Shirley Watson

International consensus report on the investigation and management of primary immune thrombocytopenia

Previously published guidelines for the diagnosis and management of primary immune thrombocytopenia (ITP) require updating largely due to the introduction of new classes of therapeutic agents, and a greater understanding of the disease pathophysiology. However, treatment-related decisions still remain principally dependent on clinical expertise or patient preference rather than high-quality clinical trial evidence. This consensus document aims to report on new data and provide consensus-based recommendations relating to diagnosis and treatment of ITP in adults, in children, and during pregnancy. The inclusion of summary tables within this document, supported by information tables in the online appendices, is intended to aid in clinical decision making.

Fatigue in adult patients with primary immune thrombocytopenia

Background:  Patients with primary immune thrombocytopenia (ITP) commonly describe symptoms of fatigue. However, hematologists rarely consider fatigue a manifestation of ITP.

Health-related lifestyle in adults and children with primary immune thrombocytopenia (ITP)

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 ITPspecific 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, closedfield 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 · 10/l, moderate: 20–49 · 10/l, and severe: <20 · 10/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 (44Æ7%) completed surveys were received from 696 (88Æ1%) adults and 94 (11Æ9%) children with primary ITP. The female-to-male ratio was 2Æ2:1, with respondents reporting a 5Æ0-year median (range: 0Æ1–54Æ0 years) duration of disease. Mild, moderate, and severe disease was noted in 57Æ2%, 17Æ5%, and 15Æ4% of patients. Bruising occurred ‘always’ or ‘often’ in 37Æ4% of adults and 56Æ8% of children, while bleeding of similar frequency was noted in 10Æ6% of all patients (Fig 1). Adults were twice as likely as children (89Æ4% vs. 45Æ3%; P < 0Æ001) to have been prescribed an ITP-specific treatment. Children were more likely than adults to experience frustration over activity restrictions (23Æ3% vs. 9Æ5% respectively; P < 0Æ001). However, the impact of primary ITP on healthcare, insurance coverage, and social engagement was

Addendum to corticosteroid side effects and risk for bleeding in immune thrombocytopenic purpura: patient perspectives

To the Editor: We recently published our data from surveys of patients in the Oklahoma immune thrombocytopenic purpura (ITP) Registry and practicing haematologists in the state of Oklahoma which documented a significant difference between ITP patients’ and haematologists’ perspectives about the severity of side effects of corticosteroid treatment and their concern about the risk for serious bleeding (1). In this publication, we listed multiple potential limitations of our study, principally whether our ITP patients were representative of all ITP patients and therefore whether their responses were generalizable. We were also concerned about the accuracy of the patients’ responses, which may have been biased by their recall of past experience, and the heterogeneity of the patient group, which included some patients who’s ITP had resolved, some who’s ITP remained symptomatic, and some who had persistent thrombocytopenia without symptoms. We recently had the opportunity to validate our results with a different patient group, patients who attended the Annual Convention of the ITP Support Association in Edinburgh, Scotland on 16 May 2009. Patients attending the Convention, described here as UK patients, were given the same questionnaire as patient participants in Oklahoma, described as US patients. The results are presented in the Table 1. There was no significant difference between the patient groups regarding their lowest platelet count or the duration they were treated with corticosteroids. In both groups, 80% of patients reported that their lowest platelet count had been less than 10 000 ⁄ lL and approximately one-third of the patients had received corticosteroid treatment for longer than 12 months. The questionnaire asked patients how bothered they were by 18 different side effects of corticosteroid treatment. They graded each side effect according to a four choice scale: ‘bothered a lot’, ‘bothered

Cognitive symptoms are common in immune thrombocytopenia and associate with autonomic symptom burden.

Objectives:  People with adult immune thrombocytopenia (ITP) are commonly thought to have an isolated blood disorder, but many also describe memory and concentration problems. Cognitive impairment commonly associates with autonomic dysfunction. Here, we quantified cognitive symptoms in a large cohort of patients with ITP compared with controls and explored the relationship with autonomic symptoms.

A United Kingdom Immune Thrombocytopenia (ITP) Forum review of practice: thrombopoietin receptor agonists

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Depression in adult patients with primary immune thrombocytopenia.

This work was supported by PCORI Grant Number CDRN 1501-26498. The authors thank the members of the Vanderbilt-Meharry Center of Excellence in Sickle Cell Disease for their thoughtful and helpful comments in reviewing the manuscript; Bertha Davis for regulatory matters; Brittany L. Myers, DNP, RN for support with patient recruitment; and Natasha Dean for assistance with preparation of this manuscript.

Whole exome sequencing in patients with inherited thrombocytopenia and excessive bleeding is an efficient way to identify genetic variants in known and novel genes

Reference EPFL-CONF-212539doi:10.1111/jth.12993View record in Web of Science Record created on 2015-09-28, modified on 2017-05-12