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Wilms’ tumor gene 1 ( WT1 ) expression is a well-known predictor for relapse in acute myeloid leukemia. We monitored  WT1  decrement along the treatment course to identify its significant role as a marker for residual disease in acute promyelocytic leukemia (APL) and tried to suggest its significance for relapse prediction. In this single center retrospective study, we serially measured  PML-RARa  and  WT1  expression from 117 APL patients at diagnosis, at post-induction and post-consolidation chemotherapies, and at every 3 months after starting maintenance therapy. All 117 patients were in molecular remission after treatment of at least 2 consolidation chemotherapies. We used  WT1  ProfileQuant™ kit (Ipsogen) for  WT1  monitoring. High  WT1  expression (>120 copies/10 4   ABL1 ) after consolidation and at early period (3 months) after maintenance therapy significantly predicted subsequent relapse. All paired  PML-RARa  RQ-PCR were not detected except for one sample with early relapse. Patients with high  WT1  expression at 3 months after maintenance therapy ( n  = 40) showed a significantly higher relapse rate (30.5 vs. 6.9%,  P  < 0.001) and inferior disease free survival (62.8 vs. 91.4%,  P  < 0.001). Multivariate analysis revealed that high peak leukocyte counts at diagnosis (HR = 6.4,  P  < 0.001) and high  WT1  expression at 3 months after maintenance therapy (HR = 7.1,  P  < 0.001) were significant factors for prediction of relapse. Our data showed high post-remission  WT1  expression was a reliable marker for prediction of subsequent molecular relapse in APL. In this high-risk group, early intervention with ATRA ± ATO, anti-CD33 antibody therapy, and  WT1 -specific therapy may be used for relapse prevention.    Trial registration  Clinical Research Information Service (CRIS), KCT0002079    Electronic supplementary material  The online version of this article (doi:10.1186/s13045-017-0404-4) contains supplementary material, which is available to authorized users.

journal of hematology and oncology

High WT1 expression is an early predictor for relapse in patients with acute promyelocytic leukemia in first remission with negative PML-RARa after anthracycline-based chemotherapy: a single-center cohort study