Hematology Reports最新文献

Gene Therapy: A Revolutionary Step in Treating Thalassemia. 基因疗法：治疗地中海贫血症的革命性一步。

IF 1.1

Hematology Reports Pub Date : 2024-10-21 DOI: 10.3390/hematolrep16040064

Jhancy Malay, Rasha Aziz Attia Salama, Ghania Shehzad Alam Qureshi, Ali Raafat Ali Ahmed Ammar, Gayatri Janardhan, Maryam Safdar, Hesham Amin Hamdy Elshamy

{"title":"Gene Therapy: A Revolutionary Step in Treating Thalassemia.","authors":"Jhancy Malay, Rasha Aziz Attia Salama, Ghania Shehzad Alam Qureshi, Ali Raafat Ali Ahmed Ammar, Gayatri Janardhan, Maryam Safdar, Hesham Amin Hamdy Elshamy","doi":"10.3390/hematolrep16040064","DOIUrl":"https://doi.org/10.3390/hematolrep16040064","url":null,"abstract":"Beta thalassemia is an inherited blood disorder that results in inefficient erythropoiesis due to genetic mutation that leads to the reduction or absence of the hemoglobin beta-globulin protein. Approximately 8.5% of UAE residents suffer from β-thalassemia, a significant health and financial problem. The treatment options available for β-Thalassemia major are limited and associated with a wide range of complications. β-thalassemia gene therapy is emerging as a potential novel treatment option that eliminates the complications caused by the current long-term treatment modalities and the associated economic burden. This paper reviews the scientific literature related to emerging gene therapy for β-Thalassemia by analyzing all the articles published from January 2010 to December 2023 in the English language on Databases like PubMed, Scopus, ProQuest, and CINAHL. The use of gene therapy has demonstrated promising outcomes for a permanent cure of β-Thalassemia. To conclude, gene therapy is an innovative solution. It demonstrates a promising future, but does come with its own setbacks and is something that must be tackled in order to revolutionize it in the medical world. FDA-approved ZYNTEGLO is a potentially one-time curative treatment for β-Thalassemia. Although cutting-edge, its use is limited because of the high cost-a price of USD 2.8 million per patient.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503351/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499158","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

IF 1.1

Hematology Reports Pub Date : 2024-10-18 DOI: 10.3390/hematolrep16040063

Jakub Misiak, Bernard Sokołowski, Norbert Skrobisz, Mateusz Matczak, Marcin Braun

{"title":"Pancytopenia Related to Splenic Angiosarcoma: A Case Report and Literature Review.","authors":"Jakub Misiak, Bernard Sokołowski, Norbert Skrobisz, Mateusz Matczak, Marcin Braun","doi":"10.3390/hematolrep16040063","DOIUrl":"https://doi.org/10.3390/hematolrep16040063","url":null,"abstract":"Background: Angiosarcomas are highly aggressive malignancies with endothelial differentiation, presenting considerable challenges in oncology, especially when arising in rare locations such as the spleen. These tumors predominantly affect adults and are commonly found in the skin, breast, liver, or soft tissues, with more unusual occurrences in other organs. Angiosarcomas have a high propensity for metastasis, typically spreading to the liver, lungs, lymph nodes, and gastrointestinal tract. Splenic angiosarcoma, with fewer than 300 documented cases, is an especially rare and complex form of this malignancy.Case presentation: This report details a case of splenic angiosarcoma in a 45-year-old male, where bone marrow metastases were the first clinical presentation, initially mimicking myelodysplastic syndrome (MDS) due to persistent pancytopenia.Conclusions: The eventual identification of the splenic origin underscores the diagnostic difficulties and clinical challenges inherent in managing such atypical and rare presentations.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503393/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499161","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Trabecular Attenuation of L1 in Adult Patients with Multiple Myeloma: An Observational Study on Low-Dose CT Images. 多发性骨髓瘤成人患者 L1 小梁衰减：低剂量 CT 图像观察研究

IF 1.1

Hematology Reports Pub Date : 2024-10-17 DOI: 10.3390/hematolrep16040061

Carlo Augusto Mallio, Valeria Tomarchio, Francesco Pulcini, Edoardo Verducci, Caterina Bernetti, Maria Antonietta Tafuri, Federico Greco, Luigi Rigacci, Bruno Beomonte Zobel, Ombretta Annibali

{"title":"Trabecular Attenuation of L1 in Adult Patients with Multiple Myeloma: An Observational Study on Low-Dose CT Images.","authors":"Carlo Augusto Mallio, Valeria Tomarchio, Francesco Pulcini, Edoardo Verducci, Caterina Bernetti, Maria Antonietta Tafuri, Federico Greco, Luigi Rigacci, Bruno Beomonte Zobel, Ombretta Annibali","doi":"10.3390/hematolrep16040061","DOIUrl":"https://doi.org/10.3390/hematolrep16040061","url":null,"abstract":"Background: The aim of this study was to evaluate the impact of trabecular attenuation of the L1 vertebral body in low-dose CT in adult patients with multiple myeloma (MM), smoldering multiple myeloma (SMM), and monoclonal gammopathy of undetermined significance (MGUS).Materials and methods: The study population consisted of 22 patients with MGUS and 51 consecutive patients with newly diagnosed MM (SMM, n = 21; symptomatic MM, n = 36). CT scans were conducted using a 128-slice CT scanner (Somatom go.Top, Siemens, Munich, Germany). Low-dose whole-body CT scans were performed at a single time point for each patient. Trabecular bone density values were obtained by defining regions of interest on non-contrast images at the level of L1 vertebra. A threshold of p = 0.05 was applied to determine statistical significance.Results: The median Hounsfield unit (HU) value in patients with MGUS, SMM, and MM was 148 HU (range 81-190), 130 HU (range 93-193), and 92 HU (range 26-190), respectively, with a statistically significant difference between the groups (p = 0.0015). Patients with HU values ≤ 92 had lower progression-free survival with statistically significant differences compared to the group with HU values > 92 (p < 0.0499).Conclusions: This is the earliest evidence of the importance of evaluating L1 attenuation values in low-dose CT images in patients with MGUS, SMM, and MM. Further prospective studies could contribute to reinforcing these results and exploring the clinical applicability and generalization of L1 attenuation values in low-dose whole-body CT scans in routine clinical practice.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503432/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499163","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Acute Lymphoblastic Leukemia: Results of a Single-Center Study. 急性淋巴细胞白血病成人患者异基因造血干细胞移植的结果：单中心研究结果。

IF 1.1

Hematology Reports Pub Date : 2024-10-17 DOI: 10.3390/hematolrep16040062

Davide Stella, Jessica Gill, Roberto Passera, Sofia Zompi, Chiara Maria Dellacasa, Ernesta Audisio, Marco Cerrano, Irene Dogliotti, Michele Dicataldo, Carolina Secreto, Benedetto Bruno, Roberto Freilone, Alessandro Busca, Luisa Giaccone

{"title":"Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients with Acute Lymphoblastic Leukemia: Results of a Single-Center Study.","authors":"Davide Stella, Jessica Gill, Roberto Passera, Sofia Zompi, Chiara Maria Dellacasa, Ernesta Audisio, Marco Cerrano, Irene Dogliotti, Michele Dicataldo, Carolina Secreto, Benedetto Bruno, Roberto Freilone, Alessandro Busca, Luisa Giaccone","doi":"10.3390/hematolrep16040062","DOIUrl":"https://doi.org/10.3390/hematolrep16040062","url":null,"abstract":"Background: Despite the adoption of pediatric-like chemotherapy protocols, the introduction of new immunotherapies and a better understanding of the oncogenic landscape, the outcome for adult patients with acute lymphoblastic leukemia (ALL) remain substantially dismal. The aim of the present study was to evaluate the outcome in terms of survival in a cohort of adult patients with ALL who received allogeneic hematopoietic stem cell transplantation (alloSCT) between 2013 and 2023.Methods: This was a single-center observational retrospective study including all consecutive adult patients with ALL who received an alloSCT between April 2013 and April 2023 at the Stem Cell Transplant Center AOU Città della Salute e della Scienza of Torino. The primary endpoints were overall survival (OS), graft-versus-host disease (GVHD) Relapse-Free Survival (GRFS), Leukemia-Free Survival (LFS) and cumulative incidence (CI) of Non-Relapse Mortality (NRM).Results: The 4-year OS and LFS were 63.4% and 48.1%, respectively, and the 1-year GRFS was 42.9%. The 1-year CI of bloodstream infections (BSI), invasive fungal infections and NRM were 38%, 7% and 18.4%, respectively. Multivariate analysis showed that the use of total body irradiation (TBI), a time interval from diagnosis to alloSCT 7 months and female gender were factors significantly associated with better OS. Relapse of the underlying malignancy and BSI were the main causes of death.Conclusion: Our study suggests that alloSCT from a matched sibling donor (MSD) and alternative donors may be considered an effective tool for patients with ALL achieving a CR.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503301/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Utilizing Clinical Transformation Criteria for Prognostic Stratification in Follicular Lymphoma Prior to Initial Immunochemotherapy. 在初始免疫化疗前利用临床转化标准对滤泡性淋巴瘤进行预后分层

IF 1.1

Hematology Reports Pub Date : 2024-10-04 DOI: 10.3390/hematolrep16040060

Yoshikazu Hori, Hiroki Hosoi, Takayuki Hiroi, Ke Wan, Shogo Murata, Masaya Morimoto, Toshiki Mushino, Akinori Nishikawa, Takashi Sonoki

{"title":"Utilizing Clinical Transformation Criteria for Prognostic Stratification in Follicular Lymphoma Prior to Initial Immunochemotherapy.","authors":"Yoshikazu Hori, Hiroki Hosoi, Takayuki Hiroi, Ke Wan, Shogo Murata, Masaya Morimoto, Toshiki Mushino, Akinori Nishikawa, Takashi Sonoki","doi":"10.3390/hematolrep16040060","DOIUrl":"https://doi.org/10.3390/hematolrep16040060","url":null,"abstract":"Background: Although the prognosis of follicular lymphoma (FL) has improved, some patients experience early disease progression, including progression of disease within 24 months (POD24). Histological transformation is a critical event in FL. However, the heterogeneity of FL tumors makes it challenging to diagnose transformation accurately. We retrospectively applied the clinical transformation criteria used for FL transformation assessments at relapse or disease progression to conduct transformation assessments before the initial immunochemotherapy.Methods: Sixty-six FL patients who first received immunochemotherapy between January 2009 and February 2023 at our institution were selected. Twenty-three were clinical-transformation-positive (CLT+).Results: The progression-free survival (PFS) rate of the CLT+ patients was significantly lower than that of the clinical-transformation-negative (CLT-) patients. In the POD24 assessment subgroup, the CLT+ patients had a higher incidence of POD24 than the CLT- patients. There was no significant difference in PFS between the patients treated with CHOP-like regimens and those treated with bendamustine regimens. In the CHOP-like group, the CLT+ patients exhibited significantly lower PFS than the CLT- patients. In the bendamustine group, the clinical transformation did not affect PFS.Conclusion: Clinical transformation criteria may be useful for the prognostic stratification of FL prior to immunochemotherapy. Additionally, they may serve as predictors of POD24.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503408/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499165","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comprehensive Genomic Profiling in Non-Myeloid Hematologic Malignancies Identifies Variants That Can Alter Clinical Practice. 非髓性血液恶性肿瘤的全面基因组分析发现了可改变临床实践的变异。

IF 1.1

Hematology Reports Pub Date : 2024-09-30 DOI: 10.3390/hematolrep16040059

Chenyu Lin, Katherine I Zhou, Michelle F Green, Bennett A Caughey, John H Strickler, Michael B Datto, Matthew S McKinney

