维多利珠单抗治疗儿童糖皮质激素耐药重型肠道急性移植物抗宿主病1例并文献复习

Vedolizumab for Steroid-Refractory Severe Intestinal Acute Graft-versus-Host Disease in a Child: A Case Report and Literature Review

摘要: 目的：探讨维多利珠单抗在儿童糖皮质激素耐药重型肠道急性移植物抗宿主病(aGVHD)治疗中的可能价值与局限。方法：回顾性分析1例7岁再生障碍性贫血患儿行亲缘半相合造血干细胞移植后发生以肠道受累为主并伴皮肤表现的aGVHD临床资料，并结合文献复习。结果：患儿移植后+16 d起出现腹泻、皮疹、腹痛及肉眼血便，腹泻高峰约3800 mL/d；在接受足量甲泼尼龙、ATG、芦可替尼、巴利昔单抗、布地奈德、环磷酰胺等治疗后，仍有大量腹泻、腹痛及便血，结合临床症状及内镜结果符合糖皮质激素耐药重型肠道aGVHD，于+27、+42、+56 d加用维多利珠单抗300 mg静脉输注；并联合感染控制、止血、营养及输血支持。至+68 d大便性状基本正常，腹痛和肉眼血便消失；随访示供者嵌合度100%，未再见活动性GVHD。结论：本例提示，维多利珠单抗可作为儿童糖皮质激素耐药重型肠道aGVHD的肠道靶向救治选择，但其独立疗效仍需更多儿科研究验证。

Abstract: Objective: To explore the potential value and limitations of vedolizumab in the treatment of steroid-refractory severe intestinal acute graft-versus-host disease (aGVHD) in children. Methods: A retrospective analysis was conducted on a 7-year-old child with aplastic anemia who developed aGVHD primarily involving the gastrointestinal tract, accompanied by cutaneous manifestations, following haploidentical hematopoietic stem cell transplantation from a related donor, combined with a literature review. Results: The patient developed diarrhea, rash, abdominal pain, and gross hematochezia starting at day +16 post-transplantation, with peak diarrhea volume reaching approximately 3800 mL/day. Despite treatment with high-dose methylprednisolone, ATG, ruxolitinib, basiliximab, budesonide, and cyclophosphamide, the patient continued to experience severe diarrhea, abdominal pain, and rectal bleeding. Based on clinical symptoms and endoscopic findings, the diagnosis of steroid-refractory severe intestinal aGVHD was confirmed. Vedolizumab 300 mg was administered intravenously at days +27, +42, and +56, along with infection control, hemostatic measures, nutritional support, and transfusion therapy. By day +68, stool consistency had normalized, and abdominal pain and gross hematochezia had resolved. Follow-up revealed 100% donor chimerism, with no recurrence of active GVHD. Conclusion: Vedolizumab may be considered as a gut-directed salvage option for pediatric steroid-refractory severe intestinal aGVHD, although its independent effect requires further pediatric evidence.