Blood Health

Blood Health

A Mediaplanet Guide to Innovations in Hematology

JUNE 2022 | FUTUREOFPERSONALHEALTH.COM

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The Next Generation of Hematology The American Society of Hematology (ASH) is working with scientists, research institutions, pharmaceutical companies, and policymakers to accelerate scientific discovery, drug development, and deployment of new therapies to conquer blood diseases for people worldwide.

O

ver the last six decades, hematologists have made momentous strides in research, including the development of new, cutting-edge therapeutic technologies that have paved the way for remarkable advances in the treatment of many serious blood diseases.

CAR T-cell therapy is a prime example of a revolutionary new method of care.

The therapy involves taking a person’s immune cells from their blood, reprogramming the immune cells to target the patient’s blood cancer, @MEDIAPLANETUSA

and then returning these new “guided missiles” to the patient as a transfusion. This new treatment has shown tremendous promise among people with blood cancers such as leukemia, lymphoma, and multiple myeloma. ASH is committed to improving the safety, effectiveness, and availability of revolutionary new cancer therapies such as CAR T-cell.

In addition, ASH is working to speed research and development of other promising technologies on the horizon, including gene therapies and genome editing techniques for the treatment of individu@FUTUREOFPERSONALHEALTH

Jane N. Winter M.D., President, American Society of Hematology (ASH), Robert H. Lurie Comprehensive Cancer Center at Northwestern University’s Feinberg School of Medicine

als living with chronic blood diseases.

Building the next generation Despite these advances, there are still gaps in access to quality care for people living with blood disorders. For example, sickle cell disease (SCD) is an inherited blood disorder that affects nearly 100,000 Americans, predominantly those with African ancestry. Unfortunately, there are barriers to high-quality care for individuals living with SCD in the United States, including racial and ethnic disparities, health insurance costs and coverage

issues, and socioeconomic status inequities.

There is also another important issue: the shortage of specialists trained to diagnose and treat the disease. In studies of hematology-oncology fellows, most fellows favored careers combining hematology and oncology, with more fellows identifying oncology, rather than hematology, as their primary focus.

The current demand for hematologists in the United States is projected to exceed the existing supply, meaning that people living with SCD will not have healthcare providers with the

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specialized expertise needed to manage the condition. In recognition of this issue, ASH has made an extraordinary investment in the creation of new hematology-focused fellowship tracks at academic institutions throughout the country.

This new program will foster training of the next generation of hematologists and help to ensure that all those with blood disorders can receive the care they need and deserve.

Get involved Perhaps you or a loved one has been affected by a blood disorder, and you would like to support new research that will contribute to novel treatments for the disease. By becoming an advocate for hematology, you can help increase public awareness about blood disorders and support state and federal funding for research, which is critically needed to make this exciting science count for patients.

For those interested in learning more about blood health, the importance of funding biomedical research, and how you can get involved, please visit www.hematology. org/patients. If you would like to donate to our efforts to conquer blood diseases, please visit www.hematology.org/foundation. One hundred percent of your donation is used to support ASH’s efforts to cure blood diseases. n

INQUIRIES: US.EDITORIAL@MEDIAPLANET.COM AND US.ADVERTISE@MEDIAPLANET.COM

PLEASE RECYCLE

Publisher Shannon Ruggiero Managing Director Jordan Hernandez Lead Designer Kayla Mendez Designer Celia Hazard Lead Editor Jon Adams Copy Editor Taylor Rice Director of Content and Production Jordan Hernandez Cover Photo American Red Cross All photos are credited to Getty Images unless otherwise specified. This section was created by Mediaplanet and did not involve USA Today.

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R E A D M O R E AT F U T U R E O F P E R S O N A L H E A LT H . C O M

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*Based on IMS claims data as of 2/2022.

1L=first-line; BTKi=Bruton tyrosine kinase inhibitor; CLL=chronic lymphocytic leukemia.

Indication and Usage CALQUENCE is indicated for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

Select Safety Information Serious adverse events, including fatal events, have occurred with CALQUENCE, including serious and opportunistic infections, hemorrhage, cytopenias, second primary malignancies, and atrial fibrillation and flutter. The most common adverse reactions (≥ 30%) of any grade in patients with CLL were anemia, neutropenia, thrombocytopenia, headache, upper respiratory tract infection, and diarrhea.

Please see Brief Summary of full Prescribing Information on adjacent pages.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.FDA.gov/medwatch or call 1-800-FDA-1088.

Reference: 1. Data on File, REF-63120. AstraZeneca Pharmaceuticals LP.

INTENDED FOR US AUDIENCE ONLY. CALQUENCE is a registered trademark of the AstraZeneca group of companies. ©2022 AstraZeneca.

All rights reserved. US-63072 Last Updated 3/22

CALQUENCE® (acalabrutinib) capsules, for oral use Initial U.S. Approval: 2017 Brief Summary of Prescribing Information.

For full Prescribing Information consult official package insert.

INDICATIONS AND USAGE Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma CALQUENCE is indicated for the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

DOSAGE AND ADMINISTRATION Recommended Dosage CALQUENCE as Monotherapy For patients with CLL or SLL, the recommended dose of CALQUENCE is 100 mg taken orally approximately every 12 hours until disease progression or unacceptable toxicity.

CALQUENCE in Combination with Obinutuzumab For patients with previously untreated CLL or SLL, the recommended dose of CALQUENCE is 100 mg taken orally approximately every 12 hours until disease progression or unacceptable toxicity. Start CALQUENCE at Cycle 1 (each cycle is 28 days). Start obinutuzumab at Cycle 2 for a total of 6 cycles and refer to the obinutuzumab prescribing information for recommended dosing. Administer CALQUENCE prior to obinutuzumab when given on the same day.

Advise patients to swallow capsule whole with water. Advise patients not to open, break or chew the capsules. CALQUENCE may be taken with or without food. If a dose of CALQUENCE is missed by more than 3 hours, it should be skipped and the next dose should be taken at its regularly scheduled time.

Extra capsules of CALQUENCE should not be taken to make up for a missed dose.

Recommended Dosage for Hepatic Impairment Avoid administration of CALQUENCE in patients with severe hepatic impairment.

Dose modifications are not required for patients with mild or moderate hepatic impairment [see Use in Specific Populations (8.6) and Clinical Pharmacology (12.3) in the full Prescribing Information].

Recommended Dosage for Drug Interactions Dose Modifications for Use with CYP3A Inhibitors or Inducers These are described in Table 1 [see Drug Interactions (7) in the full Prescribing Information].

Table 1: Recommended Dose Modifications for Use with CYP3A Inhibitors or Inducers CYP3A Co-administered Recommended CALQUENCE use Drug Avoid concomitant use.

Strong CYP3A If these inhibitors will be used shortinhibitor term (such as anti-infectives for up to Inhibition seven days), interrupt CALQUENCE. Moderate CYP3A 100 mg once daily. inhibitor Avoid concomitant use.

CYP3A If these inducers cannot be avoided, Induction Strong inducer increase CALQUENCE dose to 200 mg approximately every 12 hours.

Concomitant Use with Gastric Acid Reducing Agents Proton Pump Inhibitors: Avoid concomitant use [see Drug Interactions (7) in the full Prescribing Information].

H2-Receptor Antagonists: Take CALQUENCE 2 hours before taking a H2-receptor antagonist [see Drug Interactions (7) in the full Prescribing Information].

Antacids: Separate dosing by at least 2 hours [see Drug Interactions (7) in the full Prescribing Information].

Dose Modifications for Adverse Reactions Recommended dose modifications of CALQUENCE for Grade 3 or greater adverse reactions are provided in Table 2.

Table 2: Recommended Dose Modifications for Adverse Reactions Adverse Dose Modification Event Reaction (Starting dose = 100 mg Occurrence approximately every 12 hours) Grade 3 or greater Interrupt CALQUENCE. non-hematologic Once toxicity has resolved to Grade 1 First and toxicities, or baseline level, CALQUENCE may Second Grade 3 be resumed at 100 mg approximately thrombocytopenia every 12 hours. with bleeding, Interrupt CALQUENCE. Grade 4 Once toxicity has resolved to Grade 1 thrombocytopenia Third or baseline level, CALQUENCE may or be resumed at a reduced frequency of 100 mg once daily.

Grade 4 neutropenia lasting Discontinue CALQUENCE. longer than 7 days Fourth Adverse reactions graded by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE).

Refer to the obinutuzumab prescribing information for management of obinutuzumab toxicities.

