Category Archives: MARAC Statements

MARAC Encourages Clinical Research Studies

The Sickle Cell Disease Association of America (SCDAA) Medical and Research Advisory Committee (MARAC) believes that progress in sickle cell disease (SCD) is tied to clinical trials and comprehensive care. For this reason, we encourage individuals with SCD to consider participating in research. It’s because thousands of courageous children and adults with sickle cell disease signed up for research that we have improved survival, developed new medicines and found possible cures. Download the full statement. 

I’ve been invited to be in a clinical trial – what should I know before I sign up?

Here are questions to ask if you are invited to be in a clinical trial. Many of these questions are answered in the informed consent papers – it will be lot to read, and it may be confusing, but it is worthwhile to pay attention and ask questions. You should always get a copy of the consent papers for your records.

  1. What is the purpose of the study?
    There are many types of clinical research studies, but the goal of research is the same – to improve the lives and treatment of individuals with SCD. Some studies involve the use of new drugs. Others look at existing approved therapies to see whether they can be useful for SCD. Sometimes studies are simply questionnaires, X-rays and blood draws. Make sure to ask your physician about the trial’s purpose.
  2. What kind of treatments and tests are involved in the trial?
    As mentioned above, the type and number of treatments differ for each clinical trial. During the trial, a lab technician may need to draw blood or perform an electrocardiogram to ensure that you are reacting well to the treatment. Ask your clinical care team about the treatments and tests you can expect throughout the trial process.
  3. What are the possible risks or side effects of this treatment?
    Side effects vary by trial. You will be closely monitored throughout the clinical trial to ensure that any potential side effects are addressed. It is important to understand all the potential risks or side effects of the treatment before the trial begins.
  4. How will the trial affect my daily life?
    Asking this question will help you be prepared for your upcoming treatment and plan your personal schedule during the trial. Each trial has a different schedule of care. How often do you have to come to the hospital or clinic? Will you have to stay in the hospital during the clinical trial? If so, how often and for how long? How far will you need to travel to take part in the trial?
  5. Do I have to pay for any of the treatments? What costs will my health insurance cover?
    Talking to your health care team, as well as facility staff and your insurance company, will help you answer these questions. Your insurance plan should cover the standard of care or routine care associated with the trial. Research-specific tests and procedures will be paid by the sponsor the trial sponsor. Sometimes transportation assistance is provided and you may receive money to compensate for your time and effort.
  6. Who will oversee my care while I am participating in the trial?
    Your care team will consist of a physician overseeing your participation as well as a team of nurses and research personnel assisting with your treatment and day-to-day care.
  7. What questions should I ask about my other choices?
    What are my other treatment choices, including standard treatments? How does the treatment I would receive in this trial compare with other treatment options? What will happen to my SCD without treatment?
  8. What questions should I ask about my decision process?
    How long do I have to make up my mind about joining this trial? Who can I speak with about questions during and after the trial? Can I talk to who has been in the trial? What if I decide to leave the trial?
  9. What questions should I ask about my rights to privacy?
    How will my health information be kept private? What happens to my data if I decide to leave the trial? Be cautious about clinical research organizations that might want to sell your data to others and/or claim that you will pay less to participate in their trial.
  10. If I benefit from this therapy, will I be allowed to continue receiving it after the trial ends? If I get a placebo, can I switch to the active drug?
    The design of some clinical trials allows patients to continue using the therapy as long as they are benefitting. Clarifying this question with your care team is important.

WATCH: “I participated in clinical trials. I would do it again.” (Video testimony from the National Institutes of  Health)

My friend was asked to participate in a clinical trial, and I wasn’t – why not?

  • Certain kinds of clinical research study new medications with unknown side effects, so they focus on people who are the least likely to be harmed by the new medication. For example, many studies do not accept older people, or individuals who have kidney/liver problems, asthma, are pregnant, heart problems or a high risk for bleeding.
  • If you have not come regularly for sickle cell care or had difficulty taking medications on their prescribed schedule, the research investigators will wonder whether you will follow the rigid schedule of a clinical research study. You may have had valid reasons such as medication side effects or transportation issues. Discuss these barriers with the study team. They may be able to help.
  • Advocate for Ask your doctor(s) whether you are eligible for any clinical research. Some busy doctors simply need a reminder. Others might not have a research study open for enrollment but could refer you to a nearby clinical research group.
  • Go to a specialty sickle cell Clinical research requires a lot of expertise. You might need to find a specialty center with the right level of resources to offer research studies. Consider going at least once a year to an academic, reputable sickle cell center.
  • You might seek out clinical research that does not exclude Surveys, questionnaires and registries can help to change health care systems or set the stage for future research.

How do I find more clinical research opportunities?

  • Ask your doctor(s) whether you are eligible for any clinical research
  • Visit ClinicalTrials.Gov, a government sponsored listing of all approved clinical trials in the U.S. and some abroad.
  • Ask your local SCDAA member organization (or the patients’ organization you belong to) whether they have news about SCD clinical research in your region.
  • Seek studies using clinical research finders for sickle cell on the SCDAA website and oneSCDvoice.com.
  • Many registries study SCD using regular questionnaires. The SCDAA Get Connected Registry is being set up to serve this Registries may also try to relate symptoms with blood tests or genetic tests to lay a foundation for better preventive care in the future.

I’m nervous about participating in clinical research – isn’t it risky?

Clinical research participation today looks very different from the past. Many feel nervous about clinical trials after learning about problems with the Tuskegee syphilis project and the HELA cells from Henrietta Lacks. Know that modern clinical trials are set up with layers of safeguards for patients and opportunities to ask questions. Sponsors of clinical trials keep the interest of patients as a priority. Extra cautions are taken to protect children as research participants. Another new feature of clinical research is the community advisory boards for research studies and SCD research networks. Many individuals with SCD now serve on these boards and you can too.

Can clinical trials replace my regular sickle cell care?

All individuals with SCD should have regular health care appointments, but you should make sure check in with your care team before and after participating in a clinical trial. Comprehensive sickle cell care is available because of those who participated in past research. Honor those who came before you by taking care of yourself.

LEARN MORE:

Questions to ask – adapted from https://www.cancer.gov/about-cancer/treatment/clinical-trials/questions, and from https://sarahcannon.com/blog/entry/7-questions-to-ask-when-considering-a-clinical-trial accessed 9/15/22.

Here are links that describe the features of clinical research studies:

G Puppalwar, M Mourya, G Kadhe, A. Mane. Conducting clinical trials in emerging markets of sub-Saharan Africa: review of guidelines and resources for foreign sponsors. Open Access Journal of Clinical Trials, 7 (2015), pp. 23-34

*MARAC is a diverse group of SCD providers who have volunteered to provide SCDAA and the sickle cell community with trustworthy advice. The primary advice of SCDAA MARAC is directed toward the sickle cell population in the United States, but we recognize frequent implications for other countries and recognize there is wisdom to gain from other countries.

**SCDAA is the leader in promoting and advancing initiatives focused on people affected by sickle cell conditions worldwide. SCDAA is the largest national community-based organization for sickle cell disease. For 50 years SCDAA and its more than 50 member organizations have demonstrated how community-based organizations can work as partners with medical facilities and local and state government agencies to pursue national health care objectives.

Who is watching over the study?

  • IRB (Institutional Review Board) – A special group at a medical center that watches over clinical research safety.
  • DSMB (Data Safety Monitoring Board) – They regularly go over all study data looking for new problems with safety and whether the study should continue.
  • DSMC (Data and Safety Monitoring Committee) – Similar responsibilities as the DSMB.
  • FDA (Food and Drug Administration) – United States government agency that is responsible for drug safety.

MARAC Advisory Statement: Immunizations

August is National Immunization Awareness Month. What does that mean for individuals with sickle cell disease (SCD)?

The Big Picture from SCDAA MARAC
Individuals with sickle cell disease are more likely to have complications from many infections and receive a lot of benefit from immunizations. Family members can help protect a child or adult with sickle cell disease by also getting immunizations.

More Information
Immunizations and vaccines protect people from serious disease. The possible risks from a vaccine are much smaller than the possible bad outcomes from the diseases.

Streptococcus Pneumoniae

  • Streptococcus pneumoniae bacteria was a leading cause of death for children with SCD.
  • Infection with Strep pneumo can move quickly from fever to extremely sick in the intensive care in just a few hours for children with SCD. Losing spleen function due to clogging of the spleen with sickle red blood cells is what makes Strep pneumo bacteria so dangerous for SCD patients.
  • Immunizations against Strep pneumo are important for every child, and extra immunizations are recommended for individuals with SCD: Pneumovax-23 and PCV20.

Monkeypox (MPV)

  • MARAC monitoring has not found any unusual monkeypox problems in individuals with sickle cell disease. If anybody hears of unusual events with monkeypox and sickle cell disease, please pass along this information to the SCDAA Chief Medical Officer Dr. Lewis Hsu at info@sicklecelldisease.org.
  • MARAC encourages anyone with symptoms of monkeypox (MPV) to see a health care provider to get tested. If you do not have access to a provider, visit findhealthcarecenter.hrsa.gov to get connected.
  • There is a vaccine available for those who are at highest risk. If you are experiencing any symptoms contact your health care provider, avoid close contact with others and take a break from sex, going out to bars, gyms, clubs and other events. Do not share personal items, wash your hands and clean shared surfaces regularly.

COVID-19

  • The latest data continues to show that SCD does have increased vulnerability to severe COVID.
  • COVID can trigger SCD symptoms like vaso-occlusive pain or sickle acute chest syndrome.
  • MARAC strongly urges everyone with SCD and their families to get vaccinated and get all recommended boosters. In addition, you should consider wearing a mask, avoiding crowds and standing at least six feet away from other people.
  • Do not attend social events if you have symptoms of COVID-19 or tested positive for COVID 5 days prior, even if you’re vaccinated.
  • If you do catch COVID, SCD is among the conditions eligible to receive treatments to reduce the risk of getting severe disease and hospitalization. Contact your health care provider.

Influenza (the Flu)

  • MARAC encourages everyone to get immunized against influenza, especially individuals with SCD and their family members.
  • Influenza can lead to severe illness in individuals with SCD. Influenza can trigger a vaso-occlusive painful event or acute chest syndrome. The risks of the immunizations are less than the risks of severe influenza disease.

Other diseases

  • Immunizations against meningitis are very important in all individuals living with sickle cell disease.
  • Children with sickle cell should all be immunized following the recommendations of the CDC and American Academy of Pediatrics. Childhood immunizations can help protect against diseases that are deadly or disabling, like measles, polio, mumps, whooping cough or rubella.
  • There is currently an outbreak of Polio in communities with low rates of polio immunizations. Polio can cause permanent paralysis.
  • In tropical areas with malaria, avoid mosquito bites and take anti-malarial medicines, especially young children with SCD. Malaria immunizations began to be offered in 2021.
  • The risks of the immunizations are much smaller than the risks of these severe diseases.

Click here to download this MARAC statement.

MARAC Advisory Statement: Monkeypox

July 20 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee (MARAC) is aware of the news about monkeypox and is monitoring the situation. Please follow advisories from organizations such as the Centers for Disease Control and Prevention to avoid close, skin-to-skin contact with individuals who have a rash that looks like monkeypox. Learn more from the CDC here and read the CDC monkeypox FAQs here.

This statement has been provided by members of the Sickle Cell Disease Association of America’s Medical and Research Advisory Committee.

MARAC Advisory: COVID-19 and Sickle Cell Disease

March 2022 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee notes that news about COVID-19 continues to emerge. Monitor your state or local health department for more information. Below are some updates and suggestions for individuals with sickle cell disease.

Several studies have examined the effects of COVID-19 infection in individuals in the sickle cell community and shown concerning results to include:

  • Individuals with sickle cell disease do have higher risks for severe COVID-19 disease and hospitalization compared to those of the same age without sickle cell disease. Poor kidney function raises the risk for severe COVID-19. A blood flow problem in the lungs called pulmonary hypertension also raises the risk for severe COVID-19.
  • COVID-19 infection often triggers sickle vaso-occlusive pain in adults. This type of crisis can cause the most intense pain an individual with sickle cell has ever experienced.
  • COVID-19 infection can also trigger sickle acute chest syndrome. Not everyone gets the same problems and children might have mild cases, but severe disease and death are unpredictable.
  • The American health care system has not solved the issues of racial and ethnic disparities in health
    during the pandemic. People of color have a higher risk of COVID-19 complications and are dying from COVID-19 at younger ages.

The positive news is:

  • The risk of death from COVID-19 for those with sickle cell disease is not as high as the risk of death from COVID-19 in patients who are receiving an organ transplant or are in active chemotherapy for cancer.
  • Sickle cell trait does not present a higher risk of severe COVID-19 or death.

