Journal article

Utility of clinical comprehensive genomic characterization for diagnostic categorization in patients presenting with hypocellular bone marrow failure syndromes

P Blombery, LC Fox, GL Ryland, ER Thompson, J Lickiss, M McBean, S Yerneni, D Hughes, A Greenway, F Mechinaud, EM Wood, GJ Lieschke, J Szer, P Barbaro, J Roy, J Wight, E Lynch, M Martyn, C Gaff, D Ritchie

Haematologica | FERRATA STORTI FOUNDATION | Published : 2021

Abstract

Bone marrow failure (BMF) related to hypoplasia of hematopoietic elements in the bone marrow is a heterogeneous clinical entity with a broad differential diagnosis including both inherited and acquired causes. Accurate diagnostic categorization is critical to optimal patient care and detection of genomic variants in these patients may provide this important diagnostic and prognostic information. We performed real-time, accredited (ISO15189) comprehensive genomic characterization including targeted sequencing and whole exome sequencing in 115 patients with BMF syndromes (median age 24 years, range: 3 months - 81 years). In patients with clinical diagnoses of inherited BMF syndromes, acquired ..

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