Connect® Myeloid Disease Registry

Study Overview
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Brief Summary
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The purpose of the Connect® Myeloid disease registry is to provide unique insights into treatment decisions and treatment patterns as they relate to clinical outcomes of patients with myeloid diseases in routine clinical practice. This disease registry will also evaluate molecular and cellular markers that may provide further prognostic classification which may or may not be predictive of therapy and clinical outcomes.

Detailed Description
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This Disease Registry will collect data on patient characteristics, treatment patterns and clinical outcomes. The objective is to describe how patients with myeloid diseases are treated; and to build a knowledge base regarding the effectiveness and safety of first line and subsequent treatment regimens in both community and academic settings. Enrolled patients will receive treatment and evaluations for their disease according to the standard of care and routine clinical practice at each study site. All treatments that patients receive for their disease will be recorded, including initial treatment and any subsequent therapy. Data on treatment outcomes, including response rates as measured by the treating physician, evidence of progression, survival, and patient-reported outcomes will be collected quarterly on the electronic CRF.

Official Title
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Connect® Myeloid: The Myelofibrosis (MF), Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) Disease Registry

Conditions
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Primary Myelofibrosis, Myelodysplastic Syndromes, Leukemia, Myeloid, Acute

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients must be able to provide written informed consent form (ICF) Must be willing and able to complete baseline and follow-up HRQoL instruments, for which patients must be proficient in either English or Spanish AML patients must be at least 55 years of age at the time of informed consent. MF, ICUS, and MDS patients must be at least 18 years of age at the time of informed consent. Newly diagnosed Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML) patients: Newly diagnosed primary or secondary disease. To be considered newly diagnosed, a patient's confirmed diagnosis must be made no more than 60 days prior to the date of consent signature. (An additional 5-day window [i.e., up to 65 days prior to the date of ICF signature] may be allowed in special circumstance upon sponsor approval) Cohort assignment confirmed by central eligibility review. Cohort assignment must also be confirmed by the site. Myelofibrosis (MF) patients: Patients who initiated their first active systemic treatment for MF and/or MF-related cytopenias within 90 days prior to the date of consent signature. This cohort allows the enrollment of subjects with a diagnosis of Myelodysplastic/Myeloproliferative overlap syndromes (MDS/MPN overlap syndrome). Cohort assignment is confirmed by the site. Central eligibility review is not required. Treated Lower-Risk Myelodysplastic Syndromes (LR-MDS) patients: Patients who have initiated first active treatment regimen containing at least one non-ESA therapy, within 90 days prior to ICF Cohort assignment is confirmed by site. Central eligibility review is not required. Exclusion Criteria: Suspected or proven acute promyelocytic leukemia (APL) (FAB M3 or WHO 2008) based on morphology, immunophenotype, molecular assay or karyotype Currently enrolled in any interventional clinical trial where the patient is being treated with an investigational product that cannot be identified. Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS) patients who received or are receiving active (disease modifying) therapy for the treatment of MDS prior to the date of informed consent. Acute Myeloid Leukemia (AML) patients who initiated active (disease modifying treatment for AML more than 2 weeks prior to the date of consent. Myelofibrosis (MF) and Myelodysplastic/Myeloproliferative (MDS/MPN) overlap syndrome patients with suspected juvenile myelomonocytic leukemia (JMML).

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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Newly diagnosed Lower-Risk Myelodysplastic Syndromes (LR-MDS)<br>Newly diagnosed lower risk MDS patients as determined by International Prognostic Scoring System (IPSS). | |
| Newly diagnosed Higher-Risk Myelodysplastic Syndromes (HR-MDS)<br>Newly diagnosed higher risk MDS patients as determined by International Prognostic Scoring System (IPSS). | |
| Newly diagnosed Acute Myeloid Leukemia (AML)<br>Newly diagnosed AML patients (≥55 years old, excluding patients with acute promyelocytic leukemia (APL). | |
| Treated Myelofibrosis (MF)<br>Newly treated MF patients receiving treatment for MF or MF-related cytopenias. This cohort also includes patients with myelodysplastic syndrome (MDS)/myeloproliferative neoplasm (MPN) overlap syndromes, excluding juvenile myelomonocytic leukemia (JMML). | |
| Newly diagnosed Idiopathic cytopenia of undetermined significance (ICUS)<br>Newly diagnosed ICUS patients. | |
| Treated Lower-Risk Myelodysplastic Syndromes (LR-MDS)<br>Treated LR-MDS patients receiving first active treatment regimen containing at least one non-ESA therapy | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patient Demographics- MDS/AML/ICUS Cohorts | Describe demographics, baseline characteristics and clinical outcomes of the patients with LR or HR MDS, ICUS, and AML. | Up to 8 years | 
| Diagnostic and Treatment Patterns- MDS/AML/ICUS Cohorts | Describe current and evolving patterns for diagnosis, treatment sequencing, routine clinical practice patterns and clinical outcome measures in patients with LR or HR MDS, ICUS, and AML | Up to 8 years | 
| Safety and Effectiveness- MDS/AML/ICUS Cohorts | Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events. | Up to 8 years | 
| Patient Demographics- MF Cohort | Describe demographics, baseline characteristics, patient recorded outcomes, and clinical outcomes of patients enrolled to the MF cohort | Up to 5 years | 
| Diagnostic and Treatment Patterns- MF Cohort | Describe current and evolving patterns for diagnosis, treatment sequencing, routine clinical practice patterns and clinical outcome measures in patients enrolled in the MF Cohort | Up to 5 years | 
| Safety and Effectiveness- MF Cohort | Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events. | Up to 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Patient Reported Outcome | Summarize patient reported outcomes (including e.g., Health-Related Quality of Life (HRQOL)) and economic outcomes, and their association with patient characteristics, treatment regimens, and clinical outcomes | Up to 8 years | 
| Correlative Studies | Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples. | Up to 8 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Myelodysplastic syndromes, MDS, Acute myeloid leukemia, AML, Registry, Connect®, ICUS, Idiopathic Cytopenias of Undetermined Significance, Myelofibrosis, MF, Myelodysplastic/Myeloproliferative overlap syndromes, MDS/MPN overlap syndromes