Observational Study of Real-World Factor Utilization and Health Outcomes in Patients with Hemophilia in Canada

Introduction: Recombinant Factors VIII Fc (rFVIIIFc) and IX Fc (rFIXFc) fusion proteins are extended half-life (EHL) factor products approved in Canada in 2014 and became available through the Canadian Blood Services in January 2016. Real-world product utilization and clinician practice patterns in EHL prescription are unknown.Objectives: This is a prospective Canadian multi-centre observational study to describe product utilization, clinical and patient-reported outcomes (PROs) in patients pre- and post-switch to rFVIIIFc/ rFIXFc products compared to those who remained on standard half-life (SHL) products. The primary outcome is intra-individual change in annualized factor consumption from 12-month period pre-switch to 24-month post-switch to rFVIIIFc/rFIXFc. This analysis describes the baseline clinical characteristics and intra-individual changes in PROs from the cohort who completed baseline, 3-month and 12-month assessment before June 2018 in this ongoing study.Methods: Males aged ≥12 years with moderate and severe hemophilia A and B (factor level
Source: Blood - Category: Hematology Authors: Tags: 903. Outcomes Research-Non-Malignant Hematology: Poster III Source Type: research

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Conclusions: Valoctocogene roxaparvovec was found to be cost-saving-on average by about $6.8 million per patient-and more effective than prophylactic therapy for treatment of hemophilia A. The comparative benefit of gene therapy was observed across a broad range of simulated patients that were representative of the real-world severe hemophilia A population. PMID: 31971453 [PubMed - as supplied by publisher]
Source: Journal of Medical Economics - Category: Health Management Tags: J Med Econ Source Type: research
Abstract Haemophilia is a blood clotting disorder known as 'Christmas disease' caused when the blood has defect with the clotting factor(s). Bleeding leads various issues, such as chronic pain, arthritis and a serious complication during the surgery. Identifying this disease is mandatory to take the necessary treatment and maintains the normal clotting. It has been proved that the level of factor IX (FIX) is lesser with haemophilia patient and the attempt here is focused to quantify FIX level by interdigitated electrode (IDE) sensor. Single-walled carbon nanotube (SWCNT) was utilized to modify IDE sensing surface....
Source: International Journal of Biological Macromolecules - Category: Biochemistry Authors: Tags: Int J Biol Macromol Source Type: research
Conclusions:This study provides an estimate of the burden of inhibitors in persons with severe hemophilia A in the US, representing approximately 52.9% of all severe hemophilia A patients treated in the USHTCN (CDC, unpublished data). History of an inhibitor reduced patient productivity and increased ED and hospital utilization. Future efforts will focus on a longitudinal analysis of this cohort to better understand the natural history and outcome of inhibitors and their impact on patient quality of life and health care utilization.Acknowledgements:This study was performed with the advice of the Community Counts Inhibitor ...
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis: Poster II Source Type: research
Abstract BACKGROUND: Children and adolescents with severe hemophilia commonly suffer from acute and chronic pain as a consequence of hemophilia-related bleeding. Intervention-related pain also plays a major role. Despite its high prevalence in this patient group, hemophilia-related pain is not always adequately addressed and sufficiently treated. OBJECTIVES: This paper discusses how to improve pain management for children and adolescents (0-18 years) with hemophilia and which specific features in this population should influence decisions in pain management. MATERIALS AND METHODS: An exper...
Source: Schmerz - Category: Anesthesiology Authors: Tags: Schmerz Source Type: research
ConclusionsMeasures of joint status and HRQoL were consistently lower in participants who had higher educational levels, were unemployed, self‐reported having both acute and chronic pain, and self‐reported having anxiety/depression. A greater understanding of the association of these factors with disease outcomes may improve individualized patient management.
Source: European Journal of Haematology - Category: Hematology Authors: Tags: ORIGINAL ARTICLE Source Type: research
CONCLUSIONS: A physiotherapy program based on fascial therapy is safe in patients with hemophilia. Fascial therapy may improve joint status, pain, and mobility in patients with hemophiliac arthropathy of the knee and ankle. PMID: 29334292 [PubMed - as supplied by publisher]
Source: Physiotherapy Theory and Practice - Category: Physiotherapy Authors: Tags: Physiother Theory Pract Source Type: research
CONCLUSION: The ideal treatment for the hemophilic ankle when hematologic prophylaxis fails includes physiotherapy, orthoses, radiosynovectomy, arthroscopic ankle debridement (in the initial stages of cartilage degeneration), and ankle distraction, ankle fusion or total ankle replacement (in advanced stages of cartilage degeneration). PMID: 28891453 [PubMed - as supplied by publisher]
Source: Cardiovascular and Hematological Disorders Drug Targets - Category: Drugs & Pharmacology Tags: Cardiovasc Hematol Disord Drug Targets Source Type: research
Abstract BackgroundLimited data exist on the impact of prophylaxis on adults with severe hemophilia A and pre‐existing joint disease. ObjectivesThis analysis describes 3‐year bleeding, joint, health‐related quality‐of‐life (HRQoL), and other outcomes from the open‐label, randomized, multinational SPINART study. Patients/MethodsMales aged 12–50 years with severe hemophilia A, ≥150 FVIII exposure days, no inhibitors, and no prophylaxis for>12 consecutive months in the past 5 years were randomized to sucrose‐formulated recombinant FVIII (rFVIII‐FS) prophylaxis or on‐demand therapy (OD). Data coll...
Source: Journal of Thrombosis and Haemostasis - Category: Hematology Authors: Tags: Original Article ‐ Clinical Haemostasis and Thrombosis Source Type: research
Abstract BACKGROUND: Limited data exist on the impact of prophylaxis on adults with severe hemophilia A and pre-existing joint disease. OBJECTIVES: This analysis describes 3-year bleeding, joint, health-related quality-of-life (HRQoL), and other outcomes from the open-label, randomized, multinational SPINART study. PATIENTS/METHODS: Males aged 12-50 years with severe hemophilia A, ≥150 FVIII exposure days, no inhibitors, and no prophylaxis for>12 consecutive months in the past 5 years were randomized to sucrose-formulated recombinant FVIII (rFVIII-FS) prophylaxis or on-demand therapy (OD). Data col...
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: J Thromb Haemost Source Type: research
ConclusionsOngoing research is needed in haemophilia and VWD pain management, and on the differences in pain perception between patients, caregivers and providers. As chronic pain often begins at an early age, optimal pain management should include acknowledging patient complaints, exploring pharmacologic and non‐pharmacologic options, and optimizing prophylaxis.
Source: Haemophilia - Category: Hematology Authors: Tags: ORIGINAL ARTICLE Source Type: research
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