PDF(Pubmed)
Abstract：
Graft versus host disease (GVHD) is one of the most serious complications following stem cell transplant in children and is a major cause of morbidity and mortality. Corticosteroids remain the mainstay of treatment, and although a majority of children respond to systemic steroids, those refractory to or dependent upon corticosteroids suffer from complications secondary to long-term steroid administration. This problem has prompted consideration of steroid-sparing treatment strategies, although the time to clinical remission can be variable. Intraarterial corticosteroid delivery has been used in adults as a rescue therapy in steroid-resistant patients, but its use in children has been limited. We investigated the feasibility of intraarterial steroid administration into the bowel and/or liver in a cohort of six pediatric patients with acute GVHD. All patients successfully underwent treatment with no serious adverse effects. Five of five (100%) patients with gastrointestinal bleeding due to GVHD had rapid symptom improvement by 48 h, which was durable up to three weeks. Three of four (75%) patients with hepatic GVHD had improved cholestasis following intraarterial steroid administration. Our experience with this small cohort preliminarily demonstrated the feasibility and safety of intraarterial steroid administration in children with acute GVHD. This approach warrants consideration as a rescue therapy in steroid-refractory cases and as a \"bridge\" therapy for children with severe acute GVHD who are transitioning to steroid-sparing regimens.
摘要：
移植物抗宿主病（GVHD）是儿童干细胞移植后最严重的并发症之一，是发病率和死亡率的主要原因。皮质类固醇仍然是治疗的主要手段,尽管大多数儿童对全身性类固醇有反应，那些难以治疗或依赖皮质类固醇的患者长期服用类固醇后会出现并发症.这个问题促使人们考虑保留类固醇的治疗策略，尽管临床缓解的时间可能是可变的。动脉内皮质类固醇递送已在成人中用作类固醇耐药患者的抢救疗法。但是它在儿童中的使用受到限制。我们在6例急性GVHD儿科患者的队列中研究了向肠和/或肝脏动脉内施用类固醇的可行性。所有患者均成功接受治疗，无严重不良反应。5例（100%）因GVHD引起的消化道出血的患者中，有5例在48小时内症状迅速改善，可持续使用长达三周。动脉内类固醇给药后，四名（75％）肝性GVHD患者中有三名改善了胆汁淤积。我们在这一小组中的经验初步证明了急性GVHD儿童动脉内类固醇给药的可行性和安全性。这种方法值得考虑作为类固醇难治性病例的抢救治疗，并作为过渡到类固醇保留方案的严重急性GVHD儿童的“桥梁”治疗。
