International Journal of Hematology最新文献

This study investigated late renal, liver, endocrine, and cardiac outcomes in 52 Kurdish children aged 7-18 years who underwent bone marrow transplantation (BMT) for β-thalassemia major (β-TM). Boys had higher levels of hemoglobin and alkaline phosphatase than girls. Conversely, girls exhibited higher levels of SGPT, ferritin, T4, and eGFR. In all patients, BUN (71.15%), ALP (100%), PT (96.15%), TSB (34.62%), and serum ferritin (48.08%) were elevated, while Hb (57.69%) and serum creatinine (42.31%) were lower than normal. At ages 1-5, 6-10, and 11-15 years, patients had significantly lower serum ferritin (1053.0, 212.0, and 105.05; p = 0.0004) and SGOT (30.0, 22.0, and 26.50; p = 0.0231) levels. Echocardiography showed normal heart function in 47 patients (90.39%), with minor abnormalities observed in only 9.61%. The mean eGFR was 89.70 (SD: 22.93), with girls showing a significantly higher average (98.83) than boys (72.70; p < 0.0001). Kidney function was most often mildly decreased (55.77%), followed by normal/increased (40.39%), and mild-to-moderately decreased (3.85%). Girls were more likely to have normal/ increased kidney function (61.54%), while boys predominantly had mildly decreased kidney function (76.92%) with no significant difference between ages in all patients. Most of the children with β-TM had mildly decreased kidney function and higher liver function test values.

This study explored the relationship between body temperature (BT) and heart rate (HR) data during acute myeloid leukemia (AML) treatment, using relative bradycardia (RB) as an index. RB trends were also assessed in confirmed infections and during intensive chemotherapy. Data from AML patients who received induction therapy (anthracycline and cytarabine) at Jichi Medical University Hospital between May 2015 and December 2023 were analyzed. A total of 1000 febrile events (axillary temperature ≥ 37.8 °C) in 97 patients were included. Multivariate analysis showed an HR increase of 6.57 bpm (95% CI 4.44-8.70) per 1 °C rise in BT. RB, defined as BT ≥ 37.8 °C with HR < 90 bpm, was observed in 630 events (63.0%). RB was more likely with age ≥ 44 years, hemoglobin ≥ 6.5 g/dL, and reduced-intensity regimens. In contrast, BT ≥ 38.6 °C, diastolic BP ≥ 70 mmHg, oxygen therapy, CRP ≥ 7.13 mg/dL, and incomplete hematologic recovery decreased the likelihood of RB. In addition, RB was significantly associated with documented infection (OR = 2.32, 95% CI 1.17-4.61, p = 0.016). RB frequently occurred during AML induction therapy, and may serve as an indicator of infection.

Synchronous central nervous system (CNS) and systemic diffuse large B-cell lymphoma (synDLBCL) is a rare, aggressive entity with poor prognosis and limited data guiding optimal management. We retrospectively analyzed 25 patients with synDLBCL treated at our institution between 2012 and 2021. Among the 20 patients undergoing curative-intent treatment, most received less-intensive CNS-directed therapies, including R-CHOP with intrathecal chemotherapy or high-dose methotrexate. The 3-year progression-free survival rate was 33%, with CNS progression as the predominant site of treatment failure. Genetic profiling in 12 patients revealed a high prevalence (83%) of the MCD subtype, characterized by frequent MYD88 and CD79B mutations, irrespective of immunohistochemical cell-of-origin classification. These findings align with the genetic landscape of primary CNS lymphoma, underscoring the limitations of less-intensive CNS-directed therapies in achieving durable CNS control in synDLBCL. Our findings highlight the need for CNS-penetrant, intensified frontline strategies and support the investigation of Bruton's tyrosine kinase inhibitor-containing regimens in this population. Multicenter collaborative studies with standardized diagnostic and treatment approaches are essential to improve outcomes in synDLBCL.

Transient abnormal myelopoiesis (TAM) occurs in approximately 10% of neonates with Down syndrome (DS), and about 20% of these cases progress to myeloid leukemia associated with DS (ML-DS) within the first four years of life. TAM usually arises from a single clone harboring an identical GATA1 mutation, but approximately 8% of cases carry heterogeneous clones with distinct GATA1 mutations. To date, no reliable method has been established to determine which clone progresses to ML-DS. Here, we report a unique TAM case with multiple distinct GATA1 mutations in which a minor clone at the time of diagnosis evolved into overt ML-DS by 59 days of age. It is particularly noteworthy that changes in blast surface marker expression detected by flow cytometry (FCM) following a single course of low-dose cytarabine coincided with the emergence of the leukemogenic clone. This case illustrates that the use of FCM may serve as a valuable tool for the early detection of leukemogenic clonal expansion in TAM patients harboring multiple GATA1 mutations.

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.

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.