Distribution of Femoral Metastases; Potential Role for Extended FDG PET/CT Scanning.

This study investigates the distribution of femoral metastases in cancer patients, specifically addressing the incidence of distal femoral metastases. PET/CT examinations routinely extend only to mid-thigh level, precluding detection of distal metastases. We found a total of 208 femoral metastases in 112 patients. 30% had distal femoral metastases in addition to other areas of involvement. 7% of patients with femoral metastases had only distal femur disease. 6 patients had distal pathologic fractures. Exclusion of the distal femur during PET/CT may result in a missed or delayed diagnosis that could contribute to the development of a pathologic fracture with increased morbidity.

Cost Utility of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease.

OBJECTIVE: The gene therapy voretigene neparvovec (VN) is the first Food and Drug Administration-approved treatment for vision loss owing to the ultra-rare RPE65-mediated inherited retinal disorders. We modeled the cost-utility of VN compared with standard of care (SoC). STUDY DESIGN: A 2-state Markov model, alive and dead, with a lifetime horizon. METHODS: Visual acuity (VA) and visual field (VF) were tracked to model quality-adjusted life-years (QALYs). VN led to an improvement in VA and VF that we assumed was maintained for 10 years followed by a 10-year waning period. The cost of VN was $850 000, and other direct medical costs for depression and trauma were included for a US healthcare system perspective. A modified societal perspective also included direct nonmedical costs and indirect costs. RESULTS: VN provided an additional 1.3 QALYs over the remaining lifetime of an individual. The average total lifetime direct medical cost for individuals treated with VN was $1 039 000 compared with $213 400 for SoC, leading to an incremental cost-effectiveness ratio (ICER) of $643 800/QALY from the US healthcare system perspective. Direct nonmedical costs totalled $1 070 900 for VN and $1 203 300 for SoC, and indirect costs totalled $405 400 for VN and $482 900 for SoC, leading to an ICER of $480 100/QALY from the modified societal perspective. CONCLUSIONS: At the current price, VN was unlikely to reach traditional cost-effectiveness standards compared with SoC. VN has important implications for both development and pricing of future gene therapies; therefore clinical and economic analyses must be carefully considered.

Exploration of the cognitive and behavioural consequences of paediatric cochlear implantation.

This study followed the progress of young deaf children undergoing cochlear implantation, in terms of their non-verbal cognitive abilities and their emotional/behavioural development. A consecutive series of 20 children, between the ages of 2 and 5 years, were assessed using the Leiter International Performance Scale - Revised (LIPS-R) and the Child Behaviour Checklist (CBCL), prior to receiving a Nucleus 24 cochlear implant. They were assessed again one year after the device was switched on. Scores on seven of the 13 subtests and composite scales of the LIPS-R increased significantly during the first year of cochlear implant use. Cognitive functions post-implant (Sequential Order, Fluid Reasoning, Full IQ and Attention Sustained) were positively related to behavioural outcomes. These results may be explained by an increased capacity of the implanted children to use verbal concepts to encode visual material. There was also evidence for a reduction in internalizing behaviour problems. The results are discussed in the context of previous research, clinical and theoretical perspectives.

The cognition and behaviour of children with cochlear implants, children with hearing aids and their hearing peers: a comparison.

Hearing impairment has been associated with cognitive deficits. It is not yet clear whether cochlear implants enable children to reduce or overcome these deficits. This study examined whether hearing impaired children with cochlear implants differed from hearing impaired children without cochlear implants or from hearing children. The three age-matched groups were compared on a non-verbal measure of cognition, the Leiter International Performance Scale-Revised (LIPS-R), and a measure of behaviour, the Child Behaviour Checklist (CBCL). Apart from their scores on the Attention Sustained Subtest of the LIPS-R, the performance of the implanted group and the hearing group closely corresponded, while the hearing impaired children without cochlear implants performed below the level achieved by both these groups. There were no differences between any groups on the CBCL. It was concluded that hearing impaired children with cochlear implants were able to perform at the same non-verbal cognitive level as hearing children.