(provided by the applicant): 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.

Public Health Relevance

Although cancer is responsible for more deaths in children over one year of age than any other disease, outcomes are improving as a result of more effective anti- cancer therapy including the use of hematopoietic stem cell transplantation. Unfortunately, lung injury is a common, life-threatening condition among these patients as a result of their disease and its therapy. Consequently, for outcomes to continue to improve for this patient population, more effective therapy for the prevention and treatment of lung injury is needed.

Agency
National Institute of Health (NIH)
Institute
Food and Drug Administration (FDA)
Type
Research Project (R01)
Project #
5R01FD003410-02
Application #
7932202
Study Section
Special Emphasis Panel (ZFD1-OPD-N (S1))
Project Start
2009-09-15
Project End
2015-09-14
Budget Start
2013-09-15
Budget End
2014-09-14
Support Year
2
Fiscal Year
2013
Total Cost
Indirect Cost
Name
Pennsylvania State University
Department
Pediatrics
Type
Schools of Medicine
DUNS #
129348186
City
Hershey
State
PA
Country
United States
Zip Code
17033
Khemani, Robinder G; Thomas, Neal J; Venkatachalam, Vani et al. (2012) Comparison of SpO2 to PaO2 based markers of lung disease severity for children with acute lung injury. Crit Care Med 40:1309-16