A Prognostic Model to Predict Ruxolitinib Discontinuation and Death in Patients with Myelofibrosis
Abstract
:Simple Summary
Abstract
1. Introduction
2. Materials and Methods
2.1. Study Setting and Definitions
2.2. Ethical Aspects
2.3. Statistical Analysis
3. Results
3.1. Study Cohort
3.2. Outcome According to Timing of Ruxolitinib Discontinuation
3.3. The Short-Term Ruxolitinib Prognostic Model (STR-PM)
3.4. Expanding the STR-PM to Earlier Timepoints
4. Discussion
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Characteristics | Total Study Cohort (n.410) | |||||
---|---|---|---|---|---|---|
Follow-Up ≥ 5 Years | Follow-Up < 5 Years | |||||
Long-Term on RUX (LTR) n = 172 (42%) | Short-Term on RUX (STR) n = 115 (28%) | p | Early Death on RUX (EDR) n = 123 (30%) | p (LTR vs. EDR) | p (STR vs. EDR) | |
Age, median (range) Age ≥ 65, n. (%) | 65.9 (26.5–85.8) 94 (54.7%) | 65.4 (39.9–86.1) 59 (51.3%) | 0.34 0.58 | 73.7 (38.4–86.3) 108 (87.8%) | <0.001 <0.001 | <0.001 <0.001 |
Male sex, n. (%) | 86 (50%) | 60 (52.2%) | 0.72 | 84 (68.3%) | 0.002 | 0.01 |
Disease-specific risk score, n. (%) Int-1 Int-2 High | 120 (69.8%) 44 (25.6%) 8 (4.6%) | 70 (60.9%) 40 (34.8%) 5 (4.3%) | 0.27 | 34 (27.4%) 62 (50.8%) 26 (21.3%) | <0.001 | <0.001 |
PMF, n. (%) | 74 (43%) | 66 (57.4%) | 0.02 | 68 (55.3%) | 0.04 | 0.74 |
WBC, median (range) WBC > 25 × 109/L, n. (%) | 11.2 (2.7–70) 26 (15.1%) | 10.4 (2.2–80) 13 (11.3%) | 0.16 0.34 | 13.4 (2.2–92.3) 27 (22%) | 0.05 0.13 | 0.007 0.03 |
PLT, median (range) PLT < 100 × 109/L, n. (%) | 291 (32–1425) 6 (3.5%) | 257 (60–1084) 11 (10%) | 0.02 0.03 | 250.5 (25–1887) 14 (11.5%) | 0.12 0.007 | 0.54 0.63 |
Hb, median (range) Hb < 10 g/dL, n. (%) | 12.2 (7–16.8) 35 (20.4%) | 10.6 (5.7–16.3) 44 (38.3%) | <0.001 <0.001 | 9.9 (5–16.7) 65 (53.3%) | <0.001 <0.001 | 0.006 0.02 |
Transfusion dependence, n (%) | 17 (10.6%) | 44 (38.3%) | 0.003 | 41/118 (34.8%) | <0.001 | 0.05 |
Blasts, median (range) Blasts ≥ 1%, n. (%) | 0 (0–10) 53 (31%) | 0 (0–6) 44 (38.9%) | 0.48 0.17 | 0 (0–10) 47/121 (38.8%) | 0.25 0.16 | 0.64 0.99 |
Palpable spleen, median (range) Spleen ≥ 10 cm, n. (%) | 10 (0–35) 84 (48.8%) | 11 (0–31) 59 (51.3%) | 0.12 0.58 | 10 (0–31) 60 (49.2%) | 0.57 0.95 | 0.27 0.64 |
TSS, median (range) TSS ≥ 20, n. (%) | 20 (0–70) 83/165 (50.3%) | 20 (0–90) 69/110 (62.7%) | 0.005 0.04 | 23 (0–77) 83/115 (72.2%) | <0.001 <0.001 | 0.25 0.13 |
Median years from MF diagnosis (range), Time from MF diagnosis >2 year | 0.80 (0–22.35) 63 (36.6%) | 2 (0–31.7) 57 (49.6%) | 0.01 0.03 | 1 (0–18.2) 50 (40.7%) | 0.32 0.48 | 0.09 0.17 |
Ruxolitinib starting dose, n. (%) <10 mg BID | 15 (8.7%) | 29 (25.2%) | <0.001 | 25 (20.3%) | 0.004 | 0.37 |
Spleen response, % on evaluable At 3 months At 6 months | 35.2% 39.5% | 24.2% 32.6% | 0.07 0.29 | 18.4% 17.5% | 0.003 <0.001 | 0.05 0.02 |
Symptoms response, % on evaluable At 3 months At 6 months | 72.1% 75.8% | 61.0% 72.8% | 0.06 0.6 | 53.3% 61.5% | 0.002 0.02 | 0.26 0.10 |
Treatment-emergent anemia, % on evaluable At 3 months At 6 months | 64.2% 49% | 67.8% 49.4% | 0.58 0.96 | 53.8% 33.9% | 0.13 0.04 | 0.07 0.06 |
Treatment-emergent thrombocytopenia, % on evaluable At 3 months At 6 months | 20.6% 28.7% | 23.1% 44% | 0.63 0.008 | 29.7% 39.2% | 0.08 0.07 | 0.27 0.48 |
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Palandri, F.; Palumbo, G.A.; Bonifacio, M.; Elli, E.M.; Tiribelli, M.; Auteri, G.; Trawinska, M.M.; Polverelli, N.; Benevolo, G.; Tieghi, A.; et al. A Prognostic Model to Predict Ruxolitinib Discontinuation and Death in Patients with Myelofibrosis. Cancers 2023, 15, 5027. https://doi.org/10.3390/cancers15205027
Palandri F, Palumbo GA, Bonifacio M, Elli EM, Tiribelli M, Auteri G, Trawinska MM, Polverelli N, Benevolo G, Tieghi A, et al. A Prognostic Model to Predict Ruxolitinib Discontinuation and Death in Patients with Myelofibrosis. Cancers. 2023; 15(20):5027. https://doi.org/10.3390/cancers15205027
Chicago/Turabian StylePalandri, Francesca, Giuseppe A. Palumbo, Massimiliano Bonifacio, Elena M. Elli, Mario Tiribelli, Giuseppe Auteri, Malgorzata M. Trawinska, Nicola Polverelli, Giulia Benevolo, Alessia Tieghi, and et al. 2023. "A Prognostic Model to Predict Ruxolitinib Discontinuation and Death in Patients with Myelofibrosis" Cancers 15, no. 20: 5027. https://doi.org/10.3390/cancers15205027