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Impact of daratumumab/bortezomib/thalidomide and dexamethasone induction therapy on chemo-free stem cell mobilization in transplant-eligible newly diagnosed multiple myeloma: a multicenter real-world experience. 达拉单抗/硼替佐米/他替佐米和地塞米松诱导疗法对符合移植条件的新诊断多发性骨髓瘤患者无化疗干细胞动员的影响:多中心真实世界经验。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.286332
Marika Porrazzo, Tiziana Moscato, Giuseppe Sapienza, Fabrizio Accardi, Caterina Patti, Clotilde Cangialosi, Carmelo Costanza, Roberto Bono, Stefania Tringali, Cristina Rotolo, Emilia Gigliotta, Andrea Rizzuto, Manuela Giuseppa Ingrascè, Giulia Butera, Laura Di Noto, Alessandra Santoro, Anna Marfia, Cirino Botta, Sergio Siragusa, Massimo Martino, Luca Castagna
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引用次数: 0
Outcomes of children and young adults with B-cell acute lymphoblastic leukemia given blinatumomab as last consolidation treatment before allogeneic hematopoietic stem cell transplantation. B细胞急性淋巴细胞白血病儿童和青少年患者在接受同种异体造血干细胞移植前最后一次巩固治疗时使用blinatumomab的疗效。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.286350
Mattia Algeri, Michele Massa, Daria Pagliara, Valentina Bertaina, Federica Galaverna, Ilaria Pili, Giuseppina Li Pira, Roberto Carta, Francesco Quagliarella, Rita M Pinto, Chiara Rosignoli, Barbarella Lucarelli, Maria G Cefalo, Emilia Boccieri, Francesca Benini, Francesca Del Bufalo, Marco Becilli, Pietro Merli, Gerhard Zugmaier, Franco Locatelli

Blinatumomab has remarkable efficacy in patients with relapsed/refractory (r/r) or measurable residual disease (MRD)-positive B-cell acute lymphoblastic leukemia (B-ALL). In many patients, blinatumomab treatment is followed by allogeneic hematopoietic stem cell transplantation (HSCT). However, the influence of blinatumomab on HSCT outcomes in children and young adults (YA) remains to be fully elucidated. We conducted a single-center, retrospective analysis of patients given blinatumomab as last treatment before HSCT. Seventy-eight pediatric and YA patients were evaluated. With a median follow- up of 23.23 months, the 2-year disease-free (DFS) and overall survival (OS) probability were 72.2% and 89.2%, respectively, with a 2-year cumulative incidence of non-relapse mortality (NRM) of 2.6%. A trend toward improved 2-year DFS, but not OS, was noted in patients transplanted in first complete remission (CR1) (92.9%) compared to those in second or greater remission (CR2/3) (68.5%; P=0.18) due to a lower cumulative incidence of relapse (0% vs. 29.9%; P=0.05). Among CR2/3 patients, those receiving the sequential combination of inotuzumab and blinatumomab had a significantly lower cumulative incidence of relapse as compared to those who did not receive inotuzumab (9.5% vs. 40.4%; P=0.023). Relapse after HSCT occurred in 16 patients, all exhibiting CD19-positive blasts; ten of them received anti-CD19 chimeric antigen receptor T-cell (CAR T) therapy and two inotuzumab as salvage therapy, leading to a 2-year post-relapse OS of 52.7%. Our results indicate that HSCT following blinatumomab in children and YA with B-ALL is highly effective, being associated with low NRM and not affecting the efficacy of subsequent salvage immunotherapies, including CAR T cells.

对于复发/难治(r/r)或可测量残留病(MRD)阳性B细胞急性淋巴细胞白血病(B-ALL)患者,Blinatumomab具有显著疗效。许多患者在接受blinatumomab治疗后,会进行异基因造血干细胞移植(HSCT)。然而,blinatumomab对儿童和年轻成人(YA)造血干细胞移植结果的影响仍有待全面阐明。我们对造血干细胞移植前最后一次接受blinatumomab治疗的患者进行了单中心回顾性分析。共有 78 名儿童和青年患者接受了评估。中位随访时间为23.23个月,2年无病生存率(DFS)和总生存率(OS)分别为72.2%和89.2%,2年非复发死亡率(NRM)的累积发生率(CI)为2.6%。与第二次或更多次缓解(CR2/3)患者(68.5%,P=0.18)相比,首次完全缓解(CR1)移植患者(92.9%)的2年DFS有改善趋势,但OS没有改善,原因是复发的CI较低(0% vs. 29.9%,P=0.05)。在CR2/3患者中,与未接受伊妥珠单抗治疗的患者相比,接受伊妥珠单抗和blinatumomab序贯联合治疗的患者复发CI显著较低(9.5% vs. 40.4%,p=0.023)。造血干细胞移植后复发的患者有16例,均表现为CD19阳性囊泡,其中10例接受了抗CD19嵌合抗原受体T细胞(CAR-T)疗法,2例接受了伊妥珠单抗作为挽救疗法,复发后2年的OS为52.7%。我们的研究结果表明,B-ALL患儿和YA接受blinatumomab治疗后进行造血干细胞移植非常有效,NRM较低,而且不会影响包括CAR-T细胞在内的后续挽救性免疫疗法的疗效。
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引用次数: 0
Infections in sickle cell disease. 镰状细胞病的感染。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.285066
Lily E A Scourfield, Amina Nardo-Marino, Thomas N Williams, David C Rees

Sickle cell disease (SCD) is one of the commonest severe inherited disorders in the world. Infection accounts for a significant amount of the morbidity and mortality, particularly in sub-Saharan Africa, but is relatively poorly studied and characterized. Patients with SCD have significant immunodeficiency and are more likely to suffer severe and life-threatening complications of infection, and additionally infections can trigger complications of SCD itself. Those with more severe forms of SCD have functional asplenia from a very early age, which accounts for much of the morbidity in young children, particularly invasive infections from encapsulated bacteria including Streptococcus pneumoniae, Haemophilus influenzae, Salmonella typhi and meningococcal disease. Additionally, there are other defects in immune function in SCD, associated with anemia, tissue infarction and impaired adaptive immunity. Complications of infections in SCD include acute chest syndrome, acute painful episodes, osteomyelitis, meningitis, urinary tract infections, overwhelming sepsis and death. Viral infections cause significant morbidity, particularly severe anemia associated with parvovirus, and to a lesser extent other infections such as influenza and coronavirus disease 2019. The relationship between malaria and SCD is complicated and discussed in this review. Unlike many of the genetic risk factors for poor outcomes in SCD, it is theoretically possible to modify the risks associated with infections with established public health measures. These include the provision of vaccination, prophylactic antibiotics and access to clean water and mosquito avoidance, although current financial restraints and political priorities have made this difficult.

