The National Myelodysplastic Syndromes (MDS) Study

Conditions:Blood Cancer, Blood Cancer
Therapuetic Areas:Oncology
Age Range:18 - Any
Start Date:April 2016
End Date:September 2021
Contact:Chuck Wagner

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The National Myelodysplastic Syndromes Natural History Study

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.

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.

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 6-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

- B12 level, serum folate, ferritin, and Thyroid-Stimulating Hormone (TSH) tests
performed in prior 6 months

Exclusion Criteria:

- Prior treatment for MDS at entry and through the time of the entry bone marrow

- 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

- Enrolled in the Connect® MDS/AML Disease Registry (NCT01688011)
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