journal
https://read.qxmd.com/read/38297484/quantification-of-midostaurin-in-plasma-and-serum-by-stable-isotope-dilution-liquid-chromatography-tandem-mass-spectrometry-application-to-a-cohort-of-patients-with-acute-myeloid-leukemia
#21
JOURNAL ARTICLE
Martin H J Wiesen, Jannik Stemler, Cornelia Fietz, Carolin Joisten, Oliver A Cornely, Nick Verougstraete, Thomas Streichert, Carsten Müller
OBJECTIVES: Midostaurin is an oral multitargeted tyrosine kinase inhibitor for the treatment of acute myeloid leukemia (AML). Therapeutic drug monitoring of midostaurin may support its safe use when suspecting toxicity or combined with strong CYP3A4 inhibitors. METHODS: A stable isotope dilution liquid chromatography-tandem mass spectrometry method was developed and validated for the determination and quantification of midostaurin in human plasma and serum. Midostaurin serum concentrations were analyzed in 12 patients with FMS-like tyrosine kinase 3 (FLT3)-mutated AML during induction chemotherapy with cytarabine, daunorubicin, and midostaurin...
January 31, 2024: European Journal of Haematology
https://read.qxmd.com/read/38294126/myelodysplastic-syndromes-del-5q-pathogenesis-and-its-therapeutic-implications
#22
REVIEW
Antonella Bruzzese, Enrica Antonia Martino, Francesco Mendicino, Eugenio Lucia, Virginia Olivito, Isabella Capodanno, Antonino Neri, Fortunato Morabito, Ernesto Vigna, Massimo Gentile
Myelodysplastic syndromes (MDS) encompass a heterogeneous set of acquired bone marrow neoplastic disorders characterized by ineffective hematopoiesis within one or more bone marrow lineages. Nearly half of MDS patients carry cytogenetic alterations, with del(5q) being the most prevalent. Since its first description, del(5q) was consistently correlated with a typical clinical phenotype marked by anemia, thrombocytosis, and a low risk of evolving into acute leukemia. Presently, the World Health Organization (WHO) classification of myeloid neoplasms recognizes a specific subtype of MDS known as "myelodysplastic neoplasm with low blast and isolated del(5q)" identified by the sole presence of 5q deletion or in combination with one other abnormality excluding -7/del(7q)...
January 31, 2024: European Journal of Haematology
https://read.qxmd.com/read/38294085/the-applicability-of-the-central-line-associated-bloodstream-infection-clabsi-criteria-for-the-evaluation-of-bacteremia-episodes-in-pediatric-oncology-patients
#23
JOURNAL ARTICLE
Ceder H van den Bosch, Florine N J Frakking, Yvette G T Loeffen, Harm van Tinteren, Alida F W van der Steeg, Marc H W A Wijnen, Marianne D van de Wetering, Jan-Tom van der Bruggen
BACKGROUND: The aim of this study was to investigate the applicability of the central line-associated bloodstream infection (CLABSI) criteria of the Centers for Disease Control and Prevention in pediatric oncology patients. METHODS: Bacteremia episodes from 2020 to 2022 from a prospective cohort of pediatric oncology patients with a central venous catheter were included. Episodes were classified by three medical experts following the CLABSI criteria as either a CLABSI or non-CLABSI (i...
January 31, 2024: European Journal of Haematology
https://read.qxmd.com/read/38243840/late-transplant-associated-thrombotic-microangiopathy-verified-in-bone-marrow-biopsy-specimens-is-associated-with-chronic-gvhd-and-viral-infections
#24
JOURNAL ARTICLE
Wolfgang Hill, Karl Sotlar, Anke Hautmann, Hans-Jochem Kolb, Johanna Ullmann, Andreas Hausmann, Michael Schmidt, Johanna Tischer, Thu-Trang Pham, Andreas Rank, Manuela A Hoechstetter
OBJECTIVES: To describe late transplant-associated thrombotic microangiopathy (TA-TMA) as chronic endothelial complication in bone marrow (BM) after allogeneic hematopoietic stem cell transplantation (HSCT). METHODS: BM specimens along with conventional diagnostic parameters were assessed in 14 single-institutional patients with late TA-TMA (more than 100 days after HCST), including 11 late with history of early TA-TMA, 10 with early TA-TMA (within 100 days), and 12 non TA-TMA patients...
January 20, 2024: European Journal of Haematology
https://read.qxmd.com/read/38226781/jak2v617f-drives-gut-microbiota-differences-in-patients-with-myeloproliferative-neoplasms
#25
JOURNAL ARTICLE
Christina Schjellerup Eickhardt-Dalbøge, Henrik V Nielsen, Kurt Fuursted, Christen Rune Stensvold, Lee O' Brien Andersen, Berit Lilje, Morten Kranker Larsen, Lasse Kjaer, Sarah Friis Christensen, Trine Alma Knudsen, Vibe Skov, Anders Lindholm Sørensen, Christina Ellervik, Lars Rønn Olsen, Jens Jørgen Elmer Christensen, Xiaohui Chen Nielsen, Hans Carl Hasselbalch, Anna Cäcilia Ingham
BACKGROUND: Essential thrombocythemia (ET), polycythemia vera (PV), and primary myelofibrosis (MF) are myeloproliferative neoplasms (MPN). Inflammation is involved in the initiation, progression, and symptomology of the diseases. The gut microbiota impacts the immune system, infection control, and steady-state hematopoiesis. METHODS: We analyzed the gut microbiota of 227 MPN patients and healthy controls (HCs) using next-generation sequencing. We expanded our previous results in PV and ET patients with additional PV, pre-MF, and MF patients which allowed us to compare MPN patients collectively, MPN sub-diagnoses, and MPN mutations (separately and combined) vs...
January 16, 2024: European Journal of Haematology
https://read.qxmd.com/read/38223989/pattern-of-use-and-clinical-outcomes-with-rix-fp-in-pediatric-adolescent-patients-with-haemophilia-b-in-italy-results-from-ideal-real-world-study
#26
JOURNAL ARTICLE
Paola Giordano, Berardino Pollio, Gianluca Sottilotta, Chiara Biasoli, Filomena Daniele, Raimondo De Cristofaro, Flora Peyvandi, Maria Rosaria Villa, Giancarlo Castaman
OBJECTIVES: To evaluate pattern of use and clinical outcomes in pediatric/adolescent patients enrolled in the IDEAL study. METHODS: This post-hoc analysis of IDEAL retrospective-prospective observational study focused on patients <18 years, 100% on prophylaxis during the entire observation period. RESULTS: Thirteen subjects (median age 10.0 years; 61.5% ≤ 11 years) were analyzed. The infusion frequency changed from 2/week in 84...
January 15, 2024: European Journal of Haematology
https://read.qxmd.com/read/38213291/comparison-of-telomere-length-in-patients-with-bone-marrow-failure-syndromes-and-healthy-controls
#27
JOURNAL ARTICLE
Aruna Barade, Kavitha M Lakshmi, Anu Korula, Fouzia N Abubacker, Uday P Kulkarni, Aby Abraham, Vikram Mathews, Biju George, Eunice S Edison
INTRODUCTION: During normal aging, telomeric DNA is gradually lost in dividing somatic cells, and critically short telomeres lead to replicative senescence, apoptosis, or chromosomal instability. We studied telomere length in bone marrow failure syndromes (BMFS) compared to normal healthy population. METHODS: Peripheral blood was collected from the participants, and genomic DNA was extracted. Relative telomere length was measured using a quantitative polymerase chain reaction...
