The road not taken: Exploring non-transplant options in De Novo philadelphia positive acute myeloid leukemia

Abstract

Acute myeloid leukemia (AML) is a heterogeneous disease with diverse molecular cytogenetic characteristics. Philadelphia-positive acute myeloid leukemia, a rare subtype of AML, is traditionally considered a high-risk, with the standard recommendation being an allogeneic hematopoietic cell transplant (HCT) in first remission. More recently, with better characterization and understanding of AML biology, novel therapies have been introduced. Drawing parallels from the advances seen in Philadelphia-positive acute lymphoblastic leukemia (ALL), the question arises whether potent tyrosine kinase inhibitors (TKI), such as ponatinib, in combination with AML-directed therapies, could be used in Philadelphia-positive AML, potentially eliminating the need for HCT in the first remission.

In this report, we review the literature on Philadelphia-positive AML, study a case where HCT was omitted, and explore potential signals that could support successful HCT omission.