Acta HaematologicaPub Date : 2025-01-01Epub Date: 2024-05-24DOI: 10.1159/000539448
Yeyi Yang, Yezhen Yang, Ye Meng, Lihua Huang, Zuocheng Yang
{"title":"Novel Compound Heterogeneous Mutations in CYB5R3 Gene Leading to Methemoglobinemia (Type I) in a Chinese Boy: Case Report and Relevant Comprehensive Analysis.","authors":"Yeyi Yang, Yezhen Yang, Ye Meng, Lihua Huang, Zuocheng Yang","doi":"10.1159/000539448","DOIUrl":"10.1159/000539448","url":null,"abstract":"<p><strong>Introduction: </strong>Recessive congenital methemoglobinemia (RCM) caused by CYB5R3 deficiency due to the mutations in the reduced nicotinamide adenine dinucleotide (NADH) cytochrome b5 reductase (CYB5R) gene is an autosomal recessive inherited disease. Clinically, it can be divided into two types, namely red blood cell affected type (RCM I) and systemically affected type (RCM II).</p><p><strong>Case presentation: </strong>A 5-year-old male patient was diagnosed with cyanosis for 5 years. Physical examination showed cyanosis in areas such as the lips, fingers, and toes. Laboratory examination revealed low pulse oxygen saturation (81%) and increased blood methemoglobin (23.6%). Gene testing revealed the compound heterozygous mutations in the CYB5R3 gene, c.149G>A (p.Arg50Gln) and c.331A>G (p.Lys111Glu), respectively originating from his parents. By constructing 3D models of CYB5R3 wild-type and mutant types using SWISS-MODEL software, it was found that the mutation caused significant structural abnormalities in the CYB5R protein. The relationship between CYB5R3 gene mutation sites, amino acid change, enzyme activity, and methemoglobinemia type I and II were listed and analyzed.</p><p><strong>Conclusion: </strong>A case of congenital RCM type I caused by compound heterozygous mutations in the CYB5R3 gene was reported, with c.331A>G (p.Lys111Glu) being the newly reported mutation. The homozygosity or heterozygosity of CYB5R3 gene mutations that lead to premature termination, loss of exons, and change in amino acid properties in FAD or NADH binding domains, is positively correlated with the severity (from type I to type II) of methemoglobinemia.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"226-232"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141086453","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}
Acta HaematologicaPub Date : 2025-01-01Epub Date: 2024-05-10DOI: 10.1159/000539127
Julie Barberio, Timothy L Lash, Ajay K Nooka, Ashley I Naimi, Rachel E Patzer, Christopher Kim
{"title":"Real-World Risk of Severe Cytopenias in Multiple Myeloma Patients Sequentially Treated with Immunomodulatory Drugs.","authors":"Julie Barberio, Timothy L Lash, Ajay K Nooka, Ashley I Naimi, Rachel E Patzer, Christopher Kim","doi":"10.1159/000539127","DOIUrl":"10.1159/000539127","url":null,"abstract":"<p><strong>Introduction: </strong>Most multiple myeloma (MM) patients experience cytopenias, likely driven by both disease and treatment-related factors. Immunomodulatory agents (IMiDs), which form the backbone of most anti-myeloma regimens, are known to cause higher grade cytopenias. In this context, the impact of sequential IMiD treatments on cytopenia risk is unknown.</p><p><strong>Methods: </strong>We evaluated the cumulative risks of severe cytopenias following second line of therapy (LOT) initiation in 5,573 MM patients in the Flatiron Health database. Patients for whom both LOTs 1 and 2 contained IMiDs were considered \"sequentially exposed\"; those for whom neither contained IMiDs were \"never exposed.\"</p><p><strong>Results: </strong>For the neutropenia outcome, compared to the never exposed, the sequentially exposed had the highest 1-year risk (risk difference [RD] 12%), followed by those only recently exposed during LOT 2 (RD 8%), then by those with only past exposure during LOT 1 (RD 5%). A similar pattern was observed for leukopenia, but no meaningful differences were observed for anemia or thrombocytopenia. The associations between sequential exposure, versus never, with neutropenia and leukopenia were even stronger among those with a recent cytopenia history.</p><p><strong>Conclusion: </strong>Results suggest that sequential exposure to IMiDs is a risk factor for higher grade cytopenias. These findings have profound clinical implications in choosing newer LOTs with potential risks of cytopenia.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"135-147"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140911212","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}
Acta HaematologicaPub Date : 2025-01-01Epub Date: 2024-05-28DOI: 10.1159/000539435
Xiangyou Zeng, Kaifan Liu, Ruohao Xu, Lenghe Zhang, Peilong Lai, Xin Du, Jianyu Weng
{"title":"TES and SLC40A1 as Potential Biomarkers for Predicting Survival in T-Cell Acute Lymphoblastic Leukemia.","authors":"Xiangyou Zeng, Kaifan Liu, Ruohao Xu, Lenghe Zhang, Peilong Lai, Xin Du, Jianyu Weng","doi":"10.1159/000539435","DOIUrl":"10.1159/000539435","url":null,"abstract":"<p><strong>Introduction: </strong>Identifying patients with high-risk T-cell acute lymphoblastic leukemia (T-ALL) is crucial for personalized therapy; however, the lack of robust biomarkers hinders prognosis assessment. To address this issue, our study aimed to screen and validate genes whose expression may serve as predictive indicators of outcomes in T-ALL patients while also investigating the underlying molecular mechanisms.</p><p><strong>Methods: </strong>Differentially expressed genes (DEGs) between T-ALL patients and healthy controls were identified by integrating data from three independent public datasets. Functional annotation of these DEGs and protein-protein interactions were also conducted. Further, we enrolled a prospective cohort of T-ALL patients (n = 20) at our center, conducting RNA-seq analysis on their bone marrow samples. Survival-based univariate Cox analysis was employed to identify gene expressions related to survival, and an intersection algorithm was sequentially applied. Furthermore, we validated the identified genes using cases from the Therapeutically Applicable Research to Generate Effective Treatments database, plotting Kaplan-Meier curves for secondary validation.</p><p><strong>Results: </strong>Through the integration of survival-related genes with DEGs identified in T-ALL, our analysis revealed six T-ALL-specific genes, the expression levels of which were linked to prognostic value. Notably, the independent prognostic value of SLC40A1 and TES expression levels was confirmed in both an external cohort and a prospective cohort at our center.</p><p><strong>Conclusion: </strong>In summary, our preliminary study indicates that the expression levels of TES and SLC40A1 genes show promise as potential indicators for predicting survival outcomes in T-ALL patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"163-179"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141160096","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}
Acta HaematologicaPub Date : 2025-01-01Epub Date: 2024-05-20DOI: 10.1159/000538836
Debora S Bruno, Manoj Khanal, Xiaohong I Li, Maricer P Escalon, Katherine B Winfree, Lisa M Hess
{"title":"Racial and Ethnic Characteristics and Outcomes of Patients Diagnosed with CLL/SLL in the USA.","authors":"Debora S Bruno, Manoj Khanal, Xiaohong I Li, Maricer P Escalon, Katherine B Winfree, Lisa M Hess","doi":"10.1159/000538836","DOIUrl":"10.1159/000538836","url":null,"abstract":"<p><strong>Introduction: </strong>This study was designed to compare outcomes among patients by race and ethnicity in the post-covalent Bruton tyrosine kinase inhibitor (cBTKi) treatment era.</p><p><strong>Methods: </strong>A nationwide electronic health record (EHR)-derived de-identified database was utilized that included patients diagnosed with CLL from 2013 to 2022 who received systemic therapy for their disease. Use of cBTKi therapy, time to next treatment or death (TTNT-D), and overall survival (OS) were compared by race in unadjusted (Kaplan-Meier method) and adjusted analyses (Cox proportional hazards regression).</p><p><strong>Results: </strong>This study included 4,572 White (71.8%) and 558 Black (8.8%) patients with CLL; 270 were Hispanic or Latino (4.2%). Patients who were Black were significantly younger, more were female, had later stage disease, were of lower socioeconomic status (SES), and were more likely to have unmutated immunoglobulin heavy chain gene (IGHV) and to have received cBTKi therapy than White patients (all p ≤ 0.002). SES was also significantly different by ethnicity. TTNT-D and OS were not different by race in either unadjusted or adjusted analyses (all p > 0.05).</p><p><strong>Conclusion: </strong>In unadjusted and adjusted analyses, TTNT-D and OS were not different by race. These data did not identify racial healthcare disparities in the era following the introduction of cBTKi therapy despite differences in baseline characteristics.</p><p><strong>Introduction: </strong>This study was designed to compare outcomes among patients by race and ethnicity in the post-covalent Bruton tyrosine kinase inhibitor (cBTKi) treatment era.