Upfront use of gemtuzumab ozogamicin in young children with CD33-positive AML

Gemtuzumab ozogamicin (GO) is a humanized anti-CD33 antibody used for treating patients with CD33+ acute myeloid leukemia (AML). We report three young children (two infants and one toddler) with AML treated with GO 9 mg/m ². Two received two doses at diagnosis alone with conventional chemotherapy and one received one dose after relapse. GO was well tolerated and all three achieved remission. All were transplanted: one relapsed after 5 months and died of disease, one died a toxic death in remission due to pulmonary fibrosis, and one survived (41 months from diagnosis). In conclusion, GO was well tolerated in these young patients with evidence for efficacy.