Pub Date : 2025-08-14DOI: 10.1016/j.bcmd.2025.102955
Liana Hatoum , Hannah Song Lee , John N. Oshinski , Edward A. Botchwey , Manu O. Platt
Arterial complications in sickle cell disease (SCD), including stenoses and occlusions, are critical contributors to stroke. Townes SCD mice exhibit neurocognitive deficits and micro-vasculopathy, however stenoses and occlusions that could be causal to ischemic strokes have not yet been confirmed, which has led to challenges whether murine pathology reflects human pathology for strokes due to SCD. In our longitudinal study using label-free magnetic resonance angiography (MRA) to image carotid arteries in Townes SCD mice as they aged from 1 to 7 months, we identified multiple stenoses and occlusions consistent with abnormalities seen in individuals with SCD and stroke complications. We report three cases showing abnormalities: Case 1, middle cerebral artery occlusion associated with seizures and a T1-weighted hyperintense lesion in the brain; Case 2, persistent internal carotid artery occlusion and side-specific artery size differences; and Case 3, persistent stenosis in the common carotid artery with aging. This study highlights the utility of MRA in tracking arterial changes in SCD mice where assessing symptomatic damage due to stroke is challenging. This study provides evidence of mouse-to-mouse variability in initiation and persistence of stenoses and occlusions resembling the variability of SCD complications in humans.
{"title":"A case series of cerebrovascular abnormalities in Townes sickle cell mice visualized with magnetic resonance imaging and angiography","authors":"Liana Hatoum , Hannah Song Lee , John N. Oshinski , Edward A. Botchwey , Manu O. Platt","doi":"10.1016/j.bcmd.2025.102955","DOIUrl":"10.1016/j.bcmd.2025.102955","url":null,"abstract":"<div><div>Arterial complications in sickle cell disease (SCD), including stenoses and occlusions, are critical contributors to stroke. Townes SCD mice exhibit neurocognitive deficits and micro-vasculopathy, however stenoses and occlusions that could be causal to ischemic strokes have not yet been confirmed, which has led to challenges whether murine pathology reflects human pathology for strokes due to SCD. In our longitudinal study using label-free magnetic resonance angiography (MRA) to image carotid arteries in Townes SCD mice as they aged from 1 to 7 months, we identified multiple stenoses and occlusions consistent with abnormalities seen in individuals with SCD and stroke complications. We report three cases showing abnormalities: Case 1, middle cerebral artery occlusion associated with seizures and a T1-weighted hyperintense lesion in the brain; Case 2, persistent internal carotid artery occlusion and side-specific artery size differences; and Case 3, persistent stenosis in the common carotid artery with aging. This study highlights the utility of MRA in tracking arterial changes in SCD mice where assessing symptomatic damage due to stroke is challenging. This study provides evidence of mouse-to-mouse variability in initiation and persistence of stenoses and occlusions resembling the variability of SCD complications in humans.</div></div><div><h3>Headline</h3><div>MRA identifies arterial lesions in mice with SCD.</div></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"115 ","pages":"Article 102955"},"PeriodicalIF":1.7,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144865997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-08-12DOI: 10.1016/j.bcmd.2025.102954
Nidhi Chandrashekar Patil
{"title":"AI for patient education: Insights from DeepSeek's responses on megaloblastic anemia","authors":"Nidhi Chandrashekar Patil","doi":"10.1016/j.bcmd.2025.102954","DOIUrl":"10.1016/j.bcmd.2025.102954","url":null,"abstract":"","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"115 ","pages":"Article 102954"},"PeriodicalIF":1.7,"publicationDate":"2025-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144841503","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-07-04DOI: 10.1016/j.bcmd.2025.102941
Yonatan Diamond , Alexandra Satty , Lenat Joffe , Jonathan D. Fish
Iron overload (IOL) is an increasingly recognized complication among pediatric and young adult survivors of hematopoietic stem cell transplantation (HSCT) contributing to significant morbidity and mortality. The pathogenesis of IOL post-HSCT is multifactorial, driven by transfusion burden, impaired erythropoiesis, and increased gastrointestinal iron absorption, leading to toxic non-transferrin bound iron accumulation and oxidative tissue injury. Despite its clinical significance, consensus guidelines for the diagnosis, monitoring, and management of IOL in this population remain limited. This review examines the physiology and pathophysiology of iron metabolism, the clinical impact of post-HSCT IOL, whilst discussing evidence-based strategies for prevention and treatment. Particular attention is given to the unique challenges of managing iron overload in children, adolescents, and young adults, where individualized treatment and careful surveillance are critical.
{"title":"Burden beyond the cure: Iron overload following pediatric stem cell transplantation","authors":"Yonatan Diamond , Alexandra Satty , Lenat Joffe , Jonathan D. Fish","doi":"10.1016/j.bcmd.2025.102941","DOIUrl":"10.1016/j.bcmd.2025.102941","url":null,"abstract":"<div><div>Iron overload (IOL) is an increasingly recognized complication among pediatric and young adult survivors of hematopoietic stem cell transplantation (HSCT) contributing to significant morbidity and mortality. The pathogenesis of IOL post-HSCT is multifactorial, driven by transfusion burden, impaired erythropoiesis, and increased gastrointestinal iron absorption, leading to toxic non-transferrin bound iron accumulation and oxidative tissue injury. Despite its clinical significance, consensus guidelines for the diagnosis, monitoring, and management of IOL in this population remain limited. This review examines the physiology and pathophysiology of iron metabolism, the clinical impact of post-HSCT IOL, whilst discussing evidence-based strategies for prevention and treatment. Particular attention is given to the unique challenges of managing iron overload in children, adolescents, and young adults, where individualized treatment and careful surveillance are critical.</div></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"115 ","pages":"Article 102941"},"PeriodicalIF":2.1,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144655004","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-06-19DOI: 10.1016/j.bcmd.2025.102940
Renato Cerqueira , Josefina A.P. Braga , Elyse Moritz , João B. Pesquero , José O. Bordin
{"title":"A novel ELANE variant causing severe congenital neutropenia diagnosed in adulthood","authors":"Renato Cerqueira , Josefina A.P. Braga , Elyse Moritz , João B. Pesquero , José O. Bordin","doi":"10.1016/j.bcmd.2025.102940","DOIUrl":"10.1016/j.bcmd.2025.102940","url":null,"abstract":"","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"113 ","pages":"Article 102940"},"PeriodicalIF":2.1,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144366427","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-06-09DOI: 10.1016/j.bcmd.2025.102939
Claire Bordat , Philippe Connes , Philippe Joly , Solene Poutrel , Carine Halfon-Domenech , Anne Perez , Eric Niesor , Elie Nader
Sickle cell anemia (SCA) patients are characterized by poorly deformable and fragile red blood cells (RBCs). Few studies reported an increased cholesterol content in SCA RBC membrane. However, the consequences of this elevated cholesterol level in the above-mentioned RBC alterations are currently unknown. The aim of this study was to assess, in vitro, the effects of BRN-002 (2-Hydroxypropyl-β-cyclodextrin derivate (HPBCD)), a cholesterol-depleting molecule, on RBC membrane cholesterol, hemolysis and RBC sickling and deformability in SCA patients.
Forty patients with SCA and 10 healthy individuals (AA) were included in the different experiments of the study. SCA RBCs were incubated with BRN-002 and the following parameters were assessed: i) RBC and supernatant cholesterol content; ii) RBC deformability by oxygen-gradient ektacytometry; iii) hemolysis by measuring free hemoglobin concentration.
Results confirmed that SCA patients have increased RBC membrane cholesterol compared to AA. BRN-002 effectively removed cholesterol in SCA RBC membrane and reduced free hemoglobin release during incubation. BRN-002 also increased RBC deformability in hypoxia and decreased the pO2 at which sickling occurs.
These findings suggest that excess of RBC membrane cholesterol may participate in the typical RBC alterations found in SCA patients. Therefore, interventions focusing on RBC-cholesterol removal may be beneficial for SCA patients.
{"title":"Impact of elevated red blood cell membrane cholesterol in sickle cell anemia patients: Effects of BRN-002, a 2-hydroxypropyl-β-cyclodextrin derivate, on red blood cell lipids, deformability, sickling and hemolysis","authors":"Claire Bordat , Philippe Connes , Philippe Joly , Solene Poutrel , Carine Halfon-Domenech , Anne Perez , Eric Niesor , Elie Nader","doi":"10.1016/j.bcmd.2025.102939","DOIUrl":"10.1016/j.bcmd.2025.102939","url":null,"abstract":"<div><div>Sickle cell anemia (SCA) patients are characterized by poorly deformable and fragile red blood cells (RBCs). Few studies reported an increased cholesterol content in SCA RBC membrane. However, the consequences of this elevated cholesterol level in the above-mentioned RBC alterations are currently unknown. The aim of this study was to assess, in vitro, the effects of BRN-002 (2-Hydroxypropyl-β-cyclodextrin derivate (HPBCD)), a cholesterol-depleting molecule, on RBC membrane cholesterol, hemolysis and RBC sickling and deformability in SCA patients.</div><div>Forty patients with SCA and 10 healthy individuals (AA) were included in the different experiments of the study. SCA RBCs were incubated with BRN-002 and the following parameters were assessed: i) RBC and supernatant cholesterol content; ii) RBC deformability by oxygen-gradient ektacytometry; iii) hemolysis by measuring free hemoglobin concentration.</div><div>Results confirmed that SCA patients have increased RBC membrane cholesterol compared to AA. BRN-002 effectively removed cholesterol in SCA RBC membrane and reduced free hemoglobin release during incubation. BRN-002 also increased RBC deformability in hypoxia and decreased the pO<sub>2</sub> at which sickling occurs.</div><div>These findings suggest that excess of RBC membrane cholesterol may participate in the typical RBC alterations found in SCA patients. Therefore, interventions focusing on RBC-cholesterol removal may be beneficial for SCA patients.</div></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"113 ","pages":"Article 102939"},"PeriodicalIF":2.1,"publicationDate":"2025-06-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144262638","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-05-27DOI: 10.1016/j.bcmd.2025.102938
Eleni Gavriilaki , Paschalis Evangelidis , Maria Mainou , Theodora Maria Venou , Vasileios Theodoros Vlantos , Efthymia Vlachaki
{"title":"Pegcetacoplan for the treatment of warm autoimmune hemolytic anemia: a case report","authors":"Eleni Gavriilaki , Paschalis Evangelidis , Maria Mainou , Theodora Maria Venou , Vasileios Theodoros Vlantos , Efthymia Vlachaki","doi":"10.1016/j.bcmd.2025.102938","DOIUrl":"10.1016/j.bcmd.2025.102938","url":null,"abstract":"","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"113 ","pages":"Article 102938"},"PeriodicalIF":2.1,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144178119","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-05-23DOI: 10.1016/j.bcmd.2025.102936
Inayat Ur Rahman , Muhammad Tariq Masood Khan , Zahid Ali , Shafiq Ahmad , Muhammad Shahid , Shafaq Zafar , Faheela Faizi Aamir , Imran Khan , Muhammad Ali , Musharraf Jelani , Khalid Khan , Nafees Ahmad , Yasar Yousafzai , Afsar Ali Mian , Sami Siraj
Bacground
Transfusion-dependent β-thalassemia (TDT) requires regular transfusions, often causing iron overload and organ damage. Thalidomide, a fetal hemoglobin (HbF) inducer, may reduce transfusion needs, but scientific data are limited.
Methods
This two-arm, non-randomized clinical trial followed a total of 164 TDT patients over 30 months: 72 received thalidomide and 92 underwent standard transfusions. Complete blood count was assessed at baseline and 6, 12, 18, 24, and 30 months. SNP genotyping and β-globin mutation analysis were performed using sanger sequencing. GATA-1 and KLF gene expression were assessed at baseline and after 30 months via qRT-PCR
Results
Hemoglobin level in the thalidomide group significantly increased from 6.12 ± 0.65 g/dL to 8.36 ± 2.04 g/dL (p < 0.001). Among thalidomide-treated patients, 34.7 % were excellent responders (ER), 25 % good responders (GR), 13.9 % partial responders (PR), and 26.4 % non-responders (NR). ERs showed the highest GATA-1 [3.09 (IQR 2.0–3.38)] and KLF [3.24 (IQR 3.01–5.42)] expression levels (p < 0.001). Better response was observed in patients with AFT >12 months and those carrying the minor allele C at HBS1L-MYB rs9399137 (p < 0.05)
Conclusion
Thalidomide effectively increases hemoglobin levels and reduces transfusion needs in TDT patients, particularly through upregulation of GATA-1 and KLF. AFT and SNP genotype at HBS1L-MYB rs9399137 may help predict response
{"title":"Thalidomide confers therapeutic benefit in beta thalassemia patients by enhancing hemoglobin and hematopoietic gene expression: A non-randomized clinical trial","authors":"Inayat Ur Rahman , Muhammad Tariq Masood Khan , Zahid Ali , Shafiq Ahmad , Muhammad Shahid , Shafaq Zafar , Faheela Faizi Aamir , Imran Khan , Muhammad Ali , Musharraf Jelani , Khalid Khan , Nafees Ahmad , Yasar Yousafzai , Afsar Ali Mian , Sami Siraj","doi":"10.1016/j.bcmd.2025.102936","DOIUrl":"10.1016/j.bcmd.2025.102936","url":null,"abstract":"<div><h3>Bacground</h3><div>Transfusion-dependent β-thalassemia (TDT) requires regular transfusions, often causing iron overload and organ damage. Thalidomide, a fetal hemoglobin (HbF) inducer, may reduce transfusion needs, but scientific data are limited.</div></div><div><h3>Methods</h3><div>This two-arm, non-randomized clinical trial followed a total of 164 TDT patients over 30 months: 72 received thalidomide and 92 underwent standard transfusions. Complete blood count was assessed at baseline and 6, 12, 18, 24, and 30 months. SNP genotyping and β-globin mutation analysis were performed using sanger sequencing. <em>GATA-1</em> and <em>KLF</em> gene expression were assessed at baseline and after 30 months via qRT-PCR</div></div><div><h3>Results</h3><div>Hemoglobin level in the thalidomide group significantly increased from 6.12 ± 0.65 g/dL to 8.36 ± 2.04 g/dL (<em>p</em> < 0.001). Among thalidomide-treated patients, 34.7 % were excellent responders (ER), 25 % good responders (GR), 13.9 % partial responders (PR), and 26.4 % non-responders (NR). ERs showed the highest <em>GATA-1</em> [3.09 (IQR 2.0–3.38)] and <em>KLF</em> [3.24 (IQR 3.01–5.42)] expression levels (p < 0.001). Better response was observed in patients with AFT >12 months and those carrying the minor allele C at <em>HBS1L-MYB</em> rs9399137 (<em>p</em> < 0.05)</div></div><div><h3>Conclusion</h3><div>Thalidomide effectively increases hemoglobin levels and reduces transfusion needs in TDT patients, particularly through upregulation of <em>GATA-1</em> and <em>KLF</em>. AFT and SNP genotype at <em>HBS1L-MYB</em> rs9399137 may help predict response</div></div><div><h3>Trial registration</h3><div><span><span>ClinicalTrials.gov</span><svg><path></path></svg></span> ID: <span><span>NCT06146478</span><svg><path></path></svg></span></div></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"113 ","pages":"Article 102936"},"PeriodicalIF":2.1,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144170590","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-05-23DOI: 10.1016/j.bcmd.2025.102937
Aruna Rangan, Kenneth C. Swanson, Michelle Savedra, Xi Zhang, James D. Hoyer, Jennifer L. Herrick
Introduction
The β-globin gene cluster harbors highly homologous globin genes. Crossover events involving the δ (HBD) and β (HBB) genes result in Lepore (δβ) and anti-Lepore (βδ) hemoglobins (Hbs). Recently, double crossover (βδβ) variants have been reported. Herein, we report βδβ variants identified in our laboratory including the novel Hb Lepore Rochester-MN (LRM).
Methods
Blood samples were obtained with Institutional Review Board approval. Protein characterization included cation exchange high performance liquid chromatography (HPLC), capillary electrophoresis (CE) and mass spectrometry (MS). Molecular analysis included HBB Sanger sequencing and multiplex ligation-dependent probe amplification (MLPA).
Results
Two distinct βδβ crossover variants were identified: Novel Hb LRM (2 infants, 1 adult) and Hb Wanjiang. Hb LRM separated on protein studies (HPLC, CE and MS) and showed identical protein characteristics as Hb Lepore-Hollandia, however, it was expressed at a higher percentage. Sanger sequencing characterized the variant as NM_000518.4(HBB):c.28_68delins41 (p.Ser10_Glu23delins14TAVNALWGKVNVDA). Hb Wanjiang protein did not separate from Hb A using routine methods (HPLC, CE and IEF) but was identifiable by MS and DNA sequencing as NM_000518.4(HBB):c.255_264delinsTTTTTCTCAG (p.A87_T88delinsSQ).
Conclusions
The copy neutral incorporation of δ segments into β gene does not worsen the clinical phenotype. Some substitutions may even have a protective effect when coinherited with Hb S. These uncommon double crossover βδβ variants can pose a diagnostic challenge for laboratories as they can be mistaken for other similar variants on protein evaluation. Also, they may require specialized analysis such as MS, Sanger sequencing or NGS. Interpretation can be challenging if comparison to δ-gene is not considered.
{"title":"Hb Lepore Rochester-MN, a novel βδβ double crossover hemoglobin variant","authors":"Aruna Rangan, Kenneth C. Swanson, Michelle Savedra, Xi Zhang, James D. Hoyer, Jennifer L. Herrick","doi":"10.1016/j.bcmd.2025.102937","DOIUrl":"10.1016/j.bcmd.2025.102937","url":null,"abstract":"<div><h3>Introduction</h3><div>The β-globin gene cluster harbors highly homologous globin genes. Crossover events involving the δ <em>(HBD)</em> and β <em>(HBB)</em> genes result in Lepore (δβ) and anti-Lepore (βδ) hemoglobins (Hbs). Recently, double crossover (βδβ) variants have been reported. Herein, we report βδβ variants identified in our laboratory including the novel Hb Lepore Rochester-MN (LRM).</div></div><div><h3>Methods</h3><div>Blood samples were obtained with Institutional Review Board approval. Protein characterization included cation exchange high performance liquid chromatography (HPLC), capillary electrophoresis (CE) and mass spectrometry (MS). Molecular analysis included <em>HBB</em> Sanger sequencing and multiplex ligation-dependent probe amplification (MLPA).</div></div><div><h3>Results</h3><div>Two distinct βδβ crossover variants were identified: Novel Hb LRM (2 infants, 1 adult) and Hb Wanjiang. Hb LRM separated on protein studies (HPLC, CE and MS) and showed identical protein characteristics as Hb Lepore-Hollandia, however, it was expressed at a higher percentage. Sanger sequencing characterized the variant as NM_000518.4(HBB):c.28_68delins41 (p.Ser10_Glu23delins14TAVNALWGKVNVDA). Hb Wanjiang protein did not separate from Hb A using routine methods (HPLC, CE and IEF) but was identifiable by MS and DNA sequencing as NM_000518.4(HBB):c.255_264delinsTTTTTCTCAG (p.A87_T88delinsSQ).</div></div><div><h3>Conclusions</h3><div>The copy neutral incorporation of δ segments into β gene does not worsen the clinical phenotype. Some substitutions may even have a protective effect when coinherited with Hb S. These uncommon double crossover βδβ variants can pose a diagnostic challenge for laboratories as they can be mistaken for other similar variants on protein evaluation. Also, they may require specialized analysis such as MS, Sanger sequencing or NGS. Interpretation can be challenging if comparison to δ-gene is not considered.</div></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"113 ","pages":"Article 102937"},"PeriodicalIF":2.1,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144131137","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-05-18DOI: 10.1016/j.bcmd.2025.102935
Emmanuel Längst , Michel Prudent
Transfusion is a life-saving practice that requires regular blood donation from healthy volunteers. Red blood cells (RBCs) are isolated from blood donation and stored as RBC concentrates (RCCs) at 4 °C for 42 to 49 days depending on storage solution. RBCs have been intensively studied in this context since World War II and a plethora of data has been obtained from identification and quantification of small molecules to cell function. It has become evident that the RBC properties can be affected by different parameters such as the manufacturing process and donor characteristics. These factors among others may exert a significant influence on transfusion efficacy and clinical outcomes.
After a first part summarizing the impact of the transfusion chain on RBCs and the clinical outcomes (from donors to patients), this review will present different strategies from simple to complex models and from in vitro experiments to clinical trials to fully characterize the properties of RBCs. Furthermore, in silico modeling will be discussed. Beyond pre-analytical conditions, the experimental design might influence the findings. It is therefore essential to expose the RBCs to conditions adapted to the tested hypothesis to understand RBC behavior to optimize the transfusion outcome.
{"title":"Methodological strategies to study and elucidate RBC properties and their potential clinical impact on transfused patients","authors":"Emmanuel Längst , Michel Prudent","doi":"10.1016/j.bcmd.2025.102935","DOIUrl":"10.1016/j.bcmd.2025.102935","url":null,"abstract":"<div><div>Transfusion is a life-saving practice that requires regular blood donation from healthy volunteers. Red blood cells (RBCs) are isolated from blood donation and stored as RBC concentrates (RCCs) at 4 °C for 42 to 49 days depending on storage solution. RBCs have been intensively studied in this context since World War II and a plethora of data has been obtained from identification and quantification of small molecules to cell function. It has become evident that the RBC properties can be affected by different parameters such as the manufacturing process and donor characteristics. These factors among others may exert a significant influence on transfusion efficacy and clinical outcomes.</div><div>After a first part summarizing the impact of the transfusion chain on RBCs and the clinical outcomes (from donors to patients), this review will present different strategies from simple to complex models and from <em>in vitro</em> experiments to clinical trials to fully characterize the properties of RBCs. Furthermore, <em>in silico</em> modeling will be discussed. Beyond pre-analytical conditions, the experimental design might influence the findings. It is therefore essential to expose the RBCs to conditions adapted to the tested hypothesis to understand RBC behavior to optimize the transfusion outcome.</div></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"113 ","pages":"Article 102935"},"PeriodicalIF":2.1,"publicationDate":"2025-05-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144170588","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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