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FDA IDE validation of multiple myeloma MRD test by flow cytometry Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-18 Dragan Jevremovic, Min Shi, Pedro Horna, Gregory E. Otteson, Michael M. Timm, Shannon A. Bennett, Linda B. Baughn, Patricia T. Greipp, Wilson I. Gonsalves, Prashant Kapoor, Morie A. Gertz, Moritz Binder, Francis K. Buadi, Angela Dispenzieri, Taxiarchis Kourelis, Eli Muchtar, Jiehao Zhou, S. Vincent Rajkumar, Shaji K. Kumar, Horatiu Olteanu
Two recent decisions by the Food and Drug Administration will likely significantly impact testing for multiple myeloma (MM) minimal residual disease (MRD). First, on April 12, 2024, the FDA's Oncologic Drugs Advisory Committee (ODAC) voted to approve the use of MRD as an end point for accelerated approval of new treatments for patients with MM.1 This was a result of near 10 years effort by multiple
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The importance of autologous stem cell transplantation in improving outcomes in newly diagnosed patients with multiple myeloma Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-18 Morie A. Gertz
CONFLICT OF INTEREST STATEMENT The author declares no conflict of interest.
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Clearance of pathogenic erythrocytes is maintained despite spleen dysfunction in children with sickle cell disease Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-17 Abdoulaye Sissoko, Astan Cissé, Clémence Duverdier, Mickaël Marin, Lucie Dumas, Sandra Manceau, Blandine Maître, Anita Eckly, Aurélie Fricot‐Monsinjon, Camille Roussel, Papa Alioune Ndour, Michael Dussiot, Safi Dokmak, Béatrice Aussilhou, Jeanne Dembinski, Alain Sauvanet, François Paye, Mickaël Lesurtel, Jérôme Cros, Dominique Wendum, Magali Tichit, David Hardy, Carmen Capito, Slimane Allali, Pierre
In children with sickle cell disease (SCD), splenectomy is immediately beneficial for acute sequestration crises and hypersplenism (ASSC/HyS) but portends a long‐term risk of asplenia‐related complications. We retrieved peripheral and splenic red blood cells (RBCs) from 17 SCD children/teenagers undergoing partial splenectomy for ASSC/HyS, 12 adult subjects without RBC‐related disease undergoing splenectomy
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Comparison of prognostic scores according to WHO classification in 170 patients with advanced mastocytosis and C‐finding treated with midostaurin Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-17 Maël Heiblig, Clément Gourguechon, Philippe Guilpain, Cristina Bulai‐Livideanu, Stéphane Barete, Yannick Chantran, Julie Agopian, Fabienne Brenet, Patrice Dubreuil, Jérémie Lespinasse, Richard Lemal, Olivier Tournilhac, Louis Terriou, David Launay, Laurence Bouillet, Catharina Chatain, Ghandi Damaj, Thomas Ballul, Celine Greco, Laura Polivka, Laurent Frenzel, Cécile Meni, Hassiba Bouktit, Dina Benabou
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White blood cell count levels are associated with inflammatory response and constitute independent outcome predictors in adult patients with acute myeloid leukemia aged <60 years Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-16 Michael Ozga, Deedra Nicolet, Krzysztof Mrózek, Christopher J. Walker, James S. Blachly, Jessica Kohlschmidt, Shelley Orwick, Andrew J. Carroll, Richard A. Larson, Jonathan E. Kolitz, Bayard L. Powell, Richard M. Stone, John C. Byrd, Ann-Kathrin Eisfeld, Alice S. Mims
Acute myeloid leukemia (AML) is a biologically and clinically heterogenous disease with diverse genetic abnormalities1-6 and a wide-ranging white blood cell counts (WBC) at diagnosis.1 The 2022 European LeukemiaNet (ELN) genetic-risk classification incorporates cytogenetic and selected molecular alterations to define Favorable, Intermediate, and Adverse genetic-risk groups providing valuable prognostic
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The cognitive age in medicine: Artificial intelligence, large language models, and iterative intelligence Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-16 John Nosta
CONFLICT OF INTEREST STATEMENT The author declares no conflicts of interest.
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Thrombotic thrombocytopenic purpura masquerading as Evans syndrome Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-16 Sarah E. Mudra, Kaleb Ardoin, Vanya Aggarwal, Garrett Diltz, Pedro E. Alcedo Andrade, Catherine M. Broome
1 CASE PRESENTATION A 26-year-old female with a history of chronic urticaria (treated with omalizumab in the past) and recently treated chlamydia trachomatis infection presented to an outside emergency department after a syncopal episode. She endorsed a three-day history of nausea, vomiting, fatigue, and ecchymoses. Initial laboratory analysis revealed macrocytic anemia (hemoglobin 4.6 g/dL) with a
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Cybersecurity and the blood supply: The vulnerabilities of the technological revolution Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-12 Jeremy W. Jacobs, Nicole De Simone, Miriam Andrea Duque, Yanyun Wu, Dawn C. Ward, Jennifer S. Woo, Laura D. Stephens, Elizabeth S. Allen, Mandy F. O'Leary, Sheharyar Raza, Garrett S. Booth, Brian D. Adkins
CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.
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Real‐world efficacy and safety of luspatercept and predictive factors of response in patients with transfusion‐dependent β‐thalassemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-12 Daniele Lello Panzieri, Dario Consonni, Natalia Scaramellini, Guido Ausenda, Francesca Granata, Nancy Caponio, Lorena Duca, Simona Leoni, Silvia Elli, Marta Ferraresi, Vittorio Bolis, Cristina Curcio, Milena Agata Irrera, Diletta Maira, Giovanna Graziadei, Elena Cassinerio, Maria Domenica Cappellini, Rayan Bou‐Fakhredin, Valentina Brancaleoni, Irene Motta
Luspatercept is the first erythropoiesis-modulating agent approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for treating anemia in adult transfusion-dependent β-thalassemia (TDT) patients. As observed in clinical trials1 and real-life experience,2 response to luspatercept in TDT is heterogeneous. It can range from patients who do not respond to those who become
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Machine‐learning based subgroups of AL amyloidosis and cumulative incidence of mortality and end stage kidney disease Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-11 Shankara K. Anand, Andrew Staron, Lisa M. Mendelson, Tracy Joshi, Natasha Burke, Vaishali Sanchorawala, Ashish Verma
Immunoglobulin light chain (AL) amyloidosis is a multisystem disease with varied treatment options and disease‐related outcomes. Current staging systems rely on a limited number of cardiac, renal, and plasma cell dyscrasia biomarkers. To improve prognostication for all‐cause mortality and end‐stage kidney disease (ESKD), we applied unsupervised machine learning using a comprehensive set of clinical
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Impact of induction regimens intensity and allogeneic stem cell transplantation on survival of patients with Philadelphia chromosome‐positive acute lymphoblastic leukemia: A multi‐institutional study Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-10 Talha Badar, Ravi Narra, Alice S. Mims, Michael G. Heckman, Rory M. Shallis, Sheikh Fahad, Cameron Hunter, Vamsi Kota, Tamer Adel Othman, Brian Jonas, Shreya Desai, Guilherme Sacchi de Camargo Correia, Anand Patel, Adam S. DuVall, Neil Palmisiano, Emily Curran, Zulfa Omer, Anjali Advani, Ehab Atallah, Mark Litzow
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Quadruplets in newly diagnosed transplant‐ineligible multiple myeloma Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-10 Rajshekhar Chakraborty, Hira Mian
To the Editor: With the advent of proteasome inhibitors (PIs), immunomodulatory drugs (IMiDs), and anti-CD38 monoclonal antibodies (CD38mAbs), the outcomes of transplant-ineligible (TIE) patients with newly diagnosed multiple myeloma (MM) have substantially improved in the last decade.1-3 Currently, there are two widely accepted standard-of-care regimens in newly diagnosed TIE MM: daratumumab-lena
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Haploidentical hematopoietic cell transplantation as a platform for natural killer cell immunotherapy Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-09 Rémy Duléry, Sara Piccinelli, Mohmad Shahnawaz Beg, Ji Eun Jang, Rizwan Romee
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Hodgkin lymphoma: 2025 update on diagnosis, risk‐stratification, and management Am. J. Hematol. (IF 10.1) Pub Date : 2024-09-06 Stephen M. Ansell
Disease OverviewHodgkin lymphoma (HL) is an uncommon B‐cell lymphoid malignancy affecting 8570 new patients annually and representing ~10% of all lymphomas in the United States.DiagnosisHL is composed of two distinct disease entities: classical HL and nodular lymphocyte predominant HL (also called nodular lymphocyte predominant B‐cell lymphoma). Nodular sclerosis, mixed cellularity, lymphocyte depletion
