Imatinib treatment and longitudinal growth in pediatric patients with chronic myeloid leukemia: Influence of demographic, pharmacological, and genetic factors in the German CML-PAED cohort
This study examines demographic, pharmacological, and genetic factors associated with impaired longitudinal growth in a uniform pediatric cohort treated with imatinib. We analyzed 94 pediatric patients with chronic myeloid leukemia (CML) diagnosed in the chronic phase and treated with imatinib for >12 months who participated in the Germany-wide CML-PAEDII study between February 2006 and February 2021. During imatinib treatment, significant height reduction occurred, with medians of -0.35 standard deviation score (SDS) at 12 months and -0.76 SDS at 24 months. Cumulative height SDS change (Δheight SDS) showed a more pron...
Source: Haematologica - March 18, 2024 Category: Hematology Authors: Sophie Stiehler Stephanie Sembill Oliver Schleicher Michaela Marx Manfred Rauh Manuela Krumbholz Axel Karow Meinolf Suttorp Joachim Woelfle Carlo Maj Markus Metzler Source Type: research
Validation of LymphGen classification on a 400-gene clinical next-generation sequencing panel in diffuse large B-cell lymphoma: real-world experience from a cancer center
Haematologica. 2024 Mar 14. doi: 10.3324/haematol.2023.284565. Online ahead of print.ABSTRACTNot available.PMID:38497151 | DOI:10.3324/haematol.2023.284565 (Source: Haematologica)
Source: Haematologica - March 18, 2024 Category: Hematology Authors: Meng-Lei Zhu Esther Drill Erel Joffe Gilles Salles Alfredo Rivas Delgado Andrew Zelenetz Maria Lia Palomba Maria Arcila Ahmet Dogan Source Type: research
Bendamustine and rituximab as first-line treatment for symptomatic splenic marginal zone lymphoma: long-term outcome and impact of early unmeasurable minimal residual disease attainment from the BRISMA/IELSG36 phase II study
Haematologica. 2024 Mar 14. doi: 10.3324/haematol.2023.284109. Online ahead of print.ABSTRACTNot available.PMID:38497158 | DOI:10.3324/haematol.2023.284109 (Source: Haematologica)
Source: Haematologica - March 18, 2024 Category: Hematology Authors: Emilio Iannitto Simone Ferrero C ôme Bommier Daniela Drandi Martina Ferrante Krimo Bouabdallah Sylvain Carras Guido Gini Vincent Camus Salvatrice Mancuso Luigi Marcheselli Angela Ferrari Michele Merli Benoit Tessoulin Caterina Stelitano Kheira Beldjord G Source Type: research
Classical meets malignant hematology: a case of acquired εγδβ-thalassemia in clonal hematopoiesis
Haematologica. 2024 Mar 14. doi: 10.3324/haematol.2024.285083. Online ahead of print.ABSTRACTHemoglobinopathies including thalassemias are among the most frequent genetic disorders worldwide. Primarily, these entities result from germline variants in the globin gene clusters and their cis-acting regulatory elements, and thus the WHO classifies thalassemias as inherited diseases. Non-inherited disorders of globin chain synthesis mimicking the phenotype of thalassemias have also been described and are referred to as acquired thalassemias. These forms mainly affect the alpha-globin genes and are observed at much lower frequen...
Source: Haematologica - March 18, 2024 Category: Hematology Authors: Armin P Piehler Marietta Truger Jan-Hendrik Kozik Sandra Weissmann Martin Schwonzen Manja Meggendorfer Wolfgang Kern Torsten Haferlach Gregor Hoermann Claudia Haferlach Source Type: research
Stroke without cerebral arteriopathy in sickle cell disease children: causes and treatment
In conclusion, in a cohort of pediatric SCD patients with efficient stroke screening strategy, half of occurring ischemic strokes were related to causes other than CA. They affected a different population of SCD children and systematic long-term transfusion programs may not be necessary in these cases.PMID:38497171 | DOI:10.3324/haematol.2023.283773 (Source: Haematologica)
Source: Haematologica - March 18, 2024 Category: Hematology Authors: Sarah Liane Linguet Suzanne Verlhac Florence Missud Laurent Holvoet-Vermaut Valentine Brousse Ghislaine Ithier Alexandra Ntorkou Emmanuelle Lesprit Malika Benkerrou Mano ëlle Kossorotoff Berengere Koehl Source Type: research
Impact and utility of follicular lymphoma GELF criteria in routine care: an Australasian Lymphoma Alliance study
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284538. Online ahead of print.ABSTRACTFollicular Lymphoma (FL) treatment initiation is largely determined by tumor burden and symptoms. In the pre-rituximab era, the Group d'Etude des Lymphomes Folliculaires (GELF) developed widely adopted criteria to identify high tumor burden FL patients to harmonize clinical trial populations. The utilization of GELF criteria (GELFc) in routine therapeutic decision-making is poorly described. This multicenter retrospective study evaluated patterns of GELFc at presentation and GELFc utilization in therapeutic decision-making in newly ...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Allison Barraclough Shivam Agrawal Dipti Talaulikar Geoffrey Chong Edward Yoo Chan Y Cheah Nunzio Franco Bianca Nguyen Howard Mutsando Fatima Tahir Judith Trotman Jing Huang Colm Keane Mitchel Lincoln Tara Cochrane Anna M Johnston Michael Dickinson Stephe Source Type: research
Functional and multi-omics signatures of mitapivat efficacy upon activation of pyruvate kinase in red blood cells from patients with sickle cell disease
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284831. Online ahead of print.ABSTRACTMitapivat, a pyruvate kinase (PK) activator, shows great potential as a sickle cell disease (SCD)- modifying therapy. Safety and efficacy of mitapivat as a long-term maintenance therapy is currently being evaluated in two open-label studies. Here we apply a comprehensive multi-omics approach to investigate the impact of activating PK on red blood cells (RBCs) from 15 SCD patients. HbSS patients were enrolled in one of the open label, extended studies (NCT04610866). Leuko-depleted RBCs obtained from fresh whole blood at baseline (vis...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Angelo D'Alessandro Kang Le Maureen Lundt Quan Li Emily B Dunkelberger Troy Cellmer Andrew J Worth Spurthi Patil Chris Huston Abby Grier Monika Dzieciatkowska Daniel Stephenson William A Eaton Swee Lay Thein Source Type: research
< em > HJV < /em > mutations causing hemochromatosis: variable phenotypic expression in a pair of twins
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284134. Online ahead of print.NO ABSTRACTPMID:38450514 | DOI:10.3324/haematol.2023.284134 (Source: Haematologica)
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Akhila Vadivelan Sarah Zhang Daniel N Srole Elizabeth A Marcus George Carvalho Neto Elizabeta Nemeth Tomas Ganz Satiro De Oliveira Source Type: research
Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284622. Online ahead of print.ABSTRACTThree major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges for pharmaceutical industry; c) refining patient eligibility criteria, and endpoints definition. Currently employed AAV vectors may cause toxicity and adverse events. Furthermore, studies in animals do not fully predict risks and clinical benefits of AAV-based GT, and animal models refl...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Giovanni Di Minno Wolfgang Miesbach Giancarlo Castaman Flora Peyvandi Source Type: research
Small extracellular vesicles derived from acute myeloid leukemia cells promote leukemogenesis by transferring miR-221-3p