镰状细胞病(SCD)是世界上最常见的严重遗传性疾病之一。感染占发病率和死亡率的很大一部分,尤其是在撒哈拉以南非洲地区,但对感染的研究和描述却相对较少。SCD 患者存在严重的免疫缺陷,更有可能出现严重的、危及生命的感染并发症,此外,感染还可能引发 SCD 本身的并发症。那些患有较严重形式的 SCD 的患者在很小的时候就会出现功能性胰腺缺失,这也是导致幼儿发病的主要原因,尤其是由肺炎链球菌、流感嗜血杆菌、伤寒沙门氏菌和脑膜炎球菌病等包裹细菌引起的侵袭性感染。此外,SCD 还存在其他免疫功能缺陷,与贫血、组织梗塞和适应性免疫受损有关。SCD 感染的并发症包括急性胸部综合征(ACS)、急性疼痛发作、骨髓炎、脑膜炎、尿路感染、严重败血症和死亡。病毒感染会导致严重的发病率,尤其是与副病毒有关的严重贫血,其次是其他感染,如流感和 COVID19。疟疾与 SCD 之间的关系十分复杂,本综述将对此进行讨论。与许多导致 SCD 不良后果的遗传风险因素不同,理论上可以通过既定的公共卫生措施来改变与感染相关的风险。这些措施包括提供疫苗接种、预防性抗生素、获得清洁水和避免蚊虫叮咬,尽管目前的财政限制和政治优先事项使得这些措施难以实现。
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引用次数: 0
Expression levels of genes implicated in the working mechanism of lenalidomide predict treatment response in lower risk myelodysplastic syndrome patients. 来那度胺作用机制相关基因的表达水平可预测低风险骨髓增生异常综合征患者的治疗反应。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.286157
Florentien E M In't Hout, Thessa N Scheele, Theresia M Westers, Canan Alhan, Carolien Duetz, Eline M P Cremers, Heleen A Visser-Wisselaar, Annelies Verbrugge, Dana A Chitu, Bert A Van der Reijden, Aniek O De Graaf, Arjan A Van de Loosdrecht, Joop H Jansen
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引用次数: 0
Ixazomib, pomalidomide and dexamethasone in relapsed or refractory multiple myeloma characterized with highrisk cytogenetics: the IFM 2014-01 study. 伊沙佐米、泊马度胺和地塞米松治疗具有高风险细胞遗传学特征的复发或难治性多发性骨髓瘤:IFM 2014-01研究。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.285916
Arthur Bobin, Salomon Manier, Joe De Keizer, Jaydeep K Srimani, Cyrille Hulin, Lionel Karlin, Denis Caillot, Ingrid Lafon, Clara Mariette, Carla Araujo, Bertrand Arnulf, Benoît Bareau, Karim Belhadj, Lofti Benboubker, Thorsten Braun, Claire Calmettes, Olivier Decaux, Mamoun Dib, Hélène Demarquette, Caroline Jacquet, Cécile Sonntag, Sophie Godet, Arnaud Jaccard, Pascal Lenain, Margaret Macro, Valentine Richez-Olivier, Mourad Tiab, Laure Vincent, Hacene Zerazhi, Marie-Odile Pétillon, Sandrine Rollet, Helene Gardeney, Geraldine Durand, Anthony Levy, Cyrille Touzeau, Aurore Perrot, Philippe Moreau, Thierry Facon, Jill Corre, Stephanie Ragot, Herve Avet-Loiseau, Xavier Leleu
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引用次数: 0
Immune reconstitution dynamics after unrelated allogeneic transplantation with post-transplant cyclophosphamide compared to classical immunosuppression with anti-thymocyte globulin: a prospective cohort study. 与使用抗胸腺细胞球蛋白的传统免疫抑制相比,使用移植后环磷酰胺进行非亲属异体移植后的免疫重建动态:一项前瞻性队列研究。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.285921
Mariana Nassif Kerbauy, Fernanda Agostini Rocha, Leonardo Javier Arcuri, Priscila Silva Cunegundes, Lucila Nassif Kerbauy, Clarisse Martins Machado, Andreza Alice Feitosa Ribeiro, Pinaki P Banerjee, Luciana Cavalheiro Marti, Nelson Hamerschlak

