학술논문
Rorniplostim safety and efficacy for immune thrombocytopenia in clinical practice: 2-year results of 72 adults in a romiplostim compassionate-use program.
Document Type
Article
Author
Khellaf, Mehdi; Michel, Marc; Quittet, Philippe; Viallard, Jean-Francois; Alexis, Magda; Roudot-Thoraval, Françoise; Cheze, Stéphane; Durand, Jean-Marc; Lefrëre, François; Galicier, Lionel; Lambotte, Olivier; Panelatti, Gerard; Slama, Borhane; Damaj, Gandhi; Sebahoun, Gerard; Gyan, Emmanuel; Delbrel, Xavier; Dhedin, Nathalie; Royer, Bruno; Schleinitz, Nicolas
Source
Subject
*THROMBOCYTOPENIA treatment
*DRUG efficacy
*CONTROL groups
*ADRENOCORTICAL hormones
*IMMUNOGLOBULINS
*RITUXIMAB
*SPLENECTOMY
*
*
*
*
*
*
Language
ISSN
0006-4971
Abstract
Romiplostim, a thrombopoietlc agent with demonstrated efficacy against immune thrombocytopenla (lTP) in prospective controlled studies, was recently licensed for adults with chronic ITP. Only France has allowed romlplostim compassionate use since January 2008. lIP 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 x 1 09/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 x 1 09/L (interquartile range. 75-167 x 1091L), and 10 (21%) were still taking romiplostim. despite a median platelet count of 38 x 109/L (lnterquartile range, 35-44 x 109(L), but with clinical benefit (lower dose and/or fewer concomitant treatment(s) and/or diminshed bleeding signs). A high bleeding score and use of concomitant ITP therapy were baseline factors predicting romiplostlm 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 wwwclinicaltrials.gov as #NCT01013181. [ABSTRACT FROM AUTHOR]