Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Study Overview
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Brief Summary
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The use of genomics and functional translational studies is to identify and diagnose patients with precursor features of myeloid neoplasms and inherited bone marrow failure syndromes.

Detailed Description
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The pre-myeloid cancer and bone marrow failure clinic is an individualized medicine clinic conceptualized for the early detection of myeloid malignancies such as, myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN), MDS/MPN overlap syndromes and acute myeloid leukemia (AML). Using next-generation sequencing technologies, clonal hematopoietic stem cell changes can be detected early on in myeloid malignancies. These changes are currently classified as follows: Idiopathic cytopenias of unclear significance (ICUS). Clonal cytopenias of unclear significance (CCUS). Clonal hematopoiesis of indeterminate prognosis (CHIP). Bone marrow failure syndromes with risk for clonal myeloid transformation. Familial/Germline syndromes with increased risk for myeloid malignancies. This clinic blends next-generation sequencing technologies with functional studies to help identify, prognosticate and define management plans; including early intervention strategies, for affected patients.

Official Title
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Pre-myeloid Cancer and Bone Marrow Failure Clinic Study

Conditions
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Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion, Cytopenia

Intervention / Treatment
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* Other: follow up


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with: Idiopathic cytopenias of unclear significance (ICUS) Clonal hematopoiesis of indeterminate significance (CHIP) Age related clonal hematopoiesis (ARCH) Clonal cytopenias of unclear significance (CCUS) Marrow failure syndromes with myeloid malignancy predisposition- telomere dysfunction, chromosomal breakage disorders, etc. Germ line inherited syndromes with risk for malignant transformation Low Risk Myelodysplastic Syndromes (MDS) Exclusion Criteria: Patients who are unable to understand and sign the Informed Consent Form

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Screening
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Potential Premyeloid Cancer or Bone Marrow Failure Patients<br>Follow up provided for patients tested using Next Generation Sequencing (NGS) and other relevant functional studies | Other: follow up<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of subjects whose cytopenias are persistent or progressive over the course of the 5 year study |  | Through study completion, an average of 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of subjects who have evolved to myelodspalstic syndrome (MDS) or acute myeloid leukemia (aml) over the course of the five year study |  | Through study completion, an average of 5 years |