Blood Research最新文献

{"title":"2026 Korean expert recommendations for the use of thrombopoietin receptor agonists in severe aplastic anemia.","authors":"Silvia Park, Jun Ho Jang, Ik-Chan Song, Seo-Yeon Ahn, Dong-Yeop Shin, Yong Park, Yeung-Chul Mun","doi":"10.1007/s44313-026-00152-3","DOIUrl":"10.1007/s44313-026-00152-3","url":null,"abstract":"<p><p>The treatment landscape for severe aplastic anemia (SAA) has undergone a significant paradigm shift with the integration of thrombopoietin receptor agonists (TPO-RAs) into standard immunosuppressive therapy (IST). However, clinical practice in Korea is shaped by a distinct regulatory and therapeutic environment characterized by the exclusive availability of rabbit antithymocyte globulin (rabbit ATG) and restrictive reimbursement policies governing TPO-RA use. This expert consensus statement provides a systematic framework and age-stratified treatment algorithms tailored to Korea-specific constraints. For treatment-naïve patients with SAA who are not candidates for immediate hematopoietic stem cell transplantation, a combination of rabbit ATG-based IST and TPO-RA is recommended as the first-line strategy. While eltrombopag is supported by robust global data, the panel emphasized the clinical utility of romiplostim in the Korean setting, given its established evidence in rabbit ATG-based regimens and the limited approved dosing of eltrombopag in Korea relative to pivotal global trials. The consensus also provides practical guidance on TPO-RA initiation, retreatment, and switching in refractory or relapsed disease, while emphasizing careful evaluation of clonal evolution in patients with insufficient response or newly emerging hematologic abnormalities, regardless of the specific TPO-RA used. Ultimately, these recommendations aim to standardize SAA management in Korea, optimize patient outcomes, and support future policy refinements to better align domestic practices with evolving global evidence.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13427683/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148632229","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 heterogeneity of primary cutaneous γδ T-cell lymphoma: a 20-year single-institution experience.","authors":"Miran Han, Sang Eun Yoon, Junhun Cho, Seok Jin Kim, Won Seog Kim","doi":"10.1007/s44313-026-00147-0","DOIUrl":"10.1007/s44313-026-00147-0","url":null,"abstract":"<p><strong>Purpose: </strong>Primary cutaneous γδ T-cell lymphoma (PCGDTCL) is a rare cutaneous T-cell lymphoma defined by γδ T-cell receptor expression and a cytotoxic phenotype. It has historically been considered highly aggressive, with median survival of 15-30 months and no standardized treatment due to its rarity. However, emerging reports describe subsets with relatively indolent behavior. We report a 20-year single-institution experience to better define the clinical spectrum and therapeutic implications of PCGDTCL.</p><p><strong>Methods: </strong>Patients diagnosed with PCGDTCL at Samsung Medical Center between January 2005 and September 2025 were retrospectively reviewed. Clinical, pathologic, and immunophenotypic features were collected and confirmed by hematopathology review. Pathologic features were visualized using heatmaps, and treatment courses were visualized using swimmer plots. Tumor response was evaluated according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.</p><p><strong>Results: </strong>Six patients were identified (median age 57 years). All presented with cutaneous lesions, ranging from localized solitary lesions to multifocal cutaneous involvement. ^18F-fluorodeoxyglucose positron emission tomography (FDG-PET) demonstrated heterogeneous metabolic activity, with maximum standardized uptake values (SUVmax) ranging from absent uptake to 8.9. Immunophenotyping showed uniform CD3 positivity, T-cell receptor γδ expression, βF1 negativity, and variable CD4, CD8, and CD56 expression. First-line therapies included observation, corticosteroids, CHOP (cyclophosphamide, doxorubicin, vincristine, prednisone), and GDP (gemcitabine, dexamethasone, cisplatin). Durable complete remissions occurred with CHOP and GDP. One patient experienced multiple relapses, later developed clinicopathologic features resembling mycosis fungoides, and survived for 13 years.</p><p><strong>Conclusion: </strong>PCGDTCL appears to show broader clinical and therapeutic heterogeneity than historically appreciated. Durable responses in selected patients suggest that treatment responsiveness may vary across the disease spectrum.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13385552/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148537152","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":"Response-adapted lenalidomide-dexamethasone (LenDex) intensification after CyBorD induction for transplant-eligible multiple myeloma: the PIANO study.","authors":"Daisuke Minakata, Go Yamamoto, Shinichi Kako, Yuki Hiroshima, Shuichi Ota, Tomoyuki Handa, Atsushi Wake, Akiko Meguro, Kensuke Usuki, Nobuhiro Tsukada, Hideki Nakasone, Yoshinobu Kanda","doi":"10.1007/s44313-026-00154-1","DOIUrl":"10.1007/s44313-026-00154-1","url":null,"abstract":"<p><strong>Purpose: </strong>Combination therapy with novel agents is the standard initial treatment for transplant-eligible patients with newly diagnosed multiple myeloma. However, the benefit of additional induction therapy for insufficient response remains unclear. This multicenter prospective study investigated response-adapted intensification with lenalidomide and dexamethasone (LenDex) after cyclophosphamide, bortezomib, and dexamethasone (CyBorD) in patients who did not achieve a very good partial response (VGPR) or better, followed by autologous stem cell transplantation (ASCT) and lenalidomide maintenance.</p><p><strong>Methods: </strong>Sixty-three patients were enrolled at 10 centers between March 2014 and December 2017.</p><p><strong>Results: </strong>Of the 63 patients, 44 underwent ASCT. Six patients (9.5% [6/63; 95% confidence interval, 3.6%-19.6%]) achieved complete response (CR) or better at 100 days post-ASCT; this rate was not higher than in the previous BD-based TUBA study. Best responses following ASCT and maintenance were stringent CR in 10 patients, CR in one, VGPR in 23, and partial response in eight. Three-year overall survival (OS) and progression-free survival (PFS) from induction were 90.0% and 43.9%, respectively. Thirty-one patients received LenDex before ASCT for insufficient response to CyBorD; 17 showed improved responses. OS and PFS from ASCT were comparable between patients treated with CyBorD alone and those receiving additional LenDex.</p><p><strong>Conclusion: </strong>The primary endpoint of CR or better at 100 days after ASCT was not improved compared with the historical BD-based TUBA study. However, response-adapted LenDex intensification was feasible, improved responses pre-ASCT in some patients, and resulted in post-ASCT outcomes comparable to those observed in patients who achieved sufficient responses with CyBorD alone.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-07-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13385730/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148521195","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":"Asciminib as first-line therapy in chronic myeloid leukemia: a systematic review.","authors":"Jae Joon Han, Jeong-Ok Lee, Kyoung Ha Kim, Sung-Eun Lee, Sang Kyun Sohn, Chul Won Jung, Hawk Kim","doi":"10.1007/s44313-026-00153-2","DOIUrl":"10.1007/s44313-026-00153-2","url":null,"abstract":"<p><strong>Purpose: </strong>Chronic myeloid leukemia (CML) is a myeloproliferative neoplasm characterized by the BCR::ABL1 fusion oncogene. Asciminib, a first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket) inhibitor, represents a novel therapeutic approach distinct from conventional ATP-competitive tyrosine kinase inhibitors (TKIs). This systematic review evaluated the efficacy and safety of asciminib as first-line therapy in patients with newly diagnosed chronic-phase CML.</p><p><strong>Methods: </strong>We conducted a comprehensive systematic review following PRISMA guidelines. Electronic searches were performed in PubMed, Embase, the Cochrane Library, and clinical trial registries through December 2025. Eligible studies included randomized controlled trials, prospective cohort studies, and compassionate use programs evaluating asciminib as first-line therapy in adult patients with chronic-phase CML. Primary outcomes included molecular response rates (major molecular response [MMR], MR4, and MR4.5), whereas secondary outcomes included safety profiles and treatment discontinuation rates.</p><p><strong>Results: </strong>The systematic review identified eight eligible studies comprising more than 500 patients. In the pivotal ASC4FIRST phase 3 trial (n = 405), asciminib demonstrated superior efficacy compared with investigator-selected TKIs. At 96 weeks, MMR rates were 74.1% with asciminib compared with 52.0% with investigator-selected TKIs (treatment difference, 22.4%; 95% CI, 13.6-31.3%; P < .001) and 76.2% with asciminib compared with 47.1% with imatinib (treatment difference, 29.7%; 95% CI, 17.6-41.8%; P < .001). The ASCEND study (n = 101) reported an MMR rate of 79% at 12 months. The safety profile was distinct from that of ATP-competitive TKIs. Hypertension occurred more frequently with asciminib (all grades, 10.5%; grade ≥ 3, 5.5%), whereas arterial occlusive events (all grades: asciminib, 2.0%; investigator-selected TKIs, 1.5%) and clinical pancreatitis (all grades, 1.0% in both groups) occurred at comparable rates between the treatment groups. Treatment discontinuation due to adverse events was lower with asciminib (5.0%) than with second-generation TKIs (12.7%).