Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma, Italy.
IRCCS Policlinico San Martino IST, Genova, Italy.
Bone Marrow Transplant. 2021 Jun;56(6):1291-1296. doi: 10.1038/s41409-020-01183-9. Epub 2020 Dec 16.
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.
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。
