Treatment is indicated in Waldenström macroglobulinemia (WM) when clinical manifestations arise due to the IgM paraprotein or lymphoplasmacytic infiltrate.1 The main classes of drugs used for WM treatment include monoclonal antibodies, chemotherapeutic agents, proteasome inhibitors, and Bruton Tyrosine Kinase inhibitors (BTKi). The most frequently used chemotherapeutic agent is bendamustine, which

PIEZO1 encodes a mechanoreceptor, a cation channel activated by mechanical stimuli. Gain‐of‐function (GoF) variants in PIEZO1 cause dehydrated hereditary stomatocytosis (DHS), or xerocytosis, a pleiotropic syndrome characterized by anemia and iron overload. DHS patients develop hepatic iron overload independent of the degree of anemia and transfusion regimen. PIEZO1‐GoF variants suppress hepcidin expression

Acute myeloid leukemia (AML) with translocation t(8;16)(p11;p13) represents a rare entity that has been categorized as a disease‐defining recurring cytogenetic abnormality with adverse risk in the 2022 European LeukemiaNet classification. This rating was mainly based on a retrospective analysis comprising patients from several large clinical trials, which, however, included only 21 patients treated

According to current recommendations, older AML patients in first complete remission (CR) after induction chemotherapy should receive consolidation with intermediate‐dose cytarabine (IDAC). However, no study has demonstrated the superiority of IDAC over other regimen. In this retrospective study, we compared the efficacy of mini‐consolidations (idarubicin 8 mg/m2 day 1, cytarabine 50 mg/m2/12 h, day

Despite the introduction of JAK inhibitors, allogeneic hematopoietic cell transplant remains the only potentially curative treatment for patients with myelofibrosis but has considerable treatment‐related complications. Whether the incorporation of JAK inhibition into the transplant algorithm leads to improved outcomes is still unclear. Here, we analyzed different transplant platforms in myelofibrosis

Relapse is the major cause of treatment failure in Philadelphia chromosome‐positive (Ph+) acute lymphoblastic leukemia (ALL) undergoing allogeneic hematopoietic stem cell transplantation (allo‐HSCT). This study aimed to evaluate the effect of a prophylactic tyrosine kinase inhibitor (TKI) strategy on relapse in this population. Patients were assigned to prophylactic or control groups based on measurable

High‐grade B‐cell lymphoma not otherwise specified (HGBCL, NOS) has overlapping morphological and genetic features with diffuse large B‐cell lymphoma (DLBCL) and Burkitt lymphoma (BL), leading to uncertainty in its diagnosis and clinical management. Using functional genomic approaches, we previously characterized HGBCL and NOS, that demonstrate gene expression profiling (GEP), and genetic signatures

Click on the article title to read more.

A 76-year-old man with a history of chronic obstructive pulmonary disease with lung fibrosis, type 2 diabetes mellitus, and chronic kidney disease underwent computed tomography imaging of the chest due to increasing dyspnea. The bones appeared sclerotic, and a bone scan showed diffuse tracer uptake throughout the axial and appendicular skeleton. The prostate showed no features of malignancy on magnetic

1 BACKGROUND Globalization of clinical trials, defined operationally as conduct in the international arena, has grown over the past few decades. The pharmaceutical industry is expanding its activities not only in High-Income countries but also in Low- and Middle-Income countries (LMICs).1 For pharmaceutical companies, this shift can be associated with several benefits: a larger pool of potential participants

Bruton tyrosine kinase (BTK) inhibitor is now the standard of care for both treatment-naïve (TN) and relapsed/refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Zanubrutinib, a next-generation BTK inhibitor with better BTK specificity and less off-target inhibition, has demonstrated superior efficacy and improved safety profile compared with first-generation BTK inhibitor

Based on results from the randomized, placebo-controlled phase 3 VIALE-A (NCT02993523) and VIALE-C (NCT03069352) trials,1-4 venetoclax in combination with hypomethylating agents or low-dose cytarabine (LDAC) has become standard of care in patients with newly diagnosed acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. Cytopenias are common adverse events with venetoclax and

Acute splenic sequestration crisis (ASSC) is one of the earliest acute clinical manifestations of sickle cell anemia (SCA), with a median age at first episode of 1.8 years [range: 0.4–12.9] as reported for our recently published regional longitudinal newborn cohort, beginning with the introduction of newborn screening (1986) and ending just before the introduction of preventive intensification with

CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.

CONFLICT OF INTEREST STATEMENT Francesco Onida received lecture fees from Menarini StemLine, Takeda, Kyowa Kirin and MEDAC.

Acidosis and increased tonicity in plasma can mediate pathologic changes in the membrane and cytoplasm of sickle red blood cells (sRBCs). These changes contribute to intravascular hemolysis, endothelial damage, and endothelial adhesion with propensity to microvascular occlusion, resulting in vaso-occlusive episodes (VOE) and end organ damage.1 Kidney dysfunction, in particular, is common among adults

A 70-year-old woman with a history of successfully resected pancreatic cancer 10 years ago presented with dyspnea and fever. Laboratory tests showed leukocytosis (white cell count 20.8 × 109/L) and elevated C-reactive protein (87 mg/L) with normal hemoglobin concentration (Hb) (137 g/L) and platelet count (242 × 109/L). Computed tomography identified a hepatic abscess. The abscess was drained and

Autoimmune hemolytic anemia (AIHA) is a group of acquired autoimmune disorders characterized by red blood cell hemolysis. In a phase 2, open-label, multicenter study, adults with warm AIHA, cold agglutinin disease, or mixed-type AIHA were administered once-daily 1.0 or 2.5 mg parsaclisib (selective phosphoinositide 3-kinase δ inhibitor) orally for 12 weeks, followed by an extension period. Dose increases

Hematopoietic stem cells (HSCs) continuously produce blood cells while maintaining their self-renewal, proliferation, and differentiation potential. Normal blood cell production is balanced between myeloid and lymphoid progeny. With aging, the number of HSCs increases but their differentiation potential declines.1 One of the hallmarks of aged HSCs is a myeloid differentiation bias, with less capability

BCR::ABL1-negative myeloproliferative neoplasms (MPNs) can evolve to secondary acute myeloid leukemia (sAML) or blast-phase (BP) MPN, a very severe condition with lack of effective therapy.1 Leukemic transformation (LT) of MPNs displays a variable incidence according to MPN phenotype: 9%–13% in primary myelofibrosis (PMF), 3%–7% in polycythemia vera (PV), and 1%–4% in essential thrombocythemia (ET)

The recent report of the first pig kidney transplant in a living human brings hope to thousands of people with end‐stage kidney failure. The scientific community views this early success with caution as kidney xenotransplantation exhibits many challenges and barriers. One of these is coagulation dysregulation. This includes (i) pig von Willebrand Factor (vWF) interaction with human platelets, which

Hereditary spherocytosis (HS) is caused by mutations in genes such as ANK1, EPB42, SLC4A1, SPTA1, or SPTB,1 leading to altered red blood cell (RBC) membrane proteins, reduced deformability, and decreased RBC lifespan. Dehydrated hereditary stomatocytosis (xerocytosis) is caused by variants in the PIEZO1 gene and, less commonly, KCNN4 variants, affecting RBC hydration and stability.2 The prevalence

Beta (β)‐thalassemia and sickle cell disease (SCD) are characterized by a hypercoagulable state, which can significantly influence organ complication and disease severity. While red blood cells (RBCs) and erythroblasts continue to play a central role in the pathogenesis of thrombosis in β‐thalassemia and SCD, additional factors such as free heme, inflammatory vasculopathy, splenectomy, among other

Differences in characteristics and outcomes between incidental and symptomatic presentations of Large B-Cell Lymphoma.

The treatment of paroxysmal nocturnal hemoglobinuria (PNH) is undergoing a second revolution with the introduction of proximal complement inhibitors.1 Indeed, recent clinical studies have shown that molecules targeting either the C3, the complement factor B, or the complement factor D (this latter in association with anti-C5 therapy) have been effective in increasing hemoglobin levels in PNH patients

Click on the article title to read more.

Li-Fraumeni syndrome (LFS) is a rare inherited disorder associated with germline pathogenic TP53 variants. The absence of the functional gene product, p53 protein, results in failure to activate programmed cell death in the appropriate context and leads to uncontrolled cell proliferation. LFS patients present with a high incidence of various malignancies, often at young ages. In contrast to the high

1 INTRODUCTION Like whole blood, plasma is collected from healthy human donors. However, while much is known and has been written about whole blood donations and the worrisome trends that increasingly challenge its safety and global supply,1-4 the complexities of plasma collection and its supply chain—from the donor's arm, through the manufacturing process, to the patient's therapy—have, with few exceptions

Sickle cell disease is a somewhat ambiguous term, sometimes used as a synonym for sickle cell anemia and sometimes as a generic term, encompassing also compound heterozygous states characterized by clinicopathological features that result from sickle cell formation. There are at least 13 compound heterozygous states of relevance, the most common being sickle cell/β thalassemia, sickle cell/hemoglobin

Managing large B-cell lymphoma (LBCL) that is refractory to or relapsed after chimeric antigen receptor (CAR)-T therapy remains a significant challenge. Here we aimed to investigate the safety and efficacy of C-CAR066, an autologous fully human anti-CD20 specific CAR-T, for relapsed/refractory LBCL after failure of anti-CD19 CAR-T therapy. This first-in-human, single-arm, phase 1 study was conducted

A recent study—ASC4FIRST1—builds the case that the novel drug, asciminib, a BCR::ABL1 inhibitor, is superior to current tyrosine kinase inhibitors (TKIs) for the treatment of chronic phase (CP) chronic myeloid leukemia (CML). Some have even taken to social media to announce a post-imatinib era. As the first TKI to be approved in oncology, imatinib has been a transformative oral anti-cancer drug, improving

CONFLICT OF INTEREST STATEMENT The authors have no competing conflicts of interests to declare.

Measurable residual disease (MRD) is strongly associated with risk of relapse and long‐term survival outcomes in patients with acute myeloid leukemia (AML). Apart from its clear prognostic impact, MRD information is also increasingly used to guide therapeutic decision‐making, including selection of appropriate patients for stem cell transplant, use of post‐transplant maintenance, and candidacy for

While emerging therapies such as gene therapy are being explored, allogenic hematopoietic stem cell transplantation (HSCT) remains the most established curative treatment for sickle cell anemia (SCA). Yet its application is limited by toxicity risks, donor availability, and socioeconomic factors. In pediatric cases, myeloablative HSCT from an HLA-matched related donor (HLA-MRD), following either Busulfan

Two recent decisions by the Food and Drug Administration will likely significantly impact testing for multiple myeloma (MM) minimal residual disease (MRD). First, on April 12, 2024, the FDA's Oncologic Drugs Advisory Committee (ODAC) voted to approve the use of MRD as an end point for accelerated approval of new treatments for patients with MM.1 This was a result of near 10 years effort by multiple

CONFLICT OF INTEREST STATEMENT The author declares no conflict of interest.

In children with sickle cell disease (SCD), splenectomy is immediately beneficial for acute sequestration crises and hypersplenism (ASSC/HyS) but portends a long-term risk of asplenia-related complications. We retrieved peripheral and splenic red blood cells (RBCs) from 17 SCD children/teenagers undergoing partial splenectomy for ASSC/HyS, 12 adult subjects without RBC-related disease undergoing splenectomy

Acute myeloid leukemia (AML) is a biologically and clinically heterogenous disease with diverse genetic abnormalities1-6 and a wide-ranging white blood cell counts (WBC) at diagnosis.1 The 2022 European LeukemiaNet (ELN) genetic-risk classification incorporates cytogenetic and selected molecular alterations to define Favorable, Intermediate, and Adverse genetic-risk groups providing valuable prognostic

CONFLICT OF INTEREST STATEMENT The author declares no conflicts of interest.

1 CASE PRESENTATION A 26-year-old female with a history of chronic urticaria (treated with omalizumab in the past) and recently treated chlamydia trachomatis infection presented to an outside emergency department after a syncopal episode. She endorsed a three-day history of nausea, vomiting, fatigue, and ecchymoses. Initial laboratory analysis revealed macrocytic anemia (hemoglobin 4.6 g/dL) with a

CONFLICT OF INTEREST STATEMENT The authors declare no conflicts of interest.

Luspatercept is the first erythropoiesis-modulating agent approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for treating anemia in adult transfusion-dependent β-thalassemia (TDT) patients. As observed in clinical trials1 and real-life experience,2 response to luspatercept in TDT is heterogeneous. It can range from patients who do not respond to those who become

Immunoglobulin light chain (AL) amyloidosis is a multisystem disease with varied treatment options and disease-related outcomes. Current staging systems rely on a limited number of cardiac, renal, and plasma cell dyscrasia biomarkers. To improve prognostication for all-cause mortality and end-stage kidney disease (ESKD), we applied unsupervised machine learning using a comprehensive set of clinical

To the Editor: With the advent of proteasome inhibitors (PIs), immunomodulatory drugs (IMiDs), and anti-CD38 monoclonal antibodies (CD38mAbs), the outcomes of transplant-ineligible (TIE) patients with newly diagnosed multiple myeloma (MM) have substantially improved in the last decade.1-3 Currently, there are two widely accepted standard-of-care regimens in newly diagnosed TIE MM: daratumumab-lena

Hodgkin lymphoma (HL) is an uncommon B-cell lymphoid malignancy affecting 8570 new patients annually and representing ~10% of all lymphomas in the United States.

Click on the article title to read more.