Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study

NCT02740413 | Completed Results

• 2 other identifiers: B1821054NCT02740413
• Study Type: observational
• Target: 86
• Locations: 0 countries
• Sites: N/A

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

Conditions

Hemophilia A; Hemophilia B

Outcome Measures

Primary Outcomes (24)

• Age of Participants at Disease Diagnosis

  At disease diagnosis within 11 years before the study start date (Day 1)

• Age of Participants at Start of Treatment With Benefix or Refacto

  At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1)

• Age of Participants at Start of Replacement Treatment

  Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor. Replacement treatment could be given prophylactically or to stop the bleed and/or to stop it from becoming more severe.

  At start of replacement treatment within 11 years before the study start date (Day 1)

• Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates

  Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Data was measured in international units (IU) for factor VIII and factor IX concentrates.

  For the duration of 11 years before the study start date (Day 1)

• Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates

  The average dose per kilogram body weight of factor VIII and IX was defined as a set of two derived variables from the MHR based on information on prescribed replacement treatment (Benefix or Refacto/Refacto AF): prescribed dose per infusion divided by registered body weight (all participants) and prescribed dose per week (prescribed dose per infusion multiplied by registered number of infusions per week) divided by registered body weight (only participants on prophylaxis).

  For the duration of 11 years before the study start date (Day 1)

• Percentage of Participants on Prophylactic Treatment

  Prophylactic treatment was defined as administration of drug regularly to reduce the insufficiency of coagulation factor to prevent bleeding to occur.

  For the duration of 11 years before the study start date (Day 1)

• Average Prescribed Frequency of Infusions Per Week Dispensed for Haemophilia A or B Participants With Prophylaxis

  For the duration of 11 years before the study start date (Day 1)

• Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis

  Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate was derived from the MHR registration of prescribed annual dose of factor concentrate.

  For the duration of 11 years before the study start date (Day 1)

• Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis

  Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Prescribed annual dose of factor concentrate per kilogram body weight was derived from the MHR registration of prescribed annual dose of factor concentrate and the registered body weight.

  For the duration of 11 years before the study start date (Day 1)

• Average Annual Registered Consumption of Factor VIII and IX Concentrates

  Participants diagnosed with coagulation disorders received intravenous replacement treatment of the missing coagulation factor (Factor VIII or IX). Average annual registered consumption of factor VIII and IX concentrates was defined as MHR registered participant's reports on factor concentrate consumption during calendar year.

  For the duration of 11 years before the study start date (Day 1)

• Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate

  Participants who develop inhibitors to factor VIII or IX concentrates were treated with bypassing agents in the form of activated prothrombin complex concentrate (aPCC) which was measured in units (U) and/or recombinant factor VIIa (rFVIIa) measured in micrograms (mcg).

  For the duration of 11 years before the study start date (Day 1)

• Percentage of Time on Refacto or Benefix

  Percentage of time (in days) on Refacto/Refacto AF or Benefix was calculated as total number of days when participant was prescribed Refacto/Refacto AF or Benefix over the total number of days on any replacement treatment. Total number of days on any replacement treatment was derived from date of start of replacement treatment according to MHR and August 31, 2015 or date of death, whichever was earliest.

  For the duration of 11 years before the study start date (Day 1)

• Average Use of Factor Concentrate Per Surgery Event at Hospital for Invasive Procedures

  For the duration of 11 years before the study start date (Day 1)

• Average Factor Concentrate Use at Hospital for Invasive Procedures

  Average factor concentrate use was calculated as percentage of total annual use of factor concentrates (in IU) at hospital for invasive procedures.

  For the duration of 11 years before the study start date (Day 1)

• Average Annual Number of Filled Prescriptions of Factor Concentrate

  Data at end of every year was based on the number of filled prescriptions during the last 3 years.

  For a duration of 3 years (for up to 11 years before the study start Day 1)

• Average Annual Number of Dispensed Units of Factor Concentrate

  To assess annual consumption of factor concentrates, calculations were based on prescription date as start of use and the day before the next prescription as the last date of use of the factor concentrates retrieved. The annual number of dispensed units of factor concentrate were then summed up of all dispensed units of factor concentrate with periods within the calendar year plus estimates of average daily use periods extending over two years. Data at end of every year was based on the number of dispensed units of factor concentrate during the last 3 years.

  For a duration of 3 years (for up to 11 years before the study start Day 1)

• Average Number of Units of Benefix or Refacto

  Data from the National Board of Health and Welfare (NBHW) had information on all filled prescriptions from July 1, 2005-last date of observation (August 31, 2015). Derived variable calculated as total number of units of filled prescriptions of Refacto/Refacto AF and Benefix over the total units of all factor VIII concentrates and factor IX concentrates, respectively. Total number of units were derived from the date of start of NBHW to last available observation or date of death, whichever was earliest.

  For the duration of 11 years before the study start date (Day 1)

• Total Number of Bleeds

  For the duration of 11 years before the study start date (Day 1)

• Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants

  Joint bleeds included traumatic and spontaneous joint bleeds, muscle bleeds included traumatic and spontaneous soft tissue bleeds and other bleed events included intracranial bleed, gastrointestinal bleed and urinary tract bleed.

  For the duration of 11 years before the study start date (Day 1)

• Gilbert Joint Score

  Gilbert joint score was an instrument to measure joint health in the domain of body structure and function (i.e. impairment), of the joints most commonly affected by bleeding in haemophilia - knees, ankles, elbows. Total score ranged from 0-100, evaluating ankle, knee and elbow, where "0" indicated normal joint function, "100" indicated worst joint function, where higher values indicated more impairment in joints.

  Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009)

• Haemophilia Joint Health Score

  Haemophilia Joint Health Score (HJHS) was used to assess joint damage in participants with haemophilia. Total score ranges from 0 to 124, where, 0 indicates normal function, and 124 indicates worst joint function, higher values indicated more damage in joints.

  Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015)

• Percentage of Participants With Surgeries

  Percentage of participants who had any type of surgery (arthrodesis, surgery on foot, nose, elbow, hand or shoulder, surgery on hip, surgery on knee, tooth extraction, venous port or any other) during the data observation period were reported.

  For the duration of 11 years before the study start date (Day 1)

• Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units

  Relative dose intensity based on dispensed volume of units was calculated using annual dispensed volume of factor concentrate per participant divided by annual prescribed dose per participant. Data was analysed for 2 categories separately: children/adolescents and adults.

  For the duration of 11 years before the study start date (Day 1)

• Average Cost of Replacement Treatment (Benefix or Refacto/Refacto AF) Related to Bleed Event

  For the duration of 11 years before the study start date (Day 1)

Secondary Outcomes (2)

• Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment

  For the duration of 11 years before the study start date (Day 1)

• Average Cost of Replacement Treatment Related to Invasive Procedures

  For the duration of 11 years before the study start date (Day 1)

Other Outcomes (2)

• Average Annual Cost of Replacement Treatment Related to Invasive Procedures

  Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)

• Average Cost for Replacement Treatment Related to Invasive Procedures

  Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)

Study Arms (2)

Patients with Haemophilia A

Patients in The MHR diagnosed with Haemophilia A

Patients with Haemophilia B

Patients in The MHR diagnosed with Haemophilia B

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer, Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2015
• First Posted: April 15, 2016
• Study Start: January 11, 2016
• Primary Completion: December 31, 2017
• Study Completion: December 31, 2017
• Last Updated: July 16, 2025
• Results First Posted: May 3, 2019

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share