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Wirkung von Pamidronat auf Beschwerdebild und Knochenstoffwechsel bei fibröser Dysplasie und McCune-Albright-Syndrom

Effects of pamidronate on clinical symptoms and bone metabolism in patients with fibrous dysplasia and McCune-Albright syndrome

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Zusammenfassung

□ Hintergrund

Bei der fibrösen Dysplasie und dem durch endokrine Störungen wie eine Pubertas praecox erweiterten McCune-Albright-Syndrom findet sich eine embryonal erworbene Differenzierungsstörung der Osteoblasten mit fibrösen Knochenläsionen. Diese können zu Deformierungen, pathologischen Frakturen und chronischen Schmerzzuständen der befallenen Skelettareale führen. Wir berichten hier über unsere Erfahrungen mit einer systemischen Therapie mit dem Bisphosphonat Pamidronat.

□ Patienten

Behandelt wurden drei Patientinnen mit fibröser Dysplasie und fünf Patientinnen mit einem McCune-Albright-Syndrom über einen kumulativen Zeitraum von 37 Beobachtungsjahren.

□ Ergebnisse

Bei allen Patientinnen, die über Schmerzen klagten, ließ sich durch die intravenöse Gabe von 60 mg Pamidronat eine bis zu sechs Monate anhaltende Linderung beobachten. Bei zwei Patientinnen kam es im Langzeitverlauf auch zu einer Auffüllung einzelner osteolytischer Herde. Die Nebenwirkungen der Therapie beschränkten sich auf leichte Fieberreaktionen und einen milden, im physiologischen Bereich gelegenen Abfall des Serumcalciums während der Infusionen.

□ Schlußfolgerung

Pamidronat stellt eine nebenwirkungsarme und symptomatisch wirksame Therapieoption in der Behandlung der fibrösen Dysplasie dar, deren langfristiger Nutzen in weiteren Studien geprüft werden sollte.

Abstract

□ Background

Patients with fibrous dysplasia and the McCune-Albright syndrome which is characterized by additional endocrine dysfunctions, such as pubertas precox, suffer from a regional impairment in the differentiation of osteoblasts that is acquired during early embryogenesis and results in fibrous bone lesions. These lesions may cause bone deformities, fractures and chronical pain in the affected skeletal regions. We here report about our experience with a systemic treatment of the bisphosphonate pamidronate.

□ Patients

We treated 3 patients with fibrous dysplasia and 5 patients with Mc-Cune-Albright syndrome over a cumulative period of 37 patient-years.

□ Results

In all patients who suffered from painful lesions, intravenous infusions of 60 mg pamidronate resulted in an improvement of pain that lasted up to 6 months. In 2 patients we also observed a reduction in the size of some of the osteolytic lesions. Side effects were limited to asymptomatical fever and a small decline in total serum calcium within the physiological limits during the infusions.

□ Conclusion

Pamidronate appears to be an effective and well tolerable treatment option for patients with fibrous dysplasia.

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  1. Abteilung Innere Medizin I, Endokrinologie und Stoffwechsel, Universitätsklinikum, Bergheimer Straße 58, D-69115, Heidelberg

    Johannes Pfeilschifter & Reinhard Ziegler

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  1. Johannes Pfeilschifter
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  2. Reinhard Ziegler
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Pfeilschifter, J., Ziegler, R. Wirkung von Pamidronat auf Beschwerdebild und Knochenstoffwechsel bei fibröser Dysplasie und McCune-Albright-Syndrom. Med Klin 93, 352–359 (1998). https://doi.org/10.1007/BF03044679

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