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

Status

Terminated

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01589926

Enrollment

Registered

2012-05-02

Start date

2012-07-31

Completion date

2016-09-08

Last updated

2018-12-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Sickle Cell Anemia, Acute Chest Syndrome

Brief 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.

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.

Interventions

DEVICEBi-level positive airway pressure device

BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.

DEVICESham CPAP

Sham CPAP initiated for at least 16 hours per day for a minimum of 48hrs.

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

SUPPORTIVE_CARE

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

4 Years to 21 Years

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.	From diagnosis of ACS until meeting discharge criteria- Average 7 days.	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.

Secondary

Measure	Time frame	Description
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.	—
Rate of Exchange Transfusions.	Diagnosis until discharge. Average 7 days.	—
Difference in Pulmonary Function Tests.	48hrs after initiation of treatment	—
Difference in Mean SpO2 Recording During Sleep.	48hrs after initiation of treatment	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).
Difference in Respiratory Rate.	48hrs after initiation of treatment	—

Countries

United States

Participant flow

Recruitment details

Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.

Participants by arm

Arm	Count
Bi-level Positive Airway Pressure BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. Bi-level positive airway pressure device: BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.	0
Sham CPAP Physiologic CPAP 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.	0
Total	0

Baseline characteristics

Characteristic	—
Region of Enrollment United States	— participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	0 / 0	0 / 0
other Total, other adverse events	0 / 0	0 / 0
serious Total, serious adverse events	0 / 0	0 / 0

Outcome results

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.

Time frame: From diagnosis of ACS until meeting discharge criteria- Average 7 days.