Do's and Don'ts in Primary Aneurysmal Bone Cysts of the Proximal Femur in Children and Adolescents : Retrospective Multicenter EPOS Study of 79 Patients

Background:Aneurysmal bone cysts (ABC) are rare benign cystic bone tumors, generally diagnosed in children and adolescents. Proximal femoral ABCs may require specific treatment strategies because of an increased pathologic fracture risk. As few reports are published on ABCs, specifically for this localization, consensus regarding optimal treatment is lacking. We present a large retrospective study on the treatment of pediatric proximal femoral ABCs. Methods:All eligible pediatric patients with proximal femoral ABC were included, from 11 tertiary referral centers for musculo-skeletal oncology (2000-2021). Patient demographics, diagnostics, treatments, and complications were evaluated. Index procedures were categorized as percutaneous/open procedures and osteosynthesis alone. Primary outcomes were: time until full weight-bearing and failure-free survival. Failure was defined as open procedure after primary surgery, >3 percutaneous procedures, recurrence, and/or fracture. Risk factors for failure were evaluated. Results:Seventy-nine patients with ABC were included [mean age, 10.2 (+/- SD4.0) y, n=56 male]. The median follow-up was 5.1 years (interquartile ranges=2.5 to 8.8).Index procedure was percutaneous procedure (n=22), open procedure (n=35), or osteosynthesis alone (n=22). The median time until full weight-bearing was 13 weeks [95% confidence interval (CI)=7.9-18.1] for open procedures, 9 weeks (95% CI=1.4-16.6) for percutaneous, and 6 weeks (95% CI=4.3-7.7) for osteosynthesis alone (P=0.1). Failure rates were 41%, 43%, and 36%, respectively. Overall, 2 and 5-year failure-free survival was 69.6% (95% CI=59.2-80.0) and 54.5% (95% CI=41.6-67.4), respectively. Risk factors associated with failure were age younger than 10 years [hazard ratios (HR)=2.9, 95% CI=1.4-5.8], cyst volume >55 cm(3) (HR=1.7, 95% CI=0.8-2.5), and fracture at diagnosis (HR=1.4, 95% CI=0.7-3.3). Conclusions:As both open and percutaneous procedures along with osteosynthesis alone seem viable treatment options in this weight-bearing location, optimal treatment for proximal femoral ABCs remains unclear. The aim of the treatment was to achieve local cyst control while minimizing complications and ensuring that children can continue their normal activities as soon as possible. A personalized balance should be maintained between undertreatment, with potentially higher risks of pathologic fractures, prolonged periods of partial weight-bearing, or recurrences, versus overtreatment with large surgical procedures, and associated risks.Peer reviewe

European Cystic Fibrosis Society standards of care: best practice guidelines

Specialised CF care has led to a dramatic improvement in survival in CF: in the last four decades, well above what was seen in the general population over the same period. With the implementation of newborn screening in many European countries, centres are increasingly caring for a cohort of patients who have minimal lung disease at diagnosis and therefore have the potential to enjoy an excellent quality of life and an even greater life expectancy than was seen previously. To allow high quality care to be delivered throughout Europe, a landmark document was published in 2005 that sets standards of care. Our current document builds on this work, setting standards for best practice in key aspects of CF care. The objective of our document is to give a broad overview of the standards expected for screening, diagnosis, pre-emptive treatment of lung disease, nutrition, complications, transplant/end of life care and psychological support. For comprehensive details of clinical care of CF, references to the most up to date European Consensus Statements, Guidelines or Position Papers are provided in Table 1. We hope that this best practice document will be useful to clinical teams both in countries where CF care is developing and those with established CF centres

As simple as it sounds? The treatment of simple bone cysts in the proximal femur in children and adolescents: Retrospective multicenter EPOS study of 74 patients

Purpose: Simple bone cysts are among the most prevalent benign cystic tumor-like lesions in children. Proximal femoral simple bone cysts may require specific treatment because of increased fracture risk. With limited literature available on this specific localization, consensus regarding optimal treatment is lacking. We present a large international multicenter retrospective cohort study on proximal femoral simple bone cysts.Methods: All consecutive pediatric patients with proximal femoral simple bone cyst from 10 tertiary referral centers for musculoskeletal oncology were included (2000-2021). Demographics, primary treatment, complications, and re-operations were evaluated. Primary outcomes were time until full weight-bearing and failure-free survival.Results: Overall, 74 simple bone cyst patients were included (median age 9 years (range = 2-16), 56 (76%) male). Median follow-up was 2.9 years (range = 0.5-21). Index procedure was watchful waiting (n = 6), percutaneous procedure (n = 12), open procedure (n = 50), or osteosynthesis alone (n = 6). Median time until full weight-bearing was 8 weeks (95% confidence interval = 0.1-15.9) for watchful waiting, 9.5 (95% confidence interval = 3.7-15.3) for percutaneous procedure, 11 (95% confidence interval = -0.7 to 13.7) for open procedure, and 6.5 (95% confidence interval = 5.9-16.1) for osteosynthesis alone (p = 0.58). Failure rates were 33%, 58%, 29%, and 0%, respectively (p = 0.069). Overall failure-free survival at 1, 2, and 5 years was 77.8% (95% confidence interval = 68.2-87.4), 69.5% (95% confidence interval = 58.5-80.5), and 62.0% (95% confidence interval = 47.9-76.1), respectively.Conclusion: A preferred treatment for proximal femoral simple bone cysts remains unclear, with comparable failure rates and times until full weight-bearing. Watchful waiting may be successful in certain cases. If not feasible, osteosynthesis alone can be considered. Treatment goals should be cyst control, minimizing complications and swift return to normal activities. Therefore, an individualized balance should be made between undertreatment, with potentially higher complication risks versus overtreatment, resulting in possible larger interventions and accompanying complications