Welcome to the 9th EAPS Congress Programme Scheduling

Background and Aims

Despite international legislation to address unmet pediatric medical needs, many drugs are still prescribed off-label to the pediatric population. Although off-label drug use not supported by high level of evidence is potentially harmful, a comprehensive overview of the quality of the evidence pertaining off-label drug use in children is lacking.

Methods

The Dutch Pediatric Formulary provides best evidence-based dosing guidelines for drugs used in children and represents overall pediatric prescribing. For each drug- indication-age group combination, we scored the highest available level of evidence: authorized drug use (summary of product characteristics, SmPC), systematic review or meta-analysis, randomized controlled trial (RCT), comparative research, non-comparative research, or consensus-based expert opinions. For records that were based on selected guidelines the original sources were not reviewed. These records were scores as guideline.

Results

A total of 774 drugs included 6426 records. Of all off-label records (n=2718), 14% were supported by high quality evidence (4% meta-analysis or systematic reviews, 10% RCTs of high quality), 20% by comparative research, 14% by non-comparative research, 37% by consensus-based expert opinions and 15% by selected guidelines. 58% of all records were authorized, increasing with age from 30% in preterm neonates (n=110) up to 64% in adolescents (n=1630).

Conclusions

Many have advocated that off-label use is only justified when supported by a high level of evidence. We show that this prerequisite would seriously limit available drug treatment for children. Our data identify the drugs and therapeutic areas for which clinical evidence is clearly missing and could therefore drive the global research agenda.

Background and Aims

In recent years, evolution of surgery has led to laparoscopy and then to single port surgery. In pediatric age, single port surgery is still under discussion; the aim of this study is to report our experience with a new device used in the past 5 years

Methods

A retrospective analysis of first 350 cases was performed collecting the data of all patients treated with this new device from 2017. Epidemiological data, diagnosis, operative times and complications were analyzed. Post-operative pain was compared with standard laparoscopy

Also cosmetic results were compared to standard laparoscopy

Results

A total of 350 procedures were performed during the study period. The age range was 1-17 years. The conversion rate was 3.4% (12 patients) including 3 conversion to traditional laparoscopy and to laparotomy. Pain management was comparable to traditional laparoscopy. The complication rate was 3.2%, in one case leading to re-do surgery. All the cases in our Unit were successfully completed, with complications mainly related to the original pathology rather than to the technique itself. at follow-up (3 months), cosmetic results were better for single port surgery respect to standard laparoscopy, for both patients and parents (patients aged > 6 years).

Conclusions

The learning curve for this device and for single port surgery proved to be functional as for standard laparoscopy. In this study, we reported surgical indications for the use of single port laparoscopy discerning favorable and unfavorable procedures; A proven superiority of this technique over traditional laparoscopy is yet to be defined, but single port surgery has proved to be a safe and easy tool to reduce invasiveness of procedures in pediatric surgery with better cosmetic results

Background and Aims

Anaphylaxis is rising in prevalence amongst children and is managed with allergen avoidance and adrenaline auto-injector(AAI) use. This study seeks to determine at what age caregivers start to teach their children to recognise anaphylaxis and use AAI, the factors affecting this, how this compares to EAACI guideline’s recommendation of age 11-13 and who they believe should teach their child.

Methods

An online questionnaire was distributed to a convenience sample of 500 caregivers of paediatric allergy patients in Cork University Hospital. Statistical analysis was performed using Stata.

Results

123 responses to the questionnaire were received. Ages indicated were: <6 years for teaching recognition of anaphylaxis symptoms(65.9%) and 9-11 years for teaching to self-inject an AAI(44.7%). History of severe anaphylaxis (94.3%), the child’s ability to describe reasons to inject adrenaline(87.8%) and demonstrate AAI use(82.1%) were the most common “very important” readiness factors identified. Almost half of caregivers were “not confident”(8.94%) or “somewhat confident”(40.65%) in training their child to use AAI and the majority believed paediatric allergy specialists(30.89%) and nurses(37.40%) should be responsible for this. Caregivers with higher household incomes identified themselves more frequently as the party responsible for training their children to use AAI(p=0.04).

Conclusions

Caregivers begin to transfer the responsibility of anaphylaxis recognition and AAI use to their children significantly younger than the EAACI recommended age. Caregivers expressed suboptimal confidence in teaching their children to use AAI. Further evaluation is necessary to improve guidelines, enabling clinicians to train and support caregivers during this transition.

Background and Aims

Individuals born very preterm (<32 weeks of gestation; VP) or very low birth weight (<1500g; VLBW) have been found to be less likely to experience romantic relationships. However, it is unknown whether neonatal complications, sex, neurosensory impairments or social factors may explain the association of VP/VLBW with less romantic and sexual relationships. This was investigated in the present study.

Methods

Individual participant data (IPD) were obtained from 11 prospective cohort studies of individuals born VP/VLBW and term-born controls contributing to the ‘Research on European Children and Adults Born Preterm (RECAP-preterm)’ consortium. The combined dataset comprised 1,583 VP/VLBW and 1,641 term-born adults. IPD meta-analyses were performed for indicators of romantic and sexual relationships using a one-stage approach.

Results

Adults born VP/VLBW (≥ 18 years old) were less likely to be in a romantic relationship (OR, 0.49; 95% CI, 0.31, 0.76), to marry/cohabitate (OR, 0.70; 95% CI, 0.53, 0.92), or to have experienced sexual intercourse (OR, 0.23; 95% CI, 0.13, 0.41). When sexually active, VP/VLBW participants were more likely to have their first sexual intercourse after the age of 18 (OR, 1.93; 95% CI, 1.24, 3.01). Among VP/VLBW adults, being male or having any neurosensory impairment (NSI) decreased the likelihood to experience romantic and sexual relations

Conclusions

VP/VLBW birth decreases the likelihood of having romantic and sexual relationships in adulthood. The likelihood of not experiencing these relationships is higher for VP/VLBW adults who are male or have NSI. This has important implications for fertility and wellbeing of VP/VLBW adults.

Background and Aims

Patients with neurogenic bladder (NB) have a high risk of developing chronic kidney disease (CKD). Due to their lower muscle mass, the estimation of glomerular filtration rate (eGFR) using creatinine (Cr) may be overestimated and delay the diagnosis of renal failure.

We aim to compare eGFR calculated by different formulas based on creatinine and/or cystatin C (CysC) in children with NB, and the differences found between patients unable to walk independently, with lower muscle mass and underdeveloped lower limbs, and patients able to walk independently, with more developed lower limb musculature.

Methods

Data on pediatric patients with NB and CKD stage 1 and 2, were collected. The eGFR was calculated using CKiD-Cr, CKiD-CysC, Zappitelli-CysC, Schwartz combined-Cr/CysC and Zappitelli combined-Cr/CysC.

Results

Forty-seven patients were evaluated, 74.5% CKD stage 1, median age of 14.1 years, 59.6% had lipo/myelomeningocele. When compared with CKiD-Cr, CysC-based formulas showed lower eGFR ​​(p<0.05), (CKiD-CysC (p<0.001), Zappitelli-CysC (p<0.001), Schwartz combined-Cr/CysC (p<0.001) and Zappitelli combined-Cr/CysC (p<0.05)).

When CKiD-CysC was used, 68% of patients moved to a more advanced CKD stage.

In patients unable to walk independently, with lower muscle mass, (55.3%) the median eGFR using CKiD-Cr (118.52) and Schwartz combined-Cr/CysC (91.97) were higher (p<0.05) than in the other group (91.23, 82.80). No differences (p>0.05) were found between the two groups using the other formulas (CKiD-CysC (78.38 vs 74.08)).

Conclusions

In patients with NB and poor muscle mass, CKiD-Cr formula may overestimate renal function. CysC-based formulas may be more reliable in these patients, especially in those with greater muscular atrophy.