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Observational Study
. 2022 Mar 22;6(6):1855-1864.
doi: 10.1182/bloodadvances.2021006889.

A prognostic model to predict survival after 6 months of ruxolitinib in patients with myelofibrosis

Margherita Maffioli  1 , Barbara Mora  1   2 , Somedeb Ball  3 , Alessandra Iurlo  4 , Elena Maria Elli  5 , Maria Chiara Finazzi  6 , Nicola Polverelli  7 , Elisa Rumi  8   9 , Marianna Caramella  10 , Maria Cristina Carraro  11 , Mariella D'Adda  12 , Alfredo Molteni  13 , Cinzia Sissa  14 , Francesca Lunghi  15 , Alessandro Vismara  16 , Marta Ubezio  17 , Anna Guidetti  18 , Sabrina Caberlon  19 , Michela Anghilieri  20 , Rami Komrokji  3 , Daniele Cattaneo  4   6 , Matteo Giovanni Della Porta  17   21 , Toni Giorgino  22 , Lorenza Bertù  23 , Marco Brociner  1 , Andrew Kuykendall  3 , Francesco Passamonti  1   2
Affiliations
Observational Study

A prognostic model to predict survival after 6 months of ruxolitinib in patients with myelofibrosis

Margherita Maffioli et al. Blood Adv. .

Abstract

Ruxolitinib (RUX) is extensively used in myelofibrosis (MF). Despite its early efficacy, most patients lose response over time and, after discontinuation, have a worse overall survival (OS). Currently, response criteria able to predict OS in RUX-treated patients are lacking, leading to uncertainty regarding the switch to second-line treatments. In this study, we investigated predictors of survival collected after 6 months of RUX in 209 MF patients participating in the real-world ambispective observational RUXOREL-MF study (NCT03959371). Multivariable analysis identified the following risk factors: (1) RUX dose <20 mg twice daily at baseline, months 3 and 6 (hazard ratio [HR], 1.79; 95% confidence interval [CI], 1.07-3.00; P = .03), (2) palpable spleen length reduction from baseline ≤30% at months 3 and 6 (HR, 2.26; 95% CI, 1.40-3.65; P = .0009), (3) red blood cell (RBC) transfusion need at months 3 and/or 6 (HR, 1.66; 95% CI, 0.95-2.88; P = .07), and (4) RBC transfusion need at all time points (ie, baseline and months 3 and 6; HR, 2.32; 95% CI, 1.19-4.54; P = .02). Hence, we developed a prognostic model, named Response to Ruxolitinib After 6 Months (RR6), dissecting 3 risk categories: low (median OS, not reached), intermediate (median OS, 61 months; 95% CI, 43-80), and high (median OS, 33 months; 95% CI, 21-50). The RR6 model was validated and confirmed in an external cohort comprised of 40 MF patients. In conclusion, the RR6 prognostic model allows for the early identification of RUX-treated MF patients with impaired survival who might benefit from a prompt treatment shift.

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Figures

Figure 1.
Figure 1.
Patient disposition flowchart (training cohort). Patient flowchart reporting the total number of patients included in the RUXOREL-MF database and the number of patients excluded from the analysis with the corresponding motivations, in line with selection criteria. IPSS, International Prognostic Scoring System; LR, low risk; RUX, ruxolitinib.
Figure 2.
Figure 2.
Actuarial survival curves of the 3 risk groups of patients according to the Response to Ruxolitinib After 6 Months (RR6) model developed in RUX-treated MF patients (training cohort).
See this image and copyright information in PMC

References

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