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

Withholding transfusion therapy in children with sickle cell disease with abnormal transcranial doppler and normal magnetic resonance angiography: a retrospective analysis
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2023.284506. Online ahead of print.ABSTRACTNot available.PMID:38450542 | DOI:10.3324/haematol.2023.284506 (Source: Haematologica)
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Lydian A De Ligt Karin Fijnvandraat Bart J Biemond Harriet Heijboer Source Type: research

Hematopoietic stem cell fate under the influence of Ser/Thr protein phosphatases
Haematologica. 2024 Mar 7. doi: 10.3324/haematol.2024.285040. Online ahead of print.ABSTRACTNot available.PMID:38450554 | DOI:10.3324/haematol.2024.285040 (Source: Haematologica)
Source: Haematologica - March 7, 2024 Category: Hematology Authors: Meritxell Alberich-Jorda Libor Macurek 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