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Tissue Collection and Natural History Study of Patients with Myelodysplastic Syndrome or Myeloproliferative Neoplasms

Status
Active
Cancer Type
Cancer-Related Syndrome
Leukemia
Myelodysplastic Syndromes (MDS)
Trial Phase
Eligibility
18 Years and older, Male and Female
Study Type
Other
NCT ID
NCT02775383
Protocol IDs
NHLBI-MDS (primary)
NCI-2016-00281
Study Sponsor
ECOG-ACRIN Cancer Research Group

Summary

This research trial collects tissue samples from and studies the history of patients with myelodysplastic syndrome (MDS) or myeloproliferative neoplasms (MPN). Collecting and storing patients' bone marrow, blood, eyebrow hairs, buccal swab, skin, or other tissues to be studied in the laboratory in the future may help doctors learn more about MDS and blood disorders that may lead to MDS. Collecting information about patients and the treatments they receive may allow doctors to better understand how MDS changes over time and this knowledge may lead to better ways to prevent, detect, and treat MDS in the future.

Objectives

PRIMARY OBJECTIVES:
I. 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.
II. 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: 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; and quality management procedures to ensure minimal numbers of errors in the management of the biospecimens.
III. 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.

SECONDARY OBJECTIVES:
I. Detecting improved prognostic factors in low risk MDS. (Potential Studies)
II. Detecting markers of improved response to hypomethylating therapy. (Potential Studies)
III. Quality of life (QOL) and anemia therapy. (Potential Studies)
IV. Genetic factors in patients with complex karyotype. (Potential Studies)
V. Prognostic significance of splicing factor 3b subunit 1 (SF3B1) in MDS patients with refractory anemia with ring sideroblasts (RARS). (Potential Studies)

OUTLINE:
Patients undergo blood, bone marrow, eyebrow hairs, and buccal swab. Patients' medical records, baseline laboratory tests, quality of life, and diagnostic information including pathology reports and treatment history are also reviewed.

After the baseline visit, patients are followed up every 6 months. Follow up visits include disease evaluation, physical examination and collection of peripheral blood sample. Bone marrow biopsy and aspiration is performed when clinically necessary. Patients also complete the MDS-specific Qualify of Life in Myelodysplasia Scale (QUALMS), Functional Assessment of Cancer Therapy-General (FACT-G) (version 4), the Patient Reported Outcome Measurement Information System (PROMIS) Short Form version 1.0-Fatigue 7a, and the European Quality of Life-5 Dimensions-5 Levels (EQ-5D-5L).

Eligibility

  1. 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
  2. Diagnosed with de novo or therapy-related MDS within 12-months of enrollment per the World Health Organization (WHO) criteria and undergoing clinical evaluation and planned bone marrow assessments to confirm MDS or to evaluate disease status
  3. 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
  4. No prior treatment for MDS at entry and through the time of the entry bone marrow aspirate
  5. No treatment with hematopoietic growth factors in prior 6 months
  6. If anemic without prior MDS diagnosis, the following tests within the prior 6 months; values that are significantly outside of normal range do not exclude participation but should prompt investigation of alternative etiologies for anemia * B12 level * Serum folate * Mean corpuscular volume (MCV) * Red cell distribution width (RDW) * Ferritin * Iron studies (iron, total iron-binding capacity [TIBC] test, percent saturation)
  7. 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
  8. No treatment with radiation therapy in the two years prior to registration
  9. No non-hormonal treatment for malignancy within the two years prior to registration
  10. No established hereditary bone marrow failure syndrome
  11. No known primary diagnosis of aplastic anemia, classical paroxysmal nocturnal hemoglobinuria, amegakaryocytic thrombocytopenic purpura, or large granular lymphocyte leukemia
  12. Not enrolled in the Connect MDS/Acute Myeloid Leukemia (AML) Disease Registry
  13. 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 * Direct antiglobulin test * Antinuclear antibody (ANA) test * Creatinine * Calculated glomerular filtration rate (GFR) * Thyroid-stimulating hormone (TSH) tests performed in prior 6 months ** Based on centralized pathology review, participants will be classified into the longitudinal cohort of cases (MDS; MDS/MPN overlap disorders; AML with < 30% blasts without core binding factor or acute promyelocytic leukemia [AML < 30% blasts including chromosomal rearrangements between chromosomes 8 and 21 and within chromosome 16 as well as t(15;17)]; idiopathic cytopenia of undetermined significance [ICUS], or at risk based on selected genetic markers) and the cross-sectional cohort (all others); it is not known in advance what percentages of individuals will fall into each cohort; in addition to baseline biological samples, longitudinal samples and data will be collected for approximately 2000 cases of MDS or MDS/MPN overlap disorders and 500 cases of ICUS assigned to the longitudinal cohort; sample and data collection will cease at baseline for all cases assigned to the cross-sectional cohort; submitted samples will be reviewed by a central pathologist to determine eligibility for the longitudinal cohort (i.e., an MDS, MDS/MPN, AML with < 30% blasts without core binding factor or acute promyelocytic leukemia, or ICUS diagnosis); should a discrepancy in diagnosis occur between the central review and study site, the study site will be notified to allow for additional information to be submitted to clarify the diagnosis; such notifications will not occur in real time, and are not intended to assist in patient care; additional central sequencing of selected genetic targets will be performed

Treatment Sites in Georgia

Atlanta Cancer Care - Conyers


1498 Klondike Road
Suite 106
Conyers, GA 30094
404-303-3355
www.atlantacancercare.com

Atlanta Cancer Care - Decatur


2545 Lawrenceville Highway
Suite 300
Decatur, GA 30033
404-303-3355
www.atlantacancercare.com

Doctors:

Lijo Simpson MD

Atlanta Cancer Care - Stockbridge


7813 Spivey Station Boulevard
Suite 210
Jonesboro, GA 30236
678 466-2069
www.atlantacancercare.com

Doctors:

Gurinderjit K. Sidhu MD
Lijo Simpson MD

Georgia Cancer Specialists - CenterPointe


1100 Johnson Ferry Road
Suite 600
Sandy Springs, GA 30342
404-256-4777 ext 9242
www.gacancer.com

Doctors:

Rodolfo E. Bordoni MD
Pradeep C. Jolly MD

Georgia Cancer Specialists - Macon-Coliseum


308 Coliseum Drive
Suite 120
Macon, GA 31217
478-745-6130 x8152
www.gacancer.com

Georgia Cancer Specialists - Stemmer


2712 Lawrenceville Highway
Decatur, GA 30033
770-496-5555
www.gacancer.com

Northside Hospital Cancer Institute - Forsyth


1200 Northside Forsyth Drive
Suite 140
Cumming, GA 30041
404-303-3355
www.northside.com

**Clinical trials are research studies that involve people. These studies test new ways to prevent, detect, diagnose, or treat diseases. People who take part in cancer clinical trials have an opportunity to contribute to scientists’ knowledge about cancer and to help in the development of improved cancer treatments. They also receive state-of-the-art care from cancer experts... Click here to learn more about clinical trials.
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