CFTR调节剂和怀孕结果:全国性队列研究的早期发现
Laurent Chouchana1, Mathis Collier2, Clémence Martin3
1Regional Center of Pharmacovigilance, Department of perinatal, pediatric and adult pharmacology, Hopital Cochin, Hopital Necker, AP-HP. Paris, France; Université Paris Cité, INSERM UMR 1343 « Pharmacologie et évaluation des thérapeutiques chez l'enfant et la femme enceinte ». Paris, France.
概括
像elexacaftor/tezacaftor/ivacaftor这样的新CFTR调节剂在囊性纤维化患者的怀孕中似乎是安全的. 这些治疗没有显示出出生缺陷的增加,并且妊娠年龄较小的婴儿的发病率较低.
科学领域:
- 医学研究 医学研究
- 药理学 药理学是指药理学的学科.
- 生殖健康 生殖健康
背景情况:
- 囊性纤维化 (CF) 治疗的最新进展包括CFTR调节器.
- 患有CF的女性越来越多地使用这些疗法,导致该人口中更多的怀孕.
- 在怀孕期间评估CFTR调节器的安全性至关重要.
研究的目的:
- 评估CFTR调节剂,特别是elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) 的安全性,在患有囊性纤维化 (fwCF) 的孕妇中.
- 为了比较暴露和未暴露于CFTR调节器的fwCF之间的怀孕结果.
主要方法:
- 从2018年1月到2023年12月,利用法国医疗保险数据 (SNDS) 进行全国性队列研究.
- 包括在研究期间结束的单独怀孕.
- 基于处方数据的CFTR调节器暴露的定义,包括预设期.
主要成果:
- 在fwCF中的590例怀孕中,有148例 (24.7%) 暴露于CFTR调节剂,其中136例暴露于头三个月.
- 在暴露 (3.38%) 和未暴露 (4.66%) 组之间,重大出生缺陷的患病率是可比的 (p=0.72).
- 暴露的怀孕显示出显著较低的妊娠年龄 (SGA) 婴儿小的比率 (6.8%与16.1%,p<0.01).
结论:
- 早期发现表明,CFTR调节器在fwCF中对产性和不良妊娠结果具有令人放心的说法.
- 观察到的SGA率的降低可能表明,怀孕期间使用CFTR调节器可能有好处.
- 需要进一步的研究来证实这些结果,并调查产前暴露的儿童的长期发育影响.
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