Hematology Reports最新文献

{"title":"Establishing Reference Intervals for White Blood Cell and Absolute Neutrophil Counts in Duffy Null Individuals.","authors":"Mohammad Barouqa, Muhammad Areeb Ashfaq, Katrina J Jiang, Huseyin Kilic, Nestor Dela Cruz, Aariez Khalid, Marianne E Yassa","doi":"10.3390/hematolrep18030038","DOIUrl":"10.3390/hematolrep18030038","url":null,"abstract":"<p><p><b>Background/Objectives:</b> The Duffy (Fy) blood group system, encoded by the <i>ACKR1</i> gene, plays a key role in transfusion medicine and host susceptibility to malarial infections such as <i>Plasmodium vivax</i>. The Duffy-null phenotype [Fy(a-b-)] is associated with lower baseline white blood cell (WBC) and absolute neutrophil count (ANC) values despite a benign clinical course. This study aimed to establish specific reference intervals for WBC and ANC in Duffy-null individuals to improve diagnostic accuracy and reduce unnecessary clinical interventions. <b>Methods:</b> We conducted a retrospective analysis of 1695 adult patients who underwent complete blood count testing and red blood cell phenotyping between 1 January 2020 and 1 January 2026. Among these, 122 healthy individuals with confirmed Fy(a-b-) phenotype met inclusion criteria. Reference intervals were established using 95% reference interval nonparametric methods in accordance with Clinical Laboratory Standards Institute (CLSI) guidelines. <b>Results:</b> The median WBC and ANC values in the Duffy-null cohort were significantly lower than institutional reference medians (3.24 vs. 6.65 × 10³/µL and 1.45 vs. 4.45 × 10³/µL, respectively; <i>p</i> < 0.01). The derived 95% reference intervals (WBC: 2.43-7.05 × 10³/µL; ANC: 1.01-4.34 × 10³/µL) fell below conventional thresholds, with the lower ANC bound below the standard neutropenia cutoff. <b>Conclusions:</b> These findings support the need for Duffy-null-specific hematologic reference ranges. Adoption of such intervals may reduce misclassification, avoid unnecessary diagnostic procedures, and promote personalized clinical care.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13299395/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148316777","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perioperative Anemia, Transfusion Practices, and Patient Blood Management: Lessons from the COVID-19 Pandemic.","authors":"Alin Ionescu, Alexandra Mihăilescu, Raluca Dumache, Alexandru Capcelea, Alexander Dean Turceanu, Nicolae Albulescu, Mihai Alexandru Săndesc","doi":"10.3390/hematolrep18030037","DOIUrl":"10.3390/hematolrep18030037","url":null,"abstract":"<p><p>The COVID-19 pandemic exposed vulnerabilities in global blood supply systems and accelerated the adoption of patient blood management (PBM) strategies aimed at optimizing transfusion practices in surgical care. Perioperative anemia is a key contributor to adverse outcomes and is frequently treated with allogeneic blood transfusion (ABT), which carries infectious and immunologic risks. Iron deficiency remains the most common and potentially correctable cause of perioperative anemia. This narrative review examines various approaches to perioperative anemia, strategies to minimize reliance on ABT, and alternatives within the PBM paradigm. Evidence supports the use of iron therapy, erythropoiesis-stimulating agents, antifibrinolytic strategies, and blood conservation techniques to reduce transfusion requirements and improve clinical outcomes. Lessons from the COVID-19 pandemic highlight PBM as a framework to enhance transfusion safety and sustainability. Broader implementation of PBM may improve patient outcomes, reduce unnecessary transfusions, and preserve scarce blood resources.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13299662/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148316840","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bortezomib in Patients Who Fail BTKi in Waldenström Macroglobulinaemia.","authors":"Jahanzaib Khwaja, Nicole Japzon, Simona Gatto, Jindriska Lindsay, Charalampia Kyriakou, Shirley D'Sa","doi":"10.3390/hematolrep18030036","DOIUrl":"10.3390/hematolrep18030036","url":null,"abstract":"<p><p><b>Background:</b> Treatment options after Bruton's tyrosine kinase inhibitors (BTKi) failure in Waldenstrom macroglobulinaemia are limited. <b>Methods:</b> We retrospectively analysed the use of bortezomib after BTKi failure in 17 patients who were heavily pre-treated and chemotherapy-exposed at our centre between 2018 and 2025. <b>Results:</b> Reasons for BTKi failure were disease progression (59%) and intolerance (41%). At bortezomib initiation, the median age was 73 years and two patients experienced grade 1-2 neuropathy. The best overall response rate (ORR) was 88%. At a median follow up of 39 months (interquartile range 35-78), median treatment-free survival and overall survival were 18 (95% confidence interval [CI] 13-22) and 22 (95% CI 17-45) months, respectively. <b>Conclusion:</b> Bortezomib may be efficacious in patients who experience BTKi failure.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-05-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13300405/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148316731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Delayed Rewarming Thrombocytopenia (DRT): A Temperature-Dependent Platelet Aggregation Disorder.","authors":"Ian Joseph Cohen","doi":"10.3390/hematolrep18030035","DOIUrl":"10.3390/hematolrep18030035","url":null,"abstract":"<p><p>Below 32 °C, the second irreversible stage of platelet aggregation is absent, causing augmentation of the first reversible stage of platelet aggregation and adhesion. During rewarming, de-aggregation occurs; however, in the presence of adequate ADP (adenosine diphosphate), the second stage of aggregation occurs, leading to delayed rewarming thrombocytopenia (DRT). Erythrocytes leak ADP in sufficient amounts by 24 h to cause DRT. This is prevented by rewarming within 24 h. Heparin before hypothermia prevents platelet adhesion, as does alcohol, which also blocks the second phase of aggregation. Aspirin blocks the second phase of aggregation, and platelet infusions, stored without erythrocytes, are an effective therapy. DRT explains rewarming deaths in NCI (neonatal cold injury).</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13299634/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148316864","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sociodemographic, Clinical, and Therapeutic Characterization of Multiple Myeloma Patients (CharisMMa Study) with Symptomatic Relapse and/or Refractory Disease: An Observational, Multicenter Study in Portugal.","authors":"Rui Bergantim, José Guilherme Freitas, Cristina Gonçalves, Helena Martins, Herlander Marques, Henrique Coelho, Patrícia Seabra, Adriana Roque, Márcio Tavares, Pedro Pinto, Ana Rita Francisco, Joana Tato, Catarina Geraldes","doi":"10.3390/hematolrep18030034","DOIUrl":"10.3390/hematolrep18030034","url":null,"abstract":"<p><strong>Objectives: </strong>Real-world information on relapsed and/or refractory multiple myeloma (rrMM) clinical management in Portugal is scarce. The CharisMMa Portugal study aimed to characterize rrMM patients through socio-demographic and clinical parameters and describe treatment patterns.</p><p><strong>Methods: </strong>This was an observational, cross-sectional, multicenter study with 62 rrMM patients routinely treated at seven hospitals in Portugal. Data were collected from medical records during clinical appointments (2020-2022) after written informed consent was obtained (ClincialTrials.gov ID-NCT04135963). Patients who were diagnosed with a symptomatic MM episode in the 6 months prior to study initiation and who received treatment before their last relapse episode were enrolled.</p><p><strong>Results: </strong>Most patients were male (54.8%) and living with relatives (90.3%), and almost 50% were independent. Roughly 70% of patients were classified as Revised MM International Staging System (R-ISS) Stage II at diagnosis, with a mean age of 65.76 (±9.24) years old. Most common SliM-CRAB (SLIM: sixty percent or more clonal plasma cells in the bone marrow (S), light chain ratio ≥100 (Li), and MRI-detected focal lesions (M); CRAB: hypercalcemia (C), renal insufficiency (R), anemia (A), and bone lesions (B)) signs were bone lesions (59%), and 62.9% of the patients had at least one comorbidity. At study initiation, 70.5% of patients were on second-line treatment, with monoclonal antibodies and proteasome inhibitors (PIs) + immunomodulators (IMiDs) as leading agents. Triplet regimens were the most common across all lines, while oral and oral + subcutaneous were the most prevalent routes of administration.</p><p><strong>Conclusions: </strong>Triple treatment combinations are common in rrMM management, with PIs and IMiDs frequently used, especially in first-line settings. The use of oral formulation is substantial, suggesting a step toward more patient-centric options. This characterization underscores the complexity of rrMM management and should inform stakeholders of strategies to standardize patient care across reference centers in Portugal.