Systemic mastocytosis (SM) in adults almost always involves the bone marrow (BM) and less frequently, the skin and other organs, including the liver, spleen, and bones.1 The extent of neoplastic mast cell (MC) proliferation (i.e., MC burden), MC-associated organopathy, and co-occurrence of an associated myeloid neoplasm (AMN) determine disease severity and allow subclassification into indolent SM (ISM)

Pearson marrow pancreas syndrome (PS) is a multisystem bone marrow failure (BMF) disorder caused by deletions in mitochondrial DNA (mtDNA). Infant-onset transfusion-dependent pancytopenia, immune defects, and metabolic crises result in early mortality. However, PS often goes undiagnosed, and its rarity, comorbidities, and occasional spontaneous hematologic improvement deter timely consideration of

Myelodysplastic neoplasms (MDS) are heterogeneous clonal hematopoietic neoplasms with a high risk of evolution into acute myeloid leukemia.1 Given the highly variable clinical courses of MDS, the importance of risk stratification for distinguishing high-risk patients has been underscored.2 Currently, the most widely used survival prediction models are the International Prognostic Scoring System (IPSS)

CD19 directed CAR T-cell therapy is used to treat relapsed/refractory B-cell acute lymphoblastic leukemia. The role of the pre-CAR bone marrow (BM) stromal microenvironment in determining response to CAR T-cell therapy has been understudied. We performed whole transcriptome analysis, reticulin fibrosis assessment and CD3 T-cell infiltration on BM core biopsies from pre- and post-CAR timepoints for

Antiphospholipid syndrome (APS) is a systemic autoimmune disorder characterized by recurrent thrombosis and/or recurrent fetal loss. APS can be primary or secondary to autoimmune disorders such as systemic lupus erythematosus (SLE). It is unique from other thrombophilic disorders in that it predisposes patients to not only venous but also arterial thrombosis through endothelial cell damage and activation

Zhou et al. recently published a prognostic model for patients with chronic myelomonocytic leukemia (CMML) undergoing allogeneic hematopoietic stem cell transplantation (HSCT) in this journal.1 This score consisted of five items: Age, Blasts, Leukocytes, Anemia, and chronic Graft vs Host Disease (ABLAG). The strongest predictor in this score was the absence of chronic Graft vs Host Disease (cGvHD)

CONFLICT OF INTEREST STATEMENT Mrinal M Patnaik has received research funding from StemLine Pharmaceuticals, Kura Oncology, and Epigenetix. He has served on the advisory board for CTI pharmaceuticals.

Myelodysplastic neoplasms (MDS) with ring sideroblasts represent about 30% of all MDS patients and are characterized by severe anemia, transfusion dependence, and limited response to erythropoiesis stimulating agents (ESAs). Most patients harbor a somatic mutation of Splicing Factor 3B1 (SF3B1),1 whose presence identifies a distinct entity in the fifth edition of the WHO classification of myeloid neoplasms

Background Addition of daratumumab to lenalidomide, bortezomib, and dexamethasone (D-RVd) in the GRIFFIN study improved the stringent complete response rate by the end of consolidation in transplantation-eligible patients with newly diagnosed multiple myeloma. Here, we report the findings of the predefined final analysis. Methods GRIFFIN was an open-label, randomised, active-controlled, phase 2 trial

Diagnosing post-transplant lymphoproliferative disorder (PTLD) is challenging and often requires invasive procedures. Analyses of cell-free DNA (cfDNA) isolated from plasma is minimally invasive and highly effective for genomic profiling of tumors. We studied the feasibility of using cfDNA to profile PTLD and explore its potential to serve as a screening tool. We included seventeen patients with monomorphic

Lipid metabolic reprogramming is an emerging hallmark of cancer. In order to sustain uncontrolled proliferation and survive in unfavorable environments that lack oxygen and nutrients, tumor cells undergo metabolic transformations to exploit various ways of acquiring lipid and increasing lipid oxidation. In addition, stromal cells and immune cells in the tumor microenvironment also undergo lipid metabolic

Allogeneic hematopoietic cell transplantation (allo-HCT) is a potentially curative treatment for patients with acute leukemia. Despite this, studies have shown that only a minority of patients ultimately proceed to allo-HCT. The primary objective of this prospective, observational study was to identify the rate of allo-HCT in patients for whom it was recommended, and reasons why patients deemed appropriate

Sickle cell disease (SCD) is a severe, multisystemic hematological disorder that impacts nearly every major organ in adults. The current approved treatments for SCD directly target mutant hemoglobin or address downstream disease pathology. Several compounds targeting reduction of 2,3-DPG by activation of Pyruvate Kinase-R are currently being evaluated in SCD patients. In this study, we genetically

In the recently published International Consensus Classification (ICC) and the fifth edition of the World Health Organization classification on myeloid neoplasms,1, 2 the importance of TP53 mutation in disease pathogenesis and prognosis is recognized. The ICC goes a step further in recognizing acute myeloid leukemia (AML) with mutated TP53 as a separate distinct entity given that TP53 mutations are

Pancreatic cancer lacks effective therapy. Here, we reported two metastatic pancreatic cancer patients administrated with Claudin 18.2 (CLDN 18.2) CART therapy after the failure of standard therapy (NCT04581473 and NCT03874897). In case 1, with CLDN 18.2 expression of 2+, 70%, 250 × 106 cells were infused after lymphodepletion. Grade 1 cytokine release syndrome (CRS) occurred on d1 which was later

With the availability of effective targeted agents, significant changes have occurred in the management of patients with acute myeloid leukemia (AML) over the past several years, particularly for those considered unfit for intensive chemotherapy. While testing for measurable residual disease (MRD) is now routinely performed in patients treated with intensive chemotherapy to refine prognosis and, possibly

Background Second primary malignancies (SPMs) are known complications after chemotherapy, but the risk is not well characterised for patients with lymphoma treated with high-dose chemotherapy and autologous haematopoietic stem-cell transplantation (HSCT). We aimed to investigate the rate of SPMs in this population relative to matched control individuals from the general population. Methods In this

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Fludarabine/cyclophosphamide (Flu/Cy) is established for lymphodepletion (LD) prior to standard-of-care CAR T-cell therapy for lymphoma. There is ongoing need to test alternative LD regimens to preserve efficacy, improve safety, and address challenges including the recent national fludarabine shortage. We retrospectively evaluated outcomes among patients with relapsed/refractory aggressive B-cell lymphoma

CD19 chimeric antigen receptor (CAR) T-cells therapy has shown remarkable therapeutic effect in treating relapsed and refractory B acute lymphoblastic leukemia (r/r B-ALL). However, approximately 50% of patients experience relapse within a year due to limited CAR T-cell persistence or CD19 antigen loss.1, 2 Dual-targeting approaches have demonstrated impressive antitumor activity.3 And it has been

In one decade, immunotherapy based on immune checkpoint blockades (ICBs) has become a new pillar of cancer treatment following surgery, radiation, chemotherapy, and targeted therapies. However, not all cancer patients benefit from single or combination therapy with anti-CTLA-4 and anti-PD-1/PD-L1 monoclonal antibodies. Thus, an increasing number of immune checkpoint proteins (ICPs) have been screened