Ruxolitinib combined with prednisone, thalidomide and danazol for treatment of myelofibrosis: a pilot study

• Published in: Zhōnghuá xuèyèxué zázhì Vol. 40; no. 1; p. 24
• Main Authors: Xu, Z F, Qin, T J, Zhang, H L, Fang, L W, Pan, L J, Hu, N B, Qu, S Q, Li, B, Xiao, Z J
• Format: Journal Article
• Language: Chinese
• Published: China 14.01.2019
• Subjects: Danazol; Drug Combinations; Humans; Nitriles; Pilot Projects; Prednisone; Primary Myelofibrosis - drug therapy; Pyrazoles - therapeutic use; Pyrimidines; Thalidomide - therapeutic use; Treatment Outcome; Ruxolitinib; Prednisone; Primary myelofibrosis; Thalidomide; Danazol
• ISSN: 0253-2727
• DOI: 10.3760/cma.j.issn.0253-2727.2019.01.005

To evaluate the efficacy and tolerability of ruxolitinib combined with prednisone, thalidomide and danazol for treatment of in myelofibrosis (MF). Patients of MF according to the WHO 2016 criteria, received ruxolitinib (RUX) combined with prednisone, thalidomide and danazol (PTD). The response, changes of blood counts and adverse events were evaluated. Six PMF and one post-ET MF patients were enrolled. Four patients presented JAK2V617F mutation, one CALR mutation, one MPL mutation, one triple-negative. Responses per IWG-MRT criteria were clinical improvement in 5 patients, stable disease in 2 ones, spleen response in 6 ones. All of 7 patients were symptomatic responses, four patients achieved at least 50% improvement from baseline on MPN-SAF TSS. Three patients initially treated with RUX alone, all of 3 patients experienced treatment-associated anemia and thrombocytopenia. Then these 3 patients received RUX combined with PTD, both hemoglobin and platelet increased significantly. Four patients initially treate