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Journals Amyloid : the International Jo...

Amyloid : the International Journal of Experimental and Clinical Investigation

https://read.qxmd.com/read/37990984/longitudinal-pet-ct-imaging-with-iodine-124-i-evuzamitide-reveals-organ-response-to-plasma-cell-immunotherapy-in-a-patient-with-al-amyloidosis
#21
JOURNAL ARTICLE
Ronald Lands, Emily B Martin, Dustin Powell, Alan Stuckey, Bryan Whittle, Spencer Guthrie, Renju Raj, Stephen J Kennel, Jonathan S Wall
No abstract text is available yet for this article.
November 22, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37981706/identification-of-calcitonin-receptor-stimulating-peptide-1-derived-amyloid-in-a-feline-c-cell-carcinoma
#22
JOURNAL ARTICLE
Tomoaki Murakami, Natsumi Kobayashi, Susumu Iwaide, Yoshiyuki Itoh, Miki Hisada, Takeshi Izawa, Mitsuru Kuwamura
No abstract text is available yet for this article.
November 19, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37870967/aa-amyloidosis-in-a-father-and-daughter-as-complication-of-pstpip1-associated-myeloid-related-proteinemia-inflammatory-pami-syndrome
#23
JOURNAL ARTICLE
Anne F Brunger, Hans L A Nienhuis, Johan Bijzet, Evelien Zonneveld-Huijssoon, Jan S F Sanders, Geertje E Legger, Reinold O B Gans, Bouke P C Hazenberg
No abstract text is available yet for this article.
October 23, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37855400/hereditary-transthyretin-amyloidosis-in-middle-aged-and-elderly-patients-with-idiopathic-polyneuropathy-a-nationwide-prospective-study
#24
JOURNAL ARTICLE
Guillaume Fargeot, Andoni Echaniz-Laguna, Céline Labeyrie, Juliette Svahn, Jean-Philippe Camdessanché, Pascal Cintas, Jean-Baptiste Chanson, Florence Esselin, Céline Piedvache, Céline Verstuyft, Steeve Genestet, Emmeline Lagrange, Laurent Magy, Yann Péréon, Sabrina Sacconi, Aissatou Signate, Aleksandra Nadaj-Pakleza, Frédéric Taithe, Karine Viala, Céline Tard, Vianney Poinsignon, Cécile Cauquil, Shahram Attarian, David Adams
BACKGROUND: Hereditary transthyretin amyloidosis (ATTRv) is an adult-onset autosomal dominant disease resulting from TTR gene pathogenic variants. ATTRv often presents as a progressive polyneuropathy, and effective ATTRv treatments are available. METHODS: In this 5 year-long (2017-2021) nationwide prospective study, we systematically analysed the TTR gene in French patients with age >50 years with a progressive idiopathic polyneuropathy. RESULTS: 553 patients (70% males) with a mean age of 70 years were included...
October 19, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37822136/the-way-to-a-man-s-heart-prostate-samples-for-the-early-detection-of-transthyretin-cardiomyopathy
#25
JOURNAL ARTICLE
Lawrence Zeldin, Karan Wats, Kathleen M O'Toole, Fabrizio Remotti, Mathew S Maurer
No abstract text is available yet for this article.
October 11, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37800869/inappropriate-use-of-technetium-99m-pyrophosphate-scanning-for-the-evaluation-of-transthyretin-amyloidosis
#26
JOURNAL ARTICLE
Crystal Lihong Yan, Nina Thakkar Rivera, James Hoffman
No abstract text is available yet for this article.
October 6, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37787511/heterogeneous-worldwide-access-and-pricing-of-tafamidis
#27
JOURNAL ARTICLE
Abdirahman Wardhere, Dimitrios Bampatsias, Nowell Fine, Pablo Garcia-Pavia, Martha Grogan, Arnt V Kristen, Thibaud Damy, Yoshiki Sekijima, Mathew S Maurer
No abstract text is available yet for this article.
October 3, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37755317/intra-familial-variability-of-oculoleptomeningeal-amyloidosis-due-to-the-attr-i107m-c-381t%C3%A2-%C3%A2-g-mutation-diagnostic-challenges-of-a-rare-phenotype
#28
JOURNAL ARTICLE
Monica Alcantara, Vera Bril
No abstract text is available yet for this article.
September 27, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37668548/patient-reported-outcome-measures-for-transthyretin-cardiac-amyloidosis-the-italy-study
#29
JOURNAL ARTICLE
Alberto Aimo, Lucio Teresi, Vincenzo Castiglione, Anna Lisa Picerni, Martina Niccolai, Silvia Severino, Assunta Agazio, Anna Carnevale Baraglia, Laura Obici, Giovanni Palladini, Lucia Ponti, Alessia Argirò, Francesco Cappelli, Federico Perfetto, Matteo Serenelli, Giancarlo Trimarchi, Roberto Licordari, Gianluca Di Bella, Olena Chubuchna, Filippo Quattrone, Sabina Nuti, Sabina De Rosis, Claudio Passino, Claudio Rapezzi, Giampaolo Merlini, Michele Emdin, Giuseppe Vergaro
BACKGROUND: Transthyretin cardiac amyloidosis (ATTR-CA) has a deep impact on the quality of life (QoL), yet no specific patient-reported outcome measures (PROMs) for ATTR-CA exist. METHODS: The ITALY study involved 5 Italian referral centres (Pisa, Pavia, Ferrara, Florence, Messina) enrolling consecutive outpatients with ATTR-CA. RESULTS: Two 30-item questionnaires were created for wild-type (wt) and variant (v) ATTR-CA. Scores ranged from 100 (best condition) to 0 (worst condition)...
September 5, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37216268/a-phase-1b-dose-escalation-study-of-carfilzomib-in-combination-with-thalidomide-and-dexamethasone-in-patients-with-relapsed-refractory-systemic-immunoglobulin-light-chain-amyloidosis
#30
JOURNAL ARTICLE
Sriram Ravichandran, Andrew Hall, Matthew Jenner, Mamta Garg, Bhuvan Kishore, Helen Lachmann, Julian Gillmore, Alexandra Pitchford, Jamie B Oughton, Shameem Mahmood, Sajitha Sachchithantham, Philip Hawkins, Sarah Brown, Ashutosh Wechalekar
INTRODUCTION: Proteasome inhibitors are the backbone of AL amyloidosis treatment - bortezomib being most widely used. Carfilzomib is a proteasome inhibitor licenced to treat multiple myeloma; autonomic and peripheral neuropathy are uncommon toxicities with carfilzomib. There is limited data on the use of carfilzomib in AL amyloidosis. Here, we report the results of a phase Ib dose-escalation study of Carfilzomib-Thalidomide-Dexamethasone (KTD) in relapsed/refractory AL amyloidosis. RESULTS: The trial registered 11 patients from 6 UK centres from September 2017 to January 2019; 10 patients received at least one dose of trial treatment...
September 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/36988111/echocardiographic-findings-in-subjects-with-an-amyloidogenic-apolipoprotein-a1-pathogenic-variant
#31
JOURNAL ARTICLE
Daniela Tomasoni, Alberto Aimo, Marianna Adamo, Matilde Nardi, Carlo Mario Lombardi, Valentina Regazzoni, Maria Grazia De Angelis, Iacopo Fabiani, Giampaolo Merlini, Roberta Mussinelli, Laura Obici, Giorgia Panichella, Giuseppe Vergaro, Claudio Passino, Francesco Scolari, Stefano Perlini, Michele Emdin, Marco Metra
BACKGROUND: Very small case series of patients with apolipoprotein A1 (ApoA1) amyloidosis are available. METHODS: We described the clinical and echocardiographic characteristics of individuals with the pathogenic APOA1 variant Leu75Pro (p. Leu99Pro), referred for cardiac screening. RESULTS: We enrolled 189 subjects, 54% men, median age 55 years (interquartile range 42-67), 39% with concomitant renal disease and 31% with liver disease...
