In order to better characterize the biological features of
fibrodysplasia ossificans progressiva, we reviewed the histopathological
specimens from eleven patients (twelve biopsies) who had a confirmed
diagnosis of the disease. All of the biopsies had been performed in
children, to exclude the diagnosis of a malignant lesion. In no instance
was the diagnosis of fibrodysplasia ossificans progressiva considered
before the biopsy. The results of a lesional biopsy in all eleven patients
revealed normal endochondral osteogenesis at heterotopic sites. The results
of biopsy of an early lesion in six children were misinterpreted as
revealing a diagnosis of fibromatosis or sarcoma before the
roentgenographic appearance of ossification. Immunohistochemical studies of
sections of the earliest lesion demonstrated S-100 antigen positivity
before the histological appearance of differentiated osteochondral tissue.
The presence of congenital malformation of the great toes and of postnatal
heterotopic endochondral osteogenesis strongly suggests that fibrodysplasia
ossificans progressiva is a disorder of defective induction of endochondral
osteogenesis. This study established the predominant histopathological
findings associated with fibrodysplasia ossificans progressiva and can
serve as a basis for postulation of a candidate gene in the pathogenesis of
the disorder. A lesional biopsy is not needed to make the diagnosis; biopsy
uniformly exacerbates the condition and should be avoided.