Many second-line treatments in children with immune thrombocytopenia (ITP) may improve platelet counts, bleeding symptoms, and health-related quality of life (HRQoL) scores, though timing and effects vary, according to results published in the American Journal of Hematology.

In this prospective, multicenter, observational study conducted by the Pediatric Immune Thrombocytopenia Consortium of North America (ICON), 120 patients were enrolled. The researchers sought to compare treatment outcomes — platelet count, bleeding, and HRQoL utilizing the Kids ITP Tool — between second-line therapies. Rituximab was given to 36% (43/120) of patients, romiplostim to 26% (31/120), eltrombopag to 17% (20/120), oral immunosuppressant agents to 16% (19/120), splenectomy to 3% (4/120), and dapsone to 3% (3/120).

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The mean age of the cohort was 11.0 years, and 39% of patients were male (44/113). A majority of patients (84%) had primary ITP. ITP was chronic in 53% of patients, persistent in 30% of patients, and newly diagnosed in 17% of patients. Patients had undergone a median of 3 prior treatments.

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All treatments increased platelet counts. However, at 6 months, romiplostim appeared to be most effective, resulting in a median platelet count of 160 x 109/L and the highest percentage of patients with complete response (71%; P =.04). After 1 month of treatment, patients taking romiplostim and rituximab experienced reduced skin-related (84% to 48% for romiplostim; P =.01; 81% to 43% for rituximab; P =.004) and nonskin-related bleeding symptoms (58% to 14% for romiplostim; P =.0001; 54% to 17% for rituximab; P =.0006).

All treatments significantly improved HRQoL scores. However, at 1 month, only eltrombopag resulted in a minimal important difference in score improvement, from a median of 69.9 at baseline to 80.8 (P =.0008).

Notable patient attrition occurred at each study time point due to lack of efficacy, side effects, remission, and lack of follow-up.

“Given these findings, it is clear that clinicians should weigh and balance the reasons for treatment when choosing among therapies, as treatments do not necessarily lead to the same outcomes,” the authors concluded.


1.     Grace RF, Shimano KA, Bhat R, et al. Second-line treatments in children with immune thrombocytopenia: effect on platelet count and patient-centered outcomes [published online April 3, 2019]. Am J Hematol. doi:10.1002/ajh.25479