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Higher survival following transplantation with a mismatched unrelated donor with posttransplant cyclophosphamide‐based graft‐versus‐host disease prophylaxis than with double unit umbilical cord blood in patients with acute myeloid leukemia in first complete remission: A study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-31 Frédéric Baron, Myriam Labopin, Jurjen Versluis, Jan Vydra, Peter A. von dem Borne, Emma Nicholson, Didier Blaise, Rachel Protheroe, Alexander Kulagin, Claude Eric Bulabois, Montserrat Rovira, Patrice Chevallier, Edouard Forcade, Jenny Byrne, Jaime Sanz, Annalisa Ruggeri, Mohamad Mohty, Fabio Ciceri
The best donor option for acute myeloid leukemia (AML) patients lacking an HLA‐matched donor has remained intensively debated. We herein report the results of a large retrospective registry study comparing hematopoietic cell transplantation (HCT) outcomes between double‐unit umbilical cord blood transplantation (dCBT, n = 209) versus 9/10 HLA‐matched unrelated donor (UD) with posttransplant cyclophosphamide
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Posttraumatic stress disorder increases thrombosis risk: Evidence from a biobank data set Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-29 Hui Chong Lau, Sinead M. Sinnott, Shady Abohashem, Giovanni Civieri, Wesam Aldosoky, Krystel Karam, Maria Khalil, Iqra Qamar, Rachel P. Rosovsky, Michael T. Osborne, Ahmed Tawakol, Antonia V. Seligowski
Depression and anxiety are linked to deep venous thrombosis (DVT) and posttraumatic disorder (PTSD) increases risk of venous thromboembolism in women. However, the mechanisms underlying this relationship remain unknown. We hypothesized that PTSD would associate with increased DVT risk, that neuroimmune mechanisms would mediate the PTSD‐DVT link, and that these associations would be stronger in women
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Long‐term outcomes of renal AL amyloidosis patients undergoing autologous stem cell transplantation: Validating the performance of the renal staging system Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-29 Eli Muchtar, Morie A. Gertz, Raphael Mwangi, Hamza Hassan, Angela Dispenzieri, Nelson Leung, Francis K Buadi, David Dingli, Andrew Staron, Vaishali Sanchorawala
Renal AL amyloidosis can be complicated by end‐stage renal disease (ESRD) requiring renal replacement therapy (RRT). In this study, we describe the long‐term outcomes of renal AL amyloidosis patients undergoing autologous stem cell transplantation (ASCT) and assess the utility of the renal staging system. Retrospective study of renal AL patients (n = 697; Mayo Clinic, Boston University) who underwent
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A phase 2 trial of mini‐hyper‐CVD, blinatumomab, and ponatinib in Philadelphia positive acute lymphoblastic leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-28 Wei‐Ying Jen, Elias Jabbour, Nicholas J. Short, Ghayas C. Issa, Fadi G. Haddad, Nitin Jain, Naveen Pemmaraju, Naval G. Daver, Lucia Masarova, Gautam Borthakur, Kelly Chien, Rebecca Garris, Hagop M. Kantarjian
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Oh node: Extranodal nodular involvement of chronic lymphocytic leukemia in the colon Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-28 Michael Keith Alister Zimmerman, Lindsay Wilde
A 64-year-old male patient with a known diagnosis of chronic lymphocytic leukemia (CLL) was referred to the gastroenterology department for a follow-up colonoscopy. A previous routine colonoscopy 4 years prior found benign polyps in the colon, and the patient was recommended to have a repeat colonoscopy within 3 years. In the interim, the patient had no new gastrointestinal symptoms. However, he was
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Clinical outcomes and microenvironment profiling in relapsed/refractory multiple myeloma patients with extramedullary disease receiving anti‐BCMA CAR T‐cell‐based therapy Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-28 Yuekun Qi, Hujun Li, Kunming Qi, Feng Zhu, Hai Cheng, Wei Chen, Zhiling Yan, Depeng Li, Wei Sang, Xiaoming Fei, Weiying Gu, Yuqing Miao, Hongming Huang, Ying Wang, Tingting Qiu, Jianlin Qiao, Bin Pan, Ming Shi, Gang Wang, Zhenyu Li, Junnian Zheng, Kailin Xu, Jiang Cao
Relapsed/refractory multiple myeloma patients with extramedullary disease (EMD) have unfavorable prognosis and lack effective therapy. Chimeric antigen receptor (CAR) T‐cell activities in EMD have yet to be determined; how EMD‐specific microenvironment influences the clinical outcomes of CAR T‐cell therapy remains of great interest. In this prospective cohort study, patients with histologically confirmed
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Presentation of chronic myeloid leukemia in basophilic blast crisis Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-23 Biswadip Hazarika, Barbara J. Bain
