Outcomes of adolescents and young adults with chronic-phase chronic myeloid leukaemia treated with tyrosine kinase inhibitors

Abstract

Introduction Few studies have reported the outcomes of adolescents and young adults (AYAs) with chronic-phase chronic myeloid leukaemia (CML-CP) on tyrosine kinase inhibitors (TKIs). Materials and methods We retrospectively analysed the clinical features, treatment response, and long-term outcomes of 42 AYA patients, in comparison to older patients. The initial therapies of AYA patients between 2001 and 2016 included imatinib ( n = 24), dasatinib ( n = 13) and nilotinib ( n = 5). Results In AYA patients, the peripheral blood (PB) white blood cell count and percentage of blasts at the diagnosis were significantly higher, haemoglobin levels were lower and the spleen size was larger. The major molecular response (MMR), event-free survival (EFS) and overall survival (OS) rates were comparable. A sub-analysis comparing imatinib to second-generation TKIs as the initial therapy also showed that their prognosis was comparable. Discussion In conclusion, the tumour burden at the diagnosis of CML-CP is higher in AYA patients; however, their prognosis was not worse in comparison to older patients treated with TKIs. KEY MESSAGES Few studies have reported the outcomes of adolescents and young adults (AYAs) with chronic-phase chronic myeloid leukaemia (CML-CP) on tyrosine kinase inhibitors (TKIs). This study showed the tumour burden at the diagnosis of CML-CP is higher in AYA pa tients; however, their prognosis was not worse in comparison to older patients treated with TKIs. Understanding the biological and non-biological features of AYA patients with CML-CP on TKI therapy is essential for better management and to improve the outcomes.

Asciminib in the Treatment of Philadelphia Chromosome-Positive Chronic Myeloid Leukemia: Focus on Patient Selection and Outcomes

“… 89 Nonadherence is estimated to impact up to 60% of AYA patients with cancer and is associated with poorer outcomes. 89 AYA are also expected to have extended periods of treatment, thereby experiencing similar challenges and survivorship issues to pediatric patients. 90 , 91 Additionally, fertility is an important clinical consideration in AYA patients making the prospect of stopping treatment and attempting TFR attractive.…”

“…59,65,66 Importantly, adolescents and young adults (AYA) with CML may have a higher tumor burden at diagnosis, and some studies have reported poorer response to treatment and outcomes in this population. 89 Nonadherence is estimated to impact up to 60% of AYA patients with cancer and is associated with poorer outcomes. 89 AYA are also expected to have extended periods of treatment, thereby experiencing similar challenges and survivorship issues to pediatric patients.…”