Eguchi H, Takaue Y, Kawano Y, Watanabe A, Watanabe T, Kikuta A, Koizumi S, Matsumura T, Sawada A, Horikoshi Y, Sekine Y, Koyama T, Shimokawa T, Shimizu K, Kawasaki K, Mugishima H, Takayama J, Ohira M, Ogawa M
Department of Pediatrics and Child Health, Kurume University School of Medicine, Japan.
Bone Marrow Transplant. 1998 May;21(10):1011-4. doi: 10.1038/sj.bmt.1701207.
This is the first report of the long-term therapeutic results in 22 children more than 1 year old with stage IV neuroblastoma who were treated with autologous peripheral blood stem cell transplantation (PBSCT). The median age of the patients at PBSCT was 4 years (1 to 10 years) and seven of the 17 patients who were evaluated for N-myc amplification were positive. PBSC were collected by a median of four aphereses per patient. The patients underwent PBSCT from 6 to 21 months after the start of therapy (median 10.5 months) at which time 13 patients were in CR, seven were in PR, and two had refractory disease. Multi-drug therapy using the 'high-MEC' regimen consisting of carboplatinum (400 mg/m2) and VP-16 (200 mg/m2) on days -7 to -4, and melphalan (90 mg/m2) on days -3 and -2, was the primary cytoreductive regimen. The median number of infused MNC and CFU-GM was, respectively, 4.3 x 10(8)/kg and 2.4 x 10(5)/kg. After PBSCT, three patients died of regimen-related toxicities and one patient who was transplanted with refractory disease died of disease progression without any benefit from transplantation. Hematological recovery was evaluated in 21 patients, excluding one early death. The median number of days required to achieve an AGC of >0.5 x 10(9)/l and platelet count of >50 x 10(9)/l were, respectively, 11 and 46. Eleven patients relapsed 3 to 50 months after PBSCT, and currently seven patients (5/13 who were transplanted in CR and 2/7 in PR) are surviving disease-free at 52 to 84 months. Although the retrospective nature of this study and several variables prevent a meaningful analysis, the overall results still support the feasibility of developing a prospective study of PBSCT with a larger number of children with high-risk neuroblastoma.
这是关于22名1岁以上IV期神经母细胞瘤患儿接受自体外周血干细胞移植（PBSCT）的长期治疗结果的首份报告。接受PBSCT时患者的中位年龄为4岁（1至10岁），在接受N - myc扩增评估的17名患者中，有7名呈阳性。每位患者平均通过4次单采术采集外周血干细胞。患者在治疗开始后6至21个月（中位时间10.5个月）接受PBSCT，此时13名患者处于完全缓解（CR）状态，7名患者部分缓解（PR），2名患者疾病难治。主要的细胞减灭方案是采用“高剂量美法仑强化化疗（high - MEC）”方案进行多药治疗，即在第 - 7至 - 4天使用卡铂（400mg/m²）和依托泊苷（VP - 16，200mg/m²），在第 - 3天和 - 2天使用美法仑（90mg/m²）。输注的单个核细胞（MNC）和粒 - 巨噬细胞集落形成单位（CFU - GM）的中位数分别为4.3×10⁸/kg和2.4×10⁵/kg。PBSCT后，3名患者死于与治疗方案相关的毒性反应，1名移植时疾病难治的患者死于疾病进展，移植未带来任何益处。对21名患者（不包括1例早期死亡患者）进行了血液学恢复评估。达到绝对粒细胞计数（AGC）>0.5×10⁹/L和血小板计数>50×10⁹/L所需的中位天数分别为11天和46天。11名患者在PBSCT后3至50个月复发，目前有7名患者（13名CR状态下移植患者中的5名和7名PR状态下移植患者中的2名）在52至84个月时无病存活。尽管本研究具有回顾性且存在多个变量，无法进行有意义的分析，但总体结果仍支持开展一项针对更多高危神经母细胞瘤患儿的PBSCT前瞻性研究的可行性。
