Pub Date : 2024-03-01eCollection Date: 2024-01-01DOI: 10.4084/MJHID.2024.023
Unal Atas, Volkan Karakus, Erdal Kurtoglu
{"title":"Could the 3'UTR+101G>C Mutation Detected in Two Sibling Cases Be a Mutation Affecting the Clinical Presentation in Thalassemia Patients?","authors":"Unal Atas, Volkan Karakus, Erdal Kurtoglu","doi":"10.4084/MJHID.2024.023","DOIUrl":"10.4084/MJHID.2024.023","url":null,"abstract":"","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024023"},"PeriodicalIF":3.2,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10927219/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140101963","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}
Oral anticoagulants are widely used to treat or prevent cardiovascular diseases in millions of patients worldwide. They are the drugs of choice for stroke prevention and systemic embolism in patients with non-valvular atrial fibrillation and prosthetic heart valves, as well as for treatment/prevention of venous thromboembolism. Oral anticoagulants include vitamin K antagonists (VKAs) and direct oral anticoagulants (DOACs). The hemostasis laboratory plays a crucial role in the management of treated patients, spanning from dose adjustment based on laboratory testing that applies to VKAs to the measurement of drug concentrations in special situations that apply to DOACs. This article aims to overview how the hemostasis laboratory can help clinicians manage patients on oral anticoagulants. Special interest is devoted to the international normalized ratio, used to manage patients on VKAs and to the measurement of DOAC concentrations, for which the role of the laboratory is still not very well defined, and most interferences of DOACs with some of the most common hemostatic parameters are not widely appreciated.
{"title":"How the Hemostasis Laboratory Can Help Clinicians to Manage Patients on Oral Anticoagulants.","authors":"Armando Tripodi, Marigrazia Clerici, Erica Scalambrino, Pasquale Agosti, Paolo Bucciarelli, Flora Peyvandi","doi":"10.4084/MJHID.2024.027","DOIUrl":"10.4084/MJHID.2024.027","url":null,"abstract":"<p><p>Oral anticoagulants are widely used to treat or prevent cardiovascular diseases in millions of patients worldwide. They are the drugs of choice for stroke prevention and systemic embolism in patients with non-valvular atrial fibrillation and prosthetic heart valves, as well as for treatment/prevention of venous thromboembolism. Oral anticoagulants include vitamin K antagonists (VKAs) and direct oral anticoagulants (DOACs). The hemostasis laboratory plays a crucial role in the management of treated patients, spanning from dose adjustment based on laboratory testing that applies to VKAs to the measurement of drug concentrations in special situations that apply to DOACs. This article aims to overview how the hemostasis laboratory can help clinicians manage patients on oral anticoagulants. Special interest is devoted to the international normalized ratio, used to manage patients on VKAs and to the measurement of DOAC concentrations, for which the role of the laboratory is still not very well defined, and most interferences of DOACs with some of the most common hemostatic parameters are not widely appreciated.</p>","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024027"},"PeriodicalIF":3.2,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10927209/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140101965","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}
Pub Date : 2024-03-01eCollection Date: 2024-01-01DOI: 10.4084/MJHID.2024.017
Yi Wu, Xiaolin Yin, Kun Yang
{"title":"Protein S Deficiency with Recurrent Thromboembolism after Splenectomy in a Patient with Hemoglobin H Disease.","authors":"Yi Wu, Xiaolin Yin, Kun Yang","doi":"10.4084/MJHID.2024.017","DOIUrl":"10.4084/MJHID.2024.017","url":null,"abstract":"","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"16 1","pages":"e2024017"},"PeriodicalIF":3.2,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10927212/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140101995","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}
D. Avenoso, F. Serpenti, Liron Barnea Slonim, Styliani Bouziana, F. Dazzi, Guy Hannah, Michelle Kenyon, V. Mehra, Austin Kulasekararaj, P. Krishnamurthy, Mili Naresh Shah, Sharon Lionel, A. Pagliuca, V. Potter
