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Purpose <p>This study compared the outcomes of haploidentical-related donor (HRD) and umbilical cord blood (UCB) hematopoietic stem cell transplantation (HSCT) in pediatric patients with hematologic malignancies.</p> Methods <p>Data on patients who underwent HRD HSCT with post-transplant cyclophosphamide (<i>n</i> = 41) and UCB HSCT (<i>n</i> = 24) after targeted busulfan-based myeloablative conditioning with intensive pharmacokinetic monitoring between 2009 and 2018 were retrospectively analyzed.</p> Results <p>The median follow-up durations in the HRD and UCB groups were 7.0 and 10.9&#xa0;years, respectively. The cumulative incidence of acute graft-versus-host disease (GVHD) grades II–IV and moderate-to-severe chronic GVHD did not differ significantly between the groups. However, the HRD group demonstrated significantly lower rates of acute GVHD grades III–IV (4.9% vs. 29.2%, <i>p</i> = 0.009) and non-relapse mortality (2.6% vs. 34.2%, <i>p</i> &lt; 0.001) but a higher relapse incidence (32.1% vs. 8.8%, <i>p</i> = 0.004) than the UCB group. The 5-year event-free and overall survival rates were 65.8% and 54.2% (<i>p</i> = 0.204) and 78.0% and 65.7% (<i>p</i> = 0.142) for the HRD and UCB groups, respectively. Multivariate analysis identified disease status as a significant risk factor for overall survival (hazard ratio, 3.24; <i>p</i> = 0.016). Additionally, UCB HSCT exhibited a trend toward worse event-free survival compared to HRD HSCT (hazard ratio, 2.63; <i>p</i> = 0.05).</p> Conclusions <p>These findings indicate that HRD HSCT with post-transplant cyclophosphamide provides promising outcomes compared to UCB HSCT in pediatric patients, with a trend toward improved survival over a long-term follow-up period exceeding a median of 7&#xa0;years. Thus, HRD HSCT may be a valuable option for pediatric patients without human leukocyte antigen-matched donors.</p>

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Comparing haploidentical transplantation with post-transplantation cyclophosphamide and umbilical cord blood transplantation using targeted busulfan in children and adolescents with hematologic malignancies

  • 期刊论文

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  • 发表日期：

    2025年1月23日

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  • Kyung Taek Hong,
  • Bo Kyung Kim,
  • Hong Yul An,
  • Jung Yoon Choi,
  • Sang Hoon Song,
  • Kyung-Sang Yu,
  • In-Jin Jang,
  • Hyoung Jin Kang

摘要

Purpose

This study compared the outcomes of haploidentical-related donor (HRD) and umbilical cord blood (UCB) hematopoietic stem cell transplantation (HSCT) in pediatric patients with hematologic malignancies.

Methods

Data on patients who underwent HRD HSCT with post-transplant cyclophosphamide (n = 41) and UCB HSCT (n = 24) after targeted busulfan-based myeloablative conditioning with intensive pharmacokinetic monitoring between 2009 and 2018 were retrospectively analyzed.

Results

The median follow-up durations in the HRD and UCB groups were 7.0 and 10.9 years, respectively. The cumulative incidence of acute graft-versus-host disease (GVHD) grades II–IV and moderate-to-severe chronic GVHD did not differ significantly between the groups. However, the HRD group demonstrated significantly lower rates of acute GVHD grades III–IV (4.9% vs. 29.2%, p = 0.009) and non-relapse mortality (2.6% vs. 34.2%, p < 0.001) but a higher relapse incidence (32.1% vs. 8.8%, p = 0.004) than the UCB group. The 5-year event-free and overall survival rates were 65.8% and 54.2% (p = 0.204) and 78.0% and 65.7% (p = 0.142) for the HRD and UCB groups, respectively. Multivariate analysis identified disease status as a significant risk factor for overall survival (hazard ratio, 3.24; p = 0.016). Additionally, UCB HSCT exhibited a trend toward worse event-free survival compared to HRD HSCT (hazard ratio, 2.63; p = 0.05).

Conclusions

These findings indicate that HRD HSCT with post-transplant cyclophosphamide provides promising outcomes compared to UCB HSCT in pediatric patients, with a trend toward improved survival over a long-term follow-up period exceeding a median of 7 years. Thus, HRD HSCT may be a valuable option for pediatric patients without human leukocyte antigen-matched donors.