At the end of 2023, what are the possible treatments for children with cystic fibrosis? We talk about it with the Vaincre la Mucoviscidose association and its 2023 godmother Marine Loiseau.
- Cystic fibrosis is a fatal genetic disease linked to an abnormality of the CFTR protein, which causes poor secretion from the mucous membranes, which impairs the functioning of several organs, thereby reducing the life expectancy of patients.
- Kaftrio® triple therapy significantly improves lung function and patient weight, with a significant reduction in the need for lung transplantation.
- This year, this treatment was made accessible to more patients, with children having access to it from 2 years old, under certain conditions.
The year 2023 marked a turning point in the management of cystic fibrosis in children, with the innovative treatment Kaftrio® which was extended to patients with one of the 177 eligible transfers, from the age of six. During November, the High Authority of Health issued a favorable opinion for early access to this treatment for patients aged 2 to 5 years old, and carrying at least one F508del mutation, regardless of the nature of the second mutation and without any severity criterion. “With access to this new treatment, the management of cystic fibrosis is experiencing profound changes”, affirms the association Vaincre la Mucoviscidose. In total, thanks to these various expansions of access, 72% of people affected by cystic fibrosis can benefit from this triple therapy, or 5,500 patients.
Kaftrio® triple therapy: a treatment that reduces the need for lung transplantation
Cystic fibrosis is a fatal genetic disease linked to an abnormality of the CFTR protein. This leads to poor secretion from the mucous membranes which do not contain enough water. Not very fluid, bronchial, digestive and genital secretions are difficult to evacuate, which impairs the functioning of several organs including the lungs and the pancreas, thereby reducing the life expectancy of patients. But advances in medicine are constantly saving years of life for patients, who today have a median age of 40.8 years at death, a gain of more than 10 years over a decade, according to the conclusions of the last assessment.
Kaftrio® is composed of three molecules which act directly on the deficient CFTR protein. “Ivacaftor makes the protein work better while tezacaftor and elexacaftor increase the amount of protein present on the cell surface”, details the High Authority of Health. According to a study published in November 2020, after several months of treatment, there is a significant improvement in lung function and the patient’s weight, with a significant reduction in the need for lung transplantation. “It is an immense hope for these patients and their families, that of slowing down the progression of the disease and in particular the deterioration of their state of health, particularly on the respiratory level, from a very young age.”, adds the association.
For patients who cannot benefit from treatment due to their genetic profile, the fact that they have received a transplant, or because they are less than two years old, Vaincre la Mucoviscidose continues to support research, in particular through various animated events. by “godparents” and “godmothers”.
Marine Ltemporel, 2023 godmother of Vaincre la Cystic Fibrosis
During a press conference of the Vaincre la Mucoviscidose association at the Cystic Fibrosis Resource and Skills Center (CRCM) of the Necker-Enfants Malades hospital – AP-HP, organized this Monday, December 4, Marine Ltemporel told us expresses her enthusiasm to be the 2023 godmother of this association “which is concretely committed to improving the quality of life of patients but also to advancing research”.
The former Miss France got directly involved in the field with the Virades de l’Espoir. “These are races organized throughout Franceshe explains to Why Doctor. The funds raised help finance the association and research against cystic fibrosis. It is a wonderful message of hope to show that people not necessarily affected by the disease, but also sick people, come to run together for this cause.”
The general practitioner also applauds the latest therapeutic advances. “Thanks to treatment today, cystic fibrosis patients can resume physical activity, it’s magnificent, it’s a return to an almost normal life, so it’s a very beautiful message of hope.”
Find the interview with Marine Ltemporel in images:
@pourquoidoctor Miss France in 2013 and now a general practitioner, #marinelorphan is godmother of the association @Vaincre la Mucoviscidose was present this Monday at a #presstour at the Cystic Fibrosis Resource and Skills Center at Necker Hospital to provide an update on the care of children suffering from #cystic fibrosis in 2023 in the company of experts and families. #whydoctor ♬ original sound – whydoctor
