Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis
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Objective: To study the outcome of patients with AL amyloidosis who were ineligible for high dose melphalan (HDM) and autologous stem cell transplantation (ASCT). Methods: A real-life retrospective observational cohort study of Dutch patients with AL amyloidosis ineligible for HDM and ASCT was performed at the University Medical Center Groningen from January 2001 until April 2017. Primary outcome measure was overall survival (OS). ⋯ Incidence of toxicity was high (51%). Conclusion: OS improved in this high-risk group over the years, especially after introduction of new treatment modalities. However, early death rate remains high, illustrating the need for more effective treatment.
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Clinical Trial
The utility of repeat kidney biopsy in systemic immunoglobulin light chain amyloidosis.
Background: The diagnostic utility of repeat kidney biopsy in AL amyloidosis patients in complete (CR) or very good partial hematologic response (VGPR) but with renal organ relapse is not clear. Methods: We present eight patients with AL amyloidosis who had a repeat kidney biopsy performed. Results: AL amyloidosis was initially diagnosed by a kidney biopsy. ⋯ Amyloid deposits persist in the kidneys even after successful hematologic treatment and it is impossible to differentiate between new versus old amyloid deposits, which makes performing a repeat kidney biopsy unnecessary in most cases. Demonstration of amyloid deposits on repeat kidney biopsy would not aid in the decision making regarding re-initiation of hematologic treatment. A kidney biopsy should be considered only in cases when a specific alternative diagnosis is suspected.
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Pragmatic Clinical Trial
Immunoelectron microscopy and mass spectrometry for classification of amyloid deposits.
Amyloidosis is a shared name for several rare, complex and serious diseases caused by extra-cellular deposits of different misfolded proteins. Accurate characterization of the amyloid protein is essential for patient care. Immunoelectron microscopy (IEM) and laser microdissection followed by tandem mass spectrometry (LMD-MS) are new gold standards for molecular subtyping. ⋯ Concordance was 89.6-100% for different amyloid subtypes. Importantly, combined use of both methods increased the diagnostic classification to 100%. Some variety in performances at organ level was observed.