Gemtuzumab ozogamicin in children with relapsed/refractory myeloid leukemia
Author Information
Author(s): Brethon Benoit, Auvrignon Anne, Galambrun Claire, Yakouben Karima, Leblanc Thierry, Bertrand Yves, Leverger Guy, Baruchel André
Primary Institution: Unité de Pédiatrie à Orientation Hématologique, Hôpital Saint-Louis, Paris, France
Hypothesis
Is gemtuzumab ozogamicin effective and tolerable for children with relapsed or refractory myeloid leukemia?
Conclusion
Gemtuzumab ozogamicin shows a 25% response rate in children with relapsed or refractory myeloid leukemia, warranting further investigation.
Supporting Evidence
- Three complete responses were observed in 12 patients, indicating a 25% response rate.
- Adverse events included hematological toxicity in all patients, with one major adverse event.
- Only one patient was alive at the end of follow-up, highlighting the poor prognosis.
Takeaway
This study tested a medicine called gemtuzumab ozogamicin in kids with a tough type of leukemia, and it helped some of them get better.
Methodology
Retrospective multicenter study of 12 children treated with gemtuzumab ozogamicin on a compassionate basis.
Limitations
Small sample size and retrospective design limit the generalizability of the findings.
Participant Demographics
Median age was 5.5 years, with 7 boys and 5 girls.
Digital Object Identifier (DOI)
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