title: Romiplostim Treatment in Adults with Immune Thrombocytopenia of Varying Duration and Severity.
abstract: Romiplostim is recommended for the second- and third-line treatment of primary immune thrombocytopenia (ITP). We conducted a large, single-arm study (clinicaltrials.gov; NCT00508820) with broad entry criteria to evaluate the safety of romiplostim in adult ITP. Patients (n = 407) with ITP lasting 0.03-57.14 years and low platelet counts (median 14.0 × 10 9 /l) or uncontrolled bleeding received romiplostim for up to 4 years. The rates of treatment-related, serious adverse events, serious hemorrhage events, thromboembolic events and fatal events were similar to those reported in previous romiplostim trials (0.2, 0.4, 0.2 and 0.1/100 patient-weeks, respectively). Bone marrow reticulin was observed in 4 patients, but biopsies were not routinely performed so the true incidence of this event cannot be determined. Type I collagen (nonserious, unrelated) was reported in 1 patient who likely had myelodysplastic syndrome. No new class of adverse events was reported. Platelet responses were achieved by >90% of the patients, typically within 1-2 weeks of the initiation of romiplostim treatment. From week 8, median platelet counts were >100 × 10 9 /l; 47% of the patients received rescue medications (the use decreased over time). This study confirms and extends the tolerability/efficacy findings of previous romiplostim clinical studies. It was performed on a large ITP population, which is likely more representative of clinical practice.
authors.forename: Ann
authors.initials: A
authors.lastname: Janssens
authors.forename: Michael
authors.initials: M
authors.lastname: Tarantino
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Bird
authors.forename: Maria Gabriella
authors.initials: MG
authors.lastname: Mazzucconi
authors.forename: Ralph V
authors.initials: RV
authors.lastname: Boccia
authors.forename: Maria Fernanda López
authors.initials: MF
authors.lastname: Fernández
authors.forename: Tomas
authors.initials: T
authors.lastname: Kozak
authors.forename: Michael
authors.initials: M
authors.lastname: Steurer
authors.forename: Peter te
authors.initials: Pt
authors.lastname: Boekhorst
authors.forename: Kerry
authors.initials: K
authors.lastname: Dillingham
authors.forename: Georg
authors.initials: G
authors.lastname: Kreuzbauer
authors.forename: Paul
authors.initials: P
authors.lastname: Woodard
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D005260:Female; D006470:Hemorrhage / Q000097:blood / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000097:blood / Q000188:drug therapy*; D011961:Receptors, Fc / Q000008:administration & dosage*; D011993:Recombinant Fusion Proteins / Q000008:administration & dosage* / Q000009:adverse effects; D012720:Severity of Illness Index; D013926:Thrombopoietin / Q000008:administration & dosage* / Q000009:adverse effects; D013997:Time Factors
chemical_list: D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim