2021 yil avgust: Belzutifan (Welireg, Merck)Buyrak hujayrali karsinomasi (RCC), markaziy asab tizimi (CNS) gemangioblastomalari yoki oshqozon osti bezi neyroendokrin o'smalari bilan davolanishni talab qiladigan von Gippel-Lindau kasalligi bo'lgan katta yoshli bemorlar uchun oziq-ovqat va farmatsevtika idorasi tomonidan gipoksiya qo'zg'atuvchi omil inhibitori tasdiqlangan. (pNET), lekin darhol operatsiyani talab qilmaydi.
Ochiq etiketli klinik tadqiqotlar davom etayotgan 61 (NCT004) tadqiqotida Belzutifan, VHL germline o'zgarishi va kamida bitta aniqlanadigan qattiq o'simta aniqlangan, VHL bilan bog'liq RCC (VHL-RCC) bo'lgan 03401788 bemorda o'rganilgan. CNS gemangioblastomalari va pNET kabi VHL bilan bog'liq boshqa xavfli kasalliklari bo'lgan bemorlar ro'yxatga olindi. Belzutifan 120 mg bemorlarga kuniga bir marta, kasallik kuchayib ketguncha yoki chidab bo'lmas toksisitgacha berildi.
Umumiy javob darajasi (ORR) rentgenologik baholash bilan aniqlangan va RECIST v1.1 yordamida mustaqil tekshirish qo'mitasi tomonidan baholangan asosiy samaradorlik nuqtasi edi. Javobning davomiyligi va javob berish vaqti boshqa ikkita samaradorlik maqsadlari (TTR) edi. VHL bilan bog'liq RCC bo'lgan odamlarda 49% ORR (95% CI: 36, 62) topilgan. VHL-RCC bilan og'rigan barcha bemorlar, davolanish boshlanganidan keyin kamida 18 oy davomida kuzatilgan. Median DoR bajarilmadi; Respondentlarning 56 foizida 12 oydan kam bo'lgan DOR va o'rtacha 8 oylik TTR bor edi. O'lchanadigan CNS gemangioblastomasi bo'lgan 24 ta bemorda ORR 63 % ni, pNET o'lchanadigan 12 ta bemorda esa VHL bilan bog'liq bo'lmagan RCC bo'lmagan boshqa xavfli o'smalari bo'lgan bemorlarda 83 % ni tashkil etdi. CNS gemangioblastomalari va pNET uchun o'rtacha DoR bajarilmadi, bemorlarning 12 va 73 foizida javob muddati 50 oydan kam.
Reduced haemoglobin, anaemia, fatigue, increased creatinine, headache, dizziness, elevated hyperglycemia, and nausea were the most prevalent adverse effects, including laboratory abnormalities, reported in almost 20% of patients who took belzutifan. Belzutifan usage can cause severe anaemia and hypoxia. Anemia was seen in 90% of participants in Study 004, with 7% having Grade 3 anaemia. Patients should be transfused as needed by their doctors. In individuals on belzutifan, the use of erythropoiesis stimulating drugs to treat anaemia is not suggested. Hypoxia occurred in 1.6 percent of patients in Study 004. Belzutifan can make some hormonal contraceptives ineffective, and it can harm an embryo or foetus if taken during pregnancy.
Belzutifan kuniga bir marta, ovqat bilan yoki ovqatsiz, 120 mg dozada qabul qilinishi kerak.
Reference: https://www.fda.gov/
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