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The Model will launch in January 2025, and all 50 states may choose to begin participation anytime between January 2025 and January 2026. SCDAA looks forward to working with our community-based organizations and other stakeholders to advocate for state enrollment. The CGT Access Model will provide crucial support to patients where available, and we encourage full participation across the country.

Read the full statement from the Centers for Medicare and Medicaid Services.

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Dr. Edward Donnell Ivy (left) is director of outreach and education at The Possibilities Clinic in Toronto, Ontario, Canada. He was a medical officer at the Health Resources and Services Administration and served the National Institutes of Health in Washington, D.C., where he worked on sickle cell clinical practice guidelines and on projects involving cardiovascular disease and diabetes. As someone living with sickle cell disease, Ivy has made it his life’s work to ensure patients have access to the resources they need. 

Dr. Crawford Strunk (center) is a pediatric hematologist with experience and expertise in sickle cell care. He serves on the Sickle Cell Disease Association of America Inc.’s Medical and Research Advisory Committee. Strunk is a staff physician and co-director of the Lifespan Sickle Cell Program at the Cleveland Clinic. He directed the pediatric sickle cell center and co-directed the adult sickle cell center at Toledo Children’s Hospital. He earned his doctorate at the State University of New York Upstate Medical University and graduated from Colby College. 

Adeyinka O. Ogunlegan (right) serves as vice president of government affairs and policy at EducationSuperHighway, a national nonprofit organization with the mission to close the broadband internet affordability gap. She served as chair of the Prince George’s Chamber of Commerce’s Legislative Action Committee as well as board president of The Arc Prince George’s County. Among her honors, Ogunlegan received The Maryland Daily Record’s VIP List and Leading Women awards and was recognized as a Prince George’s County Forty Under 40 honoree. She earned a law degree from Howard University and a bachelor’s degree from Georgetown University. 

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SCDAA Medical and Research Advisory Committee (MARAC) Statement: Pfizer’s Voxelotor (Oxbryta®) Withdrawal

9/27/24

What is the news?

Pfizer announced the withdrawal of voxelotor (Oxbryta®) from national and global markets on September 25, 2024.  Clinical research was also stopped. “Pfizer’s decision is based on the totality of clinical data that now indicate the overall benefit of OXBRYTA no longer outweighs the risk in the approved sickle cell patient population. The data suggest an imbalance in vaso-occlusive crises and fatal events, which require further assessment.” [1].

What is the reason?

The Pfizer statement mentions that worrisome new information came from three reports that link voxelotor with more pain and deaths:

1. A clinical research study of children with sickle cell disease and with higher risk of stroke (GBT440-032) had eight deaths in the voxelotor group compared to two deaths in the group without voxelotor. The study recruited 236 children (2y-15y) from Egypt, Ghana, Kenya, Nigeria, Oman, Saudi Arabia, USA and the United Kingdom.
2. Another clinical research study of adolescents and adults with sickle cell disease and leg ulcers (GBT440-042) had eight deaths on voxelotor. Eighty-eight patients at least 12 years of age were enrolled in Brazil, Kenya and Nigeria.
3. Monitoring reports from people taking voxelotor as a prescribed medication, not on a clinical research study. No numbers were listed.

Following standard rules when new risks are found, Pfizer voluntarily withdrew voxelotor from use worldwide, while further investigation is conducted. Pfizer also reports that they are halting manufacturing production and clinical research.

What if I have been doing well on voxelotor (Oxbryta®)?

The FDA has received questions about whether to allow some individuals living with sickle cell disease to continue voxelotor on a “compassionate use” basis. However, for now we cannot assume that “compassionate use” will be allowed.

Do I just throw away the voxelotor?

For people with sickle cell disease who have been on voxelotor and doing well, MARAC does not have evidence on what will happen when you stop taking the medication. There is a report that going from full-dose voxelotor to completely stopping taking the drug led to intense hemolysis (breakdown of red blood cells) and severe sickle cell problems within three days that injured the kidneys and other organs and required hospital care. Thus, many doctors are suggesting that people taper off voxelotor over a period of about two weeks. However, tapering is optional and not based on strong clinical evidence. We strongly urge individuals to discuss their options and next steps with their sickle cell health care professional.

What if I have unusual problems as I discontinue voxelotor?

Contact your sickle cell health care provider. Voxelotor acts by slowing down the breakage of red blood cells (hemolysis) and blocking the red cells from changing to the sickle shape. A possible problem is that if the hemolysis suddenly increases it may damage the kidneys. Your eyes will probably become more yellow (jaundiced), and your urine will be darker yellow-orange. Tell your doctor if you have brown urine (the color of cola). You will probably need to come in for lab tests and treatment. Immediately seek medical attention if you stop making urine even when you are drinking a lot of water. Comprehensive sickle cell centers and sickle cell health care providers are collecting reports from patients about any new problems experienced when they come off voxelotor.

What about my other sickle cell medicines?

This news is only about voxelotor.  Continue taking other medications as prescribed.

What are the alternatives to voxelotor?

In the USA, sickle cell disease severity can be reduced by three other medications approved by FDA:

• hydroxyurea (Droxia®, Siklos®),
• glutamine (Endari®), and
• crizanlizumab (Adakveo®)

Blood transfusions are used for selected problems in sickle cell disease, especially to prevent stroke.

• “top-off transfusions” or simple transfusions
• “exchange transfusions” or erythrocytapheresis

Hematopoietic stem cell transplant (also known as bone marrow transplant) is a cure that requires an:

• HLA-identical (fully-matched) sibling donor, or
• haploidentical (half-matched) related donor, or
• unrelated HLA-matched donor

Two gene therapy approaches can potentially cure sickle cell disease without having to find a donor.

• Casgevy^TM (CRISPR/Vertex) or
• Lyfgenia^TM (bluebird bio)

MARAC recommends making an appointment to discuss your sickle cell treatment plan with your sickle cell health care provider.

Is this the first time a medication has been withdrawn?

No. Other situations of medication withdrawal from the market have occurred.

• 2004: Merck withdrew rofecoxib (Vioxx) as an arthritis drug.
• 1997: FDA ordered Wyeth to remove the weight-loss medications fenfluramine (Pondimin) and a related drug, dexfenfluramine (Redux) from the market.
• 1961: Chemie withdrew thalidomide as an antinausea drug. 2006 thalidomide was approved for the treatment of plasma cell myeloma.

When will there be more updates?

We do not know. MARAC is following the news as it unfolds. The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) regulations control some of the timing for release of information. SCDAA MARAC is continuing to monitor the situation and will update the community as soon as new information is released.

Additional Information — 9/30/24 Update

What details are publicly available about the deaths?
A document posted online by the European Medicines Agency has the most details that MARAC was able to find. “Notification to the CHMP/EMA Secretariat of a referral under Article 20 of Regulation (EC) 726/2004” electronically signed July 30, 2024, accessed Sept. 29, 2024. https://www.ema.europa.eu/en/documents/referral/oxbryta-article-20-procedure-notification_en.pdf The rules of EMA and FDA control release of information.

• Of the 8 deaths in the GBT440-032 randomized study of children with abnormal stroke risk: “Most of the fatal cases in the voxelotor group describe incidence of infection, including 3/8 who developed fatal malaria and 2/8 patients with sepsis.”
• Of the 8 deaths in the open-label GBT440-042 of voxelotor for leg ulcers: “In 4 cases, malaria was identified either the cause or contributing factor.”
• “The investigator and sponsor considered that none of the fatal cases were related to voxelotor in these studies.”

What information is still missing?
Quoting from the same EMA document from July 30, 2024:

• Some of the “case narratives” from GBT440-032 and GBT440-042 “are still not available, … and overall information provided to date is limited. However, given the fact that concerns due to possible immunosuppressive effects of voxelotor were raised at the time of the MA (with immunosuppressive effects observed in animal studies and decrease in WBC in clinical studies), and the study population in those studies partially overlap with the intended population based on the authorized indication, the findings from these emerging safety data need to be further reviewed, taking into account all available data, to determine whether there is an impact on the benefit-risk balance of Oxbryta in the authorized indication.”
• It is unclear how these deaths compare with the known vulnerability to infection and early death of sickle cell disease without treatment.

References

Pfizer statement, 9/25/24: https://www.pfizer.com/news/press-release/press-release-detail/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease

Pfizer letter to Health Care Providers, 9/26/24: https://webfiles.pfizer.com/dear-hcp-letter-oxbryta-us-final-092524?cmp=US-21234&campaign=US-21234&identitytype=account&tpn=1532773&LNK=GL_BD3-C2&mkt_tok=MTk1LVVMQy0yMDAAAAGV1F26wnPvgN6fKRyyOgU-kt_3Pa-xaL9I_wa6yP1ROToTo85YnZaSQ98g2cAj9j-cf9xvsubH4Rvs50AWKcAMrA4qw1nLMAsCEbu8-c6PeXuPhg

European Medicines Agency, July 2024: https://www.ema.europa.eu/en/medicines/human/referrals/oxbryta

FDA statement, 9/26/24: https://www.fda.gov/drugs/drug-safety-and-availability/fda-alerting-patients-and-health-care-professionals-about-voluntary-withdrawal-oxbryta-market-due

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Alabama

• Sept. 22: Annual Sickle Cell Walk-A-Thon | Dothan, AL
  Hosted by the Southeast Alabama Sickle Cell Association, Inc.

California

• Sept. 18-21: 16th Annual SCD Educational Summit | Los Angeles, CA or virtual
  Hosted by the Cayenne Wellness Center

Connecticut

• Sept. 21: Walk Run Bike | New Haven, CT
  Hosted by the Sickle Cell Disease Association of America, Connecticut

Delaware

• Sept. 7: Transformative Curative Gene Therapy Transplant for Sickle Cell Anemia | New Castle, DE
  Hosted by Tova Community Health

Florida

• Sept. 14: Celebrating Sickle Cell Awareness Month | Daytona Beach, FL
  Hosted by the Sickle Cell Medical Advocacy, Inc.
• Sept. 21: Sickle Cell Dinner & Musical Extravaganza | St. Petersburg, FL
  Hosted by SCDAA – St. Petersburg Chapter

Georgia

• Sept. 14: Annual Sickle Cell Race/Walk – Concert – Candlelight Vigil | Atlanta, GA
  Hosted by the Sickle Cell Foundation of Georgia
• Sept. 21: Sickle Cell Family Skate Night | Atlanta, GA
  Hosted by the Sickle Cell Foundation of Georgia

Illinois

• Sept. 28: Walk, Jog, Bike 5K/1K | Chicago, IL
  Hosted by the Sickle Cell Disease Association of Illinois

Kansas

• Sept. 14: 14th Annual Fun Walk | Kansas City, KS
  Hosted by the Uriel Owens Sickle Cell Disease Association of the Midwest

Louisiana

• Sept. 26: Tailgating for Sickle Cell | Alexandria, LA
  Hosted by the Sickle Cell Anemia Resource Foundation

Maryland

• Sept. 14: MSCDA Picnic | Baltimore, MD
  Hosted by the Maryland Sickle Cell Disease Association, Inc.

Michigan

• Sept. 14: The Annual Sickle Cell Matters Awareness Walk | Detroit, MI
  Hosted by the Sickle Cell Disease Association of America, Michigan Chapter

Minnesota

• Sept. 7: Sickle Cell-ebration of Hope | Brooklyn Park, MN
  Hosted by the Sickle Cell Foundation of Minnesota

Missouri

• Sept. 14: Sickle Cell Stroll | St. Louis, MO
  Hosted by the Sickle Cell Association of St. Louis

New Jersey

• Sept. 21: Erick Bradley Sickle Cell Disease Awareness Walk | Atlantic City, NJ
  Hosted by the Sickle Cell Association of New Jersey
• Sept. 28: 5th Annual 2K Color Fun Walk | Trenton, NJ
  Hosted by the Sickle Cell Association of New Jersey

Oregon

• Sept. 14: 12th Annual Sickle Cell Walk for Awareness & Community Marketplace | Portland, OR
  Hosted by the Sickle Cell Anemia Foundation of Oregon
• Sept. 26: 8th Annual Sickle Cell Live! Awareness Dinner | Portland, OR
  Hosted by the Sickle Cell Anemia Foundation of Oregon

South Carolina

• Sept. 3: Every Drop Counts Blood Drive | Columbia, SC
  Hosted by the James R. Clark Memorial Sickle Cell Foundation
• Sept. 14: 18th Annual Sickle Cell Walk | Columbia, SC
  Hosted by the James R. Clark Memorial Sickle Cell Foundation
• Sept. 28: Free Family Fun Day | Lancaster, SC
  Hosted by the James R. Clark Memorial Sickle Cell Foundation

Texas

• Sept. 21: Sickle Cell Houston’s Amazing Walk 5K/1K | Houston, TX
  Hosted by the Sickle Cell Assocation of Houston

Virginia

• Sept. 28: Walk for Sickle Cell | Norfolk, VA
  Hosted by the Sickle Cell Association, Inc.

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CLICK HERE TO LEARN MORE AND MAKE A NOMINATION

Do you know a community health worker who exemplifies excellence and commitment to the sickle cell community? Nominate them for a SCDAA P.O.W.E.R CHW Award! The deadline to nominate someone is Friday, August 23. Awards will be granted during the Annual National Convention in October.

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MARAC Statement: Parvovirus B19, Fever and Urgent Care

Aug. 13, 2024 – More infections with parvovirus B19, also known as “fifth disease” or “slapped-cheek disease,” are showing up in 2024 [1,2].

WHY IS THIS IMPORTANT FOR SICKLE CELL DISEASE?

• Parvovirus B19 causes a special problem in sickle cell disease called aplastic crisis [3,4,5], and infects the bone marrow and stops production red blood cells for about 10 days. Reticulocyte counts (number of young red blood cells) drop to zero. Hemoglobin can drop to dangerously low levels in people with sickle cell disease and other conditions with short- lived red blood cells (hemolytic anemias). A red blood cell transfusion is often needed.
• Parvovirus can cause other SCD problems like acute chest syndrome, enlarged spleen and damage to many parts of the body. These problems can also require hospital treatment.

HOW DO YOU DETECT PARVOVIRUS B19 INFECTION?

• Checking for parvovirus B19 infection requires blood tests: reticulocyte count and parvovirus testing by PCR.
• Symptoms of parvovirus infection overlap with the symptoms of many other respiratory infections: fever, headache, respiratory symptoms, fatigue.
• A facial rash that looks like “slapped cheeks” is seen in some children, but not always. Pregnant women usually do not get this rash.

HOW DOES PARVOVIRUS B19 SPREAD?

• Parvovirus spreads in the air from one child to another.
• Avoiding people with respiratory symptoms, wearing masks and washing hands can help to reduce the spread of parvovirus B19.
• Most adults have had parvovirus B19. The previous infection protects them from getting it again.
• Because parvovirus B19 and COVID-19 are both respiratory viruses, their spread can be prevented in similar ways. Widespread use of COVID-19 precautions likely lowered the number of people with parvovirus exposure in the past few years. However, the larger vulnerable group who were not exposed to parvovirus B19 can now catch it.

WHO IS VULNERABLE TO PARVOVIRUS B19?

• Children.
• People with sickle cell disease, thalassemia and other hemolytic anemias. They might need blood transfusion.
• People with severe immunocompromise (such as AIDS, cancer chemotherapy or transplant immunosuppression). They might need to have antibody treatment to clear the parvovirus infection.
• Pregnant women. Catching parvovirus B19 during pregnancy means a higher risk of miscarriage. Pregnant women should avoid children with parvovirus B19.

CAN A CHILD WITH SCD BE EVALUATED FOR PARVOVIRUS IN AN URGENT CARE OR PRIMARY CARE CLINIC?
Although they seem convenient, most urgent care centers and primary care clinics cannot do immediate (STAT) reticulocyte count. Emergency departments have the capacity to perform these steps in the appropriate medical evaluation of a child with SCD and fever above 101.3 F:

• Blood counts including reticulocyte count, with results within an hour or two (STAT)
• Blood culture
• Physical exam including spleen
• Pulse oximetry, blood pressure and other vital signs
• Antibiotic injection (usually ceftriaxone) to clear bacteria
• Possible chest X-ray to rule out acute chest syndrome

DON’T DOGS GET PARVOVIRUS?

Dogs get other kinds of parvovirus (CPV-1 or CPV-2), not parvovirus B19. You cannot get the infection from your pet, and you cannot infect your pet.

RECOMMENDATIONS:

1. Children with SCD and fever should be evaluated promptly in emergency departments, and families should clearly state at the intake desk (triage) that the child has sickle cell disease immunocompromise with fever.
2. Medical evaluation of a child with SCD and fever should include CBC and reticulocyte count, with STAT results. Low reticulocyte count (absolute reticulocytes below 10,000 per microliter) should raise concern about aplastic crisis from parvovirus B19, and the child should not be discharged home without a plan for possible blood transfusion.
3. Families of children with SCD and fever should not go to urgent care centers and clinics that lack the capability to do STAT labs. These types of centers and clinics should not delay sending a child with SCD and fever to a medical facility that can provide appropriate care, usually an emergency department.

REFERENCES:

1. Increase in human parvovirus activity in the United States. CDC Health Alert Network CDCHAN-00514. August 13, 2024 https://emergency.cdc.gov/han/2024/han00514.asp
2. Risks posed by reported increased circulation of human parvovirus B19 in the European Union / EEA. June 5, 2024 https://www.ecdc.europa.eu/sites/default/files/documents/Risks%20posed%20by%20reported%20increased%20circulation%20of%20human%20parvovirus%20B19%20FINAL.pdf
3. Sickle cell aplastic crisis. Texas Department of Health https://www.dshs.texas.gov/newborn-screening-program/sickle-cell-disease/more-about-sickle-cell/aplastic-crisis
4. Sickle Cell aplastic crisis. Childrens Healthcare of Atlanta. https://www.choa.org/-/media/Files/Childrens/teaching-sheets/sickle-cell—aplastic-crisis.pdf
5. Sickle cell aplastic crisis. Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/24557-aplastic-crisis

Download and print this statement. 

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READ MORE

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The Centers for Medicare & Medicaid Services (CMS) is promoting new resources to remind individuals about their rights in the emergency room. Under the Emergency Medical Treatment and Labor Act, better known as EMTALA, all hospitals that participate in the Medicare program are required to provide medical screening exams for individuals arriving at hospital emergency departments to determine if the person has an emergency medical condition. If an emergency medical condition is confirmed, hospitals must provide stabilizing medical treatment (or, if unable to stabilize the person within its capability), to appropriately transfer the patient.

SCDAA has heard countless stories of SCD warriors receiving inadequate care or even no care at all in emergency departments across the country. Under EMTALA, hospital emergency departments are required to:

1. Give you an appropriate medical screening.
2. Treat you until your condition is stable if you are found to have an emergency medical condition.
3. Transfer you if necessary.

Learn more here.

If you believe your EMTALA rights have been violated, CMS now allows you to file a complaint online. You can file anonymously or provide contact information. The federal government and the states will determine if an investigation is needed. If so, the federal government will review the case and determine if a hospital violated EMTALA – the process can take weeks or months depending on the complaint. Filing an EMTALA complaint is not a legal action. You are helping to make sure hospitals stabilize emergency medical conditions and follow the law.

If a hospital or a physician is found to have violated EMTALA, the federal government can impose a civil monetary penalty and/or may exclude physicians from participating in the Medicare and Medicaid programs.

LINKS & RESOURCES

For Warriors

• SCDAA & MedicAlert Pilot Program
• SCDAA Michigan Patient Resources
• Printable SAFER Card
• The Joint Commission – Speak Up

For Providers

• ACEP Point-of-Care Tools
• ASH Pocket Guides
• Opiate Dosing Calculator for People with Sickle Cell Disease
• Society of Hospital Medicine

The post SCDAA Statement: Know Your Rights in the ER appeared first on Sickle Cell Disease Association of America Inc..

Additional Reading:

• https://www.ama-assn.org/press-center/press-releases/statement-improving-health-through-dei#:~:text=%E2%80%9COur%20efforts%20to%20promote%20diversity,of%20our%20patients%20and%20communities
• https://www.nationalacademies.org/news/2020/09/improving-health-outcomes-for-sickle-cell-disease-care-requires-comprehensive-team-based-care-new-payment-models-and-addressing-institutional-racism-in-health
• https://nap.nationalacademies.org/resource/25632/Sickle%20Cell_Overview%20of%20Recommendations%20and%20Strategic%20Plan.pdf
• https://www.chiefhealthcareexecutive.com/view/republicans-push-to-ban-dei-in-medical-schools-bills-and-laws
• https://www.aamc.org/news/press-releases/aamc-statement-medical-school-curriculum
• https://www.nmanet.org/news/667985/The-NMA-Advocates-for-DEI-in-Medicine.htm?fbclid=IwAR07GBRDNjVBJLU67Yj1GQ5emdDe0OPbdsATvoiHzfKGxA2A_BxZJrBejks

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