Details

Autor(en) / Beteiligte

• Ghobrial, Irene M.
• Campigotto, Federico
• Murphy, Timothy J.
• Boswell, Erica N.
• Banwait, Ranjit
• Azab, Feda
• Chuma, Stacey
• Kunsman, Janet
• Donovan, Amanda
• Masood, Farzana
• Warren, Diane
• Rodig, Scott
• Anderson, Kenneth C.
• Richardson, Paul G.
• Weller, Edie
• Matous, Jeffrey

Titel

Results of a phase 2 trial of the single-agent histone deacetylase inhibitor panobinostat in patients with relapsed/refractory Waldenström macroglobulinemia

Ist Teil von

• Blood, 2013-02, Vol.121 (8), p.1296-1303

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2013

Quelle

MEDLINE

Beschreibungen/Notizen

• 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 (www.clinicaltrials.gov identifier: NCT00936611) establishes a role for histone deacetylase inhibitors as an active class of therapeutic agents in WM. •Presents the data from a phase 2 clinical trial of panobinostat in patients with relapsed WM.•Establishes a role for histone deacetylase inhibitors as an active class of therapeutic agents in WM.