EJHaem最新文献

{"title":"Autoimmune Haemolytic Anaemia in Patients With Chronic Liver Disease: Case Series","authors":"Dorien Pint, Abid Suddle, M. Mansour Ceesay","doi":"10.1002/jha2.70035","DOIUrl":"https://doi.org/10.1002/jha2.70035","url":null,"abstract":"<p>Chronic anaemia frequently accompanies chronic liver disease (CLD), contributing to increased morbidity and mortality rates [<span>1</span>]. In up to 53% of cases, the aetiology of anaemia in CLD remains unknown [<span>2</span>]. Autoimmune haemolytic anaemia (AIHA) is characterised by the destruction of red blood cells by circulating autoantibodies directed against erythrocytes. AIHA is a rare autoimmune disorder affecting approximately one to three per 100,000 annually [<span>3</span>]. AIHA has a female predisposition with a female-to-male ratio of approximately 2:1 to 3:1 [<span>4</span>]. The diagnosis of AIHA has additional challenges in CLD, as all these laboratory values can be distorted due to the CLD. For instance, haptoglobin is low in CLD due to poor synthetic function, and similarly, LDH is often raised without haemolysis [<span>5</span>]. Autoimmune liver and bile duct diseases such as autoimmune hepatitis (AIH), primary sclerosing cholangitis (PSC), and primary biliary cholangitis (PBC) often coexist with other autoimmune disorders, though AIHA is a rare comorbidity.</p><p>We conducted a retrospective study from 2002 to 2022 investigating patients with concomitant CLD and AIHA managed in King's College Hospital Institute of Liver Studies and the Department of Haematological Medicine. The patients were identified using search criteria for various causes of CLD combined with AIHA diagnosis. The diagnosis of AIHA was based on laboratory parameters. The review aimed to evaluate the relationship between AIHA and CLD as well as review the diagnostic and therapeutic challenges. Due to the retrospective nature of this study, we were unable to determine the exact number of patients with CLD seen in our institution during the study period. As a result, we could not establish a denominator for AIHA prevalence in this population. However, our objective was not to perform an epidemiological assessment but rather to describe the clinical complexities in diagnosing and managing these rare cases.</p><p>A total of 10 patients were included in this study (Table 1). The median age for the diagnosis of chronic liver disease was 34 years (range 6–72), and the median age of AIHA diagnosis was 43 (19–72 years). The median interval between CLD diagnosis and onset of AIHA was 6 (range 0–17) years. There was female predominance with 70% being female. All patients had classical features of haemolysis with an elevated unconjugated bilirubin (mean 88.5 µmol/L), increased LDH (mean 496 U/L), reduced haptoglobin, and positive direct antiglobulin test (DAT). The most common underlying liver disease was AIH in four patients. Seven patients had cirrhosis, and five received an orthotopic liver transplantation (OLT). Among the five transplanted patients, three developed AIHA after their liver transplant. Indications for liver transplantation were AIH in two patients, and primary sclerosing cholangitis (PSC) and alcohol-related liver disease (ArLD) both in one pat","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70035","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143818347","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring Cryoglobulinemia's Clinical Odyssey: A Case Series","authors":"Shivangini Duggal,&nbsp;Lakshmi Vaishnavi Prasanna Kattamuri,&nbsp;Michel Toutoungy,&nbsp;Eder Luna Ceron,&nbsp;Madhumita Rondla,&nbsp;Angelica Lehker","doi":"10.1002/jha2.70029","DOIUrl":"https://doi.org/10.1002/jha2.70029","url":null,"abstract":"<p>Cryoglobulinemia (CG) encompasses disorders in which immunoglobulins precipitate at low temperatures. According to Brouet's classification, Type I CG is linked to plasma cell disorders, while mixed CG (Types II and III) is associated with autoimmune conditions, infections (notably hepatitis C virus [HCV]), and lymphoproliferative disorders. Each type presents distinct symptoms, with Type I often causing severe vasomotor symptoms and Types II and III involving systemic features like purpura, Raynaud's phenomenon, and renal involvement. This case series presents three CG cases, illustrating diverse etiologies and presentations. Case 1 discusses CG in metastatic colon cancer with <i>Staphylococcus aureus</i> bacteremia, highlighting infection-triggered CG. Case 2 covers HCV-related mixed CG, emphasizing antiviral therapy's role. Case 3 describes a CG flare after rituximab therapy, managed with steroids. These cases emphasize a multidisciplinary approach and individualized management to address CG's complexity and improve patient outcomes.</p><p><b>Clinical Trial Registration</b>: The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70029","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143749853","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Effect of COVID-19 on Platelet Counts in Persistent and Chronic Adult ITP Patients: A Real-World Study in China","authors":"Yujiao Zhang,&nbsp;Lei Yang,&nbsp;Zhongping Xu,&nbsp;Xin Zhou","doi":"10.1002/jha2.70025","DOIUrl":"https://doi.org/10.1002/jha2.70025","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>While coronavirus disease 2019 (COVID-19)-associated thrombocytopenia is well-documented, its effects on immune thrombocytopenia (ITP) patients remain unclear. This study aimed to investigate the impact of COVID-19 infection on platelet (PLT) dynamics in chronic ITP patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This retrospective study analyzed 21 persistent and chronic ITP patients before and after mild COVID-19 infection during China's December 2022 reopening, comparing platelet parameter changes with a focus on clinical characteristics of thrombopoietin receptor agonist (TPO-RA) treated patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>TPO-RA treated patients demonstrated transient platelet surges peaking at 1 week postinfection, returning to baseline within 2–3 weeks, contrasting sharply with thrombocytopenia patterns in non-ITP populations. This suggests synergistic effects between virus-induced inflammatory cytokines and TPO-RA may drive transient megakaryopoiesis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>These findings underscore infection-related PLT fluctuations in ITP, necessitating monitoring for thrombotic and bleeding risks and TPO-RA dose optimization during infections.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70025","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143735436","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lymphocytic Variant Hypereosinophilic Syndrome: Case Series From a Tertiary Referral Center in Canada","authors":"Xiu Qing Wang,&nbsp;Kevin Shopsowitz,&nbsp;Jack Lofroth,&nbsp;Xuehai Wang,&nbsp;Erica Peterson,&nbsp;Andrew P. Weng,&nbsp;Luke Y. C. Chen","doi":"10.1002/jha2.1109","DOIUrl":"https://doi.org/10.1002/jha2.1109","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Lymphocytic variant hypereosinophilic syndrome (L-HES) is a rare disorder characterized by persistent eosinophilia driven by aberrant T-cell populations. Diagnosis remains challenging due to the lack of standardized diagnostic criteria.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We retrospectively analyzed 18 patients diagnosed with L-HES between 2016 and 2023. Comprehensive flow cytometry was performed on peripheral blood samples.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Nine patients demonstrated the classic sCD3⁻CD4⁺CD5⁺CD2⁺CD45RO⁺CD45RA⁻ immunophenotype, ranging from 0.6% to 70% of total lymphocytes. Two patients showed variant sCD3⁻CD4⁺ phenotypes, five had expanded (&gt; 10%) sCD3⁺CD4⁺CD7⁻ T-cells, and two displayed aberrant CD8⁺ T-LGL populations. Clonality was established in all patients with nonclassic phenotypes by molecular TCR testing or based on uniform TRBC1. We assessed a serial gating strategy to quantify the classic L-HES phenotype and found this to be highly sensitive and specific with an estimated limit of detection of 0.06% of lymphocytes. Using this strategy, we identified decreased but detectable abnormal T-cells in all classic phenotype patients reassessed posttreatment, down to as low as 0.3% of lymphocytes. The identification of T-LGL phenotypes with eosinophilia is a novel finding.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Our study highlights the diverse immunophenotypic spectrum of L-HES, emphasizing the importance of comprehensive flow cytometry analysis for accurate diagnosis.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1109","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143735435","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Leaky Artemis Deficiency and EBV-Related Lymphoproliferative Disease: A Novel Case and Review of the Literature","authors":"Lucie Roussel,&nbsp;Stephane Bernier,&nbsp;Gertruda Evaristo,&nbsp;Anna Perez,&nbsp;Sanabelle Zaabat,&nbsp;Romina Pace,&nbsp;Yichun Sun,&nbsp;Isabelle Angers,&nbsp;John Storring,&nbsp;Donald C. Vinh","doi":"10.1002/jha2.70026","DOIUrl":"https://doi.org/10.1002/jha2.70026","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Artemis (<i>DCLRE1C</i>) deficiency causes radiosensitive severe combined immunodeficiency (SCID), although hypomorphic cases can manifest later-onset immunodeficiency, autoimmunity, or lymphoproliferation. We report a 45-year-old man with humoral immunodeficiency, opportunistic infections, and recurrent EBV-positive diffuse large B-cell lymphoma (DLBCL).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Genetic analysis was performed to identify mutations in the <i>DCLRE1C</i> gene. Functional studies, including γH2AX assays to assess DNA damage repair and measurement of Type I interferon responses, were conducted to evaluate the impact of the variant. A literature review was performed to contextualize the findings.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Biallelic p.Leu70del frameshift mutation in <i>DCLRE1C</i> was identified, leading to significantly decreased mutant protein expression. Functional testing confirmed impaired DNA damage repair, via γH2AX measurement, and elevated Type I interferon responses, indicating cytosolic DNA damage accumulation. A literature review highlighted EBV-positive lymphomas in leaky Artemis deficiency with high mortality rate.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Our report adds hypomorphic <i>DCLRE1C</i> deficiency as an inborn error of immunity that predisposes to EBV-associated lymphoproliferative disease.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70026","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143726843","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"MYD88-mutated Lymphoplasmacytic Lymphoma With Monoclonal Immunoglobulin G: A Case Report","authors":"Morten Yung Isaksen,&nbsp;Olav Karsten Vintermyr,&nbsp;Håkon Reikvam","doi":"10.1002/jha2.70027","DOIUrl":"https://doi.org/10.1002/jha2.70027","url":null,"abstract":"<p>Lymphoplasmacytic lymphomas (LPL) are usually associated with serum monoclonal immunoglobulin M (IgM). Nevertheless, in some cases, these cells may secrete IgA or IgG monoclonal proteins or remain non-secretory. We report a case from a patient with LPL-secreting IgG who developed anaemia and splenomegaly during the disease course that necessitated treatment with bortezomib, dexamethasone, and rituximab. The case illustrates the need for clinicians and pathologists to consider LPLs as a differential diagnosis also without a serum monoclonal IgM.</p><p><b>Clinical Trial Registration</b>: The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70027","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143707368","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Striking Case of Light Chain Amyloidosis Visible on Bone Marrow Aspirate Smear","authors":"Adelaide Kwon,&nbsp;Weina Chen","doi":"10.1002/jha2.70028","DOIUrl":"https://doi.org/10.1002/jha2.70028","url":null,"abstract":"&lt;p&gt;Light chain amyloidosis is the most common form of systemic amyloidosis and is often associated with plasma cell neoplasms. Clonal or malignant plasma cells produce excess free immunoglobulin light chains, which leads to protein misfolding, aggregation, and multisystem amyloid deposition, most commonly in antiparallel beta-pleated sheets [&lt;span&gt;1&lt;/span&gt;]. Amyloid deposits can be seen in any tissue but are often found in vessel walls, which are particularly susceptible. Over 60% of bone marrow (BM) biopsies in patients with light chain amyloidosis will demonstrate amyloid deposits in vessel walls or stroma [&lt;span&gt;1&lt;/span&gt;]; however, deposits in the BM aspirate smear are rare.&lt;/p&gt;&lt;p&gt;A 50-year-old male presented with multiorgan imaging abnormalities, biventricular cardiac hypertrophy, and elevated serum lambda light chains (402 mg/L). An endomyocardial biopsy revealed cardiac amyloidosis, with liquid chromatography tandem mass spectrometry (LC/MS) demonstrating AL (lambda)-type amyloid deposition. A BM biopsy was performed for further workup. Peripheral blood (PB) demonstrated mild normocytic anemia (Hgb 12.2 g/dL) and thrombocytosis (platelets 768 × 10&lt;sup&gt;9&lt;/sup&gt;/L). BM aspirate, clot section, and core biopsy showed increased neoplastic plasma cells (∼20%) with cytologic atypia and extensive interstitial and vascular deposition of amorphous material within vessel walls and the interstitium (Figure 1A). Strikingly, these deposits were even visible on the aspirate smear as thick, amorphous, and waxy basophilic globules (Figure 1B). Congo red stains performed on the aspirate smear and core biopsy confirmed these amorphous deposits to be apple-green birefringent under polarized light (Figure 1C,D). Ancillary studies, including flow cytometric immunophenotyping and cytogenetic/FISH studies confirmed an aberrant monotypic lambda-restricted plasma cell population demonstrating gains of Chromosomes 3, 7, 11, 15, 18, 19, and 21; Trisomy 9 and 15; and an extra copy of &lt;i&gt;CCND1&lt;/i&gt; (11q13). The constellation of these findings led to a diagnosis of extensive amyloidosis (AL-lambda type) in the setting of a plasma cell neoplasm. The patient was treated with six cycles of daratumumab, cyclophosphamide, bortezomib, and dexamethasone and underwent autologous stem cell transplant. He is currently on bortezomib maintenance therapy at 35-month follow-up, postdiagnosis.&lt;/p&gt;&lt;p&gt;Amyloid deposits in AL-amyloidosis can be found in any tissue and can lead to organ dysfunction and death, with a major prognostic indicator being the extent of cardiac involvement [&lt;span&gt;1, 2&lt;/span&gt;]. In the BM, deposits are usually identified on core biopsy, most commonly within vessel walls, but also in periosteal areas and within the interstitium. Our case demonstrates very striking, extensive involvement by AL-amyloidosis, with amorphous, and Congo red birefringent amyloid deposits that also visible on aspirate smear. This is a rare and interesting finding with only a few cases reported ","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70028","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143699010","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Central Venous Access Device Complications and Premature Removal in Patients With Haematological Malignancies: A Multi-Site Cohort Study","authors":"Kerrie Curtis,&nbsp;Samantha Keogh,&nbsp;Meinir Krishnasamy,&nbsp;Karla Gough","doi":"10.1002/jha2.1090","DOIUrl":"https://doi.org/10.1002/jha2.1090","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Patients with haematological malignancies require urgent and reliable venous access for the administration of systemic anticancer therapies (SACTs) commonly via central venous access devices (CVADs). Disease pathophysiology and side effects of SACTs increase the risk of complications during the dwell time and premature removal. CVAD complications are associated with treatment disruption, increased morbidity and mortality. This study aimed to comprehensively describe CVAD performance over a 12-month period in patients with haematological malignancies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A multi-site cohort study at four tertiary hospitals in Melbourne, Australia was undertaken using multidisciplinary data from patient health records and administrative datasets including patient, device, insertion, maintenance, complication and removal data. Cases of interest were CVADs, ascertained using lists provided by the insertion services.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Findings</h3>\u0000 \u0000 <p>A total of 1078 CVADs were inserted in 673 patients between 1 September 2020 and 31 August 2021. Of the 1078 CVADs, 197 (18%) remained in situ, and 881 (82%) were removed, of which 369 (42%) were removed prematurely due to infection (<i>n</i> = 208, 57%) and non-infection related reasons (<i>n</i> = 201, 54%). Most CVADs (<i>n</i> = 919, 85%) had documented complications during their dwell time and the proportion of premature removals in these CVADs was over two-fold higher than CVADs with no documented complications. Multivariable Cox regression results indicated that CVAD type, urgency of the procedure, concurrent CVADs and insertion technology were associated with an increased risk of premature removal. Clinical variations in insertion and management care throughout the life of a CVAD and current evidence were identified.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>An unacceptably high proportion of CVADs had complications documented during the dwell time and were prematurely removed. Inconsistencies in current evidence and clinical practice highlight opportunities to positively impact CVAD outcomes in this cohort.