Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation: 20-year experience

Sanchorawala, Vaishali; Sun, Fangui; Quillen, Karen; Sloan, J. Mark; Berk, John L.; Seldin, David C.

doi:10.1182/blood-2015-08-662726

Cited by 118 publications

(87 citation statements)

References 4 publications

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“…Cohort 1: 1996–2002, Cohort 2: 2003–2009; Cohort 3: 2010–2016. Sanchorwala et al () cohorts: Cohort 1: 1994–2000, Cohort 2: 2000–2009, Cohort 3: 1994–2014. Current UK cohorts: 1: Cohort 1994–2000; Cohort 2: 2001–2006; Cohort 3: 2007–2012; Cohort 4: 2013–2018.…”

Section: Discussionmentioning

confidence: 99%

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

et al. 2019

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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.

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Section: Discussionmentioning

confidence: 99%

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

et al. 2019

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“…Remarkably, about 55% of patients in CR are projected to be alive at 14 years, with no deaths observed in patients with longer followup. 61 This observation raises the hope that a proportion of patients achieving CR according to the current definition might be cured. Bortezomib can be used in subjects who fail to achieve CR after ASCT, increasing the CR rate to almost 60%.…”

Section: What's New In Amyloidosis 163mentioning

confidence: 99%

“…57 The hematologic response rate to ASCT exceeds 70%, with 35% to 37% of patients obtaining CR. 57,61 The Boston University investigators recently updated their experience with ASCT. 61 With a median follow-up of 8 years, the overall median survival was 7.6 years.…”

Section: What's New In Amyloidosis 163mentioning

confidence: 99%

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What is new in diagnosis and management of light chain amyloidosis?

Palladini

Merlini

2016

Blood

205

171

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Light chain (AL) amyloidosis is caused by a usually small plasma cell clone producing a misfolded light chain that deposits in tissues. Survival is mostly determined by the severity of heart involvement. Recent studies are clarifying the mechanisms of cardiac damage, pointing to a toxic effect of amyloidogenic light chains and offering new potential therapeutic targets. The diagnosis requires adequate technology, available at referral centers, for amyloid typing. Late diagnosis results in approximately 30% of patients presenting with advanced, irreversible organ involvement and dying in a few months despite modern treatments. The availability of accurate biomarkers of clonal and organ disease is reshaping the approach to patients with AL amyloidosis. Screening of early organ damage based on biomarkers can help identify patients with monoclonal gammopathy of undetermined significance who are developing AL amyloidosis before they become symptomatic. Staging systems and response assessment based on biomarkers facilitate the design and conduction of clinical trials, guide the therapeutic strategy, and allow the timely identification of refractory patients to be switched to rescue therapy. Treatment should be risk-adapted. Recent studies are linking specific characteristics of the plasma cell clone to response to different types of treatment, moving toward patient-tailored therapy. In addition, novel anti-amyloid treatments are being developed that might be combined with anti-plasma cell chemotherapy.

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“…[8][9][10]15 Overall, the collective experience of bortezomibbased induction prior to AHCT shows that it is a feasible approach for most patients who are otherwise candidates for transplantation and leads to favorable long-term outcomes.…”

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confidence: 99%

Outcomes of autologous hematopoietic cell transplantation in primary amyloidosis after bortezomib-based induction therapy

Hong

Valent

Rybicki

et al. 2016

Bone Marrow Transplant

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Long-term outcome of patients with AL amyloidosis treated with high-dose melphalan and stem cell transplantation: 20-year experience

Cited by 118 publications

References 4 publications

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

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

What is new in diagnosis and management of light chain amyloidosis?

Outcomes of autologous hematopoietic cell transplantation in primary amyloidosis after bortezomib-based induction therapy

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