Workshop report First international workshop on rehabilitation management and clinical outcome measures for spinal muscular atrophy Jacqueline Montes a, *, Sally Dunaway Young a , Elena Mazzone b , Marion Main c on behalf of the International Spinal Muscular Atrophy Consortium Clinical Evaluator Working Group a Departments of Rehabilitation and Regenerative Medicine and Neurology, Columbia University Medical Center, NewYork, NY, USA b Department of Paediatric Neurology, Catholic University, Rome, Italy c Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, London, UK Received 22 June 2017 1. Introduction Twenty-one physical and occupational therapists from the USA and Europe (Italy, United Kingdom and Netherlands) met in Dallas, Texas USA on October 16 and 17, 2016. The purpose of this meeting was to review the current landscape of rehabilitation management and clinical outcome measures for spinal muscular atrophy (SMA). The workshop was organized into three sessions entitled: (1) Rehabilitation and Musculoskeletal Considerations; (2) SMA Clinical Outcome Measures; and (3) Rehabilitation Devices for Evaluation and Treatment. A closing session was included to summarize the meeting topics, next steps and proposed action items. 2. Background Existing SMA clinical research networks in the USA and Europe have made considerable contributions over the past two decades to our understanding of the natural history, development of outcome measures, and care considerations for SMA. Recently the International SMA Consortium (iSMAc) was established to formalize collaboration across international SMA networks. Physical and occupational therapists within these networks provide rehabilitative care and clinical evaluations. This meeting served as an opportunity to merge our approach, standardize assessments, and facilitate collaboration for SMA research. 3. Topics discussed 3.1. Session 1: rehabilitation and musculoskeletal considerations 3.1.1. Consensus statement of care in SMA: PT and Rehabilitation Working Group update Jacqueline Montes (USA) reviewed the results from the PhysicalTherapy and Rehabilitation Working Group’s work on the revised standard of care initiative [1]. The working group was made up of representatives from North America, Europe, and Australia. A comprehensive literature review and the Delphi technique were used to establish consensus. Patient and pharmaceutical representatives were also included. Fifty-four relevant articles were identified in the literature review but highlighted a lack of good quality and quantity of available evidence. The Delphi process helped identify important topics regarding signs and symptoms, evaluations, and interventions. Intervention for stretching and range of motion, as well as strengthening and aerobic exercise were discussed. Global and cultural considerations must be considered when developing recommendations. In summary, updating the SMA standard of care guidelines is timely but recommendations are mostly based on clinical expertise. This revision includes more specific guidelines for interventions. Future work should evaluate adherence to the new guidelines as well as the impact on SMA natural history. 3.1.2. Development and evaluation of adherence to the standard of care Shree Pandya (USA) shared her experiences from participation in development of the AAN/DMD/Corticosteroids, CDC/DMD, AAN/FSHD, MDF/DM and evaluation of implementation of care recommendations using the population based Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet). Due to the lack of randomized controlled trials, a majority of the recommendations are based on consensus using different methodologies such as Delphi and modified RAND methodology [2–5]. An important methodological consideration is the importance of blinding during the early rounds of consensus building. Adherence to care recommendations can be evaluated using patient/family surveys, provider surveys, registry data, and medical record data [6,7]. Each methodology has its benefits, limitations and cost implications that will need consideration as groups move forward with evaluating implementation of SMA care recommendations. * Corresponding author. Columbia University Medical Center, 710 West 168th Street, Room 810, NewYork, NY 10032, USA. E-mail address: jm598@columbia.edu (J. Montes). http://dx.doi.org/10.1016/j.nmd.2017.07.005 Available online at www.sciencedirect.com Neuromuscular Disorders 27 (2017) 964–969 www.elsevier.com/locate/nmd ScienceDirect