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188 Standardized treatment of pulmonary exacerbations (STOP) study: Clinical presentations of pulmonary exacerbations
S106
8. Complications of CF
Posters
187 Risk factors for pulmonary exacerbations (PEx) in pediatric cystic fibrosis (CF) patients
189 Standardized treatment of pulmonary exacerbation (STOP) study: Symptomatic treatment response
S. Lubovich1 , J. Buend´ıa2 , V. Rodr´ıguez1 , S. Zaragoza1 , B. Camargo1 , J. Alchundia1 , L. Galanternik1 , P. Ratto1 , A. Teper1 . 1 Hospital de Ni˜nos Ricardo Guti´errez, Buenos Aires, Argentina; 2 Hospital Italiano de Buenos Aires, Buenos Aires, Argentina
C.H. Goss1,2 , S.L. Heltshe2 , V. Thompson2 , N.E. West3 , D.B. Sanders4 , D.R. VanDevanter5 , P. Flume6 , STOP Study Group. 1 University of Washington, Seattle, United States; 2 Seattle Children’s Hospital, Seattle, United States; 3 Johns Hopkins University, Baltimore, United States; 4 University of Wisconsin, Pediatrics, Madison, United States; 5 Case Western Reserve University School of Medicine, Cleveland, United States; 6 Medical University of South Carolina, Charleston, United States
CF lung disease frequently has intermittent episodes of acute worsening of symptoms (PEx) responsible for much of the morbidity and mortality. Objective: To determine frequency and associated risk factors of PEx and to report the proportion of patients (pt) who do not recover the pulmonary function (PF). Material and Methods: Observational, cohort, retrospective study. Medical records of CFpt treated in 2013 were reviewed and divided in two groups: G1 (requiring intravenous antibiotic treatment) and G2 (not exacerbated). Current age, gender, p.F508 del, baseline FEV1 % and BMI Z score, % of P. aeruginosa (PA), MRSA and BCC chronic colonization, % of CFRD, FEV1 % at the end of the PEx and % pt who recovered the baseline FEV1 were recorded. The relative risk (RR) of the associations between the incidence of PEx and risk factors was estimated. Results: 117 pt (54% male) were included, 50 pt in G1 and 67 pt in G2. Statistically significant differences between both groups were: age (10 vs 7.7 years, p = 0.03), BMI z score (−0.65 vs −0.049, p = 0.007), p.F508 del (93% vs 71%, p = 0.007), FEV1 % (102% vs 68.5%, p = 0.000), CFRD (20% vs 0%, p = 0.000) and BCC chronic colonization (10% vs 0%, p = 0.000), PA (40% vs 11%, p = 0.001), MRSA (40% vs 13%, p = 0.002). The characteristics associated with the incidence of PEx were BMI Z score (RR 0.69, p = 0.002), p.F508 del (RR 3.23, p = 0.05) and BCC chronic colonization (RR 3.69, p = 0.002), PA (RR 1.89, p = 0.01) and MRSA (RR 2.32, p = 0.002). 23% of pt did not recover their previous lung function. Conclusions: Worst nutritional status, p.F508 del, chronic BCC, PA and MRSA colonization were risk factors for PEx. A quarter of the patients did not recover their baseline PF.
Objectives: Pulmonary exacerbations (PEx) are common in patients with CF, but there are few studies evaluating symptomatic resolution with intravenous (IV) antibiotics. Careful delineation of symptomatic response could provide an important tool to future studies. Methods: STOP is an observational study projected to enroll over 200 participants over 12 years old who are admitted to the hospital for treatment of a PEx. The CF Respiratory Symptom Diary-Chronic Respiratory Infection Symptom Scale (CFRSD-CRISS) was given to participants daily during the 28-day study. The CFRSD-CRISS is a validated unidimensional scale based on 8 items, with a total score ranging 0–100 (higher score indicates greater symptom severity). Prior analyses have suggested the responder criteria is 11.0 points. Results: 204 patients (56% female) have entered the study. 112 subjects had completed diaries at admission, at end of treatment, and at day 28. The mean CFRSD-CRISS of these 112 patients was 48.7 (SD = 10.9) at the beginning of treatment, 21.7 (SD = 16) at the end of treatment, and 27.0 (15.5) at day 28. The mean and median time to lowest CFRSD-CRISS was 11.6 (SD = 6.1) days and 11.5 (95% CI: 10.0, 13.0) days respectively. Ninety-six (85.7%) had a 11 point reduction at the end of treatment compared to 86 (76.8%) subjects by day 28 of the study. Conclusion: While the study is still ongoing, clear treatment responses have been noted in the CFRSD-CRISS. A high percentage of these patients had experienced a clinically meaningful improvement in the CFRSD-CRISS by the end of treatment and to a lesser degree by day 28. Final analysis data will be presented. We thank the CFFT TDN. Funding: CFF.
