Nair Velu, Kathrotiya Manthan, Shirure Vijaykumar, Bhise Shruti, Kheni Sandip, Patel Jayani, Motwani Neha, Shah Dhara, Sukhwal Grishma, Paranjape Chaitrangi, Nair Vivek, Warman Anshul, Patrikar Seema, Yanamandra Uday
Department of Clinical Haematology, BMT, and Cellular Therapy, Apollo Hospital International Limited, Gandhinagar, India.
Dr Nair's Skin Clinic and Hair Transplant Centre, Gurgaon, India.
Bone Marrow Transplant. 2025 Jul;60(7):994-1001. doi: 10.1038/s41409-025-02581-7. Epub 2025 Apr 21.
PT-CY use in T cell-replete haploidentical HCT has significantly improved outcomes. However, hyperhydration with MESNA in CY administration poses a challenge, in patients with cardiac/ renal problems. PT-CY also increases VOD risk with prior exposure to hepatotoxic drugs. Katsanis et al. in a phase Ia trial in patients undergoing HCT for hematological malignancies showed that partially replacing PT-CY with PT-BEN had comparable outcomes to conventional PT-CY. We conducted an ambispective study in 54 patients [haplo (39), MSD(14), and MUD(1)] with nonmalignant hematological disorders and hematological malignancies in pediatric and adult patients undergoing HCT (MAC/RIC) from February 2019 to May 2024. GvHD prophylaxis comprised of PT-CY/BEN (PT-CY 50 mg/kg Day +3; PT-BEN 90 mg/m Day +4) in a prospective arm (n = 21) and PT-CY/CY (50 mg/kg on Days +3, +4; comparator arm) in ambispective (prospective 12; retrospective 21) arm. In both groups, immunosuppression with CNI and MMF was also given. PT-CY/BEN was comparable to PT-CY/CY in terms of safety, efficacy, and GVHD prevention. In the PT-CY/BEN group, there was earlier neutrophil (0.008) and platelet (0.0057) engraftment with significantly lower BK viremia. Incidence of bacterial infection, TRM, EFS, and OS were comparable in both groups.
在T细胞充足的单倍体相合造血干细胞移植中使用PT-CY显著改善了治疗结果。然而，在CY给药时使用美司钠进行水化对有心脏/肾脏问题的患者构成了挑战。PT-CY在先前接触过肝毒性药物的情况下也会增加肝静脉闭塞病的风险。Katsanis等人在一项针对血液系统恶性肿瘤接受造血干细胞移植患者的Ia期试验中表明，用PT-BEN部分替代PT-CY与传统的PT-CY有相当的治疗结果。我们在2019年2月至2024年5月期间对54例接受造血干细胞移植（MAC/RIC）的儿科和成人患者进行了一项回顾性和前瞻性研究，这些患者患有非恶性血液系统疾病和血液系统恶性肿瘤[单倍体相合（39例）、半相合不相关供者（14例）和全相合不相关供者（1例）]。移植物抗宿主病预防在前瞻性组（n = 21）中采用PT-CY/BEN（PT-CY 50 mg/kg第+3天；PT-BEN 90 mg/m²第+4天），在回顾性和前瞻性组（前瞻性12例；回顾性21例）中采用PT-CY/CY（第+3天、+4天各50 mg/kg；对照臂）。在两组中，还给予了钙调神经磷酸酶抑制剂和霉酚酸酯进行免疫抑制。PT-CY/BEN在安全性、疗效和移植物抗宿主病预防方面与PT-CY/CY相当。在PT-CY/BEN组中，中性粒细胞（0.008）和血小板（0.0057）植入更早，BK病毒血症显著降低。两组的细菌感染发生率、移植相关死亡率、无事件生存期和总生存期相当。
