Acute lung injury (ALI) is a common, life-threatening complication among pediatric leukemia and lymphoma and hematopoietic stem cell transplant (HSCT) recipients. Although these children represent a relatively small and unique patient population, they account for the largest proportion of deaths of all pediatric diseases. The long-term goal of this project is to improve outcomes among these patients. Recently, the intratracheal administration of calfactant has resulted in decreased mortality among children with ALI including promising results among children with cancer and following HSCT. Consequently, the primary specific aim of this study is to assess the effect of calfactant on intensive care (PICU) survival among pediatric leukemia and lymphoma and HSCT patients with ALI. Secondary aims include assessment of the effect of calfactant on oxygenation and on the length of mechanical ventilation, PICU stay, and hospital stay. Calfactant therapy has been found to be of benefit in acute lung injury in the overall pediatric population by improving oxygenation and decreasing mortality. These findings, in conjunction with recent subgroup analysis in which calfactant therapy appeared to improve outcomes in immunocompromised children provide the rationale for assessing calfactant therapy in this patient population. Funding Source - FDA Office of Orphan Products Development (OOPD)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
43
Endotracheal calfactant, up to 3 doses if subject qualifies
Endotracheal air administration
Phoenix Children's Hospital
Phoenix, Arizona, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California San Francisco
San Francisco, California, United States
Riley Children's Hospital
Indianapolis, Indiana, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Weill Cornell Medical Center
New York, New York, United States
Maria Fareri Children's Hospital
Valhalla, New York, United States
Rainbow Babies Hospital
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Penn State College of Medicine, Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
...and 6 more locations
All-cause Mortality at the Time of Pediatric Intensive Care Unit (PICU) Discharge
Overall mortality rate from admission to PICU discharge
Time frame: Admission to PICU discharge, up to 120 days
Ventilator Free Days (VFDs)
Number of days the patient is alive and off of the ventilator
Time frame: 60 days after study enrollment
Total Duration of Stay Required
Length of stay (LOS) ,measured in days, from admission to PICU discharge and admission to hospital discharge.
Time frame: Admission to discharge, up to 120 days
Change in Oxygenation: First Intervention
The Oxygenation Index after the first intervention is calculated as the fraction of inspired oxygen, in percent, times the mean airway pressure, in mmHg, divided by the partial pressure of oxygen in arterial blood, in mmHg. Lower values are better.
Time frame: 48 hours after enrollment, up to 12 hours after each intervention
Change in Oxygenation: Second Intervention
The Oxygenation Index after the second intervention (if applicable) is calculated as the fraction of inspired oxygen, in percent, times the mean airway pressure, in mmHg, divided by the partial pressure of oxygen in arterial blood, in mmHg. Lower values are better.
Time frame: 48 hours after enrollment, up to 12 hours after each intervention
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