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1. Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor on the burden of illness in adolescents and adults with cystic fibrosis

Author

Thomas Keens, Veena Hoffman, Ia Topuria, Ken Elder, Shannon Cerf, Kyra Mulder, Jon Roberts, Jerimiah Lysinger, Maria Del Carmen Reyes, Maria Berdella, Anne Marie Cairns, Manu Jain, Vaidyanathan Ganapathy, Yiyue Lou, Bassem Morcos, Chuntao Wu, and Laura Sass

Subjects
Burden of disease, Cystic fibrosis, Elexacaftor, Ivacaftor, Real-world, Tezacaftor, Science (General), Q1-390, Social sciences (General), H1-99
Abstract

Background: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (CF) aged ≥2 years. Here, we describe results from an observational study assessing change in burden of illness following initiating ELX/TEZ/IVA in real-world settings. Methods: This US-based, multicenter, observational study used data from electronic medical records to evaluate real-world burden of illness before and after ELX/TEZ/IVA initiation in people with CF aged ≥12 years heterozygous for F508del and a minimal function mutation (F/MF) or an uncharacterized CFTR mutation. Endpoints included absolute change from baseline in percent predicted forced expiratory volume in 1 s (ppFEV1), body mass index (BMI) and BMI-for-age z-score, glycated hemoglobin (HbA1c), and numbers of pulmonary exacerbations (PEx). Results: Overall, 206 people with CF were enrolled (mean [SD] age 22.5 [11.1] years; 192 [93.2%] with F/MF genotype). Mean follow-up was 15.6 (SD, 1.6) months. Improvements in ppFEV1 (7.3 [95% CI: 5.7, 8.8] percentage points) were observed from baseline through follow-up. Increases in BMI (1.40 [95% CI: 1.07, 1.77] kg/m2) and BMI-for-age z-score (0.14 [95% CI: 0.00, 0.28]) were also observed from baseline at 12 months. The estimated annualized rate of any PEx was 1.31 at baseline and 0.61 over follow-up (rate ratio 0.47 [95% CI: 0.39, 0.55]), with annualized rates of PEx requiring antibiotics and hospitalizations of 0.55 and 0.88 in the baseline period and 0.12 and 0.36 over follow-up (rate ratios 0.22 [95% CI: 0.15, 0.31] and 0.41 [95% CI: 0.32, 0.51]), respectively. Absolute change in HbA1c was −0.22 (95% CI: -0.38, −0.06) from baseline through follow-up. Conclusions: ELX/TEZ/IVA treatment was associated with improved lung function, increased BMI, reduced frequency of PEx, and improved (i.e., reduced) HbA1c. These results confirm the broad clinical benefits of ELX/TEZ/IVA seen in clinical trials and show the potential for ELX/TEZ/IVA to improve markers of glucose metabolism.

Published
2024
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2. Airway inflammation accelerates pulmonary exacerbations in cystic fibrosis

Author

Theodore G. Liou, Natalia Argel, Fadi Asfour, Perry S. Brown, Barbara A. Chatfield, David R. Cox, Cori L. Daines, Dixie Durham, Jessica A. Francis, Barbara Glover, My Helms, Theresa Heynekamp, John R. Hoidal, Judy L. Jensen, Christiana Kartsonaki, Ruth Keogh, Carol M. Kopecky, Noah Lechtzin, Yanping Li, Jerimiah Lysinger, Osmara Molina, Craig Nakamura, Kristyn A. Packer, Robert Paine, III, Katie R. Poch, Alexandra L. Quittner, Peggy Radford, Abby J. Redway, Scott D. Sagel, Rhonda D. Szczesniak, Shawna Sprandel, Jennifer L. Taylor-Cousar, Jane B. Vroom, Ryan Yoshikawa, John P. Clancy, J. Stuart Elborn, Kenneth N. Olivier, and Frederick R. Adler

Subjects
Health sciences, Medicine, Medical specialty, respiratory medicine, Omics, Science
Abstract

Summary: Airway inflammation underlies cystic fibrosis (CF) pulmonary exacerbations. In a prospective multicenter study of randomly selected, clinically stable adolescents and adults, we assessed relationships between 24 inflammation-associated molecules and the future occurrence of CF pulmonary exacerbation using proportional hazards models. We explored relationships for potential confounding or mediation by clinical factors and assessed sensitivities to treatments including CF transmembrane regulator (CFTR) protein synthesis modulators. Results from 114 participants, including seven on ivacaftor or lumacaftor-ivacaftor, representative of the US CF population during the study period, identified 10 biomarkers associated with future exacerbations mediated by percent predicted forced expiratory volume in 1 s. The findings were not sensitive to anti-inflammatory, antibiotic, and CFTR modulator treatments. The analyses suggest that combination treatments addressing RAGE-axis inflammation, protease-mediated injury, and oxidative stress might prevent pulmonary exacerbations. Our work may apply to other airway inflammatory diseases such as bronchiectasis and the acute respiratory distress syndrome.

Published
2024
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3. Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis

Author

Theodore G. Liou, Frederick R. Adler, Natalia Argel, Fadi Asfour, Perry S. Brown, Barbara A. Chatfield, Cori L. Daines, Dixie Durham, Jessica A. Francis, Barbara Glover, Theresa Heynekamp, John R. Hoidal, Judy L. Jensen, Ruth Keogh, Carol M. Kopecky, Noah Lechtzin, Yanping Li, Jerimiah Lysinger, Osmara Molina, Craig Nakamura, Kristyn A. Packer, Katie R. Poch, Alexandra L. Quittner, Peggy Radford, Abby J. Redway, Scott D. Sagel, Shawna Sprandel, Jennifer L. Taylor-Cousar, Jane B. Vroom, Ryan Yoshikawa, John P. Clancy, J. Stuart Elborn, Kenneth N. Olivier, and David R. Cox

Subjects
Cystic fibrosis, Randomized observational trial, Study design, Sputum inflammation, HMGB-1, Neutrophil elastase, Medicine (General), R5-920
Abstract

Abstract Background Biomarkers of inflammation predictive of cystic fibrosis (CF) disease outcomes would increase the power of clinical trials and contribute to better personalization of clinical assessments. A representative patient cohort would improve searching for believable, generalizable, reproducible and accurate biomarkers. Methods We recruited patients from Mountain West CF Consortium (MWCFC) care centers for prospective observational study of sputum biomarkers of inflammation. After informed consent, centers enrolled randomly selected patients with CF who were clinically stable sputum producers, 12 years of age and older, without previous organ transplantation. Results From December 8, 2014 through January 16, 2016, we enrolled 114 patients (53 male) with CF with continuing data collection. Baseline characteristics included mean age 27 years (SD = 12), 80% predicted forced expiratory volume in 1 s (SD = 23%), 1.0 prior year pulmonary exacerbations (SD = 1.2), home elevation 328 m (SD = 112) above sea level. Compared with other patients in the US CF Foundation Patient Registry (CFFPR) in 2014, MWCFC patients had similar distribution of sex, age, lung function, weight and rates of exacerbations, diabetes, pancreatic insufficiency, CF-related arthropathy and airway infections including methicillin-sensitive or -resistant Staphylococcus aureus, Pseudomonas aeruginosa, Burkholderia cepacia complex, fungal and non-tuberculous Mycobacteria infections. They received CF-specific treatments at similar frequencies. Conclusions Randomly-selected, sputum-producing patients within the MWCFC represent sputum-producing patients in the CFFPR. They have similar characteristics, lung function and frequencies of pulmonary exacerbations, microbial infections and use of CF-specific treatments. These findings will plausibly make future interpretations of quantitative measurements of inflammatory biomarkers generalizable to sputum-producing patients in the CFFPR.

Published
2019
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4. 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

