Table 3.

Treatment outcomes in patients with NDMM and EMD.

Reference Study type	Total number of patients/ number with EMD	Time period covered	Treatments	PFS	OS
Montefusco et al. [26] Meta-analysis of 8 trials	267 EMD, n = 12	2010–2018 (across all studies)	IMiD-based therapy (n = 166), PI-based (n = 66), IMiD+ PI-based (n = 35)	Median (95% CI): 26.1 months (8.0–NR)	2-year: 35% Median (95% CI): 70.1 months (16.9–NR)
Batsukh et al. [35] Retrospective	64 EMD, n = 22	2009–2016	Bortezomib/dexamethasone (n = 7) Thalidomide/dexamethasone (n = 23) Bortezomib/thalidomide/dexamethasone (n = 11) Bortezomib/melphalan/prednisone (n = 23) Lenalidomide/dexamethasone (n = 1) Mlphalan/prednisolone or dexamethasone (n = 6) ASCT (n = 28)	Median (95% CI): 16.0 months (5.8–26.2)	Median (95% CI): 27.8 months (5.8–26.5)
Beksac et al. [31] Retrospective	130 EMD, n = 92	2010–2017	Median two lines of treatment and ASCT (44%)	Median (95% CI): 38.9 months (23.6–54.2)	Median (95% CI): 46.5 months (25.5–67.5)
Gagelmann et al. [36] EBMT registry analysis	488 EMD, n = 87	2003–2014	Induction with bortezomib (n = 355) vs non-bortezomib (n = 133) Transplants: autologous (n = 373) or tandem autologous (n = 84) or autologous-allogeneic transplant (n = 31)	3-year (range): 39% (27–52)	3-year (range): 60% (48–73)
Gagelmann et al. [28] EBMT registry analysis	682 EMD, n = 139	2005–2014	Upfront single ASCT within 12 months of diagnosis or a tandem ASCT within 6 months from first ASCT as first-line therapy	3-year (range): 39.9% (30.3–49.5)	3-year (range): 58.0% (48.1–67.9)

ASCT autologous stem-cell transplantation, EBMT European Society for Blood and Marrow Transplantation, EMD extramedullary disease, IMiD immunomodulatory drugs, OS overall survival, PFS progression-free survival, PI proteasome inhibitor.