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Published on: June 16, 2020
Disease extent, not lesion location, determines relapse risk in single-system skeletal Langerhans cell histiocytosis.
Itziar Astigarraga1, Helga Björk Arnardóttir2, Oussama Abla3
1Department of Pediatrics, Hospital Universitario Cruces, Osakidetza, Bizkaia UPV/EHU, Barakaldo, Spain.
Blood Advances
|May 8, 2026
Summary
Langerhans cell histiocytosis (LCH) in bone shows high survival. Multifocal bone lesions predict relapse, while CNS-risk lesions increase endocrine issues, guiding risk-adapted therapy.
Area of Science:
- Pediatric Oncology
- Histiocytosis Research
- Skeletal Disorders
Background:
- Langerhans cell histiocytosis (LCH) confined to bone has good survival.
- Optimal management for multifocal bone disease and special site lesions is unclear.
- Limited prospective data exists for single-system skeletal LCH.
Purpose of the Study:
- Evaluate vinblastine-prednisolone therapy in children with single-system skeletal LCH.
- Determine predictors of relapse and long-term sequelae.
- Establish a benchmark for systemic therapy in skeletal LCH.
Main Methods:
- Prospective international pilot trial (LCH-III study, NCT00276757).
- 6 months of vinblastine-prednisolone treatment.
- Evaluated 381 of 455 enrolled children with single-system skeletal LCH.
Main Results:
- 83% early response at week 6.
- 100% 5-year overall survival; 71% 5-year event-free survival (EFS).
- Multifocal bone lesions (MFB) predicted relapse (5-yr EFS 63% vs 87% without MFB).
- CNS-risk lesions increased central diabetes insipidus risk, especially with MFB.
Conclusions:
- Disease extent and location impact outcomes in skeletal LCH.
- Skeletal multiplicity predicts relapse; lesion location predicts endocrine sequelae.
- Supports risk-adapted treatment to minimize morbidity and avoid overtreatment.