A nationwide prospective registry of bortezomib-based therapy in light chain (AL) amyloidosis

Abstract

Until recently, no drug was labeled for AL amyloidosis. In 2011, the Italian Medicines Agency started a program to grant access to upfront bortezomib to patients with AL amyloidosis. All subjects were enrolled in a prospective online registry. Response was evaluated after two cycles to assess the possibility of continuing treatment. A total of 764 patients were included until 2019, and 615 were evaluable. Sixteen percent of patents had advanced (stage-IIIb) heart involvement, and 27% had severe or end-stage renal failure. Bortezomib delivery was possible in stage-IIIb patients at a reduced dose. Bortezomib discontinuation was associated with increasing age, advanced heart involvement and bi-weekly administration. Fifty-nine percent of subjects attained a hematologic response and 14% a cardiac response. Bortezomib-based therapy tends to be discontinued early in elderly patients and in advanced disease. Nevertheless, early response to therapy is possible in this challenging population.

Keywords:

• Amyloidosis
• bortezomib
• response
• prospective registry

Acknowledgements

The views expressed in this work are personal and may not be understood or quoted as being made on behalf of or reflecting the position of the Italian Medicines Agency or of one of their committees or working parties. Present composition of the AIFA’s Monitoring Registries Group (in chronological order): Giorgia Bartoccioni, Daniele Ricagni, Susanna Valentini, Antonietta Colatrella, Valeria Angelini, Paola D’Antrassi, Laura Veo, Annalisa Guarcello, Luca Tomassini. Although the AIFA monitoring registries operate within a regulatory framework that provides for the mandatory collection of data from real clinical practice on specific treatments reimbursed by the Italian National Healthcare System (NHS), by all public health facilities, the authors are, however, grateful for the contribution of all the physicians and pharmacists who filled the registers of bortezomib.

Disclosure statement

GP Honoraria and advisory board of Janssen-Cilag, Honoraria Pfizer; PM: speaker honoraria Janssen-Cilag and travel support from Celgene; AF: MN: speaker honoraria from Janssen-Cilag; the remaining authors have nothing to disclose.