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Romiplostim for chemotherapy induced thrombocytopenia in pediatric cancers
Shivam Bansal1, Aditya Kumar Gupta2, Jagdish Prasad Meena1
1Division of Pediatric Oncology, Department of Pediatrics, All India Institute of Medical Sciences (AIIMS), New Delhi, India.
Insights
Prophylactic romiplostim did not speed platelet recovery in children undergoing chemotherapy but reduced bleeding and transfusion needs. This study highlights its potential in managing chemotherapy-induced thrombocytopenia in pediatric cancer patients.
Area of Science:
- Pediatric Oncology
- Hematology
- Pharmacology
Background:
- Chemotherapy-induced thrombocytopenia (CIT) poses challenges in pediatric cancer care.
- Limited data exists on managing CIT in children.
- Romiplostim, a thrombopoietin-receptor-agonist, shows promise in adults but its pediatric use is undefined.
Purpose of the Study:
- To evaluate the efficacy of prophylactic romiplostim in preventing CIT in pediatric cancer patients.
- To assess romiplostim's impact on platelet recovery, bleeding events, and transfusion requirements.
Main Methods:
- An investigator-initiated, open-label, randomized-controlled trial was conducted in India.
- 93 children (aged 1-14) with AML, NHL, or solid tumors received either romiplostim or standard care.
- Primary outcome: platelet recovery within 3 weeks; secondary outcomes: bleeding, transfusions, and immature platelet fraction (IPF%).
Main Results:
- Platelet recovery rates were similar between romiplostim (91.3%) and control (88.1%) groups.
- No clinically significant bleeding (≥WHO grade 2) occurred in the romiplostim group versus 14% in the control group (P=0.01).
- Romiplostim group had lower median platelet transfusion volume and a significant increase in IPF% on day 16.
Conclusions:
- Prophylactic romiplostim did not accelerate platelet recovery in pediatric patients receiving myelosuppressive chemotherapy.
- Romiplostim was associated with reduced bleeding and lower transfusion requirements.
- The observed increase in IPF% supports romiplostim's mechanism of megakaryocyte stimulation.
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.
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