Single agent liposomal-encapsulated (Lipo) all-trans retinoic acid (ATRA) can cure patients with untreated acute promyelocytic leukemia (APL): An update and comparison with an ATRA+idarubicin induction regimen
Authors: A. M. Tsimberidou, M. Tirado-Gomez, M. Andreeff, S. O'Brien, H. M. Kantarjian, M. J. Keating, G. Lopez-Berestein, E. Estey
Journal: Journal of Clinical Oncology (2004)

Article Tools Leukemia, Lymphoma, and Transplantation (Adult) Article Tools OPTIONS & TOOLS Export Citation Track Citation Add To Favorites Rights & Permissions COMPANION ARTICLES No companion articles ARTICLE CITATION DOI: 10.1200/jco.2004.22.90140.6513 Journal of Clinical Oncology - published online before print July 15, 2004 PMID: 28016919 Single agent liposomal-encapsulated (Lipo) all-trans retinoic acid (ATRA) can cure patients with untreated acute promyelocytic leukemia (APL): An update and comparison with an ATRA+idarubicin induction regimen A. M. TsimberidouxA. M. TsimberidouSearch for articles by this author , M. Tirado-GomezxM. Tirado-GomezSearch for articles by this author , M. AndreeffxM. AndreeffSearch for articles by this author , S. O'BrienxS. O'BrienSearch for articles by this author , H. M. KantarjianxH. M. KantarjianSearch for articles by this author , M. J. KeatingxM. J. KeatingSearch for articles by this author , G. Lopez-BeresteinxG. Lopez-BeresteinSearch for articles by this author , E. EsteyxE. EsteySearch for articles by this author Show More U Texas M. D. Anderson Cancer Center, Houston, TX https://doi.org/10.1200/jco.2004.22.90140.6513 Abstract Abstract 6513 Background: There is interest in using "targeted therapy" in leukemia in order to avoid non-specific toxicity. This is particularly plausible in APL because of the availability of ATRA and arsenic trioxide. Methods: Lipo-ATRA 90mg/m2 I.V. every other day (Estey, Blood 94: 2230, 1999) produced a complete response (CR) rate of 79% in untreated APL. Once in CR, patients continued to receive Lipo-ATRA 90mg/m2 I.V. thrice weekly for 9 months with idarubicin added only if a PCR test for PML-RARA (sensitivity level, 10-4) performed at 3, 6 or 9 months from hematologic CR was positive. Results were compared with those obtained with a standard ATRA+idarubicin induction regimen (ATRA 45 mg/m2 daily until CR and idarubicin 12 mg/m2 daily for 4 days beginning on day 5 of ATRA). Patients in CR received two courses of idarubicin 12 mg/m2 daily for 3 days and 2 years of maintenance with three cycles of mercaptopurine, vincristine, methotrexate, and prednisone (POMP) alternating with one cycle of idarubicin. Results: Of particular note, 10 of the 26 responders (38%) with available PCR data for monitoring of PML-RARA (lost to follow-up, n=1 responder) remain relapse-free and alive after a median follow-up of 4.2 years (range, 2.6–5.6) despite never receiving idarubicin or other chemotherapy. Conclusions: These results, which are considerably superior to those reported with single agent oral ATRA (Fenaux, Leukemia, 6, Suppl. 1:64, 1992) indicate that some patients with APL are potentially cured with Lipo-ATRA without need for cytotoxic chemotherapy. Author Disclosure Employment or Leadership Consultant or Advisory Stock Ownership Honoraria Research Funding Expert Testimony Other Remuneration Antigenics Antigenics American Society of Clinical Oncology