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The role of preoperative transfusion in sickle cell disease, a systematic review and meta-analysis Blood Rev. (IF 7.4) Pub Date : 2024-02-18 Yasamin Abdu, Alaa Rahhal, Khalid Ahmed, Nada Adli, Mariam Abdou, Elrazi Awadelkarim Hamid Ali, Salam Al-Kindi, Mona Al Rasheed, Jaffer Altooq, Iheb Bougmiza, Mohamed A Yassin
This systematic review and meta-analysis aimed to provide guidance on preoperative blood transfusion strategies for patients with sickle cell disease (SCD). We included all randomized controlled and observational studies exploring the clinical outcomes of preoperative blood transfusion among patients with SCD compared to the conservative transfusion strategy until 14/09/2022.
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Depression in patients with hematologic malignancies: The current landscape and future directions Blood Rev. (IF 7.4) Pub Date : 2024-02-13 Thomas M. Kuczmarski, Lizabeth Roemer, Oreofe O. Odejide
Patients with hematologic malignancies experience high rates of depression. These patients are vulnerable to depression throughout the disease trajectory, from diagnosis to survivorship, and at the end of life. In addition to the distressing nature of depression, it has substantial downstream effects including poor quality of life, increased risk of treatment complications, and worse survival. Therefore
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Quality of life, mood disorders, and cognitive impairment in adults with β-thalassemia Blood Rev. (IF 7.4) Pub Date : 2024-02-08 Maya Bizri, Rawan Koleilat, Nathalie Akiki, Reem Dergham, Alexandra Monica Mihailescu, Rayan Bou-Fakhredin, Khaled M. Musallam, Ali T. Taher
Advances in understanding the disease process in β-thalassemia supported development of various treatment strategies that resulted in improved survival. Improved survival, however, allowed multiple morbidities to manifest and cemented the need for frequent, lifelong treatment. This has directly impacted patients' health-related quality of life and opened the door for various psychiatric and cognitive
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Prevention and management of infectious complications in patients with chronic lymphocytic leukemia (CLL) treated with BTK and BCL-2 inhibitors, focus on current guidelines Blood Rev. (IF 7.4) Pub Date : 2024-02-02 Malgorzata Mikulska, Chiara Oltolini, Emanuela Zappulo, Michele Bartoletti, Anna Maria Frustaci, Andrea Visentin, Candida Vitale, Francesca R. Mauro
CLL is associated with an increased risk of infectious complications. Treatment with BTK or BCL-2 inhibitors does not seem to increase significantly the risk of opportunistic infections, but the role of combination therapies including BTK and/or BCL-2 inhibitors remains to be established. Various infectious complications can be successfully prevented with appropriate risk management strategies. In
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Update: The molecular spectrum of virus-associated high-grade B-cell non-Hodgkin lymphomas Blood Rev. (IF 7.4) Pub Date : 2024-01-20 H. Witte, A. Kuenstner, N. Gebauer
The vast spectrum of aggressive B-cell non-Hodgkin neoplasms (B-NHL) encompasses several infrequent entities occurring in association with viral infections, posing diagnostic challenges for practitioners. In the emerging era of precision oncology, the molecular characterization of malignancies has acquired paramount significance. The pathophysiological comprehension of specific entities and the identification
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When anticoagulation management in atrial fibrillation becomes difficult: Focus on chronic kidney disease, coagulation disorders, and cancer Blood Rev. (IF 7.4) Pub Date : 2024-01-14 Panteleimon E. Papakonstantinou, Vasiliki Kalogera, Dimitrios Charitos, Dimitrios Polyzos, Dimitra Benia, Athina Batsouli, Konstantinos Lampropoulos, Sotirios Xydonas, Dhiraj Gupta, Gregory Y.H. Lip
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Harnessing multi-source data for individualized care in Hodgkin Lymphoma Blood Rev. (IF 7.4) Pub Date : 2024-01-14 Susan K. Parsons, Angie Mae Rodday, Jenica N. Upshaw, Carlton D. Scharman, Zhu Cui, Yenong Cao, Yun Kyoung Tiger, Matthew J. Maurer, Andrew M. Evens
Hodgkin lymphoma is a rare, but highly curative form of cancer, primarily afflicting adolescents and young adults. Despite multiple seminal trials over the past twenty years, there is no single consensus-based treatment approach beyond use of multi-agency chemotherapy with curative intent. The use of radiation continues to be debated in early-stage disease, as part of combined modality treatment, as
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Disparity in hematological malignancies: From patients to health care professionals Blood Rev. (IF 7.4) Pub Date : 2024-01-08 Xiaoyi Chen, Mihir Shukla, Shella Saint Fleur-Lominy
In the recent few decades, outcomes in patients diagnosed with hematological malignancies have been steadily improving. However, the improved prognosis does not distribute equally among patients from different backgrounds. Besides cancer biology, demographic and geographic disparities have been found to impact overall survival significantly. Specifically, patients from underrepresented minorities including
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Immune deficiency/dysregulation -associated lymphoproliferative disorders. Revised classification and management Blood Rev. (IF 7.4) Pub Date : 2024-01-05 Antonino Carbone, Amy Chadburn, Annunziata Gloghini, Emanuela Vaccher, Mark Bower
Significant advances in the field of lymphoma have resulted in two recent classification proposals, the International Consensus Classification (ICC) and the 5th edition WHO. A few entities are categorized differently in the ICC compared to the WHO. Nowhere is this more apparent than the immunodeficiency lymphoproliferative disorders. The three previous versions of the WHO classification (3rd, 4th and
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Chromosomal defects in multiple myeloma Blood Rev. (IF 7.4) Pub Date : 2024-01-04 Sarah E. Clarke, Kathryn A. Fuller, Wendy N. Erber
Multiple myeloma is a plasma cell neoplasm driven by primary (e.g. hyperdiploidy; translocations) and secondary (e.g. 1q21 gains/amplifications; del(17p); translocations) chromosomal events. These are important to detect as they influence prognosis, therapeutic response and disease survival. Currently, cytogenetic testing is most commonly performed by interphase fluorescence in situ hybridisation (FISH)
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Αlpha-thalassemia: A practical overview Blood Rev. (IF 7.4) Pub Date : 2024-01-03 Khaled M. Musallam, M. Domenica Cappellini, Thomas D. Coates, Kevin H.M. Kuo, Hanny Al-Samkari, Sujit Sheth, Vip Viprakasit, Ali T. Taher
α-Thalassemia is an inherited blood disorder characterized by decreased synthesis of α-globin chains that results in an imbalance of α and β globin and thus varying degrees of ineffective erythropoiesis, decreased red blood cell (RBC) survival, chronic hemolytic anemia, and subsequent comorbidities. Clinical presentation varies depending on the genotype, ranging from a silent or mild carrier state
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Heartbreaker: Detection and prevention of cardiotoxicity in hematological malignancies Blood Rev. (IF 7.4) Pub Date : 2023-12-31 Azin Vakilpour, Bénédicte Lefebvre, Catherine Lai, Marielle Scherrer-Crosbie
Cancer survivors are at significant risk of cardiovascular (CV) morbidity and mortality; patients with hematologic malignancies have a higher rate of death due to heart failure compared to all other cancer subtypes. The majority of conventional hematologic cancer treatments is associated with increased risk of acute and long-term CV toxicity. The incidence of cancer therapy induced CV toxicity depends
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Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A Blood Rev. (IF 7.4) Pub Date : 2023-12-23 Hussien Ahmed H. Abdelgawad, Rachel Foster, Mario Otto
Hemophilia A, an X-linked genetic disorder, is characterized by a deficiency or dysfunction of clotting Factor VIII. The treatment landscape has substantially changed by introducing novel extended half-life factor VIII (EHL-FVIII) replacement therapies such as efanesoctocog Alfa and non-factor replacement therapy such as emicizumab. These agents signal a shift from treatments requiring multiple weekly
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Richter's transformation: Transforming the clinical landscape Blood Rev. (IF 7.4) Pub Date : 2023-12-10 A. Barrett, N. Appleby, H. Dreau, C.P. Fox, T. Munir, T.A. Eyre
Richter transformation (RT) represents an aggressive histological transformation from chronic lymphocytic leukaemia, most often to a large B cell lymphoma. It is characterised by chemo-resistance and subsequent short survival. Drug development has struggled over recent years in light of the aggressive kinetics of the disease, lack of pivotal registrational trials and relative rarity of the phenomenon
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Optimizing high dose melphalan Blood Rev. (IF 7.4) Pub Date : 2023-12-10 Gunjan Shah, Sergio Giralt, Parastoo Dahi
Melphalan, has been a major component of myeloma therapy since the 1950s. In the context of hematopoietic cell transplantation (HCT), high dose melphalan (HDM) is the most common conditioning regimen used due to its potent anti-myeloma effects and manageable toxicities. Common toxicities associated with HDM include myelosuppression, gastrointestinal issues, and mucositis. Established approaches to
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Idiopathic multicentric Castleman disease: An update in diagnosis and treatment advances Blood Rev. (IF 7.4) Pub Date : 2023-12-05 Evan Lang, Frits van Rhee
Idiopathic multicentric Castleman disease (iMCD) is a rare disease, and it is likely underdiagnosed because of the heterogeneity of clinical manifestations and laboratory findings. While the disease leads to significant morbidity and mortality, its causes are not yet fully elucidated. There have been significant advances in diagnosis and treatment of iMCD in the past decade, including the approval
