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
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: J...
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| Format: | Article |
| Language: | English |
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Elsevier
2020-01-01
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| Series: | Hematology, Transfusion and Cell Therapy |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2531137919300549 |
| _version_ | 1819051260395913216 |
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| author | Renato Tavares Carmino Antonio De Souza Carole Paley Catherine Bouard Ranjan Tiwari Ricardo Pasquini |
| author_facet | Renato Tavares Carmino Antonio De Souza Carole Paley Catherine Bouard Ranjan Tiwari Ricardo Pasquini |
| author_sort | Renato Tavares |
| collection | DOAJ |
| description | 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: Ruxolitinib, Myelofibrosis, JUMP, Brazil |
| first_indexed | 2024-12-21T12:01:07Z |
| format | Article |
| id | doaj.art-c162ce4e672442e296264e24c201fa1d |
| institution | Directory Open Access Journal |
| issn | 2531-1379 |
| language | English |
| last_indexed | 2024-12-21T12:01:07Z |
| publishDate | 2020-01-01 |
| publisher | Elsevier |
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| series | Hematology, Transfusion and Cell Therapy |
| spelling | doaj.art-c162ce4e672442e296264e24c201fa1d2022-12-21T19:04:50ZengElsevierHematology, Transfusion and Cell Therapy2531-13792020-01-014214653A subgroup analysis of JUMP, a phase IIIb, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis in a Brazilian cohortRenato Tavares0Carmino Antonio De Souza1Carole Paley2Catherine Bouard3Ranjan Tiwari4Ricardo Pasquini5Universidade Federal de Goiás (UFG), Goiânia, GO, Brazil; Corresponding author at: Universidade Federal de Goiás, Goiânia, GO 74690-900, Brazil.Universidade Estadual de Campinas (Unicamp), Campinas, SP, BrazilNovartis Oncology, East Hanover, NJ, USANovartis Pharma S.A.S., Paris, FranceNovartis Healthcare Pvt Ltd, Hyderabad, IndiaUniversidade Federal do Paraná (UFPR), Curitiba, PR, BrazilIntroduction: 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: Ruxolitinib, Myelofibrosis, JUMP, Brazilhttp://www.sciencedirect.com/science/article/pii/S2531137919300549 |
| spellingShingle | Renato Tavares Carmino Antonio De Souza Carole Paley Catherine Bouard Ranjan Tiwari Ricardo Pasquini 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 Hematology, Transfusion and Cell Therapy |
| title | 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 |
| title_full | 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 |
| title_fullStr | 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 |
| title_full_unstemmed | 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 |
| title_short | 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 |
| title_sort | 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 |
| url | http://www.sciencedirect.com/science/article/pii/S2531137919300549 |
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