Trial Outcomes & Findings for Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study (NCT NCT02740413)
NCT ID: NCT02740413
Last Updated: 2025-07-16
Results Overview
COMPLETED
86 participants
At disease diagnosis within 11 years before the study start date (Day 1)
2025-07-16
Participant Flow
The study was a retrospective population-based register study. Detailed data on each participant diagnosed with haemophilia was retrieved from the Malmö Haemophilia Register (MHR) in January 2016 covering the period of January 2005 up to December 2015.
Participant milestones
| Measure |
Benefix/Refacto/Refacto AF
Participants diagnosed with haemophilia A and B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|
|
Overall Study
STARTED
|
86
|
|
Overall Study
COMPLETED
|
86
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Benefix/Refacto/Refacto AF
n=86 Participants
Participants diagnosed with haemophilia A and B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|
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Age, Continuous
|
34 years
STANDARD_DEVIATION 20.9 • n=86 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=86 Participants
|
|
Sex: Female, Male
Male
|
83 Participants
n=86 Participants
|
|
Year of Birth for Participants
|
1985 years
n=86 Participants
|
|
Percentage of Participants Who Had Died During the Prior 11 Year Period
|
0 percentage of participants
n=86 Participants
|
|
Percentage of Participants With Severe, Moderate and Mild Haemophilia A or B
Mild
|
20 percentage of participants
n=86 Participants
|
|
Percentage of Participants With Severe, Moderate and Mild Haemophilia A or B
Moderate
|
24 percentage of participants
n=86 Participants
|
|
Percentage of Participants With Severe, Moderate and Mild Haemophilia A or B
Severe
|
56 percentage of participants
n=86 Participants
|
|
Percentage of Participants with Inhibitor Status
Current
|
2 percentage of participants
n=86 Participants
|
|
Percentage of Participants with Inhibitor Status
Ever
|
1 percentage of participants
n=86 Participants
|
|
Percentage of Participants with Inhibitor Status
Never
|
97 percentage of participants
n=86 Participants
|
|
Percentage of Participants Infected With Hepatitis C
|
40 percentage of participants
n=86 Participants
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Percentage of Participants With Positive Human Immunodeficiency Virus (HIV) status
|
5 percentage of participants
n=86 Participants
|
PRIMARY outcome
Timeframe: At disease diagnosis within 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=28 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=50 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
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Age of Participants at Disease Diagnosis
|
5 years
Standard Deviation 10.1
|
7 years
Standard Deviation 10.7
|
PRIMARY outcome
Timeframe: At start of treatment with Benefix/Refacto within 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=32 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=52 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
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Age of Participants at Start of Treatment With Benefix or Refacto
|
18 years
Standard Deviation 16.2
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30 years
Standard Deviation 20.9
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PRIMARY outcome
Timeframe: At start of replacement treatment within 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=33 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=53 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
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Age of Participants at Start of Replacement Treatment
|
16 years
Standard Deviation 15.6
|
27 years
Standard Deviation 20.3
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PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
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Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates
Factor VIII
|
1600 IU
Standard Deviation 700
|
—
|
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Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates
Factor IX
|
2500 IU
Standard Deviation 900
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
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).
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
|
Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates
Factor VIII
|
27 IU per kilogram
Standard Deviation 10
|
—
|
|
Average Prescribed Dose Per Kilogram Bodyweight for Factor VIII and Factor IX Concentrates
Factor IX
|
37 IU per kilogram
Standard Deviation 11
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
Prophylactic treatment was defined as administration of drug regularly to reduce the insufficiency of coagulation factor to prevent bleeding to occur.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=33 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=53 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
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Percentage of Participants on Prophylactic Treatment
|
78 percentage of participants
|
56 percentage of participants
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants with prophylaxis.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=25 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=28 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
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Average Prescribed Frequency of Infusions Per Week Dispensed for Haemophilia A or B Participants With Prophylaxis
|
3.9 infusions per week
Standard Deviation 1.6
|
3.1 infusions per week
Standard Deviation 2.4
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: Analysis was performed on MHR Benefix or Refacto/Refacto AF analysis set. Here, "overall number of participants analysed" signifies participants who were evaluable for this outcome measure and "number analysed" signifies number of participants with available data for each category.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=53 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
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|---|---|---|
|
Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis
Factor VIII
|
286000 IU/year
Standard Deviation 116000
|
—
|
|
Average Prescribed Annual Dose of Factor VIII and IX Concentrates for Participants on Prophylaxis
Factor IX
|
367000 IU/year
Standard Deviation 187000
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: Analysis was performed on MHR Benefix or Refacto/Refacto AF analysis set. Here, "overall number of participants analysed" signifies participants who were evaluable for this outcome measure and "number analysed" signifies number of participants with available data for each category.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=53 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis
Factor VIII
|
4500 IU/kg/year
Standard Deviation 800
|
—
|
|
Average Prescribed Annual Dose of Factor VIII and IX Concentrates Per Kilogram Bodyweight for Participants on Prophylaxis
Factor IX
|
4700 IU/kg/year
Standard Deviation 2200
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=8 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Annual Registered Consumption of Factor VIII and IX Concentrates
Factor VIII
|
80000 IU/year
Standard Deviation 70000
|
—
|
|
Average Annual Registered Consumption of Factor VIII and IX Concentrates
Factor IX
|
73000 IU/year
Standard Deviation 53000
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
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).
