Improved quality of life for romiplostim-treated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo-controlled trials.
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.
George
James N
JN
Colleges of Medicine and Public Health, University of Oklahoma Health Sciences Center, Oklahoma City, OK 73126-0901, USA. james-george@ouhsc.edu
Mathias
Susan D
SD
Go
Ronald S
RS
Guo
Matthew
M
Henry
David H
DH
Lyons
Roger
R
Redner
Robert L
RL
Rice
Lawrence
L
Schipperus
Martin R
MR
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
D002352:Carrier Proteins; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim