Author Archives: Emma Day

Women’s History Month: Dr. Helen M. Ranney

This #WomensHistoryMonth, we are highlighting prominent women who have made lasting contributions to the SCD community. #WomenInMedicine #WomensHistoryMonth

Meet Dr. Helen M. Ranney, physician and hematologist. Born in 1920, Dr. Ranney dedicated her work to researching blood disorders. Her groundbreaking work on sickle cell anemia included the first description of abnormal blood cell structure and genetic factors, earning her the Dr. Martin Luther King Jr. Medical Achievement Award in 1972. Dr. Ranney was also the first woman president of the Association of American Physicians. Let’s take a moment to celebrate Dr. Ranney for the work she has done on behalf of the sickle cell community!  

SCDAA to promote clinical trials

The Sickle Cell Disease Association of America, a national nonprofit membership organization that advocates for people affected by sickle cell, formed the Sickle Cell Disease C.A.R.E.S. Consortium, an initiative of pharmaceutical companies united to promote clinical trial participation and education.

“We have an opportunity and a responsibility to change the perception of clinical trials and increase the participation of sickle cell patients in clinical trials,” said Regina Hartfield, president and CEO of the Sickle Cell Disease Association of America. “Together, we will collectively leverage our resources to educate and encourage more trial participation while providing opportunities and therefore better options for the treatment of sickle cell disease.

C.A.R.E.S., which stands for Collaboration of Advocates for Research, Education and Science, consists of members Agios; Beam Therapeutics; Editas Medicine; Forma Therapeutics, a Novo Nordisk Company; Hemanext; Pfizer; Sanofi and Vertex Pharmaceuticals.

The Sickle Cell Disease C.A.R.E.S. Consortium will launch with an awareness campaign for the Sickle Cell Disease Association of America’s clinical trial finder. Available on the association’s website and mobile app, the trial finder is a centralized site to help people with sickle cell disease, their families and caregivers find clinical trials.

Clinical trials help pharmaceutical companies collect data about the effectiveness and safety of sickle cell disease treatments and can provide participants with new treatments before they become widely available. Participants can use the clinical trial finder to search for sickle cell disease-focused clinical trials by keyword, location, study type and phase, all listed in one place. The Sickle Cell Disease Association of America and Forma Therapeutics partnered to develop the trial finder in 2021.

The Sickle Cell Disease C.A.R.E.S. Consortium’s awareness campaign will use stories, videos, infographics, testimonials, public relations and marketing to spotlight the clinical trial finder as well as debunk myths about clinical research and spark conversations about trial participation.  

Women’s History Month: Dr. Angella Dorothea Ferguson

This #WomensHistoryMonth, we are highlighting prominent women who have made lasting contributions to the SCD community. #WomenInMedicine #WomensHistoryMonth

Meet Dr. Angella Dorothea Ferguson, a pediatrician and sickle cell pioneer. Born in 1925, Dr. Ferguson dedicated her life to researching sickle cell disease, an unknown condition at the time. Her groundbreaking work led to the development of a blood test for infants, which is now the standard in most states. Thanks to her research, we better understand which symptoms to look for in children and can start treating sickle cell earlier. Thanks to Dr. Ferguson for the work she has done on behalf of our community!

#BlackHistoryMonthHeroes: Carlton Haywood Jr.

SCDAA’s #BlackHistoryMonthHeroes highlights prominent Black members of our community who have made their mark on our history. Stay tuned this month to learn more about these inspiring individuals. 

#BlackHistoryMonth Meet the late Carlton Haywood Jr. – a trailblazer in the field of health equity and bioethics. Born with sickle cell disease, Carlton never let his illness hold him back. He was an all-star student and held many degrees, including a Ph.D. in Bioethics and Health Policy from the John Hopkins Bloomberg School of Public Health. His research, informed by his own experiences with SCD, earned him a competitive National Institutes of Health grant and recognition as a leader in his field. Despite the challenges he faced, Carlton’s unwavering spirit and passion for health equity and bioethics inspired many. His legacy will live on as a shining example of resilience and dedication.    

#BlackHistoryMonthHeroes: Dr. Kwaku Ohene-Frempong

SCDAA’s #BlackHistoryMonthHeroes highlights prominent Black members of our community who have made their mark on our history. Stay tuned this month to learn more about these inspiring individuals. 

