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A pulmonary exacerbation risk score among cystic fibrosis patients not receiving recommended care.
Pediatric Pulmonology 2013 October
BACKGROUND: Pulmonary exacerbations (PEx) lead to substantial morbidity in cystic fibrosis (CF), and guidelines recommend chronic medication including dornase alfa and inhaled tobramycin. However PEx risk and medication use vary across patients.
OBJECTIVE: To develop a PEx risk score among CF patients not receiving guideline-recommended chronic respiratory medications.
METHODS: A cohort of patients with FEV1%-predicted between 25% and 75% without evidence of dornase alfa or inhaled tobramycin use in an index year, despite meeting guideline recommended criteria, was identified from the CF Foundation Patient Registry (2002-2008). This sample was randomly split into 2/3 for a development sample and 1/3 for a validation sample. A multivariable risk score was developed to predict PEx requiring hospitalization or home IV treatment using available patient characteristics. Its predictive performance was assessed in the validation sample.
RESULTS: Among 3,069 patient-years, 1,275 (42%) had PEx in the subsequent year. The risk score included, in order of decreasing impact on PEx risk, prior PEx, Pseudomonas aeruginosa, allergic bronchopulmonary aspergillosis, depression, methicillin-resistant Staphylococcus aureus, CF-related diabetes, Burkholderia cepacia, prior use of dornase alfa, bronchodilator use, prior use of inhaled tobramycin and lower FEV1%-predicted. Stratifying patients by risk score in the validation sample identified actual risks ranging from 14% in the lowest decile to 90% in the highest. The c-statistic was 0.8.
CONCLUSIONS: A PEx risk score for CF patients not receiving guideline-recommended chronic therapies was developed and validated, and identified patients with a wide range of risk. This score could identify high-risk patients in whom chronic therapies should be initiated or continued.
OBJECTIVE: To develop a PEx risk score among CF patients not receiving guideline-recommended chronic respiratory medications.
METHODS: A cohort of patients with FEV1%-predicted between 25% and 75% without evidence of dornase alfa or inhaled tobramycin use in an index year, despite meeting guideline recommended criteria, was identified from the CF Foundation Patient Registry (2002-2008). This sample was randomly split into 2/3 for a development sample and 1/3 for a validation sample. A multivariable risk score was developed to predict PEx requiring hospitalization or home IV treatment using available patient characteristics. Its predictive performance was assessed in the validation sample.
RESULTS: Among 3,069 patient-years, 1,275 (42%) had PEx in the subsequent year. The risk score included, in order of decreasing impact on PEx risk, prior PEx, Pseudomonas aeruginosa, allergic bronchopulmonary aspergillosis, depression, methicillin-resistant Staphylococcus aureus, CF-related diabetes, Burkholderia cepacia, prior use of dornase alfa, bronchodilator use, prior use of inhaled tobramycin and lower FEV1%-predicted. Stratifying patients by risk score in the validation sample identified actual risks ranging from 14% in the lowest decile to 90% in the highest. The c-statistic was 0.8.
CONCLUSIONS: A PEx risk score for CF patients not receiving guideline-recommended chronic therapies was developed and validated, and identified patients with a wide range of risk. This score could identify high-risk patients in whom chronic therapies should be initiated or continued.
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