Rare Disease Report

ASCO: Young AML Patients' Quality of Life Improved by Novartis Therapy CTL019

JUNE 03, 2017
Mathew Shanley
New data concludes that both pediatric and young adult patients saw clinically meaningful quality of life (QOL) improvements at 3 and 6 months after undergoing CTL019 therapy.
These data, presented at the 2017 American Society of Clinical Oncology (ASCO 2017) annual meeting in Chicago by Andrew Charles Dietz, et al., suggest improved QOL after this one-time immunocellular therapy beyond the period of acute toxicities, and further confirms the clinical benefits, safety and efficacy of CTL019.
AML, a cancer of the myeloid line of blood cells, is categorized by the expedited growth of abnormal white blood cells built up in the bone marrow. This growth interferes with the production of normal blood cells. CTL019, a single infusion of genetically modified autologous chimeric antigen, was first developed by the University of Pennsylvania. In 2012, Novartis and the entered a global collaboration to further research, develop therapies for the investigational treatment of cancers; CTL019 was among them.
81 patients were enrolled in the global ELIANA trial, a global Phase II, single arm, open-label, multicenter trial, and all were between 3-23 years, 39 of whom were younger than 8. Sixty-eight of the enrolled patients were infused with CTL019, and 59% had relapsed after SCT with median of 3 prior therapies.
Patient-reported quality of life (QOL) was measured before and after a lone 1-time infusion of CT019 using 2 instruments [Pediatric Quality of Life (PedsQL) and EuroQol (EQ-5D)]. The former is a standardized, general evaluation of QOL observations in pediatric patients with health conditions, and consists of emotional, social, and school functioning subscale scores; physical and psychosocial health summary scores; and total score.
ED-5D is a standardized measure of health status pertinent to a broad range of health conditions and treatments. It consists of a descriptive system, which comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), and a visual analog scale (VAS), which records the patient’s self-rated health state.
The percentage of patients reporting no problems on EQ-5D dimensions increased from the baseline to month 6 in all categories. Slight decreases in patient percentages were shown from month 3 to month 6 in mobility, usual activities, and pain/discomfort.
Overall, the outcomes of the trial demonstrate improved patient-reported QOL after the one-time CTL019 infusion beyond the period of acute toxicities.


Dietz AC, et al. Patient-reported quality of life (QOL) following CTL019 in pediatric and young adult patients (pts) with relapsed/refractory (r/r) b-cell acute lymphoblastic leukemia (B-ALL). J Clin Oncol 35, 2017 (suppl; abstr 10523).

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