Yeung-Chul Mun

The role of allogeneic hematopoietic cell transplantation (alloHCT) as first-line therapy in elderly patients with acquired aplastic anemia (AA) remains uncertain. While immunosuppressive therapy (IST) is the standard initial treatment for patients aged ≥40 years, recent evidence suggests that upfront alloHCT may offer improved outcomes. We conducted a retrospective multicenter study of 173 Korean patients aged ≥40 years who underwent alloHCT between 1999 and 2017. Patients received either upfront alloHCT without prior IST (n=66) or delayed alloHCT following IST failure or relapse (n=107). The upfront group had a higher rate of matched sibling donors (78.8% vs. 50.5%, p<0.001) and shorter time to transplant (median, 3.4 vs. 8.3 months; p=0.046). Rates of engraftment, hematopoietic recovery, and acute and chronic graft-versus-host disease were comparable. Five-year overall survival was higher in the upfront group (73.5% vs. 56.4%, p=0.059). In multivariate analysis, upfront alloHCT (hazard ratio [HR], 2.476; p=0.004) and ECOG performance status ≤1 (HR, 4.736; p<0.001) were independently associated with improved survival. These findings suggest that upfront alloHCT may be a viable first-line option for selected elderly AA patients and support re-evaluation of treatment algorithms that currently prioritize IST.

Background and Objectives: Essential thrombocythemia (ET) is a myeloproliferative neoplasm that overproduces platelets and is associated with life-threatening thrombosis. Medical cytoreduction either with hydroxyurea (HU) or anagrelide (AG) is widely used, but drug intolerance or resistance are major concerns. Low-dose combination of HU and AG as an alternative strategy has been explored in various studies. It showed comparable response with acceptable toxicity in second-line settings for patients who experienced side effects from prior monotherapy. In this study, we evaluated the efficacy and safety of upfront combination for ET patients. Materials and Methods: From January 2018 to June 2022, a total of 241 ET patients with intermediate to high risk were enrolled. We identified 21 patients with initial drug combinations and compared treatment outcomes and adverse events (AEs) between combination and monotherapy groups. Results: The median age was 62 years old (range, 26–87) and median platelet count was 912 × 10⁹/L (range, 520–1720). Overall treatment response did not exhibit significant differences between the groups, although there was a trend towards a lower response rate in patients treated with AG alone at 3 months post-treatment (AG + HU, 85.7% vs. AG alone, 75.4%, p = 0.068). AEs of any grade occurred in 52.3% of the combination group, 44.3% of the HU monotherapy group, and 43.4% of the AG single group, respectively. Of note was that the HU plus AG combination group suffered a lower incidence of grade 3–4 AEs compared to the other two groups, with statistical significance (p = 0.008 for HU monotherapy vs. combination therapy and p < 0.01 for AG monotherapy vs. combination therapy). Conclusions: Our findings demonstrated that the upfront low-dose combination approach showed feasible clinical outcomes with significantly lower severe AEs compared to conventional monotherapy. These results may offer valuable insights to clinicians for future prospective investigations.

This study aimed to identify the benefits of autologous-stem cell transplantation (auto-SCT) and allogeneic-SCT (allo-SCT) in patients with aggressive T-cell lymphomas to aid in the selection of transplantation type in clinical practice. This study retrospectively analyzed data from 598 patients who underwent transplantation for T-cell lymphomas from 2010 to 2020. In total, 317 patients underwent up-front SCT as consolidation therapy. The 3-year progression-free survival (PFS) and overall survival (OS) were 68.7% and 76.1%, respectively. Patients who underwent auto-SCT had significantly better OS (p = 0.026) than those who underwent allo-SCT; however, no statistical difference in PFS was found. Transplantation was used as a salvage therapy in 188 patients who had relapsed/refractory disease. Overall, 96 (51.1%) patients underwent auto-SCT and 92 (48.9%) patients underwent allo-SCT. Auto-SCT improved long-term survival in patients with complete remission (CR). Allo-SCT demonstrated better 3-year PFS in patients with partial remission and relapsed/refractory disease status. However, >50% of patients died within 1 year of allo-SCT. As a consolidative therapy, up-front auto-SCT demonstrated a survival benefit. Auto-SCT was also effective in patients who achieved CR after salvage therapy. If the disease persists or cannot be controlled, allo-SCT may be considered with reduced intensity conditioning.

Kim JS, et al. J Korean Med Sci. 2023 Sep;38:e328. https://doi.org/10.3346/jkms.2023.38.e328

The effectiveness of early treatment for biochemical relapse of multiple myeloma (MM) is unclear. To clarify this issue, this retrospective study was performed to evaluate the survival outcomes of 315 patients with relapsed MM after upfront autologous stem cell transplantation (ASCT). Over a median follow-up of 66.6 months (range, 15.1–195.5 months), 48.2% of patients showed biochemical relapse, 41.3% showed clinical relapse, and 10.5% showed significant biochemical relapse. Progression-free survival (PFS) and overall survival (OS) were inferior for patients with clinical relapse compared to the other patients. Multivariate analysis showed that clinical relapse was an independent prognostic factor for OS. In patients with biochemical relapse, there was no significant difference in survival between patients treated while asymptomatic and those treated once clinical symptoms had appeared. Relapse type after upfront ASCT was a significant prognostic factor in patients with MM. In addition, no survival benefit of early treatment at biochemical relapse was observed, but a triplet regimen may be beneficial for MM patients with biochemical relapse.

Young Hoon Park, Dae-Young Kim, Seongkoo Kim, Young Bae Choi, Dong-Yeop Shin, Jin Seok Kim, Won Sik Lee, Yeung-Chul Mun, Jun Ho Jang, Jong Wook Lee, Hoon Kook, on behalf of Korean Aplastic Anemia Working Party. Blood Res 2022;57:20-8. https://doi.org/10.5045/br.2022.2022043