Expert Review of Hematology最新文献

Introduction: Multiple myeloma (MM) progression results from complex interactions between tumor cells, cytokines, and the tumor microenvironment (TME). MM cells constantly produce paraprotein, causing endoplasmic reticulum stress (ERS). Cell survival relies on adaptive mechanisms such as the unfolded protein response (UPR), autophagy, and heat shock proteins (HSPs). This review emphasizes the role of ERS in MM cell survival and explores emerging therapeutic strategies targeting ERS-related pathways, including chemical agents, natural compounds, and inhibitors of autophagy, HSPs, or the proteasome.

Areas covered: ERS in MM cells is a process that must be understood to provide a more complete understanding of this disease. This review analyzed review articles on ERS in normal cells, cancer, and MM, ERS proteins as drug targets in MM, and reports of scientific papers on ERS and MM. The articles were selected from PubMed from 1998 to 2025. and the Global Cancer Observatory website was also consulted.

Expert opinion: The primary mechanisms regulating ERS are overexpressed in MM cells, and their inhibition can lead to apoptosis, making them potential therapeutic targets. ERS and autophagy are associated with changes in the immune cells of the TME, acting as an immune-evasive mechanism that promotes malignant progression.

Background: Anemia is one of the most common hematological disorders causing fatigue, increased vulnerability to infection, low birth weight, and threatening the physical and mental development of children. Hence, the early identification reduces the risk of patients and facilitates early intervention. On the other hand, the blood collection of the adults is simple, and the results will be precise, and directly show the cause of anemia in adults. Noninvasive techniques for anemia diagnosis are specific for young children and pediatric patients improving the cost-effectiveness.

Research design and methods: Consequently, this research focus on anemia detection for children using the proposed Deep Global Pyramid Convolutional Network (DGPCNet) model. Specifically, the proposed approach involves anemia detection through three anatomical regions, such as palm, fingernails, and eye conjunctiva images. In the proposed method, the Grayscale-based Deep Statistical Structural Edge features (GDSSE) provide enriched feature representation by integrating multiple feature perspectives.

Results: The experimental results report that the proposed method using fused features attains 97.66% accuracy, 97.19% sensitivity, 98.14% specificity, 97.90% precision, 0.018 FPR, and 0.028 FNR for 90% of training.

Conclusions: Moreover, applying fused features in the proposed DGPCNet enables hierarchy-based learning thereby significantly boosting the accuracy and reliability of detection.

Introduction: In chronic lymphocytic leukemia (CLL), progression-free survival (PFS) remains a fundamental efficacy endpoint; however, it does not fully capture patient-centric measures such as treatment discontinuation or adherence. Time to next treatment (TTNT) offers a pragmatic alternative, encompassing not only the interval until initiation of subsequent therapy but also reflecting treatment tolerability and compliance.

Areas covered: A comprehensive literature search following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines identified 40 full-text articles reporting TTNT as an outcome measure. Among these, 30 were retrospective real-world studies, while 10 were prospective phase II or phase III clinical trials involving patients with either treatment-naïve or relapsed/refractory CLL. Meta-analytic evaluation of the prospective trials, each with a minimum follow-up of four years, revealed a strong trial-level correlation between PFS and TTNT, with an r² value of 0.7410 (p = 0.0003). Additionally, TTNT demonstrated a statistically significant, though more moderate, correlation with overall survival (OS), yielding an r² value of 0.5160 (p = 0.008).

Expert opinion: The analysis suggests that TTNT enriches the CLL endpoint repertoire by capturing patient-centered outcomes and informing pragmatic clinical decision-making. Nevertheless, regulatory and methodological standards advocate a two-tier validation approach that includes both individual patient-level analyses and trial-level validation. TTNT in CLL should complement, but not substitute for, PFS in evaluating therapeutic benefit and guiding clinical decision-making.

Background: The present study aimed to evaluate cardiac global longitudinal strain (GLS) in patients diagnosed with essential thrombocytosis (ET).

Research design and methods: Patients diagnosed with ET were consecutively screened in the hematology department of our institution. Patients who underwent transthoracic echocardiography within 1 year of diagnosis were included in the study. A control group of healthy individuals matched for demographic characteristics was established. Second echocardiographic evaluation was performed during the follow-up period. The routine echocardiographic and strain parameters of both groups were compared.

Results: A total of 92 participants were included, consisting of 60 patients with ET and 32 healthy volunteers. GLS values were reduced in the ET group compared to the control group (16.5 ± 2.4% vs. 22.5 ± 2.2%; p = 0.024). Among ET patients, those with GLS values ≤ 18% had a higher prevalence of the JAK2 mutation (81.1% vs. 21.7%; p < 0.001). In univariate logistic regression analysis, presence of the JAK2 mutation (OR: 4.6; 95% CI: 1.01-21; p = 0.04) was independently associated with reduced GLS.

Conclusions: ET may reduce GLS, especially in the presence of JAK2 mutation.

Background: Pain is the most prevalent and debilitating symptom of sickle cell disease (SCD). However, there is a paucity of community-based, longitudinal data from low- and middle-income countries. This study is to systematically document phenotypic details of SCD-related pain in a cohort through a prospective, year-long study in underserved regions of five Indian states.

Research design and methods: Individuals with SCD were monitored prospectively for 24 fortnights. Pain-related data, including episode frequency, intensity, anatomical distribution, quality descriptors, and patterns, were collected. Statistical analyses comprised descriptive statistics, tests of significance and the Jonckheere - Terpstra trend test, etc.

Results: Across 6,048 visits to 252 patients, 2,042 pain episodes were reported, with 86.1% of patients experiencing at least one episode. Pain most frequently affected the lower legs and calves, with significantly higher rates among females (p < 0.001). Continuous and rhythmic pain patterns were associated with severe pain (p < 0.001). Sensory descriptors were more prevalent among high-intensity cases, suggesting neuropathic components.

Conclusion: This is the first Indian community-based longitudinal study revealing a significant prevalence of unreported pain and phenotypic variability. It contributes to the development of region-specific pain management frameworks by considering chronicity, gender, and sociocultural expressions of pain.

Objectives: To evaluate the effectiveness of PK-guided prophylaxis in severe hemophilia A.

Methods: 25 patients received PK-guided prophylaxis using a population PK tool (MyPKFiT), 30 received conventional fixed-dose prophylaxis. Outcomes over six months included bleeding frequency, joint health (HJHS), quality of life (SF-36), factor use, infusion frequency, costs, and adverse events.

Results: PK-guided group had fewer bleeds, improved HJHS and SF-36 scores, lower FVIII use, reduced infusions, and lower costs (all p<0.001).

Conclusion: PK-guided prophylaxis improves clinical outcomes and reduces costs, but the retrospective design and small sample size limit generalizability; further studies are needed for confirmation.

Introduction: Numerous risk staging systems exist for newly diagnosed multiple myeloma (NDMM), from older models which rely purely on markers of 'tumor burden' to modern scores which incorporate tumor genetics. While the 2025 IMS-IMWG high-risk criteria, based on specific cytogenetic analyses and the β2-microglobulin titer, represent a major push to harmonize NDMM risk stratification, multiple barriers to successful, comprehensive implementation in standard practice remain. There also exists substantial heterogeneity in how therapeutic trials quantify risk.

Areas covered: We herein summarize the novel 2025 IMS-IMWG criteria. We then provide a brief overview of the current state of NDMM risk stratification in standard practice, as well as the variability in 'high-risk' disease definitions in NDMM trials. Finally, we discuss ways in which such variation in 'high-risk' definition is being addressed.

Expert opinion: Attempts to improve prognostication and potentially demonstrate outcome prediction in NDMM have been confounded by variable performance of risk stratification in standard clinical practice and substantial heterogeneity of 'high-risk' definitions in clinical trials. If implemented comprehensively, the 2025 IMS-IMWG criteria will allow for apples-to-apples comparison across trials via meta-analysis and thus harmonize risk assessment in myeloma, setting the stage for investigation of risk-adapted treatment deescalation and intensification.