Riccheri Maria C, Gomez Sergio M, Deana Alejandra, Fynn Alcira B, Negri-Aranguren Pedro, Arbesu Guillermo, Elena Graciela, Caferri Horacio, Cedola Alejandra, Soria Marcela, Reichel Paola, Makiya Monika, Hollmann Carlos, Borchichi Sandra, Ferraro Cristina, Schuttenberg Virginia, Tomassetti Mercedes, Arrieta Mae, Dibar Eduardo, Sung Lillian
Hospital Nacional Profesor Dr Alejandro Posadas, Haedo, Argentina.
Hospital Sor Maria Ludovica, La Plata, Argentina.
Pediatr Blood Cancer. 2025 Oct;72(10):e31886. doi: 10.1002/pbc.31886. Epub 2025 Jul 9.
Intensified postinduction therapy improves outcomes for high-risk pediatric patients with acute lymphoblastic leukemia (ALL) in high-income countries. However, benefits are uncertain in middle-income countries where supportive care is often limited. The primary objective was to determine if augmented protocal I phase B (IB) reduced the 5-year cumulative incidence of relapse compared with standard IB among newly diagnosed pediatric patients with intermediate- and high-risk (IR and HR) ALL in a middle-income country.
This was a randomized, phase III multicenter study conducted in 30 centers from GATLA group in Argentina, as part of International BFM study group ALLIC. We included newly diagnosed pediatric patients with ALL 1-18 years of age with IR or HR B- and T-precursor ALL. Only patients who were in complete remission at end induction were randomized to augmented IB versus standard IB. Augmented IB consisted of cyclophosphamide, cytarabine, 6-mercaptopurine, vincristine, E. coli l-asparaginase and intrathecal methotrexate. Standard IB consisted of cyclophosphamide, 6-mercaptopurine, cytarabine, and intrathecal methotrexate. The primary outcome was the cumulative incidence of relapse.
There were 1060 patients randomized to standard IB (n = 527) and augmented IB (n = 533). The 5-year cumulative incidence of relapse (±standard error) was not significantly different by group (22.6 ± 0.2 vs. 22.3 ± 0.1%; p = 0.97) for standard IB and augmented IB, respectively. Treatment-related mortality (TRM) was 6.5 ± 0.1 and 7.5 ± 0.1%; p = 0.45, respectively.
Among newly diagnosed pediatric patients with IR and HR ALL treated in Argentina, postinduction intensification with augmented IB did not improve outcomes compared with standard IB. Standard IB should be used for these patients. Future trials should focus on reducing TRM.
在高收入国家,强化诱导后治疗可改善高危急性淋巴细胞白血病(ALL)儿科患者的预后。然而,在支持性护理往往有限的中等收入国家,其益处尚不确定。主要目的是确定在一个中等收入国家,与标准的强化疗方案I B期(IB)相比,强化的IB方案是否能降低新诊断的中高危(IR和HR)ALL儿科患者的5年累积复发率。
这是一项随机、III期多中心研究,在阿根廷GATLA组的30个中心进行,是国际BFM研究组ALLIC的一部分。我们纳入了1 - 18岁新诊断的ALL儿科患者,包括IR或HR B前体和T前体ALL。仅诱导结束时完全缓解的患者被随机分为强化IB组和标准IB组。强化IB方案包括环磷酰胺、阿糖胞苷、6 - 巯基嘌呤、长春新碱、大肠杆菌L - 天冬酰胺酶和鞘内注射甲氨蝶呤。标准IB方案包括环磷酰胺、6 - 巯基嘌呤、阿糖胞苷和鞘内注射甲氨蝶呤。主要结局是复发的累积发生率。
1060例患者被随机分为标准IB组(n = 527)和强化IB组(n = 533)。标准IB组和强化IB组的5年累积复发率(±标准误)无显著差异(分别为22.6 ± 0.2%和22.3 ± 0.1%;p = 0.97)。治疗相关死亡率分别为6.5 ± 0.1%和7.5 ± 0.1%;p = 0.45。
在阿根廷接受治疗的新诊断的IR和HR ALL儿科患者中,与标准IB相比,强化IB诱导后强化治疗并未改善预后。这些患者应采用标准IB方案。未来试验应侧重于降低治疗相关死亡率。