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4. Abstract 4144740: Characteristics and Natural History of Early ATTR Cardiac Amyloid Infiltration

Author

Porcari, Aldostefano, Razvi, Yousuf, Cappelli, Francesco, Nitsche, Christian, Serenelli, Matteo, Longhi, Simone, Sinigiani, Giulio, Cipriani, Alberto, Aimo, Alberto, Allegro, Valentina, Tomasoni, Daniela, zampieri, Mattia, Cantone, Anna, Masri, Ahmad, Urey, Marcus, Ioannou, Adam, Petrie, Aviva, Gustafsson, Finn, Whelan, Carol, Emdin, Michele, Metra, Marco, sinagra, Gianfranco, Lachmann, Helen, Wechalekar, Ashutosh, Hawkins, Philip, Gillmore, Julian, and Fontana, Marianna

Published
2024
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5. The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Lymphoid Neoplasms

Author

Alaggio, Rita, Amador, Catalina, Anagnostopoulos, Ioannis, Attygalle, Ayoma D, Araujo, Iguaracyra Barreto de Oliveira, Berti, Emilio, Bhagat, Govind, Borges, Anita Maria, Boyer, Daniel, Calaminici, Mariarita, Chadburn, Amy, Chan, John KC, Cheuk, Wah, Chng, Wee-Joo, Choi, John K, Chuang, Shih-Sung, Coupland, Sarah E, Czader, Magdalena, Dave, Sandeep S, de Jong, Daphne, Du, Ming-Qing, Elenitoba-Johnson, Kojo S, Ferry, Judith, Geyer, Julia, Gratzinger, Dita, Guitart, Joan, Gujral, Sumeet, Harris, Marian, Harrison, Christine J, Hartmann, Sylvia, Hochhaus, Andreas, Jansen, Patty M, Karube, Kennosuke, Kempf, Werner, Khoury, Joseph, Kimura, Hiroshi, Klapper, Wolfram, Kovach, Alexandra E, Kumar, Shaji, Lazar, Alexander J, Lazzi, Stefano, Leoncini, Lorenzo, Leung, Nelson, Leventaki, Vasiliki, Li, Xiao-Qiu, Lim, Megan S, Liu, Wei-Ping, Louissaint, Abner, Marcogliese, Andrea, Medeiros, L Jeffrey, Michal, Michael, Miranda, Roberto N, Mitteldorf, Christina, Montes-Moreno, Santiago, Morice, William, Nardi, Valentina, Naresh, Kikkeri N, Natkunam, Yasodha, Ng, Siok-Bian, Oschlies, Ilske, Ott, German, Parrens, Marie, Pulitzer, Melissa, Rajkumar, S Vincent, Rawstron, Andrew C, Rech, Karen, Rosenwald, Andreas, Said, Jonathan, Sarkozy, Clémentine, Sayed, Shahin, Saygin, Caner, Schuh, Anna, Sewell, William, Siebert, Reiner, Sohani, Aliyah R, Tooze, Reuben, Traverse-Glehen, Alexandra, Vega, Francisco, Vergier, Beatrice, Wechalekar, Ashutosh D, Wood, Brent, Xerri, Luc, and Xiao, Wenbin

Subjects
Cancer, Hematologic Neoplasms, Humans, Lymphoma, World Health Organization, Clinical Sciences, Oncology and Carcinogenesis, Immunology
Abstract

We herein present an overview of the upcoming 5th edition of the World Health Organization Classification of Haematolymphoid Tumours focussing on lymphoid neoplasms. Myeloid and histiocytic neoplasms will be presented in a separate accompanying article. Besides listing the entities of the classification, we highlight and explain changes from the revised 4th edition. These include reorganization of entities by a hierarchical system as is adopted throughout the 5th edition of the WHO classification of tumours of all organ systems, modification of nomenclature for some entities, revision of diagnostic criteria or subtypes, deletion of certain entities, and introduction of new entities, as well as inclusion of tumour-like lesions, mesenchymal lesions specific to lymph node and spleen, and germline predisposition syndromes associated with the lymphoid neoplasms.

Published
2022

6. Description and Cross-Sectional Analyses of 25,880 Adults and Children in the UK National Registry of Rare Kidney Diseases Cohort

