Latest & Greatest
NCCN Releases First
Set of Guidelines for
Myeloproliferative
Neoplasms
The National Comprehensive Cancer Network (NCCN) released its first guidelines
for myeloproliferative neoplasms (MPNs),
outlining diagnosis, treatment, and supportive care strategies for myelofibrosis
(MF). Comprehensive recommendations
for the management of essential thrombocytopenia and polycythemia vera will
be included in subsequent versions of the
MPN guidelines, which are projected to
be released in early 2017.
“Treatment of MPNs in the United
States remains heterogeneous, and it has
largely been driven by review articles,
individual opinion pieces, and, more
recently, phase III clinical trial data,”
Ruben A. Mesa, MD, professor of medicine at the Mayo Clinic Cancer Center in
Phoenix, Arizona, and chair of the NCCN
Guidelines Panel for MPN, told ASH
Clinical News.
Dr. Mesa outlined the goals for the
new guidelines including helping doctors
who may not have experience managing these disorders. “We are hopeful [the
guidelines] will provide a service for clinicians treating MPNs, particularly those
for whom managing patients with MPNs
is a small part of their practice. Next, we
hope these new guidelines highlight areas
that continue to require clinical investigation, and, third, that they will act as a
standard of care to assist with coverage
decisions by both insurance providers and
government payers.”
The guidelines stress the importance
of using available scoring systems (the
International Prognostic Scoring System
or the Dynamic International Prognostic Scoring System) to make an accurate
diagnosis and prognosis throughout the
course of the disease.
The guidelines support ruxolitinib for
symptomatic patients at each risk level
(low-risk, intermediate-1, intermediate-2,
or high-risk) and in all patients who have
intermediate-2 or high-risk disease. The
guidelines also specify that allogeneic
hematopoietic cell transplantation is an
option for patients with intermediate-1,
intermediate-2, or high-risk disease, but
the guidelines caution that “the selection
of patients for allogeneic hematopoietic
cell transplantation should be based on
age, performance status, major comorbid
conditions, psychosocial status, patient
preference, and availability of caregiver.”
The guidelines also highlight areas in
which more evidence to guide treatment is
needed. “Progressive MF to either an accelerated or overt blast phase continues to
be a very difficult clinical scenario to treat
and an important area of investigation and
need for clinical trials,” said Dr. Mesa.
Read the guidelines at nccn.org/
professionals/physician_gls/pdf/mpn.pdf.
Source: National Comprehensive Cancer Network. Clinical Practice
Guidelines in Oncology: Myeloproliferative Neoplasms. Version 1.2017,
September 26, 2016.
Investigation
Underway for WHO’s
International Agency
for Research on Cancer
U.S. government officials opened
an investigation of funding for the
World Health Organization’s (WHO)
International Agency for Research on
Cancer (IARC), which is facing criticism
over its classification of carcinogens.
Officials from the National Institutes of
Health (NIH) have agreed to provide
an in-person briefing to the committee
regarding its ongoing funding to the
controversial agency. The committee
has asked NIH to detail its standards for
awarding grants and subsequently vetting
recipients.
IARC classified coffee, mobile phones,
processed meats, and glyphosate as potential carcinogens, which has prompted
critics to argue that the agency is too quick
to conclude that certain substances might
cause cancer. These actions, they note, can
lead to unnecessary health scares.
In a letter to NIH, Rep. Jason
Chaffetz, chairman of the U.S. House
Committee on Oversight and Government
Reform, described IARC as having “a
record of controversy, retractions, and
inconsistencies. IARC’s standards and
determinations for classifying substances
as carcinogenic … appear inconsistent with
other scientific research.”
NIH’s grant database shows that
IARC has received more than $1.2 million from them in 2016, and, since 1992,
NIH has granted nearly $40 million in
funding to IARC.
Source: Reuters, October 6, 2016.
ASH Submits
Comments to FDA on
NGS-Based Tests for
Germline Diseases
The American Society of Hematology (ASH)
submitted comments to the FDA supporting the agency’s draft guidance for nextgeneration sequencing (NGS)-based tests for
germline diseases. ASH’s comments ask for
clarification and make suggestions for other
recommendations to be included in the final
guidance so that NGS-based tests can be effectively designed, developed, and validated
for various hematologic diseases.
ASH suggested that the FDA:
FDA Grants Breakthrough Designation Therapy for Novel
Bone Marrow Transplant Graft Modality
The U.S. Food and Drug Administration (FDA)
granted breakthrough designation therapy for
NiCord , a novel graft modality for hematopoietic cell transplants in patients with hematologic
malignancies. NiCord is an ex vivo expanded cell
graft that uses nicotinamide platform technology,
which expands cells from the umbilical cord blood.
NiCord is in development as an alternative to a hematopoietic cell transplant for patients who cannot
find a donor with fully matched tissue.
The FDA’s decision was based on data from
an international, multicenter, phase I/II study.
Sixteen patients who were 12 to 65 years old
with high-risk hematologic malignancies were
included in the study. They received a transplant
with NiCord as a standalone graft following
myeloablative therapy. One year after transplant,
patient outcomes were compared with outcomes
from a control group of 125 similar patients enrolled in the Center for International Blood and
Marrow Transplant Research registry who were
transplanted with cord blood.
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ASH Clinical News
Patients who received a transplant with NiCord
experienced a 101-fold increase in the number of
CD34+ cells compared with patients who received
a transplant with unmanipulated cord blood. Those
in the NiCord group also demonstrated faster neutrophil engraftment (10 days vs. 21 days p<0.0001)
and faster platelet engraftment (median = 32 vs. 46
days; p<0.001) compared with controls.
At 16 days post-transplant, 75 percent of
NiCord recipients achieved neutrophil engraftment compared with 18 percent in the control
group (p<0.0001), and at 42 days post-transplant,
56 percent versus 27 percent, respectively, achieved
platelet engraftment (p=0.015).
One-year transplant-related mortality was 19
percent for NiCord and 39 percent for the control
group (p=0.12).
An international, multicenter, phase III study of
NiCord is set to begin before the end of the year.
Sources: Gamida Cell press release, October 11, 2016; Gamida Cell press release, June 6,
2016.
December 2016