Impact of chronic Immune Thrombocytopenic Purpura (ITP) on health-related quality of life: a conceptual model starting with the patient perspective.

BACKGROUND: Immune thrombocytopenic purpura (ITP), a condition characterized by autoimmune-mediated platelet destruction and suboptimal platelet production, is associated with symptoms such as bruising, epistaxis, menorrhagia, mucosal bleeding from the gastrointestinal and urinary tracts and, rarely central nervous system bleeding. The aim of this research is to develop a conceptual model to describe the impact of ITP and its treatment on patients' health-related quality of life (HRQoL). METHODS: A literature search and focus groups with adult ITP patients were conducted to identify areas of HRQoL affected by ITP. Published literature was reviewed to identify key HRQoL issues and existing questionnaires used to assess HRQoL. Focus group transcripts were reviewed, and common themes were extracted by grouping conceptual categories that described the impact on HRQoL. RESULTS: The literature synthesis and themes from the focus group data suggest that decreased platelet counts, disease symptoms, and treatment side effects influence multiple domains of HRQoL for ITP patients. Key areas affected by ITP and its treatments include emotional and functional health, work life, social and leisure activities, and reproductive health. CONCLUSION: ITP affects various areas of HRQoL. This conceptual model will help inform the evaluation of therapeutic strategies for ITP.

Treatment of chronic immune thrombocytopenic purpura: the patients' perspective.

BACKGROUND: The 1996 ASH guidelines recommend glucocorticoids and splenectomy as standard treatment of chronic immune thrombocytopenic purpura (ITP). We sought to find out how many German ITP-patients were treated according to these guidelines and whether high-cost treatments were offered to the patients. METHODS: We handed out a questionnaire at two self-help group meetings in 2004 and 2005 and to all patients who contacted the ITP self-help group until the end of 2005. RESULTS: Eighty-five questionnaires were evaluated. Age (median 34 yr) and gender distribution (38% male, 62% female) were similar to other surveys. Median duration of disease was 5.2 yr. Seventy-five percent had platelets <20,000/microL at the time of diagnosis. Twenty-four percent still had <20,000 platelets/microL at the time of this survey. Forty-two percent had oropharyngeal mucosal bleeds, 28% gastrointestinal or urological bleeds, 11% bleedings in the eye with visual impairment or intracerebral bleeds. 96% had received a trial of glucocorticoid therapy. Seventy-five percent of the patients treated with glucocorticoids perceived this treatment as particularly bothersome. Seventy-five percent of the patients with low platelet count still had their spleen. Complementary and alternative medical treatments had been used by 46% of the patients. Only 33% of the patients had ever heard of rituximab. CONCLUSION: Despite literature suggesting that patients wish to be well informed this survey shows that chronic ITP patients know little about their disease and the various treatment modalities. This and the frequent use of complementary and alternative medicines reflects inadequate communication between doctors and ITP patients.

A disease-specific measure of health-related quality of life for use in adults with immune thrombocytopenic purpura: its development and validation.

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]. CONCLUSION: 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.