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《工程(英文)》 >> 2021年 第7卷 第2期 doi: 10.1016/j.eng.2020.12.005

疾病危险度-共患病指数在单倍型造血干细胞移植中的应用

a Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, 100044, China
b Peking–Tsinghua Center for Life Sciences, Academy for Advanced Interdisciplinary Studies, Peking University, Beijing 100871, China
c Research Unit of Key Technique for Diagnosis and Treatments of Hematologic Malignancies, Chinese Academy of Medical Sciences (2019RU029), Beijing 100073, China

收稿日期: 2019-04-16 修回日期: 2019-12-18 录用日期: 2020-01-15 发布日期: 2021-01-05

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摘要

本文的研究目的是基于疾病危险度指数(disease risk index, DRI)和造血干细胞移植共患病指数(hematopoietic cell transplantation-specific comorbidity index, HCT-CI),提出适合单倍型造血干细胞移植(haploidentical hematopoietic stem cell transplantation, haplo-HSCT)患者的疾病危险度-共患病指数(disease risk comorbidity index, DRCI)。文中通过一个训练队列(n = 593)中确定了无疾病生存(disease-free survival, DFS)的预测因素,然后对这些因素进行赋值,从而建立DRCI,并通过验证队列(n = 296)检验积分系统的有效性。多因素分析确定了DFS的两个独立影响因素:移植前DRI以及HCT-CI。我们为极高危DRI赋值2分,为高危DRI和中、高危HCT-CI各赋值1分,从而形成适合haplo-HSCT的DRCI(即haplo-DRCI)。在验证队列中,低危、中危、高危组患者移植后3年的累积DFS率分别为65.2% (95%CI, 58.2%~72.2%)、55.8% (95%CI, 44.9%~66.7%)和32.0% (95%CI, 5.8%~58.2%) (P = 0.005)。Haplo-DRCI 还可以预测不同疾病亚组,尤其是急性白血病患者移植后的DFS。此外,在独立的历史队列中 (n = 526),较高的haplo-DRCI积分同样与较高的复发率、较高的非复发死亡率(non-relapse mortality, NRM)、较差的DFS率和较差的总体生存率(overall survival, OS)相关。这些结果表明,haplo-DRCI积分可以有效地对接受haplo-HSCT的患者进行危险分层,更好地预测哪些患者更能获益于haplo-HSCT。

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参考文献

[ 1 ] Huang XJ, Chang YJ. Unmanipulated HLA-mismatched/haploidentical blood and marrow hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2011;17(2):197–204. 链接1

[ 2 ] Lv M, Chang Y, Huang X. Everyone has a donor: contribution of the Chinese experience to global practice of haploidentical hematopoietic stem cell transplantation. Front Med 2019;13(1):45–56. 链接1

[ 3 ] Lv M, Huang XJ. Allogeneic hematopoietic stem cell transplantation in China: where we are and where to go. J Hematol Oncol 2012;5(1):10. 链接1

[ 4 ] Ciceri F, Labopin M, Aversa F, Rowe JM, Bunjes D, Lewalle P, et al; Acute Leukemia Working Party (ALWP) of European Blood and Marrow Transplant (EBMT) Group. A survey of fully haploidentical hematopoietic stem cell transplantation in adults with high-risk acute leukemia: a risk factor analysis of outcomes for patients in remission at transplantation. Blood 2008;112 (9):3574–81. 链接1

[ 5 ] Federmann B, Bornhauser M, Meisner C, Kordelas L, Beelen DW, Stuhler G, et al. Haploidentical allogeneic hematopoietic cell transplantation in adults using CD3/CD19 depletion and reduced intensity conditioning: a phase II study. Haematologica 2012;97(10):1523–31. 链接1

[ 6 ] Luznik L, O’Donnell PV, Symons HJ, Chen AR, Leffell MS, Zahurak M, et al. HLAhaploidentical bone marrow transplantation for hematologic malignancies using nonmyeloablative conditioning and high-dose, posttransplantation cyclophosphamide. Biol Blood Marrow Transplant 2008;14(6):641–50. 链接1

[ 7 ] Kasamon YL, Bolaños-Meade J, Prince GT, Tsai HL, McCurdy SR, Kanakry JA, et al. Outcomes of nonmyeloablative HLA-haploidentical blood or marrow transplantation with high-dose post-transplantation cyclophosphamide in older adults. J Clin Oncol 2015;33(28):3152–61. 链接1

[ 8 ] Wang Y, Liu QF, Xu LP, Liu KY, Zhang XH, Ma X, et al. Haploidentical vs identical-sibling transplant for AML in remission: a multicenter, prospective study. Blood 2015;125(25):3956–62. 链接1

[ 9 ] Wang Yu, Liu QF, Xu LP, Liu KY, Zhang XH, Ma X, et al. Haploidentical versus matched-sibling transplant in adults with Philadelphia-negative high-risk acute lymphoblastic leukemia: a biologically phase III randomized study. Clin Cancer Res 2016;22(14):3467–76. 链接1

[10] Wang Y, Wang HX, Lai YR, Sun ZM, Wu DP, Jiang M, et al. Haploidentical transplant for myelodysplastic syndrome: registry-based comparison with identical sibling transplant. Leukemia 2016;30(10):2055–63. 链接1

[11] Xu LP, Wu DP, Han MZ, Huang H, Liu QF, Liu DH, et al. A review of hematopoietic cell transplantation in China: data and trends during 2008– 2016. Bone Marrow Transplant 2017;52(11):1512–8. 链接1

[12] Xu L, Chen Hu, Chen J, Han M, Huang He, Lai Y, et al. The consensus on indications, conditioning regimen, and donor selection of allogeneic hematopoietic cell transplantation for hematological diseases in Chinarecommendations from the Chinese Society of Hematology. J Hematol Oncol 2018;11(1):33. 链接1

[13] Lee KH, Lee JH, Lee JH, Kim DY, Seol M, Lee YS, et al. Reduced-intensity conditioning therapy with busulfan, fludarabine, and antithymocyte globulin for HLA-haploidentical hematopoietic cell transplantation in acute leukemia and myelodysplastic syndrome. Blood 2011;118(9):2609–17. 链接1

[14] Velardi A. Haplo-BMT: which approach? Blood 2013;121(5):719–20. 链接1

[15] Di Bartolomeo P, Santarone S, De Angelis G, Picardi A, Cudillo L, Cerretti R, et al. Haploidentical, unmanipulated, G-CSF-primed bone marrow transplantation for patients with high-risk hematologic malignancies. Blood 2013;121 (5):849–57. 链接1

[16] Handgretinger R. Haploidentical transplantation: the search for the best donor. Blood 2014;124(6):827–8. 链接1

[17] Wang Y, Chen H, Chen J, Han M, Hu JD, Hu J, et al. The consensus on the monitoring, treatment, and prevention of leukemia relapse after allogeneic hematopoietic stem cell transplantation in China. Cancer Lett 2018;438:63–75. 链接1

[18] Wang Yu, Liu DH, Liu KY, Xu LP, Zhang XH, Han W, et al. Long-term follow-up of haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for the treatment of leukemia: nine years of experience at a single center. Cancer 2013;119(5):978–85. 链接1

[19] Huang XJ, Liu DH, Liu KY, Xu LP, Chen H, Han W, et al. Treatment of acute leukemia with unmanipulated HLA-mismatched/haploidentical blood and bone marrow transplantation. Biol Blood Marrow Transplant 2009;15 (2):257–65. 链接1

[20] Armand P, Gibson CJ, Cutler C, Ho VT, Koreth J, Alyea EP, et al. A disease risk index for patients undergoing allogeneic stem cell transplantation. Blood 2012;120(4):905–13. 链接1

