Skip to main content Accessibility help
×
Hostname: page-component-586b7cd67f-2brh9 Total loading time: 0 Render date: 2024-11-27T21:20:40.680Z Has data issue: false hasContentIssue false

Chapter 13 - Childhood Myelodysplastic Syndromes (MDSs)

from Section IV - Neoplastic Disorders of Bone Marrow

Published online by Cambridge University Press:  25 January 2024

Xiayuan Liang
Affiliation:
Children’s Hospital of Colorado
Bradford Siegele
Affiliation:
Children’s Hospital of Colorado
Jennifer Picarsic
Affiliation:
Cincinnati Childrens Hospital Medicine Center
Get access

Summary

Childhood myelodysplastic syndromes (MDSs) are clonal myeloid neoplasms characterized by impaired production of circulating hematopoietic elements (generally, hemoglobin concentration < 10 g/dL, platelet count < 100 × 109/L, or absolute neutrophil count < 1.8 × 109/L), frequently accompanied by morphologic dysplasia and recurrent genetic abnormalities and lacking features of overproduction of additional hematopoietic lineage elements. In light of its rarity in comparison to its clinicopathologic mimics, minimal diagnostic criteria have been proposed (see Table 13.1).

Type
Chapter
Information
Publisher: Cambridge University Press
Print publication year: 2024

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

Hasle, H, Niemeyer, CM, Chessells, JM, et al. A pediatric approach to the WHO classification of myelodysplastic and myeloproliferative diseases. Leukemia. 2003;17(2):277–82. doi: 10.1038/sj.leu.2402765Google Scholar
Hasle, H, Niemeyer, CM. Advances in the prognostication and management of advanced MDS in children. Br J Haematol. 2011;154(2):185–95. doi: 10.1111/j.1365-2141.2011.08724.xGoogle Scholar
Kardos, G, Baumann, I, Passmore, SJ, et al. Refractory anemia in childhood: a retrospective analysis of 67 patients with particular reference to monosomy 7. Blood. 2003;102(6):19972003. doi: 10.1182/blood-2002-11-3444Google Scholar
Swerdlow, S, Campo, E, Harris, N, et al., eds. WHO classification of tumors of haematopoietic and lymphoid tissues. Rev. 4th ed. IARC Press; 2017.Google Scholar
Rau, AT, Shreedhara, AK, Kumar, S. Myelodysplastic syndromes in children: where are we today? Ochsner J. 2012;12(3):216–20.Google Scholar
Polychronopoulou, S, Panagiotou, JP, Kossiva, L, et al. Clinical and morphological features of paediatric myelodysplastic syndromes: a review of 34 cases. Acta Paediatr. 2004;93(8):1015–23. doi: 10.1111/j.1651-2227.2004.tb02708.xGoogle Scholar
Hofmann, I. Pediatric myelodysplastic syndromes. J Hematopathology. 2015;8:127–41.Google Scholar
Fleming, MD. Congenital sideroblastic anemias: iron and heme lost in mitochondrial translation. Hematology Am Soc Hematol Educ Program. 2011;2011:525–31. doi: 10.1182/asheducation-2011.1.525Google Scholar
Westers, TM, Ireland, R, Kern, W, et al. Standardization of flow cytometry in myelodysplastic syndromes: a report from an international consortium and the European LeukemiaNet Working Group. Leukemia. 2012;26(7):1730–41. doi: 10.1038/leu.2012.30Google Scholar
Ogata, K, Della Porta, MG, Malcovati, L, et al. Diagnostic utility of flow cytometry in low-grade myelodysplastic syndromes: a prospective validation study. Haematologica. 2009;94(8):1066–74. doi: 10.3324/haematol.2009.008532Google Scholar
Porwit, A, van de Loosdrecht, AA, Bettelheim, P, et al. Revisiting guidelines for integration of flow cytometry results in the WHO classification of myelodysplastic syndromes-proposal from the International/European LeukemiaNet Working Group for Flow Cytometry in MDS. Leukemia. 2014;28(9):1793–8. doi: 10.1038/leu.2014.191Google Scholar
Aalbers, AM, van den Heuvel-Eibrink, MM, de Haas, V, et al. Applicability of a reproducible flow cytometry scoring system in the diagnosis of refractory cytopenia of childhood. Leukemia. 2013;27(9):1923–5. doi: 10.1038/leu.2013.81Google Scholar
Sperling, AS, Gibson, CJ, Ebert, BL. The genetics of myelodysplastic syndrome: from clonal haematopoiesis to secondary leukaemia. Nat Rev Cancer. 2017;17(1):519. doi: 10.1038/nrc.2016.112Google Scholar
Nazha, A, Seastone, D, Radivoyevitch, T, et al. Genomic patterns associated with hypoplastic compared to hyperplastic myelodysplastic syndromes. Haematologica. 2015;100(11):e434–7. doi: 10.3324/haematol.2015.130112Google Scholar
Schwartz, JR, Ma, J, Lamprecht, T, et al. The genomic landscape of pediatric myelodysplastic syndromes. Nat Commun. 2017;8(1):1557. doi: 10.1038/s41467-017-01590-5CrossRefGoogle ScholarPubMed
Pastor, V, Hirabayashi, S, Karow, A, et al. Mutational landscape in children with myelodysplastic syndromes is distinct from adults: specific somatic drivers and novel germline variants. Leukemia. 2017;31(3):759762. doi: 10.1038/leu.2016.342Google Scholar
Hirabayashi, S, Flotho, C, Moetter, J, et al. Spliceosomal gene aberrations are rare, coexist with oncogenic mutations, and are unlikely to exert a driver effect in childhood MDS and JMML. Blood. 2012;119(11):e96–9. doi: 10.1182/blood-2011-12-395087Google Scholar
Mikhailova, N, Sessarego, M, Fugazza, G, et al. Cytogenetic abnormalities in patients with severe aplastic anemia. Haematologica. 1996;81(5):418–22.Google Scholar
Dolan, MM, Singleton, TP, Neglia, J, et al. Aplastic anemia and monosomy 7-associated dysmegakaryocytopoiesis. Am J Clin Pathol. 2006;126(6):925–30. doi: 10.1309/50GWDKVWU3VWL5XWGoogle Scholar
Göhring, G, Michalova, K, Beverloo, HB, et al. Complex karyotype newly defined: the strongest prognostic factor in advanced childhood myelodysplastic syndrome. Blood. 2010;116(19):3766–9. doi: 10.1182/blood-2010-04-280313Google Scholar
Woodard, P, Carpenter, PA, Davies, SM, et al. Unrelated donor bone marrow transplantation for myelodysplastic syndrome in children. Biol Blood Marrow Transplant. 2011;17(5):723–8. doi: 10.1016/j.bbmt.2010.08.016Google Scholar
Yusuf, U, Frangoul, HA, Gooley, TA, et al. Allogeneic bone marrow transplantation in children with myelodysplastic syndrome or juvenile myelomonocytic leukemia: the Seattle experience. Bone Marrow Transplant. 2004;33(8):805–14. doi: 10.1038/sj.bmt.1704438Google Scholar
Bejar, R, Stevenson, K, Abdel-Wahab, O, et al. Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011;364(26):2496–506. doi: 10.1056/NEJMoa1013343Google Scholar

Save book to Kindle

To save this book to your Kindle, first ensure [email protected] is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about saving to your Kindle.

Note you can select to save to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be saved to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

Available formats
×

Save book to Dropbox

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Dropbox.

Available formats
×

Save book to Google Drive

To save content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about saving content to Google Drive.

Available formats
×