CLINICAL NEWS
treatment,” the authors wrote, showing that
“ruxolitinib reduces splenomegaly in patients
with thalassemia.”
The researchers also observed 1.7-fold
increases in hepcidin levels from baseline
to week 30 (standard deviation [SD] = 1.15;
n=24), which suggest improved handling
of iron absorption in the long term. a Other
biomarkers (including serum iron, serum fer-
ritin, transferrin, and transferrin saturation)
experienced limited change from baseline.
Though there were no significant changes in
these factors, “increased levels of hepcidin
may suggest that the handling of iron absorp-
tion could be improved in the long term” with
ruxolitinib treatment, the researchers noted.
Also, although the investigators ob-
served “a trend for improvement” in the
median pretransfusion hemoglobin levels
over time at each six-week interval, there
was no clinically relevant improvement in
this secondary endpoint.
“Since the major purpose of reducing
spleen size in patients with transfusion-
dependent thalassemia is to improve pre-
transfusion hemoglobin and related reduction
in transfusion needs where ruxolitinib had
shown a limited effect, no further phase II
studies are planned,” the authors concluded.
The study is limited by its small patient
population and the lack of a comparator arm.
Novartis Pharmaceuticals Corporation
supported the study. ●
The authors report receiving funding
from Novartis, Celgene, Janssen-Cilag,
Roche, Pfizer, ApoPharma, Ionis Phar-
maceuticals, Shire, and Cerus. Novartis
provided editorial support for the original
manuscript.
REFERENCE
Taher AT, Karakas Z, Cassinerio E, et al. Efficacy and safety of ruxolitinib in
regularly transfused patients with thalassemia: results from a phase 2a
study. Blood. 2017 November 2. [Epub ahead of print]