Table 4.
Treatment outcomes in patients with RRMM and EMD.
| Reference Study type | Total number of patients/ number with EMD | Time period covered | Treatments | PFS | OS | Other |
|---|---|---|---|---|---|---|
| Novel agents (various) and/or SCT | ||||||
|
Avivi et al. [37] Retrospective |
127 (all EMD) | 2010–2018 | First treatment included PIs (50%), IMiDs (39%), monoclonal antibodies (10%), and chemotherapy (53%) | – | – | 57% ORR ( ≥ PR) across all treatments |
|
Rasche et al. [21] Single-center registry |
24 (all EMD) | 2007–2010 |
Radiotherapy (n = 16) ASCT + intense dose chemotherapy (n = 6) Bortezomib (n = 16) Lenalidomide (n = 12) Thalidomide (n = 8) |
Median (95% CI): 2 months (0.08–3.92) | Median (95% CI): 7 months (3.56–10.43) | – |
|
Retrospective |
96 EMD, n = 84 |
2010–2017 | Median two lines of treatment and ASCT (44%) | Median (95% CI): 9.1 months (11.6–15.6) | Median (95% CI): 11.4 months (0.6–16.2) | Complete remission rate: 9% (vs 54.5% in patients with paraskeletal, p < 0.001) |
| IMiDs | ||||||
|
Short et al. [30] Phase 2 trial |
16 (all EMD) | 2007–2010 | Pomalidomide + low-dose dexamethasone | – | Median: 16 months | – |
| PIs | ||||||
|
Zhou et al. [39] Retrospective |
45 EMD, n = 25 |
Carfilzomib + dexamethasone-based | EMD significantly inferior PFS vs paraskeletal (p = 0.004) | EMD significantly inferior OS vs paraskeletal (p = 0.04) | – | |
|
Papanikolaou et al. [40] Retrospective |
28 (all EMD) | 1998–2011 |
At relapse:Bortezomib-containing regimens (32%) Platinum-containing (21%) Lenalidomide (21%) VAD (8%) |
– |
Median following relapse: 5 months Median from MM diagnosis: 38 months |
– |
| Chemotherapy/radiotherapy | ||||||
|
Rasche et al. [41] Retrospective |
11 (all EMD) | 2007–2012 | Dexa-BEAM (including dexamethasone, carmustine, cytarabine, etoposide, and melphalan) | Median: 4 months | – | Objective response (≥PR) achieved in 6/11 patients |
| Recently approved and investigational agents | ||||||
|
Richardson et al. [42] Phase 2 |
55 EMD, n = 27 |
Patients enrolled between Dec 2016 and Oct 2019 | Melflufen + dexamethasone | – | – |
ORR: Non-EMD, 32% Paraskeletal, 25% EMD, 22% |
|
Wang et al. [43] Single-center |
57 EMD, n = 17 |
2016–2018 | LCAR-B38M | Median: 8.1 months (vs 25 months in non-EMD; p < 0.001) | Median: 13.9 months (vs NR in non-EMD; p = 0.0019) | 82% ORR (≥ PR) vs 90% for non-EMD |
ASCT autologous stem cell transplantation, EMD extramedullary disease, MM multiple myeloma, NC not calculable, NR not reached, ORR overall response rate, OS overall survival, PFS progression-free survival, PR partial response, VAD vincristine/adriamycin/dexamethasone.