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15岁以上血液系统疾病患者的脐带血移植:智利天主教大学的长期经验

Umbilical cord blood transplantation in hematologic diseases in patients over 15 years old: long-term experience at the Pontificia Universidad Católica de Chile.

作者信息

Ramirez P, Nervi B, Bertin P, Poggi H, Lagos M, Selman C, Pizarro I, Jara V, Wiestruck A, Barriga F

机构信息

Department of Hematology Oncology, School of Medicine, Pontificia Universidad Católica de Chile, Santiago, Chile.

出版信息

Transplant Proc. 2013;45(10):3734-9. doi: 10.1016/j.transproceed.2013.08.093.

Abstract

Most patients who require a sibling stem cell transplantation do not have a matched donor. In our experience, only 1/3 patients have a matched unrelated donor (MUD); therefore, the majority of the patients will require umbilical cord blood (UCB). Patients treated for hematologic diseases with UCB transplants were included. UCB selection and conditioning regimens were performed according to the Minnesota group. Graft-versus-host disease (GVHD) prophylaxis, infection prevention, and patient care were performed according to institutional guidelines. We analyzed patients and graft demography, neutrophil and platelet recovery, chimerism kinetics, GVHD incidence, overall (OS), progression-free survival (PFS) and transplant-related mortality (TRM). We included 29 patients with a median age of 34.8 years (range 15-55). Eighteen were male and the median weight was 72.6 kg (range 54-100). Nineteen patients had acute leukemia. Myeloablative (MA) conditioning was used in 27 patients. Seventeen received double UCB (DUCB) grafts. Median total nucleated cell (10(7)/kg) was 4.2 (range 3.9-4.9) and 4.4 (range 2.8-6.3) for single UCB (SUCB) and DUCB transplants, respectively. Median time for neutrophil engraftment was 24.7 (range 14-43) and 25.8 days (range 14-52) after SUCB and DUCB transplants, respectively. Median time for platelet engraftment was 147 (range 30-516) and 81 days (range 37-200) after SUCB and DUCB transplants, respectively. All the patients receiving MA conditioning had >95% chimerism shortly after transplant. Cumulative incidence of grades II-IV and III-IV acute GVHD was 41% and 20%, respectively. Localized chronic GVHD was seen in 14% of the patients. Median follow-up was 16.7 months (range 1-63). Five-year OS and PFS were 38% and 39%, respectively. One-year TRM was 42%. UCB transplantation is associated with potential cure of hematologic malignancies and our results are similar to other series. Studies are needed to decrease mortality and improve immune reconstitution.

摘要

大多数需要同胞干细胞移植的患者没有匹配的供体。根据我们的经验,只有1/3的患者有匹配的非亲属供体(MUD);因此,大多数患者将需要脐带血(UCB)。纳入接受UCB移植治疗血液系统疾病的患者。UCB的选择和预处理方案按照明尼苏达组的方法进行。移植物抗宿主病(GVHD)的预防、感染预防和患者护理按照机构指南进行。我们分析了患者和移植物的人口统计学数据、中性粒细胞和血小板的恢复情况、嵌合动力学、GVHD发生率、总生存期(OS)、无进展生存期(PFS)和移植相关死亡率(TRM)。我们纳入了29例患者,中位年龄为34.8岁(范围15 - 55岁)。18例为男性,中位体重为72.6 kg(范围54 - 100 kg)。19例患者患有急性白血病。27例患者采用清髓性(MA)预处理。17例接受双份UCB(DUCB)移植。单份UCB(SUCB)和DUCB移植的中位总有核细胞数(10⁷/kg)分别为4.2(范围3.9 - 4.9)和4.4(范围2.8 - 6.3)。SUCB和DUCB移植后中性粒细胞植入的中位时间分别为24.7天(范围14 - 43天)和25.8天(范围14 - 52天)。SUCB和DUCB移植后血小板植入的中位时间分别为147天(范围30 - 516天)和81天(范围37 - 200天)。所有接受MA预处理的患者在移植后不久嵌合率均>95%²⁻⁴。II - IV级和III - IV级急性GVHD的累积发生率分别为41%和20%。14%的患者出现局限性慢性GVHD。中位随访时间为16.7个月(范围1 - 63个月)。5年OS和PFS分别为38%和39%。1年TRM为42%。UCB移植与血液系统恶性肿瘤的潜在治愈相关,我们的结果与其他系列相似。需要开展研究以降低死亡率并改善免疫重建。

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