In a nutshell
This study observed the safety and effectiveness of the drug Gemtuzumab Ozagamicin (Mylotarg) when given to children with acute myeloid leukemia unresponsive to treatment. The authors found the treatment to be effective in children who became unresponsive to bone marrow transplant therapy.
Some background
Acute Myeloid Leukemia (AML) is a cancer of the bone marrow which may lead to abnormal immune cells. Gemtuzumab Ozagamicin (GO) is a monoclonal antibody (a drug that attacks certain proteins on cancer cells). This means that the drug can be targeted directly at the cancer cells and deliver the drug only where it's needed. However it may cause some side effects.
It is important to research if GO can be effectively used in combination with other treatment for AML such as the chemotherapy drug, cytarabine (Cytosar-U).
Methods & findings
A total of 76 patients with unresponsive AML resistant to treatment were included in the study. Eighty-eight percent of these patients had AML at high risk of becoming unresponsive to treatment and 56% had been previously treated with chemotherapy with no response. These patients received GO. GO was given by itself (47%) or with cytarabine (47%). The remaining 6% received GO in conjunction with different drugs. The most common side effect observed was fever with low white blood cell count. Some gastrointestinal problems and allergic reactions were also observed.
64% of patients were able to go on to receive a bone marrow transplant, showing GO can successfully bridge the gap to transplants in high-risk cases. Four years after treatment 13-23% of patients survived overall. Survival rate was between 20-34% in children who received a bone marrow transplant and 0% in children who did not.
The bottom line
The study concluded that Gemtuzumab Ozagamicin was found to be an effective treatment for children with AML that became unresponsive to transplant therapy.
The fine print
More studies are needed on a larger variety of patients to confirm results.
What’s next?
Talk to your doctor about using GO as part of AML treatment.
Published By :
Haematologica
Date :
Aug 09, 2018
