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浙江大学学报(医学版)  2022, Vol. 51 Issue (4): 470-473    DOI: 10.3724/zdxbyxb-2022-0245
病例报告     
托珠单抗联合芦可替尼治疗造血干细胞移植后激素耐药的移植物抗宿主病患儿六例
陈洁,陆婧媛,洪秀理,鹿全意()
厦门大学附属中山医院血液科,福建 厦门 361000
Tocilizumab combined with ruxolitinib in the treatment of children with steroid resistant graft versus host disease after hematopoietic stem cell transplantation: report of 6 cases
CHEN Jie,LU Jingyuan,HONG Xiuli,LU Quanyi()
Department of Hematology, Zhongshan Hospital, Xiamen University, Xiamen 361000, Fujian Province, China
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摘要:

6例患儿在造血干细胞移植后出现激素耐药的移植物抗宿主病(GVHD),其中急性GVHD 4例,慢性GVHD 2例。4例急性GVHD患儿主要表现为大面积皮疹、发热2例,主要表现为腹痛、腹泻2例;2例慢性GVHD患儿主要表现为大面积皮肤苔藓样变1例,主要表现为反复口腔溃疡、张口受限1例。6例患儿均采用托珠单抗联合芦可替尼治疗,给药方案为托珠单抗8?mg/kg静脉注射,每 3周一次;芦可替尼 5~10?mg/d口服,连续 28?d, 至少完成两个疗程治疗。6例患儿的治疗反应率为100%,其中5例患儿接受两个周期治疗后完全缓解,中位缓解时间 为26.7?d。中位随访11(7~25)个月,未观察到明显治疗相关的不良反应。

关键词: 移植物抗宿主病激素耐药托珠单抗芦可替尼疗效病例报告    
Abstract:

Six children with steroid resistant graft versus host disease (GVHD) after hematopoietic stem cell transplantation admitted in the hospital, including 4 cases of acute GVHD and 2 cases of chronic GVHD. Among the 4 acute GVHD cases, the main manifestations were large area rash and fever in 2 cases, and abdominal pain and diarrhea in 2 cases. In 2 chronic GVHD cases, one presented lichenoid dermatosis, and the other showed repeated oral ulcers with difficult mouth opening. Patients received tocilizumab (8 mg/kg per dose every 3 weeks) and ruxolitinib (5-10 mg/d, 28 d), at least 2 courses were completed. All patients had complete responses (100%), and 5 patients responded after completion of two treatment courses, with the median time of remission was 26.7 d. The median follow-up period was 11 (7-25) months, and no severe treatment-related adverse reactions were observed.

Key words: Graft versus host disease    Steroid refractory    Tocilizumab    Ruxolitinib    Efficacy    Case report
收稿日期: 2022-05-13 出版日期: 2022-11-16
CLC:  R733  
基金资助: 福建省科技计划引导项目(2019D009); 厦门市科技计划指导项目(3502Z20209024)
通讯作者: 鹿全意     E-mail: nyi
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陈洁
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引用本文:

陈洁,陆婧媛,洪秀理,鹿全意. 托珠单抗联合芦可替尼治疗造血干细胞移植后激素耐药的移植物抗宿主病患儿六例[J]. 浙江大学学报(医学版), 2022, 51(4): 470-473.

CHEN Jie,LU Jingyuan,HONG Xiuli,LU Quanyi. Tocilizumab combined with ruxolitinib in the treatment of children with steroid resistant graft versus host disease after hematopoietic stem cell transplantation: report of 6 cases. J Zhejiang Univ (Med Sci), 2022, 51(4): 470-473.

链接本文:

https://www.zjujournals.com/med/CN/10.3724/zdxbyxb-2022-0245        https://www.zjujournals.com/med/CN/Y2022/V51/I4/470

例序

GVHD类型

发生时间

临床表现

托珠单抗前用药情况

治疗结果

1

急性

移植后 19?d

肠道(Ⅳ)/肝脏(Ⅰ)/皮肤(Ⅰ)受累

甲强龙、芦可替尼、布地奈德

用药 23?d后排便次数、粪便量明显减少,性状转为糊状便

2

慢性

移植后 122?d

皮肤苔藓样改变

甲强龙、甲氨蝶呤

用药后 40?d, 皮肤苔藓样改变较前好转

3

急性

移植后 25?d

皮肤(Ⅱ)/肝脏(Ⅰ)受累

甲强龙

用药 30?d后瘙痒、充血明显好转

4

急性

移植后 20?d

肠道(Ⅱ)/肝脏(Ⅰ)受累

甲强龙、布地奈德

起效快,用药后 7?d后大便即恢复正常

5

慢性

移植后 144?d

口腔溃疡、张口受限

甲强龙

用药 45?d(三剂)后, 张口受限明显好转

6

急性

移植后 35?d

皮肤(Ⅲ)受累

甲强龙

用药 22?d后瘙痒、充血明显好转

表1  患儿一般资料及治疗效果
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14 王晓宁, 张 莹, 刘欣慰, 等. 异基因造血干细胞移植后激素耐药胃肠道急性移植物抗宿主病的临床分析[J/CD]. 中华细胞与干细胞杂志(电子版), 2020, 10(6): 336-340
WANG Xiaoning, ZHANG Ying, LIU Xinwei, et al. Clinical analysis of steroid-resistant acute gastrointestinal graft versus host disease after allogeneic hematopoietic stem cell transplantation[J/CD]. Chinese Journal of Cell and Stem Cell (Electronic Edition), 2020, 10(6): 336-340. (in Chinese)
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