Acta Haematologica最新文献

{"title":"Outcomes of Fedratinib in routine Treatment of ruxolitinib-resistant or refractory patients with Primary and post-polycythemia vera or essential thrombocythemia Myelofibrosis: A nationalwide retrospective study.","authors":"Adrian Duek,Alexandra Tzinman,Kira Maziuk,Assaf Levy,Martin Ellis,Galia Stemer,Adi Shacham Abulafia,Amos Cohen,Noa Lavi,Aaron Ronson,Andrey Braester,Shirley Shapira,Jonathan Canaani,Yulia Volchek,Ronit Leiba,Merab Leiba","doi":"10.1159/000540906","DOIUrl":"https://doi.org/10.1159/000540906","url":null,"abstract":"INTRODUCTIONIn recent years, fedratinib, a selective JAK2 inhibitor, has emerged as a potential therapeutic option for patients who have failed or are intolerant to ruxolitinib. Despite the promising results observed in clinical studies, real-world evidence from the United States and Europe suggests that the efficacy of fedratinib may be less conclusive. We report the characteristics, treatment patterns, and clinical outcomes of patients with myelofibrosis (MF) treated with fedratinib following ruxolitinib failure in Israel's clinical practice.METHODSThis retrospective patient chart review included adults with a physician-reported diagnosis of MF, who initiated fedratinib after discontinuing ruxolitinib. Descriptive analyses characterized patient characteristics, clinical outcomes, and treatment patterns from MF diagnosis through ruxolitinib and fedratinib treatment.RESULTSWe extracted data for 16 eligible patients. Approximately 62.5 % of the patients were female, and the median age was 77 (range, 63-85) years. The median duration of ruxolitinib therapy was 17 months (range 3-84 ) months. Before the initiation of fedratinib, the median spleen size by palpation was 15.5cm below the costal margin (range 4-22cm). After three months the median spleen size was 13cm below the costal margin (range 2-21 cm). Only two patients showed minimal improvement after six months, while three patients progressed, and two patients showed no change in the spleen size. The spleen response did not improve after 12 months of treatment. At this point, the median spleen size was 19 cm below the costal margin (range 2-30 cm). Regarding the MF-related symptoms, 43.75% (n =7) of patients reported some improvement, 37.5% (n =6) showed no changes, whereas 18.75% (n =3) of the population complained of worsening. Gastrointestinal toxicity was the most frequent adverse effect of the drug, while 31% of patients died.CONCLUSIONOur observations showed that in MF patients who have failed to ruxolitinib, the therapeutic value from fedratinib may be modest especially when exposure time to ruxolitinib was more than 12 months. We may hypothesize that earlier switching from ruxolitinib to fedratinib may yield a better result.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"15 1","pages":"1-11"},"PeriodicalIF":2.4,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142250121","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Beneficial effect of integrated nutritional interventions in patients with hematological diseases undergoing hematopoietic stem cell transplant.","authors":"Mimi Geng,Zihui Sun","doi":"10.1159/000541154","DOIUrl":"https://doi.org/10.1159/000541154","url":null,"abstract":"INTRODUCTIONThe nutritional status of patients undergoing hematopoietic stem cell transplantation (HSCT) is critically important. This study was aimed to assess the impact of comprehensive nutritional interventions on the well-being of individuals with hematological diseases who underwent HSCT.METHODSA total of 175 patients with hematological diseases who underwent HSCT were included, with 94 in the control group and 81 in the research group. Patients in the control group received standard nursing care, while those in the research group underwent integrated nutritional interventions. Nutritional status was evaluated using the mini nutritional assessment (MNA) and subjective global assessment (SGA), along with body measurements and serum levels of albumin, prealbumin, and hemoglobin.RESULTSThere were no significant differences in the proportion of malnourished patients evaluated by MNA or SGA between the control and research groups at admission. However, at discharge and 3 months post-discharge, fewer patients in the research group were malnourished compared to the control group, as assessed by both MNA and SGA. At admission, there were no significant differences in albumin, prealbumin, hemoglobin levels, weight, calf circumference (CC), triceps skinfold thickness (TSF), or subscapular skinfold thickness (SSF) between groups. However, at discharge and 3 months post-discharge, the levels of these indicators significantly decreased compared to those upon admission. Levels of albumin, prealbumin, and hemoglobin, as well as weight, CC, TSF, and SSF, were significantly higher in the research group than in the control group at both discharge and 3 months post-discharge.CONCLUSIONHSCT led to a decline in nutritional status among patients with hematological diseases, but integrated nutritional interventions effectively improved their nutritional status.