Bortezomib-Containing Regimens for the Treatment of Newly Diagnosed and Relapsed Amyloid Light Chain Amyloidosis: A Single-Center Experience.
BACKGROUND
The proteasome is an exciting target for the development of novel anticancer therapies. Recent evidence has suggested that bortezomib, a dipeptide boronate proteasome inhibitor, exhibits unprecedented single-agent activity in amyloid light chain (AL) amyloidosis.


METHODS
We performed a retrospective review of the use of bortezomib-containing regimens to assess the rapidity and quality of response at our institution.


RESULTS
A total of 52 patients with documented newly diagnosed and relapsed AL amyloidosis treated with bortezomib-containing regimens were identified from our institutional database. After a median of 4 cycles (range, 1-22 cycles), a hematologic response was seen in 49 patients (94.2%), including a complete response in 15 (28.8%), a very good partial response in 25 (48.1%), and a partial response in 9 (17.3%). At 6 weeks, 37 patients had already achieved a partial response. An organ response at 6 months was documented in 31 patients (59.6%). With respect to the cardiac response, a > 30% decrease in N-terminal pro-hormone brain natriuretic peptide (NT-proBNP) was observed in 17 of 35 evaluable patients (48.6%; NT-proBNP > 650 ng/L) at a median of 6 months. Overall survival was shorter for the patients with NT-proBNP > 5000 ng/L and for those who achieved less than a very good partial response.


CONCLUSIONS
Bortezomib is a safe and well-tolerated therapy for patients with AL amyloidosis with a rapid hematologic response and cardiac response, as assessed by the NT-proBNP level.
Department of Medical Oncology and Hematology, Tom Baker Cancer Center, Calgary, Alberta, Canada. Electronic address: victor.zepeda@albertahealthservices.ca.
Victor H
VH
Jimenez-Zepeda
Department of Medical Oncology and Hematology, Tom Baker Cancer Center, Calgary, Alberta, Canada.
Peter
P
Duggan
Department of Medical Oncology and Hematology, Tom Baker Cancer Center, Calgary, Alberta, Canada.
Paola
P
Neri
Department of Medical Oncology and Hematology, Tom Baker Cancer Center, Calgary, Alberta, Canada.
Nizar J
NJ
Bahlis
D000328:Adult; D000368:Aged; D058227:Amyloidogenic Proteins / Q000378:metabolism*; D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy* / Q000378:metabolism* / Q000401:mortality; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D015415:Biomarkers; D000069286:Bortezomib / Q000008:administration & dosage; D015897:Comorbidity; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000378:metabolism*; D008297:Male; D008875:Middle Aged; D012008:Recurrence; D012720:Severity of Illness Index; D016019:Survival Analysis; D016896:Treatment Outcome
D058227:Amyloidogenic Proteins; D015415:Biomarkers; D007147:Immunoglobulin Light Chains; D000069286:Bortezomib
AL amyloidosis; Bortezomib; Cardiac response; CyBorD; Hematologic response