EJHaem最新文献

{"title":"Red blood cell transfusion and the use of intravenous iron in iron-deficient patients presenting to the emergency department","authors":"Kevin Thomas,&nbsp;Arvand Barghi,&nbsp;Robert Balshaw,&nbsp;Emily Rimmer,&nbsp;Murdoch Leeies,&nbsp;Donald S. Houston,&nbsp;Allan Garland,&nbsp;Ryan Zarychanski,&nbsp;Brett L. Houston","doi":"10.1002/jha2.1061","DOIUrl":"10.1002/jha2.1061","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background and aims</h3>\u0000 \u0000 <p>Red blood cell transfusions are often used to treat iron-deficient patients in the emergency department (ED), while treatment with intravenous (IV) iron is preferred, as it increases hemoglobin concentration rapidly and durably. We aim to evaluate the incidence of iron deficiency anemia, frequency of blood transfusion and iron supplementation, and factors associated with blood transfusion in the ED.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a retrospective cohort study of adult patients presenting to the St. Boniface Hospital (Winnipeg, Canada) ED from 2014 to 2018. Electronic data obtained from the Emergency Department Information System and Laboratory Information Services databases identified patients presenting with iron deficiency anemia, defined as microcytic (mean corpuscular volume &lt; 75 fL) anemia (hemoglobin &lt; 120 g/L) with either a transferrin saturation &lt;20% or ferritin &lt; 30 µmol/L. Ferritin &gt; 100 µmol excluded iron deficiency anemia. The use of blood transfusions or iron supplementation was determined for each patient. Factors associated with blood transfusion were determined using logistic regression analyses.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 39,222 patients, 17,945 (45%) were anemic. In anemic patients, iron parameters were ordered in 1848 (10.3%) and iron deficiency anemia was diagnosed in 910 (5.1%). Ninety-five patients (10.4%) received one red blood cell unit, and 197 patients (21.6%) received ≥2 units. Oral iron and IV iron were prescribed for 64 (7.0%) and 14 (1.5%) patients, respectively. Hemoglobin concentration was the main determinant for treatment with blood transfusion.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Iron deficiency is underinvestigated among anemic patients presenting to the ED. The only clinical factor associated with red blood cell transfusion in the ED was hemoglobin level, irrespective of symptoms or clinical stability.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11756976/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143049018","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":"Increased serum B-cell maturation antigen levels evaluated with an Elecsys-based serum B-cell maturation antigen assay have a negative prognostic value in patients with newly diagnosed multiple myeloma","authors":"Evangelos Terpos,&nbsp;Ioannis Ntanasis-Stathopoulos,&nbsp;Panagiotis Malandrakis,&nbsp;Despina Fotiou,&nbsp;Magdalini Migkou,&nbsp;Evangelos Eleutherakis-Papaiakovou,&nbsp;Foteini Theodorakakou,&nbsp;Maria Roussou,&nbsp;Maria Gavriatopoulou,&nbsp;Meletios A Dimopoulos,&nbsp;Efstathios Kastritis","doi":"10.1002/jha2.889","DOIUrl":"10.1002/jha2.889","url":null,"abstract":"<p>Background: Serum B-cell maturation antigen (sBCMA) levels have emerged as a potential biomarker for disease monitoring in multiple myeloma (MM) with prognostic value.</p><p>Methods: Herein, we evaluated the sBCMA levels in 166 patients with newly diagnosed MM with an Elecsys-based sBCMA assay.</p><p>Results: Increased sBCMA levels at diagnosis were correlated with inferior survival outcomes in terms of both progression-free and overall survival. In a subset of patients with available samples at the time of disease progression, there was a trend for decreasing sBCMA values.</p><p>Conclusion: Sequential evaluation of sBCMA in prospective studies will determine the value of incorporating sBCMA measurement in clinical practice.