The European Acquired Haemophilia (EACH) Registry.

Abstract

Acquired hemophilia is a rare autoimmune syndrome, characterized by depletion of FVIII, more rarely of FIX, with high morbidity and mortality. Treatment modalities to control bleeding and to suppress the antibodies synthesis are controversial. The purpose of the EACH registry is to collect data on management of these patients (pts) in European countries in order to provide a basis for accepted guidelines. Method. The following information was requested: underlying condition (primary or secondary), causes (spontaneous or provoked), site, type and severity of bleeding, type and titre of the inhibitor, regimens of the anti-hemorrhagic and immunosuppressive therapy (IST), clinical response, side effects and outcome.Inclusion criteria: pts diagnosed after January 1st, 2003; patients diagnosed before 2003 were included if new bleedings occurred or if already on follow-up.Results. From January 2003 to May 15th, 214 pts from 77 centers in 18 countries were registered; 19 were not evaluable because of missing data. The analysis includes 195 pts (102 males, 93 females). Underlying conditions (pts no): idiopathic 107, seconadary 88 (autoimmune diseases 23, neoplasia 27, pregnancy 23, other 15). Inhibitor type: anti FVIII 193, anti FV 1, anti FIX 1. Laboratory data (median and range) age 72 years (6–95); Hb 10 g/dL (4.9–13.6), inhibitor titre 15.5 BU/mL (1–2,800); no bleedings in 13 pts; bleeding in 182 pts; no anti hemorrhagic therapy in 45 pts; 28 recurrences in the follow up. The evaluable events were 165, treated with one or several therapeutic modalities: 89 rFVIIa, 26 APCC, 35 FVIII, 10 fresh frozen plasma, 9 antifibrinolyticic, 5 DDAVP, 5 immunoadsorption; more than 1 treatment was given in some events. The overall response was 79.3%. IST (steroid alone or in combination with other drugs) was given in 153 pts received with complete remission in 95 pts. To date 10 inhibitor recurrences were registered. Patients mortality (%): 39 (20); 8 (4.1) of bleeding, 7 (3.6) of the underlying condition, 13 (6.6) IST related infections, 11 (5.7) others.Comments. Distribution in primary and secondary is in accordance with the data in the literature; 20% of the pts did not required treatment. The factor level and the inhibitor titre are not correlated with the clinical picture. Control of bleeding and an overall response to IST is achieved in about 80% of the episodes and in 61.4% of the pts respectively. To date bleeding mortality is lower than reported in the literature.