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

Primary outcomes

• Number and Percentage of Patients Achieving an Initial Platelet Response Within 12 Weeks of Start of Treatment [12 weeks]: Initial platelet response is defined as a platelet count ≥50 × 10\^9/L.

Secondary outcomes

• Number and Percentage of Patients Achieving an Initial Complete Platelet Response Within 12 Weeks of Start of Treatment [12 weeks]: Complete platelet response is defined as a platelet count of ≥100 × 10\^9/L.
• Number and Percentage of Patients Achieving a Partial Platelet Response Within 12 Weeks of Start of Treatment [12 weeks]: Partial platelet response is defined as a platelet count of ≥30 × 10\^9/L.
• Number and Percentage of Patients With No or Suboptimal Platelet Response Within 12 Weeks of Start of Treatment [12 weeks]: No or suboptimal platelet response is defined as a platelet count of <30 × 10\^9/L.
• Number and Percentage of Patients Achieving Clinically Meaningful Platelet Response Thresholds Within 12 Weeks of Start of Treatment [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.
• Time to Platelet Response [Up to 4 years and 6 months after treatment initiation]: Platelet response is defined as a platelet count ≥50 ×10\^9/L.
• Platelet Counts at Eltrombopag Treatment Initiation [Day 1]
• Platelet Counts at Eltrombopag Treatment Discontinuation [Up to 4 years and 6 months after treatment initiation]
• Number and Percentage of Patients With Bleeding Symptoms During Eltrombopag Treatment [Up to 4 years and 6 months after treatment initiation]
• Duration Between ITP Diagnosis and Eltrombopag Treatment Initiation [Up to 6 years prior to Day 1 of treatment]
• Number and Percentage of Patients With Concomitant Corticosteroid Treatment During Eltrombopag Treatment [Up to 4 years and 6 months after treatment initiation]
• Number and Percentage of Patients With Concomitant Corticosteroid Treatment Withdrawn During Eltrombopag Treatment [Up to 4 years and 6 months after treatment initiation]
• Time to Eltrombopag Treatment Discontinuation [Up to 4 years and 6 months after treatment initiation]
• Number of Patients by Reason for Eltrombopag Initiation [Up to 6 years prior to Day 1 of treatment]
• Number of Patients by Reason for Eltrombopag Discontinuation [Up to 4 years and 6 months after treatment initiation]
• Baseline Demographics [Up to 6 years prior to Day 1 of treatment]
• Number of Prior Corticosteroid Treatment Courses [Up to 6 years prior to Day 1 of treatment]
• Duration of Prior Corticosteroid Treatment Courses [Up to 6 years prior to Day 1 of treatment]
• Number of Patients by Comorbidities [Up to 6 years prior to Day 1 of treatment]
• Number of Patients by HCP-reported Reason for Eltrombopag Initiation [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 [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.
• Number of Patients by Location of Eltrombopag Prescription [Up to 4 years and 6 months after treatment initiation]
• Number of Patients by Type of Provider of Eltrombopag Treatment Initiation [Day 1]: Providers include general practitioner, immunologist, hematologist, internal medicine specialist, and other.
• Number of Patients Categorized by Typical Duration Between ITP Diagnosis and Eltrombopag Initiation as Reported by the HCP [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.
• Number of Patients by Corticosteroid Use During Eltrombopag Treatment as Reported by the HCP [Up to 4 years and 6 months after treatment initiation]: Corticosteroid use categorized as never, rarely, sometimes, often, or always.
• Number of Patients by HCP-reported Indicators for Treatment Escalation or Discontinuation [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.
• Number of Patients by Common Reasons for Eltrombopag Discontinuation as Reported by the HCP [Up to 4 years and 6 months after treatment initiation]
• Number of Patients by the HCP-reported Treatment Success With Eltrombopag [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.
• Number of Patients by Patient Characteristics Associated With Better Eltrombopag Response as Reported by the HCP [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
• Number of Patients by Patient Characteristics Associated With Poor Eltrombopag Response as Reported by the HCP [Up to 4 years and 6 months after treatment initiation]: Patient characteristics include multiple comorbidities, delayed initiation, prior treatment failure, and other.

Sex: All, ages: 18 Years to 100 Years

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.

• Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals