Li XG, Wang RM, Chen W, Yao T, Chen F, Xu YF, Lang T. Recombinant human thrombopoietin safety and efficacy in pediatric allogeneic hematopoietic stem cell transplantation: A cohort study. World J Stem Cells 2025; 17(7): 106579 [DOI: 10.4252/wjsc.v17.i7.106579]
Corresponding Author of This Article
Tao Lang, MD, Chief Physician, Department of Hematology, The People’s Hospital of Xinjiang Uygur Autonomous Region, No. 91 Tianchi Road, Urumqi 830001, Xinjiang Uygur Autonomous Region, China. langt2024@163.com
Research Domain of This Article
Hematology
Article-Type of This Article
Observational Study
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
World J Stem Cells. Jul 26, 2025; 17(7): 106579 Published online Jul 26, 2025. doi: 10.4252/wjsc.v17.i7.106579
Recombinant human thrombopoietin safety and efficacy in pediatric allogeneic hematopoietic stem cell transplantation: A cohort study
Xue-Guo Li, Ru-Min Wang, Wei Chen, Tong Yao, Fen Chen, Yan-Fang Xu, Tao Lang
Xue-Guo Li, Ru-Min Wang, Yan-Fang Xu, Tao Lang, Department of Hematology, The People’s Hospital of Xinjiang Uygur Autonomous Region, Urumqi 830001, Xinjiang Uygur Autonomous Region, China
Wei Chen, Department of Blood Transfusion, The People’s Hospital of Xinjiang Uygur Autonomous Region, Urumqi 830001, Xinjiang Uygur Autonomous Region, China
Tong Yao, Fen Chen, Department of Pediatric Hematology, Children’s Hospital of Xinjiang Uygur Autonomous Region, Xinjiang Hospital of Beijing Children’s Hospital, Urumqi 830000, Xinjiang Uygur Autonomous Region, China
Co-first authors: Xue-Guo Li and Ru-Min Wang.
Author contributions: Li XG and Wang RM both contributed to drafting the initial manuscript; Li XG, Wang RM, Chen W, and Yao T designed and performed the research study; Chen F and Xu YF provided help and advice on experiments and analyzed the data; Lang T contributed equally to the conceptualization, supervision, and final manuscript revisions, justifying their corresponding author; All authors contributed significantly to the work and agreed to be accountable for all aspects of the work, contributed to editorial changes in the manuscript, and read and approved the final manuscript. Li XG and Wang RM are co-first authors who contributed equally to this work.
Institutional review board statement: This study was approved by the Ethics Committee of the People’s Hospital of the Xinjiang Uygur Autonomous Region (approval No. XJS2018080701).
Informed consent statement: Written informed consent was obtained from parents of all patients.
Conflict-of-interest statement: The authors have no conflicts of interest to declare.
STROBE statement: The authors have read the STROBE Statement-checklist of items, and the manuscript was prepared and revised according to the STROBE Statement-checklist of items.
Data sharing statement: Data supporting the findings of this study are available from the corresponding author upon request.
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Tao Lang, MD, Chief Physician, Department of Hematology, The People’s Hospital of Xinjiang Uygur Autonomous Region, No. 91 Tianchi Road, Urumqi 830001, Xinjiang Uygur Autonomous Region, China. langt2024@163.com
Received: March 7, 2025 Revised: April 15, 2025 Accepted: June 25, 2025 Published online: July 26, 2025 Processing time: 139 Days and 2.1 Hours
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.
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.
