A phase II trial of thalidomide and cyclophosphamide in patients with recurrent or refractory pediatric malignancies Journal Article


Authors: Gilheeney, S. W.; Lyden, D. C.; Sgouros, S.; Antunes, N.; Gerald, W.; Kramer, K.; Lis, E.; Meyers, P.; Rosen, N.; Thaler, H. T.; Trippett, T.; Wexler, L.; Dunkel, I. J.
Article Title: A phase II trial of thalidomide and cyclophosphamide in patients with recurrent or refractory pediatric malignancies
Abstract: Background. Previous clinical and pre-clinical research has demonstrated synergy between anti-angiogenic agents and cytotoxic chemotherapy. This trial was undertaken to investigate whether the combination of cyclophosphamide and thalidomide would be active against pediatric tumors. Procedure. Patients with pediatric malignancies who had no remaining conventional therapeutic options were recruited from January 1999 to May 2001. They received thalidomide (6-12 mg/kg po every day; maximum daily dose 800 mg) and cyclophosphamide (1,200 mg/m2 IV every 28 days). Results. Twenty-seven patients were enrolled on the study. Seventeen were male and 10 were female. Median age at the time of registration was 15 years (range 1-54 years). The median number of prior treatment regimens was four. Twenty-one patients were evaluable for response; 1 had a partial response (Hodgkin disease), 1 demonstrated stable disease (neuroendocrine tumor), and 19 had progressive disease. The most common toxicities were hematological (leukocytopenia and neutropenia) and gastrointestinal. One patient experienced a grade 3 rash. Fatigue and daytime somnolence were variable. No peripheral neuropathy was observed. Conclusion. The combination of thalidomide and cyclophosphamide as described herein has a modest and tolerable toxicity profile but little evidence of efficacy. © 2006 Wiley-Liss, Inc.
Keywords: adolescent; adult; child; clinical article; treatment outcome; child, preschool; middle aged; survival analysis; thalidomide; clinical trial; constipation; drug tolerability; fatigue; neutropenia; angiogenesis inhibitor; cytotoxic agent; cancer combination chemotherapy; drug efficacy; drug withdrawal; side effect; solid tumor; recurrent cancer; neoplasms; multiple cycle treatment; phase 2 clinical trial; leukopenia; thrombocytopenia; vomiting; antineoplastic combined chemotherapy protocols; peripheral neuropathy; cyclophosphamide; dose-response relationship, drug; angiogenesis; childhood cancer; hodgkin disease; drug dose escalation; drug hypersensitivity; neuroendocrine tumor; rash; gastrointestinal toxicity; infant; drug toxicity; corticosteroid; angiogenesis inhibitors; infection risk; pediatric oncology; therapeutics; docusate sodium; daytime somnolence
Journal Title: Pediatric Blood and Cancer
Volume: 49
Issue: 3
ISSN: 1545-5009
Publisher: Wiley Periodicals, Inc  
Date Published: 2007-09-01
Start Page: 261
End Page: 265
Language: English
DOI: 10.1002/pbc.21045
PUBMED: 16972243
PROVIDER: scopus
DOI/URL:
Notes: --- - "Cited By (since 1996): 3" - "Export Date: 17 November 2011" - "CODEN: PBCEA" - "Source: Scopus"
Altmetric Score
MSK Authors
  1. Ira J Dunkel
    256 Dunkel
  2. David C Lyden
    84 Lyden
  3. Kim Kramer
    171 Kramer
  4. Eric Lis
    105 Lis
  5. Leonard H Wexler
    135 Wexler
  6. Nuno L Antunes
    22 Antunes
  7. Nancy S Rosen
    27 Rosen
  8. William L Gerald
    370 Gerald
  9. Paul Meyers
    247 Meyers
  10. Howard T Thaler
    194 Thaler