The wide use of ruxolitinib, approved for treating primary and secondary myelofibrosis (MF), has revolutionized the landscape of these diseases. This molecule can reduce spleen volume and constitutional symptoms, guaranteeing patients a better quality of life and survival or even a valid bridge to bone marrow transplantation. Despite a rapid response within the first 3 to 6 months of treatment, some patients fail to achieve a significant benefit or lose early response. After ruxolitinib failure, new drugs are available to provide an additional therapeutic option for these patients. However, the correct timing point for deciding on a therapy shift is still an open challenge. Recently, a clinical prognostic score named RR6 (Response to Ruxolitinib after 6 months) was proposed to determine survival after 6 months of treatment with ruxolitinib in patients affected by MF. We applied this model to a cohort of consecutive patients treated at our center to validate the results obtained in terms of median overall survival (mOS): for the low-risk class, mOS was not reached (as in the training cohort); for the intermediate-risk, mOS was 52 months (95% CI 39-106); for the high-risk, it was 33 (95% 8.5-59). Moreover, in addition to the other studies present in the literature, we evaluated how the new RR6 score could better identify primary MF patients at high risk, with a slight or no agreement compared to DIPSS, contrary to what occurs in secondary MF. Thus, we were able to confirm the predictive power of the RR6 model in our series, which might be of help in guiding future therapeutic choices.

Prediction of Survival and Prognosis Migration from Gold-Standard Scores in Myelofibrosis Patients Treated with Ruxolitinib Applying the RR6 Prognostic Model in a Monocentric Real-Life Setting

Duminuco, Andrea
Primo
;
Nardo, Antonella;Garibaldi, Bruno;Vetro, Calogero;Longo, Anna;Giallongo, Cesarina;Di Raimondo, Francesco;Palumbo, Giuseppe A
Ultimo
2022-01-01

Abstract

The wide use of ruxolitinib, approved for treating primary and secondary myelofibrosis (MF), has revolutionized the landscape of these diseases. This molecule can reduce spleen volume and constitutional symptoms, guaranteeing patients a better quality of life and survival or even a valid bridge to bone marrow transplantation. Despite a rapid response within the first 3 to 6 months of treatment, some patients fail to achieve a significant benefit or lose early response. After ruxolitinib failure, new drugs are available to provide an additional therapeutic option for these patients. However, the correct timing point for deciding on a therapy shift is still an open challenge. Recently, a clinical prognostic score named RR6 (Response to Ruxolitinib after 6 months) was proposed to determine survival after 6 months of treatment with ruxolitinib in patients affected by MF. We applied this model to a cohort of consecutive patients treated at our center to validate the results obtained in terms of median overall survival (mOS): for the low-risk class, mOS was not reached (as in the training cohort); for the intermediate-risk, mOS was 52 months (95% CI 39-106); for the high-risk, it was 33 (95% 8.5-59). Moreover, in addition to the other studies present in the literature, we evaluated how the new RR6 score could better identify primary MF patients at high risk, with a slight or no agreement compared to DIPSS, contrary to what occurs in secondary MF. Thus, we were able to confirm the predictive power of the RR6 model in our series, which might be of help in guiding future therapeutic choices.
2022
RR6 prognostic score
model validation
myelofibrosis
overall survival
ruxolitinib
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/20.500.11769/548107
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