American Journal of Hematology最新文献

{"title":"Successful 13-year ongoing remission with C5 inhibitor therapy following renal transplant in atypical hemolytic uremic syndrome.","authors":"Mariam A Mostafa, Martin Zand, Jeremy Taylor, Peter Kouides","doi":"10.1002/ajh.27461","DOIUrl":"https://doi.org/10.1002/ajh.27461","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142035023","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Presentation of chronic myeloid leukemia in basophilic blast crisis.","authors":"Biswadip Hazarika, Barbara J Bain","doi":"10.1002/ajh.27464","DOIUrl":"https://doi.org/10.1002/ajh.27464","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142035022","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative clinical efficacy and safety of biosimilar ABP 959 and eculizumab reference product in patients with paroxysmal nocturnal hemoglobinuria.","authors":"Austin Kulasekararaj, Francesco Lanza, Alexandros Arvanitakis, Saskia Langemeijer, Satheesh Chonat, Anil Tombak, Vladimir Hanes, Jia Cao, Mieke Jill Miller, Alexander Colbert, Benjamin Shander, Daniel T Mytych, Vincent Chow, Haby Henary","doi":"10.1002/ajh.27456","DOIUrl":"https://doi.org/10.1002/ajh.27456","url":null,"abstract":"<p><p>ABP 959 is a biosimilar to the eculizumab reference product (RP), which is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). This multicenter, randomized, double-blind, active-controlled, two-period crossover study randomized eculizumab RP-treated patients with PNH to one of two treatment sequences (ABP 959/eculizumab RP or eculizumab RP/ABP 959) to evaluate the clinical similarity of ABP 959 when compared with eculizumab RP. This study evaluated the efficacy of ABP 959 when compared with eculizumab RP based on control of intravascular hemolysis as measured by lactate dehydrogenase (LDH) and by the time-adjusted area under the effect curve of LDH. Secondary outcomes included safety, pharmacokinetics, and immunogenicity. Forty-two patients were randomized (20 in the ABP 959/eculizumab RP group and 22 in the eculizumab RP/ABP 959 group) across 25 centers. Similarity of efficacy was established by a ratio of geometric least squares means of LDH (ABP 959/eculizumab RP) of 1.0628, with a one-sided 97.5% upper CI of 1.1576 at week 27, and a geometric means ratio of time-adjusted area under the effect curve (ABP 959 vs. eculizumab RP) of LDH of 0.981, with a 90% CI of 0.9403-1.0239 from week 13 to 27, week 39 to 53, and week 65 to 79. All secondary efficacy endpoints were comparable between treatment groups. No new safety concerns were identified. The results of this study in patients with PNH, along with previously demonstrated similarity of analytical, nonclinical, and clinical pharmacokinetics and pharmacodynamics in healthy volunteers support a demonstration of no clinically meaningful differences between ABP 959 and eculizumab RP. Clinical Trial Registration: NCT03818607.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016107","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global characteristics and outcomes of autologous hematopoietic stem cell transplantation for newly diagnosed multiple myeloma: A study of the worldwide network for blood and marrow transplantation (WBMT).","authors":"Laurent Garderet, Luuk Gras, Linda Koster, Laurien Baaij, Nada Hamad, Anita Dsouza, Noel Estrada-Merly, Parameswaran Hari, Wael Saber, Andrew J Cowan, Minako Iida, Shinichiro Okamoto, Hiroyuki Takamatsu, Shohei Mizuno, Koji Kawamura, Yoshihisa Kodera, Bor-Sheng Ko, Christopher Liam, Kim Wah Ho, A Sim Goh, S Keat Tan, Alaa M Elhaddad, Ali Bazarbachi, Qamar Un Nisa Chaudhry, Rozan Alfar, Mohamed-Amine Bekadja, Malek Benakli, Cristobal Augusto Frutos Ortiz, Eloisa Riva, Sebastian Galeano, Francisca Bass, Hira S Mian, Arleigh McCurdy, Feng Rong Wang, Ly Meng, Daniel Neumann, Mickey Koh, John A Snowden, Stefan Schönland, Donal P McLornan, Patrick John Hayden, Anna Sureda, Hildegard T Greinix, Mahmoud Aljurf, Yoshiko Atsuta, Dietger Niederwieser","doi":"10.1002/ajh.27451","DOIUrl":"https://doi.org/10.1002/ajh.27451","url":null,"abstract":"<p><p>Autologous hematopoietic cell transplantation (AHCT) is a commonly used treatment in multiple myeloma (MM). However, real-world global demographic and outcome data are scarce. We collected data on baseline characteristics and outcomes from 61 725 patients with newly diagnosed MM who underwent upfront AHCT between 2013 and 2017 from nine national/international registries. The primary endpoint was overall survival (OS), and the secondary endpoints were progression-free survival (PFS), relapse incidence (RI) and non-relapse mortality (NRM). Median OS amounted to 90.2 months (95% CI 88.2-93.6) and median PFS 36.5 months (95% CI 36.1-37.0). At 24 months, cumulative RI was 33% (95% CI 32.5%-33.4%) and NRM was 2.5% (95% CI 2.3%-2.6%). In the multivariate analysis, superior outcomes were associated with younger age, IgG subtype, complete hematological response at auto-HCT, Karnofsky score of 100%, international staging scoring (ISS) stage 1, HCT-comorbidity index (CI) 0, standard cytogenetic risk, auto-HCT in recent years, and use of lenalidomide maintenance. There were differences in the baseline characteristics and outcomes between registries. While the NRM was 1%-3% at 12 months worldwide, the OS at 36 months was 69%-84%, RI at 12 months was 12%-24% and PFS at 36 months was 43%-63%. The variability in these outcomes is attributable to differences in patient and disease characteristics as well as the use of maintenance and macroeconomic factors. In conclusion, worldwide data indicate that AHCT in MM is a safe and effective therapy with an NRM of 1%-3% with considerable regional differences in OS, PFS, RI, and patient characteristics. Maintenance treatment post-AHCT had a beneficial effect on OS.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141999299","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Intra‐tumoral and peripheral blood TIGIT and PD‐1 as immune biomarkers in nodular lymphocyte predominant Hodgkin lymphoma","authors":"Jay Gunawardana, Soi C. Law, Muhammed B. Sabdia, Éanna Fennell, Aoife Hennessy, Ciara I. Leahy, Paul G. Murray, Karolina Bednarska, Sandra Brosda, Judith Trotman, Leanne Berkahn, Andreea Zaharia, Simone Birch, Melinda Burgess, Dipti Talaulikar, Justina N. Lee, Emily Jude, Eliza A. Hawkes, Sanjiv Jain, Karthik Nath, Cameron Snell, Fiona Swain, Joshua W. D. Tobin, Colm Keane, Mohamed Shanavas, Emily Blyth, Christian Steidl, Kerry Savage, Pedro Farinha, Merrill Boyle, Barbara Meissner, Michael R. Green, Francisco Vega, Maher K. Gandhi","doi":"10.1002/ajh.27459","DOIUrl":"https://doi.org/10.1002/ajh.27459","url":null,"abstract":"In classical Hodgkin lymphoma (cHL), responsiveness to immune‐checkpoint blockade (ICB) is associated with specific tumor microenvironment (TME) and peripheral blood features. The role of ICB in nodular lymphocyte predominant Hodgkin lymphoma (NLPHL) is not established. To gain insights into its potential in NLPHL, we compared TME and peripheral blood signatures between HLs using an integrative multiomic analysis. A discovery/validation approach in 121 NLPHL and 114 cHL patients highlighted &gt;2‐fold enrichment in programmed cell death‐1 (PD‐1) and T‐cell Ig and ITIM domain (TIGIT) gene expression for NLPHL versus cHL. Multiplex imaging showed marked increase in intra‐tumoral protein expression of PD‐1+ (and/or TIGIT+) CD4+ T‐cells and PD‐1+CD8+ T‐cells in NLPHL compared to cHL. This included T‐cells that rosetted with lymphocyte predominant (LP) and Hodgkin Reed–Sternberg (HRS) cells. In NLPHL, intra‐tumoral PD‐1+CD4+ T‐cells frequently expressed TCF‐1, a marker of heightened T‐cell response to ICB. The peripheral blood signatures between HLs were also distinct, with higher levels of PD‐1+TIGIT+ in TH1, TH2, and regulatory CD4+ T‐cells in NLPHL versus cHL. Circulating PD‐1+CD4+ had high levels of TCF‐1. Notably, in both lymphomas, highly expanded populations of clonal TIGIT+PD‐1+CD4+ and TIGIT+PD‐1+CD8+ T‐cells in the blood were also present in the TME, indicating that immune‐checkpoint expressing T‐cells circulated between intra‐tumoral and blood compartments. In <jats:italic>in vitro</jats:italic> assays, ICB was capable of reducing rosette formation around LP and HRS cells, suggesting that disruption of rosetting may be a mechanism of action of ICB in HL. Overall, results indicate that further evaluation of ICB is warranted in NLPHL.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":12.8,"publicationDate":"2024-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141994356","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Secondary acquisition of the Philadelphia chromosome in acute lymphoblastic leukemia","authors":"Qiudan Shen, Elias J. Jabbour, Guilin Tang, Hong Fang, Wei Wang, Nicholas J. Short, M. James You, L. Jeffrey Medeiros, Shimin Hu","doi":"10.1002/ajh.27462","DOIUrl":"https://doi.org/10.1002/ajh.27462","url":null,"abstract":"<p>The Philadelphia chromosome (Ph), resulting from the t(9;22)(q34;q11), is the defining cytogenetic abnormality in chronic myeloid leukemia (CML).<span>¹</span> This chromosomal aberration harbors the <i>BCR::ABL1</i> fusion gene, which encodes a constitutively active tyrosine kinase that initiates downstream signaling pathways crucial for leukemogenesis. Beyond CML, the Ph is also observed in other hematologic malignancies, including acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), and mixed-phenotype acute leukemia.</p>\u0000<p>Ph-positive B-ALL (Ph+ B-ALL) is the most common subtype of B-ALL in adults, accounting for approximately 25% of cases. While the Ph is typically detected at the initial diagnosis of CML and Ph+ acute leukemia, its secondary acquisition during treatment of Ph-negative leukemia has been reported, predominantly in refractory AML and myelodysplastic syndrome (MDS) in progression. The secondary acquisition of the Ph during treatment of patients with Ph-negative ALL is uncommon, and its emergence as a subclone at the time of initial diagnosis of ALL is exceedingly rare.<span>²</span> The clinical characteristics, management, and outcomes in these scenarios remain unclear. Here we report eight such cases identified through a retrospective review of 732 cases of ALL (B-ALL 727, T-ALL 5) diagnosed between 2000 and 2023. In these cases, the Ph was detected at initial diagnosis or at relapse using fluorescence in situ hybridization (FISH), with or without karyotyping.</p>\u0000<p>The study group consisted of three men and five women, with a median age of 38 years (range, 14–66) at the time of emergence of the Ph (Table 1). Seven patients were initially diagnosed with B-ALL, and one with T-ALL (#6). Six patients (#1–6) acquired the Ph secondarily during treatment of ALL at a median of 37.5 months (range, 19–192) from initial diagnosis of ALL. Two patients (#7 and 8) presented with the Ph as a subclone at the time of initial diagnosis of ALL.</p>\u0000<div>\u0000<header><span>TABLE 1. </span>Clinical characteristics of patients with the Ph chromosome as a secondary event.</header>\u0000<div tabindex=\"0\">\u0000<table>\u0000<thead>\u0000<tr>\u0000<th>No.</th>\u0000<th>Age (years)</th>\u0000<th>Sex</th>\u0000<th>Initial diagnosis</th>\u0000<th>Disease status at emergence of Ph</th>\u0000<th>Interval to Ph emergence (months)</th>\u0000<th>Treatment and response after Ph</th>\u0000<th>OS from Ph (months)</th>\u0000<th>Patient and disease status at last FU</th>\u0000</tr>\u0000</thead>\u0000<tbody>\u0000<tr>\u0000<td>1</td>\u0000<td>49</td>\u0000<td>M</td>\u0000<td>B-ALL</td>\u0000<td>2nd relapse</td>\u0000<td>192</td>\u0000<td>Ponatinib + blinatumomab + mini-hyper-CVD, ponatinib + blinatumomab + POMP maintenance, CR</td>\u0000<td>30.7</td>\u0000<td>Alive; B-ALL in CR</td>\u0000</tr>\u0000<tr>\u0000<td>2</td>\u0000<td>66</td>\u0000<td>M</td>\u0000<td>B-ALL</td>\u0000<td>1st relapse</td>\u0000<td>25</td>\u0000<td>Bosutinib + inotuzumab ozogamicin, CR; HSCT + bosutinib maintenance; 2nd relapse: ponatinib + chemo, CR; 3rd relapse: dasatinib + blinatumomab, CR</td>\u0000<td>65.3</td>\u0000<td>Died from ","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":12.8,"publicationDate":"2024-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141994668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Serum iron and transferrin saturation variation are circadian regulated and linked to the harmonic circadian oscillations of erythropoiesis and hepatic Tfrc expression in mice","authors":"Cavan Bennett, Anne Pettikiriarachchi, Alistair R. D. McLean, Rebecca Harding, Marnie E. Blewitt, Cyril Seillet, Sant‐Rayn Pasricha","doi":"10.1002/ajh.27447","DOIUrl":"https://doi.org/10.1002/ajh.27447","url":null,"abstract":"Serum iron has long been thought to exhibit diurnal variation and is subsequently considered an unreliable biomarker of systemic iron status. Circadian regulation (endogenous ~24‐h periodic oscillation of a biologic function) governs many critical physiologic processes. It is unknown whether serum iron levels are regulated by circadian machinery; likewise, the circadian nature of key players of iron homeostasis is unstudied. Here we show that serum iron, transferrin saturation (TSAT), hepatic transferrin receptor (TFR1) gene (<jats:italic>Tfrc</jats:italic>) expression, and erythropoietic activity exhibit circadian rhythms. Daily oscillations of serum iron, TSAT, hepatic <jats:italic>Tfrc</jats:italic> expression, and erythropoietic activity are maintained in mice housed in constant darkness, where oscillation reflects an endogenous circadian period. Oscillations of serum iron, TSAT, hepatic <jats:italic>Tfrc,</jats:italic> and erythropoietic activity were ablated when circadian machinery was disrupted in <jats:italic>Bmal1</jats:italic> knockout mice. Interestingly, we find that circadian oscillations of erythropoietic activity and hepatic <jats:italic>Tfrc</jats:italic> expression are maintained in opposing phase, likely allowing for optimized usage and storage of serum iron whilst maintaining adequate serum levels and TSAT. This study provides the first confirmatory evidence that serum iron is circadian regulated, discerns circadian rhythms of TSAT, a widely used clinical marker of iron status, and uncovers liver‐specific circadian regulation of TFR1, a major player in cellular iron uptake.","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":12.8,"publicationDate":"2024-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141994355","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Imatinib versus newer generation TKIs for upfront therapy in chronic phase chronic myeloid leukemia: What is the rationale for paying more to get the same survival benefit?","authors":"Jeffrey H Lipton","doi":"10.1002/ajh.27455","DOIUrl":"https://doi.org/10.1002/ajh.27455","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141974876","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The seasonal distribution of immune thrombotic thrombocytopenic purpura is influenced by geography: Epidemiologic findings from a multi-center analysis of 719 disease episodes.","authors":"Jeremy W Jacobs, Caroline G Stanek, Garrett S Booth, Argiris Symeonidis, Andrew W Shih, Elizabeth S Allen, Eleni Gavriilaki, Brenda J Grossman, Katerina Pavenski, Amy Moorehead, Flora Peyvandi, Pasquale Agosti, Ilaria Mancini, Laura D Stephens, Jay S Raval, Maria Eva Mingot-Castellano, Elizabeth P Crowe, Laetitia Daou, Menaka Pai, Donald M Arnold, Marisa B Marques, Ryan Henrie, Tyler W Smith, Gayatri Sreenivasan, Rance C Siniard, Lisa R Wallace, Chisa Yamada, Miriam Andrea Duque, Yanyun Wu, Thomas J Harrington, Diana M Byrnes, Aikaterini Bitsani, Amanda K Davis, Danielle H Robinson, Quentin Eichbaum, Cristina A Figueroa Villalba, Justin E Juskewitch, Georgia Kaiafa, Eleni Kapsali, Ellen Klapper, Ingrid Perez-Alvarez, Monica S Klein, Nikolaos Kotsiou, Chrysavgi Lalayanni, Evdokia Mandala, Fatima Aldarweesh, Rahaf Alkhateb, Lisandro Fortuny, Zois Mellios, Apostolia Papalexandri, Meredith G Parsons, Annette J Schlueter, Christopher A Tormey, Cameron Wellard, Erica M Wood, Shiyang Jia, Allison P Wheeler, Amy A Powers, Christopher B Webb, Sean G Yates, Raïda Bouzid, Paul Coppo, Evan M Bloch, Brian D Adkins","doi":"10.1002/ajh.27458","DOIUrl":"https://doi.org/10.1002/ajh.27458","url":null,"abstract":"<p><p>Prior studies have suggested that immune thrombotic thrombocytopenic purpura (iTTP) may display seasonal variation; however, methodologic limitations and sample sizes have diminished the ability to perform a rigorous assessment. This 5-year retrospective study assessed the epidemiology of iTTP and determined whether it displays a seasonal pattern. Patients with both initial and relapsed iTTP (defined as a disintegrin and metalloprotease with thrombospondin type motifs 13 activity <10%) from 24 tertiary centers in Australia, Canada, France, Greece, Italy, Spain, and the US were included. Seasons were defined as: Northern Hemisphere-winter (December-February); spring (March-May); summer (June-August); autumn (September-November) and Southern Hemisphere-winter (June-August); spring (September-November); summer (December-February); autumn (March-May). Additional outcomes included the mean temperature in months with and without an iTTP episode at each site. A total of 583 patients experienced 719 iTTP episodes. The observed proportion of iTTP episodes during the winter was significantly greater than expected if equally distributed across seasons (28.5%, 205/719, 25.3%-31.9%; p = .03). Distance from the equator and mean temperature deviation both positively correlated with the proportion of iTTP episodes during winter. Acute iTTP episodes were associated with the winter season and colder temperatures, with a second peak during summer. Occurrence during winter was most pronounced at sites further from the equator and/or with greater annual temperature deviations. Understanding the etiologies underlying seasonal patterns of disease may assist in discovery and development of future preventative therapies and inform models for resource utilization.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141970440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exercise and training in sickle cell disease: Safety, potential benefits, and recommendations","authors":"Philippe Connes,&nbsp;Emeric Stauffer,&nbsp;Robert I. Liem,&nbsp;Elie Nader","doi":"10.1002/ajh.27454","DOIUrl":"10.1002/ajh.27454","url":null,"abstract":"<p>Sickle cell disease (SCD) is a genetic disorder characterized by complex pathophysiological mechanisms leading to vaso-occlusive crisis, chronic pain, chronic hemolytic anemia, and vascular complications, which require considerations for exercise and physical activity. This review aims to elucidate the safety, potential benefits, and recommendations regarding exercise and training in individuals with SCD. SCD patients are characterized by decreased exercise capacity and tolerance. Acute intense exercise may be accompanied by biological changes (acidosis, increased oxidative stress, and dehydration) that could increase the risk of red blood cell sickling and acute clinical complications. However, recent findings suggest that controlled exercise training is safe and well tolerated by SCD patients and could confer benefits in disease management. Regular endurance exercises of submaximal intensity or exercise interventions incorporating resistance training have been shown to improve cardiorespiratory and muscle function in SCD, which may improve quality of life. Recommendations for exercise prescription in SCD should be based on accurate clinical and functional evaluations, taking into account disease phenotype and cardiorespiratory status at rest and in response to exercise. Exercise programs should include gradual progression, incorporating adequate warm-up, cool-down, and hydration strategies. Exercise training represents promising therapeutic strategy in the management of SCD. It is now time to move through the investigation of long-term biological, physiological, and clinical effects of regular physical activity in SCD patients.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":null,"pages":null},"PeriodicalIF":10.1,"publicationDate":"2024-08-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141915909","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}