Background: Allogeneic haematopoietic stem-cell transplant is a potentially curative option for high-risk acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) patients. Post-transplant cyclophosphamide administration allows for selection of haploidentical donors in patients who are eligible for the procedure, but do not have a fully matched donor, since it can overcome the HLA barrier. There is still an active debate on whether intensification of the conditioning regimen is necessary with haploidentical donors when peripheral blood stem cells are used as the source of the graft. Herein we report our decennial experience of haploidentical stem-cell transplant using peripheral blood stem cells at King’s College Hospital. Objectives: The primary objective was to evaluate overall survival (OS) for patients with less than two previous lines of therapy. Secondary objectives were total OS, OS according to cytomegalovirus (CMV) reactivation, incidence of transplant-related mortality (TRM), graft-versus-host disease (GVHD) and GVHD-relapse-free survival (GRFS). Results: One-year and three-year total OS were 62% and 43%, respectively, with a median OS of 22 months. One-year and three-year OS for patients with ≤2 and in patients with >2 previous lines of therapy were 72% and 55%, and 60% and 22%, respectively (p-value=0.04). The median OS in patients with >2 previous lines of therapy and ≤2 lines of therapy was 16 and 49 months, respectively. Cumulative incidence (CI) of relapse was 25% with a median time to relapse of 5 months (range 1 – 38 months). Conclusions: Haploidentical haematopoietic stem-cell transplant is potentially curative in chemo-sensitive AML and MDS and offers a high rate of prolonged remission. Our cohort further confirms the role of consolidative haploidentical transplant in patients in complete remission and highlights that patients with heavily pre-treated disease may not benefit from this strategy.
{"title":"HAPLOIDENTICAL TRANSPLANT WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE FOR ACUTE MYELOID LEUKAEMIA AND MYELODYSPLASTIC SYNDROMES PATIENTS: THE ROLE OF PREVIOUS LINES OF THERAPY.","authors":"D. Avenoso, F. Serpenti, Liron Barnea Slonim, Styliani Bouziana, F. Dazzi, Guy Hannah, Michelle Kenyon, V. Mehra, Austin Kulasekararaj, P. Krishnamurthy, Mili Naresh Shah, Sharon Lionel, A. Pagliuca, V. Potter","doi":"10.4084/mjhid.2024.002","DOIUrl":"https://doi.org/10.4084/mjhid.2024.002","url":null,"abstract":"Background: Allogeneic haematopoietic stem-cell transplant is a potentially curative option for high-risk acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) patients. Post-transplant cyclophosphamide administration allows for selection of haploidentical donors in patients who are eligible for the procedure, but do not have a fully matched donor, since it can overcome the HLA barrier. There is still an active debate on whether intensification of the conditioning regimen is necessary with haploidentical donors when peripheral blood stem cells are used as the source of the graft. Herein we report our decennial experience of haploidentical stem-cell transplant using peripheral blood stem cells at King’s College Hospital. Objectives: The primary objective was to evaluate overall survival (OS) for patients with less than two previous lines of therapy. Secondary objectives were total OS, OS according to cytomegalovirus (CMV) reactivation, incidence of transplant-related mortality (TRM), graft-versus-host disease (GVHD) and GVHD-relapse-free survival (GRFS). Results: One-year and three-year total OS were 62% and 43%, respectively, with a median OS of 22 months. One-year and three-year OS for patients with ≤2 and in patients with >2 previous lines of therapy were 72% and 55%, and 60% and 22%, respectively (p-value=0.04). The median OS in patients with >2 previous lines of therapy and ≤2 lines of therapy was 16 and 49 months, respectively. Cumulative incidence (CI) of relapse was 25% with a median time to relapse of 5 months (range 1 – 38 months). Conclusions: Haploidentical haematopoietic stem-cell transplant is potentially curative in chemo-sensitive AML and MDS and offers a high rate of prolonged remission. Our cohort further confirms the role of consolidative haploidentical transplant in patients in complete remission and highlights that patients with heavily pre-treated disease may not benefit from this strategy.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"56 19","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139126845","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}
Ugo Testa, F. D'Alo', E. Pelosi, G. Castelli, Giuseppe Leone
Follicular lymphoma is the second most diagnosed lymphoma in Western Europe. Significant advancements have considerably improved the survival of FL patients. However, 10-20% of these patients are refractory to standard treatments, and most of them will relapse. The treatment of follicular lymphoma patients with multiply relapsed or refractory disease represents an area of high-unmet needing new treatments with stronger efficacy. Chimeric antigen receptor (CAR)-T cell therapy targeting B-cell antigens, such as CD19 or CD20, is emerging as an efficacious treatment for R/R follicular lymphoma patients, particularly for those with early relapse and refractory to alkylating agents and to anti-CD20 monoclonal antibodies, resulting in a high rate of durable responses in a high proportion of patients.
