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2. Effect of Acoramidis on Myocardial Structure and Function in Transthyretin Amyloid Cardiomyopathy: Insights From the ATTRibute-CM Cardiac Magnetic Resonance (CMR) Substudy

Author

Razvi, Yousuf, Judge, Daniel P., Martinez-Naharro, Ana, Ioannou, Adam, Venneri, Lucia, Patel, Rishi, Gillmore, Julian D., Kellman, Peter, Edwards, Laura, Taubel, Jorg, Du, Jing, Tamby, Jean-François, Castaño, Adam, Siddhanti, Suresh, Katz, Leonid, Fox, Jonathan C., and Fontana, Marianna

Published
2024
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4. Risk Prediction and Follow-Up

Author

Aimo, Alberto, Vergaro, Giuseppe, Gillmore, Julian D., Emdin, Michele, editor, Vergaro, Giuseppe, editor, Aimo, Alberto, editor, and Fontana, Marianna, editor

Published
2024
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5. From Red Flags to Diagnosis

Author

Gillmore, Julian D., Aimo, Alberto, Garcia-Pavia, Pablo, Emdin, Michele, editor, Vergaro, Giuseppe, editor, Aimo, Alberto, editor, and Fontana, Marianna, editor

Published
2024
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12. Stratifying Disease Progression in Patients With Cardiac ATTR Amyloidosis

Author

Ioannou, Adam, Cappelli, Francesco, Emdin, Michele, Nitsche, Christian, Longhi, Simone, Masri, Ahmad, Cipriani, Alberto, Zampieri, Mattia, Colio, Federica, Poledniczek, Michael, Porcari, Aldostefano, Razvi, Yousuf, Aimo, Alberto, Vergaro, Giuseppe, De Michieli, Laura, Rauf, Muhammad U., Patel, Rishi K., Villanueva, Eugenia, Lustig, Yael, Venneri, Lucia, Martinez-Naharro, Ana, Lachmann, Helen, Wechalekar, Ashutosh, Whelan, Carol, Petrie, Aviva, Hawkins, Philip N., Solomon, Scott, Gillmore, Julian D., and Fontana, Marianna

Published
2024
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13. Complete responses in AL amyloidosis are unequal: the impact of free light chain mass spectrometry in AL amyloidosis

Author

Bomsztyk, Joshua, Ravichandran, Sriram, Giles, Hannah V., Wright, Nicola, Berlanga, Oscar, Khwaja, Jahanzaib, Mahmood, Shameem, Wisniowski, Brendan, Cohen, Oliver, Foard, Darren, Gilbertson, Janet, Rauf, Muhammad U., Starr, Neasa, Martinez-Naharro, Ana, Venneri, Lucia, Whelan, Carol, Fontana, Marianna, Hawkins, Philip N., Gillmore, Julian D., Lachmann, Helen, Harding, Stephen, Pratt, Guy, and Wechalekar, Ashutosh D.

Published
2024
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14. Clinical and Prognostic Implications of Right Ventricular Uptake on Bone Scintigraphy in Transthyretin Amyloid Cardiomyopathy

Author

Porcari, Aldostefano, Fontana, Marianna, Canepa, Marco, Biagini, Elena, Cappelli, Francesco, Gagliardi, Christian, Longhi, Simone, Pagura, Linda, Tini, Giacomo, Dore, Franca, Bonfiglioli, Rachele, Bauckneht, Matteo, Miceli, Alberto, Girardi, Francesca, Martini, Anna Lisa, Barbati, Giulia, Costanzo, Egidio Natalino, Caponetti, Angelo Giuseppe, Paccagnella, Andrea, Sguazzotti, Maurizio, La Malfa, Giovanni, Zampieri, Mattia, Sciagrà, Roberto, Perfetto, Federico, Rowczenio, Dorota, Gilbertson, Janet, Hutt, David F., Hawkins, Philip N., Rapezzi, Claudio, Merlo, Marco, Sinagra, Gianfranco, and Gillmore, Julian D.

Published
2024
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17. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Author

Coelho, Teresa, Ando, Yukio, Benson, Merrill D, Berk, John L, Waddington-Cruz, Márcia, Dyck, Peter J, Gillmore, Julian D, Khella, Sami L, Litchy, William J, Obici, Laura, Monteiro, Cecilia, Tai, Li-Jung, Viney, Nicholas J, Buchele, Gustavo, Brambatti, Michela, Jung, Shiangtung W, St. L. O’Dea, Louis, Tsimikas, Sotirios, Schneider, Eugene, Geary, Richard S, Monia, Brett P, and Gertz, Morie

Subjects
Pharmacology and Pharmaceutical Sciences, Biomedical and Clinical Sciences, Rare Diseases, Clinical Trials and Supportive Activities, Neurosciences, Orphan Drug, Clinical Research, Peripheral Neuropathy, Neurodegenerative, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, AKCEA-TTR-L-rx, Antisense oligonucleotide, Clinical trial design, Hereditary transthyretin-mediated amyloid polyneuropathy, Phase 3 clinical trial, AKCEA-TTR-Lrx, Clinical sciences
Abstract

IntroductionAKCEA-TTR-LRx is a ligand-conjugated antisense (LICA) drug in development for the treatment of hereditary transthyretin amyloidosis (hATTR), a fatal disease caused by mutations in the transthyretin (TTR) gene. AKCEA-TTR-LRx shares the same nucleotide sequence as inotersen, an antisense medicine approved for use in hATTR polyneuropathy (hATTR-PN). Unlike inotersen, AKCEA-TTR-LRx is conjugated to a triantennary N-acetylgalactosamine moiety that supports receptor-mediated uptake by hepatocytes, the primary source of circulating TTR. This advanced design increases drug potency to allow for lower and less frequent dosing. The NEURO-TTRansform study will investigate whether AKCEA-TTR-LRx is safe and efficacious, with the aim of improving neurologic function and quality of life in hATTR-PN patients.Methods/designApproximately 140 adults with stage 1 (independent ambulation) or 2 (requires ambulatory support) hATTR-PN are anticipated to enroll in this multicenter, open-label, randomized, phase 3 study. Patients will be assigned 6:1 to AKCEA-TTR-LRx 45 mg subcutaneously every 4 weeks or inotersen 300 mg once weekly until the prespecified week 35 interim efficacy analysis, after which patients receiving inotersen will receive AKCEA-TTR-LRx 45 mg subcutaneously every 4 weeks. All patients will then receive AKCEA-TTR-LRx through the remainder of the study treatment period. The final efficacy analysis at week 66 will compare the AKCEA-TTR-LRx arm with the historical placebo arm from the phase 3 trial of inotersen (NEURO-TTR). The primary outcome measures are between-group differences in the change from baseline in serum TTR, modified Neuropathy Impairment Score + 7, and Norfolk Quality of Life-Diabetic Neuropathy questionnaire.ConclusionNEURO-TTRansform is designed to determine whether targeted delivery of AKCEA-TTR-LRx to hepatocytes with lower and less frequent doses will translate into clinical and quality-of-life benefits for patients with hATTR-PN.Trial registrationThe study is registered at ClinicalTrials.gov (NCT04136184) and EudraCT (2019-001698-10).

Published
2021

18. Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen

Author

Coelho, Teresa, Waddington Cruz, Márcia, Chao, Chi-Chao, Parman, Yeşim, Wixner, Jonas, Weiler, Markus, Barroso, Fabio A., Dasgupta, Noel R., Jung, Shiangtung W., Schneider, Eugene, Viney, Nicholas J., Dyck, P. James B., Ando, Yukio, Gillmore, Julian D., Khella, Sami, Gertz, Morie A., Obici, Laura, and Berk, John L.

Published
2023
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19. Tracking Multiorgan Treatment Response in Systemic AL-Amyloidosis With Cardiac Magnetic Resonance Derived Extracellular Volume Mapping

Author

Ioannou, Adam, Patel, Rishi K., Martinez-Naharro, Ana, Razvi, Yousuf, Porcari, Aldostefano, Hutt, David F., Bandera, Francesco, Kotecha, Tushar, Venneri, Lucia, Chacko, Liza, Massa, Paolo, Hanger, Melissa, Knight, Daniel, Manisty, Charlotte, Moon, James, Quarta, Cristina, Lachmann, Helen, Whelan, Carol, Kellman, Peter, Hawkins, Philip N., Gillmore, Julian D., Wechelakar, Ashutosh, and Fontana, Marianna

Published
2023
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24. Abstract 11740: Redefining Cardiac Involvement in Systemic Immunoglobulin Light Chain Amyloidosis and Treatment Implications

Author

Porcari, Aldostefano, Masi, Ambra, Martinez-Naharro, Ana, Razvi, Yousuf, Patel, Rishi, Ioannou, Adam, Rauf, Muhammad, Sinigiani, Giulio, Wisniowski, Brendan, Filisetti, Stefano, CURRIE-CATHEY, Jasmine, OʼBeara, Sophie, Kotecha, Tushar, Knight, Daniel, Moon, James, sinagra, Gianfranco, Virsinskaite, Ruta, GILBERTSON, Janet A, Venneri, Lucia, Petrie, Aviva, Lachmann, Helen, Whelan, Carol, Kellman, Peter, Ravichandran, Sriram, Cohen, Oliver, Mahmood, Shameem, Hawkins, Philip N, Gillmore, Julian D, Wechalekar, Ashutosh, and Fontana, Marianna

Published
2023
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28. Extracellular Volume Fraction by Computed Tomography Predicts Long-Term Prognosis Among Patients With Cardiac Amyloidosis

Author

Gama, Francisco, Rosmini, Stefania, Bandula, Steve, Patel, Kush P., Massa, Paolo, Tobon-Gomez, Catalina, Ecke, Karolin, Stroud, Tyler, Condron, Mark, Thornton, George D., Bennett, Jonathan B., Wechelakar, Ashutosh, Gillmore, Julian D., Whelan, Carol, Lachmann, Helen, Taylor, Stuart A., Pugliese, Francesca, Fontana, Marianna, Moon, James C., Hawkins, Philip N., and Treibel, Thomas A.

Published
2022
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29. Addendum to ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI Expert Consensus Recommendations for Multimodality Imaging in Cardiac Amyloidosis: Part 1 of 2—Evidence Base and Standardized Methods of Imaging

Author

Dorbala, SHARMILA, ANDO, YUKIO, BOKHARI, SABAHAT, DISPENZIERI, ANGELA, FALK, RODNEY H., FERRARI, VICTOR A., FONTANA, MARIANNA, GHEYSENS, OLIVIER, GILLMORE, JULIAN D., GLAUDEMANS, ANDOR W.J.M., HANNA, MAZEN A., HAZENBERG, BOUKE P.C., KRISTEN, ARNT V., KWONG, RAYMOND Y., MAURER, MATHEW S., MERLINI, GIAMPAOLO, MILLER, EDWARD J., MOON, JAMES C., MURTHY, VENKATESH L., QUARTA, C. CRISTINA, RAPEZZI, CLAUDIO, RUBERG, FREDERICK L., SHAH, SANJIV J., SLART, RIEMER H.J.A., VERBERNE, HEIN J., and BOURQUE, JAMIESON M.

Published
2022
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33. Expansion of the National Amyloidosis Centre staging system to detect early mortality in transthyretin cardiac amyloidosis.

Author

Nitsche, Christian, Ioannou, Adam, Patel, Rishi K., Razvi, Yousuf, Porcari, Aldostefano, Rauf, Muhammad U., Bandera, Francesco, Aimo, Alberto, Emdin, Michele, Martinez‐Naharro, Ana, Venneri, Lucia, Petrie, Aviva, Wechalekar, Ashutosh, Lachmann, Helen, Hawkins, Philip N., Gillmore, Julian D., and Fontana, Marianna

Subjects
RECEIVER operating characteristic curves, CARDIAC amyloidosis, GLOMERULAR filtration rate, PEPTIDES, HEART failure, TRANSTHYRETIN, DISEASE progression
Abstract

Aims: Transthyretin cardiac amyloidosis (ATTR‐CA) is stratified into prognostic categories using the National Amyloidosis Centre (NAC) staging system. The aims of this study were to further expand the existing NAC staging system to incorporate an additional disease stage that would identify patients at high risk of early mortality. Methods and results: The traditional NAC staging system (stage 1: N‐terminal pro‐B‐type natriuretic peptide [NT‐proBNP] ≤3000 ng/L and estimated glomerular filtration rate [eGFR] ≥45 ml/min; stage 3: NT‐proBNP >3000 ng/L and eGFR <45 ml/min; stage 2: remainder) was expanded by the introduction of a new stage 4 (defined as NT‐proBNP ≥10 000 ng/L irrespective of eGFR) and studied in 2042 patients. The optimal NT‐proBNP cut‐point was established using time‐dependent receiver operating characteristic curves in the subgroup of patients with NAC stage 3 disease. Mortality at 1 year according to NAC stage was 2.3% (n = 20/886) for stage 1, 8.8% (n = 62/706) for stage 2, 10.4% (n = 28/270) for stage 3, and 30.6% (n = 55/180) for stage 4 (log‐rank p < 0.001). After adjustment for age, mortality hazard for stage 4 was >15‐fold higher than that of stage 1 (hazard ratio [HR] 15.5; 95% confidence interval [CI] 9.3–26.1) and >3‐fold higher than that of stage 3 (HR 3.4; 95% CI 2.2–5.4). The increased risk of early mortality was consistent across the different genotypes and subclasses of patients based on the severity of heart failure symptoms and echocardiographic parameters. Conclusions: The proposed modification of the NAC staging system identifies patients with ATTR‐CA at a high risk of early mortality, who may benefit from a more intensive treatment strategy, and who are most likely to experience an event early in the course of a clinical trial. [ABSTRACT FROM AUTHOR]

Published
2024
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34. Clinical Importance of Left Atrial Infiltration in Cardiac Transthyretin Amyloidosis

Author

Bandera, Francesco, Martone, Raffaele, Chacko, Liza, Ganesananthan, Sharmananthan, Gilbertson, Janet A., Ponticos, Markella, Lane, Thirusha, Martinez-Naharro, Ana, Whelan, Carol, Quarta, Cristina, Rowczenio, Dorota, Patel, Rishi, Razvi, Yousuf, Lachmann, Helen, Wechelakar, Ashutosh, Brown, James, Knight, Daniel, Moon, James, Petrie, Aviva, Cappelli, Francesco, Guazzi, Marco, Potena, Luciano, Rapezzi, Claudio, Leone, Ornella, Hawkins, Philip N., Gillmore, Julian D., and Fontana, Marianna

Published
2022
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35. Cardiopulmonary Exercise Testing in Evaluating Transthyretin Amyloidosis

Author

Patel, Rishi K., primary, Bandera, Francesco, additional, Venneri, Lucia, additional, Porcari, Aldostefano, additional, Razvi, Yousuf, additional, Ioannou, Adam, additional, Chacko, Liza, additional, Martinez-Naharro, Ana, additional, Rauf, Muhammad U., additional, Knight, Daniel, additional, Brown, James, additional, Petrie, Aviva, additional, Wechalekar, Ashutosh, additional, Whelan, Carol, additional, Lachmann, Helen, additional, Muthurangu, Vivek, additional, Guazzi, Marco, additional, Hawkins, Philip N., additional, Gillmore, Julian D., additional, and Fontana, Marianna, additional

Published
2024
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36. Addendum to ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI expert consensus recommendations for multimodality imaging in cardiac amyloidosis: Part 1 of 2—evidence base and standardized methods of imaging

Author

Dorbala, Sharmila, Ando, Yukio, Bokhari, Sabahat, Dispenzieri, Angela, Falk, Rodney H., Ferrari, Victor A., Fontana, Marianna, Gheysens, Olivier, Gillmore, Julian D., Glaudemans, Andor W.J.M., Hanna, Mazen A., Hazenberg, Bouke P.C., Kristen, Arnt V., Kwong, Raymond Y., Maurer, Mathew S., Merlini, Giampaolo, Miller, Edward J., Moon, James C., Murthy, Venkatesh L., Quarta, C. Cristina, Rapezzi, Claudio, Ruberg, Frederick L., Shah, Sanjiv J., Slart, Riemer H.J.A., Verberne, Hein J., and Bourque, Jamieson M.

Published
2021
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38. Redefining Cardiac Involvement and Targets of Treatment in Systemic Immunoglobulin AL Amyloidosis

Author

Porcari, Aldostefano, Masi, Ambra, Martinez-Naharro, Ana, Razvi, Yousuf, Patel, Rishi, Ioannou, Adam, Rauf, Muhammad U., Sinigiani, Giulio, Wisniowski, Brendan, Filisetti, Stefano, Currie-Cathey, Jasmine, O’Beara, Sophie, Kotecha, Tushar, Knight, Dan, Moon, James C., Sinagra, Gianfranco, Virsinskaite, Ruta, Gilbertson, Janet, Venneri, Lucia, Petrie, Aviva, Lachmann, Helen, Whelan, Carol, Kellman, Peter, Ravichandran, Sriram, Cohen, Oliver, Mahmood, Shameem, Manisty, Charlotte, Hawkins, Philip N., Gillmore, Julian D., Wechalekar, Ashutosh D., and Fontana, Marianna

Abstract

IMPORTANCE: Cardiac amyloid infiltration is the key determinant of survival in systemic light-chain (AL) amyloidosis. Current guidelines recommend early switching therapy in patients with a nonoptimal or suboptimal response regardless of the extent of cardiac amyloid infiltration. OBJECTIVE: To assess the differences between serum biomarkers, echocardiography, and cardiovascular magnetic resonance (CMR) with extracellular volume (ECV) mapping in characterizing cardiac amyloid, the independent prognostic role of these approaches, and the role of ECV mapping to guide treatment strategies. DESIGN, SETTING, AND PARTICIPANTS: Consecutive patients newly diagnosed with systemic AL amyloidosis (2015-2021) underwent echocardiography, cardiac biomarkers, and CMR with ECV mapping at diagnosis. Data were analyzed from January to June 2024. MAIN OUTCOMES AND MEASURES: The primary outcomes of the study were all-cause mortality and hematological response as defined according to validated criteria: no response (NR), partial response (PR), very good partial response (VGPR), and complete response (CR). Secondary outcomes were the depth and speed of hematological response and overall survival according to ECV. RESULTS: Of 560 patients with AL amyloidosis, the median (IQR) age was 68 years (59-74 years); 346 patients were male (61.8%) and 214 female (38.2%). Over a median (IQR) 40.5 months 9-58 months), ECV was independently associated with mortality. In the landmark analysis at 1 month, long-term survival was independent of the achieved hematological response in ECV less than 0.30% and ECV of 0.31% to 0.40%, while it was dependent on the depth of the hematological response in ECV greater than 0.40%. In the landmark analysis at 6 months, survival was independent of the achieved hematological response in ECV less than 0.30% and dependent on achieving at least PR in ECV of 0.31% to 0.40%. Survival was dependent on achieving CR in ECV of 0.41% to 0.50% and ECV greater than 0.50%. Achieving a deep hematological response at 1 month was associated with better survival compared with 6 months in patients with ECV greater than 0.40% but not with ECV less than 0.40%. CONCLUSIONS AND RELEVANCE: This study found that ECV mapping, in systemic AL amyloidosis, is an independent predictor of prognosis, can help define the hematological response associated with better long-term outcomes for each patient and potentially inform treatment strategies.

