2021 kolovoz: Nakon neuspjeha najmanje dvije prethodne linije sistemske terapije, Uprava za hranu i lijekove odobrila je belumosudil (Rezurock, Kadmon Pharmaceuticals, LLC), inhibitor kinaze, za odrasle i pedijatrijske bolesnike od 12 godina i starije s kroničnom bolešću presatka protiv domaćina (kronični GVHD).
Za procjenu učinkovitosti korišten je KD025-213 (NCT03640481), randomizirani, otvoreni, multicentrični eksperiment s rasponom doze u kojem je 65 pacijenata s kroničnim GVHD-om liječeno belumosudilom od 200 mg oralno jednom dnevno.
The overall response rate (ORR) through Cycle 7 Day 1 was the primary efficacy end measure, with overall response defined as a full response (CR) or partial response (PR) according to the 2014 NIH Consensus Development Project on Clinical Trials in Chronic Graft-versus-Host Disease guidelines. The ORR was 75% (95 percent CI: 63, 85); 6% of patients had a complete response, and 69 percent had a partial response. The average time it took to get a first answer was 1.8 months (95 percent CI: 1.0, 1.9). The median duration of response for chronic GVHD was 1.9 months, measured from first response through progression, death, or new systemic treatments (95 percent CI: 1.2, 2.9). No mortality or new systemic medication initiation occurred in 62 percent (95 percent CI: 46, 74) of patients who achieved response for at least 12 months after response.
Infekcije, astenija, mučnina, proljev, dispneja, kašalj, edem, krvarenje, bol u trbuhu, mišićno-koštana bol, glavobolja, smanjenje fosfata, povećanje gama glutamil transferaze, smanjenje limfocita i hipertenzija bile su najčešće nuspojave (20%), uključujući laboratorijske abnormalnosti.
Belumosudil treba uzimati jednom dnevno, uz obrok, u dozi od 200 mg.
