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  1. Care Approaches
  2. Understanding Roles

Care approaches in spinal muscular atrophy (SMA)

Supportive care

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Improvements in the medical care of spinal muscular atrophy have increased survival for some individuals1

Since the 1990s, 3 main areas of focus have advanced the treatment of spinal muscular atrophy1:

  • Expansion of the use of noninvasive ventilatory support
  • Awareness of the need to identify sleep-disordered breathing
  • Multidisciplinary approach to care

Survival in infantile-onset (Type I) spinal muscular atrophy has improved substantially2

TIME PERIOD

% OF PATIENTS
12-MONTH SURVIVAL

MEDIAN SURVIVAL
(months)

1980-1994

37%

7.5

1995-2006

79%

24


Adoption of comprehensive supportive care in infantile-onset (Type I) spinal muscular atrophy has increased2,7

SUPPORTIVE CARE

1980-1994
(% of patients receiving)

1995-2006
(% of patients receiving)

Noninvasive positive pressure ventilation (NIPPV)

31%

82%

Mechanical insufflation/exsufflation (MI-E)

8%

63%

Supplementary feeding

40%

78%

Spinal muscular atrophy is a neuromuscular disease that usually requires multidisciplinary medical care, or in some instances, a palliative approach1

OR

PROACTIVE CARE1,2

An approach to intensively manage the symptoms of the disease. For children with SMA, pulmonary and nutritional intervention may include earlier placement of a feeding tube, intensive respiratory support (e.g. cough assist machine), and tracheostomy

PALLIATIVE CARE1,3,4

An approach that aims to improve quality of life and relieve stress and discomfort. For children with SMA, the use of noninvasive ventilation may help avoid hospitalisation and the need for tracheostomy

As SMA progresses, the approach to care may need to change over time.1

While some children receive multidisciplinary care from physicians in their community, others go to a neuromuscular disease centre specialising in SMA

Assembling a multidisciplinary care team in the community

While spinal muscular atrophy has a single genetic cause, its presentation, progression, and needs can vary widely.5,6

Clinical specialties involved in a child’s care team may vary based on individual needs, but can include1:

SMA Paediatric Neurologist

Paediatric Neurologist*

SMA Paediatric Pulmonologist

Paediatric Pulmonologist

SMA Physiotherapist

Physiotherapist

SMA Orthopaedist

Orthopaedist*

SMA Dietitian

Dietitian*

Spinal Muscular Atrophy Caregiver

Parent/Caregiver

Parents/caregivers of children with spinal muscular atrophy, like Dany and Terence S., generally become experts in their child’s care, and should also be considered a critical part of the multidisciplinary care approach and decision-making.

For more information on the roles and responsibilities of those caring for children with SMA, click here.

Neuromuscular disease centres specialising in SMA offer expert, coordinated multidisciplinary care, but may not be accessible or practical for all children

Spinal Muscular Atrophy Care Team

A dedicated multidisciplinary care team is the emerging paradigm at these centres

Spinal Muscular Atrophy Centers

At some centres, centralised (shared) appointmentsallow families to see all necessary physicians on a single day at one facility

Spinal Muscular Atrophy Family

These centres provide care for the entire family, which may include genetic counselling and education

Spinal Muscular Atrophy Travel

These centres may provide assistance (e.g. parking, meals, lodging) for families travelling to the centre

*Actor portrayal.

REFERENCES

1. Wang CH, Finkel RS, Bertini ES, et al; and Participants of the International Conference on SMA Standard of Care. Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol. 2007;22(8):1027-1049. 2. Oskoui M, Levy G, Garland CJ, et al. The changing natural history of spinal muscular atrophy type 1. Neurology. 2007;69(20):1931-1936.3. World Health Organization. WHO definition of palliative care. http://www.who.int/cancer/palliative/definition/en/. Accessed April 25, 2016. 4. The World Federation of Right to Die Societies. Comfort and palliative care. http://www.worldrtd.net/comfort-palliative-care. Accessed April 25, 2016. 5. Darras BT, Royden Jones H Jr, Ryan MM, De Vivo DC, eds. Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach. 2nd ed. London, UK: Elsevier; 2015. 6. Markowitz JA, Singh P, Darras BT. Spinal muscular atrophy: a clinical and research update. Pediatr Neurol. 2012;46(1):1-12. 7. Chatwin M, Bush A, Simonds AK. Outcome of goal-directed non-invasive ventilation and mechanical insufflation/exsufflation in spinal muscular atrophy type I. Arch Dis Child. 2011;96:426-432.

The 2007 Consensus Statement for Standard of Care in Spinal Muscular Atrophy expresses the urgent need for a care standard to help with the multidisciplinary care of children with SMA.1

Access Wang et al, J Child Neurol, 2007