Department of Pulmonology, Second Faculty of Medicine, Charles University and Motol University Hospital, Prague, Czech Republic.
Faculty of Medicine, Masaryk University, Brno, Czech Republic.
BMC Pulm Med. 2024 May 28;24(1):260. doi: 10.1186/s12890-024-03069-8.
Physical activity is a crucial demand on cystic fibrosis treatment management. The highest value of oxygen uptake (VO) is an appropriate tool to evaluate the physical activity in these patients. However, there are several other valuable CPET parameters describing exercise tolerance (W, VO, VO VO/HR, etc.), and helping to better understand the effect of specific treatment (V, V, V/V etc.). Limited data showed ambiguous results of this improvement after CFTR modulator treatment. Elexacaftor/tezacaftor/ivacaftor medication improves pulmonary function and quality of life, whereas its effect on CPET has yet to be sufficiently demonstrated.
We performed a single group prospective observational study of 10 adolescent patients with cystic fibrosis who completed two CPET measurements between January 2019 and February 2023. During this period, elexacaftor/tezacaftor/ivacaftor treatment was initiated in all of them. The first CPET at the baseline was followed by controlled CPET at least one year after medication commencement. We focused on interpreting the data on their influence by the novel therapy. We hypothesized improvements in cardiorespiratory fitness following treatment. We applied the Wilcoxon signed-rank test. The data were adjusted for age at the time of CPET to eliminate bias of aging in adolescent patients.
We observed significant improvement in peak workload, VO, VO, VO, V/VCO slope, V, V, RQ, VO/HR peak and RR peak. The mean change in VOpeak was 5.7 mL/kg/min, or 15.9% of the reference value (SD ± 16.6; p= 0.014). VO improved by 15% of the reference value (SD ± 0.1; p= 0.014), VOimproved by 0.5 (SD ± 0.4; p= 0.01). There were no differences in other parameters.
Exercise tolerance improved after elexacaftor/tezacaftor/ivacaftor treatment initiation. We suggest that the CFTR modulator alone is not enough for recovering physical decondition, but should be supplemented with physical activity and respiratory physiotherapy. Further studies are needed to examine the effect of CFTR modulators and physical therapy on cardiopulmonary exercise tolerance.
体力活动是囊性纤维化治疗管理的重要需求。最大摄氧量（VO）是评估这些患者体力活动的合适工具。然而，还有其他一些有价值的 CPET 参数可以描述运动耐量（W、VO、VO/HR 等），并有助于更好地理解特定治疗的效果（V、V、V/V 等）。有限的数据显示，CFTR 调节剂治疗后这种改善的结果存在矛盾。依伐卡托/泰他卡托/维加特治疗可改善肺功能和生活质量，但其对 CPET 的影响尚未得到充分证明。
我们对 2019 年 1 月至 2023 年 2 月期间完成两次 CPET 测量的 10 名囊性纤维化青少年患者进行了单组前瞻性观察研究。在此期间，他们均开始接受依伐卡托/泰他卡托/维加特治疗。第一次 CPET 在基线时进行，然后在药物开始至少一年后进行对照 CPET。我们专注于解释新疗法对数据的影响。我们假设治疗后心肺适能会有所改善。我们应用了 Wilcoxon 符号秩检验。为了消除青少年患者年龄对 CPET 的偏倚，我们对 CPET 时的年龄进行了数据调整。
我们观察到峰值工作量、VO、VO、VO、V/VCO 斜率、V、V、RQ、VO/HR 峰值和 RR 峰值显著改善。VOpeak 的平均变化为 5.7ml/kg/min，或参考值的 15.9%（SD±16.6;p=0.014）。VO 提高了参考值的 15%（SD±0.1;p=0.014），VO 提高了 0.5（SD±0.4;p=0.01）。其他参数没有差异。
依伐卡托/泰他卡托/维加特治疗开始后，运动耐量提高。我们认为，CFTR 调节剂单独使用不足以恢复身体不适，还应辅以体力活动和呼吸物理治疗。需要进一步研究 CFTR 调节剂和物理治疗对心肺运动耐量的影响。
