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.
Key Inclusion Criteria
For a patient to be eligible for participation in this study, all of the following criteria must apply.
- 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
Key Exclusion Criteria
A patient will not be eligible for participation in this study if any of the following criteria apply.
- 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)
To learn more, visit ClinicalTrials.Gov
National Heart, Lung, and Blood Institute (NHLBI)