Romiplostim shows potential for treating chemo-induced thrombocytopenia in paediatric cancers

In children receiving myelosuppressive chemotherapy for cancer, prophylactic treatment with romiplostim helps reduce bleeding and the need for blood transfusion despite having no effect on the speed of platelet recovery, as shown in a study.

The study included 93 paediatric patients ages 1–14 years (median age 3 years) who were receiving myelosuppressive chemotherapy for acute myeloid leukaemia (AML), non-Hodgkin’s lymphoma (NHL), or solid tumour. These patients were randomly assigned to receive romiplostim at 3 μg/kg (n=47) or standard care (n=46). Treatment was administered subcutaneously.

The primary outcome was platelet recovery, defined as ≥100 × 10⁹/L and transfusion-free for 72 h, within 3 weeks. Secondary outcomes included bleeding, transfusion requirements, chemotherapy delays, and immature platelet fraction percentage (IPF%) trends.

Within 3 weeks, platelet recovery occurred in 91.3 percent of patients in the romiplostim group vs 88.1 percent in the control group. The risk difference of 3.2 percentage points (95 percent confidence interval [CI], –9.5 to 15.9) did not reach significance (p=0.73).

However, results for most secondary outcomes favoured romiplostim. None of the patients in the romiplostim group experienced clinically significant bleeding (≥WHO grade 2) as compared with 14 percent in the control group (absolute risk difference, –14 percentage points, 95 percent CI, –27 to –2; p=0.01). The romiplostim group had numerically lower median platelet transfusion volume (19.3 vs 29.6 mL/kg; median difference, –10.3 mL/kg, 95 percent CI, –21.5 to 0.9; p=0.06). Meanwhile, IPF% significantly increased from baseline to day 16 in the romiplostim vs the control group (258 percent vs 47.7 percent; p=0.016).

There were no thrombotic events reported.

The findings reinforce the biological plausibility of romiplostim’s effect on megakaryocyte stimulation.