Classification of Childhood Aplastic Anemia and Myelodysplastic Syndrome

Niemeyer, Charlotte M.; Baumann, Irith

doi:10.1182/asheducation-2011.1.84

Cited by 115 publications

(135 citation statements)

References 39 publications

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“…5 Nonetheless, because the majority of children with RCC have a normal karyotype and 80% of patients have a hypocellular BM, differentiating RCC from (v)SAA can be challenging. 4,6 Similar challenges are encountered in distinguishing (v)SAA and other non-clonal cytopenias from low-grade MDS in adults, especially in cases without specific morphological or cytogenetic aberrations. Flow cytometric immunophenotyping is a valuable addition to morphology in the diagnosis of MDS in adults.…”

Section: Introductionmentioning

confidence: 99%

Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood

Aalbers¹,

Heuvel‐Eibrink²,

Baumann³

et al. 2014

Haematologica

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Refractory cytopenia of childhood is the most common type of childhood myelodysplastic syndrome. Because the majority of children with refractory cytopenia have a normal karyotype and a hypocellular bone marrow, differentiating refractory cytopenia from the immune-mediated bone marrow failure syndrome (very) severe aplastic anemia can be challenging. Flow cytometric immunophenotyping of bone marrow has been shown to be a valuable diagnostic tool in differentiating myelodysplastic syndrome from non-clonal cytopenias in adults. Here, we performed the first comprehensive flow cytometric analysis of immature myeloid, lymphoid cells and erythroid cells, and granulocytes, monocytes, and lymphoid cells in bone marrow obtained from a large prospective cohort of 81 children with refractory cytopenia. Children with refractory cyotopenia had a strongly reduced myeloid compartment, but not as severe as children with aplastic anemia. Furthermore, the number of flow cytometric abnormalities was significantly higher in children with refractory cytopenia than in healthy controls and in children with aplastic anemia, but lower than in advanced myelodysplastic syndrome. We conclude that flow cytometric immunophenotyping could be a relevant addition to histopathology in the diagnosis of refractory cytopenia of childhood. (The multi-center studies EWOG-MDS RC06 and EWOG-MDS 2006 are registered at clinicaltrials.gov identifiers 00499070 and 00662090, respectively).

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Section: Introductionmentioning

confidence: 99%

Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood

Aalbers¹,

Heuvel‐Eibrink²,

Baumann³

et al. 2014

Haematologica

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show abstract

“…BM cellularity was reduced in about half of the children with low-grade MDS [3,5]. In children, cytopenias with decrease of BM cellularity might be caused by various underlying disorders, including AA, IBMFS and other hematological and nonhematological disorders [7]. Morphological abnormalities may also be found in these disorders.…”

Section: Morphology and Histologymentioning

confidence: 98%

“…Patchy erythropoiesis with impaired maturation, accompanied by sparsely distributed granulopoiesis, in an otherwise adipocytic BM is characteristic of hypoplastic low-grade MDS (Fig. 2b) [2,7,8]. Megakaryocytes are markedly decreased or absent.…”

Section: Morphology and Histologymentioning

confidence: 99%

“…Since hematopoietic cells in hypocellular low-grade MDS are often distributed in a patchy pattern (Fig. 2a), information obtained from aspiration cytology is limited [7,8]. Therefore, BM biopsies, which mirror the topography and cellularity of the local hematopoiesis, should be performed at least twice to diagnose hypoplastic BM disorders in children [2,7,8].…”

Section: Morphology and Histologymentioning

confidence: 99%

“…2a), information obtained from aspiration cytology is limited [7,8]. Therefore, BM biopsies, which mirror the topography and cellularity of the local hematopoiesis, should be performed at least twice to diagnose hypoplastic BM disorders in children [2,7,8]. Patchy erythropoiesis with impaired maturation, accompanied by sparsely distributed granulopoiesis, in an otherwise adipocytic BM is characteristic of hypoplastic low-grade MDS (Fig.…”

Section: Morphology and Histologymentioning

confidence: 99%

See 2 more Smart Citations

The current perspective of low-grade myelodysplastic syndrome in children

Hasegawa

2016

Int J Hematol

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Pediatric aplastic anemia and refractory cytopenia: A retrospective analysis assessing outcomes and histomorphologic predictors

et al. 2015

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Pediatric acquired aplastic anemia (AA) is a bone marrow disorder that is difficult to distinguish from inherited bone marrow failure syndromes and hypocellular refractory cytopenia of childhood (RCC). Historically, patients with hypocellular RCC have been given the diagnosis of AA. To assess the clinical and histologic distinction between RCC and AA, we performed a retrospective analysis of 149 patients previously diagnosed with AA between 1976-2010. We evaluated event free survival (EFS), overall survival (OS), response rates to immunosuppressive therapy, treatment-related toxicities and clonal evolution. The 5-year EFS and OS were 50.8%±5.5% and 73.1%±4.7%, respectively. Patients with very severe AA had worse OS compared to patients with severe and moderately severe AA. Seventy-two patients had diagnostic pathology specimens available for review. Three pediatric hematopathologists reviewed and reclassified these specimens as AA, RCC or Other based on 2008 WHO Criteria. The concordance between pathologists in the diagnosis of AA or RCC was modest. RCC was associated with a trend towards improved OS and EFS and was not prognostic of immunosuppression therapy treatment failure. There was a low rate of clonal evolution exclusively associated with moderately severe AA. Our findings indicate that a diagnosis of RCC is difficult to establish with certainty and does not predict outcomes, calling into question the reproducibility and clinical significance of the RCC classification and warranting further studies.

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Classification of Childhood Aplastic Anemia and Myelodysplastic Syndrome

Cited by 115 publications

References 39 publications

Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood

Bone marrow immunophenotyping by flow cytometry in refractory cytopenia of childhood

The current perspective of low-grade myelodysplastic syndrome in children

Pediatric aplastic anemia and refractory cytopenia: A retrospective analysis assessing outcomes and histomorphologic predictors

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