title: A disease-specific measure of health-related quality of life for use in adults with immune thrombocytopenic purpura: its development and validation.
abstract: BACKGROUND
  No validated disease-specific measures are available to assess health-related quality of life (HRQoL) in adult subjects with immune thrombocytopenic purpura (ITP). Therefore, we sought to develop and validate the ITP-Patient Assessment Questionnaire (ITP-PAQ) for adult subjects with ITP.
  
  
  METHODS
  Information from literature reviews, focus groups with subjects, and clinicians were used to develop 50 ITP-PAQ items. Factor analyses were conducted to develop the scale structure and reduce the number of items. The final 44-item ITP-PAQ, which includes ten scales [Symptoms (S), Bother-Physical Health (B), Fatigue/Sleep (FT), Activity (A), Fear (FR), Psychological Health (PH), Work (W), Social Activity (SA), Women's Reproductive Health (RH), and Overall (QoL)], was self-administered to adult ITP subjects at baseline and 7-10 days later. Test-retest reliability, internal consistency reliability, construct and known groups validity of the final ITP-PAQ were evaluated.
  
  
  RESULTS
  Seventy-three subjects with ITP completed the questionnaire twice. Test-retest reliability, as measured by the intra-class correlation, ranged from 0.52-0.90. Internal consistency reliability was demonstrated with Cronbach's alpha for all scales above the acceptable level of 0.70 (range: 0.71-0.92), except for RH (0.66). Construct validity, assessed by correlating ITP-PAQ scales with established measures (Short Form-36 v.1, SF-36 and Center for Epidemiologic Studies Depression Scale, CES-D), was demonstrated through moderate correlations between the ITP-PAQ SA and SF-36 Social Function scales (r = 0.67), and between ITP-PAQ PH and SF-36 Mental Health Scales (r = 0.63). Moderate to strong inter-scale correlations were reported between ITP-PAQ scales and the CES-D, except for the RH scale. Known groups validity was evaluated by comparing mean scores for groups that differed clinically. Statistically significant differences (p < 0.01) were observed when subjects were categorized by treatment status [S, FT, B, A, PH, and QoL, perceived effectiveness of ITP treatment [S], and time elapsed since ITP diagnosis [PH].
  
  
  CONCLUSIONS
  Results provide preliminary evidence of the reliability and validity of the ITP-PAQ in adult subjects with ITP. Further work should be conducted to assess the responsiveness and to estimate the minimal clinical important difference of the ITP-PAQ to more fully understand the impact of ITP and its treatments on HRQoL.
authors.affiliation: Ovation Research Group, 188 Embarcadero, San Francisco, CA 94105, USA. smathias@ovation.org
authors.forename: Susan D
authors.initials: SD
authors.lastname: Mathias
authors.forename: James B
authors.initials: JB
authors.lastname: Bussel
authors.forename: James N
authors.initials: JN
authors.lastname: George
authors.forename: Robert
authors.initials: R
authors.lastname: McMillan
authors.forename: Gary J
authors.initials: GJ
authors.lastname: Okano
authors.forename: Janet L
authors.initials: JL
authors.lastname: Nichol
mesh_terms: D000328:Adult; D005163:Factor Analysis, Statistical; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011696:Purpura, Thrombocytopenic / Q000188:drug therapy / Q000503:physiopathology / Q000523:psychology*; D011788:Quality of Life; D015203:Reproducibility of Results; D013156:Splenectomy; D011795:Surveys and Questionnaires