Gui Ruirui, Li Zhen, Wang Juan, Zu Yingling, Zhang Binglei, Zhao Juanjuan, Song Yongping, Zhou Jian
Department of Hematology, Affiliated Cancer Hospital of Zhengzhou University and Henan Cancer Hospital, Zhengzhou, Henan, China.
Department of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, Henan, China.
Heliyon. 2024 Nov 13;10(22):e40356. doi: 10.1016/j.heliyon.2024.e40356. eCollection 2024 Nov 30.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective measure for the treatment of hematological disease. With the progress and wide use of allo-HSCT, post-transplant central nervous system complications (CNSC) have gotten more and more attention because of its poor prognosis and high mortality. Since there is no standard treatment for patients with immune-mediated CNSC currently, new treatments are needed to be developed urgently. Here, we attempted a novel therapy regimen of intrathecal tocilizumab injection in two pediatric patients with immune-mediated CNSC manifested as cytokine release syndrome (CRS) after haploidentical hematopoietic stem cell transplantation (halpo-HSCT). In the two patients, persistent seizure symptoms could not be resolved 7 h after intravenous tocilizumab, while the symptoms were controlled rapidly only 2 hours or 1 hour after the first intrathecal injection of tocilizumab. Moreover, the level of interleukin 6 in the cerebrospinal fluid returned to normal after the fifth intrathecal injection. Even more appealing, no acute or chronic adverse reactions were observed during injection and subsequent follow-up. In conclusion, intrathecal tocilizumab seems to be more rapid and effective than intravenous administration for immune-mediated CNSC manifested as CRS in haplo-HSCT recipients. We recommend this treatment modality for further investigation.
异基因造血干细胞移植（allo-HSCT）是治疗血液系统疾病的有效措施。随着allo-HSCT的进展和广泛应用，移植后中枢神经系统并发症（CNSC）因其预后差和死亡率高而受到越来越多的关注。由于目前对于免疫介导的CNSC患者尚无标准治疗方法，迫切需要开发新的治疗方法。在此，我们尝试对两名小儿患者采用鞘内注射托珠单抗的新型治疗方案，这两名患者在单倍体造血干细胞移植（halpo-HSCT）后出现表现为细胞因子释放综合征（CRS）的免疫介导的CNSC。在这两名患者中，静脉注射托珠单抗7小时后持续性癫痫症状仍未缓解，而在首次鞘内注射托珠单抗后仅2小时或1小时症状就迅速得到控制。此外，在第五次鞘内注射后脑脊液中的白细胞介素6水平恢复正常。更吸引人的是，在注射及随后的随访期间未观察到急性或慢性不良反应。总之，对于单倍体造血干细胞移植受者中表现为CRS的免疫介导的CNSC，鞘内注射托珠单抗似乎比静脉给药更快速有效。我们建议对这种治疗方式进行进一步研究。
