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: James N George, Susan D Mathias, Ronald S Go, Matthew Guo, David H Henry, Roger Lyons, Robert L Redner, Lawrence Rice, Martin R Schipperus
• Journal: British journal of haematology (Br J Haematol) Vol. 144 Issue 3 Pg. 409-15 (Feb 2009) ISSN: 1365-2141 [Electronic] England
• PMID: 19016720 (Publication Type: Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Carrier Proteins
• Receptors, Fc
• Recombinant Fusion Proteins
• Thrombopoietin
• romiplostim

Topics

• Adult
• Aged
• Carrier Proteins (therapeutic use)
• Chronic Disease
• Combined Modality Therapy
• Double-Blind Method
• Female
• Health Status Indicators
• Humans
• Male
• Middle Aged
• Psychometrics
• Purpura, Thrombocytopenic, Idiopathic (drug therapy, psychology, surgery)
• Quality of Life
• Receptors, Fc (therapeutic use)
• Recombinant Fusion Proteins
• Splenectomy
• Thrombopoietin
• Treatment Outcome