5. A Randomized Clinical Trial of Antimicrobial Duration for Cystic Fibrosis Pulmonary Exacerbation Treatment

Author

Christopher H. Goss, Sonya L. Heltshe, Natalie E. West, Michelle Skalland, Don B. Sanders, Raksha Jain, Tara L. Barto, Barbra Fogarty, Bruce C. Marshall, Donald R. VanDevanter, Patrick A. Flume, Gregory Omlor, Brenda Bourne, Dion Roberts, Vicki Roberts, Samya Nasr, Dawn Kruse, Rachel Linnemann, Tsion Hailemichael, Caralee Forseen, Heidi Stapp, Natalie West, S. Patel, A. Claudio, Jerimiah Lysinger, Amy Harmala, George Solomon, Latona Kersh, Karen Miller, Dixie Durham, Ahmet Uluer, Robert Fowler, Carla Frederick, Nadine Caci, Charlotte Teneback, Julie Sweet, Michael Parkins, Clare Smith, Jennifer Goralski, Kelsey Haywood, Patrick Flume, Caroline Brailsford, Dana Albon, Christie Aderholt, Kimberly McBennett, Cindy Schaefer, Alpa Patel, April Hunt, Lauren Schumacher, Hari Polenakovik, Linda Clark, Jerry Nick, Katie Poch, Dana Kissner, James Cahill, Jorge Lascano, Erin Silverman, John McArdle, Alison Champagne, Robert Vender, Lisa Allwein, Lance Cohen, Norma (Jean) Barton, Tara Barto, Ami Patel, Cynthia Brown, Nia Vorhees, Michael Crosser, Lawrence Scott, Alix Ashare, Barbara Rodgers, Robert Zanni, Lisa Koval, Andrew Braun, Sophia Chiron Stevens, Maria Tupayachi Ortiz, Patricia Graham, Julie Biller, Erin Hubertz, Kathryn Moffett, Tammy Clark, Rebecca Griffith, Nancy Martinez, Sabiha Hussain, Fei Chen, Marie Egan, Catalina Guzman, Janice Wang, Aileen Espinal, Patricia Walker, Anne Kukral, Emily DiMango, Sarah Fracasso Francis, Carlos Milla, Colleen Dunn, Subramanyam Chittivelu, Ashley Scott, Daniel Dorgan, Sharon Ng, Joseph Pilewski, Rose Lanzo, Nauman Chaudary, Ryan Hayden, Steven Scofield, Barb Johnson, Brian Morrissey, Brandt Robinson, Douglas Conrad, Jenna Mielke, Moira Aitken, Chami Sanlors, Ravi Nayak, Freda Branch, Daniel Rosenbluth, Molly Siegel, Anil Ghimire, Mary Forell, Cori Daines, Monica Varela, Leslie Couch, Rebekah Hibbard, Allen Dozor, Armando Ramirez, Victor Ortega, Kathryn Kennedy, David Fish, and Karen Longtine

Subjects
Adult, Male, Pulmonary and Respiratory Medicine, medicine.medical_specialty, Time Factors, Cystic Fibrosis, Exacerbation, Critical Care and Intensive Care Medicine, Cystic fibrosis, law.invention, Randomized controlled trial, law, Internal medicine, medicine, Humans, Pseudomonas Infections, Respiratory system, Respiratory Tract Infections, business.industry, Cystic Fibrosis Pulmonary Exacerbation, Editorials, Respiratory infection, Original Articles, Middle Aged, medicine.disease, Antimicrobial, Anti-Bacterial Agents, Respiratory Function Tests, Clinical trial, Pseudomonas aeruginosa, Disease Progression, Female, business
Abstract

RATIONALE: People with cystic fibrosis (CF) experience acute worsening of respiratory symptoms and lung function known as pulmonary exacerbations. Treatment with intravenous antimicrobials is common; however, there is scant evidence to support a standard treatment duration. OBJECTIVES: To test differing durations of intravenous antimicrobials for CF exacerbations. METHODS: STOP2 (Standardized Treatment of Pulmonary Exacerbations 2) was a multicenter, randomized, controlled clinical trial in exacerbations among adults with CF. After 7–10 days of treatment, participants exhibiting predefined lung function and symptom improvements were randomized to 10 or 14 days’ total antimicrobial duration; all others were randomized to 14 or 21 days’ duration. MEASUREMENTS AND MAIN RESULTS: The primary outcome was percent predicted FEV(1) (ppFEV(1)) change from treatment initiation to 2 weeks after cessation. Among early responders, noninferiority of 10 days to 14 days was tested; superiority of 21 days compared with 14 days was compared for the others. Symptoms, weight, and adverse events were secondary. Among 982 randomized people, 277 met improvement criteria and were randomized to 10 or 14 days of treatment; the remaining 705 received 21 or 14 days of treatment. Mean ppFEV(1) change was 12.8 and 13.4 for 10 and 14 days, respectively, a ‒0.65 difference (95% CI [‒3.3 to 2.0]), excluding the predefined noninferiority margin. The 21- and 14-day arms experienced 3.3 and 3.4 mean ppFEV(1) changes, a difference of ‒0.10 (‒1.3 to 1.1). Secondary endpoints and sensitivity analyses were supportive. CONCLUSIONS: Among adults with CF with early treatment improvement during exacerbation, ppFEV(1) after 10 days of intravenous antimicrobials is not inferior to 14 days. For those with less improvement after one week, 21 days is not superior to 14 days. Clinical trial registered with www.clinicaltrials.gov (NCT02781610).