</p><p><strong>Methods: </strong>A nationwide electronic health record (EHR)-derived de-identified database was utilized that included patients diagnosed with CLL from 2013 to 2022 who received systemic therapy for their disease. Use of cBTKi therapy, time to next treatment or death (TTNT-D), and overall survival (OS) were compared by race in unadjusted (Kaplan-Meier method) and adjusted analyses (Cox proportional hazards regression).</p><p><strong>Results: </strong>This study included 4,572 White (71.8%) and 558 Black (8.8%) patients with CLL; 270 were Hispanic or Latino (4.2%). Patients who were Black were significantly younger, more were female, had later stage disease, were of lower socioeconomic status (SES), and were more likely to have unmutated immunoglobulin heavy chain gene (IGHV) and to have received cBTKi therapy than White patients (all p ≤ 0.002). SES was also significantly different by ethnicity. TTNT-D and OS were not different by race in either unadjusted or adjusted analyses (all p > 0.05).</p><p><strong>Conclusion: </strong>In unadjusted and adjusted analyses, TTNT-D and OS were not different by race. These data did not identify racial healthcare disparities in the era following the introduction of cBTKi therapy despite differences in baseline characteristics.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"148-162"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11935746/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070244","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}
{"title":"Novel Approaches of Cellular Therapy in Multiple Myeloma: Focus on Chimeric Antigen Receptor T-Cells.","authors":"Ravi Kishore Narra, Supriya Peshin, Binod Dhakal","doi":"10.1159/000543265","DOIUrl":"10.1159/000543265","url":null,"abstract":"<p><strong>Background: </strong>Recent advancements in cellular therapies, particularly chimeric antigen receptor T-cells (CAR-T) and T-cell-engaging bispecific antibodies have significantly altered the therapeutic landscape for multiple myeloma. There are two US FDA approved CAR-T products targeting BCMA available for commercial use at this time. Though these innovative therapies have demonstrated considerable efficacy in heavily pretreated multiple myeloma patients, many challenges remain, including accessibility, potential toxicities such as cytokine release syndrome and neurotoxicity and development of resistance through targeted antigen loss and T-cell exhaustion and various other mechanisms. CRISPR edited allogeneic CAR-T cells, CAR-NK cells, and structural makeover of autologous CART with safety switches are being studied to address current limitations in cellular therapy. Additionally, newer target antigens such as GPRC5D, FcRH5, armored CAR-T cells that resist immunosuppressive cytokines such as TGF-β are being investigated.</p><p><strong>Summary: </strong>This review summarizes safety and efficacy of currently available CART, discusses challenges with these therapies, and ongoing research efforts aimed at addressing resistance, mitigate treatment-related toxicities, and refining for broader applicability and prolonged efficacy.</p><p><strong>Key messages: </strong>CART cell therapy has shown significant benefit in treatment of multiple myeloma. Many challenges persist. Novel strategies with structural modifications are being incorporated to overcome the limitations.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2024-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142902550","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}
Idan Goldberg, Galia Spectre, Pia Raanani, Hugo Ten Cate, Avi Leader
{"title":"Clinical Challenges in Treating Cancer-Associated Thrombosis: A Clinically Oriented Review.","authors":"Idan Goldberg, Galia Spectre, Pia Raanani, Hugo Ten Cate, Avi Leader","doi":"10.1159/000542872","DOIUrl":"10.1159/000542872","url":null,"abstract":"<p><strong>Background: </strong>Managing cancer-associated thrombosis (CAT) is a significant clinical challenge due to several factors such as increased bleeding tendency, frailty, and drug-drug interactions. For many years, the drug of choice for treating CAT was low molecular weight heparin (LMWH). Recently, direct oral anticoagulants (DOACs) entered to the therapeutic milieu of CAT. However, due to the large diversity among patients with CAT in clinical and laboratory characteristics, not all patients will equally benefit from treatment with DOACs. Furthermore, several subgroups of patients with CAT have specific characteristics that influence the anticoagulant decision-making process.</p><p><strong>Summary: </strong>In this review, we present four different theoretical clinical case scenarios, each representing a different challenge that is associated with thrombosis management; brain metastasis, malignancies of the gastrointestinal tract, drug-drug interactions (DDIs), and thrombocytopenia. By reviewing current literature, we suggest our clinical approach for managing these cases in the era of DOACs.</p><p><strong>Key messages: </strong>(1) The management of patients with brain tumors and CAT is challenging due to increased risk for both intracranial hemorrhage and recurrent venous thromboembolism. Both LMWH and DOACs are optional treatment in this setting. (2) There are conflicting data regarding the bleeding risk in patients with GI malignancies. Treatment with LMWH should be considered specifically in patients with advanced disease and unresectable tumors. (3) There is a paucity of data regarding DDI in patients with CAT. However, caution should be exercised when prescribing DOACs to patients receiving concurrent medications that either affect DOAC metabolism or influence bleeding risk. (4) The management of patients with CAT and thrombocytopenia depends on the severity of thrombocytopenia and the timing of the thrombotic event.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-17"},"PeriodicalIF":1.7,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142811911","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}
Gilad Itchaki, Ohad Benjamini, Mor Levi, Anatoly Nemets, Shai Ygna, Mahdi Assaly, Anna Gourevitch, Moshe Gatt, Revital Saban, Pia Raanani, Iuliana Vaxman
{"title":"\"Real-Life\" Data of Zanubrutinib in Patients with Waldenström Macroglobulinemia: A Multicenter Retrospective Study.","authors":"Gilad Itchaki, Ohad Benjamini, Mor Levi, Anatoly Nemets, Shai Ygna, Mahdi Assaly, Anna Gourevitch, Moshe Gatt, Revital Saban, Pia Raanani, Iuliana Vaxman","doi":"10.1159/000542936","DOIUrl":"10.1159/000542936","url":null,"abstract":"<p><strong>Introduction: </strong>Waldenström macroglobulinemia (WM) is a rare indolent lymphoma. Zanubrutinib (ZAN), a second-generation BTK inhibitor, has been approved for the treatment of WM in any line of therapy in 2021. Between November 2020 and January 2022, an expanded access program of ZAN opened in Israel for the treatment of patients with relapsed/refractory (R/R)-WM or those ineligible for chemotherapy or ibrutinib in first line.</p><p><strong>Methods: </strong>This is a multicenter retrospective study aiming to provide real-world data on ZAN in patients with WM in Israel. Demographic and clinical data were collected and coded from electronic files. Response was evaluated by the investigator's assessment. As the program closed, patients transitioned to commercial ZAN.</p><p><strong>Results: </strong>Thirteen patients (12 R/R; 1 treatment-naive) were enrolled across 8 centers in Israel. The median age at ZAN initiation was 71 years (range, 50-85); 6 were males; 10 had high IPSS-WM. R/R patients had a median of 1 (1-4) prior lines of therapy. Other than progressive disease after chemoimmunotherapy, the most common considerations for choosing ZAN were patients' age and/or comorbidities (n = 5), as well as ibrutinib toxicity. The initial ZAN dose was reduced in 4 patients. The median time on ZAN was 19.5 months (2.9-29.5). Of 12 evaluable patients, the ORR was 83% with 3 minor responses, 6 PRs, and 1 VGPR. With a median follow-up of 19.6 months, 7 patients were still on ZAN, 5 progressed, 4 while on ZAN, and 1 after ZAN was stopped due to AE. Eighteen-month PFS and OS were 60.5% and 77%, respectively. Eight (61%) patients had AEs of any grade, and 3 (23%) of grade 3-4; 2 stopped ZAN due to congestive heart failure and extreme fatigue.</p><p><strong>Conclusion: </strong>The results of this real-world high-risk population are consistent with prospective studies highlighting the efficacy and safety of ZAN.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-6"},"PeriodicalIF":1.7,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142779024","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":"Haploidentical Allogeneic Hematopoietic Cell Transplantation following Two Courses of Venetoclax and Azacytidine Therapy in Patients over 55 Years Old with Acute Myelogenous Leukemia: Comment.","authors":"Hinpetch Daungsupawong, Viroj Wiwanitkit","doi":"10.1159/000542843","DOIUrl":"10.1159/000542843","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-2"},"PeriodicalIF":1.7,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142765464","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}
Osnat Itzhaki Ben Zadok, Iuliana Vaxman, Sara Hoss, Yeela Talmor-Barkan, Tali Steinmetz, Pia Raanani, Ran Kornowski, Ashraf Hamdan
