Treatment patterns and blood counts in patients with polycythemia vera treated with hydroxyurea in the United States: An analysis from the REVEAL study

BACKGROUND: Polycythemia vera (PV) is associated with increased blood cell counts, risk of thrombosis, and symptoms including fatigue and pruritus. National guidelines support the use of hydroxyurea (HU) in high-risk patients or those with some other clinical indication for cytoreduction. PATIENTS AND METHODS: REVEAL is a prospective, observational study designed to collect data pertaining to demographics, disease burden, clinical management, patient-reported outcomes, and health care resource utilization of patients with PV in the United States. In this analysis, HU treatment patterns and outcomes were assessed from 6 months prior to enrollment to the time of discontinuation, death, or data cutoff. RESULTS: Of the 1381 patients who received HU for ≥ 3 months, the median HU exposure was 23.6 months (range, 3.1-38.5 months). The most common maximum daily HU doses were 1000 mg (30.6%) and 500 mg (30.1%); only 6.4% received ≥ 2 g/d HU. Approximately one-third (32.3%) of patients had dose adjustments, 23.8% had dose interruptions, and 257 (18.6%) discontinued HU. The most common reasons for HU discontinuations and interruptions were adverse events/intolerance (37.1% and 54.5%, respectively) and lack of efficacy (35.5% and 22.1%, respectively). Of those who received HU for ≥ 3 months, 57.1% had hematocrit values \u3e 45% on ≥ 1 occasion, 33.1% continued to receive phlebotomies, and 27.4% had uncontrolled myeloproliferation. CONCLUSION: The results of this analysis emphasize the need for active management of patients with PV with appropriate HU dose titration to maintain blood count control while monitoring for signs and symptoms of HU intolerance

Established and novel agents for myelodysplastic syndromes

The myelodysplastic syndromes (MDS) are the most commonly diagnosed myeloid malignancy, with > 15 000 new cases identified in the United States yearly. Prognostic scoring systems supplant a formal staging approach and, in general, divide patients into those with lower-risk and those with higher-risk MDS. Although treatment goals for patients with lower-risk disease focus on minimizing transfusions and optimizing quality of life, in higher-risk MDS, the goal is to delay transformation to acute leukemia and to prolong survival. In lower-risk patients, isolated cytopenias are treated with erythropoiesis-stimulating agents or growth factors such as thrombopoietin mimetics. For patients with the del(5q) cytogenetic abnormality or those who fail these initial approaches, lenalidomide may be tried, as can experimental agents. Lower-risk patients with multiple cytopenias may be treated with immunosuppressive drugs or low-dose hypomethylating agents. For patients with higher-risk disease, hypomethylating agents are the preferred initial treatment approach, with evaluation for hematopoietic cell transplantation at diagnosis. Several novel agents are being developed for MDS patients who have failed hypomethylating drugs

Mitigating Fear and Loathing in Managing Acute Myeloid Leukemia

The contemporary care of patients with acute myeloid leukemia (AML) is made complex by potentially toxic treatments, continuously advancing science, aging patients, and individual treatment goals. By taking a survey of present-day approaches, we aim to dispel some of the trepidation surrounding that care of patients with AML. At the beginning is the initial presentation, and we will discuss whether or not AML should be considered a medical emergency. We will explore the complex realm of patient decision-making about initial therapy, including the intricate straits of patient doctor communication, and available options for initial treatment. We will then address post-remission approaches and the controversies that lie therein, and survivorship issues. Finally, we will investigate the current role molecular assessments are playing in therapy recommendations. (C) 2015 Elsevier Inc. All rights reserved