滤泡性淋巴瘤是西欧第二大确诊淋巴瘤。医学的重大进步大大提高了滤泡性淋巴瘤患者的生存率。然而,10%-20%的患者对标准疗法难治,其中大多数会复发。治疗多次复发或难治的滤泡性淋巴瘤患者是一个亟需疗效更强的新疗法的领域。以 CD19 或 CD20 等 B 细胞抗原为靶点的嵌合抗原受体(CAR)-T 细胞疗法正在成为治疗复发/难治滤泡性淋巴瘤患者的有效疗法,特别是对于那些早期复发、对烷化剂和抗 CD20 单克隆抗体难治的患者,这种疗法能使很高比例的患者产生持久反应。
{"title":"CAR-T CELL THERAPY FOR FOLLICULAR LYMPHOMAS","authors":"Ugo Testa, F. D'Alo', E. Pelosi, G. Castelli, Giuseppe Leone","doi":"10.4084/mjhid.2024.012","DOIUrl":"https://doi.org/10.4084/mjhid.2024.012","url":null,"abstract":"Follicular lymphoma is the second most diagnosed lymphoma in Western Europe. Significant advancements have considerably improved the survival of FL patients. However, 10-20% of these patients are refractory to standard treatments, and most of them will relapse. The treatment of follicular lymphoma patients with multiply relapsed or refractory disease represents an area of high-unmet needing new treatments with stronger efficacy. Chimeric antigen receptor (CAR)-T cell therapy targeting B-cell antigens, such as CD19 or CD20, is emerging as an efficacious treatment for R/R follicular lymphoma patients, particularly for those with early relapse and refractory to alkylating agents and to anti-CD20 monoclonal antibodies, resulting in a high rate of durable responses in a high proportion of patients.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"50 8","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139127059","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}
Elena Amabile, Matteo Totaro, Luca Cappelli, Clara Minotti, Alessandra Micozzi
Central venous catheter-related infections are of particular importance in onco-hematological patients. Candida parapsilosis is generally reported as a mild pathogen, however it is able to effectively colonize intravascular devices and potentially give rise to sustained fungemias. Here we report a case of invasive, potentially lethal C. parapsilosis disseminated infection in a neutropenic patient affected by chronic myeloid leukemia with blast crisis. We underline the importance of removing the central venous catheter as potential source of infection as soon as possible during the course of candidemia, and not replacing it with other polyurethan intravascular devices, which pose a risk for the maintenance of the fungemia despite the administration of the best antifungal therapy available.
