Document Detail

Results of a phase 2 trial of the single-agent histone deacetylase inhibitor panobinostat in patients with relapsed/refractory Waldenström macroglobulinemia.
MedLine Citation:
PMID:  23287861     Owner:  NLM     Status:  MEDLINE    
The present study aimed to determine the safety and activity of the histone deacetylase inhibitor panobinostat in patients with relapsed/refractory Waldenström macroglobulinemia (WM). Eligibility criteria included patients with relapsed/refractory WM with any number of prior therapies. Patients received panobinostat at 30 mg 3 times a week; 12 of 36 (33%) patients were enrolled at 25 mg dose. A total of 36 patients received therapy. The median age was 62 years (range, 47-80) and the median number of prior therapies was 3 (range, 1-8). All of the patients had received prior rituximab. Minimal response (MR) or better was achieved in 47% of patients (90% confidence interval [CI], 33-62), with 22% partial remissions and 25% MR. In addition, 18 (50%) patients achieved stable disease and none showed progression while on therapy. The median time to first response was 1.8 months (range, 1.7-3.2). The median progression-free survival was 6.6 months(90% CI, 5.5-14.8). Grade 3 and 4 toxicities included thrombocytopenia (67%), neutropenia (36%), anemia (28%), leukopenia (22%), and fatigue (11%). We conclude that panobinostat is an active therapeutic agent in patients with relapsed/ refractory WM. This study ( identifier: NCT00936611) establishes a role for histone deacetylase inhibitors as an active class of therapeutic agents in WM.
Irene M Ghobrial; Federico Campigotto; Timothy J Murphy; Erica N Boswell; Ranjit Banwait; Feda Azab; Stacey Chuma; Janet Kunsman; Amanda Donovan; Farzana Masood; Diane Warren; Scott Rodig; Kenneth C Anderson; Paul G Richardson; Edie Weller; Jeffrey Matous
Publication Detail:
Type:  Clinical Trial, Phase II; Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't     Date:  2013-01-03
Journal Detail:
Title:  Blood     Volume:  121     ISSN:  1528-0020     ISO Abbreviation:  Blood     Publication Date:  2013 Feb 
Date Detail:
Created Date:  2013-02-22     Completed Date:  2013-04-24     Revised Date:  2014-05-15    
Medline Journal Info:
Nlm Unique ID:  7603509     Medline TA:  Blood     Country:  United States    
Other Details:
Languages:  eng     Pagination:  1296-303     Citation Subset:  AIM; IM    
Data Bank Information
Bank Name/Acc. No.:
Export Citation:
APA/MLA Format     Download EndNote     Download BibTex
MeSH Terms
Administration, Oral
Aged, 80 and over
Disease Progression
Disease-Free Survival
Follow-Up Studies
Histone Deacetylase Inhibitors / administration & dosage*,  adverse effects
Hydroxamic Acids / administration & dosage*,  adverse effects
Immunoglobulin M / blood
Indoles / administration & dosage*,  adverse effects
Middle Aged
Treatment Outcome
Waldenstrom Macroglobulinemia / drug therapy*,  mortality,  pathology
Grant Support
1R01F0003743//PHS HHS; P01 CA078378/CA/NCI NIH HHS
Reg. No./Substance:
0/Histone Deacetylase Inhibitors; 0/Hydroxamic Acids; 0/Immunoglobulin M; 0/Indoles; 0/panobinostat

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

Previous Document:  Bif-1 haploinsufficiency promotes chromosomal instability and accelerates Myc-driven lymphomagenesis...
Next Document:  Targeting transcription factor SALL4 in acute myeloid leukemia by interrupting its interaction with ...