1. 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
- Author
-
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, and Deepika Polineni
- Subjects
Pulmonary and Respiratory Medicine - Abstract
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 hypertonic saline or dornase alfa in individuals using the CFTR modulator elexacaftor plus tezacaftor plus ivacaftor (ETI).The SIMPLIFY study included two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials at 80 participating clinics across the USA in the Cystic Fibrosis Therapeutics Development Network. We included individuals with cystic fibrosis aged 12-17 years with percent predicted FEVFrom Aug 25, 2020, to May 25, 2022, a total of 672 unique participants were screened for eligibility for one or both trials, resulting in 847 total random assignments across both trials with 594 unique participants. 370 participants were randomly assigned in the hypertonic saline trial and 477 in the dornase alfa trial. Participants across both trials had an average ppFEVIn individuals with cystic fibrosis on ETI with relatively well preserved pulmonary function, discontinuing daily hypertonic saline or dornase alfa for 6 weeks did not result in clinically meaningful differences in pulmonary function when compared with continuing treatment.
- Published
- 2023
- Full Text
- View/download PDF