Tosco Antonella, Cerchione Raffaele, Gelzo Monica, Cimbalo Chiara, Castaldo Alice, Terracciano Rosamaria, Raia Valeria, Sepe Angela
Paediatric Unit, Cystic Fibrosis Regional Reference Center, Department of Maternal and Child Health, University Hospital Federico II, 80131 Naples, Italy.
Paediatric Unit, Cystic Fibrosis Regional Reference Center, Department of Translational Medical Sciences, University of Naples Federico II, 80131 Naples, Italy.
J Clin Med. 2025 Jul 9;14(14):4856. doi: 10.3390/jcm14144856.
An increase in hemoglobin (Hb) has been reported in subjects with CF treated with the CFTR modulator Ivacaftor and with the combination Lumacaftor/Ivacaftor (LI), while the literature about the impact of Elexacaftor/Tezacaftor/Ivacaftor (ETI) on Hb levels in the pediatric population is lacking. We retrospectively evaluated Hb levels in 35 subjects with CF (18 males, median age: 8 years; interquartile range (IQR): 6-13 years) treated with LI and 60 (24 males, median age: 10 years; IQR: 6-14 years) treated with ETI. For each subject we considered the values of Hb, serum potassium, total bilirubin (TB), and conjugated bilirubin (CB) at baseline, after 3 days, and 1, 3, 6, 9, and 12 months from the start of treatment. In subjects with CF treated with LI, we observed a significant increase in Hb values 3 days after the introduction of the drug, which remained constant throughout the year of treatment. In subjects treated with ETI, a significant decrease in Hb was observed 3 days after the first dose up to 1 month. At 6 months, Hb returned to pre-treatment values remaining stable for up to 12 months. At 3 days of treatment, we also observed a significant increase in serum potassium, which returned to normal at one month, while both TB and CB values significantly increased at 3 days of treatment and remained significantly higher for the whole one-year period of ETI therapy. We confirmed an increase in Hb values over time in subjects treated with LI. While the Hb response in those treated with ETI showed a transient reduction that lasted for one month, this may have depended on hemolysis, and returned to pre-treatment levels. Further studies will clarify the mechanisms that govern changes in Hb in subjects with CF treated with ETI.
据报道，接受囊性纤维化跨膜传导调节因子（CFTR）调节剂依伐卡托以及鲁马卡托/依伐卡托组合（LI）治疗的囊性纤维化（CF）患者血红蛋白（Hb）水平升高，而关于依列卡托/替扎卡托/依伐卡托（ETI）对儿科人群Hb水平影响的文献尚缺。我们回顾性评估了35例接受LI治疗的CF患者（18例男性，中位年龄：8岁；四分位间距（IQR）：6 - 13岁）和60例接受ETI治疗的CF患者（24例男性，中位年龄：10岁；IQR：6 - 14岁）的Hb水平。对于每例患者，我们记录了治疗开始时、3天后以及治疗开始后1、3、6、9和12个月时的Hb、血清钾、总胆红素（TB）和结合胆红素（CB）值。在接受LI治疗的CF患者中，我们观察到用药3天后Hb值显著升高，且在整个治疗年中保持稳定。在接受ETI治疗的患者中，首次用药3天后至1个月时Hb显著下降。6个月时，Hb恢复到治疗前水平并保持稳定长达12个月。治疗3天时，我们还观察到血清钾显著升高，1个月时恢复正常，而TB和CB值在治疗3天时均显著升高，且在ETI治疗的整个1年期内均显著高于治疗前。我们证实接受LI治疗的患者Hb值随时间增加。而接受ETI治疗的患者Hb反应出现了持续1个月的短暂下降，这可能与溶血有关，随后恢复到治疗前水平。进一步的研究将阐明ETI治疗CF患者时Hb变化的机制。
