Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients: Results from an independent study

Francesca Palandri, Mario Tiribelli, Giulia Benevolo, Alessia Tieghi, Francesco Cavazzini, Massimo Breccia, Micaela Bergamaschi, Nicola Sgherza, Nicola Polverelli, Monica Crugnola, Alessandro Isidori, Gianni Binotto, Florian H. Heidel, Francesco Buccisano, Bruno Martino, Roberto Latagliata, Marco Spinsanti, Lydia Kallenberg, Giuseppe Alberto Palumbo, Elisabetta AbruzzeseLuigi Scaffidi, Antonio Cuneo, Michele Cavo, Nicola Vianelli, Massimiliano Bonifacio

Research output: Contribution to journalArticle

6 Citations (Scopus)

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 languageEnglish
JournalHematological Oncology
DOIs
Publication statusAccepted/In press - 2017

Fingerprint

Primary Myelofibrosis
Safety
INCB018424
Splenomegaly
Anemia
Therapeutics
Spleen
Quality of Life
Guidelines
Survival

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 journalArticle

Palandri, F, Tiribelli, M, Benevolo, G, Tieghi, A, Cavazzini, F, Breccia, M, Bergamaschi, M, Sgherza, N, Polverelli, N, Crugnola, M, Isidori, A, Binotto, G, Heidel, FH, Buccisano, F, Martino, B, Latagliata, R, Spinsanti, M, Kallenberg, L, Palumbo, GA, Abruzzese, E, Scaffidi, L, Cuneo, A, Cavo, M, Vianelli, N & Bonifacio, M 2017, 'Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients: Results from an independent study', Hematological Oncology. https://doi.org/10.1002/hon.2429
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. / Efficacy and safety of ruxolitinib in intermediate-1 IPSS risk myelofibrosis patients : Results from an independent study. In: Hematological Oncology. 2017.
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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.",
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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

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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.

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KW - Ruxolitinib

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