Published
2024
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39. Correction to: ASNC/AHA/ASE/EANM/HFSA/ISA/SCMR/SNMMI Expert consensus recommendations for multimodality imaging in cardiac amyloidosis: Part 2 of 2—Diagnostic criteria and appropriate utilization

Author

Dorbala, Sharmila, Ando, Yukio, Bokhari, Sabahat, Dispenzieri, Angela, Falk, Rodney H., Ferrari, Victor A., Fontana, Marianna, Gheysens, Olivier, Gillmore, Julian D., Glaudemans, Andor W. J. M., Hanna, Mazen A., Hazenberg, Bouke P. C., Kristen, Arnt V., Kwong, Raymond Y., Maurer, Mathew S., Merlini, Giampaolo, Miller, Edward J., Moon, James C., Murthy, Venkatesh L., Quarta, C. Cristina, Rapezzi, Claudio, Ruberg, Frederick L., Shah, Sanjiv J., Slart, Riemer H. J. A., Verberne, Hein J., and Bourque, Jamieson M.

Published
2021
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41. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

Author

Vita, Giuseppe, Rizzo, Vincenzo, Russo, Massimo, Mazzeo, Anna, Gentile, Luca, Berk, John L, Brueckner, Caitlin, Lazzari, Victoria, Wiesman, Janice, DeLong, Douglas, Victory, Jennifer, Dalton, James, May, John, Gilmore, Catherine, Attarian, Shahram, Diallo, Saran, Delmont, Emilien, Pouget, Jean, Verschueren, Annie, Grapperon, Aude-Marie, Campana-Salort, Emmanuelle, Conceição, Isabel M, Lopes, Ana, Lamas, Filipa, Neves, Carlos, Castro, Jose, Pereira, Pedro, Castro, Isabel, Franco, Ana, Santos, Miguel Oliveira, de Azevedo Coutinho, Conceição, Falcao de Campos, Catarina, Coelho, Teresa, Hipólito Reis, Antonio, Correia, Nuno, Perez, Javier M, Martins da Silva, Ana, Alves, Cristina, Cardoso, Marcio, Valdrez, Katia, Monte, Julia R, Pessoa, Bernardete, Guimaraes, Nadia, Freitas, Monica, Ramalho, Joana, Ferreira, Natalia, Kuzume, Daisuke, Tard, Celine, Waucquier, Nawal, Rougeaux, Isabelle, Brice, Sylvie, Kasprzyk, Emmanuelle, Elrezzi, Elise, Meguig, Sayah, Hachulla, Eric, Gauvain, Clement, Migaud-Chervy, Maria-Claire, Deplanque, Dominique, Jozefowicz, Elsa, Lebellec, Loic, Adams, David, Balaya-Gouraya, Line, Jehan Lacour, Nathalie, Bournane, Halima, Martin, Nathalie, Elabed, Mongia, Sacko, Niamey, Boubrit, Yasmine, Gaouar, Amina, Rakotondratafika, Fetra, Théaudin-Saliou, Marie, Cauquil-Michon, Cécile, Labeyrie, Celine, Not, Adeline, Al-Salameh, Abdallah, Lecoq, Anne-Lise, Stephant, Maeva, Echaniz-Laguna, Andoni, Becquemont, Laurent, Beaudonnet, Guillemette, Algalarrondo, Vincent, Eliahou, Ludivine, Slama, Michel S, Rousseau, Antoine, Signate, Aissatou, Berthelot, Emeline, Inamo, Jocelyn, Planté-Bordeneuve, Violaine, Vervoitte, Laetitia, Focseneanu, Cecile, Gendre, Thierry, Arrouasse, Raphaele, Ayache, Samar S., Ernande, Laura, Le Corvoisier, Philippe, Salhi, Hayet, Choumert, Ariane, Ehinger, Vincent, Ruiz, Julie, Charlin, Cyril, Megelin, Thomas, Brannagan III, Thomas H, Fayerman, Raisy, Kim, Arreum, Paras, Allan, Gonzalez, Leidy J, Tsang, Steven, Wajnsztajn, Fernanda, Shije, Jeffrey, Ulane, Christina, Kleyman, Inna, Weimer, Louis, Cioroiu, Comana, Lambrianides, Sakis, Abu-Manneh, Rana, Zamba-Papanicolaou, Eleni, Agathangelou, Petros, Leonidou, Eleni, Tada, Satoshi, Fujita, Akemi, Nagai, Masahiro, Ando, Rina, Hosokawa, Yuko, Yamanishi, Yuki, Overcash, J. Scott, Giardino, Elena, Boyer, Leslie, Dang, Lien, Le, An, Nguyen, Tyler, Giang, Lien, Sellers, Peter, Tran, Leyla, Truong, Nghi, Vinas, Maita, Hrkman, Nicole, Miller, Sarah, Nguyen, David, Smith, Ashley, Pu, Helen, Li, Steve, Vuong, Thao, Dioso, Holly, Green, Sinikka, Lee, Kia, Chu, Hanh, Waters, Michael, Coskun, Derya J, Zepeda, Karla A, O'Riordan, William, Obici, Laura, Cortese, Andrea, Lozza, Alessandro, Merlini, Giampaolo, Rosti, Vittorio, Sabatelli, Mario, Bisogni, Giulia, Bernardo, Daniela, Luigetti, Marco, Di Paolantonio, Andrea, Guglielmino, Valeria, Romano, Angela, Nienhuis, Hans, Bulthuis-Kuiper, Janita, Kristen, Arnt V, Gerk, Olga, Ulbricht, Hannah, Taylor, Lenka, Meyle, Eva, Kleinschmidt, Natalia, Meyrath, David, Noe-Schwenn, Simone, Meng, Ulrike, Bauer, Ralf, aus dem Siepen, Fabian, Hein, Selina, Takahashi, Tetsuya, Oshita, Tomohiko, Koujin, Yoko, Neshige, Shuichiro, Nezu, Tomohisa, Segawa, Akiko, Ueno, Hiroki, Morino, Hiroyuki, Campistol, Josep M, Rodas Marin, Lida Maria, Blasco Pelicano, Josep Miquel, Dávila, Lucía Galán, Palacios, Marta, Pytel Cordoba, Vanesa, Guerrero Sola, Antonio, Horga, Alejandro, García Feijoo, Julián, Perez de Isla, Leopoldo, Marques Júnior, Wilson, Moscardini, Mariana, Litcanov, Debora Cristina, Viera Lima, Ana Flavia, Rodrigues, Leonardo, Marques Coutinho, Barbara, Moreira, Carolina Lavigne, Daccach Marques, Vanessa, Munoz Beamud, Francisco, Gragera Martínez, Álvaro, Borrachero, Cristina, Losada López, Inés Asunción, Cisneros Barroso, Eugenia, Rodríguez Rodríguez, Adrián, Sanz, Monica, Rigo Oliver, Elena, González Moreno, Juan, Gamez Martinez, Jose M, Descals, Cristina, Uson, Mercedes, Jose Vega, Francisco, Figuerola, Antoni, Montala, Carles, Waddington-Cruz, Márcia, Dias da Silva, Moises, Gervais de Santa Rosa, Renata, Pinto, Luiz Felipe, Pinto, Marcus Vinicius, Cardoso Berensztejn, Amanda, Barroso, Fabio, Lautre, Andrea, Orellana, Lucas G, González-Duarte Briseño, Maria Alejandra, Cárdenas-Soto, Karla, Jiménez López, Brenda Poled, Pérez-Castañeda, Sandra Lorena, Cantú Brito, Carlos Gerardo, Rivera de la Parra, David, Hernandez Reyes, Jose Pablo, del Mar Saniger Alba, Maria, Criollo Mora, Elia, Parman, Yesim, Rezzan, Kus Jülide, Sahin, Erdi, Serbest, Nail G, Durmus, Hacer, Cakar, Arman, Tugal Tutkun, Nuriye Ilknur, Karamursel, Sacit, Elitok, Ali, Sirin Inan, Nermin G, Altinkurt, Emre, Polydefkis, Michael, Ye, Jing, Allen, Adriane C, Chaudhry, Vinay, Jarrett, Raquel, Bressler, Neil, Burks, Kathleen L, Liu, Qingfeng, Khoshnoodi, Mohammad, Judge, Daniel P, Vista, Geno, Shah, Syed Mahmood, Hamaguchi, Hirotoshi, Oda, Junko, Fukase, Emi, Taniguchi, Ikuko, Oda, Tetsuya, Endo, Hironobu, Shimomura, Masahiro, Katanazaka, Kimitaka, Koto, Shusuke, Nakano, Takahiro, Scheid, Christof, Zueiter, Andreas, Pester, Lars, Walter, Doreen, Özdemir, Betül, Frenzel, Lukas F, Holtick, Udo, Oh, Jeeyoung, Kim, Hee Jin, Shin, Hyun Jin, Choi, Kyomin, Yamashita, Taro, Ueda, Mitsuharu, Masuda, Teruaki, Misumi, Yohei, Ueda, Akihiko, Nakahara, Keiichi, Yorita, Akiko, Tsuruhisa, Seiko, Taniwaki, Takayuki, Harada, Masaya, Moritaka, Taiga, Sakurada, Naonori, Mauricio, Elizabeth A, Baskin, Amber, Dimberg, Elliot, Dispenzieri, Angela, Fonder, Amie, Hobbs, Miriam, Russell, Stephen J, Dyck, Peter, Gonsalves, Wilson, Leung, Nelson, Witzig, Thomas E, Zeldenrust, Steven R, Hwa, Lisa, Kapoor, Prashant, Kumar, Shaji K, Lin, Yi, Lust, John A, Rajkumar, Vincent S, Dingli, David, Gertz, Morie A, Go, Ronald, Hayman, Suzanne R, Dalia, Samir, Carrillo, Esmeralda, Gorevic, Peter, Mason, Garnette, Chao, Chi-Chao, Lee, Ming-Jen, Su, Jen-Jen, Hsieh, Sung-Tsang, Tsai, Li-Kai, Yeh, Shin-Joe, Yang, Chih-Chao, Ajroud-Driss, Senda Ajroud-Driss, Casey, Patricia, Joslin, Benjamin C, Freimer, Miriam, Sankey, Alison, Kenepp, Amanda, Heintzman, Sarah, LoRusso, Samantha, Hokezu, Youichi, Kim, Byoung-Joon, Kim, JuHyeon, Lee, Ga Yeon, Cho, Eun Bin, Jeon, Eun-Seok, Min, Ju-Hong, Seok, Jin Myoung, Lee, Hye Lim, Park, Jae Hong, Sekijima, Yoshiki, Miyazawa, Chinatsu, Kato, Nagaaki, Kishida, Dai, Hineno, Akiyo, Kodaira, Minori, Yoshinaga, Tsuneaki, Miyahara, Teruyoshi, Imai, Akira, Matsumoto, Kazuhiko, Lin, Kon-Ping, Lee, Yi-Chung, Wixner, Jonas, Falk, Malin, Pilebro, Bjorn, Suhr, Ole, Lindqvist, Per, Soderberg, Karin, Pedrosa-Domellöf, Fatima, Anan, Intissar, Nordh, Erik, Tournev, Ivaylo, Zhelyazkova-Glaveeva, Sashka, Cherneva, Zheyna, Sarafov, Staiko, Chamova, Teodora, Cherninkova-Gopina, Sylvia, Schmidt, Hartmut H, Friebel, Frauke, Zibert, Andree, Mihailovic, Natasa, Schubert, Friederike, Vorona, Elena, Lahme, Larissa, Huesing-Kabar, Anna, Schilling, Matthias, Kabar, Iyad, Gillmore, Julian D, Martinez-Naharro, Ana, Chacko, Liza, Cohen, Oliver, Law, Steven, Rezk, Tamer, Lachmann, Helen J, Quan, Dianna, Blume, Brianna, Dixon, Stacy, Low, Soon Chai, Chan, Soo Looi, Lim, He Eng Li, Goh, Khean Jin, Mezei, Michelle M, Kraus, Deborah, Jack, Kristin, Wade, N. Kevin, Lopate, Glenn, Zwijack, Brittany, Florence, Julaine, Sommerville, R. Brian, Stewart, Graeme, Ryder, Julie, Mekhael, Linda, Taylor, Mark, Suan, Daniel, Wells, Karen, Stone, Paula, Itoya, Amenze, Owusu-Sekyere, Mercy, Thai, Desmond, Chahine, Ilonah, Pedrosa, Salve, Do, Thi Hoa (Therese), González-Duarte, Alejandra, Kyriakides, Theodoros, Ajroud-Driss, Senda, Mauricio, Elizabeth, Brannagan, Thomas H, III, Aldinc, Emre, Wang, Jing Jing, White, Matthew T, Vest, John, Berber, Erhan, and Sweetser, Marianne T

