TY - JOUR
T1 - Intensive immunosuppression therapy for aplastic anemia associated with dyskeratosis congenita
T2 - Report of a case
AU - Comoli, Patrizia
AU - Basso, Sabrina
AU - Huang, Guan Cheng
PY - 2005/7
Y1 - 2005/7
N2 - Dyskeratosis congenita (DC) is a very rare inherited disorder characterized by skin pigmentation, nail dystrophy, and mucosal leukoplakia. It is also associated with a variety of noncutaneous abnormalities, such as fatal pulmonary complications, malignancy, and bone marrow failure. We report the case of a 32-year-old man with DC associated with severe aplastic anemia (SAA). The traditional treatment of DC-associated SAA is allogeneic hematopoietic stem cell transplantation (HSCT). However, in this case, an HLA-matched donor was not available. Therefore our patient was given intensive immunosuppressive therapy with antilymphocyte globulin (ALG) and cyclosporine A (CsA). The hemogram findings improved after the treatment, but the patient died of pulmonary complications after being in stable condition for 6 months. The results support the possible use of intensive immunosuppression with ALG and CsA for DC-associated SAA as an alternative treatment for patients who are not eligible for HSCT.
AB - Dyskeratosis congenita (DC) is a very rare inherited disorder characterized by skin pigmentation, nail dystrophy, and mucosal leukoplakia. It is also associated with a variety of noncutaneous abnormalities, such as fatal pulmonary complications, malignancy, and bone marrow failure. We report the case of a 32-year-old man with DC associated with severe aplastic anemia (SAA). The traditional treatment of DC-associated SAA is allogeneic hematopoietic stem cell transplantation (HSCT). However, in this case, an HLA-matched donor was not available. Therefore our patient was given intensive immunosuppressive therapy with antilymphocyte globulin (ALG) and cyclosporine A (CsA). The hemogram findings improved after the treatment, but the patient died of pulmonary complications after being in stable condition for 6 months. The results support the possible use of intensive immunosuppression with ALG and CsA for DC-associated SAA as an alternative treatment for patients who are not eligible for HSCT.
KW - Antilymphocyte globulin
KW - Aplastic anemia
KW - Dyskeratosis congenita
KW - Immunosuppression therapy
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U2 - 10.1532/IJH97.A10416
DO - 10.1532/IJH97.A10416
M3 - Article
C2 - 16105756
AN - SCOPUS:27244442204
VL - 82
SP - 35
EP - 37
JO - International Journal of Hematology
JF - International Journal of Hematology
SN - 0925-5710
IS - 1
ER -