Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments

Patients with spinal muscular atrophy (SMA) benefit from shared decision making around SMA-enhancing treatment decisions, according to a systematic literature review published in Neurology that engaged with multiple health care provider and community working groups around the issue.¹

Patients with SMA and their caregivers stand to benefit from shared decision-making processes | image credit: chapicha - stock.adobe.com

“Providing treatment to all individuals with SMA is essential. For infants and young children, providing treatment as soon after diagnosis as possible yields the best outcomes. For older children, adolescents and adults, providing treatment slows SMA disease progression, which is essential to maintain function, independence and improved quality of life. Selecting the best treatment for an individual with SMA is best determined through shared decision making between the patient and health care provider,” lead author Mary Schroth, MD, told The American Journal of Managed Care^®.

SMA is now recognized as a clinical emergency, following the development of 3 SMA-enhancing treatments for children under 2 years of age, and 2 treatments for older patients with SMA: nusinersen, onasemnogene abeparvovec-xioi (OA), and risdiplam. Clinicians must quickly confirm a genetic diagnosis and decide a course of treatment. But even in this short timeframe, health care providers should educate patients and caregivers about possible outcomes, then weigh their opinions before deciding to start, change, add, or discontinue a treatment, in addition to considerations of safety and side effects, the study found.

The study cites a systematic literature review on spinal motor neuron (SMN)-enhancing treatments for SMA and related studies, and opinions gathered from a working group of American and European health care providers with expertise in SMA care. The health care working group included 3 European physician neurologists, 14 US physician neurologists, and 1 US pediatric critical care physician. Members were instructed to identify barriers and create recommendations through a modified Delphi technique, a structured method to elicit and combine anonymous expert judgments.² The community working group consisted of a patient living with SMA and their caregivers.

Barriers to SMN-enhancing, disease-modifying therapies (DMTs) identified by the health care working group ranked as follows, in order of highest to lowest importance: insurance/payer issues, time to treatment initiation, insufficient data, and expectations. The group achieved a consensus rate of 89% when it came to identifying patient and family perspectives, along with treatment safety and side effects, as the prime factors to consider, around treatment.

Guidelines for communicating with the patients and caregivers included that healthcare providers should first become knowledgeable about all treatment options, including expected and possible side effects, safety monitoring requirements, currently known efficacy, and best practice regarding each treatment. Facts around treatment should be presented objectively, and informational resources should be provided, including websites of patient advocacy organizations. Other team members such as genetic counselors and care coordinators should be considered, the study found.

Other important factors to consider when initiating, altering, or adding treatment include current clinical status and comorbidities. For patients newly diagnosed with SMA, age and SMN2 gene copy number must be considered. Monitoring should occur for at least 6-12 months for medication or treatment plan changes, unless there is an urgent indication. For adolescents and adults, crucial factors to consider include quality of life, burden versus benefit of treatment analysis, and reproductive issues. Interdisciplinary and multidisciplinary care, as well as coordination among providers, were found to be critical to treatment outcomes.

SMA affects roughly 1 in 15,000 live births, according to the study.¹

“Sharing information about current knowledge of treatments and shared decision-making between health care providers and patients living with SMA and caregivers are essential to overcoming barriers to providing SMN-enhancing treatments,” the authors wrote.

References

1. Schroth M, Deans J, Bharucha Goebel DX, et al. Spinal muscular atrophy update in best practices. Neurology. 2024. doi:10.1212/CPJ.0000000000200374
2. Rowe G, Wright G. Expert opinions in forecasting: the role of the Delphi technique. In: Armstrong JS, ed. Principles of Forecasting: A Handbook for Researchers and Practitioners. Springer; 2001:125-144.