Zwaan CM, et al. – A phase 4, multicentre, single–arm, observational, open–label trial was set up to provide additional data on the safety and efficacy of nelarabine under licensed conditions of use in children and young adults ≤21 years of age. Data demonstrated a consistency of the response rate, median OS, and safety profile of nelarabine in this disease setting and population with those reported previously.

• As per observations, patients (N = 28) had a mean ± standard deviation age of 11.5 ± 4.6 years.
• Among these patients 71% were male and 61% had a diagnosis of T-ALL.
• 46% and 21% experienced adverse events (AEs) and treatment-related AEs.
• These adverse events included few haematological AEs and no haematological serious AEs.
• In four patients, clinicians reported neurological AEs from one of four predefined categories (peripheral and central nervous systems, mental status change and uncategorized).
• No AE-related treatment discontinuations/withdrawals were observed.
• Moreover, the overall response rate was 39.3%: complete response (CR), 35.7%; CR without full haematological recovery (CR*), 3.6%.
• For 46% of the cohort, post-treatment stem cell transplantation was performed.
• Furthermore, median overall survival (OS) was 3.35 months for non-responders and not reached for responders (CR + CR*).