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Epigenetic Therapy in a Patient With Down Syndrome and Refractory Acute Myeloid Leukemia

Becktell, Kerri, MD*; Houser, Kerri, MD; Burke, Michael J., MD*

Journal of Pediatric Hematology/Oncology: January 2019 - Volume 41 - Issue 1 - p e38–e40
doi: 10.1097/MPH.0000000000001158
Online Articles: Clinical and Laboratory Observations

Acute myeloid leukemia (AML) associated with Down syndrome (DS-AML) is a unique entity of AML with superior treatment response and overall survival compared with children with non–DS-AML. Despite good outcomes in DS-AML, those who relapse or have refractory disease have poor survival. Successful treatment of these patients is challenged by increased incidence of treatment-related toxicities often encountered with high-dose chemotherapy. Here we report the experience of epigenetic modifying agents (decitabine and vorinostat) followed by fludarabine, cytarabine, and granulocyte colony stimulating growth factor for a child with refractory DS-AML. This combination was well tolerated and resulted in a brief clinical response.

*Department of Pediatrics, Division of Hematology/Oncology/Blood and Marrow Transplantation, Medical College of Wisconsin/Children’s Hospital of Wisconsin

Department of Pediatrics, Children’s Hospital of Wisconsin, Milwaukee, WI

Supported by Midwest Athletes Against Cancer (MACC) Fund and the Children’s Hospital of Wisconsin Pediatric Leukemia & Lymphoma Program.

The authors declare no conflict of interest.

Reprints: Kerri Becktell, MD, Department of Pediatrics, Division of Hematology/Oncology/Blood and Marrow Transplantation, Medical College of Wisconsin, 8701 Watertown Plank Road, Milwaukee, WI 53226 (e-mail: kbecktell@mcw.edu).

Received September 18, 2017

Accepted December 17, 2017

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