January 12, 2024: European Journal of Haematology
https://read.qxmd.com/read/38200687/soluble-mannose-receptor-a-potential-biomarker-in-gaucher-disease
#28
JOURNAL ARTICLE
Brendan Beaton, Derralynn A Hughes
PURPOSE: Soluble mannose receptor (sMR) relates to mannose receptor expression on macrophages, and is elevated in inflammatory disorders. Gaucher disease (GD) has altered macrophage function and utilises mannose receptors for enzyme replacement therapy (ERT) endocytosis. sMR has not previously been studied in GD. METHODS: sMR was measured by ELISA and correlated with GD clinical features including spleen and liver volume, haemoglobin and platelet count, bone marrow burden (BMB) scores and immunoglobulin levels...
January 10, 2024: European Journal of Haematology
https://read.qxmd.com/read/38197567/droplet-digital-pcr-for-sensitive-relapse-detection-in-acute-myeloid-leukaemia-patients-transplanted-by-reduced-intensity-conditioning
#29
JOURNAL ARTICLE
Jonas Kassow Gronlund, Christopher Veigaard, Caroline Juhl-Christensen, Anne-Sofie Skou, Dorte Melsvik, Hans Beier Ommen
INTRODUCTION: Follow-up after allogeneic transplantation in acute myeloid leukaemia (AML) is guided by measurable residual disease (MRD) testing. Quantitative polymerase chain reaction (qPCR) is the preferred MRD platform but unfortunately, 40%-60% of AML patients have no high-quality qPCR target. This study aimed to improve MRD testing by utilising droplet digital PCR (ddPCR). ddPCR offers patient-specific monitoring but concerns of tracking clonal haematopoiesis rather than malignant cells prompt further validation...
January 10, 2024: European Journal of Haematology
https://read.qxmd.com/read/38193596/a-post-hoc-analysis-of-protect-viii-kids-assessing-long-term-efficacy-and-safety-of-damoctocog-alfa-pegol-in-adolescents-with-severe-haemophilia-a
#30
JOURNAL ARTICLE
Sanjay Ahuja, Tina Biss, Monika Maas Enriquez, Maria Elisa Mancuso, MacGregor Steele, Gili Kenet
INTRODUCTION: The safety and efficacy of the extended half-life factor VIII (FVIII) product damoctocog alfa pegol (BAY 94-9027, Jivi®) has been demonstrated in the PROTECT VIII Kids study (NCT01775618), where male previously-treated patients (PTPs) aged <12 years old with severe haemophilia A and ≥ 50 exposure days (EDs) were treated prophylactically. The PROTECT VIII Kids extension study assessed the long-term safety and efficacy of damoctocog alfa pegol in the same population...
January 9, 2024: European Journal of Haematology
https://read.qxmd.com/read/38192186/transcriptome-analysis-of-primary-adult-b-cell-lineage-acute-lymphoblastic-leukemia-identifies-pathogenic-variants-and-gene-fusions-and-predicts-subtypes-for-in-depth-molecular-diagnosis
#31
JOURNAL ARTICLE
Mirjam Podgorica, Elsa Drivet, Jonas Krag Viken, Alyssa Richman, Johanne Vestbøstad, Peter Szodoray, Ann Kristin Kvam, Hilde Skuterud Wik, Geir E Tjønnfjord, Ludvig A Munthe, Seth Frietze, Hilde Schjerven
BACKGROUND: B-cell acute lymphoblastic leukemia (B-ALL) is classified into subgroups based on known driver oncogenes and molecular lesions, including translocations and recurrent mutations. However, the current diagnostic tests do not identify subtypes or oncogenic lesions for all B-ALL samples, creating a heterogeneous B-ALL group of unknown subtypes. METHODS: We sorted primary adult B-ALL cells and performed transcriptome analysis by bulk RNA sequencing (RNA-seq)...
January 8, 2024: European Journal of Haematology
https://read.qxmd.com/read/38183302/trends-in-underlying-causes-of-death-in-allogeneic-hematopoietic-cell-transplant-recipients-over-the-last-decade
#32
JOURNAL ARTICLE
Andreas Søborg, Joanne Reekie, Henrik Sengeløv, Caspar Da Cunha-Bang, Thomas Kromann Lund, Christina Ekenberg, Isabelle Paula Lodding, Kasper Sommerlund Moestrup, Louise Lundgren, Jens D Lundgren, Neval Ete Wareham
OBJECTIVES: Improved survival after hematopoietic cell transplantation (HCT) and an increasingly comorbid transplant population may give rise to new trends in the causes of death. METHODS: This study includes all adult allogeneic HCT recipients transplanted at Rigshospitalet between January 1, 2010 and December 31, 2019. Underlying causes of death were determined using the Classification of Death Causes after Transplantation (CLASS) method. RESULTS: Among 802 HCT recipients, 289 died during the study period...
January 6, 2024: European Journal of Haematology
https://read.qxmd.com/read/38183299/risk-factors-for-hemorrhagic-cystitis-after-allogeneic-hematopoietic-stem-cell-transplantation-in-a-letermovir-exposed-cmv-free-population-receiving-ptcy
#33
JOURNAL ARTICLE
Eugenio Galli, Elisabetta Metafuni, Carlo Gandi, Maria Assunta Limongiello, Sabrina Giammarco, Andrea Mattozzi, Rosaria Santangelo, Andrea Bacigalupo, Federica Sorà, Patrizia Chiusolo, Simona Sica
Hemorrhagic cystitis (HC) is a highly impacting complication in allogeneic hematopoietic stem cell transplantation (HSCT), occurring in 12%-37% of patients. The impact of transplant- and patient-specific variables has been described, with a possible role for JCV and BKV, which may be cooperating with cytomegalovirus (CMV). Here, we analyze 134 letermovir-exposed, CMV-free patients, treated with the same cyclophosphamide-based graft-versus-host disease (GVHD) prophylaxis, describing risk factors for HC. The overall incidence of HC was 23%...
January 6, 2024: European Journal of Haematology
https://read.qxmd.com/read/38168871/feasibility-accuracy-and-usability-analysis-of-mapaml-a-first-in-class-app-for-integrated-diagnosis-in-acute-myeloid-leukemia
#34
JOURNAL ARTICLE
Thaís B Moyen, Victoria Tomaz, Paulo V Campregher
Performing a comprehensive diagnosis of acute myeloid leukemia (AML) is complex and involves the integration of clinical information, bone marrow morphology, immunophenotyping, cytogenetic, and molecular analysis, which can be challenging to the general hematologist. The aim of this study was to evaluate the usability and accuracy of MapAML, a smartphone app for integrated diagnosis in AML, created to aid the hematologist in its clinical practice. App performance was evaluated in dedicated sessions, in which 21 hematologists or fellows in hematology performed an integrated diagnosis of deidentified real-world clinical AML cases, first without and posteriorly with MapAML use...
January 3, 2024: European Journal of Haematology
https://read.qxmd.com/read/38168033/current-development-of-chimeric-antigen-receptor-t-cell-therapy-for-diffuse-large-b-cell-lymphoma-and-high-grade-b-cell-lymphoma
#35
REVIEW
Nobuhiko Yamauchi, Dai Maruyama
Chimeric antigen receptor (CAR) T-cell therapy has become a commercially available treatment option for relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) with two or more lines of prior therapies, and recently for high-risk r/r DLBCL with one prior line of therapy. The successful development of CAR T-cell therapy for multiple relapsed DLBCL has led to a boom in subsequent trials that investigated its utility in patients with other r/r B-cell lymphoma subtypes. However, CAR T-cell therapy is a multistep process that includes leukapheresis and manipulation which take several weeks...
January 3, 2024: European Journal of Haematology
https://read.qxmd.com/read/38164819/relevance-of-different-prognostic-scores-in-primary-cns-lymphoma-in-the-era-of-intensified-treatment-regimens-a-retrospective-multicenter-analysis-of-174-patients
#36
JOURNAL ARTICLE
Vanja Zeremski, Louisa Adolph, Sina Beer, Mirjeta Berisha, Benedikt Jacobs, Christoph Kahl, Christian Koenecke, Siegfried Kropf, Jens Panse, Judith Petersen, Martin Schmidt-Hieber, Jessica Schneider, Vladan Vucinic, Jeanette Walter, Oliver Weigert, Hanno M Witte, Dimitrios Mougiakakos
OBJECTIVES: Treatment intensification (including consolidative high-dose chemotherapy with autologous stem cell transplantation [HDT-ASCT]) significantly improved outcome in primary central nervous system lymphoma (PCNSL) patients. METHODS: We conducted a multicenter, retrospective analysis of newly diagnosed PCNSL patients, treated with intensified treatment regimens. The following scores were evaluated in terms of overall survival (OS) and progression-free survival (PFS): Memorial Sloan-Kettering Cancer Center (MSKCC), International Extranodal Lymphoma Study Group (IELSG), and three-factor (3F) prognostic score...
January 2, 2024: European Journal of Haematology
https://read.qxmd.com/read/37985225/chronic-myeloid-leukemia-cml-in-children-and-adolescents-clinicopathological-findings
#37
JOURNAL ARTICLE
Louis Nevejan, Veerle Labarque, Nancy Boeckx
BACKGROUND: Barely two per million Belgian children/adolescents are diagnosed with chronic myeloid leukemia (CML) annually. In this retrospective study, we aimed to investigate the diagnostic features, clinical and laboratory characteristics, and treatment outcome of this rare entity. METHODS: Medical records of all pediatric CML patients (age ≤ 17 years) diagnosed at the University Hospitals Leuven between 1986 and 2021 were reviewed...
March 2024: European Journal of Haematology
https://read.qxmd.com/read/37929654/real-world-outcomes-with-immunosuppressive-therapy-for-aplastic-anemia-in-patients-treated-at-the-university-of-michigan
#38
JOURNAL ARTICLE
Charles E Foucar, Daniel H Foley, Jessica Aldous, Patrick W Burke, Kristen R Pettit, Lydia L Benitez, Anthony J Perissinotti, Bernard L Marini, Philip Boonstra, Dale L Bixby
Aplastic anemia (AA) is a rare bone marrow failure disorder that is treated with either allogeneic stem cell transplant or immunosuppressive therapy (IST) consisting of antithymocyte globulin (ATG), cyclosporine (CSA), and eltrombopag. While outcomes are favorable in younger patients, older patients (>60) have significantly worse long-term survival. The dose of ATG is often reduced in older patients and those with multiple comorbidities given concerns for tolerability. The efficacy and safety of dose-attenuated IST in this population is largely undescribed...
March 2024: European Journal of Haematology
https://read.qxmd.com/read/38105392/chimeric-antigen-receptor-t-cell-therapy-where-are-we-now-and-where-are-we-heading-for
#39
EDITORIAL
Liang Wang, Heng Mei
No abstract text is available yet for this article.
January 2024: European Journal of Haematology
https://read.qxmd.com/read/38105391/determinants-of-outcomes-and-advances-in-cd19-directed-chimeric-antigen-receptor-therapy-for-b-cell-acute-lymphoblastic-leukemia
#40
REVIEW
Supriya Gupta, Mira Kohorst, Hassan B Alkhateeb
Relapsed and refractory B-cell acute lymphoblastic leukemia (B-ALL) is an aggressive B-cell neoplasm associated with poor outcomes. Conventional multiagent chemotherapy and bispecific antibody therapy may induce remission; however, relapse rates remain high and overall survival is poor. Chimeric antigen receptor T-cell (CAR-T) therapy provides durable, deep complete remission, and long-term cures in relapsed and refractory B-ALL. However, with this new treatment modality, 10%-30% of patients do not achieve remission, and over 50% experience relapse after therapy...
January 2024: European Journal of Haematology
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