September 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/36795029/comparison-between-tafamidis-and-liver-transplantation-as-first-line-therapy-for-hereditary-transthyretin-amyloidosis
#32
JOURNAL ARTICLE
Pierre Socie, Anouar Benmalek, Cécile Cauquil, Eve Piekarski, Ilias Kounis, Ludivine Eliahou, Antoine Rousseau, François Rouzet, Andoni Echaniz-Laguna, Didier Samuel, David Adams, Michel S Slama, Vincent Algalarrondo
BACKGROUND: By stabilizing transthyretin, tafamidis delays progression of amyloidosis due to transthyretin variant (ATTRv) and replaced liver transplantation (LT) as the first-line therapy. No study compared these two therapeutic strategies. METHODS: In a monocentric retrospective cohort analysis, patients with ATTRv amyloidosis treated with either tafamidis or LT were compared using a propensity score and a competing risk analysis for three endpoints: all-cause mortality, cardiac worsening (heart failure or cardiovascular death) and neurological worsening (worsening in PolyNeuropathy Disability score)...
September 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/36779691/impact-of-cytogenetic-abnormalities-on-treatment-outcomes-in-patients-with-amyloid-light-chain-amyloidosis-subanalyses-from-the-andromeda-study
#33
RANDOMIZED CONTROLLED TRIAL
Shaji Kumar, Angela Dispenzieri, Divaya Bhutani, Morie Gertz, Ashutosh Wechalekar, Giovanni Palladini, Raymond Comenzo, Rafael Fonseca, Arnaud Jaccard, Efstathios Kastritis, Stefan Schönland, Charles la Porte, Huiling Pei, NamPhuong Tran, Giampaolo Merlini
BACKGROUND: Cytogenetic abnormalities are common in patients with amyloid light-chain (AL) amyloidosis; some are associated with poorer outcomes. This post hoc analysis of ANDROMEDA evaluated the impact of certain cytogenetic abnormalities on outcomes in this patient population. METHODS: Patients with newly diagnosed AL amyloidosis were randomised 1:1 to daratumumab, bortezomib, cyclophosphamide, and dexamethasone (D-VCd) or VCd. Outcomes were evaluated in the intent-to-treat (ITT) population and in patients with t(11;14), amp1q21, del13q14, and del17p13...
September 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37599395/reduction-in-99m-tc-dpd-myocardial-uptake-with-therapy-of-attr-cardiomyopathy
#34
JOURNAL ARTICLE
René Rettl, Raffaella Calabretta, Franz Duca, Christina Binder, Christina Kronberger, Robin Willixhofer, Michael Poledniczek, Carolina Donà, Christian Nitsche, Dietrich Beitzke, Christian Loewe, Michaela Auer-Grumbach, Diana Bonderman, Stefan Kastl, Christian Hengstenberg, Roza Badr Eslam, Johannes Kastner, Jutta Bergler-Klein, Marcus Hacker, Andreas Kammerlander
Aims: Novel ribonucleic acid interference (RNAi) therapeutics such as patisiran and inotersen have been shown to benefit neurologic disease course and quality of life in patients with hereditary transthyretin amyloidosis (ATTRv). We aimed to determine the impact of RNAi therapeutics on myocardial amyloid load using quantitative single photon emission computed tomography/computed tomography (SPECT/CT) imaging in patients with ATTRv-related cardiomyopathy (ATTRv-CM). We furthermore compared them with wild-type ATTR-CM (ATTRwt-CM) patients treated with tafamidis...
August 20, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37563759/correction
#35
(no author information available yet)
No abstract text is available yet for this article.
August 10, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37530216/correlation-of-99mtc-dpd-bone-scintigraphy-with-histological-amyloid-load-in-patients-with-attr-cardiac-amyloidosis
#36
JOURNAL ARTICLE
Maria Ungericht, Valeria Groaz, Moritz Messner, Thomas Schuetz, Luca Brunelli, Marc-Michael Zaruba, Daniela Lener, Eva Stocker, Axel Bauer, Alexander Stephan Kroiss, Agnes Mayr, Christoph Röcken, Gerhard Poelzl
BACKGROUND: The significance of measuring 99mTc-labelled-3,3-diphosphono-1,2-propanodicarboxylic acid (99mTc-DPD) in transthyretin (ATTR) cardiac amyloidosis has not been adequately studied. This single-centre observational study evaluated the correlation between 99mTc-DPD scintigraphy and histological amyloid load in endomyocardial biopsy (EMB). METHODS: Twenty-eight patients with biopsy-proven ATTR amyloidosis and concomitantly available 99mTc-DPD scintigraphy were included...
August 2, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37493395/diagnostic-and-prognostic-contribution-of-dpd-scintigraphy-in-transthyretin-v30m-cardiac-amyloidosis
#37
JOURNAL ARTICLE
Maria C Azevedo Coutinho, Nuno Cortez-Dias, Guilhermina Cantinho, Susana Gonçalves, Nelson Cunha, Tiago Rodrigues, Laura Santos, Isabel Conceição, João Agostinho, Fausto J Pinto
BACKGROUND: Early diagnosis and prognostic stratification of cardiac transthyretin amyloidosis are crucial. Although 99m Tc 3,3-diphosphono-1,2-propanedicarboxylic acid (DPD) scintigraphy is the preferred method for the non-invasive diagnosis, its accuracy appears to be limited in transthyretin amyloidosis protein (ATTR) V30M mutation. Furthermore, its prognostic value in this mutation is unknown. This study investigated the diagnostic value of DPD scintigraphy to detect ATTR cardiomyopathy in V30M mutation and explored its prognostic value regarding mortality...
July 26, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37486102/technetium-99m-pyrophosphate-imaging-based-computed-tomography-guided-core-needle-biopsy-of-internal-oblique-muscle-in-wild-type-transthyretin-cardiac-amyloidosis
#38
JOURNAL ARTICLE
Koji Takahashi, Yoshiyasu Hiratsuka, Takaaki Iwamura, Daisuke Sasaki, Nobuhisa Yamamura, Sohei Kitazawa, Mitsuharu Ueda, Hiroe Morioka, Takafumi Okura, Daijiro Enomoto, Shigeki Uemura, Taizo Kono, Tomoki Sakaue, Shuntaro Ikeda
BACKGROUND: Technetium-99m-pyrophosphate (99m Tc-PYP) uptake in the internal oblique muscle (IOM), which is often observed in patients with wild-type transthyretin cardiac amyloidosis (ATTR-CA), indicates amyloid transthyretin (ATTR) deposition. OBJECTIVE: This study aimed to assess the safety and efficacy of 99m Tc-PYP imaging-based computed tomography (CT)-guided core-needle biopsy of the IOM as a new extracardiac screening biopsy for confirming the presence of ATTR deposits...
July 24, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37469249/treatment-response-and-neurofilament-light-chain-levels-with-long-term-patisiran-in-hereditary-transthyretin-mediated-amyloidosis-with-polyneuropathy-24-month-results-of-an-open-label-extension-study
#39
JOURNAL ARTICLE
Simina Ticau, Emre Aldinc, Michael Polydefkis, David Adams, Teresa Coelho, Mitsuharu Ueda, Cecilia Hale, John Vest, Paul Nioi
BACKGROUND: Longitudinal changes in neurofilament light chain (NfL) levels were evaluated alongside prespecified clinical assessments 24 months into the patisiran Global open-label extension (OLE) study in patients with ATTRv amyloidosis with polyneuropathy. METHODS: All patients enrolled in the Global OLE, from phase III APOLLO and phase II OLE parent studies, received patisiran. Assessments included measures of polyneuropathy (modified Neuropathy Impairment Score+7 (mNIS+7)), quality of life (QOL; Norfolk QOL-Diabetic Neuropathy questionnaire (Norfolk QOL-DN)), and plasma NfL...
July 20, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
https://read.qxmd.com/read/37459334/patients-with-transthyretin-amyloidosis-enrolled-in-thaos-between-2018-and-2021-continue-to-experience-substantial-diagnostic-delay
#40
JOURNAL ARTICLE
Teresa Coelho, Angela Dispenzieri, Martha Grogan, Isabel Conceição, Márcia Waddington-Cruz, Arnt V Kristen, Jonas Wixner, Igor Diemberger, Juan Gonzalez-Moreno, Mathew S Maurer, Violaine Planté-Bordeneuve, Pablo Garcia-Pavia, Ivailo Tournev, Jose Gonzalez-Costello, Eve Cariou, Alejandra González-Duarte, Oliver Glass, Doug Chapman, Leslie Amass
No abstract text is available yet for this article.
July 17, 2023: Amyloid: the International Journal of Experimental and Clinical Investigation
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