Saposnik Gustavo, Camacho Ana, Díaz-Abós Paola, Brañas-Pampillón María, Sánchez-Menéndez Victoria, Cabello-Moruno Rosana, Terzaghi María, Maurino Jorge, Málaga Ignacio
Clinical Outcomes and Decision Neuroscience Unit, Li Ka Shing Institute, University of Toronto, Toronto, Canada.
Division of Neurology, Department of Medicine, St. Michael's Hospital, University of Toronto, Toronto, Canada.
Neurol Ther. 2022 Sep;11(3):1209-1219. doi: 10.1007/s40120-022-00366-4. Epub 2022 Jun 3.
There are many uncertainties about treatment selection and expectations regarding therapeutic goals and benefits in the new landscape of spinal muscular atrophy (SMA). Our aim was to assess treatment preferences and expectations of pediatric neurologists caring for patients with SMA.
DECISIONS-SMA is a non-interventional, cross-sectional pilot study that assessed pediatric neurologists with expertise in SMA from across Spain. Participants were presented with 11 simulated case scenarios of common encounters of patients with SMA type 1 and 2 to assess treatment initiation, escalation, or switches. We also asked for the expected benefit with new therapies for four simulated case scenarios. Participants completed a behavioral battery to address their tolerance to uncertainty and aversion to ambiguity. The primary outcome was therapeutic inertia (TI), defined as the number of simulated scenarios with lack of treatment initiation or escalation when warranted over the total (11) presented cases.
A total of 35 participants completed the study. Participants' mean (SD) expectation for achieving an improvement by starting a new therapy for SMA type 1 (case 1, a 5-month-old) and SMA type 2 (case 6, a 1-year-old) were both 59.6% (± 21.8), but declined to 20.2% (± 12.2) for a case scenario of a 16-year-old treatment-naïve patient with long-standing SMA type 2 with severe disability. The mean (SD) TI score was 4.2 (1.7), and 3.29 (1.5) for treatment initiation. Of a total 385 individual responses, TI was observed in 147 (38.2%) of treatment choices. The multivariable analysis showed that lower aversion to ambiguity (p = 0.019) and lower expectation of treatment response (p = 0.007) were associated with higher TI after adjustment for participants' age and years of experience. Older age (p = 0.019), lower years of experience (p = 0.035), lower aversion to ambiguity (p = 0.015), and lower expectation of treatment benefits (p = 0.006) were associated with inertia for treatment initiation.
Pediatric neurologists managing patients with SMA were optimistic regarding treatment improvement in cases with early diagnosis, but had lower expectations when treatment delays and advanced patient age were present. Low aversion to ambiguity, low expectation of treatment benefits, and lower clinical experience were more likely to make suboptimal decisions, resulting in lack of treatment initiation, escalation, and TI.
在脊髓性肌萎缩症（SMA）的新领域中，关于治疗选择以及对治疗目标和益处的期望存在许多不确定性。我们的目的是评估照顾SMA患者的儿科神经科医生的治疗偏好和期望。
DECISIONS-SMA是一项非干预性横断面试点研究，评估了来自西班牙各地的具有SMA专业知识的儿科神经科医生。向参与者展示了11个1型和2型SMA患者常见情况的模拟病例场景，以评估治疗的开始、升级或转换。我们还询问了四个模拟病例场景中使用新疗法的预期益处。参与者完成了一系列行为测试，以评估他们对不确定性的耐受性和对模糊性的厌恶程度。主要结果是治疗惰性（TI），定义为在有必要进行治疗但未开始或升级治疗的模拟场景数量占所呈现的总病例数（11个）的比例。
共有35名参与者完成了研究。参与者对于为1型SMA（病例1，一名5个月大的婴儿）和2型SMA（病例6，一名1岁的儿童）开始新疗法实现改善的平均（标准差）期望均为59.6%（±21.8），但对于一名从未接受过治疗、患有长期严重残疾的16岁2型SMA患者的病例场景，这一期望降至20.2%（±12.2）。治疗开始时的平均（标准差）TI评分为4.2（1.7），治疗启动时为3.29（1.5）。在总共385个个体反应中，147个（38.2%）治疗选择中观察到了TI。多变量分析显示，在对参与者的年龄和经验进行调整后，较低的对模糊性的厌恶（p = 0.019）和较低的治疗反应期望（p = 0.007）与较高的TI相关。年龄较大（p = 0.019）、经验年限较低（p = 0.035）、对模糊性的厌恶较低（p = 0.015）以及对治疗益处的期望较低（p = 0.006）与治疗启动的惰性相关。
管理SMA患者的儿科神经科医生对于早期诊断病例的治疗改善持乐观态度，但当存在治疗延迟和患者年龄较大时，期望较低。对模糊性的厌恶程度低、对治疗益处的期望低以及临床经验较少更有可能导致做出次优决策，并导致缺乏治疗启动、升级以及治疗惰性。
