Currie Gillian R, Storek Jan, MacDonald Karen V, Hazlewood Glen, Durand Caylib, Bridges John F P, Mosher Dianne, Marshall Deborah A
Department of Community Health Sciences, University of Calgary, Calgary, AB, Canada.
Department of Hematology, University of Calgary, Calgary, AB, Canada.
Patient. 2025 Mar;18(2):161-171. doi: 10.1007/s40271-024-00724-4. Epub 2024 Dec 12.
Allogeneic bone marrow transplantation (BMT) may be a curative treatment for patients with rheumatoid arthritis (RA), but it has serious risks, including death. It is uncertain whether patients would accept the risks and benefits of BMT and participate in clinical trials. We conducted a discrete choice experiment (DCE) to quantify risk tolerance and benefit-risk trade-offs to inform the design of a clinical trial for BMT.
We conducted a DCE with three attributes (three levels each): chance of stopping disease progression (50-90%), increased chance of death in year after transplant (3-15%), and chance of chronic graft-versus-host disease (cGVHD) (3-15%). An orthogonal main effects design of nine binary choice tasks were presented for two scenarios: one considering their current situation and a second scenario where the patient has failed seven anti-rheumatic drugs. Participants were recruited from the Rheum4U inflammatory arthritis registry. Choice data were analyzed using a logit model accounting for multiple responses per participant.
Sixty patients participated. Most (82%) had severe disease, and the median number of anti-rheumatic drugs previously taken was 6 (range 0-18). As expected, an increased chance of stopping disease progression increases the probability of choosing BMT, while increased chance of both risks decreases the probability. Patients were willing to accept a 3% increase in risk of death or 6% increase in chance of chronic GVHD for a 10% increase in the chance of stopping disease progression. For the most clinically likely BMT risk-benefit profiles, and the likely initial target population of patients who have failed multiple biologics, between 72% and 91% of patients would choose BMT.
Patients with RA are willing to accept substantial risks for a chance to stop disease progression with BMT, suggesting that a pilot trial of BMT for RA could successfully recruit patients. Preference studies have an important role in informing patient-centered clinical trial planning and design.
异基因骨髓移植（BMT）可能是类风湿关节炎（RA）患者的一种治愈性治疗方法，但它有严重风险，包括死亡。患者是否会接受BMT的风险和益处并参与临床试验尚不确定。我们进行了一项离散选择实验（DCE），以量化风险耐受性和利弊权衡，为BMT临床试验的设计提供信息。
我们进行了一项DCE，有三个属性（每个属性三个水平）：疾病进展停止的几率（50 - 90%）、移植后一年内死亡几率增加（3 - 15%）以及慢性移植物抗宿主病（cGVHD）几率（3 - 15%）。针对两种情况呈现了九个二元选择任务的正交主效应设计：一种考虑他们的当前状况，另一种情况是患者已使用七种抗风湿药物治疗失败。参与者从Rheum4U炎症性关节炎登记处招募。使用考虑每位参与者多个反应的logit模型分析选择数据。
60名患者参与。大多数（82%）患有严重疾病，之前服用抗风湿药物的中位数为6种（范围0 - 18种）。正如预期的那样，疾病进展停止几率增加会增加选择BMT的概率，而两种风险几率增加则会降低概率。为使疾病进展停止几率增加10%，患者愿意接受死亡风险增加3%或慢性GVHD几率增加6%。对于临床上最可能的BMT风险 - 益处概况，以及可能的初始目标人群即多种生物制剂治疗失败的患者，72%至91%的患者会选择BMT。
RA患者愿意接受相当大的风险，以换取通过BMT停止疾病进展的机会，这表明针对RA的BMT试点试验可以成功招募患者。偏好研究在以患者为中心的临床试验规划和设计中具有重要作用。
