Recent advances in the treatment of polycythemia vera.

Polycythemia vera (PV) has long been recognized as a disease characterized by excess blood cell production leading to thromboembolic phenomena. While the focus of treatment is on prevention of thromboembolic complications, achieved by hematocrit control and administration of low dose aspirin, attention has begun to shift to other elements of this chronic neoplasm, namely symptom control and arrest of disease progression. Clearly, phlebotomy is not able to accomplish these goals, and the ability of cytoreductive agents such as hydroxyurea (HU), to influence these elements of the disease is limited. Novel and repurposed drugs have recently entered this space, based on promising initial studies demonstrating their effects on biologic outcomes such as JAK2 V617F variant allele frequency (VAF). In this review, we present updated results of randomized clinical trials of pegylated interferon (IFN) and ruxolitinib and summarize emerging data from early phase trials of novel agents in PV.

Diagnosis and management of catastrophic antiphospholipid syndrome.

INTRODUCTION: Catastrophic antiphospholipid syndrome (CAPS) is a rare, life-threatening disease. In 1992, Asherson defined it as a widespread coagulopathy related to the antiphospholipid antibodies (aPL). CAPS requires rapid diagnosis and prompt initiation of treatment. Areas covered: This paper discusses all aspects of CAPS, including its pathophysiology, clinical manifestations, diagnostic approaches, differential diagnoses, management and treatment of relapsing CAPS, and its prognosis. To obtain the information used in this review, scientific databases were searched using the key words antiphospholipid antibodies, catastrophic antiphospholipid syndrome, hemolytic anemia, lupus anticoagulant, and thrombotic microangiopathic hemolytic anemia. Expert commentary: CAPS is a rare variant of the antiphospholipid syndrome (APS). It is characterized by thrombosis in multiple organs and a cytokine storm developing over a short period, with histopathologic evidence of multiple microthromboses, and laboratory confirmation of high aPL titers. This review discusses the diagnostic challenges and current approaches to the treatment of CAPS.

Treatment of high-risk patients with diabetes: motivation and teaching intervention: a randomized, prospective 8-year follow-up study.

The aim of this study was to examine whether motivating patients to gain expertise and closely follow their risk parameters will attenuate the course of microvascular and cardiovascular sequelae of diabetes. A randomized, prospective study was conducted of 165 patients who had type 2 diabetes, hypertension, and hyperlipidemia and were referred for consultation to a diabetes clinic in an academic hospital. Patients were randomly allocated to standard consultation (SC) or to a patient participation (PP) program. Both groups were followed by their primary care physicians. The mean follow-up was 7.7 yr. The SC group attended eight annual consultations. The PP patients initiated on average one additional consultation per year. There were 80 cardiovascular events (eight deaths) in the SC group versus 47 events (five deaths) in the PP group (P = 0.001). The relative risk (RR) over 8 yr for a cardiovascular event in the intervention (PP) versus the control (SC) group was 0.65 (95% confidence interval, 0.89 to 0.41). There were 17 and eight cases of stroke in the SC and PP groups, respectively (P = 0.05). RR for stroke was 0.47 (95% confidence interval, 0.85 to 0.32). In the SC group, 14 patients developed overt nephropathy (four ESRD) versus seven (one ESRD) in the PP group (P = 0.05). Throughout the study period, BP, LDL cholesterol, and hemoglobin A1c were significantly lower in the PP than in the SC patients. Well informed and motivated patients were more successful in obtaining and maintaining good control of their risk factors, resulting in reduced cardiovascular risk and slower progression of microvascular disease.