Asciminib and pulsed ATRA as post-remission therapy in T315I mutated PML-RARA-positive blast crisis of chronic myeloid leukemia

Asciminib 和脉冲式 ATRA 作为 T315I 突变型 PML-RARA 阳性慢性粒细胞白血病急变期的缓解后治疗

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Abstract

Promyelocytic blast crisis (BC) of CML is an extremely rare event, with only seven cases described as arising during therapy with TKIs. We present a 68-year-old male who developed promyelocytic blast crisis 12 months after CML diagnosis and start of Imatinib therapy, confirmed by the concomitant presence of the t(15;17) and t(9;22) translocations in the leukemic cells. Molecular remission for the PML-RARA clone was achieved with standard acute promyelocytic leukemia induction therapy with ATRA and idarubicin. However, BCR-ABL showed resistance to first-line Dasatinib and second-line Ponatinib, principally due to the presence of multiple mutations in ABL1 kinase domain, including T315I. Hematological and molecular response was achieved with Asciminib, a first‐in‐class STAMP (Specifically Targeting the ABL Myristoyl Pocket) inhibitor in combination with pulsed ATRA as post-remission strategy. Of the 7 cases of promyelocytic BC reported in the era of TKI therapy for CML, this is the first case effectively treated with Asciminib therapy.

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