Montoro Juan, Eikema Dirk-Jan, Piepenbroek Brian, Tuffnell Joe, Halahleh Khalid, Kulagin Alexander, AlAhmari Ali, Aksoy Basak Adakli, Reményi Péter, Itäla-Remes Maija, Gulbas Zafer, McDonald Andrew, Apte Shashikant, Kwon Mi, Rovira Montserrat, Kharya Gaurav, Potter Victoria, Gambella Massimiliano, Schroeder Thomas, Giammarco Sabrina, Bazarbachi Ali, Aljurf Mahmoud, Ho Aloysius, Dalle Jean-Hugues, Vydra Jan, Sanz Jaime, Pérez-Simon José Antonio, Colita Anca, Collin Matthew, Tanase Alina, Halkes Constantijn, Kulasekararaj Austin, Risitano Antonio, Peffault de Latour Régis
Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain.
Universidad Católica de Valencia, Valencia, Spain.
Bone Marrow Transplant. 2025 May 27. doi: 10.1038/s41409-025-02633-y.
The use of post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis in severe aplastic anemia (SAA) remains understudied, particularly beyond haploidentical transplants. We analyzed outcomes of SAA patients who underwent stem cell transplantation (SCT) with PTCy from haploidentical donors (n = 209), HLA-matched sibling donors (MSD, n = 70), and unrelated donors (UD, n = 69) using EBMT data from 2010 to 2022. Median age was 22 years, and median time to transplantation was 8.6 months. For haploidentical, MSD, and UD cohorts, the 100-day cumulative incidence of grade II-IV acute GVHD was 19%, 11%, and 14% (p = 0.15), while grade III-IV was 6%, 3%, and 2% (p = 0.1). Two-year chronic and extensive chronic GVHD were 14%, 13%, and 14% (p = 0.1) and 5%, 6%, and 2% (p = 0.5), respectively. Non-relapse mortality at two years was 24% for haploidentical, 7% for MSD, and 10% for UD (p = 0.003). Two-year overall survival (OS) and GVHD- and relapse-free survival were 66% and 54% for haploidentical, 92% and 70% for MSD, and 81% and 66% for UD (p < 0.001, p = 0.06). In multivariable analysis, MSD and UD were associated with superior OS and GRFS compared to haploidentical. PTCy is safe and effective in SAA patients, though haploidentical SCT had higher NRM, leading to lower survival.
在严重再生障碍性贫血（SAA）中，移植后环磷酰胺（PTCy）用于预防移植物抗宿主病（GVHD）的研究仍不充分，尤其是在单倍体移植之外。我们利用2010年至2022年欧洲血液和骨髓移植组（EBMT）的数据，分析了接受来自单倍体供者（n = 209）、人类白细胞抗原（HLA）匹配的同胞供者（MSD，n = 70）和无关供者（UD，n = 69）的PTCy进行干细胞移植（SCT）的SAA患者的结局。中位年龄为22岁，移植的中位时间为8.6个月。对于单倍体、MSD和UD队列，II-IV级急性GVHD的100天累积发生率分别为19%、11%和14%（p = 0.15），而III-IV级为6%、3%和2%（p = 0.1）。两年慢性和广泛性慢性GVHD分别为14%、13%和14%（p = 0.1）以及5%、6%和2%（p = 0.5）。两年时非复发死亡率单倍体为24%，MSD为7%，UD为10%（p = 0.003）。两年总生存率（OS）以及无GVHD和无复发生存率单倍体分别为66%和54%，MSD为92%和70%，UD为81%和66%（p < 0.001，p = 0.06）。在多变量分析中，与单倍体相比，MSD和UD与更好的OS和无GVHD复发生存率（GRFS）相关。PTCy在SAA患者中安全有效，尽管单倍体SCT有更高的非复发死亡率，导致生存率较低。
