Abstract
Patients with myelofibrosis at intermediate-1 risk according to the International Prognostic Score System are projected to a relatively long survival; nonetheless, they may carry significant splenomegaly and/or systemic constitutional symptoms that hamper quality of life and require treatment. Since registrative COMFORT studies included only patients at intermediate-2/high International Prognostic Score System risk, safety and efficacy data in intermediate-1 patients are limited. We report on 70 intermediate-1 patients treated with ruxolitinib according to standard clinical practice that were evaluated for response using the 2013 IWG-MRT criteria. At 6 months, rates of spleen and symptoms response were 54.7% and 80% in 64 and 65 evaluable patients, respectively. At 3 months, ruxolitinib-induced grade 3 anemia and thrombocytopenia occurred in 40.6% and 2.9% of evaluable patients, respectively. Notably, 11 (15.9%) patients experienced at least one infectious event ≥grade 2. Most (82.6%) patients were still on therapy after a median follow-up of 27 months. These data support the need for standardized guidelines that may guide the decision to initiate ruxolitinib therapy in this risk category, balancing benefit expectations and potential adverse effects.
| Original language | English |
|---|---|
| Journal | Hematological Oncology |
| DOIs | |
| Publication status | Accepted/In press - 2017 |
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Keywords
- Intermediate-1 risk
- IPSS
- MF
- Myelofibrosis
- Ruxolitinib
ASJC Scopus subject areas
- Hematology
- Oncology
- Cancer Research
Cite this
Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients : Results from an independent study. / Palandri, Francesca; Tiribelli, Mario; Benevolo, Giulia; Tieghi, Alessia; Cavazzini, Francesco; Breccia, Massimo; Bergamaschi, Micaela; Sgherza, Nicola; Polverelli, Nicola; Crugnola, Monica; Isidori, Alessandro; Binotto, Gianni; Heidel, Florian H.; Buccisano, Francesco; Martino, Bruno; Latagliata, Roberto; Spinsanti, Marco; Kallenberg, Lydia; Palumbo, Giuseppe Alberto; Abruzzese, Elisabetta; Scaffidi, Luigi; Cuneo, Antonio; Cavo, Michele; Vianelli, Nicola; Bonifacio, Massimiliano.
In: Hematological Oncology, 2017.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients
T2 - Results from an independent study
AU - Palandri, Francesca
AU - Tiribelli, Mario
AU - Benevolo, Giulia
AU - Tieghi, Alessia
AU - Cavazzini, Francesco
AU - Breccia, Massimo
AU - Bergamaschi, Micaela
AU - Sgherza, Nicola
AU - Polverelli, Nicola
AU - Crugnola, Monica
AU - Isidori, Alessandro
AU - Binotto, Gianni
AU - Heidel, Florian H.
AU - Buccisano, Francesco
AU - Martino, Bruno
AU - Latagliata, Roberto
AU - Spinsanti, Marco
AU - Kallenberg, Lydia
AU - Palumbo, Giuseppe Alberto
AU - Abruzzese, Elisabetta
AU - Scaffidi, Luigi
AU - Cuneo, Antonio
AU - Cavo, Michele
AU - Vianelli, Nicola
AU - Bonifacio, Massimiliano
PY - 2017
Y1 - 2017
N2 - Patients with myelofibrosis at intermediate-1 risk according to the International Prognostic Score System are projected to a relatively long survival; nonetheless, they may carry significant splenomegaly and/or systemic constitutional symptoms that hamper quality of life and require treatment. Since registrative COMFORT studies included only patients at intermediate-2/high International Prognostic Score System risk, safety and efficacy data in intermediate-1 patients are limited. We report on 70 intermediate-1 patients treated with ruxolitinib according to standard clinical practice that were evaluated for response using the 2013 IWG-MRT criteria. At 6 months, rates of spleen and symptoms response were 54.7% and 80% in 64 and 65 evaluable patients, respectively. At 3 months, ruxolitinib-induced grade 3 anemia and thrombocytopenia occurred in 40.6% and 2.9% of evaluable patients, respectively. Notably, 11 (15.9%) patients experienced at least one infectious event ≥grade 2. Most (82.6%) patients were still on therapy after a median follow-up of 27 months. These data support the need for standardized guidelines that may guide the decision to initiate ruxolitinib therapy in this risk category, balancing benefit expectations and potential adverse effects.
AB - Patients with myelofibrosis at intermediate-1 risk according to the International Prognostic Score System are projected to a relatively long survival; nonetheless, they may carry significant splenomegaly and/or systemic constitutional symptoms that hamper quality of life and require treatment. Since registrative COMFORT studies included only patients at intermediate-2/high International Prognostic Score System risk, safety and efficacy data in intermediate-1 patients are limited. We report on 70 intermediate-1 patients treated with ruxolitinib according to standard clinical practice that were evaluated for response using the 2013 IWG-MRT criteria. At 6 months, rates of spleen and symptoms response were 54.7% and 80% in 64 and 65 evaluable patients, respectively. At 3 months, ruxolitinib-induced grade 3 anemia and thrombocytopenia occurred in 40.6% and 2.9% of evaluable patients, respectively. Notably, 11 (15.9%) patients experienced at least one infectious event ≥grade 2. Most (82.6%) patients were still on therapy after a median follow-up of 27 months. These data support the need for standardized guidelines that may guide the decision to initiate ruxolitinib therapy in this risk category, balancing benefit expectations and potential adverse effects.
KW - Intermediate-1 risk
KW - IPSS
KW - MF
KW - Myelofibrosis
KW - Ruxolitinib
UR - http://www.scopus.com/inward/record.url?scp=85019261019&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85019261019&partnerID=8YFLogxK
U2 - 10.1002/hon.2429
DO - 10.1002/hon.2429
M3 - Article
AN - SCOPUS:85019261019
JO - Hematological Oncology
JF - Hematological Oncology
SN - 0278-0232
ER -