Search results for "Octapharma"
showing 6 items of 6 documents
Hemophagocytic Lymphohistiocytosis in Early Infancy- Pitfall of Differentiation between Hereditary and Infectious Reasons
2018
Abstract Hemophagocytic Lymphohistiocytosis (HLH) is characterized by pathologic immune activation which occurs either as a familial disorder or as an acquired condition. The diagnosis of HLH requires the presence of five out of nine criteria: fever, splenomegaly, pancytopenia, hypertriglyceridemia, hypofibrinogenemia, hemophagocytosis in bone marrow, hyperferritinemia, low or absent natural killer cell activity and high level of soluble interleukin-2 receptor. Here we present a 6-month-old girl with parents from Southern Italy. She suffered from hepatosplenomegaly and a recurrent high fever for 3 months' duration. On admission, she showed neurological symptoms including irritability and ne…
Descriptive Evaluation of Age-Related Differences in Clinical Manifestation and Treatment of Von Willebrand's Disease in an Open-Label, Prospective, …
2012
Abstract Abstract 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…
Final Evaluation Of a Prospective, Non-Interventional Study On Efficacy and Tolerability Of a New Generation VWF/FVIII Concentrate In The Treatment O…
2013
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 suf…
Results of a Prospective, Open-Label Trial to Assess the Efficacy, Safety and Immunogenicity of Wilate® in Children Under 6 Years of Age with Inherit…
2011
Abstract Abstract 3330 Introduction: Wilate® is a new-generation plasma-derived concentrate of von Willebrand factor (VWF) and factor VIII (FVIII) developed for treatment of patients with von Willebrand disease (VWD) and haemophilia A. The objective of this study was to assess efficacy, safety and immunogenicity of Wilate in children below 6 years of age with inherited VWD. Methods: The prospective, multi-centre trial included patients with inherited VWD of any type below 6 years of age with insufficient response to DDAVP. Any clinical event requiring VWF/FVIII concentrate treatment within 1 year was treated with Wilate. Efficacy was recorded by using a 4-point VRS and in vivo recovery of F…
Narratives of Patients with Fatal Outcomes During the Phase 2 TITAN and Phase 3 HERCULES Studies
2019
Background: Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare but life-threatening thrombotic microangiopathy, with an untreated mortality rate of >90%. Prompt treatment with therapeutic plasma exchange (TPE) and immunosuppression improves outcomes in patients with aTTP, but 10-20% of patients still die acutely from this disease. The aim of this analysis was to describe in more detail the characteristics and disease courses of the patients who died during the caplacizumab clinical development program. Methods: Patient narratives on all deaths occurring during the phase 2 TITAN and phase 3 HERCULES studies were extracted. Results: In the overall study periods, a total of 6 pat…
Early Mortality in Patients with Multiple Myeloma Treated with Novel Agents - Analysis from Polish Myeloma Study Group
2020
Background Although the introduction of novel agents improved the survival outcomes in patients with multiple myeloma (MM), some patients died within one year (early mortality, EM) following diagnosis. In this study, we evaluated the EM rate, and investigated the risk factors associated with EM in MM patients. Aims In this study we investigated risk factors associated with EM in MM patients initially treated with novel-agent containing regimen. Methods We conducted a multicenter (15 Polish sites) retrospective study a cohort of symptomatic MM pts diagnosed between October 2006 and November 2019 and living < 365 days (d) after diagnosis. All pts were dead at the time of the analysis. …