Epigenomic diagnosis and prognosis of Acute Myeloid Leukemia.

Despite the critical role of DNA methylation, clinical implementations harnessing its promise have not been described in acute myeloid leukemia. Utilizing DNA methylation from 3314 leukemia patient samples across 11 harmonized cohorts, we describe the Acute Leukemia Methylome Atlas, which includes robust models capable of accurately predicting AML subtypes. A genome-wide prognostic model as well as a targeted panel of 38 CpGs significantly predict five-year survival in our pediatric and adult test cohorts.

Patient and Advanced Practitioner Perspectives on Symptom Burden and Symptom Management in Indolent Systemic Mastocytosis.

Background: Symptom burden is the primary driver for patients with indolent systemic mastocytosis (ISM) to seek medical care, whether or not they are diagnosed.

Purpose: This descriptive study aimed to describe the advanced practitioner (AP) and ISM patient perspective relative to the symptom burden of ISM, multidisciplinary diagnosis and management of ISM, barriers to symptom management, strategies for collaborative management of ISM, and communicative health literacy in patients with ISM.

Methods: An ISM patient survey and AP survey were developed by an AP-led steering committee incorporating validated tools to measure symptom burden, symptom burden impact, barriers, and strategies for improving symptom burden.

Cytarabine Pharmacogenomics and Outcomes Among Children and Young Adults With Acute Myeloid Leukemia.

Importance: Therapeutic responses in acute myeloid leukemia (AML) demonstrate considerable variability both across and within established risk stratifications and age groups. Moreover, significant racial disparities persist, with Black patients experiencing inferior survival outcomes compared with their White counterparts.

Objective: To validate the association of the previously reported 10 single nucleotide variant (SNV)-based ara-C pharmacogenomics score (ACS10) with survival outcomes in a large cohort of pediatric AML patients; to evaluate whether ACS10 remains relevant in an adolescent and young adult (AYA) population of patients with AML treated with similar intensive induction chemotherapy protocols; and to assess the association of ACS10 with race and treatment outcomes in both cohorts.

Hematopoietic Stem Cell Transplantation Outcomes for High-Risk AML: A Report From the Children's Oncology Group.

Purpose: Hematopoietic stem cell transplantation (HSCT) is used as consolidation for pediatric patients with high-risk AML in first complete remission (CR1). The definition of high-risk AML has evolved considerably over the past two decades with the successive identification of new unfavorable risk factors. We conducted a cross-study analysis to determine whether HSCT improves the outcomes of patients with contemporarily defined high-risk AML.