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From two U.K. centres 23 children with severe congenital coagulopathy had a total of 27 port-a-cath devices inserted to facilitate factor VIII or IX prophylaxis (eight patients), domiciliary therapy (seven patients), immunotolerance (four patients), or a combination thereof (four patients). Six children had a factor VIII inhibitor at the time of insertion. The mean age at operation was 30 months, with a range of 9-76 months. The cumulative length of follow-up is 639 months with a mean of 27.8 months and a range of 5-79 months. Haemostasis was achieved peri- and post-operatively with high-purity concentrate in the majority of patients without an inhibitor. All those with an inhibitor had porcine factor VIII, except one who had recombinant factor VIIa. The post-operative complication rate was 27% (6/23): three had a port-site haematoma (one required removal and replacement), two had post-operative infection, and one had swelling caused by extravasation. To date there have been 13 documented infections in 10/23 patients (five with inhibitor): a rate of 0.24 per follow-up year or 0.67 per 1000 patient-days. Six were caused by Gram-positive and seven by Gram-negative organisms. Six infections could not be eradicated by antibiotics and the port-a-cath system had to be removed; in three it was replaced by a second port-a-cath. Although there are risks involved in the use of port-a-caths in this population, both clinicians and parents involved in the care of these children believe that the benefits are considerable and the potential hazards are acceptable.

Introduction: Nuwiq® (human‐cl rhFVIII, simoctocog alfa) is a 4th generation recombinant human FVIII, without chemical modification or fusion with any other protein, produced in a human cell line. Aim/Methods: This study (GENA‐13) was an extension of the GENA‐03 study in which previously treated children aged 2‐12 years with severe haemophilia A received Nuwiq® prophylaxis for ≥6 months. GENA‐13 examined long‐term tolerability, immunogenicity and efficacy of Nuwiq® prophylaxis in children. Results: Of 59 patients enrolled in GENA‐03, 49 continued Nuwiq® prophylaxis in GENA‐13 for a median (range) of 30.0 (9.5‐52.0) months. No patient withdrew due to drug‐related adverse events or developed inhibitors. Only 2 of 20 518 infusions were associated with possibly related adverse events (dyspnoea, fever). The estimated annualized bleeding rate (ABR) was 0.67 (95% CI: 0.44, 1.02) for spontaneous and 2.88 (95% CI: 1.86, 4.46) for all bleeds. Younger children (2‐5 years) had lower ABRs than children aged 6‐12 years. Annualized bleeding rates were reduced in GENA‐13 vs GENA‐03, especially for spontaneous bleeds in younger children (71% reduction; ABR ratio 0.29 [95% CI: 0.11, 0.74]). Nuwiq® efficacy was rated as excellent/good in the treatment of 83.0% of 305 evaluated breakthrough bleeds. Surgical prophylaxis with Nuwiq® was rated as excellent for all 17 assessed procedures. Conclusion: Long‐term treatment with Nuwiq® for the prevention of bleeds in children with severe haemophilia A was well tolerated, effective and reduced spontaneous bleeding by up to 70% compared with GENA‐03. [ABSTRACT FROM AUTHOR]