Magnetic resonance imaging is a valuable tool to evaluate the therapeutic efficacy of burosumab in children with X-linked hypophosphatemia

in European Journal of Endocrinology
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  • 1 AP-HP, Reference Center for Rare Disorders of the Calcium and Phosphate Metabolism, Filière OSCAR, EndoRare, BOND ERN and Platform of Expertise for Rare Diseases Paris-Saclay, Bicêtre Paris-Saclay Hospital, Le Kremlin-Bicêtre, France
  • | 2 Genethon, Paris-Saclay University, Univ Evry, Inserm, Integrare research unit UMR_S951, Evry, France
  • | 3 Division of Endocrinology, Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy
  • | 4 AP-HP, Department of Pediatric Radiology, Bicêtre Paris-Saclay Hospital, Le Kremlin-Bicêtre, France
  • | 5 University Paris Saclay, Le Kremlin-Bicêtre, France
  • | 6 AP-HP, Reference Center for Rare Disorders of the Calcium and Phosphate Metabolism, Dental Medicine Department, Bretonneau Hospital, GHN-Université de Paris, Paris, France
  • | 7 Université de Paris, Paris, France
  • | 8 Université de Paris, Institut des maladies musculo-squelettiques, Laboratory Orofacial Pathologies, Imaging and Biotherapies URP2496 and FHU-DDS-Net, Dental School, and Plateforme d’Imagerie du Vivant (PIV), Montrouge, France
  • | 9 AP-HP, Department of Endocrinology and Reproductive Diseases, Bicêtre Paris-Saclay Hospital, Le Kremlin-Bicêtre, France
  • | 10 AP-HP, Departments of Endocrinology and Diabetology for Children and Adolescent Medicine, Bicêtre Paris-Saclay Hospital, Le Kremlin-Bicêtre, France
  • | 11 AP-HP, Department of ORL, Bicêtre Paris-Saclay Hospital, Le Kremlin Bicêtre, France
  • | 12 AP-HP, Department of Pediatric Orthopaedic Surgery, Necker – Sick Kids University Hospital, Paris, France
  • | 13 AP-HP, Department of Rheumatology Hospital Cochin, Paris, France
  • | 14 Pediatric Neurosurgery, Hospital Femme Mere Enfant, Hospices Civiles de Lyon and University Claude Bernard Lyon, Bron Cedex, France
  • | 15 Reference Center for Craniosynostosis, INSERM 1033, Lyon, France
  • | 16 AP-HP, Department of Molecular Genetics, Pharmacogenetics and Hormonology, Bicêtre Paris-Saclay Hospital, Le Kremlin-Bicêtre, France
  • | 17 AP-HP, Hôpital Necker Enfants Malades, INSERM U1151, Paris, France

Correspondence should be addressed to V V Zhukouskaya Email volha.zhukouskaya@aphp.fr
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Purpose

To examine the MRI diagnostic performance in the assessment of therapeutic response to burosumab in children with X-linked hypophosphatemia (XLH).

Design

Prospective longitudinal open cohort.

Patients

Seventeen children with XLH, average age of 10.2 ± 2.7 years, had a knee MRI at baseline and after 1 year of burosumab.

Intervention

Children received burosumab at an average dose of 1.4 ± 0.5 mg/kg during 1 year for the treatment of severe rickets (the target serum phosphate ≥ 1.2 mmol/L (≥3.7 mg/dL). The primary endpoint was the change from baseline to 12 months in rachitic lesions on knee MRI. Secondary endpoints were changes in biochemical parameters of phosphate and alkaline phosphatase (ALP).

Results

One year of treatment with burosumab significantly reduced radiological disease activity on knee MRI (by 44 ± 29% in the transverse extent of widening) which was accompanied by a significant reduction in biochemical activity, namely in serum ALP activity, by 28 ± 17%. Additionally, MRI parameters after 1 year of treatment with burosumab (the maximum width of medial physis at 12 months and the change from baseline in the maximum width of lateral physis) were associated with ALP activity at 12 months.

Conclusion

We suggest that MRI is a valuable and quantitative tool to evaluate the therapeutic response to burosumab. MRI could be an excellent alternative to standard bone radiographs for evaluation of the rachitic lesions in a clinical setting avoiding repeated exposition to ionizing radiation.

 

     European Society of Endocrinology

Sept 2018 onwards Past Year Past 30 Days
Abstract Views 2436 2436 60
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