Real World Patient Characteristics and Treatment Patterns From Crizanlizumab Use: Preliminary Analysis From Select Sickle Cell Centers

SEG-2022-01 Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers

This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with patients who had initiated crizanlizumab between 2019 and 2022.

Study Overview

• Status: Completed
• Conditions: Sickle Cell Disease

• Study Type: Observational
• Enrollment (Actual): 376

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

Description

Inclusion criteria

• Any patient with a diagnosis of SCD based on high performance liquid chromatography (HPLC) or hemoglobin electrophoresis at treating institution.
• Treatment order for crizanlizumab; Index date was defined as the date for the first crizanlizumab treatment.
• At least 12 months of available electronic medical record (EMR) data pre-index date (baseline).
• Patients must have received at least one dose of crizanlizumab.
• For inclusion in pre/post analysis of effectiveness, patients must have received crizanlizumab for at least 12 months.

Exclusion criteria None identified.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Crizanlizumab Cohort; Patients with sickle cell disease (SCD) who had received at least one dose of crizanlizumab between 2019 and 2022.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Age at Index Date	Index date was defined as the date of the first crizanlizumab infusion	Day 1 of Year 2
Gender at Index Date	Index date was defined as the date of the first crizanlizumab infusion	Day 1 of Year 2
Race		Day 1 of Year 2
Ethnicity		Day 1 of Year 2
Insurance Type at Index Date	Index date was defined as the date of the first crizanlizumab infusion	Day 1 of Year 2
Number of Patients Categorized by Comorbidity		Baseline
Number of Patients who Received Long-acting Opioid Medication Before Starting Crizanlizumab		Baseline
Number of Patients who Received Chronic Red Blood Cell Transfusion Therapy Before Starting Crizanlizumab Categorized by Duration of Therapy	Red blood cell transfusion therapy time intervals: 0-3 months; 3-6 months; 6-12 months; >12 months; Unknown	Baseline
Number of Patients who Received Chronic Red Blood Cell Transfusion Therapy After Starting Crizanlizumab Categorized by Duration of Therapy	Red blood cell transfusion therapy time intervals: 0-3 months; 3-6 months; 6-12 months; >12 months; Unknown	Up to 1 year
Number of Patients by Type of Disease Modifying Therapy (DMT) Received Pre- and Post-index Date	Index date was defined as the date of the first crizanlizumab infusion. DMTs included: Any SCD specific therapy; Hydroxyurea; Voxelotor; L-Glutamine; Other	Up to 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Doses Received by Patients Who Continued and Discontinued Crizanlizumab Treatment		1 year
Number of SCD-related Hospitalizations and Emergency Department Visits Pre- and Post-index Date	Index date was defined as the date of the first crizanlizumab infusion.	Up to 2 years
Length of Stay of SCD-related Hospitalization Visits Pre- and Post-index Date	Index date was defined as the date of the first crizanlizumab infusion.	Up to 2 years

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): December 13, 2022
• Primary Completion (Actual): November 1, 2023
• Study Completion (Actual): November 1, 2023

Study Registration Dates

• First Submitted: October 27, 2024
• First Submitted That Met QC Criteria: October 27, 2024
• First Posted (Actual): October 29, 2024

Study Record Updates

• Last Update Posted (Actual): October 29, 2024
• Last Update Submitted That Met QC Criteria: October 27, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: CSEG101AUS20