Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial
Erythropoiesis-stimulating agents (ESAs) achieve hematological improvement-erythroid (HIE) in only 30% of ESA-naïve lower risk myelodysplastic syndrome (LR-MDS) patients with anemia, highlighting the need for developing novel drugs or new treatment strategies to improve the outcome of these patient...
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Ferrata Storti Foundation
2025-06-01
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| Series: | Haematologica |
| Online Access: | https://haematologica.org/article/view/12102 |
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| author | Chen Mei Gaixiang Xu Cuiping Zheng Yaping Xie Minming Li Yanping Shao Rongxin Yao Shi Tao Wei Jiang Jun Guo Zhiyin Zheng Wei Wang Xinping Zhou Liya Ma Li Ye Yingwan Luo Chunmei Yang Wenjuan Yu Wanzhuo Xie Jie Jin Hongyan Tong |
| author_facet | Chen Mei Gaixiang Xu Cuiping Zheng Yaping Xie Minming Li Yanping Shao Rongxin Yao Shi Tao Wei Jiang Jun Guo Zhiyin Zheng Wei Wang Xinping Zhou Liya Ma Li Ye Yingwan Luo Chunmei Yang Wenjuan Yu Wanzhuo Xie Jie Jin Hongyan Tong |
| author_sort | Chen Mei |
| collection | DOAJ |
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Erythropoiesis-stimulating agents (ESAs) achieve hematological improvement-erythroid (HIE) in only 30% of ESA-naïve lower risk myelodysplastic syndrome (LR-MDS) patients with anemia, highlighting the need for developing novel drugs or new treatment strategies to improve the outcome of these patients. We conducted this multicenter, single-arm trial to investigate the efficacy and safety of a triple regimen consisting of recombinant human erythropoietin (rhEPO), all-trans retinoic acid (ATRA) and testosterone undecanoate in patients with anemia due to lower-risk MDS based on Revised International Prognostic Scoring System. Eligible patients received rhEPO 10000 IU/day, oral ATRA 25 mg/m2/day and oral testosterone undecanoate 80 mg twice daily for 12 weeks. The primary endpoint was the proportion of patients achieving HI-E during 12 weeks of treatment. Of 52 eligible patients, 32 (61.5%, 95%CI 48.0%-73.5%) achieved HI-E, meeting the primary endpoint. Fifteen patients (65.2% [15/23]) with baseline serum erythropoietin ≤500 IU/L had HI-E versus 58.6% of those (17/29) with baseline serum erythropoietin >500 IU/L. More patients with very low or low risk had HI-E than those with intermediate risk (73.3% vs. 45.5%, P = 0.041) and fewer patients with mutated ASXL1 had HI-E than those with wildtype ASXL1 (33.3% vs. 70.0%, P = 0.040). The regimen had an acceptable safety profile compatible with individual agents. In conclusion, the triple regimen of rhEPO combined with ATRA and testosterone undecanoate attained HI-E in approximately 61.5% of patients regardless of baseline serum EPO levels, supporting further development of this regimen for LR-MDS patients with anemia. This study was registered at CHICTR.ORG.CN as ChiCTR2000032845.
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| format | Article |
| id | doaj-art-ccc4dc6fea754fba9b505b8288b47a14 |
| institution | OA Journals |
| issn | 0390-6078 1592-8721 |
| language | English |
| publishDate | 2025-06-01 |
| publisher | Ferrata Storti Foundation |
| record_format | Article |
| series | Haematologica |
| spelling | doaj-art-ccc4dc6fea754fba9b505b8288b47a142025-08-20T02:32:42ZengFerrata Storti FoundationHaematologica0390-60781592-87212025-06-01999110.3324/haematol.2024.287055Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trialChen Mei0Gaixiang Xu1Cuiping Zheng2Yaping Xie3Minming Li4Yanping Shao5Rongxin Yao6Shi Tao7Wei Jiang8Jun Guo9Zhiyin Zheng10Wei Wang11Xinping Zhou12Liya Ma13Li Ye14Yingwan Luo15Chunmei Yang16Wenjuan Yu17Wanzhuo Xie18Jie Jin19Hongyan Tong20Myelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangDepartment of hematology, Wenzhou Central Hospital, Wenzhou, ZhejiangDepartment of Hematology, Hangzhou First People's Hospital, Hangzhou, ZhejiangDepartment of Hematology, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University, Guangzhou, GuangdongDepartment of Hematology, Taizhou Hospital of Zhejiang Province Affiliated with Wenzhou Medical University, Taizhou, ZhejiangDepartment of Hematology, The Second Affiliated Hospital of Wenzhou Medical University, Wenzhou, ZhejiangDepartment of Hematology, Shaoxing Second Hospital, Shaoxing, ZhejiangDepartment of Hematology, Shangyu People's Hospital of Shaoxing, Shaoxing, ZhejiangDepartment of Hematology, Changxing County People's Hospital, Huzhou, ZhejiangDepartment of Haematology, The First Affiliated Hospital of Zhejiang Chinese Medical University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangDepartment of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangDepartment of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangDepartment of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangDepartment of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, ZhejiangMyelodysplastic Syndromes Diagnosis and Therapy Center, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China; Zhejiang Provincial Key Laboratory of Hematopoietic Malignancy, Zhejiang University, Hangzhou, Zhejiang, China; Cancer Center, Zhejiang University, Hangzhou, Zhejiang Erythropoiesis-stimulating agents (ESAs) achieve hematological improvement-erythroid (HIE) in only 30% of ESA-naïve lower risk myelodysplastic syndrome (LR-MDS) patients with anemia, highlighting the need for developing novel drugs or new treatment strategies to improve the outcome of these patients. We conducted this multicenter, single-arm trial to investigate the efficacy and safety of a triple regimen consisting of recombinant human erythropoietin (rhEPO), all-trans retinoic acid (ATRA) and testosterone undecanoate in patients with anemia due to lower-risk MDS based on Revised International Prognostic Scoring System. Eligible patients received rhEPO 10000 IU/day, oral ATRA 25 mg/m2/day and oral testosterone undecanoate 80 mg twice daily for 12 weeks. The primary endpoint was the proportion of patients achieving HI-E during 12 weeks of treatment. Of 52 eligible patients, 32 (61.5%, 95%CI 48.0%-73.5%) achieved HI-E, meeting the primary endpoint. Fifteen patients (65.2% [15/23]) with baseline serum erythropoietin ≤500 IU/L had HI-E versus 58.6% of those (17/29) with baseline serum erythropoietin >500 IU/L. More patients with very low or low risk had HI-E than those with intermediate risk (73.3% vs. 45.5%, P = 0.041) and fewer patients with mutated ASXL1 had HI-E than those with wildtype ASXL1 (33.3% vs. 70.0%, P = 0.040). The regimen had an acceptable safety profile compatible with individual agents. In conclusion, the triple regimen of rhEPO combined with ATRA and testosterone undecanoate attained HI-E in approximately 61.5% of patients regardless of baseline serum EPO levels, supporting further development of this regimen for LR-MDS patients with anemia. This study was registered at CHICTR.ORG.CN as ChiCTR2000032845. https://haematologica.org/article/view/12102 |
| spellingShingle | Chen Mei Gaixiang Xu Cuiping Zheng Yaping Xie Minming Li Yanping Shao Rongxin Yao Shi Tao Wei Jiang Jun Guo Zhiyin Zheng Wei Wang Xinping Zhou Liya Ma Li Ye Yingwan Luo Chunmei Yang Wenjuan Yu Wanzhuo Xie Jie Jin Hongyan Tong Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial Haematologica |
| title | Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial |
| title_full | Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial |
| title_fullStr | Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial |
| title_full_unstemmed | Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial |
| title_short | Recombinant human erythropoietin plus all-trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower-risk myelodysplastic syndromes: a multicenter, single-arm, prospective trial |
| title_sort | recombinant human erythropoietin plus all trans retinoic acid and testosterone undecanoate for the treatment of anemia in patients with lower risk myelodysplastic syndromes a multicenter single arm prospective trial |
| url | https://haematologica.org/article/view/12102 |
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