PMID- 35609279 OWN - NLM STAT- MEDLINE DCOM- 20221007 LR - 20221011 IS - 1099-1069 (Electronic) IS - 0278-0232 (Linking) VI - 40 IP - 4 DP - 2022 Oct TI - Ruxolitinib combined with prednisone, thalidomide and danazol in patients with myelofibrosis: Results of a pilot study. PG - 787-795 LID - 10.1002/hon.3026 [doi] AB - Ruxolitinib is a safe and effective therapy of myeloproliferative neoplasm-associated (MPN) myelofibrosis. However, often there are dose reductions and/or therapy interruptions because of therapy-related adverse events (AEs), especially anemia and thrombocytopenia. We previously reported combined therapy with prednisone, thalidomide and danazol (PTD) reversed anemia and thrombocytopenia in people with MPN-associated myelofibrosis. We wondered whether adding PTD to ruxolitinib might mitigate the hematologic AEs and thereby avoid the dose reduction of ruxolitinib and improve the efficacy. To test this hypothesis, we conducted a baseline hemoglobin and platelet concentration assignment prospective observational study in 72 patients comparing 3-month dose adjustment and efficacy of ruxolitinib with (N = 53, the study group) or without (N = 19, the control group) PTD. According to the platelet counts, the median daily ruxolitinib doses in the study group increased from 30 to 40 mg by week 12, whereas in the control group it remained at 30 mg (p = 0.019). In the study group 35 patients had a hemoglobin increase >/=10 g/L compared with no patient receiving ruxolitinib only (p < 0.001). Platelet increases >100 x 10E+9/L were seen in 56.6% and 5.3% of patients in the two groups, respectively (p < 0.001). In patients with anemia and thrombocytopenia, 18 patients in the study group had an anemia response at week 12 and 12 had a platelet increase of >/=50 x 10E+9/L. No patient in the control group achieved either response (p < 0.001 and p = 0.078). The study group had a more spleen response than the control group (p = 0.046). Peripheral edema and transaminase elevation were the main nonhematologic AEs of PTD. These AEs can be alleviated by adjusting the danazol dose. In conclusion, adding PTD to ruxolitinib improved ruxolitinib-associated anemia and thrombocytopenia, and resulted in a higher ruxolitinib dose. CI - (c) 2022 John Wiley & Sons Ltd. FAU - Qu, Shiqiang AU - Qu S AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Xu, Zefeng AU - Xu Z AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Qin, Tiejun AU - Qin T AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Li, Bing AU - Li B AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Pan, Lijuan AU - Pan L AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Chen, Jia AU - Chen J AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Yan, Xin AU - Yan X AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Wu, Junying AU - Wu J AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Zhang, Yudi AU - Zhang Y AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Zhang, Peihong AU - Zhang P AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - Hematologic Pathology Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. FAU - Gale, Robert Peter AU - Gale RP AD - Department of Immunology and Inflammation, Haematology Research Centre, Imperial College London, London, UK. FAU - Xiao, Zhijian AU - Xiao Z AUID- ORCID: 0000-0002-1099-4489 AD - State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - MDS and MPN Centre, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. AD - Hematologic Pathology Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China. LA - eng GR - 81530008/National Natural Science Funds/ GR - 81870104/National Natural Science Funds/ GR - 82070134/National Natural Science Funds/ GR - 18JCZDJC34900/Tianjin Natural Science Funds/ GR - 16JCQNJC11400/Tianjin Natural Science Funds/ GR - 19JCQNJC09400/Tianjin Natural Science Funds/ GR - 2020-I2M-C&T-A-020/CAMS Initiative Fund for Medical Sciences/ GR - 2020-I2M-C&T-B-090/CAMS Initiative Fund for Medical Sciences/ PT - Journal Article PT - Observational Study DEP - 20220528 PL - England TA - Hematol Oncol JT - Hematological oncology JID - 8307268 RN - 0 (Hemoglobins) RN - 0 (Nitriles) RN - 0 (Pyrazoles) RN - 0 (Pyrimidines) RN - 4Z8R6ORS6L (Thalidomide) RN - 82S8X8XX8H (ruxolitinib) RN - EC 2.6.1.- (Transaminases) RN - N29QWW3BUO (Danazol) RN - VB0R961HZT (Prednisone) SB - IM MH - *Anemia/chemically induced/drug therapy MH - Danazol/therapeutic use MH - Hemoglobins/therapeutic use MH - Humans MH - *Myeloproliferative Disorders/drug therapy MH - Nitriles MH - Pilot Projects MH - Prednisone/therapeutic use MH - *Primary Myelofibrosis/drug therapy/etiology MH - Pyrazoles MH - Pyrimidines MH - Thalidomide MH - *Thrombocytopenia/chemically induced/drug therapy MH - Transaminases/therapeutic use MH - Treatment Outcome OTO - NOTNLM OT - combination therapy OT - danazol OT - myelofibrosis OT - prednisone OT - ruxolitinib OT - thalidomide EDAT- 2022/05/25 06:00 MHDA- 2022/10/12 06:00 CRDT- 2022/05/24 17:02 PHST- 2022/04/26 00:00 [revised] PHST- 2022/01/29 00:00 [received] PHST- 2022/05/21 00:00 [accepted] PHST- 2022/05/25 06:00 [pubmed] PHST- 2022/10/12 06:00 [medline] PHST- 2022/05/24 17:02 [entrez] AID - 10.1002/hon.3026 [doi] PST - ppublish SO - Hematol Oncol. 2022 Oct;40(4):787-795. doi: 10.1002/hon.3026. Epub 2022 May 28.