The COVID-19 vaccine is safe, and the vaccine’s risks to individuals with sickle cell disease are very low when compared to the risks of a COVID-19 infection.

COVID-19 vaccines have now been given to tens of thousands of people with sickle cell disease across many countries for at least 14 months. People with sickle cell report about one to two days of side effects, which is about the same as reports from the general population.

SCDAA MARAC members have found that very rare cases of sickle cell pain have been reported after vaccination for COVID-19.

ACTIONS YOU CAN TAKE

  1. Stay up to date with your COVID-19 vaccines.
  2. If you feel comfortable wearing a mask, you can. You are at risk. Don’t be bullied.
  3. Continue medications and preventive care for sickle cell disease.
  4. Seek care when needed. Do not delay getting care for your medical condition. Health care professionals have infection prevention plans to help protect you from getting COVID-19 if you need care. If you have sickle cell disease and do not have a regular doctor, we strongly recommend that you connect with one.
  5. Keep up with your state or local health department for more information.

TEST SOON AND TREAT EARLY (CDC infographic in English and Spanish)

  1. Americans can request free COVID-19 tests mailed to your home. To learn more, visit COVIDtests.gov.
  2. If you have symptoms that are suspicious for COVID-19, take a home COVID-19 test or get tested in a medical facility.
  3. Several treatment options are now available for an individual with sickle cell disease who tests positive for COVID-19. Treatments can prevent a mild infection from leading to severe disease, hospitalization or death. Click here to learn more. Ask your doctor or pharmacist for the treatment that is used for your location, age and medical condition.

This statement has been provided by members of the Sickle Cell Disease Association of America’s Medical and Research Advisory Committee.

Download this statement.

MARAC Advisory Statement: Update About COVID-19

December 23, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee reminds the sickle cell community that the COVID-19 pandemic is having another increase in infections. New variants like omicron are emerging, as are common for RNA viruses, and vaccination rates need to consequently improve. Vaccinations can protect against severe illness. A new medication might also help as early treatment for infected individuals.