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":"39 1","pages":"1-14"},"PeriodicalIF":2.4,"publicationDate":"2024-09-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142250122","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Impact of Routine Addition of Antithymocyte Globulin to Standard GVHD Prophylaxis in Myeloablative Unrelated Donor Transplants: Important Gains in Graft-versus-Host Disease Prevention though No Difference in Overall Survival.","authors":"Ni Bai, Wasithep Limvorapitak, Robert Henderson, Yasser Abou Mourad, Shanee Chung, Donna Forrest, Kevin Hay, Florian Kuchenbauer, Stephen Nantel, Sujaatha Narayanan, Thomas Nevill, Maryse Power, Judith Rodrigo, Claudie Roy, David Sanford, Kevin Song, Ryan Stubbins, Heather Sutherland, Cynthia Toze, Jennifer White","doi":"10.1159/000541071","DOIUrl":"10.1159/000541071","url":null,"abstract":"<p><strong>Introduction: </strong>Antithymocyte globulin (ATG) has been demonstrated to reduce the incidence of graft-versus-host disease (GVHD); however, it remains controversial whether these gains are offset by an increase in relapse.</p><p><strong>Methods: </strong>We conducted a retrospective historical control study consisting of patients (n = 210) who underwent myeloablative allogeneic hematopoietic stem-cell transplantation (HSCT) from 2014 to 2020.</p><p><strong>Results: </strong>The incidence of acute GVHD was lower in the ATG group (51.4%) than the non-ATG group (control) (70.0%, p = 0.010). The incidence of chronic GVHD was also lower in the ATG group at 1-year (36.4% vs. 62.9%, p &lt; 0.001) and 2-year (40.0% vs. 65.7%, p &lt; 0.001) post-HSCT. The mortality due to GVHD was higher in the control (18.5%) than the ATG group (4.3%; p = 0.024). The severe GVHD-relapse-free survival was higher in the ATG group (36.4%) than the control (12.9%; p &lt; 0.001). Nevertheless, the 2-year overall survival was similar.</p><p><strong>Conclusion: </strong>Our results confirm the effectiveness of ATG in prevention of GVHD in the real-world setting and enhanced GVHD-free survival. An important result is the equalization of overall survival between the ATG and control groups at 1- and 2-year post-HSCT and implies that earlier GVHD-associated mortality may be offset by later relapse mortality producing similar overall survival over time.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.7,"publicationDate":"2024-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142091382","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Value of the Pretransplant Fibrosis-4 Index on Non-Relapse and Overall Mortality following Unrelated Single-Unit Cord Blood Transplantation in Adults.","authors":"Takaaki Konuma, Maki Monna-Oiwa, Seiko Kato, Masamichi Isobe, Satoshi Takahashi, Yasuhito Nannya","doi":"10.1159/000541157","DOIUrl":"10.1159/000541157","url":null,"abstract":"<p><strong>Introduction: </strong>The fibrosis-4 (FIB-4) index is a noninvasive marker of liver fibrosis. The FIB-4 index predicts poor outcomes in patients with hepatic and non-hepatic diseases. However, the association of the FIB-4 index with mortality and liver-related clinical outcomes following cord blood transplantation (CBT) is unclear.</p><p><strong>Methods: </strong>We retrospectively evaluated the impact of the pretransplant FIB-4 index on outcomes in 336 adults following single-unit unrelated CBT at our institution.</p><p><strong>Results: </strong>In multivariate analyses, when the FIB-4 index &lt;1.3 group was used as the reference, non-relapse mortality was significantly higher in the FIB-4 index 1.3-2.67 (hazard ratio [HR], 2.51; 95% confidence interval [CI], 1.19-5.30) and FIB-4 index &gt;2.67 (HR, 2.34; 95% CI, 1.12-4.90) groups. Overall mortality was significantly higher in the FIB-4 index &gt;2.67 group (HR, 1.66; 95% CI, 1.00-2.73), but with only marginal significance in the FIB-4 index 1.3-2.67 group (HR, 1.59; 95% CI, 0.96-2.64). Hematopoietic recovery, acute and chronic graft-versus-host disease of the liver, and veno-occlusive disease/sinusoidal obstruction syndrome were not associated with the pretransplant FIB-4 index.