Pediatric Hematology-Oncology and Bone Marrow Transplant by Michigan Medicine

PEDIATRIC HEMATOLOGY-ONCOLOGY AND BONE MARROW TRANSPLANT

New Answers, Greater Hope

Advancing

Discovery and Possibilities for Children and Young Adults with Cancer and Blood Disorders

Shaping the Future of Care for Pediatric Cancer and Blood Disorders

The Pediatric Hematology-Oncology and Bone Marrow Transplant team at University of Michigan Health

C.S. Mott Children’s Hospital wants children, adolescents and young adults with cancer and blood disorders to know one thing: you are not alone.

Our team of expert physicians, nurses, nurse practitioners, fellows, pharmacists, psychologists, child and family life specialists, social workers and dietitians work together to make kids, young adults and families feel supported and empowered during every step of their care journey. With a multidisciplinary approach that combines leading-edge therapies with innovative science, we are driving progress in treating even the most complex cancers and blood disorders.

• Leading the way in cancer care and blood disorders

MULTIDISCIPLINARY TEAM OF PROVIDERS

ENSURES PATIENTS RECEIVE COMPREHENSIVE, EXPERT CARE

Families come to Mott from all over the world, many with referrals from other oncology and hematology programs, or to participate in our clinical trials. Our team has experience in caring for all childhood cancers and blood disorders, including the most complex ones and those that haven’t responded to prior therapies. And as part of University of Michigan Health, we partner with experts in virtually every medical specialty.

• Working together to drive discovery and hope

Our teams of pediatric oncologists, hematologists, transplant physicians, surgeons, radiation oncologists, pathologists, radiologists, nuclear medicine physicians and nurse practitioners specialize in childhood cancer and blood disorders. Mott is also home to researchers who study the conditions our physicians treat every day. Collaboration between Mott physicians and scientists is accelerating advances that are informing care and benefiting patients.

Cancers We Treat:

• Leukemia

• Lymphoma

• Brain tumors

• Neuroblastoma

• Bone sarcomas

• Eye cancers (Retinoblastoma)

• Germ cell tumors

• Hepatoblastoma and other rare liver cancers

• Melanoma

• Rhabdomyosarcoma

• Wilms tumor

• Relapsed and resistant pediatric cancers

Blood Disorders We Treat:

• Hemophilia

• Bleeding disorders

• Thrombosis

• Bone Marrow Failure syndromes

• Immunodeficiencies

• Lymphoproliferative diseases

• Neutrophil disorders

• Sickle Cell disease

• Providing comfort and support

When a child or young adult is diagnosed with cancer or a blood disorder, the road ahead can seem overwhelming. We offer a level of support that is not always available in other centers. We provide families with the most current cancer information, as well as social work services, access to financial counselors and programs that support survivors. We also offer a hospital school program, spiritual care and a wide array of other options catered to the needs of our patients.

• Training tomorrow’s leaders

Our team has made great progress in advancing research, clinical trials and care for cancer and blood disorders. To build on this momentum and bring cures to new generations of children and young adults, we are committed to teaching, mentoring and preparing U-M medical students, residents and fellows to become the next influential leaders in the field.

“We see the children and families who come to us at Mott as people, not just patients. These are kids, parents, siblings, and loved ones who are likely overwhelmed or frightened from a cancer or blood disorder diagnosis. Our teams are here to provide them answers and share knowledge and expertise. We can ensure each and every family that their child will receive state-of-the-art, personalized, and comprehensive care from the most dedicated and experienced leaders in the field.”

Rajen J. Mody, MBBS, M.S.

Ruth Heyn Professor of Pediatric Oncology

BY THE NUMBERS

OVER 10,000 PATIENT VISITS EACH YEAR

OVER 500 CHEMOTHERAPY AND IMMUNOTHERAPY PATIENTS EACH YEAR

1,100 PROCEDURES PERFORMED ANNUALLY

THREE TEAMS OF ONCOLOGISTS, HEMATOLOGISTS AND TRANSPLANT PHYSICIANS, TREATING 27–32 ADMITTED PATIENTS EVERY DAY ON A DEDICATED CANCER FLOOR

OUR CORE TEAM: 30 FACULTY MEMBERS 15 NURSE PRACTITIONERS 10 FELLOWS

OUR TEAM SAW PATIENTS COMING FROM 40 STATES OVER THE PAST FIVE YEARS

Brain Tumor Research and Care

Led by the collaborative efforts of Mott, Rogel Cancer Center and more than 1,500 dedicated donors, the Chad Carr Pediatric Brain Tumor Center was established at Michigan Medicine in 2018 to pool the diverse ideas and knowledge that can solve childhood brain cancers.

The center brings together unmatched expertise from across U-M Health departments of pediatrics, radiology, neurosurgery, radiation oncology, genetics and pathology and U-M schools of engineering and public health — as well as renowned international collaborators.

As a national center of excellence for pediatric brain cancer, the Chad Carr Center has expanded Michigan Medicine’s clinical and research capacity, delivering great promise in the search for cures. We’re already on the cusp of breakthroughs. We are doing pivotal research on diffuse intrinsic pontine glioma (DIPG) and high-grade gliomas, closing gaps in understanding their causes and mechanisms. We are also working to develop innovative treatments for DIPG and other pediatric brain tumors.

Types of brain tumors we treat:

• Low-grade glioma (grade 1, 2)

• High-grade glioma (grade 3, 4)

• Optic pathway glioma

Our Inspiration

The Chad Carr Pediatric Brain Tumor Center honors the legacy of Chad Carr, the grandson of former U-M football coach Lloyd Carr. Chad was diagnosed with DIPG three days before his fourth birthday. He endured 30 rounds of chemotherapy and a clinical trial before dying of the inoperable brain tumor at the age of 5. His memory drives our progress and efforts to advance the understanding of pediatric brain tumors, improve therapies and move us closer to finding much-needed cures.

Children’s Brain Tumor Network (CBTN)

The largest pediatric brain tumor biorepository in the world, CBTN has more than 40 partner foundations and 30 member institutions. U-M sequencing data and pediatric brain tumor autopsies are used by CBTN researchers.

• Diffuse midline glioma, including DIPG

• Atypical teratoid/rhabdoid tumor (ATRT)

• Choroid plexus tumors

• Craniopharyngioma

• Desmoplastic infantile astrocytoma

• Cysts

• Ependymoma

• Germ cell tumors

• Medulloblastoma

• Neurofibromatosis

• Oligodendroglioma

Pacific Pediatric Neuro-Oncology Consortium (PNOC)

PNOC has researchers in Australia, Canada, Europe and the U.S. Teams at PNOC hospitals are made up of renowned brain tumor specialists, nurses, social workers, psychologists and staff whose expertise enhances care for each patient. The Chad Carr Center is the only PNOC institution in Michigan.

Collaborative Network for Neuro-oncology Clinical Trials (CONNECT)

U-M recently joined CONNECT, an international collaborative network of pediatric cancer centers with the objective to improve outcomes for children with newly-diagnosed, high-risk brain tumors.

Collaborating Departments at U-M:

• Biological Chemistry

• Computational Medicine and Bioinformatics

• Internal Medicine

• Michigan Center for Translational Pathology

• Molecular & Integrative Physiology

• Neurosurgery

• Pathology

• Pediatrics

• Pediatric Surgery

• Radiation Oncology

DRIVING DISCOVERY THROUGH COLLABORATION

A highly collaborative environment with experts from diverse specialties, the Chad Carr Center has already made promising discoveries that are affecting our understanding and treatment of high-risk pediatric brain tumors. The center is inspiring innovative research, promising clinical trials and breakthroughs by spurring institutional, national and international partnerships.

U-M Brain Tumor Precision Medicine Conference

The Chad Carr Center’s Brain Tumor Precision Medicine Conference maps out personalized treatment plans for kids with solid tumors and provides coordinated, seamless care from diagnosis to cure. The conference is held monthly to identify precision medicine-based therapies for pediatric brain tumor patients using information from U-M Health research and clinical tests. We also welcome physicians, scientists and caregivers from other institutions so they can grow their knowledge and share insights.

See how an outside-the-box treatment is helping a teen with aggressive brain cancer.

WATCH VIDEO: michmed.org/sam

>165 PAPERS PUBLISHED

FOCUSED ON PEDIATRIC BRAIN TUMORS INCLUDING DIFFUSE MIDLINE GLIOMAS, DIPG, PEDIATRIC GLIOMAS, EPENDYMOMA, MEDULLOBLASTOMA AND ATYPICAL TERATOID/RHABDOID TUMORS

CHAD CARR CENTER PILOT GRANTS HAVE LED TO MORE THAN FIVE TIMES THE AMOUNT AWARDED IN ADDITIONAL FUNDING

$31 MILLION IN FUNDING

SINCE CHAD CARR CENTER INCEPTION 70% OF FUNDING COMES FROM NATIONAL INSTITUTES OF HEALTH (NIH) AND DEPARTMENT OF DEFENSE (DOD)

CHAD CARR CENTER RESEARCHERS

1 OF ONLY 5

CHILDREN’S HOSPITALS IN THE U.S. ABLE TO CONDUCT SEQUENCING IN-HOUSE

Precision Oncology: Personalized Approaches to Care

NATIONAL LEADERS

IN PERSONALIZED PEDIATRIC AND YOUNG ADULT CANCER CARE

ESTABLISHED IN 2011, OUR GENE SEQUENCING PROGRAM

IS ONE OF THE NATION’S LARGEST AND MOST SOPHISTICATED

At U-M Health, we have the resources and expertise to personalize care for each patient. And we are doing more than just treating a disease. We are delivering cancer screening and prevention tips based on individual cancer risks, providing the most successful combination of therapies with the least harmful side effects, predicting whether cancer will return and developing a proactive plan if it does.

Precision Oncology Program

Precision oncology enables doctors to create treatment plans for each patient based on that individual and their tumor’s genetic makeup. Gene sequencing, the process that analyzes a patient’s DNA and RNA to identify key genetic changes, tells physicians why a cancer occurred, how it is functioning and what the most effective and least harmful treatment will be for the patient. Sequencing can be especially beneficial for patients with relapsed and resistant cancers.

Creating a Global Standard of Care

OUR TRANSLATIONAL STUDIES INCLUDE OUR CANCER GENOMICS TRIAL, Peds-MiOncoSeq, AND A LIQUID BIOPSY STUDY THAT MONITORS CANCER VIA NON-INVASIVE METHODS

In 2015, a U-M team of researchers led by Rajen Mody, MBBS, M.S., and Arul Chinnaiyan, M.D., Ph.D., director of the Michigan Center for Translational Pathology, was the first to publish findings on the use of real-time sequencing approaches to treat relapsed or refractory pediatric cancers. The study uncovered new information from 60% of patients that led to new treatment approaches, changes in diagnosis and recommendations for cancer screening family members. Most of these patients had no known treatment available before the study. The treatment changes recommended by the study helped several patients achieve longlasting remission for leukemia, solid tumors and brain tumors, among other cancer types. This approach has now become the standard for analyzing pediatric tumors.

