Natural History and Outcomes of Treatment of Scoliosis in Osteogenesis Imperfecta

Project Investigator: Paul Sponseller, M.D., John Hopkins Hospital
Project Co-Investigator: Peter Smith, M.D., Shriners Hospitals for Children – Chicago
Consultants: Gerald Harris, Ph.D., Sahar Hassani, M.S.

Objectives:
Osteogenesis imperfecta (OI) patients often develop scoliosis due to bone structural weakness, laxity, or multiple fractures due to the course of the disease. Treatment of scoliosis in this syndrome can be challenging; since bone mineral content is lower than in the general population, complications, including fractures and implant failures, can occur. As brace treatment is mostly ineffective in these patients, surgery may be the only option for severe curves; however, indications for surgery in OI patients are not clear and documented outcomes studies do not exist.

Methods:
OI patients with scoliosis ages 11 and over of both genders, treated either surgically or non-surgically, will be recruited. This multi-center study will also include patients from Montreal Children’s Hospital, Shriners Hospitals for Children - Montreal, Johns Hopkins-Krieger Institute, and The Hospital for Special Surgery at New York. Subjects will be identified during regular clinic visits; those who choose to participate in the study may leave at any time. A total of 100 patients will be enrolled from participating sites. Shriners Hospitals for Children - Chicago will test 25 patients. Patients in both the surgical and the non-surgical group will be followed for up to five years. Testing will be administered at two-year intervals. The following tests will be administered:

• Motion analysis
• Radiological analysis
• Evaluations by a physical therapist and an orthopedic surgeon
• Patient questionnaires, including a scoliosis survey, health survey, and pain survey

Data collected from all participating centers will be gathered at Johns Hopkins.