In a nutshell
This study evaluated the safety and effectiveness of blinatumomab (Blincyto) for the treatment of children with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL). The data showed that blinatumomab was safe and effective for these patients.
B-ALL is a type of cancer in which the body makes too many lymphocytes (a type of white blood cell). This is the most common type of cancer in children. A high number of patients with B-ALL experience relapse (worsening of the disease) or refractory (not responsive to the treatment) disease despite treatment. Highly effective targeted therapies have been developed for B-ALL as it can be challenging to treat.
Blinatumomab is an antibody (immunotherapy) that uses the body’s own immune system to identify and kill cancerous B-cells (a type of white blood cell). It is approved and used as a second-line treatment for r/r B-ALL. However, the safety and effectiveness of blinatumomab for the treatment of pediatric patients with r/r B-ALL are still under investigation.
Methods & findings
This study involved 110 children with r/r B-ALL. Patients received blinatumomab treatment for up to 5 six-week cycles (4 weeks of continuous treatment, 2 weeks off). The average follow-up time was 19.5 months.
1.8% of the patients experienced severe cytokine release syndrome (CRS). CRS is a side effect of immunotherapy caused by the immune system becoming highly active. CRS can cause fever, nausea, skin rash, an increase in heartbeat, low blood pressure, and trouble breathing. 3.6% of the patients experienced severe effects affecting the nervous system such as headache, seizure, and depressed level of consciousness.
The average overall survival was 14.6 months. The average survival without cancer coming back was 8.5 months.
52% of the patients achieved a complete response (CR) with negative minimal residual disease (MRD; no cancer cells that remain after treatment) after blinatumomab treatment. The average overall survival was not reached (longer than the follow-up period) for complete MRD responders compared to 9.3 months for MRD non-responders. The average survival without cancer coming back for complete MRD responders was 8 months for MRD responders compared to 2.8 months for the MRD non-responders.
After 1 year, the probability of survival was higher (87%) for patients who received stem cell transplantation compared to patients who did not receive stem cell transplantation after blinatumomab treatment (29%).
The bottom line
This study concluded that blinatumomab was safe and effective for the treatment of children with r/r B-ALL.
The fine print
This study was sponsored by Amgen Inc, the manufacturer of blinatumomab. This study did not have a comparison group. Further studies are needed.
Published By :
Jan 04, 2022