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Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Acute Promyelocytic Leukemia

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Molecular Variants of APL Other than PML-RARA

Uncommon molecular variants of APL produce fusion proteins that join distinctive gene partners (e.g., PLZF, NPM, STAT5B, and NuMA) to RARA.[55] Recognition of these rare variants is important as they differ in their sensitivity to ATRA and to arsenic trioxide.[56] The PLZF-RARA variant, characterized by t(11;17)(q23q21), represents about 0.8% of APL, expresses surface CD56, and has very fine granules compared with t(15;17) APL.[57,58,59] APL with PLZF-RARA has been associated with a poor prognosis and does not usually respond to ATRA or to arsenic trioxide.[56,57,58,59] The rare APL variants with NPM-RARA (t(5;17)(q35;q21)) or with NuMA-RARA (t(11;17)(q13;q21)) translocations are responsive to ATRA.[56,60,61,62,63]

Treatment Options Under Clinical Evaluation

The following is an example of a national and/or institutional clinical trial that is currently being conducted. Information about clinical trials is available from the NCI Web site.

  • COG-AAML0631(Combination Chemotherapy in Treating Young Patients With Newly Diagnosed APL): The Children's Oncology Group is conducting a study evaluating the addition of two courses of arsenic trioxide plus ATRA to a backbone treatment regimen based on the Italian AIDA treatment regimen,[64] but with modifications to reduce the cumulative doses of anthracyclines. The primary objective is to decrease the total anthracycline dose from that used in regimens with the best current published results while still maintaining a comparable EFS. Promising results from pilot studies using arsenic trioxide and ATRA in newly diagnosed patients with APL also support evaluation of this combination.[45,46,47]

Current Clinical Trials

Check for U.S. clinical trials from NCI's list of cancer clinical trials that are now accepting patients with childhood acute promyelocytic leukemia (M3). The list of clinical trials can be further narrowed by location, drug, intervention, and other criteria.

General information about clinical trials is also available from the NCI Web site.

References:

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Last Updated: February 25, 2014
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