Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFⅧ Contained Regular Prophylaxis

NCT02263066 | Completed

• 1 other identifier: 17653
• Study Type: observational
• Target: 181
• Locations: 1 country
• Sites: 1

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)

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (3)

• Duration of regular prophylaxis

  Up to 1.5 years

• Prophylaxis infusion dose

  Up to 1.5 years

• Percentage of rFVIII usage in prophylaxis

  Up to 1.5 years

Secondary Outcomes (4)

• Bleeding frequency

  Up to 1.5 years

• Joint radiographic score

  Up to 1.5 years

• Joint physical examination score

  Up to 1.5 years

• Quality of life by questionnaire

  Up to 1.5 years

Study Arms (1)

Group 1

Chinese hemophilia A pediatric patients with medical records who had accepted regular prophylaxis, totally/partially with rFⅧ between Nov. 1st 2007 and May 31st 2013

Biological: BAY14-2222_Kogenate-FS FVIII

Interventions

BAY14-2222_Kogenate-FS FVIII BIOLOGICAL

Depends on Doctor's decision

Group 1

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Unknown Facility

Many Locations, China

Location

Related Publications (2)

• 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.

  PMID: 28532239 DERIVED

• 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.

  PMID: 28326849 DERIVED

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Bayer Study Director

  Bayer

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2014
• First Posted: October 13, 2014
• Study Start: October 1, 2014
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: July 17, 2015

Record last verified: 2015-07

Locations

CN (1)