Journal of Clinical Oncology, Vol 10, 1857-1864, Copyright © 1992 by American Society of Clinical Oncology
Treatment of recurrent and refractory pediatric solid tumors with high- dose busulfan and cyclophosphamide followed by autologous bone marrow rescue
ML Graham, AM Yeager, BG Leventhal, JM Wiley, CI Civin, LC Strauss, CA Hurwitz, RL Dubowy, MD Wharam Jr and PM Colombani
Johns Hopkins Oncology Center, Baltimore, MD.
PURPOSE: The purpose of this study was to determine the toxicities of and
responses to high-dose busulfan and cyclophosphamide with autologous bone
marrow transplant (ABMT) in patients with recurrent or refractory pediatric
solid tumors. PATIENTS AND METHODS: We treated 18 patients (ages, 2 to 38
years; median, 14) who had tumors that were resistant to conventional
chemotherapy and radiotherapy with busulfan 16 mg/kg and cyclophosphamide
200 mg/kg. Seventeen patients received bone marrow purged with
4-hydroperoxycyclophosphamide; one received unpurged marrow. RESULTS:
Despite extensive prior treatment, including radiotherapy in 16 patients,
toxicity generally was acceptable. For seven patients with measurable
disease, there were three partial responses of 2, 10, and 20 months'
duration, three patients with stable disease (SD), and one early, toxic
death. Of the 11 patients with no measurable disease at the time of
transplantation, one patient with osteosarcoma continues in remission at
57+ months and one third of the patients survived for at least 16 months.
Mucositis was the predominant nonhematopoietic toxicity. CONCLUSION:
Although the high-dose busulfan and cyclophosphamide combination showed
modest activity, changes in the preparative regimen should be considered to
improve the response rate in refractory tumors.
