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二次单倍体相合干细胞移植治疗原发性移植物失败。

Second haploidentical stem cell transplantation for primary graft failure.

机构信息

Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma, Italy.

IRCCS Policlinico San Martino IST, Genova, Italy.

出版信息

Bone Marrow Transplant. 2021 Jun;56(6):1291-1296. doi: 10.1038/s41409-020-01183-9. Epub 2020 Dec 16.

Abstract

We report the outcome of 19 patients who experienced primary graft failure (PrGF) after a haploidentical (HAPLO), unmanipulated bone marrow transplant. The median age of patients was 52 years; the conditioning regimen of the first HAPLO transplant was either full dose total body irradiation (TBI) or fludarabine, busulfan, and thiotepa (TBF); PTCY was given to all patients together with cyclosporine and mycophenolate. All 19 patients with PrGF received a second HAPLO graft, at a median interval of 42 days (34-82) after HSCT, using the Baltimore protocol and G-CSF mobilized PB from the same (n = 13) or another HAPLO family donor (n = 6). GvHD prophylaxis was again PTCY-based; 14/19 patients had trilineage recovery (74%) and 1-year survival was 66%. Engraftment at second HAPLO was seen in 7/8 patient with, and in 5/7 patients without donor-specific antibodies (DSA). In a multivariate logistic regression analysis on the original group of 503 patients, there was a trend for a reduced dose of busulfan, to increase the risk of PrGF (p = 0.1). In conclusion, patients with PrGF following a HAPLO transplant, can be rescued with a second early HAPLO transplant, using the same or a different donor.

摘要

我们报告了 19 例在单倍体(HAPLO)、未经处理的骨髓移植后发生原发性移植物失败(PrGF)的患者的结果。患者的中位年龄为 52 岁;第一次 HAPLO 移植的预处理方案为全剂量全身照射(TBI)或氟达拉滨、白消安和噻替哌(TBF);所有患者均给予 PTCY 联合环孢素和霉酚酸酯。所有 19 例 PrGF 患者均在 HSCT 后中位间隔 42 天(34-82 天)接受第二次 HAPLO 移植,使用巴尔的摩方案和 G-CSF 动员来自同一(n=13)或另一个 HAPLO 家族供体(n=6)的 PB。再次采用 PTCY 进行 GvHD 预防;19/19 例患者三线恢复(74%),1 年生存率为 66%。在第二次 HAPLO 中有 7/8 例患者出现移植物嵌合,而在 5/7 例无供体特异性抗体(DSA)的患者中出现移植物嵌合。在对 503 例患者的原始组进行的多变量逻辑回归分析中,降低白消安剂量有增加 PrGF 风险的趋势(p=0.1)。总之,HAPLO 移植后发生 PrGF 的患者可以通过第二次早期 HAPLO 移植挽救,使用同一或不同供体。

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