Background With marketing authorization in 2005, a non-interventional study (SET = Surveillance of Efficacy and Tolerability) with a double virus inactivated VWF/FVIII concentrate (Wilate®) was initiated in Germany. In 2012, the inclusion of patient documentation was terminated for final evaluation of the study data, representing the treatment of 170 patients suffering from von Willebrand’s disease (VWD). Aim The presented study was performed to assess the haemostatic efficacy and safety of a newly introduced VWF/FVIII product in the treatment of all types of VWD patients in every day clinical setting and to validate the results from pivotal clinical trials. Methods Patients of any age suffering from hereditary or acquired VWD, requiring replacement therapy were included. Apart from demographic and anamnestic data, details of all injections for treatment of bleeding episodes, surgeries and prophylactic treatment were documented and assessed. Clinical efficacy and tolerability were rated by the treating physicians using four-point verbal scales. All data underwent a pre-defined data management process including double data entry and plausibility checks by an independent statistical institute. Results Data of 170 patients suffering from VWD and representing the broad spectrum of disease severity was provided by 26 treatment centers. About two thirds of the patients are female; with an age range from 1 to 85 years at study entry. Six cases of acquired VWD and 7 type 3 VWD patients were included. Type 2 VWD of various subtypes accounts for about 30% of the patients with the remaining patients suffering from type 1 VWD. 108 surgical procedures in 82 patients were documented. In all evaluated surgical procedures, the efficacy of the concentrate was rated to be “excellent” or “good”. Efficacy in 156 bleeding episodes was assessed as “excellent/good” in 95.2% of ratings. 13 patients underwent a prophylactic treatment regimen which resulted in a drop of bleeding frequency to below 1 bleeding episode per month. Five patients had experienced suspected adverse drug reactions - all without sequelae. The ADR rate per injection was as low as 0.1%. Conclusion The results presented reflect the experience of routine use of Wilate® in all types of VWD in various clinical settings. They confirm the excellent efficacy and tolerability which had been demonstrated previously during an extensive panel of clinical trials. Disclosures: Alesci: Octapharma AG: Investigator Other. Von Depka:Octapharma AG: Investigator Other, Speakers Bureau. Feddern:Octapharma AG: Employment. Halimeh: Octapharma AG: Investigator Other, Research Funding. Hegener: Octapharma AG: Employment. Kadar:Octapharma AG: Investigator Other. Miesbach: Octapharma AG: Investigator Other. Nowak-Göttl:Octapharma AG: Investigator Other, Research Funding. Scharrer:Octapharma AG: Investigator Other.
4630 Efficacy, tolerability and dosing of a VWF/FVIII concentrate may differ in paediatric, adults, and elderly patients. It is therefore reasonable to collect and evaluate clinical data of patients from different age groups and to look into differences in treatment and reason for treatment with VWF/FVIII concentrate. A cohort of 120 patients suffering from all types of von Willebrand's disease (VWD) from an on-going German post-marketing surveillance study was analysed for age-related differences in treatment with a high-purity, double virus inactivated VWF/FVIII concentrate (wilate®). Results: Thirteen children up to 12 years of age and 14 patients being 65 or older were treated for prophylaxis, haemorrhages or peri-operatively. Obvious deviations were found in general condition, bleeding locations and types of surgeries for the elderly patients. Further, the data provide evidence of age group-related differences regarding reason for administration (see table 1) and dosages administered. The efficacy and tolerability of wilate®treatment in children and elderly was as high as in the total group. Conclusion: Young or old age seem to have an impact on clinical requirements of VWD. However, larger cohorts are required to confirm these first findings of the non-interventional study “Wilate SET”. Disclosures: Hegener: Octapharma AG: Employment. Feddern:Octapharma: Employment.
1424 Objective: The assessment of the clinical efficacy during the conduct of a clinical trial is critical to the potential acceptance of that product to be a viable therapeutic option. This is especially true in the conduct of clinical trials involving rare bleeding disorders such as VWD, where there is predictably small patient enrollment and limited use of a control group. A 4-point Likert hemostatic efficacy scale with mostly subjective general criteria (excellent, good, moderate, none) has been used previously in most clinical trials published to date in the VWD field. Methods: A validated and more objective set of criteria, intended to more specifically assess the therapeutic efficacy of VWF/FVIII replacement therapy was developed. These objective criteria include: quantization of transfusions of blood components; the need for salvage therapy utilizing alternative VWF/FVIII concentrates or other hemostatically active products; the requirement for significant dosing increases of replacement products; etc. Patients in a VWD clinical trial with a new VWF/FVIII replacement product, wilateÒ (Octapharma AG, Lachen, Switzerland), were assessed for clinical hemostatic efficacy in the treatment of their acute bleeding episodes, using both the traditional 4 point Likert scale, as assessed by the treating physician, and the newly proposed set of objective success and failure criteria for bleeding episodes during treatment, as determined by evaluation of the actual clinical data generated for each individual patient. Summary: Results of the analysis using the new objective criteria when compared to the previous subjective efficacy assessment approach showed a comparable efficacy profile. This proposed objective hemostatic efficacy assessment examined specific clinical criteria that could be considered as indicative of a more accurate status of treatment. Conclusions: This objective approach avoids the potential pitfalls of observer bias and inconsistency, which form the basis of criticism of the previously used subjective assessment tools. Furthermore, this more objective type of assessment approach has been requested and accepted in recent registration trials by FDA in the US. Disclosures: Kessler: Grifols S.A.: Research Funding; Baxter-Immuno: Research Funding; NovoNordisk: Research Funding. Schwartz:Octapharma: Employment. Hegener:octapharma: Employment. Walter:Octapharma: Employment. Knaub:Octapharma: Employment.
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