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Call to Action From ASH Draws Attention to Sickle Cell Disease


A report released by a group of sickle cell disease (SCD) stakeholders, including the American Society of Hematology, aims to identify unmet medical needs for those diagnosed with SCD to improve their quality of life.

With more than a 100,000 individuals living with sickle cell disease (SCD) in the United States, there is an urgent need to raise awareness and improve patient access to the growing number of treatment options for the disease. With this in mind, the American Society of Hematology (ASH), in partnership with over 20 other organizations (including the CDC and the FDA), has formed the Sickle Cell Disease Coalition (SCDC). A report released by the SCDC today aims to identify unmet medical needs for those diagnosed with SCD to improve their quality of life (QOL).

The report, State of Sickle Cell Disease: 2016 Report, is focused on 4 areas:

  • Access to care
  • Training and professional education
  • Research and clinical trials
  • Global health

Triggered by a point mutation in the gene encoding hemoglobin, the resulting abnormal protein causes a rigid cell structure that can lead to lifelong health problems, including anemia, infections, stroke, tissue damage, organ failure, intense painful episodes, and even premature death. SCD—associated pain can prove debilitating in both children and adults, preventing them from leading a normal life.

“Not only are individuals with SCD burdened by the pain and disability that comes with a chronic condition, but they also have very few accessible treatment options due to our fragmented health care system,” said ASH President Charles S. Abrams, MD, of the University of Pennsylvania in an associated press release. “This status quo is unacceptable, and we are setting out to change it. ASH is proud to make a difference for people battling this disease worldwide by teaming up with partners from around the globe to launch the Sickle Cell Disease Coalition.”

According to the report, significant discrepancies exist in the diagnosis and care of these patients. For example, one study found that more than 75% of adults with SCD who experience frequent pain fail to get the recommended, and only FDA-approved treatment of hydroxyurea. Another health services report found that nearly 31% of diagnosed cases of SCD in newborns were lost to long-term follow-up.

Adherence and treatment issues due to provider incompetence are commonly reported in children, and several children diagnosed with SCD lack necessary supportive care, the report shows. The condition is expensive to treat, with lifetime healthcare costs for a person with SCD who reaches 45 years of age estimated at $1 million.

With this evidence, SCDC has devised the following strategy to find solutions to the 4 key focus areas:

  • Access to care: Creating a healthcare delivery model based on evidence-based guidelines and coordinated care to prevent discrepancy based on age, location, and socioeconomic status.
  • Training and education: Educating the existing physician force to care for and treat SCD complications. Also encourage medical trainees to specialize in SCD.
  • Research and clinical trials: Formulate strategies to develop new drugs and also increase use of existing treatments.
  • Global issues: On the global front, efforts to improve early screening and diagnosis in newborns (symptoms develop by 5 to 6 months of age), increase SCD awareness and education, and better access to quality care.

According to the press release, ASH is developing targeted guidelines for hematologists, primary care providers, and hospitalists so they can accurately recognize and respond to symptoms of SCD. Additionally, several different projects have been launched in the United States to improve care coordination among stakeholders. A few of these include:

  • A collaboration between the American College of Emergency Physicians and the Emergency Department Sickle Cell Care Coalition to improve the emergency care of patients with SCD.
  • A campaign by the FDA to improve minority participation in all clinical trials, including those for SCD. SCD is diagnosed in 1 of every 365 African American newborns.
  • The National Marrow Donor Program influenced CMS to expand national coverage for hematopoietic stem cell transplantation for 3 conditions, including SCD.
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