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Back  / Patient Registries / TREAT-NMD Core Datasets / Limb Girdle Muscular Dystrophies (LGMD) Core Dataset

Limb Girdle Muscular Dystrophies (LGMD) Core Dataset

The current LGMD registry landscape is fragmented. There are number of small, subtype registries that do not routinely communicate with each other or with professional organizations such as TREAT-NMD. In addition, there is currently no universally agreed mandatory core dataset that the multiple registries have agreed to collect.  

The acceleration of therapeutic development in LGMDs, the potential need for clinical trial development and ultimately the need for post market surveillance, require collaboration and consensus on a core data set for LGMD registries. Creating a universal core data set at this point will lead to the production of more appropriate, harmonized and reusable data, which can be reliably used for the analysis of natural history data, to support clinical trial design and improve patient recruitment to trials  

In May 2020, TREAT-NMD embarked on a 3 Phase project with the aim of bringing together LGMD registries and associated stakeholders, creating and agreed Core LGMD Dataset and supporting as many registries as possible to adopt the dataset which would then be collected within a Central Global Repository, for use by academics, industry, regulators and the wider LGMD community.  

Contacts

Project Manager: Sonia Segovia (sonia.segovia@treat-nmd.com) 

Principal Investigator: Michela Guglieri (michela.guglieri@newcastle.ac.uk) 

This work is under the governance of the TREAT-NMD Global Database Oversight Committee (TGDOC) Chairs:

Chair: Anna Ambrosini (Fondazione Telethon, Milan, Italy)
Chair-Elect: Michela Guglieri (Newcastle University and Newcastle Hospitals NHS Foundation Trust, United Kingdom)
Outgoing Chair: Craig Campbell (Children’s Hospital, London Health Sciences Centre and Western University, Canada)

LGMD Dataset development phase

This phase of the project aimed to establish the LGMD global registry network as affiliates of TREAT-NMD and reach consensus on a feasible core data set, that all members agree to collect.

The project goals and objectives include:

  • Identification of appropriate stakeholders and working group for LGMD Core Data Set Project
  • Achievement of an agreed LGMD Core Data Set
  • Creation of an Implementation Plan for LGMD Core Data Set
  • Determine the Requirements & Costs for Core Dataset Roll Out and Adoption by LGMD Registries

In recognition of the urgent need to bring cohesion to the various LGMD registries, TREAT-NMD established an LGMD Advisory Group. This group includes highly recognized academics and clinical experts in LGMD.

To gain a better understanding of the existing registries collecting LGMD data, TREAT NMD undertook an initial stakeholder mapping and survey. This provided us with an initial overview of the current status of LGMD registry provision,  and gave us some insight into the current data sets employed by different registries. A poster with the results of this mapping was presented in the last TREAT-NMD Conference in 2019.

Using the information obtained from the stakeholder mapping survey, we defined a LGMD Stakeholder Working Group with representations from different stakeholders.  

The dataset development phase has been conducted through 4 virtual workshops, each with a different objective:

1) Meeting 1. Define the objectives of LGMD registries 

2) Meeting 2. Define the LGMD Core Dataset 

3) Meeting 3. Assess the feasibility of the LGMD Core Dataset 

4) Meeting 4. Review and approve the definitive version (V1.0) of LGMD Core dataset. 

LGMD Dataset Workshops

The aim of the Core Dataset Scoping Workshops is to reach consensus on a feasible LGMD core data set and to establish the LGMD global registry network. 

Workshops structure

The workshop has been divided into 4 virtual meetings, each of them with a specific objective.  

1) Meeting 1. Define the objectives of LGMD registries 

2) Meeting 2. Define the LGMD Core Dataset 

3) Meeting 3. Assess the feasibility of the LGMD Core Dataset 

4) Meeting 4. Review and approve the definitive version (V1.0) of LGMD Core dataset. 

1st Workshop Meeting – LGMD registry objectives

This online meeting took place on 17 September 2020, with the aim of discussing possible objectives for the LGMD registry, considering the perspectives of different stakeholders. The meeting report and appendices and speaker slides are available here:

2nd Workshop Meeting – LGMD Dataset definition

This online meeting took place on 5thNovember 2020, with the purpose of to reach consensus on the data items that together will form the LGMD Core Dataset, assessing the importance and value of each data item. The aim of the meeting was focus especially in defining the “must” (mandatory) items. The meeting report and appendices are available here:

3rd Workshop Meeting – LGMD Dataset Feasibility assessment

This online meeting took place on 12nd November 2020. The purpose of the 3rd workshop meeting was to: 

  • Continue the discussion on mandatory and non-mandatory data items in the proposed LGMD Core Dataset, which were not discussed during the 2nd workshop meeting due to lack of time.  
  • Assess the feasibility of the selected LGMD core data set items for clinician reported items; patient reported items and dual reported items.   

The meeting report and appendices are available here:

4th Workshop Meeting – LGMD Dataset revision and final version

This online meeting will take place on 11th November 2020.

LGMD Core Dataset (Version 0.1: April 2021)

Please read the notes before viewing the dataset:

  1. Please note that this dataset is not the final version. The dataset is currently being piloted to assess the feasibility of data collection. The final version will be released in September 2021.
  2. Take time to read the dataset introduction; it is essential to understand how to implement the dataset in your registry.
  3. 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.
  4. 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.
  5. Personal identifying information on patients will never be requested nor accepted as part of a data submission to TREAT-NMD.
  6. This is a minimum core dataset; registries may collect additional items of local interest or relevance.
  7. TREAT-NMD is an inclusive network. If registries are not able to implement any mandatory items, they are encouraged to identify support needs, and/or a plan to work towards their inclusion, and discuss with us.

TREAT-NMD LGMD Core Dataset V 0.1 (dataset specification)

TREAT-NMD