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Weight increase in people with cystic fibrosis on CFTR modulator therapy is mainly due to increase in fat mass.
Mouzaki M, Dupuis A, Avolio J, Griffin K, Ratjen F, Tullis E, Gonska T. Mouzaki M, et al. Among authors: ratjen f. Front Pharmacol. 2023 Jul 13;14:1157459. doi: 10.3389/fphar.2023.1157459. eCollection 2023. Front Pharmacol. 2023. PMID: 37521467 Free PMC article.
Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. The mechanism as well as the long-term impact of ivacaftor on weight, resting energy expenditure (REE) and body composition remains to …
Background: Ivacaftor, the first CFTR modulator drug, leads to significant long-term improvement in lung function and weight gain. Th …
Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial.
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ahluwalia N, Chin A, Chu C, Lu M, Menon P, Waltz D, Weinstock T, Zelazoski L, Davies JC. Wainwright C, et al. Among authors: ratjen f. Am J Respir Crit Care Med. 2023 Jul 1;208(1):68-78. doi: 10.1164/rccm.202301-0021OC. Am J Respir Crit Care Med. 2023. PMID: 37154609
Rationale: A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children aged 6-11 years with cystic fibrosis (CF) and one or more F508del-CFTR alleles. Objectives: To assess long-term safety and efficacy of …
Rationale: A 24-week, phase 3, open-label study showed elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was safe and efficacious in children a …
Impact of antibiotic eradication therapy of Pseudomonas aeruginosa on long term lung function in cystic fibrosis.
Casaredi IG, Shaw M, Waters V, Seeto R, Blanchard AC, Ratjen F. Casaredi IG, et al. Among authors: ratjen f. J Cyst Fibros. 2023 Jan;22(1):98-102. doi: 10.1016/j.jcf.2022.08.007. Epub 2022 Aug 20. J Cyst Fibros. 2023. PMID: 35995678
INTRODUCTION: While antibiotic eradication therapy (AET) of early Pseudomonas aeruginosa infection is considered standard of care, its long-term effect on the subsequent course of cystic fibrosis (CF) lung disease remains unclear. ...
INTRODUCTION: While antibiotic eradication therapy (AET) of early Pseudomonas aeruginosa infection is considered standard of care, its long- …
Aquagenic wrinkling of the palms in cystic fibrosis patients treated with ivacaftor.
Jacobi E, Solomon M, Avolio J, Shaw M, Gonska T, Ratjen F, Grasemann H. Jacobi E, et al. Among authors: ratjen f. J Cyst Fibros. 2022 Mar;21(2):e102-e105. doi: 10.1016/j.jcf.2022.01.005. Epub 2022 Jan 19. J Cyst Fibros. 2022. PMID: 35063397 Free article.
Aquagenic wrinkling of palms (AWP) in cystic fibrosis (CF) patients and common CFTR mutations is recognized as a frequent symptom of the disease. The long-term effect of CFTR targeting therapy on AWP has not been studied. ...
Aquagenic wrinkling of palms (AWP) in cystic fibrosis (CF) patients and common CFTR mutations is recognized as a frequent symptom of the dis …
Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study.
Hoppe JE, Chilvers M, Ratjen F, McNamara JJ, Owen CA, Tian S, Zahigian R, Cornell AG, McColley SA. Hoppe JE, et al. Among authors: ratjen f. Lancet Respir Med. 2021 Sep;9(9):977-988. doi: 10.1016/S2213-2600(21)00069-2. Epub 2021 May 6. Lancet Respir Med. 2021. PMID: 33965000
BACKGROUND: A previous phase 3 study showed that lumacaftor-ivacaftor was generally safe and well tolerated over 24 weeks of treatment in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation. In this study, we aimed to assess the long-term …
BACKGROUND: A previous phase 3 study showed that lumacaftor-ivacaftor was generally safe and well tolerated over 24 weeks of treatment in ch …
A multimodal approach to detect and monitor early lung disease in cystic fibrosis.
Mondéjar-López P, Horsley A, Ratjen F, Bertolo S, de Vicente H, Asensio de la Cruz Ò. Mondéjar-López P, et al. Among authors: ratjen f. Expert Rev Respir Med. 2021 Jun;15(6):761-772. doi: 10.1080/17476348.2021.1908131. Epub 2021 Apr 12. Expert Rev Respir Med. 2021. PMID: 33843417
MRI is a radiation-free imaging alternative that provides both morphological and functional information. The role of MRI for short-term follow-up and pulmonary exacerbations is currently being investigated.Expert opinion: The roles of LCI and MRI are expected to expand con …
MRI is a radiation-free imaging alternative that provides both morphological and functional information. The role of MRI for short-term …
PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy.
Nichols DP, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Sagel SD, Rosenfeld M, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Kirby S, VanDalfsen JM, Clancy JP, Rowe SM. Nichols DP, et al. Among authors: ratjen f. J Cyst Fibros. 2021 Mar;20(2):205-212. doi: 10.1016/j.jcf.2021.02.003. Epub 2021 Feb 19. J Cyst Fibros. 2021. PMID: 33619012 Free PMC article. Review.
Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on long-term outcomes, patient care needs, and future research priorities. ...
Multiple observational research studies are now being conducted to better understand the impacts of this important therapeutic milestone on …
Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study.
Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Chilvers MA, et al. Among authors: ratjen f. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28. Lancet Respir Med. 2021. PMID: 33516285 Clinical Trial.
BACKGROUND: The safety and efficacy of 24 weeks of lumacaftor-ivacaftor combination therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation was previously shown in two phase 3 studies. Here, we report long-term safety and efficacy …
BACKGROUND: The safety and efficacy of 24 weeks of lumacaftor-ivacaftor combination therapy in children aged 6-11 years with cystic fibrosis …
Evaluating the Impact of Stopping Chronic Therapies after Modulator Drug Therapy in Cystic Fibrosis: The SIMPLIFY Clinical Trial Study Design.
Mayer-Hamblett N, Nichols DP, Odem-Davis K, Riekert KA, Sawicki GS, Donaldson SH, Ratjen F, Konstan MW, Simon N, Rosenbluth DB, Retsch-Bogart G, Clancy JP, VanDalfsen JM, Buckingham R, Gifford AH. Mayer-Hamblett N, et al. Among authors: ratjen f. Ann Am Thorac Soc. 2021 Aug;18(8):1397-1405. doi: 10.1513/AnnalsATS.202010-1336SD. Ann Am Thorac Soc. 2021. PMID: 33465316 Free PMC article.
The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of discontinuing hypertonic saline or dornase alfa, enabling individuals on both therapies to participate in one or both trials. ...
The protocol is composed of two concurrent randomized controlled trials designed to evaluate the independent short-term effects of di …
Immunoreactive trypsinogen levels in newborn screened infants with an inconclusive diagnosis of cystic fibrosis.
Ooi CY, Sutherland R, Castellani C, Keenan K, Boland M, Reisman J, Bjornson C, Chilvers MA, van Wylick R, Kent S, Price A, Mateos-Corral D, Hughes D, Solomon M, Zuberbuhler P, Brusky J, Durie PR, Ratjen F, Gonska T. Ooi CY, et al. Among authors: ratjen f. BMC Pediatr. 2019 Oct 22;19(1):369. doi: 10.1186/s12887-019-1756-4. BMC Pediatr. 2019. PMID: 31640630 Free PMC article.
CF transmembrane conductance regulator (CFTR)-related metabolic syndrome (CRMS) or CF screen positive inconclusive diagnosis (CFSPID). These infants have an uncertain long-term outcome and it is currently unclear around time of diagnosis, which infants are at higher risk o …
CF transmembrane conductance regulator (CFTR)-related metabolic syndrome (CRMS) or CF screen positive inconclusive diagnosis (CFSPID). These …
38 results