Abstract

Low-dose thalidomide and prednisone alone or combined are effective therapies in some persons with primary myelofibrosis (PMF) and anemia with or with RBC transfusion dependence. Danazol is also effective in some persons with PMF and anemia. Responses to these drugs are typically incomplete and not sustained. It is unclear whether adding danazol to thalidomide and prednisone would improve efficacy. We retrospectively compared the outcomes of 88 subjects with PMF and anemia receiving thalidomide and prednisone without (n = 46) or with danazol (n = 42). The primary end point was anemia response, which was 71% (95% confidence interval (CI), 57, 85%) in subjects receiving thalidomide/prednisone/danazol compared with 46% (32, 60%; P = 0.014) in those receiving thalidomide/prednisone. Response rates in subjects who were RBC transfusion dependent was also higher in the danazol cohort (61% (38, 84%)) vs. 25% (6, 44%); P = 0.024). Time to response was rapid (median, 2 months (range, 1–11 months)) and similar between the cohorts. Response duration was longer in the thalidomide/prednisone/danazol cohort (HR 2.18 (1.18–5.42); P = 0.019). Adverse effects were mild and similar between the cohorts. In conclusion, thalidomide/prednisone/danazol seems superior to thalidomide/prednisone in persons with PMF and anemia. Our conclusion requires confirmation in a randomized trial.

Details

Title
Thalidomide plus prednisone with or without danazol therapy in myelofibrosis: a retrospective analysis of incidence and durability of anemia response
Author
Luo, Xueping 1 ; Xu, Zefeng 1 ; Li, Bing 1 ; Qin, Tiejun 2 ; Zhang, Peihong 3 ; Zhang, Hongli 2 ; Fang, Liwei 2 ; Pan, Lijuan 2 ; Hu, Naibo 2 ; Qu, Shiqiang 2 ; Zhang, Yue 1 ; Huang, Gang 4 ; Gale, Robert Peter 5 ; Xiao, Zhijian 1 

 MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China; State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China 
 MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China 
 Department of Pathology, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China 
 Divisions of Experimental Hematology and Cancer Biology, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA 
 Haematology Section, Division of Experimental Medicine, Department of Medicine, Imperial College London, London, UK 
Pages
1-5
Publication year
2018
Publication date
Jan 2018
Publisher
Springer Nature B.V.
e-ISSN
20445385
Source type
Scholarly Journal
Language of publication
English
ProQuest document ID
2019484064
Copyright
© 2018. This work is published under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.