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The Effectiveness and Safety of the Thrombopoetin Agonist Romiplostim in the Treatment of Adults with Chronic Idiopathic Thrombocytopenic Purpura

14 August 2010


Background: The treatment of chronic idiopathic thrombocytopenic purpura (ITP) has been a challenge for many hematologists and clinicians. Since the development of thrombopoietin (TPO) in 1994 there has been a new surge of research attempting to boost the platelet production potential in patients affected by this disorder. This new attempt is headed by TPO agonist medications, one of them being romiplostim.

Methods: An exhaustive search for studies analyzing the efficacy and safety of romiplostim in the treatment of chronic ITP was conducted. Studies must have had patients with a mean platelet count of 30 x 109/L and were otherwise medically uncompromised.

Results: Four studies were selected; two randomized control studies and two cohort studies were selected. Study A had two phases in their study. Phase I revealed that the target goal was not reached in all but one of the patients who received lower dose group (doses less than 1μg/kg). In Phase II 16 patients evaluated in the 1 and 3 μg/kg cohorts, 10 had reached the target range while 2, who were from the 3 μg/kg cohort, had even exceeded this range. Study Bdemonstrated that the overall platelet response was 79% (33/42) in splenectomized patients and 88% (36/41) in non-splenectomized patients receiving romiplostim. Study C determined that the most effective way to achieve the target platelet response was to convert the dosage based on patient weight. The researchers in Study D determined that the best starting dose of romiplostim in a phase III trial for Japanese adults with chronic ITP was 3 μg/kg.

Conclusion: The new addition of romiplostim in the treatment of chronic ITP in these studies were effective in raising platelet counts while having only mild to moderate side effects in the majority of the patients. Serious adverse events were not attributed to the use of romiplostim.


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