Fischer K, Poonnoose P, Dunn A L, Babyn P, Manco-Johnson M J, David J A, van der Net J, Feldman B, Berger K, Carcao M, de Kleijn P, Silva M, Hilliard P, Doria A, Srivastava A, Blanchette V
Van Creveldkliniek, University Medical Center Utrecht, Utrecht, The Netherlands.
Department of Orthopaedics, Christian Medical College, Vellore, Tamil Nadu, India.
Haemophilia. 2017 Jan;23(1):11-24. doi: 10.1111/hae.13088. Epub 2016 Sep 15.
The implementation of early long-term, regular clotting factor concentrate (CFC) replacement therapy ('prophylaxis') has made it possible to offer boys with haemophilia a near normal life. Many different regimens have reported favourable results, but the optimum treatment regimens have not been established and the cost of prophylaxis is very high. Both for optimizing treatment and reimbursement issues, there is a need to provide objective evidence of both short- and long-term results and benefits of prophylactic regimens.
This report presents a critical review of outcome measures for use in the assessment of musculoskeletal health in persons with haemophilia according to the International Classification of Functioning, Disability and Health (ICF). This framework considers structural and functional changes, activities and participation in a context of both personal and environmental factors.
Results were generated by a combination of a critical review of available literature plus expert opinion derived from a two day consensus conference between 48 health care experts from different disciplines involved in haemophilia assessment and care. Outcome tools used in haemophilia were reviewed for reliability and validity in different patient groups and for resources required.
Recommendations for choice of outcome tools were made according to the ICF domains, economic setting, and reason for use (clinical or research). The next step will be to identify a 'core' set of outcome measures for use in clinical care or studies evaluating treatment.
早期长期规律的凝血因子浓缩物（CFC）替代疗法（“预防性治疗”）的实施，使得为血友病男孩提供近乎正常的生活成为可能。许多不同的治疗方案都报告了良好的效果，但最佳治疗方案尚未确定，且预防性治疗的费用非常高。无论是为了优化治疗还是解决报销问题，都需要提供预防性治疗方案短期和长期结果及益处的客观证据。
本报告根据国际功能、残疾和健康分类（ICF），对用于评估血友病患者肌肉骨骼健康的结局指标进行了批判性综述。该框架在个人和环境因素的背景下考虑结构和功能变化、活动及参与情况。
通过对现有文献的批判性综述以及来自48位参与血友病评估和护理的不同学科医疗保健专家为期两天的共识会议得出的专家意见相结合来产生结果。对血友病中使用的结局工具在不同患者群体中的可靠性和有效性以及所需资源进行了审查。
根据ICF领域、经济背景和使用原因（临床或研究）对结局工具的选择提出了建议。下一步将是确定一套用于临床护理或评估治疗的研究的“核心”结局指标。
