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

Novartis Pharmaceuticals3,714 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

3,714

Conditions

Immune Thrombocytopenia

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Novartis

East Hanover, New Jersey, United States

Outcomes

Primary Outcomes

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

Time frame: Up to approximately 8 years

Secondary Outcomes

Duration of Patient Follow-up

Time frame: Up to approximately 8 years

Time to Treatment Discontinuation (TTD)

Time frame: Up to approximately 8 years

Time to Next Treatment (TTNT)

Time frame: Up to approximately 8 years

Time to Next Treatment or Initiation of Rescue Treatment

Time frame: Up to approximately 8 years

Number and Percentage of Patients Who Discontinued Treatment Within 1, 3, and 6 Months of Second-line Treatment Initiation

Time frame: 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

Time frame: 1, 3, and 6 months

Number of Patients Who Entered a Treatment-free Interval

Data from ClinicalTrials.gov

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Treatment free interval was defined as at least 6 months with no second-line treatment or rescue treatment.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: Up to approximately 8 years

Time to Initiation of Add-on or Rescue Treatment During Second-line Treatment

Time frame: Up to approximately 8 years

Time to Initiation of Supportive Treatment During Second-line Treatment

Time frame: Up to approximately 8 years

Platelet Counts

Time frame: 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

Time frame: 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.

Time frame: 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)

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 12 months

Number of Thrombotic Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years

Time frame: 12 months

Number and Percentage of Patients With Infection Events Within 12 Months After Second-line Treatment Initiation

Time frame: 12 months

Number of Infection Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years

Time frame: 12 months

Number and Percentage of Patients With Bleeding Events Within 12 Months After Second-line Treatment Initiation

Time frame: 12 months

Number of Bleeding Events Within 12 Months After Second-line Treatment Initiation, per 1000 Person-years

Time frame: 12 months

Overall Survival (OS)

Time frame: 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.

Time frame: 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.

Time frame: 12 months

Number of Oral Corticosteroid Medication Fills Within 12 Months After Second-line Treatment Initiation, PPPY

Time frame: 12 months

Number of Days With Active Oral Corticosteroid Prescriptions Within 12 Months After Second-line Treatment Initiation, PPPY

Time frame: 12 months

Duration of IP and ICU Admissions Among Patients Who Were Hospitalized After Second-line Treatment Initiation

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: Up to 3 months