Health-related quality of life in children with chronic immune thrombocytopenia treated with eltrombopag in the PETIT study.

The PETIT (Eltrombopag in Pediatric Patients with Thrombocytopenia from Chronic ITP) trial showed that in children aged 1-17 years with chronic or persistent immune thrombocytopenia (ITP), eltrombopag improved platelet counts, decreased clinically significant bleeding and reduced rescue medication need. We report the health-related quality of life (HRQoL) results from the PETIT study using the Kids' ITP Tools (KIT). A limitation was that PETIT was not powered for the HRQoL analysis.

Cost-Effectiveness of Eltrombopag versus Romiplostim for the Treatment of Chronic Immune Thrombocytopenia in England and Wales.

Objectives: To evaluate the cost-effectiveness of eltrombopag compared with romiplostim to be used in the treatment of chronic immune thrombocytopenia in patients in England and Wales who are splenectomized or ineligible for splenectomy and are refractory to other treatments.

Methods: A Markov cohort model in which patients were administered a sequence of treatments was used to predict long-term outcomes associated with each treatment. The model was informed by data from the eltrombopag clinical trial program and the available literature.

Health-related quality of life in thrombocytopenic patients with chronic hepatitis C with or without cirrhosis in the ENABLE-1 and ENABLE-2 studies.

Background: Despite changes in the treatment paradigm towards non-interferon-based therapies, interferon-based treatments are still used in some geographical regions for treating patients with hepatitis C virus (HCV) infection. Use of eltrombopag with interferon-based treatment for patients with thrombocytopenia and HCV was assessed in two similarly designed phase 3 trials (Eltrombopag to Initiate and Maintain Interferon Antiviral Treatment to Benefit Subjects With Hepatitis C-Related Liver Disease [ENABLE-1 and ENABLE-2]). These trials also aimed to determine whether response to antiviral therapy (e.

Treatment patterns and clinical outcomes in patients with chronic immune thrombocytopenia (ITP) switched to eltrombopag or romiplostim.

This observational study aimed to assess real-world treatment patterns and clinical outcomes for patients with chronic immune thrombocytopenia (ITP) currently being treated with eltrombopag or romiplostim after switching from corticosteroids, rituximab, or the alternate thrombopoietin receptor agonist (TPO-RA). The study examined the rationale for switching to TPO-RA therapy using aided responses. Dosing patterns were also analyzed before and after switching.

Incident severe thrombocytopenia in veterans treated with pegylated interferon plus ribavirin for chronic hepatitis C infection.

Purpose: We sought to determine the incidence and risks for severe thrombocytopenia (platelets < 50,000/μL) in United States Veteran patients treated with pegylated interferon (PEG-IFN) plus ribavirin for hepatitis C virus-positive (HCV) chronic liver disease (CLD).

Methods: Using a retrospective, observational cohort study design to analyze databases from the New England Veterans Healthcare System, we identified 979 patients diagnosed with HCV-positive CLD treated solely with PEG-IFN plus ribavirin. The cohort was stratified by pre-treatment platelet counts of 50,000-100,000/μL (N = 90), >100,000-150,000/μL (N = 162), and >150,000μL (N = 727).

Patient-reported treatment burden of chronic immune thrombocytopenia therapies.

Background: 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.

Selective validation of the WHO Bleeding Scale in patients with chronic immune thrombocytopenia.

Objective: To evaluate the World Health Organization's (WHO) Bleeding Scale in two studies of eltrombopag in adults with chronic immune thrombocytopenia (ITP).

Research Design And Methods: Validated scales assessing bleeding in adults with ITP are lacking. Data from two long-term, phase 3 clinical trials (RAISE: NCT00370331; EXTEND: NCT00351468) that assessed eltrombopag in adults with chronic ITP were analyzed to evaluate the performance of the WHO Bleeding Scale.

Validation of the FACIT-fatigue subscale, selected items from FACT-thrombocytopenia, and the SF-36v2 in patients with chronic immune thrombocytopenia.

Purpose: To assess the validity and reliability of the fatigue subscale of the Functional Assessment of Chronic Illness Therapy (FACIT-F), a 6-item subset from the thrombocytopenia subscale of the Functional Assessment of Cancer Therapy (FACT-Th6) and the Short Form-36 Version 2 (SF-36v2) in 2 clinical trials of the thrombopoietin receptor agonist eltrombopag in chronic immune thrombocytopenia (ITP) patients.

Methods: In the 6-month, RAndomized placebo-controlled ITP Study with Eltrombopag (RAISE; n = 197), the FACIT-F, FACT-Th6, and SF-36v2 were administered at baseline, day 43, weeks 14 and 26, or early withdrawal. In the ongoing open-label extension study, Eltrombopag EXTENDed Dosing Study (EXTEND; n = 154), measures were administered at baseline, at the beginning of each stage, and at permanent discontinuation of study medication.

Patients' benefit-risk preferences for chronic idiopathic thrombocytopenic purpura therapies.

Background: Idiopathic thrombocytopenic purpura (ITP) primarily is a disorder of adults characterized by autoantibody-induced platelet destruction and reduced platelet production, leading to a low peripheral blood platelet count. The long-term management of many patients with chronic ITP is unsatisfactory, largely due to the variable efficacy and risks of severe adverse effects associated with current treatment options.

Objective: To estimate patients' benefit-risk preferences for treatments for ITP.

Analysis of the impact and burden of illness of adult chronic ITP in the US.

Objective: Chronic idiopathic thrombocytopenic purpura (ITP), the predominant diagnosis in the ICD-9-CM category of primary thrombocytopenia in adults, is an autoimmune disease characterized by autoantibody-mediated platelet destruction and reduced platelet production. The objective of this study was to describe ITP patient demographics, treatment, medical care resource utilization, and costs from a real-world situation.

Research Design And Methods: Managed-care administrative claims data from January 1 2000 to February 29 2004 were used in a retrospective, longitudinal cohort study to evaluate the burden of illness of chronic idiopathic primary thrombocytopenia among adults in the US, with particular emphasis on chronic ITP.

Phase III study comparing oral topotecan to intravenous docetaxel in patients with pretreated advanced non-small-cell lung cancer.

Purpose: This open-label, randomized, multicenter, phase III study compared oral topotecan versus intravenous (IV) docetaxel in patients with previously treated non-small-cell lung cancer (NSCLC).

Patients And Methods: Patients with stage III or IV NSCLC, performance status < or = 2, who had received only one prior chemotherapy regimen, were randomly assigned to treatment with oral topotecan 2.3 mg/m2/d on days 1 to 5 or IV docetaxel 75 mg/m2 day 1 every 21 days.