Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials

Autor: Nicole Mayer-Hamblett, Felix Ratjen, Renee Russell, Scott H Donaldson, Kristin A Riekert, Gregory S Sawicki, Katherine Odem-Davis, Julia K Young, Daniel Rosenbluth, Jennifer L Taylor-Cousar, Christopher H Goss, George Retsch-Bogart, John Paul Clancy, Alan Genatossio, Brian P O'Sullivan, Ariel Berlinski, Susan L Millard, Gregory Omlor, Colby A Wyatt, Kathryn Moffett, David P Nichols, Alex H Gifford, Margaret Kloster, Katie Weaver, Claire Chapdu, Jing Xie, Michelle Skalland, Melita Romasco, Sonya Heltshe, Noah Simon, Jill VanDalfsen, Anna Mead, Rachael Buckingham, Kathy Seidel, Nikita Midamba, Laurel Couture, Brooke Zappone Case, Wendy Au, Elsie Rockers, Diane Cooke, Amber Olander, Irene Bondick, Miya Johnson, Lisya VanHousen, Boris Nicholson, Michelle Parrish, Dion Roberts, Jillian Head, Jessica Carey, Lindsay Caverly, Joy Dangerfield, Rachel Linnemann, Jason Fullmer, Chelsea Roman, Peter Mogayzel, Deanne Reyes, Amy Harmala, Jerimiah Lysinger, Jonathan Bergeron, Isabel Virella-Lowell, Perry Brown, Lejla Godusevic, Alicia Casey, Lauren Paquette, Thomas Lahiri, Julie Sweet, Scott Donaldson, Joshua Harris, Shelia Parnell, Sylvia Szentpetery, Deborah Froh, Erica Tharrington, Manu Jain, Rachel Nelson, Sharon Kadon, Gary McPhail, Kimberly McBennett, Tia Rone, Elliott Dasenbrook, Dave Weaver, Terri Johnson, Karen McCoy, Raksha Jain, Maria Mcleod, Mary Klosterman, Preeti Sharma, Amy Jones, Gary Mueller, Rachel Janney, Jennifer Taylor-Cousar, Mary Cross, Jordana Hoppe, James Cahill, Zubin Mukadam, Jill Finto, Karen Schultz, Silvia Delgado Villalta, Alexa Smith, Susan Millard, Thomas Symington, Gavin Graff, Diane Kitch, Don Sanders, Misty Thompson, Tahuanty Pena, Mary Teresi, Jennifer Gafford, David Schaeffer, Joel Mermis, Lawrence Scott, Hugo Escobar, Kristen Williams, Dana Dorman, Brian O'Sullivan, Ryan Bethay, Zoran Danov, Kat Turbeville, Jimmy Johannes, Angelica Rodriguez, Bridget Marra, Robert Zanni, Ronald Morton, Terri Simeon, Andrew Braun, Nicole Dondlinger, Julie Biller, Erin Hubertz, Nicholas Antos, Laura Roth, Joanne Billings, Catherine Larson, Priya Balaji, John McNamara, Tammy Clark, Rebecca Griffith, Nancy Martinez, Sabiha Hussain, Halina Malveaux, Marie Egan, Catalina Guzman, Joan DeCelie-Germana, Susan Galvin, Adrienne Savant, Nicole Falgout, Patricia Walker, Teresa Demarco, Emily DiMango, Maria Ycaza, Julie Ballo, Pornchai Tirakitsoontorn, Daniel Layish, Desiree Serr, Floyd Livingston, Sherry Wooldridge, Carlos Milla, Jacquelyn Spano, Rebecca Davis, Okan Elidemir, Subramanyam Chittivelu, Ashley Scott, Sarah Alam, Daniel Dorgan, Matt Butoryak, Daniel Weiner, Harmony Renna, Colby Wyatt, Brendan Klein, Anne Stone, Meg Lessard, Michael S. Schechter, Barbara Johnson, Steven Scofield, Theodore Liou, Jane Vroom, Kathryn Akong, Marissa Gil, Legna Betancourt, Jonathan Singer, Ngoc Ly, Courtney Moreno, Moira Aitken, Teresa Gambol, Ronald Gibson, Allison Lambert, Joan Milton, Sarah Smith, Deanna Green, Diana Hodge, Christopher Fortner, Mary Forell, Rachel Karlnoski, Kapil Patel, Cori Daines, Elizabeth Ryan, Rodolfo Amaro-Galvez, Elizabeth Dohanich, Alison Lennox, Zachary Messer, Holly Hanes, Kay Powell, Deepika Polineni

