CALIPSO: A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients

for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network

Research output: Contribution to journalArticle

3 Citations (Scopus)

Abstract

To assess if calfactant reduces mortality among children with leukemia/lymphoma or after hematopoietic cell transplantation (HCT) with pediatric acute respiratory distress syndrome (PARDS), we conducted a multicenter, randomized, placebo-controlled, double-blinded trial in 17 pediatric intensive care units (PICUs) of tertiary care children's hospitals. Patients ages 18 months to 25 years with leukemia/lymphoma or having undergone HCT who required invasive mechanical ventilation for bilateral lung disease with an oxygenation index (OI) > 10 and <37 were studied. Interventions used were intratracheal instillation of either calfactant or air placebo (1 or 2 doses). Forty-three subjects were enrolled between November 2010 and June 2015: 26 assigned to calfactant and 17 to placebo. There were no significant differences in the primary outcome, which was survival to PICU discharge (adjusted hazard ratio of mortality for calfactant versus placebo, 1.78; 95% confidence interval,.53 to 6.05; P =.35), OI, functional outcomes, or ventilator-free days, adjusting for risk strata and Pediatric Risk of Mortality (PRISM) score. Despite the risk-stratified randomization, more allogeneic HCT patients received calfactant (76% and 39%, respectively) due to low recruitment at various sites. This imbalance is important because independent of treatment arm and while adjusting for PRISM score, those with allogeneic HCT had a nonsignificant higher likelihood of death at PICU discharge (adjusted odds ratio, 3.02; 95% confidence interval,.76 to 12.06; P =.12). Overall, 86% of the patients who survived to PICU discharge also were successfully discharged from the hospital. These data do not support the use of calfactant among this high mortality group of pediatric leukemia/lymphoma and/or HCT patients with PARDS to increase survival. In spite of poor enrollment, allogeneic HCT patients with PARDS appeared to be characterized by higher mortality than even other high-risk immunosuppressed groups. Conducting research among these children is challenging but necessary, because survival to PICU discharge usually results in successful discharge to home.

Original languageEnglish (US)
Pages (from-to)2479-2486
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Volume24
Issue number12
DOIs
StatePublished - Dec 2018

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Acute Lung Injury
Cell Transplantation
Pediatric Intensive Care Units
Stem Cells
Randomized Controlled Trials
Pediatrics
Adult Respiratory Distress Syndrome
Placebos
Mortality
Lymphoma
Leukemia
Survival
Confidence Intervals
Child Mortality
Tertiary Healthcare
Mechanical Ventilators
Random Allocation
Artificial Respiration
Lung Diseases
calfactant

All Science Journal Classification (ASJC) codes

  • Hematology
  • Transplantation

Cite this

for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network (2018). CALIPSO: A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients. Biology of Blood and Marrow Transplantation, 24(12), 2479-2486. https://doi.org/10.1016/j.bbmt.2018.07.023
for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network. / CALIPSO : A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients. In: Biology of Blood and Marrow Transplantation. 2018 ; Vol. 24, No. 12. pp. 2479-2486.
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title = "CALIPSO: A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients",
abstract = "To assess if calfactant reduces mortality among children with leukemia/lymphoma or after hematopoietic cell transplantation (HCT) with pediatric acute respiratory distress syndrome (PARDS), we conducted a multicenter, randomized, placebo-controlled, double-blinded trial in 17 pediatric intensive care units (PICUs) of tertiary care children's hospitals. Patients ages 18 months to 25 years with leukemia/lymphoma or having undergone HCT who required invasive mechanical ventilation for bilateral lung disease with an oxygenation index (OI) > 10 and <37 were studied. Interventions used were intratracheal instillation of either calfactant or air placebo (1 or 2 doses). Forty-three subjects were enrolled between November 2010 and June 2015: 26 assigned to calfactant and 17 to placebo. There were no significant differences in the primary outcome, which was survival to PICU discharge (adjusted hazard ratio of mortality for calfactant versus placebo, 1.78; 95{\%} confidence interval,.53 to 6.05; P =.35), OI, functional outcomes, or ventilator-free days, adjusting for risk strata and Pediatric Risk of Mortality (PRISM) score. Despite the risk-stratified randomization, more allogeneic HCT patients received calfactant (76{\%} and 39{\%}, respectively) due to low recruitment at various sites. This imbalance is important because independent of treatment arm and while adjusting for PRISM score, those with allogeneic HCT had a nonsignificant higher likelihood of death at PICU discharge (adjusted odds ratio, 3.02; 95{\%} confidence interval,.76 to 12.06; P =.12). Overall, 86{\%} of the patients who survived to PICU discharge also were successfully discharged from the hospital. These data do not support the use of calfactant among this high mortality group of pediatric leukemia/lymphoma and/or HCT patients with PARDS to increase survival. In spite of poor enrollment, allogeneic HCT patients with PARDS appeared to be characterized by higher mortality than even other high-risk immunosuppressed groups. Conducting research among these children is challenging but necessary, because survival to PICU discharge usually results in successful discharge to home.",
author = "{for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network} and Thomas, {Neal J.} and Debbie Spear and Emily Wasserman and Steven Pon and Barry Markovitz and Singh, {Aalok R.} and Simon Li and Gertz, {Shira J.} and Rowan, {Courtney M.} and Allen Kunselman and Tamburro, {Robert F.}",
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for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network 2018, 'CALIPSO: A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients', Biology of Blood and Marrow Transplantation, vol. 24, no. 12, pp. 2479-2486. https://doi.org/10.1016/j.bbmt.2018.07.023

CALIPSO : A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients. / for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network.

