Clinical trial improves neurocognitive outcomes for childhood craniopharyngioma (Proton therapy better overall to photon)

Medical Xpress / St. Jude Children's Research Hospital / The Lancet Oncology ^ | April 19, 2023 | Thomas E Merchant et al

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St. Jude Children's Research Hospital is reporting phase 2 clinical trial results treating the brain tumor craniopharyngioma with proton therapy. The researchers found a similar survival rate between more targeted proton therapy and photon therapy but improved neurocognitive outcomes with the proton therapy. The clinical trial may set the new "gold standard" for pediatric craniopharyngioma treatment.

Craniopharyngioma is a rare brain tumor that forms in the central region of the brain, making it difficult to treat without exposing surrounding healthy brain tissue to radiation. Conventional radiation therapy, called photon therapy, has raised the five-year survival rate for this cancer to 90%. However, patients may experience treatment-related neurocognitive late effects.

Photons (X-rays) pass through the tumor, whereas protons (a charged particle and the nucleus of the hydrogen atom) can be directed to stop within the tumor. This is why proton therapy spares normal brain tissue and reduces collateral effects.

The St. Jude group compared proton therapy results over five years to a historical control group treated with photon therapy at the hospital. The five-year progression-free survival was 93.6% in the proton group. This result was not statistically significantly different from photon therapy (~90.0%), but it did show proton therapy maintained the same high survival rate.

For neurocognitive outcomes, proton therapy was superior to photon therapy. Patients treated with photon therapy experienced an average loss of 1.09 Intelligence Quotient (IQ) points more than those treated with proton therapy every year for the five years of the study. Similarly, patients exposed to photon therapy lost 1.48 adaptive behavior points, a parental report of self-care skills—more than those treated with proton therapy—each year. The cognitive deficits of those treated with proton therapy were stable by the end of the study.

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