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Relevance of donor-specific HLA antibodies in hematopoietic cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-08-28 Thuong Hien Tran, Andreas Heinold, Magdalena Spackova, Lien Pham, Matthias Stelljes, Peter Dreger
Advances in hematopoietic cell transplantation have expanded the use of alternative donors such as haploidentical family donors or mismatched unrelated donors. However, donor-specific HLA antibodies (DSA) have been recognized as a significant risk factor of primary graft failure after HLA incompatible transplantation. Therefore, screening for HLA antibodies and taking DSA into consideration in the
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From clones to immunopeptidomes: New developments in the characterization of permissive HLA-DP mismatches in hematopoietic cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-08-28 Esteban Arrieta-Bolaños
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Interfering with KIR and NKG2A immune checkpoint axes to unleash NK cell immunotherapy Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-31 Nicky A. Beelen, Vera T.C. Valckx, Gerard M.J. Bos, Lotte Wieten
Due to their intrinsic ability to eliminate malignant cells, natural killer (NK) cells emerge as a promising immunotherapy for cancer. While clinical studies have affirmed the safety of NK cell infusions and combination therapies have demonstrated encouraging outcomes in hematological malignancies, the efficacy of NK cell immunotherapeutic interventions remains heterogeneous across patient cohorts
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Cytomegalovirus in haematopoietic cell transplantation - The troll is still there Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-26 Sebastian Voigt
Patients undergoing allogeneic haematopoietic cell transplantation are prone to complications caused by viral infections. Cytomegalovirus (CMV) considerably impacts transplantation as it frequently requires antiviral intervention that evokes substantial side effects depending on the antiviral drug. Intermittent antiviral treatment may become necessary if CMV DNAemia cannot be permanently suppressed
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Deciphering the role of the major histocompatibility complex, the intestinal microbiome and metabolites in the pathogenesis of acute graft-versus-host disease Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-26 Valentin Wenger, Robert Zeiser
Allogeneic hematologic stem cell transplantation is a cornerstone in modern hematological treatment, yet its efficacy is compromised by acute Graft-versus-Host Disease. In acute Graft-versus-Host Disease, conditioning regimen induced epithelial damage leads to release of damage and pathogen associated molecular patters which in turns triggers activation of alloreactive donor T cells, ultimately resulting
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HLA structure and function in hematopoietic-cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-25 Effie W. Petersdorf
The degree of HLA compatibility between a patient and donor has formed the basis of donor selection since the development of allogeneic hematopoietic cell transplantation over 50 years ago and has advanced understanding of the basic immunobiology of HLA. New evidence supports a role for germline variation in the patient and the donor that do not require HLA matching for their effects to have clinical
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Allogeneic “Off-the-Shelf” CAR T cells: Challenges and advances Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-25 Sophia Chen, Marcel R.M. van den Brink
Chimeric antigen receptor (CAR) T cell therapy has shown impressive clinical efficacy in B cell malignancies and multiple myeloma, leading to the approval of six CAR T cell products by the U.S. Food and Drug Administration (FDA) to date. However, broad application of these autologous (patient-derived) CAR T cells is limited by several factors, including high production costs, inconsistent product quality
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Registries and observational databases in haematology Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-18 Gregorio Jaimovich, Robert Peter Gale
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Is HLA-E with its receptors an immune checkpoint or an antigenic determinant in allo-HCT? Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-10 Hana Rohn, Vera Rebmann
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The most frequent HLA alleles around the world: A fundamental synopsis Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-08 Alicia Sanchez-Mazas, José Manuel Nunes, PGAE HLA Consortium of the 18th International HLA and Immunogenetics Workshop, Da Di, Enrique Alanis Dominguez, Pascale Gerbault, Ndeye Khady Faye, Wassim Almawi, Marco Andreani, Esteban Arrieta-Bolanos, Danillo G. Augusto, Stéphane Buhler, Natasja G. de Groot, Roberto Díaz-Peña, Selma D'Silva, Valérie Dubois, Gehad ElGhazali, Clara Gorodezky, Zorana Grubić
A comprehensive knowledge of human leukocyte antigen (HLA) molecular variation worldwide is essential in human population genetics research and disease association studies and is also indispensable for clinical applications such as allogeneic hematopoietic cell transplantation, where ensuring HLA compatibility between donors and recipients is paramount. Enormous progress has been made in this field
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HLA-DM and HLA-DO interplay for the peptide editing of HLA class II in healthy tissues and leukemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-06 Bettina Budeus, Miguel Álvaro-Benito, Pietro Crivello
HLA class II antigen presentation is modulated by the activity of the peptide editor HLA-DM and its antagonist HLA-DO, with their interplay controlling the peptide repertoires presented by normal and malignant cells. The role of these molecules in allogeneic hematopoietic cell transplantation (alloHCT) is poorly investigated. Balanced expression of HLA-DM and HLA-DO can influence the presentation of
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Shape of the art: TCR-repertoire after allogeneic hematopoietic cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-05 Heike Uhlemann, Katharina Epp, Christian Klesse, Cornelia S. Link-Rachner, Vineeth Surendranath, Ulf-Peter Günther, Johannes Schetelig, Falk Heidenreich
The human adaptive immune repertoire is characterized by specificity and diversity to provide immunity against past and future tasks. Such tasks are mainly infections but also malignant transformations of cells. With its multiple lines of defense, the human immune system contains both, rapid reaction forces and the potential to capture, disassemble and analyze strange structures in order to teach the
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HLA typing: A review of methodologies and clinical impact on haematopoietic cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-05 Neema P. Mayor, Steven G.E. Marsh
The importance of the gene system in haematopoietic cell transplant outcomes was established early on and advances in both fields have led to ever increasing success of this clinical therapy. In large part, improvements in the understanding of have been driven by the advancement in typing technologies. Each iteration of typing technology has improved the resolution of typing, and often enabled the
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The role of registries in hematological disorders Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-07-05 Helen Baldomero, Daniel Neumann, Nada Hamad, Yoshiko Atsuta, Anna Sureda, Minako Iida, Amado Karduss, Alaa M. Elhaddad, Nosa G. Bazuaye, Carmem Bonfim, Rafael de la Camara, Naeem A. Chaudhri, Fabio Ciceri, Cinthya Correa, Cristobal Frutos, Sebastian Galeano, Laurent Garderet, Raffaella Greco, Gregorio Jaimovich, Yoshihisa Kodera, Mickey BC. Koh, Kaiyan Liu, Per Ljungman, Donal P. McLornan, Gayathri
Hematopoietic cell transplantation (HCT) was developed more than 65 years ago to treat malignant blood disorders and irreversible bone marrow failures, with the aim of replacing a diseased hematopoietic system with a healthy one (allogeneic HCT). Decades later, the procedure was adapted to apply maximal chemotherapy or radiotherapy, which would result in bone marrow failure, but could be remedied by
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Celebrating the registration of 9.000 patients treated with CAR T cells in the EBMT registry: Collection of real-world data in the context of hematopoietic cellular therapies Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-06-28 Christian Chabannon, Annalisa Ruggeri, Silvia Montoto, Anja van Biezen, Steffie van der Werf, Annemiek Markslag, Isabel Sanchez-Ortega, Rafael de la Camara, Per Ljungman, Mohamad Mohty, Nicolaus Kröger, Ana Sureda, Eoin McGrath, Chiara Bonini, Jurgen Kuball
The European society for Blood and Marrow Transplantation (EBMT) has a long-standing interest in the evaluation of hematopoietic cell transplantation. More than three decades ago, its members established a continental registry. Today, more than 700,000 patients have been registered, and information has been gathered on more than 800,000 transplants. This huge amount of information has allowed conducting
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Minor histocompatibility antigens to predict, monitor or manipulate GvL and GvHD after allogeneic hematopoietic cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-05-15 Kyra J. Fuchs, J.H. Frederik Falkenburg, Marieke Griffioen
Allogeneic hematopoietic cell transplantation (alloHCT) provides a potential curative treatment for haematological malignancies. The therapeutic Graft-versus-Leukaemia (GvL) effect is induced by donor T cells attacking patient hematopoietic (malignant) cells. However, if healthy non-hematopoietic tissues are targeted, Graft-versus-Disease (GvHD) may develop. After HLA-matched alloHCT, GvL and GvHD
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Corrigendum to “HIV-associated lymphoma” [Best Pract Res Clin Haematol 25 (2012) 101–117] Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-04-30 Lawrence D. Kaplan
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Cytogenetics in haematology Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-04-10 Yanming Zhang, Brigitte Schlegelberger
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Cytogenetics and genomics in CML and other myeloproliferative neoplasms Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-04-03 Hans H. Kreipe, Brigitte Schlegelberger
Chronic myeloid leukemia is defined by the presence of the Philadelphia translocation t (9; 22) resulting in the fusion. The other myeloproliferative neoplasms (MPN) subtypes also carry typical chromosomal abnormalities, which however are not pathognomonic for a specific entity of MPN. According to the WHO classification the distinction between these entities is still based on the integration of cytological
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The Collaborative Biobank (CoBi): Donor and recipient samples & data to facilitate future research on hematopoietic cell transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-04-01 Claudia Spielau, Carolin Bunzel, Stefan Abert, Henning Baldauf, Alexander H. Schmidt, Johannes Schetelig
Biobanking provides benefit for future generations by facilitating medical research and subsequent translation and application of research findings. Long-term storage and research involving biological material and associated data necessitate the proper implementation of ethical and legal standards. A key principle includes recognizing informed consent as a crucial element for legitimizing the collection
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Providing hematopoietic stem cell products from unrelated donors to the world: DKMS donor centers and DKMS Registry Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-03-01 Alexander H. Schmidt, Jürgen Sauter, Johannes Schetelig, Elke Neujahr, Julia Pingel
Allogeneic hematopoietic stem cell (HSC) transplantation is a curative therapy for many severe blood diseases. As many patients have no suitable family donor, large unrelated donor registries and donor centers have been established in many countries, along with an international system for the provision of unrelated donor HSC products. As an essential part of this system, DKMS operates donor centers
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Corrigendum to “Endpoint selection and evaluation in hematology studies” [Best Pract Res Clin Haematol 36 (2023) 101479] Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-02-23 Ruta Brazauskas, Mary Eapen, Tao Wang
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The landscape of cytogenetic and molecular genetic methods in diagnostics for hematologic neoplasia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-02-05 Yvonne Lisa Behrens, Stefan Pietzsch, Željko Antić, Yanming Zhang, Anke K. Bergmann
Improvements made during the last decades in the management of patients with hematologic neoplasia have resulted in increase of overall survival. These advancements have become possible through progress in our understanding of genetic basis of different hematologic malignancies and their role in the current risk-adapted treatment protocols. In this review, we provide an overview of current cytogenetic
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Corrigendum to “Designing and conducting a clinical trial in blood and marrow transplantation” [Best Pract Res Clin Haematol 36 (2023) 101471] Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-02-02 Michael J. Martens, Yan Gao, Aniko Szabo
Abstract not available
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Germline predisposition to myeloid neoplasms: Characteristics and management of high versus variable penetrance disorders Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-02-01 Amy M. Trottier, Simone Feurstein, Lucy A. Godley
Myeloid neoplasms with germline predisposition have been recognized increasingly over the past decade with numerous newly described disorders. Penetrance, age of onset, phenotypic heterogeneity, and somatic driver events differ widely among these conditions and sometimes even within family members with the same variant, making risk assessment and counseling of these individuals inherently difficult
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Using real-world evidence in haematology Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2024-01-27 Francesco Passamonti, Giovanni Corrao, Gastone Castellani, Barbara Mora, Giulia Maggioni, Matteo Giovanni Della Porta, Robert Peter Gale
Most new drug approvals are based on data from large randomized clinical trials (RCTs). However, there are sometimes contradictory conclusions from seemingly similar trials and generalizability of conclusions from these trials is limited. These considerations explain, in part, the gap between conclusions from data of RCTs and those from registries termed data (RWD). Recently, real-world evidence (RWE)
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Editorial Board Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-12-12
Abstract not available
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Cytogenetics and genomics of acute myeloid leukemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-12-10 Oraine Snaith, Corey Poveda-Rogers, Dorottya Laczko, Guang Yang, Jennifer J.D. Morrissette
The diversity of genetic and genomic abnormalities observed in acute myeloid leukemia (AML) reflects the complexity of these hematologic neoplasms. The detection of cytogenetic and molecular alterations is fundamental to diagnosis, risk stratification and treatment of AML. Chromosome rearrangements are well established in the diagnostic classification of AML, as are some gene mutations, in several
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Best practice & research clinical haematology: Screening for breast cancer in hodgkin lymphoma survivors Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-11-25 Stephanie M. Wong
Childhood and young adult survivors of Hodgkin lymphoma are at an elevated risk of developing breast cancer. Breast cancer risk is felt to originate from chest wall radiation exposure prior to the third decade of life, with incidence beginning to rise approximately eight to ten years following Hodgkin lymphoma treatment. Although incidence varies according to age at radiation exposure, dosage, and
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Progress and challenges in the acute leukemia field Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-11-07 Daniel A. Pollyea
Abstract not available
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The role of randomized controlled trials, registries, observational databases in evaluating new interventions Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-31 Robert Peter Gale, Mei-Jie Zhang, Hillard M. Lazarus
Approaches to comparing safety and efficacy of interventions include analyzing data from randomized controlled trials (RCTs), registries and observational databases (ODBs). RCTs are regarded as the gold standard but data from such trials are sometimes unavailable because a disease is uncommon, because the intervention is uncommon, because of structural limitations or because randomization cannot be
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The international cooperative Gaucher group (ICCG) Gaucher registry Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-31 Neal J. Weinreb
Gaucher disease GD), is a rare lysosomal storage disorder caused by deficient acid β-glucosylceramidase activity and accumulation of glucosylceramide in tissue macrophages. With the 1991 advent of alglucerase enzyme replenishment therapy (ERT), the manufacturer (Genzyme Corporation) created the ICGG Gaucher Registry to collect longitudinal observational “real word” information about GD world-wide in
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The Australian Aplastic Anaemia and other Bone Marrow Failure Syndromes Registry Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-23 Lucy C. Fox, Zoe K. McQuilten, Frank Firkin, Vanessa Fox, Xavier Badoux, Ashish Bajel, Pasquale Barbaro, Merrole F. Cole-Sinclair, Cecily Forsyth, John Gibson, Devendra K. Hiwase, Anna Johnston, Anthony Mills, Fernando Roncolato, Robyn Sutherland, Jeff Szer, Stephen B. Ting, Shahla Vilcassim, Lauren Young, Neil A. Waters, Erica M. Wood
The bone marrow failure syndromes (BMFS) are a diverse group of acquired and inherited diseases which may manifest in cytopenias, haematological malignancy and/or syndromic multisystem disease. Patients with BMFS frequently experience poor outcomes, and improved treatment strategies are needed. Collation of clinical characteristics and patient outcomes in a national disease-specific registry represents
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Mechanisms of resistance to hypomethylating agents and BCL-2 inhibitors Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-20 Sudhamsh Reddy Desai, Samarpana Chakraborty, Aditi Shastri
Myeloid malignancies such as myelodysplastic syndrome (MDS) & acute myeloid leukemia (AML) are clonal diseases that emerge and progress due to the expansion of disease-initiating aberrant hematopoietic stem cells, that are not eliminated by conventional cytotoxic therapies. Hypomethylating agents(HMA), azacytidine and decitabine are the first line agents for treatment of MDS and a combination with
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WHO, what, when, where, and why: New classification systems for acute myeloid leukemia and their impact on clinical practice Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-20 Frederick R. Appelbaum
