We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Ruxolitinib rechallenge in resistant or intolerant patients with myelofibrosis: Frequency, therapeutic effects, and impact on outcome.
- Authors
Palandri, Francesca; Tiribelli, Mario; Breccia, Massimo; Bartoletti, Daniela; Elli, Elena M.; Benevolo, Giulia; Martino, Bruno; Cavazzini, Francesco; Tieghi, Alessia; Iurlo, Alessandra; Abruzzese, Elisabetta; Pugliese, Novella; Binotto, Gianni; Caocci, Giovanni; Auteri, Giuseppe; Cattaneo, Daniele; Trawinska, Malgorzata M.; Stella, Rossella; Scaffidi, Luigi; Polverelli, Nicola
- Abstract
BACKGROUND: After ruxolitinib discontinuation, the outcome of patients with myelofibrosis (MF) is poor with scarce therapeutic possibilities. METHODS: The authors performed a subanalysis of an observational, retrospective study (RUX‐MF) that included 703 MF patients treated with ruxolitinib to investigate 1) the frequency and reasons for ruxolitinib rechallenge, 2) its therapeutic effects, and 3) its impact on overall survival. RESULTS: A total of 219 patients (31.2%) discontinued ruxolitinib for ≥14 days and survived for ≥30 days. In 60 patients (27.4%), ruxolitinib was rechallenged for ≥14 days (RUX‐again patients), whereas 159 patients (72.6%) discontinued it permanently (RUX‐stop patients). The baseline characteristics of the 2 cohorts were comparable, but discontinuation due to a lack/loss of spleen response was lower in RUX‐again patients (P =.004). In comparison with the disease status at the first ruxolitinib stop, at its restart, there was a significant increase in patients with large splenomegaly (P <.001) and a high Total Symptom Score (TSS; P <.001). During the rechallenge, 44.6% and 48.3% of the patients had spleen and symptom improvements, respectively, with a significant increase in the number of patients with a TSS reduction (P =.01). Although the use of a ruxolitinib dose > 10 mg twice daily predicted better spleen (P =.05) and symptom improvements (P =.02), the reasons for/duration of ruxolitinib discontinuation and the use of other therapies before rechallenge were not associated with rechallenge efficacy. At 1 and 2 years, 33.3% and 48.3% of RUX‐again patients, respectively, had permanently discontinued ruxolitinib. The median overall survival was 27.9 months, and it was significantly longer for RUX‐again patients (P =.004). CONCLUSIONS: Ruxolitinib rechallenge was mainly used in intolerant patients; there were clinical improvements and a possible survival advantage in many cases, but there was a substantial rate of permanent discontinuation. Ruxolitinib rechallenge should be balanced against newer therapeutic possibilities. This observational, retrospective study investigates the frequency and reasons for ruxolitinib rechallenge, its therapeutic effects, and its impact on overall survival in a cohort of 219 patients with myelofibrosis discontinuing ruxolitinib for ≥14 days and surviving for ≥30 days. In comparison with 159 patients discontinuing ruxolitinib permanently, discontinuation due to a lack/loss of spleen response is lower (P =.004) in 60 patients in whom ruxolitinib is rechallenged for ≥14 days (RUX‐again patients): there is a significant increase in the number of patients with large splenomegaly and a high Total Symptom Score (P <.001) between the first ruxolitinib stop and restart, and there is a significant increase in the number of patients with a Total Symptom Score reduction (P =.01) during the rechallenge. The use of a ruxolitinib dose >10 mg twice daily is associated with spleen improvements (P =.05) and symptom improvements (P =.02), and overall survival is significantly longer in RUX‐again patients (P =.004).
- Subjects
RUXOLITINIB; OVERALL survival; SURVIVAL rate; TREATMENT effectiveness; SPLEEN; MYELOFIBROSIS
- Publication
Cancer (0008543X), 2021, Vol 127, Issue 15, p2657
- ISSN
0008-543X
- Publication type
Article
- DOI
10.1002/cncr.33541