A 65-year-old man presented with fatigue. His spleen was just palpable below the left costal margin. His blood count showed hyperleukocytosis (WBC 242 × 109/L) with blast cells predominating, hemoglobin concentration (Hb) 64 g/L, and platelet count 22 × 109/L. Strikingly, the majority of blast cells had cytoplasm packed with large basophilic granules; others had more scanty granules so that the typical
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Successful 13-year ongoing remission with C5 inhibitor therapy following renal transplant in atypical hemolytic uremic syndrome Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-23 Mariam A. Mostafa, Martin Zand, Jeremy Taylor, Peter Kouides
Hemolytic uremic syndrome (HUS) is a thrombotic microangiopathy (TMA) characterized by nonimmune hemolytic anemia, thrombocytopenia, and renal impairment. Atypical HUS (aHUS) refers to cases of HUS that are caused by defects in the regulation of the alternative complement pathway and unlike other TMA are not associated with Shiga-toxin producing Escherichia coli (STEC) or ADAMTS13 deficiency. While
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Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-22 Austin Kulasekararaj, Francesco Lanza, Alexandros Arvanitakis, Saskia Langemeijer, Satheesh Chonat, Anil Tombak, Vladimir Hanes, Jia Cao, Mieke Jill Miller, Alexander Colbert, Benjamin Shander, Daniel T. Mytych, Vincent Chow, Haby Henary
ABP 959 is a biosimilar to the eculizumab reference product (RP), which is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). This multicenter, randomized, double‐blind, active‐controlled, two‐period crossover study randomized eculizumab RP‐treated patients with PNH to one of two treatment sequences (ABP 959/eculizumab RP or eculizumab RP/ABP 959) to evaluate the
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Global characteristics and outcomes of autologous hematopoietic stem cell transplantation for newly diagnosed multiple myeloma: A study of the worldwide network for blood and marrow transplantation (WBMT) Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-19 Laurent Garderet, Luuk Gras, Linda Koster, Laurien Baaij, Nada Hamad, Anita Dsouza, Noel Estrada-Merly, Parameswaran Hari, Wael Saber, Andrew J. Cowan, Minako Iida, Shinichiro Okamoto, Hiroyuki Takamatsu, Shohei Mizuno, Koji Kawamura, Yoshihisa Kodera, Bor-Sheng Ko, Christopher Liam, Kim Wah Ho, A. Sim Goh, S. Keat Tan, Alaa M. Elhaddad, Ali Bazarbachi, Qamar un Nisa Chaudhry, Rozan Alfar, Mohamed-Amine
Autologous hematopoietic cell transplantation (AHCT) is a commonly used treatment in multiple myeloma (MM). However, real-world global demographic and outcome data are scarce. We collected data on baseline characteristics and outcomes from 61 725 patients with newly diagnosed MM who underwent upfront AHCT between 2013 and 2017 from nine national/international registries. The primary endpoint was overall
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Secondary acquisition of the Philadelphia chromosome in acute lymphoblastic leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-17 Qiudan Shen, Elias J. Jabbour, Guilin Tang, Hong Fang, Wei Wang, Nicholas J. Short, M. James You, L. Jeffrey Medeiros, Shimin Hu
The Philadelphia chromosome (Ph), resulting from the t(9;22)(q34;q11), is the defining cytogenetic abnormality in chronic myeloid leukemia (CML).1 This chromosomal aberration harbors the BCR::ABL1 fusion gene, which encodes a constitutively active tyrosine kinase that initiates downstream signaling pathways crucial for leukemogenesis. Beyond CML, the Ph is also observed in other hematologic malignancies
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Serum iron and transferrin saturation variation are circadian regulated and linked to the harmonic circadian oscillations of erythropoiesis and hepatic Tfrc expression in mice Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-17 Cavan Bennett, Anne Pettikiriarachchi, Alistair R. D. McLean, Rebecca Harding, Marnie E. Blewitt, Cyril Seillet, Sant‐Rayn Pasricha
Serum iron has long been thought to exhibit diurnal variation and is subsequently considered an unreliable biomarker of systemic iron status. Circadian regulation (endogenous ~24‐h periodic oscillation of a biologic function) governs many critical physiologic processes. It is unknown whether serum iron levels are regulated by circadian machinery; likewise, the circadian nature of key players of iron
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Intra‐tumoral and peripheral blood TIGIT and PD‐1 as immune biomarkers in nodular lymphocyte predominant Hodgkin lymphoma Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-17 Jay Gunawardana, Soi C. Law, Muhammed B. Sabdia, Éanna Fennell, Aoife Hennessy, Ciara I. Leahy, Paul G. Murray, Karolina Bednarska, Sandra Brosda, Judith Trotman, Leanne Berkahn, Andreea Zaharia, Simone Birch, Melinda Burgess, Dipti Talaulikar, Justina N. Lee, Emily Jude, Eliza A. Hawkes, Sanjiv Jain, Karthik Nath, Cameron Snell, Fiona Swain, Joshua W. D. Tobin, Colm Keane, Mohamed Shanavas, Emily
