Department of Respiratory Medicine (Adult Cystic Fibrosis Unit), Vall d'Hebron University Hospital, Vall d'Hebron Research Institute (VHIR), Universitat Autònoma de Barcelona (UAB), Barcelona, Spain.
CIBER de Enfermedades Respiratorias (CIBERES), Instituto de Salud Carlos III, Madrid, Spain.
Eur Respir Rev. 2024 Oct 30;33(174). doi: 10.1183/16000617.0071-2024. Print 2024 Oct.
The prognosis of people with cystic fibrosis (pwCF) has improved dramatically with the introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators (CFTRm). The ageing of the cystic fibrosis (CF) population is changing the disease landscape with the emergence of different needs and increasing comorbidities related to both age and long-term exposure to multiple treatments including CFTRm. Although the number of pwCF eligible for this treatment is expected to increase, major disparities in care and outcomes still exist in this population. Moreover, the long-term impact of the use of CFTRm is still partly unknown due to the current short follow-up and experience with their use, thus generating some uncertainties. The future spread and initiation of these drugs at an earlier stage of the disease is expected to reduce the systemic burden of systemic inflammation and its consequences on health. However, the prolonged life expectancy is accompanied by an increasing burden of age-related comorbidities, especially in the context of chronic disease. The clinical manifestations of the comorbidities directly or indirectly associated with CFTR dysfunction are changing, along with the disease dynamics and outcomes. Current protocols used to monitor slow disease progression will need continuous updates, including the composition of the multidisciplinary team for CF care, with a greater focus on the needs of the adult population.
囊性纤维化跨膜电导调节因子（CFTR）调节剂（CFTRm）的引入极大地改善了囊性纤维化（CF）患者的预后。随着 CF 人群的老龄化，与年龄和长期暴露于包括 CFTRm 在内的多种治疗相关的不同需求和日益增加的合并症不断出现，改变了疾病的格局。尽管有资格接受这种治疗的 CF 患者人数预计会增加，但该人群的护理和结局仍存在明显差异。此外，由于目前的短期随访和使用经验，CFTRm 的长期影响仍部分未知，因此存在一些不确定性。预计这些药物在疾病早期的广泛使用和启动将减少全身性炎症及其对健康影响的全身负担。然而，预期寿命的延长伴随着与年龄相关的合并症负担的增加，尤其是在慢性疾病的背景下。与 CFTR 功能障碍直接或间接相关的合并症的临床表现正在发生变化，与疾病动态和结局一起变化。目前用于监测疾病缓慢进展的方案需要不断更新，包括 CF 护理的多学科团队的组成，更加关注成年人群体的需求。
