Saphnelo Use in Females of Child-bearing Potential

N/ANot Yet RecruitingNCT07431775

AstraZeneca26,000,000 enrolled

Overview

Systemic lupus erythematosus (SLE) is a chronic autoimmune disease characterized by substantial clinical burden-including organ damage, increased morbidity, and mortality-that often presents in young adulthood and disproportionately affects female patients. SAPHNELO™ (anifrolumab-fnia), a fully human IgG1 κ monoclonal antibody, is a novel therapeutic option approved for add-on treatment of moderate-to-severe SLE in the United States (US) on 30 July 2021 and in the European Union on 14 February 2022. To fulfill US Food and Drug Administration (FDA) post-marketing requirements for the evaluation of anifrolumab safety in pregnancy, additional evidence is needed to better understand the real-world drug utilization of anifrolumab in female patients of reproductive potential.

Research Questions: 1. What is the incidence and prevalence of anifrolumab use among females of reproductive potential in selected countries where anifrolumab is marketed? 2. What are the baseline characteristics and drug utilization patterns in females of reproductive potential who initiate treatment with anifrolumab in selected countries where anifrolumab is marketed? Primary Objectives: 1a. To describe trends in the annual incidence of anifrolumab utilization among females of reproductive potential overall and stratified by age group and, separately, by SLE diagnosis status. 1b. To describe trends in the annual prevalence of anifrolumab utilization among females of reproductive potential overall and stratified by age group and, separately, by SLE diagnosis status. Secondary Objectives: 2\. To describe the demographic and clinical characteristics of females of reproductive potential who initiate anifrolumab use, at the point of treatment initiation. 3\. To describe SLE treatment regimens at anifrolumab initiation among females of reproductive potential with an SLE diagnosis. 4\. To describe anifrolumab utilization patterns among females of reproductive potential who initiate anifrolumab use. 5\. To enumerate and describe pregnancies with anifrolumab exposure among females of reproductive potential who initiate anifrolumab use. This is a non-interventional, retrospective, descriptive cohort study using secondary healthcare data from selected countries where anifrolumab is marketed (specifically, Denmark, France, Germany, and the US) to assess trends in, and patterns of, anifrolumab use among females of reproductive potential. The study population for the primary objectives will be female patients of reproductive potential (defined as females aged 15 to 50 years old) who are observable during the patient identification period (country-specific; market launch date through the end of available data). The study population for the secondary objectives will be female patients of reproductive potential who newly initiated anifrolumab use during the patient identification period. Secondary Objective 3 is restricted to female patients of reproductive potential who initiate anifrolumab use and have a prior record of SLE diagnosis. Study populations will be identified separately within each country-specific secondary healthcare data source from Denmark, France, Germany, and the US. Final study report delivery is planned for April 2032.

Eligibility

Sex: FEMALEMin age: 15 YearsMax age: 50 Years

Medical Language ↔ Plain English

Patients will be included in the primary objective cohort for a given calendar year interval, if they satisfy the following inclusion and exclusion criteria within that year: Inclusion Criteria: * Observability (enrollment or activity) in the data source for at least one day during the patient identification period within that calendar year (set earliest qualifying date as "index date" for primary objectives) * Female sex at index date * Age ≥15 and ≤50 years at index date Exclusion Criteria: * (Primary Objective 1a only) Evidence of prior anifrolumab use in all available data prior to the index date through 1 day prior to the index date Patients will be included in the secondary objective cohort if they satisfy the following inclusion and exclusion criteria: Inclusion criteria * Receipt of anifrolumab therapy during the patient identification period (identify earliest record of anifrolumab in this period and set as "index date" for secondary objectives). * Female sex at index date. * Age ≥15 and ≤50 years at index date. * ≥180 days of continuous observability (e.g., enrollment or activity) in the dataset prior to and including the index date. * (For Secondary Objective 3 only) SLE diagnosis, defined as having at least one unique health care encounter record with an International Classification of Diseases, 10th Revision (ICD-10) code of M32\* (excluding M32.0: drug-induced SLE) within the 180-day baseline period prior to and including the index date. Exclusion criteria * Evidence of prior anifrolumab use in all available data prior the index date through 1 day prior to the index date.

