Ongoing study at Yale suggests full sickle cell cure possible even without a strong donor match
Rehana Konate used to spend a lot of time in the hospital.
“The hospital became my second home,” said Konate, a 21-year-old UConn student who grew up in Stamford, the daughter of a single mother.
Konate suffered from sickle cell disease, an inherited disorder that causes red blood cells to contort into a sickle shape, resulting in severe bone pain and fatigue. The Centers for Disease Control and Prevention say it occurs in 1 in every 365 Black or African American births.
But now, Konate is fully cured of the disease after a bone marrow transplant at Smilow Cancer Hospital in 2019. Konate had a strong donor match – her sister from Mali, Africa.
And researchers at Yale say they are seeing a full cure after bone marrow transplants even with donors who are not the optimum match.
Transplant patients can develop a complication called graft-versus-host disease. But Yale researchers say they have found a solution.
“We have an ongoing study at Yale that adding one of the agent[s] or new drug[s] into this transplant process has helped us reduce the risk of graft-versus-host disease, even if the donor is some unknown donor, or if the donor is not [a] 100% match,” Dr. Niketa Shah told Connecticut Public Radio’s Where We Live. Shah is the director of the Pediatric Bone Marrow Transplant Program and the Pediatric Cellular Therapy Program at Yale Medicine.
Shah emphasized that success rates are almost 100% for younger patients, with younger donors. “Before they experience [a] crisis and as soon as they’re diagnosed with sickle cell disease at birth, maybe we should think about that: Can we cure this disease?”
UConn Health Center is also making advancements in sickle cell treatment. New drugs and an apheresis machine, which replaces sickled blood cells with healthy blood cells, are improving outcomes, said Teresa Works, a sickle cell social worker at UConn Health.
“Anecdotally, [it] seems to have really made a difference in a lot of our patients both in terms of reducing hospitalizations but also in terms of reducing their pain and suffering,” Works said.