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post-transplantation cyclophosphamide
Post-transplantation cyclophosphamide selectively eliminates alloreactive T cells after hematopoietic stem cell transplantation to reduce graft-versus-host disease while preserving graft-versus-leukemia effects.
Post-transplantation cyclophosphamide selectively eliminates alloreactive T cells after hematopoietic stem cell transplantation to reduce graft-versus-host disease while preserving graft-versus-leukemia effects. Used for Prevention of graft-versus-host disease in hematopoietic stem cell transplantation, Allogeneic hematopoietic stem cell transplantation for hematologic malignancies.
At a glance
| Generic name | post-transplantation cyclophosphamide |
|---|---|
| Sponsor | Peking University People's Hospital |
| Drug class | Alkylating agent |
| Modality | Small molecule |
| Therapeutic area | Oncology / Immunology |
| Phase | FDA-approved |
Mechanism of action
Cyclophosphamide is administered in the early post-transplant period (typically days 3 and 4) when alloreactive donor T cells are actively proliferating. The drug preferentially targets rapidly dividing cells, including alloreactive lymphocytes, while sparing slower-dividing hematopoietic stem cells and regulatory T cells. This selective depletion reduces severe acute and chronic GVHD while maintaining beneficial anti-tumor immunity.
Approved indications
- Prevention of graft-versus-host disease in hematopoietic stem cell transplantation
- Allogeneic hematopoietic stem cell transplantation for hematologic malignancies
Common side effects
- Hemorrhagic cystitis
- Myelosuppression
- Infection
- Nausea and vomiting
- Mucositis
Key clinical trials
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Pediatric-Inspired Regimen Combined With Venetoclax and Immunotherapy for Adult Ph-Negative Acute Lymphoblastic Leukemia (NA)
- Ibrutinib Before and After Stem Cell Transplant in Treating Patients With Relapsed or Refractory Diffuse Large B-cell Lymphoma (PHASE3)
- Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1 (PHASE3)
- Autologous T Cells Transduced With Retroviral Vectors Expressing TCRs for Participant-specific Neoantigens in Patients With Hematologic Malignancies (PHASE1)
- Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists (PHASE1, PHASE2)
- MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases (PHASE2)
- Expanded/Activated Gamma Delta T-cell Infusion Following Hematopoietic Stem Cell Transplantation and Post-transplant Cyclophosphamide (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
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