Fishman J, Anderson S, Talbird SE, Dingli D. Analysis of costs per responder in US adults with a paroxysmal nocturnal hemoglobinuria with suboptimal response to prior eculizumab treatment. Hematol Rep. 2023 Oct 13;15(4):578-91. doi: 10.3390/hematolrep15040060
Ward A, Caro JJ, Green T, Huybrechts K, Arana A, Wait S, Eleftheriou A. An international survey of patients with thalassemia major and their views about sustaining life-long desferrioxamine use. BMC Clin Pharmacol. 2002;2(3).