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Refractory cytopenia of childhood

MedGen UID:
417430
•Concept ID:
C2826323
•
Neoplastic Process
Synonyms: RCC; Refractory Cytopenia of Childhood; refractory cytopenia of childhood
 
Monarch Initiative: MONDO:0044874

Definition

The most common subtype of the myelodysplastic syndromes affecting children. It is characterized by persistent cytopenia with less than 5% blasts in the bone marrow and less than 2% blasts in the peripheral blood. [from NCI]

Professional guidelines

PubMed

Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia.
Yoshida N
Int J Hematol 2024 Mar;119(3):240-247. Epub 2023 Mar 3 doi: 10.1007/s12185-023-03564-4. PMID: 36867357
Clinical characteristics and treatment outcome in 65 cases with refractory cytopenia of childhood defined according to the WHO 2008 classification.
Hasegawa D, Chen X, Hirabayashi S, Ishida Y, Watanabe S, Zaike Y, Tsuchida M, Masunaga A, Yoshimi A, Hama A, Kojima S, Ito M, Nakahata T, Manabe A
Br J Haematol 2014 Sep;166(5):758-66. Epub 2014 Jun 4 doi: 10.1111/bjh.12955. PMID: 24894311
Morphological differentiation of severe aplastic anaemia from hypocellular refractory cytopenia of childhood: reproducibility of histopathological diagnostic criteria.
Baumann I, Führer M, Behrendt S, Campr V, Csomor J, Furlan I, de Haas V, Kerndrup G, Leguit RJ, De Paepe P, Noellke P, Niemeyer C, Schwarz S
Histopathology 2012 Jul;61(1):10-7. Epub 2012 Mar 28 doi: 10.1111/j.1365-2559.2011.04156.x. PMID: 22458667

Recent clinical studies

Etiology

The International Consensus Classification (ICC) of hematologic neoplasms with germline predisposition, pediatric myelodysplastic syndrome, and juvenile myelomonocytic leukemia.
Rudelius M, Weinberg OK, Niemeyer CM, Shimamura A, Calvo KR
Virchows Arch 2023 Jan;482(1):113-130. Epub 2022 Nov 29 doi: 10.1007/s00428-022-03447-9. PMID: 36445482
The histopathology of bone marrow failure in children.
Iwafuchi H
J Clin Exp Hematop 2018;58(2):68-86. doi: 10.3960/jslrt.18018. PMID: 29998978Free PMC Article
How I treat myelodysplastic syndromes of childhood.
Locatelli F, Strahm B
Blood 2018 Mar 29;131(13):1406-1414. Epub 2018 Feb 8 doi: 10.1182/blood-2017-09-765214. PMID: 29438960
Myelodysplastic and myeloproliferative disorders of childhood.
Hasle H
Hematology Am Soc Hematol Educ Program 2016 Dec 2;2016(1):598-604. doi: 10.1182/asheducation-2016.1.598. PMID: 27913534Free PMC Article
Childhood myelodysplastic syndrome.
Chatterjee T, Choudhry VP
Indian J Pediatr 2013 Sep;80(9):764-71. Epub 2013 Aug 3 doi: 10.1007/s12098-013-1130-8. PMID: 23912822

Diagnosis

Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia.
Yoshida N
Int J Hematol 2024 Mar;119(3):240-247. Epub 2023 Mar 3 doi: 10.1007/s12185-023-03564-4. PMID: 36867357
Childhood Myelodysplastic Syndrome.
Chisholm KM, Bohling SD
Clin Lab Med 2023 Dec;43(4):639-655. Epub 2023 Aug 7 doi: 10.1016/j.cll.2023.06.005. PMID: 37865508
The International Consensus Classification (ICC) of hematologic neoplasms with germline predisposition, pediatric myelodysplastic syndrome, and juvenile myelomonocytic leukemia.
Rudelius M, Weinberg OK, Niemeyer CM, Shimamura A, Calvo KR
Virchows Arch 2023 Jan;482(1):113-130. Epub 2022 Nov 29 doi: 10.1007/s00428-022-03447-9. PMID: 36445482
Myelodysplastic syndromes in children.
Galaverna F, Ruggeri A, Locatelli F
Curr Opin Oncol 2018 Nov;30(6):402-408. doi: 10.1097/CCO.0000000000000488. PMID: 30222640
How I treat myelodysplastic syndromes of childhood.
Locatelli F, Strahm B
Blood 2018 Mar 29;131(13):1406-1414. Epub 2018 Feb 8 doi: 10.1182/blood-2017-09-765214. PMID: 29438960

Therapy

Incidence and outcomes of refractory immune thrombocytopenic purpura in children: a retrospective study in a single institution.
Ito M, Yagasaki H, Kanezawa K, Shimozawa K, Hirai M, Morioka I
Sci Rep 2021 Jul 12;11(1):14263. doi: 10.1038/s41598-021-93646-2. PMID: 34253777Free PMC Article
The current perspective of low-grade myelodysplastic syndrome in children.
Hasegawa D
Int J Hematol 2016 Apr;103(4):360-4. Epub 2016 Mar 3 doi: 10.1007/s12185-016-1965-7. PMID: 26939774
Comparison of immune manifestations between refractory cytopenia of childhood and aplastic anemia in children: A single-center retrospective study.
Wu J, Cheng Y, Zhang L
Leuk Res 2015 Dec;39(12):1347-52. Epub 2015 Sep 21 doi: 10.1016/j.leukres.2015.09.012. PMID: 26427728
Pediatric aplastic anemia and refractory cytopenia: A retrospective analysis assessing outcomes and histomorphologic predictors.
Forester CM, Sartain SE, Guo D, Harris MH, Weinberg OK, Fleming MD, London WB, Williams DA, Hofmann I
Am J Hematol 2015 Apr;90(4):320-6. Epub 2015 Mar 2 doi: 10.1002/ajh.23937. PMID: 25580823Free PMC Article
Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood.
Aalbers AM, van den Heuvel-Eibrink MM, Baumann I, Dworzak M, Hasle H, Locatelli F, De Moerloose B, Schmugge M, Mejstrikova E, Nováková M, Zecca M, Zwaan CM, Te Marvelde JG, Langerak AW, van Dongen JJ, Pieters R, Niemeyer CM, van der Velden VH
Haematologica 2015 Mar;100(3):315-23. Epub 2014 Nov 25 doi: 10.3324/haematol.2014.107706. PMID: 25425683Free PMC Article

Prognosis

Incidence and outcomes of refractory immune thrombocytopenic purpura in children: a retrospective study in a single institution.
Ito M, Yagasaki H, Kanezawa K, Shimozawa K, Hirai M, Morioka I
Sci Rep 2021 Jul 12;11(1):14263. doi: 10.1038/s41598-021-93646-2. PMID: 34253777Free PMC Article
The current perspective of low-grade myelodysplastic syndrome in children.
Hasegawa D
Int J Hematol 2016 Apr;103(4):360-4. Epub 2016 Mar 3 doi: 10.1007/s12185-016-1965-7. PMID: 26939774
Comparison of immune manifestations between refractory cytopenia of childhood and aplastic anemia in children: A single-center retrospective study.
Wu J, Cheng Y, Zhang L
Leuk Res 2015 Dec;39(12):1347-52. Epub 2015 Sep 21 doi: 10.1016/j.leukres.2015.09.012. PMID: 26427728
Pediatric aplastic anemia and refractory cytopenia: A retrospective analysis assessing outcomes and histomorphologic predictors.
Forester CM, Sartain SE, Guo D, Harris MH, Weinberg OK, Fleming MD, London WB, Williams DA, Hofmann I
Am J Hematol 2015 Apr;90(4):320-6. Epub 2015 Mar 2 doi: 10.1002/ajh.23937. PMID: 25580823Free PMC Article
Childhood myelodysplastic syndrome.
Chatterjee T, Choudhry VP
Indian J Pediatr 2013 Sep;80(9):764-71. Epub 2013 Aug 3 doi: 10.1007/s12098-013-1130-8. PMID: 23912822

Clinical prediction guides

Clinical impact of dysplastic changes in acquired aplastic anemia: A systematic study of bone marrow biopsies in children and adults.
Marchesi RF, Velloso EDRP, Garanito MP, Leal AM, Siqueira SAC, Azevedo Neto RS, Rocha V, Zerbini MCN
Ann Diagn Pathol 2020 Apr;45:151459. Epub 2019 Dec 30 doi: 10.1016/j.anndiagpath.2019.151459. PMID: 32000075
How I treat myelodysplastic syndromes of childhood.
Locatelli F, Strahm B
Blood 2018 Mar 29;131(13):1406-1414. Epub 2018 Feb 8 doi: 10.1182/blood-2017-09-765214. PMID: 29438960
The current perspective of low-grade myelodysplastic syndrome in children.
Hasegawa D
Int J Hematol 2016 Apr;103(4):360-4. Epub 2016 Mar 3 doi: 10.1007/s12185-016-1965-7. PMID: 26939774
Pediatric aplastic anemia and refractory cytopenia: A retrospective analysis assessing outcomes and histomorphologic predictors.
Forester CM, Sartain SE, Guo D, Harris MH, Weinberg OK, Fleming MD, London WB, Williams DA, Hofmann I
Am J Hematol 2015 Apr;90(4):320-6. Epub 2015 Mar 2 doi: 10.1002/ajh.23937. PMID: 25580823Free PMC Article
Childhood myelodysplastic syndrome.
Chatterjee T, Choudhry VP
Indian J Pediatr 2013 Sep;80(9):764-71. Epub 2013 Aug 3 doi: 10.1007/s12098-013-1130-8. PMID: 23912822

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