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Blood, 1 February 2002, Vol. 99, No. 3, pp. 759-767
PERSPECTIVE
Acute promyelocytic leukemia: evolving therapeutic
strategies
Martin S. Tallman,
Chadi Nabhan,
James H. Feusner, and
Jacob M. Rowe
From the Division of Hematology and Oncology, Robert H. Lurie Comprehensive Cancer Center, Northwestern University Medical
School, Chicago, IL; Children's Hospital of Oakland, CA; and the
Department of Hematology and Bone Marrow Transplantation, Technion
Israel Institute of Technology, Rambam Medical Center, Haifa, Israel.
Acute promyelocytic leukemia (APL) is now the most curable subtype
of acute myeloid leukemia in adults. All-trans retinoic acid (ATRA), which induces differentiation of the leukemic cells into
mature granulocytes, represents the important advance. The incorporation of ATRA in induction results in a high complete remission
rate, leads to rapid resolution of the characteristic life-threatening
coagulopathy, and, most importantly, decreases the relapse rate
compared with treatment with chemotherapy alone. However, ATRA is
associated with unique toxicities not observed with conventional
cytotoxic chemotherapy. A number of clinical trials have been performed
to define the optimal role of ATRA in the treatment of patients. The
therapeutic strategies have rapidly evolved as a result of both single
institution and large cooperative group trials. Arsenic trioxide and
stem cell transplantation are effective treatments for patients with
APL who relapse after or are refractory to ATRA-based therapy. As
experience with ATRA and arsenic trioxide in patients with APL
accumulates, a number of important questions arise that need to be addressed.

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