Kanold Justyna, Merlin Etienne, Halle Pascale, Paillard Catherine, Marabelle Aurelien, Rapatel Chantal, Evrard Bertrand, Berger Claire, Stephan Jean-Louis, Galambrun Claire, Piguet Christophe, D'Incan Michel, Bordigoni Pierre, Deméocq François
CHU Clermont-Ferrand, Centre Régional de Cancérologie et Thérapie Cellulaire Pédiatrique, Hôtel-Dieu, INSERM, CIC501, Faculté de Médecine, Université Clermont1, Clermont-Ferrand, France.
Transfusion. 2007 Dec;47(12):2276-89. doi: 10.1111/j.1537-2995.2007.01469.x. Epub 2007 Aug 30.
Extracorporeal photochemotherapy (ECP) gives positive results in the management of graft-versus-host disease (GVHD), but in children, specific difficulties can outweigh this benefit. These difficulties must be taken into consideration when establishing a standardized reproducible procedure for implementation under a quality management plan.
Twenty-seven children underwent ECP for severe acute GVHD (aGVHD) or chronic GVHD (cGVHD) after allogeneic marrow transplantation. Data were collected prospectively, with particular emphasis placed on technical, biologic, immunologic, clinical, and long-term follow-up issues.
The 27 children underwent a total of 750 sessions. Mononuclear cells were collected on a commercially available apheresis system (COBE Spectra, Gambro BCT). Overall survival was 73 percent, and ECP led to significant improvement in 21 of the 27 patients (11 with complete response and 10 with partial response, i.e., >50% of organ involvement). Tolerance was good overall, the main limiting factors being vascular access and the psychological impact of repeated apheresis procedures. Children weighing less than 25 kg were not more susceptible to side effects.
A specifically pediatric-dedicated and -experienced team faces only limited difficulties when treating children with GVHD by ECP. Overall, ECP is efficient and well tolerated. Our experience was therefore pooled together with available pediatric data to establish clinical practice guidelines. These guidelines consider ECP as a first-line therapy in Grade IV aGVHD (in association with conventional pharmacologic approaches) and limited cGVHD and as a second-line therapy in steroid-resistant Grades II to III aGVHD and extensive cGVHD.
体外光化学疗法（ECP）在移植物抗宿主病（GVHD）的治疗中取得了积极效果，但对于儿童而言，特定困难可能会超过这种益处。在制定质量管理计划下可标准化重复实施的程序时，必须考虑这些困难。
27名儿童在接受异基因骨髓移植后因严重急性GVHD（aGVHD）或慢性GVHD（cGVHD）接受了ECP治疗。前瞻性收集数据，特别强调技术、生物学、免疫学、临床和长期随访问题。
27名儿童共接受了750次治疗。单核细胞通过市售的血细胞分离系统（COBE Spectra，Gambro BCT）采集。总生存率为73%，ECP使27例患者中的21例有显著改善（11例完全缓解，10例部分缓解，即器官受累>50%）。总体耐受性良好，主要限制因素是血管通路和重复血细胞分离程序的心理影响。体重小于25kg的儿童对副作用并不更敏感。
一个专门针对儿科且经验丰富的团队在通过ECP治疗儿童GVHD时仅面临有限困难。总体而言，ECP有效且耐受性良好。因此，我们将自身经验与现有的儿科数据汇总，以制定临床实践指南。这些指南将ECP视为IV级aGVHD（与传统药物方法联合使用）和局限性cGVHD的一线治疗方法，以及类固醇抵抗性II至III级aGVHD和广泛性cGVHD的二线治疗方法。
