BACKGROUND: The natural history of focal splenic lesions in paediatric Gaucher disease (GD) is unknown and these lesions are thought to persist despite enzyme replacement therapy (ERT). OBJECTIVE: To assess the prevalence, evolution and resolution of splenic nodules in a cohort of paediatric Gaucher patients treated with ERT. MATERIALS AND METHODS: The US findings in 37 children with GD were retrospectively reviewed. A total of 28 children underwent serial abdominal US examinations as part of their initial assessment and during routine follow-up. All patients received ERT. RESULTS: Six children (21%) had splenic nodules on US examination, either at presentation or on follow-up examination. In all six patients, the nodules had resolved on follow-up imaging, with resolution taking 17 months to 4 years 8 months. Two sets of siblings developed nodules that resolved over a similar time period. CONCLUSION: Disappearance of focal splenic lesions in children with GD during follow-up has not been previously reported. The development of new focal splenic lesions in children with GD whilst on ERT has not been previously documented.
BACKGROUND: The natural history of focal splenic lesions in paediatric Gaucher disease (GD) is unknown and these lesions are thought to persist despite enzyme replacement therapy (ERT). OBJECTIVE: To assess the prevalence, evolution and resolution of splenic nodules in a cohort of paediatric Gaucherpatients treated with ERT. MATERIALS AND METHODS: The US findings in 37 children with GD were retrospectively reviewed. A total of 28 children underwent serial abdominal US examinations as part of their initial assessment and during routine follow-up. All patients received ERT. RESULTS: Six children (21%) had splenic nodules on US examination, either at presentation or on follow-up examination. In all six patients, the nodules had resolved on follow-up imaging, with resolution taking 17 months to 4 years 8 months. Two sets of siblings developed nodules that resolved over a similar time period. CONCLUSION: Disappearance of focal splenic lesions in children with GD during follow-up has not been previously reported. The development of new focal splenic lesions in children with GD whilst on ERT has not been previously documented.
Authors: J Charrow; J A Esplin; T J Gribble; P Kaplan; E H Kolodny; G M Pastores; C R Scott; R S Wappner; N J Weinreb; J S Wisch Journal: Arch Intern Med Date: 1998-09-14