Clinical characteristics and outcomes of pediatric oncology patients with aggressive biology enrolled in phase I clinical trials designed for adults: The university of Texas MD Anderson cancer center experience

Fernando F. Corrales-Medina1, Cynthia Herzog1, Kenneth Hess2, Daniela Egas-Bejar1, David S. Hong3, Gerald Falchook3, Pete Anderson1,4, Cesar Nunez1, Winston W. Huh1, Aung Naing3, Apostolia M. Tsimberidou3, Jennifer Wheler 3, Sarina Piha Paul3, Filip Janku3, Eugenie S Kleinerman1,* Razelle Kurzrock3,* Vivek Subbiah1,3,*

1 Children’s Cancer Hospital, Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

2 Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

3 Department of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA

4 Pediatric Hematology/Oncology/BMT, Levine Children’s Hospital/Levine Cancer Institute, Charlotte, North Carolina, USA

5 Center for Personalized Cancer Therapy and Division of Hematology & Oncology, University of California San Diego - Moores Cancer Center, La Jolla, California, USA

* These authors contributed equally to this work


Vivek Subbiah, email:

Keywords: phase I trials, children, prognostic scores, targeted therapy

Received: April 29, 2014 Accepted: July 26, 2014 Published: July 27, 2014


Background: Children (patients ≤ 18 years of age) are not usually included on pharmaceutical industry sponsored Phase I trials.

Methods: We reviewed the medical records of 40 patients ≤ 18 years treated in ≥ 1 phase I trial at MD Anderson.

Results: The median OS was 8.5 months (95% CI, 5.5-13.2 months). In the multivariate analysis, age ≥15 only predicted increased OS (P = 0.0065), and >3 prior therapies (P = 0.053) predicted decreased OS. The median PFS was 2.8 months (95% CI, 2.3-4.1 months). In the multivariate analysis, independent factors that predicted increased PFS were age ≥15 years (P < 0.001) and prior radiation therapy (P = 0.049); performance status >1 (P < 0.001) and >3 prior therapies (P = 0.002) predicted decreased PFS. RMH score ≥ 2 and MDACC score ≥ 3 were associated with decreased median OS (P = 0.029 and P = 0.031 respectively). 

Conclusions: It is feasible to conduct phase I studies in pediatric patients based on adult protocols. In the era of targeted therapy more trials should allow pediatric patients earlier in the drug development especially if deemed safe in adults in early phase trials.

Translational Relevance: Most pharmaceutical industry sponsored trials exclude patients less than 18 years in phase I clinical trials. Even in the era of targeted therapy pediatric patients usually have to wait for most phases of trials to be completed in adults before being allowed to enroll in clinical trials of new therapies, even in the advanced metastatic and relapsed setting. Some investigator initiated phase 1 trials of combinations of US FDA approved agents allow patients less than 18 years. We report the preliminary analyses of the outcomes of pediatric patients enrolled in phase I studies initially designed for adults, but allowing for enrollment of patients under 18.

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