Review Article

Smoldering Multiple Myeloma

Table 2

Clinical trials for patients with SMM.

Author yearTrial designTherapyNumber and type of patientsOutcome

Hjorth et al. 1993 [7]RCTInitial versus deferred MP50 stage I MM (DSS) No difference in RR, response duration, or OS

Riccardi et al. 2000 and 1994 [5, 6]RCTInitial versus deferred MP145 stage I MM (DSS)No difference in RR or OS

Rajkumar et al. 2003 [8]Single-arm phase 2Thalidomide16 SMM or IMM11 of 16 patients responded to therapy

Martín et al. 2002 [32]Single-arm pilotPamidronate12 SMM or IMMReduces bone turnover but has no antitumour effect

Weber et al. 2003 [9]Single-arm phase 2Thalidomide28 SMMRR was 36% and median time to remission was 4.2 m

Musto et al. 2008 [35]RCTZoledronic acid versus observation163 SMMNo difference in PFS and TTP; reduce skeletal-related events

Barlogie et al. 2008 [10]Phase 2Thalidomide with monthly pamidronate76 SMM4-y OS and PFS were 91% and 60%, respectively

D’Arena et al. 2011 [33]RCTPamidronate versus observation177 SMMNo difference in PFS, TTP, or OS; reduce skeletal events

Witzig et al. 2013 [11]RCTThalidomide plus zoledronic acid versus zoledronic acid 68 SMMSignificant difference in TTP and PFS; no difference in OS

Mateos et al. 2013 [12]RCTLenalidomide plus dexamethasone versus observation119 high-risk SMMTTP: treatment: not reached; observation: 21 m; 3-y OS: treatment: 94%; observation: 80%

MP: melphalan and prednisone; DSS: Durie and Salmon stage; RR: response rate; OS: overall survival; PFS: progression-free survival; RCT: randomized controlled trial; IMM: indolent multiple myeloma; m: month; y: year.