What are the Standards of Care for SMA?

A committee of international healthcare and patient experts made recommendations for the care and management of children, young people, and adults with SMA. These were published for families in 2007 as the ‘International Standards of Care for SMA’ 1. Since then, there has been increasing evidence that people with SMA and their families can expect a better quality of life than was possible in the past, largely due to improvements in medical care and management.

It became clear that these improvements and changes in care and management practices were possibly no longer accurately reflected in the original standards. As management of SMA can require the expertise of many specialists, 26 experts and patient representatives from nine countries met during 2016 to work to update them 2. During this time, the first treatment for SMA, called Spinraza® (sometimes referred to by its generic name, nusinersen), was approved in the USA. Though a significant achievement, such treatments are not a cure and must go hand in hand with best supportive care and management. Therefore, the need to update these guidelines became even more important.

Whether or not someone is receiving drug treatment, getting good supportive care right – including symptom management, mental and social well-being – is key to achieving the best possible quality of life.

The experts were divided into working groups according to their speciality area. Within their areas of expertise, they were tasked with looking at both the latest evidence and the views of specialists from around the world as to what care and management is best. They reviewed scientific publications and sought the opinions of many other international experts.

The group acknowledged there were limited studies that related to adults with SMA but that many of the discussions and recommendations applied to best supportive care for both children and adults. The key to the most appropriate intervention and care depended critically on the child’s, young person’s or adult’s ‘functional ability’ – whether they could sit, stand or walk; whether their breathing was affected by their SMA; what other daily living activities they could manage. These SoC are therefore for all ages. However, it was agreed there needs to be more work focusing on understanding the impact of SMA over a person’s lifetime and the implications of this for adult supportive care. There are already initiatives underway or being planned in many countries to help with this which will lead to the evolution of future SoC.

The group were aware that countries have different levels of resources so made recommendations based on the minimal care and support that anyone with SMA should receive, wherever they live.

In November 2017, following these meetings and discussions, two scientific articles were published with the updated guidance3, 4. These were written for medical professionals and contained a lot of complex medical terms and detail for them to refer to in their day-to-day care for people with SMA.

The 2017 Scientific Articles

  • Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for
    diagnosis, rehabilitation, orthopaedic and nutritional care
  • Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care;
    medications, supplements and immunisations; other organ systems; and ethics