{"title":"Coronary Artery Disease and Microvascular Ischemia in Patients with Cardiac Amyloidosis.","authors":"Osnat Itzhaki Ben Zadok, Iuliana Vaxman, Sara Hoss, Yeela Talmor-Barkan, Tali Steinmetz, Pia Raanani, Ran Kornowski, Ashraf Hamdan","doi":"10.1159/000542510","DOIUrl":"10.1159/000542510","url":null,"abstract":"<p><strong>Introduction: </strong>The prevalence of preexisting obstructive coronary artery disease (CAD) and the occurrence of anginal chest pain as a presenting symptom in patients with light-chain (AL) and transthyretin (ATTR) cardiac amyloidosis (CA) are undetermined.</p><p><strong>Methods: </strong>A single-center analysis of clinical, laboratory, imaging, and angiographic characteristics of CA cohort was performed.</p><p><strong>Results: </strong>Included were 98 CA patients (43 AL, 47 wtATTR, 8 mutant ATTR). Eighteen patients (18%) had preexisting obstructive CAD at the time of CA diagnosis. These patients were older and had worse left ventricular ejection fraction, yet revealed similar cardiac biomarkers' levels. The 3-year survival rate was comparable between patients with versus without preexisting CAD (p = 0.974). Anginal chest pain was a presenting symptom of newly diagnosed CA in 24% of patients (n = 19) with no preexisting CAD, 53% (n = 10) of which had AL-CA. Two patients had an acute myocardial infarction. The prevalence of diabetes mellitus, dyslipidemia, hypertension, and smoking was similar among CA patients presenting with versus without chest pain. Of the newly diagnosed CA patients with no preexisting CAD who underwent symptoms evaluation (n = 37), 99mTc-Sestamibi myocardial perfusion scintigraphy demonstrated stress-induced perfusion defects in 45% (9/20) and normal study in 45% (9/20) of patients. Coronary evaluation revealed nonobstructive coronary artery lesions or normal coronaries in 75% of patients (18/24).</p><p><strong>Conclusion: </strong>CA patients may initially present with anginal chest pain and myocardial perfusion defects which may reflect coronary microvascular ischemia. CA should be considered in the differential diagnosis of patients presenting with chest pain, nonobstructive CAD, and elevated cardiac biomarkers.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142714996","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}
Muhammad Faisal Aslam, Asfand Yar Cheema, Daniyal Shahid, Bibi Maryam, Debduti Mukhopadhyay, Mishaal Munir, Ali Najam, Hossam M Ali, Qaiser Bashir, Faiz Anwer
{"title":"Historical Perspective of Allogeneic Hematopoietic Stem Cell Transplantation for Multiple Myeloma.","authors":"Muhammad Faisal Aslam, Asfand Yar Cheema, Daniyal Shahid, Bibi Maryam, Debduti Mukhopadhyay, Mishaal Munir, Ali Najam, Hossam M Ali, Qaiser Bashir, Faiz Anwer","doi":"10.1159/000542704","DOIUrl":"10.1159/000542704","url":null,"abstract":"<p><strong>Background: </strong>Advances in novel therapies have improved outcomes for multiple myeloma (MM) patients and the use of allo-SCT has decreased. Current guidelines no longer support allo-SCT as consolidation therapy for newly diagnosed MM, even in high-risk cases.</p><p><strong>Summary: </strong>Allo-SCT is now typically considered only within clinical trials for young, high-risk patients with relapsed or refractory MM (RRMM). It has not proven favorable despite its historical use. CAR T-cell therapy and bispecific antibodies have shown promise in treating triple- and penta-exposed/refractory MM, yet relapse remains common with poor survival rates. The efficacy of allo-SCT following BCMA-directed therapy and other new T-cell-directed therapies is unclear. Allo-SCT might be a viable option for eligible patients who relapse after these therapies, or where such options are unavailable. Advancements in reduced-intensity conditioning regimens have led to lower toxicity and transplant-related (TR) morbidity, lower graft-versus-host disease (GvHD), and TR mortality. Expanded use of alternative donors, like haploidentical donors, has yielded comparable outcomes. Better post-transplant GvHD regimens and maintenance strategies to prevent relapse have been developed.</p><p><strong>Key messages: </strong>This review analyzes available literature to better understand the safety, efficacy, and current role of allo-SCT in managing MM. Newer regimens are needed as routine use of allo-SCT cannot be recommended.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":1.7,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142714998","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}