Classification, risk stratification and response assessment in myelodysplastic syndromes/neoplasms (MDS): A state-of-the-art report on behalf of the International Consortium for MDS (icMDS).

The guidelines for classification, prognostication, and response assessment of myelodysplastic syndromes/neoplasms (MDS) have all recently been updated. In this report on behalf of the International Consortium for MDS (icMDS) we summarize these developments. We first critically examine the updated World Health Organization (WHO) classification and the International Consensus Classification (ICC) of MDS. We then compare traditional and molecularly based risk MDS risk assessment tools. Lastly, we discuss limitations of criteria in measuring therapeutic benefit and highlight how the International Working Group (IWG) 2018 and 2023 response criteria addressed these deficiencies and are endorsed by the icMDS. We also address the importance of patient centered care by discussing the value of quality-of-life assessment. We hope that the reader of this review will have a better understanding of how to classify MDS, predict clinical outcomes and evaluate therapeutic outcomes.

Burden of chemotherapy in patients with relapsed/refractory acute myeloid leukemia in the United States: a retrospective claims database study.

BACKGROUND: Real-world estimates of relapsed or refractory (R/R) acute myeloid leukemia (AML) chemotherapy episode costs are scarce. We quantified chemotherapy episode-related costs and healthcare resource use (HRU) in R/R AML. RESEARCH DESIGN AND METHODS: This real-world, retrospective analysis of United States claims from IQVIA's PharMetrics® Plus database (October 2008-September 2019) identified adults with R/R AML and ≥1 chemotherapy episode. Chemotherapy episode (ie, low- [LIC] or high-intensity [HIC] chemotherapy) costs and HRU were determined using inpatient, outpatient, and pharmacy claims. RESULTS: Mean (SD) and median total all-cause healthcare costs per R/R AML chemotherapy episode were $230,799 ($300,770) and $129,117. Mean (SD) and median adjusted direct R/R AML chemotherapy episode costs were $116,384 ($151,425) and $63,298, with increases noted from the first to the second and subsequent episodes and with HIC. Hospitalizations were the major cost driver; 64.1% of patients had ≥1 hospitalization and 36% required an intensive care unit stay. CONCLUSIONS: R/R AML chemotherapy episode costs were high, with higher costs reported with HIC and increasing lines of chemotherapy. Hospitalizations were a main cost driver. Novel therapies with comparable or improved effectiveness and decreased need for hospitalizations versus chemotherapy may help alleviate the clinical and economic burden of R/R AML.

Nonbiological factors affecting survival in younger patients with acute myeloid leukemia.

BACKGROUND: Progress has been made in determining the biological variants of acute myelogenous leukemia (AML) and their prognostic implications. However, to the authors' knowledge, little is known regarding the impact of nonbiological factors (NBFs) on the survival of patients with AML. METHODS: The impact of NBFs (marital status, insurance status, county-level income, and education) on survival was assessed along with biological factors (disease subtype, sex, age, and race/ethnicity) using a cohort of patients aged 19 to 64 years who were diagnosed with AML between 2007 and 2011 and reported to the Surveillance, Epidemiology, and End Results program registry (SEER 18). RESULTS: There were 5541 patients included. The median overall survival for the entire study population was 16 months. On multivariate analysis, an increased risk of death was independently linked to being a Medicaid beneficiary, uninsured, single, divorced, and residing in a county within the lower 3 quintiles of median household income. NBFs affected the risk of early (<2 months) and late mortality and their impact was confirmed among patients known to have received chemotherapy. CONCLUSIONS: Insurance status, marital status, and county-level income were found to independently affect the survival of younger patients with AML and should be integrated into outcome comparisons. Interventions are needed to mitigate the impact of social factors on survival among patients with AML.

Myelodysplastic syndromes, version 2.2015.

The NCCN Guidelines for Myelodysplastic Syndromes (MDS) comprise a heterogeneous group of myeloid disorders with a highly variable disease course that depends largely on risk factors. Risk evaluation is therefore a critical component of decision-making in the treatment of MDS. The development of newer treatments and the refinement of current treatment modalities are designed to improve patient outcomes and reduce side effects. These NCCN Guidelines Insights focus on the recent updates to the guidelines, which include the incorporation of a revised prognostic scoring system, addition of molecular abnormalities associated with MDS, and refinement of treatment options involving a discussion of cost of care.