TY  - JOUR
TI  - Autologous stem cell transplantation vs bortezomib based chemotheraphy for the first?line treatment of systemic light chain amyloidosis in the UK
SP  - 537
AV  - public
VL  - 106
Y1  - 2021/04//
EP  - 545
IS  - 4
N1  - This version is the author accepted manuscript. For information on re-use, please refer to the publisher?s terms and conditions.
N2  - OBJECTIVES: The benefit of autologous stem cell transplantation (ASCT) in the treatment of light chain (AL) amyloidosis requires re-evaluation in the modern era. This retrospective case-matched study compares ASCT to bortezomib for the treatment of patients with AL amyloidosis. METHODS: Newly diagnosed patients with AL amyloidosis treated with ASCT or bortezomib between 2001-2018 were identified. Patients were excluded if the time from diagnosis to treatment exceeded 12 months. Patients were matched on a 1:1 basis, using a propensity matched scoring approach. RESULTS: A total of 136 propensity-score matched patients were included (ASCT n= 68, bortezomib n=68). There was no significant difference in overall survival at two years (p=0.908, HR: 0.95, CI:0.41-2.20). For ASCT vs. bortezomib: overall haematological response rate at six months was 90.6% vs. 92.5%; organ response at 12 months: cardiac (70.0% vs. 54%, p>0.999), renal (74% vs.24%, p=0.463)) liver (21% vs. 22%, p=0.048); median progression free survival (50 vs. 42 months p=0.058, HR:0.61, CI:0.37-1.02) and time to next treatment (68 vs. 45 months, p=0.145, HR:0.61, CI:0.31-1.19). More patients required treatment in the bortezomib group compared to ASCT group at 24 months (41 vs. 23, Chi squared p=0.004) and 48 months (57 vs 41, Chi squared p= 0.004). CONCLUSIONS: This small retrospective study suggests that there is no clear survival advantage of ASCT over bortezomib therapy. A prospective randomised controlled trial evaluating ASCT in AL amyloidosis is critically needed.
ID  - discovery10119873
UR  - https://doi.org/10.1111/ejh.13582
JF  - European Journal of Haematology
A1  - Sharpley, FA
A1  - Manwani, R
A1  - Petrie, A
A1  - Mahmood, S
A1  - Sachchithanantham, S
A1  - Lachmann, HJ
A1  - Martinez De Azcona Naharro, A
A1  - Gillmore, JD
A1  - Whelan, CJ
A1  - Fontana, M
A1  - Cohen, O
A1  - Hawkins, PN
A1  - Wechalekar, AD
KW  - multiple myeloma
KW  -  plasma cell neoplasms
KW  -  transplantation
ER  -