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Allogeneic stem cell transplantation replaces a patient's blood system with a donor's after conditioning chemotherapy, so donor immune cells hunt down leukaemia left behind. It remains the only cure for adverse-risk acute myeloid leukaemia, high-risk acute lymphoblastic leukaemia and Richter transformation, at the price of graft-versus-host disease.
Conditioning chemotherapy (myeloablative or reduced-intensity) followed by donor stem cells. The graft-versus-leukaemia effect provides ongoing immune surveillance; graft-versus-host disease is the price. Indicated in ELN adverse-risk AML in first remission, intermediate-risk with MRD positivity, relapsed disease, high-risk ALL, and Richter transformation. Post-transplant cyclophosphamide has made haploidentical donors routine; post-transplant maintenance (FLT3 inhibitors, azacitidine, menin inhibitors in trials) is reducing relapse.
Myeloablation eradicates host haematopoiesis; donor T cells recognise residual leukaemia via minor histocompatibility antigens.
Tregzi, formerly Orca-T, is a donor stem cell transplant sorted with high precision and given with regulatory T cells, so that patients with blood cancers keep the graft's anti-cancer effect while being protected from chronic graft-versus-host disease. The FDA approved it on 30 June 2026 for adults having a matched-donor transplant.
Belumosudil is a pill for chronic graft-versus-host disease, the long-term immune complication of donor stem-cell transplants used to cure blood cancers.
Busulfan is one of the oldest cancer drugs. As a tablet it controlled chronic myeloid leukaemia before imatinib; as an infusion it is used to clear the bone marrow before a stem cell transplant.
Cyclophosphamide is an alkylating chemotherapy that damages DNA in dividing cells. It is part of CHOP for lymphoma, AC for breast cancer and VAC for childhood sarcomas, clears lymphocytes before CAR-T and prevents graft-versus-host disease after transplant; bladder bleeding, infertility and secondary leukaemia are its harms.
Defibrotide (Defitelio) is the only approved treatment for hepatic veno-occlusive disease, a life-threatening liver complication of high-dose chemotherapy before stem cell transplant, in patients whose kidneys or lungs are also failing.
Zemcelpro is a laboratory-expanded umbilical cord blood transplant for adults with blood cancers who need a donor stem cell transplant but have no suitable matched donor; expanding the cells lets a single small cord blood unit be enough.
Fludarabine is a chemotherapy infusion for chronic lymphocytic leukaemia. It anchored the FCR regimen that was the first to give long remissions, and today it is used above all to prepare patients for CAR-T cells and transplants.
An expanded umbilical cord blood graft that shortens the dangerous wait for neutrophils to return after a transplant for blood cancer, the first cell therapy approved to speed engraftment.
Ruxolitinib was the first JAK inhibitor: it shrinks the spleen and relieves symptoms in myelofibrosis and controls blood counts in polycythaemia vera, without eliminating the disease clone.
Thiotepa is an old chemotherapy with a modern job: at high doses it prepares patients for stem cell transplants, and it is still instilled into the bladder or body cavities for superficial bladder cancer and malignant effusions.
A conditioning chemotherapy given before donor stem-cell transplant in AML and MDS; it was long used in Europe and reached the US in 2025.
QuANTUM-First gave FLT3-ITD AML patients a second front-line targeted option and showed that continuing a FLT3 inhibitor as long-term maintenance, including after transplant, pays off. Quizartinib was approved for this indication in 2023. Head-to-head data against midostaurin are lacking, and the design leaves open how much of the benefit came from maintenance.
ADMIRAL showed that a targeted oral drug can beat chemotherapy outright in relapsed AML, and made gilteritinib the standard bridge to transplant for FLT3-mutated relapse. Its success also underpinned FLT3 inhibitor use in first-line combinations. Resistance through FLT3-independent clones and RAS pathway mutations limits durability without transplant.
INO-VATE made inotuzumab a standard salvage therapy for adult ALL and a common route to transplant, and, with the TOWER trial of blinatumomab, moved adult ALL from chemotherapy-only salvage to immunotherapy. Both drugs have since been pulled into front-line regimens. The liver toxicity signal shaped how transplant conditioning is chosen after inotuzumab.
Query for this technology: (TITLE:"Allogeneic stem cell transplantation" OR ABSTRACT:"Allogeneic stem cell transplantation") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Allogeneic stem cell transplantation, not a curated reading list.
Shares Defibrotide, John F. DiPersio, Study 2005-01, Stem cell clinics and stem cell tourism.
Shares Extracorporeal photopheresis for graft-versus-host disease, Belumosudil, After ruxolitinib: belumosudil, axatilimab and ibrutinib in chronic GvHD, Preventing graft-versus-host disease, and what prevention costs.
Shares MRD-negative complete remission, Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL), Plasma cell leukaemia, Philadelphia chromosome-positive acute lymphoblastic leukaemia in children (Ph-positive ALL).
Shares MRD-guided transplant decisions in intermediate-risk AML, Transplant-free Ph-positive ALL for MRD-negative adults, MRD-negative complete remission, Philadelphia chromosome-like acute lymphoblastic leukaemia (Ph-like or BCR::ABL1-like ALL).
Shares Treosulfan, Preventing graft-versus-host disease, and what prevention costs, Bone, eyes, kidneys and lungs after transplant, Hormones, metabolism and the heart after transplant.
Shares Mesenchymal stromal cells for tissue damage, Stem cell clinics and stem cell tourism, Iron overload after transplant, Survival after transplant, and why the curve never quite rejoins the population.
Shares Iron overload after transplant, Survival after transplant, and why the curve never quite rejoins the population, The lungs after transplant: bronchiolitis obliterans syndrome, Bone, eyes, kidneys and lungs after transplant.
Shares Iron overload after transplant, When steroids fail: ruxolitinib for steroid-refractory GvHD, Rebuilding an immune system: the timeline, lineage by lineage, Infection risk after transplant and cell therapy, phase by phase, and the prophylaxis that follows it.