Utility and Safety of Romiplostim in Pediatric Allogeneic Stem Cell Transplantation.

BACKGROUND: The use of romiplostim, a thrombopoietin agonist, has increased in the last decade for the treatment of immune mediated thrombocytopenia and severe aplastic anemia. Its utility has been explored in the management of delayed platelet engraftment and secondary platelet failure during stem cell transplant (SCT), but its use has remained largely anecdotal in pediatric allogeneic SCT. METHODS: In this single centre, retrospective study we report the largest pediatric SCT cohort use of romiplostim. RESULTS: Romiplostim was used in 17 children for several indications, principally including poor graft function (PGF) and immune-mediated cytopenia (IMC), including multi-lineage cytopenia. The overall response rate (ORR) was 76.5% and the median time to achieve OR was 42 days. No toxicity was observed with romiplostim including marrow fibrosis, clonal evolution and thrombosis with a median follow-up of 18 months. Romiplostim averted the need for second allogeneic SCT in two patients with late graft failure and the need for stem cell boost (SCB) in three patients. CONCLUSION: We propose that romiplostim can be safely used in the cytopenia in pediatric SCT to good effect.