Haemophilia care in Europe – a survey of 35 countries

O’Mahony, Brian; Noone, Declan; Giangrande, Paul; Prihodová, Lucia

doi:10.1111/hae.12125

Cited by 62 publications

(80 citation statements)

References 5 publications

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“…The registry collects any trends or changes in product usage and monitors the development of alloantibodies. Data collected by the registry on FVIII and FIX consumption in Italy, regardless of the product used, yielded values of 9.0 IU per capita and 1.2 IU per capita, respectively; these figures for the use of prophylaxis treatment are in keeping with data collected in other European countries [20]. The increasing trend in FVIII consumption per capita, shown in Figure 3, is reasonable due to an increased use of secondary prophylaxis, according to AICE Recommendations, as well as to an increase in tertiary prophylaxis.…”

Section: Discussionsupporting

confidence: 58%

Italian Registry of Congenital Bleeding Disorders

Giampaolo

Abbonizio

Arcieri

et al. 2017

JCM

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In Italy, the surveillance of people with bleeding disorders is based on the National Registry of Congenital Coagulopathies (NRCC) managed by the Italian National Institute of Health (Istituto Superiore di Sanità). The NRCC collects epidemiological and therapeutic data from the 54 Hemophilia Treatment Centers, members of the Italian Association of Hemophilia Centres (AICE). The number of people identified with bleeding disorders has increased over the years, with the number rising from approx. 7000 in 2000 to over 11,000 in 2015. The NRCC includes 4020 patients with hemophilia A and 859 patients with hemophilia B. The prevalence of the rare type 3 vWD is 0.20/100,000 inhabitants. Less common congenital bleeding disorders include the following deficiencies: Factor I (fibrinogen), Factor II (prothrombin), Factor V, Factor VII, Factor X, Factor XI and Factor XIII, which affect 1953 patients. Hepatitis C Virus (HCV) infection affects 1561 patients, more than 200 of whom have two infections (HCV + HIV). Estimated hemophilia-related drug consumption in 2015 was approx. 550 million IU of FVIII for hemophilia A patients and approx. 70 million IU of FIX for hemophilia B patients. The NRCC, with its bleeding disorder data set, is a tool that can provide answers to fundamental questions in public health, monitoring care provision and drug treatment, as well as facilitating clinical and epidemiological research.

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Section: Discussionsupporting

confidence: 58%

Italian Registry of Congenital Bleeding Disorders

Giampaolo

Abbonizio

Arcieri

et al. 2017

JCM

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“…In the terminology used in clinical hemophilia literature 27 and the relevant European guidelines for clinical evaluation of therapeutic hemophila products 29, 30, the accepted definition of ‘exposure days’ is equivalent to the definition of ‘treatment days’ given in Table 1.…”

Section: Patient Populations (Evaluated Subjects) and Treatment Historymentioning

confidence: 99%

Standardizing terms, definitions and concepts for describing and interpreting unwanted immunogenicity of biopharmaceuticals: recommendations of the Innovative Medicines Initiative ABIRISK consortium

Rup

Pallardy

Sikkema

et al. 2015

Clinical and Experimental Immunology

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SummaryBiopharmaceuticals (BPs) represent a rapidly growing class of approved and investigational drug therapies that is contributing significantly to advancing treatment in multiple disease areas, including inflammatory and autoimmune diseases, genetic deficiencies and cancer. Unfortunately, unwanted immunogenic responses to BPs, in particular those affecting clinical safety or efficacy, remain among the most common negative effects associated with this important class of drugs. To manage and reduce risk of unwanted immunogenicity, diverse communities of clinicians, pharmaceutical industry and academic scientists are involved in: interpretation and management of clinical and biological outcomes of BP immunogenicity, improvement of methods for describing, predicting and mitigating immunogenicity risk and elucidation of underlying causes. Collaboration and alignment of efforts across these communities is made difficult due to lack of agreement on concepts, practices and standardized terms and definitions related to immunogenicity. The Innovative Medicines Initiative (IMI; www.imi-europe.org), ABIRISK consortium [Anti‐Biopharmaceutical (BP) Immunization Prediction and Clinical Relevance to Reduce the Risk; www.abirisk.eu] was formed by leading clinicians, academic scientists and EFPIA (European Federation of Pharmaceutical Industries and Associations) members to elucidate underlying causes, improve methods for immunogenicity prediction and mitigation and establish common definitions around terms and concepts related to immunogenicity. These efforts are expected to facilitate broader collaborations and lead to new guidelines for managing immunogenicity. To support alignment, an overview of concepts behind the set of key terms and definitions adopted to date by ABIRISK is provided herein along with a link to access and download the ABIRISK terms and definitions and provide comments (http://www.abirisk.eu/index_t_and_d.asp).

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“…These patients have both medical and social challenges to face as they require prompt access to complex and integrated care from multidisciplinary teams of professionals and they often need life-long access to expensive treatments. Despite the advances in healthcare and the many efforts to develop guidelines for quality standards of care in recent years, the lack of clear pathways and coordination among healthcare stakeholders, as well as organizational gaps in healthcare delivery, still exist [15–18]. …”

Section: Discussionmentioning

confidence: 99%

Comparing regional models of congenital bleeding disorders: preliminary steps in the Italian context

et al. 2017

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BackgroundAmong these diseases, congenital bleeding disorders (CBD) represent a significant societal burden in terms of high morbidity costs and health outcomes. In Italy, the organization and provision of health care is a regional responsibility and regions must assure equity and quality to all their residents. This is also true for CBD care which is provided by 54 multidisciplinary Hemophilia Treatment Centers (HTCs) distributed among the regions. With the present study, we intend to stimulate a debate on the effect that the decentralization process have in the delivery of services to CBD patients across Italy.MethodsThe available comparable measures of caseloads per center and interregional patient mobility, as proxies of quality and responsiveness of the regional network of HTCs, were first analyzed for the using data from the Italian Hemophilia Centers Association for the year 2012.ResultsNine thousand one hundred and thirty four Italian residents with CBD received care in at least one of the Italian HTC in 2012. Preliminary findings suggested room for improvement in health care delivery for CBD patients. In 2012, 16 HTCs out of 51 (31.4%) treated a number of patients under the minimum requirement for treatment center accreditation (10 severe patients). Moreover, data on interregional patient mobility highlighted differences in the ability of each region to retain its own residents or to attract residents from other regions.ConclusionsPreliminary study results showed significant disparities among regions in terms of volumes and mobility of residents with CBDs that cannot be completely explained by the different geographical characteristics. Therefore, the central government should consider taking concrete measures to bridge the gap between regions to assure access to quality care for all individuals with CBD independently from where they live and therefore to move toward a more integrated and homogeneous national network of care centers. Typology of disease, patients’ needs, and cost for outcomes, should have high priority on the political agenda. For CBD patients, even in a federal healthcare system, the national government should have the global responsibility to guaranteeing uniform levels of quality care over the country and overcome local institutions when necessary.

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Haemophilia care in Europe – a survey of 35 countries

Cited by 62 publications

References 5 publications

Italian Registry of Congenital Bleeding Disorders

Italian Registry of Congenital Bleeding Disorders

Standardizing terms, definitions and concepts for describing and interpreting unwanted immunogenicity of biopharmaceuticals: recommendations of the Innovative Medicines Initiative ABIRISK consortium

Comparing regional models of congenital bleeding disorders: preliminary steps in the Italian context

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