Overview

This trial is active, not recruiting.

Conditions hemophilia a, hemophilia b
Sponsor Pfizer
Start date January 2016
End date December 2016
Trial size 1 participant
Trial identifier NCT02740413, B1821054

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective retrospective
Arm
Patients in The MHR diagnosed with Haemophilia A
Patients in The MHR diagnosed with Haemophilia B

Primary Outcomes

Measure
Year of birth for patients with Haemophilia A or B
time frame: Baseline
Percentage of children, adolescents and adults with Haemophilia A or B
time frame: Baseline
Percentage of men and women with Haemophilia A or B
time frame: Baseline
Percentage of diseased patients diagnosed with Haemophilia A or B
time frame: Baseline
Percentage of subjects diagnosed with Haemophilia A
time frame: Baseline
Percentage of subjects diagnosed with Haemophilia B
time frame: Baseline
Percentage of patients with severe, moderate and mild Haemophilia A or B
time frame: Baseline
Age at diagnosis for Haemophilia A or B
time frame: Baseline
Age at start of replacement treatment for Haemophilia A or B
time frame: Baseline
Age at start of treatment with Benefix or Refacto for Haemophilia A or B
time frame: Baseline
Percentage of patients with inhibitor status current development of inhibitors, ever, never for patients with Haemophilia A or B
time frame: Baseline
Percentage of patients that has ever been infected with Hepatitis C for patients diagnosed with Haemophilia A or B
time frame: Baseline
Percentage of Haemophilia A or B patients who have a positive HIV status
time frame: Baseline
Average prescribed dose per infusion in international units for factor IIIV and factor IX concentrates
time frame: Baseline
Average prescribed dose per kilogram in international units for factor IIIV and factor IX concentrates
time frame: Baseline
Percentage of patients on prophylactic treatment
time frame: Baseline
Average prescribed dose per kilogram bodyweight in international units for factor IIIV and factor IX concentrates for patients on prophylaxis
time frame: Baseline
Average prescribed frequency of infusion of factor IIIV and factor IX factor concentrates
time frame: Baseline
Average prescribed number of infusions of factor IIIV and factor IX concentrates per week for patients on prophylaxis
time frame: Baseline
Average prescribed annual dose of factor IIIV and IX concentrates
time frame: Baseline
Average prescribed annual dose of factor IIIV and IX concentrates per kilogram bodyweight for patients on prophylaxis
time frame: Baseline
Average annual registered consumption of factor IIIV and IX concentrates
time frame: Baseline
Number of patients with consumption of factor VIII concentrate
time frame: Baseline
Number of patients with consumption of factor IX concentrate
time frame: Baseline
Number of patients with consumption of factor rVIIa and/or aPCC concentrate
time frame: Baseline
Percentage of time on Refacto or Benefix of total time on replacement treatment
time frame: Baseline
Average use of factor concentrate at hospital for invasive procedures
time frame: Baseline
Average annual use of factor concentrate at hospital for invasive procedures
time frame: Baseline
Percentage of annual use of factor concentrate at hospital for invasive procedures of total use of factor concentrates
time frame: Baseline
Average annual number of filled prescriptions of factor concentrate
time frame: Baseline
Average annual number of dispensed units of factor concentrate
time frame: Baseline
Percentage of units of Benefix or Refacto
time frame: Baseline
Average number of bleed events
time frame: Baseline
Average number of traumatic joint bleed events
time frame: Baseline
Average number of spontaneous joint bleed events
time frame: Baseline
Average number of traumatic bleeds in soft tissue
time frame: Baseline
Average number of spontaneous bleeds in soft tissue
time frame: Baseline
Average number of intracranial bleeds
time frame: Baseline
Average number of gastrointestinal bleeds
time frame: Baseline
Average number of urinary tract bleeds
time frame: Baseline
Average value of Gilbert Joint Score
time frame: Baseline
Average value of Haemophilia Joint Health Score
time frame: Baseline
Total number of invasive procedures by type, calculated over all patients in the cohort
time frame: Baseline
To identify the number of patients that have had 1, 2, 3 or more respectively joint surgeries
time frame: Baseline
Average relative dose intensity of factor concentrate for patients on prophylaxis
time frame: Baseline
Average relative dose intensity of factor concentrate dispensed for patients on prophylaxis
time frame: Baseline

Secondary Outcomes

Measure
Average annual cost of prescribed factor concentrate
time frame: Baseline
Average annual cost of dispensed factor concentrate
time frame: Baseline
Average cost of replacement treatment related to bleed event
time frame: Baseline
Average annual cost of replacement treatment for bleed events as registered in the MHR
time frame: Baseline
Cost for bleed events registered in the MHR as percentage of total annual cost of replacement treatment
time frame: Baseline
Average cost of replacement treatment related to joint surgery as registered in the MHR
time frame: Baseline
Average annual cost of replacement treatment related to joint surgery
time frame: Baseline
Cost for replacement treatment related to joint surgery registered in the MHR as percentage of total annual cost of replacement treatment
time frame: Baseline
Average cost of replacement treatment related to invasive procedures as registered in the MHR
time frame: Baseline
Average annual cost of replacement treatment related to invasive procedures
time frame: Baseline
Cost for replacement treatment related to invasive procedures registered in the MHR as percentage of total annual cost of replacement treatment
time frame: Baseline

Eligibility Criteria

Male or female participants of any age.

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

Additional Information

Official title Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto Af - A Swedish Cohort Study
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.
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Pfizer.