Limited surgery and proton therapy in children and adolescents with craniopharyngioma


Matthew Stenger

Posted: 2023/5/5 12:07:00 PM

Last update: May 5, 2023 11:40:18 AM

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A phase II trial (RT2CR) reported in Lancet Oncology, Thomas E. Merchant, DO, and colleagues found that limited surgery and proton therapy were associated with higher rates of disease control and lower rates of severe complications in pediatric and adolescent patients with craniopharyngioma. Survival outcomes did not differ from those observed in previous cohorts of patients treated with limited surgery and photon therapy.

Survey details

In this trial, 94 patients enrolled between August 2011 and January 2016 at St. Jude Children’s Research Hospital and the University of Florida Health Proton Therapy Institute underwent limited surgery and proton therapy. I was. Primary endpoints were progression-free survival and overall survival. Outcomes were compared with those of his cohort of 101 patients treated with limited surgery and photon therapy at St. Jude Children’s Research Hospital between April 1998 and December 2013 (photon cohort). it was done.

Thomas E. Merchant, DO

Thomas E. Merchant, DO

Main findings

In the study population, the 3-year progression-free survival rate was 96.8% (95% confidence interval). [CI] = 90.4%–99.0%), and the 5-year PFS was 93.6% (95% CI = 86.3%–97.1%). Overall survival was 100% at 3 and 5 years.

In the historical photon cohort, the 3-year PFS was 96.0% (95% CI = 89.7%–98.5%) and the 5-year PFS was 90.0% (95% CI = 82.2%–94.5%). Overall survival was 100% at 3 years and 98.0% at 5 years (95% CI = 92.3%–99.5%).

Over the 5-year period in the study population, central nervous system (CNS) necrosis occurred in 2% of patients, severe vasculopathy occurred in 4%, and persistent neurological conditions unrelated to necrosis or vasculopathy occurred. Occurred in 3%. A decrease in visual acuity from normal to abnormal occurred in her 7% of her 54 patients with normal visual acuity at baseline. The most commonly reported grade 3 or 4 adverse events were headache (6%), vascular disturbance (6%), and seizure (5%).

In the historical photon cohort, 2% of patients developed CNS necrosis, 7% developed severe vasculopathy, and 3% developed a permanent neurological condition unrelated to necrosis or vasculopathy. . A decline in visual acuity from normal to abnormal occurred in 10% of 50 patients with normal visual acuity at baseline.

Compared to the study group, the photon cohort longitudinal scores showed a decline in intelligence quotient (IQ; –1.09 points per year, P. = .0070) and adaptive functioning (self-care skills) as measured by the Adaptive Behavior Scale (–1.48 points per year, P. = .030). The 5-year estimated difference in favor of the study group was 4.58 points in IQ and 7.34 points in adaptive functioning.

The researchers concluded that “proton therapy did not improve survival outcomes in pediatric and adolescent patients with craniopharyngioma compared with historical cohorts, and rates of severe complications were similar. Cognitive outcomes were improved over photon beam therapy.Children and adolescents treated for craniopharyngioma using limited surgery and postoperative proton therapy showed high rates of tumor control, [a] Low incidence of serious complications. The results achieved with this treatment represent a new benchmark by which other regimens can be compared. ”

Thomas E. Merchant, DOof the Department of Radiation Oncology, St. Jude Children’s Research Hospital. Lancet Oncology article.

Disclosure: This study was funded by Syria-related charities in Lebanon, USA, the American Cancer Society, the National Cancer Institute, and Blindness Prevention Research. For full study author disclosures, please visit thelancet.com.

The content of this post has not been reviewed by the American Society of Clinical Oncology (ASCO®) and does not necessarily reflect the views or opinions of ASCO®.




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