从青春期到成年期脊柱裂患者报告的结果测量-系统回顾。

IF 1.9 3区医学 Q2 PEDIATRICS

Journal of Pediatric Urology Pub Date : 2025-10-01 DOI:10.1016/j.jpurol.2025.05.023

Pyry Jaakkola , Seppo Taskinen , Niklas Pakkasjärvi

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引用次数: 0

摘要

目的：脊柱裂（SB）是一种复杂的疾病，使个体在行动能力、自理能力和整体健康方面面临终身挑战。护理模式是多模式的，进步提高了生存率，将临床重点转向优化长期功能和生活质量。本系统综述的目的是概述用于评估过渡性SB患者健康相关生活质量（HRQOL）和其他状况的患者报告结局指标（PROMs），并分析其提供结果的影响因素。方法：我们根据PRISMA指南进行了系统综述，包括16项研究，2009名参与者。结果：参与者年龄从12岁到74岁不等，性别分布59%为女性。72%使用CIC， 48%有尿失禁，32%有肠失禁。儿科全球健康7 （PGH-7）和神经源性膀胱症状评分（NBSS）是最常用的工具，分别捕获一般健康和膀胱特异性结局。生活质量评估在脊柱裂（QUALAS）措施出现作为一个简洁的，sb特定的工具，解决身体和社会心理领域。CIC与膀胱特异性结果的改善有关，尽管其对总体HRQOL的影响不一致。心理健康、文化水平和过渡准备显著影响HRQOL。结论：在SB个体的治疗和研究中，PROMs的使用被证明是惊人的多样化和碎片化。针对特定疾病的prom，如QUALAS，是对这一问题的明显和必要的反应，强调不断开发这些工具，使其更适合目标群体。当与NBSS等有针对性的措施相辅相成时，它们为了解和改善SB患者的HRQOL提供了实用而全面的方法。优化膀胱和肠道管理、心理健康和社会支持仍然是改善结果的关键。证据水平：III。

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Patient-reported outcome measures in Spina Bifida from adolescence to adulthood – A systematic review

Aims

Spina bifida (SB) is a complex condition, predisposing individuals to lifelong challenges in mobility, continence, and overall health. Care modalities are multimodal, and advancements have improved survival, shifting clinical focus towards optimizing long-term function and quality of life.

The aim of this systematic review is to outline patient-reported outcome measures (PROMs) used to assess health-related quality of life (HRQOL) and other conditions in transitional SB patients, and to analyze factors influencing the results they provide.

Methods

We conducted a systematic review according to PRISMA guidelines, including sixteen studies with 2009 participants.

Results

Participants' ages ranged from 12 to 74 years, with a gender distribution of 59 % female. 72 % used CIC, 48 % had urinary incontinence and 32 % had bowel incontinence. The Pediatric Global Health 7 (PGH-7) and Neurogenic Bladder Symptom Score (NBSS) were the most frequently used tools, capturing general health and bladder-specific outcomes, respectively. The Quality of Life Assessment in Spina bifida (QUALAS) measures emerged as a concise, SB-specific tool addressing physical and psychosocial domains. CIC was linked to improved bladder-specific outcomes, though its effect on overall HRQOL was inconsistent. Mental health, literacy, and transition readiness significantly influenced HRQOL.

Conclusions

The use of PROMs in the treatment and research of individuals with SB proved to be surprisingly variable and fragmented. Disease-specific PROMs, such as QUALAS, are an obvious and necessary response to the problem emphasizing the continuous development of these tools to be more tailored to the target group. When complemented by targeted measures like NBSS, they offer a practical and comprehensive approach in understanding and improving HRQOL in SB patients. Optimizing bladder and bowel management, mental health, and social support remains critical for improving outcomes.

Level of evidence

III.

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来源期刊

Journal of Pediatric Urology PEDIATRICS-UROLOGY & NEPHROLOGY

CiteScore

3.70

自引率

15.00%

发文量

330

审稿时长

4-8 weeks

期刊介绍： The Journal of Pediatric Urology publishes submitted research and clinical articles relating to Pediatric Urology which have been accepted after adequate peer review. It publishes regular articles that have been submitted after invitation, that cover the curriculum of Pediatric Urology, and enable trainee surgeons to attain theoretical competence of the sub-specialty. It publishes regular reviews of pediatric urological articles appearing in other journals. It publishes invited review articles by recognised experts on modern or controversial aspects of the sub-specialty. It enables any affiliated society to advertise society events or information in the journal without charge and will publish abstracts of papers to be read at society meetings.