{"title":"Efficacy of Anti-CD38 Monoclonal Antibodies for Relapsed or Refractory Multiple Myeloma in Stem Cell Transplant-Ineligible Patients Aged over 65 Years: A Propensity Score-Matched Study.","authors":"Satoshi Yamasaki, Michitoshi Hashiguchi, Nao Yoshida-Sakai, Hiroto Jojima, Koichi Osaki, Takashi Okamura, Yutaka Imamura","doi":"10.3390/hematolrep16040068","DOIUrl":"10.3390/hematolrep16040068","url":null,"abstract":"<p><strong>Background: </strong>The development of newer agents, including anti-CD38 monoclonal antibodies (mAbs), has significantly improved overall survival (OS) in patients with relapsed or refractory multiple myeloma (RRMM). However, the treatment of older patients with RRMM who are transplant-ineligible remains challenging.</p><p><strong>Methods: </strong>We retrospectively evaluated OS in 78 transplant-ineligible patients with RRMM who were aged ≥ 65 years and treated at our institution between February 2012 and November 2023.</p><p><strong>Results: </strong>Unadjusted OS was significantly longer in the anti-CD38 mAb-exposed group (i.e., those previously treated with daratumumab and receiving isatuximab plus pomalidomide and low-dose dexamethasone because of disease progression during treatment with daratumumab [<i>n</i> = 6], daratumumab plus pomalidomide and low-dose dexamethasone [<i>n</i> = 9], or isatuximab plus pomalidomide and low-dose dexamethasone without daratumumab-exposure [<i>n</i> = 14]) than in the anti-CD38 mAb-naïve group (no exposure to daratumumab or isatuximab [<i>n</i> = 49]) (<i>p</i> < 0.001). To address potential confounder factors associated with use or nonuse of anti-CD38 mAbs, we performed propensity score matching (PSM) using age, sex, performance status, and Geriatric 8 and Instrumental Activities of Daily Living scores. PSM identified 14 subjects from the anti-CD38 mAb-exposed group with baseline characteristics similar to those of 14 subjects from the anti-CD38 mAb-naïve group. After PSM, the adjusted OS was significantly longer in the anti-CD38 mAb-exposed group than in the anti-CD38 mAb-naïve group (<i>p</i> < 0.001).</p><p><strong>Conclusion: </strong>These findings provide insights into the optimal use of anti-CD38 mAbs in patients with RRMM who are transplant-ineligible and aged ≥65 years and on candidates who are appropriate for novel approaches, such as chimeric antigen receptor T-cell or bispecific T-cell engager therapy.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"714-723"},"PeriodicalIF":1.1,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587159/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709529","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}
Massimo Martino, Martina Pitea, Annalisa Sgarlata, Ilaria Maria Delfino, Francesca Cogliandro, Anna Scopelliti, Violetta Marafioti, Simona Polimeni, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Maria Pellicano, Giovanni Leanza, Caterina Alati
{"title":"Treatment Strategies Used in Treating Myelofibrosis: State of the Art.","authors":"Massimo Martino, Martina Pitea, Annalisa Sgarlata, Ilaria Maria Delfino, Francesca Cogliandro, Anna Scopelliti, Violetta Marafioti, Simona Polimeni, Gaetana Porto, Giorgia Policastro, Giovanna Utano, Maria Pellicano, Giovanni Leanza, Caterina Alati","doi":"10.3390/hematolrep16040067","DOIUrl":"10.3390/hematolrep16040067","url":null,"abstract":"<p><strong>Background: </strong>Current drug therapy for myelofibrosis does not alter the natural course of the disease or prolong survival, and allogeneic stem cell transplantation is the only curative treatment modality. For over a decade, the Janus kinase (JAK) inhibitor ruxolitinib has been the standard of care. More recently, newer-generation JAK inhibitors have joined the ranks of accepted treatment options.</p><p><strong>Objectives: </strong>The primary goal of treatment is to reduce spleen size and minimize disease-related symptoms. Prognostic scoring systems are used to designate patients as being at lower or higher risk. For transplant-eligible patients, transplant is offered to those with a bridge of a JAK inhibitor; patients who are not eligible for transplant are usually offered long-term therapy with a JAK inhibitor. Limited disease-modifying activity, dose-limiting cytopenias, and other adverse effects have contributed to discontinuation of JAK inhibitor treatment.</p><p><strong>Conclusions: </strong>Novel JAK inhibitors and combination approaches are currently being explored to overcome these shortcomings. Further research will be essential to establish optimal therapeutic approaches in first-line and subsequent treatments.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"698-713"},"PeriodicalIF":1.1,"publicationDate":"2024-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709722","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}
Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli
{"title":"Real-World Study of US Adults with Paroxysmal Nocturnal Hemoglobinuria Treated with Pegcetacoplan.","authors":"Brian Mulherin, Apeksha Shenoy, Lily Arnett, Weiqi Jiao, Joseph Guarinoni, Sujata Sarda, Jinny Min, David Dingli","doi":"10.3390/hematolrep16040065","DOIUrl":"10.3390/hematolrep16040065","url":null,"abstract":"<p><p><b>Background</b>: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease characterized by complement-mediated hemolysis. OPERA is the first US longitudinal real-world study on C3 inhibitor therapy, known as pegcetacoplan. <b>Methods</b>: OPERA enrolled US patients with PNH, age ≥18, who were prescribed pegcetacoplan, and data were collected from routine care. Hemoglobin was reported by patients during regular follow-up (censored from transfusions). The Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue (0-52 score) and Patient-Reported Outcomes Measurement Information System scale for Cognitive Function Abilities (PROMIS-CF; 23.27-67.09 t-score) were completed electronically (low score = negative outcome). Patients self-reported incidence of healthcare resource utilization (HCRU). <b>Results</b>: By January 2024, 70 patients (mean age 44.6 years; 57.1% female) reported up to 9 months of pegcetacoplan treatment, with a median [IQR] follow-up of 6.6 [3.8] months. The latest reported hemoglobin levels improved by a mean (SD) of 2.6 (1.9) g/dL from baseline. At 3, 6 and 9 months, patients reported clinically meaningful improvements (≥5 points) in FACIT-F (53.3-69.0%) and (≥2 points) PROMIS-CF (46.7-55.2%). Patients reported a <10% incidence rate per person month of all HCRU events. <b>Conclusions</b>: This first longitudinal real-world US study indicates a positive trend in Hb, fatigue, and cognition with limited HCRU during pegcetacoplan treatment in adults with PNH.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"669-681"},"PeriodicalIF":1.1,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587045/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709629","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}
{"title":"Alternative Splicing: A Potential Therapeutic Target in Hematological Malignancies.","authors":"Gazmend Temaj, Silvia Chichiarelli, Sarmistha Saha, Pelin Telkoparan-Akillilar, Nexhibe Nuhii, Rifat Hadziselimovic, Luciano Saso","doi":"10.3390/hematolrep16040066","DOIUrl":"10.3390/hematolrep16040066","url":null,"abstract":"<p><p>Leukemia represents the most prevalent malignancy in children, constituting 30% of childhood cancer cases, with acute lymphoblastic leukemia (ALL) being particularly heterogeneous. This paper explores the role of alternative splicing in leukemia, highlighting its significance in cancer development and progression. Aberrant splicing is often driven by mutations in splicing-factor genes, which can lead to the production of variant proteins that contribute to oncogenesis. The spliceosome, a complex of small nuclear RNAs and proteins, facilitates RNA splicing, a process critical for generating diverse mRNA and protein products from single genes. Mutations in splicing factors, such as U2AF1, SF3B1, SRSF2, ZRSR2, and HNRNPH1, are frequently observed across various hematological malignancies and are associated with poor prognosis and treatment resistance. This research underscores the necessity of understanding the mechanisms of RNA splicing dysregulation in order to develop targeted therapies to correct these aberrant processes, thereby improving outcomes for patients with leukemia and related disorders.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"682-697"},"PeriodicalIF":1.1,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11587037/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142709360","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}
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":"<p><p>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.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"656-668"},"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}
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":"<p><strong>Background: </strong>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.</p><p><strong>Case presentation: </strong>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.</p><p><strong>Conclusions: </strong>The eventual identification of the splenic origin underscores the diagnostic difficulties and clinical challenges inherent in managing such atypical and rare presentations.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"648-655"},"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}
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":"<p><strong>Background: </strong>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).</p><p><strong>Materials and methods: </strong>The study population consisted of 22 patients with MGUS and 51 consecutive patients with newly diagnosed MM (SMM, <i>n</i> = 21; symptomatic MM, <i>n</i> = 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 <i>p</i> = 0.05 was applied to determine statistical significance.</p><p><strong>Results: </strong>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 (<i>p</i> = 0.0015). Patients with HU values ≤ 92 had lower progression-free survival with statistically significant differences compared to the group with HU values > 92 (<i>p</i> < 0.0499).</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"624-635"},"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}
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":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"636-647"},"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}
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":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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+).</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>Clinical transformation criteria may be useful for the prognostic stratification of FL prior to immunochemotherapy. Additionally, they may serve as predictors of POD24.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"612-623"},"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}
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":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"16 4","pages":"603-611"},"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}