Completed

NCT Number: NCT00842621

Long Term Effects of Erythrocyte Lysis

In this prospective observational trial, participants with chronic hemolysis will be assessed with echocardiogram for elevated tricuspid jet velocity and other evidence of pulmonary hypertension. Participants will have laboratory studies evaluating: severity of hemolysis, splenic function, inflammation, endothelial dysfunction, and hypercoagulability. There will be 3 main categories of participants enrolled in this study: (1) pediatric participants with severe sickle cell disease (SCD) (HbSS, HbS/β° thalassemia ) who are not receiving treatment (e.g., hydroxyurea or chronic transfusions); (2) pediatric participants with other forms of SCD or severe SCD (HbSS, HbS/β° thalassemia) patients being treated with hydroxyurea or chronic transfusions; and (3) pediatric and adult participants with other non-sickling hematological disorders.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Hemolysis Hemolytic Anemia Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

5 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38115, United States

About this study

The study will investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia)
The Study will estimate the prevalence of elevated TRV (≥ 2.5 m/s) in untreated children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia), as measured by echocardiography.

Secondary objectives for this study include the following:

To estimate the prevalence of elevated TRV in children with severe sickle cell disease (HbSS or Hb S/β°-thalassemia) receiving hydroxyurea or chronic transfusion therapy.
To estimate the prevalence of elevated TRV in children with other forms of hemolytic anemia, including other sickling disorders (such as HbSC or HbS/β+-thalassemia) and non-sickling hemolytic anemia (such as hereditary spherocytosis).
To estimate the prevalence of elevated TRV in adults with non-sickling hemolytic anemia, with or without splenic function.
To investigate the association between TRV and splenic function
To investigate the associations between TRV and laboratory parameters of inflammation and hypercoagulability, such as white blood cell count, platelet count, serum N-terminal pro-brain natriuretic peptide (NT-proBNP),endothelial dysfunction, and other markers of hemolysis (bilirubin, plasma free hemoglobin, haptoglobin, etc.)
To evaluate genetic determinants of elevated TRV in children and adults with hemolytic anemia.
To investigate changes in TRV and hemolysis over time using serial measurements 2 ± 0.5 years after initial enrollment testing.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Established Diagnosis of Hemolysis

Sickle Cell Disease (e.g., HbSS, HbS/β-thalassemia, HbSC)
Other conditions with hemolysis (e.g., RBC membranopathies, enzymopathies, unstable hemoglobinopathies, PNH)
Age

SCD participants: 5 years of age up to 19th birthday
All other participants: 5 years of age and up (no age limit)

Exclusion criteria

Previous cardiac surgery
Known left ventricle dysfunction (i.e. shortening fraction < 28%)
Known right sided congenital heart defect such as atrial septal defect or pulmonary valve stenosis

Treatment and study plan

Clinical Evaluations

Procedure

All clinical evaluations should be performed greater than 30 days from illness or hospitalization. Participants will have weight, height, body mass index (BMI), heart rate, respiratory rate, blood pressure (systolic and diastolic) and pulse oximetry recorded while at rest on room air by clinical staff. Cardiac examination will be performed by echocardiogram.

Laboratory Studies

Other

Blood for:

Complete Blood Count with Differential, Reticulocyte Count, Micronuclei enumeration, α-globin genotype, G6PD activity, genotype, SNP array,

Serum for:

Soluble VCAM-1, ICAM-1, Soluble CD40L, Arginase, Endothelin-1, Prothrombin Fragment 1+2, IL-6, -8, -10, Soluble E-selectin, NOx, Haptoglobin, Tumor necrosis factor alpha

Plasma for:

Chemistry 18, Cystatin-C, Ferritin, Lactate Dehydrogenase, C-reactive protein, Erythropoietin, NT-proBNP, Haptoglobin, Von Willibrand Factor Antigen, D-dimer, Factor VIII Assay, Quantitative amino acids, Free hemoglobin, Tumor necrosis factor- α, Thrombin-Antithrombin complex, endothelin-1

Urine for:

Urinalysis, Urine spot protein, creatinine, Microalbumin, Urine hemosiderin, Urine hepcidin

Primary outcomes

1.To investigate the relationship between tricuspid regurgitation jet velocity (TRV) and intravascular hemolysis, as measured by serum lactate dehydrogenase (LDH), in untreated children with severe sickle cell disease(HbSS or Hb S/β°-thalassemia.

Time frame: 2 years

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Important dates

Study start: 2009
Primary completion: 2016
Study completion: 2016
First posted: Feb 12, 2009
Registry last updated: Jun 27, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed