Successful umbilical cord blood hematopoietic stem cell transplantation in pediatric patients with MDS/AML associated with underlying GATA2 mutations: two case reports and review of literature.

Abstract:

:Germline GATA2 mutations have been associated with a vast array of clinical manifestations, as well as hematological deficiencies and a propensity to AML or MDS. We present two cases of pediatric AML/MDS with underlying GATA2 mutations who underwent a successful umbilical cord hematopoietic stem cell transplantation using two different conditioning regimens. These cases illustrate the importance of recognizing the clinical features associated with GATA2 mutations and performing the appropriate molecular testing. Diagnosis of heritable gene mutations associated with familial AML/MDS has significant clinical implication for the patients and affected families.

journal_name

Pediatr Transplant

authors

Mallhi K,Dix DB,Niederhoffer KY,Armstrong L,Rozmus J

doi

10.1111/petr.12764

subject

Has Abstract

pub_date

2016-11-01 00:00:00

pages

1004-1007

issue

7

eissn

1397-3142

issn

1399-3046

journal_volume

20

pub_type

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