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Plasma cell leukemia (PCL) is a rare and aggressive plasma cell disorder with a short survival duration despite the use of novel therapeutics. PCL remains an understudied disease for which there is no current standard of care treatment. Knowledge on optimal novel drug sequencing, including the role and timing of hematopoietic stem cell transplant as primary and salvage therapy is needed. We conducted

Myelofibrosis (MF) is a progressive disease characterized by accumulation of extracellular matrix (ECM) in the bone marrow (BM) which impairs normal hematopoiesis. While Janus kinase (JAK) inhibitors reduce spleen volume and provide symptomatic improvement, they do not resolve BM fibrosis and may cause or exacerbate anemia and thrombocytopenia. An anti-fibrotic therapy capable of normalising BM microenvironment

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Novel agents inducing deeper responses have improved the prognosis of patients with multiple myeloma (MM). To assess minimal residual disease (MRD) and stratify patients achieving complete response (CR), advanced technologies such as EuroFlow next-generation flow cytometry (NGF) and next-generation sequencing (NGS) are increasingly utilized. This prospective study evaluated responses in newly diagnosed

Anti-CD47 antibodies targeting macrophage immune checkpoints demonstrate benefit in clinical trial, particularly in combination with targeted therapies. This strategy faces challenges from suboptimal efficacy and on-target toxicity due to immunosuppressive tumor microenvironment (TME) and ubiquitous CD47 expression. Here, we report a novel oncolytic vaccine virus (OVV) that expressed therapeutic transgenes

CD47 overexpression has been associated with tumor cell survival. We present the safety, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of evorpacept, a novel fusion protein comprising a high-affinity CD47-SIRPα immune checkpoint inhibitor to promote tumor-cell phagocytosis and inactive Fc domain to spare healthy cells, plus rituximab in patients with relapsed/refractory B-cell

Infections are an important cause of morbidity and mortality in patients with lower-risk myelodysplastic syndromes (LR-MDS). Studies regarding risk factors for infections are, however, limited in this population. This study aimed to investigate the prevalence and risk factors for infections and infection-related death in patients with LR-MDS. This study included 2552 patients from the European MDS

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Chromosomal translocation of the KMT2A gene represents the cytogenetic hallmark of acute lymphoblastic leukemia diagnosed in infants (<1 year of age), driving a highly aggressive malignancy. For decades the event-free survival rates for these very young patients were at best ~40%. However, recent advances adding immunotherapy in the form of the bi-specific T-cell engager blinatumomab to the treatment

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The multi-center randomized phase 3 NHL-004 study compared etoposide, dexamethasone and pegaspargase (ESA) versus methotrexate, etoposide, dexamethasone and pegaspargase (MESA) regimen, combined with sandwiched radiotherapy, in newly diagnosed early-stage nasal natural killer/T-cell lymphoma (NKTCL). Here we report the long-term outcomes (median follow-up, 64 months) and biomarker analysis. 256 eligible

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Advances in HLA typing, conditioning regimens, GVHD prophylaxis/treatment, and supportive care have led to significant improvement in survival after allogeneic hematopoietic cell transplantation (alloHCT). Despite this progress, disease relapse after transplantation remains a daunting challenge and continues to be a major driver of mortality in patients with acute myeloid leukemia (AML). To assess

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Desmopressin increases plasma factor VIII and von Willebrand factor in non-severe hemophilia A (Hem A) and type 1 von Willebrand disease (VWD), following intravenous infusions, subcutaneous injections or nasal spray. This medication, with almost 50 years of clinical experience and proven safety and efficacy, is often unavailable. Thus, there is a need to establish how many patients can benefit from

Non-factor therapies are changing the treatment paradigm in hemophilia A, which was previously dominated by replacement-therapy using factor VIII (FVIII)-concentrates. However, the FVIIIequivalence of these new therapies has remained unclear, since in vitro assays generate variable responses. Here we used four different in vivo bleeding models to compare FVIII to emicizumab and to a sequence-identical

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Functional high-risk (FHR) multiple myeloma (MM) patients, defined as those with early relapse despite optimal initial therapy, represent an unmet clinical need. Diffusionweighted whole-body MRI (DW-MRI) is increasingly used in MM management due to its high sensitivity in assessing treatment response. The Myeloma Response Assessment and Diagnosis System (MY-RADS) established the Response Assessment

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Acute leukaemias represent the 1st cause of cancer in children. Their prognosis has improved significantly due to remarkable advances in therapeutic management, despite the risk of long-term consequences, especially for patients who underwent allogenic hematopoietic stem cell transplantation (aHSCT). Through the Leukaemia in Children and Adolescents (LEA) long-term follow-up cohort, we conducted a

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The main objective of the current study was to provide a detailed account on the prognostic relevance of abnormal karyotype (AK) and associated specific cytogenetic abnormalities in polycythemia vera (PV). 669 PV patients were informative, of which 436 (65%) were evaluated within 1 year of diagnosis. Karyotype was abnormal in 67 (15%) patients, including isolated abnormalities of loss of Y chromosome

Megakaryocytes (MKs) are canonically viewed as specialized hematopoietic cells merely producing platelets and generated through a series of hematopoietic stem and progenitor cells in the bone marrow (BM). Despite essential physiological functions, the generation and function of MKs remain incompletely understood. Recent studies, almost in mice, have begun to redefine the cellular hierarchy of megakaryopoiesis

