Long-term follow-up of CD19-CAR T-cell therapy in children and young adults with B-ALL

Abstract

The tremendous successes of CD19-directed CAR T cells in children and young adults with B-cell acute lymphoblastic leukemia (B-ALL) has led to the more widespread use of this important treatment modality. With an ability to induce remission and potentially lead to long-term survival in patients with multiply relapsed/chemotherapy refractory disease, more children are now receiving this therapy with the hope of inducing a long-term durable remission (with or without consolidative hematopoietic cell transplantation). While overcoming the acute toxicities was critical to its broad implementation, the emerging utilization requires close evaluation of subacute and delayed toxicities alongside a consideration of late effects and issues related to survivorship following CAR T cells. In this underexplored area of toxicity monitoring, this article reviews the current state of the art in relationship to delayed toxicities while highlighting areas of future research in the study of late effects in children and young adults receiving CAR T cells.

Advanced-Therapy Medicinal Products: Challenges for Implementation in Pediatric Clinical Practice

“…В настоящее время в отношении острых токсических реакций стандартизированы как методы диагностики, так и терапевтические подходы, в том числе и у детей [45][46][47]. Вместе с тем имеются ограниченные сведения и о долгосрочной токсичности терапии CD19 CAR-Т-клетками [48], что требует проведения регистровых наблюдательных исследований. Наряду с профилактикой и своевременной диагностикой токсических эффектов CAR-T для снижения рисков их развития предпринимаются попытки модификации самих Т-лимфоцитов.…”