Haploidentical hematopoietic stem cell transplantation for beta-thalassemia: a meta-analysis_Chinese Journal of Evidence-Based Medicine

Authors：

YUAN Jiaqi , MING Jian , QIAO Jingyi , WENG Junling , LUO Bingxing , YAN Juntao ,  CHEN Yingyao

School of Public Health, National Health Commission Key Laboratory of Health Technology Assessment, Fudan University, Shanghai 200032, P. R. China;

Corresponding author：

CHEN Yingyao, Email: yychen@shmu.edu.cn

Keywords：

Beta-thalassemia; Haploidentical hematopoietic stem cell transplantation; Survival; Safety; Meta-analysis

DOI：

10.7507/1672-2531.202209083

Video：

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Objective To systematically review the survival outcome and safety of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for β-thalassemia. Methods The PubMed, EMbase, CNKI, WanFang Data and CBM databases were electronically searched to collect studies on haplo-HSCT for β-thalassemia from January 1, 2017 to December 31, 2021. Two reviewers independently screened the literature, extracted data and assessed the risk of bias of the included studies. Meta-analysis was then performed by using RevMan 5.4.1 software and Stata 16.0 software. Results A total of 6 case-series studies involving 286 patients were included. The results of meta-analysis indicated that overall survival (OS) and thalassemia-free survival (TFS) for β-thalassemia patients undergoing haplo-HSCT were 92.5% (95%CI 86.1% to 96.1%) and 88.5% (95%CI 74.6% to 95.3%), the incidence of Ⅲ-Ⅳ degree acute graft versus host disease (Ⅲ-Ⅳ aGvHD) and chronic graft versus host disease (cGvHD) were 11.5% (95%CI 6.5% to 20.0%) and 23.1% (95%CI 12.3% to 39.8%), and the transplantation related mortality was 6.5% (95%CI 3.8% to 10.7%). Conclusion Relevant clinical studies published in the past 5 years provide the latest information and progress of haplo-HSCT for β-thalassemia. At present, great efficacy has been shown in NF-14-TM therapeutic regimen, but the long-term efficacy remains unclear. Due to the limited quality and quantity of the included studies, more high-quality evidence from long-term comparative studies is still needed.

Citation： YUAN Jiaqi, MING Jian, QIAO Jingyi, WENG Junling, LUO Bingxing, YAN Juntao, CHEN Yingyao. Haploidentical hematopoietic stem cell transplantation for beta-thalassemia: a meta-analysis. Chinese Journal of Evidence-Based Medicine, 2023, 23(5): 544-548. doi: 10.7507/1672-2531.202209083 Copy

1.	中华医学会医学遗传学分会遗传病临床实践指南撰写组. β-地中海贫血的临床实践指南. 中华医学遗传学杂志, 2020, 37(3): 243-251.
2.	中华医学会儿科学分会血液学组, 《中华儿科杂志》编辑委员会. 重型β地中海贫血的诊断和治疗指南(2017年版). 中华儿科杂志, 2018, 56(10): 724-729.
3.	Oikonomopoulou C, Goussetis E. HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe. Bone Marrow Transplant, 2021, 56(12): 2882-2888.
4.	王丽, 王三斌, 方建培, 等. 造血干细胞移植治疗重型β地中海贫血儿科专家共识. 中国实用儿科杂志, 2018, 33(12): 935-939.
5.	Li Q, Luo J, Zhang Z, et al. G-CSF-mobilized blood and bone marrow grafts as the source of stem cells for HLA-identical sibling transplantation in patients with thalassemia major. Biol Blood Marrow Transplant, 2019, 25(10): 2040-2044.
6.	He Y, Jiang H, Li C, et al. Long-term results of the NF-08-TM protocol in stem cell transplant for patients with thalassemia major: a multi-center large-sample study. Am J Hematol, 2020, 95(11): E297-E299.297-299.
7.	Munn Z, Barker TH, Moola S, et al. Methodological quality of case series studies: an introduction to the JBI critical appraisal tool. JBI Evid Synth, 2020, 18(10): 2127-2133.
8.	陈月红, 杜亮, 耿兴远, 等. 无对照二分类数据的Meta分析在RevMan软件中的实现. 中国循证医学杂志, 2014, 14(7): 889-896.
9.	徐倚琪. 地中海贫血儿童造血干细胞移植后生存率及并发症的年龄分析. 广州: 南方医科大学, 2019.
10.	肖玉华. 不同来源外周血干细胞改善脐血干细胞治疗地中海贫血预后及相关因素分析. 广州: 南方医科大学, 2020.
11.	Anurathapan U, Hongeng S, Pakakasama S, et al. Hematopoietic stem cell transplantation for severe thalassemia patients from haploidentical donors using a novel conditioning regimen. Biol Blood Marrow Transplant, 2020, 26(6): 1106-1112.
12.	Marwah P, Soni R, Ramprakash S, et al. The impact of Host vs. Graft mismatches on rejection of haploidentical bone marrow transplants in thalassemia patients using posttransplant cyclophosphamide. Bone Marrow Transplant, 2020, 55(9): 1824-1828.
13.	Wang X, Zhang X, Yu U, et al. Co-transplantation of haploidentical stem cells and a dose of unrelated cord blood in pediatric patients with thalassemia major. Cell Transplant, 2021, 30: 963689721994808.
14.	Vellaichamy Swaminathan V, Ravichandran N, Ramanan KM, et al. Augmented immunosuppression and PTCY-based haploidentical hematopoietic stem cell transplantation for thalassemia major. Pediatr Transplant, 2021, 25(2): e13893.
15.	Gaziev J, Isgrò A, Sodani P, et al. Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ+/CD19+-depleted grafts. Blood Adv, 2018, 2(3): 263-270.
16.	Farmakis D, Porter J, Taher A, et al. 2021 Thalassaemia international federation guidelines for the management of transfusion-dependent thalassemia. Hemasphere, 2022, 6(8): e732.
17.	施玲玲. 比较同胞脐血联合骨髓与外周血联合骨髓移植治疗重型β地贫的疗效和免疫重建. 南宁: 广西医科大学, 2015.
18.	Inati A, Kahale M, Sbeiti N, et al. One-year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation. Pediatr Blood Cancer, 2017, 64(1): 188-196.
19.	Angelucci E, Pilo F. Management of iron overload before, during, and after hematopoietic stem cell transplantation for thalassemia major. Ann N Y Acad Sci, 2016, 1368(1): 115-121.
20.	Caocci G, Orofino MG, Vacca A, et al. Long-term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment. Am J Hematol, 2017, 92(12): 1303-1310.
21.	Santarone S, Pepe A, Meloni A, et al. Secondary solid cancer following hematopoietic cell transplantation in patients with thalassemia major. Bone Marrow Transplant, 2018, 53(1): 39-43.
22.	王鹏, 陈月, 张海英. 治疗成人β-地中海贫血新药: luspatercept. 中国新药与临床杂志, 2021, 40(8): 568-571.
23.	Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med, 2020, 382(13): 1219-1231.
24.	Thompson AA, Walters MC, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med, 2018, 378(16): 1479-1493.
25.	Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med, 2021, 384(3): 252-260.

1. 中华医学会医学遗传学分会遗传病临床实践指南撰写组. β-地中海贫血的临床实践指南. 中华医学遗传学杂志, 2020, 37(3): 243-251.
2. 中华医学会儿科学分会血液学组, 《中华儿科杂志》编辑委员会. 重型β地中海贫血的诊断和治疗指南(2017年版). 中华儿科杂志, 2018, 56(10): 724-729.
3. Oikonomopoulou C, Goussetis E. HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe. Bone Marrow Transplant, 2021, 56(12): 2882-2888.
4. 王丽, 王三斌, 方建培, 等. 造血干细胞移植治疗重型β地中海贫血儿科专家共识. 中国实用儿科杂志, 2018, 33(12): 935-939.
5. Li Q, Luo J, Zhang Z, et al. G-CSF-mobilized blood and bone marrow grafts as the source of stem cells for HLA-identical sibling transplantation in patients with thalassemia major. Biol Blood Marrow Transplant, 2019, 25(10): 2040-2044.
6. He Y, Jiang H, Li C, et al. Long-term results of the NF-08-TM protocol in stem cell transplant for patients with thalassemia major: a multi-center large-sample study. Am J Hematol, 2020, 95(11): E297-E299.297-299.
7. Munn Z, Barker TH, Moola S, et al. Methodological quality of case series studies: an introduction to the JBI critical appraisal tool. JBI Evid Synth, 2020, 18(10): 2127-2133.
8. 陈月红, 杜亮, 耿兴远, 等. 无对照二分类数据的Meta分析在RevMan软件中的实现. 中国循证医学杂志, 2014, 14(7): 889-896.
9. 徐倚琪. 地中海贫血儿童造血干细胞移植后生存率及并发症的年龄分析. 广州: 南方医科大学, 2019.
10. 肖玉华. 不同来源外周血干细胞改善脐血干细胞治疗地中海贫血预后及相关因素分析. 广州: 南方医科大学, 2020.
11. Anurathapan U, Hongeng S, Pakakasama S, et al. Hematopoietic stem cell transplantation for severe thalassemia patients from haploidentical donors using a novel conditioning regimen. Biol Blood Marrow Transplant, 2020, 26(6): 1106-1112.
12. Marwah P, Soni R, Ramprakash S, et al. The impact of Host vs. Graft mismatches on rejection of haploidentical bone marrow transplants in thalassemia patients using posttransplant cyclophosphamide. Bone Marrow Transplant, 2020, 55(9): 1824-1828.
13. Wang X, Zhang X, Yu U, et al. Co-transplantation of haploidentical stem cells and a dose of unrelated cord blood in pediatric patients with thalassemia major. Cell Transplant, 2021, 30: 963689721994808.
14. Vellaichamy Swaminathan V, Ravichandran N, Ramanan KM, et al. Augmented immunosuppression and PTCY-based haploidentical hematopoietic stem cell transplantation for thalassemia major. Pediatr Transplant, 2021, 25(2): e13893.
15. Gaziev J, Isgrò A, Sodani P, et al. Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ+/CD19+-depleted grafts. Blood Adv, 2018, 2(3): 263-270.
16. Farmakis D, Porter J, Taher A, et al. 2021 Thalassaemia international federation guidelines for the management of transfusion-dependent thalassemia. Hemasphere, 2022, 6(8): e732.
17. 施玲玲. 比较同胞脐血联合骨髓与外周血联合骨髓移植治疗重型β地贫的疗效和免疫重建. 南宁: 广西医科大学, 2015.
18. Inati A, Kahale M, Sbeiti N, et al. One-year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation. Pediatr Blood Cancer, 2017, 64(1): 188-196.
19. Angelucci E, Pilo F. Management of iron overload before, during, and after hematopoietic stem cell transplantation for thalassemia major. Ann N Y Acad Sci, 2016, 1368(1): 115-121.
20. Caocci G, Orofino MG, Vacca A, et al. Long-term survival of beta thalassemia major patients treated with hematopoietic stem cell transplantation compared with survival with conventional treatment. Am J Hematol, 2017, 92(12): 1303-1310.
21. Santarone S, Pepe A, Meloni A, et al. Secondary solid cancer following hematopoietic cell transplantation in patients with thalassemia major. Bone Marrow Transplant, 2018, 53(1): 39-43.
22. 王鹏, 陈月, 张海英. 治疗成人β-地中海贫血新药: luspatercept. 中国新药与临床杂志, 2021, 40(8): 568-571.
23. Cappellini MD, Viprakasit V, Taher AT, et al. A phase 3 trial of luspatercept in patients with transfusion-dependent β-thalassemia. N Engl J Med, 2020, 382(13): 1219-1231.
24. Thompson AA, Walters MC, Kwiatkowski J, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med, 2018, 378(16): 1479-1493.
25. Frangoul H, Altshuler D, Cappellini MD, et al. CRISPR-cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med, 2021, 384(3): 252-260.

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Haploidentical hematopoietic stem cell transplantation for beta-thalassemia: a meta-analysis

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