Latest & Greatest
Ibrutinib and
Obinutuzumab Combo
Granted Approval for
CLL/SLL
The U.S. Food and Drug Administration
(FDA) approved the combination of
ibrutinib and obinutuzumab for the treat-
ment of patients with treatment-naïve
chronic lymphocytic leukemia/small
lymphocytic lymphoma (CLL/SLL).
The agency’s decision was based on
results from the phase III iLLUMINATE
trial, which compared the efficacy of the
ibrutinib-obinutuzumab combination
with chlorambucil plus obinutuzumab
in 229 patients with treatment-naïve
CLL/SLL. At a median follow-up of 31
months (interquartile range = 29.4-33.2
months), the median progression-free
survival was significantly longer in the
ibrutinib-treated patients (not reached
vs. 19 months; p<0.0001). This translated
to a significantly lower risk of disease
progression or death with the ibrutinib-
based combination (hazard ratio = 0.23;
95% CI 0.15-0.37; p<0.0001).
The most common adverse events
(AEs; occurring in ≥20% of patients) in the
ibrutinib-treated arm included neutro-
penia, thrombocytopenia, rash, diarrhea,
musculoskeletal pain, bruising, cough,
infusion-related reaction, hemorrhage, and
arthralgia.
Source: Janssen press release, January 28, 2019.
FDA Approves
Caplacizumab-yhdp
for Acquired TTP
Caplacizumab-yhdp, a bivalent anti–von
Willebrand factor nanobody, was approved
for the treatment of adult patients with ac-
quired thrombotic thrombocytopenic pur-
pura (aTTP), in combination with plasma
exchange and immunosuppressive therapy.
The FDA’s decision was based on results
from the multicenter, randomized, double-
blind, placebo-controlled HERCULES
trial, which randomized 145 patients to
receive either caplacizumab-yhdp (n=72)
or placebo (n=73). All participants also
received plasma exchange and immuno-
suppressive therapy.
Per study protocol, patients received
caplacizumab-yhdp 11 mg or placebo as
a single 11-mg bolus intravenous injec-
tion prior to the first plasma exchange,
followed by a daily subcutaneous injection
of caplacizumab-yhdp or placebo after
completion of plasma exchange, for the
duration of the daily plasma exchange
period and for 30 days subsequently. If pa-
tients had persistent signs of aTTP (such
as suppressed ADAMTS13 activity levels),
treatment was extended for seven-day
intervals for a maximum of 28 days.
In patients who received caplacizumab-
yhdp, the time to platelet-count response
(defined as a platelet count ≥150×10 9 /L
plus cessation of daily plasma exchange
within 5 days) was significantly shorter
than those who received placebo: 2.69
days (95% CI 1.89-2.83] versus 2.88 days
(95% CI 2.68-3.56; p=0.01).
Caplacizumab-yhdp also resulted in a
lower number of TTP-related deaths (0 vs.
3; p<0.001) and TTP recurrence (3 vs. 28;
p<0.001) during the treatment period.
The most common AEs (occurring ≥15%
of patients) associated with caplacizumab-
yhdp were epistaxis, headache, and
gingival bleeding.
The agent was approved through
priority review and with orphan product
designation.
Source: FDA news release, February 6, 2019.
Liquid Biopsy Test
Cleared for Use in CML
The FDA approved the QXDx AutoDG
ddPCR System, a liquid biopsy test
designed to precisely and reproducibly
monitor molecular response to treatment
in patients with chronic myeloid leukemia
(CML). This clearance represents the first
digital polymerase chain reaction (PCR)
to receive approval in the U.S.
Standard monitoring of response in
patients with CML includes using reverse
transcription quantitative PCR. The
QXDx liquid biopsy system demonstrated
that it produced reliable results, even
when measuring low levels of disease.
Source: Bio-Rad Laboratories press release, February 14, 2019.
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Visit www.hematology.org/Patients/FAH.aspx
to add your information!
14
ASH Clinical News
April 2019