PMID- 30222640
OWN - NLM
STAT- MEDLINE
DCOM- 20190503
LR  - 20190503
IS  - 1531-703X (Electronic)
IS  - 1040-8746 (Linking)
VI  - 30
IP  - 6
DP  - 2018 Nov
TI  - Myelodysplastic syndromes in children.
PG  - 402-408
LID - 10.1097/CCO.0000000000000488 [doi]
AB  - PURPOSE OF REVIEW: Myelodysplastic syndromes (MDSs) are rare disorders in 
      children, showing peculiar clinical manifestations and biological features. This 
      review will summarize biological, genetic and clinical features of childhood MDS 
      and will provide an update of the algorithm of treatment of the different disease 
      variants. RECENT FINDINGS: The most recent classification of MDS includes 
      refractory cytopenia of childhood (RCC), advanced and therapy-related MDS. 
      Importantly, in children, these clonal hematopoietic disorders may be often 
      associated with inherited bone marrow failure syndromes, this representing a 
      challenge for diagnostic work-up and treatment. Moreover, germline syndromes 
      predisposing to develop MDS/acute myeloid leukemia have been recently identified, 
      such as those caused by mutations in GATA2, ETV6, SRP72 and SAMD9/SAMD9-L. 
      SUMMARY: Treatment of childhood MDS varies according to specific disease 
      features; allogeneic hematopoietic stem cell transplantation (HSCT) using a Human 
      Leukocyte antigen (HLA)-identical donor, whenever available, represents the 
      treatment of choice for most of these children. HSCT is indicated in MDS with 
      excess of blasts, or in therapy-related MDS. For RCC patients, HSCT is 
      recommended for RCC associated with monosomy 7, or complex karyotype and for 
      patients showing severe neutropenia or transfusion dependence. Novel approaches 
      of HSCT from an HLA-haploidentical relative after selective graft manipulation 
      allow reducing transplant-related complications.
FAU - Galaverna, Federica
AU  - Galaverna F
AD  - Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico 
      Bambino Gesu, Rome.
FAU - Ruggeri, Annalisa
AU  - Ruggeri A
AD  - Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico 
      Bambino Gesu, Rome.
FAU - Locatelli, Franco
AU  - Locatelli F
AD  - Department of Pediatric Hematology and Oncology, IRCCS Ospedale Pediatrico 
      Bambino Gesu, Rome.
AD  - Department of Pediatric Sciences, University of Pavia, Pavia, Italy.
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Curr Opin Oncol
JT  - Current opinion in oncology
JID - 9007265
SB  - IM
MH  - Child
MH  - Humans
MH  - Myelodysplastic Syndromes/*diagnosis/genetics/pathology/*therapy
EDAT- 2018/09/18 06:00
MHDA- 2019/05/06 06:00
CRDT- 2018/09/18 06:00
PHST- 2018/09/18 06:00 [pubmed]
PHST- 2019/05/06 06:00 [medline]
PHST- 2018/09/18 06:00 [entrez]
AID - 10.1097/CCO.0000000000000488 [doi]
PST - ppublish
SO  - Curr Opin Oncol. 2018 Nov;30(6):402-408. doi: 10.1097/CCO.0000000000000488.