Clinical Trial: Long Term Effects of Erythrocyte Lysis

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Long Term Effects of Erythrocyte Lysis

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.

Detailed Summary:

  1. 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)
  2. 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:

  1. 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.
  2. 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).
  3. To estimate the prevalence of elevated TRV in adults with non-sickling hemolytic anemia, with or without splenic function.
  4. To investigate the association between TRV and splenic function
  5. 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.)
  6. To evaluate genetic determinants of elevated TRV in children and adults with hemolytic anemia.
  7. Sponsor: St. Jude Children's Research Hospital

    Current Primary Outcome: 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 ]

    Original Primary Outcome: 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 HbSS. [ Time Frame: 2 years ]

    Current Secondary Outcome:

    Original Secondary Outcome:

    Information By: St. Jude Children's Research Hospital

    Dates:
    Date Received: February 11, 2009
    Date Started: March 2009
    Date Completion:
    Last Updated: June 24, 2016
    Last Verified: June 2016