Naslov
European retrospective study of real-life haemophilia treatment

Autori
Berntorp, Erik ; Dolan, Gerard ; Hay, Charles ; Linari, Silvia ; Santagostino, Elena ; Tosetto, Alberto ; Castaman, Giancarlo ; Álvarez-Román, Maria Teresa ; Parra Lopez, Rafael ; Oldenburg, Johannes ; Tosseto, Albert ; Scholz, Ute ; Holmström, Margareta ; Schved, Jean- François ; Trossaërt, Mark ; Hermans, cedric ; Boban, Ana ; Ludlam, Christopher ; Lethagen, Stephan

Izvornik
Haemophilia (1351-8216) 23 (2017), 1; 105-114

Vrsta, podvrsta i kategorija rada
Radovi u časopisima, članak, znanstveni

Ključne riječi
factor VIII, factor IX, haemophilia A, haemophilia B, retrospective study, treatmen

Sažetak
Introduction: Haemophilia treatment varies significantly between individuals, countries and regions and details ofbleed rates, factor consumption and injection frequency are often not available. Aim: To provide an overview ofthe FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) acrossEurope. Methods: Non-interventional, 12-month retrospective study where anonymized data were retrieved fromhaemophilia centres/registers in Belgium, France, Germany, Italy, Spain, Sweden and the United Kingdom. Malepatients (all ages) receiving coagulation factor treatment 24 months prior to the study, with basal FVIII/FIX levels≤5IUdL1, without inhibitors, were included. Data were summarized descriptively. Results: In total, 1346patients with HA and 312 with HB were included in the analysis ; 75% and 57% had severe disease (FVIII/FIX < 1IUdL1) respectively. Prophylaxis was most common for severe haemophilia, especially for children, whereas on- demand treatment was more common for moderate haemophilia in most countries. The mean (SD)prescribed prophylactic treatment ranged from 67.9 (30.4) to 108.4 (78.1) (HA) and 32.3 (10.2) to 97.7 (32.1)(HB) IU kg1per week, across countries. Most patients on prophylaxis were treated ≥3 times/week (HA) or twotimes/week (HB). The median annual bleeding rate (ABR) for patients on prophylaxis ranged from 1.0 to 4.0 forsevere HA, and from 1.0 to 6.0 for severe HB, while those with moderate haemophilia generally had slightlyhigher ABRs. Median ABRs for on-demand- treated severe HA ranged from 4.5 to 18.0, and for HB, 1.5 to 14.0.Conclusion: Treatment practice varied greatly between centres and countries and patients treated on-demand andprophylactically both experienced bleeds, emphasizing the need for further optimization of care

Izvorni jezik
Engleski

Znanstvena područja
Kliničke medicinske znanosti

POVEZANOST RADA