Recombinant human thrombopoietin in pediatric allogeneic hematopoietic stem cell transplantation: Clinical insights and future directions

Abstract

The cohort study by Li et al provides timely and clinically relevant evidence on the use of recombinant human thrombopoietin (rhTPO) in pediatric allogeneic hematopoietic stem cell transplantation. The authors report enhanced platelet engraftment and a favorable safety profile, particularly in younger children aged 0-9 years. This age-dependent difference not only highlights the physiological responsiveness of early hematopoietic environments to rhTPO but also raises important questions about tailoring supportive therapies across pediatric age groups. While the findings are promising, the lack of a control group and single-center limitations warrant further multicenter, long-term investigations. Nevertheless, the study lays a compelling foundation for integrating rhTPO more broadly into pediatric transplant protocols and for advancing individualized post-transplant care.

Key Words: Recombinant human thrombopoietin; Pediatric hematopoietic stem cell transplantation; Platelet engraftment; Age-dependent response; Allogeneic transplantation; Supportive care

Core Tip: This cohort study evaluates the safety and efficacy of recombinant human thrombopoietin (rhTPO) in pediatric allogeneic hematopoietic stem cell transplantation. The findings suggest that rhTPO promotes earlier platelet engraftment, particularly in younger children aged 0-9 years. This age-dependent response highlights the influence of developmental hematopoiesis on treatment outcomes. Despite the limitations of a single-center, non-randomized design, the study provides novel insights that support rhTPO as a promising supportive therapy in pediatric allogeneic hematopoietic stem cell transplantation and underscores the need for age-specific post-transplant strategies.

TO THE EDITOR

Hematopoietic stem cell transplantation (HSCT) is used to treat a variety of hematological malignancies, and bleeding is a common complication[1,2]. The recent observational study by Li et al[3], published in the World Journal of Stem Cells, provides important clinical insights into the safety and efficacy of recombinant human thrombopoietin (rhTPO) in pediatric patients undergoing allogeneic HSCT (allo-HSCT). Examining a cohort of 79 pediatric patients aged between 0 and 17 years, the study highlights the notable clinical benefits of rhTPO in enhancing platelet recovery and potentially reducing complications related to transplantation.

A particularly compelling observation from this study is the age-dependent response to rhTPO, with younger children (0-9 years) demonstrating quicker platelet engraftment compared to adolescents (10-17 years). This finding emphasizes that age-specific physiological, immunological factors and haematopoietic stem cell niche may significantly influence treatment outcomes in pediatric allo-HSCT[3]. Additionally, the higher incidence yet lower severity of acute graft-vs-host disease observed in younger children merits further immunological exploration, as it may guide tailored interventions and supportive care strategies.

Nevertheless, from my perspective, several limitations need addressing to strengthen these promising findings. Primarily, the absence of a comparative control group in this single-center observational design restricts the interpretative robustness of the outcomes. Prospective randomized controlled studies or extensive multicenter collaborative trials are essential to conclusively validate the efficacy of rhTPO. Furthermore, long-term studies assessing outcomes such as sustained platelet function and overall quality of life are vital to establish a comprehensive safety and efficacy profile[4].

Moreover, a number of studies have shown that bone marrow microenvironment and cytokines can affect platelet formation and prognosis of HSCT[5]. Mouse experiments have been conducted to explore the differences in the effects of megakaryocytes in different growth cycles, but there is still a lack of specific studies related to human age[6,7]. Further improving comparisons between pediatric engraftment times and adult research findings, and exploring the biological mechanism behind the observed age-dependent changes can greatly enhance the clinical application and potentially extend these benefits beyond pediatric populations.

In summary, the study by Li et al[3] significantly advances our understanding of rhTPO’s clinical potential in pediatric allo-HSCT. Additional rigorous research is necessary to optimize treatment protocols and confirm long-term benefits. Future studies should prioritize multicenter randomized trials and mechanistic exploration of age-specific responses, which will further refine this promising therapeutic approach.