How effective and safe is high-dose chemotherapy and stem cell transplant for children with lymphoma?

This study examined the effectiveness and safety of high-dose chemotherapy and stem cell transplants for children with lymphoma. The study showed that the combination treatment was very effective but that the dose may need to be adjusted for some patients.

Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL) have very effective treatment options. However, some patients stop responding to treatment (refractory). Other patients see a return of cancer after treatment has stopped (relapse). Patients with relapsed or refractory HL/NHL require more aggressive treatment.

High-dose chemotherapy treatments such as BEC (BCNU, etoposide, and cyclophosphamide) and AEC (ACNU, etoposide, and cyclophosphamide) can be used with stem cell transplants (SCT). Autologous SCT involves removing healthy immune cells from patients and freezing them. Then, patients receive chemotherapy to kill cancer cells. After this, the healthy cells are injected back into the patient. This gives the patient healthy immune cells that travel to the bone marrow and begin producing new blood cells. High-dose chemotherapy and SCT is an effective treatment for adult patients with relapsed or refractory HL/NHL.  However, it is not known how effective and safe it is for pediatric patients.

Data from 56 pediatric patients with relapsed or refractory HL or NHL who received high-dose chemotherapy and autologous SCT was analyzed. 44 patients had NHL and 12 had HL. Patients were followed for an average of 42.9 months.

5 years after treatment, 83.6% of patients survived. 74.8% of patients did not have any cancer growth at 5 years. Overall, 81.6% of patients with NHL and 91.7% of those with HL were alive at 5 years. 72.7% of patients with NHL and 83.3% of those with HL did not have cancer growth at 5 years. 

NK/T-cell lymphoma (NKTL) is a rare and very aggressive type of NHL. After 5 years, 75% of patients with NKTL were alive and 75% did not have cancer growth.

14 patients experienced cancer growth, side effects or mortality. Eight patients had a return of cancer, three patients developed another cancer and three patients passed away. A poor response to initial chemotherapy was the only factor that gave patients a higher chance of relapse.

Low immune cells with a fever was the most common side effect experienced by 53 patients. 9 patients developed inflammation in the lung. 24 patients developed a cytomegalovirus infection.

The authors concluded that high-dose chemotherapy with SCT was an effective treatment for children with relapsed or refractory HL/NHL. However, the dose of chemotherapy may need to be changed in some patients to lessen side effects.

This trial was limited by the small number of patients analyzed. Also, there were many different types of lymphoma included. The fact that this trial was based on medical records meant that not all information was available for some patients. There was no comparison treatment used to show if high-dose chemotherapy and SCT is the best treatment available for patients.