Clinical trial NCT05833022: Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
Official title: Patient Characteristics and Treatment Patterns From Early Crizanlizumab Use in Real-world Setting: Preliminary Analysis From Select Sickle Cell Centers
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Sickle Cell Disease
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2023-04-27
Enrollment: 297
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study

Brief summary: This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with at least five patients who initiated crizanlizumab. Patients who were prescribed crizanlizumab were included in the cohort.


Eligibility criteria: Inclusion criteria:

* Any patient with 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 at least 6 doses of medication. The rates could be annualized if total post-index time was \<12 months.

Exclusion criteria:

None specified

Primary outcomes:
measure: Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) prior to treatment with crizanlizumab
timeFrame: Up to 12 months
measure: Percentage of patients who received sickle cell disease treatment (use of hydroxyurea, l-glutamine, chronic transfusion therapy, and voxelotor) during treatment with crizanlizumab
timeFrame: Up to 12 months
Secondary outcomes:
measure: Number of patients who discontinued treatment with crizanlizumab
timeFrame: Up to 24 months
measure: Number of doses of crizanlizumab for patients who discontinued treatment with crizanlizumab
timeFrame: Up to 24 months
measure: Reasons for early discontinuation of crizanlizumab treatment
timeFrame: Up to 24 months
measure: Number of sickle cell disease (SCD)-related hospitalizations
timeFrame: Up to 24 months
measure: Number of SCD-related emergency department (ED) visits
timeFrame: Up to 24 months
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama Birmingham
state: Alabama
city: Wilmington
country: United States
facility: Cristiana Care
state: Delaware
city: Saint Petersburg
country: United States
facility: All Children's Hospital
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Saint Louis
country: United States
facility: Washington University of St. Louis
state: Missouri
city: East Hanover
country: United States
facility: Beth Israel Hospital
state: New Jersey
city: New York
country: United States
facility: Montefiore
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Cleveland
country: United States
facility: University Hospital at Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Houston
country: United States
facility: University of Texas (Houston)
state: Texas
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: