eprintid: 10119873 rev_number: 25 eprint_status: archive userid: 608 dir: disk0/10/11/98/73 datestamp: 2021-01-28 12:03:31 lastmod: 2022-04-08 09:59:25 status_changed: 2021-01-28 12:03:31 type: article metadata_visibility: show creators_name: Sharpley, FA creators_name: Manwani, R creators_name: Petrie, A creators_name: Mahmood, S creators_name: Sachchithanantham, S creators_name: Lachmann, HJ creators_name: Martinez De Azcona Naharro, A creators_name: Gillmore, JD creators_name: Whelan, CJ creators_name: Fontana, M creators_name: Cohen, O creators_name: Hawkins, PN creators_name: Wechalekar, AD title: Autologous stem cell transplantation vs bortezomib based chemotheraphy for the first‐line treatment of systemic light chain amyloidosis in the UK ispublished: pub divisions: UCL divisions: A01 divisions: B02 divisions: C10 divisions: D17 divisions: G90 divisions: D21 keywords: multiple myeloma, plasma cell neoplasms, transplantation note: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. abstract: 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. date: 2021-04 date_type: published official_url: https://doi.org/10.1111/ejh.13582 oa_status: green full_text_type: other language: eng primo: open primo_central: open_green verified: verified_manual elements_id: 1843362 doi: 10.1111/ejh.13582 lyricists_name: Cohen, Oliver lyricists_name: Fontana, Marianna lyricists_name: Gillmore, Julian lyricists_name: Hawkins, Philip lyricists_name: Lachmann, Helen lyricists_name: Manwani, Richa lyricists_name: Petrie, Aviva lyricists_name: Sharpley, Faye lyricists_name: Wechalekar, Ashutosh lyricists_id: OCOHE36 lyricists_id: MFONT56 lyricists_id: JGILL78 lyricists_id: PNHAW77 lyricists_id: HJLAC80 lyricists_id: RMANW28 lyricists_id: APETR26 lyricists_id: FSHAR53 lyricists_id: AWECH53 actors_name: Flynn, Bernadette actors_id: BFFLY94 actors_role: owner full_text_status: public publication: European Journal of Haematology volume: 106 number: 4 pagerange: 537-545 event_location: England citation: Sharpley, FA; Manwani, R; Petrie, A; Mahmood, S; Sachchithanantham, S; Lachmann, HJ; Martinez De Azcona Naharro, A; ... Wechalekar, AD; + view all <#> Sharpley, FA; Manwani, R; Petrie, A; Mahmood, S; Sachchithanantham, S; Lachmann, HJ; Martinez De Azcona Naharro, A; Gillmore, JD; Whelan, CJ; Fontana, M; Cohen, O; Hawkins, PN; Wechalekar, AD; - view fewer <#> (2021) Autologous stem cell transplantation vs bortezomib based chemotheraphy for the first‐line treatment of systemic light chain amyloidosis in the UK. European Journal of Haematology , 106 (4) pp. 537-545. 10.1111/ejh.13582 <https://doi.org/10.1111/ejh.13582>. Green open access document_url: https://discovery.ucl.ac.uk/id/eprint/10119873/1/ejh.13582.pdf