Neurologists OK With Ambiguity More Likely to Pick Suboptimal Therapies

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Pediatric neurologists who are more comfortable with ambiguity are more likely to make suboptimal treatment decisions for their patients with spinal muscular atrophy (SMA), according to a new study from Spain.

“Our results may contribute to the growing evidence of the relevance of value-based shared decision-making for the current management of SMA,” its researchers wrote.

The study, “Therapeutic Decision-Making Under Uncertainty in the Management of Spinal Muscular Atrophy: [Results From DECISIONS-SMA Study,” was published in Neurology and Therapy.

Over the last several years, several disease-modifying treatments for SMA have become widely available. These medicines have been shown to drastically alter the course of the disease, resulting in clinical outcomes that have never been seen before.

In this rapidly evolving treatment landscape, pediatric neurologists have to make judgments about what treatments to recommend. In this study, scientists working in collaboration with the Spanish Society of Pediatric Neurology conducted a survey of pediatric neurologists to better understand how these decisions are being made.

Specifically, the researchers were interested in understanding factors related to treatment inertia, which is when a doctor recommends against starting a new treatment even when official guidelines suggest that doing so is probably the optimal call.

A total of 35 pediatric neurologists completed the survey. Among them, 62.9% were women, and the average age was just over 40 years. The participants had over a decade of experience on average, and 14 of them were SMA specialists.

In the survey, participants were asked a series of questions designed to score their aversion to ambiguity — that is, the extent to which they are willing to accept uncertain outcomes.

Participants were also presented with four clinical scenarios — for example, a 5-month-old with type 1 SMA or a 16-year-old with advanced type 2 disease — and were asked to rate, as a percentage, how much they would expect the patient to improve with treatment. Responses were used to assess participants’ general expectations about treatment.

The average scores for expectations of improvement were markedly higher for patients treated earlier in the course of disease: 59.6% for a 5-month-old with type 1 SMA, compared to 20.2% for the 16-year-old with advanced type 2.

“Pediatric neurologists had expectations of improvement with new therapies for simulated cases with early diagnosis of SMA type 1 and 2 at a young age. Their expectation of improvement markedly decreased for scenarios with a delayed diagnosis at an older age,” the researchers wrote.

To assess treatment inertia, participants were presented with 11 different clinical scenarios, and were asked to make a treatment recommendation. Out of 385 responses to these prompts, 147 (38.2%) were suboptimal recommendations indicating treatment inertia.

“[Treatment inertia] was observed in nearly all participants affecting on average over one-third of therapeutic decisions,” the researchers noted.

Statistical models, which adjusted for participant age and years of experience, indicated that treatment inertia scores tended to be higher among neurologists who had lower expectations about treatment benefits. Higher treatment inertia also was associated with lower aversion to ambiguity; in other words, neurologists who were more comfortable with uncertainty were more likely to have treatment inertia.

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“The relationship between [treatment inertia] and aversion to ambiguity is intriguing. Participants who were more prone to choose options with unknown probability were more likely to select treatment options that were not based on recommended guidelines (higher treatment inertia scores),” the researchers wrote.

“This finding may reflect that those participants may feel more comfortable in dealing with ambiguity resulting in making choices outside best practice recommendations,” the team added. The researchers noted that prior research has linked low aversion to ambiguity with a tendency toward overconfidence, which may help explain these findings.

They stressed that this was a small pilot study conducted in a single country, so the results may not be directly applicable for the SMA community worldwide. The team also acknowledged that, while judgments about treatment inertia were made based on recommended guidelines, it is probable that some neurologists were trying to prioritize patient safety in deciding not to administer treatments when efficacy is uncertain.

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