Guidelines on the management of <scp>AL</scp> amyloidosis

Wechalekar, Ashutosh; Gillmore, Julian D.; Bird, Jenny; Cavenagh, Jamie; Hawkins, S.J.; Kazmi, Majid; Lachmann, HJ; Hawkins, Philip N.; Pratt, Guy

doi:10.1111/bjh.13155

Cited by 114 publications

(124 citation statements)

References 115 publications

(106 reference statements)

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“…As there is no diagnosis code specific to AL amyloidosis, the following algorithm, which was used in a previously published study, 16 was used to identify patients in each calendar year during the study period ( [17][18][19][20][21] for AL amyloidosis (bendamustine, bortezomib, carfilzomib, cyclophosphamide, dexamethasone, prednisone, doxycycline, lenalidomide, melphalan, pomalidomide, thalidomide, or hematopoietic stem cell transplant [HSCT]) on or after the first amyloidosis diagnosis in the study period. A similar population-identification algorithm was used in a recent publication and confirmed with clinical input.…”

Section: Study Population and Measuresmentioning

confidence: 99%

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

et al. 2018

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Section: Study Population and Measuresmentioning

confidence: 99%

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

et al. 2018

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“…Recently, the Mayo Clinic and British Society for Hematology published two guidelines on the management of AL amyloidosis. Both guidelines present an extensive review of the literature and were aimed to make recommendations in the context of the best evidence and expert opinion [18,19]. Current treatment strategies target the clone to decrease the production of the pathologic light chains and thereby stop or reverse organ toxicity and damage.…”

Section: Management Of Al Amyloidosismentioning

confidence: 99%

“…Proteasome inhibitor-based regimens are a preferred choice due to better response rates and outcomes in phase II studies, and a bortezomib-alkylator steroid combination is preferred where a rapid response is desirable (cardiac involvement, renal impairment, severe hypoalbuminemia, and fluid retention) [19]. Several studies have confirmed that bortezomib combined with dexamethasone (BD) is an active and fast-acting regimen for AL amyloidosis, even in pretreated patients [24,25].…”

Section: Management Of Al Amyloidosismentioning

confidence: 99%

The Clinical Presentation and Management of Systemic Light-Chain Amyloidosis in China

Huang

Liu

2016

Kidney Dis

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Background: Amyloidosis includes a group of diseases characterized by the extracellular deposition of various fibrillary proteins that can autoaggregate in a highly abnormal fibrillary conformation. The amyloid precursor protein of systemic light-chain (AL) amyloidosis is comprised of monoclonal light chains that are due to plasma cell dyscrasia. The clinical presentation of patients with AL amyloidosis varies from patient to patient. Current treatment strategies target the clone in order to decrease the production of the pathologic light chains. Recent advances in therapy have helped many patients with AL amyloidosis achieve hematologic and organ responses. Summary: AL amyloidosis is the most common type of systemic amyloidosis in China with increasing morbidity and a high mortality rate. The clinical presentation of AL amyloidosis is variable, and the median overall survival was found to be 36.3 months. The disease prognosis and risk stratification are linked to serialized measurement of cardiac biomarkers and free light chains. The treatment of AL amyloidosis is mainly based on chemotherapy and autologous hematopoietic stem cell transplantation (ASCT). The use of novel agents (thalidomide, lenalidomide, and bortezomib) alone and in combination with steroids and alkylating agents has shown efficacy and continues to be explored. Key Messages: AL amyloidosis is the most common type of systemic amyloidosis in China with increasing morbidity and a high mortality rate. The lack of prospective clinical trials using the current therapies is a challenge for evidence-based decision making concerning the treatment of AL amyloidosis. Facts from East and West: (1) AL amyloidosis is the most prevalent type of amyloidosis accounting for 65% of the amyloidosis-diagnosed patients in the UK and for 93% of the amyloidosis-diagnosed patients in China. The predisposition of men over women to develop AL amyloidosis might be higher in China than in Western countries (2:1 vs. 1.3:1). Both in the East and West, incidence increases with age. At the time of diagnosis, edema is twice as frequent and the proportion of renal involvement is higher in Chinese compared to Western patients. (2) Melphalan followed by ASCT is the current standard therapy but is restricted to eligible patients. The efficacy and safety of bortezomib combined with dexamethasone were proven in Western patients and recently confirmed in a Chinese cohort. Recent studies in China and the US indicate that bortezomib induction prior to ASCT increases the response rate. Thalidomide and lenalidomide have shown benefit, but toxicity and lack of clinical evidence exclude these agents from first-line therapy. The green tea extract epigallocatechin-3-gallate is under investigation as an inhibitor of AL amyloid formation and a compound that might dissolve amyloid.

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“…The course of the disease is relatively benign in most patients with no effect on life expectancy [41], but severe damage to the affected organ can ultimately occur. If symptomatic, localized amyloidosis can be treated by radiotherapy or by local excision using either classic surgical techniques or laser-based excision [8, 39]. Coexisting autoimmune diseases were reported in 7% of patients [39].…”

Section: Localized Versus Systemic Amyloidosismentioning

confidence: 99%

“…Making the diagnosis remains a great challenge even for hematologists who are experts in MM. Whenever possible, patients should be treated in the context of clinical trials and preferably treatment undertaken in selected centers experienced in treating complex patients [8]. In this review, we summarize the data on the pathogenesis, diagnosis, risk stratification, and management of AL amyloidosis patients.…”

Section: Introductionmentioning

confidence: 99%

Recent Advances in the Diagnosis, Risk Stratification, and Management of Systemic Light-Chain Amyloidosis

Vaxman

Gertz

2019

Acta Haematol

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The term amyloidosis refers to a group of disorders in which protein fibrils accumulate in certain organs, disrupt their tissue architecture, and impair the function of the effected organ. The clinical manifestations and prognosis vary widely depending on the specific type of the affected protein. Immunoglobulin light-chain (AL) amyloidosis is the most common form of systemic amyloidosis, characterized by deposition of a misfolded monoclonal light-chain that is secreted from a plasma cell clone. Demonstrating amyloid deposits in a tissue biopsy stained with Congo red is mandatory for the diagnosis. Novel agents (proteasome inhibitors, immunomodulatory drugs, monoclonal antibodies, venetoclax) and autologous stem cell transplantation, used for eliminating the underlying plasma cell clone, have improved the outcome for low- and intermediate-risk patients, but the prognosis for high-risk patients is still grave. Randomized studies evaluating antibodies that target the amyloid deposits (PRONTO, VITAL) were recently stopped due to futility and currently there is an intensive search for novel treatment approaches to AL amyloidosis. Early diagnosis is of paramount importance for effective treatment and prognosis, due to the progressive nature of this disease.

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Guidelines on the management of AL amyloidosis

Cited by 114 publications

References 115 publications

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

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

The Clinical Presentation and Management of Systemic Light-Chain Amyloidosis in China

Recent Advances in the Diagnosis, Risk Stratification, and Management of Systemic Light-Chain Amyloidosis

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