title: Adult severe haemophilia A patients under long-term prophylaxis with factor VIII in routine clinical practice.
abstract: There is no clear consensus on the best practice for long-term prophylaxis in adults with severe haemophilia A. This is a single-centre prospective case series study. We describe here the demographic data, type and reason of prophylaxis in adult patients (>18 years old) with severe (<1%) haemophilia A, treated in our centre from 2006 to 2013. Prophylaxis was tailored according to pharmacokinetic studies and posterior factor VIII (FVIII) trough level adjustment. We analysed FVIII consumption, bleeding rate, adherence and adverse events in this group of patients. In adult patients who initiated long-term prophylaxis during this period, we compared FVIII consumption and bleeding rate with the previous on-demand period. We analysed data from 18 patients. Median annual FVIII consumption was 2374.2 IU/kg/year. Among the patients receiving tertiary prophylaxis, initiated from 2006 onwards, the annual FVIII consumption was 2557.8 vs. 1696.8 IU/kg per year during the on-demand period (P = 0.312). In this group of patients, there was a decrease in annual bleeding events of 88.3% during prophylaxis compared with the on-demand therapy (P < 0.0001). A high adherence to prophylaxis was observed (84%). No cases of anaphylaxis or symptomatic thromboembolic events were recorded. In adult severe haemophilia A patients, the type of and reason to indicate long-term prophylaxis are diverse nowadays. FVIII consumption varies depending on the justification of prophylaxis. The observations reported provide further support for the efficacy of long-term prophylaxis in adult haemophilia A patients.
authors.affiliation: aHematology Department bPharmacy Department, Regional University Hospital of Malaga, Malaga, Spain.
authors.forename: María E
authors.initials: ME
authors.lastname: Mingot-Castellano
authors.forename: Laura
authors.initials: L
authors.lastname: González-Díaz
authors.forename: Rocio
authors.initials: R
authors.lastname: Tamayo-Bermejo
authors.forename: Anabel I
authors.initials: AI
authors.lastname: Heiniger-Mazo
mesh_terms: D000328:Adult; D005169:Factor VIII / Q000008:administration & dosage* / Q000493:pharmacokinetics; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D008297:Male; D011446:Prospective Studies; D055815:Young Adult
chemical_list: C427184:recombinant factor VIII SQ; D005169:Factor VIII