OBJECTIVE: To assess the impact of calfactant (a modified natural bovine lung surfactant) in immunocompromised children with acute lung injury and to determine the number of patients required for a definitive clinical trial of calfactant in this population. DESIGN: Post hoc analysis of data from a previous randomized, control trial. SETTING:Tertiary care pediatric intensive care units. PATIENTS: All children, defined as immunocompromised, enrolled in a multicenter, masked, randomized, control trial of calfactant for acute lung injury conducted between July 2000 and July 2003. INTERVENTIONS: Patients received either an intratracheal instillation of calfactant or an equal volume of air placebo in a protocolized manner. MEASUREMENTS AND MAIN RESULTS:Eleven of 22 (50%) calfactant-treated patients died when compared with 18 of 30 (60%) placebo patients (absolute risk reduction 10.0%, 95% confidence interval [CI] -17.3, 37.3). Among the 23 patients with an initial oxygen index (OI) >/=13 and </=37, 44% (4 of 9) of calfactant-treated patients died in comparison with 71% (10 of 14) of placebo (absolute risk reduction 27.0%, 95% CI -13.2, 67.2). Only 33% (3 of 9) of calfactant patients died before intensive care discharge in comparison with 71% (10 of 14) of placebo (absolute risk reduction 38.1%, 95% CI -0.7, 76.9). Calfactant therapy was associated with improved oxygenation in these 23 patients. Using an OI entry criterion of (13 </= OI </= 37), stratifying on the presence of hematopoietic stem cell transplantation, and accepting the 27% difference in mortality observed in this analysis, 63 patients would be required in each arm of a randomized, control trial to demonstrate a significant effect of calfactant on mortality in this patient population assuming a two-sided alpha of 0.05 and a power of 0.85. CONCLUSIONS: These preliminary data suggest a potential benefit of calfactant in this high-risk population. A clinical trial powered to appropriately assess these findings seems warranted and feasible.
RCT Entities:
OBJECTIVE: To assess the impact of calfactant (a modified natural bovine lung surfactant) in immunocompromised children with acute lung injury and to determine the number of patients required for a definitive clinical trial of calfactant in this population. DESIGN: Post hoc analysis of data from a previous randomized, control trial. SETTING: Tertiary care pediatric intensive care units. PATIENTS: All children, defined as immunocompromised, enrolled in a multicenter, masked, randomized, control trial of calfactant for acute lung injury conducted between July 2000 and July 2003. INTERVENTIONS:Patients received either an intratracheal instillation of calfactant or an equal volume of air placebo in a protocolized manner. MEASUREMENTS AND MAIN RESULTS: Eleven of 22 (50%) calfactant-treated patients died when compared with 18 of 30 (60%) placebo patients (absolute risk reduction 10.0%, 95% confidence interval [CI] -17.3, 37.3). Among the 23 patients with an initial oxygen index (OI) >/=13 and </=37, 44% (4 of 9) of calfactant-treated patients died in comparison with 71% (10 of 14) of placebo (absolute risk reduction 27.0%, 95% CI -13.2, 67.2). Only 33% (3 of 9) of calfactant patients died before intensive care discharge in comparison with 71% (10 of 14) of placebo (absolute risk reduction 38.1%, 95% CI -0.7, 76.9). Calfactant therapy was associated with improved oxygenation in these 23 patients. Using an OI entry criterion of (13 </= OI </= 37), stratifying on the presence of hematopoietic stem cell transplantation, and accepting the 27% difference in mortality observed in this analysis, 63 patients would be required in each arm of a randomized, control trial to demonstrate a significant effect of calfactant on mortality in this patient population assuming a two-sided alpha of 0.05 and a power of 0.85. CONCLUSIONS: These preliminary data suggest a potential benefit of calfactant in this high-risk population. A clinical trial powered to appropriately assess these findings seems warranted and feasible.
Authors: Rahul Bhatia; Thomas H Shaffer; Jobayer Hossain; Alicia Olivant Fisher; Liana M Horner; M Elena Rodriguez; Scott Penfil; Mary C Theroux Journal: Pediatr Pulmonol Date: 2011-05-26
Authors: Daniele De Luca; Paola Cogo; Martin C Kneyber; Paolo Biban; Malcolm Grace Semple; Jesus Perez-Gil; Giorgio Conti; Pierre Tissieres; Peter C Rimensberger Journal: Crit Care Date: 2021-02-22 Impact factor: 9.097