International Journal of Hematology最新文献

The European LeukemiaNet recommendations for the management of chronic myeloid leukemia (CML) consider achievement of major molecular response (MMR) within 12 months to be an optimal response (OR). However, no currently available tool can predict treatment response at diagnosis. This study aimed to develop a predictive scoring tool for OR and subsequent deep molecular response (DMR) using peripheral blood parameters at diagnosis. A retrospective analysis was conducted in 535 patients with CML from the CML Cooperative Study Group database. Patients were categorized into OR (MMR within 12 months; n = 355) and non-OR groups (MMR after 13 months; n = 180). Logistic regression analysis identified white blood cell count, platelet count, eosinophil percentage, and imatinib use as significant predictors of OR. These variables were used to construct a novel score. The score was significantly higher in the non-OR group and showed superior predictive accuracy for OR compared with existing prognostic scores. Furthermore, lower scores correlated with higher rates of DMR achievement. Notably, the score effectively predicted OR achievement among patients with low/intermediate ELTS risk treated with imatinib. This new scoring tool may facilitate individualized treatment selection in CML, guiding upfront tyrosine kinase inhibitor selection and advancing precision medicine.

Acute graft-versus-host disease (GVHD) remains a major complication after allogeneic hematopoietic stem cell transplantation. Traditional risk stratification based on clinical symptoms provides only modest prognostic accuracy for treatment response and long-term outcomes. Several biomarkers have shown promise in improving predictive and prognostic precision. Biomarker-guided strategies can identify high-risk patients before extensive target organ damage occurs and low-risk patients who may benefit from lower-intensity treatment, thereby reducing GVHD-related organ injury and adverse effects such as infection and disease relapse. These approaches have facilitated more personalized treatment, with some studies reporting reduced infection rates and improved GVHD-related outcomes in appropriately selected populations. Biomarker-driven clinical trial designs represent a promising framework for developing risk-adapted GVHD management strategies; however, continued validation and refinement of biomarker algorithms is essential to optimize patient outcomes.

Central nervous system (CNS) involvement in plasma cell neoplasms is rare and associated with poor prognosis, and indications for cerebrospinal fluid (CSF) analysis remain undefined. We describe three cases: two with advanced-stage, high-risk cytogenetics, and one stage I case meeting criteria for plasma cell leukemia (PCL). In two patients, delayed CSF evaluation after neurological symptoms led to rapid deterioration, whereas early CSF analysis in an asymptomatic patient enabled timely diagnosis. One case worsened acutely after treatment with plerixafor. These findings suggest that CSF evaluation may merit consideration in high-risk patients, including those with PCL or before plerixafor administration.

Neutropenic patients with hematological malignancies are at high risk for infectious complications. Whether associated lymphopenia or hypogammaglobulinemia further increase this risk and affect outcome remains unclear. This retrospective single-center study included 321 patients hospitalized with lymphoma or leukemia and severe neutropenia whose serum immunoglobulin levels were measured. Overall, 60% of patients had isolated lymphopenia, 9% isolated hypogammaglobulinemia, 24% both, and 7% none. There was no correlation between absolute lymphocyte count and IgG. Severe lymphopenia was not associated with infection or mortality. Hypogammaglobulinemia was more prevalent in lymphoid leukemias and lymphomas. A multivariate logistic regression analysis adjusting for age, underlying hematological malignancy, absolute neutrophil and lymphocyte counts, and duration of neutropenia and lymphopenia showed that hypogammaglobulinemia was associated with infection (OR 1.85, 95% CI 1.07-3.26, p = 0.03), pneumonia (OR 2.04, 95% CI 1.13-3.72, p = 0.02), and sepsis or septic shock (OR 2.43, 95% CI 1.26-4.72, p = 0.01). This indicates a need for further investigation into the role of immunoglobulin replacement therapy in this setting.

Background: Iron deficiency anemia (IDA) represents a major public health concern in India, affecting mostly women of reproductive age due to increased iron demands throughout their reproductive life. However, specific erythrocyte morphology and its possible consequences in this population remain unexplored.

Objective: This study systematically examined erythrocyte abnormalities in reproductive-age women with IDA.

Methods: Twenty-five women diagnosed with IDA and 25 age-matched healthy women were recruited. Scanning electron microscopy, atomic force microscopy (AFM), flow cytometry, confocal laser scanning microscopy, and spectrophotometry were used in this study.

Results: Patients with IDA showed significant counts of elliptocytes, microcytes, stomatocytes, and spherocytes, which were negatively correlated with hemoglobin concentration. Serum iron and total iron binding capacity were strongly correlated with microcytes and mean corpuscular hemoglobin concentration. AFM revealed a notable presence of disrupted membrane topology and roughness in IDA patients. Redox balance assessed by ferric reducing antioxidant power of plasma and intracellular ROS levels was significantly impaired. IDA erythrocytes also showed increased intracellular Ca²⁺ and phosphatidyl serine externalization.

Conclusion: Patients with IDA exhibited not only significant hematologic impairment but also morphologically altered erythrocytes and redox imbalance, which could promote eryptosis.

Background: Elranatamab, a bispecific antibody targeting B-cell maturation antigen and CD3, has demonstrated efficacy in relapsed/refractory multiple myeloma (RRMM). However, real-world evidence is limited, and prognostic factors have not been sufficiently evaluated.

Methods: We retrospectively analyzed patients with RRMM receiving elranatamab between June 2024 and July 2025. Responses were assessed using the International Myeloma Working Group criteria. Progression-free survival (PFS) and overall survival (OS) were estimated using the Kaplan-Meier method. Prognostic factors were evaluated using univariate log-rank analysis, including R2-ISS at elranatamab initiation (dynamic R2-ISS) and modified MyCARe risk, with positivity defined as any detectable plasma cells in peripheral blood.

Results: Thirty-seven patients were evaluated (median age, 67 years; prior lines, 5). Median follow-up was 7.5 (median PFS, 9.7) months. One-year OS was 66.3%. The overall response rate was 67.6%, and 45.9% achieved at least a very good partial response. Extramedullary disease, plasma cells in the peripheral blood, and high-risk cytogenetics were adverse features. A baseline monocyte count < 300/µL, high-risk classification by modified MyCARe, and dynamic R2-ISS stage IV were significantly associated with shorter PFS and OS.

Conclusion: Elranatamab was effective in real-world settings. Baseline monocyte count, modified MyCARe risk, and dynamic R2-ISS may serve as practical prognostic tools.

Polatuzumab vedotin plus rituximab, cyclophosphamide, doxorubicin, and prednisone (Pola-R-CHP) was approved in Japan for the treatment of previously untreated diffuse large B-cell lymphoma (DLBCL) in 2022, based on findings of the POLARIX study (NCT03274492). Reports on real-world usage of Pola-R-CHP are lacking. Here we report safety and response rates at end of treatment (EOT) for Pola-R-CHP in Japan from the real-world observational POLASTAR study (jRCT1071220082). Patients (≥ 18 years) with previously untreated DLBCL who were scheduled to receive Pola-R-CHP were enrolled. The primary endpoint was overall survival. As of December 20, 2023, the full analysis set (FAS) included 192 of the initial 199 patients enrolled. Median age was 71.0 years (range 30-91). In the FAS, 99 (51.6%) patients had Grade ≥ 3 adverse events (AEs), 29 (15.1%) had serious AEs, and 15 (7.8%) discontinued polatuzumab vedotin due to AEs. In the efficacy-evaluable population at EOT (n = 141), the overall response rate was 95.0% [95% confidence interval (CI), 90.1-97.6], and the complete response rate was 87.9% (95% CI, 81.5-92.3). These results are consistent with published data from POLARIX. The POLASTAR study is ongoing; the recruitment target of 500 patients has been reached.Clinical trial registration: Japan Registry of Clinical Trials (jRCT1071220082).

This study characterized the phenotypic and cytogenetic features of circulating tumor plasma cells (CTPCs) in patients with monoclonal gammopathy of undetermined significance (MGUS) and smoldering multiple myeloma (SMM). A total of 120 patients were analyzed (MGUS, n = 70; SMM, n = 50). CTPCs were detected in 38.2% of MGUS and 61.8% of SMM. Immunophenotypic analysis based on mean fluorescence intensity revealed that CD200, CD117, CD38, CD56, and CD28 were significantly downregulated and CD45 was significantly overexpressed in CTPCs compared with BMPCs, whereas CD138 and CD81 expression levels did not differ. Cytogenetic analysis demonstrated higher prevalence of del(13q) and high-risk chromosomal abnormalities in CTPC-positive patients than CTPC-negative patients (24.0% vs. 4.5%, p = 0.048; 26.5% vs. 7.0%, p = 0.011, respectively). No significant differences were observed in other cytogenetic abnormalities including 1q gain/amplification, t(4;14), t(11;14), t(14;16), or del(17p). Serum soluble B cell maturation antigen levels did not differ between patients with or without detectable CTPCs in MGUS or SMM. These findings highlight the distinct phenotypic and cytogenetic characteristics of CTPCs in MGUS and SMM, and provide insights regarding their biological features and pathogenesis.

Patients with primary plasma cell leukemia (pPCL), particularly those with extramedullary disease (EMD), face a poor prognosis even with chimeric antigen receptor (CAR)-T cell therapy. This case report describes a patient with relapsed/refractory pPCL and life-threatening malignant pleural effusion (PE) treated with intrapleural CAR-T cells targeting B-cell maturation antigens. CAR-T cell expansion within the PE was observed, along with a rapid reduction in leukemia cell count and PE volume. Subsequent lymphodepletion chemotherapy and intravenous CAR-T cell therapy induced complete remission with 9 months of progression-free survival. This case highlights the potential advantages of integrating local and systemic CAR-T cell therapy in managing complex EMD cases.