CPX-351 Real-World Effectiveness and Safety Study

Jazz Pharmaceuticals147 enrolled

Overview

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).

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.

Study Type

OBSERVATIONAL

Enrollment

147

Conditions

Acute Myeloid Leukemia

Interventions

CPX-351DRUG

This is a retrospective, non-interventional, observational study. No study drug will be administered in this study.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (15)

Belfast City Hospital

Belfast, United Kingdom

Bristol Royal Infirmary

Bristol, United Kingdom

University Hospitals Derby and Burton

Outcomes

Primary Outcomes

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

Time frame: 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

Time frame: First infusion of Vyxeos liposomal (post-August 2018 approval) to date of death, up to approximately 3 years 4 months

Secondary Outcomes

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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Data from ClinicalTrials.gov

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