(SMA) Core Dataset
The TREAT-NMD Core Dataset for SMA supports patient registries to collect robust longitudinal data which capture natural history, measure the effectiveness of interventions and inform standards of care. The current version of the dataset V2 was released on the 16th of October 2020. V2 of the dataset was funded by Biogen. Prior to this version of the dataset there were two previous versions: version 1 (September 2018 to October 2020) & version 0 (March 2014 to September 2018).
The dataset is for all registries collecting data on individuals with SMA (clinician/patient/dual reported). However, not all items are mandatory for all registries. A mandatory data item in the core dataset means that designated registries must include it in their data collection forms and be able to store and provide the data, even if the data is not always available for all individuals. Previous versions of the SMA dataset can be found in the dropdowns below.
Version 2 of the SMA dataset has:
- 120 unique data items
- 77 mandatory items for clinically reported registries
- 55 mandatory items for patient reported registries
- 17 defined categories
- Examples, data rules & tools
Version 2 of the core SMA dataset can be found here: SMA Specification tool
This was a Biogen funded project (Biogen do not receive any data as a result of funding this work) which had deliverables:
- 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.
he SMA dataset was created by consensus with key opinion leaders, regulators, and industry representatives. There were a series of workshops regarding the dataset, this spanned over 4 years. The first workshop updated stakeholders on the current SMA landscape and coordinated a global approach to the core dataset expansion by gathering input, building consensus, and producing recommendations. SMA Core Dataset Pilot Workshop 2 (2018) brought together the 12 Pilot Registries to review feedback from the Pilot Study and other relevant initiatives. The outputs were final recommendations on the content and structure of the expanded SMA Core Dataset, and the 3-year Implementation Plan.
We received an extraordinary amount of feedback was extraordinary, the SMA community/ network was engaged and proactive within this project. It is never possible to implement all suggestions; some were judged outside the scope of a core dataset, some were flagged for consideration in future revisions, and others represent opposing viewpoints so a decision was made about which to apply. In all cases, final decisions were made by the leads of our TGDOC SMA Subgroup and the TGDOC Chairs .
Annual Reports :
TREAT NMD SMA Core Dataset V2 Appendix (guidance outside core dataset specification; for example suggested text for consent and ethics)
Outcome Measure Library
The SMA Outcome Measure (OM) Library is a quick-reference tool to help patient registries in the TREAT-NMD Network choose the right OMs for their patients. This is an open resource and may prove useful to other organisations or individuals.
To comply with the TREAT-NMD Core Dataset for SMA, clinician-reported registries must collect at least one validated motor measure for each SMA patient. In addition, all registries are encouraged to collect a validated patient-reported outcome measure (PROM). 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 because the best measure for an individual depends on many factors. Clinicians/registries should check national or local guidelines, review this library and other information available, and use professional judgement to identify measures which (a) will pick up meaningful change in their patient cohorts and (b) are 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
You may also find these resources helpful:
SMA Dataset Workshops
SMA Core Dataset Year 4 workshop (December 2022 – Vancouver, Canada).
SMA Core Dataset Year 3 workshop (19 – 20 May 2021, online)
SMA Core Dataset Year 2 Workshop 2 (27 and 30 October 2020, online)
This workshop provided a detailed introduction to version 2 of the SMA Core Dataset, to support TGDOC Curators with implementation.
SMA Core Dataset Year 2 Welcome Meeting (13 August 2020, online)
This meeting welcomed registries taking part in year 2 of the SMA Core Dataset implementation project, introduced them to the project team and each other, gave an overview of the requirements and support available, and addressed any early questions.
SMA Core Dataset Year 1 Workshop (13 December 2019, Leiden University, Netherlands)
SMA Core Dataset Pilot Workshop 2 (2018)
This workshop brought together the 12 Pilot Registries to review feedback from the Pilot Study and other relevant initiatives. The outputs were final recommendations on the content and structure of the expanded SMA Core Dataset, and the 3-year Implementation Plan.
SMA Core Dataset Pilot Workshop 1 (2017)
This workshop updated stakeholders on the current SMA landscape and coordinated a global approach to the core dataset expansion by gathering input, building consensus, and producing recommendations.
Share your data collection forms:
To support sharing of best practice we have created a shared repository where registry curators can upload their blank data collection forms for other curators to view.
The repository is in Google Drive. Anyone with this link can upload or download documents so please do not share anything sensitive or confidential.
- PLEASE DO NOT UPLOAD PATIENT DATA. This folder is for blank data collection forms only.
- When uploading a data collection form, please give it a clear name such as “UK SMA Registry data collection form”
Previous dataset versions
v1 SMA Core Dataset (September 2018 to October 2020)
If you have further questions about the SMA dataset or any questions regarding the implementation, please contact:
The Registries team – email@example.com