August 2021: Etter svikt i minst to tidligere linjer med systemisk terapi, godkjente Food and Drug Administration belumosudil (Rezurock, Kadmon Pharmaceuticals, LLC), en kinasehemmer, for voksne og pediatriske pasienter 12 år og eldre med kronisk graft-versus-host-sykdom (kronisk GVHD).
KD025-213 (NCT03640481), et randomisert, åpent, multisenter-dosevarierende eksperiment der 65 pasienter med kronisk GVHD ble behandlet med belumosudil 200 mg administrert oralt én gang daglig, ble brukt for å vurdere effekt.
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.
Infeksjoner, asteni, kvalme, diaré, dyspné, hoste, ødem, blødninger, magesmerter, muskel- og skjelettsmerter, hodepine, redusert fosfat, økt gamma-glutamyltransferase, redusert lymfocytter og hypertensjon var de vanligste bivirkningene (20 %), inkludert laboratoriereaksjoner (XNUMX %) abnormiteter.
Belumosudil bør tas en gang daglig, sammen med mat, i en dose på 200 mg.
