Annals of Hematology最新文献

This study aims to evaluate and predict mortality risks among pediatric patients with hemophagocytic lymphohistiocytosis (HLH). We conducted a retrospective analysis of pediatric patients with HLH diagnosed at the Affiliated Hospital of Zunyi Medical University between January 2012 and April 2023. Patients were divided into a death group and a survival group based on their outcomes. Risk factors for mortality were analyzed using a lasso-logistic regression model. This study included 142 pediatric patients with HLH, with a median age of 40.5 (14.75-84) months, of whom 78 (54.93%) were male. The overall mortality rate was 34.51%. Through lasso-logistic regression analysis, five independent prognostic factors were identified: concurrent central nervous system involvement, multiple organ dysfunction syndrome involving three or more organs, platelet count ≤ 42.5 × 10⁹/L, activated partial thromboplastin time ≥ 54.05 s, and the utilization of blood purification in conjunction with the HLH-94/2004 treatment protocol. The predictive value of the lasso-logistic regression model is better than that of the traditional logistic regression model (AUC: 0.906 vs 0.811, P = 0.001). Subsequently, a lasso-logistic regression-based predictive model incorporating these identified risk factors was developed. Our lasso-logistic regression-based prediction model may help to identify high-risk patients with HLH early, thereby enabling the timely initiation of appropriate treatment interventions.

Acute myeloid leukemia (AML) is the most prevalent hematologic malignancy in adults. In 2022, the European LeukemiaNet (ELN) has updated its prognostic system that incorporates cytogenetics and molecular genetics based on data from patients undergoing intensive chemotherapy (IC). Recently, a risk stratification framework has been established for hypomethylating agents (HMA)-based low-intensity treatment (LIT) to fill the gaps in stratification for this treatment modality, but this needs further refinement. Venetoclax (VEN), a BH3 mimetic, targets BCL-2 to modulate apoptosis and metabolism in AML cells. Its combination with HMA or low-dose cytarabine (LDAC) has been shown to enhance the response rates and prolong the survival outcomes of older or unfit patients with AML. In this review, we delved into the prognostic significance of FLT3-ITD and IDH mutations when used in combination with VEN and HMA, as well as in conjunction with their specific inhibitors. We also explored the role of VEN in NPM1-mutated AML and its efficacy in splicing factor mutations AML. Additionally, we examined the response rates and survival outcomes of CBF-AML when treated with a VEN-based regimen. Moving forward, it is imperative that risk stratification for LIT becomes more nuanced to better align with the requirements of personalized diagnosis and treatment strategies.

This study examined the factors associated with spontaneous remission in children with chronic immune thrombocytopenia (ITP). We retrospectively analyzed the medical records of patients diagnosed with ITP from January 1988 to December 2019 at our institute. A total of 104 children with chronic ITP were identified. The median follow-up time from diagnosis of chronic ITP was 3.6 years (IQR 1.2-8.3, range 0.1-31.4). Fifteen (14.4%) patients with severe symptoms received specific platelet-elevating therapies, including splenectomy, rituximab, and thrombopoietin receptor agonists. Seven of them achieved remission. Among the patients with a platelet count < 30 × 10⁹/L at the time of diagnosis of chronic ITP, those who received specific platelet-elevating therapies had a higher remission rate compared to those who did not (HR: 4.66, 95% CI: 1.36-16.0). Sixteen patients (15.4%) developed systemic lupus erythematosus, 46 (44.2%) still had thrombocytopenia after a median follow-up of 6.8 years, and 42 (40.4%) achieved remission with a median time to remission of 2.0 years (IQR 0.6-4.1, range 0.1-15.7). The two independent predictive factors for spontaneous remission in childhood chronic ITP were platelet counts > 30 × 10⁹/L at the time of diagnosis of chronic ITP (HR: 3.16, 95% CI: 1.51-6.62) and persistently negative ANA at follow-up (HR: 6.12, 95% CI: 1.46-25.7). The cumulative probabilities of spontaneous remission at 10 years post-diagnosis of chronic ITP were 72.2% for patients without risk factor compared to 0% for patients with two risk factors.

Pure red cell aplasia is a rare condition that may be congenital or associated with an underlying disease.Immunosuppressants are a commonly employed therapeutic option for the treatment of pure red cell aplasia;however, they are associated with considerable adverse effects, including nephrotoxicity. This case report describesa 74-year-old patient with pure red cell aplasia who developed long-term kidney injury following cyclosporinetherapy. Renal anemia is a common complication after chronic kidney injury and contributes to the poor outcome ofanemia treatment. Following a series of medication adjustments, the final treatment with roxarestat combined withsirolimus proved effective in delaying the impairment of this patient's kidney function, with the hemoglobin levelremaining above 100 g/L throughout. This case report demonstrates the efficacy of roxarestat in conjunction with animmunosuppressive agent in the treatment of pure red cell aplasia combined with kidney injury, with a dual effect ofalleviating the anemia and reducing serum creatinine levels.

Infection risk during high-dose cytarabine (HiDAC) consolidation following induction therapy for acute myeloid leukemia (AML) is not well understood complicating decisions regarding antimicrobial prophylaxis during this period. We performed a retrospective chart review of adult patients with AML undergoing HiDAC consolidation between June 2016 and November 2021 at our institution. The primary endpoint was microbiologically confirmed infection within 30 days of HiDAC administration. This study included 111 patients who received a total of 264 cycles of HiDAC therapy. 36% of patients undergoing HiDAC consolidation had at least 1 infection over the course of their consolidation therapy. Infection complicated 18% of HiDAC cycles. The majority of infections were bacterial (81%), primarily caused by gram-negative organisms. Fluoroquinolone prophylaxis was associated with a lower hazard of bacterial infection (HR 0.46, 95% CI 0.24, 0.88). However, 26% of bacterial infections broke through antibiotic therapy with multiple cases concerning for fluoroquinolone resistance. Viral and fungal infections were rare (14% and 3% of infections respectively).

Understanding the early features and characteristics of hemophagocytic lymphohistiocytosis (HLH) is essential for identifying high-risk individuals and also providing valuable pathological insights. This study aims to investigate the characteristics and trends of blood and hepatic parameters before an HLH diagnosis was established. Longitudinal hematological and hepatic test results from pediatric patients with HLH and an age- and sex-matched control group were analyzed. According to the length of time between hospital admission and the establishment of the HLH diagnosis, the HLH cases were divided into early-onset (≤ 7 days) and late-onset (> 7days) groups. Among the 229 pediatric HLH patients, the length of time between hospital admission and the establishment of an HLH diagnosis ranged from 0 to 41 days (median = 4 days). Over 80% of pediatric HLH patients presented abnormal laboratory results for aspartate aminotransferase (AST), triglycerides, lactate dehydrogenase (LDH), and hemoglobin at admission. The abnormal rates in the initial platelet count, neutrophil count, and fibrinogen tests were 67.3%, 48.3%, and 52.2%, respectively. The initial test results for AST, alanine aminotransferase (ALT), LDH, serum sodium, and albumin showed AUCs > 80% for discriminating early-onset HLH. For the discrimination of late-onset HLH, the performance of initial test results was poor. To conclude, abnormalities in AST, triglycerides, LDH, and hemoglobin are early presentations of pediatric HLH; platelet, neutrophil, and fibrinogen levels may become abnormal at a relatively late stage of the HLH disease trajectory; and the initial test results for AST, ALT, LDH, serum sodium, and albumin can be used to identify suspected early-onset HLH.

The combination of venetoclax with hypomethylating agents is currently the standard of care for elderly patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy. Despite its favorable efficacy, clinical use is often associated with post-remission cytopenia, frequently necessitating treatment delays and dose modifications. This study aims to evaluate the efficacy and safety of shortened venetoclax treatment durations. A multicenter analysis was conducted involving 20 adult AML patients receiving venetoclax (7 or 14 days with 9 and 11 patients, respectively) combined with 5-azacitidine (5-7 days) between 2021 and 2024. The cohort included patients from four German academic centers all treated in first line. Outcome measures included bone marrow response, transfusion dependence, overall survival (OS) and progression-free survival (PFS). Median age was 73.5 years, with 70% of patients having secondary AML. Adverse molecular risk was observed in 75% of patients. The overall response rate (ORR) was 100%, with a composite complete remission rate of 78%. No significant differences in response rates were observed between the 7-day and 14-day venetoclax regimens. Median OS for the cohort was 15 months. Infection-related complications were observed in 55% of patients, with severe sepsis in 20% of cases. In this cohort, shortened venetoclax regimens demonstrated efficacy comparable to standard treatment protocols, with a potential reduction in hematologic toxicity. These findings support the individualization of treatment regimens to optimize clinical outcomes while potentially minimizing adverse effects.

Tucidinostat has been approved by the Chinese FDA for relapsed/refractory Peripheral T cell lymphoma (PTCL), but its efficacy in newly diagnosed PTCL has not been confirmed. In this study, we aimed to compare the efficacy of tucidinostat combined with CHOP-like (C + CHT) versus CHOP-like alone (CHT) in newly diagnosed PTCL patients. Of the PTCL patients, 109 were newly diagnosed. Patients in the C + CHT group who achieved objective response received tucidinostat maintenance therapy. A total of 36 pairs (n = 72) were matched at a ratio of 1:1 using propensity scoring. The matching criteria included: whether the Prognostic index for the peripheral T-cell lymphoma-not otherwise specified subtype (PIT) was ≥ 2, the pathological subtype, age > 60 years, and gender (matching tolerance = 0.024). A significantly higher objective response rate (ORR) (P = 0.016), 2-year progression-free survival (PFS) (P = 0.026), and 2-year survival rate (P = 0.017) was observed for the C + CHT group as compared to the CHT group. After propensity score matching (PSM), the C + CHT group as compared to the CHT group displayed significantly longer PFS (P = 0.035) and overall survival (OS) (P = 0.029). For the C + CHT group in the per-protocol set, the effect values showed a significant benefit in terms of both PFS (P = 0.027) and OS (P = 0.019). Common grade 3-4 haematological adverse events (AEs), had comparable incidence in each group; while common non-haematological AEs, including elevated AST and ALT were higher in the C + CHT group than in the CHT group. Our study suggests that the tucidinostat with CHOP-like regimen and sequential tucidinostat maintenance after objective remission provides a promising therapeutic approach for treating newly diagnosed PTCL patients.