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate
Factor VIII
|
33 Participants
|
—
|
|
Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate
Factor IX
|
53 Participants
|
—
|
|
Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate
Factor rVIIa
|
0 Participants
|
—
|
|
Number of Participants With Consumption of Factor VIII, Factor IX, Factor rVIIa and/or aPCC Concentrate
Factor aPCC
|
0 Participants
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=76 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Percentage of Time on Refacto or Benefix
Percentage of time on Refacto
|
0 days
Interval 0.0 to 100.0
|
—
|
|
Percentage of Time on Refacto or Benefix
Percentage of time on Benefix
|
19 days
Interval 0.0 to 100.0
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=33 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=53 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Use of Factor Concentrate Per Surgery Event at Hospital for Invasive Procedures
|
13447 IU per surgery event
Standard Deviation 10695
|
24171 IU per surgery event
Standard Deviation 20976
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
Average factor concentrate use was calculated as percentage of total annual use of factor concentrates (in IU) at hospital for invasive procedures.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Factor Concentrate Use at Hospital for Invasive Procedures
|
2 percentage of factor concentrate
Standard Deviation 14
|
—
|
PRIMARY outcome
Timeframe: For a duration of 3 years (for up to 11 years before the study start Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
Data at end of every year was based on the number of filled prescriptions during the last 3 years.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Annual Number of Filled Prescriptions of Factor Concentrate
2005
|
5 prescriptions
Standard Deviation 4
|
—
|
|
Average Annual Number of Filled Prescriptions of Factor Concentrate
2008
|
11 prescriptions
Standard Deviation 8
|
—
|
|
Average Annual Number of Filled Prescriptions of Factor Concentrate
2011
|
11 prescriptions
Standard Deviation 7
|
—
|
|
Average Annual Number of Filled Prescriptions of Factor Concentrate
2014
|
11 prescriptions
Standard Deviation 7
|
—
|
PRIMARY outcome
Timeframe: For a duration of 3 years (for up to 11 years before the study start Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor IX (2005)
|
68000 dispensed units
Standard Deviation 71000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor IX (2008)
|
163000 dispensed units
Standard Deviation 140000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor IX (2011)
|
194000 dispensed units
Standard Deviation 217000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor IX (2014)
|
212000 dispensed units
Standard Deviation 200000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor VIII (2005)
|
109000 dispensed units
Standard Deviation 79000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor VIII (2008)
|
219000 dispensed units
Standard Deviation 151000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor VIII (2011)
|
220000 dispensed units
Standard Deviation 138000
|
—
|
|
Average Annual Number of Dispensed Units of Factor Concentrate
Factor VIII (2014)
|
235000 dispensed units
Standard Deviation 130000
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Number of Units of Benefix or Refacto
Refacto - Factor VIII
|
229000 units
Standard Deviation 130000
|
—
|
|
Average Number of Units of Benefix or Refacto
Benefix - Factor IX
|
191000 units
Standard Deviation 163000
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=33 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Total Number of Bleeds
|
1.1 bleeds
Standard Deviation 1.4
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=279 bleeds
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants
Joint bleed
|
68 bleeds
|
—
|
|
Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants
Muscle bleed
|
179 bleeds
|
—
|
|
Number of Joint Bleed, Muscle Bleeds and Other Bleed Events in Participants
Other bleed
|
32 bleeds
|
—
|
PRIMARY outcome
Timeframe: Data analyzed on study start Day 1 for the duration of 4 years (2005 to 2009)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=59 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Gilbert Joint Score
|
6 scores on a scale
Standard Deviation 8
|
—
|
PRIMARY outcome
