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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01439321
Recruitment Status : Completed
First Posted : September 23, 2011
Last Update Posted : June 25, 2012
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment
Immune Thrombocytopenic Purpura Biological: Eltrombopag Biological: Romiplostim

Study Type : Observational
Actual Enrollment : 280 participants
Observational Model: Cohort
Time Perspective: Retrospective
Official Title: Outcomes Comparison of Chronic Immune Thrombocytopenic Purpura (ITP) Patients Switched to Eltrombopag and Romiplostim
Study Start Date : November 2010
Actual Primary Completion Date : June 2011
Actual Study Completion Date : June 2011

Group/Cohort Intervention/treatment
Patient with Chronic Immune Thrombocytopenic Purpura
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
Biological: Eltrombopag
Switched to Eltrombopag
Other Name: Promacta® is a registered trademark of GlaxoSmithKline

Biological: Romiplostim
Switched to Romiplostim
Other Name: Nplate® is a registered trademark of Amgen

Primary Outcome Measures :
  1. treatment satisfaction scores by domain [ Time Frame: 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 :
  1. Short-form 36 (SF-36) score [ Time Frame: 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

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01439321

Sponsors and Collaborators
Study Director: GSK Clinical Trials GlaxoSmithKline

Responsible Party: GlaxoSmithKline Identifier: NCT01439321     History of Changes
Other Study ID Numbers: 113922
First Posted: September 23, 2011    Key Record Dates
Last Update Posted: June 25, 2012
Last Verified: June 2012

Keywords provided by GlaxoSmithKline:
treatment satisfaction
chronic immune thrombocytopenic pupura

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases