Berger Massimo, Faraci Maura, Saglio Francesco, Giardino Stefano, Ernestina Vassallo Elena, Prete Arcangelo, Fagioli Franca
Pediatric Onco-Hematology, Regina Margherita Children Hospital, City of Health and Science, University of Turin, Turin, Italy.
Hematopoietic Stem Cell transplantation Unit IRCSS, Istituto G. Gaslini, Genova, Italy.
Pediatr Transplant. 2021 Aug;25(5):e13909. doi: 10.1111/petr.13909. Epub 2020 Nov 3.
PGF is historically associated with high morbidity and mortality after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
In this study, we report our multicenter experience on stem cell boost (SCB) for PGF, or incomplete donor engraftment, in 16 pediatric patients. Donors were HLA-matched siblings (n = 4), unrelated donors (n = 11), or haploidentical family members (n = 1). Ten patients had two-lineage cytopenia, 5 had one-lineage cytopenia, and 1 had poor immunological reconstitution together with a low percentage of donor cell engraftment. A median of 6.6x10 selected CD34+/Kg was infused after 194 days from allo-HSCT (48-607).
In 4 out of 5 patients, one-lineage cytopenia was resolved, while among the 10 patients with two-lineage cytopenia, 4 resolved both cytopenia, 5 resolved one-lineage, and one did not respond. All patients reverted their mixed chimera to full donor chimera. OS was 56%, transplant-related mortality (TRM) 32%, and RI 12%. The main causes of failure were related to infections with 4 out of 7 deaths caused by this.
SCB may rescue over 50% of patients with PGF after allo-HSCT. An earlier treatment may reduce the infectious complications and improve survival.
历史上，原发性移植物功能不良（PGF）与异基因造血干细胞移植（allo-HSCT）后的高发病率和死亡率相关。
在本研究中，我们报告了16例儿科患者接受干细胞增强（SCB）治疗PGF或不完全供体植入的多中心经验。供体为HLA匹配的同胞（n = 4）、无关供体（n = 11）或单倍体亲属（n = 1）。10例患者有两系血细胞减少，5例有一系血细胞减少，1例免疫重建不良且供体细胞植入率低。allo-HSCT后194天（48 - 607天）输注了中位数为6.6x10个选定的CD34 + / Kg。
5例患者中有4例一系血细胞减少得到缓解，10例两系血细胞减少患者中，4例两系血细胞减少均得到缓解，5例一系血细胞减少得到缓解，1例无反应。所有患者的混合嵌合体均转变为完全供体嵌合体。总生存率（OS）为56%，移植相关死亡率（TRM）为32%，复发率（RI）为12%。失败的主要原因与感染有关，7例死亡中有4例由此导致。
SCB可能挽救超过50%的allo-HSCT后发生PGF的患者。早期治疗可能减少感染并发症并提高生存率。
