journal
https://read.qxmd.com/read/38697732/clotting-and-bleeding-in-beh%C3%A3-et-s-syndrome
#41
JOURNAL ARTICLE
Jagdish R Nair, Zoe J Syrimi, Caroline V Cotton, Robert J Moots
No abstract text is available yet for this article.
May 2024: Lancet Haematology
https://read.qxmd.com/read/38697731/diagnosis-treatment-and-surveillance-of-diamond-blackfan-anaemia-syndrome-international-consensus-statement
#42
REVIEW
Marcin W Wlodarski, Adrianna Vlachos, Jason E Farrar, Lydie M Da Costa, Antonis Kattamis, Irma Dianzani, Cristina Belendez, Sule Unal, Hannah Tamary, Ramune Pasauliene, Dagmar Pospisilova, Josu de la Fuente, Deena Iskander, Lawrence Wolfe, Johnson M Liu, Akiko Shimamura, Katarzyna Albrecht, Birgitte Lausen, Anne Grete Bechensteen, Ulf Tedgard, Alexander Puzik, Paola Quarello, Ugo Ramenghi, Marije Bartels, Heinz Hengartner, Roula A Farah, Mahasen Al Saleh, Amir Ali Hamidieh, Wan Yang, Etsuro Ito, Hoon Kook, Galina Ovsyannikova, Leo Kager, Pierre-Emmanuel Gleizes, Jean-Hugues Dalle, Brigitte Strahm, Charlotte M Niemeyer, Jeffrey M Lipton, Thierry M Leblanc
Diamond-Blackfan anaemia (DBA), first described over 80 years ago, is a congenital disorder of erythropoiesis with a predilection for birth defects and cancer. Despite scientific advances, this chronic, debilitating, and life-limiting disorder continues to cause a substantial physical, psychological, and financial toll on patients and their families. The highly complex medical needs of affected patients require specialised expertise and multidisciplinary care. However, gaps remain in effectively bridging scientific discoveries to clinical practice and disseminating the latest knowledge and best practices to providers...
May 2024: Lancet Haematology
https://read.qxmd.com/read/38697730/alexis-thompson-advancing-care-for-sickle-cell-disease
#43
JOURNAL ARTICLE
Udani Samarasekera
No abstract text is available yet for this article.
May 2024: Lancet Haematology
https://read.qxmd.com/read/38697729/diversity-equity-and-inclusion-in-ash-guidelines-authors-reply
#44
LETTER
Jeremy W Jacobs, Garrett S Booth, Julie K Silver
No abstract text is available yet for this article.
May 2024: Lancet Haematology
https://read.qxmd.com/read/38697728/diversity-equity-and-inclusion-in-ash-guidelines
#45
LETTER
Matthew D Seftel, Deirdra Terrell, Adam Cuker, Matthew Cheung, Menaka Pai
No abstract text is available yet for this article.
May 2024: Lancet Haematology
https://read.qxmd.com/read/38697727/universal-health-coverage-for-children-with-cancer
#46
EDITORIAL
The Lancet Haematology
No abstract text is available yet for this article.
May 2024: Lancet Haematology
https://read.qxmd.com/read/38604205/methodological-challenges-in-the-development-of-endpoints-for-myelofibrosis-clinical-trials
#47
REVIEW
Giovanni Barosi, Ayalew Tefferi, Naseema Gangat, Natasha Szuber, Alessandro Rambaldi, Olatoyosi Odenike, Nicolaus Kröger, Nico Gagelmann, Moshe Talpaz, Hagop Kantarjian, Robert Peter Gale
Myelofibrosis is a myeloid neoplasm characterised by the presence of JAK2, CALR, or MPL mutations (with a 90% mutation frequency) and trilineage myeloid proliferation with prominent megakaryocyte atypia. People with myelofibrosis have a lower survival rate and poorer quality of life than healthy individuals. Therapy for myelofibrosis uses Janus kinase inhibitors, which reduce splenomegaly and alleviate symptoms. Regulatory approvals for Janus kinase inhibitors have focused on this dual endpoint. In this Viewpoint, we discuss the validity of using spleen reduction as a surrogate endpoint for the disease-modifying activity of candidate drugs for myelofibrosis...
May 2024: Lancet Haematology
https://read.qxmd.com/read/38555923/anti-cd30-car-t-cells-as-consolidation-after-autologous-haematopoietic-stem-cell-transplantation-in-patients-with-high-risk-cd30-lymphoma-a-phase-1-study
#48
MULTICENTER STUDY
Natalie S Grover, George Hucks, Marcie L Riches, Anastasia Ivanova, Dominic T Moore, Thomas C Shea, Mary Beth Seegars, Paul M Armistead, Kimberly A Kasow, Anne W Beaven, Christopher Dittus, James M Coghill, Katarzyna J Jamieson, Benjamin G Vincent, William A Wood, Catherine Cheng, Julia Kaitlin Morrison, John West, Tammy Cavallo, Gianpietro Dotti, Jonathan S Serody, Barbara Savoldo
BACKGROUND: Chimeric antigen receptor (CAR) T cells targeting CD30 are safe and have promising activity when preceded by lymphodepleting chemotherapy. We aimed to determine the safety of anti-CD30 CAR T cells as consolidation after autologous haematopoietic stem-cell transplantation (HSCT) in patients with CD30+ lymphoma at high risk of relapse. METHODS: This phase 1 dose-escalation study was performed at two sites in the USA. Patients aged 3 years and older, with classical Hodgkin lymphoma or non-Hodgkin lymphoma with CD30+ disease documented by immunohistochemistry, and a Karnofsky performance score of more than 60% planned for autologous HSCT were eligible if they were considered high risk for relapse as defined by primary refractory disease or relapse within 12 months of initial therapy or extranodal involvement at the start of pre-transplantation salvage therapy...
May 2024: Lancet Haematology
https://read.qxmd.com/read/38552650/improving-quality-of-blood-transfusion-practices-in-nigeria
#49
JOURNAL ARTICLE
Dalhat Gwarzo, Kathleen Selleng, Halima Ismail, Kabiru Sulaiman, Sophie Reichelt, Renate Asare, Andreas Greinacher, Aisha Kuliya-Gwarzo
No abstract text is available yet for this article.
April 2024: Lancet Haematology
https://read.qxmd.com/read/38552649/be-compliant-or-compromise
#50
JOURNAL ARTICLE
May Anne Cheong
No abstract text is available yet for this article.
April 2024: Lancet Haematology
https://read.qxmd.com/read/38548404/reduced-dose-azacitidine-plus-venetoclax-as-maintenance-therapy-in-acute-myeloid-leukaemia-following-intensive-or-low-intensity-induction-a-single-centre-single-arm-phase-2-trial
#51
JOURNAL ARTICLE
Alexandre Bazinet, Hagop Kantarjian, Alex Bataller, Naveen Pemmaraju, Gautam Borthakur, Kelly Chien, Yesid Alvarado, Prithviraj Bose, Elias Jabbour, Musa Yilmaz, Courtney DiNardo, Ghayas Issa, Guillermo Montalban-Bravo, Nicholas Short, Koji Sasaki, Debra Bull-Linderman, Naval Daver, Guillermo Garcia-Manero, Farhad Ravandi, Tapan Kadia
BACKGROUND: Patients with acute myeloid leukaemia have high rates of relapse, especially if they are unable to complete standard consolidation strategies or allogeneic haematopoietic stem-cell transplantation (HSCT). The phase 3 QUAZAR AML-001 study showed an overall survival benefit with oral azacitidine maintenance. The BCL2 inhibitor venetoclax is highly active in acute myeloid leukaemia and synergistic with azacitidine. We aimed to evaluate the efficacy and safety of low dose azacitidine plus venetoclax as maintenance therapy in acute myeloid leukaemia...
April 2024: Lancet Haematology
https://read.qxmd.com/read/38548403/time-to-define-and-refine-maintenance-strategies-in-acute-myeloid-leukaemia
#52
JOURNAL ARTICLE
Chong Chyn Chua, Andrés Gómez-De León
No abstract text is available yet for this article.
April 2024: Lancet Haematology
https://read.qxmd.com/read/38548402/blood-deserts-a-vision-to-tackle-blood-inaccessibility
#53
EDITORIAL
The Lancet Haematology
No abstract text is available yet for this article.
