{"title":"Dapagliflozin in Patients with Homozygous β-Thalassemia and Albuminuria.","authors":"Konstantinos Tsiotsios, Sotirios Spyridon Vamvakas, Vasiliki Labropoulou, Vasileios Lazaris, Panagiota Davoulou, Dimitra Kalavrizioti, Adamantia Bratsiakou, Themistoklis Paraskevas, Alexandra Kourakli, Dimitrios Goumenos, Evangelos Papachristou, Argiris Symeonidis, Marios Papasotiriou","doi":"10.1159/000552285","DOIUrl":"10.1159/000552285","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with homozygous β-thalassemia show continuously improved survival, which has contributed to the development of kidney complications, predominantly expressed as increased albuminuria. In particular, patients with transfusion-dependent thalassemia frequently develop chronic kidney complications related to chronic anemia, iron overload, and exposure to iron chelation therapy. Although sodium-glucose co-transporter 2 inhibitors have demonstrated renoprotective effects in patients with chronic kidney disease (CKD) from other causes, their efficacy in patients with homozygous β-thalassemia and established CKD remains unknown.</p><p><strong>Methods: </strong>In this prospective, open-label study, we investigated the impact of dapagliflozin (10 mg/day), in patients with homozygous β-thalassemia and an albumin-to-creatinine ratio (ACR) >30 mg/g. Study end-points included the effect on ACR and kidney function at 6 months after treatment initiation. Furthermore, we examined the effect on systolic blood pressure (SBP) and diastolic blood pressure, and on urine monocyte chemoattractant protein-1 (MCP-1) and matrix metalloproteinase-9 (MMP-9) levels.</p><p><strong>Results: </strong>Twelve patients with ACR >30 mg/g received dapagliflozin, and 18 patients with normal ACR were followed up for 6 months. Twenty-three healthy adult individuals age- and sex-matched to the β-thalassemia group were used as normal controls. Patients with ACR >30 mg/g showed a significant reduction of ACR and SBP at 6 months (p = 0.034, p = 0.025, respectively), while kidney function remained stable. Patients with homozygous β-thalassemia showed increased baseline urine MCP-1 levels, while MMP-9 showed no differences versus normal controls. Those who received dapagliflozin exhibited a significant decrease in urine MCP-1 (p = 0.022), while MMP-9 remained stable. There were no significant adverse events.</p><p><strong>Conclusion: </strong>Dapagliflozin at a standard dose of 10 mg daily is safe and effectively reduces albuminuria and urinary MCP-1 in patients with homozygous β-thalassemia and established CKD, suggesting potential kidney anti-inflammatory effects. Further studies with larger cohorts and longer follow-up are needed to determine its impact on CKD progression and long-term outcomes.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":2.8,"publicationDate":"2026-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148337205","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Efficacy of Second-Generation FLT3 Inhibitors in FLT3-Mutated AML: A Meta-Analysis of Randomized Controlled Trials.","authors":"Qili Yu, Jingni Cao, Yixia Chen, Xiongtao Ma, Kangshuo Hu, Yixian Qiu","doi":"10.1159/000553180","DOIUrl":"10.1159/000553180","url":null,"abstract":"<p><strong>Introduction: </strong>Second-generation FLT3 inhibitors have demonstrated clinical efficacy in FLT3-mutated acute myeloid leukemia (AML), although results across randomized trials remain inconsistent. This meta-analysis aimed to evaluate the efficacy of second-generation FLT3 inhibitors in patients with FLT3-mutated AML.</p><p><strong>Methods: </strong>We systematically searched databases including PubMed, Web of Science, and the Cochrane Library (from inception to August 2025) to identify randomized controlled trials (RCTs) evaluating second-generation FLT3 inhibitors for treating FLT3-mutated AML. The primary endpoints were overall survival (OS) and progression-free survival (PFS). A random-effects model was used to calculate pooled hazard ratios (HRs) and their 95% confidence intervals (95% CIs). The study protocol was registered in PROSPERO (CRD420251132477).