Transplant approach showed 96% survival — News-Medical

Researchers at the Sidney Kimmel Comprehensive Cancer Center and Johns Hopkins Children’s Center evaluated bone marrow transplant outcomes in children, adolescents and young adults with severe aplastic anemia. Of 23 patients who received cyclophosphamide after transplantation, 22 survived — overall survival was 96%, News-Medical reports.

A study of 31 patients

The team analyzed data from 31 patients aged 2 to 20 who underwent bone marrow transplantation at Johns Hopkins between 2014 and 2024. Of these, 23 received a reduced-intensity conditioning regimen and cyclophosphamide after the procedure. This group included 21 patients who received bone marrow from a half-matched relative, as well as two recipients with fully matched siblings.

Another eight patients received standard transplants from matched siblings with a different combination of drugs before and after the procedure. All of them survived. In the cyclophosphamide group, all 14 patients whose disease had relapsed or had not fully responded to previous treatment survived, as did eight of nine previously untreated patients.

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Cell engraftment and complications

Severe aplastic anemia occurs when the bone marrow does not produce enough red blood cells, white blood cells and platelets. A transplant can replace damaged blood-forming cells with healthy donor cells. The authors note that only about 25–30% of patients have a fully matched sibling donor, whereas half-matched donors can be parents, siblings or other relatives.

One year after transplantation, at least 95% of blood cells in all surviving patients who received the new approach were of donor origin. At the last follow-up, none of them required blood transfusions. Severe graft-versus-host disease, in which donor immune cells attack the recipient’s tissues, occurred infrequently. After one year, 95% of surviving patients no longer needed drugs to suppress the immune system. At the same time, the researchers cautioned that the sample was small and that longer follow-up is needed to assess long-term outcomes.