RESUMO
BACKGROUND: Antenatal betamethasone is recommended before preterm delivery to accelerate fetal lung maturation. However, its optimal dose remains unknown. A 50% dose reduction was proposed to decrease the potential dose-related long-term neurodevelopmental side effects, including psychological development, sleep, and emotional disorders. Because noninferiority of the half dose in terms of the need for exogenous surfactant was not shown in the primary analysis, its impact on survival without major neonatal morbidity needs to be investigated. OBJECTIVE: This study aimed to investigate the impact of antenatal betamethasone dose reduction on survival of very preterm infants without severe neonatal morbidity, a factor known to have a strong correlation with long-term outcomes. STUDY DESIGN: We performed a post hoc secondary analysis of a randomized, multicenter, double-blind, placebo-controlled, noninferiority trial, testing half (11.4 mg once; n=1620) vs full (11.4 mg twice, 24 hours apart; n=1624) antenatal betamethasone doses in women at risk of preterm delivery. To measure survival without severe neonatal morbidity at hospital discharge among neonates born before 32 weeks of gestation, we used the definition of the French national prospective study on preterm children, EPIPAGE 2, comprising 1 of the following morbidities: grade 3 to 4 intraventricular hemorrhage, cystic periventricular leukomalacia, necrotizing enterocolitis stage ≥2, retinopathy of prematurity requiring anti-vascular endothelial growth factor therapy or laser, and moderate-to-severe bronchopulmonary dysplasia. RESULTS: After exclusion of women who withdrew consent or had pregnancy termination and of participants lost to follow-up (8 in the half-dose and 10 in the full-dose group), the rate of survival without severe neonatal morbidity among neonates born before 32 weeks of gestation was 300 of 451 (66.5%) and 304 of 462 (65.8%) in the half-dose and full-dose group, respectively (risk difference, +0.7%; 95% confidence interval, -5.6 to +7.1). There were no significant between-group differences in the cumulative number of neonatal morbidities. Results were similar when using 2 other internationally recognized definitions of severe neonatal morbidity and when considering the overall population recruited in the trial. CONCLUSION: In the BETADOSE trial, severe morbidity at discharge of newborns delivered before 32 weeks of gestation was found to be similar among those exposed to 11.4-mg and 22.8-mg antenatal betamethasone. Additional studies are needed to confirm these findings.
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BACKGROUND: Early detection of pulmonary exacerbations (PEx) in patients with cystic fibrosis is important to quickly trigger treatment and reduce respiratory damage. An intervention was designed in the frame of the MucoExocet research study providing patients with cystic fibrosis with connected devices and educating them to detect and react to their early signs of PEx. OBJECTIVE: This study aims to identify the contributions and conditions of home monitoring in relation to their care teams from the users' point of view to detect PEx early and treat it. This study focused on the patients' experiences as the first and main users of home monitoring. METHODS: A qualitative study was conducted to explore patients' and professionals' experiences with the intervention. We interviewed patients who completed the 2-year study using semistructured guides and conducted focus groups with the care teams. All the interviews were recorded and transcribed verbatim. Their educational material was collected. A grounded analysis was conducted by 2 researchers. RESULTS: A total of 20 patients completed the study. Three main categories emerged from the patients' verbatim transcripts and were also found in those of the professionals: (1) task technology fit, reflecting reliability, ease of use, accuracy of data, and support of the technology; (2) patient empowerment through technology, grouping patients' learnings, validation of their perception of exacerbation, assessment of treatment efficacy, awareness of healthy behaviors, and ability to react to PEx signs in relation to their care team; (3) use, reflecting a continuous or intermittent use, the perceived usefulness balanced with cumbersome measurements, routinization and personalization of the measurement process, and the way data are shared with the care team. Furthermore, 3 relationships were highlighted between the categories that reflect the necessary conditions for patient empowerment through the use of technology. CONCLUSIONS: We discuss a theorization of the process of patient empowerment through the use of connected devices and call for further research to verify or amend it in the context of other technologies, illnesses, and care organizations. TRIAL REGISTRATION: ClinicalTrials.gov NCT03304028; https://clinicaltrials.gov/ct2/show/results/NCT03304028.
RESUMO
Children deserve to be treated with appropriate medicines based on robust assessments. Despite the introduction of new regulations, the availability of medicines for children is suboptimal because of the frequent lack of relevant clinical trials due to the difficulty of conducting such trials. Thus, the Transparency Committee (TC) of the French National Authority for Health, who oversees the assessment of medicinal products in France, set up a pediatric working group with two aims: (1) The first aim was to review all opinions on medicines for pediatric use. Out of 536 opinions delivered between 2020 and 2022, 181 (34 %) concerned medicines for pediatric use. Whereas oncology largely dominated the medicines for adults, medicines for infectious diseases, endocrinology/metabolism, neurology, and hematology mostly prevailed for children. (2) The second aim was to clarify the evaluation criteria assessed by the TC, namely, the clinical benefit (CB), the clinical added value (CAV), and the public health impact (PHI) for pediatric medicinal products. An important CB was given to 113 out of 161 (71 %) opinions on medicines for pediatric use when it concerned pathologies with a severe prognosis. The quality of the demonstration (e.g., double-blind randomized trial vs. placebo or another active medicine) played a major role in the CB level. Clinical pediatric studies were also consistently associated with higher CAV levels: levels I (major) to III (moderate) in 26 out of 42 (62 %) opinions, level IV (minor) and level V (no therapeutic progress) in 43 out of 84 (51 %) and 30 out of 43 (70 %) opinions granting a sufficient CB, respectively. Conversely, 22 out of 30 (73 %) dossiers based only on literature reviews were given a level V. The main criteria leading to the qualification of a medicine for pediatric use as providing a PHI included a significant change in the morbidity and mortality of the disease and an improvement in the care pathway. Assessments were mostly aligned on the adults in the case of subsequent extensions of indications to children. Lastly, new measures were taken aimed at shortening median delays in the assessment process in order to reduce off-label use of medicines in France.