Arquivos de neuro-psiquiatria最新文献

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Dual versus single antiplatelet therapy in patients with nonminor ischemic stroke: a meta-analysis.
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-02-01 Epub Date: 2025-02-24 DOI: 10.1055/s-0045-1802551
Izabela Orlandi Môro, Gabriel Marinheiro, Marianna Leite, Gabriel de Almeida Monteiro, Agostinho C Pinheiro, João Paulo Mota Telles
{"title":"Dual versus single antiplatelet therapy in patients with nonminor ischemic stroke: a meta-analysis.","authors":"Izabela Orlandi Môro, Gabriel Marinheiro, Marianna Leite, Gabriel de Almeida Monteiro, Agostinho C Pinheiro, João Paulo Mota Telles","doi":"10.1055/s-0045-1802551","DOIUrl":"https://doi.org/10.1055/s-0045-1802551","url":null,"abstract":"<p><strong>Background: </strong> Patients with ischemic stroke present a higher risk of stroke recurrence, neurological deterioration, and death. The benefit of dual antiplatelet therapy (DAPT) over single antiplatelet therapy (SAPT) among patients with minor ischemic stroke is well established; however, robust evidence is lacking for those with nonminor stroke.</p><p><strong>Objective: </strong> To describe the benefits and risks of DAPT versus SAPT in patients with nonminor ischemic stroke.</p><p><strong>Methods: </strong> We searched the PubMed, Embase, and Cochrane Library databases for articles published from inception to April 2024. Data were collected from randomized clinical trials and observational studies comparing DAPT to SAPT following nonminor ischemic stroke, defined by a score ≥ 4 on the National Institutes of Health Stroke Scale (NIHSS).</p><p><strong>Results: </strong> In total, 6 studies were included, comprising 12,480 patients. The NIHSS score at baseline from the selected studies ranged from 4 to 15. There was no significant difference between DAPT and SAPT for recurrent stroke (risk ratio [RR] = 0.91; 95% confidence interval [95%CI] = 0.82-1.01; <i>p</i> = 0.09; I<sup>2</sup> = 0%), ischemic stroke (RR = 0.89; 95%CI = 0.80-1.00; <i>p</i> = 0.05; I<sup>2</sup> = 0%) or hemorrhagic stroke (RR = 1.23; 95%CI = 0.41-3.99; <i>p</i> = 0.66; I<sup>2</sup> = 27%). Major bleeding was not significantly increased in the DAPT group compared with the SAPT group (RR = 0.87; 95%CI = 0.29-2.66; <i>p</i> = 0.81; I<sup>2</sup> = 44%). The overall analysis did not show a significant difference in all-cause mortality (RR = 0.72; 95%CI = 0.50-1.02; <i>p</i> = 0.07; I<sup>2</sup> = 0%).</p><p><strong>Conclusion: </strong> There was no difference between DAPT and SAPT regarding recurrent stroke, ischemic stroke, hemorrhagic stroke, major bleeding, or overall mortality.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 2","pages":"1-10"},"PeriodicalIF":1.0,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490487","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Sixty years of the first studies by Horácio Martins Canelas on Wilson's disease.
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-02-01 Epub Date: 2025-02-24 DOI: 10.1055/s-0045-1802553
Egberto Reis Barbosa, Jacy Bezerra Parmera, Rubens Gisbert Cury, Eduardo Luiz Rachid Cançado, Patrícia Áurea Andreucci Martins Bonilha, Hélio Afonso Ghizoni Teive
{"title":"Sixty years of the first studies by Horácio Martins Canelas on Wilson's disease.","authors":"Egberto Reis Barbosa, Jacy Bezerra Parmera, Rubens Gisbert Cury, Eduardo Luiz Rachid Cançado, Patrícia Áurea Andreucci Martins Bonilha, Hélio Afonso Ghizoni Teive","doi":"10.1055/s-0045-1802553","DOIUrl":"https://doi.org/10.1055/s-0045-1802553","url":null,"abstract":"<p><p>Research into Wilson's disease (WD) in Brazil had the effective participation of Professor Horácio Martins Canelas, from the Neurologic Clinic of the Teaching Hospital of the School of Medicine of Universidade de São Paulo (USP). His exponential contributions to the study of WD placed Brazil on the international stage, making USP's Neurologic Clinic one of the world's leading research centers in the area of neurodegenerative diseases with metal accumulation.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 2","pages":"1-3"},"PeriodicalIF":1.0,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490490","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Comment on "Stroke awareness in a Brazilian Northeastern capital city and the burden of the COVID-19 pandemic".
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-02-01 Epub Date: 2025-02-04 DOI: 10.1055/s-0045-1802310
Diogo Gonçalves Dos Santos Martins, Thiago Gonçalves Dos Santos Martins
{"title":"Comment on \"Stroke awareness in a Brazilian Northeastern capital city and the burden of the COVID-19 pandemic\".","authors":"Diogo Gonçalves Dos Santos Martins, Thiago Gonçalves Dos Santos Martins","doi":"10.1055/s-0045-1802310","DOIUrl":"https://doi.org/10.1055/s-0045-1802310","url":null,"abstract":"","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 2","pages":"1-2"},"PeriodicalIF":1.0,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188149","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Validity and reliability of the Turkish version of the Innsbruck RBD-9 diagnostic inventory (IRBD-9-TR).
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-01-28 DOI: 10.1055/s-0044-1800816
Kezban Aslan-Kara, Ayşın Kısabay Ak, Ayşegül Şeyma Sarıtaş, Hikmet Yılmaz, Kübra Mehel Metin, Burcu Gökçe Çokal, Kadriye Ağan, Murat Aksu, Utku Oğan Akyıldız, Aylin Bican Demir, Betül Çevik, Ahmet Yusuf Ertürk, Derya Karadeniz, İbrahim Öztura, Gülin Sünter, Selma Tekin, İrsel Tezer, Deniz Tuncel Berktaş, Nazlı Totik, Gülçin Benbir Şenel
{"title":"Validity and reliability of the Turkish version of the Innsbruck RBD-9 diagnostic inventory (IRBD-9-TR).","authors":"Kezban Aslan-Kara, Ayşın Kısabay Ak, Ayşegül Şeyma Sarıtaş, Hikmet Yılmaz, Kübra Mehel Metin, Burcu Gökçe Çokal, Kadriye Ağan, Murat Aksu, Utku Oğan Akyıldız, Aylin Bican Demir, Betül Çevik, Ahmet Yusuf Ertürk, Derya Karadeniz, İbrahim Öztura, Gülin Sünter, Selma Tekin, İrsel Tezer, Deniz Tuncel Berktaş, Nazlı Totik, Gülçin Benbir Şenel","doi":"10.1055/s-0044-1800816","DOIUrl":"https://doi.org/10.1055/s-0044-1800816","url":null,"abstract":"<p><strong>Background: </strong> Isolated rapid eye movement (REM) sleep behavior disorder (iRBD) is characterized by loss of the normal atonia of REM sleep accompanied by repetitive motor and behavior phenomena of dream content.</p><p><strong>Objective: </strong> To evaluate the reliability and validity of the Turkish version of the original form of the Innsbruck Rapid Eye Movement Sleep Behavior Disorder Diagnostic Inventory (IRBD-9) scale (IRBD-9-TR) and ensure that this screening test can be easily used in the Turkish language.</p><p><strong>Methods: </strong> The present is a multicenter and prospective study involving 184 patients: 51 with iRBD and 133 healthy controls. The iRBD patients were not diagnosed before submitted to video polysomnography (vPSG) and filling out the IRBD-9-TR.</p><p><strong>Results: </strong> The optimal cut-off value for the IRBD-9-TR symptom score was of 0.28, with a sensitivity of 0.941 and a specificity of 0.947, and 94.4% of the patients were correctly diagnosed. The rotated factor loadings for the diagnostic accuracy of each individual question showed that the short version of the IRBD-9-TR (questions 1, 2, 3, 6, and 8) presented higher specificity and excellent discrimination of iRBD patients from healthy controls. The Cronbach's α coefficient for the symptom section of the IRBD-9-TR was of 0.857, and the Kappa coefficient, of 0.885.</p><p><strong>Conclusion: </strong> The short version of the IRBD-9-TR presents good validity and reliability to be used as a screening test to assess iRBD patients. It is convenient and potentially useful in both outpatient clinical and epidemiologic research settings.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-7"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143057875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Thanks to the Editorial Board of Arquivos de Neuro-Psiquiatria (2024).
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-01-31 DOI: 10.1055/s-0045-1802322
{"title":"Thanks to the Editorial Board of Arquivos de Neuro-Psiquiatria (2024).","authors":"","doi":"10.1055/s-0045-1802322","DOIUrl":"https://doi.org/10.1055/s-0045-1802322","url":null,"abstract":"","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-5"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143073566","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Jitter and muscle fiber conduction velocity in long COVID fatigue.
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-02-24 DOI: 10.1055/s-0045-1802961
João Aris Kouyoumdjian, Leticia Akemi Rama Yamamoto, Carla Renata Graca
{"title":"Jitter and muscle fiber conduction velocity in long COVID fatigue.","authors":"João Aris Kouyoumdjian, Leticia Akemi Rama Yamamoto, Carla Renata Graca","doi":"10.1055/s-0045-1802961","DOIUrl":"https://doi.org/10.1055/s-0045-1802961","url":null,"abstract":"<p><strong>Background: </strong> Long coronavirus disease (long COVID, LC) is defined as the continuation or development of new symptoms 3 months after the acute stage of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection. In LC, the rate of fatigue/postexertional malaise (F-PEM) has been described to be as high as 70%, regardless of age or severity of the acute symptoms.</p><p><strong>Objective: </strong> To evaluate the neuromuscular junction (NMJ) function and the isolated muscle fiber conduction velocity (MFCV) in situ in LC cases and controls.</p><p><strong>Methods: </strong> We studied 37 subjects without SARS-CoV-2 (controls) and 32 cases of SARS-CoV-2 infection, half with LC symptoms (LC-yes) and half without them (LC-no). Single-fiber electromyography (jitter measured with a concentric electrode), MFCV, the fast-to-slow MFCV ratio (F/S ratio), and the motor unit potentials (MUPs) were taken in the tibialis anterior muscle.</p><p><strong>Results: </strong> At least 1 jitter parameter was abnormal in 1/37 controls, in 1/16 LC-no patients, and in 2/16 LC-yes patients, without significant differences among them. None of the subjects with abnormal jitter presented fluctuation symptoms or positive acetylcholine-receptor antibody. The MFCV and F/S ratios did not show abnormalities in any of the participants. The MUPs did not show myopathic or neurogenic abnormality in needle electromyography. The most frequent symptom in LC was F-PEM, which occurred in all LC-yes patients and was significantly different from the other groups.</p><p><strong>Conclusion: </strong> Fatigue/postexertional malaise was found in all cases of LC, and the electrophysiological findings did not indicate the muscle fiber or the NMJ as a relevant factor in this condition.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-8"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490485","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Diagnosing preclinical and clinical Alzheimer's disease with visual atrophy scales in the clinical practice.
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-02-28 DOI: 10.1055/s-0045-1802960
Karen Luiza Ramos Socher, Douglas Mendes Nunes, Deborah Cristina P Lopes, Artur Martins Novaes Coutinho, Daniele de Paula Faria, Paula Squarzoni, Geraldo Busatto Filho, Carlos Alberto Buchpiguel, Ricardo Nitrini, Sonia Maria Dozzi Brucki
{"title":"Diagnosing preclinical and clinical Alzheimer's disease with visual atrophy scales in the clinical practice.","authors":"Karen Luiza Ramos Socher, Douglas Mendes Nunes, Deborah Cristina P Lopes, Artur Martins Novaes Coutinho, Daniele de Paula Faria, Paula Squarzoni, Geraldo Busatto Filho, Carlos Alberto Buchpiguel, Ricardo Nitrini, Sonia Maria Dozzi Brucki","doi":"10.1055/s-0045-1802960","DOIUrl":"https://doi.org/10.1055/s-0045-1802960","url":null,"abstract":"<p><strong>Background: </strong> Visual atrophy scales from the medial temporal region are auxiliary biomarkers of neurodegeneration in Alzheimer's disease (AD). Therefore, they may correlate with progression from cognitively unimpaired (CU) status to mild cognitive impairment (MCI) and AD, and they become a valuable tool for diagnostic accuracy.</p><p><strong>Objective: </strong> To compare the medial temporal lobe atrophy (MTA) and entorhinal cortex atrophy (ERICA) scores measured through magnetic resonance image (MRI) scans as a useful method for probable AD diagnosis regarding clinical diagnosis and amyloid positron-emission tomography (PET).</p><p><strong>Methods: </strong> Two neurologists blinded to the diagnoses classified 113 older adults (age > 65 years) through the MTA and ERICA scores. We investigated the correlations involving these scores and sociodemographic data, amyloid brain cortical burden measured through PET imaging with (11)C-labeled Pittsburgh Compound-B (11C-PIB PET), and clinical cognitive status, in individuals diagnosed as CU (CU; <i>N</i> = 30), presenting mild cognitive impairment (MCI, <i>N</i> = 52), and AD patients (<i>N</i> = 31).</p><p><strong>Results: </strong> The inter-rater reliability of the atrophy scales was excellent (0.8-1) according to the Cohen analysis. The CU group presented lower MTA scores (median value: 0) than ERICA (median value: 1) scores in both hemispheres. The 11C-PIB-PET was positive in 45% of the sample. In the MCI and AD groups, the ERICA score presented greater sensitivity, and the MTA score presented greater specificity. The accuracy of the clinical diagnosis was sufficient and no more than 70% for both scores in AD.</p><p><strong>Conclusion: </strong> In the present study, we found moderate sensitivity for the ERICA score, which could be a better screening tool than the MTA score for the diagnosis of AD or MCI. However, none of the scores were useful imaging biomarkers in preclinical AD.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-7"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143530998","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Erratum.
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-02-10 DOI: 10.1055/s-0045-1802550
{"title":"Erratum.","authors":"","doi":"10.1055/s-0045-1802550","DOIUrl":"https://doi.org/10.1055/s-0045-1802550","url":null,"abstract":"","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-5"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143389899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Guidelines for Parkinson's disease management part II: consensus from the movement disorders scientific department of the Brazilian Academy of Neurology - non-motor symptoms.
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-02-24 DOI: 10.1055/s-0045-1802962
Débora Palma Maia, Rubens Gisbert Cury, Pedro Renato P Brandão, Francisco E C Cardoso, Ana Paula Bertholo, André Carvalho Felicio, Arlete Hilbig, Bruno Lopes Santos Lobato, Eline Rozária F Barbosa, Elizabeth Maria A B Quagliato, Gustavo H C Sousa, Jacy Bezerra Parmera, Márcia Rúbia, Marcus Vinícius Della Coletta, Maria Sheila Guimarães Rocha, Mariana Spitz, Mônica Haddad, Nina Rosa A F Murta, Paulo Caramelli, Raimundo N D Rodrigues, Ricardo Nitrini, Roberto Prado, Vitor Tumas, Ylmar Corrêa Neto, Roberta Arb Saba
{"title":"Guidelines for Parkinson's disease management part II: consensus from the movement disorders scientific department of the Brazilian Academy of Neurology - non-motor symptoms.","authors":"Débora Palma Maia, Rubens Gisbert Cury, Pedro Renato P Brandão, Francisco E C Cardoso, Ana Paula Bertholo, André Carvalho Felicio, Arlete Hilbig, Bruno Lopes Santos Lobato, Eline Rozária F Barbosa, Elizabeth Maria A B Quagliato, Gustavo H C Sousa, Jacy Bezerra Parmera, Márcia Rúbia, Marcus Vinícius Della Coletta, Maria Sheila Guimarães Rocha, Mariana Spitz, Mônica Haddad, Nina Rosa A F Murta, Paulo Caramelli, Raimundo N D Rodrigues, Ricardo Nitrini, Roberto Prado, Vitor Tumas, Ylmar Corrêa Neto, Roberta Arb Saba","doi":"10.1055/s-0045-1802962","DOIUrl":"https://doi.org/10.1055/s-0045-1802962","url":null,"abstract":"<p><p>The treatment of Parkinson's disease (PD) is a challenge, especially because it is considered highly individualized. The Brazilian Academy of Neurology (ABN) has identified the need to disseminate knowledge about its management, adapting the best evidence to the Brazilian population. The present article aims to report the recommendations for the treatment of non-motor symptoms of PD, developed by a group of specialists in movement disorders from the ABN's scientific department. In 2021, the first part, referring to the motor symptoms of PD, was published. The main non-motor symptoms were addressed-among them neuropsychiatric symptoms, such as depression, anxiety, cognitive alteration, and psychosis-as well as the possible recommended therapies and medications used to control pain, sleep disorders, and dysautonomia.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-15"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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
Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil. 在巴西遗传性甲状腺素转淀粉样变性伴周围神经病变的真实世界的他法米底的经验。
IF 1 4区 医学
Arquivos de neuro-psiquiatria Pub Date : 2025-01-01 Epub Date: 2025-01-15 DOI: 10.1055/s-0044-1793936
Luiz Felipe Pinto, Marcus V Pinto, Paula Accioli, Gabriela Amorim, Renata Gervais de Santa Rosa, Moises Dias, Mariana Guedes, Carlos P Gomez, Roberto C Pedrosa, Marcia Waddington-Cruz
{"title":"Real-world tafamidis experience in hereditary transthyretin amyloidosis with peripheral neuropathy in Brazil.","authors":"Luiz Felipe Pinto, Marcus V Pinto, Paula Accioli, Gabriela Amorim, Renata Gervais de Santa Rosa, Moises Dias, Mariana Guedes, Carlos P Gomez, Roberto C Pedrosa, Marcia Waddington-Cruz","doi":"10.1055/s-0044-1793936","DOIUrl":"10.1055/s-0044-1793936","url":null,"abstract":"<p><strong>Background: </strong> Tafamidis is a kinetic stabilizer that binds to the transthyretin (TTR) gene, inhibiting its dissociation. It is the only disease-modifying treatment for hereditary TTR amyloidosis with peripheral neuropathy (ATTRv-PN) available in the National Therapeutic Form (Formulário Terapêutico Nacional, FTN, in Portuguese) of the Brazilian Unified Health System (Sistema Único de Saúde, SUS, in Portuguese).</p><p><strong>Objective: </strong> To assess if the efficacy and safety of tafamidis in the Brazilian real-world experience are comparable to the results of clinical trials.</p><p><strong>Methods: </strong> We retrospectively studied all patients with ATTRv-PN evaluated at our center from September 2011 to March 2022 (data cut-off) who were initiated on tafamidis and had at least 1 follow up visit 6 months after the initiation of the drug treatment. Neurologic and functional outcomes were compared from day 1 (D1) of the tafamidis treatment (baseline) to the last follow-up.</p><p><strong>Results: </strong> In total, 33 patients were included, 18 (55%) of whom were female. All patients were carriers of the V30M mutation of ATTRv-PN, and 20 (61%) presented early onset (EO) ATTRv-PN. At baseline, the median age of the sample was of 40 (interquartile range [IQR]: 36-68) years, the median Neuropathy Impairment Score (NIS) was of 10 (6-24) points, and the median body mass index (BMI) was of 26 (23-28) kg/m<sup>2</sup>. The mean follow-up time was of 3.4 years. At the last follow-up, the BMI, the neurological impairment, and the level of disability slightly worsened compared with baseline, while the findings of the nerve conduction studies remained stable. These same results were observed across EO and late-onset (LO) ATTRv-PN patients. A total of 25 (75.8%) patients were considered responders, and 8 (24.2%), non-responders.</p><p><strong>Conclusion: </strong> The efficacy and safety of tafamidis reported in clinical trials is expandable to the Brazilian real-world scenario in EO and late-onset (LO) ATTRv-PN.</p>","PeriodicalId":8694,"journal":{"name":"Arquivos de neuro-psiquiatria","volume":"83 1","pages":"1-6"},"PeriodicalIF":1.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11735064/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142999159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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