造血干细胞移植治疗对黏多糖贮积症Ⅰ型和Ⅱ型腺样体大小的影响研究  

Study of the effect of hematopoietic cell transplantation on the size of adenoids in MPS typeⅠand typeⅡ

作　　者：白鑫茹 杨洋[3] 冯顺乔[4] 刘嵘[4] 谷庆隆[1,2] Bai Xinru;Yang yang;Feng Shunqiao;Liu Rong;Gu Qinglong(Department of Otorhinolaryngology,Children's Hospital Affiliated to the Capital Institute of Pediatrics,Beijing 100020,China;Graduate School of Peking Union Medical College,Beijing 100730,China;Department of Imaging,Children's Hospital Affiliated to the Capital Institute of Pediatrics,Beijing 100020,China;Department of Hematology,Children's Hospital Affiliated to the Capital Institute of Pediatrics,Beijing 100020,China)

机构地区：[1]首都儿科研究所附属儿童医院耳鼻咽喉科,北京100020 [2]北京协和医学院研究生院,北京100730 [3]首都儿科研究所附属儿童医院影像科,北京100020 [4]首都儿科研究所附属儿童医院血液科,北京100020

出　　处：《国际耳鼻咽喉头颈外科杂志》2024年第4期187-191,共5页International Journal of Otolaryngology-Head and Neck Surgery

基　　金：首都卫生发展科研专项(首发2022-1-2101);北京市自然科学基金(7232010)。

摘　　要：目的分析黏多糖贮积症(mucopolysaccharidosis,MPS)Ⅰ型和Ⅱ型患儿造血干细胞移植(hematopoietic stem cell transplantation,HSCT)后腺样体大小的变化,为MPS患儿的治疗方案制定提供帮助。方法回顾性分析已接受HSCT治疗的14例MPSⅠ型和Ⅱ型患儿的病历及影像学资料,评估患儿腺样体大小的变化。结果14例患儿的临床表现各异,86%(12/14)在确诊前有耳鼻咽喉科表现,主要包括鼻塞、睡眠打鼾、反复呼吸道感染、耳痛、听力下降。入院时经头颅MRI发现均有不同程度的腺样体肥大(A/N>0.6),且HSCT前、后腺样体大小比较,缓解程度有统计学意义(P<0.05)。结论MPS患儿的耳鼻咽喉科症状表现较早,上气道阻塞在MPSⅠ型和Ⅱ型患者中较为普遍,主要是由腺样体肥大所致。成功的HSCT治疗可以显著改善MPSⅠ型和Ⅱ型的腺样体肥大程度,可避免行不必要的腺样体切除术。Objective To analyze the changes of adenoid size after hematopoietic stem cell transplantation(HSCT)in children with mucopolysaccharidosis(MPS)type I and type II,and to provide help for the formulation of treatment plan for children with MPS.MethodssThe medical records and imaging data of 14 children with type I and type II MPS treated with HSCT were retrospectively analyzed to evaluate the change of adenoid size.Results The clinical manifestations of the 14 patients were dferent.86%(12/14)of the patients had otolaryngology manifestations before diagnosis,including nasal congestion,sleep snoring,recurrent respiratory infection,earache and hearing loss.All the 14 patients were found to have adenoid hypertrophy of varying degrees by head MRI at admission(A/N>0.6),and the comparison of adenoid size before and after HSCT showed statistically significant remission(P<0.05).Conclusions The symptoms of otolaryngology in children with MPS appear early and are common.Upper airway obstruction is more common in MPS typeⅠand typeⅡpatients,which is mainly caused by adenoid hypertrophy.Successful HSCT treatment can significantly reduce the degree of adenoid hypertrophy in MPS typeⅠand typeⅡ,unnecessary adenoidectomy can be avoided.

关 键 词：造血干细胞移植 黏多糖贮积症 

分 类 号：R457.7[医药卫生—治疗学] R725.9[医药卫生—临床医学] R766.9