{"title":"A case of central venous catheter-related Candida parapsilosis fungemia evolved to disseminated infection in a neutropenic patient with blast crisis of chronic myeloid leukemia.","authors":"Elena Amabile, Matteo Totaro, Luca Cappelli, Clara Minotti, Alessandra Micozzi","doi":"10.4084/mjhid.2024.013","DOIUrl":"https://doi.org/10.4084/mjhid.2024.013","url":null,"abstract":"Central venous catheter-related infections are of particular importance in onco-hematological patients. Candida parapsilosis is generally reported as a mild pathogen, however it is able to effectively colonize intravascular devices and potentially give rise to sustained fungemias. Here we report a case of invasive, potentially lethal C. parapsilosis disseminated infection in a neutropenic patient affected by chronic myeloid leukemia with blast crisis. We underline the importance of removing the central venous catheter as potential source of infection as soon as possible during the course of candidemia, and not replacing it with other polyurethan intravascular devices, which pose a risk for the maintenance of the fungemia despite the administration of the best antifungal therapy available.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"14 5","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139127111","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}
V. De Sanctis, Ashraf T Soliman, S. Daar, Ploutarchos Tzoulis, Christos Kattamis
Background: Prediabetes and diabetes mellitus (DM) are complications in adult patients with transfusion dependent β-thalassemia (β-TDT), with their incidence increasing with age. Objective: This retrospective observational study describes the glycemic trajectories and evaluates predictive indices of β-cell function and insulin sensitivity/resistance in β-TDT patients with prediabetes, both in a steady state and during 3-h oral glucose tolerance test (OGTT), in order to identify patients at high risk for incipient diabetes. Setting: The study was mainly conducted at the Pediatric and Adolescent Outpatient Clinic, Quisisana Hospital, Ferrara (Italy) in collaboration with thalassemia referring centers across Italy. Patients: The study included 11 β-TDT (aged 15.11- 31.10 years) with history of prediabetes. Methods: The ADA criteria for the diagnosis of glucose dysregulation were adopted. Investigations included evaluation of plasma glucose levels and insulin secretion, analysis of glycemic trajectories and indices of β-cell function and insulin sensitivity/resistance assessed in steady state and during OGTT. Results: The duration of progression from prediabetes to DM, expressed in years, showed a positive direct correlation with corrected insulin response (CIR-30 = r: 0.7606, P: 0.0065), insulinogenic index (IGI 0-120 = r: 0.6121, P:0.045), oral disposition index (oDI = r: 0.7119, P:0.013), insulin growth factor-1 (IGF-1= r: 0.6246, P: 0.039) and an inverse linear correlation with serum ferritin (SF = r: -0.7197, P: 0.012). Conclusions: Progressive β-cell failure, peripheral resistance to the action of insulin and reduction of oDI were the principal factors responsible for the progression from prediabetes to incipient DM.
{"title":"CAN WE PREDICT INCIPIENT DIABETES MELLITUS IN PATIENTS WITH TRANSFUSION DEPENDENT β-THALASSEMIA (β-TDT) REFERRED WITH A HISTORY OF PREDIABETES?","authors":"V. De Sanctis, Ashraf T Soliman, S. Daar, Ploutarchos Tzoulis, Christos Kattamis","doi":"10.4084/mjhid.2024.005","DOIUrl":"https://doi.org/10.4084/mjhid.2024.005","url":null,"abstract":"Background: Prediabetes and diabetes mellitus (DM) are complications in adult patients with transfusion dependent β-thalassemia (β-TDT), with their incidence increasing with age. Objective: This retrospective observational study describes the glycemic trajectories and evaluates predictive indices of β-cell function and insulin