CONTRAINDICATIONS None.

WARNINGS AND PRECAUTIONS Serious and Opportunistic Infections Fatal and serious infections, including opportunistic infections, have occurred in patients with hematologic malignancies treated with CALQUENCE. Serious or Grade 3 or higher infections (bacterial, viral, or fungal) occurred in 19% of 1029 patients exposed to CALQUENCE in clinical trials, most often due to respiratory tract infections (11% of all patients, including pneumonia in 6%). These infections predominantly occurred in the absence of Grade 3 or 4 neutropenia, with neutropenic infection reported in 1.9% of all patients. Opportunistic infections in recipients of CALQUENCE have included, but are not limited to, hepatitis B virus reactivation, fungal pneumonia, Pneumocystis jiroveci pneumonia, Epstein-Barr virus reactivation, cytomegalovirus, and progressive multifocal leukoencephalopathy (PML). Consider prophylaxis in patients who are at increased risk for opportunistic infections. Monitor patients for signs and symptoms of infection and treat promptly.

Hemorrhage Fatal and serious hemorrhagic events have occurred in patients with hematologic malignancies treated with CALQUENCE. Major hemorrhage (serious or Grade 3 or higher bleeding or any central nervous system bleeding) occurred in 3.0% of patients, with fatal hemorrhage occurring in 0.1% of 1029 patients exposed to CALQUENCE in clinical trials. Bleeding events of any grade, excluding bruising and petechiae, occurred in 22% of patients.

Use of antithrombotic agents concomitantly with CALQUENCE may further increase the risk of hemorrhage. In clinical trials, major hemorrhage occurred in 2.7% of patients taking CALQUENCE without antithrombotic agents and 3.6% of patients taking CALQUENCE with antithrombotic agents. Consider the risks and benefits of antithrombotic agents when co-administered with CALQUENCE. Monitor patients for signs of bleeding.

Consider the benefit-risk of withholding CALQUENCE for 3-7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.

Cytopenias Grade 3 or 4 cytopenias, including neutropenia (23%), anemia (8%), thrombocytopenia (7%), and lymphopenia (7%), developed in patients with hematologic malignancies treated with CALQUENCE. Grade 4 neutropenia developed in 12% of patients. Monitor complete blood counts regularly during treatment. Interrupt treatment, reduce the dose, or discontinue treatment as warranted [see Dose Modifications for Adverse Reactions (2.4) in the full Prescribing Information].

Second Primary Malignancies Second primary malignancies, including skin cancers and other solid tumors, occurred in 12% of 1029 patients exposed to CALQUENCE in clinical trials. The most frequent second primary malignancy was skin cancer, reported in 6% of patients. Monitor patients for skin cancers and advise protection from sun exposure.

Atrial Fibrillation and Flutter Grade 3 atrial fibrillation or flutter occurred in 1.1% of 1029 patients treated with CALQUENCE, with all grades of atrial fibrillation or flutter reported in 4.1% of all patients. The risk may be increased in patients with cardiac risk factors, hypertension, previous arrhythmias, and acute infection. Monitor for symptoms of arrhythmia (e.g., palpitations, dizziness, syncope, dyspnea) and manage as appropriate.

ADVERSE REACTIONS The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling: • Serious and Opportunistic Infections [see Warnings and Precautions (5.1) in the full Prescribing Information] • Hemorrhage [see Warnings and Precautions (5.2) in the full Prescribing Information] • Cytopenias [see Warnings and Precautions (5.3) in the full Prescribing Information] • Second Primary Malignancies [see Warnings and Precautions (5.4) in the full Prescribing Information] • Atrial Fibrillation and Flutter [see Warnings and Precautions (5.5) in the full Prescribing Information] Clinical Trials Experience As clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.

The data in the Warnings and Precautions reflect exposure to CALQUENCE 100 mg approximately every 12 hours in 1029 patients with hematologic malignancies. Treatment includes CALQUENCE monotherapy in 820 patients in 6 trials, and CALQUENCE with obinutuzumab in 209 patients in 2 trials. Among these recipients of CALQUENCE, 88% were exposed for at least 6 months and 79% were exposed for at least one year.

In this pooled safety population, adverse reactions in ≥ 30% of 1029 patients were anemia, neutropenia, upper respiratory tract infection, thrombocytopenia, headache, diarrhea, and musculoskeletal pain.

Chronic Lymphocytic Leukemia The safety data described below reflect exposure to CALQUENCE (100 mg approximately every 12 hours, with or without obinutuzumab) in

511 patients with CLL from two randomized controlled clinical trials [see Clinical Studies (14.2) in the full Prescribing Information].

The most common adverse reactions (≥ 30%) of any grade in patients with CLL were anemia, neutropenia, thrombocytopenia, headache, upper respiratory tract infection, and diarrhea.

ELEVATE-TN The safety of CALQUENCE plus obinutuzumab (CALQUENCE+G), CALQUENCE monotherapy, and obinutuzumab plus chlorambucil (GClb) was evaluated in a randomized, multicenter, open-label, actively controlled trial in 526 patients with previously untreated CLL [see Clinical Studies (14.2) in the full Prescribing Information].

Patients randomized to the CALQUENCE+G arm were treated with CALQUENCE and obinutuzumab in combination for six cycles, then with CALQUENCE as monotherapy until disease progression or unacceptable toxicity. Patients initiated obinutuzumab on Day 1 of Cycle 2, continuing for a total of 6 cycles. Patient randomized to CALQUENCE monotherapy received CALQUENCE approximately every 12 hours until disease progression or unacceptable toxicity. The trial required age ≥ 65 years of age or 18 to < 65 years of age with a total Cumulative Illness Rating Scale (CIRS) > 6 or creatinine clearance of 30 to 69 mL/min, hepatic transaminases ≤ 3 times upper limit of normal (ULN) and total bilirubin ≤ 1.5 times ULN, and allowed patients to receive antithrombotic agents other than warfarin or equivalent vitamin K antagonists.

During randomized treatment, the median duration of exposure to CALQUENCE in the CALQUENCE+G and CALQUENCE monotherapy arms was 27.7 months (range 0.3 to 40 months), with 95% and 92% and 89% and 86% of patients with at least 6 months and 12 months of exposure, respectively. In the obinutuzumab and chlorambucil arm the median number of cycles was 6 with 84% of patients receiving at least 6 cycles of obinutuzumab, 70% of patients received at least 6 cycles of chlorambucil.

Eighty-five percent of patients in the CALQUENCE+G arm received at least 6 cycles of obinutuzumab.

In the CALQUENCE+G and CALQUENCE monotherapy arms, fatal adverse reactions that occurred in the absence of disease progression and with onset within 30 days of the last study treatment were reported in 2% for each treatment arm, most often from infection. Serious adverse reactions were reported in 39% of patients in the CALQUENCE+G arm and 32% in the CALQUENCE monotherapy arm, most often due to events of pneumonia (2.8% to 7%).

In the CALQUENCE+G arm, adverse reactions led to treatment discontinuation in 11% of patients and a dose reduction of CALQUENCE in 7% of patients. In the CALQUENCE monotherapy arm, adverse reactions led to discontinuation in 10% and dose reduction in 4% of patients.

Tables 5 and 6 presents adverse reactions and laboratory abnormalities identified in the ELEVATE-TN trial.

Table 5: Common Adverse Reactions (≥ 15% Any Grade) with CALQUENCE in Patients with CLL (ELEVATE-TN) CALQUENCE CALQUENCE Obinutuzumab Body System plus Monotherapy plus Adverse Reaction* Obinutuzumab N=179 Chlorambucil N=178 N=169 All Grade All Grade All Grade Grades ≥ 3 Grades ≥ 3 Grades ≥ 3 (%) (%) (%) (%) (%) (%) Infections 69 22‡ 65 14‡ 46 13‡ Infection† Upper respiratory 39 2.8 35 0 17 1.2 tract infectiona Lower respiratory 24 8 18 4.5 7 1.8 tract infectionb Urinary tract 15 1.7 15 2.8 5 0.6 infection § Blood and lymphatic system disorders Neutropeniac 53 37 23 13 78 50 Anemiad 52 12 53 10 54 14 Thrombocytopeniae 51 12 32 3.4 61 16 Lymphocytosisf 12 11 16 15 0.6 0.6 Nervous system disorders Headache 40 1.1 39 1.1 12 0 Dizziness 20 0 12 0 7 0 Gastrointestinal disorders Diarrhea 39 4.5 35 0.6 21 1.8 Nausea 20 0 22 0 31 0 Musculoskeletal and connective tissue disorders g Musculoskeletal pain 37 2.2 32 1.1 16 2.4 Arthralgia 22 1.1 16 0.6 4.7 1.2 General disorders and administration site conditions h Fatigue 34 2.2 23 1.1 24 1.2 Skin and subcutaneous tissue disorders i 31 0 21 0 5 0 Bruising Rashj 26 2.2 25 0.6 9 0.6 Vascular disorders 20 1.7 20 1.7 6 0 Hemorrhagek * Per NCI CTCAE version 4.03 † Includes any adverse reactions involving infection or febrile neutropenia

CALQUENCE® (acalabrutinib) capsules, for oral use ‡

Includes 3 fatal cases in the CALQUENCE plus obinutuzumab arm, 3 fatal cases in the CALQUENCE monotherapy arm and 1 fatal case in the obinutuzumab plus chlorambucil arm § Derived from adverse reaction and laboratory data a Upper respiratory tract infection, nasopharyngitis and sinusitis b Includes pneumonia, lower respiratory tract infection, bronchitis, bronchiolitis, tracheitis, and lung infection c Includes neutropenia, neutrophil count decreased, and related laboratory data d Includes anemia, red blood cell count decreased, and related laboratory data e Includes thrombocytopenia, platelet count decreased, and related laboratory data f Includes lymphocytosis, lymphocyte count increased, and related laboratory data g Includes back pain, bone pain, musculoskeletal chest pain, musculoskeletal pain, musculoskeletal discomfort, myalgia, neck pain, pain in extremity and spinal pain h Includes asthenia, fatigue, and lethargy i Includes bruise, contusion, and ecchymosis j Includes rash, dermatitis, and other related terms k Includes hemorrhage, hematoma, hemoptysis, hematuria, menorrhagia, hemarthrosis, and epistaxis

Other clinically relevant adverse reactions (all grades incidence < 15%) in recipients of CALQUENCE (CALQUENCE in combination with obinutuzumab and monotherapy) included: • Neoplasms: second primary malignancy (10%), non-melanoma skin cancer (5%) • Cardiac disorders: atrial fibrillation or flutter (3.6%), hypertension (5%) • Infection: herpesvirus infection (6%) Table 6: Select Non-Hematologic Laboratory Abnormalities (≥ 15% Any Grade), New or Worsening from Baseline in Patients Receiving CALQUENCE (ELEVATE-TN) CALQUENCE CALQUENCE Obinutuzumab plus Monotherapy plus Obinutuzumab N=179 Chlorambucil Laboratory N=178 N=169 Abnormality*,a All Grade All Grade All Grade Grades ≥ 3 Grades ≥ 3 Grades ≥ 3 (%) (%) (%) (%) (%) (%) Uric acid increase 29 29 22 22 37 37 ALT increase 30 7 20 1.1 36 6 AST increase 38 5 17 0.6 60 8 Bilirubin increase 13 0.6 15 0.6 11 0.6 *Per NCI CTCAE version 4.03 a Excludes electrolytes

Increases in creatinine 1.5 to 3 times the upper limit of normal occurred in 3.9% and 2.8% of patients in the CALQUENCE combination arm and monotherapy arm, respectively.

ASCEND The safety of CALQUENCE in patients with relapsed or refractory CLL was evaluated in a randomized, open-label study (ASCEND) [see Clinical Studies (14.2) in the full Prescribing Information]. The trial enrolled patients with relapsed or refractory CLL after at least one prior therapy and required hepatic transaminases ≤ 2 times upper limit of normal (ULN), total bilirubin ≤ 1.5 times ULN, and an estimated creatinine clearance ≥ 30 mL/min. The trial excluded patients having an absolute neutrophil count < 500/μL, platelet count < 30,000/μL, prothrombin time or activated partial thromboplastin time > 2 times ULN, significant cardiovascular disease, or a requirement for strong CYP3A inhibitors or inducers. Patients were allowed to receive antithrombotic agents other than warfarin or equivalent vitamin K antagonist.

In ASCEND, 154 patients received CALQUENCE (100 mg approximately every 12 hours until disease progression or unacceptable toxicity), 118 received idelalisib (150 mg approximately every 12 hours until disease progression or unacceptable toxicity) with up to 8 infusions of a rituximab product, and 35 received up to 6 cycles of bendamustine and a rituximab product. The median age overall was 68 years (range: 32-90); 67% were male; 92% were white; and 88% had an ECOG performance status of 0 or 1.

In the CALQUENCE arm, serious adverse reactions occurred in 29% of patients. Serious adverse reactions in > 5% of patients who received CALQUENCE included lower respiratory tract infection (6%). Fatal adverse reactions within 30 days of the last dose of CALQUENCE occurred in 2.6% of patients, including from second primary malignancies and infection.

In recipients of CALQUENCE, permanent discontinuation due to an adverse reaction occurred in 10% of patients, most frequently due to second primary malignancies followed by infection. Adverse reactions led to dosage interruptions of CALQUENCE in 34% of patients, most often due to respiratory tract infections followed by neutropenia, and dose reduction in 3.9% of patients.

Selected adverse reactions are described in Table 7 and nonhematologic laboratory abnormalities are described in Table 8. These tables reflect exposure to CALQUENCE with median duration of 15.7 months with 94% of patients on treatment for greater than 6 months and 86% of patients on treatment for greater than 12 months.

The median duration of exposure to idelalisib was 11.5 months with 72% of patients on treatment for greater than 6 months and 48% of

patients on treatment for greater than 12 months. Eighty-three percent of patients completed 6 cycles of bendamustine and rituximab product.

Table 7: Common Adverse Reactions (≥ 15% Any Grade) with CALQUENCE in Patients with CLL (ASCEND) Idelalisib plus Bendamustine CALQUENCE Rituximab plus Rituximab N=154 Product Product Body System N=118 N=35 Adverse Reaction* All Grade All Grade All Grade Grades ≥ 3 Grades ≥ 3 Grades ≥ 3 (%) (%) (%) (%) (%) (%) Infections 56 15‡ 65 28‡ 49 11 Infection† 29 1.9 26 3.4 17 2.9 Upper respiratory a tract infection 23 6 26 15 14 6 Lower respiratory tract infectionb Blood and lymphatic system disorders § Neutropeniac 48 23 79 53 80 40 47 15 45 8 57 17 Anemiad 6 41 13 54 6 Thrombocytopeniae 33 26 19 23 18 2.9 2.9 Lymphocytosisf Nervous system disorders Headache 22 0.6 6 0 0 0 Gastrointestinal disorders 18 1.3 49 25 14 0 Diarrheag Vascular disorders h 16 1.3 5 1.7 6 2.9 Hemorrhage General disorders 15 1.9 13 0.8 31 6 Fatiguei Musculoskeletal and connective tissue disorders 0 Musculoskeletal painj 15 1.3 15 1.7 2.9 *Per NCI CTCAE version 4.03 †

Includes any adverse reactions involving infection or febrile neutropenia Includes 1 fatal case in the CALQUENCE monotherapy arm and 1 fatal case in the Idelalisib plus Rituximab arm § Derived from adverse reaction and laboratory data a Upper respiratory tract infection, rhinitis and nasopharyngitis b Includes pneumonia, lower respiratory tract infection, bronchitis, bronchiolitis, tracheitis, and lung infection. c Includes neutropenia, neutrophil count decreased, and related laboratory data d Includes anemia, red blood cell decreased, and related laboratory data e Includes thrombocytopenia, platelet count decreased, and related laboratory data f Includes lymphocytosis, lymphocyte count increased and related laboratory data g Includes colitis, diarrhea, and enterocolitis h Includes hemorrhage, hematoma, hemoptysis, hematuria, menorrhagia, hemarthrosis, and epistaxis i Includes asthenia, fatigue, and lethargy j Includes back pain, musculoskeletal chest pain, musculoskeletal pain, musculoskeletal discomfort, pain in extremity, myalgia, spinal pain and bone pain ‡

Other clinically relevant adverse reactions (all grades incidence < 15%) in recipients of CALQUENCE included: • Skin and subcutaneous disorders: bruising (10%), rash (9%) • Neoplasms: second primary malignancy (12%), non-melanoma skin cancer (6%) • Musculoskeletal and connective tissue disorders: arthralgia (8%) • Cardiac disorders: atrial fibrillation or flutter (5%), hypertension (3.2%) • Infection: herpesvirus infection (4.5%) Table 8: Select Non-Hematologic Laboratory Abnormalities (≥ 10% Any Grade), New or Worsening from Baseline in Patients Receiving CALQUENCE (ASCEND) Idelalisib plus Bendamustine CALQUENCE Rituximab plus Rituximab N=154 Product Product Laboratory N=118 N=35 Abnormality a All Grade All Grade All Grade Grades ≥ 3 Grades ≥ 3 Grades ≥ 3 (%) (%) (%) (%) (%) (%) Uric acid increase 15 15 11 11 23 23 ALT increase 15 1.9 59 23 26 2.9 AST increase 13 0.6 48 13 31 2.9 Bilirubin increase 13 1.3 16 1.7 26 11 Per NCI CTCAE version 5 a Excludes electrolytes

Increases in creatinine to 1.5 to 3 times ULN occurred in 1.3% of patients who received CALQUENCE. USE IN SPECIFIC POPULATIONS Pregnancy Risk Summary Based on findings in animals, CALQUENCE may cause fetal harm and dystocia when administered to a pregnant woman. There are no available data in pregnant women to inform the drug-associated risk. In animal

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reproduction studies, administration of acalabrutinib to animals during organogenesis resulted in dystocia in rats and reduced fetal growth in rabbits at maternal exposures (AUC) 2 times exposures in patients at the recommended dose of 100 mg approximately every 12 hours (see Data).

Advise pregnant women of the potential risk to a fetus.

The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.

Data Animal Data In a combined fertility and embryo-fetal development study in female rats, acalabrutinib was administered orally at doses up to 200 mg/kg/day starting 14 days prior to mating through gestational day [GD] 17. No effects on embryo-fetal development and survival were observed. The AUC at 200 mg/kg/day in pregnant rats was approximately 9-times the AUC in patients at the recommended dose of 100 mg approximately every 12 hours. The presence of acalabrutinib and its active metabolite were confirmed in fetal rat plasma.

In an embryo-fetal development study in rabbits, pregnant animals were administered acalabrutinib orally at doses up to 200 mg/kg/day during the period of organogenesis (from GD 6-18). Administration of acalabrutinib at doses ≥ 100 mg/kg/day produced maternal toxicity and 100 mg/kg/day resulted in decreased fetal body weights and delayed skeletal ossification. The AUC at 100 mg/kg/day in pregnant rabbits was approximately 2-times the AUC in patients at 100 mg approximately every 12 hours.

In a pre- and postnatal development study in rats, acalabrutinib was administered orally to pregnant animals during organogenesis, parturition and lactation, at doses of 50, 100, and 150 mg/kg/day.

Dystocia (prolonged or difficult labor) and mortality of offspring were observed at doses ≥ 100 mg/kg/day. The AUC at 100 mg/kg/day in pregnant rats was approximately 2-times the AUC in patients at 100 mg approximately every 12 hours. Underdeveloped renal papilla was also observed in F1 generation offspring at 150 mg/kg/day with an AUC approximately 5-times the AUC in patients at 100 mg approximately every 12 hours.

Lactation Risk Summary No data are available regarding the presence of acalabrutinib or its active metabolite in human milk, its effects on the breastfed child, or on milk production. Acalabrutinib and its active metabolite were present in the milk of lactating rats. Due to the potential for adverse reactions in a breastfed child from CALQUENCE, advise lactating women not to breastfeed while taking CALQUENCE and for at least 2 weeks after the final dose.

Females and Males of Reproductive Potential Pregnancy Pregnancy testing is recommended for females of reproductive potential prior to initiating CALQUENCE therapy.

Contraception Females CALQUENCE may cause embryo-fetal harm and dystocia when administered to pregnant women [see Use in Specific Populations (8.1) in the full Prescribing Information]. Advise female patients of reproductive potential to use effective contraception during treatment with CALQUENCE and for at least 1 week following the last dose of CALQUENCE. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be informed of the potential hazard to a fetus.

Pediatric Use The safety and efficacy of CALQUENCE in pediatric patients have not been established.

Geriatric Use Of the 929 patients with CLL or MCL in clinical trials of CALQUENCE, 68% were 65 years of age or older, and 24% were 75 years of age or older. Among patients 65 years of age or older, 59% had Grade 3 or higher adverse reactions and 39% had serious adverse reactions. Among patients younger than age 65, 45% had Grade 3 or higher adverse reactions and 25% had serious adverse reactions. No clinically relevant differences in efficacy were observed between patients ≥ 65 years and younger.

Hepatic Impairment Avoid administration of CALQUENCE in patients with severe hepatic impairment. The safety of CALQUENCE has not been evaluated in patients with moderate or severe hepatic impairment [see Recommended Dosage for Hepatic Impairment (2.2) and Clinical Pharmacology (12.3) in the full Prescribing Information].

Distributed by: AstraZeneca Pharmaceuticals LP, Wilmington, DE 19850 CALQUENCE is a registered trademark of the AstraZeneca group of companies. ©AstraZeneca 2019 11/19 US-34117 11/19

Screenings Reveal if Donors Carry Sickle Cell Trait

Eugene James makes his first blood donation through the Red Cross at the Jack and Jill of America in Atlanta, GA.

Karleen James’ donation screening informed her thatshe had the sickle cell trait.

PHOTOS: AMERICAN RED CROSS

It’s often said that donated blood benefits recipients in need, but in the case of the James family, giving blood changed their lives, both the giver and the receiver.

Last December, Karleen James and her son Eugene donated blood at an American Red Cross drive in Atlanta.

The event’s goal was simple: host a blood drive that would encourage diverse blood donors to give in support of patients with sickle cell disease, with the hope of encouraging participants to become lifelong blood donors. The drive mobilized many families to come together for this goal, including the James family.

After the Jameses donated blood with the Red Cross, their blood was tested in a Red Cross immunohematology-reference lab for sickle

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cell trait. The Red Cross now provides this screening on all blood, platelet, and plasma donations from self-identified African American donors in addition to a free mini-health screening with insights on pulse, blood pressure, and hemoglobin. When Karleen and Eugene received their blood screening results, the report showed Karleen something she wasn’t expecting: she was a carrier of the sickle cell trait.

Sickle cell awareness “I knew that Eugene carried it at birth, but I never had the idea [I did] until I got the report,” says Karleen.

Along their path of education and awareness, Karleen and her son also discovered that individuals who carry the sickle cell trait are eligible to donate plasma and platelets

R E A D M O R E AT F U T U R E O F P E R S O N A L H E A LT H . C O M

to help other patients in need of lifesaving blood products.

“Giving blood with the trait is very much needed in our community, and I don’t think that it is out there enough,” says Karleen. “Had we not come into the Red Cross, we would have never realized that.”

It is estimated that about 1 in 13 Black people in the United States are born with the sickle cell trait, which means they have inherited the sickle cell gene from one of their parents. While carrying the sickle cell trait does not mean that an individual has sickle cell disease, many individuals are unaware they carry this trait, as sickle cell trait testing at birth was not widely provided until 2006.

Community support Biomedical partnership officer of the American Red Cross

Wendy Tabron shares why partnering with national and local organizations within the Black community is critical for helping people identify if they have the sickle cell trait and save the lives of patients with sickle cell.

“Partnerships with trusted community organizations help to ensure blood donation opportunities are reflective of the diverse communities the Red Cross serves,” says Tabron. “Working alongside partners to raise awareness about sickle cell disease and to encourage blood donations from individuals who are Black is critical to improving the overall health of communities. Sickle cell trait screening provides donors who are Black with vital health insight and helps the Red Cross identify compatible blood types more quickly to help patients

with sickle cell disease. With partners helping us build new relationships and providing convenient locations that bring donation and screening opportunities closer to home, we’re able to collaboratively address health disparities associated with the disease and support families.”

As soon as Karleen and Eugene became eligible to donate blood again, they were back in the donation chair — an act of charity Eugene says he’s eager to continue doing.

“I believe these donations are going to make a positive impact in the Black community and in every community,” says Eugene. “I know that I’m doing a good deed today, and that will make me smile.” n Evan Peterson, American Red Cross

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The Blood Specialists Saving Lives in the Lab

The COVID-19 pandemic has impacted every aspect of society. People dealing with serious health issues have felt its impact far more. The American Red Cross declared a blood crisis in early 2022, noting blood donations had declined 10% since March 2020. That’s especially worrisome for individuals with sickle cell disease (SCD).

Sickle cell disease affects red blood cells. It is more common among African American and Latino people. Blood transfusions are essential to helping individuals with sickle cell disease manage their illness.

“During the pandemic, nationally we saw a 50% reduction from African American donors,” notes Kimberly W. Sanford, MD, MASCP, MT(ASCP), immediate past president of The American Society for Clinical Pathology (ASCP) and medical director of transfusion medicine at Virginia Commonwealth University Health. “This depleted our blood supply for our patients with sickle cell anemia.”

The blood shortage brought to the forefront the crucial work performed by healthcare professionals who are involved in sickle cell disease: medical laboratory scientists (MLS).

“SCD patients are transfused frequently; finding com-

PHOTO: AMERICAN SOCIETY FOR CLINICAL PATHOLOGY

Patients with sickle cell disease need frequent blood transfusions. Medical Laboratory Scientists ensure their safety.

ASCP Patient Champion Malik learned about the important role that laboratory testing played in the diagnosis and treatment of his sickle cell disease.

patible blood can be difficult, and alloimmunization from transfusions are highly probable,” explains Tiffany Channer, MPH, MLS(ASCP)CM, a laboratory leader and ASCP Board Member in the New York City area. (Alloimmunization is an immune response to foreign substances from another human, most commonly occurring after blood transfusions.) Laboratories have seen the dire consequences the blood shortage has had on SCD patients.

Challenges of blood transfusion The blood needs for SCD patients are complex. Generally, patients with sickle cell

disease receive transfusions of red blood cells. In individuals with SCD, their red blood cells expire much sooner than ordinary red blood cells. The process of transfusing blood is complicated, and there is always a risk of adverse reactions in patients.

“In general, we find that patients are able to receive donated blood from any race or ethnic groups,” Dr. Sanford says. “However, owing to the presence of some unique red cell antigens that people of African heritage share, it is preferable for patients fighting sickle cell disease to receive donations from individuals who are Black; they are likely to produce better health outcomes.”

The challenge, says Channer, is that “less than 4% of the blood donor population is African American. This unfortunate circumstance creates a strain on the system and thereby impacts individuals with SCD.” Medical detectives Medical laboratory scientists play a critical role with SCD patients. They are part of the team of laboratory professionals who safeguard each unit of blood for all patients, ensuring all blood transfused is safe and compatible or approved for transfusion by the medical director prior to issuing blood.

Medical laboratory professionals don’t get the attention

they deserve — in terms of helping people dealing with blood disorders and in terms of the career and life it affords.

“Medical laboratory scientists complete all laboratory testing, which provides vital information for diagnosis and treatment,” Channer says.

Medical laboratory scientists are essential to hospital systems and healthcare organizations, from community hospitals to the Centers for Disease Control and Prevention or the National Institutes of Health.

Channer is proud to be a medical laboratory scientist and a problem solver in the healthcare profession. “Laboratory professionals save lives every day by carrying out their passion for science and enforce quality measures to ensure the public’s health is protected,” she says.

Even if they are under-recognized, medical laboratory scientists can take enormous pride in knowing that they are having a positive impact on patients with sickle cell disease and other illnesses.

“I know that I’m saving lives every single day,” notes Channer. “We’re all on a team, and it’s a well-oiled machine.

Every single specimen—we know and regard each person.” n Jeff Somers

Visit www.ascp.org/careers for more information.

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PHOTO: THE EMILY WHITEHEAD FOUNDATION

The Girl Who Lived: The Emily Whitehead Story When traditional cancer treatments failed, the Whitehead family turned to CAR T-cell for their young daughter — and found a miracle.

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mily was perfectly healthy until just after her fifth birthday,” Tom Whitehead says. “It was Memorial Day weekend in 2010. Looking back, we noticed bruising all over her. My wife said that twice when Emily was brushing her teeth, she saw blood on her gums and she had terrible knee pain.”

Emily was diagnosed with acute lymphoblastic leukemia (ALL), and initially the Whiteheads were almost relieved.

“It’s the most curable of them all,” Tom says. “There’s a 90% chance.”

Emily was treated and went into remission. The Whiteheads thought their nightmare was over, but 16 months after her diagnosis, her cancer returned.

CAR T-cell immunotherapy Doctors now recommended a bone marrow transplant, but they warned that there was only a 30% chance of survival.

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When the transplant couldn’t be arranged, things looked grim. “They told us to go home and put Emily in hospice,” Whitehead says somberly.

“I said, we’re not going to do that.”

Emily’s doctor told them that a clinical trial for CAR T-cell immunotherapy had just opened, and they signed Emily up the next day. CAR T-cell immunotherapy is a process where a cancer patient’s own immune cells are extracted from their body, genetically engineered to target cancer cells, and then put back into the patient. The T-cells target cancer cells in the blood, destroying them.

In 2012, CAR T-cell was untested and cutting-edge medicine, and Emily experienced terrible side effects.

“They ended up putting her into a coma and putting her on a ventilator,” Whitehead recalls. “That was pretty brutal. There was one night when the head of the PICU came in

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and said, ‘Tom, there’s a 1 in 1,000 chance she’s alive when the sun comes up.’”

Emily made it through the night. “She woke up on her seventh birthday, May 2012. It was eight days after she woke up that they retested her bone marrow and there was no leukemia. We did 22 months of failed standard treatments, and then 23 days later, she was cancer-free after we tried CAR T-cell therapy.”

A family mission Emily was the first pediatric cancer patient to be treated with CAR T-cell immunotherapy, and her miraculous recovery made headlines. “Ever since then, I’ve been trying to pay it forward,” Tom says.

“Families started calling us from all over the world saying

they need help. That’s when we started the Emily Whitehead Foundation.”

The foundation raises money (more than $2 million so far) to support CAR T-cell research. Aside from speaking engagements where Tom, Kari, and Emily share their story, the foundation also offers a clinical trial search engine that’s much simpler and easier to use than alternatives. They also organize a Believe Ball every year where cancer survivors, researchers, and healthcare providers can gather to meet, share stories, and celebrate life.

After appearing in documentarian Ken Burns’ 2015 film “Cancer: The Emperor of All Maladies,” Burns wrote the foreword to “Praying for Emily,” a book that Tom

explains is his attempt to bring faith and science together in the fight against cancer. The film “Of Medicine and Miracles,” focused on the work of Emily’s doctors Dr. Carl June and Dr. Steven Grupp, premiered at the Tribeca Film Festival in June.

Today, Emily is 17, still cancer free, and thriving. She’s learning to drive, looking forward to going to college, and might want to become a filmmaker. For Tom Whitehead, it really is all about paying it forward. “We really want to keep it going and do whatever we can to help this revolution move forward so there are fewer toxic treatments for patients in the future. We’re very proud of Emily for that.” n Jeff Somers

PHOTO:TC BIOPHARM

The Future of Cancer Treatments Is Here, Now

A cell therapy — very effective against a form of leukemia — points towards a wide range of future cancer treatments.

options cancer patients had, and resolved to spend my future developing medicines which were less toxic and that would help save lives.”

Acute myeloid leukemia (AML) is the most common type of leukemia in adults — about 20,000 Americans will be diagnosed with the disease this year alone. The disease is deadly and progresses rapidly if left untreated. Until recently, those treatments would be painful, uncomfortable, and potentially fatal.

“My first job as a scientist was working at the Imperial Cancer Research Fund,” says Angela Scott, founder and chief operating officer at TC BioPharm, a biopharmaceutical company developing advanced allogeneic CAR-T cell therapies for cancer. “I was disappointed with the often toxic treatment

The ‘special sauce’ Scott, who was part of the ground-breaking team that cloned ‘Dolly the sheep,’ co-founded TC BioPharm with Dr. Michael Leek in 2014.

“They came across this technology around this really unique immune cell called the gamma-delta when most cell therapies were focused on the alpha-beta,” explains Bryan Kobel, chief executive officer at TC BioPharm. “The first thing they did was build a manufacturing facility.”

Manufacturing the cell therapy in-house is the ‘secret sauce’ at TC BioPharm, according to Kobel. “Mike and Angela understood that owning your

manufacturing process was going to be core to being successful — these cells are living, breathing organisms that are changing. The best way to understand them is by knowing how to manufacture them and working with them day in and day out.”

What makes the gamma-delta cells so unique is their function as the body’s ‘bodyguards.’ They detect and hunt down an antigen emitted by all tumors called isopentenyl pyrophosphate (IPP). Healthy cells don’t emit IPP, so gamma-deltas can be used to target cancer cells very accurately.

