Maglione Marco, Borrelli Melissa, Dorato Alessandro, Cimbalo Chiara, Del Giudice Luigi Antonio, Santamaria Francesca
Department of Translational Medical Sciences, Federico II University, 80131 Naples, Italy.
Pediatric Emergency Unit, "Santobono-Pausilipon" Children's Hospital, 80129 Naples, Italy.
Children (Basel). 2024 Jul 25;11(8):895. doi: 10.3390/children11080895.
Although, in most children with asthma, good symptom control is achieved with a low to moderate dose of inhaled corticosteroids, a small group of patients still experiences frequent symptoms, and even severe exacerbations, impairment of lung function, and reduced quality of life. Some of these subjects with severe asthma require biologic drugs as add-on therapy. In the past decade, numerous monoclonal antibodies have been approved for children or adolescents with severe asthma, in addition to their increasing use in adult asthma. However, the available evidence on how to select the most appropriate biologic based on a single patient's clinical, functional, and laboratory characteristics is still scant, and is insufficient to guide clinicians in the decision-making process of a personalized treatment.
We report a case series of four patients with severe eosinophilic asthma treated with mepolizumab, an anti-interleukin-5 monoclonal antibody, and review the existing literature on this treatment in children and adolescents.
Our patients, all with blood eosinophilia and elevated fractional exhaled nitric oxide levels, developed poor symptom control despite prolonged treatment with high-dose inhaled corticosteroids a second controller, addressing the addition of a biologic drug. In all of them, a 12-month treatment with subcutaneous mepolizumab showed a reduction in the blood eosinophil count and in asthma exacerbations, as well as an improvement on the Asthma Control Test. The results of the literature search focused on the strengths and limitations of the pediatric use of mepolizumab and highlighted the areas worthy of further research.
Mepolizumab has proven effective in improving symptom control in pediatric patients with severe asthma. Additional well-powered clinical trials will be helpful in developing evidence-based guidelines regarding biologic drugs in the pediatric population.
尽管在大多数哮喘儿童中，低至中等剂量的吸入性糖皮质激素可实现良好的症状控制，但仍有一小部分患者频繁出现症状，甚至发生严重发作、肺功能受损及生活质量下降。这些重度哮喘患者中的一些需要生物药物作为附加治疗。在过去十年中，除了在成人哮喘中越来越多地使用外，许多单克隆抗体已被批准用于患有重度哮喘的儿童或青少年。然而，关于如何根据单个患者的临床、功能和实验室特征选择最合适的生物制剂的现有证据仍然很少，不足以指导临床医生进行个性化治疗的决策过程。
我们报告了一组4例接受美泊利珠单抗（一种抗白细胞介素-5单克隆抗体）治疗的重度嗜酸性粒细胞性哮喘患者的病例系列，并回顾了关于该治疗在儿童和青少年中的现有文献。
我们的患者均有血嗜酸性粒细胞增多和呼出一氧化氮分数升高，尽管长期使用高剂量吸入性糖皮质激素及第二种控制药物治疗，但症状控制不佳，因此加用生物药物。在所有患者中，皮下注射美泊利珠单抗治疗12个月后，血嗜酸性粒细胞计数和哮喘发作次数减少，哮喘控制测试结果有所改善。文献检索结果聚焦于美泊利珠单抗儿科应用的优势和局限性，并突出了值得进一步研究的领域。
美泊利珠单抗已被证明可有效改善重度哮喘儿科患者的症状控制。更多有力的临床试验将有助于制定关于儿科人群生物药物的循证指南。
