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佛罗里达儿科骨髓移植与细胞治疗联盟(FPBCC)的发展历程:一项旨在改善移植结果的全州性倡议。

Evolution of the Florida Pediatric Bone Marrow Transplant and Cell Therapy Consortium (FPBCC): A Statewide Initiative Toward Improving Transplant Outcomes.

作者信息

Alperstein Warren, Lee Jin-Ju, Chellapandian Deepakbabu, Booth Natalie, Galvez-Silva Jorge, Joyce Michael, Milner Jordan, Castillo Paul, Kashif Reema, Dalal Mansi, Ligon John, Crawford David, Alicea Marrero Minelys M, Peters Jessica, Horn Biljana, Dela Ziga Edward

机构信息

Pediatric Hematology and Oncology, University of Miami School of Medicine, Miami, Florida, USA.

Pediatric BMT and Cell Therapy, University of Florida, Gainesville, Florida, USA.

出版信息

Pediatr Transplant. 2025 May;29(3):e70059. doi: 10.1111/petr.70059.

Abstract

BACKGROUND

Florida Pediatric Bone Marrow Transplant and Cell Therapy Consortium (FPBCC) was formed in 2018 by five pediatric transplant programs in Florida. The key objectives of the consortium are to improve outcomes for children undergoing HSCT through collaboration among centers, data sharing, implementation of best practices, QI projects, and prospective clinical trials. The first step in that process was to analyze HSCT outcomes from all participating centers and identify areas for improvement. In this report, we describe the effectiveness of the activities of this consortium, focused on improving patients' outcomes.

METHODS

A retrospective data review of allogeneic transplant 1-year survival, obtained from the annual CIBMTR report, from the five FPBCC centers was compared to survival from 38 other pediatric centers in the country over two periods: preconsortium establishment, from 2016 to 2018, and postconsortium establishment, from 2019 to 2021. Of the 38 other pediatric centers, 22 were defined as small, similar to consortium centers by number of transplants (20-70 first allogeneic transplants per center in a 3-year period) and 16 were larger centers (> 71 first allogeneic transplants per center in a 3-year period).

RESULTS

The 1-year posttransplant survival for the FPBCC centers significantly improved from 77.5% (2016-2018) to 89.5% (2019-2021; p = 0.0313). During the same respective time periods, other small centers improved from 82.4% to 87.9% (p = 0.0059), and large centers maintained stable survival at 85.6%-85.4% (p = 0.2676).

CONCLUSIONS

There was a substantial improvement in the 1-year survival of allogeneic transplant recipients treated in FPBCC centers, achieved after the initiation of consortium activities. Within a 3-year period, consortium centers, which had a lower starting point, reached 1-year survival comparable to that of other small and large centers. A significant improvement in survival, although a lesser percentage of change, was seen in other programs of similar size across the country, but not in larger programs. We consider that the magnitude of improvement in survival (12% points or 4% per year), which was not seen among other programs, attests to the effectiveness of consortium activities. A blueprint for improvement in outcomes established by the FPBCC can be shared with other programs around the world that strive to improve posttransplant survival.

摘要

背景

佛罗里达儿科骨髓移植与细胞治疗联盟(FPBCC)于2018年由佛罗里达州的五个儿科移植项目组成。该联盟的主要目标是通过中心间的合作、数据共享、最佳实践的实施、质量改进项目和前瞻性临床试验,改善接受造血干细胞移植(HSCT)儿童的治疗结果。该过程的第一步是分析所有参与中心的HSCT结果,并确定需要改进的领域。在本报告中,我们描述了该联盟活动的有效性,重点是改善患者的治疗结果。

方法

从年度CIBMTR报告中获取了FPBCC五个中心的异基因移植1年生存率的回顾性数据,并与该国其他38个儿科中心在两个时期的生存率进行了比较:联盟成立前,即2016年至2018年;联盟成立后,即2019年至2021年。在其他38个儿科中心中,22个被定义为小型中心,按移植数量(3年内每个中心20 - 70例首次异基因移植)与联盟中心相似,16个为大型中心(3年内每个中心>71例首次异基因移植)。

结果

FPBCC中心的移植后1年生存率从77.5%(2016 - 2018年)显著提高到89.5%(2019 - 2021年;p = 0.0313)。在相同的相应时间段内,其他小型中心从82.4%提高到87.9%(p = 0.0059),大型中心的生存率保持稳定,为85.6% - 85.4%(p = 0.2676)。

结论

在联盟活动启动后,FPBCC中心治疗的异基因移植受者的1年生存率有了显著提高。在3年的时间里,起点较低的联盟中心达到了与其他小型和大型中心相当的1年生存率。在全国其他规模相似的项目中也观察到了生存率的显著提高,尽管变化百分比较小,但在大型项目中未观察到。我们认为,其他项目中未出现的生存率提高幅度(12个百分点或每年4%)证明了联盟活动的有效性。FPBCC制定的改善治疗结果的蓝图可以与世界各地其他致力于提高移植后生存率的项目分享。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3111/11883450/a23462e62e1b/PETR-29-e70059-g001.jpg

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