Androgen derivatives improve blood counts and elongate telomere length in adult cryptic dyskeratosis congenita.

Kirschner M; Vieri M; Kricheldorf K; Ferreira MSV; Wlodarski MW; Schwarz M; Balabanov S; Rolles B; Isfort S; Koschmieder S; Höchsmann B; Panse J; Brümmendorf TH; Beier F

doi:10.1111/bjh.16997

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Journal article

Androgen derivatives improve blood counts and elongate telomere length in adult cryptic dyskeratosis congenita.

Kirschner M Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Vieri M Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Kricheldorf K Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Ferreira MSV Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Wlodarski MW Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, University of Freiburg, Freiburg, Germany.
Schwarz M Department of Hematology and Oncology, University Hospital Charité, Berlin, Germany.
Balabanov S Hematology, University Hospital Zurich and University of Zurich, Zurich, Switzerland.
Rolles B Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Isfort S Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Koschmieder S Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Höchsmann B Institute of Transfusion Medicine, University of Ulm, Ulm, Germany.
Panse J Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Brümmendorf TH Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.
Beier F Department of Hematology, Oncology, Hemostaseology and Stem Cell Transplantation, Medical Faculty, RWTH Aachen University, Aachen, Germany.

2020-08-04

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British journal of haematology. - 2020

English Dyskeratosis Congenita (DKC) is a systemic disorder caused by mutations resulting in impaired telomere maintenance. Clinical features include bone marrow failure and an increased risk of developing hematological malignancies. There are conflicting data whether androgen derivatives (AD) can elongate telomeres in vivo and whether AD treatment enhances the risk of gaining myelodysplastic syndrome-related mutations. Seven TERC or TERT-mutated DKC patients underwent AD treatment. All patients revealed hematological response. Telomere length of lymphocytes and granulocytes increased significantly and no MDS-related mutations were detected. Pending longer follow-up, treatment with AD seems to represent an efficient and safe therapy for DKC patients.

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