Pub Date : 2024-12-03DOI: 10.1016/s2352-3026(24)00343-0
Charles A Schiffer
{"title":"Antifibrinolytics do not add to the benefits of platelet transfusion","authors":"Charles A Schiffer","doi":"10.1016/s2352-3026(24)00343-0","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00343-0","url":null,"abstract":"","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"10 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142790125","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-12-03DOI: 10.1016/s2352-3026(24)00317-x
TREATT Trial Investigators, Lise J Estcourt, Zoe K McQuilten, Peter Bardy, Merrole Cole-Sinclair, Graham P. Collins, Philip J. Crispin, Elinor Curnow, Jennifer Curnow, Amber Degelia, Claire Dyer, Adam Friebe, Lajos Floro, Effie Grand, Cara Hudson, Gail Jones, Joanne Joseph, Charlotte Kallmeyer, Marina Karakantza, Paul Kerr, Sara Last, Maria Lobo-Clarke, Matthew Lumley, Mary F McMullin, Patrick G. Medd, Suzy M. Morton, Andrew D. Mumford, Maria Mushkbar, Joseph Parsons, Gillian Powter, Mallika Sekhar, Laura Smith, Richard Soutar, William S. Stevenson, Elango Subramoniapillai, Jeff Szer, Helen Thomas, Neil A. Waters, Andrew H. Wei, David A. Westerman, Sarah A. Wexler, Erica M. Wood, Simon J. Stanworth, Adrienne Abioye, Rabia Afghan, Sylvia Ai Ai, Magbor Akanni, Rajesh Alajangi, Usmaan Alam, Bahaa Al-Bubseeree, Sophie Alderson, Craig Alderson, Sayed Ali, Kabir Ali, Rookmeen Alighan, Rebecca Allam Allam, Tania Allen, Wesam Al-Sakkaf, Kate Ames, Jacqueline Anderson, Colin Andrews, Ann-Marie Angel, Manuela Anlya Anlya, Farah Ansari, Rowan Appleby, Claire Arnold, Hulda Asbjornsdottir, Biruk Asfaw, Elissa Atkins, Leela Atkinson, Clare Aubrey, Noor Ayesha, Lola Babbola, David Badcock, Samuel Badcock, Diva Baggio, Ben Bailiff, Kizzy Baines, Holly Baker, Victoria Baker, Lindsay Ball, Martin Ball, Irwin Balquin, Emma Banks, George Banos, Jaytee Barnett, Claire Barrie, Claire Barron, Rebecca Barton, Nina Bason, Bindu Batta, Dianne Bautista, Angela Bayley, Emma Bayly, Fionnuala Baynes, Ali Bazargan, Rachel Bazeley, Yvonne Beadle, Claire Beardsmore, Kate Beattie, Kate Beattie, Caroline Bedford, Rachna Behal, Daniel Behan, Lilihna Bejan, Sarah Bell, Karen Bell, Louise Bell, Kaitlyn Bell, Reuben Benjamin, Sam Bennett, Gary Benson, Warwick Benson, Cameron Bent, Krystal Bergin, Alex Berry, Stephanie Besenyei, Caroline Besley, Scott Betteridge, Leigh Beveridge, Abir Bhattacharyya, Annelies Billen, Ian Bilmon, Emma Binns, Mark Birt, David Bishop, Andrea Blanco, Lisa Bleby, Richard Blemnerhet, Piers Blombery, Emily Blyth, Nicola Blythe, Lauren Boal, Ali Boden, Syed W.I. Bokhari, Elisa Bongetti, Stephen Booth, Jayne Borley, David Bowen, Dawn Bowers, Stephen Boyd, Sarah Bradley, Helen Bradman, Peta Bretag, Maria Brillante, Rachel Brockbank, Yasmin Brough, Ellen Brown, Jo Brown, Eleanor Brown, Claire Brown, Jenny Brown, Susan Brown, Joe Browning, Alex Brownsdon, David Bruce, Ruth Brydon-Hill, Andrea Buckwell, Dannielle Burgess, Glenda Burke, Kate Burley, Claire Burney, David Burns, Samuel Burrows, Kieran burton, Jason Butler, Lenka Cambalova, Maria C. Camozzi, Philip Campbell, Karen Campfield, Victoria Campion, Catherine Cargo, Julia Carmona, Dennis Carney, Joshua Casan, Helen Cashman, Lorraine Catt, Michael Cattell, Megan Cavill, Rachel Chadbone, Sridhar Chaganti, Yee Chai, Khai Li Chai, Joshua Chang, Judith Chapman, Oliver G. Chapman, Tamika Chapter, Andrew Charlton, Celina Chau, Saleena Chauhan, Nikesh Chavda, Frederick Chen, Melody Chen, Meng Xi Chen, Melanie Chen, Melissa Chen, Kathleen Cheok, Mai Cheung, Luke Chidgey, Karolina Chmielokliec, Philip Choi, Jae Choi, Anne Chok, Ruchika Chopra, Louise Christopherson, Vicky Chu, Chong Chyn Chua, Pavel Chudakou, Vidushi Chugh, Chi Chung, Erin Clark, Peter Clarke, Kathleen Clarke, Jennifer Clay, Laura Clayton, Mitch Clements, Jonathan Clemmens, Ruth Clifford, Dave Collett, Maia Collins, Emily Collyer, Maureen Connolly, Mark Cook, Sarah Coombs, Jason Coppell, Sophie Cornwell, Claire Corrigan, Elizabeth Coughlin, Jennifer Couling, Tony Cousins, Catriona Cowan, Christine Cox, Catherine Cox, Luke Coyle, Emily Craig, Thomas Creasey, Laura Croan, Jane Croft, Nicola Crosbie, Josephine Crowe, Helen Crowther, Jane Crozier, Naomi Culleton, Jonathan Cullis, Anita Cumming, Michelle Cummins, Adam Cunningham, Cameron Curley, Samantha Curtis, Robert Cuthbert, Kirsty Cuthill, Dinusha A Dahahayake, Amy Dang, Marc Davies, Ceri Davies, Emily Dawson, Tom Day, Kanchana De Abrew, Hugues De Lavallade, Neelaskshi De Silva, Georgina Dean, Christopher Deane, Lisa Demosthenous, Amisha Desai, Michael Desborough, Ian Devanny, Jay Dhanapal, Sundip Dhani, Vicky Di Martino, Emmy Dickens, Carmen DiCorleto, Louise Dinnett, Divya Dirisan, Karen Dixon, Kiri Dixon, Inderjit Doal, J Dobivh, Maria Docanto, Helve Doecke, David Donaldson, Kylee Donaldson, Carrie Donohoe, Ashley Douglas, Stephen Doung, Susan Downer, James D'Rozario, Malcolm Drummond, Mark Drummond, Samantha Drummond, Elizabeth Drysdale, Ross D'Souza, Eugene D'Souza, Alex Dunn, David Dutton, Martin Dyson, Kushani Ediriwicurena, Sharon Edleston, Dawn Edwards, Morgan Edwards, Anita Edwards, Nicole Eise, Steven Ellis, Hayley Ellis, Shareef Elmonley, Rosemarie Enstone, Agnes Eordogh, Sharon Erb, Shannon Evans, Megan Evans, Shannon Evans, Megan Evans, Joanne Ewing, Toby Eyre, Adam Facey, Mina Fammy, Jon Farman, Rachel Farnell, Laura Favero, Keith Fay, Karen Ferguson, Laura Fernon, Robin Filshie, Damian Finnegan, Lisa Fisher, Asia Flanagan, Emma Fleck, Simon Fletcher, Harpreet Flora, Catherine Flower, Ioana Fodor, Heather Foley, Emma Folland, Comfort Folorunso, Molly Forbes, Katrina Fordwor, Polly Foster, Vanessa Fox, Thomas Fox, Olesya Francis, Louise Fryearson, Madonna Fuery, Jiin Fung, Michelle Furtado, Leanne Galloway-Browne, Louise Gamble, Jeanette Gamgee, Arundathi Ganapathy, Hayley Gardner, Clare Gardner, Noha Gasmelsheed, Amy Gately, Lynda Gaynor, Alex Gebreid, Ruth Geffens, Rachel George, Aniko Gertner, Manar Ghebeh, Emanuela Ghirardini, Melainie Giddings, Sandra Gillett, Karen Gillett, Pratyush Giri, Chris Glass, Sarah Glewis, Sarah Gooding, Olivia Gordon, Joanne Gordon, David Gottlieb, Koushik Gowda, Elysie Gower, Nicola Gray, Jo Grayer, Elaine Greaves, Sally Anne Greenaway, Graeme Greenfield, Matthew Greenwood, Gareth Gregory, James Griffin, Julia Griffith, James Griffith, Lindsey Griffiths, Paulina Grzegrzolka, Yisu Gu, Jo Guest, Rosanna Guinai, Veena Gullapalli, A. Gunolr, Lina Guo, Wayne H, Sophia Hagua, Senait Haile, Richard Hall, Maryam Hamdollah-Zadeh, Zahra Hanif, Kathleen Hanlon, Nicholas Hann, Ramez Hanna, Guy Hannah, Sameera Hapuarachchi, Jacinta Hardman, Alison Hardy, Anthony Harris, Kylie Harris, Beth Harrison, Simon Harrison, Lea-Anne Harrison, Sean Harrop, Caroline Harvey, Faye Hatcliffe, Jo Hawking, Matthew Hawkins, Janet Hayden, Michelle Hayman, Elizabeth Haynes, Nicholas Heaney, Andrew Hebbard, Jenny Hempton, Sasanka Hendunneti, Maeve Henry, Jonathan Heywood, Catherine Hildyard, Lydia Hill, Annette Hilldrith, Petar Hitev, Smita Hiwase, Devendra Hiwase, Chris Hoare, Renate Hodge, Amy Holloway, Chloe Holt, Kelly Holton, Lauren Homer, Gillian Horne, Noemi Horvath, Linda Hotong, Kristen Houdyk, Katy Houseman, Ilda Hoxhallari, Hannah Hsu, Nina Hsu, Gillian Huang, Kerryn Hudson, Melanie Hufton, Timothy Hughes, Siobhan Hughes, Kate Hurley, Rosie Huxley, Temitope Ibitoye, Abubaken Ibrouf, Farha Inam, Tishya Indran, Karen Ingham, Calum Innes, David Irvine, Sarah Jaafar, Manish Jain, Laura Jameson, Pardeep Janjua, Rebecca Jarvis, Abirami Jatheendran, Abbie Javed, Sheila Jen, Shailesh