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.