The goal of a disease classification system is (or should be) to provide a tool for researchers and clinicians to study and treat the disease. The last decade has seen a markedly improved understanding of the pathophysiology of acute myeloid leukemia (AML), the development of new methods to measure the disease, and approval by the Food and Drug Administration (FDA) of at least ten new therapies targeted
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The best GVHD prophylaxis: Or at least progress towards finding it Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-20 Daniel Weisdorf, Najla El Jurdi, Shernan G. Holtan
Options for GVHD prophylaxis after allogeneic hematopoietic cell transplantation can best be chosen by understanding the pathophysiology of GVHD. Interventions to limit T cell activation, expansion and subsequent tissue injury can each be utilized in designing successful GVHD prevention strategies Depleting, tolerizing or blunting T cells or host antigen presenting cells (APCs), blocking co-stimulation
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When to use which molecular prognostic scoring system in the management of patients with MDS? Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-17 Tariq Kewan, Jan Philipp Bewersdorf, Carmelo Gurnari, Zhuoer Xie, Maximilian Stahl, Amer M. Zeidan
Myelodysplastic syndromes/neoplasms (MDS) are a heterogeneous group of hematopoietic cancers characterized by recurrent molecular alterations driving the disease pathogenesis with a variable propensity for progression to acute myeloid leukemia (AML). Clinical decision making for MDS relies on appropriate risk stratification at diagnosis, with higher-risk patients requiring more intensive therapy. The
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Asparaginase dosing for obese patients with acute lymphoblastic leukemia and factors that contribute to outcomes Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-14 Ryan D. Cassaday
Asparaginase in various forms is a standard part of the treatment of acute lymphoblastic leukemia (ALL) in children and adolescents. However, its use is more selective in adults. One of the key reasons is the toxicity observed from this class of agents. In a series of recent post hoc analyses of large prospective studies, obesity has emerged as a key factor that contributes to the challenges with administering
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Cytogenomics of B-cell non-Hodgkin lymphomas: The “old” meets the “new” Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-10 Marta Grau, Cristina López, José Ignacio Martín-Subero, Sílvia Beà
For the routine diagnosis of haematological neoplasms an integrative approach is used considering the morphology, and the immunophenotypic, and molecular features of the tumor sample, along with clinical information. The identification and characterization of recurrent chromosomal aberrations mainly detected by conventional and molecular cytogenetics in the tumor cells has a major impact on the classification
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Hodgkin Lymphoma: A disease shaped by the tumor micro- and macroenvironment Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-07 Rebecca Masel, Megan E. Roche, Ubaldo Martinez-Outschoorn
The tumor microenvironment (TMicroE) and tumor macroenvironment (TMacroE) are defining features of classical Hodgkin lymphoma (cHL). They are of critical importance to clinicians since they explain the common signs and symptoms, allow us to classify these neoplasms, develop prognostic and predictive biomarkers, bioimaging and novel treatments. The TMicroE is defined by effects of cancer cells to their
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Cardiac surgery for radiation associated heart disease in Hodgkin lymphoma patients Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-10-07 Daniel Shell
Much of the modern focus of Hodgkin's Lymphoma (HL) treatment involves the prevention of secondary organ injury. Despite rationalisations of radiotherapy fields, many patients still develop late radiation-related cardiotoxicity that is severe and requires interventional management. No guidelines exist to direct management of these complex patients who often present with multiple concurrent cardiac
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Editorial Board / Aims & Scope Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-08-21
Abstract not available
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Cytogenetics and genomics in pediatric acute lymphoblastic leukaemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-08-17 Željko Antić, Jana Lentes, Anke K. Bergmann
The last five decades have witnessed significant improvement in diagnostics, treatment and management of children with acute lymphoblastic leukaemia (ALL). These advancements have become possible through progress in our understanding of the genetic and biological background of ALL, resulting in the introduction of risk-adapted treatment and novel therapeutic targets, e.g., tyrosine kinase inhibitors
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Hodgkin lymphoma: Focus on evolving treatment paradigms Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-08-17 Joseph L. Roswarski, Dan L. Longo
Hodgkin lymphoma (HL) is a highly curable B-cell malignancy of germinal center origin. Biologically it is a hematologic malignancy that is highly dependent on the immune microenvironment and utilizes immune escape through upregulation of the programmed-death ligands on the neoplastic cells. Despite being highly curable, consensus is lacking nationally and internationally about the optimal approach
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Cytogenetics and molecular genetics of myelodysplastic neoplasms Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-08-16 Yi Ning, Yanming Zhang, Michael A. Kallen, Ashkan Emadi, Maria R. Baer
According to the 2022 World Health Organization (WHO) Classification (5th edition), the term myelodysplastic neoplasms (abbreviated MDS) has been introduced to replace myelodysplastic syndromes. MDS are a group of clonal hematopoietic stem cell diseases characterized by cytopenia(s), dysplasia in one or more of lineages, ineffective hematopoiesis, and an increased risk of progression to bone marrow
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Transplantation and cellular therapy in acute leukemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-22 Michael Grunwald, Belinda Avalos, Edward Copelan
Abstract not available
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Graft versus Leukemia in 2023 Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-10 Zane Chiad, Aleksander Chojecki
Allogeneic hematopoietic stem cell transplantation (HSCT) is commonly utilized in the management of leukemia across multiple subtypes. Graft versus leukemia (GVL) is a critical component of successful transplantation and involves donor cells eradicating residual leukemia within the recipient. Graft versus host disease (GVHD) by contrast is a common complication of the transplantation process in which
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Describing and analyzing complex disease history in retrospective studies Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-10 Ariane Boumendil, Myriam Labopin
Blood-related diseases are complex diseases with diverse origins, treatments and prognosis. In haematology studies, investigators are interested in multiple outcomes and multiple prognostic variables that may change value over the course of follow-up. These time-dependent variables can be of different nature. Time-dependent events such as treatment with haematopoeitic stem cell transplant (HCT) and
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Preface of special edition “Biostatistics in clinical haematology” Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-08 Myriam Labopin, Ariane Boumendil
Abstract not available
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Donor lymphocyte infusion in Acute Myeloid Leukemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-06 Nilay A. Shah
Donor lymphocyte infusion (DLI) is an important treatment modality in the management of relapsed hematological malignancies after allogeneic hematopoietic cell transplantation (allo-HCT). Donor T lymphocytes can be used in a therapeutic, pre-emptive or prophylactic manner in an attempt to stimulate a graft versus leukemia (GVL) effect and eradicate residual disease or even prevent relapse in a high-risk
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The current landscape: Allogeneic hematopoietic stem cell transplant for acute lymphoblastic leukemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-04 Susan Prockop, Franziska Wachter
One of the consistent features in development of hematopoietic stem cell transplant (HCT) for Acute Lymphoblastic Leukemia (ALL) is the rapidity with which discoveries in the laboratory are translated into innovations in clinical care. Just a few years after murine studies demonstrated that rescue from radiation induced marrow failure is mediated by cellular not humoral factors, E. Donnall Thomas reported
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“The State of the Science” of Childhood, Adolescent and Young Adult Non Hodgkin Lymphoma (NHL) Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-06-01 Mitchell S. Cairo
Abstract not available
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Reducing barriers of access and care related to hematopoietic cell transplantation and cellular therapy: The mission-driven role of the national marrow donor program Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-25 Rafeek A. Yusuf, Jaime M. Preussler, Christa L. Meyer, Katie Schoeppner, Jennifer A. Sees Coles, Amber Ruffin, Meggan McCann, Steven M. Devine, Jeffery J. Auletta
