J M Cedarbaum1, N Stambler. 1. Regeneron Pharmaceuticals Inc, Tarrytown, NY 10591, USA. jesse.cedarbaum@regpha.com
Abstract
INTRODUCTION: Use of mechanical ventilation (MV), administered either invasively via tracheostomy, or more commonly non-invasively (CPAP, BiPAP), appears to be increasing in ALS. No prospective databases exist that describe the behavior of physicians and patients and the criteria for instituting MV in ALS. METHODS:387placebo patients in a Phase III trial of r-metHuBDNF were followed for 9 months. Although the use of MV was not the primary end-point of the trial, information was gathered regarding it by cataloging respiratory adverse events and tracking health resource utilization. RESULTS: 35 of 387 patients utilized MV during the trial. Twenty-eight (7%) patients received BiPAP. Seven (2%) were tracheotomized without first receiving BiPAP. Forced vital capacity (FVC): BiPAP patients had a mean ( SEM) FVC% of 71.8 +/- 2.8% and ALSFRS of 27.7 +/- 1.0 at baseline; non-BiPAP patients had a mean baseline FVC% of 88.7 +/- 1.0%, and an ALSFRS of 30.3 +/- 0.3. Symptom duration at entry was similar for both groups (2.1 +/- 0.4 years vs. 2.1 +/- 0.1 years). At the time of first use of BiPAP, average FVC% was 47.5 +/- 4.0% and ALSFRS score was 22.4 +/- 1.5. The range of FVC% at start of BiPAP was 15-87. The nine-month survival was 67.9% for BiPAP patients vs. 86% for non-BiPAP patients. The use of BiPAP varied tremendously among the 38 study sites, with some not employing it at all and others using it in as many as 40% of patients. CONCLUSIONS: Of the 9% of placebo patients who received MV, BiPAP patients were more rapidly progressing than non-BIPAP patients, and showed a greater eventual mortality rate. Patients began MV at a wide range of values of FVC%, and centers differed in their prescribing practices. Factors influencing BiPAP use are complex, and not strictly related to FVC%.
RCT Entities:
INTRODUCTION: Use of mechanical ventilation (MV), administered either invasively via tracheostomy, or more commonly non-invasively (CPAP, BiPAP), appears to be increasing in ALS. No prospective databases exist that describe the behavior of physicians and patients and the criteria for instituting MV in ALS. METHODS: 387 placebo patients in a Phase III trial of r-metHuBDNF were followed for 9 months. Although the use of MV was not the primary end-point of the trial, information was gathered regarding it by cataloging respiratory adverse events and tracking health resource utilization. RESULTS: 35 of 387 patients utilized MV during the trial. Twenty-eight (7%) patients received BiPAP. Seven (2%) were tracheotomized without first receiving BiPAP. Forced vital capacity (FVC): BiPAPpatients had a mean ( SEM) FVC% of 71.8 +/- 2.8% and ALSFRS of 27.7 +/- 1.0 at baseline; non-BiPAPpatients had a mean baseline FVC% of 88.7 +/- 1.0%, and an ALSFRS of 30.3 +/- 0.3. Symptom duration at entry was similar for both groups (2.1 +/- 0.4 years vs. 2.1 +/- 0.1 years). At the time of first use of BiPAP, average FVC% was 47.5 +/- 4.0% and ALSFRS score was 22.4 +/- 1.5. The range of FVC% at start of BiPAP was 15-87. The nine-month survival was 67.9% for BiPAPpatients vs. 86% for non-BiPAPpatients. The use of BiPAP varied tremendously among the 38 study sites, with some not employing it at all and others using it in as many as 40% of patients. CONCLUSIONS: Of the 9% of placebo patients who received MV, BiPAPpatients were more rapidly progressing than non-BIPAPpatients, and showed a greater eventual mortality rate. Patients began MV at a wide range of values of FVC%, and centers differed in their prescribing practices. Factors influencing BiPAP use are complex, and not strictly related to FVC%.
Authors: Lori A Lind; Ellyn M Andel; Angela L McCall; Justin S Dhindsa; Katherine A Johnson; Olivia E Stricklin; Christian Mueller; Mai K ElMallah; Teresa E Lever; Nicole L Nichols Journal: Hum Gene Ther Date: 2020-07-13 Impact factor: 5.695
Authors: Nicole L Nichols; Genevieve Gowing; Irawan Satriotomo; Lisa J Nashold; Erica A Dale; Masatoshi Suzuki; Pablo Avalos; Patrick L Mulcrone; Jacalyn McHugh; Clive N Svendsen; Gordon S Mitchell Journal: Am J Respir Crit Care Med Date: 2012-12-06 Impact factor: 21.405
Authors: Hans D Katzberg; Adam Selegiman; Lee Guion; Nancy Yuan; Sungho C Cho; Jonathan S Katz; Robert G Miller; Yuen T So Journal: J Clin Sleep Med Date: 2013-04-15 Impact factor: 4.062
Authors: Özden O Dalgıç; F Safa Erenay; Kalyan S Pasupathy; Osman Y Özaltın; Brian A Crum; Mustafa Y Sir Journal: J Neurol Date: 2019-01-25 Impact factor: 4.849
Authors: Nicole L Nichols; J Van Dyke; L Nashold; I Satriotomo; M Suzuki; G S Mitchell Journal: Respir Physiol Neurobiol Date: 2013-05-18 Impact factor: 1.931