Department of Hematology, Shanghai General Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China.
Engineering Technology Research Center of Cell Therapy and Clinical Translation, Shanghai Science and Technology Committee (STCSM), Shanghai, China.
Front Immunol. 2023 Oct 26;14:1252879. doi: 10.3389/fimmu.2023.1252879. eCollection 2023.
The novel low-dose anti-thymocyte (ATG, 5 mg/kg) plus low-dose post-transplant cyclophosphamide (PTCy, 50 mg/kg) (low-dose ATG/PTCy)-based regimen had promising activity for prevention of graft-versus-host disease (GVHD) in haploidentical-peripheral blood stem cell transplantation (haplo-PBSCT), but its impacts on long-term outcomes remain to be defined.
We performed a large sample, long-term follow-up retrospective study to evaluate its efficacy for GVHD prophylaxis.
The study enrolled 260 patients, including 162 with myeloid malignancies and 98 with lymphoid malignancies. The median follow-up time was 27.0 months. For the entire cohort, the cumulative incidences (CIs) of grade II-IV and III-IV acute GVHD (aGVHD) by 180 days were 13.46% (95% CI, 9.64%-17.92%) and 5.77% (95% CI, 3.37%-9.07%); while total and moderate/severe chronic GVHD (cGVHD) by 2 years were 30.97% (95% CI, 25.43%-36.66%) and 18.08% (95% CI, 13.68%-22.98%), respectively. The 2-year overall survival (OS), relapse-free survival (RFS), GVHD-free, relapse-free survival (GRFS), non-relapse mortality (NRM), and CIs of relapse were 60.7% (95% CI, 54.8%-67.10%), 58.1% (95% CI, 52.2%-64.5%), 50.6% (95% CI, 44.8-57.1%), 23.04% (95% CI, 18.06%-28.40%), and 18.09% (95% CI, 14.33%-23.97%, respectively. The 1-year CIs of cytomegalovirus (CMV) and Epstein-Barr virus (EBV) reactivation were 43.46% (95% CI, 37.39%-49.37%) and 18.08% (95% CI, 13.68%-22.98%), respectively. In multivariate analysis, the disease status at transplantation was associated with inferior survivor outcomes for all patients and myeloid and lymphoid malignancies, while cGVHD had superior outcomes for all patients and myeloid malignancies, but not for lymphoid malignancies.
The results demonstrated that the novel regimen could effectively prevent the occurrence of aGVHD in haplo-PBSCT.
新型低剂量抗胸腺细胞（ATG，5mg/kg）联合低剂量移植后环磷酰胺（PTCy，50mg/kg）（低剂量 ATG/PTCy）方案在单倍体外周血造血干细胞移植（haplo-PBSCT）中预防移植物抗宿主病（GVHD）具有良好的活性，但它对长期结果的影响仍有待确定。
我们进行了一项大型样本、长期随访的回顾性研究，以评估其预防 GVHD 的疗效。
本研究纳入了 260 例患者，其中 162 例为髓系恶性肿瘤患者，98 例为淋巴系恶性肿瘤患者。中位随访时间为 27.0 个月。对于整个队列，180 天内 II-IV 级和 III-IV 级急性 GVHD（aGVHD）的累积发生率（CI）分别为 13.46%（95%CI，9.64%-17.92%）和 5.77%（95%CI，3.37%-9.07%）；而总慢性 GVHD（cGVHD）和中重度/严重 cGVHD 的 2 年发生率分别为 30.97%（95%CI，25.43%-36.66%）和 18.08%（95%CI，13.68%-22.98%）。2 年总生存率（OS）、无复发生存率（RFS）、GVHD 无复发生存率（GRFS）、无复发死亡率（NRM）和复发的 CI 分别为 60.7%（95%CI，54.8%-67.10%）、58.1%（95%CI，52.2%-64.5%）、50.6%（95%CI，44.8-57.1%）、23.04%（95%CI，18.06%-28.40%）和 18.09%（95%CI，14.33%-23.97%）。1 年巨细胞病毒（CMV）和 EBV 再激活的 CI 分别为 43.46%（95%CI，37.39%-49.37%）和 18.08%（95%CI，13.68%-22.98%）。多因素分析显示，移植时的疾病状态与所有患者和髓系、淋巴系恶性肿瘤患者的生存结局较差相关，而 cGVHD 则与所有患者和髓系恶性肿瘤患者的生存结局较好相关，但与淋巴系恶性肿瘤无关。
结果表明，该新型方案可有效预防单倍体 PBSCT 中 aGVHD 的发生。
