Silverman L B, Gelber R D, Dalton V K, Asselin B L, Barr R D, Clavell L A, Hurwitz C A, Moghrabi A, Samson Y, Schorin M A, Arkin S, Declerck L, Cohen H J, Sallan S E
Department of Pediatric Oncology, Dana-Farber Cancer Institute, the Division of Hematology/Oncology, Children's Hospital, Harvard Medical School, Boston, MA 02215, USA.
Blood. 2001 Mar 1;97(5):1211-8. doi: 10.1182/blood.v97.5.1211.
The Dana-Farber Cancer Institute (DFCI) acute lymphoblastic leukemia (ALL) Consortium Protocol 91-01 was designed to improve the outcome of children with newly diagnosed ALL while minimizing toxicity. Compared with prior protocols, post-remission therapy was intensified by substituting dexamethasone for prednisone and prolonging the asparaginase intensification from 20 to 30 weeks. Between 1991 and 1995, 377 patients (age, 0-18 years) were enrolled; 137 patients were considered standard risk (SR), and 240 patients were high risk (HR). Following a 5.0-year median follow-up, the estimated 5-year event-free survival (EFS) +/- SE for all patients was 83% +/- 2%, which is superior to prior DFCI ALL Consortium protocols conducted between 1981 and 1991 (P =.03). There was no significant difference in 5-year EFS based upon risk group (87% +/- 3% for SR and 81% +/- 3% for HR, P =.24). Age at diagnosis was a statistically significant prognostic factor (P =.03), with inferior outcomes observed in infants and children 9 years or older. Patients who tolerated 25 or fewer weeks of asparaginase had a significantly worse outcome than those who received at least 26 weeks of asparaginase (P <.01, both univariate and multivariate). Older children (at least 9 years of age) were significantly more likely to have tolerated 25 or fewer weeks of asparaginase (P <.01). Treatment on Protocol 91-01 significantly improved the outcome of children with ALL, perhaps due to the prolonged asparaginase intensification and/or the use of dexamethasone. The inferior outcome of older children may be due, in part, to increased intolerance of intensive therapy.
达纳-法伯癌症研究所（DFCI）急性淋巴细胞白血病（ALL）联盟方案91 - 01旨在改善新诊断ALL患儿的治疗结果，同时将毒性降至最低。与先前的方案相比，缓解后治疗得到强化，用 dexamethasone 替代 prednisone，并将天冬酰胺酶强化治疗从20周延长至30周。1991年至1995年期间，招募了377例患者（年龄0 - 18岁）；137例患者被认为是标准风险（SR），240例患者是高风险（HR）。经过5.0年的中位随访，所有患者的估计5年无事件生存率（EFS）±SE为83%±2%，优于1981年至1991年期间实施的先前DFCI ALL联盟方案（P = 0.03）。基于风险组的5年EFS无显著差异（SR组为87%±3%，HR组为81%±3%，P = 0.24）。诊断时的年龄是一个具有统计学意义的预后因素（P = 0.03），婴儿和9岁或以上儿童的治疗结果较差。耐受天冬酰胺酶25周或更少周数的患者的治疗结果明显比接受至少26周天冬酰胺酶治疗的患者差（单因素和多因素分析P均<0.01）。年龄较大的儿童（至少9岁）耐受25周或更少周数天冬酰胺酶的可能性明显更高（P < 0.01）。方案91 - 01治疗显著改善了ALL患儿的治疗结果，这可能归因于天冬酰胺酶强化治疗时间的延长和/or dexamethasone的使用。年龄较大儿童治疗结果较差可能部分归因于对强化治疗耐受性的增加。
