Dealing With High-Risk AL Amyloidosis Patients: A Single Hematologic Center Experience

• Published in: Clinical lymphoma, myeloma and leukemia myeloma and leukemia, 2021-12, Vol.21 (12), p.e970-e974
• Main Authors: Riva, Marcello, Berno, Tamara, Cipriani, Alberto, Altinier, Sara, Fedrigo, Marny, Noventa, Franco, Adami, Fausto
• Format: Article
• Language: English
• Subjects: Amyloidosis - diagnosis; Amyloidosis - drug therapy; Bortezomib; Bortezomib - therapeutic use; Dexamethasone; Hematology; Humans; Immunoglobulin Light-chain Amyloidosis - diagnosis; Immunoglobulin Light-chain Amyloidosis - drug therapy; Prognosis; Retrospective Studies; Treatment Outcome
• ISSN: 2152-2650; 2152-2669
• DOI: 10.1016/j.clml.2021.07.015

To assess the feasibility and the results of Bortezomib-based treatment of “high-risk” AL-amyloidosis patients in a hematology ward. We report on 52 high-risk amyloidosis patients treated with first-line bortezomib-based chemotherapy. At day 30 from the beginning of the therapy, 23 patients (44%) achieved a hematological response (complete response plus very good partial response); 14 patients (27%) achieved a partial response; 15 patients (29%) were non-responders. After a median follow-up of 28.5 months, the survival rates were 18/23 (78%) for responders; 9/14 (64%) for partial responders and 3/15 (20%) for nonresponders with a median overall survival of 43, 24 and 11 months, respectively (log-rank test: P < .001). NHYA class I-II, NTproBNP < 6500 ng/L, the hematologic response, and the partial hematological response at day 30 independently predicted the survival. There has been no significant difference (P = .173) in survival between revised Mayo stage III and IV patients although there was a trend toward a better prognosis for Mayo stage III. A suboptimal hematological response at day 30 allowed a later organ response in 12/14 patients (85%) even without therapy change and no modification of the hematological status. These results show that high-risk AL-amyloidosis patients can be managed safely and effectively in a hematology ward. A partial hematologic response may herald a later better response, organ response, and can allow a subsequent second-line therapy and a good survival. In this limited retrospective analysis, we report on 52 “high risk” AL amyloidosis patients treated with first-line bortezomib-based chemotherapy. Achieving a hematological complete response (CR + VGPR) at day 30 confirmed its prognostic value. Nevertheless, patients who reached a suboptimal response (PR) at day 30 have a reasonable chance of survival, and do not necessarily need a change in therapeutic strategy even in the face of permissive clinical conditions. On the other hand, an early identification of NR patients should prompt a shift in therapy trying to improve the well-known dismal prognosis of these patients. We have also shown that even “high-risk” AL amyloidosis patients can be managed safely and effectively in a hematology ward.