Hematology/ Oncology and Stem Cell Therapy最新文献

{"title":"Familial Emberger Syndrome With Autoimmunity, Hyper-Immunoglobulin E and Lymphatic Impairment Caused by a Novel GATA2 Mutation.","authors":"Moneerah AlGassim, Ahad F Al Seraihi, AlFadel AlShaibani, Walter Conca, Saleem AlShehri, Moheieldin Moustafa Abouzied, Issam Hamadah, Saleh AlReshoodi, Majed Dasouki, Farrukh Sheikh","doi":"10.1016/j.hemonc.2020.05.004","DOIUrl":"https://doi.org/10.1016/j.hemonc.2020.05.004","url":null,"abstract":"","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":"15 2","pages":"63-65"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2020.05.004","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10619926","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Spectrum of Myelodysplastic Syndrome in Patients Evaluated for Cytopenia(s). A Report from a Reference Centre in Saudi Arabia.","authors":"Nour AlMozain,&nbsp;Ayman Mashi,&nbsp;Qasem Alneami,&nbsp;Amal Al-Omran,&nbsp;Nasir Bakshi,&nbsp;Tarek Owaidah,&nbsp;Salem Khalil,&nbsp;Haitham Khogeer,&nbsp;Shahrukh Hashmi,&nbsp;Suleimman Al-Sweedan,&nbsp;Thomas Morris,&nbsp;Randa AlNounou","doi":"10.1016/j.hemonc.2020.11.001","DOIUrl":"https://doi.org/10.1016/j.hemonc.2020.11.001","url":null,"abstract":"<p><strong>Background/objective: </strong>Myelodysplastic syndrome (MDS) is a clonal disorder of hematopoietic stem cells, characterized by ineffective hematopoiesis, peripheral cytopenias along with hypercellularity of the bone marrow, and marked dysplastic features. Establishing MDS diagnosis is difficult due to nonspecific clinical presentation and imprecise morphological criteria. In anticipation to improve the diagnostic approach in this field, we aimed to characterize the clinical and morphological features of patients presented with cytopenias with a special focus on MDS.</p><p><strong>Methods: </strong>We comprehensively reviewed all medical record of patients who were referred to the hematology laboratory at KFSH-RC, Riyadh, Saudi Arabia, between January 2009 and March 2016 for evaluation of bone marrow aspirates and trephine biopsies due to severe and persistent cytopenia(s) to rule out MDS.</p><p><strong>Results: </strong>A total of 183 patients, 155 adult and 28 pediatric, were identified. In the adult group, MDS was diagnosed in 82 (52.9%) patients, with a male-to-female (M:F) ratio of 1.6:1 and mean age at diagnosis of 50 years. According to the World Health Organization (WHO) 2017 criteria, MDS subtypes were as follows: MDS with single lineage dysplasia (SLD, 5%), MDS with ring sideroblasts and SLD (MDS-RS-SLD 7%), MDS with multilineage dysplasia (MDS-MLD 21%), MDS with deletion of chromosome 5q (MDS del(5q), 2%), MDS unclassifiable (MDS-U7%), hypoplastic MDS (h-MDS 4%), MDS with excess blasts-1 (MDS-EB1, 20%), MDS with excess blasts-2 (MDS-EB2, 28%), and therapy-related MDS (6%). Laboratory and morphological features were described. In both groups, cytogenetic abnormalities were classified according to the Revised International Prognostic Scoring System cytogenetic risk groups. In adults, the dominating cytogenetic abnormalities were monosomy 5 and monosomy 7 seen in 20.7% and 24.4% of patients, respectively. Peripheral cytopenia not due to MDS was diagnosed in 54 (34.8%) patients, with a mean age of 43 years and M:F ratio of 1:1. The cause of these cytopenias were as follows: bone marrow failure (BMF, 22%), peripheral destruction (20%), drug induced (20%), anemia of chronic disease (16%), B12 deficiency (7%), infection (7%), paroxysmal nocturnal hemoglobinuria (4%), idiopathic cytopenia of undetermined significance (2%), and idiopathic dysplasia of undetermined significance (2%). A definite diagnosis of MDS was not possible in 19 patients due to insufficient clinical data. In the pediatric group, MDS was diagnosed in 14/28 (50%) patients, with M:F ratio of 1.8:1 and mean age at diagnosis of 4 years. MDS subtypes (WHO 2017) in 14 patients were as follows: refractory cytopenia of childhood (RCC, 42.8%), MDS-EB1 (42.8%), and MDS-EB2 (14.2%). Laboratory and morphological features were described. The prevalent cytogenetic abnormality was monosomy 7 in six/14 (42.8%) patients. Cytopenias due to other causes were diagnosed in eight/28 p","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"39-44"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2020.11.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38643627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Does Race Play a Role in Complications and Outcomes of Philadelphia Chromosome-Negative Myeloproliferative Neoplasms?","authors":"Andrew M Peseski,&nbsp;Antoine N Saliba,&nbsp;Sandra K Althouse,&nbsp;Hamid Sayar","doi":"10.1016/j.hemonc.2021.01.005","DOIUrl":"https://doi.org/10.1016/j.hemonc.2021.01.005","url":null,"abstract":"<p><strong>Background: </strong>Philadelphia chromosome-negative myeloproliferative neoplasms (MPN) are a group of hematologic malignancies with known vascular complications. The role race and ethnicity play in these complications is less defined. We aimed to further evaluate the role of race in patients without a history of previous thrombotic or hemorrhagic events.</p><p><strong>Methods: </strong>In this retrospective study, 300 adult patients with MPN were included; 270 (90.0%) were White and 30 (10.0%) were non-White. The non-White group primarily consisted of African American or Black (26 patients), followed by others. Median age at diagnosis was 58 years for White patients and 61.5 years for non-White patients. The interaction between outcomes and vascular events with race was evaluated using multivariate logistical regression models.</p><p><strong>Results: </strong>The incidence of thrombotic events was inversely correlated with age at diagnosis, with younger patients demonstrating a higher rate of thrombotic events over time (p < .001). The incidence of thrombotic or hemorrhagic events did not differ between White and non-White patients. A statistically significant difference in median survival was observed between White and non-White patients: 29 years (95% confidence interval [CI]: 21.8-not reached) versus 13 years (95% CI: 5.7-22.7), respectively (p=.016).</p><p><strong>Conclusion: </strong>This study did not find a significant difference in the rate of thrombotic or hemorrhagic events between White and non-White patients with MPN but suggested that non-White patients had significantly shorter median survival than White patients. Such observations may inform future studies to further characterize racial disparities in outcomes.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"30-38"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2021.01.005","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25385732","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Hispanic Paradox in Non-Small Cell Lung Cancer.","authors":"Rohit Kumar,&nbsp;Franklin Castillero,&nbsp;Shruti Bhandari,&nbsp;Sindhu Malapati,&nbsp;Goetz Kloecker","doi":"10.1016/j.hemonc.2021.02.004","DOIUrl":"https://doi.org/10.1016/j.hemonc.2021.02.004","url":null,"abstract":"<p><strong>Objective/background: </strong>According to the U.S. Census Bureau, 18% of the total population in the United States identified themselves as Hispanic in 2016 making it the largest minority group. This study aimed to evaluate the effect of Hispanic ethnicity on the overall survival of patients with non-small cell lung cancer (NSCLC) using a large national cancer database.</p><p><strong>Methods: </strong>We used the National Cancer Database to identify patients diagnosed with NSCLC between 2010 and 2015. The two comparative groups for this study were non-Hispanic Whites (NHWs) and Hispanics. The primary outcome was overall survival.</p><p><strong>Results: </strong>Of the 555,475 patients included in the study, 96.9% and 3.1% were NHWs and Hispanics with a median follow up of 12.6 months (interquartile range 4.1-30.6) and 12.1 months (interquartile range 3.8-29.5), respectively. Hispanics were more likely to be uninsured, and live in areas with lower median household income or education level. In the age-, sex-, and comorbidities-adjusted Cox model, the overall survival was significantly better in Hispanics compared with NHWs (hazard ratio [HR] 0.92, 95% confidence interval 0.90-0.93, p < .001). In a demographic, socioeconomic, clinical, and facility characteristics adjusted Cox model, Hispanics had further improvement in survival (HR 0.79, 95% confidence interval 0.78-0.81, p < .001). The survival advantage was seen in all cancer stages: Stage I-HR 0.76 (0.71-0.80), Stage II-HR 0.85 (0.79-0.92), Stage III-HR 0.81 (0.77-0.85), and Stage IV-HR 0.79 (0.77-0.81).</p><p><strong>Conclusion: </strong>Hispanic ethnicity was associated with better survival in NSCLC. This survival advantage is likely the result of complex interactions amongst several physical, social, cultural, genomic, and environmental factors.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"21-29"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2021.02.004","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25523652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mutation Profile in BCR-ABL1-Negative Myeloproliferative Neoplasms: A Single-Center Experience From India.","authors":"Madhavi Maddali,&nbsp;Uday Prakash Kulkarni,&nbsp;Niveditha Ravindra,&nbsp;Arun Kumar Arunachalam,&nbsp;Arvind Venkatraman,&nbsp;Sharon Lionel,&nbsp;Marie Therese Manipadam,&nbsp;Anup J Devasia,&nbsp;Anu Korula,&nbsp;N A Fouzia,&nbsp;Aby Abraham,&nbsp;Alok Srivastava,&nbsp;Biju George,&nbsp;Poonkuzhali Balasubramanian,&nbsp;Vikram Mathews","doi":"10.1016/j.hemonc.2021.03.002","DOIUrl":"https://doi.org/10.1016/j.hemonc.2021.03.002","url":null,"abstract":"<p><strong>Objective/background: </strong>Recurrent somatic mutations in the JAK2, calreticulin (CALR), and the MPL genes are described as drivers of BCR-ABL1-negative myeloproliferative neoplasms (MPN) that includes polycythemia vera (PV), essential thrombocytosis (ET), primary myelofibrosis (PMF), and MPN unclassified (MPN-U).</p><p><strong>Methods: </strong>We describe the mutation profile and clinical features of MPN cases diagnosed at a tertiary care center. JAK2V617F and MPL (S505/W515) mutations were screened by allele-specific polymerase chain reaction, while CALR exon 9 and JAK2 exon 12 mutations were screened by fragment analysis/Sanger sequencing. Among the 1,570 patients tested for these mutations during the study period, 407 were classified as MPN with a diagnosis of PV, ET, PMF, and MPN-U seen in 30%, 17%, 36%, and 17%, respectively, screened.</p><p><strong>Results: </strong>Similar to previous reports from Asian countries, the incidence of PMF was the highest among the classic MPN. JAK2V617F mutation was detected in 90% of PV, 38% of ET, 48% of PMF, and 65% of MPN-U. JAK2 exon 12 mutations were seen in 5.7% of PV and 1.4% of PMF. CALR exon 9 mutations were seen in 33% of ET, 33% of PMF, and 12% of MPN-U. MPL mutations were detected in 2.8%, 2.7%, and 2.9% of ET, PMF, and MPN-U, respectively. Fifteen % of PMF, 26% of ET, and 22% of MPN-U were triple negative.</p><p><strong>Conclusion: </strong>There was a significantly higher incidence of CALR mutation in PMF and ET cases. Our study highlights the challenges in the diagnosis of JAK2-negative PV and the need for harmonization of criteria for the same.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"13-20"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2021.03.002","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25534677","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":"Hepatitis-Associated Aplastic Anemia.","authors":"Alfadel Alshaibani,&nbsp;Carlo Dufour,&nbsp;Antonio Risitano,&nbsp;Regis de Latour,&nbsp;Mahmoud Aljurf","doi":"10.1016/j.hemonc.2020.10.001","DOIUrl":"https://doi.org/10.1016/j.hemonc.2020.10.001","url":null,"abstract":"<p><p>Hepatitis-associated aplastic anemia (HAAA) is a rare illness, characterized by onset of pancytopenia with a hypoplastic bone marrow that traditionally occurs within 6 months of an increase in serum aminotransferases. HAAA is observed in 1% to 5% of all newly diagnosed cases of acquired aplastic anemia. Several hepatitis viruses have been linked to the disease, but in many cases no specific virus is detected. The exact pathophysiology is unknown; however, immune destruction of hematopoietic stem cells is believed to be the underlying mechanism. HAAA is a potentially lethal disease if left untreated. Management includes immunosuppression with antithymocyte globulin and cyclosporine and allogeneic hematopoietic stem cell transplantation.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"8-12"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2020.10.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38607600","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinicopathological Significance of Common Genetic Alterations in Patients With Acute Promyelocytic Leukemia.","authors":"Sukanta Nath,&nbsp;Jina Bhattacharyya,&nbsp;Prem Chandra,&nbsp;Renu Saxena,&nbsp;Sudha Sazawal,&nbsp;Kandarpa Kumar Saikia","doi":"10.1016/j.hemonc.2020.07.004","DOIUrl":"https://doi.org/10.1016/j.hemonc.2020.07.004","url":null,"abstract":"<p><strong>Objective/background: </strong>Acute myeloid leukemia (AML) is one of the common forms of hematological malignancy and acute promyelocytic leukemia (APL) is a unique subtype of AML conferring favorable prognosis. We aimed to determine the prevalence and prognostic impact of Fms-like tyrosine kinase 3 (FLT3), nucleophosmin 1 (NPM1) mutation, epidermal growth factor receptor (EGFR), and flow marker's expression in patients with APL.