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
{"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":"10.1002/ajh.27458","url":null,"abstract":"<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%; <i>p =</i> .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":"99 11","pages":"2063-2074"},"PeriodicalIF":10.1,"publicationDate":"2024-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27458","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":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Philippe Connes, Emeric Stauffer, Robert I. Liem, Elie Nader
{"title":"Exercise and training in sickle cell disease: Safety, potential benefits, and recommendations","authors":"Philippe Connes, Emeric Stauffer, Robert I. Liem, 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":"99 10","pages":"1988-2001"},"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}
Christin B. DeStefano, Nicholas Burwick, Drew A. Helmer
{"title":"Are veterans at increased risk of myeloproliferative neoplasms?","authors":"Christin B. DeStefano, Nicholas Burwick, Drew A. Helmer","doi":"10.1002/ajh.27453","DOIUrl":"10.1002/ajh.27453","url":null,"abstract":"","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 10","pages":"1852-1854"},"PeriodicalIF":10.1,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27453","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141905553","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Self-pay laboratory charges for iron deficiency diagnosis in the Boston and New Haven metropolitan areas","authors":"Layla Van Doren, Carlo Brugnara","doi":"10.1002/ajh.27457","DOIUrl":"10.1002/ajh.27457","url":null,"abstract":"<p>The economic inequality of healthcare has been well documented. Out-of-pocket costs, which include laboratory costs, contribute to the financial burden that patients must bear for healthcare. This is particularly true for those patients living with a condition that requires frequent laboratory monitoring. Such is the case for those suffering from iron deficiency and iron deficiency anemia. Anemia accounts for 68.4 million years of life lived with a disability.<span><sup>1</sup></span> Data from Scripps-Kaiser and the National Health and Nutrition Examination Survey highlights the prevalence of anemia in the United States to be highest in Black women (or those who menstruate).<span><sup>2</sup></span> Recent data from Canada showed the correlation between anemia and socioeconomic status with lower household income associated with the greatest odds of anemia.<span><sup>3</sup></span> Various factors contribute to the burden of anemia in women of color, including disproportionately higher rates of heavy menstrual bleeding (HMB) from fibroids.<span><sup>4</sup></span> HMB, defined as greater than 80 mL blood loss per month or excessive menstrual blood loss that interferes with a woman's physical, emotional, social, and material quality of life, depletes iron stores leading to iron deficiency.<span><sup>5</sup></span> HMB has been reported to affect between 10% and 30% of reproductive age women in the United States, with a symptom duration of years commonly reported.<span><sup>6</sup></span> These numbers are even higher in persons with a bleeding disorder or on anticoagulation.<span><sup>7</sup></span> Iron deficiency leads to fatigue, impaired cognitive function, and decreased mood, leading to loss of quality of life and personal productivity.<span><sup>8</sup></span></p><p>HMB and resultant iron deficiency require frequent healthcare visits and laboratory monitoring. The correlation between race, income, and healthcare spending are profound. One study found Black insured participants were more likely to reduce spending on basic needs, leisure activities, and skip medications to cover the cost of medical care compared to non-Black participants.<span><sup>9</sup></span></p><p>To address disparities in healthcare access and affordability, it is necessary to understand the financial implications of obtaining necessary testing. Federal regulations require hospitals to post charges for procedures and laboratory tests. Charge data are available as master files or as on-line calculators for a limited number of tests. Price estimates can also be directly requested from financial service offices. Price estimates may include a discount for self-pay. We set out to determine the self-pay charges for iron deficiency and anemia testing including a complete blood cell count (CBC) and a serum/plasma ferritin at major medical centers in Boston, Connecticut, and two national laboratories.</p><p>In this cross-sectional study, we manually collected laboratory charge","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 11","pages":"2233-2235"},"PeriodicalIF":10.1,"publicationDate":"2024-08-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27457","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141905554","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Samer Al Hadidi, Obada Ababneh, Carolina Schinke, Sharmilan Thanendrarajan, Clyde Bailey, Guido Tricot, John Shaughnessy Jr, Fenghuang Zhan, Jeffrey Sawyer, Eric R. Siegel, Maurizio Zangari, Bart Barlogie, Frits van Rhee
{"title":"Second primary malignancies after tandem autologous hematopoietic stem cell transplantation for multiple myeloma","authors":"Samer Al Hadidi, Obada Ababneh, Carolina Schinke, Sharmilan Thanendrarajan, Clyde Bailey, Guido Tricot, John Shaughnessy Jr, Fenghuang Zhan, Jeffrey Sawyer, Eric R. Siegel, Maurizio Zangari, Bart Barlogie, Frits van Rhee","doi":"10.1002/ajh.27452","DOIUrl":"10.1002/ajh.27452","url":null,"abstract":"<p>Autologous hematopoietic stem cell transplantation (HSCT) is a standard treatment for eligible multiple myeloma (MM) patients.<span><sup>1</sup></span> Before high-dose chemotherapy and autologous HSCT, patients typically receive induction therapy with a proteasome inhibitor and an immunomodulatory (IMiD) agent. This approach, along with posttransplant maintenance therapies, has improved survival, with some patients achieving a median overall survival (OS) of over 10 years.<span><sup>1</sup></span></p><p>While late mortality rates have decreased over the past three decades, the incidence of second primary malignancies (SPM) has not.<span><sup>2</sup></span> Given improved survival rates, understanding long-term complications like SPM is crucial, particularly as most phase III MM trials lack extended follow-up.<span><sup>3</sup></span></p><p>Autologous HSCT enhances long-term disease control and survival in newly diagnosed MM patients but increases the risk of SPM, including second hematologic malignancies (SHM). A Swedish study found that higher cumulative doses of melphalan were linked to a 2.8-fold increased risk of therapy-related myeloid neoplasms.<span><sup>4</sup></span> In a study of 3948 MM patients, 4% developed SPM, with 64% being solid tumors, 20% myeloid, 14% SHM, and 2% lymphoid malignancies. This study included only single autologous HSCT patients and had a median follow-up of 37 months, limiting the assessment of late SPM development.<span><sup>5</sup></span></p><p>The principal objective of this study was to examine the occurrence and attributes of SPM and their subsequent effects on OS. Additionally, we sought to investigate whether the utilization of tandem autologous HSCT was associated with an elevated risk of SPM in comparison with single autologous HSCT. Secondary aims included the classification of various SPM types that emerged following autologous HSCT. Methods are detailed in supplementary.</p><p>A total of 1379 patients with newly diagnosed MM enrolled on four TT trials. An overview of the patients' characteristics can be found in [Table S1]. The median follow-up durations varied across different treatment groups: 25.3 years for TT I, 20.4 years for TT II (Arm A), 19.8 years for TT II (Arm B), 17.1 years for TT IIIA, and 15.4 years for TT IIIB. When considering the entire cohort of 1379 patients in the study, the median follow-up period was 16.6 years, with an interquartile range (IQR) spanning from 13.5 to 20 years. A total of 2640 transplants were performed on patients in our study cohort, with most of the transplants as first autologous HSCT (<i>n</i> = 1267) and second autologous HSCT (<i>n</i> = 1034) [Table S2]. Patients' baseline characteristics according to the type of transplant are summarized in [Table S3]. Overall, among the total of 1379 enrolled patients in the study, 974 patients (71%) underwent tandem autologous HSCT, with an average time interval of approximately 3.2 months between the first and secon","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 11","pages":"2222-2224"},"PeriodicalIF":10.1,"publicationDate":"2024-08-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27452","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141896493","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"2025 update on clinical trials in immune thrombocytopenia","authors":"Hanny Al-Samkari","doi":"10.1002/ajh.27448","DOIUrl":"10.1002/ajh.27448","url":null,"abstract":"<p>Although the development and regulatory approval of the thrombopoietin receptor agonists revolutionized aspects of the immune thrombocytopenia (ITP) treatment landscape over the past two decades, there remain many areas of high unmet need. Therefore, a number of investigational and repurposed agents are currently undergoing clinical development in ITP. In a departure from historical trials, which largely focused on the indefinite treatment of persistent or chronic ITP, ongoing trials run the gamut of disease phases, and include novel agents being evaluated in early phases of the disease to attempt to modify the disease course. Many agents in development target disease pathophysiologic mechanisms not previously targeted by agents in current use, including platelet autoantibody recycling, B-cell maturation and differentiation, long-lived plasma cells, and the complement system, among others. These agents represent promising treatment options for patients with otherwise refractory disease or who are intolerant of currently available therapies. Additionally, with our increasing understanding of the diverse immune mechanisms at play in ITP, the expansion of the therapeutic armamentarium to include agents targeting diverse pathophysiologic mechanisms may allow a more personalized therapeutic selection in the future. This manuscript provides an up-to-date, in-depth overview of recently completed and ongoing clinical trials in ITP.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 11","pages":"2178-2190"},"PeriodicalIF":10.1,"publicationDate":"2024-08-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27448","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141892703","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Robert T. Means Jr, Caixia Bi, Edward C. C. Wong, Lance A. Bare, Michael J. McPhaul
{"title":"Ferritin reference intervals in a population of working-age adults without anemia","authors":"Robert T. Means Jr, Caixia Bi, Edward C. C. Wong, Lance A. Bare, Michael J. McPhaul","doi":"10.1002/ajh.27444","DOIUrl":"10.1002/ajh.27444","url":null,"abstract":"<p>Iron deficiency anemia is recognized as one of the most important contributors to the worldwide burden of illness. It has a particularly high incidence in children and females of childbearing years in both the developed and less-developed world. It is generally recognized that serum ferritin concentration is the single most useful laboratory test in the diagnosis of iron deficiency.<span><sup>1</sup></span> However, it has significant limitations. Ferritin is an acute phase reactant and can be increased out of proportion to iron stores when inflammation is present and even without inflammation the specific ferritin concentration that represents iron deficiency is not clearly established, particularly in females.<span><sup>1, 2</sup></span></p><p>The authors reviewed anonymized data from 28,134 Quest Diagnostics employees participating in the Quest Blueprint For Wellness (BFW) screening program to evaluate age-specific ferritin reference intervals in a population of adults aged 18–80 years without anemia or other red cell abnormalities. In addition, changes in red cell indices in relation to serum ferritin concentrations (mean corpuscular volume (MCV); mean corpuscular hemoglobin (MCH); mean corpuscular hemoglobin concentration (MCHC); and the coefficient of variation of the red blood cell distribution width (RDW)) as well as the hemoglobin concentration, red blood cell concentration (RBC), and the hematocrit were analyzed in order to identify a more physiologic ferritin cutoff at which changes suggestive of iron deficiency may begin to appear. Individuals with values outside the Quest reference intervals for hemoglobin concentration, hematocrit, RBC, MCV, MCH, MCHC, and RDW (reference values shown in Table S2); serum total iron binding capacity (TIBC) saturation <20%; or an International Classification of Diseases-10 (ICD 10) code indicating an anemia diagnosis or a self-reported history of anemia on their BFW health questionnaire were excluded. To exclude individuals whose serum ferritin concentrations might be elevated out of proportion to iron stores by inflammation, individuals with high sensitivity C-reactive protein (hsCRP) >3 mg/L were excluded. Data from the remaining 24 812 individuals (55% female) were analyzed as presented below. Specific details of the study population including racial/ethnic and geographic demographics are shown in Table S1 and Figure S1.</p><p>The statistical technique multimodal decomposition was utilized to determine the reference ranges for serum ferritin concentrations in the final study population. Results were expressed by age deciles, with the lower limit representing the 2.5% confidence interval of population distribution for the individuals studied. A detailed description of methodology is provided in Supplemental Methods.</p><p>The ferritin reference interval lower limits for females 50 years of age or younger ranged between 11 and 13 ng/mL and were substantially lower than the lower limits for ma","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 10","pages":"2047-2049"},"PeriodicalIF":10.1,"publicationDate":"2024-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27444","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141888268","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Abdul-Hamid Bazarbachi, Divaya Bhutani, Jai Radhakrishnan, Markus Mapara, Mathew S. Maurer, Suzanne Lentzsch, Rajshekhar Chakraborty
