Mirra Virginia, Montella Silvia, Santamaria Francesca
Department of Translational Medical Sciences, Federico II University, Via Sergio Pansini 5, 80131, Naples, Italy.
BMC Pediatr. 2018 Feb 21;18(1):73. doi: 10.1186/s12887-018-1019-9.
The primary goal of asthma management is to achieve disease control for reducing the risk of future exacerbations and progressive loss of lung function. Asthma not responding to treatment may result in significant morbidity. In many children with uncontrolled symptoms, the diagnosis of asthma may be wrong or adherence to treatment may be poor. It is then crucial to distinguish these cases from the truly "severe therapy-resistant" asthmatics by a proper filtering process. Herein we report on four cases diagnosed as difficult asthma, detail the workup that resulted in the ultimate diagnosis, and provide the process that led to the prescription of omalizumab.
All children had been initially referred because of asthma not responding to long-term treatment with high-dose inhaled steroids, long-acting β-agonists and leukotriene receptor antagonists. Definitive diagnosis was severe asthma. Three out four patients were treated with omalizumab, which improved asthma control and patients' quality of life. We reviewed the current literature on the diagnostic approach to the disease and on the comorbidities associated with difficult asthma and presented the perspectives on omalizumab treatment in children and adolescents. Based on the evidence from the literature review, we also proposed an algorithm for the diagnosis of pediatric difficult-to-treat and severe asthma.
The management of asthma is becoming much more patient-specific, as more and more is learned about the biology behind the development and progression of asthma. The addition of omalizumab, the first targeted biological treatment approved for asthma, has led to renewed optimism in the management of children and adolescents with atopic severe asthma.
哮喘管理的主要目标是实现疾病控制，以降低未来病情加重和肺功能进行性丧失的风险。治疗无反应的哮喘可能导致严重的发病率。在许多症状未得到控制的儿童中，哮喘的诊断可能有误，或者治疗依从性可能较差。因此，通过适当的筛选过程将这些病例与真正“严重治疗抵抗性”哮喘患者区分开来至关重要。在此，我们报告4例被诊断为难治性哮喘的病例，详细说明最终诊断所进行的检查，并提供导致使用奥马珠单抗处方的过程。
所有儿童最初均因哮喘对高剂量吸入性糖皮质激素、长效β受体激动剂和白三烯受体拮抗剂的长期治疗无反应而被转诊。最终诊断为重度哮喘。4例患者中有3例接受了奥马珠单抗治疗，哮喘控制情况和患者生活质量均得到改善。我们回顾了关于该疾病诊断方法以及与难治性哮喘相关的合并症的当前文献，并介绍了儿童和青少年使用奥马珠单抗治疗的观点。基于文献综述的证据，我们还提出了一种小儿难治性和重度哮喘的诊断算法。
随着对哮喘发生和发展背后生物学机制的了解越来越多，哮喘管理正变得越来越针对个体患者。首个获批用于哮喘的靶向生物治疗药物奥马珠单抗的加入，为患有特应性重度哮喘的儿童和青少年的管理带来了新的希望。
