LINKS TO ABSTRACTS:
Presentation ID p204-1
Presenter: Oriana Marques, Germany
The study authors have discovered that GPI-anchoring of HJV is crucial for its function in regulating hepcidin. The results demonstrate that PIGA mutations are responsible for a novel form of juvenile hemochromatosis.
Presentation ID p204-2
Presenter: Ali Taher, Beirut, Lebanon
The study authors conclude that treatment with luspatercept resulted in significant improvements of anemia, as measured by hemoglobin levels, versus placebo in adults with NTD β-thalassemia. Changes in NTDT-PRO T/W correlated with increases in hemoglobin. Luspatercept was well tolerated in this patient population.
Presentation ID p204-3
Presenter: Maria Ester Bernardo, Milano, Italy
The study authors suggest with their results that GT accomplishes extensive metabolic correction of peripheral and central compartments and promising preliminary clinical outcomes together with a favorable safety profile, highlighting the therapeutic potential of HSC-GT for the treatment of MPSIH.
Presenter: Hanny Al-Samkari, Boston, United States
The study authors conclude that Pazopanib was safe and effective to manage severe bleeding in Hereditary hemorrhagic telangiectasia, durably liberating all patients from transfusion dependence and normalizing hematologic parameters at doses much lower (median 100 mg daily) than the dose used to treat malignancies (800 mg daily).
Presentation ID p204-5
Presenter: Xiao-Hui Zhang, Beijing, China
The study authors conclude that impaired sympathetic innervation of the spleen and bone marrow plays a role in the pathogenesis of immune thrombocytopenia (ITP). Terbutaline treatment corrects the dysregulated polarization of T helper cells during T-cell differentiation, suggesting a potential novel therapeutic approach for patients with ITP.
Presentation ID p204-6
(LB1900) FIRST INTERIM ANALYSIS OF ALPINE STUDY: RESULTS OF A PHASE 3 RANDOMIZED STUDY OF ZANUBRUTINIB VS IBRUTINIB IN PATIENTS WITH RELAPSED/REFRACTORY CHRONIC LYMPHOCYTIC LEUKEMIA/SMALL LYMPHOCYTIC LYMPHOMA
Presenter: Peter Hillmen, Leeds, United Kingdom
The study authors conclude that in this interim analysis of a randomized, phase 3 ALPINE study in patients with R/R CLL/SLL, zanubrutinib was shown to have a superior response rate, an improved progression-free survival and a lower rate of atrial fibrillation/flutter as compared with ibrutinib. These data confirm that more selective BTK inhibition, with more complete and sustained BTK occupancy results in improved efficacy and safety outcomes. NCT03734016