LONGSTANDING THERAPEUTIC EFFECT OF BIPHOSPHONATE THERAPY IN A 12 YEARS OLD PATIENT WITH FIBROUS DYSPLASIA JAFFE-LICHTENSTEIN

G Göhlich-Ratmann. J Schaper, W Havers, Th Voit,

University Pediatric Hospital and University Institute of Radiology, Essen, Germany

OBJECTIVE: In Jaffe-Lichtenstein disorder polyostotic dysplasia occurs with lesions isolated to the skeletal system, in our patient the skull. Biphosphonates were investigated for treatment in a 5 years old child with hypercalcaemia with positive acute effect, but no long time follow up was performed. To us presented an 9 year old boy, who had been found at age 5 years to have polyostotoc fibrous dysplasia of the skull base, what extended to the os occipitale and upper cervical spine (first vertebra) at age 8 years. Cerebellar tonsils were hemiated into the fourth ventricle and pons was displaced versus ventral - cranial. Clinically at the same time he started to develop chronic headache and dizziness, movements of the head were limited. At age 9 years the patient was readmitted with increasing localized pain parietal and signs of beginning rightsided oculomotor paresis.

METHODS: A now 12 years old patient with Jaffe-Lichtenstein disorder was prospectively investigated over a period of more than 3 years under the treatment with biphosphonate 30 mg once every 3 months given intravenously over 4 hours. The aim was to evaluate efficacy of treatment and possible negative side effects.

RESULTS: At age 9 years visual evoked potentials showed slowed latencies after stimulation of the right eye. EEG showed polymorphic subtheta - and delta waves left occipitally localized. 3 months after first treatment control of clinical findings, VEP and EEG showed normal results. Over the whole time of investigation there were no side effects noted apart from rised temperature to 38° and slight sickness for about half hour after initiating infusion. Neuroradiological follow-up showed no further progression of the disease and alcalic phosphatase remained at the level of in the mean 500 units / liter. The patient, who is now 12 2/12 years old has grown 19 cm in the treatment period and has now 157 cm (75^th percentile).

CONCLUSIONS: This preliminary report of the prospective follow up of a child with Jaffe Lichtenstein disorder successfully treated with biphosphonate over the last 3 years, when he was in the active phase of growing suggests not only the benefit of patients with polyostotic fibrous dysplasia by this treatment, but also biphosphonate as a therapy without harsh side effects.