RH genotype matched red cell transfusions for patients with sickle cell disease
镰状细胞病患者 RH 基因型匹配的红细胞输注
基本信息
- 批准号:10470880
- 负责人:
- 金额:$ 83.48万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2019
- 资助国家:美国
- 起止时间:2019-08-15 至 2024-07-31
- 项目状态:已结题
- 来源:
- 关键词:AddressAdverse effectsAfrican AmericanAfrican ancestryAllelesAlloimmunizationAntibodiesAntibody FormationAntigensBiological AssayBloodBlood BanksBlood donorBlood group antibody DCaringCellsChronicClinicalClinical ResearchClinical TrialsCollaborationsComplexComplicationCustomD CellsDNADonor SelectionEffectivenessEquipment and supply inventoriesErythrocyte TransfusionErythrocytesEventExposure toFrequenciesGene DuplicationGene FamilyGene FrequencyGenesGeneticGenetic HeterogeneityGenetic RecombinationGenetic VariationGenotypeGoalsHemolysisHospitalsHybridsIncidenceIndividualInstitutionKnowledgeLeadLifeMethodsMinorityModelingMonitorMorbidity - disease rateMulticenter StudiesPatient CarePatient MonitoringPatientsPhasePhenotypeProspective StudiesProteinsRecording of previous eventsResearch PersonnelRh-Hr Blood-Group SystemRisk FactorsSafetySavingsSerologyServicesSickle Cell AnemiaSystemTechnologyTransfusionVariantVisitbasebench to bedsideclinical practiceclinically significantcohortcostcost effectivedata managementdesigneffectiveness evaluationfeasibility testinggenetic variantimprovedinnovationnew technologynext generation sequencingnovelnovel sequencing technologyprecision medicinepreventprophylacticprospectiverecruit
项目摘要
ABSTRACT
Red blood cell transfusion remains a life-saving therapy for patients with sickle cell disease (SCD). A major
problem is the high rate of alloimmunization (antibody formation against transfused red cells) that occurs in
transfused patients with SCD. Alloimmunization leads to delays in care, increases costs, and makes transfusion
therapy unsafe and impossible for some patients. The most common antibodies formed by patients with SCD
are directed against the Rh blood group system. Genetic diversity in Rh antigens in patients and blood donors
of African descent contributes to this high incidence and complexity of antibodies found in patients with SCD.
Recent studies performed by our group and others demonstrate RH genetic variants in patients and donors is a
major risk factor leading to Rh alloimmunization. Routine blood bank assays can not identify these variants, so
although Rh-matched red cells are transfused to patients with SCD, they would only be truly Rh-matched with
DNA-based matching. Genetic matching of donor units at the RH loci may prevent Rh alloimmunization but no
clinical trials have been conducted. Major barriers are the current cost of RH genotyping as well as inventory
and data management of donor genotypes to identify RH matched units for patients. The major goal of this
proposal is to perform pilot clinical studies to provide RH genotype matched red cells to chronically transfused
patients with SCD. We will determine the feasibility of identifying adequate genotype matched donor units in real
clinical practice, identify barriers, and prospectively monitor patients for Rh alloimmunization. A second goal for
this proposal is to apply new technologies to improve our knowledge of the two RH genes, RHD and RHCE,
which due to their high sequence similarity, currently makes it challenging to apply low cost sequencing methods
to provide RH genotype matched red cells for all patients with SCD. In this application, we propose three
integrated aims that can address the current challenges of transfusion therapy for SCD, and will drive the field
forward by providing innovative solutions to these current obstacles.
摘要
项目成果
期刊论文数量(0)
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会议论文数量(0)
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RH genotype matched red cell transfusions for patients with sickle cell disease
镰状细胞病患者 RH 基因型匹配的红细胞输注
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10259737 - 财政年份:2019
- 资助金额:
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Improving transfusion therapy for patients with sickle cell disease with pluripotent stem cell-derived red cells
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Improving transfusion therapy for patients with sickle cell disease with pluripotent stem cell-derived red cells
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