This Article Free Full Text (Free PDF) Free References Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in Web of Science Similar articles in PubMed Alert me to new issues of the journal Add to Saved Citations Download to citation manager Request Permissions Request Reprints Add to My Marked Citations Citing Articles Citing Articles via HighWire Citing Articles via Web of Science (20) Citing Articles via Google Scholar Citing Articles via Scopus Google Scholar Articles by Wang, C. H. Search for Related Content PubMed PubMed Citation Articles by Wang, C. H. Pubmed/NCBI databases Genetics Home Reference Medline Plus Health Information Spinal Muscular Atrophy Social Bookmarking                         What's this?

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

Journal of Child Neurology, Vol. 22, No. 8, 1027-1049 (2007)
DOI: 10.1177/0883073807305788


CiteULike    Complore    Connotea    Del.icio.us    Digg    Reddit    Technorati    Twitter    What's this?

This article has been cited by other articles:

				L. van den Engel-Hoek, C. E. Erasmus, H. W. van Bruggen, B.J.M. de Swart, L. T.L. Sie, M. H. Steenks, and I. J.M. de Groot Dysphagia in spinal muscular atrophy type II: More than a bulbar problem? Neurology, November 24, 2009; 73(21): 1787 - 1791. [Abstract] [Full Text] [PDF]

				R. A. Brumback Child Neurologists Should Be Interested In Brain Tumors! J Child Neurol, November 1, 2009; 24(11): 1338 - 1340. [PDF]

				C. J. Sumner, C. D. Wee, L. C. Warsing, D. W. Choe, A. S. Ng, C. Lutz, and K. R. Wagner Inhibition of myostatin does not ameliorate disease features of severe spinal muscular atrophy mice Hum. Mol. Genet., September 1, 2009; 18(17): 3145 - 3152. [Abstract] [Full Text] [PDF]

				J. Montes, A. M. Gordon, S. Pandya, D. C. De Vivo, and P. Kaufmann Clinical Outcome Measures in Spinal Muscular Atrophy J Child Neurol, August 1, 2009; 24(8): 968 - 978. [Abstract] [PDF]

				H. B. Panitch The Pathophysiology of Respiratory Impairment in Pediatric Neuromuscular Diseases Pediatrics, May 1, 2009; 123(Supplement_4): S215 - S218. [Abstract] [Full Text] [PDF]

				G. D. Sharma Pulmonary Function Testing in Neuromuscular Disorders Pediatrics, May 1, 2009; 123(Supplement_4): S219 - S221. [Abstract] [Full Text] [PDF]

				M. K. Schroth Special Considerations in the Respiratory Management of Spinal Muscular Atrophy Pediatrics, May 1, 2009; 123(Supplement_4): S245 - S249. [Abstract] [Full Text] [PDF]

				C. Landon Novel Methods of Ambulatory Physiologic Monitoring in Patients With Neuromuscular Disease Pediatrics, May 1, 2009; 123(Supplement_4): S250 - S252. [Abstract] [Full Text] [PDF]

				L. van den Engel-Hoek, B. J. M. de Swart, C. E. Erasmus, and I. J. M. de Groot Is Head Balance a Major Determinant for Swallowing Problems in Patients With Spinal Muscular Atrophy Type 2? J Child Neurol, August 1, 2008; 23(8): 919 - 921. [Abstract] [PDF]