Patient Engagement Forum

Hello! My name is Dr. Tom Kerlow. I am presently a board member of aHUS Canada. aHUS (atypical Hemolytic Uremic Syndrome) is a chronic, life threatening ultra rare disease affecting less than 2 people per million annually. In those living with aHUS, part of their immune system becomes uncontrolled, resulting in blood clotting and leading to vital organ damage.
I, like most other board members, have either been affected personally or have family members that have the disease. In my case, both my wife and one of my sons have the disease and both have undergone successful kidney transplants.
At present, there is only one medication, eculizumab, (Soliris) that can treat and keep the disease under control. However, it is extremely expensive and it is often very challenging to get approval from the government. Treatments such as plasma exchange and plasma transfusion can be used to help treat patients with aHUS while they await government approval for Soliris. One may require up to twenty units of plasma per day. Anemia is another common symptom of active aHUS and patients often require multiple blood transfusions.
I am looking forward to working with all of you in an effort to benefit all patients afflicted with aHUS.

Hi everyone! I am Caroline Mitchell, National Senior Community Services Manager for the Leukemia and Lymphoma Society of Canada. In Canada, there are currently 155,593 people living with or in remission from one of the 137 types of blood cancers. The LLSC is the largest registered charitable health agency dedicated to supporting the blood cancer community in Canada. Our focus over the years has grown and now includes: funding research from bench to bedside, rethinking how a person navigates their blood cancer experience, provider of targeted blood cancer information, offering tools for psychosocial and emotional support and empowering Canadians to take charge of their blood cancer experience through practical support and advocacy. It is a pleasure to meet everyone.

Hello! Nice to meet you all! I am Liz Charyna, Manager of Programs and People with the Canadian Hemochromatosis Society. The Canadian Hemochromatosis Society was incorporated in 1982 by Marie Warder after her husband was diagnosed with a disorder they had never heard of called hereditary hemochromatosis. Marie wanted to be sure that Canadians were aware of the common genetic disorder of iron overload that when recognized early can be easily treated, yet left undiagnosed can be fatal. 2022 marks the 40th anniversary of the Canadian Hemochromatosis Society and starting May 1st (Hemochromatosis Awareness Month) we are driving a bus across Canada to spread more awareness. Our members have many stories of living with extreme fatigue and pain for years before getting the diagnosis and some members have been diagnosed without ever having any symptoms. Treatment for people with hereditary hemochromatosis is phlebotomy or blood donation so we have a natural relationship with Canadian Blood Services and are proud to have a Partners for Life team. We look forward to strengthening our relationship with Canadian Blood Services and learning more from all the Forum participants.

Hi Everyone! I'm Whitney Ayoub Goulstone, currently the Executive Director of the Canadian Immunodeficiencies Patient Organization (CIPO). It's great to see you all here!
CIPO represents those affected by primary immunodeficiency disease (PI) in Canada, which represents over 400 different genetic disorders affecting the immune system. Patients are normally treated with IG (immunoglobulin) replacement therapy, either through IV or at home with SCIG.
PI is vastly underdiagnosed and undertreated in Canada, with some studies pointing at up to 70-90% of patients either underdiagnosed or undertreated in some regions. The average time to diagnosis currently is 6-12 years in Canada.
I am looking forward to working with you all in this new forum.

Greetings!

My name is Jennifer DiRaimo, MS, CCGC. I am a research program manager working to enhance the lives of those living with a platelet disorder, primarily immune thrombocytopenia (ITP). ITP is a rare disease that is an autoimmune bleeding disorder that results in a low platelet count that leads to increased risks for bleeding and prolonged bleeding. ITP affects individuals of all ages. In children, the disorder is usually self-limiting lasting only a few months. When ITP lasts for over a year, a diagnosis of chronic ITP is given.

Treatment is reserved for those who have a history of bleeding, or a very low platelet count. In children, treatment is provided as rescue therapy or for preventative measures if there has been a history of serious bleeding. IVIG and steroids are the main first-line treatments for ITP. Most respond well to these therapies, however, the effect on the platelet count is often very limited lasting only days or a few weeks at best, and are not a long-term solution to reducing risk. Second-line therapies are available however they are difficult to access without private insurance or self-pay.

My first-born son was diagnosed with ITP in Feb (2015). He was 7 years old at the time and was not considered a bleeder. Things changed over time, his condition changed and worsened, and he died in May (2018) from a brain bleed secondary to a critically low platelet count from ITP. As a Canadian certified clinical genetic counselor with 14 years of experience working in a hospital environment, I switched gears and left my clinical job to work with the Platelet Disorder Support Association (PDSA). PDSA is a global not-for-profit organization that works with patients and families with ITP and other platelet disorders. PDSA is registered as a non-profit group in Canada and has partnered with organizations such as CORD and NRBDO to better support and advocate for Canadian ITP patients. PDSA has Canadian and American hematologists on the medical advisory board, who are world-renown experts in ITP and platelet disorders.

I look forward to the great work I know we will all do together in this group.

Jenn

Hello All,

My name is Biba Tinga - President/ED of the Sickle Cell Disease Association of Canada/Association d'anémie falciforme du Canada ( SCDAC/AAFC), the national umbrella for Sickle Cell provincial groups.

Sickle Cell disease is an inherited blood disorder that affects about 6000 people in Canada. One of the most frequent manifestation of SCD is vaso-occlusive crisis which occurs when microcirculation is obstructed by sickled red blood cells.
Treatment options are limited with red blood cell transfusion and hydroxyurea as the main disease-modifying therapies. We recognize the need for blood for our community and we collaborate with various partners including CBS and Héma Quebec to Identify barriers and enablers to blood donation.

In 2021, SCDAC, its member organization SCFA from Alberta, Carleton University, Concordia University of Edmonton, and Canadian Blood Services are leading a Social Sciences and Humanities Research Council funded project with the aim of increasing donation by the African, Caribbean, and Black young adults.
My adult son lives with SCD. He has been on blood exchange for over 10 years.
Looking forward to building stronger allyships.

Hi everyone, my name is Ashley Oakes and I am a Board Member and former Chair of the Aplastic Anemia and Myelodysplasia Association of Canada (AAMAC). I have served on the AAMAC Board for 7 years and in that role participated as a member of the CBS National Liaison Committee since I joined the AAMAC Board. I was diagnosed with Aplastic Anemia in 2010 while 7 months pregnant with my son and received treatment including immune-suppressive chemo therapy and many dozens of red cell and platelet transfusions. In 2013 I relapsed and was again dependent on transfusions throughout treatment. AAMAC supports patients across Canada who have been diagnosed with Aplastic Anemia, Myelodysplastic Syndromes and Paroxysmal nocturnal hemoglobinuria which are all bone marrow failure disorders. In my career life I am the Executive Director for Howe Sound Women's Centre Society supporting and advocating for survivors of gender-based violence in the Sea to Sky Corridor region of British Columbia

Hello, everyone. My name is Angela and I am the Executive Director of Alpha-1 Canada. I have been advocating on behalf of Canadian alpha-1 patients and their families since 2016. Alpha-1 Antitrypsin Deficiency (AATD) is a genetic condition that affects approximately 1 in 5,000 Canadians. AATD is a rare hereditary condition characterized by low circulating levels of alpha-1 antitrypsin protein in the blood, leading to increased risk of severe lung and liver disease. Alpha-1 is a laboratory diagnosis and augmentation therapy (a plasma protein replacement therapy) is the only specific treatment for severely deficient alpha-1 patients. To learn more about alpha-1 antitrypsin deficiency visit www.alpha1canada.ca or www.accessforalphas.ca

Looking forward to working with all of you and building a closer partnership between CBS and Alpha-1 Canada as well as all the other organizations taking part in this engagement forum.

Durhane Wong-Rieger, President & CEO, Canadian Organization for Rare Disorders, an umbrella organization for rare disease groups

Hello all!

I'm Carin Lin, Chair of the Support Committee at Kawasaki Disease Canada (https://kdcanada.org/)

Kawasaki disease (KD) is the leading cause of acquired heart disease in children in North America. It is an acute illness (a form of vasculitis) that causes inflammation in the walls of blood vessels throughout the body, including the coronary arteries which supply blood to the heart.

The majority of children who develop KD are 5 years of age or younger (75-80%), although it can affect older children and teenagers. Currently, the incidence of KD in Canada is estimated yearly to be close to 30 per 100,000 children under the age of 5.

Treatment with IVIG within 10 days of the start of the fever is highly effective in the prevention of coronary artery complications and most children will make a complete recovery without lasting damage. Without treatment, approximately 25% of children will develop some abnormalities of the coronary arteries. With IVIG treatment, that number is reduced to 5%.

My son has coronary aneurysms as a result of KD, despite having received IVIG treatment. In addition, he has also received blood transfusions during his bypass surgery and after being admitted to the hospital due to extremely low hemoglobin, resulting from unending nosebleeds caused by his anticoagulation and antiplatelet therapy.

Looking forward to connecting with everyone and learning from other patient organizations.

Carin

Hello! My name is Daphne and I am proud to be the COO of Hereditary Angioedema (HAE) Canada, a patient advocacy group with a membership of both HAE patients and their caregivers. HAE is a rare inherited disorder characterized by recurrent painful episodes of severe swelling in different parts of the body including the hands, feet, face and airway. C1-esterase inhibitor (C1-INH) activity is impaired in the majority of patients but a subset of patients exhibit the same symptoms despite normal C1-INH (HAEnC1-INH). In addition, patients often have bouts of excruciating abdominal pain, nausea and vomiting that is caused by swelling in the intestinal wall. Airway swelling is particularly dangerous and can lead to death by asphyxiation.
Many HAE patients across Canada receive their treatments from CBS, making HAEC thrilled to be a part of this engagement forum. We look forward to taking part and want to thank you for giving us this great opportunity to share our thoughts and perspectives.

Hello Friends.
I'm Mekki, representing Chronic Lymphocytic Leukemia Canada (CLL Canada). I was diagnosed with CLL in 2014 (with symptoms dating back to 2009). I've received a blood transfusion during the course of my disease and currently receive weekly at-home sub-cutaneous immunoglobulin treatments with Cutaqig, which is provided by Canadian Blood Services. Happy to contribute the CLL patient perspective to this forum.

Hello Everyone, My name is Chavakaye' Cameisha and I'm currently a a patient advocate with The Sickle Cell Awareness Groupof Ontario (SCAGO). As someone with lived Sickle Cell experiences I use my experiences and knowledge to help educate others while working to create settings where individuals receive the best care possible without discrimination or fear of judgment.

Hi All,
I'm Anna, Coordinator for the Canadian Association for Porphyria. Porphyria is a family of rare disorders related to heme synthesis. The disease experience varies depending on the type of porphyria and can include intense abdominal pain, nerve damage, seizures, skin burning and blistering in regular light. More information is available on our website canadianassociationforporphyria.ca. Looking forward to connecting and contributing with all of you.

Hello,
My name is Bojan Pirnat and I am the Manager of Member and Clinic Relations for Hemophilia Ontario. It's a pleasure to join you all here as the representative for Hemophilia Ontario. Hemophilia Ontario represents all inherited bleeding disorders in Ontario including Hemophilia, Von Willebrand's Disease, platelet function disorders, and other rare bleeding disorders. I look forward to working with all of you and building a closer partnership between CBS and Hemophilia Ontario as well as all the patient organizations taking part in this engagement forum.

Hello All!
My name is Wendy Quinn. I am currently the President of the Canadian Hemophilia Society, an organization that serves all bleeding disorders which include the Hemophilias , Von Willebrands disease, platelet disorders and any rare condition that causes bleeding. We have had a very close relationship with CBS stemming from the tainted blood tragedy in 80's and 90's, and the commitment that CBS has made to always closely consider our members needs is demonstrative. This patient engagement forum is further evidence of that commitment and we are grateful to be included and look forward to contributing any way we can .

