Immune Thrombocytopenia
Conditions
Keywords
Primary Immune Thrombocytopenia, Eltrombopag
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.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 100 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Switzerland
Contacts
CONTACTNovartis Pharmaceuticals
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed