Management of Children with Spinal Muscular Atrophy Type 1

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Management of Children with Spinal Muscular
Atrophy Type 1

• Audit, context and ethics

Benjamin Tassie David Isaacs Henry Kilham Ian
Kerridge
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• Professor David Isaacs
• Paediatric Infectious Diseases Physician,
  Children Hospital Westmead
• Associate Professor Henry Kilham
• Paediatric Respiratory Physician, Childrens
  Hospital Westmead
• Associate Professor Ian Kerridge
• Haematologist and Bone Marrow Transplant
  Physician, Royal North Shore Hospital Sydney

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Outline

• Background
• Audit
• Results
• International Context
• Discussion

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Spinal Muscular Atrophy

• Genetic 1 in 6-10 000 live births
• Features within 6 months of life
• Progressive weakness
• Intact sensorium and cognition
• Death within 1 year
• No cure

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Audit

• Aim
• To describe current practices in Australia
• To identify potential ethical issues
• Method
• National audit of 8 paediatric centres
• HREC and governance approvals
• ICD-10 search 2005-2010
• Cross checked
• Standardised audit tool

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Results
Identified 42 Excluded 4 Not located 3 Total
audited 35 (approx. 65 of all SMA 1 in time
frame)
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Results
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Results
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Results
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Results
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Discussion

• Actively managed dying
• ?Aversion re withholding food
• Limiting ventilation Vs limiting feeding
• Which standard
• Best interests
• Parental wishes
• Futility
• Burdens Vs benefits

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International context

• Guidelines
• USA
• UK
• Japan
• France
• Italy

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Discussion

• Actively managed dying
• ?Aversion re withholding food
• Limiting ventilation Vs limiting feeding
• Which standard
• Best interests
• Parental wishes
• Futility
• Burdens Vs benefits