title: Improved quality of life for romiplostim-treated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo-controlled trials.
abstract: Health-related quality of life (HRQoL) is a major concern for adults with chronic immune thrombocytopenic purpura (ITP) due to the symptoms associated with the disease and its treatment. This study utilized the ITP-patient assessment questionnaire (ITP-PAQ), a specialized HRQoL questionnaire for ITP, to investigate the humanistic burden of ITP and the impact of romiplostim therapy on HRQoL in two, placebo-controlled, phase 3 clinical trials of splenectomized and non-splenectomized patients. ITP-PAQ was self-administered to ITP patients at baseline, and weeks 4, 12 and 24 of treatment. Splenectomized patients had lower baseline HRQoL scores than non-splenectomized patients in seven of 10 scales (P < 0.05). After 24 weeks of romiplostim therapy, splenectomized patients showed significant improvements over placebo in four of 10 ITP-PAQ Scales (Symptoms, P = 0.0337; Bother, P = 0.0126; Social Activity, P = 0.0145; and Women's Reproductive Health, P = 0.0184). Non-splenectomized patients demonstrated significant improvement over placebo in the Activity Scale (P = 0.0458). Data pooled from the two trials, adjusted for splenectomy status, showed significant improvement for romiplostim-treated patients in six scales; Symptoms, Bother, Activity, Fear, Social Activity and Women's Reproductive Health. These results suggest that adult patients with chronic ITP have improved HRQoL following romiplostim therapy.
authors.affiliation: Colleges of Medicine and Public Health, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73126-0901, USA. james-george@ouhsc.edu
authors.forename: James N
authors.initials: JN
authors.lastname: George
authors.forename: Susan D
authors.initials: SD
authors.lastname: Mathias
authors.forename: Ronald S
authors.initials: RS
authors.lastname: Go
authors.forename: Matthew
authors.initials: M
authors.lastname: Guo
authors.forename: David H
authors.initials: DH
authors.lastname: Henry
authors.forename: Roger
authors.initials: R
authors.lastname: Lyons
authors.forename: Robert L
authors.initials: RL
authors.lastname: Redner
authors.forename: Lawrence
authors.initials: L
authors.lastname: Rice
authors.forename: Martin R
authors.initials: MR
authors.lastname: Schipperus
mesh_terms: D000328:Adult; D000368:Aged; D002352:Carrier Proteins / Q000627:therapeutic use*; D002908:Chronic Disease; D003131:Combined Modality Therapy; D004311:Double-Blind Method; D005260:Female; D006305:Health Status Indicators; D006801:Humans; D008297:Male; D008875:Middle Aged; D011594:Psychometrics; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000523:psychology* / Q000601:surgery; D011788:Quality of Life; D011961:Receptors, Fc / Q000627:therapeutic use*; D011993:Recombinant Fusion Proteins; D013156:Splenectomy; D013926:Thrombopoietin; D016896:Treatment Outcome
chemical_list: D002352:Carrier Proteins; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim