Therapeutic drug monitoring-guided dosing for pediatric cystic fibrosis patients: recent advances and future outlooks

References

• Cystic Fibrosis Trust. UK cystic fibrosis registry 2020 annual data report 2021. available from: 2020 annual data report - version 4.Pdf (cysticfibrosis.Org.uk).
   Google Scholar
• Poulter C, Doull I, Bhatt JM. Epidemiology,genetics,pathophysiology and prognosis of CF. In: Eber E, Midulla F, editors. ERS Handbook of Paediatric Respiratory Medicine. UK: European Respiratory Society; 2021. p. 435–445. https://books.ersjournals.com/content/ers-handbook-of-paediatric-respiratory-medicine
   Google Scholar
• Waters V, Stanojevic S, Atenafu EG, et al. Effect of pulmonary exacerbations on long-term lung function decline in cystic fibrosis. Eur Respir J. 2012;40(1):61–66. doi: 10.1183/09031936.00159111
   PubMed Web of Science ®Google Scholar
• Nixon GM, Armstrong DS, Carzino R, et al. Clinical outcome after early Pseudomonas aeruginosa infection in cystic fibrosis. J Pediatr. 2001;138(5):699–704. doi: 10.1067/mpd.2001.112897
   PubMed Web of Science ®Google Scholar
• Emerson J, Rosenfeld M, McNamara S, et al. Pseudomonas aeruginosa and other predictors of mortality and morbidity in young children with cystic fibrosis. Pediatr Pulmonol. 2002;34(2):91–100. doi: 10.1002/ppul.10127
   PubMed Web of Science ®Google Scholar
• Davies JC. Pseudomonas aeruginosa in cystic fibrosis: pathogenesis and persistence. Paediatr Respir Rev. 2002;3(2):128–134. doi: 10.1016/s1526-0550(02)00003-3
   PubMedGoogle Scholar
• Bhatt JM. Treatment of pulmonary exacerbations in cystic fibrosis. Eur Respir Rev. 2013;22(129):205–216. doi: 10.1183/09059180.00006512
   PubMedGoogle Scholar
• Duckers J, Lesher B, Thorat T, et al. Real-world outcomes of ivacaftor treatment in people with cystic fibrosis: a systematic review. J Clin Med. 2021;10(7). doi: 10.3390/jcm10071527
   PubMed Web of Science ®Google Scholar
• King JA, Nichols AL, Bentley S, et al. An update on CFTR modulators as new therapies for cystic fibrosis. Paediatr Drugs. 2022;24(4):321–333. doi: 10.1007/s40272-022-00509-y
   PubMed Web of Science ®Google Scholar
• Joseph PD, Craig JC, Caldwell PH. Clinical trials in children. Br J Clin Pharmacol. 2015;79(3):357–369. doi: 10.1111/bcp.12305
   PubMed Web of Science ®Google Scholar
• Stockmann C, Barrett JS, Roberts JK, et al. Use of modeling and simulation in the design and conduct of pediatric clinical trials and the optimization of individualized dosing regimens. CPT Pharmacometrics Syst Pharmacol. 2015;4(11):630–640. Epub 20151113. doi: 10.1002/psp4.12038
   PubMedGoogle Scholar
• Smits A, Annaert P, Cavallaro G, et al. Current knowledge, challenges and innovations in developmental pharmacology: a combined conect4children expert group and european society for developmental Perinatal And Paediatric Pharmacology White Paper. Br J Clin Pharmacol. 2022;88(12):4965–4984. doi: 10.1111/bcp.14958
   PubMed Web of Science ®Google Scholar
• De Sutter PJ, Gasthuys E, Van Braeckel E, et al. Pharmacokinetics in patients with cystic fibrosis: a systematic review of data published between 1999 and 2019. Clin Pharmacokinet. 2020;59(12):1551–1573. doi: 10.1007/s40262-020-00932-9
   PubMed Web of Science ®Google Scholar
• De Sutter PJ, Van Haeverbeke M, Van Braeckel E, et al. Altered intravenous drug disposition in people living with cystic fibrosis: A meta-analysis integrating top-down and bottom-up data. CPT Pharmacometrics Syst Pharmacol. 2022;11(8):951–966. doi: 10.1002/psp4.12832
   PubMedGoogle Scholar
• Choong E, Sauty A, Koutsokera A, et al. Therapeutic drug monitoring of ivacaftor, lumacaftor, tezacaftor, and elexacaftor in cystic fibrosis: where are we now? Pharmaceutics. 2022;14(8):1674. doi: 10.3390/pharmaceutics14081674
   PubMed Web of Science ®Google Scholar
• Sanz-Codina M, Bozkir H, Jorda A, et al. Individualized antimicrobial dose optimization: a systematic review and meta-analysis of randomized controlled trials. Clin Microbiol Infect. 2023;29(7):845–857. doi: 10.1016/j.cmi.2023.03.018
   PubMed Web of Science ®Google Scholar
• Rybak MJ, Le J, Lodise TP, et al. Therapeutic monitoring of vancomycin for serious methicillin-resistant Staphylococcus aureus infections: a revised consensus guideline and review by the American society of health-system pharmacists, the infectious diseases society of america, the pediatric infectious diseases society, and the society of infectious diseases pharmacists. Am J Health Syst Pharm. 2020;77(11):835–864. doi: 10.1093/ajhp/zxaa036
   PubMed Web of Science ®Google Scholar
• Blix HS, Viktil KK, Moger TA, et al. Drugs with narrow therapeutic index as indicators in the risk management of hospitalised patients. Pharm Pract (Granada). 2010;8(1):50–55. doi: 10.4321/s1886-36552010000100006
   PubMedGoogle Scholar
• Roberts JA, Norris R, Paterson DL, et al. Therapeutic drug monitoring of antimicrobials. Br J Clin Pharmacol. 2012;73(1):27–36. doi: 10.1111/j.1365-2125.2011.04080.x
   PubMed Web of Science ®Google Scholar
• Gilbert DN, Saag MS, Pavia AT, et al. The sanford guide to antimicrobial therapy. VA USA: Sperryville; 2022.
   Google Scholar
• Mingeot-Leclercq MP, Glupczynski Y, Tulkens PM. Aminoglycosides: activity and resistance. Antimicrob Agents Chemother. 1999;43(4):727–737. doi: 10.1128/aac.43.4.727
   PubMed Web of Science ®Google Scholar
• Ochs MA, Dillman NO, Caverly LJ, et al. Aminoglycoside dosing and monitoring for Pseudomonas aeruginosa during acute pulmonary exacerbations in cystic fibrosis. Pediatr Pulmonol. 2021;56(12):3634–3643. doi: 10.1002/ppul.25441
   PubMed Web of Science ®Google Scholar
• Praet A, Bourguignon L, Vetele F, et al. Population pharmacokinetic modeling and dosing simulations of tobramycin in pediatric patients with cystic fibrosis. Antimicrob Agents Chemother. 2021;65(10):e0073721. doi: 10.1128/aac.00737-21
   PubMed Web of Science ®Google Scholar
• Dong M, Rodriguez AV, Blankenship CA, et al. Pharmacokinetic modelling to predict risk of ototoxicity with intravenous tobramycin treatment in cystic fibrosis. J Antimicrob Chemother. 2021;76(11):2923–2931. doi: 10.1093/jac/dkab288
   PubMed Web of Science ®Google Scholar
• UK Cystic Fibrosis Trust Antibiotic Working Group. Antibiotic Treatment for Cystic Fibrosis Guidelines 2009. https://www.cysticfibrosis.org.uk/sites/default/files/2020-11/Anitbiotic%20Treatment.pdf.
   Google Scholar
• Flume PA, Mogayzel PJ Jr., Robinson KA, et al. Cystic fibrosis pulmonary guidelines: treatment of pulmonary exacerbations. Am J Respir Crit Care Med. 2009;180(9):802–808. doi: 10.1164/rccm.200812-1845PP
   PubMed Web of Science ®Google Scholar
• Smyth A, Tan KH, Hyman-Taylor P, et al. Once versus three-times daily regimens of tobramycin treatment for pulmonary exacerbations of cystic fibrosis–the TOPIC study: a randomised controlled trial. Lancet. 2005;365(9459):573–578. doi: 10.1016/s0140-6736(05)17906-9
   PubMed Web of Science ®Google Scholar
• Larcombe R, Coulthard K, Eaton V, et al. Is there a multinational consensus of tobramycin prescribing and monitoring for cystic fibrosis? Survey of current therapeutic drug monitoring practices in USA/Canada, UK/Ireland, and Australia/New Zealand. Eur J Hosp Pharm. 2022; doi:10.1136/ejhpharm-2022-003545
   PubMedGoogle Scholar
• Mouton JW, Jacobs N, Tiddens H, et al. Pharmacodynamics of tobramycin in patients with cystic fibrosis. Diagn Microbiol Infect Dis. 2005;52(2):123–127. doi: 10.1016/j.diagmicrobio.2005.02.011
   PubMed Web of Science ®Google Scholar
• Burgard M, Sandaradura I, van Hal SJ, et al. Evaluation of tobramycin exposure predictions in three bayesian forecasting programmes compared with current clinical practice in children and adults with cystic fibrosis. Clin Pharmacokinet. 2018;57(8):1017–1027. doi: 10.1007/s40262-017-0610-9
   PubMed Web of Science ®Google Scholar
• Imani S, Fitzgerald DA, Robinson PD, et al. Personalized tobramycin dosing in children with cystic fibrosis: a comparative clinical evaluation of log-linear and Bayesian methods. J Antimicrob Chemother. 2022;77(12):3358–3366. doi: 10.1093/jac/dkac324
   PubMed Web of Science ®Google Scholar
• Ambrose PG, Bhavnani SM, Rubino CM, et al. Antimicrobial resistance: pharmacokinetics-pharmacodynamics of antimicrobial therapy: it’s not just for mice anymore. Clin Infect Dis. 2007;44(1):79–86. doi: 10.1086/510079
   PubMed Web of Science ®Google Scholar
• Craig WA. Interrelationship between pharmacokinetics and pharmacodynamics in determining dosage regimens for broad-spectrum cephalosporins. Diagn Microbiol Infect Dis. 1995;22(1–2):89–96. doi: 10.1016/0732-8893(95)00053-d
   PubMed Web of Science ®Google Scholar
• Imburgia TA, Kussin ML. A review of extended and continuous infusion beta-lactams in pediatric patients. J Pediatr Pharmacol Ther. 2022;27(3):214–227. doi: 10.5863/1551-6776-27.3.214
   PubMedGoogle Scholar
• Abdul-Aziz MH, Alffenaar JC, Bassetti M, et al. Antimicrobial therapeutic drug monitoring in critically ill adult patients: a position paper(). Intensive care Med. 2020;46(6):1127–1153. doi: 10.1007/s00134-020-06050-1
   PubMed Web of Science ®Google Scholar
• Bui S, Facchin A, Ha P, et al. Population pharmacokinetics of ceftazidime in critically ill children: impact of cystic fibrosis. J Antimicrob Chemother. 2020;75(8):2232–2239. doi: 10.1093/jac/dkaa170
   PubMed Web of Science ®Google Scholar
• Pettit RS, Neu N, Cies JJ, et al. Population pharmacokinetics of meropenem administered as a prolonged infusion in children with cystic fibrosis. J Antimicrob Chemother. 2016;71(1):189–195. doi: 10.1093/jac/dkv289
   PubMed Web of Science ®Google Scholar
• Du X, Li C, Kuti JL, et al. Population pharmacokinetics and pharmacodynamics of meropenem in pediatric patients. J Clin Pharmacol. 2006;46(1):69–75. doi: 10.1177/0091270005283283
   PubMed Web of Science ®Google Scholar
• Hong LT, Liou TG, Deka R, et al. Pharmacokinetics of continuous infusion beta-lactams in the treatment of acute pulmonary exacerbations in adult patients with cystic fibrosis. Chest. 2018;154(5):1108–1114. doi: 10.1016/j.chest.2018.06.002
   PubMed Web of Science ®Google Scholar
• Kuti JL, Pettit RS, Neu N, et al. Meropenem time above the MIC exposure is predictive of response in cystic fibrosis children with acute pulmonary exacerbations. Diagn Microbiol Infect Dis. 2018;91(3):294–297. doi: 10.1016/j.diagmicrobio.2018.01.020
   PubMed Web of Science ®Google Scholar
• Hubert D, Le Roux E, Lavrut T, et al. Continuous versus intermittent infusions of ceftazidime for treating exacerbation of cystic fibrosis. Antimicrob Agents Chemother. 2009;53(9):3650–3656. doi: 10.1128/aac.00174-09
   PubMed Web of Science ®Google Scholar
• Katz JB, Shah P, Trillo CA, et al. Therapeutic drug monitoring in cystic fibrosis and associations with pulmonary exacerbations and lung function. Respir med. 2023;212:107237. doi:10.1016/j.rmed.2023.107237
   PubMed Web of Science ®Google Scholar
• Cowart MC, Ferguson CL. Optimization of aztreonam in combination with ceftazidime/avibactam in a cystic fibrosis patient with chronic stenotrophomonas maltophilia pneumonia using therapeutic drug monitoring: a case study. Ther Drug Monit. 2021;43(2):146–149. doi: 10.1097/ftd.0000000000000857
   PubMed Web of Science ®Google Scholar
• Saffioti C, Barco S, Cangemi G, et al. Ceftazidime plasma concentration in a patient with cystic fibrosis treated with ceftazidime/avibactam plus trimethoprim/sulfametoxazole for Bulkholderia cepacia reacutization. J Chemother. 2019;31(7–8):436–438. doi: 10.1080/1120009x.2019.1671654
   PubMed Web of Science ®Google Scholar
• Liebchen U, Paal M, Jung J, et al. Therapeutic drug monitoring-guided high dose meropenem therapy of a multidrug resistant Acinetobacter baumannii - a case report. Respir Med Case Rep. 2020;29:100966. doi:10.1016/j.rmcr.2019.100966
   PubMed Web of Science ®Google Scholar
• Mortensen JS, Jensen BP, Zhang M, et al. Preanalytical stability of piperacillin, tazobactam, meropenem, and ceftazidime in plasma and whole blood using liquid chromatography-tandem mass spectrometry. Ther Drug Monit. 2019;41(4):538–543. doi: 10.1097/ftd.0000000000000650
   PubMed Web of Science ®Google Scholar
• Kipper K, Barker CIS, Standing JF, et al. Development of a novel multipenicillin assay and assessment of the impact of analyte degradation: lessons for scavenged sampling in antimicrobial pharmacokinetic study design. Antimicrob Agents Chemother. 2018;62(1). doi: 10.1128/aac.01540-17
   Web of Science ®Google Scholar
• McDade EJ, Hewlett JL, Moonnumakal SP, et al. Evaluation of vancomycin dosing in pediatric cystic fibrosis patients. J Pediatr Pharmacol Ther. 2016;21(2):155–161. doi: 10.5863/1551-6776-21.2.155
   PubMedGoogle Scholar
• Rybak M, Lomaestro B, Rotschafer JC, et al. Therapeutic monitoring of vancomycin in adult patients: a consensus review of the American society of health-system pharmacists, the infectious diseases society of america, and the society of infectious diseases pharmacists. Am J Health Syst Pharm. 2009;66(1):82–98. doi: 10.2146/ajhp080434
   PubMedGoogle Scholar
• Tsutsuura M, Moriyama H, Kojima N, et al. The monitoring of vancomycin: a systematic review and meta-analyses of area under the concentration-time curve-guided dosing and trough-guided dosing. BMC Infect Dis. 2021;21(1):153. doi: 10.1186/s12879-021-05858-6
   PubMed Web of Science ®Google Scholar
• Fusco NM, Prescott WA Jr., Meaney CJ. Pharmacokinetic monitoring of vancomycin in cystic fibrosis: is it time to move past trough concentrations? Pediatr Infect Dis J. 2019;38(3):258–262. doi: 10.1097/inf.0000000000002088
   PubMed Web of Science ®Google Scholar
• Stockmann C, Olson J, Rashid J, et al. An evaluation of vancomycin area under the curve estimation methods for children treated for acute pulmonary exacerbations of cystic fibrosis due to methicillin-resistant Staphylococcus aureus. J Clin Pharmacol. 2019;59(2):198–205. doi: 10.1002/jcph.1323
   PubMed Web of Science ®Google Scholar
• Mitchell B, Kormelink L, Kuhn R, et al. Retrospective review of vancomycin monitoring via trough only versus two-point estimated area under the curve in pediatric and adult patients with cystic fibrosis. Pediatr Pulmonol. 2023;58(1):239–245. doi: 10.1002/ppul.26190
   PubMed Web of Science ®Google Scholar
• Prentice AG, Glasmacher A. Making sense of itraconazole pharmacokinetics. J Antimicrob Chemother. 2005;56(Suppl 1):i17–i22. doi: 10.1093/jac/dki220
   PubMedGoogle Scholar
• Conway SP, Etherington C, Peckham DG, et al. Pharmacokinetics and safety of itraconazole in patients with cystic fibrosis. J Antimicrob Chemother. 2004;53(5):841–847. doi: 10.1093/jac/dkh175
   PubMed Web of Science ®Google Scholar
• Bentley S, Gupta A, Balfour-Lynn IM. Subtherapeutic itraconazole and voriconazole levels in children with cystic fibrosis. J Cyst Fibros. 2013;12(4):418–419. doi: 10.1016/j.jcf.2012.09.007
   PubMed Web of Science ®Google Scholar
• Sermet-Gaudelus I, Lesne-Hulin A, Lenoir G, et al. Sputum itraconazole concentrations in cystic fibrosis patients. Antimicrob Agents Chemother. 2001;45(6):1937–1938. doi: 10.1128/aac.45.6.1937-1938.2001
   PubMed Web of Science ®Google Scholar
• Hennig S, Wainwright CE, Bell SC, et al. Population pharmacokinetics of itraconazole and its active metabolite hydroxy-itraconazole in paediatric cystic fibrosis and bone marrow transplant patients. Clin Pharmacokinet. 2006;45(11):1099–1114. doi: 10.2165/00003088-200645110-00004
   PubMed Web of Science ®Google Scholar
• Aaron SD, Vandemheen KL, Freitag A, et al. Treatment of Aspergillus fumigatus in patients with cystic fibrosis: a randomized, placebo-controlled pilot study. Plos One. 2012;7(4):e36077. doi: 10.1371/journal.pone.0036077
   PubMed Web of Science ®Google Scholar
• Gothe F, Schmautz A, Häusler K, et al. Treating allergic bronchopulmonary aspergillosis with short-term prednisone and itraconazole in cystic fibrosis. J Allergy Clin Immunol Pract. 2020;8(8):2608–14.e3. doi: 10.1016/j.jaip.2020.02.031
   PubMed Web of Science ®Google Scholar
• Willems L, van der Geest R, de Beule K. Itraconazole oral solution and intravenous formulations: a review of pharmacokinetics and pharmacodynamics. J Clin Pharm Ther. 2001;26(3):159–169. doi: 10.1046/j.1365-2710.2001.00338.x
   PubMed Web of Science ®Google Scholar
• Ashbee HR, Barnes RA, Johnson EM, et al. Therapeutic drug monitoring (TDM) of antifungal agents: guidelines from the british society for medical mycology. J Antimicrob Chemother. 2014;69(5):1162–1176. doi: 10.1093/jac/dkt508
   PubMed Web of Science ®Google Scholar
• Warris A, Lehrnbecher T, Roilides E, et al. ESCMID-ECMM guideline: diagnosis and management of invasive aspergillosis in neonates and children. Clin Microbiol Infect. 2019;25(9):1096–1113. doi: 10.1016/j.cmi.2019.05.019
   PubMed Web of Science ®Google Scholar
• Gómez-López A. Antifungal therapeutic drug monitoring: focus on drugs without a clear recommendation. Clin Microbiol Infect. 2020;26(11):1481–1487. doi: 10.1016/j.cmi.2020.05.037
   PubMed Web of Science ®Google Scholar
• Hilliard T, Edwards S, Buchdahl R, et al. Voriconazole therapy in children with cystic fibrosis. J Cyst Fibros. 2005;4(4):215–220. doi: 10.1016/j.jcf.2005.05.019
   PubMedGoogle Scholar
• Ikeda Y, Umemura K, Kondo K, et al. Pharmacokinetics of voriconazole and cytochrome P450 2C19 genetic status. Clin Pharmacol Ther. 2004;75(6):587–588. doi: 10.1016/j.clpt.2004.02.002
   PubMed Web of Science ®Google Scholar
• Medicines and Healthcare Products Regulatory Agency. Voriconazole: reminder of risk of liver toxicity, phototoxicity, and squamous cell carcinoma. [2023 February 23rd]. Available from: https://www.gov.uk/drug-safety-update/voriconazole-reminder-of-risk-of-liver-toxicity-phototoxicity-and-squamous-cell-carcinoma
   Google Scholar
• Luong ML, Al-Dabbagh M, Groll AH, et al. Utility of voriconazole therapeutic drug monitoring: a meta-analysis. J Antimicrob Chemother. 2016;71(7):1786–1799. doi: 10.1093/jac/dkw099
   PubMed Web of Science ®Google Scholar
• Markantonis SL, Katelari A, Pappa E, et al. Voriconazole pharmacokinetics and photosensitivity in children with cystic fibrosis. J Cyst Fibros. 2012;11(3):246–252. doi: 10.1016/j.jcf.2011.12.006
   PubMed Web of Science ®Google Scholar
• Bentley S, Davies JC, Gastine S, et al. Clinical pharmacokinetics and dose recommendations for posaconazole gastroresistant tablets in children with cystic fibrosis. J Antimicrob Chemother. 2021;76(12):3247–3254. doi: 10.1093/jac/dkab312
   PubMed Web of Science ®Google Scholar
• Summary of Product Characteristics (SmPC). Noxafil 100mg gastro-resistant tablets. merck sharp & dohme (UK) limited. Updated: 6th Sep 2022. [Online].: [2023 Feb 22nd]. Available from: https://www.medicines.org.uk/emc/product/5388/smpc
   Google Scholar
• Patel D, Popple S, Claydon A, et al. Posaconazole therapy in children with cystic fibrosis and Aspergillus-related lung disease. Med Mycol. 2020;58(1):11–21. doi: 10.1093/mmy/myz015
   PubMed Web of Science ®Google Scholar
• Shearin S, Bell T. Treatment of Aspergillus fumigatus infection with posaconazole delayed-release tablets. Am J Health Syst Pharm. 2018;75(13):958–961. doi: 10.2146/ajhp170534
   PubMed Web of Science ®Google Scholar
• Yakut N, Kadayifci EK, Eralp EE, et al. Successful treatment of allergic bronchopulmonary aspergillosis with posaconazole in a child with cystic fibrosis: case report and review of the literature. Lung India. 2020;37(2):161–163. doi: 10.4103/lungindia.lungindia_288_19
   PubMed Web of Science ®Google Scholar
• Bentley S, Davies JC, Carr SB, et al. Combination antifungal therapy for scedosporium species in cystic fibrosis. Pediatr Pulmonol. 2020;55(8):1993–1995. doi: 10.1002/ppul.24789
   PubMed Web of Science ®Google Scholar
• Agarwal N, Apperley L, Taylor NF, et al. Posaconazole-induced hypertension masquerading as congenital adrenal hyperplasia in a child with cystic fibrosis. Case Rep Med. 2020;2020:1–5. doi:10.1155/2020/8153012
   Web of Science ®Google Scholar
• Mills R, Rautemaa-Richardson R, Wilkinson S, et al. Impact of airway Exophiala spp. on children with cystic fibrosis. J Cyst Fibros. 2021;20(4):702–707. doi: 10.1016/j.jcf.2021.03.012
   PubMed Web of Science ®Google Scholar
• Boonsathorn S, Cheng I, Kloprogge F, et al. Clinical pharmacokinetics and dose recommendations for posaconazole in infants and children. Clin Pharmacokinet. 2019;58(1):53–61. doi: 10.1007/s40262-018-0658-1
   PubMed Web of Science ®Google Scholar
• Walsh TJ, Raad I, Patterson TF, et al. Treatment of invasive aspergillosis with posaconazole in patients who are refractory to or intolerant of conventional therapy: an externally controlled trial. Clin Infect Dis. 2007;44(1):2–12. doi: 10.1086/508774
   PubMed Web of Science ®Google Scholar
• Konstan MW, Byard PJ, Hoppel CL, et al. Effect of high-dose ibuprofen in patients with cystic fibrosis. N Engl J Med. 1995;332(13):848–854. doi: 10.1056/nejm199503303321303
   PubMed Web of Science ®Google Scholar
• Lands LC, Milner R, Cantin AM, et al. High-dose ibuprofen in cystic fibrosis: Canadian safety and effectiveness trial. J Pediatr. 2007;151(3):249–254. doi: 10.1016/j.jpeds.2007.04.009
   PubMed Web of Science ®Google Scholar
• Lands LC, Stanojevic S. Oral non-steroidal anti-inflammatory drug therapy for lung disease in cystic fibrosis. Cochrane Database Syst Rev. 2019;9(9):Cd001505. Epub 20190909. doi: 10.1002/14651858.CD001505.pub5
   PubMedGoogle Scholar
• Konstan MW, VanDevanter DR, Sawicki GS, et al. Association of high-dose ibuprofen use, lung function decline, and long-term survival in children with cystic fibrosis. Ann Am Thorac Soc. 2018;15(4):485–493. doi: 10.1513/AnnalsATS.201706-486OC
   PubMed Web of Science ®Google Scholar
• Lands LC, Dauletbaev N. High-dose ibuprofen in cystic fibrosis. Pharmaceuticals (Basel). 2010;3(7):2213–2224. doi: 10.3390/ph3072213
   PubMedGoogle Scholar
• Mogayzel PJ Jr., Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187(7):680–689. doi: 10.1164/rccm.201207-1160oe
   PubMed Web of Science ®Google Scholar
• Konstan MW, Krenicky JE, Finney MR, et al. Effect of ibuprofen on neutrophil migration in vivo in cystic fibrosis and healthy subjects. J Pharmacol Exp Ther. 2003;306(3):1086–1091. doi: 10.1124/jpet.103.052449
   PubMed Web of Science ®Google Scholar
• Arranz I, Martín-Suárez A, Lanao JM, et al. Population pharmacokinetics of high dose ibuprofen in cystic fibrosis. Arch Dis Child. 2003;88(12):1128–1130. doi: 10.1136/adc.88.12.1128
   PubMed Web of Science ®Google Scholar
• Han EE, Beringer PM, Louie SG, et al. Pharmacokinetics of Ibuprofen in children with cystic fibrosis. Clin Pharmacokinet. 2004;43(3):145–156. doi: 10.2165/00003088-200443030-00001
   PubMed Web of Science ®Google Scholar
• Bruch BA, Singh SB, Ramsey LJ, et al. Impact of a cystic fibrosis transmembrane conductance regulator (CFTR) modulator on high-dose ibuprofen therapy in pediatric cystic fibrosis patients. Pediatr Pulmonol. 2018;53(8):1035–1039. doi: 10.1002/ppul.24024
   PubMed Web of Science ®Google Scholar
• Konstan MW. Ibuprofen therapy for cystic fibrosis lung disease: revisited. Curr Opin Pulm Med. 2008;14(6):567–573. doi: 10.1097/MCP.0b013e32831311e8
   PubMed Web of Science ®Google Scholar
• Lopes-Pacheco M. CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine. Front Pharmacol. 2019;10:1662. doi: 10.3389/fphar.2019.01662
   PubMed Web of Science ®Google Scholar
• Trittler R, Hug M. PKP-017 monitoring of ivacaftor serum levels. Eur J Hosp Pharm Sci Pract. 2014;21(Suppl 1):A143.2–A144. doi: 10.1136/ejhpharm-2013-000436.352
   Google Scholar
• Masson A, Schneider-Futschik EK, Baatallah N, et al. Predictive factors for lumacaftor/ivacaftor clinical response. J Cyst Fibros. 2019;18(3):368–374. doi: 10.1016/j.jcf.2018.12.011
   PubMed Web of Science ®Google Scholar
• Hanafin PO, Sermet-Gaudelus I, Griese M, et al. Insights into patient variability during ivacaftor-lumacaftor therapy in cystic fibrosis. Front Pharmacol. 2021;12:577263. doi:10.3389/fphar.2021.577263
   PubMed Web of Science ®Google Scholar
• van der Meer R, Wilms EB, Sturm R, et al. Pharmacokinetic interactions between ivacaftor and cytochrome P450 3A4 inhibitors in people with cystic fibrosis and healthy controls. J Cyst Fibros. 2021;20(5):e72–e6. doi: 10.1016/j.jcf.2021.04.005
   PubMed Web of Science ®Google Scholar
• Summary of Product Characteristics (SmPC). Kaftrio 75 mg 50 mg 100 mg film-coated tablets. vertex pharmaceuticals (Europe) Ltd. Updated: 15th Dec 2022. [Online]. Available from: https://www.medicines.org.uk/emc/product/11724/smpc [Accessed 22nd Feb 2023].
   Google Scholar
• Summary of Product Characteristics (SmPC). Kalydeco 150mg film-coated tablets. vertex pharmaceuticals (Europe) Ltd. Updated: 16th Nov 2022. [Online]. Available from: https://www.medicines.org.uk/emc/product/3040/smpc [Accessed 22nd Feb 2023].
   Google Scholar
• Vonk SEM, van der Meer-Vos M, Bos LDJ, et al. Quantitative method for the analysis of ivacaftor, hydroxymethyl ivacaftor, ivacaftor carboxylate, lumacaftor, and tezacaftor in plasma and sputum using liquid chromatography with tandem mass spectrometry and its clinical applicability. Ther Drug Monit. 2021;43(4):555–563. doi: 10.1097/ftd.0000000000000829
   PubMed Web of Science ®Google Scholar
• Baroud E, Chaudhary N, Georgiopoulos AM. Management of neuropsychiatric symptoms in adults treated with elexacaftor/tezacaftor/ivacaftor. Pediatr Pulmonol. 2023;58(7):1920–1930. doi: 10.1002/ppul.26412
   PubMed Web of Science ®Google Scholar
• Spoletini G, Gillgrass L, Pollard K, et al. Dose adjustments of Elexacaftor/Tezacaftor/Ivacaftor in response to mental health side effects in adults with cystic fibrosis. J Cyst Fibros. 2022;21(6):1061–1065. doi: 10.1016/j.jcf.2022.05.001
   PubMed Web of Science ®Google Scholar
• Heo S, Young DC, Safirstein J, et al. Mental status changes during elexacaftor/tezacaftor/ivacaftor therapy. J Cyst Fibros. 2022;21(2):339–343. doi: 10.1016/j.jcf.2021.10.002
   PubMed Web of Science ®Google Scholar
• Zhang L, Albon D, Jones M, et al. Impact of elexacaftor/tezacaftor/ivacaftor on depression and anxiety in cystic fibrosis. Ther Adv Respir Dis. 2022;16:17534666221144211. doi: 10.1177/17534666221144211
   Web of Science ®Google Scholar
• Sergeev V, Chou FY, Lam GY, et al. The extrapulmonary effects of cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis. Ann Am Thorac Soc. 2020;17(2):147–154. doi: 10.1513/AnnalsATS.201909-671CME
   PubMed Web of Science ®Google Scholar
• Drummond D, Dana J, Berteloot L, et al. Lumacaftor-ivacaftor effects on cystic fibrosis-related liver involvement in adolescents with homozygous F508 del-CFTR. J Cyst Fibros. 2022;21(2):212–219. doi: 10.1016/j.jcf.2021.07.018
   PubMed Web of Science ®Google Scholar
• Valamparampil JJ, Gupte GL. Cystic fibrosis associated liver disease in children. World J Hepatol. 2021;13(11):1727–1742. doi: 10.4254/wjh.v13.i11.1727
   PubMed Web of Science ®Google Scholar
• Jain R, Kazmerski TM, Zuckerwise LC, et al. Pregnancy in cystic fibrosis: review of the literature and expert recommendations. J Cyst Fibros. 2022;21(3):387–395. doi: 10.1016/j.jcf.2021.07.019
   PubMed Web of Science ®Google Scholar
• Nash EF, Middleton PG, Taylor-Cousar JL. Outcomes of pregnancy in women with cystic fibrosis (CF) taking CFTR modulators - an international survey. J Cyst Fibros. 2020;19(4):521–526. doi: 10.1016/j.jcf.2020.02.018
   PubMed Web of Science ®Google Scholar
• Jain R, Magaret A, Vu PT, et al. Prospectively evaluating maternal and fetal outcomes in the era of CFTR modulators: the MAYFLOWERS observational clinical trial study design. BMJ Open Respir Res. 2022;9(1):e001289. doi: 10.1136/bmjresp-2022-001289
   PubMed Web of Science ®Google Scholar
• Schneider EK, Reyes-Ortega F, Li J, et al. Optimized LC-MS/MS method for the high-throughput analysis of clinical samples of ivacaftor, its major metabolites, and lumacaftor in biological fluids of cystic fibrosis patients. J Vis Exp. 2017128: doi:10.3791/56084-v
   PubMedGoogle Scholar
• Reyes-Ortega F, Qiu F, Schneider-Futschik EK. Multiple reaction monitoring mass spectrometry for the drug monitoring of ivacaftor, tezacaftor, and elexacaftor treatment response in cystic fibrosis: a high-throughput method. ACS Pharmacol Transl Sci. 2020;3(5):987–996. doi: 10.1021/acsptsci.0c00103
   PubMedGoogle Scholar
• Habler K, Kalla AS, Rychlik M, et al. Isotope dilution LC-MS/MS quantification of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, lumacaftor, tezacaftor, elexacaftor, and their major metabolites in human serum. Clin Chem Lab Med. 2022;60(1):82–91. doi: 10.1515/cclm-2021-0724
   PubMed Web of Science ®Google Scholar
• European Commission. State of paediatric medicines in the eu. 10 years of the eu paediatric regulation. report from the commission to the european parliament and the council. COM p. 626. 2017. [Online]. Available from: https://health.ec.europa.eu/system/files/2017-11/2017_childrensmedicines_report_en_0.pdf [ Accessed 23rd February 2023].
   Google Scholar
• Abdolrasouli A, Scourfield A, Rhodes J, et al. High prevalence of triazole resistance in clinical Aspergillus fumigatus isolates in a specialist cardiothoracic centre. Int J Antimicrob Agents. 2018;52(5):637–642. doi: 10.1016/j.ijantimicag.2018.08.004
   PubMed Web of Science ®Google Scholar
• Zhang Y, Wang T, Zhang D, et al. Therapeutic drug monitoring coupled with bayesian forecasting could prevent vancomycin-associated nephrotoxicity in renal insufficiency patients: a prospective study and pharmacoeconomic analysis. Ther Drug Monit. 2020;42(4):600–609. doi: 10.1097/ftd.0000000000000750
   PubMed Web of Science ®Google Scholar
• Stocco G, Lucafò M, Decorti G. Pharmacogenomics of Antibiotics. Int J Mol Sci. 2020;21(17). doi: 10.3390/ijms21175975
   Web of Science ®Google Scholar
• Ayers S, Muller I, Mahoney L, et al. Understanding needle-related distress in children with cystic fibrosis. Br J Health Psychol. 2011;16(Pt 2):329–343. doi: 10.1348/135910710x506895
   PubMedGoogle Scholar
• Spencer H, Kozlowska W, Davies JC, et al. Measurement of tobramycin and gentamicin in saliva is not suitable for therapeutic drug monitoring of patients with cystic fibrosis. J Cyst Fibros. 2005;4(3):209. doi: 10.1016/j.jcf.2005.05.016
   PubMedGoogle Scholar
• Vanstraelen K, Maertens J, Augustijns P, et al. Investigation of saliva as an alternative to plasma monitoring of voriconazole. Clin Pharmacokinet. 2015;54(11):1151–1160. doi: 10.1007/s40262-015-0269-z
   PubMed Web of Science ®Google Scholar
• Kim HY, Märtson AG, Dreesen E, et al. Saliva for precision dosing of antifungal drugs: saliva population pk model for voriconazole based on a systematic review. Front Pharmacol. 2020;11:894. doi:10.3389/fphar.2020.00894
   PubMed Web of Science ®Google Scholar
• Singh KD, Osswald M, Ziesenitz VC, et al. Personalised therapeutic management of epileptic patients guided by pathway-driven breath metabolomics. Commun Med (Lond). 2021;1(1):21. doi: 10.1038/s43856-021-00021-3
   PubMedGoogle Scholar