{"title":"Timing and outcomes of second-line therapy in the era of daratumumab-based frontline therapy in AL amyloidosis","authors":"Abdul-Hamid Bazarbachi, Divaya Bhutani, Jai Radhakrishnan, Markus Mapara, Mathew S. Maurer, Suzanne Lentzsch, Rajshekhar Chakraborty","doi":"10.1002/ajh.27450","DOIUrl":"10.1002/ajh.27450","url":null,"abstract":"<p>The therapeutic goal in immunoglobulin light-chain amyloidosis (AL) is to eradicate the plasma cell (PC) clone producing misfolded amyloid fibrils. Therapies for AL often draw inspiration from successful treatments in multiple myeloma (MM). However, the field faced a challenge when second-generation proteasome inhibitors and immunomodulatory drugs (IMiDs) used for MM proved poorly tolerated by AL patients, creating a significant treatment gap between the two diseases. The ANDROMEDA trial marked a turning point by combining daratumumab, a CD38-directed monoclonal antibody, with the previous standard of care—bortezomib, cyclophosphamide, and dexamethasone (VCd). This combination dramatically improved response rates, with approximately 80% of patients achieving a “≥” very good partial response (VGPR), compared to 50% with VCd alone.<span><sup>1</sup></span></p><p>Before the ANDROMEDA trial, approximately one-third of AL patients eventually required a second line of treatment (2nd LoT), often due to an unsatisfactory hematologic response (less than partial response [PR]), hematologic progression/relapse, and lack of organ response.<span><sup>2</sup></span> The landscape of 2nd LoT in the daratumumab era and the outcomes of such treatments are poorly characterized. Our study addresses this knowledge gap by examining the real-world outcomes of 100 consecutive patients with newly diagnosed AL who received upfront daratumumab-based therapy between January 2018 and December 2023.</p><p>The median age was 67.6 years [39.6–91.1]; 60% of patients were male; and the majority were White (67.3%) (Table S1). NYHA Class was III/IV in 44.3% at diagnosis. Most had lambda light chain involvement (76%), and median dFLC was 229 mg/L [2–9069]. Median NT-proBNP was 3317 ng/L [22–70 000], 26.9% ≥8500 ng/L. Median HS troponin-T was 64 ng/L [10–405]. Median BMPC percentage was 15% [2–70]. Regarding cytogenetics, 46% had <i>t</i>(11;14), 39.4% had del(13q), 40% had 1q gain/amplification, 28.6% had hyperdiploidy, and 13.4% had high-risk myeloma abnormalities. Most patients had modified Mayo 2004 stage IIIA (39.2%) or IIIB (24.7%) and renal stage II (46.3%). The most common frontline regimen was Dara-VCd (91%), followed by Dara-Vd/Dara-ixazomib-d (4%), Dara-d (4%), and Dara-Cd (1%), and 13% underwent ASCT consolidation. Comparing baseline characteristics between patients who received 2nd LoT versus those who did not, significant differences were noted in dFLC and %BMPC, with dFLC >180 mg/L in 78.8% versus 51.5% respectively (<i>p</i> = .005) and BMPC >15% in 67.6% versus 40.7% respectively (<i>p</i> = .02).</p><p>Median follow-up after 1st LoT was 22.3 months [2–69.7], with hematologic response evaluable for 84/100 patients. Most achieved either CR (46.4%), low-dFLC-PR (3.6%), or VGPR (21.4%) (Table S2). Twenty-three patients had either a PR (13.1%) or no response (NR) (15.5%) and received 2nd LoT. Overall, 34% required a 2nd LoT. Three patients who did not respond t","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 11","pages":"2225-2228"},"PeriodicalIF":10.1,"publicationDate":"2024-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27450","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141878174","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Echinocytes in pyruvate kinase deficiency, post-splenectomy","authors":"Sarah A. Bassiony, Asad Luqmani, Barbara J. Bain","doi":"10.1002/ajh.27449","DOIUrl":"10.1002/ajh.27449","url":null,"abstract":"<p>Mitapivat has been shown in a randomized clinical trial to significantly improve hemoglobin levels and reduce transfusion requirements,<span><sup>3</sup></span> offering an improved quality of life, whilst also reducing the compounded risks associated with chronic transfusion in a young population.</p><p>The authors declare no conflict of interest.</p>","PeriodicalId":7724,"journal":{"name":"American Journal of Hematology","volume":"99 12","pages":"2379-2380"},"PeriodicalIF":10.1,"publicationDate":"2024-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/ajh.27449","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141873971","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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