Table 4 Summary of studies evaluating treatment options for EMD.
Author | Patient group | Treatment arm (% of patients) | Type of EMD | Complete response rate (%) | Median PFS (months) | Median OS (months) | Limitation of study | Ref |
|---|---|---|---|---|---|---|---|---|
Gagelmann et al. [28] | Newly diagnosed MM with EMD (488)—40% with high risk cytogenetics | Bortezomib-based induction (73) | 21 | 4 year PFS-42% | 4 year OS-69% | Absence of data on maintenance therapy, salvage treatment, or details on induction therapy beyond whether bortezomib was used or not | [37] | |
Non-bortezomib-based induction (27) | 17 | 4 year PFS-34% | 4 year OS-64% | |||||
First line ASCT (77) | 4 year PFS-43% | 4 year OS-70% | ||||||
Tandem ASCT (17) | 4 year PFS-52% | 4 year OS-83% | ||||||
Auto–allogeniec transplant (6) | 4 year PFS-58% | 4 year OS-88% | ||||||
Beksac et al. [46] | Newly diagnosed EMD (130/226) | Initial therapy—IMiD-based (74.7%)/ PI-based (10%) followed by ASCT (51.5%) | Bone-independent MM | 19.3 | 38.9 | 46.5 | Selection bias—age < 45 not included | [56] |
Bone-associated MM | 34.2 | 51.7 | N.R. | |||||
EMD at relapse (96/226) | Initial therapy—IMiD-based (10.4)/ PI- based(41.7%) followed by ASCT (4.1%) | Bone-independent MM | 9 | 13.6 | 11.4 | |||
Bone-associated MM | 54.5 | 20.9 | 39.8 | |||||
Gagelmann et al. [9] | Adult patients with EMD at diagnosis who received single ASCT within 12 months of diagnosis or a tandem ASCT within six months from first ASCT as first line therapy (682/3744) | Pre-ASCT | Bone-independent MM | 11.7 | N.R. (3 year PFS-59.8%) | N.R. (3 year OS-83.6%) | Selection bias—elderly patients not transplanted are not included | [10] |
Bone-associated MM | 21.5 | |||||||
Post-ASCT | Bone-independent MM | 36.1 | 24 | N.R. (3 year OS-58%) | ||||
Bone-associated MM | 41.6 | 36 | N.R. (3 year OS-77.7%) | |||||
Post-tandem ASCT | Bone-independent MM | N.R. (3 year PFS-56.2%) | N.R. (3 year OS-52%) | |||||
Bone-associated MM | N.R. (3 year PFS-59.4%) | N.R. (3 year OS-82.6%) | ||||||
Kumar L et al. [31] | EMD at diagnosis or prior to ASCT (44/271) with 200 mg/m2 melphan conditioning | Initial therapy- Novel agents (52.3%) | EMD | 52.2% (12/23) | 18 | 32 | Small sample size. Lack of cytogenetic data. | [42] |
VDD and alkylating agents (47.7%) | 9% (2/21) | |||||||
Shin et al. [24] | EMD at diagnosis or prior to ASCT with 88.2% patients receiving 200 mg/m2 melphan conditioning (93/239) | Initial Therapy- TCD (34.5%)/ VAD (27.6%)/ RT (51.7%) | Bone-independent MM | 31 | 12 | 37 | [33] | |
Initial Therapy- TCD (29.7%)/ VAD (37.5%)/ RT (45.3%) | Bone- associated MM | 40.6 | 28 | 67 | ||||
Gozzetti et al. [35] | Intra cranial-MM (50) | Autologous/ allogenic SCT- (24%) | CNS EMD and osteodural EMD | 50% | 34 | 46 | [63] | |
Chemotherapy- novel and old agents (72%) | 5 | 12 | ||||||
RT (32%) | 12 | 25 | ||||||
Short et al. [7] | EMD in relapsed refractory MM (13/174) Patients had prior exposure to bortezomib (78%), IMiD agents -thalidomide or lenalidomide (100%) before the diagnosis of EMD | Pomalidomide plus low-dose dexamethasone in phase II clinical trial | Primary bone-independent MM | 15.4 | 16 | Only included bone-independent MM. Bone-associated MM were excluded | [7] | |
Treatment-emergent Bone-independent MM |
