Romiplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program.

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.

• Authors: Mehdi Khellaf, Marc Michel, Philippe Quittet, Jean-François Viallard, Magda Alexis, Françoise Roudot-Thoraval, Stéphane Cheze, Jean-Marc Durand, François Lefrère, Lionel Galicier, Olivier Lambotte, Gérard Panelatti, Borhane Slama, Gandhi Damaj, Gérard Sebahoun, Emmanuel Gyan, Xavier Delbrel, Nathalie Dhedin, Bruno Royer, Nicolas Schleinitz, Jean-François Rossi, Matthieu Mahévas, Laetitia Languille, Philippe Bierling, Bertrand Godeau
• Journal: Blood (Blood) Vol. 118 Issue 16 Pg. 4338-45 (Oct 20 2011) ISSN: 1528-0020 [Electronic] United States
• PMID: 21832276 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)

Chemical References

• Receptors, Fc
• Receptors, Thrombopoietin
• Recombinant Fusion Proteins
• Thrombopoietin
• romiplostim

Topics

• Adult
• Aged
• Aged, 80 and over
• Blood Platelets (drug effects)
• Compassionate Use Trials
• Female
• Humans
• Male
• Middle Aged
• Platelet Count
• Purpura, Thrombocytopenic, Idiopathic (drug therapy)
• Receptors, Fc (therapeutic use)
• Receptors, Thrombopoietin (agonists)
• Recombinant Fusion Proteins (adverse effects, therapeutic use)
• Retrospective Studies
• Thrombopoietin (adverse effects, therapeutic use)
• Treatment Outcome
• Young Adult