Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial

• Main Authors: Veroniek E. M. Harbers (Author), Lilly G. J. M. Zwerink (Author), Gerard A. Rongen (Author), Willemijn M. Klein (Author), Carine J. M. van derVleuten (Author), Ingrid M. P. vanRijnsoever (Author), Lynda Gerdsen‐Drury (Author), Uta E. Flucke (Author), Bas H. Verhoeven (Author), Peter C. J. deLaat (Author), Chantal M. A. M. van derHorst (Author), Leo J. Schultze Kool (Author), D. Maroeska W. M. teLoo (Author)
• Format: Book
• Published: Wiley, 2023-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract The clinical presentation of patients with slow‐flow vascular malformations is very heterogeneous. High clinical burden and subsequent reduced health‐related quality of life is something they have in common. There is an unmet medical need for these patients for whom regular treatments like...