European Journal of Haematology最新文献

{"title":"Do we Need to Perform Bone Marrow Examination in all Subjects Suspected of MDS? Evaluation and Validation of Non-Invasive (Web-Based) Diagnostic Algorithm","authors":"Howard S. Oster,&nbsp;Ariel M. Polakow,&nbsp;Roi Gat,&nbsp;Noa Goldschmidt,&nbsp;Jonathan Ben-Ezra,&nbsp;Moshe Mittelman","doi":"10.1111/ejh.14379","DOIUrl":"10.1111/ejh.14379","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Bone marrow examination (BME) is the gold standard of diagnosing myelodysplastic syndromes (MDS). Problems: it is invasive, painful, causing possible bleeding, inaccurate (aspirate hemodilution), and subjective (inter-observer interpretation discordance). We developed non-invasive diagnostic tools: A logistic regression formula [LeukRes 2018], then a web algorithm using 10 variables (age, gender, Hb, MCV, WBC, ANC, monocytes, PLT, glucose, creatinine) to diagnose/exclude MDS [BldAdv 2021]. Here, we perform external validation of the model.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>From the TASMC BM registry (2019–22) we identified and compared the model performance between MDS patients and controls (&gt; 50 year with unexplained anemia, not MDS), all BME diagnosed, and not used in model building.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The model was accurate and predicted MDS in 63% of 103 patients, and excluded (correctly) in 83% of 101 controls. It miss-classified in 11%/7% respectively, and was indeterminate in 26%/10% respectively. The positive predictive value (PPV), NPV, sensitivity, and specificity (excluding the indeterminate group) were 90%, 88%, 86%, and 92%, respectively. Subgroup (Lower/higher risk, LR/HR) analysis results were similar.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The MDS diagnostic model was validated and can be used, mainly for MDS exclusion, especially in suspected LR-MDS, avoiding BME in some patients. In the future incorporating peripheral blood genetics and morphometry can further improve the model.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"672-678"},"PeriodicalIF":2.3,"publicationDate":"2025-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142925981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Mortality of Adults With Sickle Cell Disease at a Comprehensive Sickle Cell Center","authors":"Jennifer A. Afranie-Sakyi,&nbsp;Eldrida Randall,&nbsp;Ross Fasano,&nbsp;Morgan L. McLemore,&nbsp;Fuad El Rassi","doi":"10.1111/ejh.14360","DOIUrl":"10.1111/ejh.14360","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Sickle cell disease (SCD) is the most common hemoglobinopathy in North America. The life expectancy of SCD has extended into adulthood with screenings, preventative care, and hydroxyurea. However, comorbidities arise as adults with SCD age, leading to early mortality.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a retrospective chart review of the Georgia Comprehensive Sickle Cell Center at Grady Health System, analyzing records of deceased SCD patients from 2013 to 2020.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Amongst the 72 patients analysed, majority had severe complications from SCD and at least 1 cardiovascular comorbidity. The median age of death was 44 (STD = 15.5) for all genotypes with the median age of death at 39 (STD = 14.26) for SS and Sβ0 genotypes (<i>n</i> = 51). There was no difference in the median age of death for patients who maintained regular clinic visits (a visit in the last 6 months prior to death) compared to those who did not. Despite hydroxyurea's known benefits in reducing SCD morbidity and mortality, less than 50% of patients had a prescription.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>As new therapies are approved, their impact on SCD-related morbidity and mortality must be evaluated. Improving access to, and education about, disease-modifying therapies like hydroxyurea for both patients and clinicians is essential to improving outcomes.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"663-671"},"PeriodicalIF":2.3,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142920820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimized GVHD Prevention in HLA-Mismatched Unrelated Allogeneic HCT Using a PTCY-Based Approach","authors":"Filipe R. Pinto,&nbsp;María Suárez-Lledó,&nbsp;Laia Guardia,&nbsp;Paola Charry,&nbsp;Joan Cid,&nbsp;Miquel Lozano,&nbsp;Alexandra Pedraza,&nbsp;Noemi de Llobet,&nbsp;Gerard Corrius,&nbsp;Cristina Moreno,&nbsp;Jordi Esteve,&nbsp;Carles Serra,&nbsp;Enric Carreras,&nbsp;Laura Rosiñol,&nbsp;Francesc Fernández-Avilés,&nbsp;Montserrat Rovira,&nbsp;Carmen Martinez,&nbsp;María Queralt Salas","doi":"10.1111/ejh.14378","DOIUrl":"10.1111/ejh.14378","url":null,"abstract":"<div>\u0000 \u0000 <p>Although post-transplant cyclophosphamide (PTCY)-based prophylaxis has become a widely adopted strategy for preventing graft-versus-host disease (GVHD) in 9 out of 10 HLA-mismatched unrelated donors (MMUDs), allogeneic hematopoietic cell transplants (allo-HCTs), data on the safety and efficacy of PTCY in this setting remain limited. This single-center study investigates the outcomes of 94 adults with hematological malignancies undergoing MMUD allo-HCT with PTCY and tacrolimus (Tac) (PTCY-Tac) between 2014 and 2023. The median age was 53 years, and 60.6% were male. Peripheral blood stem cells were infused in all cases. By Day +100, the cumulative incidence of Grades II–IV and Grades III and IV acute GVHD were 33.0% and 9.7%, with 2-year incidence of moderate-to-severe chronic GVHD at 12.6%. By Day +30, 40.8% of patients experienced bacterial bloodstream infections, and 52.4% had cytomegalovirus (CMV) reactivation before letermovir prophylaxis. With letermovir's introduction, CMV reactivation rates dropped significantly, with only one case reported. At 3 years, overall survival was 60.8%, non-relapse mortality was 23%, and the cumulative incidence of relapse was 24.5%. HLA Class I or II mismatches did not affect key outcomes or GVHD rates. These findings demonstrate that PTCY-Tac offers effective GVHD prevention and favorable outcomes in MMUD allo-HCT, supporting its application for patients without fully matched donors.</p>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"650-662"},"PeriodicalIF":2.3,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909407","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Post-Relapse Outcomes of Older Patients With NPM1-Mutated AML Are Favorable With Allo Transplant in Second Remission","authors":"Avraham Frisch,&nbsp;Chezi Ganzel,&nbsp;Yishai Ofran,&nbsp;Baher Krayem,&nbsp;Arnon Haran,&nbsp;Vladimir Vainstein,&nbsp;Shlomzion Aumann,&nbsp;Noa Gross Even-Zohar,&nbsp;Boaz Nachmias","doi":"10.1111/ejh.14375","DOIUrl":"10.1111/ejh.14375","url":null,"abstract":"<p>Molecular assessment of measurable residual disease (MRD) in <i>NPM1</i>-mutated AML patients is a powerful prognostic tool to identify the risk of relapse. There is limited data regarding MRD-guided decisions against alloSCT in elderly patients and <i>FLT3</i>–ITD co-mutation. We describe the outcome of <i>NPM1</i>-mutated AML patients in whom alloSCT was deferred based on ELN 2017 risk and MRD response. We report a relapse rate of 53% in this group, with a much higher incidence for older than 60 years patients than for younger patients (73% vs. 37%). When comparing outcomes of alloSCT in CR1 to intensive chemotherapy consolidation within each age group, patients over 60 years and patients with FLT3–ITD co-mutation had significantly lower RFS with intensive consolidation. Yet, in all subgroups, the lower RFS did not translate into OS difference, suggesting that relapsed NPM1 patients can often be salvaged and consequently achieve long-term remission. Our study supports the use of MRD response along with <i>FLT3</i>–ITD status in the decision to use post-remission therapy. We demonstrate that older patients and patients with <i>FLT3</i>–ITD-mutated AML have a high relapse rate but can be salvaged, leading to long-term survival.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"641-649"},"PeriodicalIF":2.3,"publicationDate":"2024-12-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14375","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909424","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Underrepresentation of Small Lymphocytic Lymphoma in Clinical Trials for Chronic Lymphocytic Leukemia","authors":"Robert Puckrin,&nbsp;Carolyn Owen,&nbsp;Anthea Peters","doi":"10.1111/ejh.14376","DOIUrl":"10.1111/ejh.14376","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Although chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) are the same biologic disease entity and warrant identical treatment approaches, patients with SLL have frequently been excluded from clinical trials in CLL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This study assessed the representation of patients with SLL among Phase II or III clinical trials cited in the 2024 National Comprehensive Cancer Network (NCCN) treatment guidelines.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patients with SLL were explicitly eligible for only 21 (38%) of the 56 clinical trials for CLL, comprising 222 (6%) of the 3440 enrolled patients. Notably, 380 patients with SLL were enrolled in 16 separate non-CLL clinical trials alongside patients with indolent B-cell lymphomas such as follicular lymphoma. In CLL trials, patients with SLL were included in a greater proportion of studies evaluating BTK inhibitors (67%) or BTK/BCL2 inhibitor combinations (67%) compared to BCL2 inhibitors (0%) or chemoimmunotherapy (0%).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Although recent and upcoming trials show a promising trend toward the inclusion of patients with SLL, further advocacy is needed to raise awareness of the biological similarities between CLL and SLL and to promote the representation of patients with SLL in CLL/SLL clinical research.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"636-640"},"PeriodicalIF":2.3,"publicationDate":"2024-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14376","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893120","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Tumor Lysis Syndrome in Acute Myeloid Leukemia Patients Treated With a Venetoclax Based Regimen","authors":"Margaret Rowe,&nbsp;Daria Babushok,&nbsp;Martin Carroll,&nbsp;Alison Carulli,&nbsp;Noelle Frey,&nbsp;Saar Gill,&nbsp;Elizabeth Hexner,&nbsp;Rebecca Hirsh,&nbsp;Nasheed Hossain,&nbsp;Catherine Lai,&nbsp;Alison Loren,&nbsp;Selina Luger,&nbsp;Ivan Maillard,&nbsp;Shannon McCurdy,&nbsp;Andrew Matthews,&nbsp;Mary Ellen Martin,&nbsp;Vikram R Paralkar,&nbsp;Alexander Perl,&nbsp;David Porter,&nbsp;Keith Pratz,&nbsp;Edward Stadtmauer,&nbsp;Ximena Jordan Bruno","doi":"10.1111/ejh.14371","DOIUrl":"10.1111/ejh.14371","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <p>Venetoclax with hypomethylating agents (HMA) is the standard of care for acute myeloid leukemia (AML) in patients ineligible for intensive chemotherapy and is associated with tumor lysis syndrome (TLS). TLS prophylaxis and the use of Cairo Bishop versus Howard diagnostic criteria are not standardized. Here we report TLS prophylaxis and incidence in a retrospective cohort of 100 consecutive AML patients treated with venetoclax and HMA. Thirty four patients developed laboratory Cairo Bishop TLS; 8 of these met criteria for clinical Cairo Bishop TLS. Only 6 of patients met Howard TLS criteria. Fourteen patients had spontaneous TLS. Ninety two out of 100 patients had a white blood cell count (WBC) &lt; 25 000 cells/μL at treatment start. Prophylaxis like the original venetoclax trial with allopurinol (56%), intravenous fluids (21%), and frequent lab monitoring (56%) was less common. There was a trend toward increased Cairo Bishop TLS in patients with WBC ≥ 15 000 cells/μL. In our study Howard TLS criteria better identified patients with significant TLS. Aggressive TLS prophylaxis was uncommon in our cohort and is likely unnecessary for most patients at low risk of TLS.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"626-635"},"PeriodicalIF":2.3,"publicationDate":"2024-12-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14371","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparison of HiDAC Versus FLAG-IDA in the Treatment of Relapsed Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome","authors":"Man Wai Tang,&nbsp;Deborah Van der Tuin,&nbsp;Mesire Aydin,&nbsp;Jarom Heijmans,&nbsp;Arjan A. Van de Loosdrecht,&nbsp;Ellen Meijer,&nbsp;Caroline E. Rutten,&nbsp;Mariëlle Wondergem,&nbsp;Jeroen J. W. M. Janssen,&nbsp;Marjolein L. Donker,&nbsp;Mette D. Hazenberg,&nbsp;Sonja Zweegman,&nbsp;Bart J. Biemond,&nbsp;David C. De Leeuw,&nbsp;Erfan Nur","doi":"10.1111/ejh.14370","DOIUrl":"10.1111/ejh.14370","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Relapsed acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (HR-MDS) are associated with a poor prognosis. It is unknown which re-induction therapy provides the highest chance of durable remission. Commonly used therapies are high dose cytarabine (HiDAC) and triple therapy consisting of fludarabine, cytarabine, and idarubicin combined with granulocyte colony-stimulating factor (FLAG-IDA).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Two patient cohorts with relapsed AML or HR-MDS treated with HiDAC or FLAG-IDA between October 2015 and December 2021 in two academic hospitals in the Netherlands were retrospectively analyzed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patients were treated with either HiDAC (n=22) or FLAG-IDA (n=25). Rates of CR (71% vs. 74%, P=0.85), 1-year OS (47% vs. 51%, P=0.99) and EFS (38% vs. 35%, P=0.71) were comparable between HiDAC and FLAG-IDA. Durations of neutropenia (median 24 days (IQR 20-26) vs. 30 days (IQR 22-39), P=0.014) and thrombocytopenia (22 days (IQR 17-26) vs. 36 days (IQR 26-53)) were significantly shorter in the HiDAC group than in the FLAG-IDA group.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>While remission rates and survival outcomes were similar, FLAG-IDA was associated with longer periods of myelosuppression and transfusion dependency compared to HiDAC in these two cohorts. HiDAC can be considered as a salvage chemotherapyfor relapsed AML/HR-MDS based on our study.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"620-625"},"PeriodicalIF":2.3,"publicationDate":"2024-12-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14370","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893111","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing Disability in Thalassaemia: A Position Statement by the Thalassaemia International Federation","authors":"Dimitrios Farmakis,&nbsp;Michael Angastiniotis,&nbsp;Georgios Papingiotis,&nbsp;Lily Cannon,&nbsp;Androulla Eleftheriou","doi":"10.1111/ejh.14367","DOIUrl":"10.1111/ejh.14367","url":null,"abstract":"<p>Thalassemia is not currently conceived per se as a disability, but it can be a disability-inducing condition if poorly treated or as complications increase with age. People living with thalassemia do not wish, on the one hand, to be considered disabled persons to avoid stigma and loss of opportunities to achieve social inclusion in all paths of life while, on the other, they are in need of lifelong appropriate, disease-specific health and social care, including disability allowances and schemes, in order to be able to smoothly integrate into society and achieve professional, educational, personal, and social goals. The ongoing debate on whether thalassemia is a disability or not is thus complex and inconclusive and has created a vast heterogeneity of policies and approaches across the globe. Given that the risk to develop disabilities is subject to individualised assessment, the thalassemia International Federation (TIF) proposes a specific disability risk assessment model for thalassaemia (DRAM-Thal), based on the findings of a targeted literature review and of the TIF survey 2022–2023. This model considers both clinical features and social parameters and is addressed to national healthcare and social services and all other relevant stakeholders. At the same time, this work prompts further research on this understudied topic that heavily affects the rights and daily life of people living with the disease.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"411-422"},"PeriodicalIF":2.3,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14367","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142876305","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Healthcare Utilization Patterns in Sickle Cell Patients and Their Association With Sickle Cell Retinopathy","authors":"Caterina P. Minniti,&nbsp;Pamela D. Suman,&nbsp;Kevin Dahlan,&nbsp;Andrew Crouch,&nbsp;Michelle Goodman,&nbsp;Umar K. Mian","doi":"10.1111/ejh.14369","DOIUrl":"10.1111/ejh.14369","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background/Aim</h3>\u0000 \u0000 <p>A few shave identified systemic and hematologic risk factors for Proliferative Sickle Cell Retinopathy (PSR) development. The relevance of healthcare utilization as a risk factor for PSR has not been defined. This study evaluates patterns of healthcare utilization among patients with sickle cell disease (SCD) and retinopathy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A retrospective study of adults with SCD, using EMR, was conducted from January 2017 to December 2019, seen at the Montefiore Medical Center eye clinic.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Four hundred twelve patients with SCD were included in this analysis (65.8% HbSS (SS)) and 34.2% HbSC (SC). HBSS patients had higher utilization of hematology outpatient visits and inpatient admissions than HBSC. For individuals with either HBSC or HBSS disease, higher outpatient healthcare utilization was associated with higher number of inpatient admissions. The prevalence of retinopathy was 24% and 55% in SS and SC patients respectively, with PSR in 60% SS and 87.6% SC of these patients. Patients with HBSC with higher outpatient visits and inpatient admissions experienced significantly lower PSR rates. In contrast, for patients with HBSS, higher outpatient visits were significantly associated with higher PSR prevalence.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Healthcare utilization patterns in individuals with HBSS and HBSC varied according to their prevalence of PSR. These differences may be useful in stratifying patients' risk for retinopathy development and in deciding follow-up eye examination frequency.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 4","pages":"615-619"},"PeriodicalIF":2.3,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142876369","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"TP53 Mutation Is the Only Robust Mutational Biomarker for Outcome Found in a Consecutive Clinical Cohort of Real-Word Patients With Primary Large B-Cell Lymphoma","authors":"Marie Fredslund Breinholt,&nbsp;Lone Schejbel,&nbsp;Anne Ortved Gang,&nbsp;Ib Jarle Christensen,&nbsp;Torsten Holm Nielsen,&nbsp;Lars Møller Pedersen,&nbsp;Estrid Høgdall,&nbsp;Peter Nørgaard","doi":"10.1111/ejh.14364","DOIUrl":"10.1111/ejh.14364","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Large B-cell lymphoma (LBCL) taxonomy has moved in the direction of a molecular classification, but further clinical experience is needed. We present high-risk gene mutations, which predict outcome in an exploratory study of a consecutive real-world cohort of patients with primary LBCL treated with R-CHOP or R-CHOP-like therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The study was a Registry Study Research Project. Sixty-one patients with LBCL, who had a diagnostic tumor sample successfully examined with a 59-gene next-generation sequencing (NGS) panel as a part of routine clinical work, were included in an otherwise unselected cohort. Data were extracted from patient files and pathology reports.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Mutations in <i>NOTCH2</i> (HR 9.69; 95% CI [2.46–38.11]; <i>p</i> = 0.0012), <i>PRDM1</i> (HR 3.54; 95% CI [1.03–12.22]; <i>p</i> = 0.045), and <i>TP53</i> (HR 5.89; 95% CI [1.71–20.32]; <i>p</i> = 0.005) were significantly associated with inferior survival in patients with primary LBCL treated with intention to cure with at least three series of R-CHOP or R-CHOP-like therapy. Neither <i>MYD88</i> (HR 0.66; 95% CI (0.17–2.49), <i>p</i> = 0.54) nor <i>CD79B</i> (HR 0.84;95% CI (0.18–3.88), <i>p</i> = 0.82) mutations were associated with inferior survival.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>With a targeted gene panel and NGS methodology feasible in daily diagnostic routine, we identified high-risk gene mutations with a significant prognostic impact of which <i>TP53</i> mutations were reproducible across validation cohorts.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 3","pages":"573-579"},"PeriodicalIF":2.3,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142846101","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}