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Blood Adv. 2018 Jul 10;2(13):1532-1540. doi: 10.1182/bloodadvances.2018018945.

Low-dose cytarabine to prevent myeloid leukemia in children with Down syndrome: TMD Prevention 2007 study.

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Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
Pediatric Hematology and Oncology, Pediatrics III, University Hospital Essen, Essen, Germany.
Dutch Childhood Oncology Group, Den Haag, The Netherlands.
Department of Immunology, Laboratory Medical Immunology, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Department of Pediatric Psychiatry and Psychosomatic Medicine, Landschaftsverband-Rheinland Clinic, University Duisburg/Essen, Essen, Germany.
Department of Pediatric Oncology/Hematology, Erasmus Medical Center/Sophia Children's Hospital, Rotterdam, The Netherlands; and.
Department of Pediatric Hematology and Oncology, Martin Luther University Halle-Wittenberg, Halle, Germany.


Approximately 5% to 10% of children with Down syndrome (DS) are diagnosed with transient myeloproliferative disorder (TMD). Approximately 20% of these patients die within 6 months (early death), and another 20% to 30% progress to myeloid leukemia (ML-DS) within their first 4 years of life. The aim of the multicenter, nonrandomized, historically controlled TMD Prevention 2007 trial was to evaluate the impact of low-dose cytarabine treatment on survival and prevention of ML-DS in patients with TMD. Patients received cytarabine (1.5 mg/kg for 7 days) in case of TMD-related symptoms at diagnosis (high white blood cell count, ascites, liver dysfunction, hydrops fetalis) or detection of minimal residual disease (MRD) 8 weeks after diagnosis. The 5-year probability of event-free and overall survival of 102 enrolled TMD patients was 72 ± 5% and 91 ± 3%, respectively. In patients eligible for treatment because of symptoms (n = 43), we observed a significantly lower cumulative incidence (CI) of early death as compared with symptomatic patients in the historical control (n = 45) (12 ± 5% vs 33 ± 7%, PGray = .02). None of the asymptomatic patients in the current study suffered early death. However, the treatment of symptomatic or MRD-positive patients did not result in a significantly lower CI of ML-DS (25 ± 7% [treated] vs 14 ± 7% [untreated], PGray = .34 [per protocol analysis]; historical control: 22 ± 4%, PGray = .55). Thus, low-dose cytarabine treatment helped to reduce TMD-related mortality when compared with the historical control but was insufficient to prevent progression to ML-DS. This trial was registered at EudraCT as #2006-002962-20.

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