CLINICAL NEWS
self-limited gastrointestinal (GI) events (in-
cluding diarrhea [n=27; 55%] and nausea
[n=25; 51%]) and upper respiratory tract
infections (n=28; 55%).
Seventy-six percent of patients (n=37)
experienced grade 3/4 AEs, the most
common of which were peripheral blood
cytopenias, including neutropenia (n=26;
53%), thrombocytopenia (n=8; 16%), ane-
mia (n=7; 14%), febrile neutropenia (n=6;
12%), and leukopenia (n=6; 12%). The
most common serious AEs were pyrexia
(n=6; 12%), febrile neutropenia (n=5;
10%), lower respiratory tract infection
(n=3; 6%), and pneumonia (n=3; 6%).
Five patients experienced tumor lysis
syndrome (TLS), which resulted in one
death. Following those events, the study
protocol was amended to include a lower
starting dose of venetoclax (20 mg) and
modified TLS prophylaxis. Among the
32 patients in whom venetoclax was
resumed, no clinical TLS was observed.
Twenty patients (41%) had dose
reductions, most commonly because of
cytopenias (n=9; 18%) and GI events
(n=4; 6%). Three deaths were reported,
from metabolism and nutrition disorders
(n=1) and neoplasms (n=2).
Time to first response was 2.9 months
(range = 0.7-15.7 months), and CRs
were attained for a median of 9.2 months
(range = 6.4-28.6 months). By the end of
therapy, 25 patients (51%) achieved CR
or CR with incomplete marrow recovery
(CRi) as best response. The best respons-
es according to venetoclax dose level
were as follows:
• 200 mg: 33% CR/CRi, 67%
nodular PR/PR
• 300 mg: 50% CR/CRi, 30%
nodular PR/PR, 10% stable
disease
• 400 mg: 75% CR/CRi, 13% stable
disease (not assessed in 1 patient)
• 500 mg: 57% CR/CRi, 29%
nodular