Toxicity and outcome of children and adolescents participating in phase I/II trials of novel anticancer drugs: the Royal Marsden experience

Daniel A Morgenstern, Darren Hargrave, Lynley V Marshall, Susanne A Gatz, Giuseppe Barone, Tracey Crowe, Kathy Pritchard-Jones, Stergios Zacharoulis, Donna L Lancaster, Sucheta J Vaidya, Julia C Chisholm, Andrew D J Pearson, Lucas Moreno

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19 Citations (Scopus)


Early phase trials are crucial in developing new therapies for poor prognosis childhood malignancies. Outcomes and toxicities of children treated on phase I/II trials at the Royal Marsden, one of the largest pediatric oncology early phase trial units in Europe, were examined to provide a baseline dataset and generate hypotheses. All patients recruited over a 10-year period to December 2011 were included. Variables including baseline characteristics, time on study, survival, toxicities, and admissions were collected. Seventy-two patients were recruited to 21 trials (5 phase I, 16 phase II; overall 12 involved molecularly targeted agents). Median age at consent was 12.4 years. Dose-limiting toxicities were rare in phase I trial participants (2 of 15 evaluable patients, 13%); the most common reason for leaving trials was disease progression (76%), rather than drug toxicity (1.7%). Median time on trial was 1.3 months (phase I patients) and 3.3 months (phase II). Early phase trials in children are safe and unexpected toxic side effects are infrequent. Patients and their families are willing to travel to access novel therapies, although the overall prognosis for these individuals is poor. Continued expansion of the portfolio is needed ultimately to improve the outcomes for those with resistant disease.

Original languageEnglish
Pages (from-to)218-223
Number of pages6
JournalJournal of pediatric hematology/oncology
Issue number3
Publication statusPublished - Apr 2014


  • Adolescent
  • Adult
  • Antineoplastic Agents/toxicity
  • Child
  • Child, Preschool
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Drug Design
  • Drug Discovery/methods
  • Drug-Related Side Effects and Adverse Reactions
  • Female
  • Follow-Up Studies
  • Hospitalization/statistics & numerical data
  • Humans
  • Infant
  • Male
  • Neoplasms/drug therapy
  • Patient Admission/statistics & numerical data
  • Patient Dropouts
  • Patient Selection
  • Retrospective Studies
  • Treatment Outcome
  • Young Adult
  • drug development
  • childhood cancer
  • dose limiting toxicity


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