Hematopoietic Stem Cell Transplantation Methods and Outcomes
Summary
Haematopoietic stem cell transplantation (HSCT) is an established therapeutic strategy for malignant and non-malignant blood disorders. Broadly, HSCT comprises autologous procedures, in which patients receive their own stem cells, and allogeneic procedures, in which donor cells are infused into genetically matched or mismatched recipients. Allogeneic approaches include matched sibling or unrelated donors, haploidentical relatives and umbilical cord blood units, each necessitating specific conditioning regimens that balance cytoreduction with acceptable toxicity. Advances such as reduced-intensity conditioning, post-transplant cyclophosphamide (PTCy) and cellular therapies have expanded eligibility and improved outcomes by mitigating graft-versus-host disease (GVHD) while preserving graft-versus-leukaemia effects. Key clinical endpoints encompass overall and disease-free survival, relapse incidence, transplant-related mortality and the quality of immune reconstitution. Recent trends highlight growing use of haploidentical donors to address shortages, combined protocols such as haplo-cord co-infusion to accelerate engraftment, and targeted prophylaxis to reduce chronic complications. Ongoing efforts aim to refine donor selection, tailor conditioning intensity and integrate immunomodulatory agents to extend global access and enhance long-term survival and quality of life.
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Hematopoietic Stem Cell Transplantation Methods and Outcomes publication trend
The graph below shows the total number of articles in hematopoietic stem cell transplantation methods and outcomes across all publications each year (not limited to Nature Index journals).
Technical terms
Haematopoietic stem cell transplantation (HSCT): infusion of blood-forming stem cells to restore bone marrow function.
Autologous transplant: patient’s own stem cells harvested and reinfused following conditioning.
Allogeneic transplant: donor-derived stem cells infused into a genetically distinct recipient.
Haploidentical transplant: allogeneic HSCT using a half-matched family donor.
Conditioning regimen: chemotherapy and/or radiotherapy administered before HSCT to eliminate disease and suppress immunity.
Graft-versus-host disease (GVHD): immunological attack by donor cells against host tissues.
Graft-versus-leukaemia (GvL) effect: donor immune-mediated eradication of residual malignant cells.
Post-transplant cyclophosphamide (PTCy): high-dose cyclophosphamide given after graft infusion to prevent GVHD.
Engraftment: successful establishment and proliferation of donor haematopoietic cells.
Mesenchymal stem cells (MSCs): multipotent stromal cells with immunomodulatory properties used in GVHD prophylaxis.
References
- Haploidentical hematopoietic cell transplantation with or without an unrelated cord blood unit for adult acute myeloid leukemia: a multicenter, randomized, open-label, phase 3 trial. Signal Transduction and Targeted Therapy (2024).
- GVHD occurrence does not reduce AML relapse following PTCy-based haploidentical transplantation: a study from the ALWP of the EBMT. Journal of Hematology & Oncology (2023).
- Mesenchymal Stem Cells for Prophylaxis of Chronic Graft-vs-Host Disease After Haploidentical Hematopoietic Stem Cell Transplant. JAMA Oncology (2024).
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