Congenital Dyserythropoietic Anemia Type II: An Update from the Congenital Dyseryhtropoietic Anemia Registry of North America (CDAR)
Elgammal Y, Risinger M, Husami A, Walden J, Gupta S, Shah N, Boyer J, Abajas Y, Winstead M, Miller D, Vidal-Anaya V, Vlachos A, Lal A, Ritchey A, Lorsbach R, Zhang W, Kalfa T, Niss O. Congenital Dyserythropoietic Anemia Type II: An Update from the Congenital Dyseryhtropoietic Anemia Registry of North America (CDAR). Blood 2023, 142: 1079. DOI: 10.1182/blood-2023-189389.Peer-Reviewed Original ResearchIron overloadIneffective erythropoiesisCongenital dyserythropoietic anemiaBlood transfusionHepatic hemosiderosisHemolytic anemiaIron levelsFrequency of transfusionGDF-15 levelsWhole-genome sequencingSeverity of anemiaAge of diagnosisCDA IINatural history studiesBody iron levelsDeep intronic variantsCDA II patientsCDA type IIIntronic variantsAutosomal recessive diseaseBone marrow erythroblastsBM examinationFerritin ratioGDF-15Anemia severity