</p><p><strong>Conclusion: </strong>Asciminib demonstrates superior efficacy and a favorable benefit-risk profile as first-line therapy for CML, with higher molecular response rates than both imatinib and second-generation TKIs. Asciminib also has a favorable tolerability and safety profile; however, close monitoring for cardiovascular and pancreatic toxicities is required. These findings support asciminib as an effective treatment option for newly diagnosed chronic-phase CML.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13373123/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148457229","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":"Recent advances in the treatment of FLT3-mutated acute myeloid leukemia.","authors":"Jeong Suk Koh, Ik-Chan Song","doi":"10.1007/s44313-026-00155-0","DOIUrl":"10.1007/s44313-026-00155-0","url":null,"abstract":"<p><p>FMS-like tyrosine kinase 3 (FLT3) mutations are among the most common driver alterations in acute myeloid leukemia (AML), occurring in approximately 25-30% of patients, and have historically been associated with high relapse rates and poor survival. The therapeutic landscape for FLT3-mutated AML has evolved substantially with the development of FLT3-targeted agents. In newly diagnosed AML, the addition of midostaurin or quizartinib to intensive induction chemotherapy has demonstrated significant improvements in overall survival (OS), event-free survival, and relapse-free survival, establishing these regimens as current standards of care. In the relapsed or refractory setting, gilteritinib has become the preferred treatment based on superior survival and remission outcomes compared with salvage chemotherapy. Molecular studies have identified genomic factors associated with response and resistance to FLT3 inhibition, highlighting the importance of co-occurring mutations in treatment selection and prognosis. Maintenance therapy following allogeneic hematopoietic stem cell transplantation has emerged as a promising strategy, with evidence supporting the use of sorafenib and gilteritinib to reduce relapse risk and improve posttransplant outcomes. Recent clinical investigations have focused on novel combination approaches, particularly triplet regimens, incorporating FLT3 inhibitors, venetoclax, and hypomethylating agents, which have demonstrated encouraging responses and survival outcomes. Next-generation FLT3 inhibitors, including crenolanib and dapolsertib, are being evaluated in ongoing studies. These advances continue to improve outcomes in patients with FLT3-mutated AML and are expected to further refine treatment strategies across disease settings.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13350562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148413318","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":"Primary breast follicular lymphoma without a BCL2 rearrangement: insights into extranodal lymphoma biology.","authors":"Neha Seth, Anam Khan, Elaine Zhong, Wayne Tam","doi":"10.1007/s44313-026-00150-5","DOIUrl":"10.1007/s44313-026-00150-5","url":null,"abstract":"<p><strong>Background: </strong>Primary breast lymphoma is a rare malignancy that accounts for less than 1% of non-Hodgkin lymphomas. Diffuse large B-cell lymphoma is the most common subtype, whereas follicular lymphoma (FL) accounts for only a small proportion of cases. Most FL cases involving the breast represent secondary involvement by systemic disease, and true primary breast FL is uncommon. Molecular characterization of primary breast FL has rarely been reported, and its genetic landscape remains poorly understood.</p><p><strong>Case presentation: </strong>We report a case of primary breast FL, grade 3A, in a 55-year-old woman who presented with a palpable breast mass. Core needle biopsy revealed an atypical follicular lymphoid proliferation composed of centrocytes and numerous centroblasts (> 15 cells/high-power field). Immunohistochemistry revealed positivity for CD20, PAX5, CD10, and BCL6, with weak BCL2 expression, and a Ki-67 proliferation index of approximately 30%. Fluorescence in situ hybridization did not detect IGH::BCL2, BCL6, or MYC rearrangements but demonstrated copy-number gains involving BCL2, BCL6, MYC, and IGH. Next-generation sequencing identified biallelic CREBBP mutations and a FOXO1 mutation. Clonality studies confirmed a clonal IGK rearrangement in the breast lesion. Staging work-up with positron emission tomography/computed tomography showed disease confined to the breast (stage IE). The patient was treated with R-CHOP chemotherapy followed by involved-site radiotherapy.</p><p><strong>Conclusion: </strong>Our findings support the existence of a subset of extranodal follicular lymphomas lacking an IGH::BCL2 rearrangement and highlight alternative pathways of lymphomagenesis with implications for classification and diagnosis. The identification of biallelic CREBBP mutations and a FOXO1 mutation provides molecular insight into extranodal FL biology, suggesting an overlap with nodal FL while supporting alternative oncogenic pathways in extranodal disease.