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Diagnosis of acquired bone marrow failure syndrome during childhood using the 2008 World Health Organization

Wenyu Yang1, Peihong Zhang, Asahito Hama

  • 1Diagnostic and Therapeutic Center of Children's Blood Disease, Blood Disease Hospital, Institute of Hematology, Chinese Academy of Medical Sciences, 288 Nanjing Road, Tianjin, China.

International Journal of Hematology
|May 8, 2012
PubMed

Insights

Differentiating pediatric aplastic anemia (AA) from refractory cytopenia (RCC/RCMD) is difficult. This study found RCC/RCMD patients had milder disease and bone marrow hypocellularity than AA patients, suggesting distinct clinical courses.

Area of Science:

  • Pediatric Hematology
  • Hematopathology
  • Bone Marrow Failure Syndromes

Background:

  • Distinguishing hypoplastic myelodysplastic syndromes from aplastic anemia (AA) presents diagnostic challenges.
  • Accurate diagnosis is crucial for appropriate treatment and prognosis in pediatric bone marrow failure syndromes.

Purpose of the Study:

  • To evaluate the diagnostic consensus for pediatric acquired bone marrow failure syndromes using WHO 2008 criteria.
  • To compare clinical and laboratory findings between aplastic anemia and refractory cytopenia subtypes in children.

Main Methods:

  • Joint review of bone marrow smears and trephine biopsies from 100 children with acquired bone marrow failure syndrome.
  • Application of the 2008 World Health Organization classification criteria for hematopoietic and lymphoid tissues.
  • Consensus diagnosis by Japanese and Chinese pediatric hematologists and pathologists.

Main Results:

  • Consensus diagnoses included aplastic anemia (AA) in 29%, refractory cytopenia of childhood (RCC) in 58%, and refractory cytopenia with multilineage dysplasia (RCMD) in 13%.
  • No significant differences in clinical/laboratory findings or diagnosis distribution were observed between Japanese and Chinese children.
  • Patients diagnosed with RCC/RCMD exhibited milder disease severity and less bone marrow hypocellularity compared to those with AA.

Conclusions:

  • The study established diagnostic consensus for pediatric bone marrow failure syndromes, identifying RCC/RCMD as distinct entities from AA.
  • RCC/RCMD patients demonstrated a potentially milder clinical presentation than AA patients.
  • Further prospective studies comparing clinical outcomes between AA and RCC groups are essential for establishing provisional entities for RCC.

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