MARAC Recommendations

  • No general recommendation for all individuals with SCD to stay home nor for all to return to in-person activities. Patients and families need to make individualized assessments of the risks and trade-offs of returning to work or school in person. Factors to consider include vaccination status, the rate of COVID-19 and variants in your community, family socioeconomic situation, protective measures in the building (proper ventilation, physical distancing, etc.), mental health needs and educational needs.
  • Rare vaccine-related side effects, such as pain crises, have been reported after receiving a COVID-19 vaccine. However, they are not more likely to occur as a result of the COVID-19 vaccine compared to other common vaccines. No serious adverse events have been reported to date, and the risk/benefit ratio is in favor of vaccination for all individuals with SCD over age 5.
  • Continue to recommend vaccination against COVID-19.
  • Booster shots: All Americans are eligible, except children under 5. We recommend that individuals with SCD should receive a booster dose of the COVID-19 vaccine (Pfizer or Moderna).
  • Continue to recommend general precautions like wearing masks, maintaining physical distancing, promoting good ventilation and washing hands. These are public health measures.
  • The FDA has granted emergency use authorization of early treatments for people who test positive for coronavirus and who are at high risk for severe COVID-19, including hospitalization or death.
    • FDA announced on Dec. 22 and 23 that two new antiviral pills can be available to treat people with symptomatic COVID-19: Paxlovid (ritonavir plus nirmatrelvir, Pfizer) and Molnupiravir (Merck). Paxlovid is for patients ages 12 and up who weigh at least 88 pounds. It is available by prescription only and would be taken as soon as possible after diagnosis and within 5 days of the start of symptoms. Molnupiravir (Merck) is for adults, also byprescription only, also as soon as possible after diagnosis. MARAC is still learning more about these treatments.
    • Neutralizing monoclonal antibodies (bamlanivimab plus etesevimab (Lilly); casirivimab plus imdevimab (Regeneron); Sotrovimab (Glaxo Smith Kline) have been effective as early treatment for mild, symptomatic COVID-19 in some individuals with SCD [<10d after the test, age 12y+, wt 88 lb+ (40kg+), not hospitalized, not newly on oxygen]. However, these neutralizing antibodies might no longer be effective against the omicron variant of coronavirus. MARAC is monitoring the situation.
  1. Please remember that you can take precautions to stop COVID-19. The coronavirus spreads in the tiny droplets breathed out by an infected person. The person might not know that he or she is infected. COVID-19 spreads quickly because many people breathe these contagious tiny droplets in the air.
    • Please stay aware of local conditions that impact the risk of making contact with someone who is infected. Keep track of the rate of new cases of COVID-19, the rate of vaccination against COVID-19 and whether new variants of the coronavirus are appearing near you.
    • Where vaccination rates are low, the coronavirus spreads quickly. Continue to take precautions so that you can reduce your risk of COVID-19 infection: wear masks, maintain physical distance from others, and avoid people who are coughing or otherwise contagious as well as indoor spaces and crowds.
    • Wearing a mask is not officially required in some situations but MARAC strongly advises that individuals with SCD should still wear masks in many situations.
    • Wear a mask if:
      • you must be in a crowded indoor space.
      • you are surrounded by strangers or friends who might have been exposed to people who are sick.
        You can remove the mask when you and all those around you have been vaccinated and you are certain that nobody around you is sick.
  2. MARAC continues to advise individuals with sickle cell disease to get vaccinated against COVID-19.
    • Any of the three types of vaccines available in the U.S.A. are safe for individuals with SCD.
    • Vaccinations against coronavirus have now been shown to be safe for millions of people, with a known pattern of temporary aches that go away in about two days. It is rare for people to get severe side effects after receiving the vaccine.
    • The vaccines against coronavirus protect very well against serious symptoms of COVID-19 and reduce the chances of hospitalization and death. Break-through infections after vaccination have resulted in mild cases of COVID-19, not severe.
    • People who are not vaccinated may be barred from some travel and entertainment opportunities.
    • Protect the ones you love. Most senior citizen centers will not allow entry by people who are not fully vaccinated. Young children under 5 are not yet eligible for vaccination and depend on adults around them to be protected from infection.
  3. How much riskier is COVID-19 for individuals with SCD? Research indicates that the risk of death from COVID-19 could be 1.5 to 2 times greater for individuals with SCD compared to the general population (based on the Brandow & Panepinto SECURESCD database, U.S. single-institution reports, UK and French databases). While still significant, the risk is not as high as MARAC feared at the beginning of the pandemic.
    • Risk of complications for COVID-19 in SCD is probably higher than in the general population. The rate of COVID-19 hospitalizations is higher because pain or fever from COVID-19 could result in SCD hospitalization.
    • Death and moderate to severe complications are higher for younger SCD patients. Organ damage risk factors such as kidney damage are also higher compared to the general population.
    • There are probably excess deaths during the pandemic because of deferred care for other problems (e.g., poor care in pregnancy, delayed diagnosis of cancer).
    • Individuals with SCD and kidney or lung problems are more likely to have severe COVID-19 infections.
    • People with SCD-SC and SCD-Sbeta+thalassemia can have moderate or severe COVID-19 cases.
    • Over half of the reports of COVID-19 in SCD were associated with intense vaso-occlusive pain or sickle acute chest syndrome.
    • Individuals with SCD can have mild cases of COVID-19.
    • COVID-19 remains dangerous for everyone, especially minority groups.

    SCDAA News Advisory: Partial Hold on Gene Therapy Trial

    On December 20, the FDA placed a partial hold on bluebird bio’s clinical program for lovotibeglogene autotemcel (lovo-cel) gene therapy, temporarily stopping testing on study volunteers under age 18. This partial hold was a response to the investigation of one adolescent with sickle cell disease who has persistent anemia (not dependent on transfusion) a year and a half following treatment. Read more.

    Adults can continue enrollment and treatment in bluebird bio studies. Other clinical research studies of gene therapy for sickle cell disease are continuing.

    The clinical research process has many built-in safety precautions, which include the partial hold implemented by the FDA. MARAC will continue to monitor the situation closely on behalf of the individuals and families living with sickle cell disease.

    MARAC Advisory Statement Update About COVID-19 Vaccines

    September 24, 2021 — News about COVID-19 continues to move quickly as we weather the pandemic. The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee provides the following updates on vaccination recommendations:

    1. Individuals with SCD are now eligible for the third dose of the COVID-19 vaccine. The CDC recommends that people who are immunocompromised or have medical conditions with poor antibody production receive vaccine boosters. One of the eligible conditions is asplenia (no spleen function). Because of the relationship between asplenia and SCD, sickle cell patients are eligible to receive a third dose of the vaccine if they have already received the first two doses.

    2. If you have not had the first dose of the vaccine, please do so as soon as possible. Nearly everybody is eligible to receive their first shot. Only children under age 12 or individuals with known allergies to the vaccine ingredients are ineligible. Thousands of individuals with SCD have now had the COVID-19 vaccine. Like the public, many had some side effects that went away after three days. Only a tiny number of individuals experienced sickle cell pain after their shot. A COVID-19 infection poses a great risk for people with SCD. The vaccine is a safe way to reduce that risk. To find a vaccine location near you, visit www.vaccines.gov.

    To view a full list of MARAC members and to download this statement, click here.

    MARAC Issues Updated COVID-19 Guidance


    August 26, 2021 – The Sickle Cell Disease Association of America Medical and Research Advisory Committee has released two statements with updated COVID-19 guidance.
    MARAC recommendations are making a minor shift to emphasize a more individualized approach:

    • Continue to recommend vaccination against COVID. For booster dose of vaccine, currently SCD is not eligible but expect changes in CDC guidance.
    • Continue to recommend general precautions: wearing masks, keeping physical distancing, good ventilation, and washing hands. These are public health measures.
    • Recommend neutralizing monoclonal antibodies (mAb) – (Regeneron) as early treatment for mild symptomatic COVID in individuals with SCD [<10d after the test, age 12y+, wt 88 lb+ (40kg+), not hospitalized, not newly on oxygen]. Encourage getting tested if you have COVID symptoms. Possible prophylactic treatment with Regeneron antibodies if exposed to COVID.
    • No general recommendation for all individuals with SCD to stay home nor all to return to in-person activities. Help patients and families make individualized assessments of risk and trade-offs of returning to work or school in-person. Factors to consider were listed in MARAC July 2020 “checklist for return to school” (plus vaccination status): the community’s rates of COVID and variants, vaccination status, family socioeconomic situation, ventilation and other protective measures in the building, mental health needs, educational needs.

    Read the full statement for more information on the above.
    Additionally, MARAC recommends that people with sickle cell disease should have access to monoclonal antibody (mAbs) treatment according to established guidelines, to prevent mild COVID from progressing to severe disease. Read the full recommendations for providers, policy makers and patients.
     

    MARAC Statement on Gene Therapy & Bone Marrow Therapies


    Please note: A previous version of this advisory incorrectly stated that MARAC is aware of three cases of
    leukemia and other cancer-like conditions in patients who have undergone gene therapy. MARAC is only aware
    of two such cases. The below statement has been updated accordingly.
    May 15, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee is aware of two cases of leukemia or other cancer-like conditions in people who have undergone gene therapy for sickle cell disease. This is more than would be expected given the small number of individuals who have undergone gene therapy to date. It is unclear why cancer developed in these individuals. The mission of SCDAA is to promote the best interests of individuals living with SCD. Accordingly, SCDAA MARAC urges action in three directions:
    1. Informing patients, families and others about the current understanding of cancer risk as it pertains to sickle cell disease in a clear and accurate way.

    • Educate individuals with SCD who are participating in gene therapy (gene editing as well as lentiviral gene transfer) and other stem cell transplants (bone marrow therapies). Ensure that the consent forms reflect the recent findings and potential risks of leukemia and MDS.
    • Educate the greater SCD community by providing cancer risk estimates in the general population of individuals with SCD treated with other types of therapies.
    • Enlist experts in cancer genetic risk communication (e.g., NCI) and SCD stakeholders to develop education about risks of cancer in SCD.

    2. Funding for research to better understand and address cancer-related risks of gene therapy and stem cell transplant (bone marrow therapies) in sickle cell disease.

    • Understand whether sickle cell disease intrinsically involves an increased risk of cancer; and, if so, what causes the increased risk.
    • Identify people living with sickle cell disease who are at increased risk for the development of cancer as they undergo “bone marrow therapies.”
    • Identify components of “bone marrow therapies” that increase risk of cancer.
    • Bring together scientific experts from the fields of SCD, cancer, stem cell transplant and gene therapy to answer these questions.

    3. Funding for and creation of a way to collect information about cancer-related risks from all studies that involve “bone marrow therapies” for sickle cell disease globally.
    To view a full list of MARAC members and to download this statement, click here.