40% of patients with cystic fibrosis have returned to almost normal life thanks to a revolutionary triple therapy that has been on the market since 2020. your name? caftio. Today, “Vaincre la mucoviscidose” is committed to extending its prescription.
“It was the first time in forty years that a doctor had said to me: “I hear the air circulating”. » When David Fiant first swallows a pill of Kaftrio, a triple therapy for cystic fibrosis (article in link below), he notices an improvement in his health almost immediately. “After the treatment, I emptied my lungs for several hours. It was pretty spectacular.” explains the director of the association Vaincre la mucoviscidose.
Reimbursed by health insurance
He is among the first cohort of European patients aged 12 and over to benefit in 2020 from Kaftrio, a disease-modifying treatment marketed by a single laboratory in the world: Vertex Pharmaceuticals and reimbursed by health insurance. This drug acts on the disease’s genetic mutations, which decrease the number of “CFTR” proteins on the surface of cells, causing excess mucus and digestive fluids, with an increased risk of lung infections and digestive problems. The active ingredients in Kaftrio thus improve the activity of the defective CFTR protein and reduce the symptoms of cystic fibrosis. Whereas David had to before “Take three breaks to climb the stairs” due to shortness of breath, almost four of them can now climb the stairs. He also stopped intravenous antibiotic treatments, reduced physical therapy sessions, and even stopped using aerosols. “It changed my life” rejoices the forties. “I went from 13 to 103% breathing”‘ he says proudly.
What about transplant patients?
However, the news of this therapeutic breakthrough was not received equally by everyone. She leaves a ” bitter taste “ to Sabrina Perquis, who, unlike David, benefited from a lung transplant at the age of 26. Back then, transplantation was the only palliative offered in the treatment of cystic fibrosis, but today it prevents patients who have benefited from Kaftrio. “I often tell myself that if I had lasted a while before the surgery, I could have been one of those patients, too. And what about all the friends I buried who didn’t have time for a transplant? regret the young woman. The treatment would have saved him from taking anti-rejection drugs with proven side effects. “I developed a tumor in the thymus gland (a gland in the thorax, editor’s note) attributed to these aggressive drugs”, explains Sabine. She also mentions repeated pneumonia, despite lungs ” New “.
60% of patients are not suitable for Kaftrio
In reality, 60% of patients do not have access to Kaftrio. These include transplant recipients, but also people whose genetic mutation is incompatible with the drug, and finally very young patients under the age of six, for whom international studies are underway. “Our goal is that none of them are left behind and that patients can benefit from treatment as soon as possible to reduce the destruction of their lungs and avoid transplantation,” says Thierry Nouvel, CEO of Vaincre la mucoviscidose. Because the benefit of the treatment goes far beyond the disappearance of the symptoms David mentioned. “For example, it could have a positive effect on fertility by reducing mucus, which is associated with infertility in some women,” says Thierry Nouvel. It also helps improve the patient’s quality of life, saving time for rehabilitation and oxygen therapy, for some up to six hours a day. “In fact, many families tell us they worry about not hearing their loved ones cough because the life change is so radical,” says the board of directors of the association.
The question after
On the other hand, when improvement in daily life can come quickly, enrolling in a post-cystic fibrosis life project may take some time. “How can patients be supported who have had a failed educational and professional career because of their illness and who today see their future wide open? », asks David Fiant, who observes many cases of depression around him. Predicting the consequences is the whole goal of Vaincre la mucoviscidose. The association wants to devote part of its budget to training and orientation systems, in particular the “Pass souffle d’espoir” project, which offers professional integration or reintegration paths. To support these initiatives, she organizes the “Virades de l’espoir” on September 25, 2022. A national day of leisure and collection mobilizing 250 sites and more than 10,000 volunteers across France (link below). In 2019, the event brought in almost five million euros.
“All reproduction and display rights reserved. © Handicap.fr. This article was written by Clotilde Costil, journalist of Handicap.fr.”
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