切-希二氏综合征患者异基因骨髓移植后的嵌合现象分离

Split chimerism after allogeneic bone marrow transplantation in Chediak-Higashi syndrome.

作者信息

Yamazaki S, Takahashi H, Fujii H, Miyamae T, Mori M, Fujioka K, Funabiki T, Yokota S, Arai N, Ikuta K

机构信息

Department of Pediatrics, Yokohama City University School of Medicine, Yokohama, Japan.

出版信息

Bone Marrow Transplant. 2003 Jan;31(2):137-40. doi: 10.1038/sj.bmt.1703789.

DOI:10.1038/sj.bmt.1703789

PMID:12621497

Abstract

Chediak-Higashi Syndrome (CHS) is a hereditary multiorgan disease associated with a lymphoproliferative disorder termed 'accelerated phase' (AP). As AP is often life-threatening, hematopoietic stem cell transplantation has been proposed as the only curative treatment for CHS. Here, we report a 1-year-old Japanese boy with CHS who received an HLA-matched unrelated BMT at the AP stage, which resulted in split chimerism. We evaluated the chimerism status of isolated leukocytes and found that only a limited population of T and NK cells was of donor origin and the majority of these and other hematopoietic cells was of host origin. Clinical outcome was successful, and the patient is currently alive and well, free of AP and serious infections more than 18 months after BMT.

摘要

切迪阿克-希加什综合征（CHS）是一种遗传性多器官疾病，与一种称为“加速期”（AP）的淋巴增殖性疾病相关。由于AP往往危及生命，造血干细胞移植已被提议作为CHS的唯一治愈性治疗方法。在此，我们报告一名1岁的日本CHS男孩，他在AP阶段接受了 HLA 匹配的无关供体骨髓移植（BMT），结果出现了混合嵌合体。我们评估了分离白细胞的嵌合状态，发现只有有限数量的T细胞和自然杀伤（NK）细胞是供体来源，而这些细胞和其他造血细胞的大多数是宿主来源。临床结果成功，患者目前健在，在BMT后18个月以上未出现AP且未发生严重感染。