Organ responses after highdose melphalan and stemcell transplantation in AL amyloidosis

Szalat, Raphaël; Sarosiek, Shayna; Havasi, Andrea; Brauneis, Dina; Sloan, J. Mark; Sanchorawala, Vaishali

doi:10.1038/s41375-020-1006-7

Cited by 24 publications

(15 citation statements)

References 16 publications

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“…Generally, a deep hematologic response (VGPR or better) is required for organ response, but even those who achieve CR can endure persistent organ dysfunction. It is believed that incomplete elimination of clonal PCs after treatment may hinder organ recovery (Staron et al 2020;Szalat et al 2020). In our study, patients with a deep hematologic response who achieved MRD negativity had a higher rate of response in any organ, especially cardiac response (93% (13/14) vs 25% (1/4), p = 0.019), than those who had MRD positivity.…”

Section: The Relationship Between Mrd and Survivalmentioning

confidence: 99%

Clinical value of minimal residual disease assessed by multiparameter flow cytometry in amyloid light chain amyloidosis

Huang

Liu

et al. 2021

J Cancer Res Clin Oncol

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Purpose: To assess the feasibility and prognostic value of minimal residual disease (MRD) evaluated by multiparameter ow cytometry (MFC) in newly diagnosed amyloid light chain (AL) amyloidosis.Methods: Clinical data from 25 consecutive newly diagnosed AL amyloidosis patients with MRD data tested at 3 months after rst-line therapy completion were retrospectively analysed in a single centre from 2012 to 2019. First-line therapy included 8 courses of VD or 4 courses of VD plus sequential autologous stem cell transplantation (ASCT), both without maintenance therapy.Results: Of 25 patients with very good partial response (VGPR) or better, 19 (76%) achieved MRD negativity. Baseline characteristics were not different between MRD-negative and MRD-positive patients. More ASCT patients than non-ASCT patients (90.0% vs 53.3%, P 0.043) achieved MRD negativity. In the MRD-negative and MRD-positive groups, cardiac response was observed in 93% and 25% (P=0.019) and any organ response in 94% and 50%, respectively (P=0.023). At a median follow-up of 25.1 months, MRDnegative patients showed signi cantly longer progression-free survival (PFS) from diagnosis than MRDpositive patients (24.52 vs 76.39 months, P=0.004).Conclusions: MRD negativity measured by MFC at 3 months after rst-line therapy completion in patients with AL amyloidosis is measurable and associated with improved organ response rates and PFS over a long follow-up.

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Section: The Relationship Between Mrd and Survivalmentioning

confidence: 99%

Clinical value of minimal residual disease assessed by multiparameter flow cytometry in amyloid light chain amyloidosis

Huang

Liu

et al. 2021

J Cancer Res Clin Oncol

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“…High dose chemotherapy and autologous stem cell transplantation (HDT-AuSCT) has been in use since the early 1990s and is a proven treatment modality in AL with hematologic and organ responses as well as long term survival data to support its use. (37)(38)(39) Some studies have reported increased rates of treatmentrelated mortality (TRM) as high as 24%, typically in patients with advanced cardiac involvement, though in our experience with careful patient selection these mortality rates are lower at 3-5%, though still increased compared to patients with multiple myeloma. (40) The track record of proven decades long favorable outcomes for patients with AL, who often are observed without maintenance therapy after HDT-AuSCT, is one advantage that we cannot yet claim to know with daratumumab based therapies in the upfront setting.…”

Section: What Is the Current Role Of Autologous Stem Cell Transplantation For These Patients?mentioning

confidence: 80%

How I Manage Systemic Light Chain Amyloidosis

Kaufman

Cerchione

2021

Hematology Reports

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Light chain amyloidosis (AL) is a protein deposition disorder with a heterogenous pattern of organ involvement and dysfunction that varies by affected patient. Often diagnosed late after the onset of symptoms, appropriate and correct diagnosis, and prompt initial management, typically with anti-plasma cell therapy targeting the underlying cell producing the aberrant light chain protein, can lead to significant improvement in patient symptoms, organ function and lifespan. With recent publication of phase III studies focused on AL, and a changing regulatory landscape providing greater availability of novel therapies, the management of AL is in some ways more complex with greater options to consider. In this clinical review, a discussion of the diagnosis, staging, and goals of therapy transitions to a focus on contemporary management questions to outline our clinical approach to multi-disciplinary management and long term follow up for patients with suspected or confirmed AL.

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“…Regarding melphalan conditioning dose, a higher dose (70 - 200 mg/m 2 ) preceding ASCT was associated with a better hematological response and increased median OS than a lower dose [ 17 , 18 ]. Moreover, a high melphalan induction dose of 200 mg/m 2 was tolerable in patients above the age of 65 with good performance status (Karnofsky Performance status scale of ≥ 90) with no increase in TRM except for a noticeable increase in post-transplant febrile neutrophilia and infections [ 57 ].…”

Section: Autologous Stem Cell Transplantation (Asct)mentioning

confidence: 99%

Current Updates on the Management of AL Amyloidosis

El‐Sayed¹,

Usher²,

Habib³

et al. 2021

J Hematol

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Systemic immunoglobulin light chain (AL) amyloidosis is a rare but fatal disease. It results from clonal proliferation of plasma cells with excessive production of insoluble misfolded proteins that aggregate in the extracellular matrix, causing damage to the normal architecture and function of various organs. For decades, treatment for AL amyloidosis was based mainly on therapeutic agents previously studied for its more common counterpart, multiple myeloma. As the prevalence and incidence of AL amyloidosis have increased, ongoing research has been conducted with treatments typically used in myeloma with varying success. In this review, we focus on current treatment strategies and updates to clinical guidelines and therapeutics for AL amyloidosis.

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Organ responses after highdose melphalan and stemcell transplantation in AL amyloidosis

Cited by 24 publications

References 16 publications

Clinical value of minimal residual disease assessed by multiparameter flow cytometry in amyloid light chain amyloidosis

Clinical value of minimal residual disease assessed by multiparameter flow cytometry in amyloid light chain amyloidosis

How I Manage Systemic Light Chain Amyloidosis

Current Updates on the Management of AL Amyloidosis

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