EJHaem最新文献

{"title":"Beyond Childhood: Adult and Adolescent Sickle Cell Disease and Outcomes in Northern Ghana","authors":"Kwadwo Faka Gyan,&nbsp;Solomon Gyabaah,&nbsp;Eunice Agyeman Ahmed,&nbsp;Lesley Osei,&nbsp;Mohammed Najeeb Naabo,&nbsp;Michael Asiedu Owiredu,&nbsp;Yaw Obeng Opare-Addo,&nbsp;Jessey Mahama Holu,&nbsp;Ohene Kwaku Opare-Sem","doi":"10.1002/jha2.70023","DOIUrl":"https://doi.org/10.1002/jha2.70023","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Adults and adolescents face different barriers to healthcare utilization compared to children.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To describe adult and adolescent sickle cell disease (SCD) and outcomes in northern Ghana.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This was a retrospective cohort study of SCD patients aged 13 years and above, admitted between January 1, 2021 and December 31, 2022 at the Komfo Anokye Teaching Hospital. The data was summarized with descriptive statistics and a multivariate logistics regression analysis was fitted to identify factors independently associated with prolonged hospital stay of more than 4 days.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of the 326 admissions, 68.9% regularly attended their sickle cell clinics. Approximately 3% of all admissions into the internal medicine ward were due to SCD. Commonest complications observed were painful vaso-occlusive crisis (VOC) (78.1%), infection (51.2%), and hyperhemolysis (24.0%). Presented as adjusted odds ratio (95% CI), the predictors of prolonged hospital stay were: presence of comorbidities, 2.71 [(1.28, 5.97), <i>p</i> = 0.011]; infection, 1.78 [(1.08, 2.94), <i>p</i> = 0.024]; acute chest syndrome, 2.42 [(1.22, 4.970), <i>p</i> = 0.013]; hyperhemolysis, 2.02 [(1.08, 3.80), <i>p</i> = 0.028]; sequestration crisis, 3.80 [(1.50, 11.0), <i>p</i> = 0.008]; and requirement for transfusion, 3.58 [(1.80, 7.36), <i>p</i> &lt; 0.001]. Mortality rate was 2.5%.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>SCD and its related complications constitute a significant proportion of all admissions into the adult medical ward. Approximately one in every three Ghanaian adult and adolescent SCD patients does not regularly attend the SCD clinic.</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":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70023","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143689503","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":"Real World Management of Cytopenias and Infections in Patients With Myelofibrosis Treated With Ruxolitinib","authors":"Liesl A. Butler,&nbsp;Cecily Forsyth,&nbsp;Claire Harrison,&nbsp;Andrew C. Perkins","doi":"10.1002/jha2.70007","DOIUrl":"https://doi.org/10.1002/jha2.70007","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Ruxolitinib was the first JAK2 inhibitor approved for the treatment of primary and secondary myelofibrosis. It is currently used worldwide as first-line therapy for advanced disease (intermediate-2 and high-risk) and is effective in polycythaemia vera (PV) and essential thrombocythaemia (ET), but not funded for this indication in many countries. Ruxolitinib has proven benefits with respect to symptom control, reduction in spleen size and prolongation of survival; however, it rarely induces a substantial reduction in allele burden and never provides a cure. Moreover, there are frequently encountered adverse effects and dosing issues that require careful management to optimise its therapeutic benefit.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods and Results</h3>\u0000 \u0000 <p>In this case-based review, we use seven informative common clinical scenarios to discuss appropriate investigation and management of cytopenias and infection issues.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>We make recommendations based on 15 years of experience in using ruxolitinib and other JAK inhibitors for the treatment of myelofibrosis. We discuss when allogeneic haematopoietic stem cell transplantation (AHSCT) should be considered and some of the currently available alternative JAK inhibitors and trial options when AHSCT is not an option.</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/jha2.70007","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143689501","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":"Sézary-Like Features in Atypical Vacuolated Cells of Adult T-Cell Leukemia/Lymphoma","authors":"Radu Chiriac,&nbsp;Lucile Baseggio","doi":"10.1002/jha2.70024","DOIUrl":"10.1002/jha2.70024","url":null,"abstract":"&lt;p&gt;A man in his 40s presented with a 2-week history of fatigue, headaches, diffuse abdominal pain, and sweating, without fever. Clinical examination revealed multiple bilateral cervical and inguinal lymphadenopathies, splenomegaly, and a back rash without evidence of papules. Additionally, a whole-body CT scan demonstrated extensive lymphadenopathy above and below the diaphragm, as well as lytic bone lesions in the pelvis.&lt;/p&gt;&lt;p&gt;Laboratory investigations revealed hypercalcemia (3.2 mmol/L), elevated lactate dehydrogenase (2000 U/L), anemia (78 g/L), and leukocytosis (95 × 10⁹/L) with 80% monomorphic atypical lymphoid cells, characterized by folded chromatin nuclei, weakly basophilic cytoplasm, and rare vacuoles (Figure 1A). Additionally, HTLV-1 serology was positive.&lt;/p&gt;&lt;p&gt;Flow cytometry of peripheral blood (Figure 1B, red population) identified a CD4+ T-cell population that had lost surface CD3, with weak CD2, weak CD30, strong CD5, and no CD7 expression. The cells expressed CD25 and were negative for CD26, KIR3DL2, CCR4, and T follicular helper markers (ICOS, CXCR5, PD1). Further, an inguinal lymph node biopsy demonstrating the same phenotypic profile strongly supported the diagnosis of adult T-cell leukemia/lymphoma (ATLL).&lt;/p&gt;&lt;p&gt;Extensive lymphoma infiltration of the bone marrow was observed, with no evidence of central nervous system involvement. Conventional bone marrow karyotyping revealed no chromosomal abnormalities and targeted next-generation sequencing of the lymph node identified mutations in the &lt;i&gt;TP53&lt;/i&gt; (variant allele frequency [VAF] 32%) and &lt;i&gt;PLCG1&lt;/i&gt; (VAF 36%) genes.&lt;/p&gt;&lt;p&gt;After the third cycle of Brentuximab vedotin in combination with cyclophosphamide, doxorubicin, etoposide, and prednisone, there was no clinical, microscopic, or radiographic evidence of disease, according to the adapted Lugano and ATLL staging criteria. In complete remission, a plan for consolidation therapy involving allogeneic stem cell transplantation is currently being pursued.&lt;/p&gt;&lt;p&gt;This case highlights the morphological pleomorphism observed in an acute leukemic presentation of ATLL. While medium to large-sized lymphocytes with multilobulated nuclei, commonly referred to as “flower cells,” are a characteristic feature in the acute form of the disease, the present case presents lymphocytes with convoluted nuclei, a morphology more frequently associated with Sézary syndrome. Furthermore, the identification of documented variants, including chronic lymphocytic leukemia-like morphology and prolymphocytic features, emphasizes the considerable morphological variability inherent to this entity [&lt;span&gt;1, 2&lt;/span&gt;].&lt;/p&gt;&lt;p&gt;In addition to the detection of integrated human T-cell leukemia virus type 1 in isolated lymphoma cells or the presence of anti-human T-cell leukemia virus type 1/2 antibodies in the serum, which serve as imperfect surrogates, ATLL lacks distinct clinical, morphological, immunophenotypic, or molecular characteristics. The variability in ","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11923451/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143671760","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":"Clinical Impact of CD19 Expression Assessed by Quantitative PCR in Lymphoma Patients Undergoing CAR-T Therapy","authors":"Rafael Hernani,&nbsp;Laura Ventura,&nbsp;Begoña Heras,&nbsp;Alicia Serrano,&nbsp;Marcos Rivada,&nbsp;Carolina Martínez-Ciarpaglini,&nbsp;Ana Benzaquén,&nbsp;Blanca Ferrer-Lores,&nbsp;Ariadna Pérez,&nbsp;José Luis Piñana,&nbsp;Juan Carlos Hernández-Boluda,&nbsp;Ignacio Arroyo,&nbsp;Paula Amat,&nbsp;Irene Pastor-Galán,&nbsp;María José Remigia,&nbsp;Rosa Goterris,&nbsp;Montse Gómez,&nbsp;Anabel Teruel,&nbsp;Ana Saus,&nbsp;Consejo Ortí,&nbsp;María José Terol,&nbsp;Antonio Ferrández-Izquierdo,&nbsp;Carlos Solano","doi":"10.1002/jha2.70015","DOIUrl":"https://doi.org/10.1002/jha2.70015","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Current guidelines do not mandate CD19 tumor expression assessment before chimeric antigen receptor T-cell (CAR-T) therapy in large B-cell lymphoma (LBCL) patients due to limitations of immunohistochemistry (IHC) or flow cytometry. Quantitative polymerase chain reaction (qPCR) offers a more sensitive alternative for detecting CD19 expression, with the primary advantage that mRNA can be easily extracted from paraffin-embedded tissues.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods &amp; Results</h3>\u0000 \u0000 <p>In our study, we included 51 adult patients with LBCL treated with axicabtagene ciloleucel. Among them, 16 were classified as CD19-negative by IHC; however, qPCR reclassified six (37.5%) as CD19-positive. We then compared the outcomes between consistently CD19-negative (IHC⁻qPCR⁻) and CD19-positive (IHC⁺ and IHC⁻qPCR⁺) patients. CD19-negative cohort showed worse 1-year progression-free survival (15 vs. 45%, <i>p</i> = 0.044) and a trend toward shorter duration of response (29 vs. 55%, <i>p</i> = 0.065). Only one (10%) of the CD19-negative patients remained alive and disease-free at last follow-up (6 months), having previously responded to bridge therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion</h3>\u0000 \u0000 <p>If confirmed in a large patient cohort, these findings could form the basis for modifying current patient selection criteria. Consistently negative patients may be suboptimal candidates for anti-CD19 CAR-T therapy. Alternative therapeutic options, such as bispecific antibodies or polatuzumab-based regimens, could be considered for this subset of patients.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70015","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143645725","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":"Safety and Efficacy of Busulphan Based on Dosing Patterns in the Real-World Management of Myeloproliferative Neoplasms","authors":"Ali Mahdi,&nbsp;Alexandros Rampotas,&nbsp;Patrick Roberts,&nbsp;Joanna Stokes,&nbsp;Eamon Mahdi,&nbsp;Ruth Witherall,&nbsp;Deepak Mannari,&nbsp;Naheed Ibrahim,&nbsp;Georgina Naylor,&nbsp;Mamta Garg,&nbsp;Imran Manjra,&nbsp;Paula Glancy,&nbsp;George Katis,&nbsp;Sahil Bhagat,&nbsp;Jason Coppell,&nbsp;Andrew McGregor,&nbsp;Rebecca Frewin,&nbsp;Nauman M. Butt","doi":"10.1002/jha2.1097","DOIUrl":"https://doi.org/10.1002/jha2.1097","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Myeloproliferative neoplasms (MPNs), such as polycythaemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF), are primarily treated by managing blood counts to reduce the thrombotic risk using cytoreductive agents. Busulphan, an oral alkylating agent, has been historically used for MPN management due to its myelosuppressive effects, but concerns about its risk of leukaemic transformation have limited its use.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This real-world retrospective study evaluated the safety and efficacy of busulphan in 115 MPN patients across 13 UK hospitals. Responses in patients with ET and PV only were assessed using European LeukemiaNet (ELN) criteria.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>With a median age of 78 years, the overall response rate was 78.1%, with 29% of PV and 18% of ET patients achieving complete responses. Dosing regimens were similarly distributed between repeated single doses of busulphan (31%), courses of treatment lasting 1–4 weeks (30%) and continuous therapy for more than 4 weeks (35%). No cases of disease progression to acute leukaemia or myelofibrosis were recorded during the median follow-up of 23 months. Adverse events were infrequent, with fatigue and cytopaenia being the most common (4% each).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Busulphan demonstrated a favourable safety profile and is a viable cytoreductive option, particularly for elderly patients who are intolerant to hydroxycarbamide.</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":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1097","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143645724","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":"Bortezomib, Rituximab and Dexamethasone Regimen (BDR) in Waldenström Macroglobulinaemia: A Retrospective Real-World Analysis","authors":"Thomas Hueso,&nbsp;Grégory Lazarian,&nbsp;Paul Chauvet,&nbsp;Adrien Chauchet,&nbsp;Ramy Rahmé,&nbsp;Sabine Brechignac,&nbsp;Vincent Lévy,&nbsp;Salomon Manier,&nbsp;Damien Roos-Weil,&nbsp;David Ghez,&nbsp;Claude Gardin,&nbsp;Fanny Baran-Marszak,&nbsp;Eric Durot,&nbsp;Pierre Morel,&nbsp;Thorsten Braun","doi":"10.1002/jha2.70019","DOIUrl":"https://doi.org/10.1002/jha2.70019","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>We retrospectively analysed bortezomib–dexamethasone–rituximab (BDR) combination in patients with Waldenström macroglobulinaemia (WM) in a real world setting.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A total of 87 patients were included: 49 patients (56%) were treated in frontline, 22 (25%) in second line and 16 (19%) in third or further line settings. A log-rank test was used to compare overall and event-free survival (OS and EFS) whereas a Gray's test was performed to compare cumulative incidence of deaths and relapse (CID and CIR) according to the IPSS-WM groups, MYD88/CXCR4 mutational status and line of therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The overall response rate was 88% with five patients (6%) achieving complete response, 20 (24%) very good partial response, 38 (45%) partial response and 11 (13%) minor response. The median time to achieve the best overall response was 9 months and the median EFS was 33 months for whole cohort. Patients treated in third line or further relapse settings had significantly lower median EFS compared to those treated in second- or first-line setting (13 vs. 36 vs. 47 months, respectively, <i>p</i> = 0.01) and a higher 7-year CID (50% vs. 13% vs. 12% respectively, <i>p</i> = 0.02). Among patients for whom mutational status was available, MYD88<i>^L265P</i> mutation or double mutation MYD88/CXCR4 did not influence OS or EFS. Severe peripheral neurotoxicity affected 7% of patients and 52 (62%) patients relapsed or died as result of WM whereas 21 patients (24%) died of unrelated causes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>BDR represents an interesting chemo-free, fixed duration regimen for patients in first or second line, regardless of mutational status.</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":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70019","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143645723","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":"Underrecognised Functional Hyposplenism Associated With Chronic Graft-Versus-Host Disease: A Case Report","authors":"Kaori Uchino,&nbsp;Yuya Nakagami,&nbsp;Megumi Enotomoto,&nbsp;Nozomi Shimizu,&nbsp;Kenichi Kondo,&nbsp;Takahiro Yamamoto,&nbsp;Yukie Sugita,&nbsp;Hideshige Seki,&nbsp;Sakura Saigusa,&nbsp;Yusuke Iida,&nbsp;Saki Shinohara,&nbsp;Soichi Takasugi,&nbsp;Tomohiro Horio,&nbsp;Satsuki Murakami,&nbsp;Shohei Mizuno,&nbsp;Kazuhiro Ikegame,&nbsp;Ichiro Hanamura,&nbsp;Akiyoshi Takami","doi":"10.1002/jha2.70017","DOIUrl":"https://doi.org/10.1002/jha2.70017","url":null,"abstract":"&lt;p&gt;Haematopoietic stem cell transplantation (HSCT) is a curative treatment for haematologic malignancies; however, complications such as graft-versus-host disease (GVHD) and infections remain major causes of morbidity and mortality [&lt;span&gt;1-4&lt;/span&gt;]. Functional hyposplenism, often linked to chronic GVHD, is an underrecognised complication of HSCT that increases the risk of severe infections caused by encapsulated organisms [&lt;span&gt;5&lt;/span&gt;]. This report describes a case of a patient with chronic GVHD who developed progressive splenic atrophy and persistent Howell–Jolly bodies (HJBs) in peripheral blood smears, indicative of functional hyposplenism [&lt;span&gt;6-9&lt;/span&gt;]. The case highlights the importance of blood smear examinations and imaging studies for early diagnosis and management of this overlooked complication.&lt;/p&gt;&lt;p&gt;A 44-year-old man with acute myeloid leukaemia (AML) harbouring &lt;i&gt;NPM1&lt;/i&gt; and &lt;i&gt;DNMT3&lt;/i&gt; mutations underwent allogeneic HSCT from an HLA-identical sibling donor after induction therapy, which caused persistent pancytopenia despite achieving complete remission. Myeloablative conditioning with cyclophosphamide and busulphan was administered, and GVHD prophylaxis included cyclosporine (CSA) and short-term methotrexate. Engraftment was achieved with full donor chimerism, and no acute GVHD or initial complications occurred, allowing CSA tapering.&lt;/p&gt;&lt;p&gt;On day 185 post-HSCT, cryptogenic organising pneumonia (COP) developed, requiring prednisolone (PSL; 0.5 mg/kg/day). PSL tapering followed symptom improvement. However, oral lichenoid lesions and elevated liver enzyme levels on day 380 led to a diagnosis of chronic GVHD. PSL was increased to 10 mg/day, but tapering PSL and CSA proved challenging due to worsening GVHD symptoms.&lt;/p&gt;&lt;p&gt;At 2.5 years post-HSCT, HJBs were detected in peripheral blood smears (Figure 1), and computed tomography (CT) scans revealed significant splenic atrophy. The spleen volume, normal at HSCT (136 mL, Figure 2A), progressively declined—34 mL at 2.5 years (Figure 2B), 22 mL at 3.5 years (Figure 2C), 12 mL at 4.5 years (Figure 2D), and 8 mL at 5.5 years (Figure 2E).&lt;/p&gt;&lt;p&gt;This case highlights the need to recognise functional hyposplenism as a complication of chronic GVHD post-HSCT. The patient's progressive splenic atrophy, identified via routine CT imaging, and persistent HJBs in peripheral blood smears indicated functional hyposplenism. Despite its clinical significance, functional hyposplenism is often underdiagnosed due to overlooked splenic atrophy in radiology reports and the limited use of blood smear examinations.&lt;/p&gt;&lt;p&gt;The spleen plays a key role in immune defence and erythrocyte filtration, and its dysfunction increases susceptibility to infections by encapsulated microorganisms [&lt;span&gt;7, 10&lt;/span&gt;]. This patient received PCV13 and PPV23 vaccinations and prophylactic antibiotics, including trimethoprim-sulphamethoxazole and levofloxacin, which effectively prevented pneumococcal infections. Such prev","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70017","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143612395","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":"Characterization of Immunoglobulin Heavy Locus Rearrangements in Molecular Subtypes of Childhood B-Cell Precursor Acute Lymphoblastic Leukemia","authors":"Guilherme Navarro Nilo Giusti,&nbsp;Patrícia Yoshioka Jotta,&nbsp;Caroline de Oliveira Lopes,&nbsp;Natacha Azussa Migita,&nbsp;Amilcar Cardoso de Azevedo,&nbsp;Sílvia Regina Brandalise,&nbsp;João Meidanis,&nbsp;José Andrés Yunes","doi":"10.1002/jha2.70003","DOIUrl":"https://doi.org/10.1002/jha2.70003","url":null,"abstract":"<p>Biased <i>IGH</i> VDJ recombination has been previously described in childhood B-cell precursor acute lymphoblastic leukemia (BCP-ALL), although its causes are not yet fully understood. This study assesses differential features in 565 <i>IGH</i> clonotypes from BCP-ALL molecular subsets against 560 clonotypes from bone marrow donors. Leukemia clonotypes were enriched for <i>IGHV6-1</i> segments in the KMT2A rearranged and B-other subtypes, while <i>IGHV3-23</i> was enriched in TCF3::PBX1. ETV6::RUNX1 presented a topological gap in the usage of central IGHV segments. BCP-ALL also presented shorter CDR3 regions, higher GC content, and lower productivity. Interestingly, productive clonotypes tended to be absent after induction therapy.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70003","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143595044","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":"Invasive Fungal Disease Associated With Targeted Agents for Acute Myeloid Leukaemia: A Systematic Review","authors":"Samir Agrawal,&nbsp;Anjaneya Bapat,&nbsp;Christopher P. Eades,&nbsp;Shreyans Gandhi","doi":"10.1002/jha2.1105","DOIUrl":"https://doi.org/10.1002/jha2.1105","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To examine the incidence of invasive fungal disease (IFD) in patients receiving targeted agents for acute myeloid leukaemia (AML).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Literature for this systematic review was identified through a PubMed search in April 2024, using AML, IFD and targeted therapy terms. The following filters were applied: published in the last 10 years and published in English.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The PubMed search yielded 54 results, of which 16 were deemed relevant for inclusion. Four additional references were identified through manual searches. The majority of publications focused on the incidence of IFD during treatment with targeted agents; the remainder focused on the efficacy of targeted treatments and reported IFD as an adverse event. Most publications were retrospective analyses. Prophylaxis use and agents differed across studies. In several studies, IFD incidence was above the 8% threshold identified for anti-mould prophylaxis. <i>Aspergillus</i> was the most commonly reported pathogen, and most IFD cases occurred in the lungs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>IFD is relatively common among patients with AML receiving targeted therapies, despite the use of prophylaxis. Prospective studies with detailed IFD reporting, together with large epidemiological studies, are required to better understand the risk factors for, and incidence and nature of IFD in this patient population.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.1105","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143595043","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":"Lisocabtagene Maraleucel for Richter's Transformation—A Case Series","authors":"Courtney J. Smith,&nbsp;Anmol Goyal,&nbsp;Brian R. Smith,&nbsp;Dasom Lee,&nbsp;Alexandria Jensen,&nbsp;Jonathan Alexander,&nbsp;Melody Smith,&nbsp;Matthew Frank,&nbsp;Saurabh Dahiya,&nbsp;David Miklos,&nbsp;Sushma Bharadwaj","doi":"10.1002/jha2.70011","DOIUrl":"https://doi.org/10.1002/jha2.70011","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Richter's transformation (RT) from chronic lymphocytic leukemia (CLL) to lymphoma carries poor prognosis. This case series examines the efficacy of lisocabtagene maraleucel (liso-cel) in six RT patients, highlighting the impact of concurrent ibrutinib therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Six patients were with RT who received liso-cel from were included in this single institution case series. Clinical data related to efficacy, safety, CAR-T expansion kinetics, and measurable residual disease were collected.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The best overall response was 83.3%. The four patients who received ibrutinib concurrent with liso-cel therapy continue to show MRD-negative complete response till date. None experienced severe (grade 3+) cytokine release syndrome while 1 had severe (grade 3+) immune-effector cell-associated neurotoxicity syndrome (ICANS). All patients were noted to have in vivo CAR expansion.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This series of real cases suggests liso-cel with concurrent ibrutinib may be a promising treatment for RT, warranting further exploration.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 2","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70011","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143581676","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}