Romiplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program |
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Authors: | Khellaf Mehdi Michel Marc Quittet Philippe Viallard Jean-François Alexis Magda Roudot-Thoraval Françoise Cheze Stéphane Durand Jean-Marc Lefrère François Galicier Lionel Lambotte Olivier Panelatti Gérard Slama Borhane Damaj Gandhi Sebahoun Gérard Gyan Emmanuel Delbrel Xavier Dhedin Nathalie Royer Bruno Schleinitz Nicolas Rossi Jean-François Mahévas Matthieu Languille Laetitia Bierling Philippe Godeau Bertrand |
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Institution: | Department of Internal Medicine, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-H?pitaux de Paris, Université Paris Est Créteil, Créteil, France. |
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Abstract: | Romiplostim, a thrombopoietic agent with demonstrated efficacy against immune thrombocytopenia (ITP) in prospective controlled studies, was recently licensed for adults with chronic ITP. Only France has allowed romiplostim compassionate use since January 2008. ITP patients could receive romiplostim when they failed to respond to successive corticosteroids, intravenous immunoglobulins, rituximab, and splenectomy, or when splenectomy was not indicated. We included the first 80 patients enrolled in this program with at least 2 years of follow-up. Primary platelet response (platelet count ≥ 50 × 10(9)/L and double baseline) was observed in 74% of all patients. Long-term responses (2 years) were observed in 47 (65%) patients, 37 (79%) had sustained platelet responses with a median platelet count of 106 × 10(9)/L (interquartile range, 75-167 × 10(9)/L), and 10 (21%) were still taking romiplostim, despite a median platelet count of 38 × 10(9)/L (interquartile range, 35-44 × 10(9)/L), but with clinical benefit (lower dose and/or fewer concomitant treatment(s) and/or diminished bleeding signs). A high bleeding score and use of concomitant ITP therapy were baseline factors predicting romiplostim failure. The most frequently reported adverse events were: arthralgias (26%), fatigue (13%), and nausea (7%). Our results confirmed that romiplostim use in clinical practice is effective and safe for severe chronic ITP. This trial was registered at www.clinicaltrials.gov as #NCT01013181. |
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