Post-transplant cyclophosphamide (PTCy) has contributed to the success of haploidentical hematopoietic stem cell transplantation (HSCT) and is also used in transplantation from matched donors. However, limited data on the immune reconstitution after this type of immunosuppression is available. We aimed to evaluate immune reconstitution after HSCT from unrelated donors, comparing anti-thymocyte globulin (ATG) and PTCy. Consecutive patients undergoing HSCT from unrelated donors and receiving either ATG or PTCy were prospectively included. Immune reconstitution analyses were performed by flow cytometry pre-transplant and on days 30, 60, 90, and 180 post-transplant. We included 36 patients, 20 in the ATG group and 16 in the PTCy group. In the early post-transplant period (day [d]+30), the ATG group showed a higher number of total lymphocytes, T, B, and natural killer (NK) cells compared to the PTCy group. However, at d+180, the PTCy group exhibited a higher number of B cells. On d+60 and d+90, the ATG group displayed higher number of NK cells CD56dim compared to the PTCy group, while on d+180, the PTCy group showed higher number of CD56-, CD16+, and, NKG2D+ NK cells. Naive CD4+, transition CD4+, and naive CD8+ T cells on d+60 were identified as risk factors for acute graft-versus-host disease grade 2-4, and a higher count of CD4+ memory cells on d+180 was identified as a risk factor for chronic graft-versus-host disease. In the context of unrelated allogeneic transplantation, immunosuppression with PTCy was associated with later B-, T- and NK-cell reconstitution compared to ATG.

移植后环磷酰胺为单倍体造血干细胞移植(HSCT)的成功做出了贡献,也被用于匹配供体的移植。然而,有关这种免疫抑制后免疫重建的数据十分有限。我们旨在评估非亲属供者造血干细胞移植后的免疫重建情况,比较抗胸腺细胞球蛋白(ATG)和移植后环磷酰胺(PTCy)。前瞻性地纳入了接受非亲缘供体造血干细胞移植并接受 ATG 或 PTCy 的连续患者。移植前和移植后第 30、60、90 和 180 天通过流式细胞术进行免疫重建分析。我们共纳入了 36 例患者,其中 ATG 组 20 例,PTCy 组 16 例。在移植后早期(D+30),ATG 组的总淋巴细胞、T、B 和 NK 细胞数量高于 PTCy 组。但在 D+180 时,PTCy 组的 B 细胞数量较多。在 D+60 和 D+90 时,ATG 组比 PTCy 组显示出更多的 CD56dim NK 细胞,而在 D+180 时,PTCy 组显示出更多的 CD56neg、CD16pos 和 NKG2Dpos NK 细胞。D+60时的幼稚CD4+、过渡CD4+和幼稚CD8+T细胞被确定为急性移植物抗宿主病(GVHD)II-IV级的危险因素,而D+180时CD4+记忆细胞数量较多被确定为慢性GVHD的危险因素。在非亲缘异体移植中,与 ATG 相比,PTCy 免疫抑制与 B、T 和 NK 细胞重建的时间较晚有关。
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引用次数: 0
HDAC6 inhibitors sensitize resistant t(11;14) multiple myeloma cells to a combination of bortezomib and BH3 mimetics. HDAC6抑制剂能使耐药的t(11;14)多发性骨髓瘤细胞对硼替佐米和BH3模拟物联合疗法敏感。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.286279
Cristina Florean, Manon Lernoux, Anne Lorant, Helene Losson, Guy Bormans, Michael Schnekenburger, Marc Diederich
{"title":"HDAC6 inhibitors sensitize resistant t(11;14) multiple myeloma cells to a combination of bortezomib and BH3 mimetics.","authors":"Cristina Florean, Manon Lernoux, Anne Lorant, Helene Losson, Guy Bormans, Michael Schnekenburger, Marc Diederich","doi":"10.3324/haematol.2024.286279","DOIUrl":"10.3324/haematol.2024.286279","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"784-790"},"PeriodicalIF":8.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11873690/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142390072","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Early death and intracranial hemorrhage prediction in acute promyelocytic leukemia: validation of a risk score in a cohort from an international consortium treated with chemotherapy plus all-trans retinoic acid. 急性早幼粒细胞白血病早期死亡和颅内出血预测:在一个国际联盟的化疗加ATRA队列中验证风险评分。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-03-01 DOI: 10.3324/haematol.2024.286338
Wellington F Silva, Haesook T Kim, Maria S Undurraga, Juan R Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A M Melo, Ana Beatriz F Gloria, Katia B B Pagnano, Elenaide C Nunes, Rosane Isabel Bittencourt, Ninoska Rojas, Shirley M Q Truyenque, Ana Ilda Ayala-Lugo, Ana Carolina Oliver, Lorena L Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M Fagundes, Bruno K L Duarte, Analí Pamela Mora-Alferez, Percy Ortiz, Jose Luis Untama, Martin S Tallman, Raul C Ribeiro, Arnold Ganser, Richard James Dillon, Peter J M Valk, Miguel A Sanz, Bob Löwenberg, Nancy Berliner, Eduardo M Rego
{"title":"Early death and intracranial hemorrhage prediction in acute promyelocytic leukemia: validation of a risk score in a cohort from an international consortium treated with chemotherapy plus all-<i>trans</i> retinoic acid.","authors":"Wellington F Silva, Haesook T Kim, Maria S Undurraga, Juan R Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A M Melo, Ana Beatriz F Gloria, Katia B B Pagnano, Elenaide C Nunes, Rosane Isabel Bittencourt, Ninoska Rojas, Shirley M Q Truyenque, Ana Ilda Ayala-Lugo, Ana Carolina Oliver, Lorena L Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M Fagundes, Bruno K L Duarte, Analí Pamela Mora-Alferez, Percy Ortiz, Jose Luis Untama, Martin S Tallman, Raul C Ribeiro, Arnold Ganser, Richard James Dillon, Peter J M Valk, Miguel A Sanz, Bob Löwenberg, Nancy Berliner, Eduardo M Rego","doi":"10.3324/haematol.2024.286338","DOIUrl":"10.3324/haematol.2024.286338","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"795-798"},"PeriodicalIF":8.2,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11873706/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The third form of thrombotic thrombocytopenic purpura shows up again: what is it, does it even exist, how to find out? Comment on "Acquired thrombotic thrombocytopenic purpura without detectable anti-ADAMTS13 antibodies: a possible underlying autoimmune mechanism" and on "The different faces of thrombotic thrombocytopenic purpura".
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-02-27 DOI: 10.3324/haematol.2025.287535
Bernhard Lammle

