Treatment of patients who have fibrodysplasia ossificans progressiva with isotretinoin

Michael A. Zasloff, David M Rocke, Leslie J. Crofford, Gregory V. Hahn, Frederick S. Kaplan

Research output: Contribution to journalArticle

42 Citations (Scopus)

Abstract

Retinoids are a plausible family of therapeutic agents for fibrodysplasia ossificans progressiva due to their ability to inhibit differentiation of mesenchymal tissue into cartilage and bone. A prospective study was conducted to assess the efficacy of isotretinoin (13-cis-retinoic acid) in the prevention of heterotopic ossification in patients who had fibrodysplasia ossificans progressiva. Eleven anatomic regions were assessed in each of 21 patients by clinical examination, radiographs, and bone scans. An anatomic region was considered to be involved if there was clinical, radiographic, or radionuclide evidence of orthotopic or heterotopic ossification anywhere in the region. There were 143 involved anatomic regions and 88 uninvolved anatomic regions at the beginning of the study. Only one of the 88 anatomic regions that was completely uninvolved at the beginning of the study became involved during isotretinoin therapy. However, 16 of the 21 patients (76%) had major flare ups develop in 38 of 143 (27%) previously involved anatomic regions while administered isotretinoin therapy. Isotretinoin at steady state doses of 1 to 2 mg/kg per day decreased the incidence of heterotopic ossification at uninvolved anatomic regions compared with an external control group, as long as the medication was started before the appearance of any orthotopic or heterotopic ossification in that anatomic region. The data did not allow the determination of whether isotretinoin was effective or detrimental in preventing disease flareups in regions that had even minimal orthotopic or heterotopic ossification at the time the therapy began. Extreme caution should be exercised when using this medication in patients who have fibrodysplasia ossificans progressiva.

Original languageEnglish (US)
Pages (from-to)121-129
Number of pages9
JournalClinical Orthopaedics and Related Research
Issue number346
StatePublished - Jan 1998

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Myositis Ossificans
Isotretinoin
Heterotopic Ossification
Therapeutics
Bone and Bones
Aptitude
Retinoids
Radioisotopes
Cartilage
Prospective Studies
Control Groups
Incidence

ASJC Scopus subject areas

  • Surgery
  • Orthopedics and Sports Medicine

Cite this

Treatment of patients who have fibrodysplasia ossificans progressiva with isotretinoin. / Zasloff, Michael A.; Rocke, David M; Crofford, Leslie J.; Hahn, Gregory V.; Kaplan, Frederick S.

In: Clinical Orthopaedics and Related Research, No. 346, 01.1998, p. 121-129.

Research output: Contribution to journalArticle

Zasloff, Michael A. ; Rocke, David M ; Crofford, Leslie J. ; Hahn, Gregory V. ; Kaplan, Frederick S. / Treatment of patients who have fibrodysplasia ossificans progressiva with isotretinoin. In: Clinical Orthopaedics and Related Research. 1998 ; No. 346. pp. 121-129.
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