Blood advances最新文献_第10页

Predictive Value of Free Light Chain Burden in Patients with AL Amyloidosis Treated with Bortezomib-Based Regimens. 以硼替佐米为基础的方案治疗AL淀粉样变性患者游离轻链负担的预测价值。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-30 DOI: 10.1182/bloodadvances.2024015528

Brendan Saunders, Foteini Theodorakakou, Despina V Fotiou, Samuel Boullt, Benjamin Evans, Meletios Athanasios Dimopoulos, Efstathios Kastritis, Giada Bianchi

{"title":"Predictive Value of Free Light Chain Burden in Patients with AL Amyloidosis Treated with Bortezomib-Based Regimens.","authors":"Brendan Saunders, Foteini Theodorakakou, Despina V Fotiou, Samuel Boullt, Benjamin Evans, Meletios Athanasios Dimopoulos, Efstathios Kastritis, Giada Bianchi","doi":"10.1182/bloodadvances.2024015528","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015528","url":null,"abstract":"A difference between involved and uninvolved free light chain (dFLC) equal or higher than 180 mg/L is part of the 2012 Mayo staging system for AL amyloidosis given its negative impact on overall survival. However, none of the 758 patients evaluated to develop and validate this staging system received bortezomib or daratumumab-containing regimens. Over the past two decades, cyclophosphamide-bortezomib-dexamethasone (CyBorD) and, more recently, daratumumab-CyBorD (DaraCyBorD) have become cornerstone treatments for AL amyloidosis, demonstrating high efficacy in rapidly normalizing FLC levels. We hypothesized that in newly diagnosed AL amyloidosis patients treated with bortezomib and daratumumab-based regimens, a baseline high free light chain burden may no longer predict adverse prognosis. In this retrospective, multicenter study of 223 newly diagnosed AL amyloidosis patients treated with CyBorD or DaraCyBorD therapy, we investigated: 1) the association between baseline involved FLC (iFLC) and dFLC hematological response at 28 days and 3, 6, 9 and 12 months following commencement of therapy ; 2) the overall survival of patients with baseline low (<180mg/L), medium (180-400 mg/L) and high (>400 mg/L) dFLC ; and 3) the prognostic value of bone marrow plasma cell burden in determining response to CyBorD or DaraCyBorD therapy and overall survival; and finally, the prognostic value of the 2012 Mayo staging system in the CyBorD and DaraCyBorD cohorts. Our findings suggest that a dFLC over 180 mg/L no longer holds prognostic value in the era of CyBorD/DaraCyBorD-based AL amyloidosis therapy and question the utility of the Mayo 2012 staging system in the era of highly effective chemo-immunotherapies.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144062393","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study. Lisocabtagene maraleucel用于非HSCT患者的R/R LBCL: 2期PILOT研究的最终结果

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-30 DOI: 10.1182/bloodadvances.2024015262

Alison Sehgal, Daanish Hoda, Peter A Riedell, Nilanjan Ghosh, Mehdi Hamadani, Gerhard C Hildebrandt, John E Godwin, Patrick M Reagan, Nina D Wagner-Johnston, James H Essell, Rajneesh Nath, Scott R Solomon, Rebecca Champion, Edward Licitra, Suzanne Fanning, Neel K Gupta, Victor A Chow, Brenda Yuan, Zhi Yang, Ken Ogasawara, Jerill Thorpe, Leo I Gordon

{"title":"Lisocabtagene maraleucel for R/R LBCL in patients not intended for HSCT: final results of the phase 2 PILOT study.","authors":"Alison Sehgal, Daanish Hoda, Peter A Riedell, Nilanjan Ghosh, Mehdi Hamadani, Gerhard C Hildebrandt, John E Godwin, Patrick M Reagan, Nina D Wagner-Johnston, James H Essell, Rajneesh Nath, Scott R Solomon, Rebecca Champion, Edward Licitra, Suzanne Fanning, Neel K Gupta, Victor A Chow, Brenda Yuan, Zhi Yang, Ken Ogasawara, Jerill Thorpe, Leo I Gordon","doi":"10.1182/bloodadvances.2024015262","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015262","url":null,"abstract":"We report final analysis results from the PILOT study of lisocabtagene maraleucel (liso-cel) for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL). Sixty-one adults with R/R LBCL who received 1 prior line of therapy and met ≥1 transplant not intended criterion received liso-cel. Overall response rate (primary end point) was 80%; 54% achieved complete response. After median on-study follow-up of 18.2 months, median duration of response was 23.3 months (95% confidence interval [CI], 6.2‒not reached [NR]). Median progression-free survival (PFS) was 9.0 months (95% CI, 4.2‒NR), median overall survival (OS) was NR (95% CI, 16.3‒NR), and 18-month PFS and OS rates were 43% (95% CI, 30‒55) and 59% (95% CI, 45‒70), respectively. In the treatment-emergent (TE) period (≤90 days after liso-cel administration), 79% had grade ≥3 adverse events (AE), 38% had cytokine release syndrome (2% grade 3; no grade 4/5), 31% had neurological events (5% grade 3; no grade 4/5), and 7% had grade ≥3 infections. Of 57 patients in the post-TE period (≥91 days after liso-cel administration), 18% experienced grade ≥3 AEs; 1 patient had grade ≥3 infections. Thirty patients in the leukapheresis set (n = 74) died, mostly of disease progression (n = 24). In this population with high incidence of high-grade B-cell lymphoma, primary-refractory disease, advanced age, and comorbidities, liso-cel demonstrated durable efficacy and a favorable safety profile, consistent with previous reports. These results support liso-cel as second-line therapy for this underserved population of patients with R/R LBCL not intended for hematopoietic stem cell transplantation. ClinicalTrials.gov: #NCT03483103.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143953649","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Real-world effectiveness of immunoglobulin replacement for hypogammaglobulinemia and infections in multiple myeloma. 免疫球蛋白替代治疗多发性骨髓瘤低γ球蛋白血症和感染的实际有效性。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-30 DOI: 10.1182/bloodadvances.2024015477

Elizabeth O'Donnell, Thais Gift, Zaid Yousif, Nikhil Khandelwal, Raj Desai, Lynn Huynh, Louise Clear, Megan Pinaire, Mingchen Ye, Azeem Banatwala, Gregory Belsky, Yichuan Grace Hsieh, Christopher Herrick, Mei Sheng Duh, Shawn N Murphy, Marie Sanchirico

{"title":"Real-world effectiveness of immunoglobulin replacement for hypogammaglobulinemia and infections in multiple myeloma.","authors":"Elizabeth O'Donnell, Thais Gift, Zaid Yousif, Nikhil Khandelwal, Raj Desai, Lynn Huynh, Louise Clear, Megan Pinaire, Mingchen Ye, Azeem Banatwala, Gregory Belsky, Yichuan Grace Hsieh, Christopher Herrick, Mei Sheng Duh, Shawn N Murphy, Marie Sanchirico","doi":"10.1182/bloodadvances.2024015477","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015477","url":null,"abstract":"This study assessed the real-world effectiveness of immunoglobulin replacement therapy (IgRT) for treatment of hypogammaglobulinemia and infections in patients with multiple myeloma (MM). A retrospective study was conducted in adult patients diagnosed with MM on or after January 1, 2010 using the Mass General Brigham Research Patient Data Registry. Infections were compared before versus after IgRT initiation. Generalized estimating equation logistic regression models were used to calculate odds ratios (ORs) and 95% confidence intervals (CIs). In patients with accessible serum protein electrophoresis (SPEP) tests, a natural language processing program supported the extraction of immunoglobulin G (IgG) data. IgG assessments and determination of hypogammaglobulinemia (defined as IgG level <500 mg/dL) were compared before versus after IgRT initiation. Results were reported with descriptive statistics. 6,062 patients with MM were identified (56.2% men, median age 65.0 years). 471/6,062 (7.8%) received ≥1 IgRT administrations. At 3 months, significantly lower odds of infections (OR [95% CI]: 0.71 [0.56, 0.89], P=0.0004) were observed after IgRT initiation versus before. Among patients with accessible SPEP tests (n=3,405), 3231 (94.9%) received ≥1 IgG test, with a median of 18.0 (interquartile range [IQR]: 7.0, 40.0) IgG tests per patient. Hypogammaglobulinemia was experienced by 2,075/3,231 (64.2%) patients who had ≥1 IgG test. Significantly fewer patients had hypogammaglobulinemia after IgRT initiation. In conclusion, IgRT use was associated with significant reductions in hypogammaglobulinemia and infections. While IgRT is currently used for MM treatment, there is potential to optimize its dosing and treatment duration to reduce the morbidity and mortality associated with infections.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143953900","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Chronic kidney disease in adults with sickle cell trait: A systematic review and meta-analysis. 慢性肾脏疾病成人镰状细胞特征：系统回顾和荟萃分析。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-29 DOI: 10.1182/bloodadvances.2025015920

Yagahira Castro Sesquen, Carmela Gelida Barboza Justiniano, Diego J Aparcana-Granda, Andres R Aquino, Pierre Luckens Adelson, Ronald Diego Mondragón Calixto, John Kwagyan, Vimal K Derebail, Seyed Mehdi Nouraie, Santosh L Saraf, Marina Jerebtsova

{"title":"Chronic kidney disease in adults with sickle cell trait: A systematic review and meta-analysis.","authors":"Yagahira Castro Sesquen, Carmela Gelida Barboza Justiniano, Diego J Aparcana-Granda, Andres R Aquino, Pierre Luckens Adelson, Ronald Diego Mondragón Calixto, John Kwagyan, Vimal K Derebail, Seyed Mehdi Nouraie, Santosh L Saraf, Marina Jerebtsova","doi":"10.1182/bloodadvances.2025015920","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025015920","url":null,"abstract":"Sickle cell trait (SCT) may increase the risk of chronic kidney disease (CKD). We aim to determine the pooled statistics of the association between SCT and CKD. Studies published up to May 2024 on PubMed, Embase, Global Health Library, and Web of Science were screened. We included studies reporting odds ratios (OR) or hazard ratios (HR) of CKD and/or end-stage renal disease (ESRD) comparing adults with and without SCT. The risk of bias was evaluated using the ROBINS-E tool. The pooled SCT prevalence was calculated among patients with CKD/ESRD. Random-effects analysis was performed. Only studies with low or some concerns of bias were included, corresponding to 18,847 SCT participants and 1,060,818 without SCT. SCT participants had higher odds of having an eGFR ≤ 60 ml/min/1.73 m² (OR: 1.62, 95% CI: 1.39-1.89), proteinuria (OR: 2.02, 95% CI: 1.61-2.54), and eGFR ≤ 60 ml/min/1.73 m² and/or proteinuria (OR: 1.79, 95% CI: 1.44-2.22). The pooled prevalence of SCT among African Americans with ESRD was 10% (95% CI: 8-12%); however, heterogeneity was very high (I²: 85.6%). There was a higher HR for ESRD (p = 0.04) in the studies that included both males and females (HR: 1.72, 95% CI: 1.13-2.61) compared to the study that included only females (HR: 0.75, 95% CI: 0.39 - 1.44), suggesting that males have a higher risk of ESRD. Controversial results were observed for the association of CKD with hypertension and diabetes. SCT increases the risk of developing CKD and ESRD. PROSPERO registration: CRD42021275274.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143961783","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Reassessing the Villalta Score's Use of Signs and Symptoms. 重新评估Villalta评分对体征和症状的使用。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-29 DOI: 10.1182/bloodadvances.2025016750

Steven Yale, Halil Tekiner, Regina Wilson, Eileen Yale

引用次数: 0

High rate of complete responses induced by platinum-based regimen with BTK inhibitors in Richter's Syndrome. 以铂为基础的BTK抑制剂治疗里希特氏综合征的完全缓解率高。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-29 DOI: 10.1182/bloodadvances.2025015913

Georgios Pongas, Maryam Alasfour, Daniel P Cassidy, Juan Pablo Alderuccio, Joseph D Rosenblatt, Izidore S Lossos

引用次数: 0

An Ethical Allocation Scheme for Scarce Gene Therapies in Sickle Cell Disease and Transfusion-Dependent β-Thalassemia. 镰状细胞病和输血依赖性β-地中海贫血稀缺基因治疗的伦理分配方案。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-24 DOI: 10.1182/bloodadvances.2025016053

Amar H Kelkar, Maureen O Achebe, Andrew Hantel

引用次数: 0

2025 ASH ISTH NBDF WFH Monitoring Report on Their 2021 Guidelines on Diagnosis and Management of von Willebrand Disease. 2025年ASH ISTH NBDF WFH监测报告他们2021年血管性血友病诊断和管理指南。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-24 DOI: 10.1182/bloodadvances.2025016512

Paula D James, Veronica H Flood, Nathan T Connell

{"title":"2025 ASH ISTH NBDF WFH Monitoring Report on Their 2021 Guidelines on Diagnosis and Management of von Willebrand Disease.","authors":"Paula D James, Veronica H Flood, Nathan T Connell","doi":"10.1182/bloodadvances.2025016512","DOIUrl":"https://doi.org/10.1182/bloodadvances.2025016512","url":null,"abstract":"The American Society of Hematology (ASH), International Society on Thrombosis and Hemostasis (ISTH), National Hemophilia Foundation (NHF, now National Bleeding Disorders Foundation NBDF) and World Federation of Hemophilia (WFH) 2021 Guidelines on the Diagnosis and Management of von Willebrand disease (VWD) included 11 recommendations on diagnosis of VWD, and 12 recommendations on management of VWD, the most common inherited bleeding disorder. We describe the results of a review of the 2021 guidelines by the clinical co-chairs of the guideline panels requested by ASH to inform decision-making about the need for and timing of a guideline revision. An updated Medline and Embase search applied the same terms as the ASH ISTH NHF WFH 2021 guidelines limited to studies from 2020 through July 30, 2024 (diagnosis) or July 24, 2024 (management). For the diagnosis of VWD, 432 studies were identified and underwent title and abstract review, with 17 undergoing full text review, and for management of VWD, 288 studies were identified and underwent title and abstract review, with 37 undergoing full text review, to determine if the data would change the strength or directionality of the existing recommendation or merit development of a new recommendation. Based on this review, the clinical co-chairs noted that none of the reviewed studies would change the direction or strength of the existing guideline recommendations. There will be continued monitoring of the ASH ISTH NHF WFH 2021 Guidelines on the Diagnosis and Management of VWD to evaluate whether there is sufficient new evidence to warrant additional revisions.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143954409","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Frontline allo-SCT in MRD-negative, intermediate-risk AML. mrd阴性、中度风险AML的一线alloc - sct治疗。

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-24 DOI: 10.1182/bloodadvances.2025016610

Eduardo Rodríguez-Arbolí, Teresa Caballero-Velázquez, Cristina Marrero-Cepeda, José Antonio Pérez-Simón

引用次数: 0

Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment. 成人PNH患者在接受ravulizumab或eculizumab治疗时的临床显著性血管外溶血特征

IF 7.4 1区医学

Blood advances Pub Date : 2025-04-24 DOI: 10.1182/bloodadvances.2024015490

Austin G Kulasekararaj, Jong Wook Lee, Christopher J Patriquin, Caroline I Piatek, Alexander Röth, Wilma Barcellini, Robert A Brodsky, Jun-Ichi Nishimura, Ji Yu, Mohammed Mahdi, Régis Peffault De Latour

{"title":"Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment.","authors":"Austin G Kulasekararaj, Jong Wook Lee, Christopher J Patriquin, Caroline I Piatek, Alexander Röth, Wilma Barcellini, Robert A Brodsky, Jun-Ichi Nishimura, Ji Yu, Mohammed Mahdi, Régis Peffault De Latour","doi":"10.1182/bloodadvances.2024015490","DOIUrl":"https://doi.org/10.1182/bloodadvances.2024015490","url":null,"abstract":"In patients with paroxysmal nocturnal hemoglobinuria (PNH), complement component 5 (C5) inhibitors ravulizumab and eculizumab control terminal complement activity and intravascular hemolysis, drivers of morbidity and mortality. During C5 inhibitor treatment, ongoing C3 fragment deposition on surviving PNH red blood cells may cause clinically significant extravascular hemolysis (csEVH) in some patients. csEVH is not thought to be life-threatening but may cause symptomatic anemia and need for transfusion. This post-hoc analysis of studies 301 (NCT02946463) and 302 (NCT03056040) evaluated the prevalence of csEVH (symptomatic anemia [hemoglobin levels < 9.5 g/dL] with absolute reticulocyte count ≥ 120 × 109/L) in adult patients with PNH treated with ravulizumab or eculizumab for 6 months (183 days). The association between csEVH and fatigue (measured using the Functional Assessment of Chronic Illness Therapy - Fatigue [FACIT-F] scale) was also evaluated in study 302 patients with stable disease. On Day 183 of studies 301 and 302, respectively, csEVH prevalence was 23.2% (29/125) and 20.2% (19/94) with ravulizumab, and 24.8% (30/121) and 21.3% (20/94) with eculizumab. All patients in study 302 with csEVH experienced fatigue, which was mostly mild (ravulizumab: 79%, eculizumab: 65%) or moderate (ravulizumab: 21%, eculizumab: 30%). FACIT-F scores remained stable from baseline (ravulizumab: 42.2; eculizumab: 38.3) to Day 183 (ravulizumab: 44.3; eculizumab: 38.3); close to general population normative values. This analysis demonstrated that csEVH affected 20-25% of patients with PNH, most of whom experienced mild fatigue, with fatigue remaining stable during treatment. Ravulizumab and eculizumab continue to provide benefit to adults with PNH with csEVH.","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":""},"PeriodicalIF":7.4,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143961832","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0