Randomized trial of romiplostim for chemotherapy-induced thrombocytopenia in pediatric cancers

Chemotherapy-induced thrombocytopenia (CIT) comprises cancer care. Romiplostim, a thrombopoietin-receptor agonist, has shown efficacy in adults with CIT, but its role in pediatric patients is unclear. A randomized trial at the All India Institute of Medical Science (New Delhi) evaluated prophylactic romiplostim for prevention of CIT in children aged 1-14 years receiving myelosuppresive chemotherapy for acute myeloid leukemia, non-Hodgkin lymphoma, or solid tumors. Patients were randomized to receive romiplostim of standard-of care (SOC). The primary outcome was platelet recovery (≥ 100 x 10⁹/L and transfusion-free for 72 hours) within 3 weeks. A total of 93 patients (median age 3 years; IQR 2-5) were randomized: 47 to romiplostim and 46 to SOC. Platelet recovery occurred in 91.3% versus 88.1% (p=0.73). Clinically significant bleeding (≥ WHO grade 2) occurred in none of the patients in the romiplostim group and 14% of the patients in the SOC group (p=0.01). Median platelet transfusion volume was lower with romiplostim (19.3 versus 29.6 mL/kg; p=0.06). Romiplostim significantly increased immature platelet fraction percentage (IPF%) on day 16 from baseline (258% versus 47.7%; p=0.016). No thrombotic events occurred. The authors conclude that compared with SOC, romiplostim did not improve platelet recovery in pediatric patients receiving but was associated with reduced bleeding, lower transfusion requirements, and IPF% increment on day 16.