Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Status: | Recruiting |
---|---|
Conditions: | Skin and Soft Tissue Infections |
Therapuetic Areas: | Dermatology / Plastic Surgery |
Healthy: | No |
Age Range: | Any |
Updated: | 2/7/2015 |
Start Date: | February 2012 |
Contact: | Haydar Frangoul, MD |
Email: | haydar.frangoul@vanderbilt.edu |
Phone: | 615-936-6989 |
This is a feasibility study to see if Granulocyte Colony Stimulating Factor (GCSF) is
effective as a treatment of Dystrophic Epidermolysis Bullosa (EB.) Patients will receive
one course of treatment with the study drug. The course will be 7 days in length. After
receiving GCSF, patients will be followed at 7 and 30 days following the discontinuation of
the drug. Thirty day follow up can be done via telephone communication with the patient or
family.
effective as a treatment of Dystrophic Epidermolysis Bullosa (EB.) Patients will receive
one course of treatment with the study drug. The course will be 7 days in length. After
receiving GCSF, patients will be followed at 7 and 30 days following the discontinuation of
the drug. Thirty day follow up can be done via telephone communication with the patient or
family.
Inclusion Criteria:
- Each patient must have the diagnosis of severe generalized recessive dystrophic EB
(formerly known as Hallopeau-Siemens RDEB) confirmed by clinical criteria and either
of the following:
1. transmission electron microscopy
2. immunofluorescence antigenic mapping and type VII collagen monoclonal antibody
staining
3. COL7A1 mutational analysis
Exclusion Criteria:
- The patient must not have a history of squamous cell carcinoma or any internal
malignancy.
- Female patients who are pregnant.
- Patients with active signs and symptoms of infection.
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