Several measures of quality of life (QoL) are available for children with haemophilia. However, most are not disease-specific and few focus on children's perspectives. The purpose of this study was to develop a psychometrically sound measure of QoL that included the perspectives of boys with haemophilia. A list of potential items was developed from the literature, other measures, and input from five discussion sessions with adults with haemophilia, children with haemophilia and their parents and haemophilia nurses. The list was augmented with items generated by three focus groups with children and three focus groups with parents. These groups also prioritized items and recommended a domain structure. Supplemental information was gathered by surveying haematologists. Data from all sources were analysed to reduce the number of items using a two-step approach, based on rules that weighted the children's priorities most heavily. The remaining items were compiled into a questionnaire that was pilot tested with 10 children and their parents. The total item pool contained 228 potential items. Of these, 33 were removed based on three focus groups and survey responses, 72 were removed after the completion of all focus groups and 46 were removed due to redundancy. This resulted in a 77-item version of the CHO-KLAT. Pilot testing identified the need to subdivide two items, resulting in a 79-item CHO-KLAT. The CHO-KLAT is a promising disease-specific measure of QoL that reflects children's unique perspectives. This child-centric focus distinguishes the CHO-KLAT from alternative measures of QoL. Further research will assess the measurement properties of the CHO-KLAT.

The CHO-KLAT(35) is a reliable and valid measure of QoL for boys with haemophilia.

No abstract

BackgroundAvascular necrosis (AVN) is a significant and potentially devastating complication following the treatment of developmental dysplasia of the hip (DDH). The reported rate of AVN following closed reduction for DDH ranges from 4 to 60%, and the resultant influence on hip development remains unclear.PurposeA systematic review of the literature was undertaken to evaluate the frequency of AVN after more than 5 years of follow-up in children that underwent closed reduction at younger than 2-years of age for DDH.MethodsThe search strategy was formulated with key-concepts and keywords identified using the patient problem, intervention, comparison and outcome process. Searches were undertaken using Pubmed, Scopus and Web of Science up to and including May, 2016 to identify potential studies.ResultsA total of seven papers met the a priori inclusion and exclusion criteria of this review. The overall rate of significant AVN in 441 patients (538 hips) was 10% at a mean length of follow-up of 7.6 years (5–18.8) following closed reduction. This finding can be used to inform the feasibility of future intervention studies, and act as a baseline for which surgeons to compare their results to a ‘standard’.Electronic supplementary materialThe online version of this article (doi:10.1007/s11832-016-0776-y) contains supplementary material, which is available to authorized users.

The incidence of clinically significant avascular necrosis (AVN) following medial open reduction of the dislocated hip in children with developmental dysplasia of the hip (DDH) remains unknown. We performed a systematic review of the literature to identify all clinical studies reporting the results of medial open reduction surgery. A total of 14 papers reporting 734 hips met the inclusion criteria. The mean follow-up was 10.9 years (2 to 28). The rate of clinically significant AVN (types 2 to 4) was 20% (149/734). From these papers 221 hips in 174 children had sufficient information to permit more detailed analysis. The rate of AVN increased with the length of follow-up to 24% at skeletal maturity, with type 2 AVN predominating in hips after five years' follow-up. The presence of AVN resulted in a higher incidence of an unsatisfactory outcome at skeletal maturity (55% vs 20% in hips with no AVN; p < 0.001). A higher rate of AVN was identified when surgery was performed in children aged < 12 months, and when hips were immobilised in ≥ 60° of abduction post-operatively. Multivariate analysis showed that younger age at operation, need for further surgery and post-operative hip abduction of ≥ 60° increased the risk of the development of clinically significant AVN.

Disease-specific measures of quality of life (QoL) for children with haemophilia are now available for use in clinical studies [Haemophilia, 10, 2004, 9-16]. One of these measures, the Canadian Haemophilia Outcomes - Kids' Life Assessment Tool (CHO-KLAT), was developed in Canada with emphasis on the perspectives of children [Pediatr Blood Cancer, 47, 2006, 305-11; Haemophilia, 10, 2004, 34-43]. Another, the Haemo-QoL, was developed in Europe, with emphasis on the perspectives of clinicians [Haemophilia, 8, 2002, 47-54; Haemophilia, 10, 2004, 17-25]. While these two measures are unique and independent, researchers from both studies were collaboratively linked throughout development and testing. This study presents the results of a joint assessment of the two measures with respect to their strengths, limitations and unique contributions. The primary questions addressed were: 1 What is the relationship between the CHO-KLAT and the Haemo-QoL in terms of summary scores and item content? 2 What are the methodological strengths, limitations and unique contributions of each measure? We conducted a retrospective analysis of data from field testing of both measures. The analysis included a comparative assessment of the basic validity, reliability and items used in each measure. Overall, the CHO-KLAT and the Haemo-QoL are promising and valuable measures of QoL for children with haemophilia. Our analyses confirmed the basic psychometric properties of both tools, but identified some discrepancies between them. Additional data will allow for greater understanding of these discrepancies and lend clarity to how the tools should be used in clinical studies (separately or merged). The present recommendation is that the measures be run independently, but preferably concurrently in studies of children with haemophilia.

Update This article was updated on August 14, 2020, because of a previous error. On page 1, in the byline, the text that had read “S.P. Kelley, MBChB, PhD, FRCS(Tr&Orth), M.M. Feeney, BSc, MSc, BMBS, C.L. Maddock, BSc, MMASc, M.L. Murnaghan, MD, MEd, FRCS, and C.S. Bradley, BScPT, MSc, on behalf of the International Hip Dysplasia Institute (IHDI) Study Group*” now reads “S.P. Kelley, MBChB, PhD, FRCS(Tr&Orth), M.M. Feeney, BSc, MSc, BMBS, C.L. Maddock, BSc, MMASc, M.L. Murnaghan, MD, MEd, FRCS, C.S. Bradley, BScPT, MSc, and the International Hip Dysplasia Institute (IHDI) Study Group*”. An erratum has been published: JBJS Open Access. 2020;5(3):e18.00054ER. Background: Developmental dysplasia of the hip (DDH) is the most common orthopaedic disorder in newborns. While the Pavlik harness is one of the most frequently used treatments for DDH, there is immense variability in treatment parameters reported in the literature and in clinical practice, leading to difficulties in standardizing teaching and comparing outcomes. In the absence of definitive quantitative evidence for the optimal Pavlik harness management strategy for DDH, we addressed this problem by obtaining international expert-based consensus on the subject. Methods: An initial list of items relevant to Pavlik harness treatment was derived by a review of the literature. Delphi methodology was used to guide serial rounds of surveying and obtaining feedback from content matter experts from the International Hip Dysplasia Institute (IHDI), which continued in the same manner until consensus based on standard statistical analysis was reached. This was followed by a corroboration of face validity to derive the final set of management principles. Results: Four rounds of structured surveying were required to reach consensus. Following 2 rounds of peer review, and from an initial list of 66 items in 8 categories, we were able to derive 2 simplified, yet comprehensive, print-friendly tables consisting of 28 items in 8 categories to assist clinicians in managing DDH with a Pavlik harness. The tables contain principles of treatment initiation, application and follow-up of the harness, complications, weaning, and end-of-treatment decision-making as well as specific criteria based on the severity of the DDH. Furthermore, highly contentious items were identified as important areas of future study. Conclusions: We developed a comprehensive set of principles based on expert consensus to assist clinicians in the management of DDH using the Pavlik harness. This study also generated a list of the most controversial areas in the nonoperative management of DDH, which should be considered high priority for future study to further refine and optimize outcomes. Level of Evidence: Therapeutic Level V. See Instructions for Authors for a complete description of levels of evidence.

The average age at diagnosis of 88 patients with JIS was 8.4 ± 1.4 years, with a female to male ratio of approximately 8:1. Pretreatment, Risser score was zero for 80 patients (91%); 72 (92%) of the females were premenarche; and primary Cobb angles ranged from 20° to 71°. Of the 88 patients, 60 (68%) had used a thoracolumbosacral orthosis exclusively; 28 (32%) patients used "other braces" (Milwaukee, Charleston, or a combination of braces), with an average treatment duration of 3.6 ± 1.9 years.As per Scoliosis Research Society definitions, a "non-curve-progression" (≤5° change) group consisted of 25 (28%) patients; and a "curve-progression" group consisted of 63 (72%) patients where the curve had progressed 6° or more.Of the 88 patients, 44 (50%) underwent surgery. The operative rate was higher for patients with curves 30° or more than those with curves 20° to 29° prior to brace treatment (37/58 [64%] vs. 7/30 [23%], respectively; P = 0.001); other braces compared with thoracolumbosacral orthosis (19/28 [68%] vs. 25/60 [42%], respectively; P = 0.02); Lenke I and III curves compared with Lenke VI curves (33/54 [61%] vs. 2/14 [14%], respectively; P = 0.007).