Genetic haploidentical peripheral blood stem cell transplantation for treatment of myelodysplastic syndrome: a 2-year follow-up visit of 21 cases

doi:10.3969/j.issn.2095-4344.0432

Abstract

Abstract:

BACKGROUND: In recent years, genetic haploidentical peripheral blood stem cell transplantation has been gradually improved, and haploid allogeneic hematopoietic stem cell transplantation has become an important treatment choice for malignant hematopoietic disease.
OBJECTIVE: To observe the clinical efficacy of genetic haploidentical peripheral blood stem cell transplantation for myelodysplastic syndrome.
METHODS: The clinical data of 21 myelodysplastic syndrome cases undergoing genetic haploidentical peripheral blood stem cell transplantation were retrospectively analyzed. Modified BU/CY+ATG administration was performed as a pretreatment strategy for haploidentical peripheral blood stem cell transplantation, and the combined use of cyclosporine A+mycophenolate mofetil+short-range methotrexate±basiliximab was adopted to prevent graft-versus-host disease (GVHD).
RESULTS AND CONCLUSION: (1) The 21 cases were followed for an median of 333 days (22-1 222 days), with 76% (16/21) infection of granulocyte lack period, 100% (21/21) neutrophil reconstruction, the median implantation time of 12 days (7-17 days), 81% (17/21) platelet engraftment, and the median implantation time of 14 days (7-68 days). (2) The accumulative incidence of GVHD was 52.4% (11/21), including 29% (6/21) of acute GVHD and 24% (5/21) of chronic GVHD. The incidence of hemorrhagic cystitis was 38.1% (8/21). The recurrence rate after transplantation was 4.8% (1/21). (3) The 2-year non-relapse mortality was 48% (10/21), and the 2-year disease-free survival rate was 46.8%. These results show that in the absence of HLA-identical related donors and unrelated donor, genetic haploidentical peripheral blood stem cell transplantation is a safe, effective, feasible and alternative treatment option for myelodysplastic syndrome.

中国组织工程研究杂志出版内容重点：干细胞；骨髓干细胞；造血干细胞；脂肪干细胞；肿瘤干细胞；胚胎干细胞；脐带脐血干细胞；干细胞诱导；干细胞分化；组织工程

Key words: Hematopoietic Stem Cell Transplantation, Myelodysplastic Syndromes, Graft vs Host Disease, Tissue Engineering

CLC Number:

R394.2

Wan Ding-ming, Liu Yu-ye, Cao Wei-jie, Xing Hai-zhou, Xie Xin-sheng, Wang Dao, Zhang Su-ping, Li Li, Chen Xiao-na, Sun Lin-lin. Genetic haploidentical peripheral blood stem cell transplantation for treatment of myelodysplastic syndrome: a 2-year follow-up visit of 21 cases[J]. Chinese Journal of Tissue Engineering Research, 2018, 22(5): 662-668.

Figures/Tables 2

2.2 感染的临床特点 21例患者中性粒细胞缺乏期间发生细菌、真菌感染者16例，感染率76%，首次出现感染的中位时间为移植-3 d(移植-10 d至移植+7 d)，感染最多见于移植-7 d至+6 d。 2.3 移植后植入情况 21例患者中，粒细胞100%植入(21/21)，粒细胞植活时间为移植后7-17 d，中位时间为+12 d；血小板植入率81%(17/21)，血小板植活时间为移植后7-68 d，中位时间为+14 d，其中4例血小板未植入。 2.4 移植后移植物抗宿主病的发生率 21例粒系植活患者中，确诊发生移植物抗宿主病共11例，发生率52%，Ⅰ，Ⅱ级急性移植物抗宿主病4例，Ⅲ，Ⅳ级急性移植物抗宿主病2例，局限型慢性移植物抗宿主病3例，广泛型慢性移植物抗宿主病2例；其中发生皮肤移植物抗宿主病8例，肠道移植物抗宿主病2例，肝脏移植物抗宿主病1例(后因肝衰竭引起多器官衰竭死亡)。 2.5 出血性膀胱炎的累计发生率 21例患者中出现出血性膀胱炎共8例，发生率为38%，出现的中位时间为移植后34.5 d(移植+24 d至移植+82 d)；7例给予碱化水化尿液、抗病毒治疗、移植物抗宿主病治疗、膀胱冲洗后均好转，另1例经选择性膀胱动脉栓塞后好转。 2.6 移植后复发率 21例患者2年内共复发1例，其复发时间为10个月，复发率5%。 2.7 无病生存率 21例患者中10例存活，无病生存曲线见图2。21例患者2年内非复发死亡率48%(10/21)；2年无病生存率46.8%。 2.8 死亡率 21例患者2年内共死亡11例，死亡率52%，6例因重症感染引发呼吸衰竭死亡，1例因复发进展至急性髓系白血病后死亡，1例死于心跳骤停，1例因肝脏移植物抗宿主病引发肝衰竭死亡，1例突发昏迷死亡，1例因意外跌倒脑出血死亡。余经性别染色体或嵌合状态检查均完全供者植入。"

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