188 Standardized treatment of pulmonary exacerbations (STOP) study: Clinical presentations of pulmonary exacerbations
190 Frequency and costs of pulmonary exacerbations and association with % predicted FEV1 in patients with cystic fibrosis
D.B. Sanders1 , C.H. Goss2 , D.R. VanDevanter3 , S.L. Heltse4 , V. Thompson5 , P. Flume6 . 1 University of Wisconsin, Pediatrics, Madison, United States; 2 University of Washington, Medicine, Seattle, United States; 3 Case Western Reserve University School of Medicine, Pediatrics, Cleveland, United States; 4 University of Washington, Pediatrics, Seattle, United States; 5 Seattle Children’s Hospital, Seattle, United States; 6 Medical University of South Carolina, Medicine, Charleston, United States
J.L. Rubin-Cahill1 , S. Thayer2 , A. Watkins2 , J. Wagener1 , P. Hodgkins1 , M.S. Schechter3 . 1 Vertex Pharmaceuticals Incorporated, Boston, United States; 2 Optum, Eden Prairie, United States; 3 Virginia Commonwealth University, Richmond, United States
Objectives: Pulmonary exacerbations (PEx) are common in patients with CF, but there is no standard definition. Our objective was to describe the phenotypes of PExs treated with IV antibiotics in adults and adolescents with CF. Methods: STOP is an observational study at 10 US CF centers that will enroll over 200 participants 12 years of age admitted to the hospital for treatment of PEx, determined at the discretion of the treating physician. Demographic and clinical data are collected at the time of enrollment. Results: After 12 months of enrollment, 204 patients (56% female) have entered the study. The mean age is 27.2 years (SD 9.8) with a range of 12.1–74.9 years. There are 27 (15%) patients <18 years of age. Among 157 patients with available admission spirometry, the mean FEV1 is 52% predicted (range 16–108%; SD 22). FEV1 fell >10% from best in the 6 months prior in 45%. Among 173 patients with available physician assessment, the duration of symptoms prior to admission was <7 days in 13%, 7−21 days in 53%, or >21 days in 33%. 46% had failed outpatient antibiotics prior to admission and 43% had received IV antibiotics in the 6 months prior to admission. Signs and symptoms present at admission included chest pain (24%), hemoptysis (11%), and wheezing (17%). Conclusion: Most patients enrolled in the study are adults, but there is a goal to include up to 30% pediatric patients. Most patients have had symptoms for more than one week before admission, and nearly half had first been treated as an outpatient with oral antibiotics. These are only preliminary data and results may change as numbers increase. Enrollment will be completed in January, 2015. Funded by CFFT TDN.
Objectives: To investigate the frequency and costs of pulmonary exacerbations (PEx) and the association between FEV1 and PEx in patients (pts) with cystic fibrosis (CF). Methods: Chart data were linked to claims from pts with CF aged 6 y enrolled for 18 months in a US commercial health plan, identified between July 2008 and May 2013. The most recent 12 months (follow-up) were used to calculate outcomes. PEx were defined as 1 of: inpatient (IP) stay coded ICD-9-CM 277.02; IP staywith ICD-9-CM for respiratory infection; new IV or oral antibiotics administered IP or outpatient (chronic use antibiotics were excluded). New PEx required a preceding 7 days with no claims meeting PEx criteria. The best % predicted FEV1 (ppFEV1 ) recorded in charts during the 6 months prior to the follow-up period (baseline) was used to categorize lung dysfunction as severe (<40), moderate (40−<70), and mild (70). Results: 268 pts were included (mean age 24.0 y). 88.1% of pts had 1 PEx; 48.1% had 1 PEx treated with IV antibiotics and/or IP stay (overall rate 2.9/y and 0.87/y, respectively). For any PEx, the mean cost was $13.1K and duration was 21 days; for a PEx requiring IV and/or IP stay, it was $36.6K and 27 days, respectively; 69% of these required an IP stay, with mean cost $47.1K. Of n = 241 pts with baseline ppFEV1 , 71.8% were mild, 23.2% moderate, and 5.0% severe. A higher percentage of pts with moderate and severe lung disease had 1 PEx and 1 IV/IP PEx, and incurred higher unadjusted mean annual PEx costs (severe: $119.9K; moderate: $40.8K; mild: $30.1K; P < 0.001). Conclusion: The majority of CF pts experience 1 PEx/y; frequency and costs of PEx increase in pts with lower ppFEV1 .
8. Complications of CF
Posters
187 Risk factors for pulmonary exacerbations (PEx) in pediatric cystic fibrosis (CF) patients
189 Standardized treatment of pulmonary exacerbation (STOP) study: Symptomatic treatment response
S. Lubovich1 , J. Buend´ıa2 , V. Rodr´ıguez1 , S. Zaragoza1 , B. Camargo1 , J. Alchundia1 , L. Galanternik1 , P. Ratto1 , A. Teper1 . 1 Hospital de Ni˜nos Ricardo Guti´errez, Buenos Aires, Argentina; 2 Hospital Italiano de Buenos Aires, Buenos Aires, Argentina
C.H. Goss1,2 , S.L. Heltshe2 , V. Thompson2 , N.E. West3 , D.B. Sanders4 , D.R. VanDevanter5 , P. Flume6 , STOP Study Group. 1 University of Washington, Seattle, United States; 2 Seattle Children’s Hospital, Seattle, United States; 3 Johns Hopkins University, Baltimore, United States; 4 University of Wisconsin, Pediatrics, Madison, United States; 5 Case Western Reserve University School of Medicine, Cleveland, United States; 6 Medical University of South Carolina, Charleston, United States
CF lung disease frequently has intermittent episodes of acute worsening of symptoms (PEx) responsible for much of the morbidity and mortality. Objective: To determine frequency and associated risk factors of PEx and to report the proportion of patients (pt) who do not recover the pulmonary function (PF). Material and Methods: Observational, cohort, retrospective study. Medical records of CFpt treated in 2013 were reviewed and divided in two groups: G1 (requiring intravenous antibiotic treatment) and G2 (not exacerbated). Current age, gender, p.F508 del, baseline FEV1 % and BMI Z score, % of P. aeruginosa (PA), MRSA and BCC chronic colonization, % of CFRD, FEV1 % at the end of the PEx and % pt who recovered the baseline FEV1 were recorded. The relative risk (RR) of the associations between the incidence of PEx and risk factors was estimated. Results: 117 pt (54% male) were included, 50 pt in G1 and 67 pt in G2. Statistically significant differences between both groups were: age (10 vs 7.7 years, p = 0.03), BMI z score (−0.65 vs −0.049, p = 0.007), p.F508 del (93% vs 71%, p = 0.007), FEV1 % (102% vs 68.5%, p = 0.000), CFRD (20% vs 0%, p = 0.000) and BCC chronic colonization (10% vs 0%, p = 0.000), PA (40% vs 11%, p = 0.001), MRSA (40% vs 13%, p = 0.002). The characteristics associated with the incidence of PEx were BMI Z score (RR 0.69, p = 0.002), p.F508 del (RR 3.23, p = 0.05) and BCC chronic colonization (RR 3.69, p = 0.002), PA (RR 1.89, p = 0.01) and MRSA (RR 2.32, p = 0.002). 23% of pt did not recover their previous lung function. Conclusions: Worst nutritional status, p.F508 del, chronic BCC, PA and MRSA colonization were risk factors for PEx. A quarter of the patients did not recover their baseline PF.
Objectives: Pulmonary exacerbations (PEx) are common in patients with CF, but there are few studies evaluating symptomatic resolution with intravenous (IV) antibiotics. Careful delineation of symptomatic response could provide an important tool to future studies. Methods: STOP is an observational study projected to enroll over 200 participants over 12 years old who are admitted to the hospital for treatment of a PEx. The CF Respiratory Symptom Diary-Chronic Respiratory Infection Symptom Scale (CFRSD-CRISS) was given to participants daily during the 28-day study. The CFRSD-CRISS is a validated unidimensional scale based on 8 items, with a total score ranging 0–100 (higher score indicates greater symptom severity). Prior analyses have suggested the responder criteria is 11.0 points. Results: 204 patients (56% female) have entered the study. 112 subjects had completed diaries at admission, at end of treatment, and at day 28. The mean CFRSD-CRISS of these 112 patients was 48.7 (SD = 10.9) at the beginning of treatment, 21.7 (SD = 16) at the end of treatment, and 27.0 (15.5) at day 28. The mean and median time to lowest CFRSD-CRISS was 11.6 (SD = 6.1) days and 11.5 (95% CI: 10.0, 13.0) days respectively. Ninety-six (85.7%) had a 11 point reduction at the end of treatment compared to 86 (76.8%) subjects by day 28 of the study. Conclusion: While the study is still ongoing, clear treatment responses have been noted in the CFRSD-CRISS. A high percentage of these patients had experienced a clinically meaningful improvement in the CFRSD-CRISS by the end of treatment and to a lesser degree by day 28. Final analysis data will be presented. We thank the CFFT TDN. Funding: CFF.
