The Vaincre la cystic fibrosis association published Monday, December 9, the 2023 results of the French Cystic Fibrosis Registry. This data was collected from the Cystic Fibrosis Resource and Care Centers (CRCM), multidisciplinary centers created in 2002 to coordinate patient care. They offer an overview of patient profiles, their state of health and their follow-up.
As a reminder, cystic fibrosis is a rare genetic disease that reduces respiratory function. It is characterized in particular by congestion of the bronchi, chronic cough, and frequent bacterial infections.
In total, in 2023, 7,680 patients, 64% of whom were adults, were followed in 2023 in the CRCMs. The average age of the patients followed is 25.7 years. The median age of death reaches 38.4 years. The mortality rate per 1,000 patients decreased from 6.3 in 2019 to 5.2 in 2023.
A majority of patients on triple therapy
Now, 66% of patients have access to Kaftrio triple therapy, which, since its launch on the market 5 years ago, has considerably improved the state of health, particularly respiratory, of patients who benefit from it. In patients, the mutation in the CFTR gene prevents the protein of the same name from functioning normally. Consequences: an accumulation of thick mucus in the lungs, pancreas and other organs. Kaftrio restores the function of the CFTR protein. Initially reserved for adults, a majority of children also had access to it during 2023, with use of Kaftrio increasing by 15 points in 2023 compared to 2022.
The benefits are there. The barometer points to a decreasing use of treatments over the period with fewer courses of antibiotics and respiratory therapies. Very concretely, the average forced expiratory volume per second (FEV1) has increased by 7 points in 5 years for children and by eight for adults, reaching 98.7% and 79.5% respectively in 2023. And the percentage of patients infected with Pseudomonas aeruginosa, the most common pathogen in the disease, increased from 37.6% in 2019 to 22.9% in 2023.
Kaftrio ineffective for rare genetic mutations
Same trend for transplants, with only 12 patients transplanted in 2023, including 8 lung transplants, or nearly 10% of the annual number of transplants performed before the arrival of triple therapy. The number of patients on the transplant list also continues to decline. Transplantation now represents the last resort for people with rare mutations for which Kaftrio does not work.
But although respiratory symptoms have clearly improved, comorbidities persist and even worsen. Osteoporosis and diabetes increased between 2019 and 2023, reaching 19.9% and 22.4%.
And the mental health of patients is deteriorating with a rate of depression and anxiety at 10.8%. How to explain it? According to an article on the site The Conversationwhich details the results of the Revivre study, based on the stories of patients receiving Kaftrio, triple therapy was able to destabilize the lives of certain patients in whom the immediate physical improvement led to an “upheaval”. “Identity is also called into question due to the clear reduction in care – medical, paramedical and including self-care – in the daily struggle to protect life. The end of this fight can give way to a feeling of emptiness,” note the authors of the article published on September 16.
70% of deaths are transplant patients
And for transplant patients, the figures remain bad. These represent only 11.6% of patients and yet concern 70% of deaths. Thus, since 2021, the number of deaths of transplant patients has been higher than the number of transplants. These patients transplanted in 2023 suffer from hypertension (41.4%), diabetes (69.9%) and osteoporosis (60.7%), because of the treatments necessary to prevent rejection of the graft. They are also more at risk of developing cancer (8.1%) and 27% of them suffer from depression and anxiety. Vaincre la Mucovoscidose intends to continue its investments in research to prevent graft rejection.
To note : Pancreatic function remains abnormal in 80% of patients taking kaftrio, which tends to demonstrate the limited impact of triple therapy on functions other than respiratory.
Source: Overcoming Cystic Fibrosis, 2023 report from the French Cystic Fibrosis Registry; The Conversation
