Jaiswal Sarita Rani, Chakrabarti Suparno
Department of Blood and Marrow Transplantation, Dharamshila Hospital and Research Centre, Vasundhara Enclave, New Delhi 110096, India; Manashi Chakrabarti Foundation, Kolkata, India.
Adv Hematol. 2016;2016:3467672. doi: 10.1155/2016/3467672. Epub 2016 Mar 24.
Allogeneic hematopoietic stem cell transplantation (HSCT) remains a curative option for children with high risk and advanced acute leukemia. Yet availability of matched family donor limits its use and although matched unrelated donor or mismatched umbilical cord blood (UCB) are viable options, they fail to meet the global need. Haploidentical family donor is almost universally available and is emerging as the alternate donor of choice in adult patients. However, the same is not true in the case of children. The studies of haploidentical HSCT in children are largely limited to T cell depleted grafts with not so encouraging results in advanced leukemia. At the same time, emerging data from UCBT are challenging the existing paradigm of less stringent HLA match requirements as perceived in the past. The use of posttransplantation cyclophosphamide (PTCY) has yielded encouraging results in adults, but data in children is sorely lacking. Our experience of using PTCY based haploidentical HSCT in children shows inadequacy of this approach in younger children compared to excellent outcome in older children. In this context, we discuss the current status of haploidentical HSCT in children with acute leukemia in a global perspective and dwell on its future prospects.
异基因造血干细胞移植（HSCT）仍然是高危和晚期急性白血病患儿的一种治愈性选择。然而，匹配的家族供体的可获得性限制了其应用，尽管匹配的无关供体或不匹配的脐带血（UCB）是可行的选择，但它们无法满足全球需求。单倍体家族供体几乎普遍可获得，并且正在成为成年患者的替代供体选择。然而，在儿童中情况并非如此。儿童单倍体HSCT的研究主要限于T细胞去除的移植物，在晚期白血病中的结果并不那么令人鼓舞。与此同时，来自脐血移植（UCBT）的新数据正在挑战过去所认为的不那么严格的HLA匹配要求的现有模式。移植后环磷酰胺（PTCY）的使用在成人中取得了令人鼓舞的结果，但儿童的数据却严重缺乏。我们使用基于PTCY的单倍体HSCT治疗儿童的经验表明，与大龄儿童的良好结果相比，这种方法在年幼儿童中并不适用。在此背景下，我们从全球角度讨论儿童急性白血病单倍体HSCT的现状，并探讨其未来前景。
