PROPACT: Retrospective Prophylaxis Patient Case Collection (PROPACT)

Prophylactic Treatment With Recombinant Factor VIIa (rFVIIa, NovoSeven®) in Haemophilia Patients With Inhibitors

This study is conducted in Europe and North and South America. The primary aim of this observational study is to evaluate the frequency and pattern of bleeding episodes in haemophilia patients receiving preventative treatment with activated recombinant human factor VII. The secondary aim is to evaluate which patients are selected for this treatment, the dose and dose intervals used, and the safety of activated recombinant human factor VII when used as prevention. The study also aims to increase understanding of the unmet medical need and clinical relevance of preventative treatment in haemophilia patients.

Study Overview

• Status: Completed
• Conditions: Congenital Bleeding Disorder; Haemophilia A With Inhibitors; Haemophilia B With Inhibitors
• Intervention / Treatment: Drug: eptacog alfa (activated)

• Study Type: Observational
• Enrollment (Actual): 86

Contacts and Locations

Study Locations

• Argentina
  • Prov. de Buenos Aires, Argentina, B1636DSU
    • Novo Nordisk Investigational Site
• Canada
  • Mississauga, Canada, L4W 4XI
    • Novo Nordisk Investigational Site
• Croatia
  • Zagreb, Croatia, 10 000
    • Novo Nordisk Investigational Site
• Czech Republic
  • Prague, Czech Republic, 16000
    • Novo Nordisk Investigational Site
• France
  • Paris La défense cedex, France, 92932
    • Novo Nordisk Investigational Site
• Germany
  • Mainz, Germany, 55127
    • Novo Nordisk Investigational Site
• Ireland
  • Dublin 2, Ireland
    • Novo Nordisk Investigational Site
• Italy
  • Rome, Italy, 00144
    • Novo Nordisk Investigational Site
• Slovakia
  • Bratislava, Slovakia, 811 05
    • Novo Nordisk Investigational Site
• Spain
  • Madrid, Spain, 28033
    • Novo Nordisk Investigational Site
• Sweden
  • Malmö, Sweden, 202 15
    • Novo Nordisk Investigational Site
• Switzerland
  • Zurich, Switzerland, CH-8050
    • Novo Nordisk Investigational Site
• United Kingdom
  • Crawley, United Kingdom, RH11 9RT
    • Novo Nordisk Investigational Site
• United States
  • New Jersey
    • Princeton, New Jersey, United States, 08540
      • Novo Nordisk Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Haemophilia patients in specialist hospital clinic or private clinic settings

Description

Inclusion Criteria:

• Haemophilia A or B with inhibitors
• Prescribed use of activated recombinant human factor VII for any type of prophylaxis with a duration of at least 30 days

Exclusion Criteria:

• Prophylaxis prescribed post-surgery
• One or more coagulation disorders in addition to haemophilia

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

activated recombinant human factor VII; Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.

Drug: eptacog alfa (activated); Retrospective data collection of the use of activated recombinant human factor VII as prophylaxis in haemophilia patients with inhibitors; Other Names: activated recombinant human factor VII

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change in Total Bleed Episodes Per Month - Bleeding Population	Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population	Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population	Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population	Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing	Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric	Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent	Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult	Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric	Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent	Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult	Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).	Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population	Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (defined as 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population	Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (= 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients	Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis to the prophylaxis period.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients	Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population	Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients	Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients	Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine	Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S

Publications and helpful links

Helpful Links

• Clinical Trials at Novo Nordisk

Study record dates

Study Major Dates

• Study Start: April 1, 2009
• Primary Completion (Actual): May 1, 2010
• Study Completion (Actual): May 1, 2010

Study Registration Dates

• First Submitted: April 14, 2009
• First Submitted That Met QC Criteria: April 15, 2009
• First Posted (Estimate): April 16, 2009

Study Record Updates

• Last Update Posted (Estimate): January 9, 2017
• Last Update Submitted That Met QC Criteria: November 17, 2016
• Last Verified: November 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hemostatic Disorders; Hemophilia A; Hemophilia B; Blood Coagulation Disorders
• Other Study ID Numbers: F7HAEM-3695