In a nutshell
This study aimed to assess the long-term safety and effectiveness of adding ponatinib (Iclusig) to intensive chemotherapy in the treatment of acute lymphoblastic leukemia. The study found that this treatment is effective on the long-term for these patients.
Some background
Acute lymphoblastic leukemia (ALL) is a cancer of the bone marrow. It is often linked with abnormal genes (such as the Philadelphia chromosome or Ph). Treatment involves strong chemotherapy such as hyper-CVAD [cyclophosphamide (Cytoxan), vincristine (Oncovin), doxorubicin (Adriamycin), dexamethasone (Decadron)]. It may also be treated with drugs which target certain proteins on cancer cells. An example of this is ponatinib. It is important to research the safety and effectiveness of this treatment.
Methods & findings
76 patients with ALL and abnormal genes were included in this study. Patients received hyper-CVAD with ponatinib each day for the first 14 days of the first 21-days cycle, then daily for up to 8 cycles. The effective dose of ponatinib was then reduced gradually.
After 3 years, 70% of patients were alive with no signs of cancer. All 76 patients responded to this treatment. 97% of all patients had very few leukemia cells in the blood and 83% had no leukemia cells in the blood.
Eighty-six percent of patients had an infection and 32% had liver damage (32%) after treatment. 17% also had damage to the pancreas. 12% of patients had a skin rash. 16% of patients experienced high blood pressure. Six patients died while on study treatment. Three of these were from infection. One was from bleeding. Two died from heart attacks related to early ponatinib use.
The bottom line
The study concluded that chemotherapy with ponatinib is effective in the treatment of ALL.
The fine print
This study included a rather small number of participants. Larger studies are required on the safety of this treatment combination.
Published By :
The Lancet. Haematology
Date :
Dec 01, 2018
