A 15-year experience in pediatric palliative care: a retrospective hospital-based study.

BACKGROUND: The current Italian scenario of pediatric palliative care (PPC) services is characterized by inadequate coverage of the territory. Therefore, it is important to improve the referral of patients to the most appropriate setting (community care, general PPC, or specialized PPC) and to improve the delivery of PPC care. METHODS: Aiming at obtaining information about the referrals to the Padua Pediatric Hospice that could help estimate the investments needed to improve the provision of care, a retrospective analysis has been carried out. The rate of proper referral and discharge, the number of patients followed at the hospice, the mortality rate, and the length of follow-up were analyzed, and, when possible, data were stratified by oncological and non-oncological diseases. RESULTS: The analysis showed that of the 870 patients referred to the Padua Pediatric Hospice between 2008 and 2022, 76% were affected by non-oncological conditions. 82% of patients referred were taken in charge and most of the remaining patients have been inappropriately referred. The analysis showed a growing number of total referrals, which increased by 195% from 2008 to 2022. An increase in proper referrals and referrals of non-oncological patients was observed alongside a decrease in oncological patient referrals and a trend toward a decrease in discharge rates. A decreased mortality was observed in patients with non-oncological conditions, with only 6% of deceased patients in 2022. Moreover, a longer survival with a median follow-up length of 43 months was observed among patients with non-oncological conditions who were followed up at the Padua Pediatric Hospice for more than 12 months. Conversely, the short survival rate observed for oncological patients suggests that those patients should have been referred to PPC earlier to benefit from palliative care for longer periods. CONCLUSIONS: Considering these data, it is expected that the number of patients needing PPC services will steadily increase in the next years. Hence, there is a need to invest resources to provide the best care delivery model encompassing specific pathways for the transition into adulthood, the establishment of networks within all the Italian regions, and an efficient referral to the more suitable setting of care.

Comparison of peripheral cannula outcomes, ultrasound-guided placement of deep veins versus blind insertion of superficial veins in children aged 0-18 years: A retrospective study.

BACKGROUND: The clinical use of ultrasound has increased the chances of successful insertion of all venous access devices, including short peripheral cannulas (SPC) and long peripheral catheters (LPC). The aim of the study was to compare the clinical performance of peripheral cannulas inserted using the traditional "blind" technique versus those inserted with ultrasound guidance (US). METHODS: In this retrospective study we compared 135 peripheral intravenous cannulations (PIVCs) with ultrasound guidance and 135 PIVCs with blind technique, inserted in children (0-18 years) in the emergency department. All devices were inserted using the catheter-over-needle technique. With the blind technique, superficial arm veins were cannulated using SPCs (1.9 cm, 24G; 2.5 cm, 22G; 3.2 cm, 20G), while with US, deeper non-palpable veins were accessed using SPCs of 3.2 cm, 24G; and LPCs of 6.4 cm, 22G; 6.4 cm, 20G. RESULTS: The accesses were removed electively in 66% of US-PIVCs, with an average duration of the device of 5.3 ± 4.0 days, but only in 30% of Blind-PIVCs, which presented an average duration of 2.5 ± 1.8 days. The longest dwelling time (8.7 ± 5.1 days) was observed in 20G LPCs inserted with US into the basilic vein of the arm; among Blind-PIVCs, the longest duration (3.0 ± 2.1 days) was observed in 22G SPCs inserted into the cephalic vein at the forearm. The most commonly encountered complication in both groups was accidental dislodgment. CONCLUSIONS: PIVCs with ultrasound guidance were apparently more effective than the blind ones, reducing the number of successive cannulations. Additionally, LPCs, with their greater length compared to SPCs, have proven to be more durable and may be recommended as emergency venous access in children requiring peripheral access for 4-15 days. Dislodgement should be reduced by improving the securement of the device.

Polypharmacy in Children with Medical Complexity: A Cross-Sectional Study in a Pediatric Palliative Care Center.

BACKGROUND: Children with medical complexity (CMC) often require multiple medications, leading to polypharmacy, which seems to be linked to adverse effects, administration errors, and increased caregiver burden. This study aimed to describe the prevalence of polypharmacy, medication burden, off-label drug use, and associated costs. METHODS: Conducted at the Pediatric Palliative Care Center of Padua, Italy, from August to October 2021, this cross-sectional observational study included patients up to 23 years old with at least one prescribed drug. Data were collected from medical records and caregiver interviews. Drug costs were collected from the Italian Medicine Agency. Descriptive statistical analysis was performed. For comparisons among categorical variables, the Chi-square test was used, and for those among continuous variables, the ANOVA test was used. RESULTS: This study analyzed treatment regimens of 169 patients with a median age of 12.5 years (0.3-23). Polypharmacy was present in 52.7% of patients, and medication burden was observed in 44.4%, both varying significantly by primary diagnosis (p < 0.001). The median daily cost per patient was EUR 2.2 (IQR 0.9-7.1), with significant variation among subgroups. Only 34.6% of prescriptions were off-label. CONCLUSIONS: polypharmacy and medication burden are frequent among our CMC population, with some differences according to primary diagnosis.

Dexmedetomidine at Home for Intractable Dystonia and Insomnia in Children With Special Needs: A Case Series.

BACKGROUND: We know that syndromic conditions and severe chronic diseases can be associated with symptoms that may interfere with sleep, significantly impacting the life quality of children and caregivers. Drugs commonly used in treating insomnia, such as melatonin, benzodiazepines, niaprazine, and antihistamines, are often either ineffective or associated with adverse effects, requiring new therapeutic perspectives. Dexmedetomidine is a selective alpha-2 agonist with hypnotic and anxiolytic effects, which, by stimulating alpha-2 adrenergic receptors in the locus coeruleus, induces sleep comparable to stages 2-3 of the non-REM phase without substantially affecting the respiratory drive during sedation. Its use has already been extensively described in pediatric intensive care or procedural sedation literature. In 2018, the Italian Medicines Agency (Agenzia Italiana Del Farmaco AIFA) authorized the off-label use of dexmedetomidine outside of intensive care in Children undergoing palliative treatment to control distressing symptoms related to pathology and refractory sleep disorders, and the literature reported cases of children who received dexmedetomidine at home. OBJECTIVE: Our study aims to describe the home use of dexmedetomidine in children with insomnia or intractable dystonic states. MEASURES: We conducted a retrospective analysis through a questionnaire addressed to 12 Italian pediatric palliative care centers regarding the home use of dexmedetomidine in sleep disorders and intractable dystonic states. INTERVENTION: We collected a case series of 9 children treated with dexmedetomidine at home, 8 via intranasal and 1 via intravenous route. All children received the first drug administration in the hospital or hospice during a dedicated admission, under close monitoring of vital signs parameters for 72 hours (3 days, range 2-7 days). After discharge, the potential side effects of the drug were explained to the patient's families, and, once informed consent was obtained, the home administration of dexmedetomidine continued, with follow-up by the palliative care team. At home, dexmedetomidine was administered for 3000 days (minimum 1 month, maximum 36 months). The first patient was treated for 1095 days, from 2019 to 2021 (discontinued due to underlying condition-related death). OUTCOMES: All patients observed a persistent benefit from the treatment on symptoms, and none of them discontinued dexmedetomidine administration due to drug-related adverse effects or perceived lack of therapeutic efficacy. CONCLUSIONS: Therefore, its use at home may represent a promising therapeutic approach for intractable sleep disorders or dystonic states in pediatric palliative care children. Further studies are needed to confirm our results.

The use of medical cannabis in pediatric palliative care: a case series.

BACKGROUND: Medical cannabis may be a useful tool for managing treatment-resistant epilepsy and chronic pain, which affect many patients in pediatric palliative care (PPC); however, little evidence is available in this setting. CASE PRESENTATION: We aimed to describe a clinical experience in a setting where high-level evidence may not be obtained. We report our clinical experience in a pediatric palliative care department in Italy. Caregivers reported changes in intensity and frequency of pain and epilepsy events. Six patients received a titrated plant extract of cannabis sativa for 1 year. Only mild and transient adverse events occurred: drowsiness, euphoria, restlessness and tachycardia; the resolution was either spontaneous or obtained by modifying the administration schedule. Treatment was never discontinued. No overdoses occurred. All patients experienced seizures during the pre-treatment observation period, and obtained a reduction in seizure frequency, although with variable extent while receiving cannabis. In addition, a benefit on pain was observed, based on the caregiver's evaluation, and a reduction of analgesic use. CONCLUSION: Our experience suggests that a titrated plant extract preparation of medical cannabis may be useful to control treatment-resistant pain and epilepsy in PPC patients.

