Dagher R, Kreissman S, Robertson K A, Provisor A, Bergstein J, Burke K, Rodman J H, Emanuel D, Smith F O
Section of Pediatric Hematology/Oncology, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis 46202, USA.
J Pediatr Hematol Oncol. 1998 Jul-Aug;20(4):357-60. doi: 10.1097/00043426-199807000-00016.
An autologous peripheral blood progenitor cell (APBPC) transplant in an anephric child with multiply recurrent Wilms tumor using a conditioning regimen of high dose chemotherapy in conjunction with hemodialysis (HD) and peritoneal dialysis is described.
The child had a left nephrectomy at 9 months of age for a stage II Wilms tumor. At 6 years of age, she required a right nephrectomy because of progressive, recurrent disease unresponsive to treatment with doxorubicin, actinomycin, and vincristine. She was maintained on peritoneal dialysis. Salvage chemotherapy consisted of 5 cycles of carboplatin and cyclophosphamide after APBPCs were collected after granulocyte colony-stimulating factor mobilization. After a preparative regimen of carboplatin, cyclophosphamide, and etoposide with closely timed HD, peripheral blood progenitor cells were infused and peritoneal dialysis was resumed.
No nonhematopoietic toxicity occurred. Pharmacokinetic studies demonstrated that HD effectively eliminated carboplatin and provided safe, effective plasma concentrations in this anephric patient. Trilineage engraftment occurred by day +10 and the child was discharged from the hospital on day +14. She had a local recurrence on day +194 and died of progressive disease on day +660.
With dialysis support and dose modification, high-dose chemotherapy followed by APBPC transplantation can be successfully performed in the anephric child. Given the lack of organ toxicity in this patient, increased doses of the drugs used in this preparative regimen may be possible for anephric children.
描述了一名无肾儿童，患有多次复发的肾母细胞瘤，采用高剂量化疗联合血液透析（HD）和腹膜透析的预处理方案进行自体外周血祖细胞（APBPC）移植。
该儿童9个月大时因II期肾母细胞瘤接受了左肾切除术。6岁时，由于疾病进展、复发且对阿霉素、放线菌素和长春新碱治疗无反应，她需要进行右肾切除术。她一直维持腹膜透析。在粒细胞集落刺激因子动员后采集APBPCs，挽救性化疗包括5个周期的卡铂和环磷酰胺。在接受卡铂、环磷酰胺和依托泊苷的预处理方案并密切安排HD后，输注外周血祖细胞并恢复腹膜透析。
未发生非血液学毒性。药代动力学研究表明，HD有效消除了卡铂，并在这名无肾患者中提供了安全、有效的血浆浓度。三系造血在+10天时发生，患儿在+14天时出院。她在+194天时出现局部复发，并在+660天时死于疾病进展。
在透析支持和剂量调整的情况下，高剂量化疗后进行APBPC移植可在无肾儿童中成功实施。鉴于该患者缺乏器官毒性，对于无肾儿童，本预处理方案中使用的药物剂量可能可以增加。
