Major strides made, but challenges remain
This is a remarkably exciting time to talk about sickle cell,” said Alexis Thompson, M.D., a sickle cell specialist in Chicago, during the National Medical Association Colloquium, which brought together prominent medical experts to address various topics, including violence and its impact on health and health policy, a campaign to prescribe exercise as a prevention vehicle for heart disease and stroke, equity in cancer care, the Zika virus pandemic, the impact of breast cancer on black women, breastfeeding as a disease prevention practice and diversity in medical education.
We’ve made significant strides in sickle cell care, according to Dr. Thompson, including universal screening across the United States to identify infants with the most common inherited blood disorder in this country. We have also identified early interventions that can save lives and prevent complications.
The lifespan of children with sickle cell, once drastically shortened, is now longer. Where once children with this disease didn’t survive childhood or adolescence, “95 percent of children born with the disease today will survive into adulthood,” Dr. Thompson said.
Even with all these advances, however, there are still challenges. We have limited data on whether children are benefitting fully from early interventions. This is because there have been no large-scale studies done since 1994, Dr. Thompson said. She stressed the importance of changing this by expanding long-term newborn screening follow-up.
Another challenge is limited treatment options. Hydroxyurea, which received Food and Drug Administration approval to treat sickle cell disease in 1998, reduces the number of painful crises caused by the disease and lowers the need for blood transfusions. But it is the only disease-modifying therapy for the sickle cell. “It’s absolutely shameful that a disease that is so prominent has only one treatment,” Dr. Thompson said. And even that treatment is underutilized.
A panel of experts issued new guidelines for managing sickle cell disease in 2014. The guidelines stress the use of hydroxyurea and transfusions for many with the genetic disorder.
“This is a major step forward to try to put together all of the evidence and try to highlight what is most important,” Barbara Yawn, M.D., professor of family and community health at Olmsted Medical Center in Rochester, Minnesota and co-chair of the panel, said at the time the guidelines were issued.
But too few know about the guidelines, Dr. Thompson said. “And as sickle cell disease kids grow to adulthood, we need more doctors who treat the disease in adults.”
We also need to better manage other chronic disease sickle cell kids have, including asthma and high blood pressure.