Cavallaro Gianluca, Lazzarotto Davide, Pavoni Chiara, Valsecchi Francesca, Grassi Anna, Papayannidis Cristina, Cerrano Marco, Fracchiolla Nicola, Giglio Fabio, Dargenio Michelina, Lunghi Monia, Imbergamo Silvia, Del Principe Maria Ilaria, Trappolini Silvia, Fumagalli Monica, Zappasodi Patrizia, Salutari Prassede, Delia Mario, Pasciolla Crescenza, Mosna Federico, Scappini Barbara, Forghieri Fabio, Chiusolo Patrizia, Skert Cristina, Cambò Benedetta, Defina Marzia, Lanzarone Giuseppe, Mauro Endri, Bonifacio Massimiliano, Mazzone Carla, Santoro Lidia, Mulè Antonino, Mancini Valentina, Minetto Paola, Battipaglia Giorgia, Cignetti Alessandro, Aprile Lara, Chiaretti Sabina, Foà Robin, Candoni Anna, Lussana Federico
Hematology and Bone Marrow Transplant Unit, ASST Ospedale Papa Giovanni XXIII, Bergamo, Italy.
Clinica Ematologica - Centro Trapianti e Terapie Cellulari, Azienda Sanitaria Universitaria Friuli Centrale, Udine, Italy.
Bone Marrow Transplant. 2025 Jun 2. doi: 10.1038/s41409-025-02632-z.
Allogeneic hematopoietic stem cell transplantation (alloHSCT) is the best consolidative treatment for high-risk acute lymphoblastic leukemia (ALL). The Campus ALL study group analyzed the clinical outcomes of patients treated in the real-life with the pediatric-inspired and minimal/measurable residual disease (MRD)-oriented GIMEMA LAL1913 protocol who underwent alloHSCT. Key factors impacting on outcomes were MRD and remission status (1 complete remission vs 2 complete remission) at transplant. MRD positivity was associated with poorer outcomes, with 3-year overall survival (OS) and disease-free survival (DFS) of 47% and 41% in MRD-positive patients compared to 80% and 70% in MRD-negative patients. Additionally, MRD negativity was associated with improved outcomes also for patients in 2nd complete remission with 3-year OS and DFS rates of 60% and 56%, respectively, compared to only 13% for both outcomes in MRD-positive cases. Patients older than 55 years showed survival rates comparable to younger patients, despite having a slightly higher non-relapse mortality, which remained below 20% at 3 years. These findings underscore the crucial role of alloHSCT in high-risk ALL and emphasize the importance of an early accurate disease risk allocation. The adverse outcome observed with MRD positivity advocates for early pre-transplant intervention with immunotherapy, whenever possible.
异基因造血干细胞移植（alloHSCT）是高危急性淋巴细胞白血病（ALL）的最佳巩固治疗方法。校园ALL研究小组分析了在现实生活中按照以儿科为灵感且以微小/可测量残留疾病（MRD）为导向的GIMEMA LAL1913方案接受alloHSCT治疗的患者的临床结局。影响结局的关键因素是移植时的MRD和缓解状态（1次完全缓解与2次完全缓解）。MRD阳性与较差的结局相关，MRD阳性患者的3年总生存率（OS）和无病生存率（DFS）分别为47%和41%，而MRD阴性患者分别为80%和70%。此外，MRD阴性对于处于第2次完全缓解的患者的结局改善也有相关性，其3年OS和DFS率分别为60%和56%，而MRD阳性病例的这两个结局率仅为13%。55岁以上的患者尽管非复发死亡率略高，但其生存率与年轻患者相当，3年时仍低于20%。这些发现强调了alloHSCT在高危ALL中的关键作用，并强调了早期准确进行疾病风险分配的重要性。MRD阳性所观察到的不良结局主张尽可能在移植前尽早采用免疫疗法进行干预。
