A Breakthrough in Pediatric Cancer

A landmark study brings the first good news in decades for children battling a high-risk form of Hodgkin lymphoma.

child in handkerchief sitting with doctor.

Since the 1960s, survival rates for most pediatric cancers have improved steadily. But not for high-risk Hodgkin lymphoma. “With this cancer,” said Kara Kelly, professor of pediatrics at the Jacobs School of Medicine and Biomedical Sciences at the University at Buffalo, “there haven’t been major breakthroughs.”

Until now.

In a large multicenter clinical trial co-led by Kelly and pediatric oncologists at Emory University, a targeted therapy used in adults with the disease reduced relapse rates in children by 10%. “That’s quite a big gain,” said Kelly, senior author on the study and chair of the Roswell Park Oishei Children’s Cancer and Blood Disorders Program.

A common cancer among the young

Hodgkin lymphoma (HL) is the most common cancer in patients 12-29 years old. Although it has a high five-year survival rate, about one-third of survivors are classified as high risk; of those, approximately 15-20% relapse.

Moreover, children diagnosed with HL are at higher risk of dying from secondary cancers and cardiovascular diseases, and both the cancer and the side effects of chemotherapy put them at risk for infertility and other long-term complications.

The trial, involving 587 patients between the ages of two and 21 newly diagnosed with high-risk HL, randomized participants into two groups. One received the standard pediatric chemotherapy regimen, while the other received the standard regimen plus brentuximab vedotin (BV), a drug that targets a protein on the surface of the Hodgkin cell.

Remarkable results, immediate impact

Follow-up at about three years post-treatment showed remarkable results: 92.1% event-free survival in the BV group versus 82.5% in the control group—translating to a 59% reduction in the risk of relapse, death or a second cancer. In addition, there was no increase in the incidence of toxic effects.

One marker of the study’s importance: It resulted in the first New England Journal of Medicine coverage of the disease in decades. And then, just a week after publication, the FDA approved pediatric use of the drug.

The results, said Kelly, are that much more meaningful given her previous work with this unforgiving disease.

“My goal has been to develop better treatments that would be associated with high disease control rates, yet to do so without increasing the toxicities of the treatment. We are so pleased that this treatment regimen has now become standard of care.”