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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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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 2.1) 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
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What should be done and what should be avoided when comparing two treatments? Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-05-06 Florie Brion Bouvier, Raphaël Porcher
The preferred approach to compare two treatments is a randomized controlled trial (RCT). Indeed, randomization ensures that the groups compared are similar. Well-designed and well-conducted RCTs thus allow to draw causal conclusions on the relative efficacy and safety of treatments compared. However, it is not always possible to conduct RCTs for all clinical questions of interest, and observational
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The presentation of results from studies in clinical haematology Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-05-06 Richard M. Szydlo
Research is based on trying to find answers to specific questions or to test hypotheses. Studies are thus undertaken to generate data which, with appropriate statistical methods, will help to determine the validity of the science under investigation. The aim of this paper is not to provide answers on which statistical methods to use, but will concentrate on suggesting the best ways of presenting the
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Take a spin: Apheresis in the care of adult leukaemia patients Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-18 Jing Ai
Apheresis is an automated process to separate the whole blood of a patient or a donor, collect or remove specific blood components, and return the remaining back to the individual. Apheresis is an integral part of blood and marrow transplantation and has been increasingly utilized in novel cellular therapies for a variety of blood disorders. This review uses clinical cases to highlight the multiple
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Financial toxicity in patients with leukemia undergoing hematopoietic stem cell transplantation: A systematic review Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-19 Orrin Pail, Thomas G. Knight
Financial toxicity (FT) is a term used to describe the objective financial burden of cancer care including the associated coping behaviors used by patients and their caregivers. FT has been shown to result in both direct financial burdens and in clinically relevant outcomes, such as non-adherence with care, diminished quality of life, and even decreased overall survival. Much of the data has been described
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Pharmacogenomics in allogeneic hematopoietic stem cell transplantation: Implications on supportive therapies and conditioning regimens Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-19 Sarah A. Morris, D. Grace Nguyen, Jai N. Patel
Allogeneic hematopoietic stem cell transplantation mortality has declined over the years, though prevention and management of treatment-related toxicities and post-transplant complications remains challenging. Applications of pharmacogenomic testing can potentially mitigate adverse drug outcomes due to interindividual variability in drug metabolism and response. This review summarizes clinical pharmacogenomic
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Measurable residual disease in patients undergoing allogeneic transplant for acute myeloid leukemia Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-18 Zoë C. Wong, Laura W. Dillon, Christopher S. Hourigan
The most common indication for allogeneic hematopoietic cell transplant (alloHCT) is maintenance of remission after initial treatment for patients with acute myeloid leukemia (AML). Loss of remission, relapse, remains however the most frequent cause of alloHCT failure. There is strong evidence that detectable persistent disease burden (“measurable residual disease”, MRD) in patients with AML in remission
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Dermatologic complications in transplantation and cellular therapy for acute leukemia Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-06 Shahab Babakoohi, Stephanie L. Gu, Hamid Ehsan, Alina Markova
Adoptive cellular immunotherapy, mainly hematopoietic stem cell transplant and CAR-T cell therapy have revolutionized treatment of patients with acute leukemia. Indications and inclusion criteria for these treatments have expanded in recent years. While these therapies are associated with significant improvements in disease response and overall survival, patients may experience adverse events from
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Allogeneic hematopoietic cell transplantation in acute myeloid leukemia Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-06 Gray Magee, Brittany Knick Ragon
Allogeneic hematopoietic cell transplantation (HCT) is a curative treatment modality for select patients with acute myeloid leukemia (AML), functioning as a restorative agent following intensified chemo- and/or radiotherapy and also engendering the disease-directed immunologic threat of graft-versus-leukemia effect. Advancements in conditioning regimen intensity, donor availability, and supportive
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Cardio-oncology and transplantation for acute myeloid leukemia Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-06 Jai Singh
Despite the rapidly evolving treatment landscape for acute myeloid leukemia (AML), allogeneic hematopoietic cell transplantation (allo-HCT) remains an important and potentially curative treatment option for many high-risk AML patients. Cardiovascular disease is an important competing risk throughout allo-HCT and a key driver of morbidity and mortality long after treatment. Cardio-oncology is a new
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Diagnosis and management of mature B-cell lymphomas in children, adolescents, and young adults Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-04-02 Stanton Goldman, Mitchell S. Cairo
Mature B-cell lymphoma in children, adolescents and young adults comprises three major histological subtypes including in order of frequency Burkitt, germinal center diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma. The cure rate of the first two with aggressive short chemotherapy based on clinical grouping is ∼90% in resource rich countries. Recent data has shown that incorporation
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Editorial Board / Aims & Scope Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-03-10
Abstract not available
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Worldwide sources of data in haematology: Importance of clinician-biostatistician collaboration Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-03-01 Kristin M. Page, Stephen R. Spellman, Brent R. Logan
The field of haematology has benefitted greatly from registry-based observational research. Medical and technical advances, changes in regulations and events such as the global pandemic is changing the landscape for registries. This review describes features of high-quality registries, statistical approaches and study design needed, an overview of worldwide hematologic registries, and how registries
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Diagnosis and management of lymphoblastic lymphoma in children, adolescents and young adults Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-16 William C. Temple, Stephanie Mueller, Michelle L. Hermiston, Birgit Burkhardt
Lymphoblastic lymphoma (LBL) is the second most common type of non-Hodgkin Lymphoma (NHL) in children, adolescents, and young adults (CAYA), accounting for 25–35% of all cases. T-lymphoblastic lymphoma (T-LBL) comprises 70–80% of cases, while precursor B-lymphoblastic lymphoma (pB-LBL) makes up the remaining 20–25% of cases. Event-free and overall survival (EFS and OS) for paediatric LBL patients both
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Diagnosis and management of cutaneous lymphomas and lymphoid proliferations in children, adolescents and young adults (CAYA) Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-10 Jamie Stokke, Annabelle Tann, Nader Kim El-Mallawany, Ilske Oschlies
Cutaneous lymphomas and lymphoid proliferations (LPD) in children, adolescents, and young adults (CAYA) are a heterogeneous group of lymphoid neoplasms that present formidable diagnostic challenges to clinicians and pathologists alike. Although rare overall, cutaneous lymphomas/LPD occur in real-world settings and awareness of the differential diagnosis, potential complications, and various therapeutic
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Cellular and humoral immunotherapy in children, adolescents and young adults with non-Hodgkin lymphoma Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-02 Yaya Chu, Aliza Gardenswartz, Caroline Diorio, Lianna J. Marks, Eric Lowe, David T. Teachey, Mitchell S. Cairo
The prognosis is dismal (2-year overall survival less than 25%) for childhood, adolescent, and young adult (CAYA) with relapsed and/or refractory (R/R) non-Hodgkin lymphoma (NHL). Novel targeted therapies are desperately needed for this poor-risk population. CD19, CD20, CD22, CD79a, CD38, CD30, LMP1 and LMP2 are attractive targets for immunotherapy in CAYA patients with R/R NHL. Novel anti-CD20 monoclonal
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Biopathology of childhood, adolescent and young adult non-Hodgkin lymphoma Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-04 Megan S. Lim, Michelle Foley, Lara Mussolin, Reiner Siebert, Suzanne Turner
Mature non-Hodgkin lymphomas (NHL) in the childhood, adolescent and young adult (CAYA) population are rare and exhibit unique clinical, immunophenotypic and genetic characteristics. Application of large-scale unbiased genomic and proteomic technologies such as gene expression profiling and next generation sequencing (NGS) have led to enhanced understanding of the genetic basis for many lymphomas in
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Diagnosis and management of post-transplant lymphoproliferative disease following solid organ transplantation in children, adolescents, and young adults Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-03 Jeremy Rubinstein, Keri Toner, Thomas Gross, Birte Wistinghausen
Post-transplant Lymphoproliferative Disease (PTLD) remains a major complication of solid organ transplantation (SOT) in pediatric patients. The majority are Epstein-Barr Virus (EBV) driven CD20+ B-cell proliferations responsive to reduction to immunosuppression and anti-CD20 directed immunotherapy. This review focusses on the epidemiology, role of EBV, clinical presentation, current treatment strategies
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Diagnosis and management of Hodgkin lymphoma in children, adolescents, and young adults Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-03 Jennifer A. Belsky, Jessica Hochberg, Lisa Giulino-Roth
Advances in the management of Hodgkin lymphoma in children, adolescents and young adult have resulted in survival outcomes exceeding 90%. The risk of late toxicity, however, remains a significant concern for survivors of HL and the focus of modern trials have been to advance cure rates while reducing long term toxicity. This has been accomplished through response-adapted treatment approaches and the
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Late effects and frontline treatment selection for children with non-Hodgkin lymphoma Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-03 Matthew J. Ehrhardt, Stephanie B. Dixon, Jennifer Belsky, Jessica Hochberg
Approximately 1 in 640 adults between 20 and 40 years of age is a survivor of childhood cancer. However, survival has often come at the expense of increased risk of long-term complications, including chronic health conditions and higher mortality rates. Similarly, long-term survivors of childhood non-Hodgkin lymphoma (NHL) experience significant morbidity and mortality related to prior cancer treatments
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Diagnosis and management of ALK-positive anaplastic large cell lymphoma in children and adolescents Best Pract. Res. Clin. Haematol. (IF 2.1) Pub Date : 2023-02-02 Charlotte Rigaud, Fabian Knörr, Laurence Brugières, Wilhelm Woessmann
Anaplastic lymphoma kinase (ALK)-positive anaplastic large cell lymphoma (ALCL) is a CD30-positive T cell lymphoma characterized by signalling from constitutively activated ALK fusion proteins. Most children and adolescents present in advanced stages, often with extranodal disease and B symptoms. The current front-line therapy standard of six cycles polychemotherapy reaches an event-free survival of