SOLICITATION NOTICE
A -- National Registry of Patients with Marfan Syndrome (Request for Information)
- Notice Date
- 7/15/2004
- Notice Type
- Solicitation Notice
- NAICS
- 541710
— Research and Development in the Physical, Engineering, and Life Sciences
- Contracting Office
- Department of Health and Human Services, National Institutes of Health, National Heart, Lung, and Blood Institute, Contracts Operations Branch 6701 Rockledge Dr RKL2/6100 MSC 7902, Bethesda, MD, 20892-7902
- ZIP Code
- 20892-7902
- Solicitation Number
- NHLBI-HV-05-09
- Response Due
- 8/13/2004
- Archive Date
- 8/28/2004
- Point of Contact
- Craig Miron, Contracting Officer, Phone (301) 435-0340, Fax (301) 480-3338, - Pamela Lew, Chief, HLV Section, Phone (301) 435-0340, Fax (301) 480-3338,
- E-Mail Address
-
cm49x@nih.gov, pl116x@nih.gov
- Description
- The NHLBI plans to release a Request for Proposals for a Clinical and Data Coordinating Center to establish a registry to collect and analyze clinical data and blood and tissue samples of patients with Marfan Syndrome (and other connective tissue disorders associated with aortic disease) in order to improve understanding of cardiovascular complications and therapies for aortic aneurysmal disease and its complications. Ultimately the registry will provide an essential resource to improve clinical care for patients afflicted with cardiovascular complications of Marfan Syndrome and other genetically-triggered aortic aneurysmal disease. The NHLBI would like information from academic institutions, professional groups and private industries regarding screening, surveillance and treatment of cardiac complications of Marfan Syndrome and other connective tissue disorders associated with aortic aneurysms and or valvular disease in order to identify optimum parameters to establishing a registry and ways to design and implement an effective program. Specifically, NHLBI seeks advice concerning core information to be collected, concerns and solutions to protect rights of patients, facilities, providers and support teams. Also important are independence of data analysis, access to data and data sharing, and potential strategies for the registry to become at least partially self-supporting. Ways to involve private industry without undermining the independence of the program governance are also sought. Finally, while NHLBI is primarily interested in cardiovascular issues, expansion of the data collected in order to include extra-cardiac manifestations of the connective tissue disorders such as joint disease, craniofacial disorders or others may be possible if there interest and support from other Institutes, and/or private or professional groups. In 2002, the NHLBI and the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) met with members of the National Marfan Foundation (NMF) at their request. In follow-up, NHLBI convened a Marfan Working Group in June of 2002. The registry is considered a critically important initial step to stimulate investigator-initiated research in Marfan Syndrome. Marfan Syndrome is an autosomal-dominant, inherited connective tissue disorder that is associated with cardiovascular complications, including mitral valve prolapse, aortic aneurysmal and valvular disease, and heart failure. Premature death for Marfan patients is the result of aortic disease resulting in progressive dilatation of the ascending aorta leading to aortic valve insufficiency, heart failure, and/or aortic rupture. Cardiac surgery to treat thoracic aortic disease has improved survival. Nevertheless, Marfan patients with progressive aortic disease have a shortened life span. Treatment for Marfan Syndrome, including cardiac surgery is hampered by the small numbers of patients seen, even at tertiary referral medical centers. There is a need to evaluate new surgical techniques and emerging strategies to improve clinical outcomes. Current surgical options include different attendant risks, but they lack independent assessment. The use of pharmacologic therapies is largely empiric and would benefit from systematic evaluation. Elucidation of the functional importance of genetic mutations associated with Marfan Syndrome has excellent potential for translation to therapeutic interventions, but is limited by difficulties in obtaining tissues for investigation and identification of kindred families by centers with expertise in genomic applications. Animal models of Marfan syndrome demonstrate functional significance of genetic mutations and suggest new strategies for therapeutic prevention and treatment. These findings require confirmation in patients. A registry with a tissue repository would provide a needed resource for evaluation of disease progression and response to therapy, data for planning appropriate clinical trials, and tissue for genomic investigations, and ultimately facilitate improving treatment for the Marfan patient and possibly other patients with aortic aneurysmal disease. Marfan Syndrome occurs in all races at a frequency of about 1 per 10,000 persons, and is often undiagnosed in life. Marfan Syndrome is characterized by cardiovascular, skeletal and ocular disorders, and its cardiovascular complications result in premature death. In the general population of persons over the age of 65 years, degenerative thoracic and abdominal aortic aneurysmal disease is a common cause of morbidity and mortality. Rupture almost always results in death, and repair of aortic aneurysm is associated with significant morbidity. Surgery to repair abdominal aortic aneurysm has significant operative risk for death and substantial morbidity. Alternate strategies to slow non-Marfan degenerative aneurysms may be facilitated by insights learned about Marfan-related aortic disease which is a genetically triggered. The connective tissue weakening associated with Marfan Syndrome has common and overlapping pathogenic pathways with degenerative aneurysmal diseases secondary