Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them.

The advent of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) has revolutionized the treatment of acute promyelocytic leukemia (APL), resulting in excellent rates of remission and long-term survival. However, real-world outcomes often fall short of those observed in clinical trials due to various factors related to patient demographics and clinical practices. This review examines APL treatment outcomes in real-world settings and highlights the phenomenon of APL clusters.

Cost-effectiveness of Enasidenib versus conventional care for older patients with mutant refractory/relapsed AML.

In the randomized phase III IDHENTIFY trial, the IDH2 inhibitor enasidenib (ENA) showed improvement in event-free but not overall survival compared with conventional care regimens (CCR) among patients with relapsed/refractory (R/R), -mutant AML. We constructed a partitioned survival model to evaluate the cost-effectiveness of enasidenib for the treatment of older patients with R/R, and -mutant AML. In the base-case scenario, ENA exhibited an incremental effectiveness of 0.

Analyzing determinants of premature trial discontinuation in leukemia clinical trials.

Clinical trials are crucial for improving patient outcomes. Although a significant number of trials are discontinued prematurely, our understanding of factors influencing early termination is limited. We conducted a comprehensive search of ClinicalTrials.

Treatment of Myelodysplastic Syndromes for Older Patients: Current State of Science, Challenges, and Opportunities.

Purpose Of Review: Myelodysplastic syndromes/neoplasms (MDS) represent a diverse group of pathologically distinct diseases with varying prognoses and risks of leukemia progression. This review aims to discuss current treatment options for elderly patients with MDS, focusing on patients ineligible for intensive chemotherapy or allogenic hematopoietic stem cell transplantation (HSCT). The challenges associated with treatment in this population and emerging therapeutic prospects are also explored.

Molecular and clinical analyses of PHF6 mutant myeloid neoplasia provide their pathogenesis and therapeutic targeting.

PHF6 mutations (PHF6) are identified in various myeloid neoplasms (MN). However, little is known about the precise function and consequences of PHF6 in MN. Here we show three main findings in our comprehensive genomic and proteomic study.

When to use which molecular prognostic scoring system in the management of patients with MDS?

Myelodysplastic syndromes/neoplasms (MDS) are a heterogeneous group of hematopoietic cancers characterized by recurrent molecular alterations driving the disease pathogenesis with a variable propensity for progression to acute myeloid leukemia (AML). Clinical decision making for MDS relies on appropriate risk stratification at diagnosis, with higher-risk patients requiring more intensive therapy. The conventional clinical prognostic systems including the International Prognostic Scoring System (IPSS) and its revised version (IPSS-R) have dominated the risk stratification of MDS from 1997 until 2022.

Splenectomy outcomes in immune cytopenias: Treatment outcomes and determinants of response.

Background: Splenectomy is commonly used to treat refractory immune-mediated cytopenia, but there are no established factors that are associated with response to the procedure.

Objectives: A cohort study was conducted to evaluate the hematologic and surgical outcomes of splenectomy in adult patients with immune cytopenias and identify preoperative factors associated with response.

Methods: Data from the Cleveland Clinic Foundation for 1824 patients aged over 18 who underwent splenectomy from 2002 to 2020 were analyzed.

Ischemic or Coronary Evaluations in Patients With Monomorphic VT Electrical Storm Undergoing VT Ablation.

Background: Monomorphic ventricular tachycardia (VT) electrical storm (ES) in patients with coronary artery disease is dependent on scarred myocardium. The role of routine ischemic or coronary evaluations before ablation in patients presenting with monomorphic VT storm, without acute coronary syndrome (ACS), remains unknown.

Objectives: This study sought to assess the impact of ischemic or coronary evaluations on procedural outcomes and post-ablation mortality in monomorphic VT storm patients.

Novel scheme for defining the clinical implications of TP53 mutations in myeloid neoplasia.

Background: TP53 mutations (TP53) occur in diverse genomic configurations. Particularly, biallelic inactivation is associated with poor overall survival in cancer. Lesions affecting only one allele might not be directly leukemogenic, questioning the presence of cryptic biallelic subclones in cases with dismal prognosis.

New approaches to idiopathic neutropenia in the era of clonal hematopoiesis.

Isolated chronic idiopathic neutropenia (CIN) is a rare disease with multiple contributing etiologies that must be ruled out before establishing a diagnosis. We studied clinical and molecular data of 238 consecutive adult patients with CIN. Autoimmune neutropenia was present in 28% of our cohort.

Novel Scheme for Defining the Clinical Implications of TP53 Mutations in Myeloid Neoplasia.

mutations ( ) occur in diverse genomic configurations. Particularly, biallelic inactivation is associated with poor overall survival in cancer. Lesions affecting only one allele might not be directly leukemogenic, questioning the presence of cryptic biallelic subclones in cases with dismal prognosis.