Pomalyst for amyloidosis – pro
Pomalidomide is a next-generation immunomodulatory agent with activity in relapsed light chain (AL) amyloidosis, with high response rates in phase I/II studies. A recent review by Sharpley concluded that responses are rapid and early responses may be predictive of the sustained overall response. Deep responses (VGPR or better) are seen in only a third of all patients.
V. Sanchorawala et al, Pomalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 1 and 2 tria. Blood (2016): 128 Issue: 8 Pages: 1059-1062 Sharpley FA et al, Real world outcomes of pomalidomide for treatment of relapsed light chain amyloidosis.Br J Haematol. 2018 Nov;183(4):557-563. Angela Dispenzieri, Opening a can of worms in AL progression. Blood 2018 131:475-476;