Blood Res.  2025;60:7. 10.1007/s44313-025-00057-7.

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

• Hong K
• Kim B
• An H
• Choi J
• Song S
• Yu K
• Jang I
• Kang H

• KMID: 2564329
• DOI: http://doi.org/10.1007/s44313-025-00057-7

Abstract

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 cumu‑ lative 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 with‑ out human leukocyte antigen-matched donors.

Keyword

Haploidentical; Post-transplant cyclophosphamide; Cord blood; Children; Hematologic malignancy

Figure

• 

  Fig. 1 A Significantly faster neutrophil engraftment in the HRD group compared with the UCB group (median times to neutrophil engraftment: 15 days [range, 13–21] vs. 14 days [range, 12–40], p = 0.036). B Platelet engraftment was also faster in the HRD group (median 26 days vs. 46 days, p < 0.001)

• 

  Fig. 2 A The cumulative incidence rates of acute GVHD grades II–IV were 41.5% in the HRD group versus 54.2% in the UCB group (p = 0.589), and B grades III–IV were 4.9% in the HRD group versus 29.2% in the UCB group (p = 0.009). C Additionally, the relapse incidence was significantly higher in the HRD group compared with the UCB group (p = 0.004). D However, the cumulative incidence of non-relapse mortality (NRM) was 34.2% in the UCB group, while it was only 2.6% in the HRD group (p < 0.001)

• 

  Fig. 3 The 5-year EFS rates for the HRD and UCB groups were 65.8% and 54.2%, (p = 0.204) (A), while the OS rates were 78.0% and 65.7% (p = 0.142) (B), respectively

Reference

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