Use of novel therapies in the treatment of light chain amyloidosis

Varga, Cindy; Titus, Stephanie E.; Toskic, Denis; Comenzo, Raymond L.

doi:10.1016/j.blre.2019.05.005

Cited by 13 publications

(13 citation statements)

References 127 publications

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“…Other considerations regarding the generalizability of our results include the ever‐changing landscape of available treatments for AL amyloidosis, as well as the recent evidence suggesting the important impact of cytogenetics on prognosis and response to specific treatments 17,26,27 . The emergence of new investigational therapies over the past several years has assisted in improving both prognosis and survival in this patient population 28‐30 . So while our analysis displays compelling evidence supporting the need for early diagnosis in patients with AL amyloidosis, an analysis of more recent data taking into account these novel therapies may be necessary.…”

Section: Discussionmentioning

confidence: 88%

Patient outcomes in light chain (AL) amyloidosis: The clock is ticking from symptoms to diagnosis

Schulman

Connors

Weinberg

et al. 2020

European J of Haematology

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Introduction Symptomology of AL amyloidosis can be vague, with a broad range of manifestations and potential etiologies. We sought to determine whether time from initial patient‐reported symptom onset to diagnosis was associated with survival. Methods The Boston University Amyloidosis Patient Database was queried for patients with AL amyloidosis who presented to the Center for initial evaluation from 2010 to 2015. Results A total of 324 patients with AL amyloidosis were evaluated for initial evaluation. The median time to diagnosis from initial symptom onset was 7.1 months (range, 0‐61). At data cutoff, 60.2% (n = 195) of patients were alive; of those, the majority were diagnosed <6 months from initial symptoms (52.3%, n = 102). In contrast, time to diagnosis from symptom onset was >6 months in 63.6% (n = 82) of patients who did not survive at the time of data cutoff (P = .0005). Survival analysis of time from diagnosis to death or data cutoff stratified by time from patient‐reported symptom onset to diagnosis (<6, 6‐12, and >12 months) showed significant differences among groups (P = .001). Additionally, multivariable regression demonstrated that an increase in time from self‐reported symptom onset to diagnosis was significantly associated with an increased risk of death (HR = 1.02, 95% CI = 1.01‐1.04, P = .002). Conclusion These results support the importance of early diagnosis for patients with AL amyloidosis.

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Section: Discussionmentioning

confidence: 88%

Patient outcomes in light chain (AL) amyloidosis: The clock is ticking from symptoms to diagnosis

Schulman

Connors

Weinberg

et al. 2020

European J of Haematology

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“…Current therapeutic agents include proteasome inhibitors, cyclophosphamide, dexamethasone, and daratumumab; however, the use of thalidomide and lenalidomide are limited as a result of toxicity with worsening heart failure. 1 Although t(11;14) is relatively common in MM, it is even more prevalent in systemic light chain amyloidosis, with 60% of patients harboring this translocation. This results in a higher BCL-2:MCL-1 ratio and lower response rates to standard therapies, 2 so inhibiting BCL-2 is a logical step.…”

Section: Introductionmentioning

confidence: 99%

Venetoclax in Immunoglobulin Light Chain Amyloidosis: Is This the Beginning or the End?

Premkumar

Comenzo

Lentzsch

2019

Clinical Lymphoma Myeloma and Leukemia

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“…As a multisystemic disorder, AL amyloidosis can affect the heart, kidneys, liver, nerves, lungs, and bowel. Common management approaches include anti-plasma cell systemic chemotherapy and high-dose chemotherapy followed by ASCT; however, only 20-25% of patients with AL amyloidosis are considered eligible for stem cell transplantation based on age, renal function, and severity of heart failure (18)(19)(20). Effective therapy exists, although it is ineffective if the end-organ damage is severe.…”

Section: Discussionmentioning

confidence: 99%

A prognostic staging system for light-chain amyloidosis using hepatic and renal indicator data from 1,064 Chinese patients

et al. 2021

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Background: Light-chain (AL) amyloidosis frequently involves severe multiple end-organ damage, thus affecting prognosis. As the current disease staging system is based only on cardiac indicators, we propose a new staging system based on multiple organ indicators to supplement the existing system. Methods: Patients with AL amyloidosis (n=1,064) from 18 Chinese hospitals were enrolled and divided into test and validation cohorts (4:1). Multivariate analyses were performed to identify the clinical and laboratory factors for inclusion in the new staging system.Results: A score of 1 was assigned for each of the following-the difference between the involved and uninvolved free light chains ≥100 mg/L, estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m 2 , total bilirubin (Tbil) ≥18 µmol/L, cardiac troponin I ≥0.06 µg/L, and N-terminal pro-brain natriuretic peptide (NT-proBNP) ≥3,600 pg/mL-to divide the patients into five disease stages (0 to IV). There were 220 (20.7%), 291 (27.3%), 251 (23.6%), 178 (16.7%), and 124 (11.7%) patients with stage 0, I, II, III, and IV disease, respectively. Patients with stage II, III, and IV disease had a median overall survival (OS) of 56.9 months [95% confidence interval (CI), 33.9-not reached (NR)], 18.6 months (95% CI, 33.9-NR), and 6.5 months (95% CI, 8.0-24.6) (P<0.001), respectively. The 3-year survival estimates for patients with stages 0, I, II, III, and IV were 90.7%, 71.4%, 59.4%, 39.0%, and 22.1%, respectively.Conclusions: The new staging system has been developed that incorporates plasma cell-related characteristics in addition to cardiac, renal, and hepatic function parameters. It enhances the risk stratification of patients with AL amyloidosis and is useful when multiple organs are involved.

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Use of novel therapies in the treatment of light chain amyloidosis

Cited by 13 publications

References 127 publications

Patient outcomes in light chain (AL) amyloidosis: The clock is ticking from symptoms to diagnosis

Patient outcomes in light chain (AL) amyloidosis: The clock is ticking from symptoms to diagnosis

Venetoclax in Immunoglobulin Light Chain Amyloidosis: Is This the Beginning or the End?

A prognostic staging system for light-chain amyloidosis using hepatic and renal indicator data from 1,064 Chinese patients

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