</p><p><strong>Conclusion: </strong>The pretransplant FIB-4 index is accurate and useful in predicting mortality in adult patients undergoing single-unit unrelated CBT.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.7,"publicationDate":"2024-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142091381","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes of Patients with Myeloid Malignancies and Cardiovascular Disease Undergoing Allogeneic Stem Cell Transplantation.","authors":"Gabriela Sanchez-Petitto, Olga Goloubeva, James Childress, Tahreem Iqbal, Jack Masur, Max An, Safwan Muhammad, Justin Lawson, Grace Li, Brian Barr, Ashkan Emadi, Vu H Duong, Nancy M Hardy, Aaron P Rapoport, Maria R Baer, Sandrine Niyongere, Jean A Yared","doi":"10.1159/000541131","DOIUrl":"10.1159/000541131","url":null,"abstract":"<p><strong>Introduction/background: </strong>Reduced-intensity conditioning (RIC) and nonmyeloablative (NMA) regimens have enabled patients with cardiovascular disease (CVD) to undergo allogeneic stem cell transplantation (allo-HSCT). However, little is known about long-term outcomes, including cardiovascular (CV) complications.</p><p><strong>Methods: </strong>We retrospectively studied 99 consecutive patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who underwent allo-HSCT between September 1, 2013, and November 30, 2020. Overall survival (OS), progression-free survival (PFS), nonrelapse mortality (NRM), cumulative incidence of relapse, and cumulative incidence of acute and chronic graft-versus-host disease (GvHD) were compared in patients with and without CV risk factors or disease.</p><p><strong>Results: </strong>Preexisting CVD was present in 34 of 99 patients (34%). CVD patients more commonly had reduced-intensity conditioning (91% vs. 60%, p = 0.001) and unrelated donors (56% vs. 35%, p = 0.04). Early adverse cardiac events occurred more frequently in the CVD versus no-CVD group (38% vs. 14%), particularly arrhythmias (21% vs. 5%; p = 0.04). CVD patients tended to have poorer OS and PFS outcomes (HR = 1.98, [1.00, 3.92]; HR = 1.89, [0.96-3.72], respectively). OS rate at 1, 2, and 3 years for CVD versus no-CVD patients was 66% versus 72%, 55% versus 64%, and 46% versus 62%, respectively. Causes of death in the CVD and no-CVD groups were infections (53% vs. 28%), relapsed disease (32% vs. 52%), and CV events (10% vs. 3%).</p><p><strong>Conclusion: </strong>Based on these data, predictive models to identify patients with CVD with higher risk of post-allo-HSCT complications and mortality and strategies to mitigate these risks should be developed.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-12"},"PeriodicalIF":1.7,"publicationDate":"2024-08-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142078736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Disseminated Mucormycosis and T-Cell-Depleted Allogeneic Stem Cell Transplantation: An Unusual Case Study.","authors":"Oana Diana Dragoi, Mili Shah, Victoria Potter, Daniele Avenoso, Pramila Krishnamurthy, Alireza Abdolrasouli, Silke Schelenz, Julie Chandra, Varun Mehra","doi":"10.1159/000540640","DOIUrl":"10.1159/000540640","url":null,"abstract":"<p><strong>Introduction: </strong>Invasive fungal infections are a primary cause of morbidity and mortality in patients with haematological malignancies.</p><p><strong>Case presentation: </strong>We describe an unusual clinical and radiological presentation of invasive mucormycosis (IM) in a 69-year-old patient with relapsed acute myeloid leukaemia. The patient was diagnosed with disseminated IM with involvement of the central nervous system in an atypical location, lung, spleen, muscle, bone, and heart, after having completed induction and bridging chemotherapy to allogeneic haematopoietic stem cell transplant (HSCT). Her clinical presentation was atypical with mild neurological symptoms slowly progressing over 2 months and without appropriate signs of systemic inflammation. Mucorales was eventually confirmed from bronchoalveolar lavage and subdural collection.</p><p><strong>Conclusion: </strong>This report highlights the difficult challenges of managing disseminated IM in an immunocompromised patient, where close multidisciplinary specialist care enabled successful treatment, followed by T-cell-depleted allogeneic HSCT for a high-risk haematological malignancy.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-7"},"PeriodicalIF":1.7,"publicationDate":"2024-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142016008","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Latin-American Experience in POEMS Syndrome: A Study of the GELAMM (Grupo de Estudio Latinoamericano de Mieloma Múltiple).","authors":"Moisés Manuel Gallardo-Pérez, Paola Negrete-Rodríguez, Morie A Gertz, Camila Peña, Eloisa Riva, Virginia Gilli, Gloritza Rodríguez, César Samánez, Joaquín Ferreira, Sergio Portiño, Jacqueline Montaña, Pilar León, Yaima Gutiérrez, Caroline Del-Castanhel, Cristian Seehaus, Maria Eugenia Funes, Rodrigo Meneces-Bustillo, Patricio Duarte, Claudia Shanley, Giannini Elvira, Paola Ochoa, Hernán López-Vidal, Humberto Martinez-Cordero, Jule Vasquez, Alana von-Glasenapp, Javiera Donoso, Jose Luis Viñuela, Guillermo J Ruiz-Delgado, Guillermo J Ruiz-Argüelles","doi":"10.1159/000540890","DOIUrl":"10.1159/000540890","url":null,"abstract":"<p><strong>Introduction: </strong>POEMS syndrome is a rare paraneoplastic syndrome caused by an underlying plasma cell disorder. The acronym refers to the following features: polyradiculoneuropathy, organomegaly, endocrinopathy, monoclonal paraproteinemia, and skin changes.</p><p><strong>Methods: </strong>The study was conducted at 24 hematological centers across 8 Latin-American countries. The study included a total of 46 patients {median age was 52 years (interquartile range [IQR]: 42-61.5), 30 males and 16 females} fulfilling the POEMS syndrome criteria diagnosed over a period of 12 years (January 1, 2011, through July 31, 2023). Epidemiological and clinical data were collected in an ad hoc database sent to the members of GELAMM, as well as the Kolmogorov-Smirnov test and Kaplan-Meier estimates.</p><p><strong>Results: </strong>All patients had polyneuropathy and monoclonal gammopathy; 89% had bone marrow plasma cell infiltration, 33% had sclerotic bone lesions. Only 10 patients underwent vascular endothelial growth factor (VEGF) testing in plasma samples. The paraproteinemia was IgG λ in 32% and IgA λ in 30%. 59% patients presented with cutaneous changes, mainly hyperpigmentation, 54% had organomegaly, and 74% endocrinopathy. The median interval from symptom onset to diagnosis was 7.7 months (IQR: 4.0-12.6). 69% of patients received a single line of treatment. The median follow-up period was 25 months (IQR: 9.37-52.0) and the 2-year overall survival rate was 100%. All patients who underwent transplantation (43%) are alive, with a median follow-up of 45.62 months (IQR: 15.46-70).</p><p><strong>Conclusion: </strong>This study investigates POEMS syndrome in Latin America and presents an initial overview of the disease in the region. VEGF usage is recommended for accurate diagnosis, but only 7 hematology centers in the region used it. Survival rate in Latin America is comparable with those observed internationally.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":1.7,"publicationDate":"2024-08-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141915838","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prognostic Factors for Chronic Thrombocytopenia in Systemic Lupus Erythematosus with Immune Thrombocytopenia.","authors":"Soo Min Ahn, Eun-Ji Choi, Ji Seon Oh, Yong-Gil Kim, Chang-Keun Lee, Bin Yoo, Seokchan Hong","doi":"10.1159/000540192","DOIUrl":"10.1159/000540192","url":null,"abstract":"<p><strong>Introduction: </strong>We aimed to identify the clinical characteristics and risk factors for chronic immune thrombocytopenia (ITP) in patients with systemic lupus erythematosus (SLE).</p><p><strong>Methods: </strong>We retrospectively reviewed patients diagnosed with SLE-associated ITP between January 2000 and December 2021. Patient characteristics were analyzed according to the progression of chronic thrombocytopenia. No response was defined as a platelet count &lt;30 × 109/L or less than double the baseline count after treatment. Factors associated with chronic ITP were evaluated by logistic regression analysis.</p><p><strong>Results: </strong>Among the 121 patients with SLE-associated ITP, 27 progressed to chronic ITP lasting more than 1 year after initial diagnosis. The median initial platelet count was significantly lower in patients with chronic thrombocytopenia than in those without the disease (16 vs. 51 × 109/L). Patients who did not achieve a response within 1 month of treatment exhibited a high probability of progressing to chronic ITP (55.6 vs. 22.3%, p &lt; 0.001). Multivariable analysis revealed that severe thrombocytopenia at baseline (&lt;20 × 109/L) (adjusted odds ratio [aOR] = 13.628, 95% confidence interval [CI] = 3.976-46.791) and no response within 1 month (aOR = 9.171, 95% CI = 2.776-30.298) were significantly associated with the risk of progression to chronic ITP in patients with SLE. Approximately one-quarter of the patients with SLE-associated ITP progressed to chronic ITP.</p><p><strong>Conclusion: </strong>Severe thrombocytopenia and failure to achieve a response within 1 month were risk factors for the development of chronic ITP in those patients.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":1.7,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141791634","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Case of Recurrent Localized Pulmonary Nodular Light Chain Amyloidosis Treated with Daratumumab plus CyBorD.","authors":"Ted Raddell, Farah Ashraf, Xiaofeng Zhao, Osheen Abramian, Tulin Budak-Alpdogan","doi":"10.1159/000540272","DOIUrl":"10.1159/000540272","url":null,"abstract":"<p><strong>Introduction: </strong>Nodular pulmonary amyloidosis (NPA) is a localized form of light chain (AL) amyloidosis often found incidentally and typically has an indolent and benign disease course treated with resection or local excision. We present a patient with recurrent localized AL amyloidosis who required further treatment.</p><p><strong>Case presentation: </strong>A 63-year-old female with monoclonal gammopathy of undetermined significance (MGUS) was found to have pulmonary AL amyloid on wedge resection and later had recurrence. The patient did not have signs of clonal plasma cell proliferation or systemic AL amyloid. She was treated with daratumumab, cyclophosphamide, bortezomib, and dexamethasone. After initiation of treatment, the patient has had significant hematologic and radiographic response.</p><p><strong>Conclusion: </strong>The patient had NPA recurrence with organ dysfunction without systemic disease. Because the presentation of recurrent pulmonary AL amyloidosis is rare, there is no published evidence on treatment. However, the patient has had hematologic and radiographic improvement after initiating treatment with a systemic protocol.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-6"},"PeriodicalIF":1.7,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141791633","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Follow-Up of Eltrombopag Treatment for Patients with Cyclosporin A Refractory/Relapsed Transfusion-Dependent Non-Severe Aplastic Anemia: A Report from a Single Center in China.","authors":"Qinglin Hu, Yuan Yang, Chen Yang, Miao Chen, Bing Han","doi":"10.1159/000539905","DOIUrl":"10.1159/000539905","url":null,"abstract":"<p><strong>Introduction: </strong>Aplastic anemia (AA) is characterized by bone marrow failure and cytopenia. Eltrombopag (ELT) is effective and safe for treating refractory/relapsed AA; however, reports on the long-term outcomes of transfusion-dependent non-severe AA (TD-NSAA) are limited.</p><p><strong>Methods: </strong>Patients with TD-NSAA refractory to immunosuppressive therapy (IST) or relapsed after IST, treated with ELT alone, and followed up for at least 12 months were retrospectively enrolled. The baseline characteristics of patients, efficacy and adverse effects of ELT, and relapse and clone evolution rates after ELT were documented.</p><p><strong>Results: </strong>Of the 55 patients with TD-NSAA included, 24 (43.6%) were men. Median age at diagnosis was 46 (19-80) years. Twenty-four patients had relapsed TD-NSAA, and 31 patients had refractory TD-NSAA. During the median follow-up period of 28 (12-48) months, the overall and complete response rates at 3, 6, and 12 months of ELT treatment were 38.2, 60.0, and 52.7 and 9.1, 14.6, and 9.1%, respectively. After a median follow-up of 28 (12-48) months, 21.2% (7/33) of patients experienced relapse, with a median duration from ELT treatment to relapse of 14 (6-45) months.</p><p><strong>Conclusion: </strong>ELT was effective in patients with relapsed/refractory TD-NSAA, with tolerable adverse effects.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2024-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141632312","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}