Clinical trial NCT02263066: Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis
Official title: Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2014-10-13
Enrollment: 181
Sex: MALE
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: About 300 at most hemophilia boys (2-\<18 years) in the representative hemophilia treatment centers (HTCs) with available medical data record, having received rFVIII contained regular prophylaxis between Nov. 1st 2007 and May 31st 2013 will be enrolled from about 15 centers.

Brief summary: To assess and describe real-life treatment choices for rFⅧ contained regular prophylaxis/bleeding prevention treatment in pediatric hemophilia patients in China (2007-2013)


Eligibility criteria: Inclusion Criteria:

* Male, 2-\<18 years Hemophilia A, based on documented prior testing and/or screening laboratory Patients who had received regular prophylaxis/bleeding prevention treatment, totally/partially with rFⅧ. Regular prophylaxis/bleeding prevention treatment is defined as that proportion of weeks (≥2 infusion/week) of total treatment weeks≥80%\[10\] , and total duration ≥12 weeks. During the regular prophylaxis period, if there were consecutive 4 weeks that \<2 infusion/week, the beginning day of the 4 weeks is defined as the end of regular prophylaxis.

Available patient medical data record Written informed consent by parent/legal representative. Consent should be sought from subjects if appropriate

Exclusion Criteria:

* Patients with measurable inhibitor activity at baseline and history of FVIII inhibitor antibody formation (≥0.6BU by Bethesda assay at two different time points, documentation must be available) Any individuals with another bleeding disease that is different from hemophilia A (e.g, von Willebrand disease, hemophilia B)

Primary outcomes:
measure: Duration of regular prophylaxis
timeFrame: Up to 1.5 years
measure: Prophylaxis infusion dose
timeFrame: Up to 1.5 years
measure: Percentage of rFVIII usage in prophylaxis
timeFrame: Up to 1.5 years
Secondary outcomes:
measure: Bleeding frequency
timeFrame: Up to 1.5 years
measure: Joint radiographic score
timeFrame: Up to 1.5 years
measure: Joint physical examination score
timeFrame: Up to 1.5 years
measure: Quality of life by questionnaire
timeFrame: Up to 1.5 years
Other outcomes:
Locations:
city: Many Locations
country: China
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Li C, Zhang X, Zhao Y, Wu R, Hu Q, Xu V, Sun J, Yang R, Li X, Zhou R, Lian S, Gu J, Wu J, Hou Q. Status and trend analysis of prophylactic usage of recombinant factor VIII in Chinese pediatric patients with hemophilia A: ReCare - a retrospective, phase IV, non-interventional study. Curr Med Res Opin. 2017 Sep;33(9):1571-1578. doi: 10.1080/03007995.2017.1333489. Epub 2017 Jun 21.
citation: Li C, Zhang X, Zhao Y, Wu R, Hu Q, Xu W, Sun J, Yang R, Li X, Zhou R, Lian S, Gu J, Wu J, Hou Q. Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study. Curr Med Res Opin. 2017 Jul;33(7):1223-1230. doi: 10.1080/03007995.2017.1310720. Epub 2017 Apr 25.