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

Abstract

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.

Predictors of mortality in patients undergoing autologous hematopoietic cell transplantation admitted to the intensive care unit

“…This is consistent with earlier studies that quote treatment-related mortality rates of 13-23%. [19][20][21][22] This is also not unexpected in patients with AL amyloidosis in whom cardiac and respiratory instability are more common. Over half (54%) of the AL amyloid patients in this study were admitted to the ICU with respiratory or cardiac failure, with most patients eventually dying from multisystem organ failure because of sepsis.…”

Clinical features and treatment outcome of primary systemic light‐chain amyloidosis in Korea: Results of multicenter analysis

“…Moreover, high dose melphalan followed by autologous stem cell transplantation also has been tried for patients eligible for stem cell transplantation [5,6]. New drugs developed for multiple myeloma such as bortezomib and thalidomide are also studied for patients with AL amyloidosis [5,[7][8][9][10][11][12].…”

Outcome of AL amyloidosis after high-dose melphalan and autologous stem cell transplantation: long-term results in a series of 421 patients

“…6 Single and multicenter studies show CR rates of 16%-67%, organ responses in 25%-45% of patients, and a median overall survival (OS) of ϳ 5 years. [7][8][9][10][11][12][13][14][15][16] A case-control study demonstrated the benefit of this procedure for patients younger than 70 years compared with nontransplant regimens, most of them alkylator-based oral chemotherapy. 17 A major issue in HDM/SCT for AL amyloidosis is the potential for high treatment-related mortality (TRM) because of underlying organ dysfunction in this disease.…”