Jobanpura, Irene Jobson, Deborah John, Sophie Johns, Amanda Johnston, Hollie Jones, Francesca Jones, Karolina Joniak, Michael Jovanovic, Anita Jovic, Lauren Joyce, Andrew Judd, Sudhakar Kakarlamudi, Nick Kakaroubas, Maggie Kalita, Shirly Kam, Julie Kan, P Kandle, Alex Kanellopoulos, Chien Kao, Maria Kaparou, Charamlampos Kartsios, Vicki Katsioulas, Russell Kaye, Katie Keen, Richard Kelly, Pauline Kelly, Donna Kelly, Melanie Kelly, Glen Kennedy, Nola Kennedy, Angela Kenny, Zoe Kenworthy, Ian Kerridge, Murali Kesavan, Angelika Khafizi, Muhammad Khakwani, Amna Khalid, Kate Khamly, Anjum Khan, Dalia Khan, Mojid Khan, Lubna Khan, Mona Khoo, Asim Khwaja, Grace Kim, Andrew King, Vicky King, Donna King, Francesca Kinsella, David Kipp, Pachoo Kirandeep, Laura C. Kirui, Bhuvan Kishore, Christopher Knectlhi, Amy Knot, Armit Knot, Cathy Ko, Caitlin Kolaric, Ray Koo, Mary Kotadia, Jaimal Kothari, Panagiotis D. Kottaridis, Gajan Kuiluinathan, Austin Kulasekararaj, John Kwan, Marwan Kwok, Phillip Kwok, Fiona Kwok, Kristiina Laane, Deena Lad, Jennifer Laird, Ada Lam, Mary Lane, Monica Lanenco, Susan Lang, Alex Langridge, Catherine Langton, Michelle Lannon, Annie Latif, Maya Latimer, Ruth Latter, I-Jun Lau, Sarah Lawless, Theresa Lawless, Mike Leach, Sarah Leaney, Heather Leary, James Leavy, Abbey LeBlanc, Vivienne Lee, Edwin Lee, Jenny Lee, Tamara Lee, Marian Leischkie, Emma Leitinger, Christopher Leon, Jayne Leonard, David Lewis, Ian Lewis, Tania Lewis, Daniel Lim, Kelly Littlewood, Dara Liu, Joanna Loh, Anand Lokare, Anand Lokare, Oliver Lomas, Richard Lovell, Theresa Lowe, Lisa Lowry, Marcin Lubowiecki, Rebecca Lumb, Gail Lynch, Amanda Macaulay, Lyndsey MacDonald, Jill MacDonald-Burn, Margaret Macmillan, Karen Maddock, Tomas Mahaliyana, Cassandra Mahon, Alison Maidment, Susie Maier, Michelle Mairos, Mahseeman Majid, Ka L Mak, Anne Mak, Arunthrthy Malendrayogau, Hana Malham, Felicity Malyon, Vineela Mandadapu, Laura Mandel, Sarah Mant, Ruth Manton, Nadjoua Maouche, Muhammad G. Maqbool, Gregory Marchant, Mariana Marinho, David Marks, Mike Marner, Helen Marr, Gillian Marshall, Siobhan Martin, Abigail Martin, Maria Marzolini, Kiara Mason, Jonathan Massie, Rebecca Masson, Vidya Mathavan, Siju Mathew, Judith Mathie, Lehenta Mattocks, Bernard Maybury, Georgina Mayer, Chyrelle McAlister, Jo McAllister, Stewart McConnell, James McCracken, Liz McCullagh, Rory McCulloch, Christopher Mcdermott, Kerian Mcdonald, Laura McGinniss, Fiona McGurk, Jessica McIlwain, Kirsten McIver, Pam Mckay, Lorraine McKenna, Donal Mclornan, Coalon McMahon, Linda McNeice, Susan McNeill, Molly McNickle, Fiona McQueen, Simon McRae, Bobby McTaggart, Jenny Mehew, Varnn Mehra, Michelle Melly, Tara Menichelli, Ken Micklethwatte, Loredana Mihailescue, Aleksander Mijovic, Hannah Millband, Lucy Miller, Samuel T. Millien, James Milnthorpe, Adrian Minson, Eva Molnar, Marc Monsour, Mary Moody, Rebecca Moon, Sally Moore, Katy Moore, Kelly Morgan, Rebecca Morralley, Denise Morris, Kirk Morris, Nicole Morrison, Merinda Moss, Muhammad Mughal, Paul Muir, David Mukkath, Aasiyu Mulla, Stephen Mulligan, Joanne Mullings, Angela Mulqueen, Caitlin Muluey, Sarah Murdoch, Sura Murrani, Vidhya Murthy, Jimmy Musngi, Nadreen Mustafa, Tracey Mynes, Anastasios Nalpantidis, Harshal Nandurkar, Linda Nardone, Latifa Nasari, Latifa Nasari, Monica Nash, Georgina Naylor, Loretta Ngu, Melissa Nguethina, John Nguyen, Joseph Nguyen, Wendy Nichol, Emma Nicholls, Catherine S. Nicole, Phillip Nicolson, David Nielson, Emmanouil Nikolousis, Georgina Nix, Rita Njoku, Jane Norman, Amy Norman, Phoebe Norris, Daniel North, Megan Norwood, Gaynor Notcheva, Igor Novitzky-Basso, Joseph Nyaboko, Maria Nygren, Ingrid Obu, Siobhan O'Connell, Jody O'Connor, Deanna O'Kelly, Aideen O'Niell, Jeremy Ony, Kathy Oo, April Oo, Anne Oppermann, Anne Oppermann, Ruth Orr, Mary O'Sullivan, Jennifer Page, Emma Palfreyman, Shankaranarayana Paneesha, Shyam Panicker, Catherine Parbutt, Elesha Parigi, Gemma Paris, Tracey Parker, Caroline Parnell, Christopher Parrish, Alex Parsons, Mioara Pasat, Natasha Patel, Vijay Patel, Pooja Patel, Chaya Patel, Nalini Pati, Andrea Patterson, Lauren Paul, Danielle Payet, Elspeth Payne, Victoria Peachey, Amanda Pearson, Andy Peniket, Laura Percy, Millicent Pereyra, Omer Pervaiz, Gunjan D Phalod, Anh Pham, Jason Pho, Keir Pickard, Michael Pidcock, Anna Piggin, Anna Piggin, Yalda Pishyar, Abigail Pocock, Ranjendres Pol, Paolo Polzella, Sonia Poolan, Vicki Portingale, Claire Posnett, Sandeep Potluri, Victoria Potter, Guy Pratt, Catherine Prodger, Andres Pueblo, Anish Puliyayil, Pratheepan Puvanakumar, Abdul Qadri, Hang Quach, Michael Quinn, Mark Rafferty, Marzia Rahman, Kavita Raj, Sonia Raj, Ramina Rajendran, Radha Ramanan, Karthik Ramasamy, Alexandros Rampotas, Natasha Ranchhod, Sabia Rashid, Sunita Ratanjee, Gurpreet Rathore, Sumita Ratnasingam, Manjit Rayat, Michael Rayner, Rebecca Reddell-Denton, Nicola Redding, Udaya Reddy, Atique Rehman, Carol Rice, Iwona Riches, Thomas Rider, John Riley, Ciro Rinaldi, Kayleigh Roberts, Andrew Roberts, Bryony Robertson, Peter Robertson, Dan Robinson, Rebecca Robinson, Emma Robjohns, Laura Robledo, Ana Rodrigues, Chris Rofe, Bridie Roff, Rachel Rogers, Jill Rolt, Carmela Rooney, Kathy Rose, Hannah Rose, David Ross, Shahara Rouf, Claire Rourke, David Routledge, Janet Ruggiero, Simon Rule, Richard Rumsey, Cherry Sagge, Helen Saldhana, Richard Salisbury, Sarah Salisbury, Ross Salvaris, Kay Sanders, Mirriam Sangombe, Anna Sanigorska, Kristine Santos, Taylah Sarkis, Anita Sarma, Natalie Saunders, Kara Schmidt, Anja Schmidtmann, Ann Schumacher, Tom Scorer Scorer, Asleigh Scott, Ingrid Seath, Frances Sejman, Adrian Selim, Nadia Shamim, Jocelyn Shan, Naranie Shanmuganathan, Shaminie Shanmugaranjan, Michelle Sharpe, Faye Sharpley, Emma Shaw, Cara Sheath, Oonagh Sheehy, Vivian Shen, Solomon Sherbide, Mathew Sheridan, Jane Sheridan, Matthew Sheridon, Tracy Shields, Hau V Sim, Shirlene Sim, Matt Sims, Lydia Singaraveloo, Gurcharan Singh, Jasmine Singh, Shree Sladesal, Andrew Sloan, Peter Slobodian, Sophie Smith, Sarit Smith, Claire Smith, Alastair Smith, Neil Smith, Katherine Snowden, Joel Solis, Denise Somios, Jade Soo, Michelle Spanevello, Madeleie Spaulding, Laura Spence, Liz Spillane, Alisha Spiteri, Naomi Sprigg, Sally Springett, Lynn Stafford, Katherine Stainthorp, Kate Stark, Louise Steeden, Ella Stephen, Aisling Stephenson, Andrew Stewart, Orla Stewart, Emma Stobie, Chelsea Stokes, Jacqui Streater, Charlie-Marie Suddens, Surenthini Suntharalingam, Narinder Surana, Robyn Sutherland, Antony Sutherland, David Sutton, Connor Sweeney, Reilly Sweet, Aniko P Szucs, Leila E. Taheri, Hinesh Tailor, Constantine Tam, Constantine Tam, George Tambakis, Mary Tamplin, Chee Tan, Sui Tan, Joanne Tan, Zhi Tan, Tatiana Taran, Fiona Tarpey, Angela Taseka, Suzy Tasker, Maciej Tatarczuch, Sarrah Tayabali, Hannah Taylor, Robert Taylor, Melaine Taylor, Ella Taylor-Moore, Lesley Teasdale, Elizabeth Tebbet, Aditya Tedjasepstra, Aditya Tedjaseputra, Oummy Tepkumkun, Andrew Terpstra, Wayne Thomas, Shanice Thomas, Rachel Thompson, Thomas Thornton, Bronwyn Thorp, Moi Yap Thrift, Phillipa Thwaites, Jasmine Timbres, Lauren Tindall, Ing Soo Tiong, Nicole Tippler, Tony Todd, Shirley Todd, Neil Toghill, Eve Tomlinson, Jacinta Tooth, M. Topp, Nicola Trail, Nguyen Tran, Elizabeth Tran, Vi Tran, Bona Treder, Michelle Tribbeck, Siobhan Trochowski, Maria Truslove, Tsun Tse, Bing Tseu, David Tucker, Kelly Turner, Dianne Turner, Kelly Turner, Herleen Turner, Gillian Turner, Julie Twohig, Thomas Tylee, Micheleine Uhe, Lauren Underhill, Joanne V, Georgina Van der Vliet, Tina Van Tonder, Carrie VanderWeyden, Jerry Varghese, Lachlan Vaughan, David Veale, Nicky Vickaryyous, Kathryn Vince, Jacoba Von Welligh, Sona Vora, Sona Vora, Karan Wadehra, Rebecca Walker, Stephen Walker, Roslyn Wallace, Stephanie Wallniosve, S. Wallwork, Zoe Walmsley, Fiona Walters, Joyce Wang, Angela Wang, Chen Wang, Mercy Wanyika, Dana Warcel, Katrina Wardrobe, Kristian Warnes, Christopher Waterhouse, Adam Waterworth, Caroline Watson, Edmund Watson, Emily Watts, Emma Weaver, Nicholas Weber, Kaytie Webley, Anna Welford, Matt Wells, Sarah Westbury, Jackie Westcott, Robyn Western, Julia Weston, Jessica White, Phillipa White, Anna Whitehead, James Whitehouse, Samantha Wieringa, John Willan, Sandra Williams, Bethany Williams, Stephanie Williamson, Brett Willoughby, Gail Wilmot, Rosalind Wilmott, Joanna Wilson, Emma Wilson, Suzy Wilson, Heather Wilson, Caroline Wilson, Tanya Wilson, Margaret Wilton, Paula Wiltshire, Joanne Wincup, Julia Wolf, Henna Wong, Cyndi Wong, Daniel Wong, Jonathan Wong, Shi Qin Wong, Sarah Wood, Henry Wood, Jackie Wooding, Kelly Woolley, Myles Wright, Myles Wright, Roland Wynn-Williams, Costas Yannakou, Zhi Han Yeoh, Zhi Han Yeoh, David Yeung, Agnes Young, Flora Yuen, Agnes Yuen, Oliver Zaja, Xiao-Yin Zhang, Mei Zhang
Bleeding is common in patients with haematological malignancies undergoing intensive therapy. We aimed to assess the effect of tranexamic acid on preventing bleeding and the need for platelet transfusions.
{"title":"Tranexamic acid versus placebo to prevent bleeding in patients with haematological malignancies and severe thrombocytopenia (TREATT): a randomised, double-blind, parallel, phase 3 superiority trial","authors":"TREATT Trial Investigators, Lise J Estcourt, Zoe K McQuilten, Peter Bardy, Merrole Cole-Sinclair, Graham P. Collins, Philip J. Crispin, Elinor Curnow, Jennifer Curnow, Amber Degelia, Claire Dyer, Adam Friebe, Lajos Floro, Effie Grand, Cara Hudson, Gail Jones, Joanne Joseph, Charlotte Kallmeyer, Marina Karakantza, Paul Kerr, Sara Last, Maria Lobo-Clarke, Matthew Lumley, Mary F McMullin, Patrick G. Medd, Suzy M. Morton, Andrew D. Mumford, Maria Mushkbar, Joseph Parsons, Gillian Powter, Mallika Sekhar, Laura Smith, Richard Soutar, William S. Stevenson, Elango Subramoniapillai, Jeff Szer, Helen Thomas, Neil A. Waters, Andrew H. Wei, David A. Westerman, Sarah A. Wexler, Erica M. Wood, Simon J. Stanworth, Adrienne Abioye, Rabia Afghan, Sylvia Ai Ai, Magbor Akanni, Rajesh Alajangi, Usmaan Alam, Bahaa Al-Bubseeree, Sophie Alderson, Craig Alderson, Sayed Ali, Kabir Ali, Rookmeen Alighan, Rebecca Allam Allam, Tania Allen, Wesam Al-Sakkaf, Kate Ames, Jacqueline Anderson, Colin Andrews, Ann-Marie Angel, Manuela Anlya Anlya, Farah Ansari, Rowan Appleby, Claire Arnold, Hulda Asbjornsdottir, Biruk Asfaw, Elissa Atkins, Leela Atkinson, Clare Aubrey, Noor Ayesha, Lola Babbola, David Badcock, Samuel Badcock, Diva Baggio, Ben Bailiff, Kizzy Baines, Holly Baker, Victoria Baker, Lindsay Ball, Martin Ball, Irwin Balquin, Emma Banks, George Banos, Jaytee Barnett, Claire Barrie, Claire Barron, Rebecca Barton, Nina Bason, Bindu Batta, Dianne Bautista, Angela Bayley, Emma Bayly, Fionnuala Baynes, Ali Bazargan, Rachel Bazeley, Yvonne Beadle, Claire Beardsmore, Kate Beattie, Kate Beattie, Caroline Bedford, Rachna Behal, Daniel Behan, Lilihna Bejan, Sarah Bell, Karen Bell, Louise Bell, Kaitlyn Bell, Reuben Benjamin, Sam Bennett, Gary Benson, Warwick Benson, Cameron Bent, Krystal Bergin, Alex Berry, Stephanie Besenyei, Caroline Besley, Scott Betteridge, Leigh Beveridge, Abir Bhattacharyya, Annelies Billen, Ian Bilmon, Emma Binns, Mark Birt, David Bishop, Andrea Blanco, Lisa Bleby, Richard Blemnerhet, Piers Blombery, Emily Blyth, Nicola Blythe, Lauren Boal, Ali Boden, Syed W.I. Bokhari, Elisa Bongetti, Stephen Booth, Jayne Borley, David Bowen, Dawn Bowers, Stephen Boyd, Sarah Bradley, Helen Bradman, Peta Bretag, Maria Brillante, Rachel Brockbank, Yasmin Brough, Ellen Brown, Jo Brown, Eleanor Brown, Claire Brown, Jenny Brown, Susan Brown, Joe Browning, Alex Brownsdon, David Bruce, Ruth Brydon-Hill, Andrea Buckwell, Dannielle Burgess, Glenda Burke, Kate Burley, Claire Burney, David Burns, Samuel Burrows, Kieran burton, Jason Butler, Lenka Cambalova, Maria C. Camozzi, Philip Campbell, Karen Campfield, Victoria Campion, Catherine Cargo, Julia Carmona, Dennis Carney, Joshua Casan, Helen Cashman, Lorraine Catt, Michael Cattell, Megan Cavill, Rachel Chadbone, Sridhar Chaganti, Yee Chai, Khai Li Chai, Joshua Chang, Judith Chapman, Oliver G. Chapman, Tamika Chapter, Andrew Charlton, Celina Chau, Saleena Chauhan, Nikesh Chavda, Frederick Chen, Melody Chen, Meng Xi Chen, Melanie Chen, Melissa Chen, Kathleen Cheok, Mai Cheung, Luke Chidgey, Karolina Chmielokliec, Philip Choi, Jae Choi, Anne Chok, Ruchika Chopra, Louise Christopherson, Vicky Chu, Chong Chyn Chua, Pavel Chudakou, Vidushi Chugh, Chi Chung, Erin Clark, Peter Clarke, Kathleen Clarke, Jennifer Clay, Laura Clayton, Mitch Clements, Jonathan Clemmens, Ruth Clifford, Dave Collett, Maia Collins, Emily Collyer, Maureen Connolly, Mark Cook, Sarah Coombs, Jason Coppell, Sophie Cornwell, Claire Corrigan, Elizabeth Coughlin, Jennifer Couling, Tony Cousins, Catriona Cowan, Christine Cox, Catherine Cox, Luke Coyle, Emily Craig, Thomas Creasey, Laura Croan, Jane Croft, Nicola Crosbie, Josephine Crowe, Helen Crowther, Jane Crozier, Naomi Culleton, Jonathan Cullis, Anita Cumming, Michelle Cummins, Adam Cunningham, Cameron Curley, Samantha Curtis, Robert Cuthbert, Kirsty Cuthill, Dinusha A Dahahayake, Amy Dang, Marc Davies, Ceri Davies, Emily Dawson, Tom Day, Kanchana De Abrew, Hugues De Lavallade, Neelaskshi De Silva, Georgina Dean, Christopher Deane, Lisa Demosthenous, Amisha Desai, Michael Desborough, Ian Devanny, Jay Dhanapal, Sundip Dhani, Vicky Di Martino, Emmy Dickens, Carmen DiCorleto, Louise Dinnett, Divya Dirisan, Karen Dixon, Kiri Dixon, Inderjit Doal, J Dobivh, Maria Docanto, Helve Doecke, David Donaldson, Kylee Donaldson, Carrie Donohoe, Ashley Douglas, Stephen Doung, Susan Downer, James D'Rozario, Malcolm Drummond, Mark Drummond, Samantha Drummond, Elizabeth Drysdale, Ross D'Souza, Eugene D'Souza, Alex Dunn, David Dutton, Martin Dyson, Kushani Ediriwicurena, Sharon Edleston, Dawn Edwards, Morgan Edwards, Anita Edwards, Nicole Eise, Steven Ellis, Hayley Ellis, Shareef Elmonley, Rosemarie Enstone, Agnes Eordogh, Sharon Erb, Shannon Evans, Megan Evans, Shannon Evans, Megan Evans, Joanne Ewing, Toby Eyre, Adam Facey, Mina Fammy, Jon Farman, Rachel Farnell, Laura Favero, Keith Fay, Karen Ferguson, Laura Fernon, Robin Filshie, Damian Finnegan, Lisa Fisher, Asia Flanagan, Emma Fleck, Simon Fletcher, Harpreet Flora, Catherine Flower, Ioana Fodor, Heather Foley, Emma Folland, Comfort Folorunso, Molly Forbes, Katrina Fordwor, Polly Foster, Vanessa Fox, Thomas Fox, Olesya Francis, Louise Fryearson, Madonna Fuery, Jiin Fung, Michelle Furtado, Leanne Galloway-Browne, Louise Gamble, Jeanette Gamgee, Arundathi Ganapathy, Hayley Gardner, Clare Gardner, Noha Gasmelsheed, Amy Gately, Lynda Gaynor, Alex Gebreid, Ruth Geffens, Rachel George, Aniko Gertner, Manar Ghebeh, Emanuela Ghirardini, Melainie Giddings, Sandra Gillett, Karen Gillett, Pratyush Giri, Chris Glass, Sarah Glewis, Sarah Gooding, Olivia Gordon, Joanne Gordon, David Gottlieb, Koushik Gowda, Elysie Gower, Nicola Gray, Jo Grayer, Elaine Greaves, Sally Anne Greenaway, Graeme Greenfield, Matthew Greenwood, Gareth Gregory, James Griffin, Julia Griffith, James Griffith, Lindsey Griffiths, Paulina Grzegrzolka, Yisu Gu, Jo Guest, Rosanna Guinai, Veena Gullapalli, A. Gunolr, Lina Guo, Wayne H, Sophia Hagua, Senait Haile, Richard Hall, Maryam Hamdollah-Zadeh, Zahra Hanif, Kathleen Hanlon, Nicholas Hann, Ramez Hanna, Guy Hannah, Sameera Hapuarachchi, Jacinta Hardman, Alison Hardy, Anthony Harris, Kylie Harris, Beth Harrison, Simon Harrison, Lea-Anne Harrison, Sean Harrop, Caroline Harvey, Faye Hatcliffe, Jo Hawking, Matthew Hawkins, Janet Hayden, Michelle Hayman, Elizabeth Haynes, Nicholas Heaney, Andrew Hebbard, Jenny Hempton, Sasanka Hendunneti, Maeve Henry, Jonathan Heywood, Catherine Hildyard, Lydia Hill, Annette Hilldrith, Petar Hitev, Smita Hiwase, Devendra Hiwase, Chris Hoare, Renate Hodge, Amy Holloway, Chloe Holt, Kelly Holton, Lauren Homer, Gillian Horne, Noemi Horvath, Linda Hotong, Kristen Houdyk, Katy Houseman, Ilda Hoxhallari, Hannah Hsu, Nina Hsu, Gillian Huang, Kerryn Hudson, Melanie Hufton, Timothy Hughes, Siobhan Hughes, Kate Hurley, Rosie Huxley, Temitope Ibitoye, Abubaken Ibrouf, Farha Inam, Tishya Indran, Karen Ingham, Calum Innes, David Irvine, Sarah Jaafar, Manish Jain, Laura Jameson, Pardeep Janjua, Rebecca Jarvis, Abirami Jatheendran, Abbie Javed, Sheila Jen, Shailesh Jobanpura, Irene Jobson, Deborah John, Sophie Johns, Amanda Johnston, Hollie Jones, Francesca Jones, Karolina Joniak, Michael Jovanovic, Anita Jovic, Lauren Joyce, Andrew Judd, Sudhakar Kakarlamudi, Nick Kakaroubas, Maggie Kalita, Shirly Kam, Julie Kan, P Kandle, Alex Kanellopoulos, Chien Kao, Maria Kaparou, Charamlampos Kartsios, Vicki Katsioulas, Russell Kaye, Katie Keen, Richard Kelly, Pauline Kelly, Donna Kelly, Melanie Kelly, Glen Kennedy, Nola Kennedy, Angela Kenny, Zoe Kenworthy, Ian Kerridge, Murali Kesavan, Angelika Khafizi, Muhammad Khakwani, Amna Khalid, Kate Khamly, Anjum Khan, Dalia Khan, Mojid Khan, Lubna Khan, Mona Khoo, Asim Khwaja, Grace Kim, Andrew King, Vicky King, Donna King, Francesca Kinsella, David Kipp, Pachoo Kirandeep, Laura C. Kirui, Bhuvan Kishore, Christopher Knectlhi, Amy Knot, Armit Knot, Cathy Ko, Caitlin Kolaric, Ray Koo, Mary Kotadia, Jaimal Kothari, Panagiotis D. Kottaridis, Gajan Kuiluinathan, Austin Kulasekararaj, John Kwan, Marwan Kwok, Phillip Kwok, Fiona Kwok, Kristiina Laane, Deena Lad, Jennifer Laird, Ada Lam, Mary Lane, Monica Lanenco, Susan Lang, Alex Langridge, Catherine Langton, Michelle Lannon, Annie Latif, Maya Latimer, Ruth Latter, I-Jun Lau, Sarah Lawless, Theresa Lawless, Mike Leach, Sarah Leaney, Heather Leary, James Leavy, Abbey LeBlanc, Vivienne Lee, Edwin Lee, Jenny Lee, Tamara Lee, Marian Leischkie, Emma Leitinger, Christopher Leon, Jayne Leonard, David Lewis, Ian Lewis, Tania Lewis, Daniel Lim, Kelly Littlewood, Dara Liu, Joanna Loh, Anand Lokare, Anand Lokare, Oliver Lomas, Richard Lovell, Theresa Lowe, Lisa Lowry, Marcin Lubowiecki, Rebecca Lumb, Gail Lynch, Amanda Macaulay, Lyndsey MacDonald, Jill MacDonald-Burn, Margaret Macmillan, Karen Maddock, Tomas Mahaliyana, Cassandra Mahon, Alison Maidment, Susie Maier, Michelle Mairos, Mahseeman Majid, Ka L Mak, Anne Mak, Arunthrthy Malendrayogau, Hana Malham, Felicity Malyon, Vineela Mandadapu, Laura Mandel, Sarah Mant, Ruth Manton, Nadjoua Maouche, Muhammad G. Maqbool, Gregory Marchant, Mariana Marinho, David Marks, Mike Marner, Helen Marr, Gillian Marshall, Siobhan Martin, Abigail Martin, Maria Marzolini, Kiara Mason, Jonathan Massie, Rebecca Masson, Vidya Mathavan, Siju Mathew, Judith Mathie, Lehenta Mattocks, Bernard Maybury, Georgina Mayer, Chyrelle McAlister, Jo McAllister, Stewart McConnell, James McCracken, Liz McCullagh, Rory McCulloch, Christopher Mcdermott, Kerian Mcdonald, Laura McGinniss, Fiona McGurk, Jessica McIlwain, Kirsten McIver, Pam Mckay, Lorraine McKenna, Donal Mclornan, Coalon McMahon, Linda McNeice, Susan McNeill, Molly McNickle, Fiona McQueen, Simon McRae, Bobby McTaggart, Jenny Mehew, Varnn Mehra, Michelle Melly, Tara Menichelli, Ken Micklethwatte, Loredana Mihailescue, Aleksander Mijovic, Hannah Millband, Lucy Miller, Samuel T. Millien, James Milnthorpe, Adrian Minson, Eva Molnar, Marc Monsour, Mary Moody, Rebecca Moon, Sally Moore, Katy Moore, Kelly Morgan, Rebecca Morralley, Denise Morris, Kirk Morris, Nicole Morrison, Merinda Moss, Muhammad Mughal, Paul Muir, David Mukkath, Aasiyu Mulla, Stephen Mulligan, Joanne Mullings, Angela Mulqueen, Caitlin Muluey, Sarah Murdoch, Sura Murrani, Vidhya Murthy, Jimmy Musngi, Nadreen Mustafa, Tracey Mynes, Anastasios Nalpantidis, Harshal Nandurkar, Linda Nardone, Latifa Nasari, Latifa Nasari, Monica Nash, Georgina Naylor, Loretta Ngu, Melissa Nguethina, John Nguyen, Joseph Nguyen, Wendy Nichol, Emma Nicholls, Catherine S. Nicole, Phillip Nicolson, David Nielson, Emmanouil Nikolousis, Georgina Nix, Rita Njoku, Jane Norman, Amy Norman, Phoebe Norris, Daniel North, Megan Norwood, Gaynor Notcheva, Igor Novitzky-Basso, Joseph Nyaboko, Maria Nygren, Ingrid Obu, Siobhan O'Connell, Jody O'Connor, Deanna O'Kelly, Aideen O'Niell, Jeremy Ony, Kathy Oo, April Oo, Anne Oppermann, Anne Oppermann, Ruth Orr, Mary O'Sullivan, Jennifer Page, Emma Palfreyman, Shankaranarayana Paneesha, Shyam Panicker, Catherine Parbutt, Elesha Parigi, Gemma Paris, Tracey Parker, Caroline Parnell, Christopher Parrish, Alex Parsons, Mioara Pasat, Natasha Patel, Vijay Patel, Pooja Patel, Chaya Patel, Nalini Pati, Andrea Patterson, Lauren Paul, Danielle Payet, Elspeth Payne, Victoria Peachey, Amanda Pearson, Andy Peniket, Laura Percy, Millicent Pereyra, Omer Pervaiz, Gunjan D Phalod, Anh Pham, Jason Pho, Keir Pickard, Michael Pidcock, Anna Piggin, Anna Piggin, Yalda Pishyar, Abigail Pocock, Ranjendres Pol, Paolo Polzella, Sonia Poolan, Vicki Portingale, Claire Posnett, Sandeep Potluri, Victoria Potter, Guy Pratt, Catherine Prodger, Andres Pueblo, Anish Puliyayil, Pratheepan Puvanakumar, Abdul Qadri, Hang Quach, Michael Quinn, Mark Rafferty, Marzia Rahman, Kavita Raj, Sonia Raj, Ramina Rajendran, Radha Ramanan, Karthik Ramasamy, Alexandros Rampotas, Natasha Ranchhod, Sabia Rashid, Sunita Ratanjee, Gurpreet Rathore, Sumita Ratnasingam, Manjit Rayat, Michael Rayner, Rebecca Reddell-Denton, Nicola Redding, Udaya Reddy, Atique Rehman, Carol Rice, Iwona Riches, Thomas Rider, John Riley, Ciro Rinaldi, Kayleigh Roberts, Andrew Roberts, Bryony Robertson, Peter Robertson, Dan Robinson, Rebecca Robinson, Emma Robjohns, Laura Robledo, Ana Rodrigues, Chris Rofe, Bridie Roff, Rachel Rogers, Jill Rolt, Carmela Rooney, Kathy Rose, Hannah Rose, David Ross, Shahara Rouf, Claire Rourke, David Routledge, Janet Ruggiero, Simon Rule, Richard Rumsey, Cherry Sagge, Helen Saldhana, Richard Salisbury, Sarah Salisbury, Ross Salvaris, Kay Sanders, Mirriam Sangombe, Anna Sanigorska, Kristine Santos, Taylah Sarkis, Anita Sarma, Natalie Saunders, Kara Schmidt, Anja Schmidtmann, Ann Schumacher, Tom Scorer Scorer, Asleigh Scott, Ingrid Seath, Frances Sejman, Adrian Selim, Nadia Shamim, Jocelyn Shan, Naranie Shanmuganathan, Shaminie Shanmugaranjan, Michelle Sharpe, Faye Sharpley, Emma Shaw, Cara Sheath, Oonagh Sheehy, Vivian Shen, Solomon Sherbide, Mathew Sheridan, Jane Sheridan, Matthew Sheridon, Tracy Shields, Hau V Sim, Shirlene Sim, Matt Sims, Lydia Singaraveloo, Gurcharan Singh, Jasmine Singh, Shree Sladesal, Andrew Sloan, Peter Slobodian, Sophie Smith, Sarit Smith, Claire Smith, Alastair Smith, Neil Smith, Katherine Snowden, Joel Solis, Denise Somios, Jade Soo, Michelle Spanevello, Madeleie Spaulding, Laura Spence, Liz Spillane, Alisha Spiteri, Naomi Sprigg, Sally Springett, Lynn Stafford, Katherine Stainthorp, Kate Stark, Louise Steeden, Ella Stephen, Aisling Stephenson, Andrew Stewart, Orla Stewart, Emma