In this study, we aimed to investigate the effects of AML cells-derived sEVs (AML-sEVs) on AML cells and delineate the underlying mechanisms. We initially used high-throughput sequencing to identify miR-221-3p as the miRNA prominently enriched in AML-sEVs. Our findings revealed that miR-221-3p promoted AML cell proliferation and leukemogenesis by accelerating cell cycle entry and inhibiting apoptosis. Furthermore, Gbp2 was confirmed as a target gene of miR-221-3p by dual luciferase reporter assays and rescue experiments. Additionally, AML-sEVs impaired the clonogenicity, particularly the erythroid differentiation ability, ...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Mengyu Li Guohuan Sun Jinlian Zhao Shuangshuang Pu Yanling Lv Yifei Wang Yapu Li Xiangnan Zhao Yajie Wang Shangda Yang Tao Cheng Hui Cheng Source Type: research
Prognostic impact of < em > SF3B1 < /em > mutation and multilineage dysplasia in myelodysplastic syndromes with ring sideroblasts: a Mayo Clinic study of 170 informative cases
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284719. Online ahead of print.ABSTRACTThe revised 4th edition of the World Health Organization (WHO4R) classification lists myelodysplastic syndromes with ring sideroblasts (MDS-RS) as a separate entity with single lineage (MDS-RS-SLD) or multilineage (MDS-RS-MLD) dysplasia. The more recent International Consensus Classification (ICC) distinguishes between MDS with SF3B1 mutation (MDS-SF3B1) and MDS-RS without SF3B1 mutation; the latter is instead included under the category of MDS not otherwise specified. The current study includes 170 Mayo Clinic patients with WHO4R-d...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Faiqa Farrukh Maymona Abdelmagid Abhishek Mangaonkar Mrinal Patnaik Aref Al-Kali Michelle A Elliott Kebede H Begna Christopher C Hook William J Hogan Animesh Pardanani Mark R Litzow Rhett P Ketterling Naseema Gangat Daniel A Arber Attilio Orazi Rong He Ka Source Type: research
Incidence and outcome of central nervous system relapse after hematopoietic stem cell transplantation in patients suffering from acute myeloid leukemia and acute lymphoblastic leukemia: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284858. Online ahead of print.ABSTRACTNot available.PMID:38450525 | DOI:10.3324/haematol.2023.284858 (Source: Haematologica)
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Sabine Blum Yves Chalandon Myriam Labopin J ürgen Finke Tobias Gedde-Dahl Tarek Ben Othman Jan J Cornelissen Pavel Jindra H élène Labussière-Wallet Matthew Collin Stig Lenhoff Guido Kobbe Norma C Guti érrez Arnon Nagler Mohamad Mohty Source Type: research
Health-related quality of life in patients with hematologic malignancies treated with chimeric antigen receptor T-cell therapy: review and current progress
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2022.282363. Online ahead of print.ABSTRACTChimeric antigen receptor (CAR) T-cell therapy has transformed the care of patients with relapsed/refractory B-cell derived hematologic malignancies. To date, six CAR T-cell therapies, targeting either CD19 or B-cell maturation antigen, have received regulatory approval. Along with the promising survival benefit, CAR T-cell therapy is associated with potentially lifethreatening adverse events (AE), including cytokine release syndrome and immune effector cellassociated neurotoxicity syndrome. While clinical trials evaluating CAR T-ce...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Emmanuelle Tchernonog Aline Moignet Am élie Anota Sophie Bernard Guy Bouguet Fanny Colin Catherine Rioufol Lo ïc Ysebaert Emmanuel Gyan Source Type: research
High-risk stays high-risk: Bruton tyrosine kinase inhibitors in B-cell malignancies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2024.285029. Online ahead of print.ABSTRACTNot available.PMID:38450529 | DOI:10.3324/haematol.2024.285029 (Source: Haematologica)
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Othman Al-Sawaf Source Type: research
Cell-free DNA from nail clippings as source of normal control for genomic studies in hematologic malignancies
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2024.285054. Online ahead of print.ABSTRACTComprehensive genomic sequencing is becoming a critical component in the assessment of hematologic malignancies, with broad implications for patient management. In this context, unequivocally discriminating somatic from germline events is challenging but greatly facilitated by matched analysis of tumor:normal pairs. In contrast to solid tumors, conventional sources of normal control (peripheral blood, buccal swabs, saliva) could be highly involved by the neoplastic process, rendering them unsuitable. In this work we describe our rea...
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Melissa Krystel-Whittemore Kseniya Petrova-Drus Ryan N Ptashkin Mark D Ewalt JinJuan Yao Ying Liu Menglei Zhu Jamal Benhamida Benjamin Durham Jyoti Kumar Khedoudja Nafa Iwona Kiecka Anita S Bowman Erika Gedvilaite Jacklyn Casanova Yun-Te Lin Abhinita S Mo Source Type: research
