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Background <p>Chemotherapy-induced-thrombocytopenia (CIT) compromises cancer care, but data on its management in children is scarce. Romiplostim, a thrombopoietin-receptor-agonist, has shown efficacy in adults with CIT, though its role in paediatric patients remains undefined.</p> Methods <p>We conducted an investigator-initiated, open-label, randomized-controlled-trial at a tertiary center in India to evaluate prophylactic romiplostim for prevention of CIT in children aged 1-14 years with acute myeloid leukemia (AML), non-Hodgkin’s lymphoma (NHL), and solid-tumors receiving myelosuppressive chemotherapy. Patients were randomly assigned (1:1) to receive romiplostim (3 μg/kg-subcutaneously) or standard-care. The primary outcome was platelet recovery (≥100 × 10⁹/L and transfusion-free for 72 hours) within 3-weeks. Secondary outcomes included bleeding, transfusion requirements, chemotherapy delays, and immature platelet fraction percentagecIPF%) trends.</p> Results <p>From March 2023 to October 2024, 93 patients (median age, 3 years; interquartile range [IQR], 2–5) were randomized (47 intervention, 46 control). Platelet recovery occurred in 91.3% and 88.1%, respectively (risk difference, 3.2 percentage points; 95% confidence interval [CI], –9.5 to 15.9; P = 0.73). Clinically significant bleeding (≥WHO grade 2) occurred in none of 46 patients in romiplostim group and 6 of 43 (14%) in control group (absolute risk difference,–14 percentage points; 95% CI, –27 to –2; P = 0.01). Median platelet transfusion volume was lower with romiplostim (19.3 vs. 29.6 mL/kg; median difference, –10.3 mL/kg; 95% CI, –21.5 to 0.9; P = 0.06, rank-biserial correlation= 0.35). Romiplostim significantly increased IPF% on day-16 from baseline (258% <i>vs</i>. 47.7%, p = 0.016, Cohen’s h = 2.41). No thrombotic events occurred.</p> Conclusions <p>Prophylactic romiplostim didn’t hasten platelet recovery in pediatric patients receiving myelosuppressive chemotherapy but was associated with reduced bleeding and had lower transfusion requirements. There was a significant relative immature platelet fraction percentage (IPF)% increment on day 16 in the romiplostim arm in comparison to the control arm, reinforcing the biological plausibility of romiplostim’s effect on megakaryocyte stimulation.</p> Trial registration <p>Clinical Trials Registry of India: CTRI/2023/02/049906.</p>

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Romiplostim for chemotherapy induced thrombocytopenia in pediatric cancers

  • 期刊论文

  • 发表日期：

    2026年7月25日

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  • Shivam Bansal,
  • Aditya Kumar Gupta,
  • Jagdish Prasad Meena,
  • Tushar Sehgal,
  • Rachna Seth

摘要

Background

Chemotherapy-induced-thrombocytopenia (CIT) compromises cancer care, but data on its management in children is scarce. Romiplostim, a thrombopoietin-receptor-agonist, has shown efficacy in adults with CIT, though its role in paediatric patients remains undefined.

Methods

We conducted an investigator-initiated, open-label, randomized-controlled-trial at a tertiary center in India to evaluate prophylactic romiplostim for prevention of CIT in children aged 1-14 years with acute myeloid leukemia (AML), non-Hodgkin’s lymphoma (NHL), and solid-tumors receiving myelosuppressive chemotherapy. Patients were randomly assigned (1:1) to receive romiplostim (3 μg/kg-subcutaneously) or standard-care. The primary outcome was platelet recovery (≥100 × 10⁹/L and transfusion-free for 72 hours) within 3-weeks. Secondary outcomes included bleeding, transfusion requirements, chemotherapy delays, and immature platelet fraction percentagecIPF%) trends.

Results

From March 2023 to October 2024, 93 patients (median age, 3 years; interquartile range [IQR], 2–5) were randomized (47 intervention, 46 control). Platelet recovery occurred in 91.3% and 88.1%, respectively (risk difference, 3.2 percentage points; 95% confidence interval [CI], –9.5 to 15.9; P = 0.73). Clinically significant bleeding (≥WHO grade 2) occurred in none of 46 patients in romiplostim group and 6 of 43 (14%) in control group (absolute risk difference,–14 percentage points; 95% CI, –27 to –2; P = 0.01). Median platelet transfusion volume was lower with romiplostim (19.3 vs. 29.6 mL/kg; median difference, –10.3 mL/kg; 95% CI, –21.5 to 0.9; P = 0.06, rank-biserial correlation= 0.35). Romiplostim significantly increased IPF% on day-16 from baseline (258% vs. 47.7%, p = 0.016, Cohen’s h = 2.41). No thrombotic events occurred.

Conclusions

Prophylactic romiplostim didn’t hasten platelet recovery in pediatric patients receiving myelosuppressive chemotherapy but was associated with reduced bleeding and had lower transfusion requirements. There was a significant relative immature platelet fraction percentage (IPF)% increment on day 16 in the romiplostim arm in comparison to the control arm, reinforcing the biological plausibility of romiplostim’s effect on megakaryocyte stimulation.

Trial registration

Clinical Trials Registry of India: CTRI/2023/02/049906.