</p><p><strong>Results: </strong>This meta-analysis included eight RCTs involving 2,231 patients. The second-generation FLT3 inhibitors evaluated were gilteritinib and quizartinib, and control treatments included placebo or active non-FLT3-inhibitor regimens. Patients treated with second-generation FLT3 inhibitors exhibited significant improvements in both OS (HR: 0.72, 95% CI: 0.62-0.84; p < 0.001) and PFS (HR: 0.73, 95% CI: 0.63-0.86; p < 0.001). Subgroup analysis revealed that gilteritinib showed improvements in both OS (HR: 0.71, p = 0.001) and PFS (HR: 0.71, p < 0.001), and quizartinib significantly improved OS (HR: 0.72, p = 0.013) with no significant difference in PFS (HR: 0.75, p = 0.142). Among patients with newly diagnosed AML, second-generation FLT3 inhibitors did not significantly improve PFS (HR: 0.73, p = 0.127) but significantly improved OS (HR: 0.80, p = 0.037). Among patients with relapsed/refractory AML, significant benefits were observed in both OS (HR: 0.67, p < 0.001) and PFS (HR: 0.74, p = 0.04). Benefits were observed across both placebo-controlled and active-comparator trials.</p><p><strong>Conclusion: </strong>Second-generation FLT3 inhibitors significantly improve survival outcomes in FLT3-mutated AML, particularly for gilteritinib and in relapsed/refractory disease. Further studies are needed to clarify mutation subtype-specific and dose-specific effects.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":2.8,"publicationDate":"2026-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148282981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Edmond Chiche, Caroline Fileni, Sarah Bonnet, Sarah Bertoli, Andrew T Kuykendall, Lauris Gastaud, Michael Loschi, Geoffroy Venton, Véronique De Mas, Eric Delabesse, Alice Marceau-Renaut, Claude Preudhomme, Frederic Courtier, Anne Murati, Berengere Dadone-Montaudie, David Sallman, Christian Récher, Norbert Vey, Thomas Cluzeau
{"title":"Limited Prognostic Value of ELN Classification and Relevance of Molecular Ontogeny in Acute Myeloid Leukemia post Myeloproliferative Neoplasms: A Retrospective Multicenter Study.","authors":"Edmond Chiche, Caroline Fileni, Sarah Bonnet, Sarah Bertoli, Andrew T Kuykendall, Lauris Gastaud, Michael Loschi, Geoffroy Venton, Véronique De Mas, Eric Delabesse, Alice Marceau-Renaut, Claude Preudhomme, Frederic Courtier, Anne Murati, Berengere Dadone-Montaudie, David Sallman, Christian Récher, Norbert Vey, Thomas Cluzeau","doi":"10.1159/000552809","DOIUrl":"10.1159/000552809","url":null,"abstract":"<p><strong>Introduction: </strong>Acute myeloid leukemia (AML) post myeloproliferative neoplasm (MPN) have very poor prognosis and are often excluded from most clinical trials.</p><p><strong>Methods: </strong>We retrospectively collected data from 166 patients, including 156 with available treatment data and 83 with NGS data treated in France and USA.</p><p><strong>Results: </strong>2022 ELN risk categories were favorable, intermediate and adverse in 3 (2%), 17 (13%), and 110 (85%), respectively. Overall response rate was 57%, 20%, and 25% in patients treated by intensive chemotherapy (IC), hypomethylating agents (HMAs) and BSC (including low-intensity treatments as hydroxyurea and low-dose cytarabine), respectively. A total of 31 (22%) patients underwent allogeneic stem cell transplantation (ASCT). Median overall survival (OS) was 7.2 months without significant difference between IC and HMA (9.5 and 8.6 months, respectively). OS was significantly improved in patients allografted (6.7 vs. 1.3 months, respectively, p < 0.001). Even though 2017 and 2022 ELN risk categories were not prognostic for OS, we observed a prognostic impact on OS of Lindsley's classifier (p = 0.014). In multivariate analysis, JAK2 mutation was associated with worse OS (p = 0.037), whereas SRSF2 showed a trend toward adverse prognosis (p = 0.057). Among functional groups, only spliceosome mutations predicted poor prognosis (p = 0.015).</p><p><strong>Conclusion: </strong>We confirmed poor prognosis of AML post-MPN. Classical prognostic classification was not validated in our cohort. We observed poor outcome using IC or HMA encouraging us to propose new clinical trials in this specific subgroup. Only ASCT was able to improve prognosis.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":2.8,"publicationDate":"2026-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148256957","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Johanna Vikkula, Kari Remes, Outi Laatikainen, Merja Utriainen, Saara Tikka, Juha Lievonen
{"title":"Real-World Data on First Relapse in Multiple Myeloma: Treatment Patterns and Outcomes in Finland Using Automated Data Collection.","authors":"Johanna Vikkula, Kari Remes, Outi Laatikainen, Merja Utriainen, Saara Tikka, Juha Lievonen","doi":"10.1159/000552684","DOIUrl":"10.1159/000552684","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to characterize multiple myeloma (MM) patients at the first relapse and examine treatment outcomes.</p><p><strong>Methods: </strong>This registry-based retrospective study examined relapsed MM patients in two Finnish Wellbeing Services Counties (Helsinki and Uusimaa, and Southwest Finland) from 2013 to 2022. Relapse was identified per current guidelines using serum paraprotein, plasma immunoglobulins, and free light chain data. Data were collected from hospital data lakes, Social Insurance Institution and Digital and Population Services Agency. Patients were stratified by age (≤70 vs. >70), index year, and time to first relapse (≤2 vs. >2 years post-diagnosis).</p><p><strong>Results: </strong>Of 1,221 patients treated for MM between 2012 and 2022, 286 met the study inclusion criteria. Median overall survival from the relapse was higher in the younger patients (32.4 months vs. 20.0 months). In the first half of follow-up, lenalidomide was the most common pharmacotherapy in both age groups. After 2018, CD38-based treatments and pomalidomide increased significantly for younger patients, remaining limited in the elderly.</p><p><strong>Conclusion: </strong>While new therapies have been adopted for younger MM patients, the treatment of older patients at first relapse has had little change. Given their poorer outcomes, there is a need to improve care for older MM patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":2.8,"publicationDate":"2026-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13423317/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148238417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Maymona Abdelmagid, Naima J Hashi, Mehrdad Hefazi, Aasiya Matin, Estefania Gauto Mariotti, William J Hogan, Mark R Litzow, Hassan B Alkhateeb, Mithun V Shah, Urshila Durani, Saad S Kenderian, James M Foran, Hemant S Murthy, Mohamed A Kharfan-Dabaja, Vivek Roy, Ernesto Ayala, Mrinal M Patnaik, David Dingli, Margaret A DiGuardo, Eapen K Jacob, Abhishek A Mangaonkar
{"title":"Comparative Efficacy of Donor Lymphocyte Infusions in Augmenting Graft-versus-Leukemia Effect after Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Myeloid Malignancies.","authors":"Maymona Abdelmagid, Naima J Hashi, Mehrdad Hefazi, Aasiya Matin, Estefania Gauto Mariotti, William J Hogan, Mark R Litzow, Hassan B Alkhateeb, Mithun V Shah, Urshila Durani, Saad S Kenderian, James M Foran, Hemant S Murthy, Mohamed A Kharfan-Dabaja, Vivek Roy, Ernesto Ayala, Mrinal M Patnaik, David Dingli, Margaret A DiGuardo, Eapen K Jacob, Abhishek A Mangaonkar","doi":"10.1159/000552196","DOIUrl":"10.1159/000552196","url":null,"abstract":"<p><strong>Introduction: </strong>The efficacy of donor lymphocyte infusions (DLIs) among various myeloid malignancies (particularly genetic subtypes) and the optimal timing of DLI initiation remains unclear.</p><p><strong>Methods: </strong>This was a retrospective study of 62 patients with myeloid malignancies treated with alloHSCT and DLI from years 2001 to 2022. DLI indications were therapeutic 55 (89%), preemptive 6 (10%), and prophylactic in 1 (2%) patients with complete remission (CR/CRi) in 20 patients; diagnoses included 9 (45%) acute myeloid leukemia, 6 (30%) myelodysplastic syndromes, and 3 (15%) chronic myelomonocytic leukemia among others.</p><p><strong>Results: </strong>Among patients who received therapeutic DLI (n = 55), the best response was CR/CRi in 16 (29%) patients. At a median follow-up from the date of DLI of 75 (95% CI 35-121) months, there were 49 (79%) deaths with a median OS of 6 (95% CI 4-14) months, higher in patients in CR/CRi versus no CR/CRi (median 51 versus 4 months, p = 0.008). Presence of cytogenetic abnormalities such as complex karyotype, deletions in chromosome 5, 7, and 17p, and mutations in TP53, KRAS, NRAS, RUNX1, or JAK2 were associated with adverse outcomes.</p><p><strong>Conclusion: </strong>DLI is an effective treatment strategy for post-transplant relapse across myeloid malignancies, with specific genetic subtypes showing poorer outcomes and survival.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":2.8,"publicationDate":"2026-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148203800","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"COVID-19 Infection in Patients with Follicular Lymphoma and Related Indolent B-Cell Lymphomas Receiving Anti-CD20 Monoclonal Antibody Maintenance Therapy.","authors":"Shinto Kuniyoshi, Kohei Tanaka, Yu Akuzawa, Shunsuke Homma, Miyuki Okura, Takamitsu Okamura, Toru Kiguchi, Hiroki Sugimori, Koshi Hashimoto, Hideto Tamura","doi":"10.1159/000552825","DOIUrl":"10.1159/000552825","url":null,"abstract":"<p><strong>Introduction: </strong>Coronavirus disease 2019 (COVID-19) poses significant risks to immunocompromised individuals. Patients receiving maintenance therapy with anti-CD20 monoclonal antibodies for follicular lymphoma (FL) may be especially vulnerable because of prolonged B-cell depletion. However, data from Japan, particularly from the later stages of the pandemic, remain limited. This study aimed to investigate the incidence, severity, duration, mortality, and treatment discontinuation associated with COVID-19 in this population.</p><p><strong>Methods: </strong>We retrospectively analyzed all consecutive 72 patients with FL or related indolent B-cell lymphomas, including transformed FL, who received anti-CD20 maintenance therapy (rituximab or obinutuzumab) at our institute between January 2020 and September 2023. All patients had an indolent lymphoma background. Data on clinical features, laboratory values, disease severity at initial diagnosis according to Ministry of Health, Labour and Welfare guidelines, hospitalization, outcomes, duration of polymerase chain reaction positivity (>21 days defined as prolonged), and maintenance therapy continuation were reviewed. Fisher's exact test was used for analysis.</p><p><strong>Results: </strong>Among the 72 patients (median age: 69 years), 16 (22.2%) developed COVID-19. Of these, 5 had received rituximab and 11 obinutuzumab. The median lymphocyte count at infection was 498/μL; no patient had serum IgG <500 mg/dL. Eight infected patients developed oxygen-requiring disease at initial assessment (50.0% of infected patients; 11.1% overall). Prolonged infection occurred in 9 patients (56.3% of infected patients; 12.5% overall). All four deaths were COVID-19-related and occurred in obinutuzumab recipients (25.0% of infected patients; 5.6% overall). Lymphocyte count <200/μL was associated with mortality (p = 0.027), although this finding should be interpreted cautiously because of the small number of events. Maintenance therapy was discontinued in 5 of 8 patients infected during therapy.</p><p><strong>Conclusion: </strong>COVID-19 infection during anti-CD20 maintenance therapy led to severe illness and prolonged viral shedding in some cases. Severe lymphopenia may be a potential risk marker for mortality, but this finding should be interpreted cautiously because of the exploratory nature of the analysis.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":2.8,"publicationDate":"2026-06-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148203767","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Alexander Ambinder, Thomas W LeBlanc, M Yair Levy, Ami B Patel, James McCloskey, Erin Duffield, John Katsetos, Gabriel Mannis
{"title":"Shared Care Offers an Effective Framework for Managing Tagraxofusp Treatment in Blastic Plasmacytoid Dendritic Cell Neoplasm.","authors":"Alexander Ambinder, Thomas W LeBlanc, M Yair Levy, Ami B Patel, James McCloskey, Erin Duffield, John Katsetos, Gabriel Mannis","doi":"10.1159/000552815","DOIUrl":"10.1159/000552815","url":null,"abstract":"<p><strong>Background: </strong>Tagraxofusp, a first-in-class CD123-targeted therapy, is the only approved treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN). Shared care models enable coordination and care delivery between specialized cancer treatment centers and local institutions, allowing eligible patients to receive tagraxofusp.