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1090","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143698997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EPYSQLI (SB12; Biosimilar to Reference Eculizumab) in Asian and Non-Asian Patients With Paroxysmal Nocturnal Hemoglobinuria: Subgroup Analysis of a Global Phase III Randomized Controlled Trial","authors":"Jun Ho Jang,&nbsp;Ciprian Tomuleasa,&nbsp;Hanna Oliynyk,&nbsp;Theerin Lanamtieng,&nbsp;Jihye Park,&nbsp;Younsoo Kim,&nbsp;Jinah Jung,&nbsp;Paola Russo,&nbsp;Soo Min Lim,&nbsp;Régis Peffaultde Latour","doi":"10.1002/jha2.70020","DOIUrl":"https://doi.org/10.1002/jha2.70020","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>SB12 demonstrated equivalence to reference eculizumab (ECU) in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) in the previous randomized, double-blind, multi-national, crossover, Phase III study.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The scope of this post-hoc study was subgroup analysis by race to compare the efficacy and safety of SB12 and ECU in PNH patients in the Asian and Non-Asian subgroups of the Phase III study.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Results including lactate dehydrogenase (LDH), number of units of packed red blood cells and safety as primary and secondary endpoints demonstrated comparable efficacy and safety of SB12 and ECU in Asian and Non-Asian PNH patients, in line with the study results in the overall population. In addition, transfusion avoidance (68.1% for SB12 vs. 72.9% for ECU, <i>p</i>-value of 0.4492) and hemoglobin stabilization (SB12–ECU: 6.3%, 95% confidence interval [CI] [-21.5, 34.1] and SB12–ECU: 2.5%, 95% CI [-24.8, 29.8] using stringent criteria) as post-hoc endpoints were not substantially different between SB12 and ECU treatment groups in the overall population as well as in Asians and Non-Asians.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>In conclusion, this subgroup analysis by race (Asians and Non-Asians) supports comparable efficacy and safety between SB12 and reference eculizumab in global PNH patients including no difference in transfusion avoidance effect.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70020","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143689502","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"High Fluorescent Lymphoid Cell Levels Assessed on Initial Blood Count in Newly Diagnosed Multiple Myeloma Carry Prognostic Significance","authors":"Yasmine Aramon,&nbsp;Andrea Pieragostini,&nbsp;Pierre Jean Francin,&nbsp;Marie Lorraine Chrétien,&nbsp;Ingrid Lafon,&nbsp;Denis Caillot,&nbsp;François Bailly,&nbsp;Jean Noel Bastie,&nbsp;Cédric Rossi,&nbsp;Marc Maynadié,&nbsp;Julien Guy","doi":"10.1002/jha2.1095","DOIUrl":"https://doi.org/10.1002/jha2.1095","url":null,"abstract":"<p>The outcome of multiple myeloma (MM) has tremendously improved over the past decade, due to the development of efficient chemotherapy and mostly immunotherapy. Yet, some patients still display poor responses and outcome. This could be in part related to the presence of peripheral plasma cells, at levels not compatible with a diagnosis of plasma-cell leukaemia. Some recent publications have highlighted the prognostic influence of low levels of such cells which is around 0.1%. Automated blood cell analysers now include fluorescent staining, allowing to identify cells with higher levels or nucleic acids such as activated B-lymphocytes/plasma cells with highly active ribosomal activity related to antibody production. Here, a prospective evaluation of peripheral high fluorescent lymphoid cell (HFLC) levels was carried out on samples from patients with newly diagnosed MM, and data computed with regard to patient evolution. HFLC above a 0.1% threshold were identified as strongly correlated with poorer response in a cohort of 127 patients. The 74 patients with low HFLC had a significantly better PFS both in univariate (<i>p</i> = 0.0017) and multivariate (<i>p</i> = 0.0007) analyses. This simple test provides a significant prognostic value for patients with MM and could eventually lead clinicians to consider more aggressive strategies for patients with peripheral HFLC above 0.1%.</p><p><b>Trial Registration</b>: The authors have confirmed clinical trial registration is not needed for this submission</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1095","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143689500","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}