Bone marrow transplant as a cure for sickle cell disease

Hopkins Medicine magazine highlights a 30 year-old with sickle cell disease who successfully underwent a bone marrow transplant. Over the past 30 years, only about 400 persons with sickle cell disease have actually received a bone marrow transplants. Several limiting factors, such as the need for a “full match” sibling donor and the high risk of death following the procedure, were significant obstacles that prevented bone marrow transplants from becoming a widespread cure for the disease. Dr. Robert Brodsky, director of the Division of Hematology at Hopkins, and his colleagues, are leading a new frontier in bone marrow transplants that benefit from safer transplant protocols and a new chemotherapy regimens that are more efficient at killing red blood cells while sparing stem cells. Not only will bone marrow transplants be safer and have improved outcomes but these new chemotherapy agents may make it possible for transplanting with “half-matched” donors. I this scenario, 90% of the sickle cell population, rather than only 9%, would have an eligible donor.

Yetunde Felix-Ukwu suffered through almost 30 years of sickle cell disease related pain and complications. After 3 days of a grueling chemotherapy regimen, in the hospital testing for 7 days a week for 2 months straight following the procedure, she successfully completed the bone marrow transplant procedure and no longer had evidence of sickle cell disease in her blood. However, the pain episodes were not gone. Despite the existence of any “bad blood” Felix-Ukwu continued to experience the devastating painful episodes that she had known so well for the prior three decades. Two year following her transplant, she was still having unexplained painful episodes, severe enough to warrant a hospitalization. The experience of dealing with these recurring episodes despite the lack of sickle cell disease was so stressful, at one point Felix-Ukwu received treatment for depression. However, the painful episodes did not last forever. One day Felix-Ukwu realized she had not been to the hospital in several months and couldn’t remember the last time she had last needed her pain killers.

Now, living a “normal” life, in school pursuing her master’s degree, Felix-Ukwu experienced the happy ending that will hopefully come to many, many more people living with sickle cell disease.


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