Recommended Background Readings for Pediatric Oncology Subcommittee Meeting

November 28, 2001

Boos J

Drug Trials in Pediatric Oncology:a multi-faceted problem Int J of Clin Pharm and Therapeutics 36(11):613-620 1998

A perspective and summary of several critical factors


Rowinsky EK

The Pursuit of Optimal Outcomes in Cancer Therapy in a New Age of Rationally Designed Target-Based Anticancer Agents. Drugs 60 (Suppl 1):1-14 2000

Commentary on approaches to drug development


Shah S, Weitman S, Langevin AM, Bernstein M, Furman W, Pratt C

Phase I therapy trials in children with cancer. J Pediatr Hematol Oncol 20(5):431-8,1998

Previous results and expectations with regard to safety and efficacy in pediatric phase I studies


Estlin EJ, Ablett S, Newell DR, Lewis IJ, Lashford L, Pearson ADJ

Phase I trials in pediatric oncology- the European perspective. Invest. New Drugs 14:23-32 1996

General commentary on experience in the United Kingdom Children’s Cancer Study Group


Smith M, Bernstein M, Bleyer WA, Borsi JD, Ho P, Lewis IJ, Pearson A, Pein F, Pratt C, Reaman G, Riccardi R, Seibel N, Trueworthy R, Ungerleider R, Vassal G, Vietti T

Conduct of Phase I trials in children with cancer. J Clin Oncol 16(3) 966-78 1998

An international consensus document on the design and implementation of phase I studies


Eisenhauer EA, O’Dwyer PJ, Christian M, Humphrey JS

Phase I clinical trial design in cancer drug development. J Clin Oncol 18(3):684-692 2000

General discussions on phase I trial design


Seymour L and Eisenhauer EA

A review of dose-limiting events in phase I trials:antimetabolites show unpredictable relationships between dose and toxicity. Cancer Chemother Pharmacol 47:2-10 2001

Evidence for the rationale for proceeding with studies


Carlson L, Ho P, Smith M, Resich J, Weitman S

Pediatric Phase I Drug Tolerance: A review of recent adult and pediatric Phase I trials. J Pediatr Hematol Oncol 18(3):250-6 1996

Age and therapy dependent differences in drug tolerance


Somia N and Verma IM

Gene Therapy:Trials and Tribulations Nature Reviews Genetics 1(2):91-99 2000

Commentary on some of the special considerations involved in gene therapy studies


Burke GAA, Estlin EJ, Lowis SP

The role of pharmacokinetic and pharmacodynamic studies in the planning of protocols for the treatment of childhood cancer. Cancer Treatment Reviews 25:13-27 1999

Summary of many of the critical considerations in protocol planning


Leeder JS

Pharmacogenetics and Pharmacogenomics. Pediatr Clinics of North America 48(3):765-781 2001

Review of the changes that occur with age as well as among people


Evans WE and Relling MV

Pharmacogenomics:translating functional genomics into rational therapeutics. Science 286:487-491 1999

Review of the application of genomic knowledge to studies and therapy


Sinnett D, Krajinovic M, Labuda D

Genetic susceptibility to childhood acute lymphoblastic leukemia. Leukemia and Lymphoma 38(5-6):447-462 2000

Specific example of the potential contribution of genomics to understanding childhood malignancy


Weitman S, Ochoa S, Sullivan J, Shuster J, Winick N, Pratt C, Vietti T, Harris M

Pediatric Phase II cancer chemotherapy trials : Pediatric Oncology Group study J Pediatr Hematol Oncol 19(3):187-91 1997

A review of data and perspective on phase II studies


Dent S, Zee B, Dancey J, Hanauske A, Wonders J, Eisenhauer EA

Application of a new multinomial phase II stopping rule using response and early progression. J Clin Oncol 19(3):785-791 2001

Examples of an approach to phase II design and analysis


Smith MA and Anderson B

Phase II window studies: 10 years of experience and counting. J Pediatr Hematol Oncol 23(6):334-337 2001

Summary of phase II window experience with commentary and recommendations


Goodman SN

Toward evidence-based medical statistics 1: The P Value Fallacy Ann Intern Med 130:995-1004 1999


Goodman SN

Toward evidence-based medical statistics. 2: The Bayes Factor. Ann Intern Med 130:1005-1013 1999

Two related perspectives on analyzing results from clinical studies