Intravenous Administration of Zoledronate in Children with Bone-Associated Langerhans Cell Histiocytosis - Report - MDSpire

Intravenous Administration of Zoledronate in Children with Bone-Associated Langerhans Cell Histiocytosis

  • By

  • Sy, Jann Adriel

  • Quah, Thuan Chong

  • Tee, Pian Pian

  • Ho, Cindy Wei Li

  • April 15, 2026

  • 0 min

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Clinical Report: Intravenous Administration of Zoledronate in Children with Bone-Associated LCH

Overview

This report evaluates the use of intravenous zoledronate in eight children with bone-associated Langerhans cell histiocytosis (LCH). All symptomatic patients experienced pain improvement, and the treatment was well tolerated with no significant adverse effects.

Background

Bone involvement in pediatric LCH can lead to significant pain and functional impairment, necessitating effective adjunctive therapies. Zoledronate, a bisphosphonate, has been used off-label to manage painful osseous LCH, aiming to provide rapid relief and promote skeletal healing. Understanding its efficacy and safety in this population is crucial for optimizing treatment strategies.

Data Highlights

ParameterOutcome
Symptomatic Pain Improvement100% (4 patients)
Median Time to Improvement18 days
Radiographic Outcomes2 complete resolution, 6 improvement
Adverse EffectsTransient fever in 4 patients

Key Findings

  • All 4 symptomatic patients experienced pain improvement following zoledronate treatment.
  • The median time to pain improvement was 18 days post-infusion.
  • Among patients with imaging follow-up, 2 exhibited complete radiographic resolution and 6 showed improvement.
  • No clinically significant hypocalcemia or osteonecrosis of the jaw was observed in the cohort.
  • Zoledronate was well tolerated, with transient fever being the most common adverse effect.

Clinical Implications

The findings suggest that intravenous zoledronate can be an effective adjunctive treatment for children with painful bone lesions due to LCH. Clinicians should consider its use in symptomatic patients, particularly those with poor tolerance to oral therapies or progressive disease.

Conclusion

Intravenous zoledronate demonstrates significant potential in alleviating pain and improving radiographic outcomes in pediatric LCH, warranting further investigation in larger cohorts.

References

  1. The Journal of Clinical Endocrinology & Metabolism, 2023 -- Effectiveness of Zoledronic Acid in Reducing Fractures Among Children With Primary and Secondary Bone Fragility
  2. Blood Cancer Journal, 2023 -- Exploiting Osteoclasts as a Therapeutic Strategy for High-Risk B-Cell Acute Lymphoblastic Leukemia
  3. The ASCO Post, 2020 -- Risk for Osteonecrosis of the Jaw After Treatment With Zoledronic Acid for Metastatic Bone Disease
  4. Langerhans Cell Histiocytosis Treatment (PDQ®) - NCI, 2025
  5. The ASCO Post — FDA Approves Azacitidine for Newly Diagnosed Juvenile Myelomonocytic Leukemia
  6. Langerhans Cell Histiocytosis Treatment (PDQ®) - NCI
  7. https://cdn.who.int/media/docs/default-source/essential-medicines/2023-eml-expert-committee/applications-for-new-indications-for-existing-listed-medicines/i9_zoledronic-acid-oi.pdf?sfvrsn=5c92e6a2_2

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