Dyba J, Tinmouth A, Bredeson C, Matthews J, Allan D S
Department of Medicine, Queen's University at Kingston, Kingston, Ontario, Canada.
Division of Hematology, Department of Medicine, University of Ottawa, and Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
Transfus Med. 2016 Jun;26(3):202-7. doi: 10.1111/tme.12300. Epub 2016 Apr 5.
Late graft failure after allogeneic haematopoietic cell transplantation (HCT) can result from the failed engraftment of long-term engrafting cells. The use of thrombopoietin (TPO) receptor agonists (TRA) has been extensively studied and remains an important component of experimental ex vivo stem cell expansion protocols, but its use in allogeneic transplantation is still evolving.
We describe the use of eltrombopag, a TRA, to stimulate the rescue of late graft failure in a patient following allogeneic HCT, and we performed a systematic review of published studies describing the use of TRAs following allogeneic transplantation.
A total of eight publications were identified from our systematic search and included observational case studies (five studies, total of seven patients) that primarily addressed ITP or isolated thrombocytopenia at various time points after allogeneic HCT and prospective clinical trials (three studies, total of 177 patients with 95 patients receiving TRAs). No studies reported specifically on the use of TRAs for the treatment of trilineage graft failure as a means of in vivo stem cell expansion. The use of TRAs following allogeneic HCT appears safe and promising.
The use of eltrombopag or other TRAs to treat poor graft function after allogeneic HCT is intriguing and warrants further study.
异基因造血细胞移植（HCT）后的晚期移植物失败可能是由于长期植入细胞植入失败所致。血小板生成素（TPO）受体激动剂（TRA）的使用已得到广泛研究，并且仍然是实验性体外干细胞扩增方案的重要组成部分，但其在异基因移植中的应用仍在不断发展。
我们描述了使用TRA艾曲泊帕来挽救一名异基因HCT患者的晚期移植物失败，并对已发表的描述异基因移植后使用TRA的研究进行了系统评价。
通过系统检索共识别出8篇文献，包括观察性病例研究（5项研究，共7例患者），主要涉及异基因HCT后不同时间点的免疫性血小板减少症（ITP）或孤立性血小板减少症，以及前瞻性临床试验（3项研究，共177例患者，其中95例接受TRA）。没有研究专门报道使用TRA治疗三系移植物失败作为体内干细胞扩增的一种手段。异基因HCT后使用TRA似乎是安全且有前景的。
使用艾曲泊帕或其他TRA治疗异基因HCT后的移植物功能不良很有吸引力，值得进一步研究。
