The useful results of triple remedy for cystic fibrosis

The Vaincre la Mucoviscidose affiliation publishes at present the 2021 report of the French Cystic Fibrosis Registry. This assortment of epidemiological knowledge, up to date annually, makes it doable to evaluate the state of well being and the follow-up of sufferers with cystic fibrosis. The useful results of triple remedy for cystic fibrosis have simply been established. Rationalization.

One yr after the advertising and marketing of the brand new drug Kaftrio®, the 2021 report from the French Cystic Fibrosis Registry reveals the primary useful results of triple remedy on sick folks: enhance within the inhabitants and common age, drastic drop in recourse to transplantation, discount of remedy, higher fertility. However these developments mustn’t conceal a painful actuality that makes the illness plural. There are those that can entry triple remedy and others…

cystic fibrosis : enhance within the inhabitants and enhance within the common age

In 2021, the constructive evolution of the principle indicators studied within the French Cystic Fibrosis Registry continues.

We word, in France:

• a increase the variety of folks dwelling with cystic fibrosis (+1.8%) to succeed in 7,513 sufferers recognized whereas the variety of new instances recognized stays steady: 144 in 2021 in opposition to 157 in 2020;
• a rise within the proportion of adults (over 18) within the common inhabitants dwelling with cystic fibrosis: 61.7% in 2021 in comparison with 58.5% in 2019 or 48.5% 10 years in the past.
• a rise within the common age of sufferers : 25 years outdated in comparison with 19 years outdated 10 years in the past;
• a rise within the median age at dying which grows yr after yr : 37.8 years.

Kaftrio® : encouraging first outcomes

This remedy had been the topic of a brief authorization to be used (ATU) which had been obtained on the finish of 2019 and allowed 450 sufferers to be placed on Kaftrio® earlier than acquiring the Advertising and marketing Authorization ( MA) in July 2021.

Prescribed since July 2021 to 2,200 folks (i.e. 30% of sufferers with cystic fibrosis), Kaftrio® has already had constructive results on the standard of lifetime of sick folks. Amongst these useful results:

• a drastic lower within the variety of sufferers having recourse to a lung transplant : 17 in 2021 (in comparison with 86 in 2019);
• a big discount in processing:
  • a drop within the prescription of intravenous antibiotic programs: 19% of sufferers in 2021 (versus 26% in 2020 and 29% in 2019);
  • a discount within the prescription of digestive therapies and respiratory therapies equivalent to oxygen remedy, nasal air flow and even respiratory rehabilitation.

One other consequence most likely as a result of prescription of Kaftrio®:

• there’s a vital enhance within the variety of early pregnancies (+29%), thus assuming higher fertility in ladies. On the similar time, extra sufferers are taking contraception as a way to keep away from undesirable pregnancies.
• a fall in sufferers handled with Orkambi® (-59%) in favor of different therapies, primarily Kaftrio®.

Nonetheless a big majority of sufferers with out an efficient therapeutic answer

Sadly, nonetheless 60% of sufferers are excluded from new therapies referred to as “CFTR modulators”. These sufferers are ineligible as a consequence of genetic profiles on which these therapies are ineffective or transplant sufferers for whom these modulators are contraindicated. Overcoming Cystic Fibrosis goals, alongside researchers and caregivers, to multiply initiatives in order that ALL sufferers can entry efficient therapies.