Use and complications of high-dose disodium etidronate therapy in fibrodysplasia ossificans progressiva

Rogers, John G.; Dorst, John P.; Geho, W. Blair

doi:10.1016/s0022-3476(77)80918-9

Cited by 40 publications

(17 citation statements)

References 6 publications

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“…There are no effective medical treatment options to prevent the formation of heterotopic bone in this disease. Bisphosphonate treatment has shown improvement in ambulation, a reduction of existing calcifications and the disappearance of some new ectopic ossifications [57,58,59]. …”

Section: Bisphosphonate Use In Pediatric Patientsmentioning

confidence: 99%

The Use of Bisphosphonates in Pediatrics

Baroncelli

Bertelloni

2014

Horm Res Paediatr

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Bisphosphonates are widely used for the prevention and treatment of osteoporosis in adulthood. In the last years, bisphosphonates have been increasingly used in pediatric patients for the treatment of a growing number of disorders associated with osteoporosis, resistant hypercalcemia or heterotopic calcifications. The use of bisphosphonates in pediatric patients has been proven safe; however, the risk of potential severe consequences into adulthood should be kept in mind. Well-defined criteria for bisphosphonates treatment in pediatric patients are not specified, therefore an accurate selection of patients who could benefit from bisphosphonates is mandatory. A strict follow-up of pediatric patients receiving long-term bisphosphonate therapy is strongly recommended. The purpose of this mini review is to provide a summary of current knowledge on some main general aspects of the structure, mechanisms of action, pharmacokinetics, and bioavailability of bisphosphonates, and to focus on the latest advances of bisphosphonate treatment in pediatric patients. Particular attention has been paid to the common and potential adverse effects of bisphosphonate treatment, and some suggestions concerning the clinical approach and general measures for bisphosphonate treatment in pediatric patients are reported.

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Section: Bisphosphonate Use In Pediatric Patientsmentioning

confidence: 99%

The Use of Bisphosphonates in Pediatrics

Baroncelli

Bertelloni

2014

Horm Res Paediatr

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“…In our patient the drug was given over some five years on an intermittent schedule in order to delay osteopenia. However, during treatment the nodules did not stop appearing and bone demineralization did become a significant side effect, although in the literature it has been described only with higher doses 19 . Inflammatory signs preceding ossification were generally less severe when corticosteroid was used and indeed the drug is recommended per os on these occasions.…”

Section: Discussionmentioning

confidence: 95%

“…Several types of treatment have been tried 18 . Administration of calcium chelants such as sodium etidronate have been proposed since 1969 with variable results 19 . In our patient the drug was given over some five years on an intermittent schedule in order to delay osteopenia.…”

Section: Discussionmentioning

confidence: 99%

Fibrodysplasia ossificans progressiva: case report

Nucci

Queiróz

Santos

et al. 2000

Arq. Neuro-Psiquiatr.

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-Fibrodysplasia ossificans progressiva is a rare genetic disease characterized by widespread soft tissue ossification and congenital stigmata of the extremities. We report on a male child followed for ten years since the age of 3 years and 9 months, when the diagnosis was made. He was born with bilateral hypoplasic hallux valgus and ventricular septal defect, corrected by transsternal approach when 32 months old. Restriction of neck mobility followed and foci of ectopic ossification appeared. Four crises of disease exacerbation were treated with oral prednisone and/or other antiinflammatory drugs. Sodium etidronate 5 to 10 mg/kg/day was prescribed intermittently during about six years but was discontinued due to osteopenia. The disease course has been relentless, with severe movement restriction including the chest wall. A review showed few similar case reports in the Brazilian literature. We revisit the criteria for diagnosis and the essentials of management and treatment.KEY WORDS: fibrodysplasia ossificans progressiva, myositis ossificans, muscle biopsy, rigid spine, Tc-MDP scan, etidronate treatment. Fibrodisplasia ossificante progressiva: relato de casoRESUMO -Fibrodisplasia ossificante progressiva é uma doença genética rara, caracterizada por ossificação disseminada em tecidos moles e estigmas congênitos nas extremidades. Relatamos sobre uma criança do sexo masculino, branca, que vem sendo acompanhada desde seus 3 anos e 9 meses quando diagnosticamos a doença. Nascida com halux valgo hipoplásico bilateral e defeito do septo interventricular, submeteu-se a correção cirúrgica através de esternotomia, aos 32 meses de vida. Seguiram-se progressiva restrição de movimentos cervicais e aparecimento de nódulos duros e dolorosos em regiões paravertebrais cérvico-torácicas. Em dez anos, assistimos a quatro episódios bem definidos de exacerbação da doença, com acréscimos de ossificação ectópica. Nessas ocasiões, prescrevíamos prednisona oral e/ou antiinflamatórios não hormonais. Etidronato de sódio, 5 a 10 mg/ kg/dia, foi utilizado em períodos de dois ou três meses, com intervalos de dois meses, durante seis anos. Osteopenia obrigou-nos a interromper o uso do quelante. A evolução tem sido inexorável, com limitação intensa de movimentos torácicos e corpóreos. A revisão da literatura brasileira revelou poucos casos semelhantes. Enfatizamos os critérios para diagnóstico e princípios básicos de tratamento. PALAVRAS-CHAVE: fibrodisplasia ossificante progressiva, miosite ossificante, biópsia muscular, espinha rígida, cintilografia com MDP99m

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“…Many treatments have been used, such as steroids, isotretinoin and oral etidronate, but without good results (4)(5)(6) . In vitro chemical studies have shown that bisphosphonates adsorb hydroxyapatite crystals, thus diminishing heterotopic bone formation during the active stage of the disease (7) .…”

Section: Introductionmentioning

confidence: 99%