Stobie, Chelsea Stokes, Jacqui Streater, Charlie-Marie Suddens, Surenthini Suntharalingam, Narinder Surana, Robyn Sutherland, Antony Sutherland, David Sutton, Connor Sweeney, Reilly Sweet, Aniko P Szucs, Leila E. Taheri, Hinesh Tailor, Constantine Tam, Constantine Tam, George Tambakis, Mary Tamplin, Chee Tan, Sui Tan, Joanne Tan, Zhi Tan, Tatiana Taran, Fiona Tarpey, Angela Taseka, Suzy Tasker, Maciej Tatarczuch, Sarrah Tayabali, Hannah Taylor, Robert Taylor, Melaine Taylor, Ella Taylor-Moore, Lesley Teasdale, Elizabeth Tebbet, Aditya Tedjasepstra, Aditya Tedjaseputra, Oummy Tepkumkun, Andrew Terpstra, Wayne Thomas, Shanice Thomas, Rachel Thompson, Thomas Thornton, Bronwyn Thorp, Moi Yap Thrift, Phillipa Thwaites, Jasmine Timbres, Lauren Tindall, Ing Soo Tiong, Nicole Tippler, Tony Todd, Shirley Todd, Neil Toghill, Eve Tomlinson, Jacinta Tooth, M. Topp, Nicola Trail, Nguyen Tran, Elizabeth Tran, Vi Tran, Bona Treder, Michelle Tribbeck, Siobhan Trochowski, Maria Truslove, Tsun Tse, Bing Tseu, David Tucker, Kelly Turner, Dianne Turner, Kelly Turner, Herleen Turner, Gillian Turner, Julie Twohig, Thomas Tylee, Micheleine Uhe, Lauren Underhill, Joanne V, Georgina Van der Vliet, Tina Van Tonder, Carrie VanderWeyden, Jerry Varghese, Lachlan Vaughan, David Veale, Nicky Vickaryyous, Kathryn Vince, Jacoba Von Welligh, Sona Vora, Sona Vora, Karan Wadehra, Rebecca Walker, Stephen Walker, Roslyn Wallace, Stephanie Wallniosve, S. Wallwork, Zoe Walmsley, Fiona Walters, Joyce Wang, Angela Wang, Chen Wang, Mercy Wanyika, Dana Warcel, Katrina Wardrobe, Kristian Warnes, Christopher Waterhouse, Adam Waterworth, Caroline Watson, Edmund Watson, Emily Watts, Emma Weaver, Nicholas Weber, Kaytie Webley, Anna Welford, Matt Wells, Sarah Westbury, Jackie Westcott, Robyn Western, Julia Weston, Jessica White, Phillipa White, Anna Whitehead, James Whitehouse, Samantha Wieringa, John Willan, Sandra Williams, Bethany Williams, Stephanie Williamson, Brett Willoughby, Gail Wilmot, Rosalind Wilmott, Joanna Wilson, Emma Wilson, Suzy Wilson, Heather Wilson, Caroline Wilson, Tanya Wilson, Margaret Wilton, Paula Wiltshire, Joanne Wincup, Julia Wolf, Henna Wong, Cyndi Wong, Daniel Wong, Jonathan Wong, Shi Qin Wong, Sarah Wood, Henry Wood, Jackie Wooding, Kelly Woolley, Myles Wright, Myles Wright, Roland Wynn-Williams, Costas Yannakou, Zhi Han Yeoh, Zhi Han Yeoh, David Yeung, Agnes Young, Flora Yuen, Agnes Yuen, Oliver Zaja, Xiao-Yin Zhang, Mei Zhang","doi":"10.1016/s2352-3026(24)00317-x","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00317-x","url":null,"abstract":"Bleeding is common in patients with haematological malignancies undergoing intensive therapy. We aimed to assess the effect of tranexamic acid on preventing bleeding and the need for platelet transfusions.","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"20 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142790066","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-17DOI: 10.1016/s2352-3026(24)00250-3
Nicholas J Short,Daniel Nguyen,Elias Jabbour,Jayastu Senapati,Zhihong Zeng,Ghayas C Issa,Hussein Abbas,Cedric Nasnas,Wei Qiao,Xuelin Huang,Gautam Borthakur,Kelly Chien,Fadi G Haddad,Naveen Pemmaraju,Omer S Karrar,Danielle Nguyen,Marina Konopleva,Hagop Kantarjian,Farhad Ravandi
BACKGROUNDAdvanced phase Philadelphia chromosome-positive myeloid disease-consisting of chronic myeloid leukaemia in the myeloid blast phase and in the accelerated phase, and Philadelphia chromosome-positive acute myeloid leukaemia-is associated with poor outcomes. Although previous studies have suggested the benefit of chemotherapy and BCR::ABL1 tyrosine kinase inhibitor combinations, the optimal regimen is uncertain and prospective studies for this rare group of diseases are scant. Preclinical and retrospective clinical data suggest possible synergy between the BCL-2 inhibitor venetoclax and BCR::ABL1 tyrosine kinase inhibitors. We therefore aimed to design a study to evaluate the safety and activity of a novel combination of decitabine, venetoclax, and the third-generation BCR::ABL1 tyrosine kinase inhibitor ponatinib in advanced phase Philadelphia chromosome-positive myeloid diseases.METHODSFor this phase 2 study, patients aged 18 years or older with previously untreated or relapsed or refractory myeloid chronic myeloid leukaemia-blast phase, chronic myeloid leukaemia-accelerated phase, or advanced phase Philadelphia chromosome-positive acute myeloid leukaemia, and an Eastern Cooperative Oncology Group performance status of 0-3 were eligible. Patients were eligible regardless of the number of previous lines of therapy received or previous receipt of ponatinib. Cycle 1 (induction) consisted of a 7-day lead-in of ponatinib 45 mg orally daily (days 1-7), followed by combination therapy with decitabine 20 mg/m2 intravenously on days 8-12, venetoclax orally daily with ramp-up to a maximum dose of 400 mg on days 8-28, and ponatinib 45 mg orally daily on days 8-28. Cycles 2-24 consisted of decitabine 20 mg/m2 intravenously on days 1-5, venetoclax orally 400 mg on days 1-21, and ponatinib orally daily on days 1-28. Response-based dosing of ponatinib was implemented in consolidation cycles, with reduction to 30 mg daily in patients who reached complete remission or complete remission with an incomplete haematological recovery and a reduction to 15 mg daily in patients with undetectable BCR::ABL1 transcripts. The primary endpoint was the composite rate of complete remission or complete remission with incomplete haematological recovery in the intention-to-treat population. Safety was assessed in the intention-to-treat population. This trial was registered with ClinicalTrials.gov (NCT04188405) and is still ongoing.RESULTSBetween July 12, 2020, and July 8, 2023, 20 patients were treated (14 with chronic myeloid leukaemia-blast phase, four with chronic myeloid leukaemia-accelerated phase, and two with advanced phase Philadelphia chromosome-positive acute myeloid leukaemia). The median age was 43 years (IQR 32-58); 13 (65%) patients were male and seven (35%) were female; and 12 (60%) were White, three (15%) were Hispanic, four (20%) were Black, and one (5%) was Asian. 12 (60%) patients had received 2 or more previous BCR::ABL1 tyrosine kinase inhibitors, an
{"title":"Decitabine, venetoclax, and ponatinib for advanced phase chronic myeloid leukaemia and Philadelphia chromosome-positive acute myeloid leukaemia: a single-arm, single-centre phase 2 trial.","authors":"Nicholas J Short,Daniel Nguyen,Elias Jabbour,Jayastu Senapati,Zhihong Zeng,Ghayas C Issa,Hussein Abbas,Cedric Nasnas,Wei Qiao,Xuelin Huang,Gautam Borthakur,Kelly Chien,Fadi G Haddad,Naveen Pemmaraju,Omer S Karrar,Danielle Nguyen,Marina Konopleva,Hagop Kantarjian,Farhad Ravandi","doi":"10.1016/s2352-3026(24)00250-3","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00250-3","url":null,"abstract":"BACKGROUNDAdvanced phase Philadelphia chromosome-positive myeloid disease-consisting of chronic myeloid leukaemia in the myeloid blast phase and in the accelerated phase, and Philadelphia chromosome-positive acute myeloid leukaemia-is associated with poor outcomes. Although previous studies have suggested the benefit of chemotherapy and BCR::ABL1 tyrosine kinase inhibitor combinations, the optimal regimen is uncertain and prospective studies for this rare group of diseases are scant. Preclinical and retrospective clinical data suggest possible synergy between the BCL-2 inhibitor venetoclax and BCR::ABL1 tyrosine kinase inhibitors. We therefore aimed to design a study to evaluate the safety and