Agustus 2021: Belzutifan (Welireg, Merck), a inhibitor faktor hypoxia-inducible, geus disatujuan ku Administrasi Pangan sarta Narkoba pikeun penderita sawawa kalayan kasakit von Hippel-Lindau anu merlukeun terapi pikeun pakait carcinoma sél renal (RCC), sistim saraf pusat (SSP) hemangioblastomas, atawa tumor neuroendocrine pancreatic. (pNET) tapi teu merlukeun bedah saharita.
Belzutifan diulik dina 61 pasién anu pakait sareng VHL RCC (VHL-RCC) didiagnosis dumasar kana parobihan germinal VHL sareng sahanteuna hiji tumor padet anu tiasa dideteksi dikurung dina ginjal dina Study 004 anu lumangsung (NCT03401788), panyilidikan klinis kabuka-labél. Pasien anu gaduh panyawat VHL anu sanés, sapertos CNS hemangioblastomas sareng pNET, didaptarkeun. Belzutifan 120 mg dipasihkeun ka pasién sakali sapoé dugi ka kamajuan panyakit atanapi karacunan anu teu kaampeuh.
Tingkat réspon sacara umum (ORR) mangrupikeun titik akhir efficacy primér, sakumaha anu ditetepkeun ku penilaian radiologis sareng ditaksir ku panitia ulasan independen nganggo RESIST v1.1. Durasi réspon (DoR) sareng waktos réspon mangrupikeun dua tujuan efficacy (TTR) anu sanés. Dina individu anu ngagaduhan RCC anu pakait sareng VHL, ORR 49% (95 persén CI: 36, 62) mendakan. Sadaya pasién sareng VHL-RCC anu ngagaduhan réspon dilacak sahenteuna 18 bulan saatos pangobatan dimimitian. DoR median henteu patepung; 56% réspondén ngagaduhan DoR kirang ti 12 bulan sareng rata-rata TTR 8 bulan. 24 pasién kalayan CNS hemangioblastomas kaukur kagungan ORR 63 persén, sareng 12 pasién kalayan pNET kaukur kagungan ORR 83 persén di pasién anu gaduh hubungan ganas VHL anu sanés RCC. Pikeun CNS hemangioblastomas sareng pNET, median DoR henteu patepung, kalayan durasi réspon kirang ti 12 bulan dina 73 persén sareng 50 persén pasién, masing-masing.
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 kedah dilaksanakeun sakali sapoé, nganggo atanapi henteu nganggo tuang, dina dosis 120 mg.
rujukan: https://www.fda.gov/
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