On Location
American Society of Hematology’s
59TH ANNUAL MEETING & EXPOSITION
James R. Downing, MD, presents the E. Donnall Thomas Lecture.
he 59th ASH Annual Meeting
& Exposition featured practice-
changing research from across
the spectrum of hematologic
malignancies and blood disorders. In this
issue, ASH Clinical News is taking a look
at the plenary and late-breaking abstracts
presented at the meeting, including
exciting results with an oral KIT D816V
inhibitor in systemic mastocytosis and
the possibility of using daratumumab as
frontline treatment for myeloma.
Visit ashclinicalnews.org/on-location for
our complete coverage of the meeting.
ASH Clinical News also spoke with
presenters and moderators about the
groundbreaking research that was shared at
the meeting. Check out all of the interviews
at ashclinicalnews.org/multimedia. And
look for more coverage in our special issue
in mid-January, where we’ll take a deep dive
into the 59th ASH Annual Meeting.
44
ASH Clinical News
Phase I Trial Shows
“Exciting” Results With
Avapritinib in Advanced
Systemic Mastocytosis
The oral KIT D816V inhibitor avapritinib (formerly known as BLU-285)
could provide an alternative treatment option for patients with advanced
systemic mastocytosis (SM), including those whose disease progressed
after treatment with midostaurin. Results from an ongoing phase I trial
were presented as a plenary abstract at the 2017 ASH Annual Meeting.
“We are seeing a high rate of rapid and durable responses, with a low rate
of adverse side effects, in patients with an advanced or aggressive form of the
disease,” said lead study author and presenter Daniel J. DeAngelo, MD, PhD,
director of clinical and translational research at the Dana-Farber Cancer
Institute in Boston. “All patients had some response, which is really excit-
ing in a phase I clinical trial.”
As of November 27, 2017 (data cutoff), 32 adult patients (median
age = 63 years; range = 34-83 years) with SM (28 of whom had advanced
SM per World Health Organization [WHO] diagnostic criteria) were
evaluated in a 3+3 dose-escalation and -expansion design to establish
the safety and maximum tolerated dose (MTD; primary endpoint) of
avapritinib.
Twenty-eight patients had a KIT mutation (88%), and 14 had high-risk
mutations, defined as one or more co-occurring mutations in the bone mar-
row (BM), the most frequent of which were ASXL1, SRSF2, and RUNX1.
January 2018