Standards of care for spinal muscular atrophy
TREAT-NMD has been working with the authors of the recently published consensus statement on care for patients with spinal muscular atrophy to create useful summary factsheets based on the full published document. A TREAT-NMD working group is continuing to develop the standards for care on SMA in areas such as physical and occupational therapy, orthopaedics, nutrition and psychosocial implications. We will be looking for volunteers from amongst the partners and others to help with the generation of these further guidelines soon.
We are very interested in talking with patients and clinicians about these recommendations and suggestions for their future improvement. If you're interested, please email the TREAT-NMD team at .
Translations
We believe it is crucial to have this type of information about standards of care available in patients' and clinicians' native languages, and we are therefore in the process of translating it into a number of languages with the generous help of multilingual staff within the TREAT-NMD network and colleagues from patient organisations. If you are interested in translating our factsheets into your language for hosting on our website, please contact the TREAT-NMD team at - we'd love to hear from you!
Recently added translations include: Polish, Dutch, Italian and Turkish.
Translations that are currently in progress include: Slovenian
Document downloads
Individual factsheets are available for download, along with the original "Consensus Statement for Standard of Care in Spinal Muscular Atrophy" - see the downloads section to the right.
More about the précis
This precis outlines the objective, methods, and results of the work by the SCC on identifying guidelines for standards of care for SMA. The full report, entitled “Consensus Statement for Standard of Care in Spinal Muscular Atrophy”, J Child Neurology 2007: 22: 1027, may be obtained from the journal's website or from the downloads section to the right.
Objective
To identify practice guidelines for the care of spinal muscular atrophy (SMA). Recent progress in the understanding of the molecular pathogenesis of SMA and improvements in medical technology have not been matched by similar developments in the care for SMA patients. Variations in medical practice coupled with differences in family resources and values have resulted in variable clinical outcomes that are likely to compromise the valid measurement of treatment effects during clinical trial studies.
Methods
The International Standard of Care Committee (SCC) for SMA was formed in 2005 with the goal of establishing practice guidelines for the clinical care of SMA patients. The 12 core committee members worked with over 60 SMA experts in the field; through conference calls, e-mail communications, Delphi survey, and two in-person meetings to achieve consensus on 5 care areas: Diagnostic/New Interventions, Pulmonary, Gastrointestinal/Nutrition, Orthopedics/Rehabilitation, and Palliative Care.
Results
Consensus was achieved in several topics regarding common medical problems in SMA, the diagnostic strategies, recommendations for assessment and monitoring, and for therapeutic interventions in each care area. A consensus statement was drafted to address the 5 care areas according to three functional levels of the patients: non-sitter, sitter, and walker. Several medical practices were also identified where there is no consensus, and these warrant further investigation.
