Low-Dose Post-Transplant Cyclophosphamide in Haploidentical Stem Cell Transplantation: A Systematic Review
Abstract
BACKGROUND
Haploidentical allogeneic stem cell transplants (ASCT) has transformed the treatment landscape for patients with life-threating hematologic disorders who lack matched donors. Post-transplant cyclophosphamide (PTCy) at 50 mg/kg at days +3 and +4 is established as one of the standard regimens for graft-versus-host disease (GvHD) prophylaxis. Studies on whether reducing the dose of PTCy can reduce toxicity while maintaining efficacy suffer from significant heterogeneity and lack a robust synthesis of available data.
OBJECTIVE
We conducted a systematic review to investigate the hypothesis that low-dose PTCy in haploidentical ASCT can offer comparable or superior outcomes than the standard, 100mg/kg dose with regards to acute and chronic GvHD incidence, survival, and toxicity.
STUDY DESIGN
This review had a pre-registered protocol on PROSPERO (CRD420250650291) and followed PRISMA guidelines. Relevant literature across multiple databases was searched from inception until 2025. We included retrospective and prospective observational studies and clinical trials of patients receiving haploidentical ASCT and low-dose PTCy that reported acute/chronic GvHD incidence, overall survival (OS), relapse-free survival (RFS), hemorrhagic cystitis, infections, or immune reconstitution. ROBIS-I was used to assess risk-of-bias for observational studies and Cochrane RoB 2.0 for randomized studies. Study heterogeneity and a paucity of randomized studies precluded a meta-analysis of…
How strong is the evidence?
Evidence Hub reads this as a systematic review, done in people. Its evidence level is 1 of 5 (Highest · pooled evidence). Pooled evidence from several studies sits at the top of the evidence pyramid. Reported size: 2,500 participants or records.
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