In a phase II trial reported in the Journal of Clinical Oncology, Suzanne Lentzsch, MD, PhD, and colleagues found that the combination of bendamustine and dexamethasone showed activity in patients with relapsed/refractory systemic light-chain amyloidosis. As stated by the investigators, there currently is no established treatment in this setting.
Suzanne Lentzsch, MD, PhD
Study Details
In the multicenter study, 31 patients with persistent or progressive systemic light-chain amyloidosis after 1 or more prior therapies were treated with 100 mg/m2 of bendamustine on days 1 and 2 and 40 mg of dexamethasone weekly in 28-day cycles until disease progression or up to 6 cycles after complete hematologic response. The primary outcome measure was rate of partial hematologic response or better.
Responses
Patients received a median of four cycles of bendamustine/dexamethasone (range = 2–12 cycles).
KEY POINTS
- Partial response or better was achieved in 57% of patients.
- Organ response was observed in 29% of patients with measureable disease.
Among 28 patients evaluable for hematologic response, partial response or better was observed in 16 (57%), including compete response in 3 (11%), very good partial response in 5 (18%), and partial response in 8 (29%). Among 24 patients with measureable organ involvement, response was observed in 7 (29%). Median progression-free survival was 11.3 months. Median overall survival was 18.2 months. Median overall survival among patients with partial response or better after two cycles of treatment was not reached, compared with a median of 9.5 months among those without hematologic response after two cycles (P = .0291).
Adverse Events
Treatment-related grade 3 or 4 adverse events occurred in 65% of the total group of 31 patients, with the most common being decreased white blood cell count (26%), fatigue (19%), and renal dysfunction (13%). No grade 5 treatment-related adverse events were observed. The most common treatment-related adverse events of any grade were myelosuppression (eg, decreased white blood cell count in 12, anemia in 11, and thrombocytopenia in 6 patients), fatigue (48%), and nausea/vomiting (39%).
The investigators concluded, “Overall, [bendamustine/dexamethasone] is a viable treatment option with substantial efficacy and limited toxicity for patients with pretreated systemic light-chain amyloidosis who have limited therapeutic options.”
Dr. Lentzsch, of the Division of Hematology/Oncology, Columbia University Medical Center, is the corresponding author for the Journal of Clinical Oncology article.
Disclosure: The study was supported by the National Cancer Institute and TEVA Pharmaceuticals. For full disclosures of the study authors, visit ascopubs.org.
