Martel Arnaud, Rocher Fanny, Gerard Alexandre
Ophthalmology Department, University Hospital of Nice, 06000 Nice, France.
Pharmacovigilance Department, University Hospital of Nice, 06000 Nice, France.
J Pers Med. 2024 Sep 26;14(10):1027. doi: 10.3390/jpm14101027.
Thyroid eye disease (TED) treatment has been recently revolutionized with the approval of teprotumumab, a targeted insulin growth factor 1 receptor (IGF1R) inhibitor. To date, teprotumumab is the only FDA-approved drug for treating TED. In this article, we would like to temper the current enthusiasm around IGF1R inhibitors. critical review of the literature by independent academic practitioners. several questions should be raised. First, "" Teprotumumab infusions are expensive, costing about USD 45,000 for one infusion and USD 360,000 for eight infusions in a 75 kg patient. Teprotumumab approval was based on two randomized clinical trials investigating active (clinical activity score ≥ 4) TED patients. Despite this, teprotumumab was approved by the FDA for "the treatment of TED" without distinguishing between active and inactive forms. The second question is as follows: "" Teprotumumab has never been compared to other medical treatments in active TED nor to surgery in chronic TED. Up to 75% of patients may experience proptosis regression after treatment discontinuation. Finally, ototoxicity has emerged as a potentially devastating side effect requiring frequent monitoring. Investigation into the long-term side effects, especially in women of childbearing age, is also warranted. Teprotumumab is undoubtedly a major treatment option in TED. However, before prescribing a drug, practitioners should assess its benefit/risk ratio based on the following: (i) evidence-based medicine; (ii) their empirical experience; (iii) the cost/benefit analysis; (iv) the long-term outcomes and safety profile.
随着靶向胰岛素生长因子1受体（IGF1R）抑制剂替普罗单抗的获批，甲状腺眼病（TED）的治疗最近发生了变革。迄今为止，替普罗单抗是唯一获得美国食品药品监督管理局（FDA）批准用于治疗TED的药物。在本文中，我们想给当前围绕IGF1R抑制剂的热情降降温。独立学术从业者对文献进行了批判性综述。应提出几个问题。首先，替普罗单抗输注费用高昂，一名75公斤的患者一次输注约花费45,000美元，八次输注则花费360,000美元。替普罗单抗的获批基于两项针对活动性（临床活动评分≥4）TED患者的随机临床试验。尽管如此，FDA在未区分活动性和非活动性形式的情况下批准替普罗单抗用于“TED的治疗”。第二个问题如下：替普罗单抗从未在活动性TED中与其他药物治疗进行比较，也未在慢性TED中与手术进行比较。高达75%的患者在停药后可能会出现眼球突出消退。最后，耳毒性已成为一种潜在的严重副作用，需要频繁监测。对长期副作用的调查，尤其是对育龄女性的调查也很有必要。替普罗单抗无疑是TED的一种主要治疗选择。然而，在开处方用药之前，从业者应基于以下几点评估其效益/风险比：（i）循证医学；（ii）他们的经验；（iii）成本效益分析；（iv）长期疗效和安全性。