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13303997/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148319930","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":"Nuanced treatment decision in newly diagnosed multiple myeloma patients aged 65-75 years: a real-world study.","authors":"Naryllae Lee, Seoyeon Kim, Yugyeong Lee, Sin Young Park, Hyunsoo Cho, Ja Min Byun, Youngil Koh, Junshik Hong, Dong-Yeop Shin, Chang-Ki Min, Seonggyu Byeon, Sung-Soo Park, Dong Hyun Kim","doi":"10.1007/s44313-026-00149-y","DOIUrl":"10.1007/s44313-026-00149-y","url":null,"abstract":"<p><strong>Purpose: </strong>The optimal treatment strategy for patients aged 65-75 years with multiple myeloma (MM) remains undefined, as most clinical trials involving transplant-eligible patients have excluded this subgroup. We investigated the real-world treatment patterns, outcomes, and clinical impact of autologous stem cell transplantation (ASCT) and quadruplet therapy in this population.</p><p><strong>Methods: </strong>We retrospectively analyzed 561 patients aged 65-75 years with newly diagnosed MM treated between 2015 and 2023 at two tertiary centers. Patients were stratified by age (< 70 vs. ≥ 70 years) and ASCT status.</p><p><strong>Results: </strong>The use of quadruplet regimens and ASCT has increased markedly since 2021. Among patients aged 65-69 years, ASCT was associated with improved progression-free survival (PFS) (hazard ratio [HR], 0.37; 95% confidence interval [CI], 0.27-0.53; P < 0.001) and overall survival (HR, 0.30; 95% CI, 0.17-0.55; P < 0.001). In those aged 70-75 years, quadruplet therapy was independently associated with superior PFS (HR, 0.29; 95% CI, 0.12-0.73; P = 0.009). Similarly, in non-transplant recipients, quadruplet therapy remained a favorable predictor of PFS (HR, 0.40; 95% CI, 0.19-0.87; P = 0.021).</p><p><strong>Conclusion: </strong>Intensive treatment strategies-including ASCT and quadruplet therapy-are both feasible and beneficial for selected patients with MM aged ≥ 65 years. Therefore, chronological age alone should not be used to determine treatment eligibility. These findings provide real-world evidence that supports a shift toward individualized fitness-based treatment approaches to optimize outcomes in older patients with MM.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13309587/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148309809","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":"Novel treatment strategies in acute myeloid leukemia.","authors":"Shailesh Simkhada, Utsav Joshi, Prajwal Dhakal","doi":"10.1007/s44313-026-00146-1","DOIUrl":"10.1007/s44313-026-00146-1","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is a molecularly diverse hematologic malignancy, with clinical outcomes driven by patient fitness, disease biology, and the depth of remission. Over the past decade, the increasing use of molecular studies has enabled genotype-directed therapy and accelerated the incorporation of targeted agents into both intensive and lower-intensity treatment regimens. FLT3 inhibitors are now well established across a wide disease spectrum, including frontline settings, relapsed/refractory disease, and post-allogeneic hematopoietic cell transplantation maintenance, with emerging evidence supporting measurable residual disease (MRD)-adapted maintenance approaches. IDH1/2 inhibitors offer therapy for specific molecular subsets, particularly in older or unfit patients, and are increasingly being evaluated in doublet and triplet combinations. Venetoclax-based regimens have become a cornerstone for older or unfit patients with AML and are being optimized through response-adapted dosing and careful partner selection to mitigate cytopenias while deepening remission. Menin inhibitors represent a major therapeutic advance for KMT2A-rearranged and NPM1-mutated AML and are rapidly moving into combination and earlier-line treatment strategies. In parallel, immune and cellular therapies, including antibody-drug conjugates, immune checkpoint modulation, bispecific engagers, and chimeric antigen receptor T-cell and chimeric antigen receptor natural killer constructs, are under investigation, although durable, practice-changing benefits outside select settings remain limited to date. This review synthesizes pivotal clinical data and translational principles informing emerging targeted, immune-based, and cellular strategies in AML, with an emphasis on remission depth, MRD-driven decision-making, and relapse prevention.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13269617/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148253780","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":"Cross-cultural validation and reliability of the Korean version of the Haemophilia Activities List.","authors":"Sung Eun Kim, Ye Jee Shim, Hwi-Joong Yoon, Sang Kyu Park, Ki-Young Yoo, Eun Jin Choi, Ji Yoon Kim","doi":"10.1007/s44313-026-00148-z","DOIUrl":"10.1007/s44313-026-00148-z","url":null,"abstract":"<p><strong>Purpose: </strong>To translate, culturally adapt, and evaluate the reliability and construct validity of the Korean version of the Haemophilia Activities List (HAL) in adult patients with hemophilia.