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Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia.

In English

Författare

Summary, in English

Diamond-Blackfan anemia (DBA) is a congenital erythroid hypoplastic anemia, characterized by macrocytic anemia, reticulocytopenia, and severely reduced numbers of erythroid precursors in the bone marrow. For more than fifty years, glucocorticoids have remained the main option for pharmacological treatment of DBA. While continuous glucocorticoid administration increases hemoglobin levels in a majority of DBA patients, it also causes severe side effects. There is therefore a great need for more specific and effective treatments to boost or replace the use of glucocorticoids. Over the years, many alternative therapies have been tried out, but most of them have shown to be ineffective. Here we review previous and current attempts to develop such alternative therapies for DBA. We further discuss how emerging knowledge regarding the pathological mechanism in DBA and the therapeutic mechanism of glucocorticoids treatment may reveal novel drug targets for DBA treatment.

Publiceringsår

2012

Språk

Engelska

Publikation/Tidskrift/Serie

The Scientific World Journal

Volym

2012

Dokumenttyp

Artikel i tidskrift

Förlag

Hindawi Limited

Ämne

  • Hematology

Status

Published

Forskningsgrupp

  • Stem Cells to Red Blood Cells

ISBN/ISSN/Övrigt

  • ISSN: 2356-6140