Recombinant human thrombopoietin safety and efficacy in pediatric allogeneic hematopoietic stem cell transplantation: A cohort study

Abstract

BACKGROUND

The safety and efficacy of recombinant human thrombopoietin (rhTPO) administered after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children (0-9 years old) and adolescents (10-17 years old) with hematological disorders remain unclear.

AIM

To evaluate the safety and efficacy of rhTPO administered before platelet (PLT) engraftment in pediatric patients with hematological disorders undergoing HSCT, and to investigate its effects on the incidence of graft-vs-host disease (GVHD) and other transplant-related outcomes.

METHODS

This study enrolled 79 pediatric patients with hematological disorders who received rhTPO after allo-HSCT. The safety and tolerability of rhTPO were evaluated and compared in children (n = 36) and adolescents (n = 43) with hematological disorders. We also investigated the effects of rhTPO administration on the incidence of GVHD and other transplant-related outcomes. Additionally, we examined the efficacy of rhTPO after allo-HSCT in children and adolescents.

RESULTS

All of the children and adolescents underwent hematopoietic reconstruction. The median time to PLT engraftment was 16 days for all patients, with 14 (range, 11-24) days in the 0- to 9-year-old group and 16 (range, 11-41) days in the 10- to 17-year-old group; the difference was statistically significant (P < 0.05). The median time to neutrophil engraftment was 12 days in both groups. The median recovery times for PLT counts of ≥ 20 × 10⁹/L and ≥ 50 × 10⁹/L in the 0- to 9-year-old group were 10 (range, 2-20) and 11 (range, 2-20) days, respectively, and those for the 10- to 17-year-old group were 9 (range, 4-23) and 12 (range, 5-34) days, respectively. Children exhibited significantly shorter time to PLT engraftment (14 days vs 16 days) and shorter recovery time to PLT count ≥ 100 × 10⁹/L (16 days vs 18 days) (P < 0.05) than adolescents. The incidence of acute GVHD in all patients was 53.2%, with a higher incidence in children (61.1%) than in adolescents (46.5%). The incidence of chronic GVHD showed little difference between the two age groups, with an overall incidence of 10.1%. No adverse events, other than bleeding, were observed in either age group. The incidence of bleeding was 20.3%. The median follow-up time for all survivors was 573 days (range: 42-1803 days) after transplantation. At the final follow-up, 3 patients in the 0- to 9-year-old group died; however, none of these deaths were attributed to allo-HSCT or the use of rhTPO. All patients survived in the 10- to 17-year-old group.

CONCLUSION

rhTPO was not associated with any significant safety issues and was well tolerated by pediatric and adolescent patients with hematologic diseases who underwent allo-HSCT. Our results suggested that rhTPO may benefit allo-HSCT in children and adolescents by improving PLT recovery.

Keywords: Recombinant human thrombopoietin; Pediatric hematologic disorders; Allogeneic hematopoietic stem cell transplantation; Platelet engraftment; Graft-vs-host disease

Core Tip: This study evaluated the safety and efficacy of recombinant human thrombopoietin (rhTPO) in pediatric patients (0-17 years) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Results show that rhTPO is well tolerated and improves platelet (PLT) recovery, with shorter PLT engraftment times in children (0-9 years) compared to adolescents (10-17 years). The incidence of acute graft-vs-host disease was higher in children but less severe. No severe adverse events related to rhTPO were observed, supporting its safety and utility in pediatric allo-HSCT. This study provides foundational insights for further research in larger pediatric cohorts.