Immune Thrombocytopenia
Conditions
Keywords
Primary Immune Thrombocytopenia, Retrospective Cohort, Treatment Patterns, Effectiveness, Healthcare Resource Utilization
Brief summary
The aim of this study was to describe real-world treatment patterns and standard of care treatment effectiveness and utilization outcomes in adult primary ITP patients indicated for second-line treatment with any prior corticosteroid treatment in real-world treatment settings in the United States (US) identified in an electronic health record (EHR) and claims database.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Evidence of a diagnosis of primary ITP during the study identification period. 2. Evidence of treatment with any allowed second-line treatment during the study identification period after the first primary ITP diagnosis. 3. Evidence of treatment with any allowed first-line treatment prior to index. 4. Aged ≥18 years at index.
Exclusion criteria
1. Fewer than 12 months continuous enrollment or activity prior to index. 2. Fewer than one inpatient or fewer than two outpatient records during baseline. 3. Evidence of any allowed second-line therapies or splenectomy prior to index. 4. Evidence of diagnosis of Evans syndrome, systemic lupus erythematosus, autoimmune lymphoproliferative syndrome, human immunodeficiency virus (HIV), hepatitis C virus (HCV), Helicobacter pylori, Sjogren's antiphospholipid syndrome prior to index, or active b-cell malignancies (not in remission) in the baseline period. 5. Continuous enrollment end date or death date prior to index.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number and Percentage of Patients With Prescribed Second-line Treatments of Interest | Up to approximately 8 years | Second-line treatments of interest, either as monotherapy or in a combination therapy, included: * Thrombopoietin Receptor Agonists (TPO-RAs): romiplostim, eltrombopag, avatrombopag. * Clusters of differentiation 20 (CD20) inhibitors: rituximab * Other therapies: fostamatinib * Immunosuppressants: azathioprine, mycophenolate mofetil * Splenectomy |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Initiated a New Second-line Treatment or Added Oral Corticosteroids | Up to approximately 8 years | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. |
| Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Died | Up to approximately 8 years | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. |
| Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Reached the End of the Study Period | Up to approximately 8 years | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. |
| Among Patient Responders, Time From Second-line Treatment Initiation to Response | Up to 6 months | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. |
| Among Patient Responders, Duration of Platelet Response | Up to approximately 8 years | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. |
| Number and Percentage of Patient Responders With Treatment-free Response | Up to 3 months | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. Treatment-free response was defined as platelet count ≥30 × 10\^9/L at 3 months after discontinuing second-line treatment. |
| Duration of Patient Follow-up | Up to approximately 8 years | — |
| Time to Treatment Discontinuation (TTD) | Up to approximately 8 years | — |
| Time to Next Treatment (TTNT) | Up to approximately 8 years | — |
| Time to Next Treatment or Initiation of Rescue Treatment | Up to approximately 8 years | — |
| Number and Percentage of Patients Who Discontinued Treatment Within 1, 3, and 6 Months of Second-line Treatment Initiation | 1, 3, and 6 months | — |
| Number and Percentage of Patients by First Type of Activity Observed Following Treatment Discontinuation | 1, 3, and 6 months | Activities included: * Switch to new treatment * Restart the same second-line treatment * Have rescue treatment without second-line treatment * First-line treatment without second-line treatment * Enter a treatment-free interval * End of EHR/claims disenrollment * Death |
| Number of Patients Who Entered a Treatment-free Interval | Up to approximately 8 years | Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment. |
| Duration of Treatment-free Interval | Up to approximately 8 years | Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment. |
| Number and Percentage of Combination Second-line Treatment Treated Patients Who Underwent Reductions | Up to approximately 8 years | Reduction was defined as a discontinuation of at least one therapeutic agent with maintenance of at least one therapeutic agent. |
| Number and Percentage of Patients Who Used Add-on, Rescue, or Supportive Treatment During Second-line Treatment | Up to approximately 8 years | An add-on was the inclusion of a new, additional second-line treatment ≥28 days after initiation of the initial second-line treatment. |
| Time to Initiation of Add-on or Rescue Treatment During Second-line Treatment | Up to approximately 8 years | — |
| Time to Initiation of Supportive Treatment During Second-line Treatment | Up to approximately 8 years | — |
| Platelet Counts | 1, 3, and 6 months | — |
| Number and Percentage of Patients Who Achieved Pre-defined Platelet Counts Within 3 Months of Treatment Initiation | 3 months | Measured among patients with at least one platelet count within 3 months of treatment initiation. Pre-defined platelet counts were categorized as follows: * ≥30 × 10\^9/L, * ≥ 50 × 10\^9/L, and * ≥100 × 10\^9/L |
| Number and Percentage of Patients With no Initial Response to Second-line Treatment | 3 months | Measured among patients with at least one platelet count within 3 months of treatment initiation. No initial response was defined as platelet counts \<30 × 10\^9/L within 3 months of treatment initiation. |
| Number and Percentage of Patients Who Achieved Pre-defined Response Levels Within 6 Months of Treatment Initiation | 6 months | Measured among patients with at least one platelet count within 6 months of treatment initiation. Response levels were categorized as follows: * response (≥ 50 × 10\^9/L), and * complete response (≥100 × 10\^9/L) |
| Among Patients With At Least Two Platelet Counts From 3 to 6 Months, Number and Percentage of Patients Who Reached Real-world Durable Response | From 3 to 6 months | Real-world durable response was defined as ≥75% of platelet counts ≥50 × 10\^9/L with a minimum of 2 platelet measurements and without other therapies. |
| Among Patients With At Least Two Platelet Counts From 6 to 12 months, Number and Percentage of Patients Who Reached Real-world Durable Response | From 6 to 12 months | Real-world durable response was defined as ≥75% of platelet counts ≥50 × 10\^9/L with a minimum of 2 platelet measurements and without other therapies. |
| Number and Percentage of Patients With Emergency Department (ED) or Inpatient (IP) Thrombotic Events Within 12 Months After Second-line Treatment Initiation | 12 months | Thrombotic events may have included pulmonary embolism, deep vein thrombosis, cerebral venous thrombosis, transient ischemic attack. |
| Number of Thrombotic Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years | 12 months | — |
| Number and Percentage of Patients With Infection Events Within 12 Months After Second-line Treatment Initiation | 12 months | — |
| Number of Infection Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years | 12 months | — |
| Number and Percentage of Patients With Bleeding Events Within 12 Months After Second-line Treatment Initiation | 12 months | — |
| Number of Bleeding Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years | 12 months | — |
| Overall Survival (OS) | 12 months | — |
| Number and Percentage of Patients With At Least One Healthcare Visit After Second-line Treatment Initiation | 12 months | Healthcare visits included outpatient visits, IP admissions, intensive care unit (ICU) admissions, and ED visits. |
| Number of Healthcare Visits After Second-line Treatment Initiation, per Patient per Year (PPPY) | 12 months | Healthcare visits included outpatient visits, IP admissions, ICU admissions, and ED visits. |
| Number of Oral Corticosteroid Medication Fills Within 12 Months After Second-line Treatment Initiation, PPPY | 12 months | — |
| Number of Days With Active Oral Corticosteroid Prescriptions Within 12 Months After Second-line Treatment Initiation, PPPY | 12 months | — |
| Duration of IP and ICU Admissions Among Patients Who Were Hospitalized After Second-line Treatment Initiation | 12 months | — |
| Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Received Rescue Treatment | Up to approximately 8 years | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. |
| Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Failed Treatment | Up to approximately 8 years | Patient responders were defined as patients who reach a response level of ≥ 50 × 10\^9/L within 6 months. Treatment failure was defined as platelet count \<30 × 10\^9/L. |
Countries
United States
Contacts
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed