Outcomes Comparison of Chronic Immune Thrombocytopenic Purpura (ITP) Patients Switched to Eltrombopag and Romiplostim

Completed

• Conditions: Immune Thrombocytopenic Purpura
• Interventions: Biological: Eltrombopag; Biological: Romiplostim

• Registration Number: NCT01439321
• Lead Sponsor: GlaxoSmithKline

Brief Summary

Chronic immune thrombocytopenic purpura (ITP) is characterized by low platelet counts and the risk of severe bleeding complications. The two recently introduced TPO-RA drugs, namely, eltrombopag and romiplostim, have shown efficacious sustained response with continuous administration. Both drugs are indicated for the treatment of thrombocytopia in patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. While these trials address important clinical questions they were not intended to evaluate what happens in the real-world settings with actual patient living daily lives. The purpose of this health outcomes study is to understand how the two TPO receptor agonists (TPO-RA) currently available in the US are being used in clinical practice and how their use impacts chronic ITP patients' daily lives. The study hypothesis is that patients who switched to eltrombopag report a better health-related quality of life than those who switched to romiplostim. This study utilized a hybrid design of retrospective chart review study and cross-sectional patient survey. A customized Patient Case Report Form (CRF) will be used to retrospectively collect clinical data from patient medical charts where the primary cohorts consist of patients who have switched from other ITP medication to eltrombopag or romiplostim. A cross-sectional survey will be employed to collect patient reported outcomes (PRO) data, including health-related quality of life and treatment satisfaction, using a compository questionnaire. Analyses of cross-sectional survey data and retrospective medical chart review data in patients who switch to either eltrombopag or romiplostim from their prior primary therapy will be conducted.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-11
• Primary Completion: 2011-06
• Study Completion: 2011-06
• Study First Submitted: 2011-09-21
• Study First Submitted QC: 2011-09-21
• Study First Posted: 2011-09-23
• Status Verified: 2012-06
• Last Update Submitted: 2012-06-21
• Last Update Posted: 2012-06-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 280

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patient with Chronic Immune Thrombocytopenic Purpura	Eltrombopag	Patients with chronic ITP who switch from their previous treatment of corticosteroids, rituximab, or eltrombopag or romiplostim to eltrombopag or romiplostim and have been on the new treatment for at least four weeks
Patient with Chronic Immune Thrombocytopenic Purpura	Romiplostim	Patients with chronic ITP who switch from their previous treatment of corticosteroids, rituximab, or eltrombopag or romiplostim to eltrombopag or romiplostim and have been on the new treatment for at least four weeks

Primary Outcome Measures

Name	Time	Method
treatment satisfaction scores by domain	During a 5 month period, the questionnaire will be administered to each participant at one time point. The recall period for the TQSM in this study is 3 weeks	Treatment satisfaction will be captured with the Treatment Satisfaction Questionnaire for Medication (TSQM). The questionnaire is composed of 14 items. Mean scores will be reported for each treatment group by domain. The domains of the TSQM are effectiveness, side effects, convenience, and global satisfaction

Secondary Outcome Measures

Name	Time	Method
Short-form 36 (SF-36) score	During a 5 month period, the questionnaire will be administered to each participant at one time point. The recall period for the SF-36 in this study is 1 week	The SF-36 is a general overall health instrument consisting of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. Scales include general health, limitations of activities, physical functioning, emotional health, social activities, pain, and energy/ emotional functioning. Scores for social functioning and physicial functioning will also be reported