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The National MDS Natural History Study: design of an integrated data and sample biorepository to promote research studies in myelodysplastic syndromes.

Publication ,  Journal Article
Sekeres, MA; Gore, SD; Stablein, DM; DiFronzo, N; Abel, GA; DeZern, AE; Troy, JD; Rollison, DE; Thomas, JW; Waclawiw, MA; Liu, JJ; Walter, MJ ...
Published in: Leuk Lymphoma
December 2019

Myelodysplastic syndromes (MDS), a spectrum of heterogeneous hematopoietic stem cell diseases, vary in clinical severity, response to therapy, and propensity toward progression to acute myeloid leukemia. These are acquired clonal disorders resulting from somatic mutations within the hematopoietic stem or progenitor cell population. Understanding the natural history and the risk of developing leukemia and other adverse outcomes is dependent on access to well-annotated biospecimens linked to robust clinical and molecular data. To facilitate the acquisition and distribution of MDS biospecimens to the wider scientific community and support scientific discovery in this disease, the National MDS Natural History study was initiated by the National Heart, Lung, and Blood Institute (NHLBI) and is being conducted in collaboration with community hospitals and academic medical centers supported by the National Cancer Institute (NCI). The study will recruit up to 2000 MDS patients or overlapping myeloproliferative neoplasms (MDS/MPN) and up to 500 cases of idiopathic cytopenia of undetermined significance (ICUS). The National MDS Natural History Study (NCT02775383) will offer the world's largest disease-focused tissue biobank linked to longitudinal clinical and molecular data in MDS. Here, we report on the study design features and describe the vanguard phase of 200 cases. The study assembles a comprehensive clinical database, quality of life results, laboratory data, histopathology slides and images, genetic information, hematopoietic and germline tissues representing high-quality biospecimens and data from diverse centers across the United States. These resources will be available to the scientific community for investigator-initiated research.

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Published In

Leuk Lymphoma

DOI

EISSN

1029-2403

Publication Date

December 2019

Volume

60

Issue

13

Start / End Page

3161 / 3171

Location

United States

Related Subject Headings

  • Young Adult
  • United States
  • Research Design
  • Observational Studies as Topic
  • National Heart, Lung, and Blood Institute (U.S.)
  • National Cancer Institute (U.S.)
  • Myelodysplastic Syndromes
  • Mutation
  • Middle Aged
  • Male
 

Citation

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Chicago
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MLA
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Sekeres, M. A., Gore, S. D., Stablein, D. M., DiFronzo, N., Abel, G. A., DeZern, A. E., … Epling-Burnette, P. K. (2019). The National MDS Natural History Study: design of an integrated data and sample biorepository to promote research studies in myelodysplastic syndromes. Leuk Lymphoma, 60(13), 3161–3171. https://doi.org/10.1080/10428194.2019.1616186
Sekeres, Mikkael A., Steven D. Gore, Donald M. Stablein, Nancy DiFronzo, Gregory A. Abel, Amy E. DeZern, Jesse D. Troy, et al. “The National MDS Natural History Study: design of an integrated data and sample biorepository to promote research studies in myelodysplastic syndromes.Leuk Lymphoma 60, no. 13 (December 2019): 3161–71. https://doi.org/10.1080/10428194.2019.1616186.
Sekeres MA, Gore SD, Stablein DM, DiFronzo N, Abel GA, DeZern AE, et al. The National MDS Natural History Study: design of an integrated data and sample biorepository to promote research studies in myelodysplastic syndromes. Leuk Lymphoma. 2019 Dec;60(13):3161–71.
Sekeres, Mikkael A., et al. “The National MDS Natural History Study: design of an integrated data and sample biorepository to promote research studies in myelodysplastic syndromes.Leuk Lymphoma, vol. 60, no. 13, Dec. 2019, pp. 3161–71. Pubmed, doi:10.1080/10428194.2019.1616186.
Sekeres MA, Gore SD, Stablein DM, DiFronzo N, Abel GA, DeZern AE, Troy JD, Rollison DE, Thomas JW, Waclawiw MA, Liu JJ, Al Baghdadi T, Walter MJ, Bejar R, Gorak EJ, Starczynowski DT, Foran JM, Cerhan JR, Moscinski LC, Komrokji RS, Deeg HJ, Epling-Burnette PK. The National MDS Natural History Study: design of an integrated data and sample biorepository to promote research studies in myelodysplastic syndromes. Leuk Lymphoma. 2019 Dec;60(13):3161–3171.

Published In

Leuk Lymphoma

DOI

EISSN

1029-2403

Publication Date

December 2019

Volume

60

Issue

13

Start / End Page

3161 / 3171

Location

United States

Related Subject Headings

  • Young Adult
  • United States
  • Research Design
  • Observational Studies as Topic
  • National Heart, Lung, and Blood Institute (U.S.)
  • National Cancer Institute (U.S.)
  • Myelodysplastic Syndromes
  • Mutation
  • Middle Aged
  • Male