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- Title
Assessment of the efficacy and tolerability of ruxolitinib for the treatment of myelofibrosis patients in a real‐life setting: An Italian MYNERVA Project.
- Authors
Coltro, Giacomo; Sant'Antonio, Emanuela; Palumbo, Giuseppe A.; Mannelli, Francesco; De Stefano, Valerio; Ruggeri, Marco; Elli, Elena M.; Zanotti, Roberta; Borsani, Oscar; Bertozzi, Irene; Duminuco, Andrea; Betti, Silvia; Carli, Giuseppe; Cavalca, Fabrizio; Tanasi, Ilaria; Rumi, Elisa; Randi, Maria L.; Garibaldi, Bruno; Loscocco, Giuseppe G.; Guglielmelli, Paola
- Abstract
Background: Incorporating real‐world data in the drug development process allows the improvement of health outcomes by providing better representation of actual patterns of drug safety and efficacy. Aims and Methods: Here, we present the results of a retroprospective, observational real‐life study of 154 patients with myelofibrosis treated with ruxolitinib in a real‐life setting in seven Italian centers of the MYNERVA project. Results: Median drug exposure was 29 (range, 3–98) months. Discontinuation rate was 27% after a median time of 13 (range, 3–61). While hematological toxicities were in line with previous findings, infections occurred frequently, representing a not negligible cause of discontinuation and death. Anemia, symptoms, and spleen responses were obtained at any time in 23%, 91%, and 68% of patients, respectively; most patients achieved their responses by week 24. Larger splenomegaly and delayed treatment initiation correlated with lower spleen response at 24 weeks. Spleen response was associated with a superior overall survival, regardless of DIPSS. Of interest, both achievement and loss of spleen response had prognostic implications. Discussion and Conclusion: Overall, our findings provide insights on the efficacy and safety of ruxolitinib in a real‐world, multicenter cohort of Italian MF patients.
- Subjects
RUXOLITINIB; MYELOFIBROSIS; PROGNOSIS; TREATMENT delay (Medicine); DRUG efficacy
- Publication
Cancer Medicine, 2023, Vol 12, Issue 7, p8166
- ISSN
2045-7634
- Publication type
Article
- DOI
10.1002/cam4.5618