management of SMA: Multidisciplinary perspectives SMA, spinal - PowerPoint PPT Presentation

Presentation, diagnosis and management of SMA: Multidisciplinary perspectives SMA, spinal muscular atrophy.

What are the key roles of the multidisciplinary teams that manage SMA and what challenges do they face? Dr Oscar Henry Mayer Paediatric Pulmonologist and Director of the Pulmonary Function Testing Laboratory, The Children's Hospital of Philadelphia, PA, USA SMA, spinal muscular atrophy.

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SMA multidisciplinary team 1 – 3 Physical therapist Gastroenterologist Nurse manager • Maintain flexibility and • Swallowing problems • Clinic coordination • Gastrointestinal dysfunction muscle function and access Paediatric nurse Respiratory therapist Pulmonologist • Care, advice, support Patient • • Breathing problems and Use and management of and family respiratory assessment respiratory equipment Pharmacist • Medication advice Neurologist Orthopaedic surgeon Social workers • Neurological assessment • Skeletal muscle weakness • Access to equipment, Genetic counsellor and co-ordination of care and spinal instability resources and counselling • Advice and support Endocrinologist Occupational therapist Psychologist • Maintenance of bone health • Adaptive support equipment • Behaviour Paediatrician Nutritionists Clinical trials specialist • General health and development • Dietary advice and support • Research SMA, spinal muscular atrophy. 1. Mercuri E, et al. Neuromuscul Disord. 2018;28:103 – 115. 2. Finkel RS, et al. Nueromuscul Disord. 2018;28:197 – 207. 3. SMA UK. https://smauk.org.uk/whos-who-of-professionals. (Accessed December 2019).

Changing specialist roles as SMA develops Orthopaedic surgeon: Pulmonologist and Contractures, chest wall and spine deformities respiratory therapist: Endocrinologist: Respiratory support Nutritionists and Bone demineralization Social worker: gastroenterologist: and weak bones Family counselling Swallowing problems and nutrition Diagnosis Disease progression as patient gets older of SMA type 1 Neurologist: Determine and co-ordinate pharmacotherapy SMA, spinal muscular atrophy.

Challenges for SMA multidisciplinary teams Identifying and recruiting practitioners with expertise in the management of patients with SMA Practitioners having sufficient time to devote to an SMA-focused MDT Access to newer disease-modifying pharmacotherapies Access to medical resources to manage patients: airway clearance therapy, pulse oximetry, and later ventilatory support SMA, spinal muscular atrophy.

SMA recommendations: key points 1,2 Shift to a proactive approach Provision of equipment for acute care, including airway clearance and mechanical ventilation Focus on ensuring patients have adequate nutritional support New orthopaedic approaches to support the chest wall and spine, prevent progression of deformity, and maintain respiratory function SMA, spinal muscular atrophy. 1. Mercuri E, et al. Neuromuscul Disord. 2018;28:103 – 115. 2. Finkel RS, et al. Neuromuscul Disord. 2018;28:197 – 207.

Acute management plan for SMA 1,2 Acute problems are produced by inadequate airway clearance resulting from respiratory muscle weakness • Inability to cough prevents clearance of airway secretions at the start of a respiratory illness Families need to recognize respiratory illness early, and initiate an acute care plan based around airways clearance • Can minimize the need for, or duration of, hospitalization A consistent management programme in the hospital critical and sub-acute settings improves patient flow through treatment and recovery SMA, spinal muscular atrophy. 1. Chatwin M, et al. Resp Med. 2018;136:98 – 110. 2. Finkel RS, et al. Neuromuscul Disord. 2018;28:197 – 207.