Outcomes

Primary Outcomes

Incidence of anifrolumab use

Within each country, the incidence of anifrolumab will be assessed within each calendar year interval of the overall patient identification period. The follow-up period within each calendar year interval starts on the calendar year-specific index date and ends on the earliest of the following dates within that same year: (1) last date of observability (enrollment/activity), (2) death date, (3) 51st birthday, (4) end of the study period, (5) December 31st (end of calendar year), or (6) date of anifrolumab initiation (for objective 1a only). Incidence will be reported as the number of female patients of reproductive potential with incident anifrolumab use per (1) 1,000,000 females of reproductive potential (without prior anifrolumab use) in a given calendar year, with 95% CI and (2) per 1,000,000 person-years of follow-up among females of reproductive potential (without prior anifrolumab use) in a given calendar year, with 95% CI.

Time frame: 1 year

Prevalence of anifrolumab use

Within each country, the prevalence of anifrolumab use will be assessed within each calendar year interval of the follow-up period by calculating the number and percentage with prevalent anifrolumab use among all female patients of reproductive potential in a given calendar year (with 95% CI), as well as number of female patients of reproductive potential with prevalent anifrolumab use per 1,000,000 female patients of reproductive potential in a given calendar year (with 95% CI).

Time frame: 1 year

Secondary Outcomes

Baseline patient characteristics

Patient demographics and clinical characteristics for the secondary objective cohort will be assessed using descriptive statistics over the appropriate baseline period. Categorical variables will be reported as counts with percentages, and continuous variables will be reported as mean (SD) and median (IQR). The frequency (and proportion) of missing responses will be reported for baseline patient characteristics.

Time frame: At anifrolumab treatment initiation (index date)

Baseline SLE treatment characteristics

Baseline SLE treatment utilization characteristics will be assessed over the appropriate baseline period among the patients in the secondary objective cohort with a baseline SLE diagnosis. The number and percentage of patients who have a record for each unique SLE treatment during the baseline assessment period will be reported.

Time frame: At anifrolumab treatment initiation (index date)

Anifrolumab treatment utilization patterns

Anifrolumab treatment use outcomes will be assessed in the secondary objective cohort from one day after the index date until the earliest of: last date of observability, death, 51st birthday, end of study period, or anifrolumab discontinuation (end of first episode of use). Duration of anifrolumab use (in days) will be estimated using the Kaplan-Meier method, summarized as (1) median time on treatment (with 95% CI) and (2) proportion of patients still on treatment at 84 days (with 95% CI). Additional outcome measures include total count of doses per patient (mean, SD, median, IQR, range), and proportion with anifrolumab discontinuation (defined as a gap of 90+ days between infusions, with discontinuation date as 28 days after last dose before discontinuation) during follow-up.

Time frame: From anifrolumab treatment initiation (index date) up to 84 days post-initiation

Anifrolumab-exposed pregnancies

Pregnancies that occur during anifrolumab exposure will be identified and assessed in the secondary objective cohort from one day after the index date until the earliest of: last date of observability, death, 51st birthday, end of study period, or last date of anifrolumab exposure (set as 93 days after last dose in first episode of use). Reported outcome measures will include: (1) total count of anifrolumab-exposed pregnancy episodes (cohort-level), (2) counts with percentages of patients who initiated anifrolumab use with 0, 1, or ≥2 unique anifrolumab-exposed pregnancy episodes, (3) number (and percentage) of patients who were pregnant at time of anifrolumab initiation, (4) trimester of pregnancy at anifrolumab initiation (counts/percentages for 1st, 2nd, or 3rd trimester), and (5) duration of anifrolumab exposure (mean, SD, median, IQR, range) among anifrolumab-exposed pregnancy episodes.

Time frame: From anifrolumab treatment initiation (index date) up to 93 days after date of last dose

Saphnelo Use in Females of Child-bearing Potential

Overview

Conditions

Interventions

Eligibility

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts