Article
Medicine, General & Internal
Eva Steinke, Olaf Sommerburg, Simon Y. Graeber, Cornelia Joachim, Christiane Labitzke, Gyde Nissen, Isabell Ricklefs, Isa Rudolf, Matthias V. Kopp, Anna-Maria Dittrich, Marcus A. Mall, Mirjam Stahl
Summary: This study is a prospective, longitudinal cohort study aimed at determining influencing factors of early cystic fibrosis (CF) lung disease through the combined analysis of clinical information and biomaterials. The primary endpoints are the lung clearance index and magnetic resonance imaging scores, while secondary endpoints include pulmonary exacerbations, infection with pro-inflammatory pathogens, and anthropometric data.
FRONTIERS IN MEDICINE
(2023)
Review
Pharmacology & Pharmacy
Yizi Wang, Bin Ma, Wenya Li, Peiwen Li
Summary: Triple combination therapy for cystic fibrosis patients achieves better clinical results and comparable adverse events compared to the control group.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Respiratory System
Christopher H. Goss, Isabelle Fajac, Raksha Jain, Wolfgang Seibold, Abhya Gupta, Ming-Chi Hsu, Sivagurunathan Sutharsan, Jane C. Davies, Marcus A. Mall
Summary: BI 1265162, an ENaC inhibitor for the treatment of cystic fibrosis, did not demonstrate significant clinical benefits in a phase II study. Although there were some positive findings, such as an improvement in lung clearance index in some patients, overall, the drug showed limited clinical efficacy.
EUROPEAN RESPIRATORY JOURNAL
(2022)
Article
Microbiology
David C. Griffith, Elizabeth E. Morgan, Michael N. Dudley, Jeffery S. Loutit
Summary: The study showed that Biapenem exposure increased slightly more than dose proportionally after multiple doses, with no evidence of accumulation. Urinary recovery ranged from 14.2% at 250 mg to 42.3% at 1,250 mg. Biapenem was well tolerated up to 1,000 mg doses every 8 hours, but higher incidences of nausea, vomiting, and rash were reported at 1,250 mg.
ANTIMICROBIAL AGENTS AND CHEMOTHERAPY
(2021)
Article
Respiratory System
Christina Thornton, Ranjani Somayaji, Angel Chu, Michael D. Parkins
Summary: Human papillomavirus (HPV) is a major risk factor for cervical cancer, and transplant recipients have a higher risk of HPV complications. A retrospective study on adult female cystic fibrosis (CF) lung transplant recipients found that 35.3% had abnormal pap smear results, including refractory anogenital warts, vulvectomy, and cervical cancer. The study also revealed that lung transplant recipients had a higher likelihood of cervical dysplasia compared to the control group, emphasizing the importance of HPV vaccination to prevent future morbidity and mortality.
Article
Medical Laboratory Technology
Jean-Baptiste Woillard, Stephane Bouchet, Michael Fayon, Pierre Marquet, Caroline Monchaud, Stephanie Bui
Summary: This study aimed to develop amikacin dosing guidelines for children with cystic fibrosis and analyze the efficacy and toxicity at different MIC levels through simulations. Results showed that varying amikacin doses were effective for different bacterial MIC levels in improving treatment success rates and reducing toxicity.
THERAPEUTIC DRUG MONITORING
(2021)
Article
Microbiology
Antonin Praet, Laurent Bourguignon, Florence Vetele, Valentine Breant, Charlotte Genestet, Oana Dumitrescu, Anne Doleans-Jordheim, Philippe Reix, Sylvain Goutelle
Summary: This study developed nonparametric population pharmacokinetic (PK) models of tobramycin in children with CF for dosage design and model-guided therapeutic drug monitoring, with a recommended daily dose of 12.5 mg/kg optimizing efficacy and safety targets in most patients as validated through simulations.
ANTIMICROBIAL AGENTS AND CHEMOTHERAPY
(2021)
Article
Radiology, Nuclear Medicine & Medical Imaging
Elzbieta Jurkiewicz, Silvia Tsvetkova, Anna Grinberg, Blaise Pasquiers
Summary: The pharmacokinetic profile of gadopiclenol in children aged 2 to 17 years was similar to that observed in adults. Age-based dose adaptation is not indicated, and comparable plasma concentrations can be achieved with body weight-based dosing in this population. Gadopiclenol at 0.05 mmol/kg has a good safety profile in these patients and can improve lesion detection and visualization, thus enhancing diagnostic confidence.
INVESTIGATIVE RADIOLOGY
(2022)
Article
Oncology
Yoichi Naito, Yasutoshi Kuboki, Masafumi Ikeda, Kenichi Harano, Nobuaki Matsubara, Shigeyuki Toyoizumi, Yuko Mori, Natsuki Hori, Takashi Nagasawa, Takahiro Kogawa
Summary: The study investigated the safety and preliminary antitumor activity of talazoparib in Japanese patients with advanced solid tumors, showing that single-agent talazoparib was well tolerated and had promising antitumor effects. The recommended phase 2 dose of talazoparib was established as 1 mg once daily, with a disease control rate of 44.4%.
INVESTIGATIONAL NEW DRUGS
(2021)
Article
Psychology, Multidisciplinary
Deborah Friedman, Maysa M. Kaskas, Alexandra L. Quittner, Beth A. Smith, Anna M. Georgiopoulos
Summary: Individuals with cystic fibrosis (CF) are at high risk for depression and anxiety. This study developed a CBT-based intervention tailored to CF-specific needs, with positive feedback from patients.
FRONTIERS IN PSYCHOLOGY
(2022)
Article
Respiratory System
James F. Chmiel, Patrick Flume, Damian G. Downey, Allen J. Dozor, Carla Colombo, Henryk Mazurek, Ewa Sapiejka, Marta Rachel, Scott Constantine, Brian Conley, Nancy Dgetluck, Quinn Dinh, Barbara White, J. Stuart Elborn
Summary: The study assessed the safety and tolerability of lenabasum in treating chronic airway inflammation in CF patients. Results indicate that lenabasum has potential anti-inflammatory effects and can reduce the incidence of PEx.
JOURNAL OF CYSTIC FIBROSIS
(2021)
Article
Pediatrics
Taylor A. Imburgia, Samantha R. Engdahl, Rebecca S. Pettit
Summary: This study compares the rates of adverse drug events (ADEs) between prolonged and standard infusions of cefepime in pediatric cystic fibrosis (CF) patients. The results show that there is no significant difference in the composite safety endpoint between the two infusion methods. The clinical outcomes are also similar between the two groups.
PEDIATRIC PULMONOLOGY
(2022)
Article
Respiratory System
J. Stuart Elborn, Michael W. Konstan, Jennifer L. Taylor-Cousar, Isabelle Fajac, Alexander Horsley, Sivagurunathan Sutharsan, Shawn D. Aaron, Cori L. Daines, Ahmet Uluer, Damian G. Downey, Vincenzina V. Lucidi, Sanjeev Ahuja, Eric Springman, John Mershon, Ralph Grosswald, Steven M. Rowe
Summary: The study found that Acebilustat did not improve lung function, but there was a trend towards reduced pulmonary exacerbations in patients with early-stage lung disease.
JOURNAL OF CYSTIC FIBROSIS
(2021)
Article
Medicine, Research & Experimental
Min Zheng, Xiang Chen, Feng Wang, Jisu Chen, Kimberley Jackson, Fan Yang, Christopher Payne, HongYing Li, YiHui Wang, ZiPei Xiao, Jie Zheng
Summary: We evaluated the pharmacokinetics, safety, and efficacy of ixekizumab in Chinese patients with moderate-to-severe psoriasis. The results showed that after 12 weeks of treatment with IXE Q2W or IXE Q4W, clinically relevant treatment responses and acceptable safety profile were observed.
ADVANCES IN THERAPY
(2023)
Article
Health Care Sciences & Services
Tomasz Hildebrandt, Elzbieta Swietochowska, Agata Trzcionka, Anna Zawilska, Henryk Mazurek, Dagmara Maczkowiak, Mansur Rahnama, Marta Tanasiewicz
Summary: This study assessed the oral hygiene status of adult cystic fibrosis patients and found that their oral hygiene was satisfactory. However, professional preventive treatments were needed.
