Researchers from Spain presented the protocol of a new study on decision-making in the treatment of spinal muscular atrophy (SMA) in an article published in PLOS One.

Findings from the study will identify the treatment preferences of pediatric neurologists in SMA and clarify factors affecting these preferences, the authors said. They will also determine the role of behavioral aspects including therapeutic inertia, herding, regret, and workplace burnout in therapeutic decision-making.

With the number of treatment choices steadily increasing in SMA, pediatric neurologists are faced with the challenge of identifying the best treatment plan for their patients. To provide insight into the preferred treatment choices of pediatric neurologists who treat patients with SMA and to recognize behavioral factors that may influence decision-making, a noninterventional pilot study was designed.


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The web-based, cross-sectional study called DECISIONS-SMA involves 50 pediatric neurologists managing patients with SMA in Spain. It contains 13 simulated case scenarios of common SMA presentations.

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The primary outcome measure of the study is the pediatric neurologists’ treatment preferences according to the percentages of participants who initiate treatment when recommended, switch therapies when the disease progresses, and discontinue treatment when the disease progresses to the point that patients fall outside of the treatment recommendation. 

Secondary outcome measures include the prevalence of therapeutic inertia, defined as failure to initiate or intensify therapy when therapeutic goals are not reached; the prevalence of herding phenomenon, where neurologists copy others’ therapeutic decisions rather than deciding independently based on their own knowledge and information; the prevalence and intensity of care-related regret, where a neurologist believes that the current situation would have had a better outcome had they chosen a different course of action; the prevalence of occupational burnout, characterized by emotional exhaustion, depersonalization, and a low sense of personal accomplishment; and risk preferences and tolerance to uncertainty.

“These data will provide meaningful evidence to understand decision making when managing SMA in routine clinical practice,” the researchers wrote.

Reference

Saposnik G, Díaz-Abós P, Sánchez-Menéndez V, et al. Therapeutic decisions under uncertainty for spinal muscular atrophy: the DECISIONS-SMA study protocol. PLOS One. 2022;17(2):e0264006. doi:10.1371/journal.pone.0264006