Core Dataset for Spinal Muscular Atrophy (SMA)
This page contains information about the core dataset for TREAT-NMD registries which are collecting data on people with SMA. This dataset was expanded in September 2018 in order to better inform on the natural history of SMA, and provide data to support post-marketing surveillance (safety and effectiveness) for new treatments.
We are currently undergoing our first formal dataset revision, and a draft of v2 will be circulated to stakeholders shortly. Feedback on the dataset is always welcome; please see the ‘Dataset Revisions and Stakeholder Input’ section below for more details.
This work is under the governance of the TREAT-NMD Global Database Oversight Committee (TGDOC) Chairs:
Chair: Craig Campbell (Children’s Hospital, London Health Sciences Centre and Western University, Canada)
Chair-Elect: Anna Ambrosini (Fondazione Telethon, Milan, Italy)
Outgoing Chair: Nathalie Goemans (Department of Paediatrics and Child Neurology, University Hospital Leuven, Belgium)
Please read the important notes below before viewing the dataset documents:
- The dataset is intended for all registries collecting data on SMA (clinician/patient/dual reported), however some items are only relevant for certain types of registry and are marked accordingly.
- Personal identifying information on patients will never be requested by a TREAT-NMD enquiry. This is included in the dataset as recommended best practise but would be for registries’ internal use only.
- The dataset is split into ‘mandatory’ and ‘highly encouraged’ items. TREAT-NMD registries are required to include all relevant mandatory items in their data collection forms.
- TREAT-NMD strives to be an inclusive network. If there are any mandatory items that registries are not easily able to implement, they are encouraged to identify their support needs, and/or a plan to work towards their inclusion, and discuss with us.
- This is a minimum standard dataset only; many registries choose to collect additional items of local interest or relevance.
This SMA Outcome Measure (OM) Library has been developed as a quick-reference tool to help patient registries in the TREAT-NMD Network choose and implement the right OMs for their patients. However this is an open resource and may also prove useful to other organisations or individuals with an interest in OMs relevant to SMA.
To comply with the TREAT-NMD Core SMA Dataset, all clinician-reported registries should collect a minimum of one validated motor measure for each SMA patient. In addition, all registries are encouraged to collect a validated patient-reported outcome measure (PROM) of their choosing. The OMs listed in the core dataset should not be viewed as restrictive; registries may collect alternative validated OMs if they wish.
We encourage independent decision-making in this area, as the best OM for any given patient can depend on many factors. Clinicians/registries should check for national or local guidelines, review the information available in this library and elsewhere, and use their own professional judgement to identify measures which will (a) pick up meaningful change in their patient cohorts and (b) be feasible for their registry to collect.
The Library contains the following information (where available) for each OM:
- Type of OM (Motor or PROM)
- Suitable age range / level of function
- Available languages
- Link to scales
- Link to manual
- Training required
- Average time needed per patient
- Equipment needed
- 2 Key references
- Notes or further information
The OM Library is a working document and will be updated as needed, so please always check this web page for the most up-to-date version. If you know of a validated OM for SMA which is missing, or if you can contribute some information, please contact the SMA Dataset Project Manager Jo Bullivant (firstname.lastname@example.org).
Please note the OM Library is protected against editing. If you would like access to filter/sort the information, please contact us.
You may also find the following resources from other groups helpful:
For patient-reported registries (and for the patient-reported elements of dual-reported registries), we have prepared some suggested patient-friendly wording for relevant data items.
Please read the important notes below before viewing the document:
- This document does not contain the full dataset – only those sections/items which are relevant for collecting patient-reported data.
- This document should therefore be viewed in the context of the full dataset to see the ‘original’ text for each data item.
- We do not intend to mandate that registries must use this wording; rather it is recommended for use in English language registries, and also as a basis for translations into other languages.
Version 1 of the Core SMA Dataset was open for consultation until Friday 27th March 2020 as part of the first formal revision process. The original plan was for Version 2 to be released in draft for consultation in May 2020, and confirmed/published at the end of June 2020.
PLEASE NOTE: Due to the unexpected volume of feedback received (over 650 comments), and the challenging working conditions due to COVID-19, it will not be possible to achieve the timeline originally planned for this revision.
Version 2 documents will be circulated in draft for the second stakeholder review as soon as they are ready. You can always find the latest updates and documents on this web page. If you can’t find the information you are looking for here please email Joanna Das (SMA Dataset Project Coordinator) or Jo Bullivant (SMA Dataset Project Manager).
For more information about the revision process, please watch this 10 Minute Briefing Presentation (click [cc] icon for subtitles).
The presentation slides are also available below, however you are strongly encouraged to watch the video to benefit from the explanations and additional context given in the commentary.
Please note: consultation on the version 1 documents closed on Friday 27th March 2020.
Documents and Links
- 10 Minute Briefing Presentation (video with subtitles available)
- 10 Minute Briefing Slides (although we recommend to watch the presentation video if possible)
- Feedback Template: please complete and send by email (by Friday 27th March 2020) to Project Manager Jo Bullivant or Project Coordinator Joanna Das.
- Feedback Log: list of all feedback received on version 1 (between Sept 2018 and 13 March 2020)
- Core SMA Dataset Revision Process: full process document
To support registries with implementing and collecting the expanded core SMA dataset, we are running 3 annual dataset workshops (2019, 2020 and 2021).
Dataset Workshop 1 (2019)
Workshop 1 took place on Friday 13 December 2019 (the day after the annual TGDOC Curators’ Meeting) at Leiden University in the Netherlands. The meeting report and speaker slides are available here:
Workshop 2 (2020)
The date and location for Workshop 2 are still to be confirmed, but it is likely to be directly after the 2020 TGDOC Curators’ Meeting (date and location also still to be confirmed). This page will be updated as soon as we have any further details.
During the first SMA Dataset Workshop (December 2019, Leiden, Netherlands), participants suggested it would be helpful to have a shared repository where Curators can upload their blank data collections forms / case report forms, for other Curators to view.
This will mean that registries who have not yet implemented the expanded dataset (or who are working on implementing it) can see how other registries are collecting it, and learn from them where relevant.
We have therefore set up a TREAT-NMD SMA Registries file sharing folder in Google Drive. Anyone with this link can upload or download documents so please do not share anything of a sensitive or confidential nature.
- PLEASE DO NOT UPLOAD ANY PATIENT DATA. This folder is for blank data collection forms only.
- If you are uploading your data collection form, please give it a clear name, such as “Registry name SMA data collection form”
This project is funded by Biogen. The deliverables are:
- Expanded dataset collected by all active TREAT-NMD SMA Registries.
- Dataset manual: data dictionary, wording for patient-reported registries, outcome measure toolkits, and standardised text for consent etc.
- Financial bursaries and 3 x annual workshops to support registry curators with implementation.
- Formal revision process to gather stakeholder feedback, harmonise with other initiatives and support continuous improvement.
Biogen do not receive any data as a result of funding this work.