Key issues in inhibitor management in patients with haemophilia.
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ABSTRACT:
SUBMITTER: Gomez K
PROVIDER: S-EPMC3934290 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:The standard therapy for patients with haemophilia is prophylactic treatment with replacement factor VIII (FVIII) or factor IX (FIX). Patients who develop inhibitors against FVIII/FIX face an increased risk of bleeding, and the likelihood of early development of progressive arthropathy, alongside higher treatment-related costs. Bypassing agents can be used to prevent and control bleeding, as well as the recently licensed prophylaxis, emicizumab, but their efficacy is less predictable than that of factor replacement therapy. Antibody eradication, by way of immune tolerance induction (ITI), is still the preferred management strategy for treating patients with inhibitors. This approach is successful in most patients, but some are difficult to tolerise and/or are unresponsive to ITI, and they represent the most complicated patients to treat. However, there are limited clinical data and guidelines available to help guide physicians in formulating the next treatment steps in these patients. This review summarises currently available treatment options for patients with inhibitors, focussing on ITI regimens and those ITI strategies that may be used in difficult-to-treat patients. Some alternative, non-ITI approaches for inhibitor management, are also proposed.
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Project description:IntroductionThe Haemophilia Experiences, Results and Opportunities (HERO) Study identified sexual health as an important psychosocial issue affecting people with haemophilia (PWH) worldwide. However, sexual health is inadequately addressed at haemophilia treatment centres (HTCs), because PWH and healthcare professionals (HCPs) experience barriers to broaching the subject. There is a clear need for HCP training to support communication in this area and improve comprehensive care.AimThe Sexual Health: Strategies for Effective Communication pilot programme was trialled in Canada to assess HCP readiness and ability to discuss sexual health issues with PWH and test communication tools to facilitate these conversations.MethodsThe pilot programme consisted of two 3-h sessions attended by seven HCPs from Calgary's Alberta Children's and Foothills Hospitals. The sessions included lectures and case scenarios and explained the check-in-affirm-clarify-answer and head-heart-body tools designed by the Centre for Sexuality to aid communication. The pilot was evaluated through discussions and an online questionnaire.ResultsThe pilot was well received by all HCP participants. Questionnaire data showed improvements in participants' knowledge, skills and comfort level in conducting sexual health discussions. Greatest improvements were noted in knowledge (100% 'good' or 'excellent' after the pilot, compared with 29% beforehand). Importantly, 86% felt that the material presented would be applicable in clinical practice.ConclusionThe Canadian pilot demonstrated the effectiveness of the proposed educational programme. The underlying principles could be adapted to similar programmes for other HTCs to facilitate sexual health discussions.
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