Tandem high-dose chemotherapy with autologous stem cell rescue for stage M high-risk neuroblastoma: Experience using melphalan/etoposide/carboplatin and busulfan/melphalan regimens.
Tandem high-dose chemotherapy with autologous stem cell rescue for stage M high-risk neuroblastoma: Experience using melphalan/etoposide/carboplatin and busulfan/melphalan regimens.
复制标题
串联高剂量化疗与自体干细胞挽救治疗 M 期高危神经母细胞瘤：使用美法仑/依托泊苷/卡铂和白消安/美法仑方案的经验。
DOI：
10.1111/petr.13772
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发表时间：
2020
期刊：
影响因子：
--
通讯作者：
Hiwatari M.
中科院分区：
文献类型：
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作者：
Kato S;Kubota Y;Watanabe K;Hogetsu K;Arakawa Y;Koh K;Takita J;Hiwatari M.
已报告了串联HDCT对高危神经母细胞瘤的疗效;然而，仍需建立最佳方案。在本文中，我们报告了我们在高危神经母细胞瘤患者中使用包括MEC和BuMel方案的串联HDCT的经验。我们回顾性分析了4例M期高危神经母细胞瘤患者，这些患者接受了HDCT和MEC，随后接受了BuMel联合自体干细胞拯救。虽然在诱导化疗后没有转移性病灶消失，但3例患者在串联HDCT后显示CR。观察到胃肠道粘膜损伤和肾功能不全为3级或3级以上的非血液学不良事件。在第一次HDCT后的所有4例患者中观察到胃肠道粘膜损伤，在第二次HDCT后的1例患者中观察到胃肠道粘膜损伤，并接受肠外营养和镇痛药治疗。1例患者在首次HDCT期间发生肾功能不全，通过充分水合和利尿剂缓解，并导致第二次HDCT的美法仑剂量减少。在所有患者中均未观察到SOS。观察到本研究中检查的HDCT方案是可行的，未导致任何危及生命的不良事件。我们的研究结果表明，包括MEC和BuMel的串联HDCT是高危神经母细胞瘤患者的潜在有效方案，包括对诱导化疗反应较差的患者，尽管应在更大人群中进行额外研究以验证任何长期结局和毒性。
The efficacy of tandem HDCT against high‐risk neuroblastoma has been reported; however, an optimal regimen remains to be established. In this paper, we report our experience using tandem HDCT comprising the MEC and BuMel regimens in patients with high‐risk neuroblastoma. We retrospectively analyzed four patients with stage M high‐risk neuroblastoma who received HDCT with MEC followed by BuMel combined with autologous stem cell rescue. Although none of their metastatic lesions had disappeared after induction chemotherapy, three patients showed a CR after tandem HDCT. Gastrointestinal mucosal injuries and renal dysfunction were observed as non‐hematologic adverse events of grade 3 or higher. Gastrointestinal mucosal injuries were observed in all four patients following the first HDCT and in one patient following the second HDCT and were treated with parenteral nutrition and analgesics. One patient experienced renal dysfunction during the first HDCT, which was alleviated by sufficient hydration and diuretics and resulted in the reduction of melphalan dosage for the second HDCT. SOS was not observed in any patient. The HDCT regimens examined in this study were observed to be feasible and did not result in any life‐threatening adverse events. Our findings indicate that tandem HDCT comprising MEC and BuMel is a potentially effective regimen for patients with high‐risk neuroblastoma, including for those who respond poorly to induction chemotherapy, although additional studies in a larger population should be conducted to verify any long‐term outcomes and toxicity.