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

Study Overview
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Brief Summary
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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
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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.

Official Title
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Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial

Conditions
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Acute Chest Syndrome, Sickle Cell Disease

Intervention / Treatment
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* Drug: Nitric oxide by inhalation INOMAX
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1: INOMAX<br>Nitric oxide by inhalation INOMAX: active arm treated with nitric oxide | Drug: Nitric oxide by inhalation INOMAX<br>* Nitric oxide by inhalation INOMAX 800 ppm 40 ppm during 24 hours then 20 ppm during 24 hours then 10 ppm during 24 hours<br>* Other names: INOMAX;|
| Placebo Comparator: 2: Placebo<br>placebo arm treated with placebo at the same conditions | Drug: Placebo<br>* placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Nitric oxide therapy |  | after 7 to 10 days | 
| Duration of OXYGENOTHERAPY |  | 7 to 10 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Nitric oxide