Details
Title Protocol for the collection of information and samples to be used in the study of MDS
IRB NHLBI-MDS
CC 16-817
Hospital Akron, Fairview, Hillcrest, Main Campus, Mansfield, North Coast Cancer, Wooster
Stage Advanced/Metastatic
Phase Phase 2
Disease GI (Gastrointestinal), Myelodisplastic Syndrome (MDS) , Pancreas
Drug Capecitabine, Cisplatin, Etoposide, Temozolomide
Description
- To develop a high-quality clinical database containing clinical history, including environmental exposure history, presenting signs and symptoms, diagnostic testing results, co-existing diseases, therapies and response to therapies, disease progression, quality of life and survival.
- To develop a high-quality biorepository linked to the clinical data that will facilitate diverse studies, including genetic, epigenetic, immunologic, proteomic, and cell-functional and cell-phenotypic studies through the development of (details described further in the Manual of Procedures):
- Central communication with the biorepository to ensure timely and accurate collections and biospecimen data appended to the clinical database.
- Defined standard operating procedures for the collection, processing, storage and distribution, with special emphasis on processing protocols fit-for-purpose to sample requirements for downstream testing.
- Quality management procedures to ensure minimal numbers of errors in the management of the biospecimens.
- To facilitate broad use of these linked data and specimens to support studies focused on:
- Improving diagnostic accuracy, risk-stratification and prognostication, and medical decision-making in MDS;
- Understanding quality of life and its relationship to changing disease and treatment status
- Understanding the pathogenesis of MDS and diverse MDS subtypes, including genetic, epigenetic, immunologic mechanisms;
- Optimizing treatment strategies for specific subtypes of MDS;
- Identifying novel biomarkers for MDS outcomes; and
- Identifying novel targets for therapeutic interventions in MDS.
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
- No prior treatment for MDS at entry and through the time of the entry bone marrow aspirate
- No treatment with hematopoietic growth factors in prior 6 months
- B12 level, serum folate, ferritin, and Thyroid-Stimulating Hormone (TSH) tests performed in prior 6 months
- No 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
- No treatment with radiation therapy in the two years prior to registration
- No non-hormonal treatment for malignancy within the two years prior to registration
- No established hereditary bone marrow failure syndrome
- No known primary diagnosis of aplastic anemia, classical paroxysmal nocturnal hemoglobinuria, amegakaryocytic thrombocytopenic purpura, or large granular lymphocyte leukemia
- Not enrolled in the Connect® MDS/AML Disease Registry
Note: See Appendix II for WHO peripheral blood and bone marrow findings in MDS. In participants with suspected MDS and prior to registration with subsequent bone marrow evaluation, alternative causes for the cytopenias should be considered (e.g., internal bleeding, autoimmune cytopenias, thyroid disorders, other causes of anemia etc.). In select individuals, the following tests could be performed to assist in the diagnostic work-up. These evaluations are not required by the protocol; however, abnormal results in advance of enrollment may reduce the number of non-MDS cases.
- Copper, serum level
- Iron studies (Iron, Total Iron-Binding Capacity (TIBC) Test, Percent Saturation)
- Direct Antiglobulin Test
- Antinuclear Antibody (ANA) Test