In: Biology of Blood and Marrow Transplantation, Vol. 24, No. 12, 12.2018, p. 2479-2486.

Research output: Contribution to journalArticle

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T1 - CALIPSO

T2 - A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients

AU - for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network

AU - Thomas, Neal J.

AU - Spear, Debbie

AU - Wasserman, Emily

AU - Pon, Steven

AU - Markovitz, Barry

AU - Singh, Aalok R.

AU - Li, Simon

AU - Gertz, Shira J.

AU - Rowan, Courtney M.

AU - Kunselman, Allen

AU - Tamburro, Robert F.

PY - 2018/12

Y1 - 2018/12

N2 - To assess if calfactant reduces mortality among children with leukemia/lymphoma or after hematopoietic cell transplantation (HCT) with pediatric acute respiratory distress syndrome (PARDS), we conducted a multicenter, randomized, placebo-controlled, double-blinded trial in 17 pediatric intensive care units (PICUs) of tertiary care children's hospitals. Patients ages 18 months to 25 years with leukemia/lymphoma or having undergone HCT who required invasive mechanical ventilation for bilateral lung disease with an oxygenation index (OI) > 10 and <37 were studied. Interventions used were intratracheal instillation of either calfactant or air placebo (1 or 2 doses). Forty-three subjects were enrolled between November 2010 and June 2015: 26 assigned to calfactant and 17 to placebo. There were no significant differences in the primary outcome, which was survival to PICU discharge (adjusted hazard ratio of mortality for calfactant versus placebo, 1.78; 95% confidence interval,.53 to 6.05; P =.35), OI, functional outcomes, or ventilator-free days, adjusting for risk strata and Pediatric Risk of Mortality (PRISM) score. Despite the risk-stratified randomization, more allogeneic HCT patients received calfactant (76% and 39%, respectively) due to low recruitment at various sites. This imbalance is important because independent of treatment arm and while adjusting for PRISM score, those with allogeneic HCT had a nonsignificant higher likelihood of death at PICU discharge (adjusted odds ratio, 3.02; 95% confidence interval,.76 to 12.06; P =.12). Overall, 86% of the patients who survived to PICU discharge also were successfully discharged from the hospital. These data do not support the use of calfactant among this high mortality group of pediatric leukemia/lymphoma and/or HCT patients with PARDS to increase survival. In spite of poor enrollment, allogeneic HCT patients with PARDS appeared to be characterized by higher mortality than even other high-risk immunosuppressed groups. Conducting research among these children is challenging but necessary, because survival to PICU discharge usually results in successful discharge to home.

AB - To assess if calfactant reduces mortality among children with leukemia/lymphoma or after hematopoietic cell transplantation (HCT) with pediatric acute respiratory distress syndrome (PARDS), we conducted a multicenter, randomized, placebo-controlled, double-blinded trial in 17 pediatric intensive care units (PICUs) of tertiary care children's hospitals. Patients ages 18 months to 25 years with leukemia/lymphoma or having undergone HCT who required invasive mechanical ventilation for bilateral lung disease with an oxygenation index (OI) > 10 and <37 were studied. Interventions used were intratracheal instillation of either calfactant or air placebo (1 or 2 doses). Forty-three subjects were enrolled between November 2010 and June 2015: 26 assigned to calfactant and 17 to placebo. There were no significant differences in the primary outcome, which was survival to PICU discharge (adjusted hazard ratio of mortality for calfactant versus placebo, 1.78; 95% confidence interval,.53 to 6.05; P =.35), OI, functional outcomes, or ventilator-free days, adjusting for risk strata and Pediatric Risk of Mortality (PRISM) score. Despite the risk-stratified randomization, more allogeneic HCT patients received calfactant (76% and 39%, respectively) due to low recruitment at various sites. This imbalance is important because independent of treatment arm and while adjusting for PRISM score, those with allogeneic HCT had a nonsignificant higher likelihood of death at PICU discharge (adjusted odds ratio, 3.02; 95% confidence interval,.76 to 12.06; P =.12). Overall, 86% of the patients who survived to PICU discharge also were successfully discharged from the hospital. These data do not support the use of calfactant among this high mortality group of pediatric leukemia/lymphoma and/or HCT patients with PARDS to increase survival. In spite of poor enrollment, allogeneic HCT patients with PARDS appeared to be characterized by higher mortality than even other high-risk immunosuppressed groups. Conducting research among these children is challenging but necessary, because survival to PICU discharge usually results in successful discharge to home.

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for the CALIPSO Study Investigators and the Pediatric Acute Lung Injury and Sepsis Investigators Network. CALIPSO: A Randomized Controlled Trial of Calfactant for Acute Lung Injury in Pediatric Stem Cell and Oncology Patients. Biology of Blood and Marrow Transplantation. 2018 Dec;24(12):2479-2486. https://doi.org/10.1016/j.bbmt.2018.07.023