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

Study at a glance

From the registry record, last updated by the sponsor on 16 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

TREATMENT PATTERNS AND OUTCOMES IN PATIENTS TREATED WITH BENEFIX OR REFACTO/REFACTO AF - A SWEDISH COHORT STUDY

ClinicalTrials.gov ID

NCT02740413

Acronym / sponsor ID

B1821054

Conditions

Hemophilia A, Hemophilia B

Study type

Observational

Recruitment status

Completed

Enrolment

86 participants (actual)

Lead sponsor

PfizerIndustry (pharma, biotech, devices)

Study start

11 Jan 2016 (actual)

Primary completion

31 Dec 2017 (actual)

Study completion

31 Dec 2017 (actual)

First posted

15 Apr 2016 (estimated)

Results

Posted 3 May 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model

Cohort

Time perspective

Retrospective

Number of arms

2

Who may qualify

Sex

All

Healthy volunteers

Not accepted

Age groups

Child, Adult, Older adult

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

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

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

   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.

4. Average Prescribed Dose Per Infusion for Factor VIII and Factor IX Concentrates

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

   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.

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

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

   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).

6. Percentage of Participants on Prophylactic Treatment

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

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

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

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

   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.

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

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

   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.

10. Average Annual Registered Consumption of Factor VIII and IX Concentrates

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

    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.

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

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

    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).

12. Percentage of Time on Refacto or Benefix

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

    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.

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

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

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

15. Average Annual Number of Filled Prescriptions of Factor Concentrate

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

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

16. Average Annual Number of Dispensed Units of Factor Concentrate

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

    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.

17. Average Number of Units of Benefix or Refacto

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

    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.

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

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

    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.

20. Gilbert Joint Score

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

    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.

21. Haemophilia Joint Health Score

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

    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.

22. Percentage of Participants With Surgeries

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

    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.

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

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

    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.

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)

Results and safety

Loading the posted results from ClinicalTrials.gov…

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2025.
• Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
• To obtain contact information for a study center near you, click here.Link provided by the sponsor.
• Individual participant dataSharing planned: 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.

Related trials for Hemophilia A

• RecruitingObservational study

  The CACHE Study: Coronary Artery Care in HaEmophilia

  Haemophilia, Cardiovascular Diseases

• Active, not recruitingObservational study

  A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.

  Hemophilia A or B

• RecruitingPhase 1/2

  A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A

  Hemophilia A

• Active, not recruitingPhase 3

  A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

  Hemophilia B

• Not yet recruiting

  Safety of Intraosseous Anaesthesia in Children With Haemophilia

  Haemophilia A, Haemophilia B

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.