Ayvakit for systemic mastocytosis – pro
AYVAKIT® (avapritinib) is indicated for the treatment of adult patients with: Unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations, and Advanced SM (AdvSM) including patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL).
However, there is no medical necessity specifically for this drug, since both it and midostaurinn are effective against placebo and have not been studies one against another. Thus, both are effective and it is not known whether one is more effective than the other.
Papayannidis C, Federico V, Fianchi L, Pregno P, Pugliese N, Romano A, Grifoni FI. Treatment of Advanced Systemic Mastocytosis with Midostaurin: Practical Guidance for Optimal Therapy and Management. Mediterr J Hematol Infect Dis. 2022 Nov 1;14(1):e2022073. doi: 10.4084/MJHID.2022.073. PMID: 36425146; PMCID: PMC9652003.
Gotlib J, Castells M, Elberink HO, Siebenhaar F, Hartmann K, Broesby-Olsen S, George TI, Panse J, Alvarez-Twose I, Radia DH, Tashi T, Bulai Livideanu C, Sabato V, Heaney M, Van Daele P, Cerquozzi S, Dybedal I, Reiter A, Pongdee T, Barete S, Ustun C, Schwartz L, Ward BR, Schafhausen P, Vadas P, Bose P, DeAngelo DJ, Rein L, Vachhani P, Triggiani M, Bonadonna P, Rafferty M, Butt NM, Oh ST, Wortmann F, Ungerstedt J, Guilarte M, Taparia M, Kuykendall AT, Arana Yi C, Ogbogu P, Gaudy-Marqueste C, Mattsson M, Shomali W, Giannetti MP, Bidollari I, Lin HM, Sulllivan E, Mar B, Scherber R, Roche M, Akin C, Maurer M. Avapritinib versus Placebo in Indolent Systemic Mastocytosis. NEJM Evid. 2023 Jun;2(6):EVIDoa2200339. doi: 10.1056/EVIDoa2200339. Epub 2023 May 23. PMID: 38320129.
DeAngelo, D.J., Radia, D.H., George, T.I. et al. Safety and efficacy of avapritinib in advanced systemic mastocytosis: the phase 1 EXPLORER trial. Nat Med 27, 2183–2191 (2021). https://doi.org/10.1038/s41591-021-01538-9