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-12-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11756978/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143048883","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 characteristics and outcomes of patients with multiple myeloma receiving second-line treatment in England","authors":"Sally Moore,&nbsp;Laura Cornic,&nbsp;Christina-Jane Crossman-Barnes,&nbsp;Sophie Jose,&nbsp;Zeyad Khalaf,&nbsp;Kwee Yong,&nbsp;Megan Soutar,&nbsp;Philip Woods","doi":"10.1002/jha2.1058","DOIUrl":"10.1002/jha2.1058","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Despite recent advances in first-line therapies for multiple myeloma (MM), most patients relapse or become refractory, underscoring the need for effective second-line (2L) regimens for relapsed/refractory MM (RRMM).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This study describes the real-world baseline characteristics, treatment patterns and clinical outcomes of adult patients diagnosed with MM between 2013 and 2020 using data collated by the National Cancer Registration and Analysis Service (NCRAS) of the National Health Service in England. The study cohorts were broadly aligned to the eligibility criteria of the ongoing DREAMM-7 (D7) and DREAMM-8 (D8) clinical trials. We focus on lenalidomide-exposed/refractory patients who received daratumumab–bortezomib–dexamethasone (DaraVd) at 2L in both cohorts.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In the D7-like cohort, the lenalidomide-exposed (<i>n </i>= 282) and lenalidomide-refractory (<i>n </i>= 143) patients who received DaraVd at 2L had a median (95% confidence interval [CI]) time to next treatment or death (TTNTD) of 15.1 (12.6–22.4) and 10.3 (7.4–13.9) months, respectively. In the D8-like cohort, the lenalidomide-exposed (<i>n </i>= 269) and lenalidomide-refractory (<i>n </i>= 148) patients who received DaraVd at 2L had a median (95% CI) TTNTD of 14.5 (11.7–19.7) and 10.0 (7.3–13.7) months, respectively.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Patients with RRMM in England receiving DaraVd at 2L have poor clinical outcomes, highlighting the urgent need for new therapies, particularly for lenalidomide-refractory patients.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11756965/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143049014","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":"Terminal deoxynucleotidyl transferase-positive high-grade B-cell lymphoma with MYC and BCL2 rearrangements transformed from follicular lymphoma","authors":"Radu Chiriac,&nbsp;Lucile Baseggio,&nbsp;Marie Donzel","doi":"10.1002/jha2.1060","DOIUrl":"10.1002/jha2.1060","url":null,"abstract":"&lt;p&gt;A 48-year-old man with a 2-year history of classical follicular lymphoma (according to the 5th WHO classification) [&lt;span&gt;1&lt;/span&gt;] involving the axillary lymph nodes achieved complete remission after six cycles of obinutuzumab plus cyclophosphamide, doxorubicin, vincristine, and prednisone. By the end of the third cycle of maintenance therapy with obinutuzumab, the patient presented with epigastric and left hypochondrial pain while in complete remission for 8 months.&lt;/p&gt;&lt;p&gt;Laboratory studies revealed a lactate dehydrogenase level of 2000 U/L (reference range: 135–235 U/L) and mild anemia (90 g/L). However, the peripheral blood smear showed 10% atypical intermediate-sized lymphomatous cells, characterized by nuclei with oval to irregular contours, finely stippled chromatin, variable nucleoli, and intensely basophilic cytoplasm with numerous vacuoles (Figure 1A). Staging bone marrow was negative. Peripheral blood flow cytometry showed a kappa-restricted population of mature B-cells that were CD45+, CD19+, CD10+, CD5-, and CD20- (Figure 1B). To evaluate the abdominal pain, &lt;sup&gt;18&lt;/sup&gt;F-fluorodeoxyglucose (&lt;sup&gt;18&lt;/sup&gt;F-FDG) positron emission tomography/computed tomography revealed a left retroperitoneal solid mass with increased &lt;sup&gt;18&lt;/sup&gt;F-FDG uptake, an SUVmax of 20, and measuring 6 × 16 cm (Figure 1C).&lt;/p&gt;&lt;p&gt;Mass biopsy revealed a predominance of monomorphic medium-sized blastoid cells with scant basophilic cytoplasm, round nuclei, and conspicuous nucleoli. Mitotic figures were easily observed (Figure 1D). The neoplastic cells were positive for B-cell markers, including PAX5 and CD19, but negative for CD20. They exhibited a germinal center phenotype (CD10+, BCL6-, and MUM1+) and overexpressed both c-MYC and BCL2. Diffuse terminal deoxynucleotidyl transferase (TdT) expression and monotypic surface immunoglobulin light chain expression were also observed. CD5, CD34, P53, and Epstein–Barr virus-encoded RNA in situ hybridization were negative. The Ki-67 proliferation index was 70%. Fluorescence in-situ hybridization performed with break-apart probes on tissue samples showed &lt;i&gt;MYC&lt;/i&gt; (80%) and &lt;i&gt;BCL2&lt;/i&gt; (90%) rearrangements, with no &lt;i&gt;BCL6&lt;/i&gt; rearrangement (Figure 1D, insets).&lt;/p&gt;&lt;p&gt;Ifosfamide and etoposide-based chemotherapy were initiated, followed by anti-CD19 chimeric antigen receptor T-cell infusion, which was initially well-tolerated; however, a recurrence developed 3 months later. He was switched to dexamethasone, high-dose cytarabine, and oxaliplatin but developed tumor lysis syndrome with renal failure despite prophylaxis. He experienced progressive disease involving the kidney, lower retroperitoneum, extraperitoneal space, and testis, ultimately dying 7 months after diagnosis.&lt;/p&gt;&lt;p&gt;This case describes a complex example of an aggressive TdT-positive high-grade B-cell lymphoma (HGBCL), with &lt;i&gt;MYC&lt;/i&gt; and &lt;i&gt;BCL2&lt;/i&gt; rearrangements transformed from follicular lymphoma marked by significant tissue involvement and a concurrent l","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1351-1353"},"PeriodicalIF":0.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647685/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848238","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":"Low-dose nivolumab for extranodal natural killer/T-cell lymphoma, nasal type","authors":"Satish Maharaj,&nbsp;Simone Chang","doi":"10.1002/jha2.1059","DOIUrl":"10.1002/jha2.1059","url":null,"abstract":"&lt;p&gt;Natural killer (NK)/T-cell lymphoma is a rare subtype of non-Hodgkin lymphoma that is associated with poor outcomes. Efforts to improve treatment are urgently needed and immune checkpoint inhibitor therapies have shown promise in a series of relapsed/refractory diseases. A 59-year-old Hispanic male presented with near-total obstruction of his left nasal airway. Computed tomography imaging showed left greater than right sinusitis with near-total airway obstruction on the left side. Magnetic resonance imaging (MRI) and positron emission tomography (PET) scan showed a hypermetabolic primary mass in the left nasal cavity measuring 3.5 cm in length by 1.7 cm in width, SUV max 10.99. Nasal endoscopy showed a highly vascular exophytic tumor filling the nasal airway and sinuses; debulking and surgical biopsy were performed.&lt;/p&gt;&lt;p&gt;Pathology from this showed extranodal NK/T-cell lymphoma, nasal-type (ENKTL). Tumor cells were positive for LCA, CD2, CD3 and CD56 immunostains; negative for CD5, CD4, CD8, CD10, CD20, MUM1, CD21, BCL2, BCL6, CD79A, Cyclin D1, CD30, ALK1, Pan-keratin, HV8, CD57, and c-Myc immunostains. Karyotyping was normal, 46, XY[20], and bone marrow biopsy showed normocellular bone marrow without involvement and active maturing trilinear hematopoiesis. Flow cytometry analysis of the nasal mass demonstrated relatively increased natural killer cells, 66% of lymphocytes CD3-/CD56+ with normal expression of CD16 and no immunophenotypic aberrancies without diminished or loss of CD2, CD7, and/or CD57, or abnormal uniform expression of CD8. Epstein-Barr virus (EBV) was positive by RNA in situ hybridization (EBER Positive). Peripheral blood also was positive with EBV DNA Quantitative polymerase chain reaction level 705 copies/mL.&lt;/p&gt;&lt;p&gt;Using these evaluations, the patient was determined to have Stage II disease using the Chinese Southwest Oncology Group and Asia Lymphoma Study Group (CA) staging system (CASS) established in 2020 [&lt;span&gt;1&lt;/span&gt;]. CASS Stage II is defined as a primary tumor localized to the nasal cavity or nasopharynx involving local structures, without regional lymph node involvement. Research has shown that the Ann Arbor staging system (AASS), established for Hodgkin Lymphoma, has limited predictive ability and poor prognostication for ENKTL with CASS better in discriminating survival than AASS [&lt;span&gt;2, 3&lt;/span&gt;]. Unlike most other lymphomas, ENKTL is primarily extranodal, and therefore using the AASS, the majority of ENKTL (70%–90%) appears early (stage I/II) leading to unbalanced distribution and poor predictive accuracy.&lt;/p&gt;&lt;p&gt;Another model developed for patients treated with non-anthracycline-containing regimens is the prognostic index for NK/T-cell lymphomas (PINK-E) (negative scoring parameters: age  &gt;60 years, stage III/IV disease, distant lymph node involvement, non-nasal subtype, and detectable presentation EBV DNA) [&lt;span&gt;4&lt;/span&gt;]. Using PINK-E, this patient would be classified as low-risk. Using the PINK-E model,","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1363-1365"},"PeriodicalIF":0.0,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647695/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848601","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":"Enteropathy-associated T-cell lymphoma: A population-based cohort study on incidence, treatment, and outcome in the Netherlands","authors":"Frederik O. Meeuwes,&nbsp;Mirian Brink,&nbsp;Wouter J. Plattel,&nbsp;Joost S. P. Vermaat,&nbsp;Marie José Kersten,&nbsp;Mariëlle Wondergem,&nbsp;Otto Visser,&nbsp;Marjolein W. M. van der Poel,&nbsp;Rimke Oostvogels,&nbsp;F. J. Sherida H. Woei-A-Jin,&nbsp;Lara Böhmer,&nbsp;Tjeerd J. F. Snijders,&nbsp;Gerwin A. Huls,&nbsp;Marcel Nijland","doi":"10.1002/jha2.1049","DOIUrl":"10.1002/jha2.1049","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Enteropathy-associated T-cell lymphoma (EATL) is a peripheral T-cell lymphoma (PTCL) with a poor prognosis. Cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) with or without etoposide consolidated by autologous stem cell transplantation (ASCT) are recommended for fit PTCL patients. The role of etoposide and ASCT in EATL is unclear.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This study reports the incidence, treatment, and outcome of EATL patients using the Netherlands Cancer Registry, with nationwide coverage of &gt;95%.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>All patients diagnosed in 1989–2021 (<i>n</i> = 351, 77% treated) were identified (median age 67 years, 56% male, 50% limited stage). Time period analysis assessed trends in primary therapy and overall survival (OS). Treatment included chemotherapy (CT) (34%), surgery (18%), surgery and CT (19%) or CT followed by ASCT (7%). The 5-year OS for treated patients with limited versus advanced stage was 19% and 9% respectively. The 2-year OS improved over time (21%–33%, <i>p = </i>0.06). Surgery only (hazard ratio [HR] 2.16; 95% confidence interval [CI] 1.55–3.01, <i>p &lt;</i> 0.01) and advanced-stage disease (HR 1.67; 95% CI 1.25–2.23, <i>p =</i> 0.01) were predictors of poor prognosis. ASCT (HR 0.31; 95% CI 0.18–0.56) was associated with improved OS.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>There was no statistical difference in OS between patients treated with or without etoposide. Current first-line treatment is ineffective.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1215-1222"},"PeriodicalIF":0.0,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647706/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848557","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":"IFN-α treatment may enable discontinuation of TKIs in NK cell-licensed patients with CML-CP","authors":"Hiroshi Ureshino,&nbsp;Kazuharu Kamachi,&nbsp;Keisuke Kidoguchi,&nbsp;Shinya Kimura","doi":"10.1002/jha2.1053","DOIUrl":"10.1002/jha2.1053","url":null,"abstract":"<p>The magnitude of the natural killer (NK) cell response contributes to the achievement of treatment-free remission (TFR) in patients with chronic myeloid leukemia (CML) and is regulated by the interaction between killer immunoglobulin-like receptors (KIRs) on NK cells and human leukocyte antigen (HLA) class I molecules on target cells. The abundant combination between <i>KIR</i> and <i>HLA</i> through genetic polymorphisms determines the functional diversity of NK cells. We previously reported that <i>KIR3DL1-HLA-Bw</i> status is associated with achievement of TFR by reflecting NK cell potential. Patients with strong interaction between <i>KIR3DL1/HLA-Bw</i> were identified as having a higher molecular relapse risk, based on the “missing self” hypothesis which suggests that the lack of cognate ligands for KIRs may induce target cell lysis. However, all the patients with strong interaction between <i>KIR3DL1/HLA-Bw</i> who received prior IFN-α therapy achieved TFR (<i>p</i> = 0.007), explained by the “NK cell licensing” concept, whereby NK cells become more functional through the recognition “self” HLA class I molecules by KIRs. NK cell licensing may contribute to the potential efficacy of IFN-α treatment in patients with CML. We defined high-risk molecular relapse patients and suggest that <i>KIR3DL1/HLA-Bw</i> status may help detect patients who could benefit from IFN-α for maintaining TFR.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1278-1282"},"PeriodicalIF":0.0,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647684/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848576","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":"Incidence and risk factors of graft failure in allogeneic hematopoietic stem cell transplantation for mucopolysaccharidosis in a nationwide pediatric cohort. A study on behalf of the Francophone Society of Bone Marrow Transplantation and Cellular Therapy","authors":"Laura Danhardt,&nbsp;Arnaud Wiedemann,&nbsp;Gerard Michel,&nbsp;Jean-Hugues Dalle,&nbsp;Fanny Rialland,&nbsp;Cécile Renard,&nbsp;Charlotte Jubert,&nbsp;Johan Maertens,&nbsp;Anne Sirvent,&nbsp;Nimrod Buchbinder,&nbsp;Christine Devalck,&nbsp;Bénédicte Brichard,&nbsp;Catherine Paillard,&nbsp;Stephanie Nguyen,&nbsp;Angelo Paci,&nbsp;David Combarel,&nbsp;Martin Castelle,&nbsp;Simona Pagliuca,&nbsp;Cecile Pochon","doi":"10.1002/jha2.1056","DOIUrl":"10.1002/jha2.1056","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Context</h3>\u0000 \u0000 <p>Mucopolysaccharidosis (MPS) requires urgent treatment to prevent neurological damage. While gene therapy holds promise for effectively treating these diseases with minimal toxicity, access remains limited for most patients. Consequently, advancing allogeneic hematopoietic stem cell transplantation (HSCT) for young children is crucial. Since the 2010s, cord blood (CB) transplants with reduced-toxicity conditioning (RTC) have become the standard of care.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and methods</h3>\u0000 \u0000 <p>Recent reports in France indicate a significant incidence of graft failures (GF), prompting a large-scale retrospective study from the French-speaking bone marrow transplantation society's registry, to understand GF risks, guide clinicians in selecting transplant platforms, and describe outcomes of second HSCT in young patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>This report analyses 93 children who underwent HSCT for MPS between 2000 and 2020. The GF rate was notably high (22.6% at day 100), primarily associated with the donor's HLA compatibility and the recipient's age. Well-matched CB and RTC were not found to be risk factors for GF. This study also details the procedures for second and third transplants in patients who rejected their first HSCT.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>In the era of RTC, CB remains a viable and expedient option for MPS transplantation.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1295-1300"},"PeriodicalIF":0.0,"publicationDate":"2024-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647734/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848595","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 practitioner perceptions of CARTITUDE-4 results for patients with previously treated multiple myeloma","authors":"Alexandrina Balanean,&nbsp;Samuel Baird,&nbsp;Brooke Dulka,&nbsp;Luke Jennings-Zhang,&nbsp;Robert N. Bone,&nbsp;Yolaine Jeune-Smith,&nbsp;Bruce Feinberg,&nbsp;Muhamed Baljevic","doi":"10.1002/jha2.1047","DOIUrl":"10.1002/jha2.1047","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Initially approved for the fifth-line or later therapeutic setting, the chimeric antigen receptor (CAR) T-cell regimen ciltacabtagene autoleucel (cilta-cel) was recently approved for second-line (2L) treatment in relapsed/refractory multiple myeloma (RRMM). Oncology practitioners use clinical trials to inform treatment, but real-world impressions and impact on practice are lacking. We aimed to determine whether presenting CARTITUDE-4 clinical trial data would impact real-world preferences/perceptions around CAR T-cell therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Recruiting from the Cardinal Health Oncology Provider Extended Network (OPEN), we surveyed hematologists/oncologists to investigate fourth-line (4L) preferences in a hypothetical patient with triple-class–refractory MM. We posed the same questions and answers before and after the trial presentation and compared pre-/post-preferences toward cilta-cel and sequencing relative to bispecific antibodies (BsAbs). Using the same methodology as described above, we also performed a secondary analysis comparing pre-/post-perceptions on the use of CAR T-cell therapy in earlier lines for patients with triple-class–refractory MM.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among 50 respondents, decision-making factors before the trial presentation included CAR T-cell center availability (58%), comorbidities (52%), and center locations (34%). Additionally, 48% of 46 respondents chose 4L cilta-cel. Among 47, 40% wanted more real-world/long-term CAR T-cell therapy outcomes in any line, 38% wanted more 2L data, and 34% favored 2L/third-line (3L) use. After the presentation, the preference for cilta-cel doubled from 48% to 88% (<i>p </i>&lt; 0.001) among 50 respondents and rose from 34% to 55% (<i>p </i>= 0.001) for earlier-line CAR T-cell therapy among 49. Moreover, 55% of 49 respondents preferred CAR T-cell therapy prior to BsAbs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Discussion</h3>\u0000 \u0000 <p>We have shown that making oncology practitioners aware of trials precipitated decision-making factors and led to notable, significant shifts in future intended practice patterns. Being aware of trial data enables practitioners to make more informed decisions, tailor therapies to individual patients, and ultimately improve outcomes.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1154-1164"},"PeriodicalIF":0.0,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647711/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848079","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":"Successful treatment of low-risk myelodysplastic syndrome-related anemia in patients with chronic kidney disease with daprodustat: A report of two cases","authors":"Hiroyoshi Kunimoto,&nbsp;Takayuki Sakuma,&nbsp;Takuma Ohashi,&nbsp;Mayoko Shirafuta,&nbsp;Hiroshi Teranaka,&nbsp;Hideaki Nakajima","doi":"10.1002/jha2.1057","DOIUrl":"10.1002/jha2.1057","url":null,"abstract":"<p>Anemia is a major clinical manifestation seen in myelodysplastic syndromes (MDS). Treatment options for anemia in low-risk MDS are limited. Especially, oral medication which is uniformly effective for anemia in low-risk MDS is required. Hypoxia-inducible factor (HIF) prolyl hydroxylase (HIF-PH) inhibitors, such as daprodustat, are oral tablets effective for renal anemia. Pharmacological restoration of HIF activity by HIF-PH inhibitors may be beneficial for MDS-related anemia as well. Yet, their efficacy and safety against low-risk MDS are unclear. Here, we report two cases of low-risk MDS complicated with chronic kidney disease whose anemia responded to daprodustat treatment.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"5 6","pages":"1335-1339"},"PeriodicalIF":0.0,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11647689/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142848233","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}