to atherosclerosis and normal aging. Thus, understanding and therapy for non-Marfan aneurysmal disease may benefit from new knowledge gained through the proposed registry. The registry would provide systematic collection of tissue and clinical data intended to provide information concerning diagnostic evaluation for the disease, complications, and outcomes. The resultant data base will provide the capacity to evaluate treatments, such as surgical outcomes for aortic valve repair, pharmacologic interventions to reduce progression of aortic root dilatation, complications associated with anticoagulation associated with prosthetic valve, etc. Specific treatment issues that might be addressed include: 1) Current issues in surgical management of aortic root disease in Marfan Syndrome. (Timing of replacement surgery, valve sparing techniques, complications of anticoagulation, sex difference in utilization of surgery , etc.); 2) The role of afterload reduction in slowing progression of aortic dilation leading to valve replacement and anticoagulation; and 3) Optimal use of mechanical versus tissue valves. The registry will support infrastructure to establish a Clinical and Data Coordinating Center, a tissue and blood repository, and a scientific advisory group to develop protocols for standard procedures to collect patient and procedure/treatment data, tissue, and blood samples for genomic and proteomic investigations. Registry data would be analyzed to permit understanding of clinical outcomes, complications, and associated therapies. The registry would serve as a resource to encourage research investigations using centrally collected tissue or data, and would provide information critical to the design of clinical investigations or trials of specific therapeutic interventions. The proposed registry would build on the progress of the existing registries, and facilitate sharing of data without compromising confidentiality of patients or rights of independent investigators. Participation of patient advocacy or other professional groups with NIH will facilitate access and maximize patient and family participation and the cooperation of their doctors. NHLBI seeks comments and suggestions as requested below. 1. Registry will collect a detailed data set in patients with various genetically-triggered cardiovascular complications of the aorta, including aneurysmal disease, valvular disease and heart failure and their treatment. Patient outcomes are determined by patient characteristics, surgical provider and facilities support, and device performance. Please suggest data elements you consider essential for collection by the registry at the time entry and for follow-up. 2. A repository for blood and tissue is planned. Please suggest ways to make the repository useful and accessible to investigators interested in advancing the diagnosis and treatment or advance pathophysiologic understanding of aortic aneurysmal disease or other genetically triggered complication of registry patients. 3. There are other registries of aortic aneurysmal disease and patients with connective tissue disorders. Please suggest ways that the registry might collaborate or coordinate its data collection and analysis with existing registries. 4. Abstracting hospital and out-patient data and checking accuracy of submitted data will be required. Please provide concerns you have about the registry collecting data on patients, hospitals and medical providers. Please suggest ways the NHLBI can design registry protocols to minimize those concerns. 5. Please identify ways for NHLBI to encourage industry to participate and support the registry. 6. Because it is not likely that a single center or hospital has sufficient numbers of patients to support costs associated with a dedicated research coordinator, it is anticipated that patients will be recruited through regional centers responsible for specific large geographic areas. Please suggest how regional centers might be identified and identify elements such as scientific and clinical expertise, patient facilities and services, a regional center requires to facilitate patient enrollment, treatment and data and tissue specimen collection required for the registry to be successful. 7. Please provide methods to ensure accuracy and security of data in the registry. 8. Please suggest strategies for the registry to become at least partially self-supporting. 9. We would appreciate any additional views or opinions that you think would be useful to develop a registry that will improve outcomes for patients with genetically triggered aortic aneurysmal disease including ways to improve identification of at risk patients, screening of families and therapy to arrest or alter progression of disease. Responses in any of the areas are welcome; respondents should not feel compelled to address all items. Please respond no later than August 13, 2004. We look forward to your thoughts, opinions and suggestions and hope that you will share this document with your colleagues. All responses will be kept confidential. Thank you for your assistance. To respond please send a letter, fax or email to the following address: Patrice Desvigne-Nickens, M.D.,Leader, Cardiovascular Medicine SRG,Clinical and Molecular Medicine Program,Division of Heart and Vascular Diseases,National Heart, Lung, and Blood Institute ,National Institutes of Health,6701 Rockledge Drive, Rm. 9178, MSC-7940,Bethesda, MD 20892-7940 (Fed Ex- 20817),Tel. 301-435-0515 / Fax. 301-480-1336, Email: desvigne@mail.nih.gov or Amanda Curto,Program Specialist, Cardiovascular Medicine SRG,Clinical and Molecular Program, Division of heart and Vascular Diseases,National Heart, Lung, and Blood Institute, National Institutes of Health,6701 Rockledge Drive, Rm.9153, MSC-7940, Bethesda, MD 20892-7940 (Fed Ex- 20817),Tel. 301-435-0515 / Fax. 301-480-1336, Email: acurto@mail.nih.gov
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