Limb Girdle Muscular Dystrophies (LGMD) Core Dataset

Please read the notes before viewing the dataset:

1. Take time to read the dataset introduction; it is essential to understand how to implement the dataset in your registry.
2. The dataset is for all registries collecting data on individuals with LGMD (clinician/patient/dual reported). However, not all items are mandatory for all registries.
3. 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.
4. Personal identifying information on patients will never be requested nor accepted as part of a data submission to TREAT-NMD.
5. This is a minimum core dataset; registries may collect additional items of local interest or relevance.
6. 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 1 (dataset specification)

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 6 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.

5) Meeting 5. Present the feasibility assessment process  for the LGMD Core Dataset (Pilot).

6) Meeting 6. Review, decide and approve the definitive version (V1.0) of LGMD Core dataset. 

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. 

1^st 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:

•  1^st Workshop Meeting Report
• Appendices 1^st Workshop Meeting Report  
• Slides: LGMD Core Dataset development – Welcome and Presentation Meeting
• Slides: LGMD Core Dataset development – Objectives Survey Results

2^nd Workshop Meeting – LGMD Dataset definition

This online meeting took place on 5^thNovember 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:

•  2^nd  Workshop Meeting Report
• Appendices 2^nd  Workshop Meeting Report

3^rd Workshop Meeting – LGMD Dataset Feasibility assessment

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

• 3^rd Workshop Meeting Report
• Appendices 3^rd Workshop Meeting Report

^4th Workshop Meeting – LGMD Dataset revision and final version

This online meeting took place on 11^th November 2020.

^5th Workshop Meeting – LGMD Dataset Pilot Presentation

This online meeting took place on April 2021.

^6th Workshop Meeting – LGMD Dataset Pilot Feasibility Results

This online meeting took place on 30th September 2021. The purpose of this meeting was to review the pilot results and decide what items will be removed or remain in the dataset. The report below manifests the results and decision made the the LGMD Working Group.

• LGMD Core Dataset LGMD Data Project Working Group Feasibly Assessment Document