The drug cyclophosphamide could revolutionise blood stem cell transplants, allowing transplants from “mismatched” donors, according to a new study.
Haematologists involved in the research said it suggests most patients should be able to find a bone marrow donor.
A multi-centre study in the USA, conducted by the Center for International Blood and Marrow Transplant Research, followed 145 patients who received the immune suppressant cyclophosphamide to enable them to have peripheral blood stem cells from mismatched unrelated donors.
Reporting in the Journal of Clinical Oncology, the researchers say that 80% were alive after a year. 10% of the patients developed moderate or severe graft-versus-host disease – a proportion they claim is a similar to patients who receive fully matched donations. The majority of patients in the study had been diagnosed with acute myeloid leukaemia.
The researchers say the use of cyclophosphamide could be a “significant advance”, and call for further work to improve the safety and effectiveness of mismatched blood stem cell transplants.
One of the researchers Dr Karen Ballen, of University of Virginia Health, said: “This study is important because all patients, regardless of background, now have a stem cell or bone marrow transplant donor, a significant advance for our field, our patients and our community.
“At UVA Health, in the past year, all eligible patients for stem cell transplant were able to find a suitably matched donor.”
Source:
Al Malki MM, Bo-Subait S, Logan B, Olson J, Kou J, Smith S, Leckrone E, Wu J, Stefanski HE, Auletta JJ, Spellman SR, Malmberg C, Askar M, Cusatis R, Shaffer BC, Modi D, Khimani F, Gooptu M, Hamadani M, Madbouly A, Maiers M, Fingerson S, Cook R, Ballen K, Loren A, Larkin K, Arai S, Qayed M, Choi SW, Broglie L, Shaw BE, Devine SM, Jimenez Jimenez AM. (2025) “Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation.” Journal of Clinical Oncology, 16 June 2025, doi: 10.1200/JCO-25-00856.
Link: https://ascopubs.org/doi/10.1200/JCO-25-00856
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