CPX-351 Real-World Effectiveness and Safety Study

Study Overview
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Brief Summary
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CPX-351 Real World Effectiveness and Safety Study (CREST UK) is a real-world evidence study designed to collect data on the potential benefits and/or risks of Vyxeos liposomal (liposomal daunorubicin/cytarabine; CPX-351) in routine clinical practice in the United Kingdom (UK).

Detailed Description
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This is a retrospective, non interventional, multi-centre, single arm, observational study designed to assess patients with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or acute myeloid leukaemia with myelodysplasia related changes (AML-MRC) who have been treated with Vyxeos liposomal (liposomal daunorubicin/cytarabine; CPX-351) in routine UK clinical practice and managed as per standard local practice.

Official Title
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CREST UK: CPX-351 Real-World Effectiveness and Safety Study

Conditions
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Acute Myeloid Leukemia

Intervention / Treatment
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* Drug: CPX-351


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Aged ≥18 years at the start of treatment of AML with Vyxeos liposomal Pathological diagnosis of t-AML or AML-MRC according to World Health Organization criteria (with at least 20% blasts in the peripheral blood or bone marrow) Patient has received at least one infusion of Vyxeos liposomal, prescribed as per the SmPC Patient signs an informed consent form or is included in accordance with an informed consent waiver Exclusion Criteria: Treatment with Vyxeos liposomal as part of a clinical trial or managed access program Prior treatment intended for induction therapy of AML; only hydroxyurea is permitted for control of blood counts

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 |
| --- | --- |
| CPX-351<br>Adult patients with newly diagnosed, therapy related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) who were treated in routine practice with Vyxeos liposomal in the UK. | Drug: CPX-351<br>* This is a retrospective, non-interventional, observational study. No study drug will be administered in this study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Achieving Complete Response/Complete Response with Incomplete Platelet or Neutrophil Recovery in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months | 
| Overall Survival in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | First infusion of Vyxeos liposomal (post-August 2018 approval) to date of death, up to approximately 3 years 4 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Vyxeos Liposomal Induction and Consolidation Cycles Administered (Inpatient or Outpatient) in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months | 
| Dose Per Cycle of Vyxeos Liposomal Given During Induction and Consolidation Cycles Administered in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months | 
| Percentage of Participants Transferred for Haematopoietic Stem Cell Transplant (HSCT) in Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months | 
| Overall Survival in Participants Post-HSCT With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | Date of HSCT to date of death, up to approximately 3 years 4 months | 
| Mean Duration of Hospitalization Stay of Participants With AML Who Were Treated in Routine Practice With Vyxeos Liposomal |  | First infusion of Vyxeos liposomal (post-August 2018 approval) to inclusion in study, up to approximately 3 years 4 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acute Myeloid Leukemia, CPX-351, Vyxeos liposomal