April 2024: Lancet Haematology
https://read.qxmd.com/read/38452788/oral-decitabine-and-cedazuridine-plus-venetoclax-for-older-or-unfit-patients-with-acute-myeloid-leukaemia-a-phase-2-study
#54
JOURNAL ARTICLE
Alexandre Bazinet, Guillermo Garcia-Manero, Nicholas Short, Yesid Alvarado, Alex Bataller, Tareq Abuasab, Rabiul Islam, Kathryn Montalbano, Ghayas Issa, Abhishek Maiti, Musa Yilmaz, Nitin Jain, Lucia Masarova, Steven Kornblau, Elias Jabbour, Guillermo Montalban-Bravo, Caitlin R Rausch, Sherry Pierce, Courtney D DiNardo, Tapan Kadia, Naval Daver, Marina Konopleva, Xuelin Huang, Hagop Kantarjian, Farhad Ravandi
BACKGROUND: Hypomethylating agents combined with venetoclax are effective regimens in patients with acute myeloid leukaemia who are ineligible for intensive chemotherapy. Decitabine and cedazuridine (ASTX727) is an oral formulation of decitabine that achieves equivalent area-under-curve exposure to intravenous decitabine. We performed a single centre phase 2 study to evaluate the efficacy and safety of ASTX727 plus venetoclax. METHODS: This study enrolled patients with newly diagnosed (frontline treatment group) acute myeloid leukaemia who were ineligible for intensive chemotherapy (aged ≥75 years, an Eastern Cooperative Oncology Group [ECOG] performance status of 2-3, or major comorbidities) or relapsed or refractory acute myeloid leukaemia...
April 2024: Lancet Haematology
https://read.qxmd.com/read/38437857/etranacogene-dezaparvovec-gene-therapy-for-haemophilia-b-hope-b-24-month-post-hoc-efficacy-and-safety-data-from-a-single-arm-multicentre-phase-3-trial
#55
MULTICENTER STUDY
Michiel Coppens, Steven W Pipe, Wolfgang Miesbach, Jan Astermark, Michael Recht, Paul van der Valk, Bruce Ewenstein, Karen Pinachyan, Nicholas Galante, Sandra Le Quellec, Paul E Monahan, Frank W G Leebeek
BACKGROUND: Etranacogene dezaparvovec, the first gene therapy approved for haemophilia B treatment, was shown to be superior to treatment with continuous prophylactic factor IX in terms of bleeding protection 18 months after gene therapy in a phase 3 trial. We report post-hoc 24-month efficacy and safety data from this trial to evaluate the longer-term effects of etranacogene dezaparvovec in individuals with haemophilia B. METHODS: The phase 3 HOPE-B trial enrolled males aged 18 years or older with inherited haemophilia B, classified as severe (plasma factor IX activity level <1%) or moderately severe (plasma factor IX activity level ≥1% and ≤2%), with a severe bleeding phenotype and who were on stable continuous factor IX prophylaxis...
April 2024: Lancet Haematology
https://read.qxmd.com/read/38490233/correction-to-lancet-haematol-2024-11-e216-27
#56
(no author information available yet)
No abstract text is available yet for this article.
March 12, 2024: Lancet Haematology
https://read.qxmd.com/read/38461850/sex-and-gender-reporting-advances-in-medicine
#57
EDITORIAL
The Lancet Haematology
No abstract text is available yet for this article.
March 7, 2024: Lancet Haematology
https://read.qxmd.com/read/38452789/fully-oral-regimen-with-decitabine-and-cedazuridine-plus-venetoclax-a-new-step-forward-for-older-or-unfit-patients-with-acute-myeloid-leukaemia
#58
JOURNAL ARTICLE
Anna Candoni
No abstract text is available yet for this article.
March 4, 2024: Lancet Haematology
https://read.qxmd.com/read/38437858/breaking-ground-in-haemophilia-b-gene-therapy-insights-from-the-hope-b-trial-and-beyond
#59
JOURNAL ARTICLE
Margareth C Ozelo
No abstract text is available yet for this article.
March 1, 2024: Lancet Haematology
https://read.qxmd.com/read/38428445/health-care-transition-services-for-sickle-cell-disease-in-brazil
#60
JOURNAL ARTICLE
Jane S Hankins, Clarisse Lobo, Josefina A P Braga, Tarun Aurora, Kelly Pimenta, Maria Stella Figueiredo, Ana A Baumann
No abstract text is available yet for this article.
March 2024: Lancet Haematology
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