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Haploidentical α/β T-cell and B-cell Depleted Stem Cell Transplantation in Seve...

Faraci, M; Giardino, S; Podestà, M; Pierri, F; Dell’Orso, G; Beccaria, A; Farela Neves, J; Volpi, S; Gattorno, M

Objective: Mevalonic aciduria represents the most severe form of mevalonate kinase deficiency (MKD). Patients with mevalonic aciduria have an incomplete response even to high doses of anti-cytokine drugs such as anakinra or canakinumab and stem cell transplantation (SCT) represents a possible therapy for this severe disease. Methods: We report the first two children affected by severe MKD who received haploiden...

Date: 2021   |   Origin: Repositório do Centro Hospitalar de Lisboa Central, EPE
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