SIR 2024
Pediatric Interventions
Ryan Tang, BA
Medical Student
University of South Florida, Morsani College of Medicine
Financial relationships: Full list of relationships is listed on the CME information page.
Pavel Yarmolenko, PhD, BS, BA
Assistant Professor
The Sheikh Zayed Institute for Pediatric Surgical Innovation
Disclosure information not submitted.
Aerang Kim, MD, PhD
Oncologist
Childrens National Medical Center
Disclosure information not submitted.
Matthew Oetgen, MD
Orthopedic Surgeon
Childrens National Medical Center
Disclosure information not submitted.
Pamela Hinds, PhD, RN
Chair of Nursing Research
Children's National Medical Center
Disclosure information not submitted.
Robert Henshaw, MD
Orthopedic Oncologist
Children's National Medical Center
Disclosure information not submitted.
Karun V. Sharma, MD, PhD, FSIR
Director of Interventional Radiology
Children's National Medical Center
Disclosure information not submitted.
Osteoid osteoma (OO) is a benign but painful bone tumor that predominantly occurs in long bones of children and adolescents. Standard-of-care definitive treatment is CT-guided Radiofrequency ablation (RFA), which has high success rates, but is invasive and exposes patients and operators to ionizing radiation. MRI-guided-high-intensity focused ultrasound (MR-HIFU) ablation is non-invasive and radiation-free and has shown success in treating OO. We report the results of a pilot trial that was designed to evaluate safety and feasibility of this therapy.
Materials and Methods:
Nine patients (7 males, 2 females; 16±6 years) were treated (Clinical Trials.gov - NCT02349971). The primary objectives were to evaluate feasibility and safety. The secondary objectives were to evaluate pain relief, medication use, quality of life (QOL), and imaging response. Pain relief was evaluated using Visual Analog Scale (VAS) score and medication logs. QOL was assessed using validated patient reported outcome tools. Imaging response was assessed by comparing pre and post treatment MRI scans. Subjects were followed at 1day, 1week, 1,6,12 months, and 5 years.
Results:
MR-HIFU ablation was technically successful in all 9 patients demonstrating feasibility. No serious adverse events (SAEs) were observed based on the NCI (CTCAE) v4.0. Pre-treatment median worst VAS score was 6 (with NSAIDs), decreased to 0 by day 7, and remained at 0 (without NSAIDs) at 1-year post treatment, which is statistically significant improvement (Wilcoxon, p=0.0078). Regarding QOL, median total SDS scores decreased from 15 to 11 at one year, demonstrating significant improvement in QOL (Wilcoxon, p=0.0313). The PROMIS® Pediatric Pain Interference Short Form median total standardized t scores decreased by 21.65 at one year, demonstrating significantly decreased impact of pain on various aspects of the patients’ life following treatment (Wilcoxon, p=0.0078). PedsQL™ scores showed similar trends of improvement for patients and their parents. Regarding imaging response, 8 out of 9 patients underwent follow up MRI at 12 months. OO nidus perfusion was eliminated or reduced in the majority of patients. Soft tissue edema resolved in 8 out of 8 patients and bone marrow edema was reduced or resolved in 6 out of 8 patients.
Conclusion:
This pilot trial demonstrates that MR-HIFU ablation is safe and provides durable pain relief and improved QOL for patients with symptomatic OO. These results helped to support FDA approval and led to a larger, ongoing pivotal trial to determine efficacy of this therapy.