Successful reduced intensity allogeneic stem cell transplantation for systemic AL amyloidosis

Imamura, Tomoyuki; Ogata, Masao; Kohno, Kazuhiro; Tomo, Tadashi; Ohtsuka, Eiichi; Kojima, Hiroshi; Kadota, Jun‐ichi

doi:10.1002/ajh.20544

Cited by 8 publications

(5 citation statements)

References 9 publications

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“…Due to amyloid related organ disease, toxicity of allogeneic SCT can be substantial in AL patients [60]. However, the success of this strategy in small numbers of patients provides proof of principal that antitumor immune effects may be important in AL patients [61-64]. A review of 19 patients from the European Group for Blood and Marrow Transplantation (EBMT) registry reported one year OS and progression free survival (PFS) as 60% and 53% respectively; at two years OS and PFS were 52% and 46%, respectively [60].…”

Section: Therapeutic Options In Al Amyloidosismentioning

confidence: 99%

Light chain (AL) amyloidosis: update on diagnosis and management

Rosenzweig

Landau

2011

J Hematol Oncol

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Light chain (AL) amyloidosis is a plasma cell dyscrasia characterized by the pathologic production of fibrillar proteins comprised of monoclonal light chains which deposit in tissues and cause organ dysfunction. The diagnosis can be challenging, requiring a biopsy and often specialized testing to confirm the subtype of systemic disease. The goal of treatment is eradication of the monoclonal plasma cell population and suppression of the pathologic light chains which can result in organ improvement and extend patient survival. Standard treatment approaches include high dose melphalan (HDM) followed by autologous hematopoietic stem cell transplantation (SCT) or oral melphalan with dexamethasone (MDex). 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. A risk adapted approach to SCT followed by novel agents as consolidation reduces treatment related mortality with promising outcomes. Immunotherapeutic approaches targeting pathologic plasma cells and amyloid precursor proteins or fibrils are being developed. Referral of patients to specialized centers focusing on AL amyloidosis and conducting clinical trials is essential to improving patient outcomes.

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Section: Therapeutic Options In Al Amyloidosismentioning

confidence: 99%

Light chain (AL) amyloidosis: update on diagnosis and management

Rosenzweig

Landau

2011

J Hematol Oncol

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“…81 Anecdotal reports suggest RIC-SCT is possible in AL. 82 Currently, allogeneic stem cell transplantation may have a role in highly selected young fit patients with relapsed disease but, ideally, should only be considered in the context of a clinical trial. …”

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confidence: 99%

Update on treatment of light chain amyloidosis

et al. 2014

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Light chain amyloidosis is the most common type of amyloidosis as a consequence of protein misfolding of aggregates composed of amyloid fibrils. The clinical features are dependent on the organs involved, typically cardiac, renal, hepatic, peripheral and autonomic neuropathy and soft tissue. A tissue biopsy or fat aspirate is needed to confirm the presence/type of amyloid and prognostic tools are important in a risk stratified approach to treatment. Autologous stem cell transplant eligibility should be assessed at baseline, weighing the reversible or non-reversible contraindications, toxicity of treatment and chemotherapy alternatives available. Chemotherapy options include melphalan, thalidomide, bortezomib, lenalidomide, bendamustine in combination with dexamethasone. Many studies have explored these treatment modalities, with ongoing debate about the optimal first line and sequential treatment thereafter. Attaining a very good partial response or better is the treatment goal coupled with early assessment central to optimizing treatment. One major challenge remains increasing the awareness of this disease, frequently diagnosed late as the presenting symptoms mimic many other medical conditions. This review focuses on the treatments for light chain amyloidosis, how these treatments have evolved over the years, improved patient risk stratification, toxicities encountered and future directions. ABSTRACT © F e r r a t a S t o r t i F o u n d a t i o n S. Mahmood et al.210 haematologica | 2014; 99(2) rectum or abdominal fat aspirate (the latter being an easy bedside procedure available for all patients including those with hemostatic impairment). 7 Fibril typing is critical in deciding appropriate therapy and performed by immunohistochemistry (widely available but specific only in 75-80% of cases of AL), 8 immunoelectron microscopy (highly specific but limited availability), 9 or lately, mass spectrometry of amyloid deposits obtained by laser capture (rapidly becoming an invaluable adjunct) 10 ( Figure 2). Detecting the underlying clone requires serum and urine electrophoresis and immunofixation, serum free light chain analysis, bone marrow examination and imaging for presence of myeloma-related bone disease.Base-line assessment of organ function (Table 2) is ECG showing small QRS complexes and late gadolinium enhancement of cardiac MRI. The pre-fibrillar light chain aggregates (and possibly the misfolded light chains) can have direct tissue toxicity. Cardiac toxicity of light chains appears to be a significant contributor to myocardial dysfunction seen in AL amyloidosis. This may also be the reason for rapid improvement in NT-proBNP which parallels a hematologic response to therapy often without any evidence of structural cardiac improvement but correlating with clinical improvement in the patients' cardiac symptoms. © F e r r a t a S t o r t i F o u n d a t i o nimportant for prognosis and selection of therapy. Formal testing for autonomic and peripheral neuropathy may be needed in selected cases. 123I labeled s...

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“…Allogeneic hematopoietic transplantation has very rarely been performed as therapy of systemic AL amyloidosis [11,12]. The majority of patients with AL amyloidosis have a low plasma cell burden.…”

Section: Evolution Of Transplant For Al Amyloidosismentioning

confidence: 99%

A Short Review of Hematopoietic Transplantation for AL Amyloidosis

Danylesko¹

2022

Amyloidosis - History and Perspectives

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AL amyloidosis is an uncommon disease with variable clinical presentations; as such, it is often initially unrecognized and diagnosis is therefore frequently delayed. As a result of diagnosis at a point in their disease when patients often have significant end-organ damage, aggressive therapy with major toxicities can be extremely challenging. Nonetheless, clinical data have been accumulating over the past several decades that have demonstrated that patients who were taken to high-dose therapy—typically using single-agent L-phenylalanine mustard—with autologous hematopoietic rescue, have a dramatically improved overall survival than otherwise. In this chapter, the critical clinical data that demonstrate this, and the risk-adjusted approach to optimize the outcome for patients, are reviewed.

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Successful reduced intensity allogeneic stem cell transplantation for systemic AL amyloidosis

Cited by 8 publications

References 9 publications

Light chain (AL) amyloidosis: update on diagnosis and management

Light chain (AL) amyloidosis: update on diagnosis and management

Update on treatment of light chain amyloidosis

A Short Review of Hematopoietic Transplantation for AL Amyloidosis

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