CLINICAL NEWS
Latest & Greatest
Although this is an early proof-of-principle
study, the researchers hope that this ACT MILs
method could be used in other hematologic
malignancies infiltrating the bone marrow.
Source: Noonan KA, Huff CA, Davis J, et al. Adoptive transfer of activated
marrow-infiltrating lymphocytes induces measurable antitumor immunity in
the bone marrow in multiple myeloma. Sci Transl Med. 2015;7:288ra78.
Updated ABIM Score
Reporting for MOC
Examination
The American Board of Internal Medicine
(ABIM) is updating the score reporting for its
Maintenance of Certification (MOC) examinations. The changes are a result of input from
the Internal Medicine community, according
to the ABIM, and will provide more information on physician performance when delivering MOC scores.
Beginning with the spring 2015 MOC
examinations, physicians will receive score reports in a new electronic format that includes
a visual presentation of the results, detailed
feedback on incorrect questions, and links to
additional information.
“As the practice of medicine changes,
ABIM must work closely with the physician
community to continuously ensure that our
exams reflect foundational knowledge that
doctors in practice today need to serve their
patients,” said Nick Fitterman, MD, chair
of the ABIM Internal Medicine Board Exam
Committee, in a press release.
The ABIM has come under fire recently for
its MOC requirements, and for not providing
usable feedback to exam takers.
Source: American Board of Internal Medicine press release
FDA Approves
Eltrombopag for Pediatric Chronic Immune
Thrombocytopenia
The U.S. FDA has approved eltrombopag for
children who are 6 years or older with chronic
immune thrombocytopenia (ITP) who have
not responded to treatment with corticosteroids, immunoglobulins, or splenectomy. The
drug was previously approved by the U.S. FDA
in 2008 for the treatment of ITP in adults.
Eltrombopag is a once-daily oral thrombopoietin receptor agonist that increases platelet
production.
The recent approval for pediatric ITP was
based on data from two double-blind, controlled trials, including the largest phase III
trial in the pediatric ITP patient population.
28
ASH Clinical News
In each of these studies, eltrombopag was
shown to significantly increase and sustain
platelet counts, while also allowing for the
safe discontinuation of some concomitant
medications (including corticosteroids).
The most commonly reported adverse
events associated with eltrombopag included
upper respiratory tract infections, nasopharyngitis, rhinitis, abdominal pain, cough,
inflammation of the throat or mouth, toothache, abnormal liver function tests, diarrhea,
rash, and vitamin D deficiency.
Epigenetic suppression of the KLF4 gene
acts as a key transforming event in AML
and, through its unique mechanism of action, APTO-253 exhibited strong activity to
kill AML cells as both a single-agent and in
combination with other agents, according to
preclinical studies. Those reports also showed
that APTO-253 had a favorable safety profile
with no evidence of suppression of the normal
bone marrow.
Source: Aptose Biosciences press release
Source: Novartis press release
FDA Introduces New
REMS Tracking
AG-120 Receives Orphan Simplified
On June 17, 2015, the U.S. FDA rolled out an
updated website for its Risk Evaluation and
Drug Designation for
Mitigation Strategies (REMS). The website
aims to make REMS more accessible and the
the Treatment of AML
process of finding an REMS and determinThe U.S. FDA granted orphan drug designation
to AG-120 for the treatment of acute myeloid
leukemia (AML). AG-120 is an oral, first-inclass IDH1 mutant inhibitor, which is currently
being evaluated in a phase I clinical trial in
patients with IDH1-mutant positive AML.
Preliminary reports from the ongoing
phase I study found that AG-120 demonstrated clinical activity and a favorable safety
profile in 57 patients. The overall treatment
response rate was 31 percent and the complete remission rate was 15 percent. Patients
who have responded to treatment have remained on AG-120 for up to 11 months, with
79 percent of responders receiving treatment
for at least three months.
The most commonly reported adverse
events associated with AG-120 in this trial
included fatigue, diarrhea, pyrexia, and
nausea. So far, 13 deaths have been reported
among study participants, though the researchers did not attribute any mortalities as
related to treatment.
An additional three expansion cohorts
consisting of 175 AML patients are expected
to be studied.
Source: Agios Pharmaceuticals news release
APTO-253 Receives FDA’s
Orphan Designation for
the Treatment of AML
The U.S. FDA has granted orphan drug designation for APTO-253 for the treatment of patients with acute myeloid leukemia (AML). The
drug is a first-in-class inducer of t