Transient Leukemia (TL) occurs in approximately 10% of infants with Down syndrome(DS). Most of cases achieve spontaneous remission, but there are severe, potentially lethal cases which manifest hepatic fibrosis or cardiopulmonary failure. COG reported the incidence of 17% early death in 48 TL cases in DS, but no other large case series reported till now. We retrospectively analyzed Japanese 70 cases of TL in DS to identify the prognostic factors associated with early death. From 1992 to 2006, 46 boys and 24 girls were treated in 14 Japanese institutes. 16 cases (22.9%) were dead in their early life, and 12cases (17.1%) developed AMKL. One patient died with relapsed AMKL, so the overall survival rate was 75.7%. Univariate analysis showed that mechanical ventilation (p=0.001), pulmonary hypertension (p=0.0105), low birth weight (p=0.0063), early gestational age (p=0.0016), low Apgar score at 1min (p=0.0003), low Apgar score at 5min (p=0.0057), high WBC count (p=0.0006), elevated aspartate aminotransferase(AST) (p=0.0004) and elevated direct bilirubin (p<0.0001) are associated with early death of TL in DS. Multivariate proportional hazard model identified gestational age (p=0.0266; RR 0.708) and elevated direct bilirubin (p=0.0487; RR 1.076) as independent prognostic factors. Prospective clinical study is needed to find the optimal therapeutic approach to save neonates with TL who have these poor prognostic factors.

Topics:
down syndrome, myeloproliferative syndrome, transient, newborn, prognostic factors, apgar score low, direct bilirubin, aspartate aminotransferases, cardiorespiratory failure, hepatic fibrosis, mechanical ventilation

Disclosure: No relevant conflicts of interest to declare.

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2006, The American Society of Hematology
2006
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