Author

Abat, Sharirose, Adalat, Shazia, Agbonmwandolor, Joy, Ahmad, Zubaidah, Alejmi, Abdulfattah, Almasarwah, Rashid, Annear, Nicholas, Asgari, Ellie, Ayers, Amanda, Baharani, Jyoti, Balasubramaniam, Gowrie, Kpodo, Felix Jo-Bamba, Bansal, Tarun, Barratt, Alison, Barratt, Jonathan, Bates, Megan, Bayne, Natalie, Bendle, Janet, Benyon, Sarah, Bergmann, Carsten, Bhandari, Sunil, Bingham, Coralie, Boddana, Preetham, Bond, Sally, Braddon, Fiona, Bramham, Kate, Branson, Angela, Brearey, Stephen, Brocklebank, Vicky, Budwal, Sharanjit, Byrne, Conor, Cairns, Hugh, Camilleri, Brian, Campbell, Gary, Capell, Alys, Carmody, Margaret, Carson, Marion, Cathcart, Tracy, Catley, Christine, Cesar, Karine, Chan, Melanie, Chea, Houda, Chess, James, Cheung, Chee Kay, Chick, Katy-Jane, Chitalia, Nihil, Christian, Martin, Chrysochou, Tina, Clark, Katherine, Clayton, Christopher, Clissold, Rhian, Cockerill, Helen, Coelho, Joshua, Colby, Elizabeth, Colclough, Viv, Conway, Eileen, Cook, H. Terence, Cook, Wendy, Cooper, Theresa, Coward, Richard J., Crosbie, Sarah, Cserep, Gabor, Date, Anjali, Davidson, Katherine, Davies, Amanda, Dhaun, Neeraj, Dhaygude, Ajay, Diskin, Lynn, Dixit, Abhijit, Doctolero, Eunice Ann, Dorey, Suzannah, Downard, Lewis, Drayson, Mark, Dreyer, Gavin, Dutt, Tina, Etuk, Kufreabasi, Evans, Dawn, Finch, Jenny, Flinter, Frances, Fotheringham, James, Francis, Lucy, Gale, Daniel P., Gallagher, Hugh, Game, David, Garcia, Eva Lozano, Gavrila, Madita, Gear, Susie, Geddes, Colin, Gilchrist, Mark, Gittus, Matt, Goggolidou, Paraskevi, Goldsmith, Christopher, Gooden, Patricia, Goodlife, Andrea, Goodwin, Priyanka, Grammatikopoulos, Tassos, Gray, Barry, Griffith, Megan, Gumus, Steph, Gupta, Sanjana, Hamilton, Patrick, Harper, Lorraine, Harris, Tess, Haskell, Louise, Hayward, Samantha, Hegde, Shivaram, Hendry, Bruce, Hewins, Sue, Hewitson, Nicola, Hillman, Kate, Hiremath, Mrityunjay, Howson, Alexandra, Htet, Zay, Huish, Sharon, Hull, Richard, Humphries, Alister, Hunt, David P.J., Hunter, Karl, Hunter, Samantha, Ijeomah-Orji, Marilyn, Inston, Nick, Jayne, David, Jenfa, Gbemisola, Jenkins, Alison, Johnson, Sally, Jones, Caroline A., Jones, Colin, Jones, Amanda, Jones, Rachel, Kamesh, Lavanya, Kanigicherla, Durga, Frankl, Fiona Karet, Karim, Mahzuz, Kaur, Amrit, Kavanagh, David, Kearley, Kelly, Kerecuk, Larissa, Khwaja, Arif, King, Garry, King, Grant, Kislowska, Ewa, Klata, Edyta, Kokocinska, Maria, Lambie, Mark, Lawless, Laura, Ledson, Thomas, Lennon, Rachel, Levine, Adam P., Maggie Lai, Ling Wai, Lipkin, Graham, Lovitt, Graham, Lyons, Paul, Mabillard, Holly, Mackintosh, Katherine, Mahdi, Khalid, Maher, Eamonn, Marchbank, Kevin J., Mark, Patrick B., Masoud, Sherry, Masunda, Bridgett, Mavani, Zainab, Mayfair, Jake, McAdoo, Stephen, Mckinnell, Joanna, Melhem, Nabil, Meyrick, Simon, Moochhala, Shabbir, Morgan, Putnam, Morgan, Ann, Muhammad, Fawad, Murray, Shona, Novobritskaya, Kristina, Ong, Albert CM., Oni, Louise, Osmaston, Kate, Padmanabhan, Neal, Parkes, Sharon, Patrick, Jean, Pattison, James, Paul, Riny, Percival, Rachel, Perkins, Stephen J., Persu, Alexandre, Petchey, William G., Pickering, Matthew C., Pinney, Jennifer, Pitcher, David, Plumb, Lucy, Plummer, Zoe, Popoola, Joyce, Post, Frank, Power, Albert, Pratt, Guy, Pusey, Charles, Rabara, Ria, Rabuya, May, Raju, Tina, Javier, Chadd, Roberts, Ian SD., Roufosse, Candice, Rumjon, Adam, Salama, Alan, Saleem, Moin, Sandford, R.N., Sandu, Kanwaljit S., Sarween, Nadia, Sayer, John A., Sebire, Neil, Selvaskandan, Haresh, Shah, Sapna, Sharma, Asheesh, Sharples, Edward J., Sheerin, Neil, Shetty, Harish, Shroff, Rukshana, Simms, Roslyn, Sinha, Manish, Sinha, Smeeta, Smith, Kerry, Smith, Lara, Srivastava, Shalabh, Steenkamp, Retha, Stott, Ian, Stroud, Katerina, Swift, Pauline, Szklarzewicz, Justyna, Tam, Fred, Tan, Kay, Taylor, Robert, Tischkowitz, Marc, Thomas, Kay, Tse, Yincent, Turnbull, Alison, Turner, A. Neil, Tyerman, Kay, Usher, Miranda, Venkat-Raman, Gopalakrishnan, Walker, Alycon, Walsh, Stephen B., Waters, Aoife, Watt, Angela, Webster, Phil, Wechalekar, Ashutosh, Welsh, Gavin Iain, West, Nicol, Wheeler, David, Wiles, Kate, Willcocks, Lisa, Williams, Angharad, Williams, Emma, Williams, Karen, Wilson, Deborah H., Wilson, Patricia D., Winyard, Paul, Wong, Edwin, Wong, Katie, Wood, Grahame, Woodward, Emma, Woodward, Len, Woolf, Adrian, Wright, David, Downward, Lewis, Griffin, Sian, Hall, Matt, Karet Frankl, Fiona, Maher, Eamonn R., Pinney, Jenny, Tam, Frederick W.K., Wilson, Patricia, Sy, Karla Therese L., Huang, Kui, Ye, Jamie, Nitsch, Dorothea, and Bockenhauer, Detlef

Published
2024
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7. Correction: “The 5th edition of The World Health Organization Classification of Haematolymphoid Tumours: Lymphoid Neoplasms” Leukemia. 2022 Jul;36(7):1720–1748

Author

Alaggio, Rita, Amador, Catalina, Anagnostopoulos, Ioannis, Attygalle, Ayoma D., de Oliveira Araujo, Iguaracyra Barreto, Berti, Emilio, Bhagat, Govind, Borges, Anita Maria, Boyer, Daniel, Calaminici, Mariarita, Chadburn, Amy, Chan, John K. C., Cheuk, Wah, Chng, Wee-Joo, Choi, John K., Chuang, Shih-Sung, Coupland, Sarah E., Czader, Magdalena, Dave, Sandeep S., de Jong, Daphne, Di Napoli, Arianna, Du, Ming-Qing, Elenitoba-Johnson, Kojo S., Ferry, Judith, Geyer, Julia, Gratzinger, Dita, Guitart, Joan, Gujral, Sumeet, Harris, Marian, Harrison, Christine J., Hartmann, Sylvia, Hochhaus, Andreas, Jansen, Patty M., Karube, Kennosuke, Kempf, Werner, Khoury, Joseph, Kimura, Hiroshi, Klapper, Wolfram, Kovach, Alexandra E., Kumar, Shaji, Lazar, Alexander J., Lazzi, Stefano, Leoncini, Lorenzo, Leung, Nelson, Leventaki, Vasiliki, Li, Xiao-Qiu, Lim, Megan S., Liu, Wei-Ping, Louissaint, Jr, Abner, Marcogliese, Andrea, Medeiros, L. Jeffrey, Michal, Michael, Miranda, Roberto N., Mitteldorf, Christina, Montes-Moreno, Santiago, Morice, William, Nardi, Valentina, Naresh, Kikkeri N., Natkunam, Yasodha, Ng, Siok-Bian, Oschlies, Ilske, Ott, German, Parrens, Marie, Pulitzer, Melissa, Rajkumar, S. Vincent, Rawstron, Andrew C., Rech, Karen, Rosenwald, Andreas, Said, Jonathan, Sarkozy, Clémentine, Sayed, Shahin, Saygin, Caner, Schuh, Anna, Sewell, William, Siebert, Reiner, Sohani, Aliyah R., Suzuki, Ritsuro, Tooze, Reuben, Traverse-Glehen, Alexandra, Vega, Francisco, Vergier, Beatrice, Wechalekar, Ashutosh D., Wood, Brent, Xerri, Luc, and Xiao, Wenbin