Published
2021

6. Associations of Sputum Biomarkers with Clinical Outcomes in People with Cystic Fibrosis

Author

Theodore G Liou, Natalia Argel, Fadi Asfour, Perry S Brown, Barbara A Chatfield, David R Cox, Cori L Daines, Dixie Durham, Jessica A Francis, Barbara Glover, My Helms, Theresa Heynekamp, John R Hoidal, Judy L Jensen, Christiana Kartsonaki, Ruth Keogh, Carol M Kopecky, Noah Lechtzin, Yanping Li, Jerimiah Lysinger, Osmara Molina, Craig Nakamura, Kristyn A Packer, Robert Paine, Katie R Poch, Alexandra L Quittner, Peggy Radford, Abby J Redway, Scott D Sagel, Rhonda D Szczesniak, Shawna Sprandel, Jennifer L Taylor-Cousar, Jane B Vroom, Ryan Yoshikawa, John P Clancy, J Stuart Elborn, Kenneth N Olivier, and Frederick R Adler

Abstract

BackgroundAirway inflammation promotes bronchiectasis and lung injury in cystic fibrosis (CF). Amplification of inflammation underlies pulmonary exacerbations of disease. We asked whether sputum inflammatory biomarkers provide explanatory information on pulmonary exacerbations.Patients and MethodsWe collected sputum from randomly chosen stable adolescents and adults and prospectively observed time to next exacerbation, our primary outcome. We evaluated relationships between potential biomarkers of inflammation, clinical characteristics and outcomes and assessed clinical variables as potential confounders or mediators of explanatory models. We assessed associations between the markers and time to next exacerbation using proportional hazard models adjusting for confounders.ResultsWe enrolled 114 patients, collected data on clinical variables [December 8, 2014 to January 16, 2016; 46% male, mean age 28 years (SD 12), mean percent predicted forced expiratory volume in 1 s (FEV1%) 70 (SD 22)] and measured 24 inflammatory markers. Half of the inflammatory markers were plausibly associated with time to next exacerbation. Age and sex were confounders while we found that FEV1% was a mediator.Three potential biomarkers of RAGE axis inflammation were associated with time to next exacerbation while six potential neutrophil-associated biomarkers indicate associations between protease activity or reactive oxygen species with time to next exacerbation.ConclusionPulmonary exacerbation biomarkers are part of the RAGE proinflammatory axis or reflect neutrophil activity, specifically implicating protease and oxidative stress injury. Further investigations or development of novel anti-inflammatory agents should consider RAGE axis, protease and oxidant stress antagonists.Tweetable abstractSputum from 114 randomly chosen people with CF show RAGE axis inflammation, protease and oxidative stress injury are associated with time to next pulmonary exacerbation and may be targets for bench or factorial design interventional studies. (242 characters)

Published
2022

7. Additional file 6: of Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis

Author

Liou, Theodore, Adler, Frederick, Argel, Natalia, Asfour, Fadi, Brown, Perry, Chatfield, Barbara, Daines, Cori, Durham, Dixie, Francis, Jessica, Glover, Barbara, Heynekamp, Theresa, Hoidal, John, Jensen, Judy, Keogh, Ruth, Kopecky, Carol, Lechtzin, Noah, Yanping Li, Jerimiah Lysinger, Osmara Molina, Nakamura, Craig, Packer, Kristyn, Poch, Katie, Quittner, Alexandra, Radford, Peggy, Redway, Abby, Sagel, Scott, Sprandel, Shawna, Taylor-Cousar, Jennifer, Vroom, Jane, Yoshikawa, Ryan, Clancy, John, J. Elborn, Olivier, Kenneth, and Cox, David

Subjects
ComputingMethodologies_DOCUMENTANDTEXTPROCESSING, Hardware_CONTROLSTRUCTURESANDMICROPROGRAMMING
Abstract

Sputum Processing Instructions. Provides the laboratory flow sheet for sample collection and illustrated instructions. (PDF 706 kb)

Published
2019
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8. Additional file 5: of Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis

Author

Liou, Theodore, Adler, Frederick, Argel, Natalia, Asfour, Fadi, Brown, Perry, Chatfield, Barbara, Daines, Cori, Durham, Dixie, Francis, Jessica, Glover, Barbara, Heynekamp, Theresa, Hoidal, John, Jensen, Judy, Keogh, Ruth, Kopecky, Carol, Lechtzin, Noah, Yanping Li, Jerimiah Lysinger, Osmara Molina, Nakamura, Craig, Packer, Kristyn, Poch, Katie, Quittner, Alexandra, Radford, Peggy, Redway, Abby, Sagel, Scott, Sprandel, Shawna, Taylor-Cousar, Jennifer, Vroom, Jane, Yoshikawa, Ryan, Clancy, John, J. Elborn, Olivier, Kenneth, and Cox, David

Abstract

Final Study Protocol. Created and distributed 13 May 2013. (PDF 290 kb)

Published
2019
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9. Additional file 4: of Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis

Author

Liou, Theodore, Adler, Frederick, Argel, Natalia, Asfour, Fadi, Brown, Perry, Chatfield, Barbara, Daines, Cori, Durham, Dixie, Francis, Jessica, Glover, Barbara, Heynekamp, Theresa, Hoidal, John, Jensen, Judy, Keogh, Ruth, Kopecky, Carol, Lechtzin, Noah, Yanping Li, Jerimiah Lysinger, Osmara Molina, Nakamura, Craig, Packer, Kristyn, Poch, Katie, Quittner, Alexandra, Radford, Peggy, Redway, Abby, Sagel, Scott, Sprandel, Shawna, Taylor-Cousar, Jennifer, Vroom, Jane, Yoshikawa, Ryan, Clancy, John, J. Elborn, Olivier, Kenneth, and Cox, David

Abstract

Transcript of Video: Principles of Study Design: A Conversation Between David Cox and Ruth Keogh. (PDF 124 kb)

Published
2019
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10. Prospective multicenter randomized patient recruitment and sample collection to enable future measurements of sputum biomarkers of inflammation in an observational study of cystic fibrosis

Author

David Cox, Jessica A. Francis, Carol M. Kopecky, John P. Clancy, Osmara Molina, Barbara Glover, Frederick R. Adler, Dixie Durham, Kristyn A Packer, Ruth H. Keogh, Perry S. Brown, Peggy Radford, Theodore G. Liou, Abby J. Redway, Ryan Yoshikawa, Shawna Sprandel, Scott D. Sagel, Jerimiah Lysinger, Katie R. Poch, Jane B. Vroom, John R. Hoidal, Yanping Li, Alexandra L. Quittner, Cori L. Daines, Theresa Heynekamp, Natalia Argel, Noah Lechtzin, Kenneth N. Olivier, Craig Nakamura, Barbara A. Chatfield, Fadi Asfour, Judy L. Jensen, Jennifer L. Taylor-Cousar, and J. Stuart Elborn

Subjects
Adult, Male, Methicillin-Resistant Staphylococcus aureus, medicine.medical_specialty, Adolescent, Cystic Fibrosis, Epidemiology, Health Informatics, Cystic fibrosis, Neutrophil elastase, 03 medical and health sciences, Young Adult, 0302 clinical medicine, Internal medicine, Arthropathy, medicine, Humans, 030212 general & internal medicine, Prospective Studies, Lung, HMGB-1, lcsh:R5-920, business.industry, 030503 health policy & services, Patient Selection, Sputum, Study design, Middle Aged, Staphylococcal Infections, medicine.disease, Sputum inflammation, Clinical trial, Patient recruitment, Cohort, Randomized observational trial, Observational study, Female, Sample collection, medicine.symptom, lcsh:Medicine (General), 0305 other medical science, business
Abstract

Background Biomarkers of inflammation predictive of cystic fibrosis (CF) disease outcomes would increase the power of clinical trials and contribute to better personalization of clinical assessments. A representative patient cohort would improve searching for believable, generalizable, reproducible and accurate biomarkers. Methods We recruited patients from Mountain West CF Consortium (MWCFC) care centers for prospective observational study of sputum biomarkers of inflammation. After informed consent, centers enrolled randomly selected patients with CF who were clinically stable sputum producers, 12 years of age and older, without previous organ transplantation. Results From December 8, 2014 through January 16, 2016, we enrolled 114 patients (53 male) with CF with continuing data collection. Baseline characteristics included mean age 27 years (SD = 12), 80% predicted forced expiratory volume in 1 s (SD = 23%), 1.0 prior year pulmonary exacerbations (SD = 1.2), home elevation 328 m (SD = 112) above sea level. Compared with other patients in the US CF Foundation Patient Registry (CFFPR) in 2014, MWCFC patients had similar distribution of sex, age, lung function, weight and rates of exacerbations, diabetes, pancreatic insufficiency, CF-related arthropathy and airway infections including methicillin-sensitive or -resistant Staphylococcus aureus, Pseudomonas aeruginosa, Burkholderia cepacia complex, fungal and non-tuberculous Mycobacteria infections. They received CF-specific treatments at similar frequencies. Conclusions Randomly-selected, sputum-producing patients within the MWCFC represent sputum-producing patients in the CFFPR. They have similar characteristics, lung function and frequencies of pulmonary exacerbations, microbial infections and use of CF-specific treatments. These findings will plausibly make future interpretations of quantitative measurements of inflammatory biomarkers generalizable to sputum-producing patients in the CFFPR.

Published
2018

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