Outcomes after Haploidentical Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Patients with Primary Immunodeficiency Diseases

IF 4.3 Q1 Medicine

Biology of Blood and Marrow Transplantation Pub Date : 2020-10-01 DOI:10.1016/j.bbmt.2020.07.003

Juliana Folloni Fernandes , Samantha Nichele , Leonardo Javier Arcuri , Lisandro Ribeiro , Gabriele Zamperlini-Netto , Gisele Loth , Ana Luiza Melo Rodrigues , Cilmara Kuwahara , Adriana Koliski , Joanna Trennepohl , Julia Lopes Garcia , Liane Esteves Daudt , Adriana Seber , Alessandra Araujo Gomes , Anders Fasth , Ricardo Pasquini , Nelson Hamerschlak , Vanderson Rocha , Carmem Bonfim

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引用次数: 32

Abstract

Allogeneic hematopoietic stem cell transplantation (HCT) can cure primary immunodeficiency diseases (PID). When a HLA-matched donor is not available, a haploidentical family donor may be considered. The use of T cell-replete haploidentical HCT with post-transplantation cyclophosphamide (haplo-PTCy) in children with PID has been reported in few case series. A donor is usually readily available, and haplo-PTCy can be used in urgent cases. We studied the outcomes of 73 patients with PID who underwent haplo-PTCy, including 55 patients who did so as a first transplantation and 18 who did so as a salvage transplantation after graft failure of previous HCT. The median patient age was 1.6 years. Most of the children were male (n = 54) and had active infection at the time of transplantation (n = 50); 10 children had severe organ damage. The diagnosis was severe combined immunodeficiency (SCID) in 34 patients and non-SCID in 39 (Wiskott-Aldrich syndrome; n = 14; chronic granulomatous disease, n = 10; other PID, n = 15). The median duration of follow-up of survivors was 2 years. The cumulative incidence of neutrophil recovery was 88% in the SCID group and 84% in non-SCID group and was 81% for first transplantations and 83% after a salvage graft. At 100 days, the cumulative incidence of acute GVHD grade II-IV and III-IV was 33% and 14%, respectively. The majority of patients reached 200/μL CD4⁺ and 1000/μL CD3⁺ cell counts between 3 and 6 months. The estimated 2-year overall survival was 66%; it was 64% for SCID patients and 65% for non-SCID patients and 63% for first HCT and 77% for salvage transplantations. Twenty-five patients died, most of them due to infection early after transplantation (before 100 days). In conclusion, haplo-PTCy is a feasible procedure, can cure two-thirds of children with PID, and can be used as rescue treatment for previous graft failure.

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原发性免疫缺陷疾病患者单倍体干细胞移植后环磷酰胺治疗的结果

同种异体造血干细胞移植(HCT)可以治疗原发性免疫缺陷疾病(PID)。当没有匹配的hla供体时，可以考虑单倍体家庭供体。在少数病例系列中报道了使用T细胞充满的单倍体HCT与移植后环磷酰胺(单倍体ptcy)。通常很容易找到供体，在紧急情况下可以使用单倍- ptcy。我们研究了73例接受单倍ptcy的PID患者的预后，包括55例首次移植患者和18例既往HCT移植失败后的补救性移植患者。患者的中位年龄为1.6岁。大多数患儿为男性(n = 54)，移植时存在活动性感染(n = 50);10名儿童有严重的器官损伤。34例诊断为严重联合免疫缺陷(SCID)， 39例诊断为非SCID (Wiskott-Aldrich综合征;n = 14;慢性肉芽肿病，n = 10;其他PID, n = 15)。幸存者的中位随访时间为2年。累积中性粒细胞恢复发生率在SCID组为88%，非SCID组为84%，首次移植为81%，补救性移植后为83%。在100天，急性GVHD II-IV级和III-IV级的累积发病率分别为33%和14%。大多数患者在3 ~ 6个月达到200/μL CD4+和1000/μL CD3+细胞计数。估计2年总生存率为66%;SCID患者为64%，非SCID患者为65%，首次HCT为63%，补救性移植为77%。25例患者死亡，大多数是由于移植后早期(100天前)感染。总之，单倍ptcy是一种可行的方法，可以治愈三分之二的儿童PID，并且可以作为既往移植物失败的抢救治疗。©2020美国移植和细胞治疗学会。Elsevier Inc.出版。

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来源期刊

Biology of Blood and Marrow Transplantation 医学-免疫学

CiteScore

6.60

自引率

0.00%

发文量

1061

审稿时长

3-6 weeks

期刊介绍： Biology of Blood and Marrow Transplantation publishes original research reports, reviews, editorials, commentaries, letters to the editor, and hypotheses and is the official publication of the American Society for Transplantation and Cellular Therapy. The journal focuses on current technology and knowledge in the interdisciplinary field of hematopoetic stem cell transplantation.