Publication Date:
2018-11-29
Description:
Introduction: Gemtuzumab ozogamicin (GO; MylotargTM) is an antibody-drug conjugate composed of an anti-CD33 monoclonal antibody covalently linked to the potent antibiotic calicheamicin. Previous studies have shown GO is generally well tolerated and can induce durable second remissions when administered as monotherapy or in combination with chemotherapy in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). We report safety data from an expanded-access protocol (EAP) that allowed compassionate use of GO in patients with R/R AML or acute promyelocytic leukemia (APL) and no access to comparable or alternative therapy. Methods: Conducted in the United States, the GO EAP (NCT02312037) was an open-label study in patients aged ≥3 months with R/R AML (including myelodysplastic syndrome) or APL who were considered to have the potential to derive clinical benefit and had exhausted other treatment options. The protocol allowed for treatment regimens tested in clinical trial settings and reported in the Mylotarg Investigators Brochure or peer-reviewed journals. Data from these trials indicated these regimens could potentially benefit a patient with R/R AML or APL. For R/R AML patients, the regimens included GO as monotherapy or in combination with anthracyclines and/or nucleoside-analogue containing regimens or hypomethylating agents. For patients with APL, these included GO as monotherapy or in combination with all-trans retinoic acid and/or arsenic trioxide. Patients were permitted to re-enroll in treatment, and their data are summarized according to each enrollment treatment. Results: A total of 331 patients received GO either as monotherapy for R/R AML (adult [aged ≥18 years]: n=118; pediatric [aged
Print ISSN:
0006-4971
Electronic ISSN:
1528-0020
Topics:
Biology
,
Medicine
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