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. 2020 Sep;55(9):2302-2306.
doi: 10.1002/ppul.24854. Epub 2020 May 26.

Patients eligible for modulator drugs: Data from cystic fibrosis registry of Turkey

Nazan Çobanoğlu  1 , Uğur Özçelik  2 , Erkan Çakır  3 , Tuğba Şişmanlar Eyüboğlu  4 , Sevgi Pekcan  5 , Güzin Cinel  6 , Ebru Yalçın  2 , Nural Kiper  2 , Nagehan Emiralioğlu  2 , Velat Şen  7 , Hadice Selimoğlu Şen  8 , Ömür Ercan  5 , Haluk Çokuğraş  9 , Ayşe Ayzıt Kılınç  9 , Lina Muhammed Al Shadfan  3 , Hakan Yazan  3 , Derya Ufuk Altıntaş  10 , Dilek Karagöz  10 , Esen Demir  11 , Gökçen Kartal Öztürk  11 , Ayşen Bingöl  12 , Abdurrahman Erdem Başaran  12 , Nihat Sapan  13 , Şükrü Çekiç  13 , Ebru Çelebioğlu  14 , Ayşe Tana Aslan  4 , Tuğba Ramaslı Gürsoy  4 , Gökçen Tuğcu  6 , Ali Özdemir  15 , Koray Harmancı  16 , Gonca Kılıç Yıldırım  16 , Mehmet Köse  17 , Melih Hangül  17 , Zeynep Tamay  18 , Ayşe Süleyman  18 , Hasan Yüksel  19 , Özge Yılmaz  19 , Gizem Özcan  1 , Erdem Topal  20 , Demet Can  21 , Pervin Korkmaz Ekren  22 , Gönül Çaltepe  23 , Mehmet Kılıç  24 , Şebnem Özdoğan  25 , Deniz Doğru  2
Affiliations

Patients eligible for modulator drugs: Data from cystic fibrosis registry of Turkey

Nazan Çobanoğlu et al. Pediatr Pulmonol. 2020 Sep.

Abstract

Background: A better understanding of cystic fibrosis transmembrane conductance regulator biology has led to the development of modulator drugs such as ivacaftor, lumacaftor-ivacaftor, tezacaftor-ivacaftor, and elexacaftor-tezacaftor-ivacaftor. This cross-sectional study evaluated cystic fibrosis (CF) patients eligible for modulator drugs.

Methods: Data for age and genetic mutations from the Cystic Fibrosis Registry of Turkey collected in 2018 were used to find out the number of patients who are eligible for modulator therapy.

Results: Of registered 1488 CF patients, genetic analysis was done for 1351. The numbers and percentages of patients and names of the drugs, that the patients are eligible for, are as follows: 122 (9.03%) for ivacaftor, 156 (11.54%) for lumacaftor-ivacaftor, 163 (11.23%) for tezacaftor-ivacaftor, and 57 (4.21%) for elexacaftor-tezacaftor-ivacaftor. Among 1351 genotyped patients total of 313 (23.16%) patients are eligible for currently licensed modulator therapies (55 patients were shared by ivacaftor and tezacaftor-ivacaftor, 108 patients were shared by lumacaftor-ivacaftor and tezacaftor-ivacaftor, and 22 patients were shared by tezacaftor-ivacaftor and elexacaftor-tezacaftor-ivacaftor groups).

Conclusions: The present study shows that approximately one-fourth of the registered CF patients in Turkey are eligible for modulator drugs. As, frequent mutations that CF patients have in Turkey are different from North American and European CF patients, developing modulator drugs effective for those mutations is necessary. Furthermore, as modulator drugs are very expensive currently, financial support of the government in developing countries like Turkey is noteworthy.

Keywords: cystic fibrosis; modulator drugs; national registry.

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