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

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• Hemophilia A 866
• Hemophilia B 271

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 86 is close to the median of 80 across 314 observational studies indexed under Hemophilia A.

Lead sponsor

Pfizer is the lead sponsor of 3,244 studies on the registry; 139 are open to participants now.

Of its 582 completed or terminated interventional studies of FDA-regulated products, 381 (65%) have results posted.

Study population

Groups and cohorts

• Patients with Haemophilia A

  Patients in The MHR diagnosed with Haemophilia A

• Patients with Haemophilia B

  Patients in The MHR diagnosed with Haemophilia B

Primary outcomes

1. Age of Participants at Disease Diagnosis

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

2. Age of Participants at Start of Treatment With Benefix or Refacto

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

3. 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.

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

4. 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.

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

5. 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).

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

6. 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.

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

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

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

8. 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.

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

9. 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.

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

10. 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.

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

11. 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).

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

12. 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.

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

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

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

14. 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.

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

15. 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.

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

16. 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.

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

17. 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.

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

18. Total Number of Bleeds

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

19. 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.

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

20. 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.

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

21. 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.

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

22. 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.

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

23. 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.

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

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

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

Secondary outcomes

1. Average Annual Cost of Prescribed Factor Concentrate and Dispensed Replacement Treatment

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

2. Average Cost of Replacement Treatment Related to Invasive Procedures

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

Other outcomes

1. Average Annual Cost of Replacement Treatment Related to Invasive Procedures

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

2. Average Cost for Replacement Treatment Related to Invasive Procedures

   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.

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

Baseline characteristics

1 further baseline measures are reported on the registry.

Related links

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

Study documents

• Study protocol · Jun 16, 2015
• Statistical analysis plan · Nov 3, 2015

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No — Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.