Published
2021
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45. Serum neurofilament light chain in hereditary transthyretin amyloidosis: validation in real-life practice.

Author

Carroll, Antonia S., Razvi, Yousuf, O'Donnell, Luke, Veleva, Elena, Heslegrave, Amanda, Zetterberg, Henrik, Vucic, Steve, Kiernan, Matthew C., Rossor, Alexander M., Gillmore, Julian D., and Reilly, Mary M.

Subjects
TRANSTHYRETIN, CYTOPLASMIC filaments, AMYLOIDOSIS, GENE conversion, ASYMPTOMATIC patients
Abstract

Neurofilament light chain (NfL) has emerged as a sensitive biomarker in hereditary transthyretin amyloid polyneuropathy (ATTRv-PN). We hypothesise that NfL can identify conversion of gene carriers to symptomatic disease, and guide treatment approaches. Serum NfL concentration was measured longitudinally (2015–2022) in 59 presymptomatic and symptomatic ATTR variant carriers. Correlations between NfL and demographics, biochemistry and staging scores were performed as well as longitudinal changes pre- and post-treatment, and in asymptomatic and symptomatic cohorts. Receiver-operating analyses were performed to determine cut-off values. NfL levels correlated with examination scores (CMTNS, NIS and MRC; all p <.01) and increased with disease severity (PND and FAP; all p <.05). NfL was higher in symptomatic and sensorimotor converters, than asymptomatic or sensory converters irrespective of time (all p <.001). Symptomatic or sensorimotor converters were discriminated from asymptomatic patients by NfL concentrations >64.5 pg/ml (sensitivity= 91.9%, specificity = 88.5%), whereas asymptomatic patients could only be discriminated from sensory or sensorimotor converters or symptomatic individuals by a NfL concentration >88.9 pg/ml (sensitivity = 62.9%, specificity = 96.2%) However, an NfL increment of 17% over 6 months could discriminate asymptomatic from sensory or sensorimotor converters (sensitivity = 88.9%, specificity = 80.0%). NfL reduced with treatment by 36%/year and correlated with TTR suppression (r = 0.64, p =.008). This data validates the use of serum NfL to identify conversion to symptomatic disease in ATTRv-PN. NfL levels can guide assessment of disease progression and response to therapies. [ABSTRACT FROM AUTHOR]

Published
2024
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46. Degradation versus fibrillogenesis, two alternative pathways modulated by seeds and glycosaminoglycans

Author

Verona, Guglielmo, primary, Raimondi, Sara, additional, Canetti, Diana, additional, Mangione, P. Patrizia, additional, Marchese, Loredana, additional, Corazza, Alessandra, additional, Lavatelli, Francesca, additional, Gillmore, Julian D., additional, Taylor, Graham W., additional, Bellotti, Vittorio, additional, and Giorgetti, Sofia, additional

Published
2024
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47. Multiorgan Dysfunction and Associated Prognosis in Transthyretin Cardiac Amyloidosis

Author

Ioannou, Adam, primary, Nitsche, Christian, additional, Porcari, Aldostefano, additional, Patel, Rishi K., additional, Razvi, Yousuf, additional, Rauf, Muhammad U., additional, Martinez‐Naharro, Ana, additional, Venneri, Lucia, additional, Accietto, Antonella, additional, Netti, Lucrezia, additional, Bandera, Francesco, additional, Virsinskaite, Ruta, additional, Kotecha, Tushar, additional, Knight, Dan, additional, Petrie, Aviva, additional, Whelan, Carol, additional, Wechalekar, Ashutosh, additional, Lachmann, Helen, additional, Hawkins, Philip N., additional, Gillmore, Julian D., additional, and Fontana, Marianna, additional

Published
2024
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48. Impact of vutrisiran on exploratory cardiac parameters in hereditary transthyretin‐mediated amyloidosis with polyneuropathy

Author

Garcia‐Pavia, Pablo, primary, Grogan, Martha, additional, Kale, Parag, additional, Berk, John L., additional, Maurer, Mathew S., additional, Conceição, Isabel, additional, Di Carli, Marcelo, additional, Solomon, Scott D., additional, Chen, Chongshu, additional, Yureneva, Elena, additional, Vest, John, additional, and Gillmore, Julian D., additional

Published
2024
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49. Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy

Author

Gillmore, Julian D., primary, Judge, Daniel P., additional, Cappelli, Francesco, additional, Fontana, Marianna, additional, Garcia-Pavia, Pablo, additional, Gibbs, Simon, additional, Grogan, Martha, additional, Hanna, Mazen, additional, Hoffman, James, additional, Masri, Ahmad, additional, Maurer, Mathew S., additional, Nativi-Nicolau, Jose, additional, Obici, Laura, additional, Poulsen, Steen Hvitfeldt, additional, Rockhold, Frank, additional, Shah, Keyur B., additional, Soman, Prem, additional, Garg, Jyotsna, additional, Chiswell, Karen, additional, Xu, Haolin, additional, Cao, Xiaofan, additional, Lystig, Ted, additional, Sinha, Uma, additional, and Fox, Jonathan C., additional

Published
2024
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50. Primary Results From APOLLO-B Open-label Extension Study Of Patisiran In Patients With Transthyretin Cardiac Amyloidosis

Author

Maurer, Mathew S., primary, Berk, John L., additional, Hanna, Mazen A., additional, Gillmore, Julian D., additional, Schwartzmann, Pedro V., additional, Lairez, Olivier, additional, Hatano, Masaru, additional, Cho, Hyun-Jai, additional, van der Meer, Peter, additional, White, Matthew T., additional, Yureneva, Elena, additional, Sweetser, Marianne T., additional, Jay, Patrick Y., additional, Vest, John, additional, and Fontana, Marianna, additional

Published
2024
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