Category Archives: MARAC Statements

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.
 

Temporary Suspension of Clinical Trials

 
March 1, 2021 – The Sickle Cell Disease Association of America’s Medical and Research Advisory Committee (MARAC) is aware of the announcement on February 16, regarding the temporary suspension of bluebird bio clinical trials of LentiGlobin Gene Therapy for Sickle Cell Disease and the pause of all commercial use of bluebird bio European gene therapy.
Additionally, on February 22, the National Heart, Lung, and Blood Institute (NHLBI) temporarily suspended their unrelated gene therapy trial — Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for Sickle Cell Disease at Boston Children’s Hospital. The NHLBI stated that this temporary suspension was, “out of an abundance of caution” despite having no indications of harm.
On February 23, another gene therapy trial, Gene Transfer for Patients with Sickle Cell Disease, was also paused by the sponsor Aruvant.
MARAC has investigated the situation and met with bluebird bio to discuss the information available to the public. In the bluebird bio study, two patients developed blood cancer, and a third patient is under investigation for a related problem called myelodysplastic syndrome. The details of these patients are being examined by their doctors and the bluebird bio sponsors. Investigations are trying to determine whether the blood cancer can be linked to the gene therapy vector, the chemotherapy preparation for gene therapy, or damage of the host stem cell. No events occurred in the other clinical trials.
We value patient trust and patient concerns. SCDAA tries to express the voice of people living with sickle cell disease (SCD), and MARAC supports this mission with biomedical expertise. MARAC is monitoring developments and will continue to communicate findings to the SCD community. Nearly all the members of MARAC are involved in research to help those with SCD, and some who participated in developing this advisory statement are gene therapy investigators. The MARAC members with a potential conflict of interest due to their involvement in gene therapy clinical trials are in the full statement linked below.
MARAC acknowledges that there has been a history of clinical investigations that were unethical, including the infamous Tuskegee syphilis study, but this past week’s events highlight that clinical research is no longer in that era. The modern safeguards for clinical research are working. Preplanned “stopping rules” triggered a “pause” of enrollment by bluebird bio when unusual and concerning events occurred. The Data Safety and Monitoring Board for the NHLBI gene therapy study followed “out of an abundance of caution,” as did the Aruvant study. There were public announcements, and an intensive investigation is now underway to gather more information. The participants in the studies are being notified and are receiving appropriate medical care from the investigators.
Clinical research has been and continues to be the path for progress to improved SCD survival and quality of life. MARAC celebrates the decades of clinical research studies on which the progress in sickle cell care that we have today has been built — including penicillin, hydroxyurea, stroke screening and new medications.
SCDAA honors the SCD warriors who volunteer in clinical research. They have given their time so that others may benefit from new future treatments and cures. We pay tribute to all of those who have been lost to SCD, and we know many have died too young.

 

Statement from SCDAA MARAC


February 16, 2021 – We are aware of the announcement today from bluebird bio regarding the temporary suspension of the clinical trials of its LentiGlobin Gene Therapy for Sickle Cell Disease (bb1111). We have investigated the situation, and we have met with bluebird bio to discuss the information available to the public. MARAC is monitoring developments, and we will continue to communicate our findings to the community of people living with sickle cell disease (SCD).
SCDAA honors the SCD warriors who volunteer in clinical research. They have given their time so that others may benefit from new future treatments and cures.
We pay tribute to all of those whom we have lost to SCD, as we know many have died too young. We understand that clinical research is the path for progress to improved survival in SCD.
Click here to view a full list of the SCDAA Medical and Research Advisory Committee Members and download this statement.
 

MARAC Advisory Statement: COVID-19 Vaccines

Download the MARAC Alert | Download Spanish Translation | Download French Translation
December 14, 2020 – News is evolving rapidly about COVID-19 and COVID-19 vaccines. Early results from the COVID-19 vaccine trials are very promising, although the true benefits and risks will not be known until a larger number of people receive the vaccine.
The Centers for Disease Control and Prevention (CDC) lists sickle cell disease (SCD) as one of the populations vulnerable to severe COVID-19. Sickle cell disease raises the risk for serious problems with COVID-19, especially when compared to the same age in the general population.
What about side effects?
Side effects from the vaccine are possible. Reported side effects include redness and soreness at the injection (shot) site, headache, fever and body aches. These side effect symptoms go away after a few days. The second injection may have more of these side effects than the first injection, but they also went away after a few days. Two severe allergic reactions were reported and seemed to occur only in people with a history of severe life-threatening allergies.
Based on current information, MARAC recommends that people with sickle cell disease receive COVID-19 vaccination.

  • The benefits of vaccination outweigh the risks for people with SCD. Vaccination is worthwhile compared to the risks of having COVID-19 disease in people with SCD.
  • Consult with your doctor or health care team about whether your personal medical condition causes an exception to this general recommendation. Key risk conditions for the vaccines made by Pfizer and Moderna (mRNA vaccines) are a history of life-threatening allergic reactions to polyethylene glycol
    (PEG), another vaccine or other injectable medicine.
  • The fact that SCD affects the immune system should not cause a safety problem for COVID-19 vaccines.
    If a clinical trial is available, consider joining so that we can understand how vaccines or treatments work best for people with SCD.
  • Don’t relax your precautions right after getting the vaccine. You might still get infected in the few weeks following vaccination. You could still give infection to those around you. Continue to wear a mask covering your nose and mouth. Wash your hands often. Maintain physical distance. Avoid crowds, and avoid people who are ill.

Frequently Asked Questions based on CDC information as of 12-13-2020
Is a booster dose of vaccine necessary? Can I get two doses of two different kinds of vaccine?
We don’t really know. The testing was done with two doses of each vaccine so that is the recommended plan. Getting just one dose or a mixture of two vaccines might be a waste of the shot and leave you with incomplete protection. The v-safe smartphone app will remind you when it is time to get the second dose.
If I had COVID-19 disease should I still get a vaccination against COVID-19?
Probably yes, but wait until your isolation period is over. Talk to your doctor.
If I just had COVID-19 exposure, should I still get a vaccination against COVID-19?
Probably yes, but after a quarantine period. Talk to your doctor. If you live in a group setting, it might be worthwhile to protect others by getting the vaccine without waiting for quarantine to end.
How is the safety of these vaccines being tracked?
v-safe is a smartphone-based tool that uses text messaging and web surveys to provide personalized health check-ins after you receive a COVID-19 vaccination. This will allow you to quickly share any vaccine side effects with the CDC.
I have some allergies. What allergy history is worrisome?

  • Key risk conditions for the mRNA vaccines are a history of life-threatening allergic reactions to components of the vaccine, to another vaccine or injectable medicine, or allergy to polyethylene glycol (PEG). Talk to your doctor. You might need to be deferred from the mRNA vaccine, or just monitored for 30 minutes after the injection.
  • Allergic reactions that were not life-threatening and allergies to food, insects, oral medications, dust, or pollen are probably OK for the mRNA vaccines. Talk to your doctor. You should be watched for at least 15 minutes after the vaccine.
  • Talk to your doctor.
  • Sign up for v-safe from your smartphone’s browser at vsafe.cdc.gov.

Click here to view a full list of the SCDAA Medical and Research Advisory Committee Members and download this advisory.