# Clinical Efficacy of Focused Ultrasound (MR-HIFU) Treatment of Symptomatic Osteoid Osteoma in Pediatrics

> **NIH NIH R01** · CHILDREN'S RESEARCH INSTITUTE · 2022 · $195,039

## Abstract

ABSTRACT
 Osteoid osteoma is a painful localized bone tumor that occurs most commonly in children and adolescents
between 5-25 years of age. It accounts for 14% of all benign bone tumors. Symptoms can include severe bone pain and
deformity, limb length discrepancy, and scoliosis. The current standard of care definitive treatment is with CT-guided
radiofrequency ablation (CT-RFA), which has replaced open surgery. During CT-RFA, a bone drill is placed through the
skin, muscle and bone into the osteoid osteoma using a CT imaging guidance. An RFA needle probe is then used to burn
the osteoid osteoma. Although CT-RFA is less invasive than open surgical resection, potential complications include
bleeding, infection, skin and muscle burn, and nerve injury from drilling through tissue and heating along the RFA probe.
Furthermore, the use of CT imaging required for guidance exposes patients and operators to ionizing radiation, which can
have potential long-term negative effects, especially for growing children.
 An optimal image-guided therapy for treatment of painful localized tumors such as osteoid osteoma would ideally
be completely non-invasive, precise, and radiation-free. It would be performed without any skin incisions or bone drills,
limiting potential collateral tissue damage and complications. It would be performed with real-time imaging feedback that
could be used to prevent thermal injury to nearby critical structures and also to confirm treatment success. Magnetic
resonance imaging-guided high intensity focused ultrasound (MR-HIFU) is a novel image-guided therapy which offers
such treatment.
 Our group has performed the first Phase I clinical trial in the US to evaluate MR-HIFU treatment for painful
osteoid osteoma in a pediatric cohort (PI Sharma, NCT02349971). The results show that MR-HIFU is feasible, well
tolerated, and can be safely performed in children with painful osteoid osteoma. Based on our encouraging preliminary
results, we hypothesize that this completely non-invasive and radiation-free MR-HIFU therapy will be clinically
effective for treatment of painful osteoid osteoma; with clinical success comparable to CT-RFA. To test this
hypothesis, we propose an early Phase II clinical trial designed to perform a thorough evaluation of the safety and efficacy
of MR-HIFU ablation in patients with painful osteoid osteoma. We will also closely evaluate MR thermometry, intrinsic
contrast imaging, and diffusion-weighted MR imaging obtained during the course of treatment and determine if these
imaging changes can depict ablation zone without the use of intravenous injections of gadolinium-based contrast agents
and serve as imaging biomarkers of treatment efficacy.

## Key facts

- **NIH application ID:** 10308666
- **Project number:** 5R01CA227454-03
- **Recipient organization:** CHILDREN'S RESEARCH INSTITUTE
- **Principal Investigator:** Karun V Sharma
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $195,039
- **Award type:** 5
- **Project period:** 2019-07-01 → 2023-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10308666

## Citation

> US National Institutes of Health, RePORTER application 10308666, Clinical Efficacy of Focused Ultrasound (MR-HIFU) Treatment of Symptomatic Osteoid Osteoma in Pediatrics (5R01CA227454-03). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10308666. Licensed CC0.

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