OmnImmune TC BioPharm’s research on treatments for AML has resulted in a breakthrough with an allogeneic unmodified cell therapy consisting of activated and expanded gam-

ma-delta T-cells called OmnImmune. “OmnImmune is an exciting product,” Scott says.

“It’s one of the first cell therapies which will be available to large numbers of patients at an affordable price point.”

“OmnImmune is the first frozen-thawed gamma-delta in the clinic, meaning we can freeze it, ship it, and then store it for up to 9 months at clinical sites,” adds Kobel. “It’s a true off-the-shelf technology. Additionally, it is a donor-derived allogeneic, meaning we use healthy donors, not the patient.

The economics of the therapeutic are therefore extremely cost effective for the patient and the system.”

That makes a huge difference, because trying to use the patient’s own cells means you’re starting with a weakened system and limited in the supply you can tap. Drawing

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from healthy donors increases the potential effectiveness of the treatment without putting the patient through more physical stress.

The OmnImmune trial data was very positive. There was complete and near-complete responses starting at the lowest dose level in 28 days, and results showed no adverse side effects.

Commitment to the future While TC BioPharm’s focus in the short-term is treating AML, they have ambitious plans for the future. “Moving forward, we aim to treat other blood cancers including multiple myeloma, ALL, CLL, and various lymphomas,” Scott says.

“We are also looking into various combination therapies.”

“In the future, allogeneic cell therapies will be used to treat not just cancer but cardiovascular disease, neurological disorders, diabetes, macular degeneration, and other indications,” Scott predicts. “Over time, therapeutic use of engineered tissues and organs combined with cell therapies will extend average life expectancy to over 100 years.”

Ultimately, Kobel states that TC BioPharm will remain committed to helping the people who need it most. “We have 75 employees,” he notes, “and every day they are rowing in the same direction for an altruistic reason: they want to help patients. They want to cure patients. They want to save lives.” n Jeff Somers

Learn more about OmnImmune® and TC BioPharm’s commitment to logical, safer, and more agile clinical trials by visiting tcbiopharm.com/pipeline

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New Breakthrough in Cancer Treatments is Just the Beginning

CAR T-cell immunotherapy uses our own immune cells to fight cancer. It might eventually do even more.

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early 200,000 people will be diagnosed with a form of blood cancer this year. Most of us still think of chemotherapy and radiation when we hear the word, but CAR T-cell therapy is rapidly changing the game.

This treatment uses T-cells — a part of our immune system that normally targets viruses and other diseases.

“We take a batch of T-cells from somebody using a procedure very similar to what’s used for blood donation,” explains pathologist Michael C. Milone, M.D., Ph.D., FASCP, a pioneer in the field. “We then engineer them to express a chimeric antigen receptor (CAR) — a way of synthetically redirecting the T-cell towards a target.”

Search and destroy Once in the body, these engi-

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neered T-cells replicate very, very quickly and go on a “search and destroy” mission looking for the specific target cancer cells. “It’s pretty new,” says Laurie Adami, who was diagnosed with follicular non-Hodgkin lymphoma in 2006. “I heard about it in 2012, when I was on my fifth line of therapy. Nothing had gotten me into remission. At best, I had a stable disease.”

Unfortunately for Adami, in 2012 the therapy hadn’t gone through clinical trials for her specific disease. She had to wait six years to get into a clinical trial. Because the therapy was so new and untested, Adami experienced intense side effects. Today, it’s a different story.

“What’s happened in the last five years is we understand the safety profile,” explains Lee Greenberger, Ph.D., chief sci-

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entific officer of the Leukemia & Lymphoma Society (LLS), which has been funding CAR T-cell research since the 1990s.

“There can be significant safety issues in some patients, but they’re controllable. They don’t persist.”

A month after her treatment, Adami was shocked when her scans showed that her cancer was completely gone. She initially thought she was being deceived. “Now I’m four years out and I’m still clean,” she says. “I believe it.”

An exciting future These successes are driving further research. “The initial observations have really driven an explosion of research interest,” notes Milone. “People are looking at ways to create really smart T-cells, directing them to where they want to go through very tight regulation.”

“The efficacy is quite remarkable — it works,” stresses Greenberger. “Immunological approaches to control tumors are going to be a very, very important tool in the future.”

There are challenges to overcome, too. Currently, CAR T-cell therapies are primarily what’s known as an “autologous” therapy, meaning the engineered T-cells are created from the patient’s own cells, which are often weakened. “They won’t work very well,” explains Dr. Greenberger, “because they’re just not capable of super-activating like a healthy T-cell. So where are you going to get the T-cells? Well, it turns out, you can get them from donors.”

In what’s known as allogeneic T-cell therapy, the cells can be taken from healthy donors, engineered, and then stored so the therapy is available almost

immediately. Greenberger also speculates there may come a time when genetically engineered material could be injected directly, engineering and activating T-cells without needing to harvest them at all.

Dr. Milone sees the future impact of CAR T-cell advances more widely, noting that a single treatment could have a huge impact on poorer areas that struggle to work with “resource intense” cancer treatments, and that the therapy could be applied to different diseases.

“The potential applications of CAR T-cell approaches are quite wide ranging,” he says.

For Adami, her CAR T-cell experience wasn’t solely about survival. “All of us in those trials were really guinea pigs,” she says, “but they learned a lot. So, I have so much gratitude.” n Jeff Somers

NFL Hall of Famer Jerry Rice Overcomes Fear of Needles to Give Lifesaving Blood

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espite that fear, Rice rallied his courage and rolled up his sleeve to donate his lifesaving blood. Over time, his fear of needles dissipated as he became a blood donor helping patients in need — from accident victims to people battling cancer or sickle cell disease to mothers facing complications from childbirth.

“The first time I gave blood, I really felt proud because it was a way for me to give back off the football field, and you just never know who you may be helping,” Rice says.

That feeling inspired Rice to continue giving. Now, he is teaming up with the American Red Cross to encourage people across the country to overcome their fear of needles just like he did, so they, too, can commit to a simple act with a powerful impact — giving blood to save lives.

“I was one of those guys who was always afraid of needles. This really tough guy, but you put a needle in front of me and I’m about to run in the other direction,” Rice says. “But to be able to go ahead and withstand that to donate blood, it makes you feel good about yourself, that you have done something really positive. They have people who are experts that will hold your hand. They’ll get you through that process.”

PHOTO: AMERICAN RED CROSS

Pro football Hall of Famer Jerry Rice was never scared of big defensive players — but he was afraid of needles.

A crucial act The need for blood is constant. The Red Cross often sees a drop in blood donations around holiday weeks, like the days surrounding Memorial Day and Independence Day, so it’s crucial donors continue to make and keep appointments all season long. A single blood donation can help save more than one life, which Rice says comes down to teamwork.

“Every two seconds, someone needs blood, and blood can’t be made. It’s up to us to roll up our sleeves, go out there, and donate blood,” he says. “That’s the bot-

tom line. I’m happy to be able to have this partnership and put awareness out there.

There’s a desperate need.”

To schedule an appointment to donate blood, platelets, or plasma, download the Red Cross Blood Donor App, visit RedCrossBlood.org, or call 1-800-CROSS (1-800-733-2767).

Preparing for game day If you are unsure about what your first donation will be like, Rice encourages you to think of donating like playing sports.

Preparing to donate blood “is just like a game: you have to make the appointment, you have to eat iron [a rich meal], drink lots of water, get plenty of sleep, and make sure you show up the next day,” Rice says. “And afterward, don’t forget to post about your donation on social media to encourage others to give blood.”

A blood donation appointment typically takes about one hour from start to finish, although the actual donation only takes about 8 to 10 minutes. Upon arriving to a Red Cross blood drive or donation center, donors check in and complete a health history screening, which includes checking their temperature, blood pressure, and hemoglobin levels, as well as asking a series of questions designed to ensure they are healthy enough to donate and that their blood is as safe as possible for patients.

Donors can speed up the process by completing a RapidPass online health history questionnaire at RedCrossBlood. org/rapidpass or on the Red Cross Blood Donor app. In addition, bring your ID — such as a valid driver’s license — birth certificate, or social security card.

“Here’s your chance to make a difference,” Rice says. “You never know who you might be helping. It could be a friend or a family member who needs blood — and that’s why it’s so important.” n Victoria Goff, American Red Cross

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Understanding MPNs and What ET, PV, and MF Patients Need to Know Approximately 295,000 Americans have one of a rare group of blood cancers — polycythemia vera, essential thrombocythemia, or myelofibrosis — collectively known as myeloproliferative neoplasms (MPN), which are characterized by an increase in red blood cells, white blood cells, or platelets.

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n polycythemia vera (PV), there are too many red blood cells made in the bone marrow. Essential thrombocythemia (ET) occurs when the bone marrow produces too many platelets. Both PV and ET may put the patient at risk for heart attack, stroke, or pulmonary embolism.

People with primary myelofibrosis (PMF) have “fibrosis,” a buildup of scar tissue in the bone marrow, causing problems such as an enlarged spleen or liver.

On a mission While MPNs are chronic and there are few therapies available, there isn’t a cure. But one group — the MPN Research Foundation (MPNRF) — is on a mission to fund research toward discovering new treatments, improve patients’ quality of life, and, ultimately, cures.

The research foundation, which has been working for 21 years to fund global innovative approaches for prevention and new therapies, has awarded over $16 million for MPN blood cancer research.

“MPNs, specifically new treatment options, have

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become a hot topic. The promising news is we now have four treatments available for some MPN patients.

The hopeful news is also the several treatments in latestage development that could become available in the next 18-24 months,” says Kapila Viges, CEO of MPN Research Foundation. “Doctors are eager to learn about and gain experience with these drugs to have more options to offer and help patients through their journey.”

Symptoms and diagnosis Symptoms of MPNs, such as fatigue, bleeding or bruising, nights sweats, and itchy skin,

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often start months or years before diagnosis. However, symptoms are often overlooked since they may overlap with another diagnosis.

Diagnosis often occurs after abnormal bloodwork, such as high or low blood counts. MPNs are typically diagnosed in men and women who are over age of 50, with most being in their 60s. With proper treatment, many people with MPN live normal life spans. However, others may experience complications, including the clots and abnormal bleeding.

“In terms of prognosis, some MPN patients may progress very quickly, in a

matter of a few years,” says Richard Winneker, Ph.D., head of scientific strategies for MPNRF. “Yet many patients can go decades maintaining stable health, with the uncertainty of if or when they might get worse.

It’s this concept of MPN disease progression that MPNRF is focused on to better understand it.”

Patient voices MPNRF held an externally led, patient-focused drug development meeting with 135 in-person and web-based participants, which gave the MPN patient community an opportunity to educate Food

and Drug Administration (FDA) representatives, biopharmaceutical companies, and academic researchers about the challenges of living with an MPN. Out of that meeting, MPNRF released the “Voice of the Patient” report. For example, David A., a 64-yearold PV Patient, says his disease progression was marked a decade after diagnosis with increasing fatigue, muscle pain, and lack of concentration. One of his worst symptoms was itching.

“It was nearly body-wide pain worse than any burn I’ve ever had,” he explains, concluding, “It was so bad I was unable to fix a glass of ice water, which I thought helped, or say more than three words.”

Another PV patient, Diane R. reports, “I’m very worried about having another TIA or stroke. To quote my neurologist, ‘It’s not if but when.’”

MPNRF is committed to raising awareness among all stakeholders, including patients and providers. Their website is a comprehensive resource: www.mpnresearchfoundation.org n Kristen Castillo

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Driving groundbreaking research.

Improving lives.

The MPN Research Foundation’s mission is to help people with an MPN live a better quality of life as we work toward answers to prevention, progression and a cure for polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF) – blood cancers collectively known as myeloproliferative neoplasms (MPNs).

To learn more, visit our website mpnresearchfoundation.org

For 21 years, the MPN Research Foundation has delivered on a bold commitment to fund global pioneers studying innovative approaches to prevention, halting progression, and improving quality of life for people living with an MPN. Convening patients and caregivers, researchers and clinicians, biopharmaceutical industry leaders, and advocates around the world, we are, together, conquering MPNs. See our IMPACT@MPNRF mpnresearchfoundation.org/impact/

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Q&A: Stepping Up to the Plate for Chronic Lymphocytic Leukemia Chronic lymphocytic leukemia (CLL) is a slow-growing cancer in the blood and bone marrow. Dr. Brian Koffman, executive vice president and chief medical officer of CLL Society, shares his journey as an advocate following his CLL diagnosis.

My journey with CLL is similar to that of many others. I went to the doctor for a routine blood test that unexpectedly revealed cells in my blood were typical of CLL. The next big shock after my diagnosis was to find out my cancer was incurable and, at the time, in 2005, there were limited treatments available to even prolong my life. Furthermore, it was incredibly surprising to learn the default approach for many patients diagnosed with CLL whose disease is not aggressive was – and still is – to “watch and wait” instead of beginning treatment immediately. Unfortunately, I was one of the unlucky ones with aggressive disease.

My care team and I tried some experimental treatments. I enrolled in a Phase I clinical trial of a medicine that didn’t even have a name yet.

After seven years of disease control, I relapsed and entered another Phase I trial, which

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PHOTO: DR. BRIAN KOFFMAN

How would you describe your experience with CLL?

led to another three and a half years of disease control.

Today, I’m doing very well, thanks to the progress that’s been made in the treatment of CLL. As for my patient advocacy work – that part just happened

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naturally. My original aim was to support CLL patient education and research, but I didn’t see anybody really stepping up to the plate and being an advocate for patients the way I thought was needed, and suddenly I was an advocate.

Based on your own experience, what advice do you give to others with CLL as they are navigating a diagnosis?

The experience can be overwhelming for someone newly

diagnosed with CLL, so one of the first things I recommend is to take some deep breaths and start becoming familiar with the disease. Learning about the disease is an iterative process, so I encourage others to start by learning the vocabulary, acronyms, and other key terms. It’s good to frontload knowledge on the disease because, as we say at CLL Society, smart patients get smart care. Join a CLL support group. I would also recommend working with someone who specializes in CLL – a true CLL expert – to make care decisions.

We’re inclined to believe that all physicians are experts by nature, but CLL is a rare cancer and some may not be wellversed in the latest developments in therapy.

The treatment paradigm has quickly shifted in such a radical and positive way, it’s important that doctors understand the latest developments.

For example, it’s important to use tools such as prognostic markers or appropriate predictive testing, which can have a big impact on selecting the

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PHOTO: DR. BRIAN KOFFMAN

best therapeutic strategy for an individual patient, in the management of CLL. To help ensure patients are getting advice from the appropriate subject-matter expert, at CLL Society we encourage all patients to seek a second opinion with a specialist, whether that be consulting a physician through CLL Society’s free Expert Access program available via our website or through their own research. Results from our program indicate 80% of patients made a change in their treatment plan after consulting an expert virtually.

What role should selfadvocacy have in the blood cancer treatment process?

No one has more skin in the game than you have as a patient. I think it’s good for a patient to receive support from others to help with researching different therapeutic options and staying up to date, but it’s important for those with blood cancer to make their preferences known. Asking yourself questions about what you want out of treatment is an essential part of the decision-making process. People who have cancer need to engage with their healthcare teams and advocate for themselves to avoid letting other people, however well-intended, make assumptions about their preferences.

What do you see as the biggest barriers keeping every person with CLL/ blood cancer from receiving the highest quality of available care?

The low-hanging fruit in managing CLL is ensuring

that all patients – no matter what type of insurance they have, no matter whether they live in a city or a rural setting – have access to the latest best possible care. That is not happening for many reasons.

For example, community hematologists and oncologists are extremely busy caring for many cancer patients with common cancers and may not have time to stay up to date on the latest CLL treatments. To address this issue, CLL Society developed online resources including our “Test Before Treat™” hub and our many fact sheets on CLL and COVID-19 available for free on our website. These education materials and resources are designed so that patients can bring them with them into the doctor’s office. They can help guide patients in conversations with physicians and have appropriate references so the doctors know the information is from a trusted source.

What are the remaining needs for CLL patients?

We need more therapeutic options for patients who relapse while on existing therapies. When patients’ disease returns while taking the current prominent forms of therapy for CLL, and there are growing numbers of these patients, they have limited additional treatment options, many of which are still experimental. So those whom I call “double refractory patients” – patients whose first and second or later lines of treatments were ineffective or stopped being effective – need help and are searching for clinical trials or searching for novel combinations.

