Ruxolitinib rechallenge in resistant or intolerant patients with myelofibrosis: Frequency, therapeutic effects, and impact on outcome

Caocci, Giovanni;
2021-01-01

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.
2021
Inglese
127
15
2657
2665
9
Esperti anonimi
scientifica
Cancer; Myelofibrosis; Outcome; Rechallenge; Ruxolitinib
Palandri, Francesca; Tiribelli, Mario; Breccia, Massimo; Bartoletti, Daniela; Elli, Elena M; Benevolo, Giulia; Martino, Bruno; Cavazzini, Francesco; T ...espandi
1.1 Articolo in rivista
info:eu-repo/semantics/article
1 Contributo su Rivista::1.1 Articolo in rivista
262
35
open
Files in This Item:
File Size Format  
Ruxolitinib Rechallenge in Resistant or Intolerant Patients With Myelofibrosis- Frequency, Therapeutic Effects, and Impact on Outcome.pdf

Open Access from 02/08/2022

Type: versione post-print
Size 259.17 kB
Format Adobe PDF
259.17 kB Adobe PDF View/Open

Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.

Questionnaire and social

Share on:
Impostazioni cookie