Bi-Level Positive Airway Ventilation for Acute Chest Syndrome

Early Bi-Level Positive Airway Pressure (BiPAP) Ventilation for Acute Chest Syndrome (ACS) - a Double-Blind Randomized Controlled Pilot Study

Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by having findings on a chest x-ray and one of the following: chest pain, fever, or trouble breathing. Patients with Acute Chest Syndrome can get very sick and require an exchange transfusion (special large blood transfusion) and mechanical ventilation. Bi-level Positive Airway Pressure (also known as BLPAP or BiPAP) is a device that blows air into a patients lungs via a mask that covers the nose. The goal of this study is to determine whether giving children BiPAP when they have ACS, in addition to providing standard clinical care for ACS, alters the clinical course of these patients. The investigators hypothesize that patients receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to the intensive care unit and exchange transfusions.

Study Overview

• Status: Terminated
• Conditions: Sickle Cell Anemia; Acute Chest Syndrome
• Intervention / Treatment: Device: Bi-level positive airway pressure device; Device: Sham CPAP

Detailed Description

Acute chest syndrome (ACS) is a frequent complication of sickle cell disease and is diagnosed by a new infiltrate on chest x-ray and one of the following: chest pain, fever, or respiratory signs or symptoms (tachypnea, cough, new onset hypoxemia, or increased work of breathing.)The treatment for acute chest syndrome is focused on supportive care with hydration, antibiotics, blood transfusions and respiratory support. Unfortunately, despite these treatments many patients fail to have improvements in their respiratory status, or have respiratory decompensation. These patients require more aggressive treatments, which frequently include exchange transfusions, pediatric intensive care unit (PCCU) management, and respiratory support.

The study objective is to perform a prospective double blind randomized control trial to investigate if early initiation of effective BiPAP in addition to providing standard clinical care for ACS alters the clinical course of these patients vs. sham BiPAP and standard clinical care. Investigators hypothesize that participants receiving effective BiPAP will have milder clinical courses resulting in shorter hospital stays and fewer transfers to PCCU and exchange transfusions.

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • New York
    • Bronx, New York, United States, 10467
      • Children's Hospital @ Montefiore

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• patients diagnosed with Hemoglobin SS (HB SS), the most common type of sickle cell disease
• patients diagnosed with Hemoglobin SC (HB SC), the second most common type of sickle cell disease.
• patients diagnosed with Hemoglobin sickle beta-zero thalassemia ( HB SB0thal) or Hemoglobin sickle thalassemia (HB SBthal)

Must meet clinical criteria for ACS- an infiltrate on Chest X-ray and one of the following:

• Respiratory symptoms/signs (patients pulse oximetry < 92% or oxygen saturation < 2% below their baseline, tachypnea, cough, and increased work of breathing)
• Fever
• Chest pain AND

Patients' eligible for a simple transfusion based on one of the following criteria:

• Hypoxemia (patients pulse oximetry < 92% or oxygen saturation < 2% below their baseline)
• Hemoglobin < 5 gm/dl
• Increased work of breathing

Exclusion Criteria:

• Patient requires exchange transfusion within first 24 hours of admission
• Patient requires PCCU transfer within first 24 hours of admission
• Hemoglobin > 9gm/dl secondary to these patients requiring an exchange transfusion

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Bi-level Positive Airway Pressure Device; BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	Device: Bi-level positive airway pressure device; BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.
Sham Comparator: Sham CPAP; Physiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs.	Device: Sham CPAP; Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.	Length of stay as measured by the time from initial diagnosis of ACS until meeting discharge criteria. It is anticipated length of stay will correlate to efficacy of treatment: shorter stay is theorized to indicate more efficient treatment.	From diagnosis of ACS until meeting discharge criteria- Average 7 days.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Exchange Transfusions.		Diagnosis until discharge. Average 7 days.
Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS.		Upon completion of intervention at 48hrs.
Rate of PCCU Transfers.		Diagnosis until discharge. Average 7 days.
Difference in Respiratory Rate.		48hrs after initiation of treatment
Difference in Pulmonary Function Tests.		48hrs after initiation of treatment
Difference in Mean SpO2 Recording During Sleep.	Peripheral capillary oxygen saturation (SpO2) is an estimate of the amount of oxygen in the blood. It is the percentage of haemoglobin containing oxygen compared to the total amount of haemoglobin in the blood (i.e. oxygenated haemoglobin vs oxygenated and non-oxygenated haemoglobin).	48hrs after initiation of treatment

Collaborators and Investigators

• Sponsor: Albert Einstein College of Medicine
• Investigators: Principal Investigator: Deepa Manwani, MD, Albert Einstein College Of Medicine; Principal Investigator: Michael E Roth, MD, Albert Einstein College Of Medicine; Study Director: Kerry Morrone, MD, Albert Einstein College Of Medicine; Principal Investigator: Hiren Muzumdar, MD, Albert Einstein College Of Medicine; Principal Investigator: Ranaan Arens, MD, Albert Einstein College Of Medicine

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2012
• Primary Completion (Actual): September 8, 2016
• Study Completion (Actual): September 8, 2016

Study Registration Dates

• First Submitted: April 30, 2012
• First Submitted That Met QC Criteria: May 1, 2012
• First Posted (Estimate): May 2, 2012

Study Record Updates

• Last Update Posted (Actual): December 11, 2018
• Last Update Submitted That Met QC Criteria: November 19, 2018
• Last Verified: November 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Respiration Disorders; Lung Diseases; Disease; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Syndrome; Anemia, Sickle Cell; Acute Chest Syndrome
• Other Study ID Numbers: 12-04-139

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes