International Journal of Hematology最新文献

Azacitidine (AZA) monotherapy demonstrates efficacy for post-transplant maintenance in patients with high-risk myeloid malignancies. However, no study has directly compared combination regimens. In this retrospective cohort study, 59 patients (AML = 56, MDS = 3) received AZA-based maintenance post-allogeneic hematopoietic stem cell transplantation (allo-HSCT), as monotherapy (n = 33), AZA plus interferon-α (IFN-α) (n = 15), or AZA plus targeted agents (n = 11). At the median 31-month follow-up, the overall relapse rate was 10.2% (6/59), with comparable rates across groups (AZA: 9.1%, AZA + IFN-α: 13.3%, AZA + targeted: 9.1%) (P = 0.850). Three 3-year relapse-free survival (89.4%; 95%CI 84.7-94.1%) and overall survival (84.4%; 95%CI 78.9-89.9%) rates did not differ significantly between monotherapy and combination regimens (RFS P = 0.975; OS P = 0.770). Overall adverse event rates showed no statistical difference (P > 0.05), although febrile reactions were more common with IFN-α combination regimens (P < 0.001). These findings demonstrate that AZA monotherapy has non-inferior efficacy compared with combination regimens and has a favorable toxicity profile, establishing it as a viable backbone for maintenance therapy in MRD-negative patients. Biomarker-driven combinations warrant prospective validation.

This phase 2 study (NCT05105841) evaluated the safety and efficacy of a fixed-duration 12-cycle regimen of venetoclax plus obinutuzumab in Japanese patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The primary efficacy endpoint was the complete remission (CR)/complete remission with incomplete marrow recovery (CRi) rate, assessed by an independent review committee (IRC) according to the 2008 International Workshop on CLL criteria. Ten patients (6 male, 4 female; 9 CLL, 1 SLL) with a median age of 69.5 years (range 52-76) received venetoclax for a median duration of 11.3 months (range 9.2-12.4). The IRC-assessed CR/CRi rate based on the best overall response was 90.0% (95% confidence interval 55.5%, 99.7%). All patients experienced at least one treatment-emergent adverse event (TEAE), and three patients (30.0%) experienced at least one serious TEAE. The most common TEAEs included infusion-related reactions (60.0%), decreased neutrophil count (50.0%), and nausea (40.0%). Nine patients (90.0%) experienced TEAEs related to venetoclax, while all ten patients (100.0%) had TEAEs related to obinutuzumab. One patient (10.0%) developed COVID-19 pneumonia, necessitating the discontinuation of venetoclax. These findings demonstrate the high efficacy and manageable safety profile of venetoclax plus obinutuzumab in this patient population.

Liver involvement in systemic immunoglobulin light-chain (AL) amyloidosis is diagnosed by an increase in liver size and/or elevated serum alkaline phosphatase (ALP) levels. However, novel diagnostic approaches that reflect the organ-specific characteristics of liver involvement are needed. Shear wave elastography (SWE) is a noninvasive and repeatable technique for measuring tissue stiffness. In this study, we evaluated the clinical significance of SWE and serum biochemistry tests in the diagnosis of AL amyloidosis with liver involvement. Twenty-five patients with systemic AL amyloidosis were examined. Eight patients were diagnosed with liver involvement according to the current consensus criteria. In patients with and without liver involvement, hepatic shear wave velocity (SWV) was 1.93 ± 0.39 versus 1.36 ± 0.11 m/sec (p = 0.00099), and the gamma-glutamyltranspeptidase (γ-GTP) level was 165 ± 131 versus 36 ± 28 U/L (p = 0.00222). Receiver-operating characteristic curve analysis showed that SWV and γ-GTP levels had favorable diagnostic accuracy for liver involvement. Of note, two patients with liver involvement who achieved organ response showed a decrease in both SWV and γ-GTP levels. These results suggest that SWV and γ-GTP are useful for establishing accurate organ-specific diagnosis and response criteria for liver involvement in AL amyloidosis.

Micafungin and other echinocandins are commonly used for the prophylaxis and empirical treatment of invasive fungal infections in neutropenic patients due to their broad-spectrum activity and favorable safety profile. Drug-induced hemolytic anemia (DIHA) is a rare condition, and micafungin-related cases are exceptionally uncommon. We describe a fatal case of intravascular hemolysis triggered by re-administration of micafungin in a patient receiving chemotherapy for peripheral T cell lymphoma. The patient experienced convulsions and shock within minutes of infusion of the drug, followed by severe hemolysis and disseminated intravascular coagulation. Subsequently, hemolytic mechanisms were evaluated using drug-adsorption and immune complex models. Additionally, we had sera from eight patients treated with micafungin between January 2019 and December 2022, including our index case, tested for anti-micafungin antibodies. Immune complex-mediated hemolysis was confirmed in the index case, supported by positive indirect antiglobulin tests and in vitro hemolysis. Anti-micafungin antibodies were only detected in this single patient, indicating an extremely rare immunologic reaction. Although rare, micafungin-induced DIHA can be life-threatening. Clinicians should remain vigilant, particularly when resuming or continuing administration of micafungin.

Multiple myeloma is an incurable plasma cell malignancy. Its prognosis improved with the introduction of proteasome inhibitors, immunomodulatory drugs, and monoclonal antibodies, but outcomes for triple-class-exposed (TCE) myeloma remained poor. New therapeutic options including CAR-T cell therapy and bispecific antibodies have now further improved prognosis, even in TCE myeloma. While true cure remains challenging, functional cure, defined as long-term disease control through a favorable immune environment suppressing minimal residual disease (MRD), is currently considered a realistic therapeutic goal.

The diagnosis-to-treatment interval (DTI) has been identified as a prognostic factor in newly diagnosed diffuse large B-cell lymphoma (DLBCL). However, its significance in relapsed or refractory DLBCL (R/R DLBCL) remains unclear. This study aimed to investigate the impact of DTI on survival outcomes in patients with R/R DLBCL. We reviewed the medical records of patients with R/R DLBCL who received second-line therapy. DTI at relapse or refractory disease (r/r DTI) was defined as the time from radiological diagnosis-to-treatment initiation, with a cut-off of 28 days. The primary endpoint was overall survival (OS). A total of 184 patients with R/R DLBCL were included. Patients with short r/r DTI had significantly worse OS than those with long r/r DTI (1-year OS: 50.4% vs. 79.5%; P < 0.001). Short r/r DTI was associated with adverse clinical features, including poor performance status, elevated lactate dehydrogenase levels, and high International Prognostic Index at relapse. Multivariate analysis demonstrated that short r/r DTI was independently associated with significantly inferior OS (HR 1.77; P = 0.043). In conclusion, DTI is an independent prognostic factor in R/R DLBCL and can be considered in patient selection for clinical trials.

Peripheral T cell lymphomas (PTCLs) are a heterogeneous group of aggressive lymphomas with generally poor outcomes. CHOP or CHOP-like regimens have long been the standard frontline therapy; however, the addition of brentuximab vedotin has improved overall survival and established a new standard of care for CD30-positive PTCL. Despite this advance, the outcomes of relapsed or refractory (R/R) disease remain dismal. In recent years, multiple novel agents have been developed for the treatment of PTCL, particularly for R/R settings. Molecular profiling has refined disease classifications, identifying subtypes such as nodal T follicular helper (TFH) lymphoma, PTCL-GATA3, and PTCL-TBX21, which differ in their pathogenesis, prognosis, and potential therapeutic vulnerabilities. Epigenetic modulators, including histone deacetylase inhibitors and an EZH1/2 inhibitor, have demonstrated particularly high activity in TFH-derived subtypes, supporting the integration of biomarker-driven patient selection as a path toward precision medicine in PTCL. This review summarizes current insights into the genetic landscape, recent clinical advances in novel agents, and future perspectives on therapeutic stratification, highlighting the need to translate molecular insights into durable clinical benefits for patients with PTCL.

Purpose: Although Hodgkin and non-Hodgkin lymphoma are chemosensitive, 10-15% of patients develop refractory disease. With the increasing use of intensive chemotherapy, the role of radiotherapy (RT) in salvage settings has diminished. This study evaluated the efficacy of salvage RT in patients with refractory lymphoma.

Materials and methods: Forty-one adults with histologically confirmed Hodgkin or non-Hodgkin lymphoma and either primary refractory or relapsed/refractory disease treated between 2010 and 2022 were retrospectively analyzed. All patients received involved-site RT after systemic therapy.

Results: At a median follow-up of 67 months, 85% of patients showed a complete response to salvage RT and 10% showed a partial response. The 5- and 10-year disease-specific survival rates were 90 and 84%. All recurrences occurred within 5 years and outside the RT field.

Conclusion: Salvage RT achieved high response rates, durable survival, and minimal toxicity, supporting its role as a valuable treatment option in selected patients.

Purpose: This study investigated the correlation between plasma biomarker levels and post-transplant complications of allo-HSCT in children. The biomarkers analyzed were sST2, REG3α, TNFR1, IL-6, and IL-8.

Methods: From January 1, 2019 to October 30, 2024, specimens from 157 patients were collected at aGVHD onset or when aGVHD was clinically presumed to be likely to occur.

Results: Patients with aGVHD and intestinal aGVHD had significantly elevated sST2 levels. Patients with grade III/IV aGVHD had significantly higher sST2 and REG3α levels than patients with grade I/II aGVHD. OS and NRM differed significantly between patients with high sST2 and REG3α indexes (sST2 ≥ 124,090 pg/ml and REG3α ≥ 17,176 pg/ml) and those with low sST2 and REG3α indexes (P < 0.05). Univariate logistic regression analysis indicated that aGVHD, SOS, and TA-TMA were correlated with elevated sST2 and REG3α levels. Multivariate logistic regression analysis indicated that grade III/IV aGVHD was significantly associated with elevated sST2 and REG3α levels (each P = 0.003).

Conclusions: Elevated sST2 and REG3α can reflect the occurrence and severity of aGVHD. Acute GVHD, SOS, and TA-TMA can all lead to fluctuations in sST2 and REG3α levels, and warrant further study.