Another major barrier those with blood cancer face is our status as immunocompromised individuals. Before COVID-19, most people like myself saw their CLL as an annoyance we had to deal with. We may have had to

take pills every day, but we could still participate in activities we enjoy doing – travel, skiing, eating at restaurants, doing whatever we wanted.

But during the pandemic, it became glaringly evident to us that being immunocompromised was going to prevent us from doing those things again. The CLL treatments that we have do a great job of reducing our disease burden and knocking our CLL down, giving us longer lives, but they don’t seem to allow us to reconstitute our immune system. We’re highly susceptible to COVID-19 and other infections like pneumonia, which is still a leading cause of death in CLL patients. So I see ways to reconstitute our immune system as a major need.

Is there anything else you’d like to share with the larger CLL community?

I’ve been saying this to industry and researchers since I

entered my first clinical trial: I’m extraordinarily grateful for the way that the scientific community and the healthcare industry have cracked the biology of CLL and found ways to target cancer cells leading to improved outcomes for patients like myself. But your job is not done.

To use a baseball analogy: Don’t leave me on third base.

Get me home! Find a cure!

Don’t give up when the work isn’t done. We have great molecules, we have great combinations, but we don’t have curative therapies.

My biggest hope is that we can find a curative path for CLL in my lifetime. I’m lucky to live with a manageable case, but many are not as fortunate. So I encourage continued partnership across the cancer care continuum to help continue moving care forward.

Learn more about navigating a CLL diagnosis and exploring treatment options at LivingwithCLL.com and CLLSociety.org.

About Genentech in Hematology For more than 20 years, Genentech has been developing medicines with the goal to redefine treatment in hematology. Today, we’re investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. n

For more information, visit www.gene.com/ hematology

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How the Leukemia & Lymphoma Society Is Transforming Treatment and Care for Kids With Blood Cancer “Your child has cancer” are words parents should never have to hear. In reality, the lives of children and their families are turned upside down every day by the unthinkable.

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or many pediatric cancer patients, their diagnosis is a type of blood cancer, leukemia — the most common type of childhood cancer and the second-leading cause of cancer deaths among children.

While major advances have been made in treating children with cancer, particularly those with certain types of blood cancer, there is still much more to be done to help our youngest and one of our most vulnerable patient populations not only survive their diagnosis but thrive in their lives after treatment. Even when treatments are effective, 80% of childhood cancer survivors have a chronic health condition and 42% have a severe, disabling, or life-threatening condition 30 years after diagnosis.

The Leukemia & Lymphoma Society (LLS), the largest nonprofit dedicated to creating a world without blood cancer, is working to make this notion possible through

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the Dare to Dream Project, the next phase of The LLS Children’s Initiative. Over the next five years, LLS is investing $175M to transform treatment and care for children with blood cancer through advocacy, patient support, and ground-breaking research into new, safer therapies.

A world without blood cancer At the heart of the Dare to Dream Project is LLS PedAL, the first integrated, global, acute myeloid leukemia (AML) master clinical trial to test new, safer therapies on children, matching a specific treatment to a specific cancer in a specific child.

The need to pursue new, targeted treatments for children

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is urgent. While CAR T-cell immunotherapy, which LLS has funded since the early days of this therapeutic approach, provides a treatment option for children with highly treatment-resistant acute lymphoblastic leukemia (ALL), some pediatric patients stop responding to treatments. For children with AML, about 40% will relapse after their initial treatment and only 69% survive more than five years.

“Different treatment approaches are needed for children because their bodies work in different ways than adults’ do and undergo changes as they grow,” says Gwen Nichols, M.D., chief medical officer at LLS and co-chair of PedAL Master Clinical Trial. “PedAL has the

potential to advance progress for these children and stand as a model for future pediatric cancer trials. By bringing together worldwide leaders, we can elevate the findings from this study to offer newer, safer standards of care.”

The goal of the PedAL Master Clinical Trial is to replace one-size-fits-all chemotherapy with treatments tailored to each child’s unique tumor biology. Through partnerships with multiple organizations and institutes based in the United States and internationally, PedAL has study sites across the world, bringing the dream of safer, more effective pediatric leukemia treatments closer to home for more families.

In addition to PedAL, LLS is

funding over 32 pediatric-specific grants, researching longterm side effects of treatment and improving detection of relapse as part of the Dare to Dream Project.

LLS is also expanding its support for pediatric patients and their families, including educational resources, financial assistance, and oneon-one consultation with pediatric oncology specialists who can be reached by calling (800) 955-4572.

To learn more about the Leukemia & Lymphoma Society, the Dare to Dream Project, and LLS’s support services and resources, please visit lls.org/ daretodream. n The Leukemia & Lymphoma Society

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Rock and Roll Up Your Sleeve Like Elvis Presley The extent of Elvis Presley’s good deeds come to light decades later.

At age 24, Elvis Presley makes his first blood donation to the Red Cross

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I

f you go walking in Memphis, Tenn., you’ll most certainly see the shining lights of Beale Street, hear a chorus of local blues music floating through the air, and smell the molasses-infused BBQ sauce that has come to define Memphis-style BBQ. And if you go talking to people in Memphis, one thing you are guaranteed to discuss is Elvis Presley.

It’s been five decades since Presley’s passing, but Memphians (and the world, for that matter) still remember him fondly. Moreover, those who personally encountered Presley still recount many private acts of kindness.

Today, we are still unraveling and learning the extent of Presley’s philanthropy. He often gave without fanfare and out of the public eye.

You can still hear stories of Presley handing out teddy bears to children’s hospitals, performing free concerts, paying off mortgages, and even purchasing Cadillacs for strangers in need of cars. But he didn’t stop there — Elvis even donated his own blood to help patients in need.

A commitment to help The year was 1959 and Presley was on an intense international high following a string of popular songs.

Despite being on top of the world,

PHOTO S: AMERIC AN RED CROSS

Elvis Presley confirms his blood donor information prior to giving blood

Presley was silently suffering.

His beloved mother, Gladys, had just passed away. Presley now found himself deployed with the U.S. Army in Germany, an ocean away and separated from his family. Despite his personal turmoil, Presley, alongside his fellow soldiers, decided to donate blood on base in Friedburg, Germany. At the young age of 24, he rolled up his uniform’s sleeve and gave the lifesaving gift of blood.

Presley’s commitment to helping save patients in need through blood donations did not just stay in Germany. After

his return and transition to civilian life, his charitable giving continued. He continued to be a supporter of the American Red Cross and blood donations.

To this day, Presley’s blood donor card can still be found at his former home of Graceland in Memphis. Additionally, in 1961, Presley started a tradition of annually sending checks to more than 50 charities, including the Red Cross.

The legacy of giving Today, the American Red Cross is asking the American public to “Rock ‘N Roll Up Your

Sleeve” and give the gift of life through a blood donation. The American Red Cross supplies 40% of the nation’s blood, and the need for blood is constant.

Every two seconds, someone in the United States needs blood. Unlike other medical treatments, blood cannot be manufactured in a lab and can only come from the generosity of volunteer blood donors.

Although Elvis Presley left us many years ago, the legacy of his giving lives on.

Even with an artist as prolific as Presley, we are still learning new charitable acts that

he committed. Although he never knew the patients who received his lifesaving blood, he committed to a routine of scheduled giving and knew that those donations made a difference.

As a thank-you for helping, in honor of the new Baz Luhrmann film, “Elvis,” all who come to give blood in the month of June will be automatically entered for a chance to win a VIP trip to Graceland for two, including round-trip airfare to Memphis; a threenight stay at The Guest House and Elvis Entourage VIP

tour, courtesy of Graceland; a custom-wrapped Gibson Epiphone guitar; and more.

Additionally, those who come to donate June 1-30 will also receive a $5 e-gift card to a merchant of choice. Terms apply.

Visit RedCrossBlood.org/ElvisMovie for details.

To schedule an appointment to donate blood, platelets, or plasma, download the Red Cross Blood Donor App, visit RedCrossBlood.org, or call 1-800-CROSS (1-800-733-2767). n Emily Osment, American Red Cross

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SAVING LIVES. It’s what we do. It’s who we are.

Marc, donor and Zeke, recipient

The National Marrow Donor Program® (NMDP)/Be The Match® is a global leader working to save lives through cellular therapy. For people with life-threatening blood cancers--like leukemia and lymphoma--or other blood disorders like sickle cell, a cure exists. With more than 30 years experience managing the most diverse registry of potential unrelated blood stem cell donors and cord blood units in the world, Be The Match is a proven partner in providing cures and driving equal outcomes for all.

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