CLINICAL NEWS
Conference Coverage
BREAKTHROUGHS IN BLOOD DISORDERS
he European Hematology Asso-
ciation’s 23rd Congress was held
June 14-17 in Stockholm, Sweden,
where more than 10,000 interna-
tional hematologists met to share the lat-
est results from clinical and translational
research in hematologic disorders and
emerging techniques for diagnosis and
risk assessment.
Here, ASH Clinical News presents
highlights from the meeting, including
a combination of polatuzumab vedotin,
bendamustine, and rituximab in diffuse
large B-cell lymphoma and fostamatinib
for patients with warm antibody auto-
immune hemolytic anemia.
Look for more reporting from the con-
ference in our August issue.
Synthetic Hepcidin Options
Safe in Patients With Iron
Disorders, But Efficacy Data
Still Preliminary
Two studies presented at the 23rd Congress of the European Hematology Association
examined the safety and pharmacokinetics of two synthetic hepcidin options for the treat-
ment of hereditary blood disorders associated with iron overload: PTG-300 and LJPC-401.
Hepcidin is secreted by the liver and acts as “the master regulator of serum iron
concentrations,” explained one study’s lead author Ashutosh Lal, MD, of the UCSF
Benioff Children’s Hospital in San Francisco. “Several disorders of iron overload,
such as hereditary hemochromatosis and beta-thalassemia major, are associated with
reduced hepcidin secretion, [and] there is considerable interest in augmenting endo-
genous hepcidin for the management of iron overload.”
LJPC-401
LJPC-401 is a synthetic endogenous human hepcidin that mimics the natural hepcidin
hormone. Dr. Lal and colleagues evaluated its safety in a phase I study of 18 patients
(7 with hemoglobinopathies and 11 with hemochromatosis), who had the following
disorders and medical histories:
• transfusion-dependent anemia
• iron chelation therapy within 6 months prior to enrollment
• serum ferritin >1,000 μg/L
• hemochromatosis requiring phlebotomy at least once every 2 months
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