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Article
Open AccessLight chain mutations contribute to defining the fibril morphology in systemic AL amyloidosis
Systemic AL amyloidosis is one of the most frequently diagnosed forms of systemic amyloidosis. It arises from mutational changes in immunoglobulin light chains. To explore whether these mutations may affect th...
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Article
Open AccessPatient experiences of interprofessional collaboration and intersectoral communication in rare disease healthcare in Germany – a mixed-methods study
Rare diseases are often complex, chronic and many of them life-shortening. In Germany, healthcare for rare diseases is organized in expert centers for rare diseases. Most patients additionally have regional ge...
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Article
Versorgung von Patienten mit kardialer Amyloidose
Amyloidosen zählten historisch zu den seltenen Erkrankungen und wurden oft zentralisiert in wenigen spezialisierten Zentren versorgt. Resultierend aus den Fortschritten in der nichtinvasiven kardialen Diagnost...
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Article
Open AccessHigh-throughput electron tomography identifies centriole over-elongation as an early event in plasma cell disorders
Plasma cell disorders are clonal outgrowths of pre-malignant or malignant plasma cells, characterized by extensive chromosomal aberrations. Centrosome abnormalities are a major driver of chromosomal instabilit...
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Article
Open AccessCommon transthyretin-derived amyloid fibril structures in patients with hereditary ATTR amyloidosis
Systemic ATTR amyloidosis is an increasingly important protein misfolding disease that is provoked by the formation of amyloid fibrils from transthyretin protein. The pathological and clinical disease manifest...
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Article
Open AccessFirst third-generation CAR T cell application targeting CD19 for the treatment of systemic IgM AL amyloidosis with underlying marginal zone lymphoma
Light chain amyloidosis (AL) is a rare disease caused by the generalized deposition of misfolded free light chains. Patients with immunoglobulin M gammopathy (IgM) and indolent B-cell lymphoma such as marginal...
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Article
Open AccessBelantamab Mafodotin-assoziierte Keratopathie – eine häufige und therapiebeeinflussende Nebenwirkung
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Article
Open AccessDifferent treatment strategies versus a common standard arm (CSA) in patients with newly diagnosed AML over the age of 60 years: a randomized German inter-group study
A randomized inter-group trial comparing more intensive treatment strategies to a common standard arm 3 + 7 (CSA) was conducted in patients with non-M3 AML. Untreated patients ≥ 60 years were allocated to the ...
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Article
Open AccessThe management of light chain (AL) amyloidosis in Europe: clinical characteristics, treatment patterns, and efficacy outcomes between 2004 and 2018
Systemic light-chain (AL) amyloidosis is a rare and debilitating disease. Advances have been made in new treatments in recent years, yet real-world data on the management of the disease are scarce. EMN23 is a ...
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Article
Open AccessThiotepa–fludarabine–treosulfan conditioning for 2nd allogeneic HCT from an alternative unrelated donor for patients with AML: a prospective multicenter phase II trial
Therapeutic options for patients with AML relapsing after allogeneic HCT range from chemotherapy or hypomethylating agents with or without donor lymphocyte infusions to a 2nd allogeneic HCT. Available data are...
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Article
Open AccessCryo-EM structure of an ATTRwt amyloid fibril from systemic non-hereditary transthyretin amyloidosis
Wild type transthyretin-derived amyloid (ATTRwt) is the major component of non-hereditary transthyretin amyloidosis. Its accumulation in the heart of elderly patients is life threatening. A variety of genetic ...
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Article
Open AccessOutcomes of renal transplantation in patients with AL amyloidosis: an international collaboration through The International Kidney and Monoclonal Gammopathy Research Group
Effective systemic therapies suppress toxic light chain production leading to an increased proportion of patients with light chain (AL) amyloidosis who survive longer albeit with end-stage renal disease. There...
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Chapter
Amyloidose
Unter dem Begriff Amyloidose wird eine heterogene Gruppe von Erkrankungen zusammengefasst, bei denen unterschiedliche Proteine in unlöslicher Form deponiert werden und dadurch die umgebenden Strukturen und Org...
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Article
Open AccessRole of mutations and post-translational modifications in systemic AL amyloidosis studied by cryo-EM
Systemic AL amyloidosis is a rare disease that is caused by the misfolding of immunoglobulin light chains (LCs). Potential drivers of amyloid formation in this disease are post-translational modifications (PTM...
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Article
Correction to: Allogeneic hematopoietic stem cell transplantation improves long‑term outcome for relapsed AML patients across all ages: results from two East German Study Group Hematology and Oncology (OSHO) trials
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Article
Open AccessAllogeneic hematopoietic stem cell transplantation improves long-term outcome for relapsed AML patients across all ages: results from two East German Study Group Hematology and Oncology (OSHO) trials
Relapse of acute leukemia is a frequent complication with uncertain outcome and poorly defined risk factors. From 1621 patients entered into two prospective clinical trials (AML02; n = 740 and AML04; n = 881),...
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Article
Open AccessLetermovir prophylaxis is effective in preventing cytomegalovirus reactivation after allogeneic hematopoietic cell transplantation: single-center real-world data
Morbidity and mortality after allogeneic hematopoietic cell transplantation (alloHCT) are still essentially affected by reactivation of cytomegalovirus (CMV). We evaluated 80 seropositive patients transplanted...
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Article
Open AccessSolid state NMR assignments of a human λ-III immunoglobulin light chain amyloid fibril
The aggregation of antibody light chains is linked to systemic light chain (AL) amyloidosis, a disease where amyloid deposits frequently affect the heart and the kidney. We here investigate fibrils from the λ-...
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Article
Open AccessImpaired in vitro growth response of plasma-treated cardiomyocytes predicts poor outcome in patients with transthyretin amyloidosis
Direct toxic effects of transthyretin amyloid in patient plasma upon cardiomyocytes are discussed. However, no data regarding the relevance of this putative effect for clinical outcome are available. In this m...
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Article
Open AccessCryo-EM reveals structural breaks in a patient-derived amyloid fibril from systemic AL amyloidosis
Systemic AL amyloidosis is a debilitating and potentially fatal disease that arises from the misfolding and fibrillation of immunoglobulin light chains (LCs). The disease is patient-specific with essentially e...
