European retrospective study of real-life haemophilia treatment.

INTRODUCTION: Haemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available. AIM: To provide an overview of the FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) across Europe. METHODS: Non-interventional, 12-month retrospective study where anonymized data were retrieved from haemophilia centres/registers in Belgium, France, Germany, Italy, Spain, Sweden and the United Kingdom. Male patients (all ages) receiving coagulation factor treatment 24 months prior to the study, with basal FVIII/FIX levels ≤5 IU dL-1 , without inhibitors, were included. Data were summarized descriptively. RESULTS: In total, 1346 patients with HA and 312 with HB were included in the analysis; 75% and 57% had severe disease (FVIII/FIX < 1 IU dL-1 ) respectively. Prophylaxis was most common for severe haemophilia, especially for children, whereas on-demand treatment was more common for moderate haemophilia in most countries. The mean (SD) prescribed prophylactic treatment ranged from 67.9 (30.4) to 108.4 (78.1) (HA) and 32.3 (10.2) to 97.7 (32.1) (HB) IU kg-1 per week, across countries. Most patients on prophylaxis were treated ≥3 times/week (HA) or two times/week (HB). The median annual bleeding rate (ABR) for patients on prophylaxis ranged from 1.0 to 4.0 for severe HA, and from 1.0 to 6.0 for severe HB, while those with moderate haemophilia generally had slightly higher ABRs. Median ABRs for on-demand-treated severe HA ranged from 4.5 to 18.0, and for HB, 1.5 to 14.0. CONCLUSION: Treatment practice varied greatly between centres and countries and patients treated on-demand and prophylactically both experienced bleeds, emphasizing the need for further optimization of care.

The use of short-term central venous catheters for optimizing continuous infusion of coagulation factor concentrate in haemophilia patients undergoing major surgical procedures.

INTRODUCTION: Continuous infusion (CI) of clotting factors has facilitated surgical procedures and intensive replacement therapy in haemophilia patients. This procedure could, however, be further optimized by using a short-term central venous catheter (CVCs) instead of via peripheral venous access. AIM: In this paper, we present our results on using a short-term CVC in haemophilia patients during major surgical operations. METHODS: In total, 40 patients with haemophilia A or B (37 and 3, respectively), aged 21-81 years, underwent 67 surgeries with 65 CVCs. Patients requiring intensive treatment lasting over 5 days had the indications for CVC placement. The catheters were placed by experienced anaesthesiologists in the operating theatre under general anaesthesia and following activated partial thromboplastin time correction. RESULTS: No interruption of CI was observed and only one catheter had to be removed prematurely due to a suspected infection. There were no signs found for prosthesis or wound infection, nor was there any thrombosis documented. CONCLUSION: Our study produced encouraging results regarding the use of short-term CVCs in haemophilia patients. Even though our patient sample was small, the data corroborates short-term CVCs to be safe and convenient for factor concentrate delivery in CI during major surgical operations.