Long Term Effects of Erythrocyte Lysis

NCT00842621 | Completed

• 1 other identifier: ELYSIS
• Study Type: observational
• Target: 390
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Sickle Cell Disease; Hemolytic Anemia

Keywords

Sickle Cell Disease, Hemolytic Anemia; Hemolysis

Outcome Measures

Primary Outcomes (1)

• 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.

  2 years

Study Arms (3)

1

Pediatric participants with severe sickle cell disease (HbSS or Hb S/β°-thalassemia) who are not receiving treatment, e.g., hydroxyurea or chronic transfusions

Procedure: Clinical Evaluations; Other: Laboratory Studies

2

Pediatric participants with other forms of SCD or severe sickle cell disease patients (HbSS or Hb S/β°-thalassemia) being treated with hydroxyurea or chronic transfusions

Procedure: Clinical Evaluations; Other: Laboratory Studies

3

Pediatric and adult participants with other non-sickling hematological disorders

Procedure: Clinical Evaluations; Other: Laboratory Studies

Interventions

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.

1; 2; 3

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

1; 2; 3

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Children aged 5 years of age up to 19th birthday with sickle cell disease or other forms of hemolytic anemia; Adults over the age of 18 with non-sickling hemolytic anemia.

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38115, United States

Location

Related Publications (2)

• Rai P, Okhomina VI, Kang G, Martinez HR, Hankins JS, Joshi V. Longitudinal effect of disease-modifying therapy on left ventricular diastolic function in children with sickle cell anemia. Am J Hematol. 2023 Jun;98(6):838-847. doi: 10.1002/ajh.26911. Epub 2023 Mar 20.

  PMID: 36890729 DERIVED

• Whipple NS, Naik RJ, Kang G, Moen J, Govindaswamy SD, Fowler JA, Dowdy J, Penkert R, Joshi VM, Hankins JS. Ventricular global longitudinal strain is altered in children with sickle cell disease. Br J Haematol. 2018 Dec;183(5):796-806. doi: 10.1111/bjh.15607. Epub 2018 Nov 19.

  PMID: 30450553 DERIVED

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Biospecimen

Retention: SAMPLES WITH DNA

Blood Serum Plasma Urine

MeSH Terms

Conditions

Anemia, Sickle Cell; Anemia, Hemolytic; Hemolysis

Condition Hierarchy (Ancestors)

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

Study Officials

• Jane Hankins, MD, MS

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2009
• First Posted: February 12, 2009
• Study Start: March 1, 2009
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2016
• Last Updated: June 27, 2016

Record last verified: 2016-06

Locations

US (1)