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214959/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148029491","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel Approaches to the Management of Myelodysplastic Syndromes: The Roles of Artificial Intelligence and Oxidative Stress Biomarkers.","authors":"Ioannis Tsamesidis, Georgios Drillis, Sotirios Varlamis, Niki Smaragdaki, Philippos Klonizakis, Maria Dimou, Konstantinos Liapis, Georgios Vrahiolias, Eleni Andreadou, Stella Mitka, Maria Chatzidimitriou, Ioannis Kotsianidis, Petros Skepastianos, Anastasios G Kriebardis, Ilias Pessach","doi":"10.3390/hematolrep18030033","DOIUrl":"10.3390/hematolrep18030033","url":null,"abstract":"<p><p><b>Objectives</b>: Myelodysplastic syndromes (MDSs) are a heterogeneous group of clonal hematopoietic disorders characterized by ineffective hematopoiesis, genomic instability, and a high risk of progression to acute myeloid leukemia. Oxidative stress (OS) has emerged as a central factor in MDS pathophysiology, contributing to DNA damage, altered cellular signaling, and disease progression. Recent advances in artificial intelligence (AI) and machine learning (ML) offer a transformative approach for integrating multidimensional datasets including oxidative stress markers, hematologic parameters, and molecular profiles to enhance diagnosis, prognostication, and therapeutic monitoring in MDS. <b>Methods</b>: A comprehensive literature search was conducted in PubMed and Scopus, using the keywords \"OS biomarkers,\" \"AI,\" and \"MDS''. <b>Results</b>: Modified redox biomarkers can be correlated with oxidative imbalance and disease progression. ML models such as neural networks, decision trees, and support vector machines effectively capture complex relationships among redox biomarkers, enhancing risk stratification and prediction of treatment response. AI-driven proteomic analyses further revealed OS-related protein signatures linked to MDS pathophysiology. Overall, AI and ML enable the transformation of multidimensional OS data into clinically actionable tools for personalized management in MDS. <b>Conclusions</b>: Integrating biomarker research with AI-based analytics holds promise for advancing personalized diagnostics, prognostication, and therapeutic strategies in MDS, paving the way toward precision medicine.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214639/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148029488","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Leukemic Non-Nodal Mantle Cell Lymphoma Presenting with Traumatic Splenic Rupture.","authors":"Moinul Haque, Razie Amraei, Krasimira A Rozenova","doi":"10.3390/hematolrep18030032","DOIUrl":"10.3390/hematolrep18030032","url":null,"abstract":"<p><p><b>Background:</b> Leukemic non-nodal variant mantle cell lymphoma (nnMCL) is an uncommon subtype of mantle cell lymphoma that lacks lymphadenopathy and generally follows an indolent clinical course. Adverse genetic alterations such as <i>TP53</i> inactivation and del(13q) may have prognostic significance. Clinical findings such as splenomegaly may serve as a clue to the diagnosis and should prompt further evaluation. <b>Case Presentation:</b> We describe a 91-year-old woman who presented with a one-month history of anemia (hemoglobin 12.3 g/dL), mild thrombocytopenia (platelets 136 × 10⁹/L), isolated splenomegaly and no palpable lymphadenopathy. Despite a normal total white blood cell count, intermittent relative lymphocytosis with atypical lymphocytes (4%) and smudge cells was detected on the complete blood count. Peripheral blood flow cytometry demonstrated a monoclonal kappa-restricted B-cell population negative for CD5 and CD10, comprising approximately 20% of lymphocytes. Conventional karyotyping and fluorescent in situ hybridization (FISH) analysis identified del(13q), del(17p)/<i>TP53</i>, and <i>IGH-CCND1</i> rearrangement in 8-19.5% of peripheral blood leukocytes. A month after the initial assessment, the patient presented following a fall. CT imaging of the abdomen revealed marked splenomegaly, a large subcapsular/perisplenic hematoma, and moderate-to-large hemoperitoneum. Emergent laparotomy showed an enlarged spleen (1490 g, 23 × 16 × 7.5 cm) with laceration. Histologic evaluation showed atypical lymphoid cells positive for CD20 and cyclin D1, with strong p53 expression, negative for CD5 and SOX11, and a low Ki-67 index. Similar involvement was identified in the small bowel and appendix. Targeted sequencing of splenic tissue, performed as part of a retrospective molecular characterization, identified a pathogenic <i>TP53</i> variant <i>(</i>p.His179Gln). <b>Conclusions:</b> This case provides a rare opportunity to evaluate splenic and small intestinal involvement by nnMCL at both the gross and histologic levels. It highlights the importance of integrating clinical findings with flow cytometry, imaging, cytogenetic, and molecular data in establishing the diagnosis. Even when peripheral blood findings suggest a low disease burden, imaging may better define the extent of disease and support appropriate clinical assessment, particularly in elderly patients at risk for complications related to splenomegaly.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-05-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214678/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148029519","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Analysis of Anticoagulation Stability in Critically Ill Patients Receiving Argatroban for Suspected or Confirmed Heparin-Induced Thrombocytopenia Compared with Unfractionated Heparin: A Retrospective Cohort Study.","authors":"Imran Khan, Elizabeth Lamarche, Bernadett Kovacs, Ariel Hendin, Andy Pan, Caitlin Richler, Christine Landry, Sydney Morin, Kaouther Derouiche, Pierre Thabet","doi":"10.3390/hematolrep18030031","DOIUrl":"10.3390/hematolrep18030031","url":null,"abstract":"<p><p><b>Background:</b> Achieving and maintaining therapeutic anticoagulation with unfractionated heparin in critically ill patients is challenging due to biologic variability, heparin resistance, and limitations of activated partial thromboplastin time (aPTT) monitoring. Argatroban, a direct thrombin inhibitor, provides antithrombin-independent anticoagulation with more predictable pharmacokinetics, but real-world data describing its anticoagulation stability in the intensive care unit (ICU) remain limited. <b>Objective:</b> This study aimed to compare anticoagulation stability between continuous intravenous argatroban and unfractionated heparin in critically ill patients using time in therapeutic range (TTR) based on aPTT as the primary performance metric. <b>Methods:</b> A retrospective cohort study was conducted in the ICU and step-down unit of Hôpital Montfort (Ottawa, ON, Canada) between January 2016 and December 2024. Adult patients receiving continuous intravenous argatroban or unfractionated heparin for systemic anticoagulation were included. All aPTT values obtained during active infusion were extracted, and TTR was calculated using linear interpolation between consecutive measurements. Continuous variables were summarized as medians with interquartile ranges and compared using the Wilcoxon rank-sum test; categorical TTR strata were compared using Fisher's exact test. <b>Results:</b> Sixty-eight patients met the inclusion criteria, contributing 9 argatroban and 61 heparin infusion courses. Argatroban demonstrated a higher median TTR than heparin (83.3% [IQR 82.0-90.7] vs. 47.5% [32.9-62.4]; <i>p</i> < 0.001), with a moderate-to-large effect size (r = 0.51). Median aPTT values were similar between groups, but argatroban showed narrower dispersion and fewer prolonged subtherapeutic periods. A majority of heparin courses (56.5%) spent <50% of time within range, whereas no argatroban courses fell into this category. Conversely, 33.3% of argatroban courses achieved ≥90% TTR compared with none in the heparin group. <b>Conclusions:</b> In this real-world ICU cohort where argatroban was used for suspected or confirmed HIT, argatroban was associated with higher TTR than unfractionated heparin. These findings support the use of time-dependent metrics to evaluate anticoagulation quality and warrant prospective studies in more homogeneous populations.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214870/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148029467","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"VEXAS Syndrome: Clinical Features, Hematologic Involvement, and Clinical Outcomes of Current and Emerging Therapies.","authors":"Chanika Assavarittirong, Christopher Grant, Sandeep S Nayak, Anthony L Nguyen","doi":"10.3390/hematolrep18030030","DOIUrl":"10.3390/hematolrep18030030","url":null,"abstract":"<p><p><b>Background/Objectives</b>: VEXAS (Vacuoles, E1-Enzyme, X-linked, Autoinflammatory, and Somatic) syndrome is a recently described adult-onset autoinflammatory disorder. It is characterized by somatic mutations in the UBA1 gene, systemic inflammation, macrocytic anemia, cytopenias, and bone marrow vacuolization and frequently overlaps with Sweet's syndrome, relapsing polychondritis, and myelodysplastic syndrome (MDS). Because treatment options are evolving, we reviewed the current and latest evidence of clinical features and therapeutic methods. <b>Methods</b>: A comprehensive literature review was conducted using PubMed and MEDLINE for studies published between 1 January 2020 and 1 July 2025. Search terms included \"VEXAS\" and \"treatment.\" Eligible publications comprised clinical trials, multicenter and observational studies, and case reports containing therapeutic data. Findings were analyzed narratively with emphasis on treatment response, steroid-sparing effects, survival outcomes, and molecular responses. <b>Results</b>: Glucocorticoids remain the first-line therapy for acute management; however, this comes with near-universal steroid dependence. DMARDs and TNF-α inhibitors showed limited benefits. IL-6 inhibitors and JAK inhibitors showed improvement in overall response, with JAK inhibitors demonstrating a superior effect. Ruxolitinib showed a higher complete response rate and transfusion independence compared to other JAK inhibitors. Hypomethylating agents, particularly azacitidine, improved hematologic responses in patients with co-existing MDS and reduced UBA1 variant allele burden. Allogeneic hematopoietic stem cell transplantation may be the only current curative method, though with notable transplant-related mortality. <b>Conclusions</b>: JAK inhibitors and hypomethylating agents offer promising disease-modifying potential, while transplant may provide curative intent in selected patients. Ongoing clinical trials are taking place to dictate the treatment direction of VEXAS syndrome.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 3","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13214811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148029576","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Prognostic Value of the CD8⁺PD-1⁺/CD4⁺PD-1⁺ (PERLS) Ratio for Leukemic Transformation in MDS.","authors":"Panagiotis Panagiotidis, Emmanuel Karavanis, Konstantinos Neanidis, Eleftherios Panteris, Maria Moysidou","doi":"10.3390/hematolrep18020029","DOIUrl":"10.3390/hematolrep18020029","url":null,"abstract":"<p><p><b>Background/Objectives:</b> Myelodysplastic syndromes (MDS) are associated with a significant risk of progression to acute myeloid leukemia (AML), affecting approximately 30% of patients. In high-risk MDS, leukemic transformation may occur within a short time frame, highlighting the need for early and reliable biomarkers of disease progression. Increasing evidence suggests that immune dysregulation and cytotoxic T-cell dysfunction contribute to disease evolution. This study aimed to evaluate PD-1 and CD57 expressions on CD8⁺ T cells and to investigate the CD8⁺PD-1⁺/CD4⁺PD-1⁺ ratio (PERLS) as a potential immunological marker predictive of leukemic transformation. <b>Methods:</b> Thirty-one patients with MDS were prospectively followed over a 12-month period. At baseline, patients underwent routine clinical and laboratory evaluation, including multiparameter flow cytometric assessment of bone marrow blasts. An extended immunophenotypic analysis of bone marrow samples was performed at study entry to assess PD-1 and CD57 expression on CD8⁺ T cells. Cytogenetic and molecular analyses were conducted when clinical findings suggested disease progression. Patients who developed signs of progression were re-evaluated approximately one month later, during the progression phase, to assess dynamic immunological changes. <b>Results:</b> Of the thirty-one patients included, eighteen progressed to AML, whereas thirteen remained clinically stable. Patients who progressed demonstrated a significant increase in PD-1 and CD57 expression on CD8⁺ T cells compared with stable patients. Moreover, a markedly higher CD8⁺PD-1⁺/CD4⁺PD-1⁺ (PERLS) ratio was observed in patients who subsequently developed AML, particularly during the progression phase. <b>Conclusions:</b> Dynamic immunophenotypic monitoring reveals that increased PD-1 on CD8⁺ T cells and an elevated PERLS ratio are associated with imminent leukemic transformation in MDS. These findings support the incorporation of immune-based biomarkers, particularly the CD8⁺PD-1⁺/CD4⁺PD-1⁺ ratio, into routine risk assessment to enable earlier identification of disease progression and timely therapeutic intervention.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"18 2","pages":""},"PeriodicalIF":1.9,"publicationDate":"2026-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13116684/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147769758","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}