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外周血与骨髓作为重型Ⅰ级和Ⅱ级β地中海贫血患儿异基因移植造血干细胞来源的比较

Peripheral blood versus bone marrow as a source of hematopoietic stem cells for allogeneic transplantation in children with class I and II beta thalassemia major.

作者信息

Ghavamzadeh Ardeshir, Iravani Masood, Ashouri Aseaeh, Mousavi Seyed Asadollah, Mahdavi Nastaran, Shamshiri Ahmadreza, Hadjibabaie Molouk, Namdar Rocsanna, Nedaeifard Leila, Ghaffari Hamidollah, Alimoghaddam Kamran

机构信息

Hematology-Oncology and Stem Cell Research Center, University of Tehran/Medical Sciences, Tehran, Iran.

出版信息

Biol Blood Marrow Transplant. 2008 Mar;14(3):301-8. doi: 10.1016/j.bbmt.2007.12.491.

Abstract

Peripheral blood stem cell transplantation (PBSCT) has been extended to treating hematologic disorders, but the benefits over bone marrow transplantation (BMT) still remain unclear, especially in nonmalignant hematologic disorders. In this study, we compared class I-II thalassemic children who underwent HLA-matched PBSCT and BMT for treatment. Conditioning regimens consisted of busulfan and cyclophosphamide, followed by cyclosporine +/- methotrexate for graft-versus-host disease (GVHD) prophylaxis. Using multivariate analysis, the outcomes of 87 PBSCT patients and 96 BMT patients were reported (median follow-up: 29 and 60 months, respectively). The median time to neutrophil and platelet recovery in PBSCT patients (11 and 18 days, respectively) was significantly lower than BMT patients (19 and 26 days, respectively) (P < .001). Grade II-IV acute GVHD was more frequent in PBSCT versus BMT group (72% versus 55%; P = .003) (relative risk = 1.75, 95% confidence interval [CI]: 1.20-2.57). The incidence of chronic GVHD was more frequent in the PBSCT versus BMT group (48% versus 19%; P < .001) (relative risk = 2.62, 95% CI: 1.43-4.82). There was no difference in the 2-year overall survival after PBSCT and BMT (83% and 89%, respectively). The 2-year disease-free survival was 76% in both groups. These results show some advantages of PBSCT, but to improve the risk of GVHD in PBSCT, a better conditioning and prophylaxis regimen is needed.

摘要

外周血干细胞移植(PBSCT)已被扩展用于治疗血液系统疾病,但与骨髓移植(BMT)相比,其优势仍不明确,尤其是在非恶性血液系统疾病中。在本研究中,我们比较了接受 HLA 匹配的 PBSCT 和 BMT 治疗的 I-II 级地中海贫血儿童。预处理方案包括白消安和环磷酰胺,随后使用环孢素+/-甲氨蝶呤预防移植物抗宿主病(GVHD)。通过多变量分析,报告了 87 例 PBSCT 患者和 96 例 BMT 患者的结果(中位随访时间分别为 29 个月和 60 个月)。PBSCT 患者中性粒细胞和血小板恢复的中位时间(分别为 11 天和 18 天)显著低于 BMT 患者(分别为 19 天和 26 天)(P <.001)。PBSCT 组 II-IV 级急性 GVHD 比 BMT 组更常见(72%对 55%;P =.003)(相对风险 = 1.75,95%置信区间[CI]:1.20 - 2.57)。PBSCT 组慢性 GVHD 的发生率比 BMT 组更高(48%对 19%;P <.001)(相对风险 = 2.62,95%CI:1.43 - 4.82)。PBSCT 和 BMT 后的 2 年总生存率无差异(分别为 83%和 89%)。两组的 2 年无病生存率均为 76%。这些结果显示了 PBSCT 的一些优势,但为了降低 PBSCT 中 GVHD 的风险,需要更好的预处理和预防方案。

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