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The National Myelodysplastic Syndromes (MDS) Study (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02775383
Recruitment Status : Recruiting
First Posted : May 17, 2016
Last Update Posted : April 26, 2023
National Cancer Institute (NCI)
Information provided by (Responsible Party):
National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary:
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS). Participants will be followed long term. Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.

Condition or disease Intervention/treatment
Myelodysplastic Syndromes (MDS) Other: Therapeutic

Detailed Description:

Multi-center, prospective cohort study enrolling patients from centers in the National Cancer Institute (NCI) National Clinical Trials Network (NCTN) and NCI Community Oncology Research Program (NCORP). The accrual period is 5+ years. After central pathology review of registered participants, approximately 2,000 cases of MDS or MDS/MPN overlap disorders, and 500 cases of ICUS will be identified for the longitudinal study cohort and up to 1000 cases will be identified for the cross-sectional cohort. No more than 3500 total participants will be registered. Participants in the longitudinal cohort may be followed for life.

The goal of the National MDS Study is to establish a publicly available resource to facilitate the study of MDS natural history. This will be accomplished through: 1) Creation of a multi-institutional, longitudinal collection of consistently processed and clinically well-annotated blood and tissue specimens collected prospectively from participants with MDS and participants with idiopathic cytopenia of undetermined significance (ICUS); and 2) Support for investigator-initiated studies of MDS that will have high-impact for MDS patients, including basic science, clinical, health outcomes and epidemiological research.

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Study Type : Observational
Estimated Enrollment : 3500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: The National Myelodysplastic Syndromes Natural History Study
Study Start Date : April 2016
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : January 2025

Group/Cohort Intervention/treatment
Participants with MDS, MDS/MPN overlap disorder, AML <30% blasts without core binding factor or acute promyelocytic leukemia, ICUS, or at risk based on select karyotypic or genetic abnormalities
Other: Therapeutic
Observational Study

Participants who do not have MDS, MDS/MPN overlap disorder, or ICUS and have the baseline visit only
Other: Therapeutic
Observational Study

Primary Outcome Measures :
  1. Number of patients dying [ Time Frame: Through study completion, an average of 3 years ]
    Deaths will be reported on study case report forms

Secondary Outcome Measures :
  1. Number of participants progressing to Acute Myeloid Leukemia (AML) [ Time Frame: Through study completion, an average of 3 years ]
    Progression to AML will be reported on study case report forms

Biospecimen Retention:   Samples With DNA
bone marrow, peripheral blood, serum, plasma, eyebrow hairs with attached follicles, buccal cells, skin biopsy

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with suspected MDS or MDS/MPN overlap disorder who are undergoing diagnostic workup and bone marrow assessments, and patients diagnosed with MDS within 6-months of enrollment and undergoing clinical evaluation and bone marrow assessments to confirm MDS or evaluate disease status

Inclusion Criteria:

  • Suspected (e.g., persistent unexplained cytopenia, circulating peripheral blasts etc.) MDS or MDS/MPN overlap disorders and undergoing diagnostic work-up with planned bone marrow assessments, or diagnosed with de novo or therapy-related MDS within 12 months of enrollment per the World Health Organization (WHO) criteria1 and undergoing clinical evaluation and planned bone marrow assessments to confirm MDS or to evaluate disease status
  • Bone marrow aspirate expected to be performed within 1 week of registration, and in all cases must be performed no later than 4 weeks after enrollment
  • Age 18 or older
  • If anemic without prior MDS diagnosis, B12 level, serum folate, mean corpuscular volume (MCV), red cell distribution width (RDW), ferritin, and iron studies (Iron, total iron-binding capacity (TIBC) test, and percent saturation) must be performed within prior 6 months.

Exclusion Criteria:

  • Prior treatment for MDS at entry and through the time of the entry bone marrow aspirate
  • Treatment with hematopoietic growth factors in prior 6 months
  • Diagnosis of a solid tumor or hematologic malignancy within two years prior to enrollment except for in situ cancer of the skin (basal or squamous cell), cervix, bladder, breast, or prostate
  • Treatment with radiation therapy in the two years prior to registration
  • Non-hormonal treatment for malignancy within the two years prior to registration
  • Established hereditary bone marrow failure syndrome
  • Known primary diagnosis of aplastic anemia, classical paroxysmal nocturnal hemoglobinuria, amegakaryocytic thrombocytopenic purpura, or large granular lymphocyte leukemia
  • Enrolled in the Connect® MDS/AML Disease Registry (NCT01688011)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02775383

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Contact: Kristina Creek 301-251-1151
Contact: Seth Sherman

Show Show 281 study locations
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
National Cancer Institute (NCI)
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Study Chair: Mikkael Sekeres, MD, MS University of Miami
Study Chair: Amy DeZern, MD, MHS Johns Hopkins University
Additional Information:
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Responsible Party: National Heart, Lung, and Blood Institute (NHLBI) Identifier: NCT02775383    
Other Study ID Numbers: NHLBI-MDS
First Posted: May 17, 2016    Key Record Dates
Last Update Posted: April 26, 2023
Last Verified: April 2023
Keywords provided by National Heart, Lung, and Blood Institute (NHLBI):
National MDS Study
Myeloproliferative Neoplasm
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions