安徽省杜氏肌营养不良症患儿生命质量现状及影响因素分析

巫洁; 陈信; 毛惠芳; 程进; 魏锁; 王丽华

doi:10.16766/j.cnki.issn.1674-4152.003963

安徽省杜氏肌营养不良症患儿生命质量现状及影响因素分析

doi: 10.16766/j.cnki.issn.1674-4152.003963

巫洁¹,
陈信^1, ,,
毛惠芳²,
程进²,
魏锁³,
王丽华⁴

1.
蚌埠医科大学第一附属医院儿科，安徽蚌埠 233004
2.
安徽卫生健康职业学院，安徽池州 247099
3.
池州市疾病预防控制保健中心，安徽池州 247000
4.
复旦大学附属中山医院护理部，上海 200030

基金项目:

安徽省高等学校省级质量工程项目 2022zmgj018

详细信息

通讯作者:
陈信，E-mail：44783939@qq.com

中图分类号: R725
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出版历程
- 收稿日期: 2024-07-11
- 网络出版日期: 2025-06-30

Investigation of quality of life in Duchenne muscular dystrophy children in Anhui Province and analysis of influencing factors

WU Jie¹,
CHEN Xin^{1
, ,},
MAO Huifang²,
CHENG Jin²,
WEI Suo³,
WANG Lihua⁴

1.
Department of Pediatrics, the First Affiliated Hospital of Bengbu Medical University, Bengbu, Anhui 233004, China

摘要

摘要: 目的了解安徽省杜氏进行性肌营养不良(DMD)患儿生存现状及相关影响因素，为提高该人群的生命质量及制定相关健康管理制度提供参考。方法采取一次性横断面调查方式于2023年2—8月开展问卷调查收集符合标准的安徽省DMD患儿基本信息，采用儿童生存质量量表(PedsQL)调查DMD患儿生命质量，采用多元线性回归分析探究DMD患儿生命质量得分的影响因素。结果累计回收有效问卷179份，有效回收率为90.40%。患儿平均确诊年龄为3.00(2.00，5.00)岁, 误诊率为41.90%(75/179), 异地就诊率达69.27%(124/179)。儿童PedsQL生存质量得分为(42.22±20.38) 分。健康问卷抑郁量表(PHQ-9)显示77.65%(139/179)的主要照料者存在不同程度的抑郁倾向。多元线性回归分析显示，年龄、主要居住地、是否接受激素治疗、照料者抑郁感受均为DMD患儿生存质量得分的影响因素(P＜0.05)。结论安徽省DMD患儿生命质量仍然存在较大提升空间；生命质量与患者居住地、是否接受激素治疗、照料者的抑郁感受等相关，应积极采取措施提高DMD人群生命质量，加强疾病防治科普宣教及人文关怀等。
- 杜氏进行性肌营养不良 /
- 现状调查 /
- 生命质量
Abstract: Objective To investigate the oral health-related quality of life and associated factors in children with Duchenne muscular dystrophy (DMD) in Anhui province, providing reference for improving the quality of life in this population and developing relevant health management systems. Methods A cross-sectional survey was adopted to select the children with Duchenne muscular dystrophy who met the criteria in Anhui Province from February to August 2023. The pediatric quality of life inventory measurement models (PedsQL) was used to investigate the quality of life of DMD patients, and multiple liner regression analysis was used to explore the factors influencing quality of life scores in DMD patients. Results A total of 179 valid questionnaires were collected, with an effective response rate of 90.40%. The average age at diagnosis was 3.00 (2.00, 5.00) years old, with a misdiagnosis rate of 41.90% (75/179) and a long-distance treatment rate of 69.27% (124/179). In addition, the PedsQL quality of life score for children was (42.22±20.38) points. The patient health questionnaire-9 (PHQ-9) showed that 77.65% (139/179) of participants exhibited depressive tendencies. Multiple linear regression analysis identified age, primary place of residence, receipt of hormone therapy, and caregiver depression as significant factors influencing quality of life scores in children with DMD (P < 0.05). Conclusion There is still significant potential for improving the quality of life for children with DMD in Anhui Province. The quality of life is influenced by factors such as patient' s place of residence, hormone therapy, and the caregiver' s feelings of depression. Proactive measures should be taken to improve the quality of life for DMD patients, strengthen education on disease prevention and management, and provide humanistic care.
- Duchenne muscular dystrophy /
- Status survey /
- Quality of life

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表 1 DMD患儿基本人口学特征

Table 1. Basic demographic characteristics of DMD children

项目	例数(%)
性别
男	177(98.89)
女	2(1.11)
年龄(岁)
≤3	24(13.40)
4~6	45(25.14)
7~9	38(21.23)
10~12	48(26.82)
13~15	18(10.06)
16~18	6(3.35)
受教育水平
尚未入托	25(13.97)
幼儿园	41(22.91)
小学	79(44.13)
初中	8(4.47)
高中	2(1.11)
辍学居家	24(13.41)
主要照料者
父母	143(79.89)
祖辈	36(20.11)
主要居住地
城镇	111(62.01)
农村	68(37.99)
是否有过误诊经历
是	75(41.90)
否	104(58.10)
是否有专人陪护
是	135(75.42)
否	44(24.58)
是否出现过危及生命的并发症
是	12(6.70)
否	167(93.30)

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表 2 不同项目DMD患儿生存质量得分比较

Table 2. Comparison of quality of life scores among DMD children across different programs

项目	例数(%)	生存质量得分(分)	统计量	P值
患儿年龄(岁)			19.890^a	＜0.001
≤3	24(13.40)	52.17(32.88, 71.20)
4~6	45(25.14)	43.48(29.35, 60.87)
7~9	38(21.23)	47.83(33.15, 56.79)
10~12	48(26.82)	40.22(27.45, 51.36)
13~15	18(10.06)	30.43(22.83, 43.21)
16~18	6(3.35)	8.15 (3.26, 25.54)
主要照料者			2.113^b	0.036
父母	143(79.89)	43.82±20.41
祖辈	36(20.11)	35.87±19.21
主要居住地			3.794^b	＜0.001
城镇	111(62.01)	46.58±20.04
农村	68(37.99)	35.10±19.00
父亲文化程度			8.781^c	＜0.001
初中及以下	88(49.16)	36.72±20.24
高中及中专	38(21.23)	42.79±17.79
大专及以上	53(29.61)	50.94±19.52
母亲文化程度			13.731^a	＜0.001
初中及以下	92(51.40)	36.96(26.36，49.46)
高中及中专	33(18.44)	41.30(30.98，56.52)
大专及以上	54(30.17)	50.00(37.77，64.13)
确诊年龄			3.376^c	0.020
≤3岁	91(50.84)	46.73±20.77
4~6岁	65(36.31)	37.74±18.96
7~9岁	20(11.17)	35.71±20.24
10~12岁	3(1.68)	46.01±10.56
是否接受激素治疗			18.990^c	＜0.001
是	125(69.83)	39.58±16.96
否	20(11.17)	30.38±22.44
暂无激素应用指征	34(18.99)	58.89±21.71
康复训练			19.367^a	＜0.001
无康复训练指征	27(15.08)	44.57(29.35，59.78)
定期康复训练	90(50.28)	45.65(33.70，59.78)
偶尔康复训练	43(24.02)	36.96(25.00，47.83)
有康复训练的必要，但从未接受康复训练	19(10.61)	19.57(3.26，41.30)
照顾者抑郁感受			33.964^a	＜0.001
无	40(22.35)	59.78(44.84，71.20)
轻度	41(22.91)	43.48(30.43，55.43)
中度	28(15.64)	41.30(27.17，51.36)
中重度	32(17.88)	37.50(29.35，47.01)
重度	38(21.23)	29.35(17.12，42.66)
是否有专人陪护			3.915^b	＜0.001
是	135(75.42)	39.38±21.01
否	44(24.58)	50.94±15.48
注：^a为H值，^b为t值，^c为F值。表中仅列出P＜0.05的项目。正态分布的计量资料以x ±s表示，非正态分布的计量资料以M(P₂₅, P₇₅)表示。

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表 3 变量赋值情况

Table 3. Variable assignment

变量	赋值方法
患儿年龄(岁)	连续性变量，以实际值赋值
确诊年龄	连续性变量，以实际值赋值
主要照料者	祖辈=0，父母=1
主要居住地	农村=0，城镇=1
是否有专人陪护	否=0，是=1
父亲文化程度	初中及以下=(0, 0)，高中及中专=(1, 0)，大专及以上=(0, 1)
母亲文化程度	初中及以下=(0, 0)，高中及中专=(1, 0)，大专及以上=(0, 1)
是否接受激素治疗	暂无激素应用指征=(0, 0)，是=(1, 0)，无=(0, 1)
康复训练	无康复训练指征=(0, 0, 0)，定期康复训练=(1, 0, 0)，偶尔康复训练=(0, 1, 0)，有康复训练必要，但从未接受康复训练=(0, 0, 1)
患儿依从性	暂不需治疗=(0, 0, 0)，积极配合治疗=(1, 0, 0)，有部分抵触情绪=(0, 1, 0)，十分抵触=(0, 0, 1)
照顾者抑郁感受	无=(0, 0, 0, 0)，轻度=(1, 0, 0, 0)，中度=(0, 1, 0, 0)，中重度=(0, 0, 1, 0)，重度=(0, 0, 0, 1)

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表 4 DMD患儿生存质量影响因素的多元线性回归分析

Table 4. Multivariate regression analysis of factors influencing quality of life in children with DMD

变量	β	SE	B	t值	P值
年龄	-2.038	0.497	-0.389	-4.098	＜0.001
是否接受激素治疗
是	-16.978	4.298	-0.383	-3.950	＜0.001
否	-23.552	5.041	-0.365	-4.672	＜0.001
照料者抑郁感受
轻度	-11.340	3.701	-0.235	-3.064	0.003
中度	-11.975	4.155	-0.214	-2.882	0.005
中重	-10.685	4.041	-0.201	-2.644	0.009
重度	-16.781	3.980	-0.338	-4.216	＜0.001
主要居住地
城镇	6.464	2.667	0.154	2.424	0.017
注：R²=0.593，调整后R²=0.507，F=6.900，P＜0.001。表中仅列出P＜0.05的项目。

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