CREST‐UK: Real‐world effectiveness, safety and outpatient delivery of CPX‐351 for first‐line treatment of newly diagnosed therapy‐related AML and AML with myelodysplasia‐related changes in the UK
医学
置信区间
内科学
临床试验
移植
儿科
外科
作者
Priyanka Mehta,Victoria Campbell,Jamie Maddox,Yngvar Fløisand,Anita J. M. Kalakonda,Jenny O’Nions,Thomas Coats,Sateesh K. Nagumantry,Katherine Hodgson,Richard Whitmill,Ian Amott,Gillian Flynn,David Taussig,Rui Zhao,Nicholas Cunningham,Montse Roset,Daniel Cuadras,Greg Medalla,Hayley Kuter,Saemi Park,Alex Legg,Anjum Khan
Summary Favourable outcomes with CPX‐351 versus conventional 7 + 3 were demonstrated in the pivotal phase III trial in adults aged 60–75 years with newly diagnosed, highrisk/secondary acute myeloid leukaemia (AML). As a complement to the clinical trial and to address important data gaps, the C PX‐351 R eal‐World E ffectiveness and S afe T y (CREST‐UK; NCT05169307) study evaluated the use of CPX‐351 in routine clinical practice in the UK, in 147 patients with newly diagnosed therapy‐related AML or AML with myelodysplasia‐related changes. Best response of complete remission or complete remission with incomplete platelet or neutrophil recovery was achieved by 53% of evaluable patients. Kaplan–Meier median overall survival (OS) was 12.8 months (95% confidence interval 9.2–15.3). Fifty (34%) patients proceeded to haematopoietic cell transplantation (HCT); median OS landmarked from the HCT date was not reached. There were no new safety concerns with CPX‐351 identified in CREST‐UK. Patients treated with CPX‐351 in the outpatient setting spent an average of 24.4, 16.7, 28.2, and 27.7 fewer days on the ward compared with inpatients during first induction, second induction, first consolidation, and second consolidation, respectively. The results from CREST‐UK provide valuable insights into the effectiveness, safety, and outpatient delivery of CPX‐351 in routine clinical practice in the UK.