High Dose Therapy with Autologous Hematopoietic Stem Cell Transplantation (HSCT) for Patients with Primary Systemic Amyloidosis (AL): An Abmtr Study

Vesole, DH; Perez, W.; Reece, D.; Akasheh, M.; Horowitz, M.; Bredeson, C.

doi:10.1201/9781420037494-58

Cited by 4 publications

(5 citation statements)

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“…High TRM has been encountered widely in this setting, and even the North American high‐volume centres of excellence continue to report an incidence of around 13% (Gertz et al , 2002; Skinner et al , 2004). In non‐specialist centres, TRM was 43% in a French survey (Moreau et al , 1998), and 25% among 114 patients treated in 50 US centres (Vesole et al , 2003). Furthermore, only 33% of the latter patients had a haematological response, and observational studies do not always identify morbidity and mortality associated with stem cell mobilisation.…”

Section: Discussionmentioning

confidence: 99%

“…Anecdotal reports (Majolino et al , 1993) were followed by open studies, notably including extensive experience in Boston (Comenzo et al , 1996, 1998; Skinner et al , 2004) where treatment‐related mortality (TRM), defined as all‐cause mortality during the first 100 d following therapy, has been about 13%, compared with <2–5% in myeloma. TRM has been even greater in multicentre studies, at 43% in a French series (Moreau et al , 1998) and 25% in an Autologous Blood and Marrow Transplant Registry series of 114 patients treated in 50 US centres (Vesole et al , 2003).…”

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confidence: 99%

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Outcome of autologous stem cell transplantation for AL amyloidosis in the UK

et al. 2006

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SummaryHigh-dose chemotherapy with autologous stem cell transplantation (SCT) is widely used as a treatment for systemic AL amyloidosis, but its efficacy has not been proved and it has substantial toxicity in this setting. We report here the outcome of 92 patients evaluated at the UK National Amyloidosis Centre who underwent SCT for AL amyloidosis between 1994 and 2004 in various British centres. Median age was 53 years and median of two organs were affected by amyloidosis. All-cause day 100 mortality [treatment-related mortality (TRM)] was 23% for the entire cohort, although this was substantially greater for patients treated from 1994 to 1998 (15/47, 32%) than subsequently (6/45, 13%). Independent factors significantly associated with TRM on multivariate analysis were: number of affected organs, performance status, serum albumin and age. Response of the underlying clonal disease, defined by ‡50% reduction in the aberrant serum-free light chain concentration, occurred in 83% of evaluable patients. Overall median survival was 5AE3 years, and was 8AE5 years among patients who survived beyond day 100. Despite recent refinements in patient selection, TRM remains substantial during SCT for systemic AL amyloidosis, and its place in the therapeutic armamentarium for this disease needs to be defined in randomised controlled clinical studies.

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Section: Discussionmentioning

confidence: 99%

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confidence: 99%

Outcome of autologous stem cell transplantation for AL amyloidosis in the UK

et al. 2006

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“…A major problem in patients with amyloidosis is limited eligibility due to the risk of procedural morbidity and mortality with probably only c. 20% of cases being genuinely 'low risk' for TRM with ASCT, as per stringent criteria . Though earlier studies reported a TRM of 20-40% (Moreau et al, 1998;Vesole et al, 2003;Mollee et al, 2004), substantially higher than in myeloma, it has lately levelled out to 10-12% in the experienced North American transplant centres Gertz et al, 2005c). Use of a risk-adapted approach further reduces the TRM markedly to less than 5% (Cohen et al, 2005) but the reduction in melphalan dose probably compromises its efficacy (Gertz et al, 2004) and needs prospective study to find an optimal dose.…”

Section: Autologous Stem Cell Transplantationmentioning

confidence: 98%

Perspectives in treatment of AL amyloidosis

2007

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Light chain (AL) amyloidosis is the most frequently diagnosed form of systemic amyloid in the western world. The historically poor prognosis of AL amyloidosis appears to be improving with currently reported median survival of c. 40 months compared to 13 months in the early 1990s when low-dose oral melphalan was the mainstay of treatment. Autologous stem cell transplantation (ASCT) achieves the highest rates of complete clonal response but is confounded by substantial treatment-related mortality in AL amyloidosis unless it is restricted to highly selected patients. Newer chemotherapy regimens appear to have a balance of better safety and respectable efficacy with overall outcomes nearly similar to ASCT, but which may be used more widely. There are few data comparing durability, depth of clonal response, rate of organ response and overall survival following ASCT or chemotherapy, but a recent small, randomized trial did not suggest superiority of ASCT to oral melphalan and dexamethasone. There is a compelling need for further and larger randomized trials in this context. At the same time, various new specific anti-amyloid drugs have shown (in early phase studies or animal models) some very promising results. This review attempts to highlight the challenges, controversies and progress in AL amyloidosis.

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“…42 This substantial early mortality aligned well with that observed in contemporary retrospective single-center data sets in both the United States and the United Kingdom. 52,53…”

Section: The Role Of Autologous Stem-cell Transplantationmentioning

confidence: 99%

“…42 This substantial early mortality aligned well with that observed in contemporary retrospective single-center data sets in both the United States and the United Kingdom. 52,53 Improved patient selection and the specific application of surrogate markers for cardiac function (systolic blood pressure [BP] . 90, New York Heart Association heart failure scale less than III, patients with low frailty score, better performance score, use of limited cycles of induction to reduce plasma cell burden, lower dose of melphalan [140 mg/m 2 ] given as a single one-time dose in patients with poor glomerular filtration rate, cardiac monitoring, better fluid volume balance control, and lower cutoff for cardiac biomarkers) has led to improved early survival outcomes after ASCT over the past 15 years.…”

Section: The Role Of Autologous Stem-cell Transplantationmentioning

confidence: 99%

Diagnostic and Treatment Strategies for AL Amyloidosis in an Era of Therapeutic Innovation

Dima

Mazzoni

Anwer

et al. 2023

JCO Oncology Practice

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Despite significant progress and improving outcomes in the management of plasma cell disorders, AL amyloidosis remains diagnostically and therapeutically challenging for clinicians across practice settings. There is, however, a reason for optimism with the advent of new combination therapy approaches and novel targets offering the promise of improvement in end organ function, survival, and quality of life. This review offers a clinically applicable overview of an approach to diagnosis, risk stratification, and clinical management of AL amyloidosis in an era of rapid therapeutic innovation.

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High Dose Therapy with Autologous Hematopoietic Stem Cell Transplantation (HSCT) for Patients with Primary Systemic Amyloidosis (AL): An Abmtr Study

Cited by 4 publications

References 0 publications

Outcome of autologous stem cell transplantation for AL amyloidosis in the UK

Outcome of autologous stem cell transplantation for AL amyloidosis in the UK

Perspectives in treatment of AL amyloidosis

Diagnostic and Treatment Strategies for AL Amyloidosis in an Era of Therapeutic Innovation

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