Surgical Management and Denosumab for Aneurysmal Bone Cysts of the Spine in an Australian Tertiary Paediatric Centre.

Aneurysmal bone cysts (ABC) are rare osteolytic, benign but often locally aggressive tumours of the long bones or vertebrae. For spinal ABC, surgical management, embolisation or sclerotherapy alone often carry high morbidity and/or high recurrence rates. Interruption of receptor activator of nuclear factor-kappa B ligand (RANKL) signalling holds promise as an effective therapeutic strategy for these tumours. We aimed to review the approach to surgical management and evaluate the efficacy and safety of denosumab for ABC of the spine in children. Retrospective review of 7 patients treated with denosumab using a standardised protocol for ABC of the spine in a tertiary paediatric centre. Surgical intervention was only conducted if there was spinal instability or significant neurological impairment. Denosumab 70 mg/m2 was given 4-weekly for at least 6 months, followed by 2 doses of zoledronate 0.025 mg/kg, aiming to prevent rebound hypercalcaemia. All patients achieved stability of the spine and resolution of neurological impairment, if present. Six patients achieved metabolic remission and have ceased denosumab without recurrence to date; the other showed clinical and radiological improvement without complete metabolic remission. Three patients developed symptomatic hypercalcaemia 5-7 months after cessation of denosumab, requiring additional bisphosphonate treatment. We present our algorithm for the surgical and medical management of paediatric spinal ABC. Denosumab produced a radiological and metabolic response in all patients, with complete remission in most. Follow-up time was not long enough to evaluate the endurance of response after cessation in some patients. Incidence of rebound hypercalcaemia in this paediatric cohort was high, prompting a change to our protocol.

Denosumab for central giant cell granuloma in an Australian tertiary paediatric centre.

BACKGROUND: Central giant cell granulomas (CGCG) are rare osteolytic, benign but often locally aggressive tumours of bone. Surgical curettage may not be possible in extensive lesions and resection carries high morbidity, especially in growing children, and previous medical therapies have had variable efficacy and high recurrence rates. Interruption of receptor activator of nuclear factor-kappa B ligand (RANKL) signalling holds promise as an effective therapeutic strategy for these tumours. AIMS: To evaluate the efficacy and safety of our protocol for denosumab treatment of CGCG in children. METHODS: Retrospective review of 4 patients treated with denosumab using a standardised protocol for CGCG in a tertiary paediatric centre. Denosumab 70 mg/m2 was given 4-weekly, followed by 2 doses of zoledronate 0.025 mg/kg, aimed at preventing rebound hypercalcaemia. RESULTS: Treatment of CGCG resulted in metabolic remission in all patients, but recurrence, detected by positron emission tomography (PET), occurred at 6 months in three patients and 12 months in one patient. Three patients developed symptomatic hypercalcaemia 4-5 months and one patient asymptomatic hypercalcaemia 7 months after cessation of denosumab, with 3 requiring additional bisphosphonate treatment. CONCLUSIONS: Denosumab produced a radiological and metabolic response in our patients, but metabolic recurrence occurred in all patients. PET imaging was effective for monitoring treatment response and early detection of recurrence. Incidence of rebound hypercalcaemia in this paediatric cohort was high. We present proposed changes to our protocol with the aim of producing sustained remission and preventing rebound hypercalcaemia.

Family Impact of Child Oro-Facial Cleft.

OBJECTIVE: To describe the association between children's orofacial cleft (OFC) and families' quality of life (QoL), using the short-form Family Impact Scale questionnaire (FIS-SF). Also assessed were the psychometric properties of the FIS-SF, as well as whether certain demographic and clinical variables impacted the family. DESIGN: Observational cross-sectional study. SETTING: Tertiary care public children's hospital in New South Wales, Australia. PARTICIPANTS: Parents/caregivers of children with OFC. MAIN OUTCOME MEASURE(S): The short-form Family Impact Scale questionnaire. RESULTS: Two hundred and fourteen parents completed the FIS-SF. Excellent convergent validity was evident, but discriminant validity was weaker. Those children with velopharyngeal insufficiency/submucous cleft reported lower scores on the family/parental activity, and lower overall family impact scores than those with cleft lip (CL) or cleft palate (CP). Those with cleft lip and palate (CL/P) had poorer family/parental activity scores than those with CP. There were no systematic differences in FIS-SF scores by the child's age, gender, private health insurance, living location, the number of cleft-related operations, or whether a child had commenced orthodontic treatment. CONCLUSIONS: The FIS-SF is a valid and reliable measure for determining the impact that OFC has on family QoL. The impact of OFC on children's families differs according to cleft type.

Understanding behaviour change in context: examining the role of midstream social marketing programmes.

