TY -的T1 - o预后模型的发展f respiratory insufficiency or death in amyotrophic lateral sclerosis JF - European Respiratory Journal JO - Eur Respir J DO - 10.1183/13993003.02237-2018 VL - 53 IS - 4 SP - 1802237 AU - Ackrivo, Jason AU - Hansen-Flaschen, John AU - Wileyto, E. Paul AU - Schwab, Richard J. AU - Elman, Lauren AU - Kawut, Steven M. Y1 - 2019/04/01 UR - //www.qdcxjkg.com/content/53/4/1802237.abstract N2 - A clinically useful model to prognose onset of respiratory insufficiency in amyotrophic lateral sclerosis (ALS) would inform disease interventions, communication and clinical trial design. We aimed to derive and validate a clinical prognostic model for respiratory insufficiency within 6 months of presentation to an outpatient ALS clinic.We used multivariable logistic regression and internal cross-validation to derive a clinical prognostic model using a single-centre cohort of 765 ALS patients who presented between 2006 and 2015. External validation was performed using the multicentre Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database with 7083 ALS patients. Predictors included baseline characteristics at first outpatient visit. The primary outcome was respiratory insufficiency within 6 months, defined by initiation of noninvasive ventilation, forced vital capacity (FVC) <50% predicted, tracheostomy, or death.Of 765 patients in our centre, 300 (39%) had respiratory insufficiency or death within 6 months. Six baseline characteristics (diagnosis age, delay between symptom onset and diagnosis, FVC, symptom onset site, amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) total score and ALSFRS-R dyspnoea score) were used to prognose the risk of the primary outcome. The derivation cohort c-statistic was 0.86 (95% CI 0.84–0.89) and internal cross-validation produced a c-statistic of 0.86 (95% CI 0.85–0.87). External validation of the model using the PRO-ACT cohort produced a c-statistic of 0.74 (95% CI 0.72–0.75).We derived and externally validated a clinical prognostic rule for respiratory insufficiency in ALS. Future studies should investigate interventions on equivalent high-risk patients.ALS has a heterogenous progression to respiratory failure. A clinician can use the clinical prognostic rule to estimate a 6-month risk of respiratory failure onset, thus facilitating referrals and respiratory interventions http://ow.ly/LLzP30nqm2D ER -