Natural history and clinical characteristics of inhibitors in previously treated haemophilia A patients: a case series

• Tipo: Artigo em Revista Científica Internacional
• Data: 2017

Autores

Iorio, A (Autor) Outra; Barbara, AM (Autor) Outra; Makris, M (Autor) Outra; Fischer, K (Autor) Outra; Castaman, G (Autor) Outra; Cristina Catarino (Autor) FFUP; Gilman, E (Autor) Outra; Kavakli, K (Autor) Outra; Lambert, T (Autor) Outra; Lassila, R (Autor) Outra; Lissitchkov, T (Autor) Outra; Mauser Bunschoten, E (Autor) Outra; Mingot Castellano, ME (Autor) Outra; Ozdemir, N (Autor) Outra; Pabinger, I (Autor) Outra; Parra, R (Autor) Outra; Pasi, J (Autor) Outra; Peerlinck, K (Autor) Outra; Rauch, A (Autor) Outra; Roussel Robert, V (Autor) Outra; Serban, M (Autor) Outra; Tagliaferri, A (Autor) Outra; Windyga, J (Autor) Outra; Zanon, E (Autor) Outra

Revista

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Título: HAEMOPHILIA

Vol. 23

Páginas: 255-263

ISSN: 1351-8216

Editora: Wiley

Indexação

ISI Web of Knowledge - 5 Citações

Scopus - 9 Citações

Outras Informações

ID Authenticus: P-00M-K2Q

DOI: 10.1111/hae.13167

Abstract (EN): Background: Development of inhibitors is the most serious complication in haemophilia A treatment. The assessment of risk for inhibitor formation in new or modified factor concentrates is traditionally performed in previously treated patients (PTPs). However, evidence on risk factors for and natural history of inhibitors has been generated mostly in previously untreated patients (PUPs). The purpose of this study was to examine cases of de novo inhibitors in PTPs reported in the scientific literature and to the EUropean HAemophilia Safety Surveillance (EUHASS) programme, and explore determinants and course of inhibitor development. Methods: We used a case series study design and developed a case report form to collect patient level data; including detection, inhibitor course, treatment, factor VIII products used and events that may trigger inhibitor development (surgery, vaccination, immune disorders, malignancy, product switch). Results: We identified 19 publications that reported 38 inhibitor cases and 45 cases from 31 EUHASS centres. Individual patient data were collected for 55/83 (66%) inhibitor cases out of 12 330 patients. The median (range) peak inhibitor titre was 4.4 (0.5-135.0), the proportion of transient inhibitors was 33% and only two cases of 12 undergoing immune tolerance induction failed this treatment. In the two months before inhibitor development, surgery was reported in nine (22%) cases, and high intensity treatment periods reported in seven (17%) cases. Conclusions: By studying the largest cohort of inhibitor development in PTPs assembled to date, we showed that inhibitor development in PTPs, is on average, a milder event than in PUPs.

Idioma: Inglês

Tipo (Avaliação Docente): Científica

Nº de páginas: 9