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Full-Text Articles in Medicine and Health Sciences
Response To Androgen Therapy In Patients With Dyskeratosis Congenita, Payal P. Khincha, Ingrid M. Wentzensen, Neelam Giri, Blanche P. Alter, Sharon A. Savage
Response To Androgen Therapy In Patients With Dyskeratosis Congenita, Payal P. Khincha, Ingrid M. Wentzensen, Neelam Giri, Blanche P. Alter, Sharon A. Savage
Pediatrics Faculty Publications
Dyskeratosis congenita (DC) is an inherited bone marrow failure syndrome and telomere biology disorder characterized by dysplastic nails, reticular skin pigmentation and oral leucoplakia. Androgens are a standard therapeutic option for bone marrow failure in those patients with DC who are unable to undergo haematopoietic stem cell transplantation, but there are no systematic data on its use in those patients. We evaluated haematological response and side effects of androgen therapy in 16 patients with DC in our observational cohort study. Untreated DC patients served as controls. Seventy percent of treated DC patients had a haematological response with red blood cell …