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Leukemia. 2019 Apr;33(4):884-892. doi: 10.1038/s41375-018-0265-z. Epub 2018 Sep 28.

Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia.

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Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Children's Hospital Los Angeles and Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
National Cancer Institute, Bethesda, MD, USA.
Mayo Clinic, Rochester, MN, USA.
Cincinnati Children's Hospital Medical Center, University of Cincinnati School of Medicine, Cincinnati, OH, USA.
University of Chicago Medicine Comer Children's Hospital, Chicago, IL, USA.
Oxford University Hospitals, Oxford, UK.
University Hospital Saint-Louis, Paris, France.
University Children's Hospital of Würzburg, Würzburg, Germany.
University Children's Hospital of Münster, Münster, Germany.
Children's Hospital at West-mead, Sydney, Australia.
Children's Mercy Hospital, Kansas City, MO, USA.
The Hospital for Sick Children, Toronto, Canada.
University Children's Hospital, Zurich, Switzerland.
Erasmus MC, University Medical Center, Rotterdam, the Netherlands.
New York University Langone Medical Center, New York, NY, USA.
Benioff Children's Hospital and the Helen Diller Family Comprehensive Cancer Center, University of California San Francisco, San Francisco, CA, USA.
Childrens Hospital of Philadelphia, University of Pennsylvania, Philadelphia, PA, USA.


Although inotuzumab ozogamicin (InO) is recognized as an effective agent in relapsed acute lymphoblastic leukemia (ALL) in adults, data on safety and efficacy in pediatric patients are scarce. We report the use of InO in 51 children with relapsed/refractory ALL treated in the compassionate use program. In this heavily pretreated cohort, complete remission was achieved in 67% of patients with overt marrow disease. The majority (71%) of responders were negative for minimal residual disease. Responses were observed irrespective of cytogenetic subtype or number or type of prior treatment regimens. InO was well-tolerated; grade 3 hepatic transaminitis or hyperbilirubinemia were noted in 6 (12%) and grade 3/4 infections in 11 (22%) patients. No patient developed sinusoidal obstruction syndrome (SOS) during InO therapy; however, 11 of 21 (52%) patients who underwent hematopoietic stem cell transplantation (HSCT) following InO developed SOS. Downregulation of surface CD22 was detected as a possible escape mechanism in three patients who developed a subsequent relapse after InO. We conclude that InO is a well-tolerated, effective therapy for children with relapsed ALL and prospective studies are warranted. Identification of risk factors for developing post-HSCT SOS and strategies to mitigate this risk are ongoing.

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