Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children

Phase 2

Completed

Brief Summary: Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis.

Detailed Description: Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis in a prospective randomized double-blind placebo controlled study. 50 children in two years will be included: 25 in each arm.

Recruitment & Eligibility

Inclusion Criteria

child between 1 and 18 years old
Sickle cell anemia or equivalent (sickle beta 0 thalassemia)whoe weight is between 10kg and 65kg
presenting acute chest syndrome as defined a new radiological infiltrate with tachypnea, respiratory discomfort, cough, chest wall pain and fever more than 38.5°C
hypoxaemia with transcutaneous oxygen saturation equal or less than 92%
informed consent signed by parents and approved by the child able to express his consent
insured by the National social security system or by the universal medical insurance
previous medical physical examination

Exclusion Criteria

respiratory distress with hypoxaemia with transcutaneous oxygen saturation equal or less than 92% under more than 5l/min of oxygen or 40% oxygen inhaled, hypercapnia signs 'sweating, altered consciousness, paCO2 more than 60mmHg) with need of emergency exchange transfusion and/or tracheal intubation with mechanical ventilation
Isolated acute asthmatic crisis
stroke or priapism with emergency acute transfusion needed
acute anemia with hemoglobin drop of more than 20% as compared to steady state hemoglobin
chronic long term transfusion therapy
nitric oxyde hypersensitivity
patients with right-left extra-pulmonary cardiac shunt
patient previously included in the protocol
patient participating in another interventional protocol
pregnancy or breast feeding

Arm && Interventions

Group	Intervention	Description
2: Placebo	Placebo	placebo arm treated with placebo at the same conditions
1: INOMAX	Nitric oxide by inhalation INOMAX	Nitric oxide by inhalation INOMAX: active arm treated with nitric oxide

Primary Outcome Measures

Name	Time	Method
We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% )	Oxygenation improvement (transcutaneous O2 superior to 92%) after Gas inhalation will be evaluate 2hours after inclusion and therafter every 2 hours until 12 hours therapy and then every six hours for 3days and then once a day till hospital discharge

Secondary Outcome Measures

Name	Time	Method
Duration of Nitric oxide therapy	after 7 to 10 days
Number of blood transfusions and total transfused blood volume	7 to 10 days
Quantity of Pain-killer drugs required and particularly OPIOIDS	7 to 10 days
Duration of OXYGENOTHERAPY	7 to 10 days

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