In classical Hodgkin lymphoma (cHL), responsiveness to immune‐checkpoint blockade (ICB) is associated with specific tumor microenvironment (TME) and peripheral blood features. The role of ICB in nodular lymphocyte predominant Hodgkin lymphoma (NLPHL) is not established. To gain insights into its potential in NLPHL, we compared TME and peripheral blood signatures between HLs using an integrative multiomic
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Imatinib versus newer generation TKIs for upfront therapy in chronic phase chronic myeloid leukemia: What is the rationale for paying more to get the same survival benefit? Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-14 Jeffrey H. Lipton
To the Editor: In this issue of the American Journal of Hematology, there is an interesting analysis of a study involving an exciting, very effective newly marketed, targeted therapy for chronic myeloid leukemia (CML).1 The authors raise some interesting questions about utility and positioning of this drug in therapy. A quote from Warren Buffett, reputedly one of the 10 richest individuals in the world
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The seasonal distribution of immune thrombotic thrombocytopenic purpura is influenced by geography: Epidemiologic findings from a multi‐center analysis of 719 disease episodes Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-13 Jeremy W. Jacobs, Caroline G. Stanek, Garrett S. Booth, Argiris Symeonidis, Andrew W. Shih, Elizabeth S. Allen, Eleni Gavriilaki, Brenda J. Grossman, Katerina Pavenski, Amy Moorehead, Flora Peyvandi, Pasquale Agosti, Ilaria Mancini, Laura D. Stephens, Jay S. Raval, Maria Eva Mingot‐Castellano, Elizabeth P. Crowe, Laetitia Daou, Menaka Pai, Donald M. Arnold, Marisa B. Marques, Ryan Henrie, Tyler W.
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Exercise and training in sickle cell disease: Safety, potential benefits, and recommendations Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-12 Philippe Connes, Emeric Stauffer, Robert I. Liem, Elie Nader
Sickle cell disease (SCD) is a genetic disorder characterized by complex pathophysiological mechanisms leading to vaso-occlusive crisis, chronic pain, chronic hemolytic anemia, and vascular complications, which require considerations for exercise and physical activity. This review aims to elucidate the safety, potential benefits, and recommendations regarding exercise and training in individuals with
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Are veterans at increased risk of myeloproliferative neoplasms? Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-09 Christin B. DeStefano, Nicholas Burwick, Drew A. Helmer
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Self‐pay laboratory charges for iron deficiency diagnosis in the Boston and New Haven metropolitan areas Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-09 Layla Van Doren, Carlo Brugnara
The economic inequality of healthcare has been well documented. Out-of-pocket costs, which include laboratory costs, contribute to the financial burden that patients must bear for healthcare. This is particularly true for those patients living with a condition that requires frequent laboratory monitoring. Such is the case for those suffering from iron deficiency and iron deficiency anemia. Anemia accounts
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Second primary malignancies after tandem autologous hematopoietic stem cell transplantation for multiple myeloma Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-07 Samer Al Hadidi, Obada Ababneh, Carolina Schinke, Sharmilan Thanendrarajan, Clyde Bailey, Guido Tricot, John Shaughnessy, Fenghuang Zhan, Jeffrey Sawyer, Eric R. Siegel, Maurizio Zangari, Bart Barlogie, Frits van Rhee
Autologous hematopoietic stem cell transplantation (HSCT) is a standard treatment for eligible multiple myeloma (MM) patients.1 Before high-dose chemotherapy and autologous HSCT, patients typically receive induction therapy with a proteasome inhibitor and an immunomodulatory (IMiD) agent. This approach, along with posttransplant maintenance therapies, has improved survival, with some patients achieving
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2025 update on clinical trials in immune thrombocytopenia Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-06 Hanny Al‐Samkari
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Long-term quality of life after hematopoietic cell transplant for sickle cell disease in childhood: A STELLAR interim analysis Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-06 Staci D. Arnold, Nitya Bakshi, Diana Ross, Crystal Smith, Cynthia Sinha, Anirudh Veludhandi, Valerie Dutreuil, Shasha Bai, Lillian R. Meacham, Greg Guilcher, Monica Bhatia, Allistair Abraham, Kimberly A. Kasow, Ann Haight, Fuad El Rassi, Elizabeth Stenger, Joseph Lipscomb, Lakshmanan Krishnamurti
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Ferritin reference intervals in a population of working-age adults without anemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-05 Robert T. Means, Caixia Bi, Edward C. C. Wong, Lance A. Bare, Michael J. McPhaul
Iron deficiency anemia is recognized as one of the most important contributors to the worldwide burden of illness. It has a particularly high incidence in children and females of childbearing years in both the developed and less-developed world. It is generally recognized that serum ferritin concentration is the single most useful laboratory test in the diagnosis of iron deficiency.1 However, it has
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Timing and outcomes of second‐line therapy in the era of daratumumab‐based frontline therapy in AL amyloidosis Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-03 Abdul‐Hamid Bazarbachi, Divaya Bhutani, Jai Radhakrishnan, Markus Mapara, Mathew S. Maurer, Suzanne Lentzsch, Rajshekhar Chakraborty
The therapeutic goal in immunoglobulin light-chain amyloidosis (AL) is to eradicate the plasma cell (PC) clone producing misfolded amyloid fibrils. Therapies for AL often draw inspiration from successful treatments in multiple myeloma (MM). However, the field faced a challenge when second-generation proteasome inhibitors and immunomodulatory drugs (IMiDs) used for MM proved poorly tolerated by AL patients
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Chronic myeloid leukemia: 2025 update on diagnosis, therapy, and monitoring Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-02 Elias Jabbour, Hagop Kantarjian
Disease overviewChronic myeloid leukemia (CML) is a myeloproliferative neoplasm with an annual incidence of two cases/100 000. It accounts for approximately 15% of newly diagnosed cases of leukemia in adults.DiagnosisCML is characterized by a balanced genetic translocation, t(9;22) (q34;q11.2), involving a fusion of the Abelson murine leukemia (ABL1) gene from chromosome 9q34 with the breakpoint cluster
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Optimal frontline therapy of chronic myeloid leukemia today, and related musings Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-02 Hagop M. Kantarjian, Kebede Begna, Elias J. Jabbour, Shilpa Paul, Mary Alma Welch, Ayalew Tefferi
CONFLICT OF INTEREST STATEMENT H.K. received research grants from AbbVie, Amgen, Ascentage, BMS, Daiichi-Sankyo, Immunogen, Jazz, Novartis, Pfizer; and honoraria from AbbVie, Amgen, Aptitude Health, Ascentage, Astra Zeneca, KAHR Medical Ltd, Novartis, Pfizer. E.J. received research grants from Abbvie, adaptive biotechnologies, Amgen, Pfizer, Novartis, and Takeda; and consultancy fees from Abbvie, adaptive
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Echinocytes in pyruvate kinase deficiency, post‐splenectomy Am. J. Hematol. (IF 10.1) Pub Date : 2024-08-02 Sarah A. Bassiony, Asad Luqmani, Barbara J. Bain
A 19-year-old female patient with known pyruvate kinase deficiency was referred for evaluation of her suitability for initiation of therapy with mitapivat, a pyruvate kinase activator.1 At the age of 4 years she had been found to be homozygous for a PKLR mutation. At the age of 11 years she underwent a laparoscopic splenectomy and cholecystectomy. Despite the splenectomy, she remained transfusion dependent
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Where hematology meets rheumatology—VEXAS syndrome Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-30 Hajer Oun, William Gordon, Mike Leach, Barbara J. Bain
A 55-year-old man with a past history of gout treated with allopurinol and colchicine presented with symptoms of lethargy and arthralgia. His blood count showed a hemoglobin concentration of 97 g/L, mean cell volume of 100 fL, white cell count of 1.4 × 109 L, neutrophils of 0.5 × 109 L, and platelets of 232 × 109 L. His blood film showed poorly segmented neutrophils but unremarkable red cell morphology
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Isolated peripheral nervous system relapse after allogeneic hematopoietic cell transplantation for T-cell acute lymphoblastic leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-30 Mathijs Willemsen, Pieter Smeets, Aniek de Coninck
A 23-year-old man was admitted with left peripheral facial nerve palsy and right C7 radiculopathy. Ten weeks prior, he had received an allogeneic hematopoietic stem cell transplant (allo-HSCT) for presumed peripheral nervous system (PNS) and cerebrospinal fluid (CSF) flow cytometry-confirmed central nervous system (CNS) relapse of T-cell acute lymphoblastic leukemia (T-ALL). CNS MRI showed bilateral
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Mitapivat for phosphofructokinase deficiency Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-23 Hanny Al-Samkari
Pyruvate kinase activation is an emerging therapeutic modality under evaluation to treat congenital hemolytic anemias, including pyruvate kinase deficiency, thalassemia, and sickle cell disease, among others.1, 2 The results of a phase 3, randomized, placebo-controlled clinical trial of the pyruvate kinase activator mitapivat to treat the most common glycolytic enzyme defect, pyruvate kinase deficiency
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Network meta-analysis of upfront fixed-duration therapies in chronic lymphocytic leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-23 Stefano Molica, David Allsup, Diana Giannarelli
The emergence of Bruton's tyrosine kinase (BTK) inhibitors marked a significant advancement in chronic lymphocytic leukemia (CLL) management, especially for high-risk patients.1 Ibrutinib, the pioneering BTK inhibitor, has undergone extensive study. The final analysis of the RESONATE-2 study confirms its sustained survival benefit for first-line CLL treatment with up to 10 years of follow-up.2 Despite
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Advances in sickle cell retinopathy screening techniques, tests, and practices: A systematic review Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-23 Olivia W. Cummings, Sara Rahman, Lauren Fletcher, Adrienne W. Scott
Sickle cell retinopathy (SCR) is a progressive, sight‐threatening ophthalmic complication of sickle cell disease (SCD). Current SCR screening focuses on the detection of pathologic sea fan neovascularization, the first sign of proliferative sickle cell retinopathy (PSR). If untreated, PSR can lead to severe visual impairment and blindness through progression to vitreous hemorrhage and/or retinal detachment
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Disease burden, management strategies, and unmet needs in α‐thalassemia due to hemoglobin H disease Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-22 Ashutosh Lal, Vip Viprakasit, Elliott Vichinsky, Yongrong Lai, Meng‐Yao Lu, Antonis Kattamis
Alpha‐thalassemia is an inherited blood disorder caused by impaired α‐globin chain production, leading to anemia and other complications. Hemoglobin H (HbH) disease is caused by a combination of mutations generally affecting the expression of three of four α‐globin alleles; disease severity is highly heterogeneous, largely driven by genotype. Notably, non‐deletional mutations cause a greater degree
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A growing panoply of options for patients with paroxysmal nocturnal hemoglobinuria Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-18 David J. Young
In this issue, the COMMODORE 11 and COMMODORE 22 trials of crovalimab for previously treated and treatment-naïve paroxysmal nocturnal hemoglobinuria, respectively, are presented, demonstrating efficacy and safety for both patient populations in comparison with the benchmark, eculizumab. Paroxysmal nocturnal hemoglobinuria (PNH) is a hemolytic anemia characterized by sometimes painful intravascular
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Risk of myeloproliferative neoplasms among U.S. Veterans from Korean, Vietnam, and Persian Gulf War eras Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-18 Andrew Tiu, Zoe McKinnell, Shanshan Liu, Puneet Gill, Martha Antonio, Zoe Shancer, Nandan Srinivasa, Guoqing Diao, Ramesh Subrahmanyam, Craig M. Kessler, Maneesh Jain
The Promise to Address Comprehensive Toxics (PACT) Act expanded U.S. Veterans' health care and benefits for conditions linked to service-connected exposures (e.g., Burn Pits, Agent Orange). However, myeloproliferative neoplasms (MPN) are not recognized as presumptive conditions for Veterans exposed to these toxic substances. This study evaluated the development of MPN among U.S. Veterans from the Korean
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Optical genome mapping improves the accuracy of classification, risk stratification, and personalized treatment strategies for patients with acute myeloid leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-17 Sanam Loghavi, Qing Wei, Farhad Ravandi, Andres E. Quesada, Mark J. Routbort, Shimin Hu, Gokce A. Toruner, Sa A. Wang, Wei Wang, Roberto N. Miranda, Shaoying Li, Jie Xu, Courtney D. DiNardo, Naval Daver, Tapan M. Kadia, Ghayas C. Issa, Hagop M. Kantarjian, L. Jeffrey Medeiros, Guilin Tang
Cytogenomic characterization is crucial for the classification and risk stratification of acute myeloid leukemia (AML), thereby facilitating therapeutic decision-making. We examined the clinical utility of optical genome mapping (OGM) in 159 AML patients (103 newly diagnosed and 56 refractory/relapsed), all of whom also underwent chromosomal banding analysis (CBA), fluorescence in situ hybridization
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Frontline therapy of acute myeloid leukemia with lower intensity regimens: Where are we now and where can we go? Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-17 Jennifer Marvin-Peek, Jason S. Gilbert, Daniel A. Pollyea, Courtney D. DiNardo
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Correction to “Natural history, predictors of development of extramedullary disease, and treatment outcomes for patients with extramedullary multiple myeloma” Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-12
Zanwar S, Ho M, Lin Y, Kapoor P, Binder M, Buadi FK, Dispenzieri A, Dingli D, Fonder A, Gertz MA, Gonsalves W, Hayman SR, Hwa Y, Hobbs M, Kourelis T, Lacy MQ, Leung N, Muchtar E, Warsame R, Jevremovic D, Kyle RA, Rajkumar SV, Kumar S. Am J Hematol. 2023 Oct;98(10):1540–1549. doi: 10.1002/ajh.27023. Epub 2023 Jul 8. PMID: 37421603. In this article, we forgot to acknowledge that the work was supported
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Correction to “Risk factors for severe infection and mortality in patients with COVID-19 in patients with multiple myeloma and AL amyloidosis” Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-12
Ho M, Zanwar S, Buadi FK, Ailawadhi S, Larsen J, Bergsagel L, Binder M, Chanan-Khan A, Dingli D, Dispenzieri A, Fonseca R, Gertz MA, Gonsalves W, Go RS, Hayman S, Kapoor P, Kourelis T, Lacy MQ, Leung N, Lin Y, Muchtar E, Roy V, Sher T, Warsame R, Fonder A, Hobbs M, Hwa YL, Kyle RA, Rajkumar SV, Kumar S. Am J Hematol. 2023 Jan;98(1):49–55. doi: 10.1002/ajh.26762. Epub 2022 Oct 24. PMID: 36226510. In
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Safety and efficacy of baricitinib in steroid-resistant or relapsed immune thrombocytopenia: An open-label pilot study Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-09 Peng Zhao, Zhuo-Yu An, Hai-Xia Fu, Hui-Xin Liu, Cheng-Jie Feng, Qiu-Sha Huang, Jin Wu, Ye-Jun Wu, Li-Ping Yang, Qing-Yuan Qu, Yu-Xiu Chen, Meng-Lin Li, Chen-Cong Wang, Qi Chen, Xiao-Lu Zhu, Yun He, Yuan-Yuan Zhang, Qian Jiang, Hao Jiang, Jin Lu, Ying-Jun Chang, Xiao-Su Zhao, Xiang-Yu Zhao, Xiao-Jun Huang, Xiao-Hui Zhang
Patients with steroid-resistant or relapsed immune thrombocytopenia (ITP) suffer increased bleeding risk and impaired quality of life. Baricitinib, an oral Janus-associated kinases (JAK) inhibitor, could alleviate both innate and adaptive immune disorders without inducing thrombocytopenia in several autoimmune diseases. Accordingly, an open-label, single-arm, phase 2 trial (NCT05446831) was initiated
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Immune thrombocytopenia newly diagnosed during pregnancy: Outcome for mothers and neonates and comparison with chronic immune thrombocytopenia during pregnancy Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-09 Stéphanie Guillet, Valentine Loustau, Emmanuelle Boutin, Odile Souchaud-Debouverie, Nathalie Costedoat Chalumeau, Laurent Pascal, Laurent Gilardin, Louis Terriou, Julie Graveleau, Thibault Comont, Helene Henique, Quitterie Reynaud, Matthieu Mahévas, Marc Michel, Florence Canoui-Poitrine, Bertrand Godeau
The incidence of many autoimmune diseases is increased in women of childbearing age. Recent studies gave reassuring data for women with preexisting immune thrombocytopenia (ITP) who want to become pregnant.1, 2 In particular, we recently confirmed that pregnancy does not increase the risk of severe bleeding.2 Little is known about the outcome of ITP diagnosed during pregnancy, and conflicting results
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Evaluating the role of Day 14 bone marrow biopsy and European LeukemiaNet risk classification in predicting overall and relapse-free survival in acute myeloid leukemia Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-05 Metodi Balev, Victor Zibara, Grace Van Hyfte, Jonathan Feld, Marina Kremyanskaya, Michelle Becker, Alla Keyzner, Alan H. Shih, Bridget Marcellino, Hannah Levavi, Lewis Silverman, John Mascarenhas, Douglas Tremblay
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At what COST?: A prospective longitudinal cohort assessment of financial toxicity, quality of life, and associated risk factors in patients with plasma cell disorders Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-05 Rahma M. Warsame, Walker M. Schmidt, Angela Dispenzieri, Amylou C. Dueck
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Anxiety and depression are associated with heightened risk of incident deep vein thrombosis: Mediation through stress-related neural mechanisms Am. J. Hematol. (IF 10.1) Pub Date : 2024-07-04 Rachel P. Rosovsky, Kenechukwu Mezue, Charbel Gharios, Giovanni Civieri, Alexander Cardeiro, Hadil Zureigat, Hui Chong Lau, Roger K. Pitman, Lisa Shin, Shady Abohashem, Michael T. Osborne, Farouc A. Jaffer, Ahmed Tawakol