journal
Journals Best Practice & Research. Clin...

Best Practice & Research. Clinical Haematology

https://read.qxmd.com/read/38490769/corrigendum-to-designing-and-conducting-a-clinical-trial-in-blood-and-marrow-transplantation-best-pract-res-clin-haematol-36-2023-101471
#1
Michael J Martens, Yan Gao, Aniko Szabo
No abstract text is available yet for this article.
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38490768/corrigendum-to-endpoint-selection-and-evaluation-in-hematology-studies-best-pract-res-clin-haematol-36-2023-101479
#2
Ruta Brazauskas, Mary Eapen, Tao Wang
No abstract text is available yet for this article.
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38490767/the-landscape-of-cytogenetic-and-molecular-genetic-methods-in-diagnostics-for-hematologic-neoplasia
#3
REVIEW
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 and molecular genetic methods, commonly used in the genetic characterization of hematologic malignancies, describe the current developments in the cytogenetic and molecular diagnostics, and give an outlook into their future development...
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38490766/providing-hematopoietic-stem-cell-products-from-unrelated-donors-to-the-world-dkms-donor-centers-and-dkms-registry
#4
REVIEW
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 in 7 countries with a total of 12.2 million donors and over 114,000 donations so far, and a multinational donor registry...
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38490765/germline-predisposition-to-myeloid-neoplasms-characteristics-and-management-of-high-versus-variable-penetrance-disorders
#5
REVIEW
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. In this review, we will shed light on high malignant penetrance (e.g., CEBPA, GATA2, SAMD9/SAMD9L, and TP53) versus variable malignant penetrance syndromes (e...
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38490764/using-real-world-evidence-in-haematology
#6
REVIEW
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 real world data (RWD). Recently, real-world evidence (RWE) from RWD processed by artificial intelligence has received increasing attention. We describe the potential of using RWD in haematology concluding RWE from RWD may complement data from RCTs to support regulatory decisions...
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38490763/cytogenetics-and-genomics-of-acute-myeloid-leukemia
#7
REVIEW
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 international classification systems. Additionally, the detection of new mutational profiles at relapse and identification of mutations in the pre- and post-transplant settings are illuminating in understanding disease evolution and are relevant to the risk assessment of AML patients...
March 2024: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092485/cytogenetics-and-genomics-in-pediatric-acute-lymphoblastic-leukaemia
#8
REVIEW
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 for BCR::ABL1-positive ALL. Further advances in the taxonomy of ALL and the discovery of new genetic biomarkers and therapeutic targets, as well as the introduction of targeted and immunotherapies into the frontline treatment protocols, may improve management and outcome of children with ALL...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092484/when-to-use-which-molecular-prognostic-scoring-system-in-the-management-of-patients-with-mds
#9
REVIEW
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 conventional clinical prognostic systems including the International Prognostic Scoring System (IPSS) and its revised version (IPSS-R) have dominated the risk stratification of MDS from 1997 until 2022...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092483/cytogenomics-of-b-cell-non-hodgkin-lymphomas-the-old-meets-the-new
#10
REVIEW
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 of lymphoid neoplasms. Some of the B-cell non-Hodgkin lymphomas are characterized by particular chromosomal aberrations, highlighting the relevance of conventional and molecular cytogenetic studies in their diagnosis and prognosis...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092482/the-role-of-randomized-controlled-trials-registries-observational-databases-in-evaluating-new-interventions
#11
REVIEW
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 done for practical or (seemingly) ethical reasons. There are many examples of an unproved intervention being so widely-believed to be effective that clinical trialists and potential subjects decline randomization...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092481/best-practice-research-clinical-haematology-screening-for-breast-cancer-in-hodgkin-lymphoma-survivors
#12
REVIEW
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 treatment fields, cohort studies have documented a cumulative incidence of breast cancer of 10-20% by 40 years of age...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092480/progress-and-challenges-in-the-acute-leukemia-field
#13
EDITORIAL
Daniel A Pollyea
No abstract text is available yet for this article.
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092479/the-international-cooperative-gaucher-group-iccg-gaucher-registry
#14
REVIEW
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 heterogeneous patient populations, to annotate phenotypes and genotypes that define the natural history of GD in untreated patients, and to document and analyze treatment outcomes for alglucerase and any other future treatments...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092478/mechanisms-of-resistance-to-hypomethylating-agents-and-bcl-2-inhibitors
#15
REVIEW
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 BCL-2 inhibitor, venetoclax, is approved for AML induction in patients above 75 years and is also actively being investigated for use in high risk MDS...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092477/the-best-gvhd-prophylaxis-or-at-least-progress-towards-finding-it
#16
REVIEW
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 or more broadly suppressing inflammation have all been used. Interventions which spare regulatory T cells (Tregs) may prevent GVHD and facilitate controlled allo-responses and not compromise subsequent relapse risks...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092476/asparaginase-dosing-for-obese-patients-with-acute-lymphoblastic-leukemia-and-factors-that-contribute-to-outcomes
#17
REVIEW
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 regimens that include asparaginase. In this review, the most salient findings are highlighted from these latest publications, both from the pediatric and adult literature...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092475/the-australian-aplastic-anaemia-and-other-bone-marrow-failure-syndromes-registry
#18
REVIEW
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 a powerful tool to identify areas of need and support clinical and research collaboration. Novel treatment strategies such as gene therapy, particularly in rare diseases, will depend on the ability to identify eligible patients alongside the molecular genetic features of their disease that may be amenable to novel therapy...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092474/cardiac-surgery-for-radiation-associated-heart-disease-in-hodgkin-lymphoma-patients
#19
REVIEW
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 pathologies. Despite possessing a greater mortality risk than in the general population, cardiac surgery has an important role in treating radiation-associated heart disease...
December 2023: Best Practice & Research. Clinical Haematology
https://read.qxmd.com/read/38092473/hodgkin-lymphoma-a-disease-shaped-by-the-tumor-micro-and-macroenvironment
#20
REVIEW
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 immediate surrounding and within the tumor. Effects of cancer cells at a distance or outside of the tumor define the TMacroE...
December 2023: Best Practice & Research. Clinical Haematology
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