Thiotepa-based high-dose chemotherapy with autologous stem-cell rescue in patients with recurrent or progressive CNS germ cell tumors Journal Article


Authors: Modak, S.; Gardner, S.; Dunkel, I. J.; Balmaceda, C.; Rosenblum, M. K.; Miller, D. C.; Halpern, S.; Finlay, J. L.
Article Title: Thiotepa-based high-dose chemotherapy with autologous stem-cell rescue in patients with recurrent or progressive CNS germ cell tumors
Abstract: Purpose: To evaluate the efficacy and toxicity of high-dose chemotherapy (HDC) followed by autologous stem-cell rescue (ASCR) in patients with relapsed or progressive CNS germ cell tumors (GCTs). Patients and Methods: Twenty-one patients with CNS GCTs who experienced relapse or progression despite having received initial chemotherapy and/or radiotherapy were treated with thiotepa-based HDC regimens followed by ASCR. Results: Estimated overall survival (OS) and event-free survival (EFS) rates for the entire group 4 years after HDC were 57% ± 12% and 52% ± 14%, respectively. Seven of nine (78%) patients with germinoma survived disease-free after HDC with a median survival of 48 months. One patient died as a result of progressive disease (PD) 39 months after HDC, and another died as a result of pulmonary fibrosis unrelated to HDC 78 months after ASCR without assessable disease. However, only four of 12 patients (33%) with nongerminomatous germ cell tumors (NGGCTs) survived without evidence of disease, with a median survival of 35 months. Eight patients with NGGCTs died as a result of PD, with a median survival of 4 months after HDC (range, 2 to 17 months). Patients with germinoma fared better than those with NGGCTs (P = .016 and .014 for OS and EFS, respectively). Patients with complete response to HDC also had significantly better outcome (P < .001 for OS and EFS) compared with patients with only a partial response or stable disease. There were no toxic deaths because of HDC. Conclusion: Dose escalation of chemotherapy followed by ASCR is effective therapy for patients with recurrent CNS germinomas and might be effective in patients with recurrent NGGCTs with a low tumor burden. © 2004 by American Society of Clinical Oncology.
Keywords: survival; adolescent; adult; child; clinical article; preschool child; treatment outcome; child, preschool; disease-free survival; survival analysis; retrospective studies; disease course; mortality; salvage therapy; united states; disease free survival; chemotherapy; brain tumor; brain neoplasms; neoplasm recurrence, local; relapse; alkylating agent; autologous stem cell transplantation; hematopoietic stem cell transplantation; pathology; retrospective study; thiotepa; central nervous system; infant; tumor recurrence; antineoplastic agents, alkylating; medical record; tumor growth; toxicity; new york; germ cell tumor; lung fibrosis; fatality; medical records; germinoma; humans; human; male; female; priority journal; article
Journal Title: Journal of Clinical Oncology
Volume: 22
Issue: 10
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 2004-05-15
Start Page: 1934
End Page: 1943
Language: English
DOI: 10.1200/jco.2004.11.053
PROVIDER: scopus
PUBMED: 15143087
DOI/URL:
Notes: J. Clin. Oncol. -- Cited By (since 1996):45 -- Export Date: 16 June 2014 -- CODEN: JCOND -- Source: Scopus
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MSK Authors
  1. Ira J Dunkel
    255 Dunkel
  2. Shakeel Modak
    160 Modak
  3. Marc Rosenblum
    255 Rosenblum
  4. Jonathan Finlay
    62 Finlay