Dataset Information

Patient-reported treatment burden of chronic immune thrombocytopenia therapies.

ABSTRACT: Chronic immune thrombocytopenia (ITP) is a debilitating autoimmune disorder that causes a reduction in blood platelets and increased risk of bleeding. ITP is currently managed with various pharmacologic therapies and splenectomy.This study was conducted to assess patient perceived and reported treatment side effects, as well as the perceived burden or bother, and need to reduce or stop treatment, associated with these side effects among adult patients with chronic ITP.A Web-enabled survey was administered to members of a US-based ITP patient support group. Patients reported demographic and clinical characteristics, ITP treatments' side effects for treatments received since diagnosed, level of bother (or distress), and need to reduce or stop treatment, associated with side effects. Current and past exposure was assessed for five specific treatment types: corticosteroids (CS), intravenous immunoglobulin (IVIg), anti-D immunoglobulin (anti-D), rituximab (RT), and splenectomy (SPL), as well as for other patient-referenced therapies (captured as "other").The survey was completed by 589 patients; 78% female, 89% white, mean age 48 years (SD = 14.71), and 68% reported a typical low platelet count of < 50,000/?L. Current or past treatment with CS was reported by 92% (n = 542) of patients, 56% (n = 322) for IVIg, 36% (n = 209) for anti-D, 36% (n = 213) for RT, and 39% (n = 227) for SPL. A substantial proportion of CS-treated patients reported side effects (98%, P < 0.05), were highly bothered by their side effects (53.1%, P < 0.05), and reported the need to stop or reduce treatment due to side effects (37.8%, P < 0.05). Among patients reporting side effects of treatment, significant associations were noted for the number of side effects, aggregate bother of reported side effects, and the need to stop or reduce treatment (all P < 0.05).Current ITP treatments, particularly corticosteroids, are associated with multiple bothersome side effects that may lead to patients stopping or reducing therapy. Open, informed and complete communication between clinician and patient regarding both the benefits and the side effects of ITP treatment may better prepare patients for their prescribed regimens.

SUBMITTER: Brown TM

PROVIDER: S-EPMC3350461 | biostudies-other | 2012 Mar

REPOSITORIES: biostudies-other

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Patient-reported treatment burden of chronic immune thrombocytopenia therapies.

Brown T Michelle TM Horblyuk Ruslan V RV Grotzinger Kelly M KM Matzdorff Axel C AC Pashos Chris L CL

BMC blood disorders 20120322

<h4>Background</h4>Chronic immune thrombocytopenia (ITP) is a debilitating autoimmune disorder that causes a reduction in blood platelets and increased risk of bleeding. ITP is currently managed with various pharmacologic therapies and splenectomy.This study was conducted to assess patient perceived and reported treatment side effects, as well as the perceived burden or bother, and need to reduce or stop treatment, associated with these side effects among adult patients with chronic ITP.<h4>Meth ...[more]

PMID: 22436142

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Project description:ObjectivesTreatment burden is the workload of healthcare for people with long-term conditions (LTC) and its impact on well-being. A method of measurement is required to identify those experiencing high burden and to measure intervention efficacy. Our aim was to identify, examine and appraise validated patient-reported measures (PRMs) of treatment burden in stroke. Here, stroke serves as an exemplar LTC of older adults.DesignA systematic review of published studies that describe the development and validation of PRMs measuring treatment burden in stroke survivors.Data sourcesWe searched MEDLINE, Embase, CINAHL and PsycINFO electronic databases.Eligibility criteriaStudies published between January 2000 and 12 April 2019 inclusive, in English language. No restrictions were set based on clinical setting or geographical location.Data extraction and synthesisScreening, data extraction and quality appraisal were conducted by two independent reviewers. Content of the PRMs was compared with a published taxonomy of treatment burden. Quality appraisal was conducted using International Society for Quality of Life Research standards.ResultsFrom 3993 articles, 6 relevant PRMs were identified: 3 were stroke specific: The Satisfaction with Stroke Care questionnaire; The Stroke Patient-Reported Outcome Measure and The Barriers to Physical Activity after Stroke scale. Three were generic but validated in stroke: The WHO Quality of Life-100; The Patient's Questionnaire on Participation in Discharge Planning and The Chao Perception of Continuity scale. None comprehensively measured treatment burden. Examples of omitted burdens included developing coping strategies, managing finances and returning to driving. The most notable issue regarding quality appraisal was that three PRMs lacked any underpinning qualitative research relevant to the sample.ConclusionThere is a need to develop a comprehensive PRM of treatment burden for use in stroke, with potential for use in other older populations.

Project description:BackgroundRomiplostim, a thrombopoietin receptor agonist (TPOra), is a second-line medical treatment option for adults with chronic immune thrombocytopenia (ITP). Clinical trials have shown that romiplostim increases platelet counts, while reducing the risk of bleeding and, in turn, the need for costly rescue medications.AimsThe objective of this study was to assess the cost effectiveness of romiplostim in the treatment of adult ITP in Ireland, in comparison with eltrombopag and the medical standard of care (SoC).MethodsA lifetime treatment-sequence cost-utility Markov model with embedded decision tree was developed from an Irish healthcare perspective to compare romiplostim with eltrombopag and SoC. The model was driven by platelet response (platelet count ?50 × 10(9)/L), which determined effectiveness and progression along the treatment pathway, need for rescue therapy (e.g. intravenous immunoglobulin [IVIg] and steroids) and risk of bleeding. Probability of response, mean treatment duration, average time to initial response and utilities were derived from clinical trials and other published evidence. Treatment sequences and healthcare utilization practice were validated by Irish clinical experts. Costs were assessed in <euro> for 2011 and included drug acquisition costs and costs associated with monitoring patients and management of bleeding, as available from published Irish reimbursement lists and other relevant sources. Deterministic and probabilistic sensitivity analyses were conducted.ResultsRomiplostim treatment resulted in an average of 20.2 fewer administrations of rescue medication (IVIg or intravenous steroids) over a patient lifetime than eltrombopag, and 29.3 fewer rescue medication administrations than SoC. Romiplostim was dominant, with cost savings of <euro>13,258 and <euro>22,673 and gains of 0.76 and 1.17 quality-adjusted life-years (QALYs), compared with eltrombopag and SoC, respectively. Romiplostim remained cost effective throughout a variety of potential scenarios, including short-term TPOra treatment duration (1 year). One-way sensitivity analysis showed that the model was most sensitive to variation in the cost of IVIg and use of romiplostim and IVIg. Probabilistic sensitivity analysis showed that romiplostim was likely to be cost effective in over 90 % of cases compared with eltrombopag, and 96 % compared with SoC at a willingness-to-pay threshold of <euro>30,000 per QALY.ConclusionsUse of romiplostim in the ITP treatment pathway, compared with eltrombopag or SoC, is likely to be cost effective in Ireland. Romiplostim improves clinical outcomes by increasing platelet counts, reducing bleeding events and the use of IVIg and steroids, resulting in both cost savings and additional QALYs when compared with current treatment practices.

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Dataset Information

Patient-reported treatment burden of chronic immune thrombocytopenia therapies.

Publications

Patient-reported treatment burden of chronic immune thrombocytopenia therapies.

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