Timeframe: Data analyzed on study start Day 1 for the duration of 6 years (2009 to 2015)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=75 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Haemophilia Joint Health Score
|
15 scores on a scale
Standard Deviation 17
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Percentage of Participants With Surgeries
Other surgeries
|
34 percentage of participants
|
—
|
|
Percentage of Participants With Surgeries
Arthrodesis
|
8 percentage of participants
|
—
|
|
Percentage of Participants With Surgeries
Surgery on foot, nose, elbow, hand or shoulder
|
8 percentage of participants
|
—
|
|
Percentage of Participants With Surgeries
Surgery on hip
|
7 percentage of participants
|
—
|
|
Percentage of Participants With Surgeries
Surgery on knee
|
11 percentage of participants
|
—
|
|
Percentage of Participants With Surgeries
Tooth extraction
|
17 percentage of participants
|
—
|
|
Percentage of Participants With Surgeries
Venous port
|
15 percentage of participants
|
—
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
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.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=33 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
n=53 Participants
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units
Children/Adolescents
|
103 percentage of factor concentrate
Standard Deviation 33
|
96 percentage of factor concentrate
Standard Deviation 22
|
|
Average Relative Dose Intensity of Factor Concentrate Dispensed for Haemophilia A or B Participants on Prophylaxis Based on Dispensed Volume of Units
Adults
|
88 percentage of factor concentrate
Standard Deviation 24
|
71 percentage of factor concentrate
Standard Deviation 33
|
PRIMARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=13 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Cost of Replacement Treatment (Benefix or Refacto/Refacto AF) Related to Bleed Event
|
100000 Swedish Krona (SEK)
Standard Deviation 121000
|
—
|
SECONDARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment
Prescribed Factor Concentrate
|
2085000 SEK
Standard Deviation 875000
|
—
|
|
Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment
Dispensed Replacement Treatment
|
1351000 SEK
Standard Deviation 944000
|
—
|
SECONDARY outcome
Timeframe: For the duration of 11 years before the study start date (Day 1)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "N"= number of participants evaluable for this outcome measure.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=9 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Cost of Replacement Treatment Related to Invasive Procedures
|
71000 SEK
Standard Deviation 72000
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Annual Cost of Replacement Treatment Related to Invasive Procedures
2005
|
165000 SEK
Standard Deviation 143000
|
—
|
|
Average Annual Cost of Replacement Treatment Related to Invasive Procedures
2011
|
63000 SEK
Standard Deviation 51000
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Data analyzed on study start Day 1 for Year 1 (2005) and Year 7 (2011)Population: MHR Benefix or Refacto/Refacto AF analysis set: all participants diagnosed with haemophilia A or B in ICD-10, registered in MHR since 1977, had at least 1 registered prescription of Benefix or Refacto/Refacto AF in MHR since market authorization of respective product. Here, "n"= number of participants with available data for each category.
The average cost for replacement treatment related to invasive procedures was derived from the percentage of total costs for dispensed replacement therapy and calculated as: cost of factor concentrate used for invasive procedures/ total cost of factor concentrate.
Outcome measures
| Measure |
Benefix/Refacto/Refacto AF: Haemophilia A
n=86 Participants
Participants diagnosed with haemophilia A who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
Benefix/Refacto/Refacto AF: Haemophilia B
Participants diagnosed with haemophilia B who were registered in the MHR and had at least one registered prescription of Benefix or Refacto/Refacto AF were included in the study and their data (for the duration of 11 years, i.e. 2005-2015) was retrospectively observed.
|
|---|---|---|
|
Average Cost for Replacement Treatment Related to Invasive Procedures
2011
|
14 percentage of cost
Standard Deviation 21
|
—
|
|
Average Cost for Replacement Treatment Related to Invasive Procedures
2005
|
124 percentage of cost
Standard Deviation 245
|
—
|
Adverse Events
Benefix/Refacto/Refacto AF
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER