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NCT Number: NCT07587723

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

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.

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Key information

Conditions

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

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis

Basel, CH-4056, Switzerland

Location status: Recruiting

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

HCP inclusion criteria:

  • 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.

Patient-level inclusion criteria:

  • 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).

Exclusion criteria

HCP exclusion criteria:

  • Currently employed by a pharmaceutical company or regulatory authority involved in the approval or marketing of treatments for primary ITP.
  • Unable to provide data for patients who meet the study's inclusion criteria.
  • Not currently managing patients with primary ITP or are not involved in treatment decisions for these patients.

Patient-level exclusion criteria:

  • Diagnosis of Evans syndrome, secondary ITP, or non-immune thrombocytopenia.

Treatment and study plan

Primary outcomes

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

    Time frame: 12 weeks

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

Secondary outcomes

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

    Time frame: 12 weeks

    Complete platelet response is defined as a platelet count of ≥100 × 10^9/L.

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

    Time frame: 12 weeks

    Partial platelet response is defined as a platelet count of ≥30 × 10^9/L.

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

    Time frame: 12 weeks

    No or suboptimal platelet response is defined as a platelet count of <30 × 10^9/L.

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

    Time frame: 12 weeks

    Clinically meaningful platelet response thresholds as indicated by a platelet count increase from a pretreatment value <30 × 10^9/L, and/or documentation of improvement in clinical symptoms such as bleeding.

  5. Time to Platelet Response

    Time frame: Up to 4 years and 6 months after treatment initiation

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

  6. Platelet Counts at Eltrombopag Treatment Initiation

    Time frame: Day 1

  7. Platelet Counts at Eltrombopag Treatment Discontinuation

    Time frame: Up to 4 years and 6 months after treatment initiation

  8. Number and Percentage of Patients With Bleeding Symptoms During Eltrombopag Treatment

    Time frame: Up to 4 years and 6 months after treatment initiation

  9. Duration Between ITP Diagnosis and Eltrombopag Treatment Initiation

    Time frame: Up to 6 years prior to Day 1 of treatment

  10. Number and Percentage of Patients With Concomitant Corticosteroid Treatment During Eltrombopag Treatment

    Time frame: Up to 4 years and 6 months after treatment initiation

  11. Number and Percentage of Patients With Concomitant Corticosteroid Treatment Withdrawn During Eltrombopag Treatment

    Time frame: Up to 4 years and 6 months after treatment initiation

  12. Time to Eltrombopag Treatment Discontinuation

    Time frame: Up to 4 years and 6 months after treatment initiation

  13. Number of Patients by Reason for Eltrombopag Initiation

    Time frame: Up to 6 years prior to Day 1 of treatment

  14. Number of Patients by Reason for Eltrombopag Discontinuation

    Time frame: Up to 4 years and 6 months after treatment initiation

  15. Baseline Demographics

    Time frame: Up to 6 years prior to Day 1 of treatment

  16. Number of Prior Corticosteroid Treatment Courses

    Time frame: Up to 6 years prior to Day 1 of treatment

  17. Duration of Prior Corticosteroid Treatment Courses

    Time frame: Up to 6 years prior to Day 1 of treatment

  18. Number of Patients by Comorbidities

    Time frame: Up to 6 years prior to Day 1 of treatment

  19. Number of Patients by HCP-reported Reason for Eltrombopag Initiation

    Time frame: Up to 6 years prior to Day 1 of treatment

  20. Number of Patients by HCP-reported Factors Influencing Selection of Eltrombopag as Second-line Treatment

    Time frame: Up to 6 years prior to Day 1 of treatment

    Factors influencing selection include platelet count, bleeding symptoms, comorbidities, prior treatment response, ease of administration, safety profile, guideline recommendation, and other.

  21. Number of Patients by Location of Eltrombopag Prescription

    Time frame: Up to 4 years and 6 months after treatment initiation

  22. Number of Patients by Type of Provider of Eltrombopag Treatment Initiation

    Time frame: Day 1

    Providers include general practitioner, immunologist, hematologist, internal medicine specialist, and other.

  23. Number of Patients Categorized by Typical Duration Between ITP Diagnosis and Eltrombopag Initiation as Reported by the HCP

    Time frame: Up to 6 years prior to Day 1 of treatment

    Typical duration between diagnosis and treatment initiation categorized as <3 months, 3-6 months, 6-12 months, >12 months.

  24. Number of Patients by Corticosteroid Use During Eltrombopag Treatment as Reported by the HCP

    Time frame: Up to 4 years and 6 months after treatment initiation

    Corticosteroid use categorized as never, rarely, sometimes, often, or always.

  25. Number of Patients by HCP-reported Indicators for Treatment Escalation or Discontinuation

    Time frame: Up to 4 years and 6 months after treatment initiation

    Indicators for treatment escalation or discontinuation include lack of platelet response, bleeding symptoms, adverse events, stable response, patient preference, and other.

  26. Number of Patients by Common Reasons for Eltrombopag Discontinuation as Reported by the HCP

    Time frame: Up to 4 years and 6 months after treatment initiation

  27. Number of Patients by the HCP-reported Treatment Success With Eltrombopag

    Time frame: Up to 4 years and 6 months after treatment initiation

    Treatment success categorized as rapid platelet increase, sustained response, bleeding control, minimal side effects, improved quality of life, and other.

  28. Number of Patients by Patient Characteristics Associated With Better Eltrombopag Response as Reported by the HCP

    Time frame: Up to 4 years and 6 months after treatment initiation

    Patient characteristics include younger age, no comorbidities, early initiation post-diagnosis, prior corticosteroid response, and other

  29. Number of Patients by Patient Characteristics Associated With Poor Eltrombopag Response as Reported by the HCP

    Time frame: Up to 4 years and 6 months after treatment initiation

    Patient characteristics include multiple comorbidities, delayed initiation, prior treatment failure, and other.

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

+41613241111

Novartis Pharmaceuticals

CONTACT

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2028
Study completion
2028
First posted
May 14, 2026
Registry last updated
Jul 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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