Hello all,
I am Lanre Tunji-Ajayi, M.S.M, the president/CEO of the Sickle Cell Awareness Group of Ontario and it’s an honour to join this patient engagement forum
As part of my past roles, I have chaired the Network of Rare Blood Disorder Organizations, served as the founding president/CEO of the Sickle Cell Disease Association of Canada, and the Global Alliance of Sickle Cell Disease Organizations.
Sickle Cell Disease (SCD) is a genetic blood disease mainly affecting people of black heritage though the disease also affects people of other cultures. Life with SCD is characterized by recurrent, unpredictable episodes of severe pain, hospitalizations, and progressive end-organ damage.
Though considered rare in many parts of the world including Canada, it is very prevalent in other places such as the sub-Sahara Africa. An estimated 1.8M people also have SCD in India.
As a patient organization serving a marginalized community, the Sickle Cell Awareness Group of Ontario understands the importance of cross-disease collaboration and along with the Thalassemia Foundation of Canada, established the Ontario Hemoglobinopathy Patient Association (OHPA) in 2017. The OHPA continues to achieve monumental successes including:
1. the recent passing of Bill 255 recognizing June 19 as Sickle Cell Awareness Day and May 8 as Thalassemia Awareness Day in the province of Ontario; and
2. the recent funding of 5 hemoglobinopathy clinics in Ontario
To learn more about the SCAGO and its work, please visit: www.sicklecellanemia.ca and I truly look forward to learning from and working with all of you.
Warm regards,
Lanre Tunji-Ajayi- SCAGO

My name is Janet Pereira I’m the Executive Director of Diamond Blackfan Anemia Canada. Diamond Blackfan anaemia is a bone marrow failure where many patients rely on monthly blood transfusions to survive. In addition they must chelate nightly to rid their bodies of the iron that accumulates from chronic blood transfusions. My 13 year old son is one of these patients. You can learn more about DBA by visiting our website at www.dbacanada.com

Hello! I am Jennifer van Gennip and I'm here representing the Network of Rare Blood Disorder Organizations (NRBDO) as the Executive Director. It's an honour to support the rare blood disorder community in this role as we work together to ensure high-quality, effective, accessible, interdisciplinary care for all Canadians affected by rare blood disorders. You can learn more about the NRBDO, including a listing of member organizations and our advocacy work, at www.nrbdo.ca. I look forward to being part of this forum, and I'm optimistic about this initiative as a way for CBS and patients to engage with and hear from each other.

I am the Executive Director of GBS/CIDP Foundation of Canada, a GBS patient in 2009, and diagnosed with CIDP in 2011.

Guillain-Barré Syndrome (GBS), Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), and their variants such as Multifocal Motor Neuropathy (MMN), are rare conditions which are paralysing and potentially catastrophic inflammatory disorders of the peripheral nerves and categorised as neuromuscular conditions. GBS symptoms are rapid onset of weakness and, often, paralysis of the legs, arms, breathing muscles and face, in some cases leading to complete paralysis and requiring life-sustaining hospital care. CIDP and MMN are chronic neuromuscular conditions requiring ongoing treatment. the cause of GBS, CIDP, and MMN is unknown, and these conditions can develop in any person, regardless of age, gender or ethnic background. Immunoglobulin is the first line treatment for GBS, CIDP, and MMN.

GBS, CIDP, and MMN have a slow and unpredictable recovery, patients and their families face an uncertain future, usually requiring months of hospital care without knowing if or when they will recover, or whether they will face long-term disabilities. Earlier diagnosis, treatment, and access to rehabilitation services can improve the chances of avoiding permanent lifelong residual damage of the nerves.

In 2003, GBS/CIDP Foundation of Canada, a patient organization providing support, education, research, and advocacy, was founded so that no patient or family would go through GBS, CIDP, MMN or their variants alone.

For more information go to: www.gbscidp.ca. I look forward to being actively involved in CBS's new engagement strategy and look forward to meeting everyone!