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Successful Treatment With ATRA and Arsenic Trioxide for a Child With Down Syndrome and Acute Promyelocytic Leukemia

Tsumura, Yusuke MD; Yamada, Yuji MD; Osumi, Tomoo MD; Kato, Motohiro MD, PhD; Terashima, Keita MD, PhD; Shioda, Yoko MD, PhD; Kiyotani, Chikako MD, PhD; Matsumoto, Kimikazu MD, PhD; Tomizawa, Daisuke MD, PhD

Journal of Pediatric Hematology/Oncology: February 22, 2019 - Volume Publish Ahead of Print - Issue - p
doi: 10.1097/MPH.0000000000001438
Clinical and Laboratory Observations: PDF Only

Acute promyelocytic leukemia (APL) is rare in patients with Down syndrome (DS). Cytotoxic chemotherapy combined with all-trans retinoic acid (ATRA) has been a standard treatment for APL, but is potentially intolerable for DS patients because of their vulnerability to cytotoxic agents. We report here a case of a 10-year-old girl with DS and APL successfully treated with a combination of ATRA and arsenic trioxide, a therapy emerging as a new standard for APL. She achieved molecular remission and completed the therapy without significant toxicities. ATRA/arsenic trioxide combination therapy would be a preferable option for DS patients with APL.

Children’s Cancer Center, National Center for Child Health and Development, Tokyo, Japan

Off-label use/unapproved drugs or products: the manuscript includes off-label use of all-trans retinoic acid and arsenic trioxide.

The authors declare no conflict of interest.

Reprints: Daisuke Tomizawa, MD, PhD, Division of Leukemia and Lymphoma, Children’s Cancer Center, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan (e-mail:

Received November 10, 2018

Accepted January 9, 2019

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