SPECIAL NOTICE
A -- Request for Information (RFI): Building a National Resource to Study Myelodysplastic Syndromes (MDS) - The MDS Cohort Natural History Study
- Notice Date
- 8/29/2012
- Notice Type
- Special Notice
- NAICS
- 541712
— Research and Development in the Physical, Engineering, and Life Sciences (except Biotechnology)
- Contracting Office
- Department of Health and Human Services, National Institutes of Health, National Heart, Lung and Blood Institute, Rockledge Dr. Bethesda, MD, Office of Acquisitions, 6701 Rockledge Dr RKL2/6100 MSC 7902, Bethesda, Maryland, 20892-7902
- ZIP Code
- 20892-7902
- Solicitation Number
- HHS-NIH-NHLBI-RFI-12-147
- Point of Contact
- Allison M Cristman, Phone: (301) 435-0359, Cheryl A Jennings, Phone: (301) 435-0347
- E-Mail Address
-
allison.cristman@nih.gov, cj19f@nih.gov
(allison.cristman@nih.gov, cj19f@nih.gov)
- Small Business Set-Aside
- N/A
- Description
- This notice can also be found on The NIH Guide for Grants and Contracts (http://grants.nih.gov/grants/guide/), Notice # NOT-HL-12-147. Purpose This is a Request for Information (RFI). This is NOT a solicitation for proposals, proposal abstracts, or quotations. The purpose of this RFI is to obtain knowledge and information for project planning purposes and to enhance study design. The National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI) are considering building a national resource to better understand the disease progression of myelodysplastic syndromes (MDS), and are seeking information relevant to this project from the communities. Background An estimated 20,000 or more new cases of MDS are diagnosed in the United States each year. The incidence of MDS dramatically increases with age. MDS is a heterogeneous collection of clonal blood disorders characterized by ineffective hematopoiesis leading to anemia, neutropenia, and thrombocytopenia. While most cases occur in older adults, chemotherapy and other toxin-induced MDS and MDS due to inherited disorders occur in younger persons as well. Those suffering with this syndrome are predisposed to iron overload from repeated transfusions, lethal infections, and life-threatening bleeding. Nearly 30% of patients with MDS develop chemotherapy-resistant acute myeloid leukemia. Mortality resulting from hemorrhage or infection from failure of normal hematopoiesis is high. Most therapies for MDS are suboptimal and not curative. MDS is frequently asymptomatic, but clinically suspected in older patients with abnormal routine blood tests, recurrent infections, or excessive bleeding. Diagnosis requires evaluation of bone marrow for evidence of cells that are maturing abnormally and malignant cells. These findings, combined with peripheral cytopenia(s), abnormal bone marrow cytogenetics, and the degree of transfusion dependence, classify patients with MDS into risk groups and provide a general assessment of disease severity and possible prognosis. Biomarkers clinically useful for accurate diagnosis, prognosis, and prediction of a patient's response to therapy are not available. Currently the etiologies of MDS are not well defined. Distinct mutations and genetic modifications are thought to underlie the molecular basis of MDS, some of which may be independent predictors of poor survival. A comprehensive standardized longitudinal clinical dataset and a consistently-processed, well-annotated biospecimen collection could provide national resources that potentially can be used to: (1) identify genetic, epigenetic and biological factors associated with initiation and progression of MDS; (2) uncover fundamental information on the changes that occur in hematopoietic stem cells and bone marrow stroma during normal aging; (3) reveal clinically useful biomarkers, and; (4) identify potential targets for new interventions. These resources could facilitate the conduct of basic and translational research on the evolution of MDS over time, and improve the diagnosis and clinical management of affected individuals. Information Requested Several approaches are being considered in building a national resource to study MDS in adults. One envisioned approach is to target adults with hematological abnormalities, in particular ones that require a bone marrow aspirate in order to diagnose or eliminate MDS as the diagnosis. Aspirates could undergo centralized multi-reader histopathology review to help ensure consistent diagnosis. From these individuals, up to 2000 adults diagnosed with MDS and a smaller group of participants to serve as comparators could be enrolled. Both groups could be followed over time to determine the natural history of the disease. Academic and community sites that participate in the NCI's Clinical Trials Network would be encouraged to participate in this study. Data would be collected using a secure, web-based data management system, and