Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death

Muchtar, Eli; Gertz, Morie A.; Kumar, Shaji; Lacy, Martha Q.; Dingli, David; Buadi, Francis K.; Grogan, Martha; Hayman, Suzanne R.; Kapoor, Prashant; Leung, Nelson; Fonder, Amie; Hobbs, Miriam; Hwa, Yi L.; Gonsalves, Wilson I.; Warsame, Rahma; Kourelis, Taxiarchis; Russell, Stephen J.; Lust, John A.; Lin, Yi; Go, Ronald S.; Zeldenrust, Steven R.; Kyle, Robert A.; Rajkumar, S. Vincent; Dispenzieri, Angela

doi:10.1182/blood-2016-11-751628

Cited by 271 publications

(231 citation statements)

References 25 publications

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“…Despite the reduced survival of our cohort, our data indicate that survival for Australian patients with amyloidosis is improving. Similar improvements in survival have been reported in other countries, particularly with respect to AL amyloidosis (Kumar et al , ; Muchtar et al , ). While this may reflect the addition of drugs, such as bortezomib (Palladini et al , ) and lenalidomide (Kumar et al , ), into therapeutic regimens, randomised trial data showing a survival benefit for these regimens is not available.…”

Section: Discussionsupporting

confidence: 81%

“…While this may reflect the addition of drugs, such as bortezomib (Palladini et al , ) and lenalidomide (Kumar et al , ), into therapeutic regimens, randomised trial data showing a survival benefit for these regimens is not available. Other explanations for the improving survival among AL amyloidosis patients include earlier diagnosis (Muchtar et al , ), and greater experience with autologous stem cell transplantation (D’Souza et al , ). It is probable that improvements in amyloidosis diagnosis, coupled with the use of more effective therapies for AL amyloidosis, have resulted in the improving survival we observed in our cohort.…”

Section: Discussionmentioning

confidence: 99%

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The epidemiology of amyloidosis in Queensland, Australia

Wisniowski¹,

McLeod

Adams

et al. 2019

Br J Haematol

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Summary Published studies on the epidemiology of amyloidosis have relied on death certificate data for case ascertainment. We estimated the incidence and mortality burden of amyloidosis among residents of the Australian state, Queensland, aged ≥20 years for the years 1999–2013 based on case ascertainment from histopathology reports. Information systems for participating laboratories were scrutinised to identify histopathology reports that documented a diagnosis of amyloidosis. Case mortality status was determined via linkage to the National Death Index. A total of 447 cases of amyloidosis were identified, with a median age at diagnosis of 66 years. A plasma cell dyscrasia was identified in 72% of patients who had paraprotein studies performed. The estimated incidence for Queenslanders aged ≥20 years was 12·1 cases per million person years. The median survival was 2·45 years. Age at diagnosis, presence of a paraprotein, earlier year of diagnosis, and inner regional location of residence (compared with residence in a major city) were independently associated with reduced survival. Our data confirms previously reported incidence data for amyloidosis of approximately 10 cases per million patient years and indicates that survival for Queensland patients with amyloidosis is improving, though it remains poor for the elderly and patients with AL amyloidosis.

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Section: Discussionsupporting

confidence: 81%

Section: Discussionmentioning

confidence: 99%

The epidemiology of amyloidosis in Queensland, Australia

Wisniowski¹,

McLeod

Adams

et al. 2019

Br J Haematol

View full text Add to dashboard Cite

show abstract

“…Despite the fact that our study could not determine the mechanisms responsible for the observed change, a study by Muchtar et al with 1551 newly diagnosed AL amyloidosis patients seen at the Mayo Clinic between 2000 and 2014 found a significant improvement in overall survival over time, a change that could explain our findings. 25 Continual enhanced survival is only possible if AL amyloidosis symptoms and cases continue to be identified earlier and characterized accurately. Awareness is essential for timely detection and treatment of amyloidosis.…”

Section: Discussionmentioning

confidence: 99%

Epidemiology of AL amyloidosis: a real-world study using US claims data

et al. 2018

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“…Our UK analysis (2003–2012) also suggested that an improvement in TRM lagged behind that of the US (6·8% vs. 5% in the UK and US respectively) (Gertz et al , ; Venner et al , ). Since this last analysis (Venner et al , ), multiple highly effective bortezomib‐based chemotherapy combinations have been introduced with a corresponding improvement in the survival outcomes for patients with AL amyloidosis (Muchtar & Gertz, ). This has prompted the present retrospective analysis of the TRM, as well as long‐term survival outcomes of all patients with AL amyloidosis, treated with an ASCT over a 24‐year period (1994–2018).…”

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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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Improved outcomes for newly diagnosed AL amyloidosis between 2000 and 2014: cracking the glass ceiling of early death

Abstract: Key Points Significant improvement in outcome is a reality for newly diagnosed AL amyloidosis in the past decade. Six-month mortality among transplant ineligible patients has declined since 2005.

Cited by 271 publications

References 25 publications

The epidemiology of amyloidosis in Queensland, Australia

The epidemiology of amyloidosis in Queensland, Australia

Epidemiology of AL amyloidosis: a real-world study using US claims data

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

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