</p><p><strong>Summary: </strong>This review provides guidance on best practices for effective shared care implementation for tagraxofusp in patients with BPDCN, including appropriate patient selection, adequate local institutional readiness, proactive collaboration and communication between institutions, integrated comanagement between institutions, and requisite staff, patient, and caregiver education.</p><p><strong>Key messages: </strong>Shared care is particularly suitable for tagraxofusp treatment, as adverse events can be grade 3 or greater, but typically only occur in cycle 1, if they are to occur at all. Based on clinical experience, this article discusses the application of a shared care model for tagraxofusp treatment in BPDCN.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":2.8,"publicationDate":"2026-06-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13349363/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148161316","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Yajie Wang, Quan Rao, Yiming Ma, Yuan Zhang, Xiaofei Li
{"title":"Clinical Effect Analysis of Frozen-Thawed-Deglycerolized Red Blood Cells.","authors":"Yajie Wang, Quan Rao, Yiming Ma, Yuan Zhang, Xiaofei Li","doi":"10.1159/000552369","DOIUrl":"10.1159/000552369","url":null,"abstract":"<p><strong>Introduction: </strong>The aim of this study was to analyze the clinical infusion effect of frozen-thawed-deglycerolized red blood cells (FTD-RBCs) and provide solutions for situations such as blood supply shortages.</p><p><strong>Methods: </strong>In this retrospective study, 280 patients receiving FTD-RBCs and 280 patients receiving suspended red blood cells (SRBCs) in our hospital from December 2019 to June 2022 were included. We collected the course records and laboratory test results, including basic information, total amount of transfusion, as well as RBC count, hemoglobin concentration (HGB), and hematocrit (HCT) 24 h before and after the transfusion, and whether there were adverse blood transfusion reactions. Clinical efficacy analysis of FTD-RBCs was conducted by Mann-Whitney U test and Kruskal-Wallis H test.</p><p><strong>Results: </strong>In the 280 patients of our study, RBC count, HGB, and HCT increased significantly after transfusion of FTD-RBCs (p < 0.05). For transfusion of one FTD-RBC unit, the median differences in RBC count, HGB, and HCT before and after transfusion were 0.310 × 1012/L, 9.735 g/L, and 2.800%, respectively. The increments of the three indicators in the FTD-RBC group were all lower than those in the SRBC group, but the differences were not statistically significant (p > 0.05). None of the patients in this study had adverse transfusion reactions after transfusion of FTD-RBCs. In addition, we found that the efficacy of FTD-RBCs transfusion was associated with sex, age, body weight, disease type, and blood type of the patient (p < 0.05).</p><p><strong>Conclusion: </strong>The infusion of FTD-RBCs for clinical patients is safe and effective, and it is an effective blood supply supplement in situations of blood shortage such as public health events.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":2.8,"publicationDate":"2026-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147969421","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Catherine C Coombs, Thomas W LeBlanc, Katherine B Winfree, Catherine E Muehlenbein, Urvi Desai, Noam Y Kirson, Ashley Holub, Alice Qu, Ellen Sears, Brian Koffman, Meghan C Thompson
{"title":"Treatment Decision-Making for Individuals with Relapsed/Refractory Chronic Lymphocytic Leukemia in the USA.","authors":"Catherine C Coombs, Thomas W LeBlanc, Katherine B Winfree, Catherine E Muehlenbein, Urvi Desai, Noam Y Kirson, Ashley Holub, Alice Qu, Ellen Sears, Brian Koffman, Meghan C Thompson","doi":"10.1159/000552243","DOIUrl":"10.1159/000552243","url":null,"abstract":"<p><strong>Introduction: </strong>As available treatment options for relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) increase, treatment decisions become increasingly complex. This cross-sectional study evaluated patient and physician decision-making when selecting treatment for R/R CLL in the USA.