迈向确立肾移植试验的核心结局领域：肾脏病-肾移植标准化结局共识研讨会报告
Toward Establishing Core Outcome Domains For Trials in Kidney Transplantation: Report of the Standardized Outcomes in Nephrology-Kidney Transplantation Consensus Workshops.
作者信息
Tong Allison, Gill John, Budde Klemens, Marson Lorna, Reese Peter P, Rosenbloom David, Rostaing Lionel, Wong Germaine, Josephson Michelle A, Pruett Timothy L, Warrens Anthony N, Craig Jonathan C, Sautenet Benedicte, Evangelidis Nicole, Ralph Angelique F, Hanson Camilla S, Shen Jenny I, Howard Kirsten, Meyer Klemens, Perrone Ronald D, Weiner Daniel E, Fung Samuel, Ma Maggie K M, Rose Caren, Ryan Jessica, Chen Ling-Xin, Howell Martin, Larkins Nicholas, Kim Siah, Thangaraju Sobhana, Ju Angela, Chapman Jeremy R
机构信息
1 Sydney School of Public Health, The University of Sydney, Sydney, NSW, Australia. 2 Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, NSW, Australia. 3 Division of Nephrology, University of British Columbia, Vancouver, BC, Canada. 4 Department of Nephrology, Charité-Universitätsmedizin Berlin, Germany. 5 Transplant Unit, University of Edinburgh, Edinburgh, United Kingdom. 6 Renal Division, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, United States. 7 ESRD Network 18, Los Angeles, CA. 8 Clinique Universitaire de Nephrologie, CHU Michallon, Grenoble, France. 9 Centre for Transplant and Renal Research, Westmead Hospital, NSW, Westmead, Australia. 10 Department of Medicine, The University of Chicago, Chicago, IL. 11 Department of Surgery, University of Minnesota, Minneapolis, MN. 12 School of Medicine and Dentistry, Queen Mary University of London, London, United Kingdom. 13 Department of Nephrology and Clinical Immunology, University Francois Rabelais, Tours Hospital, Tours, France. 14 INSERM, U1246, Tours, Franc Tours, France. 15 Department of Nephrology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Los Angeles, CA. 16 William B. Schwartz Division of Nephrology, Tufts Medical Center, Boston, MA. 17 Jockey Club Nephrology & Urology Centre, Princess Margaret Hospital, Hong Kong. 18 The University of Hong Kong, Queen Mary Hospital, Hong Kong. 19 Department of Nephrology, Monash Medical Centre, Melbourne, Australia. 20 Depatment of Renal Medicine, Singapore General Hospital, Singapore.
出版信息
Transplantation. 2017 Aug;101(8):1887-1896. doi: 10.1097/TP.0000000000001774.
BACKGROUND
Treatment decisions in kidney transplantation requires patients and clinicians to weigh the benefits and harms of a broad range of medical and surgical interventions, but the heterogeneity and lack of patient-relevant outcomes across trials in transplantation makes these trade-offs uncertain, thus, the need for a core outcome set that reflects stakeholder priorities.
METHODS
We convened 2 international Standardized Outcomes in Nephrology-Kidney Transplantation stakeholder consensus workshops in Boston (17 patients/caregivers; 52 health professionals) and Hong Kong (10 patients/caregivers; 45 health professionals). In facilitated breakout groups, participants discussed the development and implementation of core outcome domains for trials in kidney transplantation.
RESULTS
Seven themes were identified. Reinforcing the paramount importance of graft outcomes encompassed the prevailing dread of dialysis, distilling the meaning of graft function, and acknowledging the terrifying and ambiguous terminology of rejection. Reflecting critical trade-offs between graft health and medical comorbidities was fundamental. Contextualizing mortality explained discrepancies in the prioritization of death among stakeholders-inevitability of death (patients), preventing premature death (clinicians), and ensuring safety (regulators). Imperative to capture patient-reported outcomes was driven by making explicit patient priorities, fulfilling regulatory requirements, and addressing life participation. Specificity to transplant; feasibility and pragmatism (long-term impacts and responsiveness to interventions); and recognizing gradients of severity within outcome domains were raised as considerations.
CONCLUSIONS
Stakeholders support the inclusion of graft health, mortality, cardiovascular disease, infection, cancer, and patient-reported outcomes (ie, life participation) in a core outcomes set. Addressing ambiguous terminology and feasibility is needed in establishing these core outcome domains for trials in kidney transplantation.
背景
肾移植的治疗决策要求患者和临床医生权衡一系列医学和外科干预措施的利弊，但移植试验中存在的异质性以及缺乏与患者相关的结果，使得这些权衡具有不确定性，因此，需要一个反映利益相关者优先事项的核心结局集。
方法
我们在波士顿（17名患者/护理人员；52名卫生专业人员）和香港（10名患者/护理人员；45名卫生专业人员）召开了2次国际肾脏病-肾移植标准化结局利益相关者共识研讨会。在分组讨论环节，参与者讨论了肾移植试验核心结局领域的制定和实施。
结果
确定了七个主题。强调移植结局的至关重要性包括对透析的普遍恐惧、提炼移植功能的含义以及承认排斥反应这一可怕且模糊的术语。反映移植健康与合并症之间的关键权衡至关重要。对死亡率进行背景化解释，说明了利益相关者对死亡优先级的差异——死亡的不可避免性（患者）、预防过早死亡（临床医生）以及确保安全性（监管机构）。获取患者报告结局的必要性源于明确患者的优先事项、满足监管要求以及解决生活参与问题。还提出了移植特异性；可行性和务实性（长期影响和对干预措施的反应性）；以及认识结局领域内的严重程度梯度等需要考虑的因素。
结论
利益相关者支持在核心结局集中纳入移植健康、死亡率、心血管疾病、感染、癌症以及患者报告结局（即生活参与）。在为肾移植试验建立这些核心结局领域时，需要解决术语模糊和可行性问题。
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