Do we always need to treat patients with spinal muscular atrophy? A personal view and experience.

BACKGROUND: We report the clinical outcomes observed in our patients with SMA type 1 or 2 receiving nusinersen, and we comment on the ethical implications of this treatment, in line with our results and those reported by Audic et al. in their analysis published in the Orphanet Journal of Rare Diseases. METHODS: We analyzed records of all children with a genetically diagnosed SMA and clinically confirmed diagnosis of SMA Type 1 or 2 to whom nusinersen was offered. Follow-up lasted 30 months. RESULTS: Among the 17 children with SMA type 1, 6 interrupted treatment with nusinersen due to adverse events or lack of efficacy. Of the remaining 11 patients, 9 are responding to therapy, though multidisciplinary complex care is still required. All those children started nusinersen at a very early age. Eighteen patients with SMA type 2 received nusinersen; five required treatment interruption. The other 13 patients are still on nusinersen therapy, and 6 are responders. Among the seven non-responders, only two met the inclusion criteria of the pivotal trial. CONCLUSIONS: Our analysis further supports the findings reported in the study by Audic et al. We believe that a wider use of nusinersen in clinical practice would require a comprehensive assessment of its actual benefits weighed against the discomfort caused to patients, as well as the identification of the patients who may obtain the best benefits from this treatment.

Effect of a short training on neonatal face-mask ventilation performance in a low resource setting.

BACKGROUND: We assessed whether a short training, effective in a high resource country, was able to improve the quality of face-mask ventilation (FMV) in a low resource setting. METHODS: Local healthcare providers at the Centre Médico-Social, Kouvè, Togo were asked to ventilate a neonatal leak-free manikin before (time-t1) and after (t2) a two-minute training session. Immediately after this section, a further two-minute training with participants aware of the data monitor was offered. Finally, a third 1-minute FMV round (t3) was performed by each participant. Ventilatory parameters were recorded using a computerized system. Primary outcome was the percentage of breaths with relevant mask leak (>25%). Secondary outcomes were percentages of breaths with a low peak inspiratory pressure (PIP<20 cm H2O), within the recommended PIP (20-35 cm H2O) and with a high PIP (>35 cm H2O). RESULTS: Twenty-six subjects participated in the study. The percentage of relevant mask leak significantly decreased (p<0.0001; ß = -0.76, SE = 0.10) from 89.7% (SD 21.5%) at t1 to 45.4% (SD 27.2%) at t2 and to 18.3% (SD 20.1%) at t3. The percentage of breaths within the recommended PIP significantly increased (p<0.0001; ß = +0.54, SE = 0.12). The percentage of breaths with PIP>35 cm H2O was 19.5% (SD 32.8%) at t1 and 39.2% (SD 37.7%) at t2 (padj = 0.27; ß = +0.61, SE = 0.36) and significantly decreased (padj = 0.01; ß = -1.61, SE = 0.55) to 6.0% (SD 15.4%) at t3. CONCLUSIONS: A 2-minute training on FMV, effective in a high resource country, had a positive effect also in a low resource setting. FMV performance further improved after an extra 2-minute verbal recall plus real time feedback. Although the training was extended, it still does not cost much time and effort. Further studies are needed to establish if these basic skills are transferred in real patients and if they are maintained over time.

Use of Zoledronic Acid in Paediatric Craniofacial Fibrous Dysplasia.

We describe a case of a paediatric patient affected by mandibular fibrous dysplasia (FD) with severe and chronic pain who was successfully treated with zoledronic acid (ZOL): a third-generation bisphosphonate. Further research is needed to assess its safety and efficacy as a treatment option for FD in the paediatric population.