Myelodysplastic syndromes in children

Federica Galaverna, Annalisa Ruggeri, Franco Locatelli

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish
Pages (from-to)402-408
Number of pages7
JournalCurrent Opinion in Oncology
Volume30
Issue number6
DOIs
Publication statusPublished - Nov 2018

Fingerprint

Myelodysplastic Syndromes
Hematopoietic Stem Cell Transplantation
HLA Antigens
Therapeutics
Transplants
Neutropenia
Karyotype
Acute Myeloid Leukemia
Tissue Donors
Mutation

Cite this

Myelodysplastic syndromes in children. / Galaverna, Federica; Ruggeri, Annalisa; Locatelli, Franco.

In: Current Opinion in Oncology, Vol. 30, No. 6, 11.2018, p. 402-408.

Research output: Contribution to journalArticle

Galaverna, Federica ; Ruggeri, Annalisa ; Locatelli, Franco. / Myelodysplastic syndromes in children. In: Current Opinion in Oncology. 2018 ; Vol. 30, No. 6. pp. 402-408.
@article{a2f78323f03e4cf282d77f3fcd1c8962,
title = "Myelodysplastic syndromes in children",
abstract = "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.",
author = "Federica Galaverna and Annalisa Ruggeri and Franco Locatelli",
year = "2018",
month = "11",
doi = "10.1097/CCO.0000000000000488",
language = "English",
volume = "30",
pages = "402--408",
journal = "Current Opinion in Oncology",
issn = "1040-8746",
publisher = "Lippincott Williams and Wilkins",
number = "6",

}

TY - JOUR

T1 - Myelodysplastic syndromes in children

AU - Galaverna, Federica

AU - Ruggeri, Annalisa

AU - Locatelli, Franco

PY - 2018/11

Y1 - 2018/11

N2 - 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.

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.

U2 - 10.1097/CCO.0000000000000488

DO - 10.1097/CCO.0000000000000488

M3 - Article

C2 - 30222640

VL - 30

SP - 402

EP - 408

JO - Current Opinion in Oncology

JF - Current Opinion in Oncology

SN - 1040-8746

IS - 6

ER -