Within the last two decades, multiple stem cell transplants and immunotherapy were added to intensive chemotherapy as the standard of care for high-risk neuroblastoma, drastically improving survival of this childhood cancer.

Most survivors, however, suffer from long-term complications of treatments, according to the first study to examine late effects in this population since the new therapies were incorporated. Results were published in Lancet Child and Adolescent Health.

“This is a seminal study in our field,” said lead author Tara Henderson, MD, MPH, childhood cancer survivorship specialist and Chair of Paediatrics at Ann & Robert H.

Lurie Children’s Hospital of Chicago, as well as Professor of Paediatrics at Northwestern University Feinberg School of Medicine.

“For the first time we have data on long-term effects of modern therapies for high-risk neuroblastoma. These findings are crucial for optimising care of survivors and in planning clinical trials. We need to make sure that we carefully consider the long-term risks and benefits of cancer treatments.”

Globally, neuroblastoma accounts for 8–10 percent of childhood cancers.

It is generally diagnosed at a median age of 17–18 months, with 90 percent of cases diagnosed in patients younger than 5 years.

More than half of children with neuroblastoma present with high-risk disease.

Until the past two decades, expected five-year survival of high-risk neuroblastoma was less than 25 percent.

Today, those who complete all components of therapy have an estimated three-year event-free survival of 66 percent.

The study included 375 survivors of high-risk neuroblastoma who were enrolled from 2017 to 2021 at 88 hospitals participating in the Children’s Oncology Group (COG) in North America, New Zealand, and Australia.

Eligible participants were aged 5–50 years at enrollent and were diagnosed with high-risk neuroblastoma on or after January 1, 2000.

Moderate-to-severe hearing loss was identified in 72 percent of 327 participants.

Growth failure was found in 24 percent of 360 participants and underweight in 51 percent of 373.

Pulmonary function tests revealed limited lung capacity (restrictive lung disease) in 8 percent of 207 participants.

Most participants had at least two clinically important late effects.

Longer follow-up was associated with a higher prevalence of late effects.

Compared with single stem cell transplant, exposure to multiple stem cell transplants was associated with increased risk of growth failure and restrictive lung disease.

Immunotherapy, however, was not associated with an increase in late effects.

“It is well established that hearing loss is associated with the type of chemotherapy used for high-risk neuroblastoma, so it was not surprising that the vast majority of survivors in our study were affected,” said Dr. Henderson.

“Early intervention and awareness of educational needs of children with hearing impairment might mitigate the quality-of-life impact of this late effect.”

“However, it was striking to find that more than half of survivors in our study were underweight, which likely impacts growth,” she added.

“Growth failure is a sign of accelerated ageing that childhood cancer survivors often face, which increases the risk of various chronic health problems usually associated with older age.”

Article: Late effects after high-risk neuroblastoma (LEAHRN): a multicentre, cross-sectional cohort study from the Children's Oncology Group

Source: Ann & Robert H. Lurie Children's Hospital of Chicago