Joint Outcome Study Continuation for Children With Severe Factor VIII Deficiency

Summary

The original Joint Outcome Study (JOS) enrolled 65 boys with hemophilia from 16 sites nationally. The subjects were randomized to one of two arms (prophylaxis or an enhanced episode-based treatment)and were followed prospectively until the age of six. At the age of six, the proportion of children on each treatment arm who developed bone or cartilage damage as determined by X-Ray or MRI was assessed. In addition, the function and structure of the index joints (defined as knees, ankles, and elbows)were evaluated using a physical assessment scale specially designed for preschool children.

The specific aim of the Joint Outcome Study Continuation (JOSC) is to extend observations of the children participating in the original JOS until the subjects reach the age of 18 years in order to determine the natural history of joint development in hemophilia and the impact of primary or secondary prophylaxis on the prevention, limitation, or reversal of hemophilic arthropathy. In addition, plasma and DNA will be collected and banked yearly for current and future studies of biomarkers and predictors of hemophilia outcomes.

Conditions

• Hemophilia

Sponsors & Collaborators

• Bayer

  collaborator INDUSTRY

• Rush University Medical Center

  collaborator OTHER

• Phoenix Children's Hospital

  collaborator OTHER

• The University of Texas Health Science Center, Houston

  collaborator OTHER

• Oregon Health and Science University

  collaborator OTHER

• Emory University

  collaborator OTHER

• University of Texas Southwestern Medical Center

  collaborator OTHER

• Prisma Health-Midlands

  collaborator OTHER

• Indiana University School of Medicine

  collaborator OTHER

• Intermountain Health Care, Inc.

  collaborator OTHER

• Ann & Robert H Lurie Children's Hospital of Chicago

  collaborator OTHER

• University of Colorado, Denver

  lead OTHER

Principal Investigators

• Marilyn Manco-Johnson, MD · University of Colorado at Denver Health and Sciences Center

Eligibility

Min Age

8 Years

Max Age

18 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2009-11-30

Primary Completion

2017-10-31

Completion

2017-10-31