The treatment of malignant and nonmalignant hematologic disorders continues to benefit from significant scientific advancement and progress in the use of hematopoietic cell transplantation and cellular therapies. However, barriers associated with receiving these lifesaving treatments and care remain, which necessitate innovative approaches to overcome, so all persons in need can receive these therapies
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Handling missing covariate data in clinical studies in haematology Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-22 Edouard F. Bonneville, Johannes Schetelig, Hein Putter, Liesbeth C. de Wreede
Missing data are frequently encountered across studies in clinical haematology. Failure to handle these missing values in an appropriate manner can complicate the interpretation of a study's findings, as estimates presented may be biased and/or imprecise. In the present work, we first provide an overview of current methods for handling missing covariate data, along with their advantages and disadvantages
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Targeted cellular therapy for treatment of relapsed or refractory leukemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-25 Lauren D. Scherer, Rayne H. Rouce
While the mainstay of treatment for high-risk or relapsed, refractory leukemia has historically revolved around allogeneic hematopoietic stem cell transplant (allo-HSCT), targeted immunotherapies have emerged as a promising therapeutic option, especially given the poor prognosis of patients who relapse after allo-HSCT. Novel cellular immunotherapies that harness the cytotoxic abilities of the immune
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Endpoint selection and evaluation in hematology studies Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-24 Ruta Brazauskas, Mary Eapen, Tao Wang
Observational studies and clinical trials in hematology aim to examine treatments for blood disorders. The outcomes being studied must address the goals of the study and provide meaningful information about treatment course, disease progression, describe patients’ survival experience and quality of life. Endpoints are the specific measures of these outcomes, and much consideration should be given to
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The analysis of multiple outcomes, multiple variables and variables selection in hematopoietic cell transplantation studies Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-24 Soyoung Kim, Xi Fang, Kwang Woo Ahn
It is common to study time-to-event data in cancer research such as hematopoietic cell transplantation (HCT) for leukemia. The extensive work has been done for the univariate survival outcome, that is, one event type. However, in practice a subject is often exposed to multiple types of outcomes. In this article, we review various types of right-censored data with multiple outcome types including competing
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Designing and conducting a clinical trial in blood and marrow transplantation Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-11 Michael J. Martens, Yan Gao, Aniko Szabo
Clinical trials form the cornerstone of the science-based approach to improving patient outcomes. A trial needs to be designed and performed carefully to provide valid evidence to inform medical science and to protect the safety and well-being of its participants. The development of a clinical trial involving blood and marrow transplant (BMT) requires special considerations, including the rare disease
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Immunopathogenic mechanisms and modulatory approaches to graft-versus-host disease prevention in acute myeloid leukaemia Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-11 Yifan Pang, Noa G. Holtzman
Allogeneic haematopoietic stem cell transplantation (HSCT) remains the only potential cure for intermediate to high-risk acute myeloid leukaemia (AML). The therapeutic effect of HSCT is largely dependent on the powerful donor-derived immune response against recipient leukaemia cells, known as graft-versus-leukaemia effect (GvL). However, the donor-derived immune system can also cause acute or chronic
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What is relative survival and what is its role in haematology? Best Pract. Res. Clin. Haematol. (IF 2.2) Pub Date : 2023-05-08 Maja Pohar Perme, Liesbeth C. de Wreede, Damjan Manevski
In many haematological diseases, the survival probability is the key outcome. However, when the population of patients is rather old and the follow-up long, a significant proportion of deaths cannot be attributed to the studied disease. This lessens the importance of common survival analysis measures like overall survival and shows the need for other outcome measures requiring more complex methodology