title: Health-related quality of life in nonsplenectomized immune thrombocytopenia patients receiving romiplostim or medical standard of care.
abstract: Immune thrombocytopenia (ITP) is an autoimmune disorder characterized by platelet destruction and insufficient platelet production. The resulting thrombocytopenia reduces patient health-related quality of life (HRQOL). In a randomized, open-label, 52-week study of non-splenectomized ITP patients treated with romiplostim or medical standard of care (SOC), patients completed the 10-scale ITP-patient assessment questionnaire (PAQ) at the start of the study and after 12, 24, 36, 48,and 52 weeks of treatment. HRQOL changes were examined for all patients in both treatment groups and by responder status, splenectomy status, and after the use of rituximab. Patients in both groups showed marked increases in all HRQOL scales over 52 weeks of treatment.These change scores exceeded the minimally important difference values (a measure of clinical relevance) for most of these scales,especially in responders to treatment. Compared with baseline,patients receiving romiplostim showed statistically significant improvements compared to SOC over 52 weeks for the ITP-PAQ scales of Symptoms, Bother, Activity, Psychological Health, Fear, Overall QOL,and Social QOL. Overall, treatment of ITP was associated with improvement in HRQOL. Patients receiving romiplostim had greater HRQOL improvements than those receiving SOC, but the magnitude ofthe difference is of uncertain clinical benefit.
authors.affiliation: Hematology Department, Massachusetts General Hospital, Boston, MA 02114, USA. kuter.david@MGH.harvard.edu
authors.forename: David J
authors.initials: DJ
authors.lastname: Kuter
authors.forename: Susan D
authors.initials: SD
authors.lastname: Mathias
authors.forename: Mathias
authors.initials: M
authors.lastname: Rummel
authors.forename: Romeo
authors.initials: R
authors.lastname: Mandanas
authors.forename: Aristoteles A
authors.initials: AA
authors.lastname: Giagounidis
authors.forename: Xuena
authors.initials: X
authors.lastname: Wang
authors.forename: Robert R
authors.initials: RR
authors.lastname: Deuson
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D058846:Antibodies, Monoclonal, Murine-Derived / Q000627:therapeutic use; D059026:Diagnostic Self Evaluation; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy / Q000523:psychology* / Q000628:therapy; D011788:Quality of Life; D011961:Receptors, Fc / Q000627:therapeutic use*; D053628:Receptors, Thrombopoietin / Q000819:agonists; D011993:Recombinant Fusion Proteins / Q000627:therapeutic use*; D000069283:Rituximab; D012720:Severity of Illness Index; D013156:Splenectomy; D059039:Standard of Care; D011795:Surveys and Questionnaires; D013926:Thrombopoietin / Q000627:therapeutic use*; D055815:Young Adult
chemical_list: D058846:Antibodies, Monoclonal, Murine-Derived; D007166:Immunosuppressive Agents; D011961:Receptors, Fc; D053628:Receptors, Thrombopoietin; D011993:Recombinant Fusion Proteins; C082218:MPL protein, human; D000069283:Rituximab; D013926:Thrombopoietin; C488777:romiplostim