ArticleHaematologica2014
Oral melphalan and dexamethasone grants extended survival with minimal toxicity in AL amyloidosis: long-term results of a risk-adapted approach.
Article in Haematologica, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 60 papers.
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Who cites it
60 citing papers in PubMed, 148 citations in OpenAlex.
- Trial
- Trial
- A phase 1/2 study of the oral proteasome inhibitor ixazomib in relapsed or refractory AL amyloidosis.Blood · 2017Trial
- Lenalidomide/melphalan/dexamethasone in newly diagnosed patients with immunoglobulin light chain amyloidosis: results of a prospective phase 2 study with long-term follow up.Haematologica · 2017Trial
- Phase 2 trial of daily, oral epigallocatechin gallate in patients with light-chain amyloidosis.International journal of hematology · 2017Trial
- First-in-Human Phase I/II Study of NEOD001 in Patients With Light Chain Amyloidosis and Persistent Organ Dysfunction.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2016Trial
- Trial
- Limited duration of dexamethasone in newly diagnosed AL amyloidosis: impact on response, toxicity, and survival.Annals of medicine · 2026Article
- A Comprehensive Review of the Management of Light-Chain (AL) and Transthyretin (ATTR) Cardiac Amyloidosis.Reviews in cardiovascular medicine · 2025Review
- BCMA CAR-T: From Multiple Myeloma to Light-Chain Amyloidosis.Current oncology (Toronto, Ont.) · 2025Review
- Case report: Light-chain amyloidosis responsive to selinexor in combination with daratumumab and dexamethasone (SDd) therapy.Frontiers in medicine · 2024Article
- Systemic AL amyloidosis: current approach and future direction.Oncotarget · 2023Review
- Efficacy of bortezomib, cyclophosphamide and dexamethasone in cardiac AL amyloidosis.Internal medicine journal · 2022Article
- Targeted treatments of AL and ATTR amyloidosis.Heart failure reviews · 2022Review
- Immunotherapy in AL Amyloidosis.Current treatment options in oncology · 2022Review
- Severe Obstructive Cholestasis and Hypercalcemia Caused by Light-Chain Amyloidosis: A Case Report.Iranian journal of medical sciences · 2022Article
- The Influence of Deterioration of Kidney Function on the Diagnostic Power of Laboratory Parameters Used in the Prognostic Classification of AL Amyloidosis.Journal of clinical medicine · 2021Article
- Congestive heart failure associated with POEMS syndrome that was adequately distinguished from cardiac amyloidosis: a case report and literature review.Annals of translational medicine · 2021Article
- Immunoglobulin light chain amyloidosis diagnosis and treatment algorithm 2021.Blood cancer journal · 2021Review
- Pathophysiology and Therapeutic Approaches to Cardiac Amyloidosis.Circulation research · 2021Review
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The combination of oral melphalan and dexamethasone is considered standard therapy for patients with light-chain amyloidosis ineligible for autologous stem cell transplantation. However, previous trials reported different rates of response and survival, mainly because of the different proportions of high-risk patients. In the present study, including a total of 259 subjects, we treated 119 patients with full-dose melphalan and dexamethasone (dexamethasone 40 mg days 1-4), and 140 patients with advanced cardiac disease with an attenuated dexamethasone schedule (20 mg). Hematologic response rates were 76% in the full-dose group and 51% in the patients receiving the attenuated schedule; the corresponding complete response rates were 31% and 12%, respectively. The median survival was 7.4 years in the full-dose group and 20 months in the attenuated-dose group. Use of high-dose dexamethasone, amino-terminal pro-natriuretic peptide type-B >1800 ng/L, a difference between involved and uninvolved free light chains of >180 mg/L, troponin I >0.07 ng/mL, and response to therapy were independent prognostic determinants. In relapsed/refractory subjects bortezomib combinations granted high hematologic response rates (79% and 63%, respectively), proving the most effective rescue treatment after melphalan and dexamethasone. In summary, melphalan plus dexamethasone was highly effective with minimal toxicity, confirming its central role in the treatment of AL amyloidosis. Future randomized trials will clarify whether bortezomib is best used in frontline combination with melphalan and dexamethasone or as rescue treatment.
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Registered trials
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