Bi-Level Positive Airway Ventilation for Acute Chest Syndrome

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

NA

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-07-31

Study Completion Date

2016-09-08

Brief Summary

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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

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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.

Conditions

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Sickle Cell Anemia Acute Chest Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Bi-level Positive Airway Pressure Device

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

Group Type EXPERIMENTAL

Bi-level positive airway pressure device

Intervention Type DEVICE

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

Sham CPAP

Physiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs.

Group Type SHAM_COMPARATOR

Sham CPAP

Intervention Type DEVICE

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

Interventions

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Bi-level positive airway pressure device

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

Intervention Type DEVICE

Sham CPAP

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

Intervention Type DEVICE

Eligibility Criteria

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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

Minimum Eligible Age

4 Years

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Deepa Manwani

Professor, Pediatrics

Responsibility Role PRINCIPAL_INVESTIGATOR