A Pilot Study of Chronic Red Blood Cell Transfusion in Sickle Cell Disease-Associated Pulmonary Hypertension

NCT00850369 | Withdrawn Phase 2 DMC

• 3 other identifiers: R01-HL79915-1HL799151R01HL079915
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Pulmonary hypertension, a complication associated with an increased risk of death, is common in patients with sickle cell disease. Despite its frequency, there remains no standard treatment for this complication in patients with sickle cell disease. In this small study, the investigators will evaluate the effect of monthly transfusion of red blood cells to patients with sickle cell disease-associated pulmonary hypertension. The investigators speculate that by increasing the hemoglobin level and decreasing the amount of sickle red blood cells, these patients would experience improvements in their PHT.

Conditions

Pulmonary Hypertension; Sickle Cell Disease

Keywords

Pulmonary hypertension; Sickle cell disease

Outcome Measures

Primary Outcomes (2)

• Pulmonary artery systolic pressure (mm Hg)

  2 years

• Pulmonary vascular resistance (dyne.s.cm-5)

  2 years

Secondary Outcomes (5)

• Six-minute walk

  2 years

• Markers of thrombin generation (TAT complexes, F1.2, d-dimers)

  2 years

• Markers of platelet activation (soluble CD40 ligand, beta thromboglobulin, platelet factor

  2 years

• Nitric oxide metabolites

  2 years

• Quality of life

  2 years

Study Arms (1)

1

EXPERIMENTAL

All subjects wil receive monthly RBC transfusions for 6 months

Other: RBC transfusion

Interventions

RBC transfusion OTHER

Study subjects will receive monthly transfusions with 2 units of red blood cells

1

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• diagnosis of sickle cell anemia (HbSS) and HbSbeta0 thalassemia;
• male and female subjects between 18 and 65 years;
• documented PHT, but with pulmonary artery systolic pressures \>/= 45 mmHg (TR jet velocity of \>/= 3.0 m/s) on at least 2 separate visits at least 1 month apart;
• ability to give written informed consent to participate in the study; and
• in non-crisis steady state at time of enrollment

You may not qualify if:

• treatment with epoprostenol (flolan) or similar prostacyclin analog, bosentan or sildenafil (or similar phosphodiesterase 5 inhibitor)
• on chronic anticoagulation
• RBC transfusion in previous 90 days;
• use of hydroxyurea
• multiple red cell alloantibodies that will make transfusion unsafe;
• baseline ferritin level \> 1000 mg/dL
• pregnancy, and/or any condition which in the opinion of investigator might make the subject unsuitable for the study;
• patients with WHO functional class IV

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• Duke University: collaborator

MeSH Terms

Conditions

Hypertension, Pulmonary; Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Kenneth I Ataga, MD

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2009
• First Posted: February 25, 2009
• Study Start: February 1, 2005
• Primary Completion: May 1, 2011
• Study Completion: May 1, 2011
• Last Updated: July 29, 2013

Record last verified: 2013-07