biospecimens processed centrally using validated procedures, stored in the program's central biorepository, and linked to their phenotypic and clinical data. Clinical data, genomic data, and biospecimens collected from the participants would be part of a national resource that could then be used by the scientific community to address important scientific questions related to MDS in adults. NHLBI and NCI request your input on some or all of the following items regarding the design of a potential MDS natural history cohort study: 1. Please comment on the approach described above, including the design and feasibility. What are the most important scientific questions that this study should answer? Are critical elements missing from the proposed study design? 2. What characteristics are most important in the selection of subjects for the comparator group? How important are age, gender, geographic location, medical history, and exposure histories in enrolling these participants and creating optimal value to the resource? 3. In addition to the information being collected for diagnosis of MDS, what clinical information should be collected from participants and how often should this information be collected? 4. Which patient reported outcomes (e.g., functionality, symptoms, or other) should be collected? 5. Biospecimens collected from study participants would be processed centrally, stored in a central biorepository, and made available to the extramural research community as an NHLBI resource. Please comment on this approach, including: a. What biospecimens (e.g., plasma, DNA, cells) should be collected from each participant for both immediate and future research questions? How frequently should biospecimens be collected from each participant? Should the frequency of collection differ between those diagnosed with MDS and the comparators? b. What assays and molecular testing should be performed as part of this study to expedite future research in MDS, for example, a mutational screen using a specific technology? c. What cell types or tissues should be collected from each participant to serve as controls for future research? For example, in addition to bone marrow and peripheral blood cells, should CD3+ cells, a skin punch, buccal swab cells, or other cells be collected from each participant? 6. What recruitment strategies could be used to optimize participation and reduce barriers to enrollment? 7. Please provide any additional information pertinent to the proposed project that we should consider. How to Submit a Response Interested extramural investigators and other interested parties are invited to respond. Responses to this RFI will be accepted until September 20, 2012 at 4:30 pm EST. You will not receive individualized feedback on any suggestions. No basis for claims against the United States government shall arise as a result of a response to this request for information or from the United States government's use of such information. All comments must be submitted via e-mail as text or as an attached electronic document. Microsoft Word documents are preferred. Please submit your response to the following address: E-mail: NHLBI_MDScohortstudy@nhlbi.nih.gov Disclaimer and Important Notes This is a Request for Information (RFI). This is NOT a solicitation for proposals, proposal abstracts, or quotations. The purpose of this RFI is to obtain knowledge and information for project planning purposes and to enhance study design. This notice does not obligate the Government to award a contract or otherwise pay for the information provided in response. The Government reserves the right to use information provided by respondents for any purpose deemed necessary and legally appropriate. Any organization responding to this notice should ensure that its response is complete and sufficiently detailed to allow the Government to determine the organization's qualifications to perform the work. Respondents are advised that the Government is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted. After a review of the responses received, a pre-solicitation synopsis and solicitation may be published in Federal Business Opportunities. However, responses to this notice will not be considered adequate responses to a solicitation. Confidentiality No proprietary, classified, confidential, or sensitive information should be included in your response. The Government reserves the right to use any non-proprietary technical information in any resultant solicitation(s).
- Web Link
-
FBO.gov Permalink
(https://www.fbo.gov/spg/HHS/NIH/NHLBI/HHS-NIH-NHLBI-RFI-12-147/listing.html)
- Record
- SN02859937-W 20120831/120829235358-ce766e5641ede68c22615e67778bf6bc (fbodaily.com)
- Source
-
FedBizOpps Link to This Notice
(may not be valid after Archive Date)
| FSG Index | This Issue's Index | Today's FBO Daily Index Page |