A Real-world Study of Treatment Patterns and Outcomes in Primary Immune Thrombocytopenia Patients Treated With Eltrombopag

NCT07587723 | Not Yet Recruiting

• 1 other identifier: CVAY736I12002
• Study Type: observational
• Target: 150
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study aims to describe the treatment patterns and clinical outcomes of treatment with eltrombopag in primary immune thrombocytopenia (ITP) patients treated with prior corticosteroid therapy. This study will use secondary data provided by health care professionals (HCPs) for a sample of primary ITP patients, and primary data from a structured HCP survey. Data will be collected from HCPs working in various clinical centers across the United States (US), United Kingdom (UK), and Germany.

Conditions

Immune Thrombocytopenia

Keywords

Primary Immune Thrombocytopenia; Eltrombopag

Outcome Measures

Primary Outcomes (1)

• Number and Percentage of Patients Achieving an Initial Platelet Response Within 12 Weeks of Start of Treatment

  Initial platelet response is defined as a platelet count ≥50 × 10\^9/L.

  12 weeks

Secondary Outcomes (29)

• Number and Percentage of Patients Achieving an Initial Complete Platelet Response Within 12 Weeks of Start of Treatment

  12 weeks

• Number and Percentage of Patients Achieving a Partial Platelet Response Within 12 Weeks of Start of Treatment

  12 weeks

• Number and Percentage of Patients With No or Suboptimal Platelet Response Within 12 Weeks of Start of Treatment

  12 weeks

• Number and Percentage of Patients Achieving Clinically Meaningful Platelet Response Thresholds Within 12 Weeks of Start of Treatment

  12 weeks

• Time to Platelet Response

  Up to 4 years and 6 months after treatment initiation

Study Arms (1)

Eltrombopag Cohort

Adult patients diagnosed with primary ITP who initiated eltrombopag treatment between 01 January 2020 and 31 December 2024 following corticosteroid treatment.

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult patients diagnosed with primary ITP who were treated with eltrombopag following corticosteroid treatment.

You may qualify if:

• A licensed HCP (e.g., internal medicine specialist, hematologist, hematologist-oncologist or immunologist etc.) involved in the management of adult patients with primary ITP.
• Actively involved in treatment decision-making for primary ITP patients at the time of the study.
• Have a minimum of 3 years of experience managing patients with primary ITP.
• Currently, managing an average of at least 2 adult primary ITP patients per month, including those treated with eltrombopag.
• Able and willing to complete a web-based survey (approximately 60 to 70 minutes) and abstract data from 2 eligible patient charts.
• Practicing in one of the study countries (US, UK, or Germany).
• Have access to a computer or mobile device with internet connectivity to complete the survey.
• Not listed as inactive or retired in the panel database.
• Confirmed diagnosis of primary ITP.
• Aged ≥18 years at the time of diagnosis.
• Received first-line corticosteroid therapy (with or without intravenous immune globulin \[IVIg\]).
• Initiated eltrombopag treatment (with or without corticosteroids) as first non-steroidal ITP treatment between 01 January 2020 and 31 December 2024.
• Have at least 6 months of follow-up data available after the index date (unless deceased within that period).

You may not qualify if:

• Currently employed by a pharmaceutical company or regulatory authority involved in the approval or marketing of treatments for primary ITP.
• Not currently managing patients with primary ITP or are not involved in treatment decisions for these patients.
• \. Diagnosis of Evans syndrome, secondary ITP, or non-immune thrombocytopenia.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2026
• First Posted: May 14, 2026
• Study Start: May 20, 2026
• Primary Completion (Estimated): August 20, 2026
• Study Completion (Estimated): August 20, 2026
• Last Updated: May 14, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share