Early Bi-Level Positive Airway Pressure (BiPAP) Ventilation for Acute Chest Syndrome (ACS) - a Double-Blind Randomized Controlled Pilot Study
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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)
- •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
排除标准
- •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
研究组 & 干预措施
Bi-level Positive Airway Pressure Device
BiPAP initiated for at least 16 hours per day for a minimum of 48hrs.
干预措施: Bi-level positive airway pressure device (Device)
Sham CPAP
Physiologic continuous positive airway pressure (CPAP) initiated for at least 16 hours per day for a minimum of 48hrs.
干预措施: Sham CPAP (Device)
结局指标
主要结局
Length of Stay as Measured by the Time From Initial Diagnosis of ACS Until Meeting Discharge Criteria.
时间窗: 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.
次要结局
- 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.)
- Difference in Pulmonary Function Tests.(48hrs after initiation of treatment)
- Difference in Respiratory Rate.(48hrs after initiation of treatment)
- Rate of Exchange Transfusions.(Diagnosis until discharge. Average 7 days.)
- Difference in Mean SpO2 Recording During Sleep.(48hrs after initiation of treatment)
研究者
Deepa Manwani
Professor, Pediatrics
Albert Einstein College of Medicine