Matthay Katherine K, Edeline Veronique, Lumbroso Jean, Tanguy Marie Laure, Asselain Bernard, Zucker Jean Michel, Valteau-Couanet Dominique, Hartmann Olivier, Michon Jean
Department of Pediatrics, University of California School of Medicine, San Francisco 94143-0106, USA.
J Clin Oncol. 2003 Jul 1;21(13):2486-91. doi: 10.1200/JCO.2003.09.122.
Metaiodobenzylguanidine (MIBG), specifically taken up in cells of sympathetic origin, provides a highly sensitive and specific indicator for the detection of metastases in neuroblastoma. The aim of this study was to correlate early response to therapy by MIBG scan, using a semiquantitative scoring method, with the end induction response and event-free survival (EFS) rate in stage IV neuroblastoma.
Seventy-five children older than 1 year and with stage IV neuroblastoma had 123I-MIBG scans at diagnosis, after two and four cycles of induction therapy, and before autologous stem-cell transplantation. The scans were read by two independent observers (concordance > 95%) using a semiquantitative method. Absolute and relative (score divided by initial score) MIBG scores were then correlated with overall pretransplantation response, bone marrow response, and EFS.
The pretransplantation response rate was 81%, and the 3-year EFS rate was 32%, similar to a concomitant group of 375 stage IV patients. The median relative MIBG scores after two, four, and six cycles were 0.5, 0.24, and 0.12, respectively. The probability of having a complete response or very good partial response before transplantation was significantly higher if the relative score after two cycles was < or = 0.5, or, if after four cycles, the relative score was < or = 0.24. Patients with a relative score of < or = 0.5 after two cycles or a score of < or = 0.24 after four cycles had an improved EFS rate (P =.053 and.045, respectively).
Semiquantitative MIBG score early in therapy provides valuable prognostic information for overall response and EFS, which may be useful in tailoring treatment.
间碘苄胍（MIBG）特异性摄取于交感神经起源的细胞中，为检测神经母细胞瘤转移提供了一种高度敏感和特异的指标。本研究的目的是通过使用半定量评分方法，将MIBG扫描对治疗的早期反应与IV期神经母细胞瘤的诱导结束反应和无事件生存率（EFS）相关联。
75例年龄大于1岁的IV期神经母细胞瘤患儿在诊断时、诱导治疗两个和四个周期后以及自体干细胞移植前进行了123I-MIBG扫描。由两名独立观察者（一致性>95%）使用半定量方法读取扫描结果。然后将绝对和相对（分数除以初始分数）MIBG分数与移植前的总体反应、骨髓反应和EFS相关联。
移植前反应率为81%，3年EFS率为32%，与一组375例IV期患者相似。两个、四个和六个周期后的中位相对MIBG分数分别为0.5、0.24和0.12。如果两个周期后的相对分数≤0.5，或者四个周期后的相对分数≤0.24，则移植前获得完全缓解或非常好的部分缓解的概率显著更高。两个周期后相对分数≤0.5或四个周期后分数≤0.24的患者EFS率有所提高（分别为P = 0.053和0.045）。
治疗早期的半定量MIBG分数可为总体反应和EFS提供有价值的预后信息，这可能有助于调整治疗方案。
