Single‐dose (4.5 mg/m2) gemtuzumab ozogamicin in combination with fludarabine, cytarabine and anthracycline as reinduction therapy in relapsed or refractory paediatric acute myeloid leukaemia

• Published in: British journal of haematology Vol. 198; no. 2; pp. 373 - 381
• Main Authors: Dhunputh, Chloé, Strullu, Marion, Petit, Arnaud, Merched, Maria, Pasquet, Marlène, Azarnoush, Saba, Leverger, Guy, Ducassou, Stéphane
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 01.07.2022
• Subjects: acute myeloid leukaemia; Acute myeloid leukemia; adjuvant chemotherapy; Anthracycline; Bone marrow transplantation; Chemotherapy; Children; Cytarabine; Cytotoxicity; Fludarabine; Gemtuzumab ozogamicin; Hematology; Leukemia; Neutropenia; paediatric; relapse; Remission; Survival; adjuvant chemotherapy; acute myeloid leukaemia; relapse; Gemtuzumab ozogamicin; paediatric
• ISSN: 0007-1048; 1365-2141
• DOI: 10.1111/bjh.18203

Summary Despite major therapeutic improvements, children with relapsed/refractory Acute Myeloid Leukaemia still have poor outcomes and overall survival does not exceed 40%. New treatments are required to improve their outcome; Gemtuzumab ozogamicin (GO), an anti‐CD33 immunoconjugate antibody, is a potent cytotoxic agent whose efficacy has been demonstrated mainly in adults. The main objective of this retrospective multicentre study was to assess the outcome of children treated, between February 2008 and August 2019, with GO at a single 4.5 mg/m2 dose, in combination with Fludarabine, Cytarabine and antssshracyclines, in context of a first relapse (n = 26) or refractory disease (n = 3). The remission rate was 83% (24/29 children) and 20 children (69%) were allografted. With a median follow‐up of 1.2 years (range: 0.1–8), the overall survival was 49% (CI95% = 33; 72). Most common adverse event was febrile neutropenia with microbiological identification in 55% of cases. Veno‐occlusive disease occurred in 6 patients (21%), of which 5 subvened after bone marrow transplantation, and resolved within 2–32 days (median 10.5 days). Administration of GO in combination with FLA‐anthracyclines chemotherapy appears to be a good reinduction regimen for relapsed or refractory AML with a good safety profile. These results warrant larger prospective study.