Primary immune thrombocytopenia (ITP) is an autoimmune disease characterized by a decreased peripheral blood platelet count in the absence of a demonstrable cause, leading to an increased susceptibility to bleeding events (Provan et al, 2010). To avoid such events, patients with primary ITP have historically been subject to considerable lifestyle restrictions. Investigation into the impact of these restrictions, of highly visible bleeding manifestations, and of patient concerns over the side effects of treatment has only recently begun (Guidry et al, 2009; Zehnder et al, 2010). Emerging research has, however, highlighted a considerable burden posed by the disease. For example, McMillan et al (2008) reported lower aggregate health-related quality of life (HRQoL) scores among 73 adults with primary ITP than in patients with hypertension, arthritis, or cancer. Such research has importantly been coupled with the development and validation of two ITP-specific HRQoL assessment instruments: the ITP-Patient Assessment Questionnaire (ITP-PAQ) and the Kids’ ITP Tools (KIT) (Klaassen et al, 2007; George et al, 2009).
To complement ongoing research in HRQoL, the ITP Support Association conducted a questionnaire-based survey to identify health-related lifestyle concerns among adults and children with primary ITP in the United Kingdom.
In collaboration with ITP specialists, a 43-question, closed-field questionnaire was developed, addressing bruising and bleeding frequency; disease management; social engagement; work and school performance; and recreational activities. Questionnaires were mailed to members of the ITP Support Association (N = 1767). Results from returned questionnaires were stratified by age [adults (>16 years) and children] and platelet count at last follow-up, which served as a surrogate marker for disease severity (mild: >50 × 109/l, moderate: 20–49 × 109/l, and severe: <20 × 109/l). Differences between groups were evaluated using generalized Cochrane–Mantel–Haenszel tests and were restricted to fields completed by at least three-quarters of responders.
In total, 790 (44·7%) completed surveys were received from 696 (88·1%) adults and 94 (11·9%) children with primary ITP. The female-to-male ratio was 2·2:1, with respondents reporting a 5·0-year median (range: 0·1–54·0 years) duration of disease. Mild, moderate, and severe disease was noted in 57·2%, 17·5%, and 15·4% of patients. Bruising occurred ‘always’ or ‘often’ in 37·4% of adults and 56·8% of children, while bleeding of similar frequency was noted in 10·6% of all patients (Fig 1). Adults were twice as likely as children (89·4% vs. 45·3%; P < 0·001) to have been prescribed an ITP-specific treatment.
Children were more likely than adults to experience frustration over activity restrictions (23·3% vs. 9·5% respectively; P < 0·001). However, the impact of primary ITP on healthcare, insurance coverage, and social engagement was more pronounced among adults; 31·3% of adults reported having surgery delayed due to a low platelet count, and 30·2% experienced difficulty obtaining travel insurance. Although the incidence of bleeding manifestations were comparable between men and women, significant gender disparities were noted with regard to suspicion of physical violence (women: 7·1% vs. men: 1·6%; P = 0·001) and efforts at bruise concealment (women: 13·5% vs. men: 3·2%; P < 0·001) (Table I). Notably, 12·5% of all patients with primary ITP reported ‘always’ or ‘often’ missing work or school due to fatigue. These absences were not significantly associated with disease severity (P = 0·301).
This study represents one of the largest HRQoL investigations in primary ITP to date and strengthens recurrent reports of fatigue in patients. A pathological basis for its onset has not been determined and warrants further investigation (Zehnder et al, 2010). The results of our study further demonstrate the need for HRQoL metrics to incorporate the effects of treatments on fatigue, bruising, and activity restriction. The ITP-PAQ and KIT do so, but our findings suggest that increased weighting of bruising and activity restriction may be merited in women and children respectively.
The reported difficulty of adults in securing travel insurance prompts concern. Population-based studies have revealed a low incidence of major bleeding events in adults with primary ITP (Frederiksen & Schmidt, 1999). Dissemination of this information to insurers may be facilitated by patient support groups, which may help to make access to coverage more straightforward and affordable.
Finally, reports of delayed surgery in one-third of adults with primary ITP highlights a need for improved coordination of care between surgeons and haematologists. Surgery may often be safely performed in thrombocytopenic patients (Provan et al, 2010); in this respect, an international consensus group of ITP specialists has recently published a table of recommended platelet counts for a variety of procedures (http://bloodjournal.hematologylibrary.org/cgi/data/blood-2009-06-225565/DC1/8).
While this large-scale survey provides useful insight into health-related lifestyle concerns among patients with primary ITP, its limitations warrant consideration. First, the questionnaire utilized was designed as a descriptive tool and is not a validated instrument. Second, the proportion of respondents (44·7%) was low, raising questions about the representativeness of the sample. It should be noted, however, that membership to the ITP Support Association was not restricted to patients with primary ITP; spouses and parents of patients comprised a minority population within the group. Although the exact number of non-patient members at the time of the survey was not known, it is reasonable to assume that the proportion of patient responders was significantly higher than 45%. Third, our use of last platelet count as a proxy for disease severity did not account for disease progression or treatment effects over time and may have biased our observation of a non-significant association between disease severity and fatigue.
Despite these limitations, the results of our health-related lifestyle survey of patients with primary ITP in the United Kingdom successfully highlight several avenues for research, including demographically-dependent variable weighting in HRQoL metrics and the pathogenesis of primary ITP associated fatigue, while uncovering the need for greater dialogue between both patient support groups and insurers and haematologists and surgeons.