• Benoit Brethon, Anne Auvrignon, Claire Galambrun, Karima Yakouben, Thierry Leblanc, Yves Bertrand, Guy Leverger, and André Baruchel.
• Unité de Pédiatrie à Orientation Hématologique, Hôpital Saint-Louis, Paris, France. benoit.brethon@sls.aphp.fr
• Bmc Cancer. 2006 Jun 28; 6: 172.

BackgroundGemtuzumab ozogamicin (GO) is a cytotoxic anti-CD33 monoclonal antibody that has given promising preliminary results in adult myeloid CD33+ AML. We conducted a retrospective multicenter study of 12 children treated with GO on a compassionate basis (median age 5.5 y). Three patients (2 MDS/AML, 1 JMML) were refractory to first-line treatment, 8 patients with de novo AML were in refractory first relapse, and one patient with de novo AML was in 2nd relapse after stem cell transplantation (SCT). CD33 expression exceeded 20% in all cases.MethodsGO was administered alone, at a unit dose of 3-9 mg/m2, once (3 patients), twice (3 patients), three (5 patients) or five times (1 patient). Mean follow-up was 128 days (8-585 d).ResultsThere were three complete responses (25%) leading to further curative treatment (SCT). Treatment failed in the other nine patients, and only one patient was alive at the end of follow-up. NCI-CTC grade III/IV adverse events comprised hematological toxicity (n = 12), hypertransaminasemia (n = 2), allergy and hyperbilirubinemia (1 case each). There was only one major adverse event (grade IV allergy). No case of sinusoidal obstruction syndrome occurred.ConclusionThese results warrant a prospective trial of GO in a larger population of children with AML.

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