#BlackHistoryMonth Meet the late Kwaku Ohene-Frempong, M.D., – a renowned pediatric hematologist-oncologist and expert in sickle cell disease. Dr. Ohene-Frempong grew up in Ghana, where he excelled as a track-and-field athlete. He completed medical school and a residency in pediatrics, followed by a fellowship in pediatric hematology-oncology in the U.S. He dedicated his career to serving patients with sickle cell disease at Tulane University School of Medicine and the Children’s Hospital of Philadelphia. Upon retiring, he returned to Ghana to establish public health initiatives for SCD screening and treatment. Dr. Ohene-Frempong’s passion for improving the lives of those affected by sickle cell disease has left a lasting impact. His dedication to improving access to care, education and public health initiatives for SCD has inspired many to continue his work and carry on his legacy.   

#BlackHistoryMonthHeroes: Hertz Nazaire

SCDAA’s #BlackHistoryMonthHeroes highlights prominent Black members of our community who have made their mark on our history. Stay tuned this month to learn more about these inspiring individuals. 
Meet the late Hertz Nazaire – a talented artist and author who used his brush to spread awareness about sickle cell disease. At just 9 years old, Hertz began honing his skills in NYC and later perfected his craft at The Art Institute of Fort Lauderdale and the University of Bridgeport. Known for his advocacy work and his “Finding Your Colors” adult coloring book series, Hertz was dedicated to using his art as therapy for those impacted by stress. Hertz lost his battle to sickle cell in late 2021, but his commitment to SCD advocacy and his impact on the art world will not be forgotten. 

MARAC Statement: Penicillin Shortage

Penicillin VK solution is suffering from intermittent supply shortages. This can affect children with sickle cell disease. Penicillin VK in liquid form is prescribed for babies and young children with sickle cell disease who are unable to swallow pills as standard care, twice a day, starting as early as 6-8 weeks of age. Children less than 3 years of age should receive oral penicillin 125 mg twice daily, and the dose should be increased to 250 mg twice daily for children older than 3 years.

WHY DO CHILDREN WITH SICKLE CELL DISEASE NEED PENICILLIN?

The strategy of using an antibiotic daily to prevent infection in people with lower immune defense is called prophylaxis. Without medical care, young children with sickle cell disease have extremely high risk of hospitalizations and death from infection. Strong scientific evidence shows that penicillin twice a day helps young children with sickle cell disease avoid an overwhelming bacterial infection, especially the bacteria called pneumococcus. Newborn screening diagnosis of sickle cell disease leading to penicillin prophylaxis are the two fundamental starting points of sickle cell comprehensive care.

WHAT CAN THE HEALTH CARE TEAM DO ABOUT PENICILLIN SHORTAGES?

  • Urge the manufacturers and distributors to improve the supply of penicillin so that we do not lose out on this effective, evidence-based standard of care.
  • Prioritize sickle cell disease as an immune-compromised condition that has specific guidelines stating the necessity for penicillin prophylaxis.

WHAT DO FAMILIES DO ABOUT PENICILLIN SHORTAGES?

  • Alternatives to penicillin liquid exist, although all are suboptimal. Discuss with your doctor:
    • Penicillin VK tablets, crushed (Miss out on residual uncrushed portions, takes time twice a day while the family is already busy with parenting a young child)
    • Penicillin G injection, once a month (Cannot be administered at home/requires visiting a medical facility, needle stick)
    • Other alternative antibiotics: Amoxicillin 20mg/kg/day or Erythromycin (Broader effects on normal bacteria than penicillin, supply shortages might also be a problem)
  • Make sure your child has all immunizations as recommended for sickle cell disease. SCDAA MARAC strongly encourages full immunization, especially pneumococcal vaccines PCV and PPSV-23.
  • Fever needs prompt attention in individuals with SCD. Go to the Emergency Department (not urgent care) for blood counts, blood cultures and injection of a strong antibiotic like ceftriaxone.

HOW STRONG ARE THE RECOMMENDATIONS FOR PENICILLIN PROPHYLAXIS?

In the United States, penicillin prophylaxis is written into public health policy, National Institutes of Health guidelines and even state laws. Brazil, Canada, France, Italy, Jamaica, Ghana, Nigeria, Tanzania, Uganda, the United Kingdom and other countries have penicillin prophylaxis in their national guidelines for sickle cell disease.

IS IT SAFE TO GIVE ANTIBIOTICS DAILY?

Penicillin prophylaxis has an excellent track record and has been used to provide safe care to thousands of children for over 30 years. Studies have showed that giving penicillin prophylaxis continuously does not cause harmful bacteria to become antibiotic resistant. On-and-off periods of penicillin prophylaxis could cause antibiotic resistant bacteria to emerge. Penicillin was chosen for prophylaxis because it is focused on the pneumococcal bacteria that can cause problems in sickle cell disease.

IS PENICILLIN EXPENSIVE?

Penicillin is one of the oldest and most affordable antibiotics. Business analysts say that the low prices of penicillin make few companies motivated to manufacture penicillin.

For references and highlight points, download the full statement.

Sickle Cell is Not a Joke

The Sickle Cell Disease Association of America, Inc., joins the Foundation For Sickle Cell Disease Research (FSCDR) in condemning the use of sickle cell disease (SCD) as a punchline on the HBO Max show Velma. For the over 100,000 Americans impacted by sickle cell and their families, this disease is anything but a laughing matter. Stereotypes and misinformation reinforced by media clips such as this have real-life consequences. Sickle cell patients struggle to be taken seriously and receive proper care, even when they present with life-threatening symptoms in the emergency room. As we work to change the perception of sickle cell and increase education surrounding this condition, insensitive and inappropriate jokes like these work against progress and contribute to the spread of misinformation. We must do better and encourage people to treat rare diseases with the respect they are due.

Click here to read the full statement from FSCDR. 

#SickleCellIsNotAJoke  

CMO Speaks: Fertility Care and SCD

CMO Speaks is a blog featuring the voices of SCDAA’s clinical leadership team. The below article was written by Dr. Lewis Hsu, SCDAA chief medical officer, with input from Dr. Lydia Pecker.

NPR recently produced this segment with sickle cell warrior and advocate Teonna Woolford on fertility care and SCD. This is an underdiscussed topic and an important aspect of sickle cell care. We congratulate Teonna and the Sickle Cell Reproductive Health Education Directive (SC RED) for getting this message out, and we are grateful for the attention from NPR. In light of this discussion, we would like to reinforce a few points about reproductive health and SCD:

  1. Even without a transplant or gene therapy, sickle cell disease can damage patients’ bodies in ways that can affect their ability to have children. We agree with the statements by Drs. Lydia Pecker and Leena Nahata in this NPR report.
  2. Currently, insurance coverage for fertility preservation is highly variable and differs from state to state. We agree with statements in this NPR report by Dr. Irene Su about the vagueness of these policies. This is a sickle cell advocacy opportunity to get coverage in more states. SCDAA praises the efforts of SC RED and the Alliance for Fertility Preservation as allies in fighting for coverage. However, just as the NPR report described for intrauterine insemination, there are high costs and many other barriers of access to these procedures. Shared decision-making should be the model for information, choices and policies.
  3. SCDAA agrees wholeheartedly with the statements in this NPR report regarding funding disparities and the lack of information dissemination about sickle cell disease. The NASEM 2020 Report has much more information about action steps to take.

Go deeper – What is not brought out in this NPR report

  1. Sickle cell disease is an inherited condition. Another aspect of reproductive decision-making for individuals with SCD is understanding the risks of having a child who also has sickle cell disease (more about family planning in this infographic from SC RED). Having a child with sickle cell disease is not a mistake, but it should not be a surprise for lack of information. Tests are available to know whether the mate has the sickle gene or another hemoglobin variant. Tests are available for prenatal diagnosis. Fertility centers also can do preimplantation genetic diagnosis and embryo selection to choose an embryo without sickle cell disease.
  2. For individuals with sickle cell, choosing to have children also means gathering a support system so that they can be a caregiver for a child when they themselves have sickle cell problems. (Currently, the mother of one of my patients cannot be at the bedside for her child with sickle acute chest syndrome because she is hospitalized herself for sickle cell pain.)
  3. Making reproductive decisions would be best done in the format of “shared decision-making.” These are complicated decisions that depend on individual situations and individual values. Individuals with sickle cell disease should try to be well-informed. Try to explain your values and reasons to your health care provider(s), and ask questions until you have a solid understanding. Please be aware that some things are not certain in medicine and in reproduction and can only be described as chances and risks.

Helpful links and resources

Lewis Hsu, MD, PhD, is a pediatric hematologist who serves as director of the Sickle Cell Center and professor of pediatrics for the University of Illinois at Chicago. He has conducted sickle cell research, published over 50 peer-reviewed papers and co-authored “Hope and Destiny: The Patient and Parent’s Guide to Sickle Cell Disease and Sickle Cell Trait.” He currently serves as the SCDAA Chief Medical Officer.