Baltimore-based Johns Hopkins Kimmel Cancer Center physicians successfully treated 16 patients with aplastic anemia, a rare and deadly form of bone marrow failure, and published their findings in Biology of Blood and Marrow Transplantation.
One in 250,000 people are diagnosed each year with aplastic anemia. Typically, physicians prescribe immunosuppressive therapy and ask patients to avoid contact with the outside world to limit the risk of infections. If the immunosuppressive therapy fails, physicians prescribe a drug called eltrombopag. The drug, however, works in about 30 percent of patients.
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The Johns Hopkins team enrolled 16 patients, between 11 and 69 years, in this study from July 2011 through August 2016. Each of the patients had failed to respond to immunosuppressive therapy or other drug treatments. The patients received partially matched bone marrow transplants followed by two high doses of cyclophosphamide, a chemotherapy drug. For the following year or so, they remained on immunosuppressive medications.
The team reported that more than a year after the treatments, all 16 patients have stopped taking immunosuppressive drugs and no longer have aplastic anemia.