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Personalised CFTR Pharmacotherapeutic Response Testing and Therapy of Cystic Fibrosis


Extract

Cystic fibrosis (CF) is a fatal, multisystem, genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride transporter critical to luminal fluid homeostasis at respiratory airway and other ductal epithelial surfaces. Therapeutic options for some CF patients were transformed by the approval of ivacaftor and lumacaftor/ivacaftor, which are indicated, respectively, for patients heterozygous for a subset of rare class III mutations [1] or homozygous for the common class II mutation Phe508del.[2] Notwithstanding, more than 40% of CF patients do not meet the CFTR mutation-specific inclusion criteria and are excluded from these therapies, and while it is impractical to conduct a clinical trial for each new pharmacotherapy-responsive CFTR mutation identified, an evolution of the drug approval process now permits drug label expansion based on in vitro physiological response testing. Indeed, the development of novel patient cell-based methods such as cultured intestinal organoids [3, 4] or nasal epithelial spheroids [5] permit in vitro testing of targeted CFTR molecular therapies. Here, we report the use of a novel preclinical test in one such patient to demonstrate patient-specific functional correction of CFTR ex vivo followed by initiation of therapy and functional correction in vivo. Further, while adverse respiratory effects have been reported with lumacaftor/ivacaftor therapy,[6] a comprehensive description of the treatment adjustment period has not been previously reported, here we provide a detailed account of this phenomenon.

Conflict of interest: Dr. McCarthy has nothing to disclose.

Conflict of interest: Dr. Brewington has nothing to disclose.

Conflict of interest: Ms. Harkness has nothing to disclose.

Conflict of interest: Dr. Clancy has nothing to disclose.

Conflict of interest: Dr. Trapnell has nothing to disclose.

sumber : https://erj.ersjournals.com/content/early/2018/03/15/13993003.02457-2017.abstract

 
 

PRAKTEK
RS Panti Waluya / RKZ Sawahan, Lt II-B1
, Dr Koentjahja, SpP
Nusakambangan 56, Malang 65117
08113777488 / 362017 ext. 88.23
Pukul 18.00 - 20.00, kec. Sabtu, Minggu / Libur


Rumah
, Dr Koentjahja, SpP
Wilis Indah A-6, Malang 65115
0818568711 / 0341-568711
Senin - Sabtu Pukul 09.00-10.00,
kec. Minggu / Libur


    


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