• DOI:10.1182/blood-2011-02-334227
• Corpus ID: 7584436

Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study.

@article{Reece2011EfficacyAS,
  title={Efficacy and safety of once-weekly and twice-weekly bortezomib in patients with relapsed systemic AL amyloidosis: results of a phase 1/2 study.},
  author={Donna Ellen Reece and Ute Hegenbart and Vaishali Sanchorawala and Giampaolo Merlini and Giovanni Palladini and Joan Blad{\'e} and Jean Paul Fermand and Hani Hassoun and Leonard T. Heffner and Robert A. Vescio and Kevin X. Liu and Christopher Enny and Dixie Lee Esseltine and Helgi van de Velde and Andrew Z Cakana and Raymond L. Comenzo},
  journal={Blood},
  year={2011},
  volume={118 4},
  pages={
          865-73
        }
}

• D. Reece, U. Hegenbart, +13 authors R. Comenzo
• Published 28 July 2011
• Medicine
• Blood

This first prospective phase 2 study of single-agent bortezomib in relapsed primary systemic AL amyloidosis evaluated the recommended (maximum planned) doses identified in phase 1 testing (1.6 mg/m² once weekly [days 1, 8, 15, and 22; 35-day cycles]; 1.3 mg/m² twice weekly [days 1, 4, 8, and 11; 21-day cycles]). Among all 70 patients enrolled in the study, 44% had ≥ 3 organs involved, including 73% and 56% with renal and cardiac involvement. In the 1.6 mg/m² once-weekly and 1.3 mg/m² twice-weekly groups, the hematologic response rate was 68.8% and 66.7% (37.5% and 24.2% complete responses, respectively); median time to first/best response was 2.1/3.2 and 0.7/1.2 months, and 78.8% and 75.5% had response durations of ≥ 1 year, respectively. One-year hematologic progression-free rates were 72.2% and 74.6%, and 1-year survival rates were 93.8% and 84.0%, respectively. Outcomes appeared similar in patients with cardiac involvement. Among all 70 patients, organ responses included 29% renal and 13% cardiac responses. Rates of grade ≥ 3 toxicities (79% vs 50%) and discontinuations/dose reductions (38%/53% vs 28%/22%) resulting from toxicities appeared higher with 1.3 mg/m² twice-weekly versus 1.6 mg/m² once-weekly dosing. Both bortezomib dose schedules represent active, well-tolerated regimens in relapsed AL amyloidosis. This study was registered at www.clinicaltrials.gov as #NCT00298766.