Effect of Romiplostim in Refractory ITP with Possibility of Maintaining Response Without Treatment – Case Report
This case report from the United States features an ideal therapeutic response to romiplostim in a patient with refractory immune thrombocytopenia (ITP). After several years of successful therapy, the medication could even be gradually discontinued while maintaining the therapeutic response.
Introduction
Primary immune thrombocytopenia is characterized by a low platelet count and increased tendency to bleed. The destruction of platelets coupled with low production, mediated by autoantibodies, can be managed in most patients either by first-line treatments such as corticosteroids or intravenous immunoglobulins (IVIG), or by second-line treatments, which include thrombopoietin receptor agonists (TPO-RA) like romiplostim for those with insufficient response. These agents can achieve sustained, long-lasting therapeutic responses without clinically significant adverse effects.
Case Description
A 44-year-old woman was monitored for refractory ITP. She initially presented in 2004 with skin purpura and menorrhagia. Her blood count showed isolated thrombocytopenia (7 × 109/l). In the first year after the ITP diagnosis, her response to various treatment types was mostly transient. She received high doses of dexamethasone, IVIG, rituximab, and underwent a splenectomy.
Romiplostim was initiated in August 2005 as part of a clinical trial at a dose of 7 μg/kg/week, which she received over approximately 50 weeks. The dose was subsequently reduced, which the patient tolerated well. By 2007, she was on a dose of 3 μg/kg/week, and continued this regimen for another 2 years as part of an ongoing phase of the clinical trial. Due to the sustained response, her hematologist began extending the intervals between romiplostim administrations in 2010, with her last dose in October 2010. Her platelet count has remained above 150 × 109/l without further treatment.
Conclusion
This case report, published in 2012, confirmed the very good effect of romiplostim in a patient with refractory ITP and exhausted previous treatment options. It also demonstrated that romiplostim offers, even for such complicated and heavily pre-treated patients, not only the potential to achieve a satisfactory response but also the possibility of completely discontinuing treatment, marking a whole new chapter in the era of treatment for the chronic and often refractory form of ITP.
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Source: Baxley A. A., George J. N., Terrell D. et al. A case report of long-term complete remission following cessation of romiplostim dosing in a previously severe ITP patient. ASCO Annual Meeting 2012, abstract e17001. J Clin Oncol 2012; 30 (15_suppl.): e17001.
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