activity of a novel combination of decitabine, venetoclax, and the third-generation BCR::ABL1 tyrosine kinase inhibitor ponatinib in advanced phase Philadelphia chromosome-positive myeloid diseases.METHODSFor this phase 2 study, patients aged 18 years or older with previously untreated or relapsed or refractory myeloid chronic myeloid leukaemia-blast phase, chronic myeloid leukaemia-accelerated phase, or advanced phase Philadelphia chromosome-positive acute myeloid leukaemia, and an Eastern Cooperative Oncology Group performance status of 0-3 were eligible. Patients were eligible regardless of the number of previous lines of therapy received or previous receipt of ponatinib. Cycle 1 (induction) consisted of a 7-day lead-in of ponatinib 45 mg orally daily (days 1-7), followed by combination therapy with decitabine 20 mg/m2 intravenously on days 8-12, venetoclax orally daily with ramp-up to a maximum dose of 400 mg on days 8-28, and ponatinib 45 mg orally daily on days 8-28. Cycles 2-24 consisted of decitabine 20 mg/m2 intravenously on days 1-5, venetoclax orally 400 mg on days 1-21, and ponatinib orally daily on days 1-28. Response-based dosing of ponatinib was implemented in consolidation cycles, with reduction to 30 mg daily in patients who reached complete remission or complete remission with an incomplete haematological recovery and a reduction to 15 mg daily in patients with undetectable BCR::ABL1 transcripts. The primary endpoint was the composite rate of complete remission or complete remission with incomplete haematological recovery in the intention-to-treat population. Safety was assessed in the intention-to-treat population. This trial was registered with ClinicalTrials.gov (NCT04188405) and is still ongoing.RESULTSBetween July 12, 2020, and July 8, 2023, 20 patients were treated (14 with chronic myeloid leukaemia-blast phase, four with chronic myeloid leukaemia-accelerated phase, and two with advanced phase Philadelphia chromosome-positive acute myeloid leukaemia). The median age was 43 years (IQR 32-58); 13 (65%) patients were male and seven (35%) were female; and 12 (60%) were White, three (15%) were Hispanic, four (20%) were Black, and one (5%) was Asian. 12 (60%) patients had received 2 or more previous BCR::ABL1 tyrosine kinase inhibitors, an","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"52 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142275331","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
BACKGROUNDMost patients with myelofibrosis develop ruxolitinib intolerance or disease that is relapsed or refractory, and survival rates after ruxolitinib discontinuation are poor. We aimed to evaluate the safety and efficacy of fedratinib versus best available therapy (BAT) in patients with myelofibrosis previously treated with ruxolitinib.METHODSFREEDOM2 was a multicentre, open-label, randomised, controlled, phase 3 trial in 86 clinics in 16 countries, in which patients aged at least 18 years with intermediate-2 or high-risk myelofibrosis that was relapsed or refractory or intolerant to ruxolitinib with Eastern Cooperative Oncology Group performance status 0-2 were stratified by spleen size by palpation, platelet count, and previous ruxolitinib treatment, and randomly assigned 2:1 by interactive response technology to receive fedratinib 400 mg per day (4 × 100 mg capsules orally once daily, open-label) or BAT. Patients received prophylactic antiemetics and thiamine supplementation, and symptomatic antidiarrhoeals as required. Primary endpoint was proportion of patients reaching spleen volume reduction (SVR) of at least 35% (SVR35) at end of cycle 6 in the intention-to-treat population. This manuscript reports the primary analysis of the trial; follow-up is ongoing. This trial is registered at clinicaltrials.gov, NCT03952039.FINDINGSBetween Sept 9, 2019 and June 24, 2022, of 316 patients screened, 201 were randomly assigned and treated (134 to fedratinib, 67 to BAT [including 52 receiving ruxolitinib]); 46 patients from the BAT group crossed over to fedratinib. Approximately half of enrolled patients were male (fedratinib 75 [56%] of 134; BAT 30 [45%] of 67) and most were White (fedratinib 106 [79%] of 134; BAT 58 [87%] of 67). At data cutoff (Dec 27, 2022), median survival follow-up was 64·5 weeks (IQR 37·9-104·9). SVR35 at end of cycle 6 was seen in 48 (36%) of 134 patients receiving fedratinib versus four (6%) of 67 patients receiving BAT (30% difference; 95% CI 20-39; one-sided p-value <0·0001). During the first six cycles 53 (40%) of 134 patients in the fedratinib group and 8 (12%) of 67 patients in the BAT group had grade 3 or greater treatment-related adverse events, most frequently anaemia (fedratinib 12 [9%] of 134; BAT 6 [9%] of 67) and thrombocytopenia (fedratinib 16 [12%] of 134; BAT 2 [3%] of 67); one patient in the fedratinib group died from acute kidney injury suspected to be related to study drug (no treatment-related deaths in the BAT group). Gastrointestinal adverse events occurred more frequently in the fedratinib group compared with the BAT group, but were mostly grade 1-2 in severity and more frequent in early cycles, and were less frequent than in prior clinical trials. A total of 28 (21%) of 134 patients in the fedratinib group and 3 (4%) of 67 patients in the BAT group had thiamine levels below lower limit of normal per central laboratory assessment, with only one case of low thiamine in the fedratinib arm after the intro
{"title":"Efficacy and safety of fedratinib in patients with myelofibrosis previously treated with ruxolitinib (FREEDOM2): results from a multicentre, open-label, randomised, controlled, phase 3 trial.","authors":"Claire N Harrison,Ruben Mesa,Moshe Talpaz,Haifa Kathrin Al-Ali,Blanca Xicoy,Francesco Passamonti,Francesca Palandri,Giulia Benevolo,Alessandro Maria Vannucchi,Clemence Mediavilla,Alessandra Iurlo,InHo Kim,Shelonitda Rose,Patrick Brown,Christopher Hernandez,Jia Wang,Jean-Jacques Kiladjian","doi":"10.1016/s2352-3026(24)00212-6","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00212-6","url":null,"abstract":"BACKGROUNDMost patients with myelofibrosis develop ruxolitinib intolerance or disease that is relapsed or refractory, and survival rates after ruxolitinib discontinuation are poor. We aimed to evaluate the safety and efficacy of fedratinib versus best available therapy (BAT) in patients with myelofibrosis previously treated with ruxolitinib.METHODSFREEDOM2 was a multicentre, open-label, randomised, controlled, phase 3 trial in 86 clinics in 16 countries, in which patients aged at least 18 years with intermediate-2 or high-risk myelofibrosis that was relapsed or refractory or intolerant to ruxolitinib with Eastern Cooperative Oncology Group performance status 0-2 were stratified by spleen size by palpation, platelet count, and previous ruxolitinib treatment, and randomly assigned 2:1 by interactive response technology to receive fedratinib 400 mg per day (4 × 100 mg capsules orally once daily, open-label) or BAT. Patients received prophylactic antiemetics and thiamine supplementation, and symptomatic antidiarrhoeals as required. Primary endpoint was proportion of patients reaching spleen volume reduction (SVR) of at least 35% (SVR35) at end of cycle 6 in the intention-to-treat population. This manuscript reports the primary analysis of the trial; follow-up is ongoing. This trial is registered at clinicaltrials.gov, NCT03952039.FINDINGSBetween Sept 9, 2019 and June 24, 2022, of 316 patients screened, 201 were randomly assigned and treated (134 to fedratinib, 67 to BAT [including 