婴儿急性淋巴细胞白血病:来自印度的观点

Debasish Sahoo, Sonali Mohapatra
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引用次数: 0

摘要

儿童急性淋巴细胞白血病(ALL)预后的改善是一个成功的故事,在各种研究中生存率接近90%。然而,ALL在婴儿中,虽然罕见,但却是一个例外,它具有独特的侵袭性疾病生物学,分子遗传学和对治疗的不良反应,并且复发过多。它的特点是白细胞增多,疾病体积大,中枢神经系统(CNS)和皮肤受累的发生率较高。免疫表型显示CD10阴性,各种髓系标记物的高共表达表明原始造血细胞的起源。位于11q23的KMT2A基因重排,即KMTA-r(以前称为MLL),是疾病病理生理学的核心,在驱动白血病发生的表观遗传修饰中起关键作用,是该亚组中最强的阴性预后标志物。白细胞总数超过30万/mm3的白细胞增多症、年龄较小(6个月大)、强的松龙反应差和MRD反应差是婴儿ALL的其他重要阴性预后指标。尽管高度强化治疗和造血干细胞移植(HSCT),复发率仍然非常高。显著的治疗相关死亡率和发病率仍然是取得成功结果的其他障碍。最近,在婴儿ALL中使用blinatumomab显示出有希望的结果,并且在各种试验中被整合到化疗骨干中,例如interant 06。早期整合免疫疗法,包括blinatumomab和嵌合抗原受体(CAR) T细胞,以及其他大型合作试验的新药,将是改善这一人群预后的关键。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Infant acute lymphoblastic leukemia: a view from India
Improvement in outcomes of pediatric acute lymphoblastic leukemia (ALL) has been a success story with survival approaching 90% in various studies. However, ALL in infants, though rare, is an exception, with its distinct aggressive disease biology, molecular genetics and poor response to therapy with an excess of relapses. It characteristically presents with hyperleukocytosis, bulky disease and a higher incidence of central nervous system (CNS) and cutaneous involvement. Immunophenotyping is notable for CD10 negativity, and higher co-expression of various myeloid markers indicating a primitive hematopoietic cell of origin. Rearrangement in KMT2A gene i.e. KMTA-r (previously MLL), located on 11q23, is central to disease pathophysiology, playing a key role in epigenetic modification that drives leukemogenesis, and is the strongest negative prognostic marker in this subgroup. Hyperleukocytosis with total leukocyte count exceeding 3,00,000/mm3, younger age group (<6 months age), poor prednisolone response and poor MRD response are other important negative prognostic markers in infant ALL. Despite highly intensive therapy and hematopoietic stem cell transplantation (HSCT), the incidence of relapse remains exceedingly high. Significant treatment related mortality and morbidity remain other barriers to successful outcomes. Recently, the use of blinatumomab has shown promising results in infant ALL and is being integrated into chemotherapy backbones in various trials e.g. Interfant 06. Early integration of immunotherapy including blinatumomab and chimeric antigen receptor (CAR) T cells, and other novel drugs in larger collaborative trials will be key in improving the prognosis in this population.
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