Background: Transplantation-related mortality (TRM) is a major obstacle in allogeneic hematopoietic cell transplantation (allo-HCT). Approximately 60–80% of TRM occurs early, within 100 days of transplantation. Methods: This was a nationwide population cohort study involving 5395 patients with acute leukemia who underwent allo-HCT between 2003 and 2015. Patient data were collected from the Korean National Health Insurance Service database. We investigated the cumulative incidence rates (CIRs) of early TRM at 50 and 100 days. Results: The CIRs of early TRM at 50 and 100 days were 2.9 and 8.3%, respectively. There was no decrease in the CIRs of early TRM over time. The early mortality was significantly higher in patients with more than 9 months between the diagnosis and transplantation (CIRs of TRM at 50, 100 days; 6.0, 13.2%), previous transplantations (CIRs of TRM at 50, 100 days; 9.4, 17.2%), and cord blood transplantation (CIRs of TRM at 50, 100 days; 6.1, 8.3%). The early TRM was significantly lower in patients who received iron chelation before transplantation (CIRs of TRM at 50, 100 days; 0.3, 1.8%). Conclusions: In conclusion, the overall CIR of early TRM was less than 10%. The predictable factors for early TRM included age, time from diagnosis to transplantation, the number of prior transplantations, the graft source, and previous iron chelation therapy.

Background: The response rate and survival improvement for rituximab, a CD20‐targeting monoclonal antibody, have been demonstrated in marginal zone lymphoma (MZL) as monotherapy and in combination with chemotherapeutic regimens, yet relapses still occur despite treatment completion. Thus, extending the period of remission in MZL patients remains an essential goal. This multicenter, single‐arm, open‐label phase II study evaluated the survival efficacy of 2 years of rituximab‐maintenance therapy in patients with stage III–IV CD20‐positive MZL who had responded to first‐line R–CVP (rituximab, cyclophosphamide, vincristine, and prednisolone). The objective of this study was to determine whether rituximab maintenance following R–CVP warrants further investigation. Methods: Prior to rituximab‐maintenance therapy, patients received 6–8 cycles of first‐line R–CVP therapy for stage III–IV MZL. Rituximab (375 mg/m ² ), cyclophosphamide (750 mg/m ² ), and vincristine (1.4 mg/m ² ; maximum 2 mg) were administered via an intravenous infusion on day 1 of each 3‐week cycle, while oral prednisolone (100 mg) was given on days 1–5 of each 3‐week cycle. The patients who achieved complete response (CR), partial response (PR), or stable disease (SD) to R–CVP treatment, were prescribed rituximab‐maintenance therapy which was administered intravenously at a dose of 375 mg/m ² every 8 weeks for up to 12 cycles. The primary endpoint was progression‐free survival (PFS). Secondary endpoints were overall survival (OS) and treatment safety. Results: 47 patients were enrolled, of whom, 45 (96%) received rituximab‐maintenance treatment. Fifteen (33%) patients had nodal MZL. Following R–CVP first‐line therapy, 20 (44%), 22 (49%), and 3 (7%) patients achieved CR, PR, and SD, respectively. After a median follow‐up of 38.2 months, their observed 3‐year PFS rate was 81%. During the rituximab‐maintenance, 6 PR and 1 SD patients achieved CR following the administration of R–CVP. Elevated LDH and the presence of B symptoms were found to be significant prognostic factors for PFS ( P = 0.003) and demonstrated a 3‐year OS rate of 90%. Rituximab‐maintenance therapy was well tolerated, and the common treatment‐emergent adverse events were sensory neuropathy (18%), myalgia (13%), fatigue (9%), and neutropenia (9%). Conclusion: Rituximab‐maintenance therapy following first‐line R–CVP demonstrated good PFS in patients with stage III–IV MZL, in addition to a favorable toxicity profile. Trial registration clinicaltrials.gov : NCT01213095

Patients with transplant‐ineligible relapsed and refractory multiple myeloma (RRMM) have a short life expectancy, especially when they have failed both the proteasome inhibitor and immunomodulator therapies. This study aimed to assess the efficacy and safety of pomalidomide, cyclophosphamide, and dexamethasone (PCd) in elderly patients with RRMM. This phase 2 clinical trial recruited 55 elderly patients with RRMM. The patients underwent a 28‐day treatment cycle: pomalidomide (4 mg/day on days 1‐21, administered orally) and cyclophosphamide (400 mg/day on days 1, 8, and 15; administered orally) plus dexamethasone. The median (range) age of the patients was 73.3 (64‐86) years, and 8 (14.5%) patients who were ≥ 80 years old. Eight (14.5%) and 31 (56.4%) patients exhibited stage III (revised international staging system) and frail status (simplified frailty scale), respectively. The overall response rate (ORR) and clinical benefit rate (CBR) of PCd therapy were 58.2% and 72.7%, respectively. The median PFS and median overall survival (OS) were 6.90 months (95% CI, 4.7‐9.0) and 18.48 months (95% CI, 9.4‐27.6), respectively. The incidence rate of grade ≥ 3 non‐hematological toxicities was 70.8%. In particular, the incidence rate of primary infection was 45.4%, including 21.8% for pneumonia, 9.0% for sepsis, and 14.6% for febrile neutropenia. In conclusion, PCd is an effective regimen for elderly patients with RRMM who had failed both bortezomib and lenalidomide treatments, but in whom the treatment‐associated infection is the main cause of morbidity and mortality.

Background: The epidemiological features of Waldenström macroglobulinemia (WM) have seldom been investigated at a national level, particularly in East Asia. The goal of our study is to present the incidence, prevalence, mortality, survival with competing risks, and causes of death of patients with WM. Methods: We used a national population-based database, operated by the Health Insurance Review and Assessment Service of the Korean government. This data includes information on all WM patients diagnosed according to uniform criteria, between 2003 and 2016. Results: The total number of patients newly diagnosed with WM during the study period was 427, with a male-to-female ratio of 3.2:1. The incidence increased from 0.03 to 0.10 per 10⁵ between 2003 and 2016, and the prevalence was 0.42 per 10⁵ in 2016. A total of 217 patients with WM died during the study period (standardized mortality ratio = 7.57), and the overall survival (OS) of WM patients was 47.5%. On multivariate analysis, older age was associated with worse OS (P < 0.0001). WM was the most common cause of death (n = 102, 48.6%), followed by other malignant neoplasms (n = 82, 39.0%). Conclusions: The national incidence of WM in Korea, a racially homogeneous country in Asia, was lower than that in previous reports from other countries, reflecting ethnic disparities. However, the incidence increased, and mortality was the highest ever reported. The main cause of death was WM in itself. This study reflects the need for greater awareness of WM, particularly in Asian countries.

Clinicians and healthcare decision-makers conduct their clinical practice based on the results of clinical trials. However, some health problems remain unresolved; in such cases, further research is required. To ensure reliable research results, it is important to understand the study design and conduct well-designed clinical trials. Many study designs can be chosen within the two broad categories of observational and interventional. Clinical studies have a variety of designs, including case series, case-control, cross-sectional, and prospective and retrospective cohort studies. Well-designed clinical studies can clarify important differences between treatment options and provide data on long-term drug efficacy and safety. Interpreting the results of clinical trials can be difficult because weaknesses in research design, data collection methods, analytic methods, and reporting can compromise their value and usefulness. However, although randomized controlled trials are limited owing to ethical and practical issues, they are optimal for investigating the effects of therapy and establishing causality. Here we present an overview of different clinical research designs and review their advantages and limitations.