Expert Review of Hematology最新文献

Introduction: Multiple myeloma is a hematological malignancy characterized by clonal plasma cell proliferation within the bone marrow, often resulting in osteolytic bone disease. Traditionally, bisphosphonates, such as zoledronic acid and pamidronate, have been used in the prophylaxis and treatment of myeloma bone disease (MBD).

Areas covered: A comprehensive, systematic review using these keywords, 'multiple myeloma,' 'myeloma bone disease,' 'bisphosphonates,' 'denosumab,' 'osteonecrosis' was conducted on Medline and Cochrane databases from 1995 to 2024. Priority was given articles reporting randomized, double-blinded clinical trials; with inclusion of large case cohort studies, in view of the limited comparison between different bone resorptive agents in the treatment of myeloma bone disease.

Expert opinion: This updated guideline recommends a two-year course of bisphosphonate treatment, with suggested extension of dosing intervals if the disease remains stable. Denosumab, a monoclonal antibody targeting receptor activator of nuclear factor ϰB ligand (RANKL), has demonstrated efficacy and non-inferiority compared to zoledronic acid in the treatment of MBD and may serve as an alternative treatment option especially with renal impairment. Further research into the utility of bone turnover markers for guiding anti-resorptive therapy in myeloma bone disease may provide significant clinical benefit. In addition, therapeutic strategies aimed at enhancing osteoblastic activity represent a potential therapeutic strategy.

Introduction: Donor variation is one of the important factor can effect on RBC storage lesion. Consequently, it is possible that the frequency of blood donation can be effective in this regard. Given the few studies that have been conducted, the aim of this study is to compare red blood cell storage damage in two groups of repeat and first-time donors.

Research design and methods: Ten red blood cell concentrates (RCCs) were collected from first-time and ten RCCs from regular blood donors. RCCs were stored for up to 42 days at 2-6°C. Research parameters were performed in both groups during RCCs storage.

Results: Nitric oxide metabolites and Na⁺ concentration were significantly decreased (p = 0.001), while LDH activity and K⁺ concentration were increased (p = 0.001) in the RCCs from regular compared to the first-time donors. In both groups, lipid peroxidation, lactate, and hemolysis increased while total antioxidant, pH and glucose concentration showed a decreasing trend during RCCs storage.

Conclusion: It seems that the RBC storage lesion in RCCs from regular donors is much more than the first-time donors. Therefore, due to the importance of regular blood donors to supply the required blood, further studies need to investigate the causes of this issue.

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.