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8 results on '"Barraco, D."'

7. Gender effect on phenotype and genotype in patients with post-polycythemia vera and post-essential thrombocythemia myelofibrosis: results from the MYSEC project.

Author

Barraco D, Mora B, Guglielmelli P, Rumi E, Maffioli M, Rambaldi A, Caramella M, Komrokji R, Gotlib J, Kiladjian JJ, Cervantes F, Devos T, Palandri F, De Stefano V, Ruggeri M, Silver RT, Benevolo G, Albano F, Merli M, Pietra D, Barbui T, Rotunno G, Cazzola M, Giorgino T, Vannucchi AM, and Passamonti F

Subjects
Adult, Aged, Aged, 80 and over, Biomarkers, Female, Genetic Association Studies, Genetic Predisposition to Disease, Humans, Male, Middle Aged, Polycythemia Vera mortality, Primary Myelofibrosis mortality, Prognosis, Severity of Illness Index, Sex Factors, Thrombocythemia, Essential mortality, Genotype, Phenotype, Polycythemia Vera diagnosis, Polycythemia Vera genetics, Primary Myelofibrosis diagnosis, Primary Myelofibrosis genetics, Thrombocythemia, Essential diagnosis, Thrombocythemia, Essential genetics
Published
2018
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8. Momelotinib therapy for myelofibrosis: a 7-year follow-up.

Author

Tefferi A, Barraco D, Lasho TL, Shah S, Begna KH, Al-Kali A, Hogan WJ, Litzow MR, Hanson CA, Ketterling RP, Gangat N, and Pardanani A

Subjects
Aged, Benzamides adverse effects, Disease-Free Survival, Female, Follow-Up Studies, Humans, Janus Kinase 2 genetics, Male, Middle Aged, Mutation, Primary Myelofibrosis genetics, Pyrimidines adverse effects, Survival Rate, Benzamides administration & dosage, Primary Myelofibrosis drug therapy, Primary Myelofibrosis mortality, Pyrimidines administration & dosage
Abstract

One-hundred Mayo Clinic patients with high/intermediate-risk myelofibrosis (MF) received momelotinib (MMB; JAK1/2 inhibitor) between 2009 and 2010, as part of a phase 1/2 trial (NCT00935987); 73% harbored JAK2 mutations, 16% CALR, 7% MPL, 44% ASXL1, and 18% SRSF2. As of July 2017, MMB was discontinued in 91% of the patients, after a median treatment duration of 1.4 years. Grade 3/4 toxicity included thrombocytopenia (34%) and liver/pancreatic test abnormalities (<10%); grade 1/2 peripheral neuropathy occurred in 47%. Clinical improvement (CI) occurred in 57% of patients, including 44% anemia and 43% spleen response. CI was more likely to occur in ASXL1-unmutated patients (66% vs 44%) and in those with <2% circulating blasts (66% vs 42%). Response was more durable in the presence of CALR type 1/like and absence of very high-risk karyotype. In multivariable analysis, absence of CALR type 1/like (HR 3.0; 95% CI 1.2-7.6) and presence of ASXL1 (HR 1.9; 95% CI 1.1-3.2) or SRSF2 (HR 2.4, 95% CI 1.3-4.5) mutations adversely affected survival. SRSF2 mutations (HR 4.7, 95% CI 1.3-16.9), very high-risk karyotype (HR 7.9, 95% CI 1.9-32.1), and circulating blasts ≥2% (HR 3.9, 95% CI 1.4-11.0) predicted leukemic transformation. Post-MMB survival (median 3.2 years) was not significantly different than that of a risk-matched MF cohort not receiving MMB.

Published
2018
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