Published
2023
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9. Healthcare Resource Utilization and Cost-of-Illness in Systemic Light Chain (AL) Amyloidosis in Europe: Results From the Real-World, Retrospective EMN23 Study

Author

Jaccard, Arnaud, Bridoux, Frank, Roeloffzen, Wilfried, Minnema, Monique C., Bergantim, Rui, Hájek, Roman, João, Cristina, Cibeira, M. Teresa, Palladini, Giovanni, Schönland, Stefan, Merlini, Giampaolo, Milani, Paolo, Dimopoulos, Meletios A., Ravichandran, Sriram, Hegenbart, Ute, Agis, Hermine, Gros, Blanca, Asra, Aisha, Magarotto, Valeria, Cheliotis, Giorgos, Psarros, Giorgos, Sonneveld, Pieter, Wechalekar, Ashutosh, and Kastritis, Efstathios

Published
2024
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11. Effects of rare kidney diseases on kidney failure: a longitudinal analysis of the UK National Registry of Rare Kidney Diseases (RaDaR) cohort

Author

Abat, Sharirose, Adalat, Shazia, Agbonmwandolor, Joy, Ahmad, Zubaidah, Alejmi, Abdulfattah, Almasarwah, Rashid, Annear, Nicholas, Asgari, Ellie, Ayers, Amanda, Baharani, Jyoti, Balasubramaniam, Gowrie, Kpodo, Felix, Bansal, Tarun, Barratt, Alison, Barratt, Jonathan, Bates, Megan, Bayne, Natalie, Bendle, Janet, Benyon, Sarah, Bergmann, Carsten, Bhandari, Sunil, Bingham, Coralie, Boddana, Preetham, Bond, Sally, Braddon, Fiona, Bramham, Kate, Branson, Angela, Brearey, Stephen, Brocklebank, Vicky, Budwal, Sharanjit, Byrne, Conor, Cairns, Hugh, Camilleri, Brian, Campbell, Gary, Capell, Alys, Carmody, Margaret, Carson, Marion, Cathcart, Tracy, Catley, Christine, Cesar, Karine, Chan, Melanie, Chea, Houda, Chess, James, Cheung, Chee Kay, Chick, Katy-Jane, Chitalia, Nihil, Christian, Martin, Chrysochou, Tina, Clark, Katherine, Clayton, Christopher, Clissold, Rhian, Cockerill, Helen, Coelho, Joshua, Colby, Elizabeth, Colclough, Viv, Conway, Eileen, Cook, H Terence, Cook, Wendy, Cooper, Theresa, Coward, Richard J, Crosbie, Sarah, Cserep, Gabor, Date, Anjali, Davidson, Katherine, Davies, Amanda, Dhaun, Neeraj, Dhaygude, Ajay, Diskin, Lynn, Dixit, Abhijit, Doctolero, Eunice, Dorey, Suzannah, Downard, Lewis, Drayson, Mark, Dreyer, Gavin, Dutt, Tina, Etuk, Kufreabasi, Evans, Dawn, Finch, Jenny, Flinter, Frances, Fotheringham, James, Francis, Lucy, Gale, Daniel P, Gallagher, Hugh, Game, David, Garcia, Eva, Gavrila, Madita, Gear, Susie, Geddes, Colin, Gilchrist, Mark, Gittus, Matt, Goggolidou, Paraskevi, Goldsmith, Christopher, Gooden, Patricia, Goodlife, Andrea, Goodwin, Priyanka, Grammatikopoulos, Tassos, Gray, Barry, Griffith, Megan, Gumus, Steph, Gupta, Sanjana, Hamilton, Patrick, Harper, Lorraine, Harris, Tess, Haskell, Louise, Hayward, Samantha, Hegde, Shivaram, Hendry, Bruce, Hewins, Sue, Hewitson, Nicola, Hillman, Kate, Hiremath, Mrityunjay, Howson, Alexandra, Htet, Zay, Huish, Sharon, Hull, Richard, Humphries, Alister, Hunt, David P J, Hunter, Karl, Hunter, Samantha, Ijeomah-Orji, Marilyn, Inston, Nick, Jayne, David, Jenfa, Gbemisola, Jenkins, Alison, Johnson, Sally, Jones, Caroline A, Jones, Colin, Jones, Amanda, Jones, Rachel, Kamesh, Lavanya, Kanigicherla, Durga, Karet Frankl, Fiona, Karim, Mahzuz, Kaur, Amrit, Kavanagh, David, Kearley, Kelly, Kerecuk, Larissa, Khwaja, Arif, King, Garry, King, Grant, Kislowska, Ewa, Klata, Edyta, Kokocinska, Maria, Lambie, Mark, Lawless, Laura, Ledson, Thomas, Lennon, Rachel, Levine, Adam P, Lai, Ling Wai Maggie, Lipkin, Graham, Lovitt, Graham, Lyons, Paul, Mabillard, Holly, Mackintosh, Katherine, Mahdi, Khalid, Maher, Eamonn, Marchbank, Kevin J, Mark, Patrick B, Masoud, Sherry, Masunda, Bridgett, Mavani, Zainab, Mayfair, Jake, McAdoo, Stephen, Mckinnell, Joanna, Melhem, Nabil, Meyrick, Simon, Moochhala, Shabbir, Morgan, Putnam, Morgan, Ann, Muhammad, Fawad, Murray, Shona, Novobritskaya, Kristina, Ong, Albert CM, Oni, Louise, Osmaston, Kate, Padmanabhan, Neal, Parkes, Sharon, Patrick, Jean, Pattison, James, Paul, Riny, Percival, Rachel, Perkins, Stephen J, Persu, Alexandre, Petchey, William G, Pickering, Matthew C, Pinney, Jennifer, Pitcher, David, Plumb, Lucy, Plummer, Zoe, Popoola, Joyce, Post, Frank, Power, Albert, Pratt, Guy, Pusey, Charles, Rabara, Ria, Rabuya, May, Raju, Tina, Javier, Chadd, Roberts, Ian S D, Roufosse, Candice, Rumjon, Adam, Salama, Alan, Saleem, Moin, Sandford, Richard, Sandu, Kanwaljit S, Sarween, Nadia, Sayer, John A, Sebire, Neil, Selvaskandan, Haresh, Sharma, Asheesh, Sharples, Edward J, Sheerin, Neil, Shetty, Harish, Shroff, Rukshana, Simms, Roslyn, Sinha, Manish, Sinha, Smeeta, Smith, Kerry, Smith, Lara, Srivastava, Shalabh, Steenkamp, Retha, Stott, Ian, Stroud, Katerina, Swift, Pauline, Szklarzewicz, Justyna, Tam, Fred, Tan, Kay, Taylor, Robert, Tischkowitz, Marc, Thomas, Kay, Tse, Yincent, Turnbull, Alison, Turner, A Neil, Tyerman, Kay, Usher, Miranda, Venkat-Raman, Gopalakrishnan, Walker, Alycon, Walsh, Stephen B, Waters, Aoife, Watt, Angela, Webster, Phil, Wechalekar, Ashutosh, Welsh, Gavin I, West, Nicol, Wheeler, David, Wiles, Kate, Willcocks, Lisa, Williams, Angharad, Williams, Emma, Williams, Karen, Wilson, Deborah H, Wilson, Patricia D, Winyard, Paul, Wong, Edwin, Wong, Katie, Wood, Grahame, Woodward, Emma, Woodward, Len, Woolf, Adrian, Wright, David, Downward, Lewis, Chrysochou, Constantina, Griffin, Sian, Hall, Matt, Maher, Eamonn R, Pinney, Jenny, Tam, Frederick W K, Turner, Andrew Neil, Wilson, Patricia, Taylor, Christopher Mark, Nitsch, Dorothea, and Bockenhauer, Detlef

Published
2024
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12. 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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13. Daratumumab plus bortezomib, cyclophosphamide, and dexamethasone in Asian patients with newly diagnosed AL amyloidosis: subgroup analysis of ANDROMEDA

Author

Suzuki, Kenshi, Wechalekar, Ashutosh D., Kim, Kihyun, Shimazaki, Chihiro, Kim, Jin Seok, Ikezoe, Takayuki, Min, Chang-Ki, Zhou, Fude, Cai, Zhen, Chen, Xiaonong, Iida, Shinsuke, Katoh, Nagaaki, Fujisaki, Tomoaki, Shin, Ho-Jin, Tran, NamPhuong, Qin, Xiang, Vasey, Sandra Y., Tromp, Brenda, Weiss, Brendan M., Comenzo, Raymond L., Kastritis, Efstathios, and Lu, Jin

Published
2023
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14. The management of light chain (AL) amyloidosis in Europe: clinical characteristics, treatment patterns, and efficacy outcomes between 2004 and 2018

Author

Palladini, Giovanni, Schönland, Stefan, Merlini, Giampaolo, Milani, Paolo, Jaccard, Arnaud, Bridoux, Frank, Dimopoulos, Meletios A., Ravichandran, Sriram, Hegenbart, Ute, Roeloffzen, Wilfried, Cibeira, M. Teresa, Agis, Hermine, Minnema, Monique C., Bergantim, Rui, Hájek, Roman, João, Cristina, Leonidakis, Alexandros, Cheliotis, Giorgos, Sonneveld, Pieter, Kastritis, Efstathios, and Wechalekar, Ashutosh

Published
2023
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15. Birtamimab plus standard of care in light-chain amyloidosis: the phase 3 randomized placebo-controlled VITAL trial

Author

Gertz, Morie A., Cohen, Adam D., Comenzo, Raymond L., Kastritis, Efstathios, Landau, Heather J., Libby, Edward N., Liedtke, Michaela, Sanchorawala, Vaishali, Schönland, Stefan, Wechalekar, Ashutosh, Zonder, Jeffrey A., Palladini, Giovanni, Walling, Jackie, Guthrie, Spencer, Nie, Christie, Karp, Carol, Jin, Yuying, Kinney, Gene G., and Merlini, Giampaolo

Published
2023
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18. 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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24. Consensus guidelines and recommendations for infection prevention in multiple myeloma: a report from the International Myeloma Working Group

Author

Raje, Noopur S, Anaissie, Elias, Kumar, Shaji K, Lonial, Sagar, Martin, Thomas, Gertz, Morie A, Krishnan, Amrita, Hari, Parameswaran, Ludwig, Heinz, O'Donnell, Elizabeth, Yee, Andrew, Kaufman, Jonathan L, Cohen, Adam D, Garderet, Laurent, Wechalekar, Ashutosh F, Terpos, Evangelos, Khatry, Navin, Niesvizky, Ruben, Yi, Qing, Joshua, Douglas E, Saikia, Tapan, Leung, Nelson, Engelhardt, Monika, Mothy, Mohamad, Branagan, Andrew, Chari, Ajai, Reiman, Anthony J, Lipe, Brea, Richter, Joshua, Rajkumar, S Vincent, Miguel, Jesús San, Anderson, Kenneth C, Stadtmauer, Edward A, Prabhala, Rao H, McCarthy, Phillip L, and Munshi, Nikhil C

Published
2022
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26. Pharmacodynamic evaluation and safety assessment of treatment with antibodies to serum amyloid P component in patients with cardiac amyloidosis: an open-label Phase 2 study and an adjunctive immuno-PET imaging study

Author

Wechalekar, Ashutosh, Antoni, Gunnar, Al Azzam, Wasfi, Bergström, Mats, Biswas, Swethajit, Chen, Chao, Cheriyan, Joseph, Cleveland, Matthew, Cookson, Louise, Galette, Paul, Janiczek, Robert L., Kwong, Raymond Y., Lukas, Mary Ann, Millns, Helen, Richards, Duncan, Schneider, Ian, Solomon, Scott D., Sörensen, Jens, Storey, James, Thompson, Douglas, van Dongen, Guus, Vugts, Danielle J., Wall, Anders, Wikström, Gerhard, and Falk, Rodney H.

Published
2022
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28. Response rates to second‐line treatment with daratumumab bortezomib dexamethasone (DVD) in relapsed/refractory light chain amyloidosis (AL) after initial Bortezomib‐based regime

Author

Bomsztyk, Joshua, primary, Ravichandran, Sriram, additional, Khwaja, Jahanzaib, additional, Cohen, Oliver, additional, Rauf, Muhammad U., additional, Foard, Darren, additional, Martinez‐Naharro, Ana, additional, Venneri, Lucia, additional, Whelan, Carol, additional, Fontana, Marianna, additional, Hawkins, Philip N., additional, Gillmore, Julian, additional, Lachmann, Helen, additional, Mahmood, Shameem, additional, and Wechalekar, Ashutosh D, additional

Published
2024
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29. Utility of 18F-PET Scintigraphy to detect inflammatory light chain proteotoxicity in cardiac AL amyloidosis

Author

Khwaja, Jahanzaib, primary, Ravichandran, Sriram, primary, Bomsztyk, Joshua, primary, Cohen, Oliver, primary, Foard, Darren, primary, Martinez-Naharro, Ana, primary, Venneri, Lucia, primary, Fontana, Marianna, primary, Whelan, Carol, primary, Hawkins, Philip, primary, Lachmann, Helen, primary, Mahmood, Shameem, primary, and Wechalekar, Ashutosh, primary

Published
2024
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30. Outcomes of Daratumumab Bortezomib Thalidomide Dexamethasone in treatment-naïve systemic AL amyloidosis

Author

Khwaja, Jahanzaib, primary, Ravichandran, Sriram, primary, Bomsztyk, Joshua, primary, Cohen, Oliver, primary, Foard, Darren, primary, Low, May, primary, Martinez-Naharro, Ana, primary, Venneri, Lucia, primary, Fontana, Marianna, primary, Hawkins, Philip, primary, Lachmann, Helen, primary, Whelan, Carol, primary, Mahmood, Shameem, primary, and Wechalekar, Ashutosh, primary

Published
2024
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31. Health-Related Quality of Life (HRQoL) in Patients With Mayo Stage IV Light Chain (AL) Amyloidosis Treated With Birtamimab Plus Standard of Care (SoC): Results From the VITAL Trial

Author

D'Souza, Anita, primary, Sanchorawala, Vaishali, primary, Kastritis, Efstathios, primary, Wechalekar, Ashutosh, primary, Schönland, Stefan, primary, Comenzo, Raymond, primary, Sprinz, Ingrid, primary, Nie, Christie, primary, Lin, Tim, primary, and Gertz, Morie, primary

Published
2024
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32. Refining prognostication in systemic AL amyloidosis

Author

Khwaja, Jahanzaib, primary, Ravichandran, Sriram, primary, Bomsztyk, Joshua, primary, Cohen, Oliver, primary, Foard, Darren, primary, Martinez-Naharro, Ana, primary, Venneri, Lucia, primary, Fontana, Marianna, primary, Whelan, Carol, primary, Hawkins, Philip, primary, Lachmann, Helen, primary, Mahmood, Shameem, primary, and Wechalekar, Ashutosh, primary

Published
2024
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33. Time to next treatment is longer in patients with a haematological complete response who are FLC-MS negative vs. positive

Author

Bomsztyk, Joshua, primary, Ravichandran, Sriram, primary, V, Hannah, primary, Wright, Nicola, primary, Berlanga, Oscar, primary, Khwaja, Jahanzaib, primary, Mahmood, Shameem, primary, Cohen, Oliver, primary, Foard, Darren, primary, Gilbertson, Janet, primary, U, Muhammad, primary, Martinez-Naharro, Ana, primary, Venneri, Lucia, primary, Whelan, Carol, primary, Fontana, Marianna, primary, Hawkins, Philip, primary, Lachmann, Helen, primary, Harding, Stephen, primary, Pratt, Guy, primary, and Wechalekar, Ashutosh, primary

Published
2024
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34. A UK experience of 874 endomyocardial biopsies for the diagnosis of amyloidosis

Author

Bomsztyk, Joshua, primary, Ravichandran, Sriram, primary, Mahmood, Shameem, primary, Cohen, Oliver, primary, Foard, Darren, primary, Smit, Angelique, primary, Gilbertson, Janet, primary, Botcher, Nicola, primary, Canetti, Diana, primary, Taylor, Graham, primary, U, Muhammad, primary, Coghlan, Gerry, primary, Martinez-Naharro, Ana, primary, Venneri, Lucia, primary, Whelan, Carol, primary, Fontana, Marianna, primary, Hawkins, Philip, primary, Lachmann, Helen, primary, and Wechalekar, Ashutosh, primary

Published
2024
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35. Prognostic Value of a 6-Minute Walk Test in Patients With Transthyretin Cardiac Amyloidosis

Author

Ioannou, Adam, primary, Fumagalli, Carlo, additional, Razvi, Yousuf, additional, Porcari, Aldostefano, additional, Rauf, Muhammad U., additional, Martinez-Naharro, Ana, additional, Venneri, Lucia, additional, Moody, William, additional, Steeds, Richard P., additional, Petrie, Aviva, additional, Whelan, Carol, additional, Wechalekar, Ashutosh, additional, Lachmann, Helen, additional, Hawkins, Philip N., additional, Solomon, Scott D., additional, Gillmore, Julian D., additional, and Fontana, Marianna, additional

Published
2024
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36. Description and cross-sectional analyses of 25,880 adults and children in the UK National Registry of Rare Kidney Diseases cohort

Author

Wong, Katie, primary, Pitcher, David, additional, Braddon, Fiona, additional, Downward, Lewis, additional, Steenkamp, Retha, additional, Masoud, Sherry, additional, Annear, Nicholas, additional, Barratt, Jonathan, additional, Bingham, Coralie, additional, Coward, Richard J., additional, Chrysochou, Tina, additional, Game, David, additional, Griffin, Sian, additional, Hall, Matt, additional, Johnson, Sally, additional, Kanigicherla, Durga, additional, Karet Frankl, Fiona, additional, Kavanagh, David, additional, Kerecuk, Larissa, additional, Maher, Eamonn R., additional, Moochhala, Shabbir, additional, Pinney, Jenny, additional, Sayer, John A., additional, Simms, Roslyn, additional, Sinha, Smeeta, additional, Srivastava, Shalabh, additional, Tam, Frederick W.K., additional, Thomas, Kay, additional, Turner, A. Neil, additional, Walsh, Stephen B., additional, Waters, Aoife, additional, Wilson, Patricia, additional, Wong, Edwin, additional, Abat, Sharirose, additional, Adalat, Shazia, additional, Agbonmwandolor, Joy, additional, Ahmad, Zubaidah, additional, Alejmi, Abdulfattah, additional, Almasarwah, Rashid, additional, Asgari, Ellie, additional, Ayers, Amanda, additional, Baharani, Jyoti, additional, Balasubramaniam, Gowrie, additional, Jo-Bamba Kpodo, Felix, additional, Bansal, Tarun, additional, Barratt, Alison, additional, Bates, Megan, additional, Bayne, Natalie, additional, Bendle, Janet, additional, Benyon, Sarah, additional, Bergmann, Carsten, additional, Bhandari, Sunil, additional, Boddana, Preetham, additional, Bond, Sally, additional, Bramham, Kate, additional, Branson, Angela, additional, Brearey, Stephen, additional, Brocklebank, Vicky, additional, Budwal, Sharanjit, additional, Byrne, Conor, additional, Cairns, Hugh, additional, Camilleri, Brian, additional, Campbell, Gary, additional, Capell, Alys, additional, Carmody, Margaret, additional, Carson, Marion, additional, Cathcart, Tracy, additional, Catley, Christine, additional, Cesar, Karine, additional, Chan, Melanie, additional, Chea, Houda, additional, Chess, James, additional, Cheung, Chee Kay, additional, Chick, Katy-Jane, additional, Chitalia, Nihil, additional, Christian, Martin, additional, Clark, Katherine, additional, Clayton, Christopher, additional, Clissold, Rhian, additional, Cockerill, Helen, additional, Coelho, Joshua, additional, Colby, Elizabeth, additional, Colclough, Viv, additional, Conway, Eileen, additional, Cook, H. Terence, additional, Cook, Wendy, additional, Cooper, Theresa, additional, Crosbie, Sarah, additional, Cserep, Gabor, additional, Date, Anjali, additional, Davidson, Katherine, additional, Davies, Amanda, additional, Dhaun, Neeraj, additional, Dhaygude, Ajay, additional, Diskin, Lynn, additional, Dixit, Abhijit, additional, Doctolero, Eunice Ann, additional, Dorey, Suzannah, additional, Downard, Lewis, additional, Drayson, Mark, additional, Dreyer, Gavin, additional, Dutt, Tina, additional, Etuk, Kufreabasi, additional, Evans, Dawn, additional, Finch, Jenny, additional, Flinter, Frances, additional, Fotheringham, James, additional, Francis, Lucy, additional, Gale, Daniel P., additional, Gallagher, Hugh, additional, Garcia, Eva Lozano, additional, Gavrila, Madita, additional, Gear, Susie, additional, Geddes, Colin, additional, Gilchrist, Mark, additional, Gittus, Matt, additional, Goggolidou, Paraskevi, additional, Goldsmith, Christopher, additional, Gooden, Patricia, additional, Goodlife, Andrea, additional, Goodwin, Priyanka, additional, Grammatikopoulos, Tassos, additional, Gray, Barry, additional, Griffith, Megan, additional, Gumus, Steph, additional, Gupta, Sanjana, additional, Hamilton, Patrick, additional, Harper, Lorraine, additional, Harris, Tess, additional, Haskell, Louise, additional, Hayward, Samantha, additional, Hegde, Shivaram, additional, Hendry, Bruce, additional, Hewins, Sue, additional, Hewitson, Nicola, additional, Hillman, Kate, additional, Hiremath, Mrityunjay, additional, Howson, Alexandra, additional, Htet, Zay, additional, Huish, Sharon, additional, Hull, Richard, additional, Humphries, Alister, additional, Hunt, David P.J., additional, Hunter, Karl, additional, Hunter, Samantha, additional, Ijeomah-Orji, Marilyn, additional, Inston, Nick, additional, Jayne, David, additional, Jenfa, Gbemisola, additional, Jenkins, Alison, additional, Jones, Caroline A., additional, Jones, Colin, additional, Jones, Amanda, additional, Jones, Rachel, additional, Kamesh, Lavanya, additional, Frankl, Fiona Karet, additional, Karim, Mahzuz, additional, Kaur, Amrit, additional, Kearley, Kelly, additional, Khwaja, Arif, additional, King, Garry, additional, King, Grant, additional, Kislowska, Ewa, additional, Klata, Edyta, additional, Kokocinska, Maria, additional, Lambie, Mark, additional, Lawless, Laura, additional, Ledson, Thomas, additional, Lennon, Rachel, additional, Levine, Adam P., additional, Maggie Lai, Ling Wai, additional, Lipkin, Graham, additional, Lovitt, Graham, additional, Lyons, Paul, additional, Mabillard, Holly, additional, Mackintosh, Katherine, additional, Mahdi, Khalid, additional, Maher, Eamonn, additional, Marchbank, Kevin J., additional, Mark, Patrick B., additional, Masunda, Bridgett, additional, Mavani, Zainab, additional, Mayfair, Jake, additional, McAdoo, Stephen, additional, Mckinnell, Joanna, additional, Melhem, Nabil, additional, Meyrick, Simon, additional, Morgan, Putnam, additional, Morgan, Ann, additional, Muhammad, Fawad, additional, Murray, Shona, additional, Novobritskaya, Kristina, additional, Ong, Albert CM., additional, Oni, Louise, additional, Osmaston, Kate, additional, Padmanabhan, Neal, additional, Parkes, Sharon, additional, Patrick, Jean, additional, Pattison, James, additional, Paul, Riny, additional, Percival, Rachel, additional, Perkins, Stephen J., additional, Persu, Alexandre, additional, Petchey, William G., additional, Pickering, Matthew C., additional, Pinney, Jennifer, additional, Plumb, Lucy, additional, Plummer, Zoe, additional, Popoola, Joyce, additional, Post, Frank, additional, Power, Albert, additional, Pratt, Guy, additional, Pusey, Charles, additional, Rabara, Ria, additional, Rabuya, May, additional, Raju, Tina, additional, Javier, Chadd, additional, Roberts, Ian SD., additional, Roufosse, Candice, additional, Rumjon, Adam, additional, Salama, Alan, additional, Saleem, Moin, additional, Sandford, R.N., additional, Sandu, Kanwaljit S., additional, Sarween, Nadia, additional, Sebire, Neil, additional, Selvaskandan, Haresh, additional, Shah, Sapna, additional, Sharma, Asheesh, additional, Sharples, Edward J., additional, Sheerin, Neil, additional, Shetty, Harish, additional, Shroff, Rukshana, additional, Sinha, Manish, additional, Smith, Kerry, additional, Smith, Lara, additional, Stott, Ian, additional, Stroud, Katerina, additional, Swift, Pauline, additional, Szklarzewicz, Justyna, additional, Tam, Fred, additional, Tan, Kay, additional, Taylor, Robert, additional, Tischkowitz, Marc, additional, Tse, Yincent, additional, Turnbull, Alison, additional, Turner, A Neil, additional, Tyerman, Kay, additional, Usher, Miranda, additional, Venkat-Raman, Gopalakrishnan, additional, Walker, Alycon, additional, Watt, Angela, additional, Webster, Phil, additional, Wechalekar, Ashutosh, additional, Welsh, Gavin Iain, additional, West, Nicol, additional, Wheeler, David, additional, Wiles, Kate, additional, Willcocks, Lisa, additional, Williams, Angharad, additional, Williams, Emma, additional, Williams, Karen, additional, Wilson, Deborah H., additional, Wilson, Patricia D., additional, Winyard, Paul, additional, Wong, Katie, additional, Wood, Grahame, additional, Woodward, Emma, additional, Woodward, Len, additional, Woolf, Adrian, additional, Wright, David, additional, Sy, Karla Therese L., additional, Huang, Kui, additional, Ye, Jamie, additional, Nitsch, Dorothea, additional, and Bockenhauer, Detlef, additional

Published
2024
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37. Inflammatory diseases underlying AA amyloidosis: analysis of 952 patients seen at a single reference centre over 34 years

Author

Lachmann, Helen, primary, Cohen, Oliver, primary, Fontana, Marianna, primary, Gilbertson, Janet, primary, D., Julian, primary, N, Philip, primary, Mahmood, Shameem, primary, Martinez-Naharro, Ana, primary, Peet, Claire, primary, Ravichandran, Sriram, primary, Rowczenio, Dorota, primary, Wechalekar, Ashutosh, primary, and Whelan, Carol, primary

Published
2024
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38. Efficacy and Safety of Belantamab Mafodotin Monotherapy in Patients with Relapsed or Refractory Light Chain Amyloidosis: A Phase 2 Study by the European Myeloma Network

Author

Kastritis, Efstathios, primary, Palladini, Giovanni, primary, Athanasios, Meletios, primary, Jaccard, Arnaud, primary, Merlini, Giampaolo, primary, Theodorakakou, Foteini, primary, Fotiou, Despina, primary, C., Monique, primary, Wechalekar, Ashutosh, primary, Ninos, Ioannis, primary, Psarros, Giorgos, primary, Sonneveld, Pieter, primary, and Schönland, Stefan, primary

Published
2024
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39. 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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40. Search for AL amyloidosis risk factors using Mendelian randomization

Author

Saunders, Charlie N., Chattopadhyay, Subhayan, Huhn, Stefanie, Weinhold, Niels, Hoffmann, Per, Nöthen, Markus M., Jöckel, Karl-Heinz, Schmidt, Börge, Landi, Stefano, Goldschmidt, Hartmut, Milani, Paolo, Merlini, Giampaolo, Rowcieno, Dorota, Hawkins, Philip, Hegenbart, Ute, Palladini, Giovanni, Wechalekar, Ashutosh, Schönland, Stefan O., Försti, Asta, Houlston, Richard, and Hemminki, Kari

Published
2021
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41. High response rates with single-agent belantamab mafodotin in relapsed systemic AL amyloidosis

Author

Khwaja, Jahanzaib, Bomsztyk, Joshua, Mahmood, Shameem, Wisniowski, Brendan, Shah, Raakhee, Tailor, Anish, Yong, Kwee, Popat, Rakesh, Rabin, Neil, Kyriakou, Charalampia, Sive, Jonathan, Esposti, Simona Degli, Larkin, Daniel F. P., Worthington, Sarah, Hart, Alyse, Dowling, Emma, Correia, Nuno, Bygrave, Ceri, Rydzewski, Andrzej, Jamroziak, Krzysztof, and Wechalekar, Ashutosh D.

Published
2022
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42. Outcomes of renal transplantation in patients with AL amyloidosis: an international collaboration through The International Kidney and Monoclonal Gammopathy Research Group

Author

Havasi, Andrea, Heybeli, Cihan, Leung, Nelson, Angel-Korman, Avital, Sanchorawala, Vaishali, Cohen, Oliver, Wechalekar, Ashutosh, Bridoux, Frank, Jaffer, Insara, Gutgarts, Victoria, Hassoun, Hani, Levinson, Maya, Rosenbaum, Cara, Milani, Paolo, Palladini, Giovanni, Merlini, Giampaolo, Hegenbart, Ute, Schönland, Stefan, Veelken, Kaya, Pogrebinsky, Alexander, Doros, Gheorghe, and Landau, Heather

Published
2022
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43. 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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44. Graded Organ Response and Progression Criteria for Kidney Immunoglobulin Light Chain Amyloidosis

Author

Muchtar, Eli, Wisniowski, Brendan, Geyer, Susan, Palladini, Giovanni, Milani, Paolo, Merlini, Giampaolo, Schönland, Stefan, Veelken, Kaya, Hegenbart, Ute, Leung, Nelson, Dispenzieri, Angela, Kumar, Shaji K., Kastritis, Efstathios, Dimopoulos, Meletios A., Liedtke, Michaela, Ulloa, Patricia, Sanchorawala, Vaishali, Szalat, Raphael, Dooley, Katharine, Landau, Heather, Petrlik, Erica, Lentzsch, Suzanne, Coltoff, Alexander, Bladé, Joan, Cibeira, M. Teresa, Cohen, Oliver, Foard, Darren, Gillmore, Jullian, Lachmann, Helen, Wechalekar, Ashutosh, and Gertz, Morie A.