Not available.

{"title":"The third form of thrombotic thrombocytopenic purpura shows up again: what is it, does it even exist, how to find out? Comment on \"Acquired thrombotic thrombocytopenic purpura without detectable anti-ADAMTS13 antibodies: a possible underlying autoimmune mechanism\" and on \"The different faces of thrombotic thrombocytopenic purpura\".","authors":"Bernhard Lammle","doi":"10.3324/haematol.2025.287535","DOIUrl":"https://doi.org/10.3324/haematol.2025.287535","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143515445","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
Community respiratory viruses are generally well-tolerated in hematopoietic stem cell transplant recipients: a brief report from the TRANSPIRE study.
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2025-02-27 DOI: 10.3324/haematol.2024.287107
Meghan Haney, Arfa Ashraf, Kelly E Lake, Lauren Strecker, Kasiani C Myers, Christopher Towe, Laura Walkup, Jason Woods, Stephanie L Edwards, Richard Cooper, Leslie E Lehmann, Gabriel Salinas Cisneros, Jason L Freedman, K Scott Baker, Erin Doherty, Margaret L MacMillan, Samuel B Goldfarb, Stella M Davies, Jane Koo, Transpire Groups

Not available.

{"title":"Community respiratory viruses are generally well-tolerated in hematopoietic stem cell transplant recipients: a brief report from the TRANSPIRE study.","authors":"Meghan Haney, Arfa Ashraf, Kelly E Lake, Lauren Strecker, Kasiani C Myers, Christopher Towe, Laura Walkup, Jason Woods, Stephanie L Edwards, Richard Cooper, Leslie E Lehmann, Gabriel Salinas Cisneros, Jason L Freedman, K Scott Baker, Erin Doherty, Margaret L MacMillan, Samuel B Goldfarb, Stella M Davies, Jane Koo, Transpire Groups","doi":"10.3324/haematol.2024.287107","DOIUrl":"https://doi.org/10.3324/haematol.2024.287107","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143515230","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
期刊
Haematologica
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