Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis.

doi:10.1182/blood-2011-01-330563

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Article

Safety and efficacy of everolimus, a mTOR inhibitor, as single agent in a phase 1/2 study in patients with myelofibrosis.

Blood - 25 Aug 2011

Guglielmelli Paola, Barosi Giovanni, Rambaldi Alessandro, Marchioli Roberto, Masciulli Arianna, Tozzi Lorenzo, Biamonte Flavia, Bartalucci Niccolò, Gattoni Elisabetta, Lupo Maria Letizia, Finazzi Guido, Pancrazzi Alessandro, Antonioli Elisabetta, Susini Maria Chiara, Pieri Lisa, Malevolti Elisa, Usala Emilio, Occhini Ubaldo, Grossi Alberto, Caglio Silvia, Paratore Simona, Bosi Alberto, Barbui Tiziano, Vannucchi Alessandro M

Abstract excerpt

In addition to dysregulated JAK/STAT signaling, activation of the AKT/mTOR pathway occurs in myelofibrosis, a myeloproliferative neoplasm with no approved therapies. We conducted a phase 1/2 study with everolimus, an mTOR inhibitor, in 39 high- or intermediate-risk primary or postpolycythemia vera/postessential thrombocythemia myelofibrosis subjects. Responses were evaluated in 30 patients of phase 2. No...

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PMID: 21725052
DOI: 10.1182/blood-2011-01-330563
PMCID: PMC3365876

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