</p><p><strong>Methods: </strong>This cross-sectional validation study included adult patients with hemophilia A and B. The HAL was translated into Korean using a standardized forward-backward translation process according to international guidelines. The participants completed the Korean HAL, the EuroQol Five-Dimension Five-Level Questionnaire (EQ-5D-5L), and the Routine Assessment of Patient Index Data 3 (RAPID3) questionnaire. Internal consistency was assessed using Cronbach's alpha, test-retest reliability was assessed using intraclass correlation coefficients (ICCs), and construct validity was assessed using Spearman's correlation analysis.</p><p><strong>Results: </strong>A total of 87 adult patients completed the questionnaire. The mean age was 37 years (range, 18-62 years), and most participants had severe hemophilia. The Korean HAL demonstrated high internal consistency, with a Cronbach's alpha coefficient of 0.97 for the total score and domain-specific values ranging from 0.78 to 0.95. The test-retest reliability was excellent, with an overall ICC of 0.95. Construct validity was supported by strong correlations between the HAL and EQ-5D-5L overall scores (r = 0.78, p < 0.01) and strong correlations with RAPID3 physical function scores (r = -0.82, p < 0.01).</p><p><strong>Conclusions: </strong>The Korean version of the HAL demonstrated excellent reliability and good construct validity in adult patients with hemophilia. These findings support the use of the Korean HAL as a reliable and culturally appropriate patient-reported outcome measure to assess functional limitations in clinical practice and research.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":" ","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13275968/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148240125","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":"Infectious complications after CAR T-cell therapy: mechanisms, risk stratification, and prevention.","authors":"Eunseuk Lee, Ji Hyun Lee, Hye Ryeon Kim, Seok Jae Huh, Suee Lee, Sung-Yong Oh, Sung-Hyun Kim, Hojin Lee","doi":"10.1007/s44313-026-00145-2","DOIUrl":"10.1007/s44313-026-00145-2","url":null,"abstract":"<p><p>Chimeric antigen receptor (CAR) T-cell therapy is an established treatment for relapsed or refractory B-cell malignancies. Despite therapeutic success, infectious complications remain a major contributor to non-relapsed morbidity and mortality. The risk of infection reflects a prolonged and multifactorial state of immunosuppression resulting from lymphodepleting chemotherapy, treatment-related cytopenia, sustained CD4⁺ T-cell lymphopenia, B-cell aplasia with hypogammaglobulinemia, and the use of corticosteroids or cytokine-directed therapies for the management of immune effector cell-associated toxicities (i.e., cytokine-release syndrome and immune effector cell-associated neurotoxicity). The spectrum of infections has evolved over time. Bacterial infections are observed most frequently in the early post-infusion period, whereas viral and opportunistic infections become more prominent later, particularly in patients with delayed immune recovery. Infection risk is also influenced by the underlying malignancy, prior exposure to treatment, and the type of CAR T-cell product used. For example, BCMA-directed CAR T-cell therapy has been associated with a higher infection burden, likely related to profound plasma cell depletion and sustained impairment of humoral immunity. Preventing infections in this setting requires a structured and individualized approach. Common strategies include antiviral and Pneumocystis jirovecii pneumonia prophylaxis, the selective use of antibacterial and antifungal prophylaxis during periods of severe or prolonged cytopenia, immunoglobulin replacement in patients with clinically significant hypogammaglobulinemia, and vaccination schedules guided by immune recovery. Therefore, coordinated and risk-adapted prevention is essential to minimize infectious complications and to improve long-term outcomes after CAR-T cell therapy. This review summarizes the current evidence and major recommendations of international guidelines to provide an updated overview of infection mechanisms, risk stratification, and preventive strategies for patients receiving CAR T-cell therapy.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"61 1","pages":""},"PeriodicalIF":3.7,"publicationDate":"2026-06-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13260542/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148220217","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}