Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic disorder with increased mortality and morbidity resulting from intravascular hemolysis. Eculizumab, a monoclonal antibody against the complement protein 5, stops the intravascular hemolysis in PNH. We evaluated 79 consecutive patients treated with eculizumab in Leeds between May 2002 and July 2010. The survival of patients treated with eculizumab was not different from age- and sex-matched normal controls (P = .46) but was significantly better than 30 similar patients managed before eculizumab (P = .030). Three patients on eculizumab, all over 50 years old, died of causes unrelated to PNH. Twenty-one patients (27%) had a thrombosis before starting eculizumab (5.6 events per 100 patient-years) compared with 2 thromboses on eculizumab (0.8 events per 100 patient-years; P < .001). Twenty-one patients with no previous thrombosis discontinued warfarin on eculizumab with no thrombotic sequelae. Forty of 61 (66%) patients on eculizumab for more than 12 months achieved transfusion independence. The 12-month mean transfusion requirement reduced from 19.3 units before eculizumab to 5.0 units in the most recent 12 months on eculizumab (P < .001). Eculizumab dramatically alters the natural course of PNH, reducing symptoms and disease complications as well as improving survival to a similar level to that of the general population.

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The study lays a foundation for creating acceptable peer assessment systems among students by soliciting their views. Merely introducing an assessment tool will not result in students' willingness to assess each other.

SummaryIn Paroxysmal nocturnal haemoglobinuria (PNH), pregnancy is associated with increased maternal and foetal complications to such an extent that the condition has been considered relatively contra-indicated in PNH. Eculizumab has revolutionized the treatment of PNH. We evaluate its use in pregnancy to date. We report on seven patients exposed to eculizumab at different stages of pregnancy including the first two patients to receive the drug from conception to delivery. There was no evidence of complement blockade from cord blood samples taken at delivery. Eculizumab appears safe to use in this setting and is likely to prevent many of the complications usually observed.

A circumscribed concept of professionalism is available to serve as a foundation for next steps in assessing professional behavior. The current array of assessment tools is rich. However, their measurement properties should be strengthened. Accordingly, future research should explore rigorous qualitative techniques; refine quantitative assessments of competence, for example, through OSCEs; and evaluate separate elements of professionalism. It should test the hypothesis that assessment tools will be better if they define professionalism as behaviors expressive of value conflicts, investigate the resolution of these conflicts, and recognize the contextual nature of professional behaviors. Whether measurement tools should be tailored to the stage of a medical career and how the environment can support or sabotage the assessment of professional behavior are central issues. FINAL THOUGHT: Without solid assessment tools, questions about the efficacy of approaches to educating learners about professional behavior will not be effectively answered.

In sum, a school-wide examination such as the QPE can be used successfully. As a longitudinal, comprehensive testing system based on the objectives of the educational program, the QPE can satisfy internal and external requirements for assessment. Such an examination system could be an effective evaluation instrument for schools fostering problem-based and independent learning by students.

In this article, the authors consider the quality and relevance of current and future medical education research by (1) presenting a framework for medical education research and reviewing basic principles of "good" empirical work, (2) extending the discussion of principles to "best practices," (3) considering the distinctive features of medical education that present challenges to the researcher, and (4) discussing opportunities for expanding the scope and influence of medical education research. Their audience is intended to be clinicians involved in education, deans and associate deans who create and direct educational curricula and processes, and those from offices critical to the educational mission such as admissions, student services and financial aid, as well as medical education researchers. The authors argue that the quality and relevance of current work can be enhanced when research is situated within a general framework and questions are asked that are based on literature and theory and push the field toward new knowledge. Obviously methods and designs must be appropriate and well-executed and sufficient data must be gathered. Multiple studies are highlighted that showcase the rigor and creativity associated with excellent quality work. However, good research is not without its challenges, most notably short timelines and the need to work within an ever-changing real-life educational environment. Most important, the field of medical education research has many opportunities to increase its impact and advance its quest to study important learners' behaviors and patients' outcomes. Programs to train and collaborate with clinical and administrative colleagues, as well as researchers in other fields, have great potential to improve the quality of research in the field.