The National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI) are seeking input from the Myelodysplastic Syndromes (MDS) community (practitioners, scientific researchers, patients and patient advocacy groups) on the feasibility and design of a proposed MDS natural history study. The study being considered would collect, store, and make available to the wider scientific community, biospecimens and data to facilitate research leading to a better understanding of disease progression. MDS represents an important disorder for scientific focus given its epidemiologic link to senescence, its significance as a pre-leukemia model, and its relevance to stem cell biology. With the aging of a large segment of the American population who are at emerging risk for these disorders, the advancement of research and treatment of MDS is one of the ASH's priorities. ASH applauds the NHLBI and the NCI for spearheading this effort, in concert with the recommendations from the 2010 ASH workshop on MDS.
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.
Specifically, the NHLBI and NCI request the community's input on the following:
- Comment on the approach described above, including the design and feasibility, the most important scientific questions that this study should answer, and/or critical elements missing from the proposed study design.
- The importance of the selection of subjects for the comparator group, as well as the importance of age, gender, geographic location, medical history, and exposure histories in enrolling these participants and creating optimal value to the resource.
- In addition to the information being collected for diagnosis of MDS, we welcome feedback about the clinical information that should be collected from participants and how often should this information be collected.
- Collection of patient reported outcomes (e.g., functionality, symptoms, or other).
- 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. Comments on this approach should include, but are not limited to:
- The type of biospecimens (e.g., plasma, DNA, cells) collection from each participant for both immediate and future research questions. Frequency of biospecimen collection from each participant. The frequency of collection differ between those diagnosed with MDS and the comparators.
- Assays and molecular testing performed as part of this study to expedite future research in MDS.
- The type of cells or tissues collected from each participant to serve as controls for future research.
- Recruitment strategies that could be used to optimize participation and reduce barriers to enrollment.
ASH members are encouraged to submit their comments on the proposed study to the ASH Senior Manager for Scientific Affairs, Ulyana Desiderio, PhD, to firstname.lastname@example.org or (202) 776-0544. Comments are being accepted through September 20.
Please see the RFI for more information.
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