0000000000704228
AUTHOR
Heinz Gisslinger
Improved Safety with the Use of Subcutaneous Bortezomib in Combination with Panobinostat and Dexamethasone: Preliminary Data from a Panobinostat Global Expanded Treatment Protocol
Abstract Introduction: Panobinostat (PAN) is a potent pan-deacetylase inhibitor that targets multiple myeloma (MM) cells via its epigenetic effects as well as its effect on the aggresome. In the PANORAMA 1 phase 3 trial, the combination of PAN, bortezomib (BTZ), and dexamethasone (Dex; PAN+BTZ+Dex) significantly increased progression-free survival compared with placebo plus BTZ and Dex, leading to approval in Europe of the combination for the treatment of patients with MM who have received ≥ 2 prior regimens, including BTZ and an immunomodulatory agent. The purpose of this expanded treatment protocol (ETP) is to further evaluate safety and to provide panobinostat prior to commercial availab…
Bone marrow fibrosis and diagnosis of essential thrombocythemia
However, there are serious issues to beraisedconcerningtheauthors’analysisoftheclinicaldata,thecriteriausedtodiagnoseET,andthequantificationoffibrosis.Altogethertheauthors compared a heterogenous patient database, which included311 patients evaluated for presenting features, 299 for response totherapy,361forcomplicationrates,97forprogressionoffibrosis,andfour for reversal of BM fibrosis, so no single cohort with consistentfeatures was described throughout the study of 361 patients. Forexample, for the analysis of progression of fibrosis, only 97 (12%) oftheoriginal809patientsenteredintheUK-PT1trial
European Bone Marrow Working Group trial on reproducibility of World Health Organization criteria to discriminate essential thrombocythemia from prefibrotic primary myelofibrosis: Haematologica 2012;97(3):360-5 - Comment
Any study of myeloproliferative neoplasms (MPNs) that lacks adequate clinical input is doomed to cause diagnostic uncertainty and increased controversy. In the paper by Buhr et al. published in Haematologica,[1][1] the authors studied 102 cases of essential thrombocythemia (ET) and early primary