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

NCT07805408 | Completed

• 1 other identifier: CVAY736I12001
• Study Type: observational
• Target: 3,714
• Locations: 1 country
• Sites: 1

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.

Conditions

Immune Thrombocytopenia

Keywords

Primary Immune Thrombocytopenia; Retrospective Cohort; Treatment Patterns; Effectiveness; Healthcare Resource Utilization

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (38)

• 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

Study Arms (1)

ITP Cohort

Primary ITP patients with prior corticosteroid treatment being treated with second-line treatment.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Evidence of a diagnosis of primary ITP during the study identification period.
• Evidence of treatment with any allowed second-line treatment during the study identification period after the first primary ITP diagnosis.
• Evidence of treatment with any allowed first-line treatment prior to index.
• Aged ≥18 years at index.

You may not qualify if:

• Fewer than 12 months continuous enrollment or activity prior to index.
• Fewer than one inpatient or fewer than two outpatient records during baseline.
• Evidence of any allowed second-line therapies or splenectomy prior to index.
• 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.
• Continuous enrollment end date or death date prior to index.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 1, 2026
• First Posted: September 4, 2026
• Study Start: May 16, 2025
• Primary Completion: November 11, 2025
• Study Completion: November 11, 2025
• Last Updated: September 4, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)