Approaches to the management of haemoptysis in young people with cystic fibrosis.

Haemoptysis occurs in up to 25 % of young people with Cystic fibrosis (CF) [1]. We undertook a literature review and described the management approach to haemoptysis in CF between 2010 and 2020 at an Australian tertiary paediatric centre, The Children's Hospital Westmead, Sydney, New South Wales, using a retrospective review of the medical records which identified 67 episodes. Sixty episodes met inclusion criteria, including 31 patients. Using the US CF Foundation guidelines, episodes were classified as scant (53.3 %), moderate (38.3 %) or massive (8.3 %). Fifty-two percent of patients were female, mean age at presentation was 15.4 years (SD+/- 2.4) and 58 % were homozygous for the Fdel508 genotype. Twelve episodes (9 patients) required bronchial artery embolization (BAE). BAE was used in all cases of massive haemoptysis 5/5 (100 %), 6/23 (22 %) episodes of moderate and 1/32 (3 %) episode of scant haemoptysis as an elective procedure for recurrent haemoptysis. Our literature review and institutional experience highlights the need for up-to-date management guidelines in the management of haemoptysis in Cystic Fibrosis. Based on our experience, we provide a proposed algorithm to help guide the management of haemoptysis in CF.

Superior Extension of Upper Instrumented Vertebrae in Distraction-based Surgery: A Surrogate for Clinically Significant Proximal Junctional Kyphosis.

BACKGROUND: Proximal junctional kyphosis (PJK) is a reported complication of distraction-based growth-friendly surgery for early-onset scoliosis (EOS). A potential consequence of PJK is revision surgery with superior extension of the upper instrument vertebrae (UIV). The purpose of this study was to determine the risk of radiographic and clinically significant PJK during growth-friendly surgery. METHODS: This is a retrospective review of children treated with distraction-based growth-friendly surgeries from two EOS registries with minimum two-year follow-up. PJK is defined as clinically significant in this study if surgery with superior extension of the UIV was performed. RESULTS: Of 419 total patients, there was a 20% risk of developing clinically significant PJK (24% rib vs. 15% spine-based anchors, p = .03). These patients had a mean preoperative age of 5.6 years (5.2-year rib vs. 6.0-year spine, p < .001), scoliosis of 73° (69° rib vs. 77° spine, p < .001), and kyphosis of 51° (47° rib vs. 56° spine, p < .01). Regression analysis demonstrated that these differences in age, scoliosis, and kyphosis between anchor type did not account for a significant proportion of the measured variance. CONCLUSIONS: There was a 20% risk of developing clinically significant PJK, with a slightly higher risk for patients treated with rib-based proximal anchors (24%) than for those patients treated with spine-based proximal anchors (15%). LEVEL OF EVIDENCE: Level III.

Newborns Referred for Therapeutic Hypothermia: Association between Initial Degree of Encephalopathy and Severity of Brain Injury (What About the Newborns with Mild Encephalopathy on Admission?).

OBJECTIVE: The aim of this article was to describe the severity of brain injury and/or mortality in a cohort of newborns referred for therapeutic hypothermia, in relation to the degree of encephalopathy on admission, and to especially look at the ones with initial mild encephalopathy. STUDY DESIGN: Term newborns with perinatal depression referred to our neonatal intensive care unit for possible hypothermia treatment from 2008 to 2012 were enrolled prospectively. The modified Sarnat score on admission was correlated with severity of brain injury on brain imaging and/or autopsy. RESULTS: A total of 215 newborns were referred for possible cooling. Sixty percent (128/215) were cooled. Most of the not-cooled newborns with an available brain magnetic resonance imaging (85% = 50/59) had an initial mild encephalopathy, and 40% (20/50) developed brain injury. Some cooled newborns had an initial mild encephalopathy (12% = 13/108); only 31% (4/13) developed brain injury. CONCLUSION: Our results demonstrated that several newborns with an initial mild encephalopathy developed subsequent brain injury, especially when they were not cooled.

Pediatric Hereditary Neuralgic Amyotrophy: Successful Treatment With Intravenous Immunoglobulin and Insights Into SEPT9 Pathogenesis.

Hereditary neuralgic amyotrophy is a rare disorder characterized by the sudden onset of recurrent episodes of painful brachial plexus neuropathies, followed by atrophy within a few weeks. The authors present the case of a 5-year-old boy who developed hereditary neuralgic amyotrophy in the right upper limb after a gastroenteritis illness. He made a full and rapid recovery with the use of intravenous immunoglobulin. A subsequent episode in the left upper limb during the course of intravenous immunoglobulin was significantly attenuated. A de novo c.262C>T mutation in exon 2 of the SEPT9 gene was identified. To our knowledge, he is the first pediatric patient with SEPT9 hereditary neuralgic amyotrophy to be treated with intravenous immunoglobulin. The authors hypothesize that the c.262C>T mutation in exon 2 of the SEPT9 gene generates pathology via the numerous isoforms under specific conditions and that intravenous immunoglobulin can play a role at the epigenetic level of improving dysfunctional SEPT9 expression.

Diet-induced weight gain produces a graded increase in behavioral responses to an acute immune challenge.

Sickness behaviors and fever during infection constitute an adaptive and tightly regulated mechanism designed to efficiently clear the invading pathogen from the body. Recent literature has demonstrated that changes in energy status can profoundly affect the fever response to an acute immune challenge. The purpose of the present study was to investigate whether the exacerbating effect of diet induced obesity (DIO) on the LPS-induced fever response demonstrated previously would generalize to other sickness behaviors and, further, whether incremental changes in body weight would influence these responses. Results showed that DIO male Wistar rats exhibited a higher number of sickness symptoms for a longer period after lipopolysaccharide (LPS) injection (100µg/kg) than lean rats. Similarly, they showed a more prolonged fever and a delayed recovery from LPS-induced suppression of social interaction. No difference in locomotor activity was observed between obese and lean groups. Comparisons among groups that varied in body weight showed that an 11% increase in body weight was sufficient to increase the number and duration of sickness symptoms displayed after an LPS-injection and that the severity of sickness symptoms increased with increasing body weight. Together these data suggest that DIO can have profound effects on multiple behavioral responses to an acute immune challenge placing obese organisms at higher risk of the consequences of prolonged inflammation.

Effectiveness of sensory integration interventions in children with autism spectrum disorders: a pilot study.

The purpose of this pilot study was to establish a model for randomized controlled trial research, identify appropriate outcome measures, and address the effectiveness of sensory integration (SI) interventions in children with autism spectrum disorders (ASD). Children ages 6-12 with ASD were randomly assigned to a fine motor or SI treatment group. Pretests and posttests measured social responsiveness, sensory processing, functional motor skills, and social-emotional factors. Results identified significant positive changes in Goal Attainment Scaling scores for both groups; more significant changes occurred in the SI group, and a significant decrease in autistic mannerisms occurred in the SI group. No other results were significant. The study discusses considerations for designing future outcome studies for children with ASD.