Clinical trial NCT05157087: XOLAIR (Omalizumab) Outcomes in Pediatric Allergic Asthma Patients in the United States
Official title: XOLAIR (Omalizumab) Outcomes in Pediatric Allergic Asthma Patients in the United States
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Asthma
Keywords: Asthma,
Omalizumab (Xolair),
Asthma Control,
Corticosteroids,
Pediatric,
Children,
Adolescents,
Healthcare Expenditures
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2021-12-14
Enrollment: 16246
Sex: ALL
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: asthmatic patients, aged 6-11, with omalizumab use

Brief summary: A retrospective database pre-post cohort study, identifying asthmatic patients, aged 6- 11, with omalizumab use over 24 months

Detailed description: This was a retrospective database pre-post cohort study, identifying asthmatic patients with omalizumab use over 24 months. Omalizumab use was analyzed in two categories. First, initiators were defined as individuals with ≥ 1 omalizumab claims. "Responders" were defined as individuals with at least 4 omalizumab claims. Descriptive statistics are provided for both groups, but outcomes are reported only for responders.

For this study, any persons from the Marketscan database with an asthma diagnosis and \>= 1 omalizumab prescription/administration during the index period were identified. From this patient population the inclusion and exclusion criteria were applied, resulting in the final study cohort. Each person was assigned an index date based on their initial use of omalizumab.

Study Period: 07/07/2015 - 12/31/2019 Index Period: 07/07/2016 - 12/31/2018 Index Date: The date of the first medical or pharmacy claim of omalizumab Baseline Period (pre-index): 12 months before index date Follow up Period (post-index): 12 months after index date

The MarketScan® Claims Database (including commercial and Medicaid claims) was used. The claims database included data from 07/08/2015 - 12/31/2019. The MarketScan® Databases capture personspecific clinical utilization, expenditures, and enrollment across inpatient, outpatient, prescription drug, and carve-out services from a selection of large employers, health plans, and government and public organizations. The current study used the Commercial, Medicare Supplemental and Multi-State Medicaid Databases.

Eligibility criteria: Inclusion criteria:

We used all eligible Marketscan beneficiaries with an asthma diagnosis and omalizumab use between 07/07/2016 - 12/31/2018 (index period). Continuous enrollment in the Marketscan database was required to ensure the availability of claims data to capture study outcomes and covariates.

* Omalizumab cohort was defined as ≥1 prescription claims within the index period, with the date of first dispensing deemed the index date.
* Asthma was defined by ≥1 diagnosis code in any available diagnosis field on or prior to index date.

ICD-9-CM: 493.xx OR ICD-10-CM: J45.x

* 6-11 years of age at the time of index
* ≥12-months pre-index and ≥12-months post-index continuous eligibility in medical and pharmacy benefits
* Enrollment gap of ≤30 days will be considered continuous enrollment

Exclusion criteria:

Patients were excluded from the study if they had one or more of the following:

* Bronchial Thermoplasty at any time during data capture. Current Procedural Terminology (CPT): 31660, 31661
* Prior asthma-indicated biologic use during the 12 months pre or post-index

Omalizumab:

NDC: 50242004062; 50242004201 or HCPCS: J2357; S0107; C9217

Mepolizumab:

NDC: 00173088101, 00173088185 or HCPCS: J2182

Reslizumab:

NDC: 5931061031 or HCPCS: J2786

Benralizumab:

NDC: 0310173030 or HCPCS: C9466

Dupilumab:

NDC: 0024591400 or 0024591800

Primary outcomes:
measure: Proportion of patients with uncontrolled asthma (impairment + risk criteria) post omalizumab treatment initiation
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Proportion of patients with excessive SABA use (≥6 SABA prescriptions dispensed in 12 months) (impairment criteria)
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Proportion of patients with exacerbations requiring IP visits (risk criteria)
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Proportion of patients with exacerbations requiring ED visits (risk criteria)
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Proportion of patients with exacerbations requiring Oral corticosteroids (OCS) prescriptions (risk criteria)
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Mean daily dose of Inhaled Corticosteroids (ICS) use
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Mean cumulative dose of OCS use
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Mean number of prescriptions of OCS use
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
measure: Mean days of supply of OCS use
timeFrame: 12 months after index date (Index date defined as the date of the first medical or pharmacy claim of omalizumab between 07/07/2016 - 12/31/2018 index period)
Secondary outcomes:
measure: Proportion of uncontrolled asthma patients (total: risk and impairment criteria)
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Proportion of uncontrolled asthma patients (per impairment criteria)
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Proportion of uncontrolled asthma patients (per risk criteria)
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Mean difference in the total number of asthma-related prescriptions
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Mean difference in daily dose (of days supply) of ICS use
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Proportion of patients with a reduction in daily dose of ICS use
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Mean difference in daily dose of OCS use
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Mean difference in number of prescriptions of OCS use
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Mean difference in days of supply of OCS use
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
measure: Proportion of patients with a reduction in total prescriptions of OCS use
timeFrame: through study completion, approximately 4 years (Study Period: 07/07/2015 - 12/31/2019)
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis Investigational Site
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: