Photo courtesy of St. Jude
Children’s Hospital
A new report suggests the current state of care for sickle cell disease (SCD) is inadequate, and improvements are needed.
The State of Sickle Cell Disease: 2016 Report outlines 4 main areas for improvement—SCD patients’ access to care, the training and education of healthcare professionals treating patients with SCD, research and clinical trials pertaining to SCD, and global health issues related to the disease.
The American Society of Hematology (ASH) published the report, with the endorsement of organizations in the SCD community.
The report includes statistics that highlight the need for improvements as well as future goals and recommended actions.
Access to care
The report states that more than 75% of adults with SCD who have frequent pain crises do not receive the recommended treatment, hydroxyurea.
One potential solution, according to the report, is to ensure that existing standard-of-care guidelines are being used. Another solution is to develop coordinated healthcare delivery models that ensure SCD patients can access quality care regardless of their age, location, and socioeconomic status.
“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 healthcare system,” said ASH President Charles S. Abrams, MD, of the University of Pennsylvania in Philadelphia.
Training and education
The report cites a national survey in which only 20.4% of family physicians said they felt comfortable treating SCD. And 69.4% of family physicians said clinical decision support tools would be useful for helping to guide their treatment decisions for SCD patients.
Therefore, the report recommends devising an “actionable plan” to educate healthcare providers about best practices in caring for SCD patients, developing clinical support tools, and encouraging medical trainees to pursue careers in SCD care, among other solutions.
“There are many unique challenges that people with SCD face,” said ASH Vice President Alexis Thompson, MD, of the Ann and Robert H. Lurie Children’s Hospital of Chicago in Illinois.
“For example, the transition from pediatric to adult care can be especially difficult, and many people struggle to find healthcare providers with comprehensive knowledge and expertise to provide proper care, especially in rural communities.”
Research and clinical trials
The report notes that hydroxyurea is the only drug approved by the US Food and Drug Administration to treat SCD. Therefore, research is needed to develop novel therapies, new drug delivery modes, and new agents that can be used in combination with hydroxyurea.
The report also highlights other areas where research is needed and recommends developing clinical trial networks to increase enrollment in trials.
Global issues
According to the report, roughly 1000 children in Africa are born with SCD every day, and more than half will die before they reach the age of 5. In addition, more than 90% of children with SCD who live in resource-poor countries do not survive to adulthood.
Therefore, the report recommends expanding newborn screening and early intervention programs, increasing SCD awareness and education, and improving access to quality care in developing regions.
Sickle Cell Disease Coalition
To address the aforementioned challenges, ASH and more than 20 other organizations launched the Sickle Cell Disease Coalition. The coalition is focused on promoting research, clinical care, education, training, and advocacy.
The aim of the coalition is to provide a platform to encourage stakeholders to work together to implement projects and activities that will ultimately help the SCD community and improve patient outcomes.
The coalition consists of leading patient advocacy groups, people with SCD and their families, researchers, clinicians, policymakers, industry stakeholders, and foundations with an interest in SCD.
