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Clinical Case Report
Evidence of Myeloid Differentiation in non-M3 Acute Myeloid Leukemia Treated with the Retinoid X Receptor Agonist Bexarotene
Donald E. Tsai, Selina Luger, Allison Kemner, Cezary Swider, Ami Goradia, Ewa Tomczak, Doris DiPatri, Adam Bagg, Peter Nowell, Alison W. Loren, Alexander Perl, Stephen Schuster, James E. Thompson, David Porter, Charlambos Andreadis, Edward A. Stadtmauer, Steven Goldstein, Richard Ghalie and Martin Carroll
volume 6 | issue 1
January 2007Pages: 18 - 21
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All-trans-retinoic acid has dramatically changed the treatment paradigm for acute promyelocytic leukemia, however, it has no significant activity in non-M3 acute myeloid leukemia (AML). In vitro, bexarotene, a retinoid X receptor agonist inhibits the proliferation of non-M3 AML cell lines and induces differentiation of leukemic blasts from patients. We hypothesized that there may be similar activity in patients with AML. We report on 2 patients with relapsed or refractory non-M3 AML treated with bexarotene monotherapy. After initiating treatment, both patients showed leukemic differentiation in their peripheral blood and reduction in bone marrow blasts to less than 5%. One patient had a significant improvement in her platelet count with loss of platelet transfusion needs. Differentiation syndrome occurred in one patient and was successfully treated with steroids and discontinuation of bexarotene. These data suggest that bexarotene has clinical activity in non-M3 AML and may be able to induce myeloid differentiation in vivo.
Authors
Donald E. Tsai
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Selina Luger
University of Pennsylvania Medical Center; Philadelphia, Pennsylvania
Allison Kemner
University of Pennsylvania Medical Center; Philadelphia, Pennsylvania
Cezary Swider
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Ami Goradia
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Ewa Tomczak
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Doris DiPatri
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Adam Bagg
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Peter Nowell
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Alison W. Loren
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Alexander Perl
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Stephen Schuster
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
James E. Thompson
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
David Porter
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Charlambos Andreadis
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Edward A. Stadtmauer
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Steven Goldstein
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
Richard Ghalie
Ligand Pharmaceuticals, San Diego, California
Martin Carroll
University of Pennsylvania Medical Center, Philadelphia, Pennsylvania
We now provide open access to journal articles published online for one year or more. This article may be downloaded at the following link:
Download PDFIf the document does not open, please right-click on the link (control-click on a Macintosh) and select the option to save the file to disk.