As yet there are no results from prospective randomised studies available to inform our choice of agent, route, dosage or duration of therapy. A better understanding will result from studies already initiated in a number of centres, but could be further enhanced if there were common goals in terms of outcome assessment. To address this issue, a questionnaire has been sent out by email, fax and/or letter to over 20 investigators and groups thought to be active in this area. The questionnaire has sought to ascertain the types of databases being used, the information being collected (clinical, biochemical, hematological, radiological/densitometric, histologic, underway (including assessment of side-effects, compliance and treatment withdrawal), and the types of outcomes that are under investigation. In particular, the investigators have been asked to identify what they regard as the most important outcome(s), and how they ensure that the outcomes are being measured accurately. The issues are those of fracture number and quality of life particularly. It is hoped that there will be a complete picture by the time the meeting begins; if so, this should act as a focus for debate, and hopefully, some consensus as to the most effective way forward for us, and more importantly, our patients.

Reference: Proceedings of the 7th International Conference on Osteogenesis Imperfecta. Montreal, Canada, 1999.