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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00850369
Recruitment Status : Withdrawn (Slow accrual onto the study)
First Posted : February 25, 2009
Last Update Posted : July 29, 2013
Duke University
Information provided by (Responsible Party):
University of North Carolina, Chapel Hill

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.

Condition or disease Intervention/treatment Phase
Pulmonary Hypertension Sickle Cell Disease Other: RBC transfusion Phase 2

Detailed Description:

As patients with sickle cell disease (SCD) age, recurrent vaso-occlusive episodes lead to progressive end-organ damage. Pulmonary hypertension (PHT) represents an example of such end-organ damage. Pulmonary hypertension, a common complication in patients with sickle cell disease (SCD), results in a shortened survival. The high mortality reported in SCD patients with PHT appears to occur particularly in those patients with moderate and severe elevations in their pulmonary artery pressure. The overall objective of this proposal is to evaluate the effect of chronic red blood cell transfusion on PHT in SCD. We hypothesize that by increasing the hemoglobin concentration and decreasing the amount of HbS, these patients would experience improvements in their PHT.

Thus, the specific aim of this clinical trial is to evaluate the effects of RBC transfusion on pulmonary hypertension in SCD, as well as the effect of chronic RBC transfusion on plasma markers of thrombin generation, platelet activation, and nitric oxide metabolites.

Study subjects will be transfused monthly for 6 months to investigate the safety and efficacy of RBC transfusion in SCD patients with PHT. All packed red blood cells will have extended antigen matching for C, D, E and Kell to minimize the risk of alloimmunization. Subjects will receive other routine treatments for SCD. Specific outcome variables will be evaluated at 1 month, 3 months, and 6 months. All study subjects will receive simple transfusion of packed red blood cell to achieve a post-transfusion hemoglobin (Hb) not greater than 10 g/dL. For those subjects who may have baseline hemoglobins in whom a post transfusion Hb would exceed 10 g/dL, they will require a limited exchange transfusion, i.e. phlebotomy of 1 unit of blood, followed by transfusion of 2 units of packed RBC. All study subjects will return for assessment of safety and/or efficacy measures every two weeks for the first month, and subsequently every four weeks till the completion of the study. Study subjects who experience a documented worsening of their disease (decreased SaO2, worsening 6-minute walk) on at least two consecutive follow up visits will be taken off the study. At the end of the study, subjects will have the option of continuing on chronic RBC transfusion.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of the Effects of Chronic Red Blood Cell Transfusion in Sickle Cell Disease On Pulmonary Hypertension in Patients With Sickle Cell Disease
Study Start Date : February 2005
Actual Primary Completion Date : May 2011
Actual Study Completion Date : May 2011

Arm Intervention/treatment
Experimental: 1
All subjects wil receive monthly RBC transfusions for 6 months
Other: RBC transfusion
Study subjects will receive monthly transfusions with 2 units of red blood cells

Primary Outcome Measures :
  1. Pulmonary artery systolic pressure (mm Hg) [ Time Frame: 2 years ]
  2. Pulmonary vascular resistance ( [ Time Frame: 2 years ]

Secondary Outcome Measures :
  1. Six-minute walk [ Time Frame: 2 years ]
  2. Markers of thrombin generation (TAT complexes, F1.2, d-dimers) [ Time Frame: 2 years ]
  3. Markers of platelet activation (soluble CD40 ligand, beta thromboglobulin, platelet factor [ Time Frame: 2 years ]
  4. Nitric oxide metabolites [ Time Frame: 2 years ]
  5. Quality of life [ Time Frame: 2 years ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. diagnosis of sickle cell anemia (HbSS) and HbSbeta0 thalassemia;
  2. male and female subjects between 18 and 65 years;
  3. 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;
  4. ability to give written informed consent to participate in the study; and
  5. in non-crisis steady state at time of enrollment

Exclusion Criteria:

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00850369

Sponsors and Collaborators
University of North Carolina, Chapel Hill
Duke University
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Principal Investigator: Kenneth I Ataga, MD University of North Carolina, Chapel Hill

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Responsible Party: University of North Carolina, Chapel Hill Identifier: NCT00850369    
Other Study ID Numbers: R01-HL79915-1
First Posted: February 25, 2009    Key Record Dates
Last Update Posted: July 29, 2013
Last Verified: July 2013
Keywords provided by University of North Carolina, Chapel Hill:
Pulmonary hypertension
Sickle cell disease
Additional relevant MeSH terms:
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Hypertension, Pulmonary
Anemia, Sickle Cell
Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn