From 7 to 9 April, the Cardiopulmonary Consensus in Duchenne took place in Rome: an international meeting, organised by Parent Project aps in collaboration with Parent Project Muscular Dystrophy (PPMD).

The event brought together around 50 specialists from the United States, Italy and France – mainly cardiologists and pulmonologists – and provided an important opportunity to compare practices in different countries, highlighting strengths and differences in the clinical management of patients.

 

Why this meeting

This meeting, as well as those that preceded it, is part of Parent Project’s activities to increasingly optimise the clinical management of patients and consolidate the network of specialists not only nationally, but internationally.

The aim of this meeting was to explore the key issues related to cardiorespiratory care in Duchenne and Becker muscular dystrophy and to create the premises for the definition of clearer and more shared global guidelines: on the horizon, the goal is to optimize the care and, therefore, the health and wellbeing of patients worldwide.

In recent years Parent Project aps and PPMD have collaborated to organise a series of international events. In Italy, two meetings have been dedicated to the endocrinological and the respiratory field, while in the USA a series of workshops on cardiological care has been organised. These experiences are contributing to the strengthening of a collaborative network between Europe and the United States.

Thanks to the increasing dissemination of international care guidelines developed starting from shared standards, the Duchenne scenario continues to evolve: clinicians today are increasingly dealing with the needs of patients becoming adults and therefore presenting new complications that need to be addressed.  The scientific and patient communities live in a dynamic context: there are new investigational therapies and approved drugs, which must be taken into account in the framework of the necessary monitoring of respiratory and cardiac health.

Guiding the work was the overall purpose of:

– defining how to optimise care for all patients at all stages of the disease;

– creating an international collaborative network to exchange views and ideas, highlight best practices and different approaches across the lifespan, address and evaluate critical challenges, gaps in care and opportunities for better disease management.

 

Photos by Massimo Mazzotta

Agenda

After the welcome address, the first day was devoted to a review of the main collaborative workshops (organised by Parent Project and PPMD) held in recent years in the cardiac and pulmonary fields. To complete the picture, there was a focus on the impact of new therapies for DMD, which provided an overall view, useful as a starting point for the work.

On the second day, participants were divided into two groups to focus on respiratory and cardiac topics respectively. Each group session was followed by an open discussion, with the aim of aligning the experts’ opinions.

On the third day, the groups met to present the main conclusions and recommendations that emerged from the discussions.

Conclusions

Some significant aspects emerged from the stimulating exchanges between the groups, including:

– The importance of adopting a multidisciplinary approach, particularly for advanced cardiovascular therapies, such as VAD, transplantation and defibrillator;

– The value of longitudinal data, crucial for understanding changes over time;

– The crucial role of networking, extending the network to other clinicians and sharing data more even between centres;

– The need for clear language in recommendations, highlighting the pros and cons of each choice, enabling patients and carers to make informed decisions.

Many participants expressed enthusiasm for the high content value of the meeting, for the concrete opportunity for discussion among colleagues and for the welcome received during the event.

From this event, in addition to a consensus paper, a series of publications will be produced on the individual topics addressed by the various working groups, and up-to-date international guidelines for cardiorespiratory disease management will be defined.

The meeting was supported by Avidity Biosciences, Capricor Therapeutics, Catalyst Pharmaceuticals, Edgewise Therapeutics, Italfarmaco, ITF Therapeutics, NS Pharma, Roche, Santhera Pharmaceuticals, Sarepta Therapeutics, Secretome Therapeutics and Wave Life Sciences.

For further information: scienza@parentproject.it