Clinical trial NCT01439321: Outcomes Comparison of Chronic Immune Thrombocytopenic Purpura (ITP) Patients Switched to Eltrombopag and Romiplostim
Official title: Outcomes Comparison of Chronic Immune Thrombocytopenic Purpura (ITP) Patients Switched to Eltrombopag and Romiplostim
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Immune Thrombocytopenic Purpura
Keywords: treatment satisfaction
romiplostim
eltrombopag
chronic immune thrombocytopenic pupura
Lead sponsor: GlaxoSmithKline
Sponsor class: INDUSTRY
First posted: 2011-09-23
Enrollment: 280
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Study population will include adult patients with a diagnosis of immune (idiopathic) thrombocytopenic purpura (ITP) who switched to eltrombopag or romiplostim.

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.


Eligibility criteria: Inclusion criteria

* Adults 18 years and older diagnosed with chronic ITP
* Switched to eltrombopag or romiplostim a minimum of 4 weeks prior to index date
* Received corticosteroids, rituximab, or one TPO-receptor agonist as prior primary therapy before switching to eltrombopag or romiplostim
* Medical history must be available from chronic ITP diagnosis through to most recent office visit
* Patients who complete the self-administered questionnaire were the same patients whose medical charts were reviewed

Exclusion criteria

* Age less than 18 years old

Primary outcomes:
measure: treatment satisfaction scores by domain
timeFrame: 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
Secondary outcomes:
measure: Short-form 36 (SF-36) score
timeFrame: 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
Other outcomes:
Locations:
Study officials:
affiliation: GlaxoSmithKline
name: GSK Clinical Trials
role: STUDY_DIRECTOR
References: