无关供者异基因造血干细胞移植治疗骨髓增生异常综合征9例患者临床分析

目的探讨无关供者异基因造血干细胞移植(UD-HSCT)治疗骨髓增生异常综合征(MDS)的疗效和可行性。方法MDS患者9例,其中男性6例,女性3例;年龄7～46岁,中位年龄30岁。其中难治性贫血(RA)1例,难治性血细胞减少伴有多系发育异常(RCMD)2例,难治性贫血伴有原始细胞过多-2(RAEB-2)5例,MDS进展为急性髓系白血病(MDS-AML)1例。接受UD-HSCT治疗的MDS患者中外周血造血干细胞移植(PBSCT)8例,骨髓移植(BMT)1例。供受者HLA高分辨配型10/10位点相合4例、9/10位点相合4例、7/10位点相合1例。预处理方案为BU+CY+Flud+Ara-C+ATG 8例、BU+Mel+Flud+Ara-C+ATG 1例,移植物抗宿主病(GVHD)预防方案为FK506+MTX+MMF 8例、CsA+MTX+MMF 1例。结果9例患者均获得造血重建,中性粒细胞≥0.5×109/L和血小板≥20×109/L的中位时间分别为移植后15(11～20)d和23(8～32)d。6例患者发生急性GVHD(aGVHD),其中Ⅰ度4例,Ⅱ度2例,5例患者发生局限型慢性GVHD(cGVHD)。中位随访20.3(6.4～50.0)个月,1例患者移植后14个月复发死亡,其余8例患者中位随访27.9(6.4～50.0)个月,均无病存活,总体生存率(OS)及无病生存率(DFS)均为85.7%±13.2%。结论UD-HSCT治疗MDS安全有效,在无同胞全合供者时,无关供者也可以作为此类患者有效治疗选择。

Nine cases of myelodysplastic syndrome receiving unrelated donor hematopoietic stem cell transplantation

Objective To explore the feasibility and efficacy of unrelated donor hematopoietic stem cell transplantation(UD-HSCT) for treatment of patients with myelodysplastic syndrome(MDS).Methods Nine patients with MDS were treated with UD-HSCT.The median age of patients(6 males and 3 females) was 30 years old(range 7-46).Patients were classified as refractory anemia(RA)(n = 1),refractory cytopenia with multilineage dysplasia(RCMD)(n = 2),RA with excess blasts-2(RAEB-2)(n = 5),and MDS progressed to acute myeloid leukemia(MDS-AML)(n = 1).Out of them 8 patients received peripheral blood hematopoietic stem cell transplantation(PBSCT),while 1 patient received bone marrow transplantation(BMT),included 4 HLA-matched,5 HLA partially matched(9/10 matched 4 cases,7/10 matched 1 case).Of the 9 cases,8 chose BU + CY + Flud + Ara-C + ATG as preparative regimens and 1 chose BU + Mel + Flud + Ara-C + ATG pretreatment.In order to prevent the occurrence of graft-versus-host disease(GVHD),8 patients were treated with FK506 + MTX + MMF,1 patient was treated with CsA + MTX + MMF.Results A total of 9 MDS patients successfully reconstructed their hematopoietic system.The time from transplantation to ANC ≥ 0.5 × 109/L and platelet ≥ 20 × 109/L were 15(11-20) days and 23(8-32) days after transplantation,respectively.The main complications were GVHD.Six cases developed acute GVHD(aGVHD)(4 cases of grade Ⅰ,2 cases of grade Ⅱ),and local chronic GVHD(cGVHD) occurred in 5 patients.During 20.3(6.4-50.0) months of follow-up,1 patient died of relapse at 14 months.Eight cases survived for median 27.9(6.4-50.0) months until the end of follow-up.The overall survival(OS) and disease-free survival(DFS) rates were as the same(85.7 % ± 13.2 %).Conclusion It is demonstrated that UD-HSCT is a safe and effective option for the patients with MDS.Unrelated donor could be an alternative in case of the absence of identical sibling donor.