What matters to children with lower limb deformities: an international qualitative study guiding the development of a new patient-reported outcome measure

Abstract

Background 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. Results 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. Conclusions 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.

Patients’ quality of life assessment following treatment of leg length discrepancy in a single center in Riyadh

“…Furthermore, the information obtained from their patient-reported outcome measure on how different treatment options could affect children with LLD confirms that HRQL may be used to educate parents and kids on the possible repercussions of various treatment options. [14] Oostenbroek et al reported that correcting LLD of 25% or higher of the original limb length proved to be associated with an increased likelihood of developing complications that may have an adverse effect on patients' QoL. They had 37 children aged 6-17 years who were treated by the Ilizarov technique for lengthening; out of them, 10 had an unsuccessful outcome and additional surgical procedure was necessary to reduce their complications.…”

LIMB-Q Kids—German Translation and Cultural Adaptation

“…Collecting information about the effects of various treatment modalities on HRQL is thus important in order to inform patients and their next of kin. PROMs are indispensable in modern health care, because, if developed and validated in an appropriate way, they not only include the patients, but genuinely put them at the center, and measure what is important to them [ 4 , 23 , 24 , 25 , 28 , 29 ]. PROMs capture more important aspects than mere radiological or other objectively measured outcomes, e.g., joint range of motion, muscle strength, etc.…”

“…PROMs capture more important aspects than mere radiological or other objectively measured outcomes, e.g., joint range of motion, muscle strength, etc. [ 24 , 29 , 30 ]. The value of PROMs is highlighted by the fact that PROMs do not necessarily correlate well with radiological or other objectively assessed outcomes [ 31 ].…”

“…Chhina et al, conducted a systematic review on PROMs used for children with lower limb deformities and after identifying the lack of a valid PROM, the development of LIMB-Q Kids was initiated [ 4 , 23 ]. Qualitative interviews were conducted with children from high-income, low-income, and lower-to-middle-income countries to determine what matters most to children with lower limb deformities [ 24 , 25 ]. Item generation and scale development for the new PROM, LIMB-Q Kids, was performed based on qualitative interviews.…”

German Translation and Cross-Cultural Adaptation of the Limb Deformity-Scoliosis Research Society (LD-SRS) Questionnaire

“…PROMs may be conducted for assessing the general health status, e.g., the European Quality of Life 5 Dimensions (EQ5D) and the Short Form Health Survey (SF-36) are widely used [ 12 ]. Disease-specific PROMs for LD are scarce, but several research groups aim to establish and validate LD-PROMs [ 13 , 14 , 15 , 16 , 17 , 18 ].…”