ANN ONCOL

The introduction of immunotherapy with monoclonal antibodies (mAbs) is a major step forward for adults and children with B-lineage acute lymphoblastic leukaemia (ALL) and will change the treatment paradigm substantially. With intensive multi-agent chemotherapy (ChT) in children with ALL, long-term cure has been achieved in ≥90% of patients; however, this approach is often associated with long-term sequelae. Until recently, aggressive ChT has also been used in adult patients, with an overall cure rate of 50%. Survival rates are higher (∼70%) in adolescents and young adults (AYAs) but lower (<20%) in elderly patients. The major hazard of treatment is myelotoxicity leading to infection, which causes death in the induction and consolidation phases in 1%-3% of children and ≤10% of adults, increasing to ≤20% of elderly patients aged >70 years. With haematopoietic stem cell transplantation (HSCT), cure can be achieved in approximately half of adults; however, this approach is also associated with substantial toxicity and treatment-related mortality (TRM) rates of 10%-20%. Immunotherapy may provide new possibilities for B-lineage ALL, with very promising response and cure rates. Immunotherapy is associated with toxicities, but these are manageable and the TRM rate is low (∼1%). Immunotherapeutic approaches have been explored in different disease settings, initially in relapsed or refractory (r/r) ALL and in patients with minimal residual disease (MRD), and more recently as first-line therapy, either as monotherapy or in combination with ChT. Table 1 provides an overview of currently available targeted therapies.