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PLX51107 is a highly effective oral bromine domain inhibitor with moderate selectivity for BD1, the first N-terminal bromine domain of bet family proteins
A 70-year-old woman with a 3-year history of isolated mononucleosis was diagnosed with cutaneous leukemia
When the authors visited their facility, there were many purple 2-5 cm tumors and plaques on the trunk and limbs (Figure 1a
Figure 1: a.
Figure 2: a.
BM biopsy is negative for AML involvement
Patients received the BET inhibitor PLX51107 120 mg PO daily on days 1 to 21 and AZA 75 mg/m2 IV every
Overall, the response of patients to the treatment of skin diseases with PLX51107/AZA was an improvement in clinical response and overall well
In this case, a significant response to bet inhibitors in combination with AZA highlights the possibility of improving skin-associated leukemia activity
A Phase 2 clinical trial evaluating PLX51107 and AZA in AML and MDS is currently underway (NCT04022785
Original source:
Kim K, Bazinet A, Loghavi S, Konopleva M, Bhalla K, Daver N, Khoury JD, Kadia T, Wilson NR, Curry JL, Heberton M, Miller D, Pierce S, Borthakur G, Pemmaraju N.