哈萨克斯坦共和国先天性耳畸形儿童医疗与组织照护的专家评估：RAND/UCLA适宜性方法

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中文摘要

背景： 先天性听觉异常是严重疾病，范围从轻度听力损失到完全耳聋，并伴有言语障碍、社会限制和残疾。我们的目标是使用RAND/UCLA适宜性方法，评估哈萨克斯坦共和国先天性耳畸形（小耳畸形和耳道闭锁）儿童医疗与组织照护干预措施的适宜性。
方法： 一个由九名成员组成的多学科专家小组（耳鼻咽喉科医生、听力学家、儿科医生和耳科外科医生）在兩轮评估中，使用结构化的9分适宜性量表对93项诊断、治疗和康复干预措施进行评分。共识定义为数值IQR（Q3-Q1）≤2分，且≥75%的评分位于同一类别。适宜性类别定义为：适宜（中位数7-9）、不确定（4-6）和不适宜（1-3）。
结果： 在93项评分的干预措施中，43项（46%）达成共识为适宜，45项（48%）被分类为不确定，5项（5%）为不适宜。最高适宜性被赋予多学科诊断会诊（耳鼻咽喉科专家、听力学家、耳科外科医生）和影像学检查（颞骨高分辨率CT、MRI）。对于双侧耳道闭锁，骨传导装置被评为适宜（均值7.55-7.66）。非干预方法获得最低评分（均值1.11-3.00），表明专家强烈共识反对期待管理。关键组织障碍作为明确评分的条目纳入问卷（条目85-93，附录A第4-5部分）。患者登记和随访延迟或缺失（条目86，均值8.00，中位数8，适宜）以及监管框架不足（条目87，均值7.22，中位数7，适宜†）是评分最高的组织条目之一，而资金不足（条目88，均值6.22，中位数6，不确定且未达成共识）未达成共识。
结论： 这是RAND/UCLA方法在哈萨克斯坦首次应用于评估先天性耳畸形儿童的照护，确定了专家共识和不确定性的明确领域。结果支持制定多学科照护路径和有针对性的康复政策。研究结果代表哈萨克斯坦九名专家小组成员的结构化专家意见，不应被解释为临床有效性、成本效益或国家代表性的证据。

英文摘要

UNLABELLED: Congenital auditory anomalies are serious conditions ranging from mild hearing loss to complete deafness, with associated speech impairment, social limitations, and disability. Our aim was to evaluate the appropriateness of medical and organizational care interventions for children with congenital ear malformations (microtia and aural atresia) in the Republic of Kazakhstan, using the RAND/UCLA Appropriateness Method.
METHODS: A nine-member multidisciplinary expert panel (otorhinolaryngologists, audiologists, pediatricians, and otologic surgeons) rated 93 diagnostic, therapeutic, and rehabilitation interventions using a structured 9-point appropriateness scale across two rounds of evaluation. Consensus was defined as a numerical IQR (Q3-Q1) ≤2 score points combined with ≥75% of ratings within the same category. Appropriateness categories were defined as: Appropriate (median 7-9), Uncertain (4-6), and Inappropriate (1-3).
RESULTS: Of 93 rated interventions, 43 (46%) reached consensus as Appropriate, 45 (48%) were classified as Uncertain, and 5 (5%) as Inappropriate. Highest appropriateness was assigned to multidisciplinary diagnostic consultations (ENT specialist, audiologist, otologic surgeon) and imaging (high-resolution CT of the temporal bones, MRI). For bilateral aural atresia, bone conduction devices were rated as Appropriate (mean 7.55-7.66). Non-intervention approaches received the lowest ratings (mean 1.11-3.00), indicating strong expert consensus against expectant management. Key organizational barriers were included as explicitly rated items in the questionnaire (items 85-93, Section 4-5 of Appendix A). Delayed or absent patient registration and follow-up (item 86, mean 8.00, median 8, Appropriate) and inadequacies in regulatory frameworks (item 87, mean 7.22, median 7, Appropriate†) were among the highest-rated organizational items, while insufficient funding (item 88, mean 6.22, median 6, Uncertain without consensus) did not reach consensus.
CONCLUSION: This first application of the RAND/UCLA method in Kazakhstan to evaluate care for children with congenital ear malformations identified clear areas of expert consensus and uncertainty. The results support the development of multidisciplinary care pathways and targeted rehabilitation policies. Findings represent structured expert opinion from nine specialist panelists in Kazakhstan and should not be interpreted as evidence of clinical effectiveness, cost-effectiveness, or national representativeness.