Amyloid-Journal of Protein Folding Disorders最新文献

Clinical impact of beta-blocker withdrawal in transthyretin amyloid cardiomyopathy. 经甲状腺素淀粉样变性心肌病患者停用β-受体阻滞剂的临床影响。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-11-20 DOI: 10.1080/13506129.2024.2431082

Eva Cabrera-Romero, Leidy Alexandra Serrao-Faria, Nerea Mora-Ayestarán, Belén Peiró-Aventín, Ana Espinoza, Daniel de Castro, Marta Cobo-Marcos, Fernando Domínguez, Esther González-López, Pablo Garcia-Pavia

引用次数: 0

Involvement of bile acid in diarrhoea and therapeutic effect of colestimide in hereditary ATTR amyloidosis. 胆汁酸对遗传性 ATTR 淀粉样变性病腹泻的影响及可乐定的治疗效果。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-11-20 DOI: 10.1080/13506129.2024.2430554

Yusuke Mochizuki, Nagaaki Katoh, Akira Matsushima, Masahide Yazaki, Naoko Kuwabara, Saori Nakagawa, Yoshiki Sekijima

{"title":"Involvement of bile acid in diarrhoea and therapeutic effect of colestimide in hereditary ATTR amyloidosis.","authors":"Yusuke Mochizuki, Nagaaki Katoh, Akira Matsushima, Masahide Yazaki, Naoko Kuwabara, Saori Nakagawa, Yoshiki Sekijima","doi":"10.1080/13506129.2024.2430554","DOIUrl":"https://doi.org/10.1080/13506129.2024.2430554","url":null,"abstract":"Background: Diarrhoea is one of the most serious complications in hereditary ATTR (ATTRv) amyloidosis. However, its precise pathomechanism remains unknown. The present study investigated the involvement of bile acid in diarrhoea along with the therapeutic effect of colestimide, a bile acid sequestrant, in ATTRv amyloidosis.Methods: We prospectively enrolled 19 ATTRv amyloidosis patients (9 with refractory diarrhoea and 10 without diarrhoea) and 20 healthy individuals for measurements of serum 7a-hydroxy-4-cholesten-3-one (C4) levels. The patients with diarrhoea were then treated with oral colestimide (1.5 g twice daily) for 28 days. The frequency of diarrhoea and C4 level were evaluated before and after colestimide treatment.Results: Mean serum C4 level was significantly higher in ATTRv patients with diarrhoea (62.3 ng/mL) than in ATTRv patients without diarrhoea (24.0 ng/mL, p = 0.03). Colestimide treatment significantly decreased mean diarrhoea frequency (pre-treatment period: 9.1 times/week, colestimide treatment period, 6.6 times/week, p = 0.04) and increased mean C4 level (before treatment: 66.2 ng/mL, after treatment: 187.1 ng/mL, p = 0.02).Conclusions: Bile acid status was significantly associated with diarrhoea in ATTRv amyloidosis. Colestimide and other bile acid sequestrants may reduce diarrhoea frequency in afflicted patients.","PeriodicalId":50964,"journal":{"name":"Amyloid-Journal of Protein Folding Disorders","volume":" ","pages":"1-7"},"PeriodicalIF":5.2,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142677439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Effects of eplontersen on symptoms of autonomic neuropathy in hereditary transthyretin-mediated amyloidosis: secondary analysis from the NEURO-TTRansform trial. 依普仑特生对遗传性转甲状腺素介导的淀粉样变性自主神经病变症状的影响：NEURO-TTRansform 试验的二次分析。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-11-17 DOI: 10.1080/13506129.2024.2427290

Jonas Wixner, John L Berk, David Adams, Michael Polydefkis, Isabel Conceição, Shahram Attarian, Julian D Gillmore, P James B Dyck, Folke Folkvaljon, Wunan Zhou, Jersey Chen, Nicholas J Viney, T Jesse Kwoh, Teresa Coelho, Márcia Waddington-Cruz

