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Replacing a patient's blood system with a donor's: chemotherapy wipes out the marrow, donor stem cells rebuild it, and the donor's immune cells hunt down leftover leukaemia. It is the only cure for adverse-risk or relapsed AML, high-risk ALL, MDS and myelofibrosis, and the riskiest treatment in oncology because of graft-versus-host disease and infection.
Used for adverse-risk or relapsed AML, high-risk ALL and MDS, myelofibrosis and some lymphomas. Donors are HLA-matched siblings, matched unrelated donors from registries, haploidentical relatives (now safe with post-transplant cyclophosphamide) or cord blood. Conditioning is myeloablative or reduced-intensity depending on age and fitness. The graft-versus-leukaemia effect is the cure mechanism; graft-versus-host disease, infection and organ toxicity are the price, with treatment-related mortality of 10-25%. MRD-guided transplant decisions, CAR-T bridging and maintenance drugs after transplant (FLT3 inhibitors, azacitidine) are refining who needs it.
Showing the technology this term belongs to: Allogeneic (off-the-shelf) cell therapy.
This is the trial behind belumosudil's July 2021 US approval for chronic graft-versus-host disease after two or more lines. For patients it means a once-daily pill with a high response rate in a condition where earlier treatments had failed; the FDA's own analysis, counting responses only through cycle 7 day 1, gave 75 percent.
This is the study behind the December 2024 US approval of remestemcel-L (Ryoncil), the first mesenchymal stromal cell therapy approved in the United States, for children whose acute graft-versus-host disease has not settled on steroids. The label reports the same 54 children as a 70 percent response.
This is the trial behind defibrotide's March 2016 US approval, the only approved treatment for hepatic veno-occlusive disease with organ failure after transplant. The historical-control design was accepted because a randomised trial in a condition this lethal and rare was judged unfeasible; the gain is meaningful but most patients still died.
Shares ROCKstar, Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study, Graft-versus-host disease (GVHD) and graft-versus-leukaemia, Acute lymphoblastic leukaemia.
Shares A phase 3, single-arm, prospective study of remestemcel-L, ex vivo culture-expanded adult human mesenchymal stromal cells for the treatment of pediatric patients who failed to respond to steroid treatment for acute graft-versus-host disease, MSB-GVHD001, Graft-versus-host disease (GVHD) and graft-versus-leukaemia.
Shares DIPSS, DIPSS-plus and MIPSS70 (myelofibrosis risk scores), IPSS-R and IPSS-M (myelodysplastic syndrome risk scores), Chronic myeloid leukaemia, accelerated and blast phase, Acute lymphoblastic leukaemia.
Shares 7+3 induction chemotherapy, IPSS-R and IPSS-M (myelodysplastic syndrome risk scores), Higher-risk myelodysplastic syndromes, Acute myeloid leukaemia.
Shares A phase 3, single-arm, prospective study of remestemcel-L, ex vivo culture-expanded adult human mesenchymal stromal cells for the treatment of pediatric patients who failed to respond to steroid treatment for acute graft-versus-host disease, MSB-GVHD001.
Shares Xiaojun Huang, Allogeneic (off-the-shelf) cell therapy, Acute lymphoblastic leukaemia, Acute myeloid leukaemia.
Shares DIPSS, DIPSS-plus and MIPSS70 (myelofibrosis risk scores), AML with myelodysplasia-related gene mutations (AML-MR), IPSS-R and IPSS-M (myelodysplastic syndrome risk scores), Acute lymphoblastic leukaemia.
Shares Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure, Study 2005-01.