Abstract | BACKGROUND: Children and adolescents with unresectable rhabdomyosarcoma fare poorly when treated with contemporary chemotherapeutic regimens. Evaluation of newly developed agents in these patients is important to improve their outcome. Based on a preclinical rhabdomyosarcoma xenograft model that accurately predicted the activity of new agents, the safety and efficacy of ifosfamide was evaluated as part of a Phase II clinical trial in previously untreated children with unresectable rhabdomyosarcoma. METHODS: RESULTS: Nineteen of 22 patients (86%) had a partial response to ifosfamide given as a single agent. No complete responses to this agent alone were observed. After administration of additional chemotherapy and local control measures ( radiation therapy and surgery), the estimated proportion of patients surviving progression-free at 2 years was 63% (95% confidence interval, 37-80%). Ifosfamide was tolerated well; the most frequent toxicity was nondose-limiting myelosuppression. Transient mild renal toxicity infrequently was observed, and no central nervous system toxicity occurred in this group of patients. CONCLUSIONS:
Ifosfamide appears to have significant clinical activity in untreated patients with unresectable rhabdomyosarcomas. These findings provide an accurate estimate of the response rate to single-agent ifosfamide in this group of previously untreated patients and thus provide a foundation for its rational incorporation into multiagent clinical trials. In addition, the potential benefits of this type of new drug development were demonstrated.
|
Authors | A S Pappo, E Etcubanas, V M Santana, B N Rao, L E Kun, J Fontanesi, P K Roberson, L C Bowman, W M Crist, D N Shapiro |
Journal | Cancer
(Cancer)
Vol. 71
Issue 6
Pg. 2119-25
(Mar 15 1993)
ISSN: 0008-543X [Print] United States |
PMID | 8443761
(Publication Type: Clinical Trial, Clinical Trial, Phase II, Journal Article, Research Support, Non-U.S. Gov't, Research Support, U.S. Gov't, P.H.S.)
|
Chemical References |
|
Topics |
- Adolescent
- Adult
- Child
- Child, Preschool
- Female
- Humans
- Ifosfamide
(adverse effects, therapeutic use)
- Infant
- Kidney
(drug effects)
- Male
- Rhabdomyosarcoma
(drug therapy)
|