The efficacy and safety of allogeneic stem cell transplantation in Mevalonate Kinase Deficiency

• Main Authors: Jerold Jeyaratnam (Author), Maura Faraci (Author), Andrew R. Gennery (Author), Katarzyna Drabko (Author), Mattia Algeri (Author), Akira Morimoto (Author), Tiarlan Sirait (Author), Arjan C. Lankester (Author), Michael Albert (Author), Benedicte Neven (Author), Joost Frenkel (Author), on behalf of the EBMT Inborn Errors Working Party (Author)
• Format: Book
• Published: BMC, 2022-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Objectives Mevalonate kinase deficiency (MKD) is a rare autoinflammatory syndrome. Several reports have described allogeneic hematopoietic stem cell transplantation in severely affected patients, sometimes with promising results. In view of the scarcity of data, this study aims to analyse t...