Hello concernedparent-ga,
I am sorry to hear of your son's diagnosis. You will no doubt have
already found that that there is no regular cure at present and that
standard therapy is orthopaedic surgery to correct the deformities
that occur. As you say you have found general information about the
condition, I have not reproduced it here in my answer. However, since
one of the papers cited below distinguishes between different
conditions, let me just summarize that fibrous dysplasia can occur as
monostotic disease (circumscribed lesion in one bone), polyostotic
disease (lesions in more than one bone, can be of extended type), or
as one of the symptoms of McCune-Albright syndrome. I only looked at
studies of McCune-Albright syndrome if they were aimed at
reducing/reversing the fibrous dysplasia.
I found some information on a new type of drug treatment. A drug
called pamidronate (which is a bisphosphonate) has been reported to
be helpful in fibrous dysplasia.
Here is a study published in August of this year which reported
encouraging results in children:
1. Calcified Tissue International 2002 August; vol 71(2):121-128
Bone Turnover in Children and Adolescents with McCune-Albright
Syndrome Treated with Pamidronate for Bone Fibrous Dysplasia.
Isaia GC, Lala R, Defilippi C, Matarazzo P, Andreo M, Roggia C, Priolo
G, Sanctis C.
Department of Internal Medicine, University of Turin, Italy.
"Bone mineral density (BMD) significantly increased during treatment.
There were signs of radiological healing as thickening of the cortical
bone was found in some cases."
URL for summary: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=12199352&dopt=Abstract
2. A study done five years ago in adults concluded "We suggest that
intravenous pamidronate alleviates bone pain, reduces the rate of bone
turnover assessed by biochemical markers, and improves radiological
lesions of FD. Few side effects were observed."
Bone Mineral Research 1997 Oct; vol. 12(10):1746-1752
Long-term effects of intravenous pamidronate in fibrous dysplasia of
bone.
Chapurlat RD, Delmas PD, Liens D, Meunier PJ.
Department of Rheumatology and Bone Diseases, Hopital Edouard Herriot,
Lyon, France
URL for summary: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=9333137&dopt=Abstract
Here is some additional information about other approaches:
3. Cryosurgery, ie local freezing of the bone cavities can be useful.
I found two recent papers on this topic.
a. Surgical Oncology 2001 March; vol 76(3):157-166
Fibrous dysplasia of bone: management and outcome of 20 cases.
Keijser LC, Van Tienen TG, Schreuder HW, Lemmens JA, Pruszczynski M,
Veth RP.
Department of Orthopaedics, University Medical Center St. Radboud,
Nijmegen, The Netherlands. K.Fruin@orthp.azn.nl
The authors concluded: "Symptomatic circumscribed lesions showed
satisfactory outcome when treated with curettage, cryosurgery and bone
grafting. Lesions of the extended type were most of all seen in
polyostotic disease and eventually needed operative treatment. In case
of bony deformity, corrective osteotomies and rigid internal fixation
were performed in addition to curettage, cryosurgery, and bone
grafting. In polyostotic disease, expected outcomes were good, but in
McCune-Albright syndrome, results were uniformly poor."
URL for summary: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=11276018&dopt=Abstract
b. Acta Orthopaedica Scandinavica 2002 Aug; vol. 73(4):483-486
Cryosurgery in fibrous dysplasia: good result of a multimodality
protocol in 16 patients.
Segev E, Kollender Y, Bickels J, Flusser G, Issakov J, Wientroub S,
Meller I.
Department of Pediatric Orthopaedics, Dana Children's Hospital,
Tel-Aviv, Israel. esegev@tasmc.health.gov.il
No summary available.
4. On clinicaltrials.gov, I found a list of clinical trials which are
currently recruiting patients. These can give some indications of
possible new therapies.
a. Bone Marrow Injection to Replace Diseased Bone in Polyostotic
Fibrous Dysplasia and McCune-Albright Syndrome
"This new treatment is intended to reduce the risk of fracture,
minimize deformities and improve overall function in these patients."
The study is currently recruiting patients aged 4 and older.
http://clinicaltrials.gov/ct/gui/show/NCT00001851;jsessionid=DA5E2E0E8D31784B421E1F42597C5BD5?order=4
b. Alendronate to Treat Polyostotic Fibrous Dysplasia and
McCune-Albright Syndrome
Alendronate is another biphosphonate. Unfortunately, the trial is only
accepting patients aged over 12.
http://clinicaltrials.gov/ct/gui/show/NCT00001728;jsessionid=DA5E2E0E8D31784B421E1F42597C5BD5?order=3
c. Screening and Natural History of Patients with Polyostotic Fibrous
Dysplasia and McCune-Albright Syndrome
This study is not looking at any treatment, but its objectives is "to
identify those patients and lesions that may be appropriate for either
experimental medical or surgical treatment as well as to define the
natural history of the disease with or without treatment." It is
recruiting patients of all ages
http://clinicaltrials.gov/ct/gui/show/NCT00001727;jsessionid=DA5E2E0E8D31784B421E1F42597C5BD5?order=2
d. A Study of the Effects of Pegvisomant on Growth Hormone Excess in
McCune-Albright Syndrome
This may be less relevant as it is only looking at effects in
McCune-Albright patients who have been found to have excess growth
hormone.
http://clinicaltrials.gov/ct/gui/show/NCT00017927;jsessionid=DA5E2E0E8D31784B421E1F42597C5BD5?order=6
Finally:
Fibrous Dysplasia Support Online http://members.cox.net/fdsupport/
includes messages about the treatment and experiences of people with
the disease, a FAQ, and also links to sites with further information,
including information on research and potential new therapies. You
might find it useful to register and monitor this site at intervals.
I was unable to find any applications of alternative therapies to
fibrous dysplasia.
I do hope this information will be of some use to you, and I wish you
and your son all the best. |
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