Fouquet G, Macro M, Decaux O, Fohrer C, Guidez S, Demarquette H, Le Grand C, Prodhomme C, Renaud L, Bories C, Herbaux C, Karlin L, Roussel M, Benboubker L, Hulin C, Arnulf B, Leleu X
Service des maladies du sang, hôpital Huriez, CHRU de Caen, rue Michel Polonovski, 59037 Lille, France.
Hématologie, hôpital Côte-de-Nacre, CHU, 1400 Caen, France.
Rev Med Interne. 2015 Sep;36(9):613-8. doi: 10.1016/j.revmed.2015.04.007. Epub 2015 Aug 6.
Once characterized by a very poor outcome, multiple myeloma (MM) now has a significantly prolonged survival, with major improvements allowed by the use of "novel agents": proteasome inhibitors (first-in-class bortezomib) and immunomodulatory compounds (IMiDs; first-in-class thalidomide and lenalidomide). However, the vast majority - if not all - of patients with MM ultimately end up being refractory to all existing drugs, including these efficient novel agents. There is a clear unmet medical need in this situation, which warrants the development of the next generation of proteasome inhibitors and IMiDs, as well as new drug classes. This review focuses on pomalidomide, the next generation IMiD, recently approved by the US FDA and the EMA for patients with relapsed or refractory MM who have received at least two prior therapies, including lenalidomide and bortezomib, and have demonstrated disease progression on their last therapy.
曾经，多发性骨髓瘤（MM）的预后非常差，但如今患者的生存期已显著延长，这主要得益于“新型药物”的使用：蛋白酶体抑制剂（首个此类药物硼替佐米）和免疫调节化合物（免疫调节剂；首个此类药物沙利度胺和来那度胺）。然而，绝大多数（即便不是所有）MM患者最终都会对所有现有药物产生耐药性，包括这些有效的新型药物。在这种情况下，显然存在尚未满足的医疗需求，这就需要研发新一代蛋白酶体抑制剂和免疫调节剂，以及新的药物类别。本综述聚焦于泊马度胺，这是新一代免疫调节剂，最近已获美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）批准，用于治疗复发或难治性MM患者，这些患者此前至少接受过两种治疗，包括来那度胺和硼替佐米，且在最后一次治疗中病情仍有进展。