</p><p><strong>Methods: </strong>After an initial pilot, 100 individuals with R/R CLL and 100 physicians who treat R/R CLL completed web-based surveys on factors that influenced treatment selection and levels of involvement during treatment selection. Categorical measures were summarized using numbers/proportions, and continuous measures were summarized using means/standard deviations.</p><p><strong>Results: </strong>Individuals with R/R CLL and physicians prioritized treatment efficacy (individuals: N = 47 of 78, 60.3%; physicians: N = 79 of 100, 79.0%) and potential side effects (individuals: N = 43 of 78, 55.1%; physicians: N = 50 of 79, 63.3%) when selecting treatment. Differences in the relative importance of specific factors were observed. Financial and cost factors were important for individuals, while medical history was important for physicians. Both cohorts reported a shared decision-making process.</p><p><strong>Conclusion: </strong>Building upon previous findings in the front-line CLL setting, this study confirms that individuals with R/R CLL and physicians commonly consider efficacy and safety of different treatment options when making their decision. Relative importance of specific factors within these and other categories differs across the samples.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-14"},"PeriodicalIF":2.8,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13368015/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147832214","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kashif Qureshi, Sujai Arakali, Jose Anzueto, Dillon Huddleston, Joshua Hobbs, Sebastian Saenz, Rhea Thakkar, Rahul H Jayaram, Carlos Goulart, Khaled Alok, Mihir Gupta
{"title":"Diagnostic Pathways and Procedural Risk in Primary Central Nervous System Lymphoma: A Nationwide Analysis.","authors":"Kashif Qureshi, Sujai Arakali, Jose Anzueto, Dillon Huddleston, Joshua Hobbs, Sebastian Saenz, Rhea Thakkar, Rahul H Jayaram, Carlos Goulart, Khaled Alok, Mihir Gupta","doi":"10.1159/000552333","DOIUrl":"10.1159/000552333","url":null,"abstract":"<p><strong>Introduction: </strong>Primary central nervous system lymphoma (PCNSL) is an aggressive malignancy in which timely diagnosis is critical. While neurosurgical biopsy remains the gold standard, cerebrospinal fluid (CSF)-based diagnostics are frequently deployed. Owing to the rarity of this condition, little is known about clinical characteristics, test utilization, social determinants of health, and complication rates in the context of securing a PCNSL diagnosis. We evaluated approaches for diagnosing PCNSL, post-procedural complications, and clinical and socioeconomic associations with adverse events.</p><p><strong>Methods: </strong>We queried the PearlDiver Mariner claims database to identify adults with newly diagnosed PCNSL between 2011 and 2023. CPT and ICD codes were used to identify lumbar puncture (LP) and/or neurosurgical biopsy performed before diagnosis. Patients were stratified by diagnostic pathway: LP-only, biopsy-only, or both LP and biopsy. We used logistic regression to identify predictors of adverse events.</p><p><strong>Results: </strong>Among 2,549 total patients, 1,957 (76.8%), 398 (15.6%), and 194 (7.6%) comprised the LP-only, biopsy-only, or both LP and biopsy pathways, respectively. LP-only patients were younger, more frequently male, Medicaid-insured, and economically disadvantaged (p < 0.001 for each). Rates of post-procedural thromboembolic complications were 5.4%, 10.1%, and 6.7% for LP-only, biopsy-only, and both LP and biopsy, respectively (p < 0.002). Biopsy-only was associated with increased odds of procedural complications (OR 3.21, 95% CI 1.79-5.69, p < 0.001), thromboembolic events (OR 1.85, 95% CI 1.39-2.45, p < 0.001), and medical complications (OR 1.87, 95% CI 1.49-2.33, p < 0.001) compared to LP alone.</p><p><strong>Conclusion: </strong>The choice of diagnostic modalities utilized to secure a diagnosis of PCNSL may be influenced by patient-level clinical and socioeconomic factors. PCNSL patients may represent a uniquely high-risk subgroup for neurosurgical biopsy, emphasizing the importance of advancing CSF-based diagnostics.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":2.8,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147832229","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}