Rita Levi Montalcini Department of Neuroscience, University of Turin Medical School, Turin, Italy.
Clinical Neurosciences, Department of Neurology and Center for Translational Neuro- and Behavioral Sciences (C-TNBS), University Hospital Essen, Essen, Germany.
Muscle Nerve. 2024 Sep;70(3):413-419. doi: 10.1002/mus.28189. Epub 2024 Jun 28.
INTRODUCTION/AIMS: The impact of treatment expectations on active treatment outcomes has not been specifically investigated in neuromuscular disorders. We thus explored in myasthenia gravis (MG) the contribution of patients' pre-treatment expectations combined with an immunosuppressant drug on treatment outcomes.
This pilot correlational study involved 17 patients with generalized MG, scheduled to start immunosuppressant azathioprine. At baseline, a healthcare professional administered: (i) the Stanford Expectations of Treatment Scale; (ii) a structured checklist paper form asking patients which side-effects they expected to develop after starting azathioprine, coupled with a standardized framing of statements. Quantitative Myasthenia Gravis (QMG) score and daily dose of concomitant drugs were assessed by neurologists as clinical outcomes. Clinical outcomes and side-effects were re-assessed at 3 and 6 months, and clinical outcomes were monitored at 18 months.
Clinically significant improvement in the QMG scores was achieved at 3 or 6 months. The level of state anxiety appeared to act as moderator of pre-treatment negative expectations (strong, positive, indicative correlation, rs = .733, p = .001). The latter were, in turn, associated with the fulfillment of side-effects that patients expected to develop with the new treatment (moderate, positive, indicative correlation, rs = .699, p = .002). No significant correlation emerged between positive and negative expectations.
Our findings show a very quick clinical response and also suggest that patients' expectations and anxiety contributed to treatment outcomes, highlighting the importance of promoting safety messages and education strategies around newly introduced treatments. Future goals include evaluating a larger cohort that includes a matched control group.
简介/目的：治疗预期对神经肌肉疾病的积极治疗结果的影响尚未被专门研究。因此，我们在重症肌无力（MG）患者中探索了患者的治疗前预期与免疫抑制剂药物联合使用对治疗结果的影响。
这项初步相关性研究涉及 17 名计划开始使用免疫抑制剂硫唑嘌呤治疗的全身性 MG 患者。在基线时，一名医疗保健专业人员进行了以下操作：（i）斯坦福治疗期望量表；（ii）一份结构化的检查表，询问患者在开始使用硫唑嘌呤后预期会出现哪些副作用，并对陈述进行标准化表述。神经科医生评估定量重症肌无力（QMG）评分和同时使用的药物的每日剂量作为临床结果。在 3 个月和 6 个月时重新评估临床结果和副作用，并在 18 个月时监测临床结果。
在 3 或 6 个月时，QMG 评分达到了显著的临床改善。状态焦虑水平似乎充当了治疗前消极预期的调节因素（强、正、指示性相关，rs =.733，p =.001）。这些预期反过来又与患者预期与新治疗相关的副作用的发生有关（中度、正、指示性相关，rs =.699，p =.002）。积极预期和消极预期之间没有显著相关性。
我们的发现表明，患者的预期和焦虑对治疗结果有贡献，治疗后很快出现临床反应，强调了在引入新治疗时推广安全信息和教育策略的重要性。未来的目标包括评估一个包括匹配对照组的更大队列。
