Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.
Sickle cell disease (SCD) and thalassemia are genetic disorders inducing anemia of differing pathophysiology. A primary therapy for preventing certain SCD complications (e.g., stroke) and for thalassemia major is regular red blood cell (RBC) transfusion, coupled with iron chelation to prevent the complications of transfusion-induced iron overload. For patients with pediatric hematology-oncology diagnoses with chemotherapy-induced aplasia, RBC transfusion is also common, but the degree of transfusion-induced iron overload and its implications for these patients is incompletely understood. Because iron-related tissue toxicity is a major cause of morbidity and mortality in regularly transfused patients, developing strategies to minimize iron loading and iron toxicity is a key objective of this proposal (study Aim #2), stemming from the objective to optimize RBC unit characteristics that patients with SCD and thalassemia receive beyond RBC phenotype matching for Rh C, E and K antigens (study Aim #1). The study will enroll patients with SCD, thalassemia or pediatric oncologic diagnoses receiving eligible transfusion at 6 hospital sites in the United States, as well as patients with SCD at 5 hemocenters in Brazil.
Study Type
OBSERVATIONAL
Enrollment
157
Simple RBC transfusion or partial manual exchange
UCSF Benioff Children's Hospital
Oakland, California, United States
Vitalant Research Institute
San Francisco, California, United States
Change in Hemoglobin A or Hemoglobin Level per day (RBC Survival)
Change in hemoglobin A or hemoglobin level per day in between subsequent transfusion episodes, for sickle cell disease and thalassemia cohorts, respectively
Time frame: Baseline (immediately pre-) to post-transfusion over 2 years
Change in Serum Iron Level
For all groups participating, change in serum iron measured from immediately prior to 2 hours post-transfusion
Time frame: Baseline (immediately before) and 2-hours after transfusion
Hemoglobin Increment
Hemoglobin increment \[defined as Hb/HbA(post-transfusion)visit(i) - Hb/HbA(pre-transfusion)visit(i)\] is associated with "RBC survival"
Time frame: Baseline (immediately pre-) to post-transfusion, over 2 years
Hemolysis Parameter Increment
Includes serum iron, indirect bilirubin, or plasma free hemoglobin
Time frame: Baseline (immediately pre-) to post-transfusion or 2-hours post-transfusion, over 2 years
Hepcidin Level
Hepcidin level at time of transfusion is a predictor of change in iron parameters (i.e., transferrin saturation, serum iron) following transfusion
Time frame: Baseline (immediately before) to 2 hours after transfusion
Non-Transferrin-Bound Iron (NTBI) Level
NTBI levels in patients with pediatric oncologic diagnoses with aplasia are elevated at baseline and increase following transfusion
Time frame: Baseline (immediately before) to 2 hours after transfusion
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Boston Children's Hospital
Boston, Massachusetts, United States
Weill Cornell Medical Collection (WCMC)/New York Presbyterian Hospital (NYPH)
New York, New York, United States
Columbia University Irving Medical Center/New York Presbyterian Hospital (NYPH)
New York, New York, United States
New York Blood Center (NYBC)
New York, New York, United States
Children's Wisconsin
Milwaukee, Wisconsin, United States
Froedtert Hospital
Milwaukee, Wisconsin, United States
Versiti Wisconsin, Inc.
Milwaukee, Wisconsin, United States
HEMOAM - Amazonas
Manaus, Amazonas, Brazil
...and 4 more locations
Number of Clinical Complications
Increased NTBI, serum iron, or transferrin saturation following transfusion is associated with increased risk of clinical adverse effects (i.e., new infections, SCD complications)
Time frame: 2 years