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.
Brief Title
Bi-Level Positive Airway Ventilation for Acute Chest Syndrome
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.
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.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Anemia
Acute Chest Syndrome
Eligibility Criteria
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
21 Years
Minimum Age
4 Years
NCT Id
NCT01589926
Org Class
Other
Org Full Name
Albert Einstein College of Medicine
Org Study Id
12-04-139
Overall Status
Terminated
Phases
Not Applicable
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Early Bi-Level Positive Airway Pressure (BiPAP) Ventilation for Acute Chest Syndrome (ACS) - a Double-Blind Randomized Controlled Pilot Study
Primary Outcomes
Outcome Description
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.
Outcome Measure
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.
Outcome Time Frame
From diagnosis of ACS until meeting discharge criteria- Average 7 days.
Secondary Outcomes
Outcome Time Frame
Diagnosis until discharge. Average 7 days.
Outcome Measure
Rate of Exchange Transfusions.
Outcome Time Frame
Upon completion of intervention at 48hrs.
Outcome Measure
Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS.
Outcome Time Frame
Diagnosis until discharge. Average 7 days.
Outcome Measure
Rate of PCCU Transfers.
Outcome Time Frame
48hrs after initiation of treatment
Outcome Measure
Difference in Respiratory Rate.
Outcome Time Frame
48hrs after initiation of treatment
Outcome Measure
Difference in Pulmonary Function Tests.
Outcome Description
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).
Outcome Time Frame
48hrs after initiation of treatment
Outcome Measure
Difference in Mean SpO2 Recording During Sleep.
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
21
Minimum Age Number (converted to Years and rounded down)
4
Investigators
Investigator Type
Principal Investigator
Investigator Name
Deepa Manwani
Investigator Email
dmanwani@montefiore.org
Investigator Phone
718-741-2342