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Corrigendum to “Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme” [Blood Rev. 2023 Sep:61:101103] Blood Rev. (IF 7.4) Pub Date : 2023-12-02 Myrthe J. van Dijk, Jonathan R.A. de Wilde, Marije Bartels, Kevin H.M. Kuo, Andreas Glenthøj, Minke A.E. Rab, Eduard J. van Beers, Richard van Wijk
Abstract not available
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Paroxysmal nocturnal hemoglobinuria: Review of the patient experience and treatment landscape Blood Rev. (IF 7.4) Pub Date : 2023-11-29 Anem Waheed, Jamile Shammo, David Dingli
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder caused by complement-mediated hemolysis and thrombosis through the alternative pathway. The most common symptom of PNH is fatigue due to chronic anemia, which can negatively impact quality of life (QoL) and affect overall well-being. The currently approved therapies for PNH significantly limit intravascular hemolysis (IVH) and reduce the
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Iron deficiency anemia among women: An issue of health equity Blood Rev. (IF 7.4) Pub Date : 2023-11-29 Grace H. Tang, Michelle Sholzberg
Iron deficiency is the most common and widespread nutritional deficiency in the world. For women, the risk of iron deficiency and iron deficiency anemia increases due to iron demands during pregnancy and regular iron losses due to menstruation during reproductive years. These interrelated conditions are of public health concern as they are highly prevalent, and the negative consequences such as chronic
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Contemporary tools for evaluation of hemostasis in neonates. Where are we and where are we headed? Blood Rev. (IF 7.4) Pub Date : 2023-11-25 Rozeta Sokou, Stavroula Parastatidou, Aikaterini Konstantinidi, Andreas G. Tsantes, Nicoletta Iacovidou, Daniele Piovani, Stefanos Bonovas, Argirios E. Tsantes
The assessment of hemostatic disorders in neonates is crucial, but remains challenging for clinicians. Although the concept of developmental hemostasis is widely accepted among hemostasis specialists globally, it is probably under-recognized by clinicians and laboratory practitioners. In parallel with age-dependent hemostatic status maturation, comprehension of the differences between normal values
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Practical considerations in clinical application of WHO 5th and ICC classification schemes for acute myeloid leukemia Blood Rev. (IF 7.4) Pub Date : 2023-11-25 Daniel J. Chandra, Curtis A. Lachowiez, Sanam Loghavi
The updated WHO 5th edition and ICC 2022 classification systems for AML aim to refine our diagnostic criteria and definitions of AML with deeper incorporation of cytogenetic and molecular aberrations. The two classification systems diverge, however, in numerous AML defining criteria and subclassifications, including the incorporation of blast enumeration and the integration of specific genomic mutations
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Down syndrome and leukemia: An insight into the disease biology and current treatment options Blood Rev. (IF 7.4) Pub Date : 2023-11-25 Sonali P. Barwe, E. Anders Kolb, Anilkumar Gopalakrishnapillai
Children with Down syndrome (DS) have a 10- to 20-fold greater predisposition to develop acute leukemia compared to the general population, with a skew towards myeloid leukemia (ML-DS). While ML-DS is known to be a subtype with good outcome, patients who relapse face a dismal prognosis. Acute lymphocytic leukemia in DS (DS-ALL) is considered to have poor prognosis. The relapse rate is high in DS-ALL
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Platelet factor 4(PF4) and its multiple roles in diseases Blood Rev. (IF 7.4) Pub Date : 2023-11-23 Zhiyan Liu, Longtu Li, Hanxu Zhang, Xiaocong Pang, Zhiwei Qiu, Qian Xiang, Yimin Cui
Platelet factor 4 (PF4) combines with heparin to form an antigen that could produce IgG antibodies and participate in heparin-induced thrombocytopenia (HIT). PF4 has attracted wide attention due to its role in novel coronavirus vaccine-19 (COVID-9)-induced immune thrombotic thrombocytopenia (VITT) and cognitive impairments. The electrostatic interaction between PF4 and negatively charged molecules
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Unraveling germline predisposition in hematological neoplasms: Navigating complexity in the genomic era Blood Rev. (IF 7.4) Pub Date : 2023-11-20 Joaquín Jerez, Marta Santiago
Genomic advancements have yielded pivotal insights into hematological neoplasms, particularly concerning germline predisposition mutations. Following the WHO 2016 revisions, dedicated segments were proposed to address these aspects. Current WHO 2022, ICC 2022, and ELN 2022 classifications recognize their significance, introducing more mutations and prompting integration into clinical practice.
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From microscope to micropixels: A rapid review of artificial intelligence for the peripheral blood film Blood Rev. (IF 7.4) Pub Date : 2023-11-19 Bingwen Eugene Fan, Bryan Song Jun Yong, Ruiqi Li, Samuel Sherng Young Wang, Min Yi Natalie Aw, Ming Fang Chia, David Tao Yi Chen, Yuan Shan Neo, Bruno Occhipinti, Ryan Ruiyang Ling, Kollengode Ramanathan, Yi Xiong Ong, Kian Guan Eric Lim, Wei Yong Kevin Wong, Shu Ping Lim, Siti Thuraiya Binte Abdul Latiff, Hemalatha Shanmugam, Moh Sim Wong, Kuperan Ponnudurai, Stefan Winkler
Artificial intelligence (AI) and its application in classification of blood cells in the peripheral blood film is an evolving field in haematology. We performed a rapid review of the literature on AI and peripheral blood films, evaluating the condition studied, image datasets, machine learning models, training set size, testing set size and accuracy. A total of 283 studies were identified, encompassing
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A personalized, organ-based approach to the treatment of chronic steroid-refractory graft-versus-host disease Blood Rev. (IF 7.4) Pub Date : 2023-11-11 Hanaa Fatoum, Robert Zeiser, Shahrukh K. Hashmi
Chronic graft-versus-host-disease (cGvHD) remains the leading cause of morbidity among transplant recipients. The efficacy of second-line treatments varies widely based on many factors, including wide differences in the organ overall response-rate response and in the current era where multiple agents are approved, and optimal sequencing of drugs based on organ ORR is unknown. We aimed to evaluate outcomes
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The many facets of immune-mediated thrombocytopenia: Principles of immunobiology and immunotherapy Blood Rev. (IF 7.4) Pub Date : 2023-11-11 Thomas Moulinet, Anthony Moussu, Ludovic Pierson, Simona Pagliuca
Immune thrombocytopenia (ITP) is a rare autoimmune condition, due to peripheral platelet destruction through antibody-dependent cellular phagocytosis, complement-dependent cytotoxicity, cytotoxic T lymphocyte-mediated cytotoxicity, and megakaryopoiesis alteration. This condition may be idiopathic or triggered by drugs, vaccines, infections, cancers, autoimmune disorders and systemic diseases. Recent
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A paradox of choice: Sequencing therapy in relapsed/refractory diffuse large B-cell lymphoma Blood Rev. (IF 7.4) Pub Date : 2023-10-28 Taylor R. Brooks, Paolo F. Caimi
The available treatments for relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) have experienced a dramatic change since 2017. Incremental advances in basic and translational science over several decades have led to innovations in immune-oncology. These innovations have culminated in eight separate approvals by the US Food and Drug Administration for the treatment of patients with R/R
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Optimal management of chemotherapy-induced thrombocytopenia with thrombopoietin receptor agonists Blood Rev. (IF 7.4) Pub Date : 2023-10-18 Hanny Al-Samkari
Chemotherapy-induced thrombocytopenia (CIT) is a common complication of antineoplastic therapy, resulting in antineoplastic therapy dose reductions, treatment delays, treatment discontinuation, and morbid bleeding events. Despite several decades of research into thrombopoietic growth factors in CIT, there are presently no available U.S. FDA- or EMA-approved agents to treat CIT. However, a respectable
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Management of transfusion-dependent β-thalassemia (TDT): Expert insights and practical overview from the Middle East Blood Rev. (IF 7.4) Pub Date : 2023-10-13 Amal El-Beshlawy, Hany Dewedar, Salwa Hindawi, Salam Alkindi, Azza A. Tantawy, Mohamed A. Yassin, Ali T. Taher
β-Thalassemia is one of the most common monogenetic diseases worldwide, with a particularly high prevalence in the Middle East region. As such, we have developed long-standing experience with disease management and devising solutions to address challenges attributed to resource limitations. The region has also participated in the majority of clinical trials and development programs of iron chelators
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Haematopoietic stem cell health in sickle cell disease and its implications for stem cell therapies and secondary haematological disorders Blood Rev. (IF 7.4) Pub Date : 2023-10-13 Vishaka Gorur, Kamil R. Kranc, Miguel Ganuza, Paul Telfer
Gene modification of haematopoietic stem cells (HSCs) is a potentially curative approach to sickle cell disease (SCD) and offers hope for patients who are not eligible for allogeneic HSC transplantation. Current approaches require in vitro manipulation of healthy autologous HSC prior to their transplantation. However, the health and integrity of HSCs may be compromised by a variety of disease processes
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Access to CAR T-cell therapy: Focus on diversity, equity and inclusion Blood Rev. (IF 7.4) Pub Date : 2023-10-05 Maria S. Odstrcil, Catherine J. Lee, Catherine Sobieski, Daniel Weisdorf, Daniel Couriel
Chimeric antigen receptor T-cell (CAR T-cell) therapy has revolutionized the treatment of hematologic malignancies in patients with relapsed or refractory disease without other treatment options. However, only a very small proportion of patients with an indication for CAR T-cell can access the treatment. The imbalance between supply and demand is magnified in minority and vulnerable populations. Limited
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Platelet transfusion refractoriness due to HLA alloimmunization: Evolving paradigms in mechanisms and management Blood Rev. (IF 7.4) Pub Date : 2023-10-02 Sandhya R. Panch, Li Guo, Ralph Vassallo
Platelet transfusion refractoriness due to HLA alloimmunization presents a significant medical problem, particularly among multiply transfused patients with hematologic malignancies and those undergoing hematopoietic stem cell transplants. HLA compatible platelet transfusions also impose significant financial burden on these patients. Recently, several novel mechanisms have been described in the development
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Revolutionizing chronic lymphocytic leukemia diagnosis: A deep dive into the diverse applications of machine learning Blood Rev. (IF 7.4) Pub Date : 2023-09-22 Mohamed Elhadary, Amgad Mohamed Elshoeibi, Ahmed Badr, Basel Elsayed, Omar Metwally, Ahmed Mohamed Elshoeibi, Mervat Mattar, Khalil Alfarsi, Salem AlShammari, Awni Alshurafa, Mohamed Yassin
Chronic lymphocytic leukemia (CLL) is a B cell neoplasm characterized by the accumulation of aberrant monoclonal B lymphocytes. CLL is the predominant type of leukemia in Western countries, accounting for 25% of cases. Although many patients remain asymptomatic, a subset may exhibit typical lymphoma symptoms, acquired immunodeficiency disorders, or autoimmune complications. Diagnosis involves blood
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Using artificial intelligence to improve body iron quantification: A scoping review Blood Rev. (IF 7.4) Pub Date : 2023-09-18 Abdulqadir J. Nashwan, Ibraheem M. Alkhawaldeh, Nour Shaheen, Ibrahem Albalkhi, Ibrahim Serag, Khalid Sarhan, Ahmad A. Abujaber, Alaa Abd-Alrazaq, Mohamed A. Yassin
This scoping review explores the potential of artificial intelligence (AI) in enhancing the screening, diagnosis, and monitoring of disorders related to body iron levels. A systematic search was performed to identify studies that utilize machine learning in iron-related disorders. The search revealed a wide range of machine learning algorithms used by different studies. Notably, most studies used a
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Androgen use in bone marrow failures and myeloid neoplasms: Mechanisms of action and a systematic review of clinical data Blood Rev. (IF 7.4) Pub Date : 2023-09-09 Alessandro Bosi, Wilma Barcellini, Francesco Passamonti, Bruno Fattizzo
Despite recent advancements, treatment of cytopenia due to bone marrow failures (BMF) and myeloid neoplasms remains challenging. Androgens promote renewal and maturation of blood cells and may be beneficial in these forms. Here we report a systematic review of androgens use as single agent in hematologic conditions. Forty-six studies, mainly retrospective with various androgen types and doses, were
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Prophylactic and therapeutic strategies for intraoperative bleeding in women with von Willebrand disease and heavy menstrual bleeding: A systematic review Blood Rev. (IF 7.4) Pub Date : 2023-09-07 H.P. Eising, M.C. Punt, J.C. Leemans, M.Y. Bongers
Background Optimal peri-operative management for women with Von Willebrand disease (VWD) and heavy menstrual bleeding (HMB) remains undetermined. Aim and methods To evaluate (pre)operative management in relation to (post)operative bleeding after endometrial ablation (EA) and hysterectomy in VWD women with HMB by performing a database search between 1994 and 2023. Results Eleven cohort studies and 1
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Targeting apoptosis dysregulation in myeloid malignancies - The promise of a therapeutic revolution Blood Rev. (IF 7.4) Pub Date : 2023-08-29 Enrico Santinelli, Maria Rosaria Pascale, Zhuoer Xie, Talha Badar, Maximilian F. Stahl, Jan P. Bewersdorf, Carmelo Gurnari, Amer M. Zeidan
In recent years, the therapeutic landscape of myeloid malignancies has been completely revolutionized by the introduction of several new drugs, targeting molecular alterations or pathways crucial for leukemia cells survival. Particularly, many agents targeting apoptosis have been investigated in both pre-clinical and clinical studies. For instance, venetoclax, a pro-apoptotic agent active on BCL-2
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Current approach to Waldenström macroglobulinemia Blood Rev. (IF 7.4) Pub Date : 2023-08-26 Prashant Kapoor, S. Vincent Rajkumar
Waldenström macroglobulinemia (WM) is a unique CD20+, B-cell non-Hodgkin lymphoma, characterized by lymphoplasmacytic infiltration of the bone marrow and circulating monoclonal immunoglobulin M. The clinical manifestations and outcomes of patients are highly variable. High-level evidence supports integration of monoclonal anti-CD20 antibody, rituximab, to the chemotherapy backbone to treat WM. However
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Classification, risk stratification and response assessment in myelodysplastic syndromes/neoplasms (MDS): A state-of-the-art report on behalf of the International Consortium for MDS (icMDS) Blood Rev. (IF 7.4) Pub Date : 2023-08-19 Maximilian Stahl, Jan Philipp Bewersdorf, Zhuoer Xie, Matteo Giovanni Della Porta, Rami Komrokji, Mina L. Xu, Omar Abdel-Wahab, Justin Taylor, David P. Steensma, Daniel T. Starczynowski, Mikkael A. Sekeres, Guillermo Sanz, David A. Sallman, Gail J. Roboz, Uwe Platzbecker, Mrinal M. Patnaik, Eric Padron, Olatoyosi Odenike, Stephen D. Nimer, Aziz Nazha, Amer M. Zeidan
The guidelines for classification, prognostication, and response assessment of myelodysplastic syndromes/neoplasms (MDS) have all recently been updated. In this report on behalf of the International Consortium for MDS (icMDS) we summarize these developments. We first critically examine the updated World Health Organization (WHO) classification and the International Consensus Classification (ICC) of
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Factor XI inhibitors for the prevention of cardiovascular disease: A new therapeutic approach on the horizon? Blood Rev. (IF 7.4) Pub Date : 2023-08-10 D. Santagata, M.P. Donadini, W. Ageno
Anticoagulant drugs that are currently used to prevent and/or treat thrombosis have some limitations that hinder their ability to meet specific clinical requirements. While these drugs effectively reduce the rates of thrombotic events, they simultaneously increase the risk of bleeding. Moreover, their risk-to-benefit balance is problematic in some patients, such as those with severe chronic kidney
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Awareness of individual goals, preferences, and priorities of persons with severe congenital haemophilia A for a tailored shared decision-making approach to liver-directed gene therapy. A practical guideline Blood Rev. (IF 7.4) Pub Date : 2023-08-01 Giovanni Di Minno, Gaia Spadarella, Nelson Mauro Maldonato, Natascia De Lucia, Giancarlo Castaman, Raimondo De Cristofaro, Cristina Santoro, Flora Peyvandi, Anna Borrelli, Angelo Lupi, Marco Follino, Gerardo Guerrino, Filomena Morisco, Matteo Di Minno
In clinical medicine, shared decision making (SDM) is a well-recognized strategy to enhance engagement of both patients and clinicians in medical decisions. The success of liver-directed gene therapy (GT) to transform severe congenital haemophilia A (HA) from an incurable to a curable disease has launched a shift beyond current standards of treatment. However, GT acceptance remains low in the community
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The core concepts of core binding factor acute myeloid leukemia: Current considerations for prognosis and treatment Blood Rev. (IF 7.4) Pub Date : 2023-07-25 Christina Darwish, Kyle Farina, Douglas Tremblay
Core binding factor acute myeloid leukemia (CBF AML), defined by t(8;21) or inv(16), is a subset of favorable risk AML. Despite its association with a high complete remission rate after induction and relatively good prognosis overall compared with other subtypes of AML, relapse risk after induction chemotherapy remains high. Optimizing treatment planning to promote recurrence free survival and increase
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Aiming for the cure in myeloma: Putting our best foot forward Blood Rev. (IF 7.4) Pub Date : 2023-07-14 Noffar Bar, Ross S. Firestone, Saad Z. Usmani
Frontline therapy for multiple myeloma (MM) is evolving to include novel combinations that can achieve unprecedented deep response rates. Several treatment strategies exist, varying in induction regimen composition, use of transplant and or consolidation and maintenance. In this sea of different treatment permutations, the overarching theme is the powerful prognostic factors of disease risk and achievement
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Future prospects for the clinical transfusion of pig red blood cells Blood Rev. (IF 7.4) Pub Date : 2023-07-14 Yevgen Chornenkyy, Takayuki Yamamoto, Hidetaka Hara, Sean R. Stowell, Ionita Ghiran, Simon C. Robson, David K.C. Cooper
Transfusion of allogeneic human red blood cell (hRBCs) is limited by supply and compatibility between individual donors and recipients. In situations where the blood supply is constrained or when no compatible RBCs are available, patients suffer. As a result, alternatives to hRBCs that complement existing RBC transfusion strategies are needed. Pig RBCs (pRBCs) could provide an alternative because of
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Do anemia treatments improve quality of life and physical function in patients with myelodysplastic syndromes (MDS)? A systematic review Blood Rev. (IF 7.4) Pub Date : 2023-07-13 Allison Mo, Matthew Poynton, Erica Wood, Jake Shortt, Susan J. Brunskill, Carolyn Doree, Josie Sandercock, Nicholas Saadah, Edwin Luk, Simon J. Stanworth, Zoe McQuilten
Anemia is common in Myelodysplastic Syndromes (MDS). Different anemia treatments have been tested in clinical studies, but the full impact on patients' health-related quality of life (HRQoL) and physical function is unknown. The main aim of this review was to assess whether improvements in anemia are associated with changes in HRQoL/physical function. Twenty-six full-text publications were identified
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Preservation of fertility in female patients with hematologic diseases Blood Rev. (IF 7.4) Pub Date : 2023-07-13 Marika Bini Antunes, Sara Pinto Cardeal, Manuel Magalhães, Emídio Vale-Fernandes, Márcia Barreiro, Rosália Sá, Mário Sousa
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Primary and secondary immune thrombocytopenia (ITP): Time for a rethink Blood Rev. (IF 7.4) Pub Date : 2023-06-29 Tomás José González-López, Drew Provan, Abelardo Bárez, Angel Bernardo-Gutiérrez, Silvia Bernat, Daniel Martínez-Carballeira, Isidro Jarque-Ramos, Inmaculada Soto, Reyes Jiménez-Bárcenas, Fernando Fernández-Fuertes
There are not many publications that provide a holistic view of the management of primary and secondary ITP as a whole, reflecting the similarities and differences between the two. Given the lack of major clinical trials, we believe that comprehensive reviews are much needed to guide the diagnosis and treatment of ITP today. Therefore, our review addresses the contemporary diagnosis and treatment of
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Activation of pyruvate kinase as therapeutic option for rare hemolytic anemias: Shedding new light on an old enzyme Blood Rev. (IF 7.4) Pub Date : 2023-06-12 Myrthe J. van Dijk, Jonathan R.A. de Wilde, Marije Bartels, Kevin H.M. Kuo, Andreas Glenthøj, Minke A.E. Rab, Eduard J. van Beers, Richard van Wijk
Novel developments in therapies for various hereditary hemolytic anemias reflect the pivotal role of pyruvate kinase (PK), a key enzyme of glycolysis, in red blood cell (RBC) health. Without PK catalyzing one of the final steps of the Embden-Meyerhof pathway, there is no net yield of adenosine triphosphate (ATP) during glycolysis, the sole source of energy production required for proper RBC function
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Artificial intelligence in sickle disease Blood Rev. (IF 7.4) Pub Date : 2023-06-08 Ahmed Adel Elsabagh, Mohamed Elhadary, Basel Elsayed, Amgad Mohamed Elshoeibi, Khaled Ferih, Rasha Kaddoura, Salam Alkindi, Awni Alshurafa, Mona Alrasheed, Abdullah Alzayed, Abdulrahman Al-Abdulmalek, Jaffer Abduljabber Altooq, Mohamed Yassin
Artificial intelligence (AI) is rapidly becoming an established arm in medical sciences and clinical practice in numerous medical fields. Its implications have been rising and are being widely used in research, diagnostics, and treatment options for many pathologies, including sickle cell disease (SCD). AI has started new ways to improve risk stratification and diagnosing SCD complications early, allowing
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Proteasome inhibition in combination with immunotherapies: State-of-the-Art in multiple myeloma Blood Rev. (IF 7.4) Pub Date : 2023-05-27 David Kegyes, Diana Gulei, Rares Drula, Diana Cenariu, Bogdan Tigu, Delia Dima, Alina Tanase, Sorina Badelita, Anca-Dana Buzoianu, Stefan Ciurea, Gabriel Ghiaur, Evangelos Terpos, Aaron Ciechanover, Hermann Einsele, Ciprian Tomuleasa
Multiple myeloma (MM) is a malignant plasma cell disorder accounting for around 1.8% of all neoplastic diseases. Nowadays, clinicians have a broad arsenal of drugs at their disposal for the treatment of MM, such as proteasome inhibitors, immunomodulatory drugs, monoclonal antibodies, bispecific antibodies, CAR T-cell therapies and antibody-drug conjugates. In this paper we briefly highlight essential
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Central nervous system prophylaxis in diffuse large B-cell lymphoma: What does the evidence tell us? Blood Rev. (IF 7.4) Pub Date : 2023-05-26 Jeffrey Lantz, Craig A. Portell, Emily C. Ayers
Secondary involvement of the central nervous system (CNS) by diffuse large b-cell lymphoma (DLBCL) is a rare yet often catastrophic event for DLBCL patients. As standard first-line therapy for DLBCL with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) does not cross the blood-brain barrier, one approach to lessen the risk of CNS relapse has been to include additional
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PTCy: The “new” standard for GVHD prophylaxis Blood Rev. (IF 7.4) Pub Date : 2023-05-16 Andrea Bacigalupo, Richard Jones
High dose Post transplant cyclophosphamide (PTCy) is now regarded as a very effective way of preventing acute and chronic GvHD, and it's use has rapidly expanded world-wide.
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The road to implementation of pharmacokinetic-guided dosing of factor replacement therapy in hemophilia and allied bleeding disorders. Identifying knowledge gaps by mapping barriers and facilitators Blood Rev. (IF 7.4) Pub Date : 2023-05-09 Tine M.H.J. Goedhart, A. Janssen, Ron A.A. Mathôt, Marjon H. Cnossen
Clinical guidelines and expert groups recommend the use of pharmacokinetic (PK)-guided dosing of factor replacement therapy for the treatment of bleeding disorders, especially for patients with hemophilia. Although PK-guided dosing is increasingly applied, it is generally not considered standard clinical practice. The aim of this scoping review is to map barriers and facilitators for the implementation
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Thinking “outside the germinal center”: Re-educating T cells to combat follicular lymphoma Blood Rev. (IF 7.4) Pub Date : 2023-05-08 Swetha Kambhampati, Geoffrey Shouse, Alexey V. Danilov
There have been significant advancements in the management of follicular lymphoma (FL), the most common indolent lymphoma. These include immunomodulatory agents such as lenalidomide, epigenetic modifiers (tazemetostat), and phosphoinotiside-3 kinase inhibitors (copanlisib). The focus of this review is T cell-engager therapies, namely chimeric antigen receptor (CAR) T-cell therapy and bispecific antibodies
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Diamond-Blackfan anemia in adults: In pursuit of a common approach for a rare disease Blood Rev. (IF 7.4) Pub Date : 2023-05-08 Deena Iskander, Noémi B.A. Roy, Elspeth Payne, Emma Drasar, Kelly Hennessy, Yvonne Harrington, Chrysi Christodoulidou, Anastasios Karadimitris, Leisa Batkin, Josu de la Fuente
Diamond-Blackfan anemia (DBA) is a rare bone marrow failure syndrome, usually caused by loss-of function variants in genes encoding ribosomal proteins. The hallmarks of DBA are anemia, congenital anomalies and cancer predisposition. Although DBA usually presents in childhood, the prevalence in later life is increasing due to an expanding repertoire of implicated genes, improvements in genetic diagnosis
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Failure of CAR-T cell therapy in relapsed and refractory large cell lymphoma and multiple myeloma: An urgent unmet need Blood Rev. (IF 7.4) Pub Date : 2023-04-29 Yasmine St. Martin, Joseph K. Franz, Mounzer E. Agha, Hillard M. Lazarus
Since its FDA approval, chimeric antigen receptor (CAR)-T cell therapy is changing the landscape of the treatment algorithm for relapsed and refractory large cell lymphoma and multiple myeloma. While initially hailed as a game changer and received widely with great enthusiasm, the reality of treatment failure soon became a major disappointment. This situation left patients and clinicians alike wondering
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Beyond current treatment of Fanconi Anemia: What do advances in cell and gene-based approaches offer? Blood Rev. (IF 7.4) Pub Date : 2023-04-28 Elena Martínez-Balsalobre, Jean-Hugues Guervilly, Jenny van Asbeck-van der Wijst, Ana Belén Pérez-Oliva, Christophe Lachaud