Immunogenicity, efficacy and safety of Nuwiq® (human-cl rhFVIII) in previously untreated patients with severe haemophilia A-Interim results from the NuProtect Study.

INTRODUCTION: Nuwiq® (Human-cl rhFVIII) is a fourth generation recombinant FVIII, produced in a human cell line, without chemical modification or protein fusion. No inhibitors developed in studies with Nuwiq® in 201 previously treated patients with haemophilia A (HA). The immunogenicity, efficacy and safety of Nuwiq® in previously untreated patients (PUPs) with severe HA are being assessed in the ongoing NuProtect study. METHODS: The study, conducted across 38 centres worldwide, is evaluating 110 true PUPs of all ages and ethnicities enrolled for study up to 100 exposure days (EDs) or 5 years maximum. The primary objective is to assess the immunogenicity of Nuwiq® (inhibitor activity ≥0.6 BU) using the Nijmegen-modified Bethesda assay at a central laboratory. RESULTS: Data for 66 PUPs with ≥20 EDs from a preplanned interim analysis were analysed. High-titre (HT) inhibitors developed in 8 of 66 patients after a median of 11.5 EDs (range 6-24). Five patients developed low-titre inhibitors (4 transient). The cumulative incidence (95% confidence interval) was 12.8% (4.5%, 21.2%) for HT inhibitors and 20.8% (10.7%, 31.0%) for all inhibitors. During inhibitor-free periods, median annualized bleeding rates during prophylaxis were 0 for spontaneous bleeds and 2.40 for all bleeds. Efficacy was rated as "excellent" or "good" in treating 91.8% of bleeds. Efficacy of surgical prophylaxis was "excellent" or "good" for 8 (89%) procedures and "moderate" for 1 (11%). No tolerability concerns were evident. CONCLUSION: These interim data show a cumulative incidence of 12.8% for HT inhibitors and convincing efficacy and tolerability in PUPs treated with Nuwiq® .

[A mouth cavity mucosa membrane illnesses and haemophilia].

To reveal the frequency of parodontitis, parodontosis and gingivitis among patients with haemophilia the structure of inflammatory diseases of mouth cavity mucosa was investigated. 224 patients (aged 2-64 years old) with the various forms of haemophilia were examined. The investigation showed that the occurrence of parodontitis, parodontosis and gingivitis in patients with haemophilia was significantly higher than in control group. In case of haemophilia relative and attributic risk of inflammatory diseases of mouth cavity mucosa rises: parodontitis (RR=2,15; 95%CI: 1,75-2,63; AR=0,48; 95%CI: 0,39-1,04); parodontosis (RR=1,41; 95%CI: 1,251,60; AR=0,26; 95%CI: 0,17-0,85) and gingivitis (RR=2,26; 95%CI: 1,86-2,74; AR=0,53; 95%CI: 0,44-0,96), but they do not correlate with the severity of illness.

National haemophilia programme development in the Republic of Georgia.

After the dissolution of Soviet Union in 1991, haemophilia care in the Republic of Georgia was negatively affected because of the expense of treatment products, lack of clinical and diagnostic facilities, and the need for trained personnel throughout the country. In 2001, the Georgian Government, working through the Ministry of Health, in collaboration with Georgian Association of Haemophilia and Donors, the Institute of Haematology and Transfusion, and the World Federation of Haemophilia, initiated a National Haemophilia Programme. As part of this programme the first Georgian Haemophilia Treatment Centre (HTC) was established. In this paper, we will describe (i) our outreach efforts to identify patients with haemophilia (PWH), (ii) the diagnostic and clinical services provided to patients by the HTC, and (iii) the results of a patient survey designed to assess patient satisfaction with the care provided. Total of 216 PWH were diagnosed, mean age was 25 years (range 4 months to 75 years); 43% had severe, 33% had moderate and 24% had mild haemophilia A or B. Overall, 183 (85%) had haemophilia A and 33 (15%) had haemophilia B, giving a ratio of 5.6. During the 2-year period, 77% of the expected number of PWH was identified by our outreach programme. Vast majority had comprehensive evaluation including joint assessment and over 60% were tested for blood-borne infections within a year and half period. Our findings showed that haemophilia care was considerably improved since the beginning of the National Haemophilia Programme and the survey of PWH showed a high degree of satisfaction with services provided in the HTC. In conclusion, close collaboration of the government, non-government entities and medical professionals in a Georgian national haemophilia care model; resulted in the successful delivery of the much needed services and care to the people living in Georgia with haemophilia.