Guidebook Home

Table of Contents

Preface

Introductory Comments

General Questions About FOP

Genetics of FOP

How FOP Affects the Body

Care and Treatment

Activities

Feelings About FOP

Helpful Addresses

Family Resources

Ideas for Independence

Medical Articles

Acknowledgments and Contributions

A Medication Guide for Patients Who Have Fibrodysplasia Ossificans Progressiva (FOP)

Frederick S. Kaplan, M.D. and Michael A. Zasloff, M.D., Ph.D.

The purpose of this report is:
1) to briefly review the major classes of medications that have been used in the treatment and management of patients who have FOP
2) to provide a perspective on indications and contraindications for the use of such medications.

The treatment of FOP can ultimately be divided into two categories:
1) symptom modifying
2) disease-modifying therapies

At the present time, there are no generally effective disease-modifying medications for patients who have FOP, except perhaps for accutane. There are, however, numerous medications that can provide some relief from acute flare-ups.

We emphasize that this report reflects the authors' experience and opinions on the various classes of symptom-modifying medications, and is meant only as a guide to this controversial area of therapeutics. Although there are common physical features shared by every person who has FOP, there are differences among individuals that may alter the potential benefits or risks of any medication discussed here. The decision to use or withhold a particular medication must ultimately rest within an individual patient and his or her physician.

The gold standard for all medication studies is a double-blind randomized placebo controlled study. Such studies would be extremely difficult to conduct in the FOP community considering the few patients afflicted with the disorder. Nevertheless, there is a wealth of information on the natural history of FOP, and therapeutic studies with historical controls have been and will continue to be of great value in assessing the potential benefits and risks of various therapeutic approaches.

Another major factor that has impaired exploration of effective therapy for FOP has been the lack of an appropriate animal model for the disease. Although heterotopic ossification can be induced in an animal by the injection or surgical implantation of bone morphogenetic proteins, there are no transgenic animal models for heterotopic ossification. Our group is collaborating with scientists at The Institute of Molecular Pathology in Vienna, Austria to develop an animal model that may be useful for testing medications in diseases like FOP.

Finally, an assiduous search for the gene responsible for FOP is likely to provide valuable insight into potentially useful medications for the condition. We all look forward to the day when effective medications are available for arresting and reversing the progress of FOP.

For additional information on medications used with FOP and other treatment issues, please consult "Medical Management of FOP: Current Treatment Considerations."

Works Cited

Bailar, J.C. et al. Statistics in practice: Studies without internal controls. New Engl. J. Med. 311: 156-162, 1994.

Bruni, L. et al. Case Report: Fibrodysplasia ossificans progressiva. An 11-year old boy treated with a diphosphonate. Acta. Pediatr. Scand. 79: 994-998, 1990.

Cohen, Randolph B. et al. The Natural History of Heterotopic Ossification in Patients who have Fibrodysplasia Ossificans Progressiva: A Study of Forty-Four Patients.J. Bone and Joint Surgery. 75-A (2): 215-219, 1993.

Connor, J.M. et al. Cardiopulminary function in fibrodysplasia ossificans progressiva. Thorax. 36: 419-423, 1981.

Connor, J.M. and Evans. D.A.P.: Fibrodysplasia ossificans progressiva. The clinical features and natural history of 34 patients. J. Bone Joint Surg. 64-B: 76 83, 1982.

DiCesare, P.E. et al. Effects of indomethacin on demineralized bone-induced heterotopic ossification in the rat. J. Ortho. Res. 9: 855-861, 1991.

Rogers, J.G. et al. Use and complications of high-dose disodium etidronate therapy in fibrodysplasia ossificans progressiva. J. Pediatrics. 91: 1011-1014, 1977.

Smith, R. et al. Myositis ossificans progressiva. Clinical features of eight patients and their response to treatment. J. Bone Joint Surg. 58-B: 48-57, 1976.

Weider, D. L. Case Report: Treatment of traumatic myositis ossificans with acetic acid iontophoresis. Phys. Ther. 72: 137, 1992.

Zasloff, M.A. Treatment of fibrodysplasia ossificans progressiva with 13-cis retinoic acid (Accutane). NIH Clinical Research Protocol. Project No. 84-CH 202.