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Outcomes With Haploidentical Stem Cell Transplantation Plus Cyclophosphamide in Adults With Hematologic Cancers


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In a meta-analysis reported in JAMA Oncology, Gagelmann et al found that overall survival with haploidentical stem cell transplantation with post-transplantation cyclophosphamide was poorer vs matched related donor transplantation and similar vs matched unrelated donor transplantation in adult hematologic cancers.

“Results of this meta-analysis suggest that matched related donors, if available, remain the optimal donors regarding mortality and [haploidentical] stem cell transplantation with posttransplant cyclophosphamide may be preferred over mismatched unrelated donors. Prospective comparisons with matched unrelated donors are needed.”
— Gagelmann et al

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Study Details  

The meta-analysis included 22,974 patients from 30 studies comparing haploidentical stem cell transplantation and post-transplantation cyclophosphamide therapy with transplantations from other donors. Comparator groups in the studies consisted of:

  • Matched related donor only (11 studies)
  • Matched unrelated donor only (seven studies)
  • Matched related donor and matched unrelated donor (eight studies)
  • Mismatched unrelated donor (one study)
  • Matched related donor and mismatched unrelated donor (two studies)
  • Matched related donor, matched unrelated donor, and mismatched unrelated donor (one study).

All-cause mortality was assessed in all 30 studies at 18 to 49 months of follow-up. Pooled odds ratios (ORs) were derived from a random-effects model.

Key Findings

Haploidentical stem cell transplantation with cyclophosphamide was associated with increased all-cause mortality vs matched related donors (OR = 1.17, 95% confidence interval [CI] = 1.05–1.30), similar mortality vs matched unrelated donors (OR = 1.06, 95% CI = 0.96–1.18), and reduced mortality vs mismatched unrelated donors (OR = 0.75, 95% CI = 0.61–0.92).

Risk of nonrelapse mortality with haploidentical stem cell transplantation plus cyclophosphamide was greater vs matched related donors (OR = 1.20, 95% CI = 1.04–1.40), reduced vs matched unrelated donors (OR = 0.75, 95% CI = 0.61–0.92), and numerically reduced vs mismatched unrelated donors (OR = 0.51, 95% CI = 0.25–1.02).

Risk of relapse with haploidentical stem cell transplantation plus cyclophosphamide was greater vs matched unrelated donors (OR = 1.20, 95% CI = 1.03–1.40) and similar to that with matched related donors (OR = 1.01, 95% CI = 0.86–1.17) and mismatched unrelated donors (OR = 1.06, 95% CI = 0.77–1.47).

The investigators concluded, “Results of this meta-analysis suggest that matched related donors, if available, remain the optimal donors regarding mortality and [haploidentical] stem cell transplantation with posttransplant cyclophosphamide may be preferred over mismatched unrelated donors. Prospective comparisons with matched unrelated donors are needed.”

Nicolaus Kröger, MD, of the Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, is the corresponding author for the JAMA Oncology article.

Disclosure: For full disclosures of the study authors, visit jamanetwork.com.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
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