Prophylactic rFIXFc, administered every 1 to 2 weeks, resulted in low annualized bleeding rates in patients with hemophilia B. (Funded by Biogen Idec; ClinicalTrials.gov number, NCT01027364.).

Agents that control bleeding and the usage of bypassing agents have made surgery an option to consider in people with hemophilia. However, the lack of consistent definitions for major or minor surgery may lead to inconsistencies in patient management. This literature review has evaluated how surgical procedures in people with hemophilia were categorized as major or minor surgery and assessed the consistency across publications. After screening 926 potentially relevant articles, 547 were excluded and 379 full-text articles were reviewed. Ninety-five articles categorized major or minor surgical procedures; of these, 35 publications categorized three or more major or minor surgical procedures and were included for analysis. Seven (20%) publications provided varying criteria for defining major or minor surgery, five of which defined surgery according to the level of surgical invasiveness. Across all 35 publications, there was considerable variance in the categorization of major and minor surgical procedures and some overlap in surgical nomenclature (eg, type of synovectomy, arthroscopy, and central venous access device insertion/removals). The lack of consistent guidance when referring to major or minor surgery in people with hemophilia needs to be addressed. Clear and consistent definitions, achieved by consensus and promoted by relevant international hemophilia committees, are desirable, to provide guidance on appropriate treatment, to increase the accuracy of trial data and may confound the interpretation of surgical outcomes.

Background. People with haemophilia face many treatment decisions, which are largely informed by evidence from observational studies. Without evidence-based 'best' treatment options, patient preferences play a large role in decisions regarding therapy. The shared decision-making (SDM) process allows patients and health care providers to make decisions collaboratively based on available evidence, and patient preferences. Decision tools can help the SDM process. The objective of this project was to develop two-sided decision tools, decision boxes for physicians and patient decision aids for patients, to facilitate SDM for treatment decisions in haemophilia. Methods. Development of the decision tools comprised three phases: topic selection, prototype development and usability testing with targeted end-users. Topics were selected using a Delphi survey. Tool prototypes were based on a previously validated framework and were informed by systematic literature reviews. Patients, through focus groups, and physicians, through interviews, reviewed the prototypes iteratively for comprehensibility and usability. Results. The chosen topics were: (i) prophylactic treatment: when to start and dosing, (ii) choosing factor source and (iii) immunotolerance induction: when to start and dosing. Intended end users (both health care providers and haemophilia patients and caregivers) were engaged in the development process. Overall perception of the decision tools was positive, and the purpose of using the tools was well received.Conclusions. This study demonstrates the feasibility of developing decision tools for haemophilia treatment decisions. It also provides anecdotal evidence of positive perceptions of such tools. Future directions include assessment of the tools' practical value and impact on clinical practice.

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