How I treat myelodysplastic syndromes of childhood

Author:

Locatelli Franco12,Strahm Brigitte3

Affiliation:

1. Department of Pediatric Hematology and Oncology, Istituto di Ricovero e Cura a Carattere Scientifico Ospedale Pediatrico Bambino Gesù, Rome, Italy;

2. Department of Pediatric Science, University of Pavia, Pavia, Italy; and

3. Department of Pediatrics and Adolescent Medicine, Division of Pediatric Hematology and Oncology, Medical Centre, Faculty of Medicine, University of Freiburg, Freiburg, Germany

Abstract

Abstract Pediatric myelodysplastic syndromes (MDSs) are a heterogeneous group of clonal disorders with an annual incidence of 1 to 4 cases per million, accounting for less than 5% of childhood hematologic malignancies. MDSs in children often occur in the context of inherited bone marrow failure syndromes, which represent a peculiarity of myelodysplasia diagnosed in pediatric patients. Moreover, germ line syndromes predisposing individuals to develop MDS or acute myeloid leukemia have recently been identified, such as those caused by mutations in GATA2, ETV6, SRP72, and SAMD9/SAMD9-L. Refractory cytopenia of childhood (RCC) is the most frequent pediatric MDS variant, and it has specific histopathologic features. Allogeneic hematopoietic stem cell transplantation (HSCT) is the treatment of choice for many children with MDSs and is routinely offered to all patients with MDS with excess of blasts, to those with MDS secondary to previously administered chemoradiotherapy, and to those with RCC associated with monosomy 7, complex karyotype, severe neutropenia, or transfusion dependence. Immune-suppressive therapy may be a treatment option for RCC patients with hypocellular bone marrow and the absence of monosomy 7 or a complex karyotype, although the response rate is lower than that observed in severe aplastic anemia, and a relevant proportion of these patients will subsequently need HSCT for either nonresponse or relapse.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Reference84 articles.

1. The epidemiology of myelodysplastic syndromes;Sekeres;Hematol Oncol Clin North Am,2010

2. Classification of childhood aplastic anemia and myelodysplastic syndrome;Niemeyer,2011

3. Germline genetic predisposition to hematologic malignancy;Furutani;J Clin Oncol,2017

4. Genetic predisposition to hematologic malignancies: management and surveillance;Godley;Blood,2017

5. Myelodysplastic and myeloproliferative disorders of childhood;Hasle;Hematology Am Soc Hematol Educ Program,2016

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