Kaftrio® triple therapy: “inequalities are widening among patients with cystic fibrosis”

The association Overcoming Cystic Fibrosis has just published the 2022 report from the French cystic fibrosis register.

“The results for 2022 confirm a clear improvement in the overall state of health of the entire population suffering from cystic fibrosis: increase in the population and average age, significant reduction in the use of transplants, reduction of treatments…”, the activists begin. “Nevertheless, this assessment also reveals an increase in disparities and considerable loss of opportunity within the community of patients between those who have access to triple therapy and those who do not.” they note.

Increase in the number of people living with cystic fibrosis

The report first notes an increase in the number of people living with cystic fibrosis (+3%) and an increase in the share of the number of adults in the entire population living with cystic fibrosis: 62.5% in 2022 compared to 61.7% in 2021 or even 48.5% 10 years ago.

An increase in the average age of patients has also been established: 25.2 years compared to 19 years 10 years ago. “For the first year, the median age at death exceeds 40 years (40.8 years), an increase of more than 10 years over a decade” can we also read in the survey.

50.7% of patients with cystic fibrosis benefit from the new Kaftrio® triple therapy

Regarding the new Kaftrio® protein triple therapy, 50.7% of patients with cystic fibrosis benefited from it in 2022.

As a result, a drastic reduction in the number of patients who had recourse to a lung transplant was noted (23 in 2022 compared to 31 in 2021 and more than a hundred before the arrival of Kaftrio®), as well as a clear improvement in respiratory health.

“The percentage of patients benefiting from triple therapy should evolve favorably due to the extension for 6/11 year olds which took place at the end of December 2022 and the effects of which will only be able to be fully measured in 2023. The extension to children from age of 2 years as part of early access should also contribute to increasing this percentage, as will the expansion of the compassionate prescribing framework which took place in June 2023. adds the patient association.

Evolution of the mental health of people with cystic fibrosis

On the digestive and hepatic levels, complications affect more patients than in 2021. “In the coming months, it is appropriate to continue efforts to improve patient care regarding these health issues,” estimate the authors of the survey.

Another worrying aspect lies in the evolution of the mental health of people with cystic fibrosis, with a slight increase in cases of depression: 9.5% of patients are now being monitored for this psychiatric pathology (compared to 8.8% in 2021). “It remains to be determined whether these trends are associated with side effects of the new CFTR modulator Kaftrio® or with paradoxical reactions in certain patients, who sometimes find themselves helpless in building their future,” they analyze.

Finally, one last worrying observation emerges from the assessment of the French cystic fibrosis registry: the state of health of transplant recipients shows little improvement or even deteriorates, with the potential occurrence of comorbidities such as diabetes, present in 69% of people. having undergone a transplant compared to 15.9% in non-transplanted patients, osteopenia/osteoporosis (55.3% vs 12.3%) and cancers (8% vs 0.5%). Nearly 3⁄4 of patients with cystic fibrosis who died in 2022 had been transplanted. “So many signs which demonstrate the importance of placing emphasis on the monitoring of transplant patients, particularly for better control of chronic dysfunction of the lung graft” concludes the association Vaincre la Cystic Fibrosis.