Natalie E West1, Valeria V Beckett2, Raksha Jain3, Don B Sanders4, Jerry A Nick5, Sonya L Heltshe6, Elliott C Dasenbrook7, Donald R VanDevanter8, George M Solomon9, Christopher H Goss10, Patrick A Flume11. 1. Department of Medicine, Johns Hopkins University, Baltimore, MD, United States. Electronic address: nwest5@jhmi.edu. 2. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, United States. 3. Department of Medicine, University of Texas Southwestern Medical Center, Dallas, TX, United States. 4. Department of Pediatrics, Indiana University, Indianapolis, IN, United States. 5. Department of Medicine, National Jewish Health, Denver, CO, United States. 6. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, United States; Department of Pediatrics, University of Washington, Seattle, WA, United States. 7. Respiratory Institute, Cleveland Clinic, Cleveland, OH, United States. 8. Department of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, OH, United States. 9. Department of Medicine and the Gregory Fleming James Cystic Fibrosis Center, University of Alabama at Birmingham, Birmingham, AL, United States. 10. Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, United States; Department of Pediatrics, University of Washington, Seattle, WA, United States; Department of Medicine, University of Washington, Seattle, WA, United States. 11. Department of Medicine, Medical University of South Carolina, Charleston, SC, United States; Department of Pediatrics, Medical University of South Carolina, Charleston, SC, United States.
Abstract
BACKGROUND: Pulmonary Exacerbations (PEx) are associated with increased morbidity and mortality in individuals with CF. PEx management practices vary widely, and optimization through interventional trials could potentially improve outcomes. The object of this analysis was to evaluate current physician treatment practices and patient outcomes for PEx. METHODS: The Standardized Treatment of Pulmonary Exacerbations (STOP) observational study enrolled 220 participants ≥12years old admitted to the hospital for PEx at 11 U.S. CF centers. Spirometry and daily symptom scores were collected during the study. Physicians were surveyed on treatment goals and their management practices were observed. Treatment outcomes were compared to stated goals. RESULTS: The mean (SD) duration of IV antibiotic treatment was 15.9 (6.0) days. Those individuals with more severe lung disease (<50% FEV1) were treated nearly two days longer than those with >50% FEV1. Physician-reported FEV1 improvement goals were 10% (95% CI: 5%, 14%) lower for patients with 6-month baseline FEV1 ≤50% predicted compared with those with 6-month baseline FEV1 >50% predicted. There were clinically and statistically significant improvements in symptoms from the start of IV antibiotic treatment to the end of IV antibiotic treatment and 28days after the start of treatment. The mean absolute increase in FEV1 from admission was 9% predicted at end of IV antibiotic treatment, and 7% predicted at day 28. Only 39% fully recovered lost lung function, and only 65% recovered at least 90% of lost lung function. Treatment was deemed successful by 84% of clinicians, although 6-month baseline FEV1 was only recovered in 39% of PEx. CONCLUSIONS: In this prospective observational study of PEx, treatment regimens and durations showed substantial variation. A significant proportion of patients did not reach physician's treatment goals, yet treatment was deemed successful.
BACKGROUND: Pulmonary Exacerbations (PEx) are associated with increased morbidity and mortality in individuals with CF. PEx management practices vary widely, and optimization through interventional trials could potentially improve outcomes. The object of this analysis was to evaluate current physician treatment practices and patient outcomes for PEx. METHODS: The Standardized Treatment of Pulmonary Exacerbations (STOP) observational study enrolled 220 participants ≥12years old admitted to the hospital for PEx at 11 U.S. CF centers. Spirometry and daily symptom scores were collected during the study. Physicians were surveyed on treatment goals and their management practices were observed. Treatment outcomes were compared to stated goals. RESULTS: The mean (SD) duration of IV antibiotic treatment was 15.9 (6.0) days. Those individuals with more severe lung disease (<50% FEV1) were treated nearly two days longer than those with >50% FEV1. Physician-reported FEV1 improvement goals were 10% (95% CI: 5%, 14%) lower for patients with 6-month baseline FEV1 ≤50% predicted compared with those with 6-month baseline FEV1 >50% predicted. There were clinically and statistically significant improvements in symptoms from the start of IV antibiotic treatment to the end of IV antibiotic treatment and 28days after the start of treatment. The mean absolute increase in FEV1 from admission was 9% predicted at end of IV antibiotic treatment, and 7% predicted at day 28. Only 39% fully recovered lost lung function, and only 65% recovered at least 90% of lost lung function. Treatment was deemed successful by 84% of clinicians, although 6-month baseline FEV1 was only recovered in 39% of PEx. CONCLUSIONS: In this prospective observational study of PEx, treatment regimens and durations showed substantial variation. A significant proportion of patients did not reach physician's treatment goals, yet treatment was deemed successful.
Authors: Sonya L Heltshe; Natalie E West; Donald R VanDevanter; D B Sanders; Valeria V Beckett; Patrick A Flume; Christopher H Goss Journal: Contemp Clin Trials Date: 2017-11-21 Impact factor: 2.226
Authors: Don B Sanders; George M Solomon; Valeria V Beckett; Natalie E West; Cori L Daines; Sonya L Heltshe; Donald R VanDevanter; Jonathan E Spahr; Ronald L Gibson; Jerry A Nick; Bruce C Marshall; Patrick A Flume; Christopher H Goss Journal: J Cyst Fibros Date: 2017-04-29 Impact factor: 5.482
Authors: Christopher H Goss; Yukihiro Kaneko; Lisa Khuu; Gail D Anderson; Sumedha Ravishankar; Moira L Aitken; Noah Lechtzin; Guolin Zhou; Daniel M Czyz; Kathryn McLean; Oyebode Olakanmi; Howard A Shuman; Mary Teresi; Ellen Wilhelm; Ellen Caldwell; Stephen J Salipante; Douglas B Hornick; Richard J Siehnel; Lev Becker; Bradley E Britigan; Pradeep K Singh Journal: Sci Transl Med Date: 2018-09-26 Impact factor: 17.956
Authors: Ranjani Somayaji; Renee Russell; Jonathan D Cogen; Cristopher H Goss; Sarah E Nick; Milene T Saavedra; Jennifer L Taylor-Cousar; Jerry A Nick; Dave P Nichols Journal: Ann Am Thorac Soc Date: 2019-07