Abstract Number: PB1336
Meeting: ISTH 2020 Congress
Background: Romiplostim is a thrombopoietin receptor agonist (TPO-RA) which has recently been approved for treatment of immune thrombocytopenic purpura (ITP) in children.
Aims: We tried to investigate the efficacy and safety of romiplostim in a heterogeneous group of children with persistent and chronic ITP and acquired aplastic anemia.
Methods: We enrolled 14 patients with the following diagnoses: chronic ITP (n=10), persistent ITP (n=3) and acquired aplastic anemia (n=1). Romiplostim dose was adjusted according to the issued treatment guideline (1). The patients were followed for 26 weeks.
Results: Mean baseline platelet count was 26714± 15774 (range 6000-49000). The mean treatment duration was 22.2± 5.5 weeks (range 8-26 weeks). Totally, 63% ± 24.7% (range 12.5-95.2%) of the patients showed partial or complete response (Table 1). The overall response was comparable in the 3 treatment category (P=0.71). Figure-1 demonstrates the trend of treatment response in the 3 diagnosis categories during the first 21 weeks of the treatment course.
There was no severe adverse effect during the intervention period. One patient reported headache and body pain in the first week of treatment which was treated with oral analgesic.
Conclusions: We observed that about 65% of patients with chronic or persistent ITP responded to romiplostim which is very close to 73% response rate reported by the Pediatric ITP Consortium of North America (ICON2) study (2). Although romiplostim has not been approved for treatment of severe aplastic anemia, we observed that our case showed partial response in 42% of observations. A larger longitudinal study is advised to elucidate the durability of response and safety of this drug in patients with ITP and aplastic anemia.
To cite this abstract in AMA style:Bordbar M, Karimi M. Efficacy and Safety of Romiplostim in Children with Chronic and Persistent Immune Thrombocytopenic Purpura [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/efficacy-and-safety-of-romiplostim-in-children-with-chronic-and-persistent-immune-thrombocytopenic-purpura/. Accessed November 28, 2023.
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