Abstract

IMPORTANCE: Kidney light chain (AL) amyloidosis is associated with a risk of progression to kidney replacement therapy (KRT) and death. Several studies have shown that a greater reduction in proteinuria following successful anticlonal therapy is associated with improved outcomes. OBJECTIVE: To validate graded kidney response criteria and their association with kidney and overall survival (OS). DESIGN, SETTING, AND PARTICIPANTS: This retrospective, multicenter cohort was conducted at 10 referral centers for amyloidosis from 2010 to 2015 and included patients with kidney AL amyloidosis that was evaluable for kidney response and who achieved at least hematologic partial response within 12 months of diagnosis. The median follow-up was 69 (54-88) months. Data analysis was conducted in 2023. EXPOSURE: Four kidney response categories based on the reduction in pretreatment 24-hour urine protein (24-hour UP) levels: complete response (kidCR, 24-hour UP ≤200 mg), very good partial response (kidVGPR, >60% reduction in 24-hour UP), partial response (kidPR, 31%-60% reduction), and no response (kidNR, ≤30% reduction). Kidney response was assessed at landmark points (6, 12, and 24 months) and best kidney response. MAIN OUTCOMES AND MEASURES: Cumulative incidence of progression to KRT and OS. RESULTS: Seven-hundred and thirty-two patients (335 women [45.8%]) were included, with a median (IQR) age of 63 (55-69) years. The median (IQR) baseline 24-hour proteinuria and estimated glomerular filtration rate was 5.3 (2.8-8.5) g per 24 hours and 72 (48-92) mL/min/1.73m2, respectively. In a competing-risk analysis, the 5-year cumulative incidence rates of progression to KRT decreased with deeper kidney responses as early as 6 months from therapy initiation (11%, 12%, 2.1%, and 0% for kidNR, kidPR, kidVGPR, and kidCR, respectively; P = .002) and were maintained at 12 months and 24 months and best kidney response. Patients able to achieve kidCR/kidVGPR by 24 months and at best response had significantly better OS compared with kidPR/kidNR. Kidney progression, defined as a 25% or greater decrease in estimated glomerular filtration rate, was associated with cumulative incidence of progression to KRT and OS. CONCLUSIONS AND RELEVANCE: The results of this cohort study suggest that graded kidney response criteria offers clinically and prognostically meaningful information for treating patients with kidney AL amyloidosis. The response criteria potentially inform kidney survival based on the depth of reduction in 24-hour proteinuria levels and demonstrate an OS advantage for those able to achieve kidCR/kidVGPR compared with kidPR/kidNR. Taken together, achievement of at least kidVGPR by 12 months is needed to ultimately improve kidney and patient survival.

Published
2024
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46. Healthcare Resource Utilization and Cost-of-Illness in Systemic Light Chain (AL) Amyloidosis in Europe: Results From the Real-World, Retrospective EMN23 Study

Author

MS Hematologie, Cancer, Infection & Immunity, Regenerative Medicine and Stem Cells, Jaccard, Arnaud, Bridoux, Frank, Roeloffzen, Wilfried, Minnema, Monique C, Bergantim, Rui, Hájek, Roman, João, Cristina, Cibeira, M Teresa, Palladini, Giovanni, Schönland, Stefan, Merlini, Giampaolo, Milani, Paolo, Dimopoulos, Meletios A, Ravichandran, Sriram, Hegenbart, Ute, Agis, Hermine, Gros, Blanca, Asra, Aisha, Magarotto, Valeria, Cheliotis, Giorgos, Psarros, Giorgos, Sonneveld, Pieter, Wechalekar, Ashutosh, Kastritis, Efstathios, MS Hematologie, Cancer, Infection & Immunity, Regenerative Medicine and Stem Cells, Jaccard, Arnaud, Bridoux, Frank, Roeloffzen, Wilfried, Minnema, Monique C, Bergantim, Rui, Hájek, Roman, João, Cristina, Cibeira, M Teresa, Palladini, Giovanni, Schönland, Stefan, Merlini, Giampaolo, Milani, Paolo, Dimopoulos, Meletios A, Ravichandran, Sriram, Hegenbart, Ute, Agis, Hermine, Gros, Blanca, Asra, Aisha, Magarotto, Valeria, Cheliotis, Giorgos, Psarros, Giorgos, Sonneveld, Pieter, Wechalekar, Ashutosh, and Kastritis, Efstathios

Published
2024

48. 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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49. Renal Transplant Outcomes in Plasma Cell Dyscrasias and AL Amyloidosis after Treatment with Daratumumab.

Author

Mohidin, Barian, Needleman, Amy, Fernando, Raymond, Lowe, David M., Wechalekar, Ashutosh, Sheaff, Michael, Salama, Alan, and Jones, Gareth

Subjects
PLASMA cell diseases, CHRONIC kidney failure, PLASMA cells, RENAL replacement therapy, SEROTHERAPY
Abstract

Background: The morbidity and mortality from AL amyloidosis has significantly improved with the development of novel treatments. Daratumumab is a highly effective treatment for AL amyloidosis, but end-stage kidney disease is a common complication of this condition. Kidney transplantation is the ideal form of renal replacement therapy but has historically been contraindicated in this group of patients. Methods: Given the improved survival and better treatments of both conditions, we argue that it is time to reconsider transplanting these patients. Results: We report our experience of transplanting four patients with AL amyloidosis who had achieved stable remission through treatment with daratumumab. Conclusions: We highlight the key challenges involved and discuss important clinical issues for patients receiving daratumumab, particularly the difficulties with interpreting the crossmatch in light of daratumumab and immunoglobulin therapy interference. We also discuss the complexities involved in balancing the risks of infection, relapse, rejection, and immunosuppression in such patients. [ABSTRACT FROM AUTHOR]

Published
2024
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