Not provided
Not provided
Not provided
Not provided
Not provided
Low enrollment
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Bi-level Positive Airway Pressure Device | Experimental | BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. |
|
| Sham CPAP | Sham Comparator | Physiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Bi-level positive airway pressure device | Device | BiPAP initiated for at least 16 hours per day for a minimum of 48hrs. |
|
| 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. |
| 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. |
Not provided
Inclusion Criteria:
Must meet clinical criteria for ACS- an infiltrate on Chest X-ray and one of the following:
Patients' eligible for a simple transfusion based on one of the following criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Deepa Manwani, MD | Albert Einstein College of Medicine | Principal Investigator |
| Michael E Roth, MD | Albert Einstein College of Medicine | Principal Investigator |
| Kerry Morrone, MD | Albert Einstein College of Medicine | Study Director |
| Hiren Muzumdar, MD | Albert Einstein College of Medicine | Principal Investigator |
| Ranaan Arens, MD | Albert Einstein College of Medicine | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital @ Montefiore | The Bronx | New York | 10467 | United States |
Not provided
Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| BG001 | Sham CPAP |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | From diagnosis of ACS until meeting discharge criteria- Average 7 days. |
|
Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.
Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Kerry Morrone, MD, Asst Prof of Pediatrics | Children's Hospital at Montefiore | 718-741-2342 | kmorrone@montefiore.org |
Not provided
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| D056586 | Acute Chest Syndrome |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Sham CPAP | Device | Sham CPAP initiated for at least 16 hours per day for a minimum of 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 |
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. |
| BG002 | Total | Total of all reporting groups |
|
| Age, Continuous |
| Sex: Female, Male |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. |
| Region of Enrollment | participants |
|
| 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. |
|
| Secondary | Rate of Exchange Transfusions. | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | Diagnosis until discharge. Average 7 days. |
|
|
| Secondary | Determine Parent and Patient Acceptability of BLPAP Administration in the Setting of ACS. | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | Upon completion of intervention at 48hrs. |
|
|
| Secondary | Rate of PCCU Transfers. | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | Diagnosis until discharge. Average 7 days. |
|
|
| Secondary | Difference in Respiratory Rate. | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | 48hrs after initiation of treatment |
|
|
| Secondary | Difference in Pulmonary Function Tests. | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | 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). | Study was terminated due to low enrollment. Only three participants were enrolled and none initiated treatment. | Posted | 48hrs after initiation of treatment |
|
|
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| 0 |
| EG001 | 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 | 0 | 0 | 0 | 0 | 0 |
Not provided
Not provided
Not provided
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D012120 | Respiration Disorders |