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Consensus Statement for Standard of Care in Spinal Muscular Atrophy

Stanford University Medical Center, Stanford, California, wangch{at}stanford.edu

Richard S. Finkel, MD

The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania

Enrico S. Bertini, MD

Bambino Gesu' Children's Research Hospital, Rome, Italy

Mary Schroth, MD

University of Wisconsin Children's Hospital, Madison

Anita Simonds, MD

Sleep & Ventilation Unit, Royal Brompton & Harefield NHS Trust, London, England

Brenda Wong, MD

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio

Annie Aloysius, MRCSLT, HPC

Hammersmith Hospital, London, England

Leslie Morrison, MD

University of New Mexico Health Sciences Center, Albuquerque

Marion Main, MCSP, MA

Hammersmith Hospital, London, England

Thomas O. Crawford, MD

The Johns Hopkins Hospital, Baltimore, Maryland

Anthony Trela, BS

Stanford University Medical Center, Stanford, California

Participants of the International Conference on SMA Standard of Care

Spinal muscular atrophy is a neurodegenerative disease that requires multidisciplinary medical care. Recent progress in the understanding of molecular pathogenesis of spinal muscular atrophy and advances in medical technology have not been matched by similar developments in the care for spinal muscular atrophy patients. Variations in medical practice coupled with differences in family resources and values have resulted in variable clinical outcomes that are likely to compromise valid measure of treatment effects during clinical trials. The International Standard of Care Committee for Spinal Muscular Atrophy was formed in 2005, with a goal of establishing practice guidelines for clinical care of these patients. The 12 core committee members worked with more than 60 spinal muscular atrophy experts in the field through conference calls, e-mail communications, a Delphi survey, and 2 in-person meetings to achieve consensus on 5 care areas: diagnostic/new interventions, pulmonary, gastrointestinal/nutrition, orthopedics/rehabilitation, and palliative care. Consensus was achieved on several topics related to common medical problems in spinal muscular atrophy, diagnostic strategies, recommendations for assessment and monitoring, and therapeutic interventions in each care area. A consensus statement was drafted to address the 5 care areas according to 3 functional levels of the patients: nonsitter, sitter, and walker. The committee also identified several medical practices lacking consensus and warranting further investigation. It is the authors' intention that this document be used as a guideline, not as a practice standard for their care. A practice standard for spinal muscular atrophy is urgently needed to help with the multidisciplinary care of these patients.

Key Words: spinal muscular atrophy • standard of care • consensus statement

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