Cystic fibrosis newborn screening implemented in Belgium in 2019

Patrick Lebecque (1), Olivier Lebecque (2), Marijke Proesmans (3), Teresinha Leal (4) Published in the journal : November 2019 Category : Pneumologie pédiatrique

In 2019, a national program of cystic fibrosis newborn screening is scheduled to be eventually implemented in Belgium. An IRT/DNA algorithm has been chosen, pretty similar to the French algorithm. In both countries, R117H is almost always combined in cis with a 7T sequence in the intron 8 acceptor splice site. It was included in the initial panel of CFTR variants looked for. This paper critically describes the program’s steps, while emphasizing the crucial roles of general practitioners and local pediatricians.

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O. Lebecque, V. Godding, P. Lebecque Published in the journal : February 2016 Category : Pneumologie pédiatrique

Your money or your life? - Kalydeco®: a milestone towards the “cure” of cystic fibrosis From February 1st 2016 on, Ivacaftor (KALYDECO™) will be reimbursed in Belgium for the treatment of cystic fibrosis (CF) in patients aged 6 years and older carrying at least one of nine well characterized gating (Class III) cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations, which account for approximately 3% of Belgian CF patients. In most of these patients, this very expensive oral drug (248,000 €/year) substantially improves lung function, lowers sweat chloride levels, and improves body weight by targeting the primary defect, thus representing a fundamental shift in the way the disease is managed. However, clinicians should be aware of the drug's potential for numerous drug interactions.

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