TY - JOUR
T1 - Venetoclax in Relapse/Refractory AL Amyloidosis—A Multicenter International Retrospective Real-World Study
AU - Lebel, Eyal
AU - Kastritis, Efstathios
AU - Palladini, Giovanni
AU - Milani, Paolo
AU - Theodorakakou, Foteini
AU - Aumann, Shlomzion
AU - Lavi, Noa
AU - Shargian, Liat
AU - Magen, Hila
AU - Cohen, Yael
AU - Gatt, Moshe E.
AU - Vaxman, Iuliana
N1 - Publisher Copyright:
© 2023 by the authors.
PY - 2023/3/10
Y1 - 2023/3/10
N2 - Therapeutic options in relapsed refractory (R/R) light-chain (AL) amyloidosis patients are limited. Given the encouraging results in t(11;14) multiple myeloma and the high prevalence of t(11;14) in AL amyloidosis, venetoclax is an attractive treatment option in this setting. We report here the results of a multi-center retrospective study on 26 R/R AL amyloidosis patients treated off-label with venetoclax. The median lines of therapy prior to venetoclax was 3.5 (range 1–7), and 88% of our cohort had t (11;14). Twenty-two patients (85%) were previously treated with daratumumab. The overall hematologic response rate was 88%, 35% achieved a CR, and 35% achieved VGPR. The median event-free survival was 25 months (m) (95% CI 9.7 m-not reached), and the median overall survival was 33 m (95% CI 25.9–39.2 m). Most of the patients in this cohort are in ongoing deep responses and continuing venetoclax therapy. The treatment was relatively safe. One patient died due to infection, and there were two grade 3 infections in our cohort. Tumor lysis syndrome (TLS) was not seen in any patient. Dose reductions were frequent but did not affect the efficacy. These promising results require confirmation in a randomized controlled trial.
AB - Therapeutic options in relapsed refractory (R/R) light-chain (AL) amyloidosis patients are limited. Given the encouraging results in t(11;14) multiple myeloma and the high prevalence of t(11;14) in AL amyloidosis, venetoclax is an attractive treatment option in this setting. We report here the results of a multi-center retrospective study on 26 R/R AL amyloidosis patients treated off-label with venetoclax. The median lines of therapy prior to venetoclax was 3.5 (range 1–7), and 88% of our cohort had t (11;14). Twenty-two patients (85%) were previously treated with daratumumab. The overall hematologic response rate was 88%, 35% achieved a CR, and 35% achieved VGPR. The median event-free survival was 25 months (m) (95% CI 9.7 m-not reached), and the median overall survival was 33 m (95% CI 25.9–39.2 m). Most of the patients in this cohort are in ongoing deep responses and continuing venetoclax therapy. The treatment was relatively safe. One patient died due to infection, and there were two grade 3 infections in our cohort. Tumor lysis syndrome (TLS) was not seen in any patient. Dose reductions were frequent but did not affect the efficacy. These promising results require confirmation in a randomized controlled trial.
KW - amyloidosis
KW - t(11;14)
KW - venetoclax
UR - http://www.scopus.com/inward/record.url?scp=85151317084&partnerID=8YFLogxK
U2 - 10.3390/cancers15061710
DO - 10.3390/cancers15061710
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C2 - 36980596
AN - SCOPUS:85151317084
SN - 2072-6694
VL - 15
JO - Cancers
JF - Cancers
IS - 6
M1 - 1710
ER -