Haemophilia B: Where are we now and what does the future hold?

Research has been lacking on the natural history, complications, and treatment of haemophilia B, which is less common than haemophilia A and was recognized as a distinct clinical entity in 1947. Although the two diseases share the same clinical manifestations, they differ in causative mutation, risk of inhibitor development, and patient quality of life. Frequently debated is whether haemophilia B is as clinically severe as haemophilia A, with much of the published data on overall and haemophilia-specific health outcomes suggesting that haemophilia B may have a less severe clinical phenotype. However, although fewer haemophilia B than haemophilia A patients appear to experience bleeding, bleeds are just as severe. We review distinguishing characteristics of haemophilia B and its treatment, including management strategies for neonates, therapeutic approaches for patients who develop inhibitors, pharmacokinetics of factor IX concentrates administered as replacement therapy, and potential future treatments.     

Mucosal healing and deep remission: What does it mean?

The use of specific terms under different meanings and varying definitions has always been a source of confusion in science.When we point our efforts towards an evidence based medicine for inflammatory bowel diseases(IBD)the same is true:Terms such as"mucosal healing"or"deep remission"as endpoints in clinical trials or treatment goals in daily patient care may contribute to misconceptions if meanings change over time or definitions are altered.It appears to be useful to first have a look at the development of terms and their definitions,to assess their intrinsic and context-independent problems and then to analyze the different relevance in present-day clinical studies and trials.The purpose of such an attempt would be to gain clearer insights into the true impact of the clinical findings behind the terms.It may also lead to a better defined use of those terms for future studies.The terms"mucosal healing"and"deep remission"have been introduced in recent years as new therapeutic targets in the treatment of IBD patients.Several clinical trials,cohort studies or inception cohorts provided data that the long term disease course is better,when mucosal healing is achieved.However,it is still unclear whether continued or increased therapeutic measures will aid or improve mucosal healing for patients in clinical remission.Clinical trials are under way to answer this question.Attention should be paid to clearly address what levels of IBD activity are looked at.In the present review article authors aim to summarize the current evidence available on mucosal healing and deep remission and try to highlight their value and position in the everyday decision making for gastroenterologists.