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Acute Promyelocytic Leukemia

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Induction and consolidation therapies result in molecular remission as measured by retrotranscriptase polymerase chain reaction (RT-PCR) for PML/RAR-alpha in the large majority of APL patients. While two negative RT-PCR assays after completion of therapy are associated with long-term remission,[5] conversion from negative to RT-PCR positivity is highly predictive of subsequent hematologic relapse.[47] Patients with persistent or relapsing disease based upon PML/RAR-alpha RT-PCR measurement may benefit from intervention with relapse therapies (see the APL section of Recurrent Childhood Acute Myeloid Leukemia).

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: The Children's Oncology Group will be conducting a study, COG-AAML0631, evaluating the addition of two courses of arsenic trioxide plus ATRA to a backbone treatment regimen based on the Italian AIDA treatment regimen,[48] 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.[43,49]

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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  25. Wells RA, Catzavelos C, Kamel-Reid S: Fusion of retinoic acid receptor alpha to NuMA, the nuclear mitotic apparatus protein, by a variant translocation in acute promyelocytic leukaemia. Nat Genet 17 (1): 109-13, 1997.
  26. Wells RA, Hummel JL, De Koven A, et al.: A new variant translocation in acute promyelocytic leukaemia: molecular characterization and clinical correlation. Leukemia 10 (4): 735-40, 1996.
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  33. Tallman MS, Kim HT, Montesinos P, et al.: Does microgranular variant morphology of acute promyelocytic leukemia independently predict a less favorable outcome compared with classical M3 APL? A joint study of the North American Intergroup and the PETHEMA Group. Blood 116 (25): 5650-9, 2010.
  34. Powell BL, Moser B, Stock W, et al.: Effect of consolidation with arsenic trioxide (As2O3) on event-free survival (EFS) and overall survival (OS) among patients with newly diagnosed acute promyelocytic leukemia (APL): North American Intergroup Protocol C9710. [Abstract] J Clin Oncol 25 (Suppl 18): A-2, 2007.
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  48. Mandelli F, Diverio D, Avvisati G, et al.: Molecular remission in PML/RAR alpha-positive acute promyelocytic leukemia by combined all-trans retinoic acid and idarubicin (AIDA) therapy. Gruppo Italiano-Malattie Ematologiche Maligne dell'Adulto and Associazione Italiana di Ematologia ed Oncologia Pediatrica Cooperative Groups. Blood 90 (3): 1014-21, 1997.
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Last Updated: May 16, 2012
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