The Sickle Cell Disease Association of Canada and Pfizer announce the First National Research Program for Sickle Cell Disease

Tuesday, November 14, 2017 - The Sickle Cell Disease Association of Canada/Association d’Anémie Falciforme du Canada(SCDAC/AAFC)and Pfizer are proud to introduce the first National Research Program for Sickle Cell Disease. This competitive research grant program aims to advance clinical research and patient care in Sickle Cell Disease (SCD).

Through this program, Canadian investigators will conduct clinical and psychosocial research on SCD including outcome evaluation. Grants will be awarded for clinical research, quality improvement, qualitative research, quality of life, and educational initiatives in fields relevant to improving the quality of life of persons with SCD.

Pfizer Canada brings therapies that significantly improve patients’ lives and this is accomplished through research into innovative medicines. Pfizer Canada works closely with patients, advocacy organizations, research centers, academic institutions, and other experts to share innovative practices from diverse perspectives.

“Evidently, Pfizer Canada is working with patient associations and advocates supporting patients’ needs and we are pleased that Pfizer Canada has struck a research partnership with SCDAC/AAFC as we support persons with SCD in Canada,” says Mrs. Lanre Tunji-Ajayi, President and Executive Director of SCDAC/AAFC.

“Pfizer Canada is proud to support this partnership with SCDAC/AAFC, their inaugural call for research applications,” says Dr. Mark Lundie, Medical Director Rare Disease for Pfizer Canada. “While discovering and developing new therapies for SCD patients is a priority for Pfizer’s Rare Disease Research Unit, it is through programs such as this national research partnership with SCDAC/AAFC that we can enhance our collective knowledge and understanding of the impact of this disease and the implementation of care for patients and their families.”

For the 2018-2019 granting period, specific areas of interest have been identified and investigators are encouraged to submit proposals within these areas. The Research Peer Review Committee (RPRC) of the SCDAC/AAFC will allocate funding to the strongest and most relevant projects including but not limited to the areas of interest listed below.

For 2018-2019 granting periods, Areas of Interest include but not limited to:

  • 1.Emergency Room Management of Vascular Occlusive Crisis
  • 2.Clinical Management of Vascular Occlusive Crisis
  • 3.Treatment Adherence to Prescribed Regimens
  • 4.Management of Adolescents and Young Adults with SCD
  • 5.Systemic Racism in SCD

For the SCDAC/Pfizer National Research Program for Sickle Cell Disease, individual grants for the 2018-2019 granting period are valued at a maximum of $20,000 per year for up to two years. Two grants of a maximum of $5,000 will also be available to applicants. If a two-year award is provided, the second year of funding is dependent on evidence of progress as described in the required progress report. Grant funding will begin on May 1st, 2018.

Application forms, general criteria and conditions will be released in January 2018 for the 2018-19 granting period and deadline to submit applications will be at 4PM EST on March 15th 2018.

About Sickle Cell Disease Association of Canada/Association d’Anémie Falciforme du Canada(SCDAC/AAFC)

  • Established in 2012, SCDAC/AAFC is committed to increasing awareness about SCD, enhancing methods of identification, diagnosis, and treatment towards improving the quality of life of affected individuals and their families.

The SCDAC/AAFC also encourages the establishment of coordinated clinical services and research initiatives that will support SCD treatments and efforts towards a cure. We believe that attracting clinical and research expertise to undertake and further SCD research in Canadian universities and healthcare settings is essential, and therefore we will support these initiatives.

To learn more about SCDAC/AAFC, you may visit: www.sicklecelldisease.ca; like us on Facebook (www.facebook.com/SickleCellDiseaseCanada) and follow us on twitter (www.twitter.com/sicklecellca)

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including hematology, neuroscience, and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

To learn more about Pfizer Canada, visit pfizer.ca or you can follow us on Twitter (twitter.com/PfizerCA) or Facebook (facebook.com/Pfizer.Canada).

The Announcement

Invitation for survey participation- June 2017:Supplemental oxygen during air travel in SCD—Patient experiences

We are inviting patients and/or families affected by sickle cell disease to complete a research survey about their experience with air travel. More specifically about their experience with health concerns due to sickle cell disease when travelling, as well as, the use of supplemental oxygen during air travel. Results from this study will help inform health care providers about how they can improve patient experience with air travel among patients/families affected by sickle cell.

We expect that this survey will take 5-10 minutes of your time. No personal information will be collected. Your participation in this study is entirely voluntary. Researchers involved in this study are affiliated to the Department of Pediatric Hematology at the University of Alberta. The University of Alberta Research Ethics Board has reviewed and approved this study.

Please use the following link (or copy and paste the link into your web browser) to access the survey: https://redcap.ualberta.ca/surveys/?s=9JA9X4J8KE

We ask that you complete and share this survey. Please feel free to contact Dr. Aisha Bruce (aisha.bruce@ahs.ca) or Amarjot Padda (padda@ualberta.ca), if you have any questions about the study. 

RESEARCH ADVISORY COMMITTEE (RAC):

The Research Advisory Committee will establish the research priorities of the SCDAC, its strategic research partnership and run the research program in the most effective way possible. SCDAC’s research agenda will not be limited to fellowships, basic research, clinical research, research by young investigators but also include social and psychosocial research initiatives. Hence RAC should include psychologists, social workers, and nurses in addition to academic clinicians. The research agenda designed by RAC must be best suited to the persons and processes served by the organization.

RESEARCH PEER REVIEW COMMITTEE (RPRC)

The Research Peer Review Committee is established to review submissions made in response to calls for competitive research proposals offered by the Sickle Cell Disease Association of Canada (SCDAC), and to advise the SCDAC with respect to continuous quality improvement in the review process. The principles inherent in the review process are identification of excellence, provision of equity of opportunity, and due diligence in the allocation of SCDAC research funds.

Guidelines for the RPRC



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The Medical Advisory Board of the SCDAC will be a three member team led by a hematologist. Other members of the MAB will include a hematology nurse and an allied health professional. The MAB will provide and advise SCDAC on medical matters.

MEDICAL ADVISORY BOARD- TERMS OF REFERENCE
Purpose:
The purpose of the MAB is to advise SCDAC on medical and scientific matters pertaining to the management and health-care of those affected with of people with sickle cell disease.
Duties and Responsibilities:
• To co-sign all documents containing medical facts including letters to the Ministry of Health and Long Term Care in Ontario and Health Ministers in all other Provinces
• To provide information to the SCDAC concerning the comprehensive management of people with sickle cell disease
• To assist the SCDAC in ensuring equal access to care and treatment for all persons with sickle cell disease
• To inform the SCDAC and RAC of research initiatives considered to be of benefit and interest to all persons with sickle cell disease.
• To represent the SCDAC, from time to time, on ad hoc committees developing policies on medical affairs issues
Nomination and Term of Office:
The MAB shall be chaired by a hematologist appointed by the SCDAC Board of Directors
The term of appointment shall be one year, and shall be renewable for up to four consecutive years

This board designs the research agenda for SCDAC/AAFC to include among other responsibilities attracting more researchers and fellows to the field of hemoglobinopathy and encourage data collection to support optimal evidence-based care and treatment

        This section is intended solely to inform patients and families of various clinical trials and patient recruitment currently underway. Their inclusion is not an endorsement of a particular product, company or study.

        CLINICAL TRIALS of medications…phases…the pros and cons…the rights of “subjects”…are trials necessary?

        You will find below links to web sites which provide answers to frequently asked questions about clinical research. 

        1. Clinical Trials FAQ- a Health Canada's Clinical Trial Database FAQ Page     
        2. ClinicalTrials.gov is a registry and results database of federally and privately supported clinical trials conducted in the United States and around the world. ClinicalTrials.gov gives you information about a trial's purpose, who may participate, locations, and phone numbers for more details. This information should be used in conjunction with advice from health care professionals.
        LOCATIONS OF CLINICAL TRIALS IN CANADA
        LIST OF CURRENT TRIALS

        2. Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor (Sickle-MAID)

Promoting and funding research to improve treatment and ultimately to find a cure

To fulfill one of its research objectives which is to attract more researchers and fellows to the field of hemoglobinopathy; SCDAC/AAFC is pleased to collaborate with the University Health Network on its Canadian Adult Comprehensive Hemoglobinopathy (CAtCH ) Fellowship program. It will continue to seek more opportunities as this across the country.

The CAtCH
There has been an improvement in survival of patients with Sickle Cell Disease over the past several decades, whereas in 1970s the life expectancy was teenage years, it has slowly improved to the 6th decade of life. This improvement in lifespan has impacted on the number of patients requiring expert ongoing medical care. To date, clinical training and research in Sickle Cell Disease has focused on the pediatric population. However, with improved life expectancy and survival into adulthood, there are new challenges in managing the complications unique to adults.

The Canadian Adult Comprehensive Hemoglobinopathy (CAtCH ) Fellowship is a combined clinical/research fellowship created in response to the needs of trainees who have shown interest in Sickle Cell Disease, and recognizes the lack of any substantive training opportunities. The Fellowship in Adult Hemoglobinopathies focusses on comprehensive care, multidisciplinary (inter-professional) collaboration, education outreach, clinical and translational research development.

The Fellowship aims to meet the need for clinicians and clinical / translational researchers in this field. It shall help fulfill current and future anticipated workforce needs across the country. This is an innovative, inter-disciplinary, and first of its kind program in Canada, focussing on comprehensive care and research in adult Sickle Cell Disease, and it has received a prestigious training award from the American Society of Hematology.

The first year encompasses the fundamentals of Sickle Cell as a chronic disorder, from prenatal through adolescence and into adulthood. Time is spent in the regional reference laboratory learning about newborn screening and diagnostic techniques, and in pediatrics gaining an appreciation for the effect of SCD on children’s physical, social and psychological development. The unique transfusion challenges faced in the Sickle Cell population such as availability of suitable donors and the role of exchange transfusion will be examined at the TGH hospital blood bank and also the Canadian Blood Service. The majority of clinical time will be at Toronto General, exposed to a large volume of patients with a diverse array of Sickle Cell related medical issues. The fellow is strongly encouraged to take a relevant MSc program, e.g clinical research, hemoglobinopathies, or public health.

In the second year, the fellow will further develop their academic interest in Sickle Cell Disease, gain additional clinical experience, and spend 2 months in the UK, to gain an appreciation of different models of care provision and building partnerships. If the Fellow is from outside of Toronto, it is expected they will return to their home institution and implement further Hemoglobinopathy training locally.

How can I be tested?

A simple blood test called the hemoglobin electrophoresis can be done at your local laboratory. This test will tell if you are a carrier of the sickle cell trait or if you have the disease.

What is Sickle Cell Disease?

Sickle Cell Disease is a disease that affects a protein called hemoglobin in the red blood cells. Hemoglobin carries oxygen around the body. When a person has Sickle Cell Disease, their hemoglobin does not work properly. This can cause pain, a higher chance of serious infections and organ damage or stroke. A person with Sickle Cell Disease needs lifelong treatment.

What does it mean to be a sickle cell carrier, or to have the sickle cell trait?

A carrier of Sickle Cell Disease is also said to have the sickle cell trait. This person has inherited a sickle cell gene from a parent, but does not have sickle cell disease and is not more likely to get sick than any other person. They do not need special medical care and will not develop sickle cell disease at any time in their life.
Why is it helpful to know that I am a carrier of Sickle Cell Disease, or whether my child is a carrier?
Knowing whether you or your child has the sickle cell trait is important for several reasons. When you are ready to have a family, your partner can have carrier testing so that you know, as a couple, if there is a chance of having a child with Sickle Cell Disease.
By having this information, you can let your child know if he or she is a carrier of Sickle Cell Disease in the future.

What does my child’s carrier result mean?
When a child is a carrier of Sickle Cell Disease, it is very likely that one of the parents is also a carrier of Sickle Cell Disease. More rarely, when a child is a carrier of Sickle Cell Disease:

  • Both parents are carriers
  • One parent actually has Sickle Cell Disease
  • One parent is a carrier and the other has Sickle Cell Disease

If I have more children, could they have Sickle Cell Disease?
Most often when a child is a carrier of Sickle Cell Disease, only one parent is a carrier and the chance to have a baby with Sickle Cell Disease is very low

When both parents are carriers, each pregnancy they have has:

  • 1 in 4 (25%) chance of having Sickle Cell Disease
  • 1 in 2 (50%) chance of being a carrier (but not having Sickle Cell Disease)
  • 1 in 4 (25%) chance of not having Sickle Cell Disease or being a carrier

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You can have a blood test to find out if you are a carrier of Sickle Cell Disease. If you want to have this test, talk to your health care provider.
A carrier does not have, and will not develop, Sickle Cell Disease.

Sickle cell disease is an inherited condition. People who are at high risk of having a child with sickle cell anemia and are planning to have children may want to consider genetic counseling. A counselor can explain the risk (likelihood) of having a child who has the disease. He or she also can help explain the choices that are available. If a person is born with sickle cell disease, steps should be taken to reduce complications. Genetic counseling is most available at major health centres, hospitals, and clinics that care for people who have sickle cell anemia.

How can I meet with a genetic counsellor?

If you and your partner are carriers of Sickle Cell Disease, a genetic counsellor can help you learn more about the genetics of Sickle Cell Disease and your options of testing in a future pregnancy.

There is no treatment for Sickle Cell Disease in pregnancy; however testing is available to couples who want to learn if their baby has Sickle Cell Disease as early as possible. You can ask your health care provider to refer you to your local Genetics clinic. If you do not have a health care provider, contact your local Genetics Clinic directly for more information.

Should I test my other children to see if they are carriers of Sickle Cell Disease?

If only one parent is a carrier of Sickle Cell Disease, there is a 1 in 2 (50%) chance your other children are carriers. Carrier testing for Sickle Cell Disease is not usually done in childhood because being a carrier does not cause health problems in childhood. Carrier testing can be done when your other children are old enough to understand the information and decide on their own if they want to be tested. Some parents have their other children tested at a young age and share this information with their child at a time they feel is right both for their child and their family. If you have more questions about testing your other children, talk to your health care provider.

If both parents are carriers, your other children should be tested for Sickle Cell Disease.

Should my relatives be tested?

Other family members (for example, cousins, aunts, uncles) might be carriers of Sickle Cell Disease. It is important to let family members know about this chance. They can then decide if they want to have carrier testing for Sickle Cell Disease. This is especially important if they are planning to have children. Rarely, family members might have Sickle Cell Disease.

NEW BORN SCREENING
The provinces of Ontario, British Columbia, Nova Scotia, New Brunswick, Prince Edward Island, Yukon and progressively Quebec have included sickle cell disease in their newborn screening programs. Only four provinces and two territories remain until the goal of universal newborn screening for SCD throughout Canada is achieved. It will continue to push for other provinces and provinces to screen all newborns for sickle cell disorder.

Screening involves a simple blood test performed at birth that detects the presence of sickle cell disease or sickle cell trait.

Other types of traits that may be discovered include:
Hemoglobin C trait
Hemoglobin E trait
Hemoglobin Barts – which indicates an alpha thalassemia trait
Beta thalassemia trait

What is Sickle Cell Trait?
Sickle cell trait is a person who carries one sickle hemoglobin producing gene inherited from their parents and one normal hemoglobin gene. Normal hemoglobin is called type A. Sickle hemoglobin called S. Sickle cell trait is the presence of hemoglobin AS on the hemoglobin electrophoresis. This will NOT cause sickle cell disease.

Are there different types of sickle cell disease?

There are three commonly diagnosed types of sickle cell disease:

  1. Hemoglobin SS or sickle cell anemia
  2. Hemoglobin SC disease
  3. Hemoglobin sickle beta-thalassemia

Each of these can cause sickle pain episodes and complications, but some are more common than others. All of these may also have an increase in fetal hemoglobin which can protect the red cell from sickling and help prevent complications. The medication hydroxyurea also increases fetal hemoglobin.

There are three commonly diagnosed types of sickle cell disease:
Hemoglobin SS or sickle cell anemia
Hemoglobin SC disease
Hemoglobin sickle beta-thalassemia
Each of these can cause sickle pain episodes and complications, but some are more common than others. All of these may also have an increase in fetal hemoglobin which can protect the red cell from sickling and help prevent complications. The medication hydroxyurea also increases fetal hemoglobin.
What are the complications?
Complications from the sickle cells blocking blood flow and early breaking apart include:

  1. Pain episodes
  2. Strokes
  3. Increased infections
  4. Leg ulcers
  5. Bone damage
  6. Yellow eyes or jaundice
  7. Early gallstones
  8. Lung blockage
  9. Kidney damage and loss of body water in urine
  10. Painful erections in men (priapism)
  11. Blood blockage in the spleen or liver (sequestration)
  12. Eye damage
  13. Low red blood cell counts (anemia)
  14. Delayed growth

What can be done to help prevent these complications?
Sickle cell patients should be under the care of a medical team that understands sickle cell disease. All newborn babies detected with sickle cell disease should be placed on daily penicillin and monitored closely to prevent serious infections and maintain their well-being. All of the childhood immunizations should be given plus the pneumococcal vaccine. Parents should know how to check for a fever because this signals the need for a quick medical checkup for serious infection.

The following are general guidelines to keep the sickle cell patient healthy:

  1. Taking the vitamin folic acid (folate) daily to help make new red cells
  2. Daily penicillin until age six to prevent serious infection
  3. Drinking plenty of water daily (8-10 glasses for adults)
  4. Avoiding too hot or too cold temperatures
  5. Avoiding over exertion and stress
  6. Getting plenty of rest
  7. Getting regular check-ups from knowledgeable health care providers

Patients and families should watch for the following conditions that need an urgent medical evaluation:

  1. Fever
  2. Chest pain
  3. Shortness of Breath
  4. Increasing tiredness
  5. Abdominal swelling
  6. Unusual headache
  7. Any sudden weakness or loss of feeling
  8. Pain that will not go away with home treatment
  9. Priapism (painful erection that will not go down)
  10. Sudden vision change

Acknowledgement: Some of the information on this page was a courtesy of:

  1. Toronto General Hospital website and Dr. Richard Ward (TGH)
  2. Ontario Newborn Screening website and Dr. Robert Klaassen (CHEO)
  3. Grady Memorial Hospital’s Sickle Cell Information Centre and National Heart, Lung, and Blood Institute websites