The overall objective of this application is to prevent the complications of hemophilia and bleeding disorders through the expansion and acquisition of clinical data sets. These data sets will include both disease-specific and general-health related information. The data acquired will be used for proactive implementation of prevention programs throughout the network of multidisciplinary comprehensive Hemophilia Treatment Centers (HTC) throughout the United States. HTC's are designed to treat patients with hemophilia A and B, von Willebrand Disease and rare factor deficiencies. Utilizing the comprehensive care model, therapeutic interventions based on previously collected data and changes in therapy based on new research continues to enhance the care of the bleeding disorders community. Chronic genetic disorders often require complex and multidisciplinary care for optimal diagnosis, treatment and management. The consequences of bleeding disorders include severe medical, financial, vocational, psychological and familial repercussions when the condition is not properly managed. The lack of proper treatment has general public health consequences such as increased costs of care caused by increased hospital and emergency utilization and patient disability that may require welfare and other government entitlements. A regional approach to planning, coordination and allocation of funds is vital to ensure that services are available to treat this and other complicated bleeding disorders in Region II, New York, New Jersey, Puerto Rico and the US Virgin Islands. The proposed program will continue a national network of federally funded HTCs organized through 12 regional core centers and funded by the CDC.
Aim 1 : To provide comprehensive prevention services to persons with bleeding disorders directed at attaining and measuring specific outcomes through research and surveillance aimed at reducing complications using a multidisciplinary approach. Under this aim we propose to expand the cohort through continued recruitment and observation individuals with genetic bleeding disorders including hemophilia A and B, von Willebrand disease, rare bleeding disorders and women with bleeding disorders; and expand the current Universal Data and Serum Specimen Collection System (UDC) database to collect additional relevant data related to women's issues in coagulation, rare bleeding disorders, prophylaxis, factor utilization, inhibitors, genetic mutations, and other important clinical outcomes. We plan to design and pilot additional studies aimed at further characterizing the bleeding disorders population through the expanded access to a) gene-based diagnosis and b) more precise definition of the biochemical phenotype (e.g. global assays) with the ultimate goal of genotype - phenotype correlation; utilize the UDC BMI data to evaluate the cost impact on the health care system from conditions with weight-based dosing; test interventions that could facilitate weight loss; and design and pilot a study to evaluate effects of rising annual out- of-pocket expenses and co-payments on the use of elective/preventive services provided by the HTC.
Aim 2 : Maintain A Regional Network Of Comprehensive Prevention Services Through Hemophilia Treatment Centers to Persons With Hemophilia and Related Disorders Including Women, Minorities and the Underserved. Under this broad aim we will lay out our comprehensive, culturally sensitive, family-centered care services and their expansion, our communication within the region and administrative mechanisms. These include outreach and education, orientation and training programs, maintenance of appropriate management and evaluation systems, maintenance of a prevention evaluation network to assess the efficacy of prevention services by increasing the capacity for data collection, moving from paper to electronic and our participation in the coordinating committee. Our plan as stated will promote health and quality of life by preventing and controlling disease, injury, and disability. Many of our aims will facilitate reducing health disparities and accelerate the adoption of health behaviors while improving prevention effectiveness through cost-based studies.
Some aims will be directly transferable to other chronic disease populations in the general public such as the evaluation of obesity and menorrhagia. ? ? ?
