Clinical trial NCT02740413: Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study
Official title: TREATMENT PATTERNS AND OUTCOMES IN PATIENTS TREATED WITH BENEFIX OR REFACTO/REFACTO AF - A SWEDISH COHORT STUDY
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Hemophilia B
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2016-04-15
Enrollment: 86
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study population will consist of all patients diagnosed with hemophilia (D66.9 (hemophilia A) D67.9 (hemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the Malmö Hemophilia Register since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product (Benefix August 27 1997, ReFacto April 13 1999, ReFacto AF July 1 2009). Diseased individuals are included.

Brief summary: The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden

Detailed description: The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, as well as the related direct treatment costs in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF, and in subgroups (e.g. level of severity) at the MHC in Sweden.

The study population will consist of all patients diagnosed with haemophilia (D66.9 (haemophilia A) D67.9 (haemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the MHR since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product (Benefix August 27 1997, ReFacto April 13 1999, ReFacto AF July 1 2009). Diseased individuals are included. Information on drugs picked up at the pharmacy is available in the Prescribed Drug Register from 2005.

Eligibility criteria: Inclusion Criteria:

* The study population will consist of all patients diagnosed with hemophilia (D66.9 (hemophilia A) D67.9 (hemophilia B) in International Statistical Classification of Diseases and Related Health Problems (ICD-10) that have been registered in the Malmö Hemophilia Register since 1977 and that have had at least one registered prescription of BeneFIX or ReFacto/ReFacto AF in the MHR since market authorization of the respective product

Exclusion Criteria:

* No exclusion criteria in this study

Primary outcomes:
measure: Age of Participants at Disease Diagnosis
timeFrame: At disease diagnosis within 11 years before the study start date (Day 1)
measure: Age of Participants at Start of Treatment With Benefix or Refacto
timeFrame: At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1)
measure: Age of Participants at Start of Replacement Treatment
timeFrame: At start of replacement treatment within 11 years before the study start date (Day 1)
measure: Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Percentage of Participants on Prophylactic Treatment
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Prescribed Frequency of Infusions Per Week Dispensed for Haemophilia A or B Participants With Prophylaxis
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Annual Registered Consumption of Factor VIII and IX Concentrates
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Percentage of Time on Refacto or Benefix
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Use of Factor Concentrate Per Surgery Event at Hospital for Invasive Procedures
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Factor Concentrate Use at Hospital for Invasive Procedures
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Annual Number of Filled Prescriptions of Factor Concentrate
timeFrame: For a duration of 3 years (for up to 11 years before the study start Day 1)
measure: Average Annual Number of Dispensed Units of Factor Concentrate
timeFrame: For a duration of 3 years (for up to 11 years before the study start Day 1)
measure: Average Number of Units of Benefix or Refacto
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Total Number of Bleeds
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Gilbert Joint Score
timeFrame: Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009)
measure: Haemophilia Joint Health Score
timeFrame: Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015)
measure: Percentage of Participants With Surgeries
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Cost of Replacement Treatment (Benefix or Refacto/Refacto AF) Related to Bleed Event
timeFrame: For the duration of 11 years before the study start date (Day 1)
Secondary outcomes:
measure: Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment
timeFrame: For the duration of 11 years before the study start date (Day 1)
measure: Average Cost of Replacement Treatment Related to Invasive Procedures
timeFrame: For the duration of 11 years before the study start date (Day 1)
Other outcomes:
measure: Average Annual Cost of Replacement Treatment Related to Invasive Procedures
timeFrame: Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)
measure: Average Cost for Replacement Treatment Related to Invasive Procedures
timeFrame: Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)
Locations:
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: