A subgroup analysis of JUMP, a phase IIIb, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis in a Brazilian cohort

Renato Tavares, Carmino Antonio De Souza, Carole Paley, Catherine Bouard, Ranjan Tiwari, Ricardo Pasquini, Renato Tavares, Carmino Antonio De Souza, Carole Paley, Catherine Bouard, Ranjan Tiwari, Ricardo Pasquini

Abstract

Introduction: Ruxolitinib has been approved for the treatment of myelofibrosis (MF). In this study, we present safety and efficacy findings from an analysis of 104 patients with intermediate- and high-risk MF in a Brazilian cohort of the JUMP study who received treatment with ruxolitinib.

Methods: JUMP is a single-arm, open-label, phase IIIb, expanded-access study. The primary endpoint was to evaluate the safety and tolerability (frequency, duration, and severity of adverse events [AEs]) of ruxolitinib.

Results: All of the 104 patients received the treatment. Median duration of exposure was 35.8 months. The most common hematologic AEs were anemia (57.7), thrombocytopenia (38.5%), neutropenia (11.5%), and leukopenia (9.6%). Second malignancies (all grades) occurred in 19.2% of patients (n=20). Serious AEs were reported in 62.5% of patients (n=65). The proportions of patients with ≥50% reduction from baseline in palpable spleen length at weeks 24 and 48 were 62.7% and 69.2%, respectively. The mean change from the baseline in the Functional Assessment of Cancer Therapy (FACT)-Lymphoma total score was 10.8 [15.6%] at week 4, 12.6 [14.1%] at week 24, and 12.2 [14.3%] at week 48. The mean change from the baseline for the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale was 3.9 [42.8%] at week 4, 4.9 [29.9%] at week 24, and 4.7 [28%] at week 48. At week 48, the estimated progression-free survival, leukemia-free survival, and overall survival probabilities were 91%, 91% and 93%, respectively Overall, 21 deaths were observed in the present study.

Conclusion: Findings from this study suggest that ruxolitinib could be evaluated as a standard-of-care treatment for the MF population in need of a viable treatment option. NCT01493414.

Keywords: Brazil; JUMP; Myelofibrosis; Ruxolitinib.

Copyright © 2019. Published by Elsevier Editora Ltda.

Figures

Figure 1
Figure 1
(A) Hemoglobin levels over time; (B) platelet count over time.
Figure 2
Figure 2
Evaluable patients with a decrease from baseline in palpable spleen length.
Figure 3
Figure 3
Best percent change from baseline in palpable spleen length at any time by week 48.
Figure 4
Figure 4
Proportion of patients achieving response (HRQoL) by the lower limit of MID.
https://www.ncbi.nlm.nih.gov/pmc/articles/instance/7031100/bin/mmc1.jpg
https://www.ncbi.nlm.nih.gov/pmc/articles/instance/7031100/bin/mmc2.jpg

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