Clarification on the definition of complete haematologic response in light-chain (AL) amyloidosis

Palladini, Giovanni; Schönland, Stefan; Sanchorawala, Vaishali; Kumar, Shaji; Wechalekar, Ashutosh; Hegenbart, Ute; Milani, Paolo; Ando, Yukio; Westermark, Per; Dispenzieri, Angela; Merlini, Giampaolo

doi:10.1080/13506129.2020.1868810

Cited by 70 publications

(47 citation statements)

References 6 publications

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“…Patients with symptomatic multiple myeloma were defined by CRAB criteria 15 . Hematologic response after first‐line treatment was evaluated as described by Palladini et al and recommended by the International Society of Amyloidosis (n = 69) 16,17 . The best hematologic response after first‐line treatment was assessed.…”

Section: Methodsmentioning

confidence: 99%

Impact of time to diagnosis on Mayo stages, treatment outcome, and survival in patients with AL amyloidosis and cardiac involvement

Oubari

Naser

Παπαθανασίου

et al. 2021

European J of Haematology

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Objective To study the impact of time to diagnosis on cardiac Mayo stages, treatment outcome, and overall survival. Methods We retrospectively analyzed 77 consecutive patients diagnosed between 2015 and 2020 with AL amyloidosis and cardiac involvement. Medical history was recorded in standardized form with the help of a questionnaire. Results Time from onset of symptoms of cardiac failure to diagnosis was correlated with the severity of cardiac involvement in modified Mayo 2004 and revised Mayo 2012 staging systems (rs = 0.30, 95% CI: 0.07‐0.50, P = .007 and rs = 0.25, 95% CI: 0.01‐0.45, P = .03). Patients with advanced Mayo 2004 stages received reduced‐intensity regimens and had a lower probability to achieve adequate hematologic‐ and cardiac response after first‐line treatment than patients with early stages (rs = 0.28, 95% CI: 0.04‐0.48, P = .01 and rs = 0.72, 95% CI: 0.55‐0.82, P < .0001) and poorer overall survival (P = .0004). Compared with patients diagnosed within the first year, patients diagnosed after 13‐18 or ≥19 months from first symptoms had a 3‐ to 5 times higher risk of dying. Our data indicate that there is a 12‐month window within which the diagnosis of AL amyloidosis needs to be established to avoid early deterioration and death. Conclusions Sensitizing physicians and raising awareness for the disease are crucial for timely diagnosis and may improve the outcome of the disease.

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Section: Methodsmentioning

confidence: 99%

Impact of time to diagnosis on Mayo stages, treatment outcome, and survival in patients with AL amyloidosis and cardiac involvement

Oubari

Naser

Παπαθανασίου

et al. 2021

European J of Haematology

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“…Haematological response was evaluated by intent-to-treat after every 3 months, according to current validated criteria 21,22 and recent response criteria for patients with a difference of involved/uninvolved FLC (dFLC) between 20 and 50 mg/L. 23,24 A very good haematological response (VGHR) was defined as the achievement of a very good partial response (VGPR), complete response (CR) or low-dFLC partial response.…”

Section: Methodsmentioning

confidence: 99%

Lenalidomide and dexamethasone in relapsed/refractory immunoglobulin light chain (AL) amyloidosis: results from a large cohort of patients with long follow‐up

et al. 2021

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Lenalidomide and dexamethasone (RD) is a standard treatment in relapsed/refractory immunoglobulin light chain (AL) amyloidosis (RRAL). We retrospectively investigated toxicity, efficacy and prognostic markers in 260 patients with RRAL. Patients received a median of two prior treatment lines (68% had been bortezomib-refractory; 33% had received high-dose melphalan). The median treatment duration was four cycles. The 3-month haematological response rate was 31% [very good haematological response (VGHR) in 18%]. The median follow-up was 56Á5 months and the median overall survival (OS) and haematological event-free survival (haemEFS) were 32 and 9 months. The 2-year dialysis rate was 15%. VGHR resulted in better OS (62 vs. 26 months, P < 0Á001). Cardiac progression predicted worse survival (22 vs. 40 months, P = 0Á027), although N-terminal prohormone of brain natriuretic peptide (NT-proBNP) increase was frequently observed. Multivariable analysis identified these prognostic factors: NT-proBNP for OS [hazard ratio (HR) 1Á71; P < 0Á001]; gain 1q21 for hae-mEFS (HR 1Á68, P = 0Á014), with a trend for OS (HR 1Á47, P = 0Á084); difference between involved and uninvolved free light chains (dFLC) and light chain isotype for OS (HR 2Á22, P < 0Á001; HR 1Á62, P = 0Á016) and hae-mEFS (HR 1Á88, P < 0Á001; HR 1Á59, P = 0Á008). Estimated glomerular filtration rate (HR 0Á71, P = 0Á004) and 24-h proteinuria (HR 1Á10, P = 0Á004) were prognostic for renal survival. In conclusion, clonal and organ biomarkers at baseline identify patients with favourable outcome, while VGHR and cardiac progression define prognosis during RD treatment.

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“…Responses were assessed at six months after each line of treatment according to the validated response criteria published by the International Society of Amyloidosis (ISA) — complete response (CR), very good partial response (VGPR), partial response (PR) and no response (NR), respectively 13,14 . All response assessment was performed on an intent‐to‐treat (ITT) basis.…”

Section: Methodsmentioning

confidence: 99%

Haematologic responses and survival do not significantly decrease with subsequent lines of therapy in systemic immunoglobulin light chain amyloidosis: results from an analysis of real‐world longitudinal data

et al. 2021

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Systemic immunoglobulin light chain amyloidosis (AL) is an incurable disorder, and the natural history is incompletely understood. In this study, we describe its natural history based on an analysis of real-world longitudinal data. All patients seen at the National Amyloidosis Centre, UK, between February 2010 and August 2019 and treated with up-front bortezomib are included. In all, 1 276 patients received the first-line treatment; 259, 85, and 32 patients received second, third, and fourth treatment lines, respectively. Among patients requiring further treatment after the first line, 77Á2% started the second line within two years of the first line; 50Á5%, 50Á6%, 40Á1% and 40Á6% of patients had achieved at least very good partial response after the first, second, third and fourth treatment lines. Median overall survival (OS) from first, second, third and fourth lines was 45 months, 56 months, 37 months and not reached, respectively (P = 0Á109). In summary, although relapses occur in AL amyloidosis, the outcomes and responses do not worsen with each subsequent relapse, making it attractive to design therapeutics with curative intent.

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Clarification on the definition of complete haematologic response in light-chain (AL) amyloidosis

Cited by 70 publications

References 6 publications

Impact of time to diagnosis on Mayo stages, treatment outcome, and survival in patients with AL amyloidosis and cardiac involvement

Impact of time to diagnosis on Mayo stages, treatment outcome, and survival in patients with AL amyloidosis and cardiac involvement

Lenalidomide and dexamethasone in relapsed/refractory immunoglobulin light chain (AL) amyloidosis: results from a large cohort of patients with long follow‐up

Haematologic responses and survival do not significantly decrease with subsequent lines of therapy in systemic immunoglobulin light chain amyloidosis: results from an analysis of real‐world longitudinal data

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