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Avapritinib is a second-generation potent and selective inhibitor of the mutant KIT D816V that, based on the results of pivotal clinical trials, was approved for the treatment of adults with AdvSM by the regulatory agencies US FDA and EMA. The present article reports the experience of treating SM patients with avapritinib in an Italian compassionate use program. The data from our case series confirm the drug as being active after multiple lines of treatment allowing rapid achievement of profound responses, making it also an effective bridging strategy to allogeneic transplant in eligible patients. However, the anticipated wider use of avapritinib in the near future will require careful monitoring of side effects, especially in heavily pretreated patients.<\/jats:p>","DOI":"10.1177\/20406207231205643","type":"journal-article","created":{"date-parts":[[2023,11,2]],"date-time":"2023-11-02T03:51:01Z","timestamp":1698897061000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["The clinical experience of compassionate use program for avapritinib: implications for drug positioning in the therapeutic scenario of systemic mastocytosis"],"prefix":"10.1177","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4810-6501","authenticated-orcid":false,"given":"Francesco","family":"Mannelli","sequence":"first","affiliation":[{"name":"SOD Ematologia, Centro Ricerca e Innovazione Malattie Mieloproliferative, AOU Careggi, Universit\u00e0 di Firenze, Largo Brambilla 3, Firenze 50134, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Francesca","family":"Crupi","sequence":"additional","affiliation":[{"name":"SOD Ematologia, Centro di Ricerca e Innovazione per le Malattie Mieloproliferative, Azienda Ospedaliero Universitaria Careggi, Firenze, Italy"},{"name":"Dipartimento di Medicina Sperimentale e Clinica, Denothe Excellence Center, Universit\u00e0 degli Studi, Firenze, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roberta","family":"Zanotti","sequence":"additional","affiliation":[{"name":"Unit\u00e0 di Ematologia, Dipartimento di Medicina, Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy"},{"name":"Gruppo Interdisciplinare per lo Studio della Mastocitosi, Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8287-928X","authenticated-orcid":false,"given":"Livio","family":"Pagano","sequence":"additional","affiliation":[{"name":"Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. 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