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

Novartis Pharmaceuticals150 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

150

Conditions

Immune Thrombocytopenia

Eligibility

Sex: ALLMin age: 18 YearsMax age: 100 Years

Medical Language ↔ Plain English

Inclusion criteria: HCP inclusion criteria: 1. A licensed HCP (e.g., internal medicine specialist, hematologist, hematologist-oncologist or immunologist etc.) involved in the management of adult patients with primary ITP. 2. Actively involved in treatment decision-making for primary ITP patients at the time of the study. 3. Have a minimum of 3 years of experience managing patients with primary ITP. 4. Currently, managing an average of at least 2 adult primary ITP patients per month, including those treated with eltrombopag. 5. Able and willing to complete a web-based survey (approximately 60 to 70 minutes) and abstract data from 2 eligible patient charts. 6. Practicing in one of the study countries (US, UK, or Germany). 7. Have access to a computer or mobile device with internet connectivity to complete the survey. 8. Not listed as inactive or retired in the panel database. Patient-level inclusion criteria: 1. Confirmed diagnosis of primary ITP. 2. Aged ≥18 years at the time of diagnosis. 3. Received first-line corticosteroid therapy (with or without intravenous immune globulin \[IVIg\]). 4. Initiated eltrombopag treatment (with or without corticosteroids) as first non-steroidal ITP treatment between 01 January 2020 and 31 December 2024. 5. Have at least 6 months of follow-up data available after the index date (unless deceased within that period). Exclusion criteria: HCP exclusion criteria: 1. Currently employed by a pharmaceutical company or regulatory authority involved in the approval or marketing of treatments for primary ITP. 2. Unable to provide data for patients who meet the study's inclusion criteria. 3. Not currently managing patients with primary ITP or are not involved in treatment decisions for these patients. Patient-level exclusion criteria: 1\. Diagnosis of Evans syndrome, secondary ITP, or non-immune thrombocytopenia.

Outcomes

Primary Outcomes

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.

Time frame: 12 weeks

Secondary Outcomes

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

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

Time frame: 12 weeks

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

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

Time frame: 12 weeks

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

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

Time frame: 12 weeks

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

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.

Time frame: 12 weeks

Time to Platelet Response

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

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

Platelet Counts at Eltrombopag Treatment Initiation

Time frame: Day 1

Platelet Counts at Eltrombopag Treatment Discontinuation

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

Number and Percentage of Patients With Bleeding Symptoms During Eltrombopag Treatment

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

Duration Between ITP Diagnosis and Eltrombopag Treatment Initiation

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

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

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

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

Time to Eltrombopag Treatment Discontinuation

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

Number of Patients by Reason for Eltrombopag Initiation

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

Number of Patients by Reason for Eltrombopag Discontinuation

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

Baseline Demographics

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

Number of Prior Corticosteroid Treatment Courses

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

Duration of Prior Corticosteroid Treatment Courses

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

Number of Patients by Comorbidities

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

Number of Patients by HCP-reported Reason for Eltrombopag Initiation

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

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

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

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

Number of Patients by Location of Eltrombopag Prescription

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

Number of Patients by Type of Provider of Eltrombopag Treatment Initiation

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

Time frame: Day 1

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Overview

Conditions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts