title: Real-life evidence in evaluating effectiveness of treatment in Haemophilia A with a recombinant FVIII concentrate: a non-interventional study in emerging countries.
abstract: Some progress has been made regarding availability of recombinant factor VIII concentrates and prophylaxis for haemophilia A in emerging countries, where plasma-derived concentrates were used in the vast majority. Clinical studies to document their introduction and effectiveness are so far not widely available in literature. This non-interventional study evaluates the real-life effectiveness and safety of prophylactic and on-demand treatment with recombinant factor VIII formulated with sucrose (rFVIII-FS) for bleed control and preservation of joints in emerging countries from Eastern Europe, North Africa and Middle East area. One hundred and eighty-six patients from 11 countries were enrolled, mean ± SD age 12.8 ± 12.7 years. At enrolment, majority (79.6%) had severe haemophilia A (<2% IU mL(-1) ), 47.8% had a target joint, 15% had an inhibitor history and one patient was on immune tolerance induction. During the 24-month observation period, 58.1% of the patients were prescribed prophylaxis at every visit, 31.7% were on an on-demand regimen. Patients with severe haemophilia A on prophylaxis (n = 82) had a mean annual rate of treated bleeds of 2.8 ± 4.4, whereas it was 19.1 ± 32.0 for the on-demand group (n = 31), and a mean total Gilbert Score of 9.9 ± 10.3 at baseline and 4.1 ± 6.7 at study end; vs. 15.2 ± 17.3 and 13.7 ± 17.1 for on-demand respectively. The majority of the bleeds (91.1%) were treated with one or two infusions. Four patients without inhibitor history had a first positive inhibitor test during the study. This study demonstrates the effective use of rFVIII-FS in emerging countries and adds to the established safety profile of rFVIII-FS.
authors: Service d'Hématologie Clinique Hôpital Aziza Othmana, Tunis, Tunisia.
authors: E
authors: E
authors: Gouider
authors: Bayer HealthCare Pharmaceuticals, Berlin, Germany.
authors: S
authors: S
authors: Rauchensteiner
authors: Comprehensive Hemophilia Care Centre, St. Petersburg, Russia.
authors: T
authors: T
authors: Andreeva
authors: Sheikh Khalifa Medical City, Abu Dhabi, United Arab Emirates.
authors: A
authors: A
authors: Al Zoebie
authors: Klinika za Pedijatriju, Sarajevo, Bosnia-Herzegovina.
authors: S
authors: S
authors: Mehadzic
authors: Akmolinskaya Regional Hospital, Kokshetau, Kazakhstan.
authors: L
authors: L
authors: Nefyodova
authors: Bayer HealthCare Pharmaceuticals, Berlin, Germany.
authors: M
authors: M
authors: Brunn
authors: Bayer HealthCare Pharmaceuticals, Wuppertal, Germany.
authors: C
authors: C
authors: Tueckmantel
authors: Service d'Hématologie Clinique Hôpital Aziza Othmana, Tunis, Tunisia.
authors: B
authors: B
authors: Meddeb
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D019774:Blood Coagulation Factor Inhibitors; D002648:Child; D002675:Child, Preschool; D005169:Factor VIII / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D006467:Hemophilia A / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007223:Infant; D007239:Infections / Q000209:etiology; D007518:Isoantibodies; D007592:Joint Diseases / Q000209:etiology; D008297:Male; D008875:Middle Aged; D011292:Premedication; D013395:Sucrose / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D019774:Blood Coagulation Factor Inhibitors; D007518:Isoantibodies; C438308:recombinant FVIII, sugar formulated; D013395:Sucrose; D005169:Factor VIII
keywords: clinical study; factor VIII; haemophilia A; prophylaxis
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