This research examines how midstream social marketing programmes that adopt a relational and community-based approach create opportunities for individuals to make incremental changes to health behaviour. Specifically, it applies Bourdieusian theory to explore how interactions between community healthcare workers (CHWs) and members of the public generate impetus for change and foster individual agency for improved health. Qualitative interviews were carried out with members of the public and CHWs engaged in a Smokefree home and cars initiative. The findings suggest that although CHWs are challenged by resource constraints, their practices in working with individuals and families build trust and enable dialogue that bridges smoking-related health insight with home logics. These interactions can promote individual agency with a transformative effect through small changes to smoking-related dispositions, norms and practices. However, tensions with the habitus of other household members and other capital deficits can inhibit progress towards embedding new practices. The study concludes that interventions built upon community relationships show potential for addressing limitations of information-focused campaigns but there is a need to also respond to key social structures relating to the field of action for new health dispositions to become embedded in practice.

Validity and reliability of the COHIP-SF in Australian children with orofacial cleft.

AIM: To measure the validity and reliability of the Child Oral Health Impact Profile-Short Form (COHIP-SF) in Australian children with orofacial cleft (OFC) and to evaluate the need for a proxy report. DESIGN: Children with OFC and their proxy completed a COHIP-SF questionnaire. Items were divided into oral health, functional and socio-emotional subscales, and scores on all subscales were compared between and within groups. RESULTS: A total of 222 children (average age 11.3, SD = 3.5, range 8.0-14.0) and 215 proxies completed the COHIP-SF, with similar mean scores of 50.8 (SD = 13.7) and 48.1 (SD = 14.0). Excellent internal consistency reliability (Cronbach's alpha 0.9) and convergent validity was evident (P < 0.001); however, discriminant validity was weaker. There was strong correlation between child and proxy reports (Pearson's r = 0.7), and intraclass correlation coefficients were high indicating good child/proxy agreement. Older children had poor socio-emotional well-being (P < 0.004), and children with bilateral cleft lip and palate (CL/P) and velopharyngeal insufficiency/submucous cleft (VPI/SMC) had poor functional well-being. Those with private health insurance reported better oral health-related quality of life (OHRQoL) (P < 0.05). CONCLUSION: The COHIP-SF is a valid and reliable measure for measuring OHRQoL in Australian children with OFC. Proxy reports did not add further information regarding a child's OHRQoL.

Association of cyclophosphamide use with dental developmental defects and salivary gland dysfunction in recipients of childhood antineoplastic therapy.

BACKGROUND: The aim of this study was to examine the effect of antineoplastic therapy on dental development and saliva function in recipients of childhood antineoplastic therapy. METHODS: Patients attending the long-term follow-up clinic at Children's Hospital at Westmead, NSW, Australia, were included if they had received treatment prior to 16 years of age and were in remission for more than 5 years. A dental examination and saliva test were performed for each participant. Holtta's Defect Index (HDI) was used to assess tooth aplasia, microdontia, and root-crown ratio on an orthopantomogram (OPG). Multivariable-adjusted regression analyses were used to estimate the association of patient characteristics and treatment modalities with dental outcomes. RESULTS: One hundred six participants (61% male) were recruited (response rate = 88%). The mean HDI score was 24.7 ± 17.8. A cumulative dose of cyclophosphamide >7500 mg/m(2) increased the HDI score by 13.06 (P = .01). Recipients of cyclophosphamide also had significantly increased odds of exhibiting very low saliva flow (<0.7 mL/min) (odds ratio = 12.43; 95% confidence interval, 2.08-74.35; P = .006). CONCLUSIONS: Children and adolescents who received high doses of cyclophosphamide were at increased risk of dental disturbances. Cyclophosphamide recipients were also at greater risk of exhibiting very low saliva flow. This study applied the HDI to patients receiving all forms of antineoplastic treatment and highlights the dose-dependent relation between cumulative dose of cyclophosphamide and dental disturbances.

A previously unreported association between Nance-Horan syndrome and spontaneous dental abscesses.

Atypical dentofacial structures may be the first indicator of other anomalies linked to a syndrome. This case describes the management of a 9-year-old girl referred for the routine management of supernumerary teeth. The anomalous form of her teeth, together with multiple supernumerary units and a history of congenital cataracts, were suggestive of a diagnosis of Nance-Horan syndrome. This is an X-linked disorder, in which females usually demonstrate mild expression; this case was unusual in respect to the marked phenotype expressed. Unusually, the girl developed 2 spontaneous abscesses of her noncarious upper incisor teeth; a feature never previously described in this syndrome. This report details the patient's dental management and discusses the possible pathogenesis of the dental abscesses, together with the genetic implications of this syndrome.