Dose-related efficacy and toxicity of gemtuzumab ozogamicin in pediatric acute myeloid leukemia

J. Parigger, C. M. Zwaan, D. Reinhardt, G. J.L. Kaspers

Research output: Contribution to journalArticlepeer-review

18 Citations (Scopus)


Gemtuzumab ozogamicin, an anti-tumour antibiotic linked to an anti-CD33 antibody (Mylotarg®), has been well studied in AML in adults but to a lesser extent in children. No review has yet been published on the dose-related toxicity and efficacy of gemtuzumab ozogamicin in pediatric AML patients. Here we looked at 14 studies then scatterplots and linear regressions were used to estimate the relationship between the dose of gemtuzumab and its toxicity and efficacy. A non-significant increase in bilirubin level and in incidence of veno-occlusive disease was seen with higher doses of gemtuzumab ozogamicin when used as single-agent. In terms of efficacy, even a low dose of 3 mg/m2 of gemtuzumab ozogamicin can have antileukemic effect, but available data do not allow conclusions on its dose-dependency. Data indicate that higher doses of gemtuzumab ozogamicin account for more adverse events. The data do not show that a high dose is required for anti-leukemic efficacy of gemtuzumab ozogamicin. This study also indicates that there seems to be a role for gemtuzumab ozogamicin in the treatment of pediatric AML and further studies are required to assess its optimal dose, schedule and balance between efficacy and side-effects.

Original languageEnglish
Pages (from-to)137-146
Number of pages10
JournalExpert Review of Anticancer Therapy
Issue number2
Publication statusPublished - 1 Feb 2016
Externally publishedYes


  • acute myeloid leukemia
  • children
  • efficacy
  • Gemtuzumab ozogamicin
  • Mylotarg®
  • toxicity


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