Incorporation of high-dose I-131-metaiodobenzylguanidine treatment into tandem high-dose chemotherapy and autologous stem cell transplantation for high-risk neuroblastoma: results of the SMC NB-2009 study

作者：Lee Ji Won EUIS; Lee Sanghoon; Cho Hee Won; Ma Youngeun; Yoo Keon Hee; Sung Ki Woong^*; Koo Hong Hoe; Cho Eun Joo; Lee Suk Koo; Lim Do Hoon

来源：Journal of Hematology & Oncology, 2017, 10(1): 108.

DOI：10.1186/s13045-017-0477-0

• Neuroblastoma
• High-dose I-131-MIBG
• High-dose chemotherapy
• Autologous stem cell transplantation

Background: In our previous SMC NB-2004 study of patients with high-risk neuroblastomas, which incorporated total-body irradiation (TBI) with second high-dose chemotherapy and autologous stem cell transplantation (HDCT/auto-SCT), the survival rate was encouraging; however, short-and long-term toxicities were significant. In the present SMC NB-2009 study, only TBI was replaced with I-131-meta-iodobenzylguanidine (MIBG) treatment in order to reduce toxicities.
Methods: From January 2009 to December 2013, 54 consecutive patients were assigned to receive tandem HDCT/auto-SCT after nine cycles of induction chemotherapy. The CEC (carboplatin + etoposide + cyclophosphamide) regimen and the TM (thiotepa + melphalan) regimen with (for metastatic MIBG avid tumors) or without (for localized or MIBG non-avid tumors) I-131-MIBG treatment (18 or 12 mCi/kg) were used for tandem HDCT/auto-SCT. Local radiotherapy, differentiation therapy with 13-cis-retinoic acid, and immunotherapy with interleukin-2 were administered after tandem HDCT/auto-SCT.
Results: Fifty-two patients underwent the first HDCT/auto-SCT and 47 patients completed tandem HDCT/auto-SCT. There was no significant immediate toxicity during the 131I-MIBG infusion. Acute toxicities during the tandem HDCT/auto-SCT were less severe in the NB-2009 study than in the NB-2004 study. Late effects such as growth hormone deficiency, cataracts, and glomerulopathy evaluated at 3 years after the second HDCT/auto-SCT were also less significant in the NB-2009 study than in NB-2004 study. There was no difference in the 5-year event-free survival (EFS) between the two studies (67.5 +/- 6.7% versus 58.3 +/- 6.9%, P = 0.340).
Conclusions: Incorporation of high-dose 131I-MIBG treatment into tandem HDCT/auto-SCT could reduce short-and long-term toxicities associated with TBI, without jeopardizing the survival rate.

• 出版日期2017-5-16