A Real World Study of Treatment Patterns and Effectiveness of Standard of Care in Primary Immune Thrombocytopenia Patients

Treatment Patterns and Real World Effectiveness of Standard of Care in Primary Immune Thrombocytopenia (ITP) Patients: A Real-world Retrospective Cohort Study Using the Optum® Market Clarity Database

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.

Study Overview

• Status: Completed
• Conditions: Immune Thrombocytopenia

• Study Type: Observational
• Enrollment (Actual): 3714

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936
      • Novartis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients diagnosed with primary ITP from a US medical claim and EHR data source during the study period.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
ITP Cohort; Primary ITP patients with prior corticosteroid treatment being treated with second-line treatment.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number and Percentage of Patients With Prescribed Second-line Treatments of Interest	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	Up to approximately 8 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival (OS)		12 months
Time to Next Treatment (TTNT)		Up to approximately 8 years
Duration of Patient Follow-up		Up to approximately 8 years
Time to Treatment Discontinuation (TTD)		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	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	1, 3, and 6 months
Number of Patients Who Entered a Treatment-free Interval	Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment.	Up to approximately 8 years
Duration of Treatment-free Interval	Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment.	Up to approximately 8 years
Number and Percentage of Combination Second-line Treatment Treated Patients Who Underwent Reductions	Reduction was defined as a discontinuation of at least one therapeutic agent with maintenance of at least one therapeutic agent.	Up to approximately 8 years
Number and Percentage of Patients Who Used Add-on, Rescue, or Supportive Treatment During Second-line Treatment	An add-on was the inclusion of a new, additional second-line treatment ≥28 days after initiation of the initial second-line treatment.	Up to approximately 8 years
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	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	3 months
Number and Percentage of Patients With no Initial Response to Second-line Treatment	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.	3 months
Number and Percentage of Patients Who Achieved Pre-defined Response Levels Within 6 Months of Treatment Initiation	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)	6 months
Among Patients With At Least Two Platelet Counts From 3 to 6 Months, Number and Percentage of Patients Who Reached Real-world Durable Response	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.	From 3 to 6 months
Among Patients With At Least Two Platelet Counts From 6 to 12 months, Number and Percentage of Patients Who Reached Real-world Durable Response	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.	From 6 to 12 months
Number and Percentage of Patients With Emergency Department (ED) or Inpatient (IP) Thrombotic Events Within 12 Months After Second-line Treatment Initiation	Thrombotic events may have included pulmonary embolism, deep vein thrombosis, cerebral venous thrombosis, transient ischemic attack.	12 months
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
Number and Percentage of Patients With At Least One Healthcare Visit After Second-line Treatment Initiation	Healthcare visits included outpatient visits, IP admissions, intensive care unit (ICU) admissions, and ED visits.	12 months
Number of Healthcare Visits After Second-line Treatment Initiation, per Patient per Year (PPPY)	Healthcare visits included outpatient visits, IP admissions, ICU admissions, and ED visits.	12 months
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 Failed Treatment	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.	Up to approximately 8 years
Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Received Rescue Treatment	Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.	Up to approximately 8 years
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	Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.	Up to approximately 8 years
Number and Percentage of Patient Responders From Date of First Response Through the End of Follow-up, Who Died	Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.	Up to approximately 8 years
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	Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.	Up to approximately 8 years
Among Patient Responders, Time From Second-line Treatment Initiation to Response	Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.	Up to 6 months
Among Patient Responders, Duration of Platelet Response	Patient responders were defined as patients who reach a response level of ≥ 50 × 10^9/L within 6 months.	Up to approximately 8 years
Number and Percentage of Patient Responders With Treatment-free Response	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.	Up to 3 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): May 16, 2025
• Primary Completion (Actual): November 11, 2025
• Study Completion (Actual): November 11, 2025

Study Registration Dates

• First Submitted: September 1, 2026
• First Submitted That Met QC Criteria: September 1, 2026
• First Posted (Actual): September 4, 2026

Study Record Updates

• Last Update Posted (Actual): September 4, 2026
• Last Update Submitted That Met QC Criteria: September 1, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Effectiveness; Treatment Patterns; Primary Immune Thrombocytopenia; Retrospective Cohort; Healthcare Resource Utilization
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: CVAY736I12001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO