title: Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy.
abstract: Romiplostim was effective, safe, and well-tolerated over 6-12 months of continuous treatment in Phase 3 trials in patients with immune thrombocytopenia (ITP). This report describes up to 5 years of weekly treatment with romiplostim in 292 adult ITP patients in a long-term, single-arm, open-label study. Outcome measures included adverse events (including bleeding, thrombosis, malignancy, and reticulin/fibrosis), platelet response (platelet count >50 × 10(9) per litre), and the proportion of patients requiring rescue treatments. Treatment-related serious adverse events were infrequent and did not increase with longer treatment. No new classes of adverse events emerged. Thrombotic events occurred in 6.5% of patients and were not associated with platelet count. Median platelet counts of 50-200 × 10(9) per litre were maintained with stable doses of romiplostim (mean 5-8 μg/kg; generally self-administered at home) throughout the study. A platelet response was achieved at least once by 95% of patients, with a platelet response maintained by all patients on a median 92% of study visits. There was a low rate of bleeding and infrequent need for rescue treatments. In conclusion, this study demonstrated that romiplostim was safe and well-tolerated over 614 patient-years of exposure in ITP patients, and that efficacy was maintained with stable dosing for up to 5 years of continuous treatment.
authors: Massachusetts General Hospital, Boston, MA 02114, USA. kuter.david@MGH.harvard.edu
authors: David J
authors: DJ
authors: Kuter
authors: James B
authors: JB
authors: Bussel
authors: Adrian
authors: A
authors: Newland
authors: Ross I
authors: RI
authors: Baker
authors: Roger M
authors: RM
authors: Lyons
authors: Jeffrey
authors: J
authors: Wasser
authors: Jean-Francois
authors: JF
authors: Viallard
authors: Gail
authors: G
authors: Macik
authors: Mathias
authors: M
authors: Rummel
authors: Kun
authors: K
authors: Nie
authors: Susie
authors: S
authors: Jun
mesh_terms: D000328:Adult; D000368:Aged; D001855:Bone Marrow Diseases / Q000139:chemically induced; D003131:Combined Modality Therapy; D004305:Dose-Response Relationship, Drug; D005260:Female; D006470:Hemorrhage / Q000209:etiology; D006801:Humans; D007279:Injections, Subcutaneous; D008297:Male; D008875:Middle Aged; D009203:Myocardial Infarction / Q000139:chemically induced; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000150:complications / Q000188:drug therapy* / Q000601:surgery; D011961:Receptors, Fc / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins / Q000009:adverse effects / Q000627:therapeutic use*; D012646:Self Administration; D013156:Splenectomy; D013921:Thrombocytopenia / Q000139:chemically induced; D013926:Thrombopoietin / Q000009:adverse effects / Q000627:therapeutic use*; D013927:Thrombosis / Q000139:chemically induced
chemical_list: D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim