Arvelige erytrocytsygdomme
Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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Arvelige erytrocytsygdomme. / Glenthøj, Andreas.
I: Ugeskrift for Laeger, Bind 183, Nr. 42, V01210080, 18.10.2021.Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt
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TY - JOUR
T1 - Arvelige erytrocytsygdomme
AU - Glenthøj, Andreas
PY - 2021/10/18
Y1 - 2021/10/18
N2 - Hereditary anaemias are the most prevalent genetic disorders worldwide. Until recently, treatment options were mostly supportive or surgical, i.e. splenectomy. Recently, several medical treatments designed for frequent haemoglobinopathies such as thalassaemia and sickle cell disease have become available, and numerous new clinical trials hold promise of many more to come. Even rare anaemias such as pyruvate kinase deficiency have promising clinical trials with targeted therapies. Together, these herald hope for future treatment options for patients living with hereditary anaemias, which is discussed in this review.
AB - Hereditary anaemias are the most prevalent genetic disorders worldwide. Until recently, treatment options were mostly supportive or surgical, i.e. splenectomy. Recently, several medical treatments designed for frequent haemoglobinopathies such as thalassaemia and sickle cell disease have become available, and numerous new clinical trials hold promise of many more to come. Even rare anaemias such as pyruvate kinase deficiency have promising clinical trials with targeted therapies. Together, these herald hope for future treatment options for patients living with hereditary anaemias, which is discussed in this review.
KW - Anemia, Hemolytic, Congenital Nonspherocytic
KW - Anemia, Sickle Cell
KW - Hemoglobinopathies
KW - Humans
KW - Splenectomy
KW - Thalassemia
M3 - Tidsskriftartikel
C2 - 34709160
VL - 183
JO - Ugeskrift for Laeger
JF - Ugeskrift for Laeger
SN - 0041-5782
IS - 42
M1 - V01210080
ER -
ID: 305547064