REPORTING. “It’s unexpected”, a revolutionary treatment against cystic fibrosis accessible to young children

Kaftrio has just been authorized for children aged 2 to 5 years suffering from this rare and incurable genetic disease.

Otto, barely 2 years old, has suffered from cystic fibrosis since he was born; the disease affected his digestive system when he was still in his mother’s womb. Caroline did not expect to be able to benefit from the innovative treatment that is Kaftrio. “He was operated on at birth: intestinal obstruction in utero which required an operation, then there was a second and a thirdexplains Caroline. So, it’s true, when we knew a few weeks ago that it could be from two years, it’s unexpected”she rejoices.

5,000 older patients are already benefiting from Kaftrio, with spectacular results. This medicine, which arrived on the market two years ago, revolutionized the treatment of cystic fibrosis. This rare and incurable genetic disease causes abnormally thick mucus to be secreted, which obstructs the respiratory tract and digestive system, and affects more than 7 500 people.

Medication has been authorized since the beginning of November for children aged 2 to 5, under pressure from associations, notably Vaincre la Cystic Fibrosis. It was also a big issue for pediatrician Isabelle Sermet: “It is fundamental, because at 2 years the disease has not yet taken hold, if we can benefit from a medication which ultimately prevents the consequences of the disease from evolving, that is to say the mucus builds up, the mucus becomes infected and the lung becomes damaged.”

“Run less out of breath”

Isabelle Sermet follows sick children at the Cystic Fibrosis Resource and Skills Center at Necker hospital in Paris. For 9-year-old Thomas, the treatment changed everything: less bronchitis, fewer physiotherapy sessions and 10 to 15% more respiratory capacity. “I play football and I find that it has helped me breathe better, to keep running less out of breath”explains the young boy.

In this specialized center at Necker hospital, the respiratory capacities of young patients are regularly tested. Achraf, 16, has been taking Kaftrio for two years and the treatment continues to have an effect. He has a clip on his nose and he has to blow very hard into a tube under Anthony’s control: “That, Ashraf, when he did it before the treatment, it made him cough, he spit, he had phlegm and everything. Whereas here…” Achraf is indeed not out of breath.

These caregivers hope to be able to give this medication from the diagnosis, at birth, to prevent the disease from developing, avoid operations, lung transplant. For the moment, there is no cure for cystic fibrosis, but patients live longer. Thanks to these treatments, their life expectancy reaches 50 years.