188 Standardized treatment of pulmonary exacerbations (STOP) study: Clinical presentations of pulmonary exacerbations
190 Frequency and costs of pulmonary exacerbations and association with % predicted FEV1 in patients with cystic fibrosis
D.B. Sanders1 , C.H. Goss2 , D.R. VanDevanter3 , S.L. Heltse4 , V. Thompson5 , P. Flume6 . 1 University of Wisconsin, Pediatrics, Madison, United States; 2 University of Washington, Medicine, Seattle, United States; 3 Case Western Reserve University School of Medicine, Pediatrics, Cleveland, United States; 4 University of Washington, Pediatrics, Seattle, United States; 5 Seattle Children’s Hospital, Seattle, United States; 6 Medical University of South Carolina, Medicine, Charleston, United States
J.L. Rubin-Cahill1 , S. Thayer2 , A. Watkins2 , J. Wagener1 , P. Hodgkins1 , M.S. Schechter3 . 1 Vertex Pharmaceuticals Incorporated, Boston, United States; 2 Optum, Eden Prairie, United States; 3 Virginia Commonwealth University, Richmond, United States
Objectives: Pulmonary exacerbations (PEx) are common in patients with CF, but there is no standard definition. Our objective was to describe the phenotypes of PExs treated with IV antibiotics in adults and adolescents with CF. Methods: STOP is an observational study at 10 US CF centers that will enroll over 200 participants 12 years of age admitted to the hospital for treatment of PEx, determined at the discretion of the treating physician. Demographic and clinical data are collected at the time of enrollment. Results: After 12 months of enrollment, 204 patients (56% female) have entered the study. The mean age is 27.2 years (SD 9.8) with a range of 12.1–74.9 years. There are 27 (15%) patients <18 years of age. Among 157 patients with available admission spirometry, the mean FEV1 is 52% predicted (range 16–108%; SD 22). FEV1 fell >10% from best in the 6 months prior in 45%. Among 173 patients with available physician assessment, the duration of symptoms prior to admission was <7 days in 13%, 7−21 days in 53%, or >21 days in 33%. 46% had failed outpatient antibiotics prior to admission and 43% had received IV antibiotics in the 6 months prior to admission. Signs and symptoms present at admission included chest pain (24%), hemoptysis (11%), and wheezing (17%). Conclusion: Most patients enrolled in the study are adults, but there is a goal to include up to 30% pediatric patients. Most patients have had symptoms for more than one week before admission, and nearly half had first been treated as an outpatient with oral antibiotics. These are only preliminary data and results may change as numbers increase. Enrollment will be completed in January, 2015. Funded by CFFT TDN.
Objectives: To investigate the frequency and costs of pulmonary exacerbations (PEx) and the association between FEV1 and PEx in patients (pts) with cystic fibrosis (CF). Methods: Chart data were linked to claims from pts with CF aged 6 y enrolled for 18 months in a US commercial health plan, identified between July 2008 and May 2013. The most recent 12 months (follow-up) were used to calculate outcomes. PEx were defined as 1 of: inpatient (IP) stay coded ICD-9-CM 277.02; IP staywith ICD-9-CM for respiratory infection; new IV or oral antibiotics administered IP or outpatient (chronic use antibiotics were excluded). New PEx required a preceding 7 days with no claims meeting PEx criteria. The best % predicted FEV1 (ppFEV1 ) recorded in charts during the 6 months prior to the follow-up period (baseline) was used to categorize lung dysfunction as severe (<40), moderate (40−<70), and mild (70). Results: 268 pts were included (mean age 24.0 y). 88.1% of pts had 1 PEx; 48.1% had 1 PEx treated with IV antibiotics and/or IP stay (overall rate 2.9/y and 0.87/y, respectively). For any PEx, the mean cost was $13.1K and duration was 21 days; for a PEx requiring IV and/or IP stay, it was $36.6K and 27 days, respectively; 69% of these required an IP stay, with mean cost $47.1K. Of n = 241 pts with baseline ppFEV1 , 71.8% were mild, 23.2% moderate, and 5.0% severe. A higher percentage of pts with moderate and severe lung disease had 1 PEx and 1 IV/IP PEx, and incurred higher unadjusted mean annual PEx costs (severe: $119.9K; moderate: $40.8K; mild: $30.1K; P < 0.001). Conclusion: The majority of CF pts experience 1 PEx/y; frequency and costs of PEx increase in pts with lower ppFEV1 .