In a nutshell
This study wanted to assess the safety and effectiveness of inotuzumab ozogamicin (InO; Besponsa) for the treatment of children with unresponsive acute lymphoblastic leukemia (ALL). Researchers found that InO was safe and effective in this group of patients.
Some background
Acute lymphoblastic leukemia (ALL) is a cancer of the bone marrow. It leads to abnormal immune cells in the blood. It is often treated with drugs that target the immune system. Sometimes patients stop responding to therapy and a newer treatment is needed. InO is a drug that targets certain proteins on the ALL cancer cells. The safety and effectiveness of this treatment in pediatric patients are still under investigation.
Methods & findings
51 children with unresponsive or relapsed ALL were included in the study and given InO treatment. Patients were followed up for an average of 112-137 days.
No signs of disease after treatment was seen in 67% of patients with severe disease. 71% of patients had no signs of cancer in the blood after treatment with InO (minimal residual disease). Previous treatments or abnormal genes did not appear to affect response to InO.
The most common side effects of InO treatment included liver dysfunction (12%) and infection (22%).
The bottom line
The study concluded that inotuzumab ozogamicin was safe and effective in the treatment of children with unresponsive ALL.
The fine print
A small sample size and short follow-up period may have affected results. Larger studies are needed to confirm these findings.
This study was partially funded by Pfizer, the manufacturer of inotuzumab ozogamicin.
Published By :
Leukemia
Date :
Sep 28, 2018