de Alencar Morais Lima Waldenice, de Souza Jackson G, García-Villén Fátima, Loureiro Julia Lira, Raffin Fernanda Nervo, Fernandes Marcelo A C, Souto Eliana B, Severino Patricia, Barbosa Raquel de M
Laboratory of Galenic Pharmacy, Department of Pharmacy, Federal University of Rio Grande Do Norte, Natal, 59012-570, Brazil.
InovAI Lab, nPITI/IMD, Federal University of Rio Grande Do Norte, Natal, RN, 59078-970, Brazil.
World J Pediatr. 2025 Jan;21(1):8-28. doi: 10.1007/s12519-024-00834-x. Epub 2024 Aug 28.
Global pediatric healthcare reveals significant morbidity and mortality rates linked to respiratory, cardiac, and gastrointestinal disorders in children and newborns, mostly due to the complexity of therapeutic management in pediatrics and neonatology, owing to the lack of suitable dosage forms for these patients, often rendering them "therapeutic orphans". The development and application of pediatric drug formulations encounter numerous challenges, including physiological heterogeneity within age groups, limited profitability for the pharmaceutical industry, and ethical and clinical constraints. Many drugs are used unlicensed or off-label, posing a high risk of toxicity and reduced efficacy. Despite these circumstances, some regulatory changes are being performed, thus thrusting research innovation in this field.
Up-to-date peer-reviewed journal articles, books, government and institutional reports, data repositories and databases were used as main data sources.
Among the main strategies proposed to address the current pediatric care situation, nanotechnology is specially promising for pediatric respiratory diseases since they offer a non-invasive, versatile, tunable, site-specific drug release. Tissue engineering is in the spotlight as strategy to address pediatric cardiac diseases, together with theragnostic systems. The integration of nanotechnology and theragnostic stands poised to refine and propel nanomedicine approaches, ushering in an era of innovative and personalized drug delivery for pediatric patients. Finally, the intersection of drug repurposing and artificial intelligence tools in pediatric healthcare holds great potential. This promises not only to enhance efficiency in drug development in general, but also in the pediatric field, hopefully boosting clinical trials for this population.
Despite the long road ahead, the deepening of nanotechnology, the evolution of tissue engineering, and the combination of traditional techniques with artificial intelligence are the most recently reported strategies in the specific field of pediatric therapeutics.
全球儿科医疗保健显示,儿童和新生儿的发病率和死亡率与呼吸、心脏和胃肠道疾病密切相关,这主要是由于儿科和新生儿科治疗管理的复杂性,因为缺乏适合这些患者的剂型,常常使他们成为“治疗孤儿”。儿科药物制剂的开发和应用面临诸多挑战,包括不同年龄组的生理异质性、制药行业利润有限以及伦理和临床限制。许多药物未经许可使用或超说明书用药,存在高毒性风险和疗效降低的问题。尽管如此,一些监管变革正在进行,从而推动了该领域的研究创新。
最新的同行评审期刊文章、书籍、政府和机构报告、数据存储库和数据库被用作主要数据来源。
在为解决当前儿科护理状况而提出的主要策略中,纳米技术对儿科呼吸系统疾病特别有前景,因为它们提供了一种非侵入性、多功能、可调节、位点特异性的药物释放。组织工程作为解决儿科心脏疾病的策略以及诊疗系统受到关注。纳米技术与诊疗系统的整合有望完善和推动纳米医学方法,为儿科患者迎来创新和个性化药物递送的时代。最后,儿科医疗保健中药物再利用与人工智能工具的交叉具有巨大潜力。这不仅有望提高一般药物开发的效率,也有望提高儿科领域的效率,有望推动针对这一人群的临床试验。
尽管前路漫漫,但纳米技术的深化、组织工程的发展以及传统技术与人工智能的结合是儿科治疗特定领域最近报道的策略。