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Haplo-HCT
Haplo-HCT is a haploidentical hematopoietic cell transplantation approach that enables transplantation from half-matched donors by using post-transplant cyclophosphamide and other immunosuppressive strategies to prevent graft-versus-host disease.
Haplo-HCT is a haploidentical hematopoietic cell transplantation approach that enables transplantation from half-matched donors by using post-transplant cyclophosphamide and other immunosuppressive strategies to prevent graft-versus-host disease. Used for Hematologic malignancies (acute leukemia, chronic leukemia, lymphoma, myeloma) in patients lacking matched donors, Severe aplastic anemia and other bone marrow failure syndromes.
At a glance
| Generic name | Haplo-HCT |
|---|---|
| Sponsor | The First Affiliated Hospital of Soochow University |
| Modality | Biologic |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Haplo-HCT leverages haploidentical (half-matched) donors, which are more readily available than fully matched donors, by employing post-transplant cyclophosphamide (PTCy) to selectively eliminate alloreactive T cells while preserving beneficial graft-versus-leukemia effects. This approach expands access to hematopoietic stem cell transplantation for patients with hematologic malignancies or bone marrow disorders who lack matched sibling or unrelated donors.
Approved indications
- Hematologic malignancies (acute leukemia, chronic leukemia, lymphoma, myeloma) in patients lacking matched donors
- Severe aplastic anemia and other bone marrow failure syndromes
Common side effects
- Graft-versus-host disease (GVHD)
- Infection
- Relapse of underlying malignancy
- Organ toxicity
- Graft failure or delayed engraftment
Key clinical trials
- The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation (PHASE1, PHASE2)
- A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT) (PHASE2)
- MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis (PHASE2)
- Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome (PHASE3)
- Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies (PHASE2)
- Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease (PHASE1, PHASE2)
- Orca-T Following Chemotherapy and Total Marrow and Lymphoid Irradiation for the Treatment of Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia or Myelodysplastic Syndrome (PHASE1)
- Two Step Haplo With Radiation Conditioning (PHASE2)
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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Haplo-HCT CI brief — competitive landscape report
- Haplo-HCT updates RSS · CI watch RSS
- The First Affiliated Hospital of Soochow University portfolio CI