How I Treat on Infant Acute Lymphoblastic Leukemia
Cet article passe en revue des cas représentatifs de leucémie lymphoblastique aiguë (LLA) chez l'enfant, identifie les différences principales entre une LLA avec réarrangement du gène KMT2A et une LLA avec mutation constitutionnelle de ce même gène, examine les nouveaux traitements et analyse comment améliorer la stratification des patients et intégrer les nouveaux anticancéreux
Infant acute lymphoblastic leukemia (ALL) is an aggressive malignancy that has historically been associated with a very poor prognosis. Despite large co-operative international trials and incremental increases in intensity of therapy, there has been no significant improvement in outcome over the last 3 decades. Using representative cases, we highlight the key differences between KMT2A-rearranged and KMT2A-germline infant ALL, and how advances in molecular diagnostics are unpicking KMT2A-germline genetics and guiding treatment reduction. We focus on KM2TA-rearranged infant B-cell ALL where the last few years have seen the emergence of novel therapies which both are more effective and less toxic than conventional chemotherapy. Of these, there is promising early data on the efficacy and tolerability of the bi-specific T-cell engager monoclonal antibody, blinatumomab, as well as the use of autologous and allogeneic chimeric antigen receptor T-cell therapy. We discuss how we can improve risk stratification and incorporate these new agents to replace the most toxic elements of currently deployed intensive chemotherapy schedules with their associated unacceptable toxicity.
Blood , résumé, 2023