By Ruben Mesa, MD
The 50th ASH Annual Meeting concludes today with a special session for the late-breaking abstracts, communications of significant scientific value which were submitted after the standard abstract deadline but reviewed and felt to be of significant impact. This is only the second time ASH has presented a “Late Breaking” session, which, similar to other meetings, includes either 1) very large clinical studies of significant impact or 2) mechanistic scientific discoveries of great impact that were not ready for submission by the initial deadline.
Today’s session begins with an important report by Dr. Tadeusz Robak and colleagues on the final results of a randomized trial of rituximab, fludarabine, and cyclophosphamide (R-FC) compared to fludarabine and cyclophosphamide (FC) alone in relapsed or refractory chronic lymphocytic leukemia. This large trial of 552 patients from 17 countries demonstrated a prolongation of progression-free survival by a median of 10 months in patients in the treatment arm containing rituximab (R-FC), with a higher complete response rate. The full results and slightly higher rate of toxicity in the R-FC arm of the trial will be presented.
Additional clinical insight adding to the discussion of the controversy regarding the use of erythropoietin-stimulating agents (ESAs) in cancer patients will be presented by Dr. Julia Bohlius and colleagues. In this analysis, data from almost 14,000 cancer patients from 53 studies are used in a structured meta-analysis and demonstrated an increase in on-study mortality and worsened survival among those patients receiving ESAs. This analysis will certainly generate great interest and discussion as we all consider the impact of such data on patients with solid tumors and whether these data have any direct relevance to hematologic or myeloid neoplasms. Separately, Dr. Lewis Hsu will present a clinical trial of tetrahydrobiopterin (6R-BH4) as therapy for endothelial cell dysfunction in sickle cell disease. Initial outcomes, responses, and toxicity of this novel approach will be discussed in the results of the first 32 affected patients on this phase I trial.
Pertinent mechanistic breakthroughs comprise the rest of this interesting session. Dr. Francois Delhommeau from the laboratory of Dr. William Vainchenker, who first identified the JAK2-V617F mutation in myeloproliferative neoplasms (MPNs), will describe the recognition that Ten-Eleven Translocation 2 (TET2) is a novel tumor suppressor gene inactivated in MPNs as a pathogenetic event preceding the JAK2-V617F mutation. Separately, Dr. Marina Cavazzana-Calvo will assert that human adenylate kinase 2 deficiency leads to differentiation blocks in myeloid and lymphoid maturation and is associated with sensorineural deafness. Finally, Dr. Paris Margaritis will describe the results of a canine model for FVIII/FIX gene-based bypassing agents.
All late-breaking abstracts will be presented today at 7:30 a.m. in Halls B and C South of the Moscone Center.
Dr. Mesa indicated no relevant conflicts of interest.
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