Chhina Harpreet, Klassen Anne F, Kopec Jacek A, Oliffe John, Iobst Christopher, Dahan-Oliel Noemi, Aggarwal Aditya, Nunn Tim, Cooper Anthony P
Department of Experimental Medicine, Faculty of Medicine, University of British Columbia, Vancouver, Canada.
Department of Orthopaedics, BC Children's Hospital, 1D 18, Orthopaedics Research Office, 4480 Oak Street, Vancouver, BC, V6H 3V4, Canada.
J Patient Rep Outcomes. 2021 Apr 1;5(1):30. doi: 10.1186/s41687-021-00299-w.
Lower limb deformities include conditions such as leg length discrepancy, lower limb deficiency and associated angular and rotational deformities of the hips, knees, ankles and feet. Children with lower limb deformities often have physical limitations due to gait irregularities and pain. The differences in the appearance and function of their lower limbs can discourage participation in social, recreational and leisure activities, which may result in behavioural, emotional, psychological and social adjustment problems. The health-related quality of life (HRQL) of these children is often impacted due to the factors discussed above, as well as by the complex surgical procedures. Surgical treatment options for limb deformities in children vary from limb lengthening and reconstruction to amputation. The lack of evidence demonstrating superiority of either treatment options and their effect on HRQL limits the ability of healthcare providers to counsel families on the best evidence-based treatment option for them. This manuscript describes the international qualitative study which guided the development of a new patient-reported outcome measure (PROM). Individual semi-structured face-to-face interviews with children with lower limb deformities and their parents were conducted at five sites: Canada (2 sites), Ethiopia, India and the USA.
Seventy-nine interviews were conducted at five international sites. Five main themes emerged from the qualitative interviews and formed the basis of the conceptual framework. These themes were: 1) appearance, 2) physical health, 3) psychological health 4) school and 5) social health.
Lower limb deformities have a substantial impact on the HRQL of children. The concepts of interest identified in our study were similar across children from all countries. The conceptual framework guided the development of outcome scales specific to these patients. The information about the impact of various treatment options on the HRQL of children with lower limb deformities, collected using this new PROM, could be used to inform parents and children about outcomes (physical, social, psychological) associated with specific treatment options. This information could supplement other objective outcome information (e.g., complication rates, how the leg will look, etc.) to help families to come to a more informed decision on a child's course of treatment.
下肢畸形包括诸如腿长差异、下肢缺损以及髋部、膝部、踝部和足部相关的角度和旋转畸形等情况。患有下肢畸形的儿童常常由于步态异常和疼痛而存在身体限制。其下肢外观和功能的差异可能会阻碍他们参与社交、娱乐和休闲活动，这可能导致行为、情绪、心理和社会适应问题。这些儿童的健康相关生活质量（HRQL）常常受到上述因素以及复杂外科手术的影响。儿童肢体畸形的外科治疗选择从肢体延长和重建到截肢不等。缺乏证据表明哪种治疗选择更具优势及其对HRQL的影响，限制了医疗服务提供者为家庭提供基于最佳证据的最佳治疗选择建议的能力。本手稿描述了一项国际定性研究，该研究指导了一种新的患者报告结局测量指标（PROM）的开发。在五个地点对患有下肢畸形的儿童及其父母进行了个体半结构化面对面访谈：加拿大（2个地点）、埃塞俄比亚、印度和美国。
在五个国际地点进行了79次访谈。定性访谈中出现了五个主要主题，并构成了概念框架的基础。这些主题是：1）外观，2）身体健康，3）心理健康，4）学校，5）社会健康。
下肢畸形对儿童的HRQL有重大影响。我们研究中确定的关注概念在所有国家的儿童中都相似。该概念框架指导了针对这些患者的结局量表的开发。使用这种新的PROM收集的关于各种治疗选择对下肢畸形儿童HRQL影响的信息，可用于告知父母和儿童与特定治疗选择相关的结局（身体、社会、心理）。这些信息可以补充其他客观结局信息（例如，并发症发生率、腿部外观等），以帮助家庭就儿童的治疗方案做出更明智的决定。