Publikováno v: The Lancet Respiratory Medicine. 11:329-340

Reducing treatment burden is a priority for people with cystic fibrosis, whose health has benefited from using new modulators that substantially increase CFTR protein function. The SIMPLIFY study aimed to assess the effects of discontinuing nebulised

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a610bdf6f80f9f99b6e9a399c3e8c0aa
https://doi.org/10.1016/s2213-2600(22)00434-9

Age-related heterogeneity in dental caries and associated risk factors in individuals with cystic fibrosis ages 6–20 years: A pilot study

Autor: Carlos Milla, Lloyd Mancl, Alan Genatossio, Margaret Rosenfeld, Alaa A. Alkhateeb, JoAnna M. Scott, Sharon McNamara, Elise W. Sarvas, Isabel Virella-Lowell, Richard B. Presland, Donald L. Chi, Whasun O. Chung, Marilynn Rothen

Publikováno v: Journal of Cystic Fibrosis. 17:747-759

BACKGROUND. Literature conflicts regarding dental caries risk in cystic fibrosis (CF) relative to controls. METHODS. Prospective, observational study of age-related heterogeneity in caries rates and potential risk factors in CF patients ages 6–20 a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::54b148283ce2e1575bf28d0cd2d6d839
https://doi.org/10.1016/j.jcf.2018.06.009

Long-term safety of tobramycin inhalation powder in patients with cystic fibrosis: phase IV (ETOILES) study

Autor: Isabel Virella-Lowell, Gerhild Angyalosi, Andrea Viegas, Urte Sommerwerck, Weihua Cao, Laurie DeBonnett

Publikováno v: Current medical research and opinion. 32(11)

Objective: Long-term treatment with inhaled antibiotics is recommended for chronic Pseudomonas aeruginosa (Pa) infection in cystic fibrosis (CF) patients. The ETOILES study (Clinicaltrials.gov identifier: NCT01519661) evaluated the safety of tobramyc

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::987ac4d5bfcd9f27fcba3c1f1046c110
https://pubmed.ncbi.nlm.nih.gov/27435882

Defective Acid Sphingomyelinase Pathway with Pseudomonas aeruginosa Infection in Cystic Fibrosis

Autor: Hong Yu, Isabel Virella-Lowell, Russell W. Jenkins, Youssef H. Zeidan, Terence R. Flotte, Bill X. Wu, Yusuf A. Hannun

Publikováno v: American Journal of Respiratory Cell and Molecular Biology. 41:367-375

Acid sphingomyelinase (ASMase) is a key enzyme in sphingolipid metabolism, which can be activated by various cellular stress mechanisms including bacterial pathogens. Activation of ASMase generates ceramide, which is important for innate immune respo

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0f0ead0ee0b8645d7e72a256bfce3aad
https://doi.org/10.1165/rcmb.2008-0295oc

Quantitative sinonasal symptom assessment in an unselected pediatric population with cystic fibrosis

Autor: Isabel Virella-Lowell, Jennifer L. Wentzel, Rodney J. Schlosser, Zachary M. Soler

Publikováno v: American journal of rhinologyallergy. 29(5)

Introduction The aim of this study was to establish baseline sinonasal quality of life scores in an unselected pediatric population with cystic fibrosis (CF) and to test the correlation of those scores with various clinical outcome measurements. Meth

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f9916296acac996122d6783e8666c03b
https://pubmed.ncbi.nlm.nih.gov/26358346