Preliminary report of an ascending dose study of gemtuzumab ozogamicin (mylotarg™, cma-676) in pediatric patients with acute myeloid leukemia

Eric L. Sievers, Robert Arceci, Janet Franklin, Beverly Lange, Kevin Shannon, Frank Smith, Mark S. Berger, Catharine Eten, Melissa Auen

Research output: Contribution to journalArticlepeer-review

14 Scopus citations


Gemtuzumab ozogamicin is an antibody-targeted chemotherapy agent which is used for the treatment of CD33-positive acute myeloid leukemia (AML). This report summarizes the preliminary treatment experience with gemtuzumab ozogamicin in 18 pédiatrie patients with CD33-positive AML enrolled in an ongoing open-label phase 1 study as of June 15, 2000. Patients had a median age of 12 years (min-max, 1-16 years). Seven patients had refractory AML, and 11 patients had AML in relapse. Gemtuzumab ozogamicin was administered at doses of 6 or 9 mg/m2 intravenously, over 2 hours for 2 doses on days 1 and 15. One of 3 patients treated with 6 mg/m2 developed NCI grade 3 gastrointestinal bleeding and congestive heart failure (possibly drug related). Three additional patients were added at this dose and evaluated for safety before escalating to 9mg/m2. At 9 mg/nr, 1 out of the first 3 patients developed prolonged hypoplasia and remained pancytopenic on approximately day 50, the beginning of conditioning for hematopoietic stem cell transplantation (HSCT). Three additional patients were enrolled, and 1 patient who had allogeneic HSCT approximately 4 months earlier experienced NCI grade 4 respiratory failure and hyperbilirubinemia and died from infection 31 days after receiving dose 2. Six additional patients were enrolled at 9 mg/m2, and 1 patient developed NCI grade 4 elevation of hepatic transaminases 7 days after receiving dose 1. The elevated transaminases improved to grade 1, and this patient received etoposide and mitoxantrone at approximately day 35 for persistent AML. Additional adverse events included an infusion-related symptom complex (transient fever, chills, and infrequently hypotension and shortness of breath) and grade 4 neutropenia and thrombocytopenia. Adverse events did not differ from those experienced by adult AML patients. Four of 11 patients treated at 9 mg/m1 for 2 doses of gemtuzumab ozogamicin had less than 5% bone marrow blasts after the second dose, A phase 2 study combining gemtuzumab ozogamicin with conventional chemotherapy in pédiatrie patients with AML is planned.

Original languageEnglish (US)
Pages (from-to)217b
Issue number11 PART II
StatePublished - 2000

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology


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