Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Primary Myelofibrosis
Conditions
Keywords
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
Brief summary
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
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.
Interventions
DRUGLuspatercept
Prescribed by treating physician as per product label.
Sponsors
Celgene
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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. Luspatercept treated patients: * Patient must have been at least 18 years of age at the start of luspatercept. * Among LR-MDS patients, patient must have initiated luspatercept on or after September 1, 2023, must be ESA-naïve and luspatercept must be the first active treatment (as monotherapy or part of a treatment regimen) for their disease. * Among all other myeloid malignancies, there is no date restriction for initiation of luspatercept .Patient may have received prior treatment for their disease. * Patient must have at least 3 months of follow-up from start of luspatercept treatment at the participating site.
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). Luspatercept treated patients: * Patient must not be currently or previously enrolled in the Connect Myeloid Registry. * Patient must not have received luspatercept as part of a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment duration - LTC Cohort | Minimum of 3-months post index date | Luspatercept treatment duration |
| Safety and Effectiveness- MF Cohort | Up to 5 years | Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events. |
| Treatment effectiveness - LTC | Minimum of 3-months post index date | Describe clinical response to treatment, transfusion information, ECOG performance status and deaths. |
| Treatment patterns and clinical outcomes - LTC Cohort | Minimum of 3-months post index date | Describe the myeloid malignancy treatment patterns and clinical outcomes before and after initiating luspatercept treatment |
| Transfusion information - LTC | Minimum of 3-months post index date | Describe changes in hemoglobin and transfusion independence status. |
| Patient Demographics- MDS/AML/ICUS Cohorts | Up to 8 years | Describe demographics, baseline characteristics and clinical outcomes of the patients with LR or HR MDS, ICUS, and AML. |
| Diagnostic and Treatment Patterns- MDS/AML/ICUS Cohorts | Up to 8 years | 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 |
| Safety and Effectiveness- MDS/AML/ICUS Cohorts | Up to 8 years | Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events. |
| Patient Demographics- MF Cohort | Up to 5 years | Describe demographics, baseline characteristics, patient recorded outcomes, and clinical outcomes of patients enrolled to the MF cohort |
| Diagnostic and Treatment Patterns- MF Cohort | Up to 5 years | Describe current and evolving patterns for diagnosis, treatment sequencing, routine clinical practice patterns and clinical outcome measures in patients enrolled in the MF Cohort |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Correlative Studies | Up to 8 years | Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples. |
| Patient demographics and clinical characteristics - LTC | Baseline | Describe demographics, baseline characteristics and clinical outcomes of the patients treated with luspatercept |
| Reason for treatment discontinuation - LTC | Minimum of 3-months post index date | Describe reasons for luspatercept treatment discontinuation |
| Patient Reported Outcome | Up to 8 years | 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 |
Countries
Puerto Rico, United States
Outcome results
None listed