Efficacy of therapeutic strategies on thrombocytopenia after hematopoietic stem cell transplantation-an evidence-based meta-analysis.

Abstract

Background: Thrombocytopenia is a common complication following hematopoietic stem cell transplantation, and there is currently no standardized therapeutic approach for its management.

Objectives: This article aims to systematically review the existing clinical treatment regimens for this condition and assess their efficacy.

Design: This is a systematic review and meta-analysis.

Data sources and methods: We conducted a comprehensive literature search in several databases, including PubMed, Web of Science, Cochrane Library, and Embase, up until October 30, 2024. A total of 43 datasets involving 1154 patients were included for further analysis. Therapeutic effectiveness was defined as a patient's platelet count being above 20 × 10⁹/L and being off platelet transfusion for more than 7 consecutive days. Drug response rates and side effect values were statistically calculated.

Results: Our study included a total of 42 articles. The results of our study show that Decitabine and Romiplostim show relatively high effectiveness in treating thrombocytopenia after hematopoietic stem cell transplantation (HSCT), with efficacy rates of 83% (95% CI: 57%-99%) and 87% (95% CI: 69%-98%), respectively. Eltrombopag and Avatrombopag also demonstrated good efficacy, with overall utilities of 68% and 73%. Subgroup analyses revealed that Eltrombopag was particularly effective in adolescent patients compared to adults. Most of the reported side effects were well tolerated, with an overall effect value of 14% (95% CI: 11%-18%, I ² = 41.08%, p = 0.04), and no patients discontinued treatment due to adverse effects. Furthermore, our network meta-analysis suggests that Decitabine-based therapy shows significant efficacy in managing thrombocytopenia after HSCT, although more high-quality clinical trials are needed to validate these conclusions.

Conclusion: Our meta-analysis suggests a significant efficacy of both Romiplostim and Decitabine in the treatment for thrombocytopenia after HSCT, while Eltrombopag showed significantly better efficacy in the pediatric subgroup compared to elder patients, and the side effects of the drugs are within acceptable range.Trial registration: CRD420251000342.