Summary
Clinical trials in pediatric oncology over the past 30 years have led to the situation today where most children with newly diagnosed cancer can be treated effectively, and many are cured. Despite this dramatic improvement in outcome for many children diagnosed with cancer, about 30–40% of children will die of their disease [1]. Although some attempts have been made to improve outcome by increasing the dose intensity of existing therapies, intolerable side effects and marginal increases in cancer cell kill limit this approach. Clearly, effective new anti-cancer agents are necessary to significantly improve the survival and quality of life in children with cancer. Well-organized pediatric Phase I trials to establish the maximum tolerated dose (MTD), and Phase II trials to establish efficacy, are critical to the identification of new anti-cancer agents.
This is a preview of subscription content, log in via an institution to check access.
Similar content being viewed by others
References
Robison LL: General principles of the epidemiology of childhood cancer. In: Pizzo PA, Poplack DG (eds.) Principles and Practice of Pediatric Oncology. 2nd ed, J.B. Lippincott, Philadelphia, p 8, 1993.
Hurwitz CA, Relling MV, Weitman SD, Ravindranath V, Vietti TJ, Strother DR, Ragab AH, Pratt CB: Phase I trial of paclitaxel in children with refractory solid tumors: a Pediatric Oncology Group Study. J Clin Oncol 11:2324–2329, 1993.
Smith MA, Adamson PC, Balis FM, Feusner J, Aronson L, Murphy RF, Horowitz ME, Reaman G, Hammond GD: Phase I and pharmacologic evaluation of all-trans retinoic acid in pediatric patients. J Clin Oncol 10:1666–1673, 1992.
Balis FM, Holcenberg JS, Poplack DG: General principles of chemotherapy. In: Pizzo PA, Poplack DG (eds.) Principals and Practice of Pediatric Oncology. 2nd ed, J.B. Lippincott, Philadelphia, pp 231–232, 1993.
Venditti JM: Preclinical drug development: trationale and methods. Semin Oncol 8:349–361, 1981.
Marsoni S, Ungerleider RS, Hurson SB, Simon RM, Hammershaimb LD: Tolerance to antineoplastic agents in children and adults. Cancer Treat Rep 69:1263–1269, 1985.
Carlson L, Ho P, Smith M, Reisch J, Weitman S: Pediatric Phase I drug tolerance: a review and comparison of recent adult and pediatric Phase I trials. Am J Fed Hem Onc: in press, dy1996.
Pieters R, Kongo T, Loonen AH, Huismans DR, Broxterman HJ, Hahlen K, Veerman AJ: Different types of non-P-glycoprotein mediated multiple drug resistance in children with relapsed acute lymphoblastic leukemia. BrJ Cancer 65:691–697, 1992.
Hecker S, Sauerbrey A, Volm M: p53 expression and poor prognosis in childhood acute lymphoblastic leukemia. Anticancer Res 14:2759–2761, 1994.
Horowitz ME, Etcubanas E, Christensen ML, Houghton JA, George SL, Green AA, Houghton PJ: Phase II testing of melphalan in children with newly diagnosed rhabdomyosarcoma: a model for anticancer drug development. J Clin Oncol 6:308–314, 1988.
Castleberry RP, Cantor AB, Green AA, Joshi V, Berkow RL, Buchanan GR, Leventhal B, Mahoney DH, Smith EI, Hayes FA: Phase II investigational window using carboplatin, iproplatin, ifosfamide, and epirubicin in children with untreated disseminated neuroblastoma: a Pediatric Oncology Group study. J Clin Oncol 12:1616–1620, 1994.
Author information
Authors and Affiliations
Additional information
Address for offprints: Craig A. McArthur, Department of Pediatrics, Box 8116, Washington University School of Medicine, One Children's Place, St. Louis, MO 63110, USA
Rights and permissions
About this article
Cite this article
MacArthur, C.A., Vietti, T. The importance of Phase I/II trials in pediatric oncology. Invest New Drugs 14, 33–35 (1996). https://doi.org/10.1007/BF00173680
Issue Date:
DOI: https://doi.org/10.1007/BF00173680