A 24‐year experience of autologous stem cell transplantation for light chain amyloidosis patients in the United Kingdom

Abstract

Summary Autologous stem cell transplantation (ASCT) is considered to be the best method to achieve deep haematological/organ responses and improve survival in selected patients with AL amyloidosis. This field has been led by US centres and is less utilised in Europe. The introduction of effective chemotherapy agents for AL prompted us to re‐evaluate UK outcomes of ASCT in affected patients. A total of 264 AL amyloidosis patients treated with an ASCT between 1994 and 2018 were identified. Patient baseline characteristics, transplant‐related mortality (TRM) and overall survival (OS) were analysed. The median OS post‐ASCT was 87 months [95% confidence interval (CI): 77–106 months]. The median time from ASCT to next treatment was 48 months (95% CI: 29–55 months). A haematological response was achieved in 94·8% of patients and was a strong predictor of time to next treatment [P < 0·0001, hazard ratio (HR) = 1·75, 95% CI = 1·35–2·28] and OS (P = 0·007, HR = 1·91, 95% CI = 1·19–3·07). Organ response was: cardiac (n = 28, 60·9%), renal (n = 101, 76%) and liver (n = 7, 13·5%). Overall TRM was 8·7%, with a significant reduction over time (1994–2000: 18·8%; 2001–2006: 13·6%; 2007–2012: 6·2%; 2013–2018: 1·1%). In conclusion, ASCT is significantly safer and remains a highly effective treatment with excellent long‐term survival; it should be more widely considered as a treatment option for systemic AL amyloidosis.

AL-amyloidosis with cardiac involvement. Diagnostic capabilities of non-invasive methods

“…(у пациента АД 120-110/80), клиренс креатинина ≥30 мл/мин/1,73 м 2 (у пациента СКФ -CKD-EPI -варьируется от 63 до 79 мл/мин/1,73 м 2 ), а также менее чем 3 органа значительного поражения [22]. Наилучшие результаты достигаются у пациентов группы низкого риска [21], к которым относятся 15-20% пациентов [23]. Пациент не относится к группе низкого риска, которым показана аутологичная трансплантация стволовых периферических клеток крови.…”

“…Важно отметить необходимость проведения высокодозной химиотерапии мелфаланом в дозе 140-200 мг/м 2 с последующей аутологичной трансплантацией стволовых периферических клеток крови в 1-й линии терапии у пациентов, удовлетворяющих критериям возможности проведения данной процедуры [22]. Важность отбора пациентов диктуется более высоким уровнем посттрансплантационной смертности по сравнению с пациентами с миеломой (до 12-40% против 5% по данным различных авторов) [23].…”

High-Dose Melphalan and Autologous Peripheral Blood Stem Cell Transplantation in AL Amyloidosis

“…Absence of heart involvement, receiving full HDM and achievement of hematologic CR remained independent predictors of survival following SCT in AL amyloidosis in this series. The UK National Amyloidosis Center also has recently reported on 264 patients undergoing HDM/SCT for AL amyloidosis with a median OS of 8.2 years and long-term survival of 33% at 20 years [38]. A large registry data from the CIBMTR from 134 centers again showed improvement in the 5-year OS in patients undergoing HDM/SCT for AL amyloidosis.…”

Autologous stem cell transplantation vs bortezomib based chemotheraphy for the first‐line treatment of systemic light chain amyloidosis in the UK

“…The French prospective, randomised trial (2007) contradicted this finding with a median OS 22.2 vs 56.9 months, for ASCT compared to oral melphalan and dexamethasone therapy 5 . Since these initial studies, the transplant‐related mortality (TRM) associated with ASCT has dramatically decreased 1,6 . This reduction in TRM should favour ASCT over standard chemotherapy 7 .…”