WATCH VIDEO: michmed.org/sequencing

THE BENEFITS OF DNA SEQUENCING IN CHILDHOOD CANCER

IN 90% OF PATIENTS, we learned new information regarding tumor drivers which could be the basis for future drug discovery.

IN 60% OF CASES, we found new actionable information which was useful for the patient’s clinical management.

IN 35% OF PATIENTS, new information yielded from tumor sequencing led to specific recommendations regarding a drug and/or targeted therapy.

The Michigan Oncology Sequencing Program

The foundation of our precision medicine efforts is the Michigan Oncology Sequencing Program (Mi-ONCOSEQ).

Launched in 2010, this program sequences the DNA and RNA of metastatic cancers and normal tissue to identify alterations that could help drive treatment. More than 10,000 adult and pediatric patients with a variety of cancer types have had their tumors sequenced through the program. Mi-ONCOSEQ brings together U-M experts in

clinical oncology, clinical genetics, genomic science and bioinformatics, clinical pathology, social and behavioral sciences, and bioethics.

The information we learn from Mi-ONCOSEQ not only confirms or clarifies the type and characteristics of cancers, but can reveal information that creates options and direction for treatment in real time.

Decoding Cancer: A Personalized Approach

Targets Genetics

When Melia arrived at Mott, the 3-year-old had endured chemotherapy, radiation, and the amputation of her left forearm. But none of those treatments halted her soft tissue cancer.

Melia joined a U-M precision medicine program that studies and treats pediatric patients who have rare forms of cancer.

The U-M team sequenced, analyzed, and identified the genetic mutation driving Melia’s cancer — and there was a clinical trail that targeted it.

She stopped chemotherapy and instead swallowed two pills each day. After three months, a scan showed Melia’s cancer had receded and was stable.

Neuroblastoma Care

Neuroblastoma is a disease in which malignant cancer cells form in nerve tissue called “ganglions” or in cells in the adrenal glands, which are located above each kidney. Other common neuroblastoma sites include the chest or nerve tissue near the spine or spinal cord.

Treatment plans for Mott patients with neuroblastoma are individualized and our team meets weekly to determine which approaches are working best for each patient. Unfortunately, 70% of neuroblastomas have already reached the bones or bone marrow by the time they are diagnosed. Once it spreads here, it is termed stage 4 with cure rates of only 40-50% for children over 18 months.

Mott’s internationally recognized Pediatric Oncology Program is a longstanding leader in treating pediatric neuroblastoma. We are also home to an active research program that has pushed groundbreaking discoveries and insights into neuroblastoma pathogenesis that have advanced treatments for high-risk neuroblastoma and patients who have failed primary therapy.

INCREASING NEUROBLASTOMA SURVIVAL RATES

In 2016, Mott physicians led a groundbreaking Children’s Oncology Group immunotherapy clinical trial of 190 centers that showed unparalleled outcomes in the treatment of relapsed or resistant neuroblastoma. This trial found that anti-GD2 therapy in combination with chemotherapy can lead to four times more successful outcomes. Thanks to this discovery, the survival rate for pediatric neuroblastoma is now 69%, a significant climb from the 19% survival rate for the disease in 1997.

Highly Specialized Neuroblastoma Therapy Delivers Hopeful Results

To see 5-year-old Dakota zipping around in her little motorized Jeep, with her pretend cell phone and sparkly shoes today, you wouldn’t believe what she has already been through in her young life.

Dakota spent close to 200 days at Mott to treat stagefour, high-risk neuroblastoma.

Because of her diagnosis, Dakota was eligible for a study at Mott, which included treatment with highdose MIBG, an experimental therapy developed at U-M more than 40 years ago and typically reserved for cancer relapses, not frontline use.

Doctors Unleash New Weapon to Fight Pediatric Neuroblastoma

At 3 years old, Emil underwent chemotherapy to fight the tangerine sized cancerous tumor in his abdomen. His tumor proved very resistant and he had a lot of treatment side effects.

Mott physicians took a new approach, combining immunotherapy and chemotherapy in an innovative way as part of an international clinical trial.

After 17 rounds of the combination treatment, Emil’s tumor shrunk considerably, but he continues to receive treatment. Of the children in the clinical trial, 53% saw either complete or partial remission — a significant jump over the typical 10–12% response rate.

Immunotherapy

The immune system is one of the most important cogs in fighting cancer. With exciting new technologies, we can take advantage of a person’s immune system to help kill cancer cells and develop antibodies against antigens present on cancer cells and use them as targets.

Immunotherapy has shown tremendous promise in treatment of many childhood cancers — including leukemia, lymphoma, and neuroblastoma. Newer antibodies are also continuously being developed against other cancers. Immune therapies have a different side effect profile than traditional cancer treatments. They can also work on their own or in combination with conventional chemotherapy, providing additional options to patients who have failed to respond to chemotherapy alone.

In addition to antibodies, immunotherapy is being used as the basis to develop cancer vaccines, which are in the early stages of development. Using commonly prevalent cancer antigens, researchers are developing vaccines to treat cancers and/or prevent cancer recurrence. Immunotherapy approaches give us hope that we will not only be able to provide more effective cancer treatments in hard-to-treat or relapsed cancers but also prevent complications that could affect patients for the rest of their lives.

MIBG Therapy

Radiolabeled MetaIodoBenzylGuanidine (MIBG) combines radiation particles with a drug that specifically targets neuroblastoma cells in a patient’s body. This therapy is unique, in that the radiation pinpoints just the tumor, sparing normal tissue from radiation side effects. And unlike other types of radiation therapy, MIBG is given as an intravenous (IV) infusion to a patient. In low doses, MIBG may also be used as a scan to identify where the neuroblastoma is located. In higher doses, MIBG can treat patients with tumors that have either failed to respond or relapsed after prior chemotherapy.

Radiolabeled MIBG was developed in 1979 by U-M scientists. Within 10 years of development, centers around the world began using MIBG to scan or treat patients with neuroblastoma. MIBG has been used to treat several thousand children and young adults worldwide, with over 150 treated at the U-M alone. Fewer than 20 U.S. hospitals have the capability to treat patients with MIBG. We are proud to be one of those centers and even prouder to be the center that developed this therapy for children around the world.

MIBG THERAPY WAS DEVELOPED AT U-M OVER 40 YEARS AGO

1 OF 18

PEDIATRIC CENTERS IN NORTH AMERICA WITH THE ABILITY TO DELIVER MIBG THERAPY TO PATIENTS WITH ADVANCED NEUROBLASTOMA

MOTT PHYSICIANS ARE INTERNATIONAL EXPERTS

IN USING IMMUNOTHERAPY AND THE NOVEL COMBINATION OF CHEMOTHERAPY AND IMMUNOTHERAPY TREATMENTS IN MANAGING HIGH-RISK NEUROBLASTOMA

New Approaches to Neuroblastoma Therapy (NANT)

We are one of 16 centers across the country that make up the NANT consortium, a group of universities and children’s hospitals with research and treatment programs specifically for neuroblastoma.

Leukemia and Lymphoma Program

Groundbreaking Leukemia Treatment “a Whole New Approach” to Fighting Cancer. READ MORE: michmed.org/cartcell

U-M IS THE FIRST SITE IN MICHIGAN TO OFFER FDA-APPROVED CAR T-CELL THERAPIES FOR LEUKEMIA AND NON-HODGKIN LYMPHOMA

U-M PARTICIPATED IN THE PIVOTAL TRIALS THAT LED TO THE FIRST FDA-APPROVED CAR T-CELL THERAPY IN 2017

MOTT HAS THE LARGEST, MOST EXPERIENCED

PEDIATRIC BONE MARROW TRANSPLANT AND CELLULAR THERAPY PROGRAM IN MICHIGAN

MOTT PHYSICIANS SPECIALIZE IN RELAPSED AND RESISTANT LEUKEMIA

OFFERING ACCESS TO INNOVATIVE TREATMENT PROTOCOLS AND PHASE 1 CLINICAL TRIALS

MOTT WAS ONE OF THE FIRST TRANSPLANT CENTERS TO RECEIVE ACCREDITATION BY THE FOUNDATION FOR THE ACCREDITATION OF CELLULAR THERAPY (FACT) IN 2001

SINCE OUR PEDIATRIC BONE MARROW TRANSPLANT PROGRAM BEGAN IN 1989, WE HAVE PERFORMED OVER 1,200 TRANSPLANTS

Leukemia occurs when white blood cells in the bone marrow grow out of control. While it is the most common childhood cancer and usually is curable, our understanding of the subtle differences between each patient’s leukemia continues to grow and guide individualized treatments.

Lymphoma is a cancer that is composed of lymphocytes, a cell subtype that plays an important role in our immune system. The third most common childhood cancer, lymphoma most frequently appears in the second decade of life, although it can occur at any time. Our group remains at the forefront of lymphoma treatment, including expanding use of immunotherapy to treat many types of pediatric lymphoma.

Our leukemia and lymphoma team is engaged in multicenter clinical trials that bring the newest therapies to our patients and help advance our knowledge. Our expertise across medical oncology, cellular therapy and bone marrow transplant ensures that our patients have immediate access to cutting-edge care.

CELLULAR THERAPY

Chimeric antigen receptor T-cell therapy, or CAR T-cell therapy, reprograms a patient’s immune system to target specific cancer cells. CAR T-cell therapy and other cell therapies are quickly revolutionizing treatment for a wide variety of cancers, including relapsed or resistant leukemia and lymphomas.

Our CAR T-cell therapy program began in 2014, serving as the first center in the state to treat patients with relapsed leukemia. We were one of only 15 North American sites involved in the FDA licensing trial using CAR T-cells to treat acute lymphocytic leukemia (ALL) in children, and have continued to remain in the forefront of this technology. We also continue to study CAR T-cell trials in a wide variety of other blood cancers.

Historically, leukemia treatment has heavily relied on the use of chemotherapy, often causing many negative side effects on a patient’s body. One of the unique aspects of CAR T-cell therapy is its ability to target cancer cells while sparing normal, healthy tissue. CAR T-cell therapy is currently used at Mott to treat children with B-cell leukemia, specifically those whose leukemia has relapsed or failed standard chemotherapy. In the future, we envision that CAR T-cell therapy may replace chemotherapy altogether and change cancer treatments for generations to come.

Bone Marrow Transplant

With a track record in innovative research and leadingedge clinical care, our Pediatric Bone Marrow Transplant (BMT) Program is the largest pediatric bone marrow transplant program in the state. We perform 50-60 transplants annually for a wide variety of conditions, including leukemias, lymphomas, brain tumors, and neuroblastoma, as well as disorders of the immune system and other rare blood disorders. We are actively participating in transplant trials for sickle cell disease, aplastic anemia, and both inherited and acquired immune system disorders.

Our faculty have led multiple national clinical trials and are recognized for their leadership roles in both research and clinical transplant care. Our team has focused on lessening the toxicities of transplant, including the prevention and treatment of graft versus host disease (GVHD). GVHD can have tremendous impacts on a transplant patient’s quality of life and overall survival, and we have made tremendous strides in managing GVHD in children and young adults. Our mission is to not only provide state-of-the-art care for the patients of today, but to identify better options for the patients of tomorrow.

NMDP. Find Cures. Save Lives. Mott is a member of NMDP, a nonprofit that is establishing, maintaining and improving a system that provides transplants of bone marrow and other hematopoietic cells from volunteer, unrelated donors for life-threatening blood diseases.

Therapeutic Advances in Childhood Leukemia & Lymphoma (TACL)

Mott is one of only 16 U.S. centers in TACL, a consortium focused on developing new and innovative clinical trials and advancing them into new therapies for relapsed or resistant leukemia.

THE PEDIATRIC BONE MARROW TRANSPLANT PROGRAM WAS AWARDED A $3.4 MILLION GRANT FROM THE NATIONAL HEART, LUNG AND BLOOD INSTITUTE (NHLBI) TO STUDY PULMONARY COMPLICATIONS IN BONE MARROW TRANSPLANT RECIPIENTS

OUR CELLULAR THERAPY AND BONE MARROW TRANSPLANT TEAMS HAVE RECEIVED OVER $10M IN NIH FUNDING FOR RESEARCH AND PRODUCED MORE THAN

300 PUBLICATIONS

Can Wearable Sensors Put Temperature Monitoring Back in Patients’ Hands?

For cancer patients who’ve had a bone marrow transplant or CAR T-cell therapy, a fever can be a sign of danger to come.

A simple FDA-approved temperature sensor in the hospital could help. A study of 62 adult and pediatric patients found a consumer-grade temperature sensing patch was able to detect fever up to five hours before routine temperature checks by hospital staff.

Pediatric oncologist Sung Won Choi, M.D. M.S., is a co-author of the study. She says the research team has already taken this approach to an outpatient setting and observed some patients were better able to care for themselves at home and get to a hospital faster than they would have with routine thermometer checks.

Hematology and Blood Disorders

Blood disorders affect children of any race, age or background, and often come with painful complications that reduce quality of life. At Mott, our management of pediatric blood disorders caters to the unique needs of each patient and family. Our Division of Pediatric Hematology is renowned for its expertise in nonmalignant hematology, including leukocyte disorders, coagulation disorders and thrombosis. Our research achievements are also changing care, with studies leading to genetic approaches for treating hemophilia.

Pediatric Hemophilia and Coagulation Disorders Program

The U-M Health Pediatric Hemophilia and Coagulation Disorders Program is the beginning of a lifetime of care. Our patients include infants, children, adolescents and young adults. Our physicians are experts in treating hemophilia, bleeding disorders and thrombosis, and we offer many clinical trials as treatment options. Our

First of Its Kind

The first and only approved gene therapy for the bleeding disorder hemophilia B remained safe and effective two years after treatment, according to new data from a clinical trial led by Steven Pipe, M.D., a hematologist-oncologist at Mott.

The gene therapy studied, Hemgenix, boosted the protein missing in hemophilia B that prevents blood from clotting properly to control bleeding. Most patients with the condition need lifelong, continuous treatment to replace the deficient clotting factor to prevent life-threatening bleeding episodes that can include excessive bleeding from cuts, an injury, surgery or dental work.

“This new gene therapy gives hope to pediatric patients that they can one day have an option that eliminates reliance on regular IV infusions and allows them to enjoy a freer, less-restricted lifestyle,” Dr. Pipe says.

Why Do Some Patients with Blood Clotting Diseases Have Worse Symptoms Than Others?

U-M Health pediatric hematologist Jordan Shavit, M.D., Ph.D., is using a novel tool to understand a potentially fatal clotting disorder that affects girls and young women. See how his research of zebrafish is helping to inform care and determine which medications are most beneficial for patients with estrogen-induced thrombosis.

Collaborative Gynecology and Hematology Clinic

Too often, adolescent girls and young women with gynecological symptoms have their concerns dismissed or misdiagnosed. Our Collaborative Gynecology and Hematology Clinic offers a multidisciplinary approach to caring for young women with bleeding and clotting disorders. Each patient is seen by a team that specializes in the care of adolescents and young adults, including a gynecologist, pediatric hematologist, nurse specialist and a social worker.

Unique and Compassionate Care for Young Women and Girls

Our Collaborative Gynecology and Hematology Clinic was open to serve patients for only one half day when it was established in 2016. The clinic was just expanded from weekly to six times per month and continues to grow. Faculty members like Angela Weyand, M.D., the co-director of our combined hematology/gynecology program, are also committed to driving research and improving quality of care and education to better serve young women and girls. Dr. Weyand has more than 70 publications on genetic modifiers of hemostasis and thrombosis, women and girls with bleeding disorders, hormone-provoked thrombosis, and von Willebrand disease.

U-M IS HOME TO TOP IMMUNOHEMATOLOGY & IMMUNODEFICIENCY PROGRAMS IN THE U.S.

1 OF ONLY 9 PROGRAMS IN THE COUNTRY TO OFFER A FELLOWSHIP IN GYNECOLOGY AND HEMATOLOGY

Improving Screening and Approaches to Care

Four out of 10 teen girls and young women may have iron levels low enough to cause symptoms such as low energy and dizziness, according to a study by Angela Weyand, M.D. Most don’t even know, since health screenings for their age group don’t include the proper blood test. Dr. Weyand’s study also showed that Black and Latina young women, as well as those near the poverty level, had higher rates of iron deficiency. Dr. Weyand hopes the findings will encourage care providers to order proper blood tests for female patients, and counsel them on eating foods rich in iron.

An Unknown Trigger for Rare Bone Marrow Disease

At age 9, Mireya started getting mysterious bruises on her body that didn’t heal easily. She was diagnosed with severe aplastic anemia, a disease that caused her bone marrow to stop making new blood cells and to function at less than 5% of normal.

This means her immune system attacks its own bone marrow, which leads to dangerously low blood cell counts and a high risk of infection. More research is needed to understand why the disease process that causes aplastic anemia suddenly develops.

Mireya stayed at Mott for nearly a week for treatment, where other diseases like cancer were ruled out. She has shown great progress, needing fewer medications and blood draws, and her blood cell count is also up.

Immuno-Hematology

Immuno-Hematology Comprehensive Program

The Immuno-Hematology Comprehensive Program is one of the nation’s longest tenured multi-specialty programs advancing research and providing care to children and young adults with rare immune and blood system disorders. We offer leading edge therapies and access to related clinical trials. Our program cares for patients with immune deficiencies, bone marrow failure syndromes, lymphoproliferative diseases, histiocytic disorders, autoinflammatory syndromes and over 700 other rare disorders. These disorders can vary in presentation and severity, so it is critical for patients to have an expert clinical team that can deliver a rapid diagnosis and personalized treatments.

For families in need of answers, our Immuno-Hematology Comprehensive Program brings together dedicated physicians, nurse practitioners, pharmacists, social workers, child life specialists and nurses from hematology, rheumatology, infectious disease, immunology, genetics and bone marrow transplant. This collaborative framework provides unsurpassed care and support.

Our program is committed to driving groundbreaking research to improve the understanding, diagnosis and treatment of these rare and serious diseases, as well as the impact the diagnostic journey and therapy has on patients and families. To ensure that we continue to make progress that can benefit future patients, we support multiple educational opportunities, including a medical student elective and mentoring programs for fellows.

U-M plays a leading role in the North American Immuno-Hematology Clinical Education & Research (NICER) Consortium, a multi-specialty group driving new research and clinical trials while also educating and caring for patients with rare immuno-hematologic disorders.

Primary Immune Deficiency Treatment Consortium

Mott is one of 47 centers in North America that is part of the PIDTC. Member institutions share information and a vision to improve outcomes for patients with rare, life threatening disorders of the immune system.

A 4-Year-Old’s Journey with Sickle Cell Disease

Every time Kennedy’s family took her to a doctor’s appointment, it seemed like they were preparing for another one right after that.

After a year of blood transfusions and spleen removal, Kennedy’s family has found a new normal.

“There’s more hope now for families,” says Sharon Singh, M.D., a Mott pediatric hematologist and oncologist. “There was only one sickle cell medication up until a few years ago. Now there are several more, and we keep getting closer to curative treatments.”

Complete Sickle Cell Care

Pediatric Comprehensive Sickle Cell Program

Ongoing assessment, intervention and active participation of the child and caregivers are essential to providing the most effective sickle cell treatments. Our coordinated, comprehensive care is available 24 hours a day, seven days a week with a physician, pain specialist, registered nurse, social worker, psychologist, dietician, school liaison or clinic coordinator. We are one of the few programs that provide care into adulthood and we are proud to be driving research that is improving understanding of sickle cell disease and leading to new treatments for patients.

Sickle Cell Gynecologic Clinic

We offer a Sickle Cell Gynecologic Clinic to provide specialized gynecologic care for adolescent females who might have unique needs due to their sickle cell diagnosis.

Living with Sickle Cell: From Isolation to Pursuing Medical School, Advocacy

Halimat Olaniyan was about 7 years old when excruciating pain sent her to an emergency room.

A series of tests revealed a diagnosis that had been missed at birth in her native country Nigeria: sickle cell disease.

With the support of Mott physicians, Halimat has been managing the disease as she attends medical school and advocates for others with sickle cell disease.

Genetic Therapy to Treat Sickle Cell Disease

The FDA has cleared the way for two groundbreaking sickle cell disease gene therapies — one of which is the first gene editing treatment to be used in humans. U-M teams are already working to bring these therapies to patients ages 12 to 25. Neither tool is a 100% cure but are intended to be one-time treatments that can significantly alleviate pain and prevent a lifetime of complications associated with the disease.

READ MORE: michmed.org/sicklecelltherapy

Surgical Expertise and Innovation

Solid Tumor Oncology Program

U-M Health has one of the nation’s largest multidisciplinary programs for children with solid tumors. Because we care for so many children with complex tumors, we have expertise in treating the rarest cancers, including bone sarcomas, germ cell tumors, hepatoblastoma, melanoma, metastatic tumors, neuroblastoma, rhabdomyosarcoma, relapsed and resistant diseases, and Wilms tumors. Our program is also driving critical research, clinical trials and quality improvement.

National Leaders in Neuroblastoma Surgery

Neuroblastoma develops in immature nerve cells, or neuroblasts, and is one of the most common pediatric cancers, affecting 700 children annually. Our surgeons have a long history of providing advanced, compassionate and multidisciplinary care. Families from all over the world come to Mott for our pediatric surgery expertise. Erika Newman, M.D., leads neuroblastoma surgeons nationally through the Children’s Oncology Group and helps set standards for care, participates in high-risk trials and reviews cases from around the country.

U-M Health surgeons delivered the fastest response for the rarest cancer.

Hyperthermic Intraperitoneal Chemotherapy (HIPEC)

The HIPEC procedure is a heated, sterilized chemotherapy solution that is used for patients with complex or advanced cancers that attach to the lining of the abdomen. Because the HIPEC procedure is administered directly into the abdomen, it’s not significantly absorbed into the bloodstream, helping minimize side effects associated with traditional chemotherapy. HIPEC can be a good option for recurrent diseases in the abdomen, like appendiceal cancer, Wilms tumor, certain types of sarcomas that have metastasized to abdominal lining, malignant ovarian germ cell tumors and metastatic colorectal cancer.

Wilms Tumor Therapy

Wilms tumor is a kidney cancer that typically affects children under the age of five. It is the most common form of childhood kidney cancer, but with early detection, the survival rate is 90%. The standard treatment is to surgically remove the tumor and follow up with chemotherapy. The Mott surgery team specializes in minimally invasive approaches and our radiation safety record is unsurpassed, with our faculty among nationally recognized experts in the safe delivery of radiation therapy.

TO SUCCESSFULLY REMOVE COMPLEX ABDOMINAL TUMORS

Clinical Trials

Oncology clinical trials test new ways of treating pediatric cancers, such as new drugs, new approaches to surgery or radiation, or new biologically targeted therapies. In a clinical trial, children receive state-of-the-art treatment and have a chance to be among the first to benefit from new therapies.

Participating in a clinical trial helps ensure that children diagnosed with cancer in the future will benefit from what is learned today. Mott scientists are actively participating in the latest innovative treatment protocols and clinical trials to improve survival rates and scientific understanding of childhood cancers.

Biorepository and Tumor Bank

Pediatric Hematology-Oncology and Bone Marrow Transplant maintains a biorepository and tumor, blood and cerebral spinal fluid bank with thousands of specimens from BMT, immuno-hematology, leukemia and solid tumor patients, including ultra rare and unique samples. This resource is driving important research and collaboration with other centers, and enabling us to reanalyze samples and develop innovative, new therapies if a patient relapses.

Relapsed and Resistant Cancer Program

Unfortunately for many families, some cancers resist therapy. Mott is one of very few children’s cancer centers in the U.S. that offers certain experimental therapies to young patients whose cancer has relapsed, has not responded to traditional treatment or does not have a reasonable cure.

Children’s Oncology Group (COG)

U-M is an active member of the Children’s Oncology Group, a clinical trials organization supported by the National Cancer Institute. COG is the world’s largest childhood and adolescent cancer research group. Our involvement enables us to offer families a wide range of clinical trials.

Our children’s cancer clinical trial program is the sixth largest in the U.S. by total enrollment

Our program offers Phase I clinical trials and first-in-children protocols available at a limited number of institutions

U-M is the only Children’s Oncology Group Phase I Consortium member in Michigan, which means that we offer unique studies unavailable at other hospitals in the state

As one of only a handful of New Approaches to Neuroblastoma Therapy and Therapeutic Advances in Childhood Leukemia consortium member institutions, we offer families access to a number of trials only available at NANT and TACL institutions

Mott is home to the largest portfolio of investigator-initiated clinical trials in oncology and transplant in the region

Our team conducts promising experimental trials sponsored by the National Cancer Institute and a number of pharmaceutical companies

Mott is one of the largest providers of clinical trials in the Midwest

Clinical Areas of Excellence

Childhood Cancer Survivorship Program

Our research seeks to better understand the needs of cancer survivors to develop more effective care and provide resources and support following the completion of treatment. We care for the patient’s total well-being by managing health and personal issues like growth and development, school challenges, organ dysfunction, neurocognitive problems or impaired fertility.

Adolescent and Young Adult Oncology Program

Adolescent and Young Adult (AYA) Oncology patients have unique needs compared to their younger and older counterparts. U-M Health’s AYA Oncology Program serves this population whose lifestyles are much different than other cancer patients. The AYA program connects patients with fertility counseling; mental, sexual, and nutritional health services; art and music therapy; school and vocational support; social work; peer mentors; support groups; and a teen room.

Cardiology-Oncology Long Term Follow-Up Care

Cardiac disease is a leading cause of non-cancer related death for childhood cancer survivors, as some treatments like chemotherapy and radiation can have detrimental side effects. A collaborative effort between Mott’s

pediatric cancer and cardiology programs, the CardioOncology Long Term Follow-Up Clinic manages care for cancer patients at high risk of developing heart issues. The clinic is one of the first of its kind in the U.S.

Psych-Oncology Program

This program aims to help children, parents and family members cope with treatments, stress and the negative social and psychological effects of a cancer or blood disorder. Patients and families can receive support in clinic, inpatient or outpatient settings, during infusions or through virtual appointments. Specialists include social workers, psychiatrists, psychologists, child and family life therapists, nurses, art therapists and complementary therapy professionals.

