Abstract 2914
Background
Acute lymphoblastic leukaemia (ALL) is associated with high survival rates in paediatrics and young adults and less favourable outcomes in older adults. Clinical trials (CTs) studying survival-based endpoints in ALL lead to significantly prolonged trial durations, resulting in delays to medicines access. Biomarkers in ALL have been correlated with longer-term outcomes, potentially accelerating the clinical development of novel treatments. The aim of this study was to characterise efficacy measures investigated in ALL CTs conducted in the European Union (EU)/Economic Area (EEA).
Methods
Interventional Phase II to Phase IV ALL CTs registered in the EU Clinical Trials Register over an 11-year period (2007-2017) were identified. Therapeutic CTs reporting efficacy data in the English language for investigational medicinal products of chemical, biological and biotechnological origin were included in the study. A protocol design or age filter was not applied to avoid limiting the scope of outcomes identified. Primary and secondary efficacy endpoints were extracted from the selected CTs and categorised according to type of measurement. A data mining process was performed to detect trends in outcomes studied.
Results
The data set comprised 68 CTs representing about 20,000 patients. The majority of trials (69%, n = 47) recruited patients from the adult population (18-64 years) and conducted only Phase II studies (62%, n = 42). Twenty-three unique efficacy endpoints were identified and stratified into the clusters of survival (n = 4), time-to-event (n = 3), response rates and biomarkers (n = 11) and other (n = 5). Fifty-three per cent (n = 36) of the trials reported 4-10 outcomes, with a mean of 4 outcomes per CT (range 1-10). The principal endpoints evaluated in CTs consisted of overall survival (CTs: 63%, n = 43), minimal residual disease (CTs: 50%, n = 34), event-free survival (CTs: 40%, n = 27) and disease-free survival (CTs: 40%, n = 27).
Conclusions
The high uptake of minimal residual disease as an efficacy parameter in ALL CTs is in line with reported findings confirming the prognostic value of this marker on clinical outcomes. Heterogeneity in the selection of efficacy endpoints was observed which warrants future work on the standardisation of efficacy outcomes in ALL CTs.
Clinical trial identification
Editorial acknowledgement
The Malta Medicines Authority. The Department of Pharmacy, Faculty of Medicine and Surgery, University of Malta.
Legal entity responsible for the study
Department of Pharmacy, Faculty of Medicine and Surgery, University of Malta.
Funding
Government of Malta, Endeavour Scholarship Scheme 2017.
Disclosure
All authors have declared no conflicts of interest.
Resources from the same session
2182 - Evaluating the prevalence of the expression of PD-L1 in NSCLC specimens with short-duration formalin fixation using IHC 22C3 pharmDx
Presenter: Keiichi Ota
Session: Poster Display session 1
Resources:
Abstract
5255 - [18F]-FDG PET/CT in predicting PD-L1 status in nasopharyngeal carcinoma
Presenter: Liang Zhao
Session: Poster Display session 1
Resources:
Abstract
4910 - Expression of PD-L1 in Chinese Patients with Common Cancers
Presenter: Min Zheng
Session: Poster Display session 1
Resources:
Abstract
4227 - The clearance of EGF by tumor-associated macrophages is suppressed by chemotherapeutic agent cisplatin
Presenter: Irina Larionova
Session: Poster Display session 1
Resources:
Abstract
5222 - VHIO-300 and a thousand one nights, a tale of Precision Medicine
Presenter: Ginevra Caratù
Session: Poster Display session 1
Resources:
Abstract
5668 - Matched Whole-Genome Sequencing and Whole-Exome Sequencing with Circulating Tumor DNA (ctDNA) Analysis are complementary modalities in clinical practice
Presenter: Robin Imperial
Session: Poster Display session 1
Resources:
Abstract
5772 - Exploring the role of genes associated with familial cancer syndromes on the development of multiple primary tumors
Presenter: Atanaska Mitkova
Session: Poster Display session 1
Resources:
Abstract
4784 - Doxorubicin resistance: early and advanced tumors can use two different strategies based on initial and profound abnormalities in microRNA expression signature
Presenter: Volodymyr Halytskiy
Session: Poster Display session 1
Resources:
Abstract
3456 - From tumor transcriptomes to underlying cell type proportions to better predict prognosis and response to treatments
Presenter: Yuna Blum
Session: Poster Display session 1
Resources:
Abstract
4976 - Optimization of automated germline DNA extraction from non-tumoral formalin-fixed paraffin embedded (FFPE) tissues
Presenter: Omar Youssef
Session: Poster Display session 1
Resources:
Abstract