Data Stream
Facts and figures from research presented at the 2017 ASH Annual Meeting
Making CLL More Chronic Hammering Sickling
Treatment with the BCL2 inhibitor venetoclax and rituximab (VR) more than doubled
the likelihood that patients with relapsed/refractory chronic lymphocytic leukemia (CLL)
survived to two years without disease progression, compared with bendamustine and
rituximab (BR). Teenagers with sickle cell disease (SCD)
may have a new treatment option that
addresses the underlying cause of the
disease, according to preliminary results
from a trial of voxelotor. “What sets this
drug apart is that it was designed spe-
cifically for SCD,” said lead study author
Carolyn C. Hoppe, MD. “This is one of
the first drugs that intentionally targets
the fundamental mechanism of SCD.”
Among 389 patients randomized to
receive either VR or the standard chemo-
immunotherapeutic regimen:
Progression-free survival
84.9 %
VR Group
55 %
Of the patients (ages 12-17 years) with
SCD who received at least 16 weeks
of voxelotor treatment, 55% (n=6/11)
experienced a >1 g/dL increase in hemoglobin.
Voxelotor also appeared to improve patients’
quality of life: 83% saw improvement in total
severity score, including 42% who experienced no
symptoms.
83 %
42 %
Safety and tolerability data were reassuring, the authors noted, but results
will need to be confirmed in larger, randomized trials.
Source: Hoppe CC, Inati AC, Brown C, et al. Initial results from a cohort in a phase 2a study
(GBT440-007) evaluating adolescents with sickle cell disease treated with multiple doses of
GBT440, a HbS polymerization inhibitor. Abstract #689. Presented at the 2017 American Society
of Hematology Annual Meeting, December 11, 2017; Atlanta, GA.
36.3 %
BR Group
A Game-Changer for aTTP
Minimal Residual Disease Clearance
83.5 %
VR Group
23.1 %
BR Group
0% 20% 40% 60% 80% 100%
“This is the first randomized trial comparing any of the new agents targeted to treat
CLL against a standard chemoimmunotherapy program,” said lead study author
John Seymour, MBBS, PhD, adding that the results “suggest that venetoclax should
replace chemotherapy for relapsed/refractory CLL.”
The researchers are continuing to monitor participants to assess long-term safety,
survival, and disease progression.
Source: Seymour JF, Kipps TJ, Eichhorst BF, et al. Venetoclax plus rituximab is superior to bendamustine plus
rituximab in patients with relapsed/refractory chronic lymphocytic leukemia - results from pre-planned interim
analysis of the randomized phase 3 Murano study. Abstract LBA-2. Presented at the 2017 American Society of
Hematology Annual Meeting, December 12, 2017; Atlanta, GA.
16
ASH Clinical News
In a phase III trial, treatment with the anti–von Willebrand factor antibody
caplacizumab reduced the immediate risks and prevented organ damage
associated with acquired thrombocytopenic purpura (aTTP).
“This is a real game cha