title: 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.affiliation: Department of Internal Medicine, Centre Hospitalier Universitaire Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris Est Créteil, Créteil, France.
authors.forename: Mehdi
authors.initials: M
authors.lastname: Khellaf
authors.forename: Marc
authors.initials: M
authors.lastname: Michel
authors.forename: Philippe
authors.initials: P
authors.lastname: Quittet
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Viallard
authors.forename: Magda
authors.initials: M
authors.lastname: Alexis
authors.forename: Françoise
authors.initials: F
authors.lastname: Roudot-Thoraval
authors.forename: Stéphane
authors.initials: S
authors.lastname: Cheze
authors.forename: Jean-Marc
authors.initials: JM
authors.lastname: Durand
authors.forename: François
authors.initials: F
authors.lastname: Lefrère
authors.forename: Lionel
authors.initials: L
authors.lastname: Galicier
authors.forename: Olivier
authors.initials: O
authors.lastname: Lambotte
authors.forename: Gérard
authors.initials: G
authors.lastname: Panelatti
authors.forename: Borhane
authors.initials: B
authors.lastname: Slama
authors.forename: Gandhi
authors.initials: G
authors.lastname: Damaj
authors.forename: Gérard
authors.initials: G
authors.lastname: Sebahoun
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Gyan
authors.forename: Xavier
authors.initials: X
authors.lastname: Delbrel
authors.forename: Nathalie
authors.initials: N
authors.lastname: Dhedin
authors.forename: Bruno
authors.initials: B
authors.lastname: Royer
authors.forename: Nicolas
authors.initials: N
authors.lastname: Schleinitz
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Rossi
authors.forename: Matthieu
authors.initials: M
authors.lastname: Mahévas
authors.forename: Laetitia
authors.initials: L
authors.lastname: Languille
authors.forename: Philippe
authors.initials: P
authors.lastname: Bierling
authors.forename: Bertrand
authors.initials: B
authors.lastname: Godeau
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001792:Blood Platelets / Q000187:drug effects; D057176:Compassionate Use Trials; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy*; D011961:Receptors, Fc / Q000627:therapeutic use*; D053628:Receptors, Thrombopoietin / Q000819:agonists*; D011993:Recombinant Fusion Proteins / Q000009:adverse effects / Q000627:therapeutic use*; D012189:Retrospective Studies; D013926:Thrombopoietin / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim