Division of Oncology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, MLC 7018, Cincinnati, OH, 45229, USA.
Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Curr Oncol Rep. 2021 Feb 12;23(3):27. doi: 10.1007/s11912-021-01021-1.
Infant leukemia is a rare, distinct subgroup of pediatric acute leukemias diagnosed in children under 1 year of age and characterized by unique, aggressive biology. Here, we review its clinical presentation, underlying molecular biology, current treatment strategies, and novel therapeutic approaches.
Infant leukemias are associated with high-risk molecular features and high rates of chemotherapy resistance. International collaborative clinical trials have led to better understanding of the underlying molecular biology, refined risk-based stratification, and investigated the use of hematopoietic stem cell transplantation. However, intensification of chemotherapy has failed to improve outcomes, and current regimens are associated with significant treatment-related and long-term toxicities. Infants with leukemia remain a challenging group to treat. We must continue collaborative efforts to move beyond traditional cytotoxic chemotherapy, incorporate molecularly targeted strategies and immunotherapy, and increase access to clinical trials to improve outcomes for this high-risk group of patients.
婴儿白血病是一种罕见的、独特的儿童急性白血病亚组，诊断于 1 岁以下的儿童，其特征为独特的侵袭性生物学特性。在此，我们综述其临床表现、潜在的分子生物学、当前的治疗策略和新的治疗方法。
婴儿白血病与高风险的分子特征和化疗耐药率高有关。国际合作临床试验使我们更好地了解了潜在的分子生物学，对基于风险的分层进行了精细化，并研究了造血干细胞移植的应用。然而，化疗的强化未能改善结局，当前的治疗方案与严重的治疗相关和长期毒性有关。患有白血病的婴儿仍然是一个具有挑战性的治疗群体。我们必须继续合作努力，超越传统细胞毒性化疗，纳入靶向治疗策略和免疫疗法，并增加临床试验的机会，以改善这一高危患者群体的结局。