[21] Armand P, Kim HT, Logan BR, Wang Z, Alyea EP, Kalaycio ME, et al. Validation and refinement of the disease risk index for allogeneic stem cell transplantation. Blood 2014;123(23):3664–71. 链接1

[22] Beauverd Y, Roosnek E, Tirefort Y, Nagy-Hulliger M, Bernimoulin M, Tsopra O, et al. Validation of the disease risk index for outcome of patients undergoing allogeneic hematopoietic stem cell transplantation after T cell depletion. Biol Blood Marrow Transplant 2014;20(9):1322–8. 链接1

[23] Lim AB, Roberts AW, Mason K, Bajel A, Szer J, Ritchie DS. Validating the allogeneic stem cell transplantation disease risk index: sample size, follow-up, and local data are important. Transplantation 2015;99(1):128–32. 链接1

[24] Törlén J, Remberger M, Le Blanc K, Ljungman P, Mattsson J. Impact of pretransplantation indices in hematopoietic stem cell transplantation: knowledge of center-specific outcome data is pivotal before making indexbased decisions. Biol Blood Marrow Transplant 2017;23(4):677–83. 链接1

[25] Paviglianiti A, Ruggeri A, Volt F, Sanz G, Milpied N, Furst S, et al. Evaluation of a disease risk index for adult patients undergoing umbilical cord blood transplantation for haematological malignancies. Br J Haematol 2017;179 (5):790–801. 链接1

[26] McCurdy SR, Kanakry JA, Showel MM, Tsai HL, Bolaños-Meade J, Rosner GL, et al. Risk-stratified outcomes of nonmyeloablative HLA-haploidentical BMT with high-dose posttransplantation cyclophosphamide. Blood 2015;125 (19):3024–31. 链接1

[27] Sorror ML, Maris MB, Storb R, Baron F, Sandmaier BM, Maloney DG, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood 2005;106(8): 2912–9. 链接1

[28] Smith AR, Majhail NS, MacMillan ML, DeFor TE, Jodele S, Lehmann LE, et al. Hematopoietic cell transplantation comorbidity index predicts transplantation outcomes in pediatric patients. Blood 2011;117(9):2728–34. 链接1

[29] Sorror ML, Sandmaier BM, Storer BE, Maris MB, Baron F, Maloney DG, et al. Comorbidity and disease status based risk stratification of outcomes among patients with acute myeloid leukemia or myelodysplasia receiving allogeneic hematopoietic cell transplantation. J Clin Oncol 2007;25(27): 4246–54. 链接1

[30] Sorror ML, Giralt S, Sandmaier BM, De Lima M, Shahjahan M, Maloney DG, et al. Hematopoietic cell transplantation specific comorbidity index as an outcome predictor for patients with acute myeloid leukemia in first remission: combined FHCRC and MDACC experiences. Blood 2007;110(13):4606–13. 链接1

[31] Pollack SM, Steinberg SM, Odom J, Dean RM, Fowler DH, Bishop MR. Assessment of the hematopoietic cell transplantation comorbidity index in non-Hodgkin lymphoma patients receiving reduced-intensity allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 2009;15(2):223–30. 链接1

[32] Sperr WR, Wimazal F, Kundi M, Baumgartner C, Nösslinger T, Makrai A, et al. Comorbidity as prognostic variable in MDS: comparative evaluation of the HCT-CI and CCI in a core dataset of 419 patients of the Austrian MDS Study Group. Ann Oncol 2010;21(1):114–9. 链接1

[33] Mo XD, Xu LP, Liu DH, Zhang XH, Chen H, Chen YH, et al. The hematopoietic cell transplantation-specific comorbidity index (HCT-CI) is an outcome predictor for partially matched related donor transplantation. Am J Hematol 2013;88(6):497–502. 链接1

[34] Bejanyan N, Brunstein CG, Cao Q, Lazaryan A, Ustun C, Warlick ED, et al. Predictive value of disease risk comorbidity index for overall survival after allogeneic hematopoietic transplantation. Blood Adv 2019;3(3):230–6. 链接1

[35] Wang Y, Wu DP, Liu QF, Xu LP, Liu KY, Zhang XH, et al. Low-dose posttransplant cyclophosphamide and anti-thymocyte globulin as an effective strategy for GVHD prevention in haploidentical patients. J Hematol Oncol 2019;12(1):88. 链接1

[36] Wang Y, Chang YJ, Xu LP, Liu KY, Liu DH, Zhang XH, et al. Who is the best donor for a related HLA haplotype-mismatched transplant? Blood 2014;124 (6):843–50. 链接1

[37] Mo XD, Zhang YY, Zhang XH, Xu LP, Wang Yu, Yan CH, et al. The role of collateral related donors in haploidentical hematopoietic stem cell transplantation. Sci Bull 2018;63(20):1376–82. 链接1

[38] Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med 1999;18(6):695–706. 链接1

[39] Abdul Wahid SF, Ismail NA, Mohd-Idris MR, Jamaluddin FW, Tumian N, SzeWei EY, et al. Comparison of reduced-intensity and myeloablative conditioning regimens for allogeneic hematopoietic stem cell transplantation in patients with acute myeloid leukemia and acute lymphoblastic leukemia: a meta-analysis. Stem Cells Dev 2014;23(21):2535–52. 链接1

[40] Rubio MT, Savani BN, Labopin M, Piemontese S, Polge E, Ciceri F, et al. Impact of conditioning intensity in T-replete haplo-identical stem cell transplantation for acute leukemia: a report from the acute leukemia working party of the EBMT. J Hematol Oncol 2016;9(1). 链接1

[41] Gratwohl A, Hermans J, Goldman JM, Arcese W, Carreras E, Devergie A, et al.; Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Risk assessment for patients with chronic myeloid leukaemia before allogeneic blood or marrow transplantation. Lancet 1998;352 (9134):1087–92. 链接1

[42] Gratwohl A. The EBMT risk score. Bone Marrow Transplant 2012;47 (6):749–56. 链接1

[43] Wang HT, Chang YJ, Xu LP, Liu DH, Wang Y, Liu KY, et al. EBMT risk score can predict the outcome of leukaemia after unmanipulated haploidentical blood and marrow transplantation. Bone Marrow Transplant 2014;49 (7):927–33. 链接1

[44] Huang XJ, Liu DH, Liu KY, Xu LP, Chen H, Han W, et al. Haploidentical hematopoietic stem cell transplantation without in vitro T-cell depletion for the treatment of hematological malignancies. Bone Marrow Transplant 2006;38(4):291–7. Erratum in: Bone Marrow Transplant 2008;42(4): 295.

[45] Mo XD, Zhang XH, Xu LP, Wang Y, Yan CH, Chen H, et al. Haploidentical hematopoietic stem cell transplantation for myelodysplastic syndrome. Biol Blood Marrow Transplant 2017;23(12):2143–50. 链接1

[46] Mo XD, Xu LP, Zhang XH, Liu DH, Wang Y, Chen H, et al. Haploidentical hematopoietic stem cell transplantation in adults with Philadelphia-negative acute lymphoblastic leukemia: no difference in the high- and low-risk groups. Int J Cancer 2015;136(7):1697–707. 链接1

[47] Huo MR, Pei XY, Li D, Chang YJ, Xu LP, Zhang XH, et al. Impact of HLA allele mismatch at HLA-A, -B, -C, -DRB1, and -DQB1 on outcomes in haploidentical stem cell transplantation. Bone Marrow Transplant 2018;53(5):600–8. 链接1

[48] Lorentino F, Labopin M, Fleischhauer K, Ciceri F, Mueller CR, Ruggeri A, et al. The impact of HLA matching on outcomes of unmanipulated haploidentical HSCT is modulated by GVHD prophylaxis. Blood Adv 2017;1(11):669–80. 链接1

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