Bortezomib-based induction for transplant ineligible AL amyloidosis and feasibility of later transplantation.
Recent studies support the use of bortezomib-based therapies in light chain amyloidosis (AL). We performed a retrospective analysis of the safety, efficacy and long-term survival (median follow-up 3 years) after bortezomib-based treatment in 28 consecutive patients with de novo AL deemed ineligible at initial presentation. The first 14 patients received bortezomib and dexamethasone (VD), and the second 14 patients received cyclophosphamide, bortezomib and dexamethasone (CVD; CyBorD). Both regimens were well tolerated with no treatment-related mortality. The overall hematological response (HR) rate was 93% in both the groups. Median time to response was shorter in the CVD group (39 days vs 96 days in the VD group; P=0.002). Hematological and organ responses induced with bortezomib-based therapy enabled 8 (33%) of initially transplant ineligible patients to undergo autologous hematopoietic stem cell transplantation (AHCT), including 4 patients with cardiac stage III or IV. Seven of the eight patients (88%) who underwent subsequent AHCT achieved sustained HR at a median of 33 months posttransplant. These data suggest that bortezomib-based induction followed by AHCT is a viable therapeutic strategy for transplant-ineligible AL. Larger, multicenter prospective trials are necessary to confirm our findings.
Department of Medicine, Division of Hematology and Oncology, Vanderbilt University Medical Center, Nashville, TN, USA.
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RF
Cornell
Department of Biostatistics, Medical College of Wisconsin, Milwaukee, WI, USA.
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X
Zhong
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
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C
Arce-Lara
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
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E
Atallah
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
L
L
Blust
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
W R
WR
Drobyski
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
T S
TS
Fenske
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
M C
MC
Pasquini
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
J D
JD
Rizzo
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
W
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Saber
Department of Medicine, Medical College of Wisconsin, Milwaukee, WI, USA.
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PN
Hari
D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D000069286:Bortezomib / Q000008:administration & dosage / Q000627:therapeutic use*; D018450:Disease Progression; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011379:Prognosis
D000970:Antineoplastic Agents; D000069286:Bortezomib