Bi-Level Positive Airway Ventilation for Acute Chest Syndrome

Sickle Cell Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01589926
• Other study ID #: 12-04-139
• Status: Terminated
• Phase: N/A
• Start date: July 2012
• Est. completion date: September 8, 2016

Study information

• Verified date: November 2018
• Source: Albert Einstein College of Medicine, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 3
• Est. completion date: September 8, 2016
• Est. primary completion date: September 8, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• Acute Chest Syndrome
• Anemia, Sickle Cell
• Sickle Cell Anemia
• Syndrome

Intervention

Device:
• Bi-level positive airway pressure device
BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.
• Sham CPAP
Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.

Locations

Country	Name	City	State
United States	Children's Hospital @ Montefiore	Bronx	New York

Sponsors (1)

Lead Sponsor	Collaborator
Albert Einstein College of Medicine, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Rate of Exchange Transfusions.		Diagnosis until discharge. Average 7 days.
Secondary	Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS.		Upon completion of intervention at 48hrs.
Secondary	Rate of PCCU Transfers.		Diagnosis until discharge. Average 7 days.
Secondary	Difference in Respiratory Rate.		48hrs after initiation of treatment
Secondary	Difference in Pulmonary Function Tests.		48hrs after initiation of treatment
Secondary	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