</p><p><strong>Methods: </strong>In the present study, 165 de novo APL patients were molecularly characterized for promyelocytic leukemia (PML) breakpoint and additional genetic alterations. Reverse transcriptase polymerase chain reaction (PCR) and real-time PCR assays were used to detect genetic alterations.</p><p><strong>Results: </strong>PML/RARα was detected in 29/165 (17.5%) samples with breakpoint cluster region 1 (bcr1) in 17/29 (58.5%) and bcr3 in 12/29 (41.5%) samples. The prevalence of FLT3-ITD, NPM1, and EGFR were detected in 5/29 (17.5%), 11/29 (38%), and 5/29 (17.5%) patients, respectively. Patients expressing bcr-3 hybrid transcript had lower overall survival compared with bcr1 ( p = .254). White blood cell (WBC) count was significantly higher in bcr3 in comparison with bcr1 patients ( p = .002). Patients with positive EGFR expression ( p = .042) and higher WBC ( p = .002) were significantly associated with poor survival ( p < .05).</p><p><strong>Conclusions: </strong>We documented the higher prevalence of bcr1 and confirmed that the association of FLT3-ITD significantly reduced the chances of survival in APL. The mortality rate of bcr3 was comparatively higher than that of bcr1. Higher WBC count and EGFR expression were significantly associated with poor survival.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":"15 2","pages":"54-57"},"PeriodicalIF":0.0,"publicationDate":"2022-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2020.07.004","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9167527","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Novel Frameshift Mutation in the ITGB3 Gene Leading to Glanzmann's Thrombasthenia in a Saudi Arabian Family.","authors":"Asma Alharbi,&nbsp;Jamil A Hashmi,&nbsp;Essa Alharby,&nbsp;Alia M Albalawi,&nbsp;Khushnooda Ramzan,&nbsp;Sulman Basit","doi":"10.1016/j.hemonc.2021.01.003","DOIUrl":"https://doi.org/10.1016/j.hemonc.2021.01.003","url":null,"abstract":"<p><p>Glanzmann's thrombasthenia (GT) is an autosomal recessive congenital bleeding disorder of platelet aggregation. Mutations in ITGA2B and ITGB3 genes result in quantitative and/or qualitative abnormalities of the glycoprotein receptor complex IIb/IIIa (integrin αIIbβ3), which in turn impairs platelet aggregation and lead to GT. In this study, whole genome single nucleotide polymorphism (SNP) genotyping as well as whole exome sequencing was performed in a large family segregating GT. Analysis of the genotypes localized the disease region to chromosome 17q21.2-q21.3. Filtration of whole exome data and candidate variants prioritization identified a pathogenic variant in the ITGB3 gene. The single nucleotide deletion variant (c.2113delC) in exon 13 of the ITGB3 gene is predicted to cause a frameshift and absence of vital C-terminal domains including the transmembrane helix and the cytoplasmic domain. Clinical variability of the bleeding phenotype in affected individuals with the same mutation suggests that other genetic and nongenetic factors are responsible for determining GT features.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"21-26"},"PeriodicalIF":0.0,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2021.01.003","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25384663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Using the Appropriate Formula for QT Measurement Can Save Lives.","authors":"Taher Al-Tweigeri,&nbsp;Susan Dent,&nbsp;Adher Al Sayed,&nbsp;Dania Mohty,&nbsp;Kausar Suleman,&nbsp;Dahish Ajarim,&nbsp;Hussein Raef,&nbsp;Najmeddine Echahidi","doi":"10.1016/j.hemonc.2021.06.001","DOIUrl":"https://doi.org/10.1016/j.hemonc.2021.06.001","url":null,"abstract":"<p><p>CDK 4/6 inhibitors, in combination with endocrine therapy, are the standard of care for patients with endocrinesensitive advanced breast cancer. This class of drug, however, is associated with QT prolongation, which serves as a surrogate marker for Torsades de Pointes (TdP), a cause of life-threatening ventricular arrhythmias and sudden cardiac death. The ICH E14 guidance document uses the Bazett formula for reporting of cardio-dynamic and safety ECG data in clinical trials. While there is substantial familiarity with the Bazett (QTcB) formula (QT/(RR) 1/2), the Fridericia (QTcF) formula (QT/(RR) 1/3 ) is preferred in the cancer population as it is often more accurate at heart rate extreme. Accordingly, the Fridericia formula is currently the standard adopted by the FDA when submitting QT data for review. At the King Faisal Specialist Hospital and Research Center, a total of 82 patients with advanced breast cancer, had a baseline ECG on day 1 before the initiation of ribociclib based therapy. Of the enrolled 82 patients, 19 (23%) were initially excluded from receiving ribociclib based due to a prolonged QTc >450ms, however, when the QTc-interval was manually measured and recalculated using Fridericia and Framingham formulae using MDCalC (https://www.mdcalc.com),17 of 19 patients successfully received their treatment without any arrhythmogenic effects. Repeat ECG on day14, and day 1 of cycle 2 demonstrated that none of these patients had QTc exceeding 480 ms. Our data highlights the complexities of evaluating the QT interval in oncology patients and the utility of the Fridericia/Framingham formulae in this population. Given these findings, we recommend the adoption of the Fridericia or Framingham formulae for measurement of QTc in all cancer patients exposed to potentially QT-prolonging cancer therapy.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":" ","pages":"79-82"},"PeriodicalIF":0.0,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2021.06.001","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39047693","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Evaluation of Eltrombopag in Thrombocytopenia Post Hematopoietic Cell Transplantation: Rertrospective Observational Trial.","authors":"H Samarkandi,&nbsp;M Al Nahedh,&nbsp;A Alfattani,&nbsp;F Alsharif,&nbsp;N Bakshi,&nbsp;W Rasheed,&nbsp;F Alfraih,&nbsp;M Alhumaid,&nbsp;Nora Alkhudair,&nbsp;S Alhayli,&nbsp;H Alsaedi,&nbsp;M Shaheen,&nbsp;A Hanbali,&nbsp;S K Hashmi,&nbsp;E Devol,&nbsp;A Alseraihy,&nbsp;H Alzahrani,&nbsp;M Aljurf","doi":"10.1016/j.hemonc.2020.07.006","DOIUrl":"https://doi.org/10.1016/j.hemonc.2020.07.006","url":null,"abstract":"<p><strong>Background: </strong>Thrombocytopenia remains a life-threatening late complication of HCT with an incidence of 5-20%. Currently, there is no approved drug for the treatment of persistent thrombocytopenia post HCT and platelet transfusion is the maintain stay of treatment. Eltrombopag is approved for the treatment of thrombocytopenia associated with different diseases, however; data on eltrombopag treatment post HCT are limited.</p><p><strong>Methods: </strong>This is a retrospective cohort study evaluating the effect of eltrombopag on platelet recovery in patients with persistent thrombocytopenia post HCT. The primary endpoint was platelet recovery to ≥ 20,000/μL for 7 consecutive days without transfusion support after starting eltrombopag. Secondary endpoint was platelet recovery to ≥ 50,000/μL for 7 consecutive days.</p><p><strong>Results: </strong>Twenty-one patients were included. Twelve (75%) of 16 patients became independent from platelet transfusions. Median time from starting eltrombopag to last transfusion was 60 days (range, 9-226 days). Ten (63%) of 16 transfusion dependent patients with platelet count < 20,000/μL achieved the primary endpoint. Seven (33%) patients of 21 included had successful platelet recovery (ie, ≥50,000/μL without transfusion support) and the median time to platelet recovery in patients who achieved it was 32 days (range, 13-265 days). Ten patients (48%) were able to successfully discontinue eltrombopag without recurrence of thrombocytopenia.</p><p><strong>Conclusion: </strong>Our findings demonstrated that eltrombopag appears to have a clinically significant impact on platelet recovery in persistent thrombocytopenic patients post HCT.</p>","PeriodicalId":39226,"journal":{"name":"Hematology/ Oncology and Stem Cell Therapy","volume":"15 1","pages":"285-290"},"PeriodicalIF":0.0,"publicationDate":"2022-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1016/j.hemonc.2020.07.006","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9249069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}