Is bortezomib safe for children with refractory acute lymphoblastic leukemia?

This study aimed to investigate the safety and efficacy of bortezomib (Velcade) when combined with induction chemotherapy in Japanese children with refractory acute lymphoblastic leukemia.

This study concluded that the combination of bortezomib and induction chemotherapy has high effectiveness but led to a high risk of infectious complications. 

Many patients with acute lymphoblastic leukemia relapse after treatment, or do not respond to treatment. Remission can be hard to achieve or maintain in these patients. New treatments are needed for this population.

Bortezomib is a proteasome inhibitor that can be used to treat cancer. Proteasomes break down proteins. The proteins needed to kill cancer cells can be broken down too quickly. By inhibiting proteasomes, the proteins can kill cancer cells.

Bortezomib is a treatment often used in patients with multiple myeloma. It was not known if bortezomib was safe and effective in children with ALL. 

This small study involved 6 young patients who had ALL. All patients were treated with four-dose bortezomib combined with two standard induction chemotherapies. The safety and effectiveness was measured.

Four of the five patients achieved a complete response to treatment.

All 6 patients experienced pancytopenia. Pancytopenia involves low levels of white blood cells, red blood cells and platelets. Four patients had serious infectious complications after being treated. Five 5 patients had peripheral neuropathy. Peripheral neuropathy is damage to nerves.

This study concluded that the combination of bortezomib and induction chemotherapy was effective but led to a high risk of infectious complications. 

The limitation to this study is that only a small number of patients were involved.