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BLOOD HEALTH Not All Rare Blood Disorders Are Treated Alike Dr. Durhane Wong-Rieger
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Dr. Durhane Wong-Rieger President & CEO, Canadian Organization for Rare Disorders
ll Canadians with rare blood disorders should have the same access to the best treatment. That's the vision for the national Rare Disease Drug Strategy to be set up in 2022-23 with a $1 billion federal commitment. Regardless of where people live or their treatment needs and cost, patients should have equitable access. Today, this scenario is true for only some rare cancers, a few rare disorders, and very few rare blood disorders. Among those with the best access are patients with hemophilia, who came together in creating a state-ofthe-art program out of the tainted blood scandal of the 1980s. In contrast, access to screening, clinics, emergency care, and appropriate medicines for thalassemia and
sickle cell disease has been less exemplary. Barriers, especially to innovative medicines, have been even greater for those with ultra-rare conditions, such as, aplastic anemia, myelodysplastic syndromes, Waldenstrom’s anemia, thrombotic thrombocytopenic purpura (TTP), and paroxysmal nocturnal hemoglobinuria (PNH). How can a national Rare Disease Drug Strategy help? Increasingly, reimbursement for treatments is based on health technology assessment (HTA), namely, cost-benefit assessment, which often disadvantages drugs that may not prolong life but improve quality of life. We encountered this with slow-release clotting factors for hemophilia and oral versus intravenous iron removal therapy for thalassemia. Traditional HTA methodology has a
bias against novel therapies that have little immediate clinical impact but that reduce future events. This results in delays and denials for eculizumab (which reduces the risk of blood clots in PNH), lenalidomide (which reduces transfusion dependency for myelodysplastic syndromes), emicizumab (which stimulates factor production for hemophilia), luspatercept (which prolongs the life of red blood cells in thalassemia), and caplacizumab (which reduces the time to recovery for acquired-TTP). The Canadian Organization for Rare Disorders has been consulting with multiple stakeholders on the Canadian Rare Disease Drug Strategy, specifically to develop innovative rare disease therapies with potential lifetime benefits.
We invite all stakeholders to join our ongoing webinar consultation series by visiting raredisorders. ca/cord-raredrug-strategyimplementationwebinars/.
Stem Cell Donors Could Give Someone the Gift of Life Obinna Okwelume
E Melissa Deleary Health Policy Analyst, Ontario Federation of Indigenous Friendship Centres
very year, hundreds of Canadian patients require life -sav ing stem cell transplants to treat over 80 diseases and disorders, including cancers. Unfortunately, three quarters of those patients won't find a match within their own families. To survive, they’ll turn instead to the Canadian Blood Services Stem Cell Registry. About seven years ago, while working as a health policy analyst for the Ontario Federation of Indigenous Friendship
Centres, Melissa Deleary organized a workplace event to recruit donors for the registry. As the event organizer, she thought that by swabbing her own cheek to collect a DNA sample for the registry, she'd inspire others to do the same. But in 2019, she got a call herself, with news that she was a good match for a patient in need of a stem cell transplant. “I know there are very few Indigenous people on the Canadian Blood Services Stem Cell Registry,” says Deleary. “So, when I got the call, I knew it was probably
because someone really needed my help.”
The need for diversity in the registry Patients with Indigenous, Asian, Hispanic, Black Canadian, and other backgrounds find it especially challenging to find a match. “We need individuals from all ancestral backgrounds to provide matches for patients,” says Dr. Heidi Elmoazzen, Stem Cells Director at Canadian Blood Services.
Healthy Canadians between 17 and 35 years of age can register online at blood.ca to receive their swab kit in the mail.
What is Myeloma? Martine Elias
M Martine Elias Executive Director, Myeloma Canada
ultiple myeloma (myeloma) — often confused with melanoma (skin cancer) — is the second most common blood cancer. Every day, nine Canadians are diagnosed with myeloma, yet it sadly remains relatively unknown, and without a cure.
Understanding Multiple Myeloma Myeloma, a blood cancer, is associated with the abnormal, uncontrolled growth of plasma cells, a type of white blood cell
made in the bone marrow that helps fight infection. In myeloma, plasma (myeloma) cells crowd out healthy blood cells and overproduce an antibody called M-protein, which often leads to health complications, fatigue and recurrent infections. When thinking about myeloma symptoms, think CRAB: elevated Calcium, Renal (kidney) issues, Anemia, Bone fractures. Every person’s experience with myeloma is unique, so when it comes to your health, trust your instincts. If you’re not
feeling right, tell your doctor, regardless of how inconsequential the changes seem. Don’t wait. Cancer doesn’t. If you or a loved one has myeloma, you don’t have to go through it alone. Myeloma Canada is there for you. The only national charitable organization exclusively devoted to helping improve the lives of Canadians impacted by myeloma, it has the resources, information, tools, and support to help you understand, cope with and manage every phase of the disease.
Visit Myeloma Canada’s website to learn more at myeloma.ca or call, toll-free: 1-888-798-5771. This article was sponsored by Myeloma Canada.
Publisher: Olivia Delbello Business Development Manager: Julia Colavecchia Strategic Account Director: Jessica Golyatov Country Manager: Nina Theodorlis Content &Production Manager: Raymond Fan Designer: Lauren Livingston Web Editor: Karthik Talwar All images are from Getty Images unless otherwise credited. This section was created by Mediaplanet and did not involve Toronto Star or its editorial departments. Send all inquiries to ca.editorial@mediaplanet.com. @MediaplanetCA
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This article was sponsored by the Canadian MPN Research Foundation and the Canadian MPN Network.
How Life Changes When Told You Have an Ultra-Rare Blood Disorder Acquired Thrombotic thrombocytopenic purpura (aTTP) is a rare and sometimes fatal blood disorder. Awareness, timely diagnosis, and prompt treatment are needed to ensure positive outcomes.
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Anne Pamphel
n September 2008, Sydney Kodatsky, stepped out of a swimming pool, removed her goggles, and suddenly found herself unable to see. The 28-year-old newlywed and recent MBA graduate visited a major Toronto emergency room, waited for five hours, and was sent home with a migraine diagnosis. With no relief from her headache or fluctuating vision the next day, she went to her family doctor, who reassured Sydney that there was nothing to worry about. It was only after seeking a third opinion, at her mother’s insistence, and getting the results of some routine bloodwork, that Sydney finally got her diagnosis — acquired thrombotic thrombocytopenic purpura (aTTP).
Rareness of aTTP can make it difficult to diagnose aTTP is a rare but life-threatening autoimmune blood disorder that creates blood clots throughout the body. Because it's so rare, diagnosis can be difficult or delayed, as in Sydney’s case, putting the patient at further risk of serious complications. “I was extremely lucky not to have experienced a heart attack, stroke, or kidney failure before my treatment was able to kick in,” she says. aTTP is diagnosed at a rate of three to four in one million people per year worldwide. The cause is not well understood, though it's believed to be hereditary in about one percent of patients. “About 65 percent of aTTP patients will go through the treatment process once and never
experience a relapse,” says Sydney. Others, like Sydney, have it on a recurrent but unpredictable basis. Since her initial diagnosis, Sydney has had seven relapses, the last one in 2016. It takes extraordinary effort to stabilize an aTTP patient during a crisis with the therapies available in Canada, and the experience for patients is truly life-altering. The current treatment options for an aTTP crisis are a combination of plasmapheresis and immunosuppressant medication. During each two- to four-hour plasmapheresis treatment, a patient’s entire blood volume is removed through a central line to replace the plasma. Sydney has required 700 or so units of plasma so far, and she's grateful to those who donate blood. Unfortunately, the immunosuppressive treatment that saves lives can also have devastating consequences. At only 35 years old, Sydney started a battle with metastatic cancer. Most pat ient s diagnosed with aTTP are young women in their childbearing years. “If you're lucky enough to recover, aTTP can really derail their plans for having a family, so these patients need to work closely with their hematologists to understand the risks associated with pregnancy and aTTP,” says Sydney. As young newlyweds, Sydney and her husband faced immense challenges they never foresaw to successfully build their family.
The daily challenges and fear of relapse are very real Living with aTTP is challenging. “There's no day-to-day treatment and there’s
nothing you can do to control the timing of a possible recurrence,” says Sydney. “It’s the ultimate disruptor because you don’t know if and when a recurrence could happen and land you in the hospital for weeks or months, so you need to have backups for everything.” Aside from the disruption, there's also the fear of relapse. “That fear is very real because not only can it derail your life, but there’s also a 20 percent chance of dying from each aTTP episode with treatments available in Canada,” says Kodatsky. As a result, many aTTP patients deal with anxiety and depression.
I was extremely lucky not to have experienced a heart attack, stroke, or kidney failure before my treatement was able to kick in. Determined to change the outlook for herself and others living with aTTP, Sydney founded the Answering TTP Foundation in 2010. The foundation focuses on fundraising, research grants, patient advocacy, and education with a view to improving diagnosis and access to improved treatment options for aTTP patients. “Getting timely access to efficient and effective treatment is challenging and there are only about 40 treatment centres across Canada that can treat aTTP,” she says. Sydney is nevertheless optimistic about the future. “Canada pioneered new treatments for aTTP that increased survival by about 75 percent, and there are currently promising treatments available in peer nations to target the mechanism of the disease to reduce harm,” she says.
To learn more visit answeringttp.org. In partnership with
National Organization for Rare Disorders (NORD). Rare disease database: Thrombotic thrombocytopenic purpura. https://rarediseases.org/rare-diseases/ thrombotic-thrombocytopenic-purpura/
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2 Kremer Hovinga J et al. Survival and relapse in patients with thrombotic thrombocytopenic purpura. Blood 2010; 115(8):1500-1511https:// doi.org/10.1182/ blood-2009-09-243790 3 Pavenski K et al. Efficacy of Caplacizumab in patients with aTTP in the HERCULES study according to initial immunosuppression regimen. Blood. 2019;134 (Supplement 1): 2365; https://doi.org/10.1182/ blood-2019-123126 4 Rock GA et al. Comparison of plasma exchange with plasma infusion in the treatment of thrombotic thrombocytopenic purpura. Canadian Apheresis Study Group. N Engl J Med. 1991 Aug 8;325(6):393-7.
The placement of this article was made possible with support from a research based pharmaceutical company
The Canadian MPN Research Foundation and the Canadian MPN Network Are Meeting the Needs of the Canadian MPN Community Cheryl Petruk
W
hen someone says "blood cancer," you may immediately think of leukemia, but there are many types of blood cancers that aren’t as well-known. For those living with myeloproliferative neoplasms (MPNs), these serious cancers are very real. MPNs are progressive blood cancers in which the bone marrow typically overproduces one or more types of blood cells. The three most common classical MPNs are polycythemia vera, essential thrombocythemia, and myelofibrosis. With stem cell research and the discovery of key gene markers, there have been significant advances in the diagnosis, understanding, and treatment of MPNs, but there are still no known
cures for most of these blood cancers. The Canadian MPN Research Foundation is on a mission to change that. Founded in 2018 by Executive Director Cheryl Petruk, the organization has a single goal: to stimulate and fund original and ongoing research in pursuit of new treatments and eventually a cure for MPNs. Petruk became an advocate for the MPN community in 2014 when she began caring for her late husband, who was diagnosed with myelofibrosis. The members of the Canadian MPN Research Foundation's board of directors each have a very personal involvement and commitment to the foundation's mission. The Canadian MPN Network (CMPNN) Patient Advocacy Group, which was founded in 2014, works
symbiotically with the Canadian MPN Research Foundation in providing the MPN patient community with the vitally-needed support system to help navigate their journey with an MPN blood cancer. The CMPNN provides the patient community, with a variety of resources to make the navigation of a rare blood cancer journey more manageable. The CMPNN has a board of directors made up of patients and care partners who work tirelessly in advocating for this patient community. Together, the Canadian MPN Research Foundation and the CMPNN are a testament of collaboration in providing the entire MPN community of physicians, researchers and patients with hope, inspiration, knowledge and education about MPN blood cancers.
For more information on the Foundation and to fund vital MPN Research, visit cmpnrf.ca. To find out about patient resources and support groups, visit canadian mpnnetwork.ca. Your donation is vitally needed to fund research for this rare blood cancer! To learn more, call 1 866 644 6766.
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New Treatments Are Providing Hope to Canadians with Hemophilia Advanced therapies reduce the burden of illness and improve the quality of life for people living with a bleeding disorder. Abigail Cukier
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Dr. Jayson Stoffman Medical Director, Manitoba Bleeding Disorders Program
Mathieu Jackson Hemophilia Patient
hen he was a baby, Mathieu Jackson bruised easily and would often bleed from his mouth. His mom repeatedly took him to the hospital, but doctors couldn't find the cause. When he was almost two, Jackson had major swelling in one knee caused by internal bleeding, which led to him being tested and finally diagnosed with hemophilia B. “Blood clotting is controlled by a series of proteins that act in sequence to create a blood clot. When you have hemophilia, you're missing one of those proteins, which means your blood takes significantly longer to clot,” says Dr. Jayson Stoffman, Medical Director of the Manitoba Bleeding Disorders Program. Hemophilia is a rare congenital bleeding disorder characterized by a deficiency of essential blood clotting proteins, coagulation factor VIII in hemophilia A patients and coagulation factor IX in hemophilia B patients. Hemophilia A is estimated to account for 80 to 85 percent of all cases, while hemophilia B is estimated to account for 15 to 20 percent of cases. While hemophilia can be hereditary, in up to a third of patients, there's no known family history. This was the case for Jackson.
