Protocol Number: 01-H-0223

Title:

Inhaled Nitric Oxide and Transfusion Therapy for Patients with Sickle Cell Anemia and Secondary Pulmonary Hypertension

Number:

01-H-0223

Summary:

This study will test whether inhaling nitric oxide (NO) gas mixed with room air can improve pulmonary hypertension (high blood pressure in the lungs) in patients with sickle cell anemia.

Patients with sickle cell disease 18 years of age or older may be eligible to participate in one or more parts of this three-stage study, as follows:

Stage 1

Patients undergo the following tests to determine the cause of their pulmonary hypertension: blood tests; echocardiogram (heart ultrasound); asthma test; oxygen breathing study with measurement of arterial blood oxygen levels; chest X-ray; lung scans; MRI of the heart; 6-minute walk test; night-time oxygen measurement while sleeping; and exercise studies.

Stage 2

Patients have a detailed MRI evaluation of the heart and are admitted to the NIH Clinical Center intensive care unit (ICU) for the following test: A plastic tube is placed in a vein in the patient's arm and another tube is placed in a deeper neck or leg vein. A third tube is inserted through the vein into the heart and the lung artery to measure blood pressures in the heart and lungs directly. Following baseline measurements, three medications (inhaled oxygen, infused prostaglandin, and inhaled NO) are delivered for 2 hours each, separated by a 30-minute washout period. A small blood sample is drawn during the NO administration.

Patients who cannot be treated with nitric oxide or for whom the treatment does not work may receive monthly exchange transfusions for 3 months. For this procedure, 3 to 5 five units of the patient's blood is removed and replaced with 3 to 5 units that do not have sickle hemoglobin. Some patients who do not respond to NO or exchange transfusions may receive an alternative therapy, such as oxygen, prostacyclin, L-arginine, bosentan or sidenafil.

Stage 3

Patients remain in the ICU with catheters in place for another 24 hours. During this time they breathe NO. Lung pressures are measured every 4 hours and blood is drawn every 8 hours. They then stay in the hospital 1 more day for observation. Patients then breathe nitric oxide continuously for 2 months using a tank of gas that delivers the NO through tubes placed in the nose. They may do this at home on an outpatient basis or may remain in the hospital for the 2 months.

Patients have an echocardiogram and blood tests every week and do a 6-minute walk test every 2 weeks.

Sponsoring Institute:

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Completed Study; data analyses ongoing

Gender: Male & Female

Referral Letter Required: No

Population Exclusion(s): Children

Eligibility Criteria: This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Special Instructions:

Currently Not Provided

Keyword(s):

Acute Chest Syndrome

Blood Flow

Nitric Oxide

NO Therapy

Vaso-Occlusive Crisis

Recruitment Keyword(s):

Sickle Cell Anemia

Sickle Cell

Acute Chest Syndrome

ACS

Pulmonary Hypertension

Condition(s):

Sickle Cell Anemia

Pulmonary Hypertension

Investigational Drug(s):

Nitric Oxide

Investigational Device(s):

INO Pulse - NO Delivery System

Interventions:

Drug: Nitric Oxide

Device: INO Pulse - NO Delivery System

Supporting Site:

National Heart, Lung and Blood Institute

Contact(s):

This study is not currently recruiting new subjects. If you have questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.

Citation(s):

Mortality in sickle cell disease Life expectancy and risk factors for early death

Acute chest syndrome in sickle cell disease: clinical presentation and course Cooperative Study of Sickle Cell Disease

The acute chest syndrome in sickle cell disease: incidence and risk factors The Cooperative Study of Sickle Cell Disease

• Questions about participating in a study, please contact the Patient Recruitment and Public Liaison Office, CC.
• Technical questions regarding the Clinical Center web site, please contact the Department Clinical Research Informatics, CC.

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