Cancer Med. 2026 Aug;15(8):e72210. doi: 10.1002/cam4.72210.
ABSTRACT
Extramedullary disease (EMD) in newly diagnosed multiple myeloma (NDMM) is aggressive and associated with worse survival. This study aimed to comprehensively evaluate the clinical outcomes and factors affecting prognosis in NDMM patients with EMD receiving novel agents-based induction therapy. EMD presented in 94 cases (21.2%) among 443 screened NDMM patients. Eighty-five EMD patients with complete clinical and follow-up data were subdivided into extramedullary bone-related disease (EMB, n = 55) and extramedullary extraosseous disease (EME, n = 30). EMD patients did not present a significantly high tumor burden or worse disease stage than non-EMD patients. With novel agents-based induction therapy, the overall response (OR) rate in the EMD group was significantly lower than in the non-EMD group (88.2% vs 96.8%, p = 0.005), but was comparable between EMB and EME (90.9% vs 83.3%, p = 0.494). The number of patients achieving complete remission (CR) or stringent CR (sCR) in the EME group was significantly less than that in the EMB or non-EMD group. After a median follow-up of 49 months, EME patients showed significantly inferior progression-free survival (PFS) compared with non-EMD and EMB patients (median PFS: 20 vs 39 vs 32 months, p = 0.000). Exploratory subgroup analyses suggested that patients with EME and high-risk cytogenetic abnormalities (HRCAs) experienced the most unfavorable survival outcomes, although these findings require validation in larger cohorts. Patients achieving sCR/CR demonstrated significantly prolonged PFS and overall survival (OS). Autologous stem cell transplantation (ASCT) was associated with improved survival. Novel agents-based induction therapy achieved favorable responses and encouraging survival outcomes in patients with EMD, particularly among those achieving deep remissions. EME patients appear to represent a clinically aggressive disease phenotype whose adverse prognosis is closely intertwined with high-risk clinical and cytogenetic characteristics rather than being entirely independent. Larger prospective multicenter studies are warranted to validate these findings and optimize treatment strategies for patients with EMD.
PMID:42626847 | DOI:10.1002/cam4.72210