Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

157 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-03-16

Study Completion Date

2024-03-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Sickle Cell Disease Thalassemia Pediatric Cancer

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Sickle cell disease (SCD)

Patients with SCD who are chronically transfused (in the U.S. and Brazil)

Red Blood Cell (RBC) Transfusion

Intervention Type BIOLOGICAL

Simple RBC transfusion or partial manual exchange

Thalassemia

Patients with thalassemia who are chronically transfused in the U.S.

Red Blood Cell (RBC) Transfusion

Intervention Type BIOLOGICAL

Simple RBC transfusion or partial manual exchange

Pediatric Hematology-Oncology

Patients in U.S. with pediatric oncologic diagnoses with hypo-proliferative bone marrow requiring single unit red blood cell transfusion

Red Blood Cell (RBC) Transfusion

Intervention Type BIOLOGICAL

Simple RBC transfusion or partial manual exchange

Blood Donors

Allogenic blood donors in the US (estimated: 10,200) and allogenic blood donors in Brazil (estimated: 2,100) with extended donation genotyping using an investigational hematology array.

No interventions assigned to this group

Interventions

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Red Blood Cell (RBC) Transfusion

Simple RBC transfusion or partial manual exchange

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Well-characterized transfusion-dependent form of SCD or thalassemia (including Hemoglobin E-thalassemia and sickle-beta thalassemia) on chronic simple transfusion therapy or partial manual exchange
* On a regular simple RBC transfusion schedule, including partial manual exchange (i.e., 1-3 units scheduled every 2-6 weeks and on a minimum 6-month chronic transfusion trial; for partial manual exchange, the phlebotomy must be completed before the transfusion is started without a back and forth between rounds of phlebotomy and transfusion)
* Seen at any participating domestic hub hospital (i.e., Columbia University Irving Medical Center/Morgan Stanley Children's Hospital of New York, Weill Cornell Medical Center/Komansky Children's Hospital, Boston Children's Hospital, Froedtert \& Medical College of Wisconsin/Children's Wisconsin, University of California San Francisco, Benioff Children's Hospital Oakland) or enrolled in the Brazil REDS-IV-P sickle cell disease cohort and seen at any participating Brazil hemocenter (i.e., Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo - HCFMUSP, HEMOAM - Amazonas, HEMOMINAS - Minas Gerais, HEMOPE -Pernambuco, and HEMORIO - Rio de Janeiro)

* Either included in Aim #1 (consented patient with SCD or thalassemia) or patient with pediatric oncologic diagnosis under care in a pediatric hematology/oncology service with anemia due to chemotherapy or primary/secondary hypo-proliferative bone marrow requiring a RBC transfusion (including HSCT)
* \[In domestic study only\] Age ≤21 years old (many pediatric services include care of patients up to age 21, therefore the protocol will not limit by age but instead on whether they are seen in a pediatric service)
* Planned transfusion of RBC from an aliquot or unit from a single donor
* Seen at any participating domestic hub hospital (i.e., Columbia University Irving Medical Center/Morgan Stanley Children's Hospital of New York, Weill Cornell Medical Center/Komansky Children's Hospital, Boston Children's Hospital, Froedtert \& Medical College of Wisconsin/Children's Wisconsin, University of California San Francisco, Benioff Children's Hospital Oakland) or at any REDS-IV-P participating Brazil hemocenter (i.e., Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo - HCFMUSP, HEMOAM - Amazonas, HEMOMINAS - Minas Gerais, HEMOPE -Pernambuco, and HEMORIO - Rio de Janeiro).

Exclusion Criteria

* Institutionalization or imprisonment
* Foster care
* Weight \<11 kg

* Institutionalization or imprisonment
* Foster care
* Current active auto-immune hemolytic anemia based on positive direct antiglobulin test (DAT) with laboratory evidence of hemolysis and increased transfusion requirement
* \[In domestic study only\] Microangiopathic hemolytic anemia
* Weight \<18 kg

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Eldad A Hod, MD

Role: PRINCIPAL_INVESTIGATOR

Columbia University

Brian Custer, PhD, MPH

Role: PRINCIPAL_INVESTIGATOR

Vitalant Research Institute

Locations

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UCSF Benioff Children's Hospital

Oakland, California, United States

Site Status

Vitalant Research Institute

San Francisco, California, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

Weill Cornell Medical Collection (WCMC)/New York Presbyterian Hospital (NYPH)

New York, New York, United States

Site Status

Columbia University Irving Medical Center/New York Presbyterian Hospital (NYPH)

New York, New York, United States

Site Status

New York Blood Center (NYBC)

New York, New York, United States

Site Status

Children's Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Froedtert Hospital

Milwaukee, Wisconsin, United States

Site Status

Versiti Wisconsin, Inc.

Milwaukee, Wisconsin, United States

Site Status

HEMOAM - Amazonas

Manaus, Amazonas, Brazil

Site Status

HEMOMINAS - Minas Gerais

Belo Horizonte, Minas Gerais, Brazil

Site Status

HEMOPE - Pernambuco

Recife, Pernambuco, Brazil

Site Status

HEMORIO - Rio De Janeiro

Rio de Janeiro, , Brazil

Site Status

Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

São Paulo, , Brazil

Site Status

Countries

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United States Brazil

References

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Karafin MS, Kelly S, Chapman KM, Kreuziger LB, Manis JP, Dinardo C, Josephson CD, Stone M, Roubinian NH, Branchford B, Sachais BS, Hailu B, Sabino EC, Hod EA, Custer B; National Heart, Lung, and Blood Institute (NHLBI) Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric (REDS-IV-P). The Red Blood Cell-Improving Transfusions for Chronically Transfused Recipients (RBC-IMPACT) study: protocol description of an international multi-site observational clinical study. Blood Transfus. 2025 Sep-Oct;23(5):418-432. doi: 10.2450/BloodTransfus.1026. Epub 2025 May 9.

Reference Type DERIVED

PMID: 40423585 (View on PubMed)