A Clinical Observation of Childhood Myelodysplastic Syndrome

Park, Sun Young; Whang, Pil Geung; Lee, Soon Yong

Abstract

BACKGROUND: Childhood myelodysplastic syndrome(MDS) is a heterogenous disease complex which has characteristics of cytopenia in one or more hemopoietic cell lines in peripheral blood and of dysmorphisms of hemopoietic precursors in bone marrow, but quite different from adult one. We experienced 22 patients with childhood myelodysplastic syndrome during 9 years from 1987 to 1995 in the Department of Pediatrics, Inje University College of Medicine, Pusan Paik Hospital, Pusan, Korea and performed a clinical study about them to analyze the clinical and hematological features and treatment outcome retrospectively. METHOD: We analyzed their clinical features including age and sex distribution, chief complaints and physical findings on first admission, distribution of subtypes, hematologic features including initial hemoglobin, total WBC and platelet count, and treatment outcome including leukmic transformation and prognostic scores.
RESULTS
1) Of the total 22 patients, 10 were in 0-4 years of age on first admission, 8 in 5-9, and 4 above 10 years of age. The male : female sex ratio was 2.1 : 1. 2) Of the 22 patients, chief complaints on first admission were bleeding tendency in 9 patients(40.9%), pallor in 8(36.4%) and fever in 3(13.6%), in order, and physical findings on first admission were purpura or ecchymoses in 17(77.3%), anemia in 15(68.2%) and hepatosplenomegaly in 8(36.4%), in order. 3) Hematologic findings on first admission were as follows : hemoglobin levels were below 3 g/dl in 1 patient(4.5%), 3-6 g/dl in 10(45.5%), and 6-9 g/dl in 11(50.0%). Initial WBC counts were below 5,000/mm3 in 13 patients(59.1%), 5,000-10,000/mm3 in 5(22.7%) and above 10,000/mm3 in 4(18.2%). Initial platelet counts were below 20,000/mm3 in 10(45.5%), 20,000-50,000/mm3 in 5(22.7%), 50,000-100,000/mm3 in 5(22.7%) and above 100,000/mm3 in 2(9.1%). 4) Of the 22 patients, 12 patients(54.6%) were RA type, 1(4.6%) RAS, and 3(13.6%) RAEB, RAEB-T and JCML types, respectively. 5) According to prognostic scores by Mufti et al(1986), none were in 'good' group, 17 patients(89.5%) in 'intermediate' group with 39.5 months of mean duration of survival(range 4-95 months) and 2(10.5%) in 'poor' group with 18 months of mean duration of surviral(range 17-19) until the last follow-up. However, the subtypes and clinical status seemed not to be related to the prognostic scores. 6) Sixteen patients were treated with low dose cytosine arabinoside(10 mg/m2/12hrs), of whom 7 patients gained long-standing event-free survival, whose treatment regimen was changed to oral 6-TG about 2 years later. All 3 of JCML were treated with A-Triple-V regimen, one of whom was died of sepsis, one was transformed into AML and died of sepsis, while the remained one gained long-standig event-free survival (62 months). 7) Leukemic transformation into AML occured in 7 patients(RA 1, RAEB 2, RAEB-T 3, JCML 1), 6 of whom were dead, while one gained long-standing event-free survival of 34 months.
CONCLUSION
We concluded that RA was the most dominant type among our patients, and the frequency to transform into AML was 31,8%, and 31.9% of the patients had long-term survival, and that reliability of prognostic scoring system by Mufti et al(1986) was not high.