Cancer Genetics Clinic

The Cancer Genetics Clinic sees people with a personal or family history of cancers that may have genetic or inherited links. This includes colon, breast, ovarian, gastric, renal and pancreatic cancers; as well as melanoma, sarcoma and certain rare cancers including some childhood cancers. Our clinic can assess a patient’s risk of developing certain cancers and recommend preventative measures and/or proactive screenings.

Palliative Care

Palliative care focuses on the physical, psychological, social and spiritual needs of patients with chronic or lifethreatening illnesses. We offer comprehensive support for managing these conditions, helping to make everyone more comfortable during the illness. Our team of doctors, social workers, nurses and spiritual care providers are available around the clock to help children and families find the best quality of life possible.

Child and Family Life Programs

To make patients and families feel more comfortable during hospital stays, Mott offers services that provide support, distraction, stress relief and even a little fun. Activities like art and music therapy can help patients to express themselves and better cope with the challenges they are facing. Our hospital school program and various activity centers can help patients find a sense of normalcy. And video games, Lego robotics and other technologies enable them to enjoy their favorite hobbies and connect with friends outside the hospital.

Lipschutz-Danzansky Family Paws4Patients Program

Animal-assisted therapy has many positive effects on patients of all ages. It can lower stress and anxiety levels, improve blood pressure, increase patient mobility and provide an alternative focus from pain. Michigan Medicine is proud to have full-time facility dogs on our team, comforting patients and families across many units and areas of care, including a dedicated Paws4Patients dog on our Pediatric Hematology-Oncology floor at Mott. The dogs visit patients at the bedside and help to motivate and assist during certain types of therapy and rehabilitation.

Family Support Groups

A cancer or blood disorder diagnosis can leave families with many questions on how to navigate new challenges and lifestyle changes for their child. Mott has many support programs for parents, including a dedicated hemophilia and bleeding disorders parent group that connects parents to discuss helpful resources and issues related to hemophilia. We also have a pediatric oncology parent host, a position dedicated to supporting families with all non-medical needs, such as assisting them with navigating the hospital, finding laundry facilities or food options, and connecting them with any resources they may need during their child’s stay. The parent host position is held by a parent who has experienced their own journey with childhood cancer, ensuring that families know what to expect and are supported by someone who has walked in their shoes.

Research and Education

DRIVING LIFESAVING DISCOVERY

U-M Health pediatric oncologists are known nationally and internationally for expertise in translational research, precision oncology, and clinical and basic science research.

450 PEER-REVIEWED MANUSCRIPTS

OUR DEPARTMENT OF PEDIATRICS IS NUMBER 12 OUT OF 83 INSTITUTIONS IN FUNDING RECEIVED FROM THE NATIONAL INSTITUTES OF HEALTH

PUBLISHED OVER THE PAST FIVE YEARS, MANY OF THEM IN HIGHIMPACT JOURNALS PEDIATRIC HEMATOLOGY-ONCOLOGY

SHAPING THE FUTURE OF PEDIATRIC HEMATOLOGY AND ONCOLOGY CARE

Advances in knowledge and treatments for pediatric cancers and blood disorders are giving patients and families better options and more hope than they had five or 10 years ago. To build on this momentum and bring cures to future generations of children, we are committed to preparing the next group of leaders in the field:

Pediatric Hematology and Oncology Fellowship

A three-year program for individuals pursuing academic careers in hematology, oncology, cellular therapy, or bone marrow transplant, or those who will assume clinical leadership positions.

Boxer Training Program in Molecular and Translational Hematology

This NIH and NHLBI funded postdoctoral research program was established to better understand and translate the molecular basis of hematologic diseases and to continue to produce the next generation of scientific investigators in this area.

Pediatric Coagulation Specialist Training Program

This program provides a unique clinical and research training experience to prepare hematology fellows for a career in hemostasis and thrombosis.

Neuro-Oncology Advanced Training Program

Trainees gain experience in the diagnosis and management of primary and metastatic brain tumors, as well as in diagnosing and management of neurologic complications of cancer and its treatment.

Medical Student Education

The Division of Pediatric Hematology-Oncology and Bone Marrow Transplant is actively involved in teaching and training U-M Medical School students, providing them with opportunities to gain experience in the clinic, lab and classroom.

JOIN THE FIGHT TO

Every year during National Childhood Cancer Awareness Month in September, our Block Out Cancer campaign rallies supporters to raise money and awareness to improve outcomes for kids with cancer.

• Since 2014, Block Out Cancer has sold thousands of T-shirts and raised more than $1.1 million for pediatric cancer research.

• Proceeds have funded groundbreaking research at Mott.

• Block Out Cancer has also helped our physicians and scientists expand personalized approaches to care, increase survival rates and enhance treatments.

GET INVOLVED!

There are many ways you can show your support throughout the year for Mott Pediatric Hematology and Oncology patients and families:

• Donate

• Create an online fundraiser

• Host an in-person event

• Buy limited edition Block Out Cancer gear

• Follow us and share our content on social media

• LEARN MORE at blockoutcancer.org

BECAUSE OF YOU

Much of the hope we are able to provide Mott families is made possible by philanthropy. The generosity and involvement of supporters like you has transformed care, driven meaningful discoveries and shared incredible comfort with our Little Victors.

TO PARTNER WITH US

To support Pediatric Hematology and Oncology at C.S. Mott Children’s Hospital, contact: Ann Kay, Associate Director of Development, annvkay@umich.edu or Jennifer Polan, Assistant Director of Development, hurandjl@umich.edu.