Department of Hematology-Oncology, Hospital de Pediatría S.A.M.I.C Prof. Dr. Juan P. Garrahan, Buenos Aires, Argentina.
Department of Immunology and Rheumatology, Hospital de Pediatría S.A.M.I.C Prof. Dr. Juan P. Garrahan, Buenos Aires, Argentina.
Pediatr Blood Cancer. 2020 Oct;67(10):e28624. doi: 10.1002/pbc.28624. Epub 2020 Jul 30.
Infant acute lymphoblastic leukemia (ALL) is an infrequent disease characterized by clinical and biological features related to poor prognosis. Adapted therapies were designed without a clear consensus regarding the best treatment options. We aimed to compare the outcome between infant ALL cases receiving Interfant versus BFM-based protocols.
This is a retrospective observational study. From April 1990 to June 2018, infant ALL cases were enrolled in one of the five consecutive treatment protocols. Clinical, demographic, and biological features and outcome were evaluated. A comparative analysis was performed between Interfant protocols and BFM-based protocols.
During the studied period, 1913 ALL patients were admitted and 116 (6%) were infants. Treatment administered was: ALL-BFM'90 (n = 16), 1-ALL96-BFM/HPG (n = 7), Interfant-99 (n = 39), Interfant-06 (n = 35), and ALLIC-BFM'2009 (n = 19). The 5-year event-free survival probability (EFSp) was 31.9(standard error [SE] 4.6)% for the entire population, with a significant difference among risk groups according to Interfant-06 criteria (P = .0029). KMT2A-rearrangement status was the strongest prognostic factor (P = .048), independently of the protocol strategy. The median time for relapse was 24.1 months for patients with minimal residual disease (MRD)-negative versus 11.5 months for those with MRD-positive (P = .0386). EFSp and cumulative relapse risk probability (CRRp) were similar. Interfant protocols showed comparable induction (8.1% vs 7.1%, P = .852) and complete remission mortality (21.6% vs 28.6%, P = .438), failing to reduce the relapse rate (48.5% vs 30.7%, P = .149).
Interfant protocols and BFM-based protocols presented comparable results. The risk group stratification proposed by Interfant-06 was validated by our results, and MRD seems useful to identify patients with an increased risk of early relapse.
婴儿急性淋巴细胞白血病（ALL）是一种罕见疾病，其临床和生物学特征与预后不良相关。适应性治疗方案的设计并未就最佳治疗选择达成明确共识。我们旨在比较接受 Interfant 与基于 BFM 的方案的婴儿 ALL 病例的结果。
这是一项回顾性观察性研究。1990 年 4 月至 2018 年 6 月，将婴儿 ALL 病例纳入五个连续治疗方案之一。评估了临床、人口统计学和生物学特征及结果。对 Interfant 方案和基于 BFM 的方案进行了比较分析。
在研究期间，收治了 1913 例 ALL 患者，其中 116 例（6%）为婴儿。治疗方法为：ALL-BFM'90（n=16）、1-ALL96-BFM/HPG（n=7）、Interfant-99（n=39）、Interfant-06（n=35）和 ALLIC-BFM'2009（n=19）。整个人群的 5 年无事件生存概率（EFSp）为 31.9%（标准误差 [SE] 4.6）%，根据 Interfant-06 标准，风险组之间存在显著差异（P=0.0029）。KMT2A 重排状态是最强的预后因素（P=0.048），独立于方案策略。微小残留病（MRD）阴性患者的复发中位时间为 24.1 个月，MRD 阳性患者的复发中位时间为 11.5 个月（P=0.0386）。EFSp 和累积复发风险概率（CRRp）相似。Interfant 方案的诱导缓解率（8.1%对 7.1%，P=0.852）和完全缓解死亡率（21.6%对 28.6%，P=0.438）相当，未能降低复发率（48.5%对 30.7%，P=0.149）。
Interfant 方案和基于 BFM 的方案的结果相当。Interfant-06 提出的风险分组得到了我们的结果验证，MRD 似乎可用于识别早期复发风险增加的患者。