52 receiving ruxolitinib]); 46 patients from the BAT group crossed over to fedratinib. Approximately half of enrolled patients were male (fedratinib 75 [56%] of 134; BAT 30 [45%] of 67) and most were White (fedratinib 106 [79%] of 134; BAT 58 [87%] of 67). At data cutoff (Dec 27, 2022), median survival follow-up was 64·5 weeks (IQR 37·9-104·9). SVR35 at end of cycle 6 was seen in 48 (36%) of 134 patients receiving fedratinib versus four (6%) of 67 patients receiving BAT (30% difference; 95% CI 20-39; one-sided p-value <0·0001). During the first six cycles 53 (40%) of 134 patients in the fedratinib group and 8 (12%) of 67 patients in the BAT group had grade 3 or greater treatment-related adverse events, most frequently anaemia (fedratinib 12 [9%] of 134; BAT 6 [9%] of 67) and thrombocytopenia (fedratinib 16 [12%] of 134; BAT 2 [3%] of 67); one patient in the fedratinib group died from acute kidney injury suspected to be related to study drug (no treatment-related deaths in the BAT group). Gastrointestinal adverse events occurred more frequently in the fedratinib group compared with the BAT group, but were mostly grade 1-2 in severity and more frequent in early cycles, and were less frequent than in prior clinical trials. A total of 28 (21%) of 134 patients in the fedratinib group and 3 (4%) of 67 patients in the BAT group had thiamine levels below lower limit of normal per central laboratory assessment, with only one case of low thiamine in the fedratinib arm after the intro","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"8 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142231512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-06DOI: 10.1016/s2352-3026(24)00249-7
Gordon Cook,A John Ashcroft,Ethan Senior,Catherine Olivier,Anna Hockaday,Jeanine Richards,Jamie D Cavenagh,John A Snowden,Mark T Drayson,Ruth de Tute,Lesley Roberts,Roger G Owen,Kwee Yong,Mamta Garg,Kevin Boyd,Hamdi Sati,Sharon Gillson,Mark Cook,David A Cairns,Christopher Parrish,
BACKGROUNDThe efficacy of consolidation and maintenance in the context of salvage autologous haematopoietic stem-cell transplantation (HSCT) for relapsed multiple myeloma remains unclear. We aimed to assess whether consolidation after salvage autologous HSCT, using ixazomib, thalidomide, and dexamethasone, followed by maintenance with single agent ixazomib is superior to observation.METHODSThis is an interim analysis of Myeloma XII (ACCorD; referred to as ACCorD hereafter), an open-label, randomised, controlled, phase 3 trial done at 79 hospitals in the UK. Eligible patients were aged 18 years or older, had relapsed multiple myeloma with measurable disease, an ECOG performance status of 2 or less with adequate renal, hepatobiliary, pulmonary, and cardiac function, and required treatment for first progressive disease occurring at least 12 months after first autologous HSCT. In a first randomisation, patients were assigned (1:1) to receive either conventional autologous HSCT with melphalan or augmented autologous HSCT with melphalan and ixazomib. In the second randomisation, reported here, patients were assigned (1:1) to consolidation using ixazomib, thalidomide, and dexamethasone (oral ixazomib 4 mg per day on days 1, 8, and 15, oral thalidomide 100 mg per day on days 1-28, and oral dexamethasone 40 mg per day on days 1, 8, 15 and 22 of 28-day cycles), followed by maintenance with single agent ixazomib (oral ixazomib 4 mg per day on days 1, 8, and 15 of 28-day cycles until disease progression or intolerance), or observation. The primary endpoint was progression-free survival, analysed by intention-to-treat. Safety was analysed per-protocol. This study is registered with ISRCTN, ISRCTN10038996, and EudraCT, 2016-000905-35, and recruitment is complete.FINDINGSBetween Dec 12, 2017, and April 21, 2023, 206 patients entered the second randomisation (103 in the consolidation and maintenance group and 103 in the observation group). This prespecified interim analysis (data cutoff April 21, 2023), was done at a median follow-up of 27 months (IQR 13-38). Median progression-free survival was 20 months (95% CI 15-29) in the consolidation and maintenance group and 13 months (11-18) in the observation group (hazard ratio 0·55 [95% CI 0·39-0·78]; p=0·0006). Serious adverse events were reported in 29 (32%) of 92 patients in the consolidation and maintenance group compared with seven (7%) of 103 patients in the observation group. The most common serious adverse events were infections and infestations in both the consolidation and maintenance group and the observation group. The most common grade 3, 4, or 5 adverse events for patients in the consolidation and maintenance group were upper respiratory infection (seven [8%] of 92 patients). No deaths in the consolidation and maintenance group were deemed treatment related.INTERPRETATIONACCorD provides evidence that an orally administered, deliverable, and tolerable post-salvage autologous HSCT treatment regimen can im
{"title":"Ixazomib as consolidation and maintenance versus observation in patients with relapsed multiple myeloma eligible for salvage autologous stem-cell transplantation (Myeloma XII [ACCoRD]): interim analysis of a multicentre, open-label, randomised, phase 3 trial.","authors":"Gordon Cook,A John Ashcroft,Ethan Senior,Catherine Olivier,Anna Hockaday,Jeanine Richards,Jamie D Cavenagh,John A Snowden,Mark T Drayson,Ruth de Tute,Lesley Roberts,Roger G Owen,Kwee Yong,Mamta Garg,Kevin Boyd,Hamdi Sati,Sharon Gillson,Mark Cook,David A Cairns,Christopher Parrish,","doi":"10.1016/s2352-3026(24)00249-7","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00249-7","url":null,"abstract":"BACKGROUNDThe efficacy of consolidation and maintenance in the context of salvage autologous haematopoietic stem-cell transplantation (HSCT) for relapsed multiple myeloma remains unclear. We aimed to assess whether consolidation after salvage autologous HSCT, using ixazomib, thalidomide, and dexamethasone, followed by maintenance with single agent ixazomib is superior to observation.METHODSThis is an interim analysis of Myeloma XII (ACCorD; referred to as ACCorD hereafter), an open-label, randomised, controlled, phase 3 trial done at 79 hospitals in the UK. Eligible patients were aged 18 years or older, had relapsed multiple myeloma with measurable disease, an ECOG performance status of 2 or less with adequate renal, hepatobiliary, pulmonary, and cardiac function, and required treatment for first progressive disease occurring at least 12 months after first autologous HSCT. In a first randomisation, patients were assigned (1:1) to receive either conventional autologous HSCT with melphalan or augmented autologous HSCT with melphalan and ixazomib. In the second randomisation, reported here, patients were assigned (1:1) to consolidation using ixazomib, thalidomide, and dexamethasone (oral ixazomib 4 mg per day on days 1, 8, and 15, oral thalidomide 100 mg per day on days 1-28, and oral dexamethasone 40 mg per day on days 1, 8, 15 and 22 of 28-day cycles), followed by maintenance with single agent ixazomib (oral ixazomib 4 mg per day on days 1, 8, and 15 of 28-day cycles until disease progression or intolerance), or observation. The primary endpoint was progression-free survival, analysed by intention-to-treat. Safety was analysed per-protocol. This study is registered with ISRCTN, ISRCTN10038996, and EudraCT, 2016-000905-35, and recruitment is complete.FINDINGSBetween Dec 12, 2017, and April 21, 2023, 206 patients entered the second randomisation (103 in the consolidation and maintenance group and 103 in the observation group). This prespecified interim analysis (data cutoff April 21, 2023), was done at a median follow-up of 27 months (IQR 13-38). Median progression-free survival was 20 months (95% CI 15-29) in the consolidation and maintenance group and 13 months (11-18) in the observation group (hazard ratio 0·55 [95% CI 0·39-0·78]; p=0·0006). Serious adverse events were reported in 29 (32%) of 92 patients in the consolidation and maintenance group compared with seven (7%) of 103 patients in the observation group. The most common serious adverse events were infections and infestations in both the consolidation and maintenance group and the observation group. The most common grade 3, 4, or 5 adverse events for patients in the consolidation and maintenance group were upper respiratory infection (seven [8%] of 92 patients). No deaths in the consolidation and maintenance group were deemed treatment related.INTERPRETATIONACCorD provides evidence that an orally administered, deliverable, and tolerable post-salvage autologous HSCT treatment regimen can im","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"382 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142165848","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-09-06DOI: 10.1016/s2352-3026(24)00277-1
Elias K Mai
{"title":"Continued need for autologous transplantation in relapsed myeloma.","authors":"Elias K Mai","doi":"10.1016/s2352-3026(24)00277-1","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00277-1","url":null,"abstract":"","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"15 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142165904","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-05-01DOI: 10.1016/s2352-3026(24)00105-4
Mohammad K Khan, T. Nasti, Joshua Y Qian, T. Kleber, Jeffrey M Switchenko, J. Kaufman, Ajay J Nooka, M. Dhodapkar, Z. Buchwald, Daby Obiekwe, S. Lonial, Rafi Ahmed
{"title":"Pembrolizumab and low-dose, single-fraction radiotherapy for patients with relapsed or refractory multiple myeloma: a prospective, single-centre, single-group, open-label, phase 2 pilot trial in the USA","authors":"Mohammad K Khan, T. Nasti, Joshua Y Qian, T. Kleber, Jeffrey M Switchenko, J. Kaufman, Ajay J Nooka, M. Dhodapkar, Z. Buchwald, Daby Obiekwe, S. Lonial, Rafi Ahmed","doi":"10.1016/s2352-3026(24)00105-4","DOIUrl":"https://doi.org/10.1016/s2352-3026(24)00105-4","url":null,"abstract":"","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"43 s7","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141141311","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-02-05DOI: 10.1016/s2352-3026(23)00377-0
Hanny Al-Samkari, Nadine Shehata, Kelly Lang-Robertson, Paola Bianchi, Andreas Glenthøj, Sujit Sheth, Ellis J Neufeld, David C Rees, Satheesh Chonat, Kevin H M Kuo, Jennifer A Rothman, Wilma Barcellini, Eduard J van Beers, Dagmar Pospíšilová, Ami J Shah, Richard van Wijk, Bertil Glader, Maria Del Mar Mañú Pereira, Oliver Andres, Theodosia A Kalfa, Rachael F Grace
Pyruvate kinase (PK) deficiency is the most common cause of chronic congenital non-spherocytic haemolytic anaemia worldwide, with an estimated prevalence of one in 100 000 to one in 300 000 people. PK deficiency results in chronic haemolytic anaemia, with wide ranging and serious consequences affecting health, quality of life, and mortality. The goal of the International Guidelines for the Diagnosis and Management of Pyruvate Kinase Deficiency was to develop evidence-based guidelines for the clinical care of patients with PK deficiency. These clinical guidelines were developed by use of GRADE methodology and the AGREE II framework. Experts were invited after consideration of area of expertise, scholarly contributions in PK deficiency, and country of practice for global representation. The expert panel included 29 expert physicians (including adult and paediatric haematologists and other subspecialists), geneticists, laboratory specialists, nurses, a guidelines methodologist, patients with PK deficiency, and caregivers from ten countries. Five key topic areas were identified, the panel prioritised key questions, and a systematic literature search was done to generate evidence summaries that were used in the development of draft recommendations. The expert panel then met in person to finalise and vote on recommendations according to a structured consensus procedure. Agreement of greater than or equal to 67% among the expert panel was required for inclusion of a recommendation in the final guideline. The expert panel agreed on 31 total recommendations across five key topics: diagnosis and genetics, monitoring and management of chronic complications, standard management of anaemia, targeted and advanced therapies, and special populations. These new guidelines should facilitate best practices and evidence-based PK deficiency care into clinical practice.
{"title":"Diagnosis and management of pyruvate kinase deficiency: international expert guidelines","authors":"Hanny Al-Samkari, Nadine Shehata, Kelly Lang-Robertson, Paola Bianchi, Andreas Glenthøj, Sujit Sheth, Ellis J Neufeld, David C Rees, Satheesh Chonat, Kevin H M Kuo, Jennifer A Rothman, Wilma Barcellini, Eduard J van Beers, Dagmar Pospíšilová, Ami J Shah, Richard van Wijk, Bertil Glader, Maria Del Mar Mañú Pereira, Oliver Andres, Theodosia A Kalfa, Rachael F Grace","doi":"10.1016/s2352-3026(23)00377-0","DOIUrl":"https://doi.org/10.1016/s2352-3026(23)00377-0","url":null,"abstract":"<p><span>Pyruvate kinase (PK) deficiency is the most common cause of chronic congenital non-spherocytic haemolytic anaemia worldwide, with an estimated prevalence of one in 100 000 to one in 300 000 people. PK deficiency results in chronic haemolytic anaemia, with wide ranging and serious consequences affecting health, </span>quality of life<span>, and mortality. The goal of the International Guidelines for the Diagnosis and Management of Pyruvate Kinase Deficiency was to develop evidence-based guidelines for the clinical care of patients with PK deficiency. These clinical guidelines were developed by use of GRADE methodology and the AGREE II framework. Experts were invited after consideration of area of expertise, scholarly contributions in PK deficiency, and country of practice for global representation. The expert panel included 29 expert physicians (including adult and paediatric haematologists and other subspecialists), geneticists, laboratory specialists, nurses, a guidelines methodologist, patients with PK deficiency, and caregivers from ten countries. Five key topic areas were identified, the panel prioritised key questions, and a systematic literature search was done to generate evidence summaries that were used in the development of draft recommendations. The expert panel then met in person to finalise and vote on recommendations according to a structured consensus procedure. Agreement of greater than or equal to 67% among the expert panel was required for inclusion of a recommendation in the final guideline. The expert panel agreed on 31 total recommendations across five key topics: diagnosis and genetics, monitoring and management of chronic complications, standard management of anaemia, targeted and advanced therapies, and special populations. These new guidelines should facilitate best practices and evidence-based PK deficiency care into clinical practice.</span></p>","PeriodicalId":501011,"journal":{"name":"The Lancet Haematology","volume":"23 6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139695694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}