Natural History of Sickle Cell Disease and Other Hemolytic Disorders - Full Text View

Sponsored by:	National Heart, Lung, and Blood Institute (NHLBI)
Information provided by:	National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier:	NCT00081523

Purpose

Under this study, NIH physicians will evaluate and treat patients with sickle cell disease and other hemolytic disorders (diseases involving damage to red blood cells) in order to learn more about the diseases and the painful attacks and lung complications that are often associated with them. Patients in this study will not receive experimental therapy, but will be given standard medical care. Patients who meet the medical criteria for another NIH protocol may be offered participation in that study, but are not obligated to enroll in it.

Patients 5 years of age and older with known or suspected sickle cell disease, sickle cell trait or another anemia or red blood cell disorder such as thalassemia may be eligible for this study. Participants will be admitted to the NIH Clinical Center as an inpatient or outpatient for evaluation and treatment. In addition to a physical examination, evaluation may include the following types of tests and procedures:

Routine blood and urine tests, including a 24-hour urine collection
Blood test for genetic analysis to look for genes that may be relevant to the development of sickle cell anemia or other hemolytic disorders
X-rays, if needed, to evaluate the patient's condition
Computerized tomography (CT) scans or magnetic resonance imaging (MRI), or both, as needed to examine the lungs, brain, or heart and to measure blood flow in these organs.
Echocardiogram (heart ultrasound) to determine if there is high blood pressure in the lungs
Pulmonary (lung) function tests and cardiopulmonary (heart and lung) exercise test to evaluate breathing and exercise capacity to determine if there are lung or heart problems associated with the patient's condition.
Heart catheterization to measure blood pressures in the heart and lung blood vessels will be offered if the results of clinical evaluation or echocardiogram suggest the presence of high blood pressure in the lungs. For this procedure, the patient is given a local anesthetic, and a catheter (plastic tube) is inserted into a vein in the arm, thigh, chest, or neck until it passes through the right side of the heart. When the catheter is in place, a tiny balloon at the end of the catheter is inflated, temporarily blocking the blood flow and allowing the physician to measure pressure in the lung artery system.
Ventilation-perfusion scan to measure breathing and circulation in all areas of the lungs. For the perfusion scan, radioactive a...

Condition
Hemoglobin SC Disease Hematologic Diseases Sickle Cell Anemia Hemolytic Anemia

Genetics Home Reference related topics: beta thalassemia paroxysmal nocturnal hemoglobinuria sickle cell disease

MedlinePlus related topics: Anemia High Blood Pressure Sickle Cell Anemia Thalassemia

Drug Information available for: Nitric oxide

U.S. FDA Resources

Study Type:	Observational
Official Title:	Studies of the Natural History of Sickle Cell Disease and Other Hemolytic Disorders

Further study details as provided by National Institutes of Health Clinical Center (CC):

Study Start Date:

April 2004

Detailed Description:

This protocol will permit the evaluation and treatment of subjects with hereditary and acquired hemolytic diseases, to facilitate understanding of the pathogenesis and natural history of vaso-occlusive painful crisis and pulmonary complications of sickle cell disease and related cardiopulmonary complications of other hereditary, acquired and iatrogenic hemolytic disorders. Patients will be evaluated with a medical history and physical examination and routine laboratory studies will be obtained as needed to assess diagnosis, disease activity, and disease complications and to monitor for treatment-related responses and toxicities. Blood can be obtained, with subject's consent, for research studies evaluating gene/protein expression and to evaluate the role of vasodialators, vasoconstrictors, inflammatory and redox stress mediators in this population. Patients identified with pulmonary hypertension will have the option to undergo invasive hemodynamic evaluation and treatment with FDA approved drugs, according to current standards of medical practice, with signed informed consent for all offered procedures. Patients eligible for other research protocols will be offered an opportunity to participate in theses studies by signed informed consent. Apart from such protocols, any medical care recommended or provided to the patient will be consistent with routine standards of practice and will be provided in consultation with the patient's referring physician. The establishment of this protocol will provide a means to evaluate and treat patients, and to generate hypotheses and protocols based on this clinical experience in sickle cell disease and in other acquired or inherited hemolytic anemias.

Eligibility

Ages Eligible for Study:	5 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

INCLUSION CRITERIA:

Patients with sickle cell disease and other hemolytic disorders will be evaluated at the NIH CCMD/NIDDK sickle cell clinic or day hospital. Patients will not be recruited or selected with reference to age, race or gender. The patient population is expected, however, to be predominantly minority in composition (mainly African-American and Latino, with some Asian, European and Native American patients as well) as function of demographics and genetics of these diseases. Children may be evaluated or treated in this protocol with parental informed consent and assent of the child when appropriate.

Known or suspected sickle cell disease and other hereditary hemolytic disorders such as thalassemia, hereditary sphyerocytosis.
Immune or other acquired hemolytic states, such as paroxysmal nocturnal hemoglobinuria.
Iatrogenic hemolytic states (e.g. post-cardiopulmonary bypass or post-transfusion).
Age from 5 years old to death (all ages).
Willingness and capacity to provide informed consent or appropriate informed consent from parent or legal guardian.

EXCLUSION CRITERIA:

-Any concurrent condition (medical, social, behavioral) which in the opinion of the research team would preclude or confound acquisition/interpretation of data or delivery of clinical care.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00081523

Contacts

Contact: Patient Recruitment and Public Liaison Office	(800) 411-1222	prpl@mail.cc.nih.gov
Contact: TTY	1-866-411-1010

Locations

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike	Recruiting
Bethesda, Maryland, United States, 20892
Suburban Hospital	Recruiting
Bethesda, Maryland, United States, 20814

Sponsors and Collaborators

National Heart, Lung, and Blood Institute (NHLBI)

More Information

NIH Clinical Center Detailed Web Page This link exits the ClinicalTrials.gov site

Publications:

Gaston MH, Verter JI, Woods G, Pegelow C, Kelleher J, Presbury G, Zarkowsky H, Vichinsky E, Iyer R, Lobel JS, et al. Prophylaxis with oral penicillin in children with sickle cell anemia. A randomized trial. N Engl J Med. 1986 Jun 19;314(25):1593-9.

Platt OS, Brambilla DJ, Rosse WF, Milner PF, Castro O, Steinberg MH, Klug PP. Mortality in sickle cell disease. Life expectancy and risk factors for early death. N Engl J Med. 1994 Jun 9;330(23):1639-44.

[No authors listed] From the Centers for Disease Control and Prevention. Mortality among children with sickle cell disease identified by newborn screening during 1990-1994--California, Illinois, and New York. JAMA. 1998 Apr 8;279(14):1059-60. No abstract available.

Study ID Numbers:	040161, 04-H-0161
Study First Received:	April 15, 2004
Last Updated:	July 18, 2008
ClinicalTrials.gov Identifier:	NCT00081523
Health Authority:	United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):

Acute Chest Syndrome
Hemoglobin
Nitric Oxide
Pulmonary Hypertension
Pain Crisis
Treatment Options

Sickle Cell Disease
Sickle Cell Trait
Red Blood Cell Disorder
Thalassemia
Paroxysmal Nocturnal Hemoglobinuria
Hemolytic Disorders

Study placed in the following topic categories:

Paroxysmal nocturnal hemoglobinuria
Hemoglobin SC disease
Hematologic Diseases
Hemoglobin SC Disease
Anemia
Anemia, Hemolytic
Pain
Thalassemia
Sickle cell anemia
Nitric Oxide

Hemoglobinuria
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hypertension, Pulmonary
Marchiafava-Micheli disease
Hemoglobinopathies
Sickle Cell Trait
Hemoglobinopathy
Anemia, Sickle Cell
Hypertension

ClinicalTrials.gov processed this record on January 16, 2009