Bleeding causes pain and joint damage Hemophilia causes bleeding in the joints and muscles, including the ankles, knees, hips, elbows, wrists, and fingers. “And every time you bleed in a joint, the joint becomes more susceptible to bleeding,” Jackson says. “For example, my left ankle will bleed frequently for no reason,
just from walking. That’s because I’ve Treatments lead to better control had so many bleeds inside that ankle. Dr. Stoffman is excited about new And when you bleed, it’s extremely treatments available, which lead to painful. A lot of people compare it to better hemophilia control and make hospital and clinic visits less neceshitting your joints with a baseball bat.” Every two days, Jackson gives sary. Tremendous advances have himself an intravenous injection been made in several aspects of of factor replacement therapy. This hemophilia management, including standard half-life treatment for extended half-life products, which hemophilia is made from human stop blood loss more effectively and plasma or genetically engineered for longer periods, meaning patients in a lab. The factor concentrate is inject the treatment less frequently. nearly identical to the protein lack“With better control, the likelihood of ing in the blood of someone with bleeding is lower and patients are less hemophilia. But the replacement is likely to need to be seen in the hosquickly removed from the body, pital, emergency room, or clinic,” which is why treatment is needed says Dr. Stoffman. every two to three days. We’ve stressed throughGene therapy on the out the pandemic that it’s horizon important for patients not “The ultimate, which is to ignore issues out of coming is gene therapy,” fear of the healthcare says Dr. Stof f ma n. Blood clotting is system. “There's already gene controlled by a series of proteins that act Treatment also therapy available for in sequence to create a includes reg ula r factor IX in clinical blood clot. When you have monitoring by a trials, which is great hemophilia, you are misshealth care team. news for people ing one of those proteins, Dr. Stoffman says living with hemowhich means your blood that some patients philia B. Factor VIII takes significantly have been anxious about gene therapy is coming. longer to clot. coming in for appointWhen that works, I'll be ments during COVID-19. the cure. You get a single treat“We’ve stressed throughout the panment and your body starts making its demic that it’s important for patients own protein again.” not to ignore issues out of fear of the “The idea of a life without bleeding health care system," he says. "COVIDor with one or two bleeds is something 19 shouldn't be a barrier when seeking I can’t even imagine," says Jackson we care, especially when you’re living with give future generations of people with a chronic condition. Communication hemophilia the potential to do more, I with your health care team is imperative think we'll all gain from it.” for better health outcomes.”
To discover more, visit hemophilia.ca.
This article was made possible with support from a research based pharmaceutical company.
Raising Awareness About Women With Bleeding Disorders Natalie Philbert, Amy Griffith, and Dr. Paula James
The most common misconception about bleeding disorders is that women can’t have them. As a result, there can be a dismissal of symptoms, lack of diagnostic investigation, and no treatment offered for bleeding symptoms. Raising awareness to undiagnosed women who may have a bleed-
Do your gums bleed frequently when you brush you teeth?
ing disorder is a challenge, as many women are unsure of what constitutes a bleeding disorder. There's also a lack of understanding about normal versus abnormal bleeding, (especially for gynecologic/menstrual bleeding and postpartum bleeding).
Do you ever feel like your periods are too long or too heavy?
Do you have frequent nosebleeds?
Do you bruise easily?
If any of these sound like you, you may have a bleeding disorder. It's estimated that nine out of 10 people are undiagnosed. If you think this could be you, check out the hemophilia.on.ca/bleeding-disorders/ following resources or contact us at 1-888-838-8846 or info@hemophilia.on.ca. women-with-bleeding-disorders
The Importance of Understanding Thrombosis
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hrombosis Canada is dedicated to education and building awareness about thrombosis. Issues around blood clots and COVID-19 have made our work more relevant than ever. The COVID-19 pandemic has negatively affected the health of millions of Canadians relating to COVID-19 and other areas of health. One of the unexpected consequences has been an increased awareness of thrombosis (blood clots), because thrombosis is very common in conjunction with COVID-19 infection. As a consequence, more Canadians are aware of the threat of thrombosis to their health than they were a year ago.
What is thrombosis? Thrombosis can occur in various parts
of the body: Deep vein thrombosis (DVT), in the leg Pulmonary embolism (PE), in the lung Cerebral venous sinus thrombosis (CVST), in the venous sinuses of the brain, which prevents blood from draining from the brain Portal vein thrombosis (PVT), the blood vessel that brings blood to the liver from the intestines. Approximately 1.3 out of 1,000 Canadians per year will develop thrombosis. The most common symptoms are leg tenderness or swelling, chest pain, light-headedness, and breathlessness. Recognizing the signs and symptoms of thrombosis can help you to seek timely medical assistance. Our website has short videos and many publications to help you learn more about thrombosis and understand your risks. Learn more
at thrombosiscanada.ca.
Thrombosis and COVID-19 COVID-19 has introduced a new concern for Canadians around thrombosis. Everyone should know that there's an increased risk of thrombosis associated with COVID-19, as we learned early in the pandemic. That's one of the reasons why we strongly recommend that all Canadians get vaccinated.
Learn More Increased awareness means that more Canadians are reviewing our education materials, including our CLOTS resources (thrombosiscanada.ca/ clots). Please consider donating to help us to continue our work at thrombosiscanada.ca/donate.
This article was sponsored by Thrombosis Canada.