sensitivity/resistance in β-TDT patients with prediabetes, both in a steady state and during 3-h oral glucose tolerance test (OGTT), in order to identify patients at high risk for incipient diabetes. Setting: The study was mainly conducted at the Pediatric and Adolescent Outpatient Clinic, Quisisana Hospital, Ferrara (Italy) in collaboration with thalassemia referring centers across Italy. Patients: The study included 11 β-TDT (aged 15.11- 31.10 years) with history of prediabetes. Methods: The ADA criteria for the diagnosis of glucose dysregulation were adopted. Investigations included evaluation of plasma glucose levels and insulin secretion, analysis of glycemic trajectories and indices of β-cell function and insulin sensitivity/resistance assessed in steady state and during OGTT. Results: The duration of progression from prediabetes to DM, expressed in years, showed a positive direct correlation with corrected insulin response (CIR-30 = r: 0.7606, P: 0.0065), insulinogenic index (IGI 0-120 = r: 0.6121, P:0.045), oral disposition index (oDI = r: 0.7119, P:0.013), insulin growth factor-1 (IGF-1= r: 0.6246, P: 0.039) and an inverse linear correlation with serum ferritin (SF = r: -0.7197, P: 0.012). Conclusions: Progressive β-cell failure, peripheral resistance to the action of insulin and reduction of oDI were the principal factors responsible for the progression from prediabetes to incipient DM.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"13 10","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139129118","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}
I. Gur, Roei Tounek, Yaniv Dotan, Elite Vainer Evgrafov, Stav Rakedzon, Eyal Fuchs
Background Hospitalized hematological patients often require bronchoalveolar lavage (BAL). Paucity of evidence exists as to the potential risks in patients with very-severe thrombocytopenia (VST). Methods This retrospective-cohort study included adult hematological in-patients with VST, defined as platelets<20x103/μL, undergoing BAL during 2012-2021. Mechanically-ventilated patients or those with known active bleeding were excluded. Primary outcomes included major bleeding halting the BAL or deemed significant by the treating physician, need for any respiratory support other than low flow O2 or death within 24 hours. Any other bleeding were recorded as secondary outcomes. Results Of the 507 patients included in the final analysis, the 281 patients with VST had lower hemoglobin (Md=0.3, p=0.003), longer prothrombin-time (Md=0.7s, p=0.025), higher chances of preprocedural platelet transfusion (RR 3.68, 95%CI[2.86,4.73]), and only one primary-outcome event (death of septic shock 21h postprocedurally) - compared with 3 (1.3%) events (two bleedings halting procedure and one need for non-invasive-ventilation) in patients with platelets ≥20x103/μL (p=0.219). Risk of minor, spontaneously resolved bleeding was higher (RR=3.217, 95%CI[0.919,11.262]) in patients with VST (4.3% vs 1.3%, p=0.051). No association was found between any of the complications recorded and preprocedural platelets, age, aPTT, PT, hematological status, or platelet transfusion. Conclusions This data suggests BAL to be safe even when platelet counts are <20x103/μL.
背景住院血液病患者经常需要进行支气管肺泡灌洗(BAL)。有关极重度血小板减少症(VST)患者潜在风险的证据不足。 方法 这项回顾性队列研究纳入了 2012-2021 年期间接受 BAL 的 VST(定义为血小板<20x103/μL)成人血液病住院患者。机械通气患者或已知有活动性出血的患者除外。主要结果包括导致 BAL 中止或主治医生认为严重的大出血、需要低流量氧气以外的任何呼吸支持或 24 小时内死亡。其他任何出血情况均作为次要结果记录在案。 结果 在纳入最终分析的 507 名患者中,281 名 VST 患者的血红蛋白较低(Md=0.3,p=0.003),凝血酶原时间较长(Md=0.7s,p=0.025),术前输注血小板的几率较高(RR 3.68,95%CI[2.86,4.相比之下,血小板≥20x103/μL的患者有3次(1.3%)事件(两次出血导致手术中止,一次需要无创通气)(P=0.219)。VST患者发生轻微、自发缓解出血的风险更高(RR=3.217,95%CI[0.919,11.262])(4.3% vs 1.3%,P=0.051)。未发现任何并发症与术前血小板、年龄、aPTT、PT、血液学状况或血小板输注有关。 结论 这些数据表明,即使血小板计数小于 20x103/μL,BAL 也是安全的。
{"title":"SAFETY OF BRONCHOALVEOLAR LAVAGE IN HEMATOLOGICAL PATIENTS WITH THROMBOCYTOPENIA – A RETROSPECTIVE COHORT STUDY","authors":"I. Gur, Roei Tounek, Yaniv Dotan, Elite Vainer Evgrafov, Stav Rakedzon, Eyal Fuchs","doi":"10.4084/mjhid.2024.006","DOIUrl":"https://doi.org/10.4084/mjhid.2024.006","url":null,"abstract":"Background Hospitalized hematological patients often require bronchoalveolar lavage (BAL). Paucity of evidence exists as to the potential risks in patients with very-severe thrombocytopenia (VST). Methods This retrospective-cohort study included adult hematological in-patients with VST, defined as platelets<20x103/μL, undergoing BAL during 2012-2021. Mechanically-ventilated patients or those with known active bleeding were excluded. Primary outcomes included major bleeding halting the BAL or deemed significant by the treating physician, need for any respiratory support other than low flow O2 or death within 24 hours. Any other bleeding were recorded as secondary outcomes. Results Of the 507 patients included in the final analysis, the 281 patients with VST had lower hemoglobin (Md=0.3, p=0.003), longer prothrombin-time (Md=0.7s, p=0.025), higher chances of preprocedural platelet transfusion (RR 3.68, 95%CI[2.86,4.73]), and only one primary-outcome event (death of septic shock 21h postprocedurally) - compared with 3 (1.3%) events (two bleedings halting procedure and one need for non-invasive-ventilation) in patients with platelets ≥20x103/μL (p=0.219). Risk of minor, spontaneously resolved bleeding was higher (RR=3.217, 95%CI[0.919,11.262]) in patients with VST (4.3% vs 1.3%, p=0.051). No association was found between any of the complications recorded and preprocedural platelets, age, aPTT, PT, hematological status, or platelet transfusion. Conclusions This data suggests BAL to be safe even when platelet counts are <20x103/μL.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"19 2","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139125967","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}
G. Bagnato, V. Stefoni, A. Broccoli, L. Argnani, C. Pellegrini, B. Casadei, Francesca Bonifazi, P. Zinzani
We report the case of 2 patients with relapsed/refractory peripheral T-cell lymphoma treated with valemetostat tosilate, a selective dual inhibitor of histone-lysine N-methyltransferases enhancer of zeste homolog 1 and 2, and subsequently bridged to allogeneic stem cell transplantation. Valemetostat led to a quick response and was well tolerated, offering as a promising bridge therapy to transplantation for patients with relapsed/refractory peripheral T-cell lymphoma which is still an unmet medical need.
我们报告了2例复发/难治性外周T细胞淋巴瘤患者接受valemetostat tosilate(一种组蛋白-赖氨酸N-甲基转移酶zeste同源增强子1和2的选择性双重抑制剂)治疗并随后接受同种异体干细胞移植的病例。Valemetostat 可使患者迅速产生反应,且耐受性良好,有望成为复发/难治性外周 T 细胞淋巴瘤患者进行移植的桥接疗法。
{"title":"Successful Bridging to Allogeneic Transplantation With Valemetostat in Two Refractory/relapsed Peripheral T-cell lymphoma patients","authors":"G. Bagnato, V. Stefoni, A. Broccoli, L. Argnani, C. Pellegrini, B. Casadei, Francesca Bonifazi, P. Zinzani","doi":"10.4084/mjhid.2024.004","DOIUrl":"https://doi.org/10.4084/mjhid.2024.004","url":null,"abstract":"We report the case of 2 patients with relapsed/refractory peripheral T-cell lymphoma treated with valemetostat tosilate, a selective dual inhibitor of histone-lysine N-methyltransferases enhancer of zeste homolog 1 and 2, and subsequently bridged to allogeneic stem cell transplantation. Valemetostat led to a quick response and was well tolerated, offering as a promising bridge therapy to transplantation for patients with relapsed/refractory peripheral T-cell lymphoma which is still an unmet medical need.","PeriodicalId":18498,"journal":{"name":"Mediterranean Journal of Hematology and Infectious Diseases","volume":"18 6","pages":""},"PeriodicalIF":3.2,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139129875","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}