Accurate measurements of the platelet count are necessary to diagnose thrombocytosis or thrombocytopenia correctly, gauge the severity of the clinical risk and identify the most appropriate therapeutic intervention. Despite increased diagnostic accuracy with the electronic counters, it is still unsatisfactory in rare situations. Conditions causing spurious thrombocytopenia include the following. 1)

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Patients with multiple myeloma meeting frailty criteria have worse outcomes than those identified as non-frail. Here, we present a post-hoc subgroup analysis of IMROZ, a global, phase III, open-label study investigating isatuximab (Isa) with bortezomib, lenalidomide, and dexamethasone (VRd) followed by Isa-Rd (n=265) versus VRd followed by Rd (n=181) in newly diagnosed transplant-ineligible multiple

Asparaginase (ASNase)-based chemotherapy regimens significantly improve survival outcomes in children, adolescent and young adult (AYA), and even adults with acute lymphoblastic leukemia/lymphoma (ALL); however, the incidence and severity of ASNase-associated adverse events (AEs) in adults may differ significantly from those reported in children. Strategies to mitigate, monitor for, and manage toxicities

We compared long-term outcomes in 78 patients with steroid-refractory acute graft-versushost disease (SR-aGVHD) treated at the University Medical Center Hamburg, Germany between December 2015 and August 2022 who received either ruxolitinib alone (Ruxo, n=29) or Ruxo plus extracorporeal photopheresis (Ruxo-ECP, n=49). Patients were well balanced between both arms except for SR-aGVHD grade IV which was

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QuANTUM-First (NCT02668653) was a randomized phase 3 trial in newly diagnosed FLT3-ITDQpositive acute myeloid leukemia (AML) patients treated with quizartinib or placebo plus standard induction and consolidation chemotherapy and/or allogeneic hematopoietic cell transplantation (allo-HCT), followed by single-agent maintenance therapy. We evaluated the impact of allo-HCT performed in first complete remission

Hemophilia is a rare X-linked bleeding disorder caused by mutations in the F8 or F9 gene (hemophilia A or B), leading to deficient factor VIII or IX proteins, respectively. Hemophilia-related complications caused by bleeding into the joints (the hallmark of hemophilia) and age-related comorbidities occur frequently and impact the functionality and quality of life of persons with hemophilia (PwH). Given

Lisocabtagene maraleucel (liso-cel) and axicabtagene ciloleucel (axi-cel) are FDA- and EMAapproved chimeric antigen receptor (CAR) T-cell therapies for relapsed/refractory large B-cell lymphoma (LBCL). However, comparative real-world analyses of their efficacy and toxicity with extended follow-up are lacking. We conducted a retrospective study of 160 LBCL patients treated at the Fred Hutchinson Cancer

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Patients with severe hemophilia A (HA) often develop undesired immune responses to therapeutic factor VIII (FVIII) that hamper replacement therapy with FVIII-derived products. The transplacental delivery of two Fc-fused FVIII domains in pregnant HA mice was shown to induce partial FVIII-specific immune tolerance in the offspring. Here, we evaluated whether the transplacental delivery of Fc-fused FVIII

Continuous treatment with ibrutinib not only exerts tumor control but also enhances T cell function in patients with chronic lymphocytic leukemia (CLL). We conducted longitudinal multi-omics analyses in samples from CLL patients receiving ibrutinib upfront to identify potential adaptive mechanisms to Bruton tyrosine kinase (BTK) inhibition during the first 12 months of continuous therapy. We found

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Mutations in the NPM1 gene (NPMc+) and in the FLT3 gene (FLT3-ITD) represent the most frequent co-occurring mutations in Acute Myeloid Leukemia (AML), yet the cellular and molecular mechanisms of their cooperation remain largely unexplored. Using mouse models that faithfully recapitulate human AML, we investigated the impact of these oncogenes on pre-leukemic and leukemic hematopoietic stem cells (HSCs)

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Bernard Soulier Syndrome (BSS) is a severe bleeding disorder with moderate to severe thrombocytopenia, giant platelets, and platelet dysfunction, caused by biallelic mutations in GP1BA, GP1BB, or GP9 genes. We generated induced pluripotent stem cells (iPSC) from a BSS patient with a novel heterozygous GP1BA p.N103D mutation, resulting in moderate macrothrombocytopenia. The mutation does not affect

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Patients with chronic lymphocytic leukemia (CLL) have impaired response to vaccination, which calls for improved vaccination strategies. This study aimed to evaluate antibody persistence five years after pneumococcal vaccination and response to revaccination. Seventyfour CLL patients and 31 controls, all primary immunized with 13-valent conjugated pneumococcal vaccine (PCV13) or 23-valent polysaccharide

Connexin (Cx) gap junction proteins are expressed by a multitude of cells and function as plasma membrane hemichannels or dock to form intercellular communication tunnels. Whilst Cx43 has garnered considerable attention, less is known about the structure and function of Cx62 channels. Platelets and megakaryocytes express Cx37, Cx40 and Cx62, which contribute to hemostatic and thrombotic responses.