TY - JOUR
T1 - Fifteen year overall survival rates after autologous stem cell transplantation for AL amyloidosis
AU - Sidana, Surbhi
AU - Sidiqi, M. Hasib
AU - Dispenzieri, Angela
AU - Buadi, Francis K.
AU - Lacy, Martha Q.
AU - Muchtar, Eli
AU - Dingli, David
AU - Hayman, Suzanne R.
AU - Gonsalves, Wilson I.
AU - Kapoor, Prashant
AU - Leung, Nelson
AU - Warsame, Rahma
AU - Kourelis, Taxiarchis V.
AU - Wolf, Robert C.
AU - Hogan, William J.
AU - Kumar, Shaji K.
AU - Gertz, Morie A.
N1 - Funding Information:
SS: Honoraria (advisory board), Janssen; AD: Research Funding from Celgene, Takeda, Prothena, Jannsen, Pfizer, Alnylam, GSK; PK: Research Funding from Celgene, Takeda. MQL: Research Funding from Celgene; SKK: Research Funding and membership on an entity's Board of Directors or advisory committees: AbbVie, Celgene, Janssen, KITE, Merck. Membership on an entity's Board of Directors or advisory committees: Oncopeptides, Takeda. Research funding from Novartis and Roche.; MAG: Honoraria/consultancy from Ionis, Alnylam, Prothena, Celgene, Janssen, Specytrum, Annexon, Apellis, Amgen, Medscape, Abbvie, Research to Practice, Physcians Education Resource and Teva.
Publisher Copyright:
© 2019 Wiley Periodicals, Inc.
PY - 2019/9
Y1 - 2019/9
N2 - In appropriately selected patients with AL amyloidosis, autologous stem cell transplant (ASCT) is an established treatment modality with excellent outcomes and decreasing transplant related mortality (TRM) over time. We report on 15-year overall survival (OS) in 159 patients undergoing ASCT from 1996 to 2003, with median follow up of 17.1 years. Day 100 TRM was 13.2% (n = 21). The OS of ≥15 years was observed in 30% (47/159) of patients. Patients surviving ≥15 years were younger (53 vs 56 years, P =.02), less likely to have lambda as the involved light chain (62% vs 78%, P =.03) and were less likely to have heart involvement (32% vs 56%, P =.005). Median OS of patients with heart involvement vs not was 4.0 vs 11.1 years, P =.006 and actuarial 15-year OS was 23% vs 43%, respectively. A higher proportion of patients with OS ≥15 years received full-dose melphalan conditioning (81% vs 61%, P =.01), and achieved day 100 complete response (CR) (64% vs 24%, P <.001). Median OS amongst patients who achieved CR vs not was 19.3 vs 5.4 years, P <.001. Heart involvement, receiving full-dose melphalan and achieving CR remained independent predictors of OS. AL amyloidosis and related complications were the cause of death in 52% of patients overall (1-5 years post-transplant: 81%; 5-10 years: 62% and 10-15 years: 55%). These results reinforce the key role of ASCT in AL amyloidosis. With improvements in TRM and more options for relapsed disease, we expect the long-term survival post-transplant to improve significantly in the future.
AB - In appropriately selected patients with AL amyloidosis, autologous stem cell transplant (ASCT) is an established treatment modality with excellent outcomes and decreasing transplant related mortality (TRM) over time. We report on 15-year overall survival (OS) in 159 patients undergoing ASCT from 1996 to 2003, with median follow up of 17.1 years. Day 100 TRM was 13.2% (n = 21). The OS of ≥15 years was observed in 30% (47/159) of patients. Patients surviving ≥15 years were younger (53 vs 56 years, P =.02), less likely to have lambda as the involved light chain (62% vs 78%, P =.03) and were less likely to have heart involvement (32% vs 56%, P =.005). Median OS of patients with heart involvement vs not was 4.0 vs 11.1 years, P =.006 and actuarial 15-year OS was 23% vs 43%, respectively. A higher proportion of patients with OS ≥15 years received full-dose melphalan conditioning (81% vs 61%, P =.01), and achieved day 100 complete response (CR) (64% vs 24%, P <.001). Median OS amongst patients who achieved CR vs not was 19.3 vs 5.4 years, P <.001. Heart involvement, receiving full-dose melphalan and achieving CR remained independent predictors of OS. AL amyloidosis and related complications were the cause of death in 52% of patients overall (1-5 years post-transplant: 81%; 5-10 years: 62% and 10-15 years: 55%). These results reinforce the key role of ASCT in AL amyloidosis. With improvements in TRM and more options for relapsed disease, we expect the long-term survival post-transplant to improve significantly in the future.
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U2 - 10.1002/ajh.25566
DO - 10.1002/ajh.25566
M3 - Article
C2 - 31254301
AN - SCOPUS:85068960292
VL - 94
SP - 1020
EP - 1026
JO - American Journal of Hematology
JF - American Journal of Hematology
SN - 0361-8609
IS - 9
ER -