Report Calls for Overhaul of Sickle Cell Treatment
/By Pat Anson, PNN Editor
A new report by the National Academies of Sciences, Engineering, and Medicine is calling for major changes in the way sickle cell disease is treated in the United States, including an end to the discrimination and stigma that many sickle cell patients face while navigating the healthcare system.
About 100,000 Americans live with sickle cell disease (SCD), a genetic disorder that mainly affects people of African or Hispanic descent. SCD causes red blood cells to form in a crescent or sickle shape, which can create blockages in blood vessels that prevent the normal delivery of oxygen throughout the body. As a result, sickle cell patients often suffer from chronic pain, anemia, infections, strokes and organ failure.
Compared to other chronic illnesses, SCD has received little attention from the healthcare community, resulting in a lag in the development of new treatments. Until 2018, only one drug was approved by the Food and Drug Administration to treat sickle cell patients. Bone marrow and stem cell transplants are currently the only curative therapies for SCD.
“People with sickle cell disease show incredible resilience. They often have to become their own advocate to get the treatment and services they deserve — whether in the health care system, at school, at work, or in the community,” said Marie McCormick, MD, a pediatrician and professor at the Harvard School of Public Health, who chaired the committee that wrote the report.
Addressing Sickle Cell Disease: A Strategic Plan and Blueprint for Action calls for the National Institutes of Health to designate SCD as a “health disparity” to incentivize more research, and for the Department of Health and Human Services to fund efforts to identify and mitigate disparities in health outcomes for sickle cell patients.
Lack of Compatible Donors
Although stem cell and bone marrow transplants are the only cures for SCD, it’s often difficult to find compatible donors, especially for children who can benefit the most from treatment.
“Unfortunately, less than one in five individuals with sickle cell disease have compatible donors. Those children fortunate to have a fully matched sibling as a donor enjoy very high success rates with very few complications after transplantation,” said Enrico Novelli, MD, an Associate Professor of Medicine and Director of the Adult Sickle Cell Disease Program at University of Pittsburgh Medical Center.
“The success of the procedure decreases as people get older. For many adults living with sickle cell disease, stem cell transplantation is not an option, either because they are too ill to undergo the procedure, or because of a lack of donor,” Novelli said in an email to PNN.
“And for those who are cured by transplantation and no longer have sickle cell disease, some challenges remain; for instance, the treatments to prepare the recipient for the donor stem cells may cause infertility, and years of exposure to the disease may leave a mark in terms of organ dysfunction and chronic pain. Thus, it may take a while for some patients to overcome their sickle cell-related chronic pain after a successful stem cell transplant.”
The report recommends that the Centers for Medicare & Medicaid Services and state Medicaid programs, as the lead insurers for most sickle cell patients, explore new payment methods to encourage and pay for coordinated care delivered by certified SCD centers. Insurance coverage is also needed to finance the cost of experimental new treatments, such as gene therapy.
The report calls for more diversity and “cultural competency training” for SCD care providers, as well as better training in managing acute and chronic pain. It also recommends that federal health agencies work with states to standardize newborn screening for SCD and to develop a national registry of people living with sickle cell disease.