{"title":"Effects of eplontersen on symptoms of autonomic neuropathy in hereditary transthyretin-mediated amyloidosis: secondary analysis from the NEURO-TTRansform trial.","authors":"Jonas Wixner, John L Berk, David Adams, Michael Polydefkis, Isabel Conceição, Shahram Attarian, Julian D Gillmore, P James B Dyck, Folke Folkvaljon, Wunan Zhou, Jersey Chen, Nicholas J Viney, T Jesse Kwoh, Teresa Coelho, Márcia Waddington-Cruz","doi":"10.1080/13506129.2024.2427290","DOIUrl":"https://doi.org/10.1080/13506129.2024.2427290","url":null,"abstract":"Background: The NEURO-TTRansform trial showed that after 66 weeks of treatment, eplontersen significantly reduced neuropathic impairment and improved quality of life (QoL) in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN). In this secondary analysis from NEURO-TTRansform, autonomic impairment, and the impact of eplontersen on autonomic impairment progression was evaluated through 85 weeks in patients randomised to eplontersen (n = 144) versus external placebo (n = 60; through Week 66 from the NEURO-TTR trial).Methods: Change from baseline in modified Neuropathy Impairment Score +7 (mNIS+7) composite score, Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score, and the Neuropathy Symptoms and Change (NSC) total score was evaluated. Exploratory assessments were change in autonomic components of these instruments, Composite Autonomic Symptom Score-31 (COMPASS-31) total score, and nutritional status (modified body mass index [mBMI]).Results: Patients reported profound autonomic dysfunction at baseline. Improvements with eplontersen versus placebo were observed up to Week 66 in autonomic components of mNIS+7, Norfolk QoL-DN, NSC, and mBMI; eplontersen results were sustained up to Week 85, including improvements in COMPASS-31 (Week 81).Conclusions: Eplontersen demonstrated benefit across multiple measures of autonomic impairment known to progress rapidly and negatively impact QoL without treatment, without deterioration in nutritional status.","PeriodicalId":50964,"journal":{"name":"Amyloid-Journal of Protein Folding Disorders","volume":" ","pages":"1-10"},"PeriodicalIF":5.2,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142649726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

High frequency of occult transthyretin and apolipoprotein AI-type amyloid in aortic valves removed by valve replacement for aortic stenosis. 在因主动脉瓣狭窄而进行瓣膜置换术切除的主动脉瓣中，隐匿性转甲状腺素和脂蛋白AI型淀粉样蛋白的出现频率很高。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-11-11 DOI: 10.1080/13506129.2024.2426508

Kohei Honda, Masayoshi Tasaki, Tetsuhiro Yamano, Mitsuharu Ueda, Hironobu Naiki, Noriyuki Tanaka, Yukiko Morinaga, Aya Miyagawa-Hayashino

{"title":"High frequency of occult transthyretin and apolipoprotein AI-type amyloid in aortic valves removed by valve replacement for aortic stenosis.","authors":"Kohei Honda, Masayoshi Tasaki, Tetsuhiro Yamano, Mitsuharu Ueda, Hironobu Naiki, Noriyuki Tanaka, Yukiko Morinaga, Aya Miyagawa-Hayashino","doi":"10.1080/13506129.2024.2426508","DOIUrl":"10.1080/13506129.2024.2426508","url":null,"abstract":"Background: A high incidence of valvular involvement of amyloid in the setting of aortic stenosis (AS) has been reported. Amyloid derived from ApoAI (AApoAI) can form local amyloid deposits in the aortic valve. Although a high prevalence of concomitant severe AS and cardiac transthyretin-type amyloidosis (ATTR) has been reported, the prevalence of valvular involvement by ATTR and AApoAI is unclear.Methods: Using immunostaining and mass spectrometry, we analysed amyloid proteins in 97 aortic valves removed for valve replacement due to AS at Kyoto Prefectural University of Medicine between 2014 and 2021. Clinical information was also reviewed.Results: Amyloid deposits were found in 44 cases (45%), of which 30 cases (68%) involved ATTR and 33 cases (75%) AApoAI. Statistical analysis showed significantly lower age and E/e' among amyloid-positive cases compared with amyloid-negative cases and significantly lower brain natriuretic peptide, higher fractional shortening, and higher left ventricular ejection fraction among ATTR-positive cases compared with ATTR-negative cases. Seven recent patients underwent bone scintigraphy and ATTR cardiomyopathy was observed in only one case.Conclusions: AS symptoms can manifest earlier in patients with amyloid or ATTR deposition in the aortic valve than in patients without such deposition, even though left ventricular function is preserved.","PeriodicalId":50964,"journal":{"name":"Amyloid-Journal of Protein Folding Disorders","volume":" ","pages":"1-7"},"PeriodicalIF":5.2,"publicationDate":"2024-11-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142631826","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Cardiac amyloidosis: the possibilities and challenges in the Ghanaian setting. 心脏淀粉样变性：加纳的可能性与挑战。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-11-04 DOI: 10.1080/13506129.2024.2422458

Andrew Sefenu Dzebu, Magalys López Cuba

引用次数: 0

Amyloidosis can be diagnosed by cardiologists in Africa: now they should be given the medicine to treat it. 非洲的心脏病专家可以诊断出淀粉样变性：现在应该给他们提供治疗药物。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-11-04 DOI: 10.1080/13506129.2024.2422474

Lucio Luzzatto

引用次数: 0

Anomalous colours, not interference colours or 'apple-green birefringence', in Congo red-stained amyloid. 刚果红染色淀粉样蛋白中的异常颜色，而非干涉色或 "苹果绿双折射"。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-10-30 DOI: 10.1080/13506129.2024.2421427

Alexander J Howie

引用次数: 0

PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study. PRX004治疗变异型淀粉样转甲状腺素（ATTRv）淀粉样变性病：1期开放标签剂量递增研究结果。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-10-29 DOI: 10.1080/13506129.2024.2420809

Ole B Suhr, Martha Grogan, Ana Martins da Silva, Chafic Karam, Pablo Garcia-Pavia, Brian Drachman, Wagner Zago, Radhika Tripuraneni, Gene G Kinney

{"title":"PRX004 in variant amyloid transthyretin (ATTRv) amyloidosis: results of a phase 1, open-label, dose-escalation study.","authors":"Ole B Suhr, Martha Grogan, Ana Martins da Silva, Chafic Karam, Pablo Garcia-Pavia, Brian Drachman, Wagner Zago, Radhika Tripuraneni, Gene G Kinney","doi":"10.1080/13506129.2024.2420809","DOIUrl":"https://doi.org/10.1080/13506129.2024.2420809","url":null,"abstract":"Background: The investigational monoclonal antibody PRX004 is designed to specifically target and deplete TTR amyloid. Here, we report on the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical activity of PRX004 in patients with ATTRv amyloidosis.Methods: This global, multicentre, phase 1 trial comprised a 3 + 3 dose-escalation phase and a long-term extension (LTE) phase (NCT03336580). In the dose-escalation phase, patients received PRX004 (0.1, 0.3, 1, 3, 10 or 30 mg/kg), administered intravenously every 28 days for 3 months. In the LTE, eligible patients could receive up to 15 additional doses. Patients who received doses of ≥3 mg/kg for ≥9 months were assessed for Global Longitudinal Strain (GLS) and Neuropathy Impairment Score (NIS). The primary objective was to determine the maximum tolerated dose (MTD) of PRX004.Results: Overall, 21 patients with ATTRv amyloidosis completed the dose-escalation phase; 17 subsequently enrolled in the LTE. The MTD was not reached. PRX004 was well tolerated at all doses, with dose-proportional exposure. GLS and NIS were improved or maintained over 9 months (n = 7).Conclusions: PRX004 was well tolerated in patients with ATTRv amyloidosis and demonstrated potential clinical activity. A phase 2 randomised controlled trial in ATTR cardiomyopathy is ongoing (NCT05442047).","PeriodicalId":50964,"journal":{"name":"Amyloid-Journal of Protein Folding Disorders","volume":" ","pages":"1-8"},"PeriodicalIF":5.2,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142548803","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

A case of the iatrogenic transmission of vascular Aß40 amyloid. 一例血管 Aß40 淀粉样蛋白的先天性传播。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-10-29 DOI: 10.1080/13506129.2024.2419857

Takeshi Kawarabayashi, Takumi Nakamura, Shin Takatama, Naoko Miyamoto, Tomoyuki Iwai, Isao Naito, Takashi Sugawara, Kunihiko Ishizawa, Kentaro Hashimoto, Masakuni Amari, Takeshi Ikeuchi, Hiroo Kasahara, Yoshio Ikeda, Masamitsu Takatama, Mikio Shoji

引用次数: 0

Identification of epidermal growth factor-containing fibulin-like extracellular matrix protein 1-derived amyloid deposition in a rhesus macaque. 在猕猴体内发现含表皮生长因子的纤维蛋白样细胞外基质蛋白 1 淀粉样沉积。

IF 5.2 2区医学

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-10-29 DOI: 10.1080/13506129.2024.2421434

Matthew F Starost, Tomoaki Murakami, Kelli L Vaughan, Christopher King, Anna Harima, Julie A Mattison

引用次数: 0