There is a substantial research gap regarding analgesic interventions for children and adolescents with chronic pain. Most clinical trials in the field focus on the evaluation of non-pharmacological interventions and are of low methodological quality. There is also a specific lack of trials involving infants and children and adolescents with long-lasting diseases.

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.

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.

BackgroundWe 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.MethodsLocal 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).ResultsTwenty-six subjects participated in the study. The percentage of relevant mask leak significantly decreased (p<0.0001; β = -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; β = +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; β = +0.61, SE = 0.36) and significantly decreased (padj = 0.01; β = -1.61, SE = 0.55) to 6.0% (SD 15.4%) at t3.ConclusionsA 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.