{"title":"Comprehensive Genomic Profiling in Non-Myeloid Hematologic Malignancies Identifies Variants That Can Alter Clinical Practice.","authors":"Chenyu Lin, Katherine I Zhou, Michelle F Green, Bennett A Caughey, John H Strickler, Michael B Datto, Matthew S McKinney","doi":"10.3390/hematolrep16040059","DOIUrl":"https://doi.org/10.3390/hematolrep16040059","url":null,"abstract":"Background: Comprehensive genomic profiling (CGP) is frequently adopted to direct the clinical care of myeloid neoplasms and solid tumors, but its utility in the care of lymphoid and histiocytic cancers is less well defined.Methods: In this study, we aimed to evaluate the frequency at which mutations identified by CGP altered management in non-myeloid hematologic malignancies. We retrospectively examined the CGP results of 105 samples from 101 patients with non-myeloid hematologic malignancies treated at an academic medical center who had CGP testing between 2014 and 2021.Results: CGP revealed one or more pathogenic or likely pathogenic variant in 92 (88%) of samples and 73 (72%) of tested patients had one or more mutations with diagnostic, prognostic, or therapeutic significance. The identification of a resistance variant resulted in the suspension of the active treatment or affected subsequent treatment choice in 9 (69%) out of 13 patients. However, the presence of a therapy sensitizing variant only led to consideration of a biomarker-directed therapy in 6 (10%) out of 61 patients.Conclusions: Overall, CGP of non-myeloid hematologic malignancies identified clinically significant variants in 72% of patients and resulted in a change in management in 22% of patients.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503333/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

The Real-World Outcomes of Relapsed/Refractory Multiple Myeloma Treated with Elotuzumab, Pomalidomide, and Dexamethasone. 埃洛珠单抗、泊马度胺和地塞米松治疗复发性/难治性多发性骨髓瘤的实际效果

IF 1.1

Hematology Reports Pub Date : 2024-09-30 DOI: 10.3390/hematolrep16040058

Hitomi Nakayama, Yoshinobu Aisa, Chisako Ito, Aki Sakurai, Tomonori Nakazato

{"title":"The Real-World Outcomes of Relapsed/Refractory Multiple Myeloma Treated with Elotuzumab, Pomalidomide, and Dexamethasone.","authors":"Hitomi Nakayama, Yoshinobu Aisa, Chisako Ito, Aki Sakurai, Tomonori Nakazato","doi":"10.3390/hematolrep16040058","DOIUrl":"https://doi.org/10.3390/hematolrep16040058","url":null,"abstract":"Introduction: A combination of elotuzumab, pomalidomide, and dexamethasone (EPd) was approved for the treatment of relapsed/refractory multiple myeloma (RRMM) following the ELOQUENT-3 phase II clinical trial. However, the clinical experience with this therapy is still limited. In this retrospective study, we analyzed the efficacy and safety of EPd in a real-world cohort of RRMM patients. Patients and Methods: The medical records of 22 patients who received EPd for RRMM at Yokohama Municipal Citizen's Hospital (Japan) between January 2020 and July 2021 were reviewed. Results: The median age of our cohort was 73.5 years. The overall response rate was 55%. With a median follow-up of 20.2 months, the median progression-free survival (PFS) was 9.1 months (95% confidence interval [CI], 2.5-23.0 months). The median PFS was shorter in patients with a poor performance status (PS) than in those with favorable PS (2.5 vs. 10.8 months; p < 0.01). Patients with prior daratumumab had significantly shorter PFS than those without prior daratumumab (2.1 vs. 23.0 months; p < 0.01). Additionally, patients with prior pomalidomide had significantly shorter PFS (1.7 vs. 10.3 months; p < 0.01). In the multivariate analysis, poor PS (hazard ratio [HR] = 4.1, 95% CI: 1.1-15.6; p = 0.04) and prior exposure to daratumumab (HR = 3.8, 95% CI: 1.1-13.8; p = 0.04) remained significantly associated with shorter PFS. Conclusions: The results of our study suggest that EPd is an active and well-tolerated regimen in RRMM, even in real-world patients. Furthermore, EPd may be useful, especially in daratumumab-naïve patients.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503276/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499162","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Two Cases of Immune Thrombocytopenia (ITP) Related to Viral Vector Vaccination ChAdOx1-S (AstraZeneca) and a Good Response after Thrombopoietin Receptor Agonist (TPO-RA) Therapy. 两例与病毒载体疫苗 ChAdOx1-S (AstraZeneca) 相关的免疫性血小板减少症 (ITP) 病例及接受促血小板生成素受体激动剂 (TPO-RA) 治疗后的良好反应。

IF 1.1

Hematology Reports Pub Date : 2024-09-27 DOI: 10.3390/hematolrep16040057

Konstantina Salveridou, Theodoros Tzamalis, Maika Klaiber-Hakimi, Sabine Haase, Stefanie Gröpper, Aristoteles Giagounidis

{"title":"Two Cases of Immune Thrombocytopenia (ITP) Related to Viral Vector Vaccination ChAdOx1-S (AstraZeneca) and a Good Response after Thrombopoietin Receptor Agonist (TPO-RA) Therapy.","authors":"Konstantina Salveridou, Theodoros Tzamalis, Maika Klaiber-Hakimi, Sabine Haase, Stefanie Gröpper, Aristoteles Giagounidis","doi":"10.3390/hematolrep16040057","DOIUrl":"https://doi.org/10.3390/hematolrep16040057","url":null,"abstract":"Background: In 2019, a new coronavirus disease emerged in Wuhan, China, known as SARS-CoV-2, severe acute respiratory syndrome coronavirus 2, and caused an ongoing pandemic. Symptomatology of the syndrome is variable, with complications extending to hematopoiesis and hemostasis. Approximately a year after onset of the virus, four vaccination formulas became available to the public, based on a viral vector or mRNA technology. These vaccine formulas have been hampered with hematological complications, like vaccine-induced immune thrombotic thrombocytopenia (VITT) and vaccine-related ITP (immune thrombocytopenic purpura). ITP is a disease with autoimmune pathogenesis characterized by antibody production against platelets and an increased hemorrhagic risk. A decent number of cases have been referred to as possible adverse effects of COVID-19 vaccinations.Case presentation: in this case report, we present two cases of newly diagnosed ITP after vaccination with ChAdOx1-S (AstraZeneca), with a good response to treatment with thrombopoietin-receptor agonists (TPO-RAs).Discussion: we observed an absence of response after corticosteroids and IVIG therapy and a positive therapeutic outcome on TPO-RA.Conclusions: in the ongoing pandemic, there is an urgent need to create therapeutic guidelines for vaccination-related clinical entities and to clarify indications for the vaccination of patients with pre-existing hematological diseases.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503447/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Complete Remission with Inotuzumab Ozogamicin as Fourth-Line Salvage Therapy in a Child with Relapsed/Refractory Acute Lymphoblastic Leukemia. 复发/难治性急性淋巴细胞白血病患儿接受因妥珠单抗奥佐加米星四线挽救疗法后病情完全缓解

IF 1.1

Hematology Reports Pub Date : 2024-09-27 DOI: 10.3390/hematolrep16040056

Athanasios Tragiannidis, Vassiliki Antari, Eleni Tsotridou, Theodoros Sidiropoulos, Aikaterini Kaisari, Maria Palabougiouki, Timoleon-Achilleas Vyzantiadis, Emmanuel Hatzipantelis, Assimina Galli-Tsinopoulou, Evgenios Goussetis

{"title":"Complete Remission with Inotuzumab Ozogamicin as Fourth-Line Salvage Therapy in a Child with Relapsed/Refractory Acute Lymphoblastic Leukemia.","authors":"Athanasios Tragiannidis, Vassiliki Antari, Eleni Tsotridou, Theodoros Sidiropoulos, Aikaterini Kaisari, Maria Palabougiouki, Timoleon-Achilleas Vyzantiadis, Emmanuel Hatzipantelis, Assimina Galli-Tsinopoulou, Evgenios Goussetis","doi":"10.3390/hematolrep16040056","DOIUrl":"https://doi.org/10.3390/hematolrep16040056","url":null,"abstract":"Background: Despite the progress achieved regarding survival rates in childhood acute lymphoblastic leukemia (ALL), relapsed or refractory disease still poses a therapeutic challenge. Inotuzumab ozogamicin is a CD22-directed monoclonal antibody conjugated to calicheamicin, which has been approved by the Food and Drug Administration for adults and pediatric patients 1 year and older with relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia. Case presentation: Herein, we present the case of a 23-month-old girl with high-risk B-ALL who experienced very early isolated medullary relapse; following the failure of conventional chemotherapy according to the ALL-IC REL 2016 protocol, she went on to receive the bispecific T-cell engager (BiTE) blinatumomab and subsequently, due to refractory disease, the combination of fludarabine, cytarabine, and the proteasome inhibitor bortezomib without achieving remission. Given the high CD22 expression by the lymphoblasts, off-label use of inotuzumab ozogamicin (InO) was chosen and administrated in a 28-day cycle as a salvage treatment. The minimal residual disease (MRD) was 0.08% on day 28, and InO was continued, thus achieving MRD negativity; the patient successfully underwent an allogeneic stem cell transplantation from a matched family donor. Conclusions: Our case highlights the efficacy and safety of InO as a salvage treatment in the setting of relapsed B-ALL refractory not only to conventional chemotherapy but also to novel treatments, such as blinatumomab and bortezomib.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503335/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499156","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Padua Prediction Score and Hospital-Acquired Proximal and Isolated Distal Deep Vein Thrombosis in Symptomatic Patients. 帕多瓦预测评分与无症状患者在医院获得的近端和孤立远端深静脉血栓。

IF 1.1

Hematology Reports Pub Date : 2024-09-25 DOI: 10.3390/hematolrep16040055

Michelangelo Sartori, Miriam Fiocca, Mario Soldati, Laura Borgese, Elisabetta Favaretto, Benilde Cosmi

{"title":"Padua Prediction Score and Hospital-Acquired Proximal and Isolated Distal Deep Vein Thrombosis in Symptomatic Patients.","authors":"Michelangelo Sartori, Miriam Fiocca, Mario Soldati, Laura Borgese, Elisabetta Favaretto, Benilde Cosmi","doi":"10.3390/hematolrep16040055","DOIUrl":"https://doi.org/10.3390/hematolrep16040055","url":null,"abstract":"Background: Hospital-acquired deep vein thrombosis (DVT) is an important cause of morbidity and mortality.Objectives: The purpose of this study was to evaluate the prevalence of proximal lower limb DVT and isolated distal DVT (IDDVT) and their relationship to the Padua Prediction Score (PPS) in acutely ill, hospitalized patients.Methods: In a single-center cross-sectional study, all inpatients from medical departments with suspected lower-extremity DVT were evaluated with whole-leg ultrasonography during 183 days from 2016 to 2017.Results: Among the 505 inpatients (age 78.0 ± 13.3, females 59.2%) from medical departments, 204 (40.2%) had PPS ≥ 4, but only 54.4% of them underwent pharmacological thrombo-prophylaxis. Whole-leg ultrasonography detected 47 proximal DVTs (9.3%) and 65 IDDVTs (12.8%). Proximal DVT prevalence was higher in patients with high PPS vs. those with low PPS (12.7% vs. 7.0% p = 0.029, respectively), whereas IDDVT prevalence was similar in patients with high and low PPS (14.7% vs. 11.6% p = 0.311, respectively). The area under the receiver operating curve (AUC) for the PPS was 0.62 ± 0.03 for all DVTs, 0.64 ± 0.04 for proximal DVTs, and 0.58 ± 0.04 for IDDVTs.Conclusions: In hospitalized patients, IDDVT had similar prevalence regardless of PPS risk stratification. Adherence to thrombo-prophylaxis in patients was still far from optimal.","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":null,"pages":null},"PeriodicalIF":1.1,"publicationDate":"2024-09-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11503401/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142499160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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