Hermans Cedric, Auerswald Günter, Benson Gary, Dolan Gerry, Duffy Anne, Jiménez-Yuste Victor, Ljung Rolf, Morfini Massimo, Lambert Thierry, Osooli Mehdi, Zupančić Šalek Silva
Division of Haematology, Cliniques Universitaires Saint-Luc, Brussels, Belgium.
Klinikum Bremen-Mitte, Professor Hess Children's Hospital, Bremen, Germany.
Eur J Haematol. 2017 Aug;99(2):103-111. doi: 10.1111/ejh.12881. Epub 2017 Apr 25.
Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15 Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population. A framework for outcome assessment in inhibitor patients incorporates traditional hemophilia outcome measures, such as bleed frequency and mortality, alongside measures of health, functioning, disability, social participation, quality of life, and economic considerations. It is important to remember that inhibitor patients differ in their clinical needs, perspectives, and priorities according to age, inhibitor status, degree of joint disease, and activity levels; as a result, the relative importance of different outcome measures will change throughout an inhibitor patient's life. Challenges inherent in measuring long-term outcomes in inhibitor patients include the small number of known patients, the subjective nature of many outcome assessment tools, and the risk of overburdening patients with repeated requests to complete questionnaires or participate in studies. Therefore, there is an urgent need to reach consensus on the most important and appropriate assessment tools for measuring outcomes in this population. These tools should ideally be standardized, easily applied, and internationally applicable in order to collect and generate quality outcome data.
几乎在血友病治疗的各个方面，近期的进展都极大地改善了患者护理与管理，新出现的治疗方法有望取得进一步进展。然而，关于血友病长期预后的数据仍然匮乏，尤其是在那些有抑制物的患者中，目前尚无经过验证的预后评估工具。在第15届苏黎世血友病论坛上，一个专家小组审查了抑制物患者最重要的预后指标，并考虑了评估该人群预后所面临的挑战。抑制物患者的预后评估框架纳入了传统的血友病预后指标，如出血频率和死亡率，以及健康、功能、残疾、社会参与、生活质量和经济考量等指标。重要的是要记住，抑制物患者根据年龄、抑制物状态、关节疾病程度和活动水平，在临床需求、观点和优先事项方面存在差异；因此，不同预后指标的相对重要性在抑制物患者的一生中会发生变化。在抑制物患者中测量长期预后所固有的挑战包括已知患者数量少、许多预后评估工具的主观性，以及因反复要求患者填写问卷或参与研究而使患者负担过重的风险。因此，迫切需要就用于测量该人群预后的最重要和最合适的评估工具达成共识。理想情况下，这些工具应标准化、易于应用且在国际上适用，以便收集和生成高质量的预后数据。
