Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
- Conditions
- ThalassemiaSickle Cell DiseasePediatric Cancer
- Interventions
- Biological: Red Blood Cell (RBC) Transfusion
- Registration Number
- NCT05255445
- Lead Sponsor
- Westat
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 157
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Pediatric Hematology-Oncology Red Blood Cell (RBC) Transfusion Patients in U.S. with pediatric oncologic diagnoses with hypo-proliferative bone marrow requiring single unit red blood cell transfusion Sickle cell disease (SCD) Red Blood Cell (RBC) Transfusion Patients with SCD who are chronically transfused (in the U.S. and Brazil) Thalassemia Red Blood Cell (RBC) Transfusion Patients with thalassemia who are chronically transfused in the U.S.
- Primary Outcome Measures
Name Time Method Change in Serum Iron Level Baseline (immediately before) and 2-hours after transfusion For all groups participating, change in serum iron measured from immediately prior to 2 hours post-transfusion
Change in Hemoglobin A or Hemoglobin Level per day (RBC Survival) Baseline (immediately pre-) to post-transfusion over 2 years Change in hemoglobin A or hemoglobin level per day in between subsequent transfusion episodes, for sickle cell disease and thalassemia cohorts, respectively
- Secondary Outcome Measures
Name Time Method Hemolysis Parameter Increment Baseline (immediately pre-) to post-transfusion or 2-hours post-transfusion, over 2 years Includes serum iron, indirect bilirubin, or plasma free hemoglobin
Hepcidin Level Baseline (immediately before) to 2 hours after transfusion Hepcidin level at time of transfusion is a predictor of change in iron parameters (i.e., transferrin saturation, serum iron) following transfusion
Non-Transferrin-Bound Iron (NTBI) Level Baseline (immediately before) to 2 hours after transfusion NTBI levels in patients with pediatric oncologic diagnoses with aplasia are elevated at baseline and increase following transfusion
Hemoglobin Increment Baseline (immediately pre-) to post-transfusion, over 2 years Hemoglobin increment \[defined as Hb/HbA(post-transfusion)visit(i) - Hb/HbA(pre-transfusion)visit(i)\] is associated with "RBC survival"
Number of Clinical Complications 2 years Increased NTBI, serum iron, or transferrin saturation following transfusion is associated with increased risk of clinical adverse effects (i.e., new infections, SCD complications)
Trial Locations
- Locations (14)
HEMOMINAS - Minas Gerais
🇧🇷Belo Horizonte, Minas Gerais, Brazil
Childrens Institute and Adult Clinics at Hospital das ClÃnicas da Faculdade de Medicina da Universidade de São Paulo
🇧🇷São Paulo, Brazil
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
New York Blood Center (NYBC)
🇺🇸New York, New York, United States
HEMOAM - Amazonas
🇧🇷Manaus, Amazonas, Brazil
HEMOPE - Pernambuco
🇧🇷Recife, Pernambuco, Brazil
Columbia University Irving Medical Center/New York Presbyterian Hospital (NYPH)
🇺🇸New York, New York, United States
HEMORIO - Rio De Janeiro
🇧🇷Rio De Janeiro, Brazil
Vitalant Research Institute
🇺🇸San Francisco, California, United States
Weill Cornell Medical Collection (WCMC)/New York Presbyterian Hospital (NYPH)
🇺🇸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