Long Term Effects of Erythrocyte Lysis
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Anemia, Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | 5 - Any |
| Updated: | 4/21/2016 |
| Start Date: | March 2009 |
| End Date: | June 2016 |
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.
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.
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. To investigate changes in TRV and hemolysis over time using serial measurements 2 ± 0.5
years after initial enrollment testing.
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. To investigate changes in TRV and hemolysis over time using serial measurements 2 ± 0.5
years after initial enrollment testing.
Inclusion Criteria:
1. 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)
2. 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:
1. Previous cardiac surgery
2. Known left ventricle dysfunction (i.e. shortening fraction < 28%)
3. Known right sided congenital heart defect such as atrial septal defect or pulmonary
valve stenosis
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