Blood Cells Molecules and Diseases最新文献

{"title":"The 50th anniversary of Blood Cells, Molecules and Diseases, 1975–2024","authors":"Marshall A. Lichtman","doi":"10.1016/j.bcmd.2024.102854","DOIUrl":"10.1016/j.bcmd.2024.102854","url":null,"abstract":"<div><p>The journal <em>Blood Cells</em> was initiated in 1975 by Marcel Bessis, a French hematologist and cell biologist, as a vehicle for the publication of papers and discussions presented at an international blood club meeting he convened at L' Institut de Pathologie Cellulaire on the campus of Hôpital Bicétre in Kremlin Bicétre, France, a commune on the southern border of Paris. The group met at the Institute for the first time in October 1972. After the first meeting, Bessis published the articles describing the presentations in the <em>Nouvelle Revue d'Hématologie Française</em>, France's principal journal for articles on the science and practice of hematology of which he was the editor. The refusal of the <em>Nouvelle Revue d'Hématologie Française</em> to continue publishing the papers from the meeting of the blood club in English prompted Bessis to start a new journal, <em>Blood Cells</em>, in 1975. <em>Blood Cells</em>, also, began to accept individual submitted papers unrelated to the blood club meeting and, thus, it evolved into a standard journal. A decade later, when Bessis became ill, he asked Brian Bull, a hematopathologist and professor at Loma Linda University School of Medicine in California to assume the position as the second editor-in-chief. He and Bessis had become scientific collaborators and good friends in the preceding years. In 1995, Ernest Beutler, Chair of Molecular and Experimental Medicine at Scripps Research Institute, assumed the editor-in-chief position and transformed the <em>Journal</em> by making three consequential changes. He expanded its title to <em>Blood Cells, Molecules and Diseases</em>, converted its editorial board to past presidents of the American Society of Hematology plus a few additional experimental hematologists of note, a few from abroad, and he converted the <em>Journal</em> to a digital format, hosted on the Scripps Research Institute server. The <em>Journal</em> was the first published solely in a digital format. It, subsequently, was bought by Academic Press, then Harcourt and, then, by Elsevier. The next three editors-in-chief were (i) Marshall A. Lichtman, then Professor of Medicine (Hematology) and of Biochemistry and Biophysics and former Dean of the School of Medicine and Dentistry at the University of Rochester Medical Center, editor from 2000 to 2013, (ii) Mohandas Narla, then Vice President for Research and Director of The Laboratory of Red Cell Physiology at the New York Blood Center, editor from 2014 to 2021 and (iii) Lionel Blanc, Professor of Molecular Medicine and Pediatrics, Center for Autoimmune, Musculoskeletal and Hematopoietic Diseases, Institute of Molecular Medicine, The Feinstein Institutes for Medical Research and the Les Nelkin Professor of Pediatric Oncology Donald and Barbara Zucker School of Medicine at Hofstra-Northwell from 2022 to the present. Although the <em>Journal</em> publishes papers on any aspect of hematology, it has developed a focus on disorders of r","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"108 ","pages":"Article 102854"},"PeriodicalIF":2.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141729243","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}

{"title":"Dysmorphic megakaryocytes in TAFRO syndrome: A case series from a single institute","authors":"Shohei Maida ,&nbsp;Hiromi Nakagawa ,&nbsp;Hiroshi Ureshino ,&nbsp;Kyoko Kajihara ,&nbsp;Shinichi Yamazaki ,&nbsp;Tatsuo Ichinohe","doi":"10.1016/j.bcmd.2024.102870","DOIUrl":"10.1016/j.bcmd.2024.102870","url":null,"abstract":"<div><p>TAFRO syndrome is a rare systemic inflammatory disorder of unknown etiology characterized by thrombocytopenia, anasarca, fever, reticulin myelofibrosis, renal dysfunction, and organomegaly. The diagnosis of TAFRO syndrome can be challenging; however, prompt diagnosis is vital because TAFRO syndrome is a progressive and life-threatening disease. We have showcased five patients with TAFRO syndrome who had similar bone marrow (BM) findings that could be considered the findings that characterize TAFRO syndrome. All patients were treated with corticosteroids and tocilizumab; three of the five patients (60 %) responded positively to the treatment. The unique BM findings observed in this study were megakaryocytes with distinct multinuclei and three-dimensional and indistinct bizarre nuclei (“dysmorphic megakaryocyte”), similar to the megakaryocyte morphology observed in myeloproliferative neoplasms (MPNs). Notably, dysmorphic megakaryocytes were observed in all five cases, whereas only two of the five patients tested positive for reticulin myelofibrosis, and three of the five patients had megakaryocytic hyperplasia, which are considered typical findings of TAFRO syndrome. Thus, the BM findings of dysmorphic megakaryocytes could help in the correct and immediate diagnosis of TAFRO syndrome.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"108 ","pages":"Article 102870"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141404724","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}

{"title":"Surgical procedures and complications in placement of totally implantable venous access port in pediatric hemophilia patients: A retrospective analysis","authors":"Wei Cheng ,&nbsp;Jinrui Zhang ,&nbsp;Xipeng Wang ,&nbsp;Guoqing Liu ,&nbsp;Wanru Yao ,&nbsp;Chunli Wang ,&nbsp;Runhui Wu ,&nbsp;Zhiqiang Li","doi":"10.1016/j.bcmd.2024.102862","DOIUrl":"10.1016/j.bcmd.2024.102862","url":null,"abstract":"<div><p>This retrospective study at Beijing Children's Hospital (2020–2023) analyzed surgical procedures and complications in 24 pediatric hemophilia patients undergoing Totally Implantable Venous Access Port (TIVAP) insertion, primarily in the right jugular vein (RJV). We detailed the surgical process, including patient demographics and intraoperative imaging use. The choice of the RJV for TIVAP placement was influenced by its larger diameter and superficial anatomical position, potentially reducing risks like thrombosis and infection. Our findings support the RJV as a safer alternative for port placement in pediatric patients, aligning with current literature. Statistical analysis revealed no significant correlation between complications and baseline characteristics like weight and diagnosis type. However, the length of hospital stay and implant brand were significant risk factors for catheter or port displacement and removal. The limited patient number may introduce bias, suggesting a need for further studies with larger samples. Despite a 14.7 %–33 % complication rate and 5 port removals, the advantages of TIVAP, including reliable venous access, reduced discomfort, and treatment convenience, were evident. Most complications improved with symptomatic treatment, and there were no deaths due to port-related complications, underscoring the impact of TIVAP on improving pediatric hemophilia treatment.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"108 ","pages":"Article 102862"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1079979624000408/pdfft?md5=2b35836cd4ee944b58be12582d724ec4&pid=1-s2.0-S1079979624000408-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141403393","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Molecular and biochemical evaluation of oxidative effects of cord blood CD34+ MPs on hematopoietic cells","authors":"Zoi Katana ,&nbsp;Kyriaki Sianidou ,&nbsp;Gregory Kaiopoulos ,&nbsp;Fani Deligianni ,&nbsp;Sarantis Tsetsakos ,&nbsp;Anastasia Kouvatsi ,&nbsp;Ioanna Sakellari ,&nbsp;Aristeidis Kritis ,&nbsp;Maria Touraki ,&nbsp;Damianos Sotiropoulos ,&nbsp;Angeliki Xagorari","doi":"10.1016/j.bcmd.2024.102871","DOIUrl":"10.1016/j.bcmd.2024.102871","url":null,"abstract":"<div><p>A graft source for allogeneic hematopoietic stem cell transplantation is umbilical cord blood, which contains umbilical cord blood mononuclear cells (MNCs and mesenchymal stem cells, both an excellent source of extracellular microparticles (MPs). MPs act as cell communication mediators, which are implicated in reactive oxygen species formation or detoxification depending on their origin. Oxidative stress plays a crucial role in both the development of cancer and its treatment by triggering apoptotic mechanisms, in which CD34+ cells are implicated. The aim of this work is to investigate the oxidative stress status and the apoptosis of HL-60 and mononuclear cells isolated from umbilical cord blood (UCB) following a 24- and 48-hour exposure to CD34 + microparticles (CD34 + MPs). The activity of superoxide dismutase, glutathione reductase, and glutathione S-transferase, as well as lipid peroxidation in the cells, were employed as oxidative stress markers. A 24- and 48-hour exposure of leukemic and mononuclear cells to CD34 + -MPs resulted in a statistically significant increase in the antioxidant activity and lipid peroxidation in both cells types. Moreover, CD34 + MPs affect the expression of BCL2 and FAS and related proteins and downregulate the hematopoietic differentiation program in both HL-60 and mononuclear cells. Our results indicate that MPs through activation of antioxidant enzymes in both homozygous and nonhomozygous cells might serve as a means for graft optimization and enhancement.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"108 ","pages":"Article 102871"},"PeriodicalIF":2.1,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141623690","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}

{"title":"Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP)","authors":"A. Guarina ,&nbsp;P. Farruggia ,&nbsp;E. Mariani ,&nbsp;P. Saracco ,&nbsp;A. Barone ,&nbsp;D. Onofrillo ,&nbsp;S. Cesaro ,&nbsp;R. Angarano ,&nbsp;W. Barberi ,&nbsp;S. Bonanomi ,&nbsp;P. Corti ,&nbsp;B. Crescenzi ,&nbsp;G. Dell'Orso ,&nbsp;A. De Matteo ,&nbsp;G. Giagnuolo ,&nbsp;A.P. Iori ,&nbsp;S. Ladogana ,&nbsp;A. Lucarelli ,&nbsp;M. Lupia ,&nbsp;B. Martire ,&nbsp;C. Dufour","doi":"10.1016/j.bcmd.2024.102860","DOIUrl":"10.1016/j.bcmd.2024.102860","url":null,"abstract":"<div><p>Acquired aplastic anemia (AA) is a rare heterogeneous disorder characterized by pancytopenia and hypoplastic bone marrow. The incidence is 2–3 per million population per year in the Western world, but 3 times higher in East Asia. Survival in severe aplastic anemia (SAA) has improved significantly due to advances in hematopoietic stem cell transplantation (HSCT), immunosuppressive therapy, biologic agents, and supportive care. In SAA, HSCT from a matched sibling donor (MSD) is the first-line treatment. If a MSD is not available, options include immunosuppressive therapy (IST), matched unrelated donor, or haploidentical HSCT. The purpose of this guideline is to provide health care professionals with clear guidance on the diagnosis and management of pediatric patients with AA. A preliminary evidence-based document prepared by a group of pediatric hematologists of the Bone Marrow Failure Study Group of the Italian Association of Pediatric Hemato-Oncology (AIEOP) was discussed, modified and approved during a series of consensus conferences that started online during COVID 19 and continued in the following years, according to procedures previously validated by the AIEOP Board of Directors.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"108 ","pages":"Article 102860"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141417573","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}

{"title":"Decreasing circ_0014614 promotes the differentiation of bone marrow flineage cells into megakaryocytes in essential thrombocythemia via activiation of miR-138-5p/caspase3 axis","authors":"Guopan Yu,&nbsp;Xiaofan Chen,&nbsp;Weixiang Lu,&nbsp;Yanlin Li,&nbsp;Yanxiao Chen,&nbsp;Changxin Yin,&nbsp;Zhongxin Zheng,&nbsp;Xiaoshan Huang,&nbsp;Dan Xu","doi":"10.1016/j.bcmd.2024.102855","DOIUrl":"https://doi.org/10.1016/j.bcmd.2024.102855","url":null,"abstract":"<div><h3>Background</h3><p>Circular RNAs (circRNA) are pivotal in hematological diseases. Previous study showed that circ_0014614 (circDAP3) was significantly underexpressed in bone marrow–derived exosomes from essential thrombocythemia (ET) patients, affecting the differentiation of bone marrow lineage cells into megakaryocytes.</p></div><div><h3>Methods</h3><p>Fluorescence in situ hybridization (FISH) was used to display circ_0014614's primary cytoplasmic location in K562 cells. Cytoscape software was used to predict the circRNA-miRNA-mRNA networks, and their expression at the cellular level was detected by Quantitative reverse transcription-polymerase chain reaction (qRT-PCR). qRT-PCR was utilized to detect the expression levels of circ_0014614，miR-138-5p and caspase3 mRNA. Western blot was used to determine the protein levels of GATA-1, RUNX-1, NF-E2, CD41 and caspase3. The proliferation of K562 cells was assessed using the Cell Counting Kit-8 (CCK-8) Assay. Furthermore, the interplay between miR-138-5p and circ_0014614 or caspase3 was elucidated through a Dual-luciferase reporter assay.</p></div><div><h3>Results</h3><p>FISH assay indicated circ_0014614's primary cytoplasmic location in K562 cells. In ET bone marrow and K562 cells, circ_0014614 and caspase3 were down-regulated, whereas miR-138-5p saw a significant surge. Overexpressing circ_0014614 curtailed K562 cells' proliferation and differentiation. Further, circ_0014614 targeted miR-138-5p, with heightened miR-138-5p levels counteracting circ_0014614's inhibition. MiR-138-5p further targeted caspase3, and caspase3 silencing neutralized suppressed miR-138-5p's effects on K562 cell differentiation.</p></div><div><h3>Conclusion</h3><p>Circ_0014614 was down-regulated in ET bone marrow and bone marrow lineage cells, and upregulating circ_0014614 can inhibit bone marrow lineage cells' proliferation and differentiation into megakaryocytes. Mechanistically, circ_0014614 functioned as ceRNA via sponging miR-138-5p and alleviated the inhibitory effect of miR-138-5p on its target caspase3, which potentially deters tumor activity in ET.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"107 ","pages":"Article 102855"},"PeriodicalIF":2.3,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140823572","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}

{"title":"PKLR mutations in pyruvate kinase deficient Polish patients: Functional characteristics of c.101-1G > A and c.1058delAAG variants","authors":"Karolina Maciak ,&nbsp;Aneta Jurkiewicz ,&nbsp;Wojciech Strojny ,&nbsp;Anna Adamowicz-Salach ,&nbsp;Magdalena Romiszewska ,&nbsp;Teresa Jackowska ,&nbsp;Kinga Kwiecinska ,&nbsp;Jaroslaw Poznanski ,&nbsp;Monika Gora ,&nbsp;Beata Burzynska","doi":"10.1016/j.bcmd.2024.102841","DOIUrl":"https://doi.org/10.1016/j.bcmd.2024.102841","url":null,"abstract":"<div><p>Pyruvate kinase (PK) deficiency is a rare autosomal recessive disorder characterized by chronic hemolytic anemia of variable severity. Nine Polish patients with severe hemolytic anemia but normal PK activity were found to carry mutations in the <em>PKLR</em> gene encoding PK, five already known ones and one novel (c.178C &gt; T). We characterized two of the known variants by molecular modeling (c.1058delAAG) and minigene splicing analysis (c.101-1G &gt; A). The former gives a partially destabilized PK tetramer, likely of suboptimal activity, and the c.101-1G &gt; A variant gives alternatively spliced mRNA carrying a premature stop codon, encoding a severely truncated PK and likely undergoing nonsense-mediated decay.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"107 ","pages":"Article 102841"},"PeriodicalIF":2.3,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140347774","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}

{"title":"Differential effects of iron chelators on iron burden and long-term morbidity and mortality outcomes in a large cohort of transfusion-dependent β-thalassemia patients who remained on the same monotherapy over 10 years","authors":"Khaled M. Musallam ,&nbsp;Susanna Barella ,&nbsp;Raffaella Origa ,&nbsp;Giovanni Battista Ferrero ,&nbsp;Roberto Lisi ,&nbsp;Annamaria Pasanisi ,&nbsp;Filomena Longo ,&nbsp;Barbara Gianesin ,&nbsp;Gian Luca Forni ,&nbsp;Webthal® project","doi":"10.1016/j.bcmd.2024.102859","DOIUrl":"10.1016/j.bcmd.2024.102859","url":null,"abstract":"<div><p>We conducted a retrospective cohort study on 663 transfusion-dependent β-thalassemia patients receiving the same iron chelation monotherapy with deferoxamine, deferiprone, or deferasirox for up to 10 years (median age 31.8 years, 49.9 % females). Patients on all three iron chelators had a steady and significant decline in serum ferritin over the 10 years (median deferoxamine: −170.7 ng/mL, <em>P</em> = 0.049, deferiprone: −236.7 ng/mL, <em>P</em> = 0.001; deferasirox: −323.7 ng/mL, <em>P</em> &lt; 0.001) yet had no significant change in liver iron concentration or cardiac T2*; while noting that patients generally had low hepatic and cardiac iron levels at study start. Median absolute, relative, and normalized changes were generally comparable between the three iron chelators. Patients receiving deferasirox had the highest morbidity and mortality-free survival probability among the three chelators, although the difference was only statistically significant when compared with deferoxamine (<em>P</em> = 0.037). On multivariate Cox regression analysis, there was no significant association between iron chelator type and the composite outcome of morbidity or mortality. In a real-world setting, there is comparable long-term iron chelation effectiveness between the three available iron chelators for patients with mild-to-moderate iron overload.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"107 ","pages":"Article 102859"},"PeriodicalIF":2.3,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141183152","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}

{"title":"Aberrant baseline cytokine profile in patients with newly diagnosed acquired aplastic anaemia correlates with disease severity and the treatment response","authors":"Rahul Vatsayan ,&nbsp;Ankur Jain ,&nbsp;Aditya Jandial ,&nbsp;Parveen Bose ,&nbsp;Man Updesh Singh Sachdeva ,&nbsp;Neelam Varma ,&nbsp;Arihant Jain ,&nbsp;Gaurav Prakash ,&nbsp;Alka Khadwal ,&nbsp;Pankaj Malhotra","doi":"10.1016/j.bcmd.2024.102857","DOIUrl":"10.1016/j.bcmd.2024.102857","url":null,"abstract":"<div><h3>Background</h3><p>Immune dysregulation is crucial in the pathogenesis of acquired aplastic anaemia (aAA). There is paucity of data regarding correlation of baseline cytokine profile with treatment response in aAA.</p></div><div><h3>Objective</h3><p>Present prospective case-control study aimed to correlate the baseline cytokines in patients with aAA with the treatment response.</p></div><div><h3>Methods</h3><p>Fifty-one patients with newly-diagnosed aAA &gt; 13 years of either sex were enrolled over 1.5 years. Twenty age-and sex-matched healthy controls (HC) were also included. The cytokine profile (IL-2, 4, 6, 8, 10, 17, IFN-γ and TNF-α) in the peripheral blood plasma of aAA patients was performed at the baseline using cytometric bead analysis. The cytokine levels were compared with HC and correlated with response to immunosuppressive therapy (IST) at 3-months.</p></div><div><h3>Results</h3><p>The median age of cases was 29 years (range,13–74). The cases had higher mean levels of IL2 (<em>p</em> = 0.326), IL4 (<em>p</em> = 0.038), IL6 (<em>p</em> = 0.000), IL10 (<em>p</em> = 0.002), TNF-α (<em>p</em> = 0.302), IFN-γ (<em>p</em> = 0.569) and IL-17 (<em>p</em> = 0.284) than the HC. The baseline levels of all the cytokines were higher (statistically non-significant) among responders (<em>n</em> = 13) than the non-responders (<em>n</em> = 14) to IST.</p></div><div><h3>Conclusions</h3><p>Baseline cytokine profile in patients with aAA might predict response to the IST. Larger studies are needed to validate our results.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"107 ","pages":"Article 102857"},"PeriodicalIF":2.3,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141030757","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}

{"title":"Endurance training and hydroxyurea have synergistic effects on muscle function and energetics in sickle cell disease mice","authors":"Constance P. Michel ,&nbsp;Laurent A. Messonnier ,&nbsp;Benoit Giannesini ,&nbsp;Christophe Vilmen ,&nbsp;Joevin Sourdon ,&nbsp;Yann Le Fur ,&nbsp;David Bendahan","doi":"10.1016/j.bcmd.2024.102853","DOIUrl":"https://doi.org/10.1016/j.bcmd.2024.102853","url":null,"abstract":"<div><p>Sickle cell disease (SCD) is an hemoglobinopathy resulting in the production of an abnormal Hb (HbS) which can polymerize in deoxygenated conditions, leading to the sickling of red blood cells (RBC). These alterations can decrease the oxygen-carrying capacity leading to impaired function and energetics of skeletal muscle. Any strategy which could reverse the corresponding defects could be of interest. In SCD, endurance training is known to improve multiples muscle properties which restores patient's exercise capacity but present reduced effects in anemic patients. Hydroxyurea (HU) can increase fetal hemoglobin production which can reduce anemia in patients. The present study was conducted to determine whether HU can improve the effects of endurance training to improve muscle function and energetics. Twenty SCD Townes mice have been trained for 8 weeks with (<em>n</em> = 11) or without (<em>n</em> = 9) HU. SCD mice muscle function and energetics were analyzed during a standardized rest-exercise-recovery protocol, using Phosphorus-31 Magnetic resonance spectroscopy (³¹P-MRS) and transcutaneous stimulation. The combination of training and HU specifically decreased fatigue index and PCr consumption while muscle oxidative capacity was improved. These results illustrate the potential synergistic effects of endurance training and HU on muscle function and energetics in sickle cell disease.</p></div>","PeriodicalId":8972,"journal":{"name":"Blood Cells Molecules and Diseases","volume":"107 ","pages":"Article 102853"},"PeriodicalIF":2.3,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140347666","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}