Sickle Cell Disease, Thalassemia
Conditions
Keywords
iron, transfusion, red blood cells, sickle cell disease, thalassemia, blood donors
Brief summary
The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: * whether a similar effect (older stored blood is associated with worse outcomes) is seen in chronically transfused patients with hemoglobinopathies. This patient population will also allow the investigators to test whether iron- chelation therapy is beneficial in this setting. * whether washing or cryopreserving the red blood cells has any effect on this outcome. These findings may explain the immunomodulatory effects of older stored blood in patients and will help us develop safer transfusion products for patients.
Detailed description
Epidemiologic studies suggest that older stored blood is associated with worse outcomes in certain hospitalized patients. Storage of red cells is associated with a storage lesion and the survival of transfused red cells decreases with increasing storage time, thus older blood is associated with an increased acute delivery of hemoglobin-iron to the reticuloendothelial system. The investigators have preliminary data in healthy human volunteers suggesting that delivery of a significant iron load to the reticuloendothelial system from aged red cells leads to the elaboration of a potentially toxic form of iron known as non-transferrin-bound iron.
Interventions
PROCEDURETransfusion
A routine medical procedure to transfuse packed red blood cells.
BIOLOGICALBlood
Processing of RBC for transfusion include the following: * Fresh * Stored * Washed * Frozen (Cryopreserved)
PROCEDUREChelation therapy
(non-experimental) A medical procedure that involves the administration of chelating agents to remove heavy metals from the body.
Sponsors
National Heart, Lung, and Blood Institute (NHLBI)
Columbia University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
(Recipient): * specific, well-characterized hemoglobinopathy * chronic simple transfusion therapy (transfusion episodes \< 6 weeks apart in frequency) * chronic iron chelation therapy * not pregnant by self-report and not planning pregnancy * age \> 1 year old
Exclusion criteria
(Recipient): * clinically unstable * treatment for mental illness * imprisonment * institutionalization Inclusion Criteria (Donor): * 21-65 years of age * male weight \> 130 lbs, female weight \> 150 lbs * male height \> 5'1, female height \> 5'5 * hemoglobin \> 15.0 g/dL * reasonably certain of intention to stay in New York City metropolitan area for study duration * previously tolerated red blood cell donation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Non-transferrin-bound Iron Level | 2 hours after transfusion |
Countries
United States
Participant flow
Recruitment details
This is a research study carried out at Columbia University Medical Center. Participants with a blood disorder and currently receive blood transfusions as treatment will be transfused fresh, stored, washed or frozen blood according to regular schedule for up to 3 years. Volunteer dedicated donors will also be asked to provide consent.
Pre-assignment details
Enrolled participants (recipients) were never randomized or received transfusion on study because study closed due to poor enrollment.
Participants by arm
| Arm | Count |
|---|---|
| Recipients Participants with a blood disorder (either sickle cell disease or thalassemia) and currently receive blood transfusions as treatment - will be receiving the following transfusion according to regular schedule: a fresh blood, a stored blood, a washed blood, and a frozen blood transfusion. | 2 |
| Donors Volunteer dedicated donors will be recruited to donate blood for each transfusion events over the next 3 years. Each patient with a chronic illness such as sickle cell disease or thalassemia will have one dedicated donor. | 1 |
| Total | 3 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Study closed due to poor enrollment | 2 | 1 |
Baseline characteristics
| Characteristic | Recipients | Donors | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 2 Participants | 0 Participants | 2 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 1 Participants | 1 Participants |
| Region of Enrollment United States | 2 participants | 1 participants | 3 participants |
| Sex: Female, Male Female | 0 Participants | 1 Participants | 1 Participants |
| Sex: Female, Male Male | 2 Participants | 0 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 2 | 0 / 1 |
| serious Total, serious adverse events | 0 / 2 | 0 / 1 |
Outcome results
Primary
Non-transferrin-bound Iron Level
Time frame: 2 hours after transfusion
Population: Enrolled participants (recipients) were never randomized or received transfusion on study because study closed due to poor enrollment. No data was collected or analyzed.