Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and McCune-Albright Syndrome
| Status: | Recruiting |
|---|---|
| Conditions: | Other Indications, Hematology |
| Therapuetic Areas: | Hematology, Other |
| Healthy: | No |
| Age Range: | Any - 105 |
| Updated: | 2/6/2019 |
| Start Date: | August 25, 1998 |
| Contact: | Lori C Guthrie, R.N. |
| Email: | guthriel@mail.nih.gov |
| Phone: | (301) 594-0844 |
Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in
the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous
tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright
Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation
of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful,
and depending on the location of the lesion, can cause significant morbidity. Lesions in
weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to
compression of vital structures such as cranial nerves.
The natural history of this disease is poorly described and there are no clearly-defined
systemic therapies for the bone disease. The purpose of this study is to define the natural
history of the disease with or without treatment.
the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous
tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright
Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation
of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful,
and depending on the location of the lesion, can cause significant morbidity. Lesions in
weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to
compression of vital structures such as cranial nerves.
The natural history of this disease is poorly described and there are no clearly-defined
systemic therapies for the bone disease. The purpose of this study is to define the natural
history of the disease with or without treatment.
Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in
the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous
tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright
Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation
of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful,
and depending on the location of the lesion, can cause significant morbidity. Lesions in
weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to
compression of vital structures such as cranial nerves.
The natural history of this disease is poorly described and there are no clearly-defined
systemic therapies for the bone disease. The purpose of this study is to define the natural
history of the disease with or without treatment.
the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous
tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright
Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation
of the skin, and precocious puberty. The bony lesions are frequently disfiguring and painful,
and depending on the location of the lesion, can cause significant morbidity. Lesions in
weight-bearing bones can lead to disabling fractures, while lesions in the skull can lead to
compression of vital structures such as cranial nerves.
The natural history of this disease is poorly described and there are no clearly-defined
systemic therapies for the bone disease. The purpose of this study is to define the natural
history of the disease with or without treatment.
- INCLUSION CRITERIA
- Any patient with a likelihood of having PFD or MAS, based on information from a
referring physician or surgeon or provided by the patient or guardian, will be
eligible for consideration for inclusion in the study. The diagnosis will be based on
typical findings on bone biopsy, or on clinical grounds.
EXCLUSION CRITERIA
- Patient, child or parents unwilling to fully cooperate with the evaluation
- Patient or guardian of minor unable to provide informed consent.
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 800-411-1222
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