Farrukh Hadia, Kunzli Abbey, Virag Olivia, O'Hara Nathan, Sprague Sheila, Cizik Amy, Gehrke-Becker Ricardo, Schubert Thomas, Ghert Michelle
Department of Surgery, McMaster University, Hamilton, ON L8S 4K1, Canada.
Department of Orthopaedics, University of Maryland, Baltimore, MD 21201, USA.
Curr Oncol. 2025 May 30;32(6):318. doi: 10.3390/curroncol32060318.
The proximal femur represents the most frequent site in the appendicular skeleton for metastatic bone disease (MBD) to occur, with a high risk for pathologic fracture. While surgical stabilization is typically used to manage fractures, reconstruction approaches are gaining popularity due to improved survival. Previous studies have focused on clinical outcomes, but patient-centered outcomes remain underexplored. This study aims to develop a patient-centered primary outcome for the Proximal FEmur Reconstruction or Internal Fixation fOR Metastases (PERFORM) Randomized Controlled Trial, employing a mixed-methods approach. First, a focus group with advanced cancer patients and caregivers identified relevant outcomes. Next, a discrete choice experiment (DCE) assessed the importance of these outcomes among stakeholders, including surgeons, patients and caregivers. The most important components for the primary outcome were identified: mortality within twelve months, physical function assessed at four months using the PROMIS Global Physical Function score, and the number of days at home within twelve months. The DCE further confirmed that survival and physical function were most prioritized. The PERFORM trial's primary outcome, developed through extensive stakeholder engagement, will guide the evaluation of surgical approaches for MBD of the proximal femur and has the potential to influence patient-centered practice.
股骨近端是四肢骨骼中转移性骨病（MBD）最常发生的部位，发生病理性骨折的风险很高。虽然手术稳定通常用于处理骨折，但由于生存率提高，重建方法越来越受欢迎。以往的研究集中在临床结果上，但以患者为中心的结果仍未得到充分探索。本研究旨在采用混合方法，为股骨近端转移瘤重建或内固定（PERFORM）随机对照试验制定以患者为中心的主要结局指标。首先，一个由晚期癌症患者和护理人员组成的焦点小组确定了相关结局。接下来，一项离散选择实验（DCE）评估了这些结局在包括外科医生、患者和护理人员在内的利益相关者中的重要性。确定了主要结局的最重要组成部分：十二个月内的死亡率、四个月时使用PROMIS全球身体功能评分评估的身体功能，以及十二个月内在家的天数。DCE进一步证实，生存和身体功能是最优先考虑的。通过广泛的利益相关者参与制定的PERFORM试验主要结局指标，将指导对股骨近端MBD手术方法的评估，并有可能影响以患者为中心的实践。
