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First subject over five years after the transplant. The second subject is not in compliance with follow ups. We do not plan to enroll more subjects.
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ARRON is a disease believed to be due to immune cells, cells which normally protect the body, but are now attacking the tissue in the retina and/or optic nerve. In addition, the disease may affect the nerves in the ear or other parts of the body . The affected nerves fail to respond, or respond only weakly, to stimuli causing numbing, tingling, pain, and progressive muscle weakness. If the nerves to the ear are affected, reduced hearing or deafness may result. The likelihood of progression of your disease is high. This study is designed to examine whether treating patients with high dose cyclophosphamide and rabbit ATG (drugs which reduce the function of the immune system) followed by return of previously collected blood stem cells will stop the progression of ARRON syndrome. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the cyclophosphamide and rabbit ATG is to destroy the cells in the immune system which are thought to be causing this disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and to produce a normal immune system that will no longer attack the body.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| stem cell transplantation | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hematopoietic stem cell transplantation. | Biological | Autologous hematopoietic stem cell transplantation will be performed. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Standard Snellen acuity clinical testing and improvement visual fields is done by using Humphrey Automated Machine with 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter) | 5 years after transplant |
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Inclusion Criteria:
Age between 18-60.
Diagnosis of ARRON syndrome. Diagnostic criteria described below.
Unexplained visual loss over weeks to months. The visual loss includes both visual acuity and field loss define as follows:
OR
Visual field: perimetric mean deviation -5b
OR
A response to immunosuppressive drugs or immune modulators (response is defined by improvement of vision or decrease the rate of decline of visual loss).
The patient has failed at least 3 months of corticosteroids (prednisone 0.5mg/kg to start), IVIG and at least one other immunosuppressive drug such as methotrexate, Imuran, cyclosporine, etc. Failure is defined by decline of visual acuity (by standard Snellen acuity clinical testing) or visual field (by Humphrey Automated Machine with the 30-2 program or using Kinetic Visual Fields on the Goldman Perimeter)
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Richard Burt, MD | Northwestern University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Northwestern University, Feinberg School of Medicine | Chicago | Illinois | 60611 | United States |
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| ID | Term |
|---|---|
| D012164 | Retinal Diseases |
| ID | Term |
|---|---|
| D005128 | Eye Diseases |
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| ID | Term |
|---|---|
| D018380 | Hematopoietic Stem Cell Transplantation |
| ID | Term |
|---|---|
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
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| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |