Acta Haematologica最新文献

{"title":"Clinical Challenges in Treating Cancer Associated Thrombosis: A Clinically Oriented Review.","authors":"Idan Goldberg, Galia Spectre, Pia Raanani, Hugo Ten Cate, Avi Leader","doi":"10.1159/000542872","DOIUrl":"https://doi.org/10.1159/000542872","url":null,"abstract":"<p><strong>Background: </strong>Managing cancer associated thrombosis (CAT) is a significant clinical challenge due to several factors such as increased bleeding tendency, frailty, and drug - drug interactions. For many years, the drug of choice for treating CAT was low molecular weight heparin (LMWH); Recently, direct oral anticoagulants (DOACs) entered to the therapeutic milieu of CAT. However, due to the large diversity among patients with CAT in clinical and laboratory characteristics not all patients will equally benefit from treatment with DOACs. Furthermore, several subgroups of patients with CAT have specific characteristics that influence the anticoagulant decision-making process.</p><p><strong>Summary: </strong>In this review, we present four different theoretical clinical case scenarios, each representing a different challenge that is associated with thrombosis management; brain metastasis, malignancies of the gastrointestinal tract, drug-drug interactions (DDI) and thrombocytopenia. By reviewing current literature, we suggest our clinical approach for managing these cases in the era of DOACs.</p><p><strong>Key messages: </strong>(1) The management of patients with brain tumors and CAT is challenging due to increased risk for both intracranial hemorrhage and recurrent venous thromboembolism (VTE). Both LMWH and DOACs are optional treatment in this setting. (2) There are conflicting data regarding the bleeding risk in patients with GI malignancies. Treatment with LMWH should be considered specifically in patients with advanced disease and unresectable tumors. (3) There is a paucity of data regarding DDI in patients with CAT. However, caution should be exercised when prescribing DOACs to patients receiving concurrent medications that either affect DOAC metabolism or influence bleeding risk. (4) The management of patients with CAT and thrombocytopenia depends on the severity of thrombocytopenia and the timing of the thrombotic event.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-27"},"PeriodicalIF":1.7,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142811911","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-life\" Data of Zanubrutinib in Patients with Waldenström Macroglobulinemia - A Multi-Center Retrospective Study.","authors":"Gilad Itchaki, Ohad Benjamini, Mor Levi, Anatoly Nemets, Shai Ygna, Mahdi Assaly, Anna Gourevitch, Moshe Gatt, Revital Saban, Pia Raanani, Iuliana Vaxman","doi":"10.1159/000542936","DOIUrl":"https://doi.org/10.1159/000542936","url":null,"abstract":"<p><strong>Introduction: </strong>Waldenström macroglobulinemia (WM) is a rare indolent lymphoma. Zanubrutinib (ZAN), a second-generation BTK inhibitor, has been approved for the treatment of WM in any line of therapy in 2021. Between November 2020 and January 2022, an expanded access program of ZAN opened in Israel for the treatment of patients with relapsed refractory (RR)-WM or those ineligible for chemotherapy or ibrutinib in first line.</p><p><strong>Methods: </strong>This is a multi-center retrospective study aiming to provide real-world data on ZAN in patients with WM in Israel. Demographic and clinical data were collected and coded from electronic files. Response was evaluated by the investigator's assessment. As the program closed, patients transitioned to commercial ZAN.</p><p><strong>Results: </strong>13 patients (12 RR; 1 treatment-naive) were enrolled across 8 centers in Israel. The median age at ZAN initiation was 71 years (range, 50-85); 6 were males; 10 had high IPSS-WM. RR pts had a median of 1 (1-4) prior lines of therapy. Other than progressive disease after chemoimmunotherapy (CIT), the most common considerations for choosing ZAN were patients' age and/or comorbidities (n=5), as well as ibrutinib toxicity. The initial ZAN dose was reduced in 4 pts. The median time on ZAN was 19.5 months (2.9-29.5). Of 12 evaluable patients, the ORR was 83% with 3 minor responses, 6 PR, and 1 VGPR. With a median follow-up of 19.6 months, 7 patients were still on ZAN, 5 progressed, 4 while on ZAN, and 1 after ZAN was stopped due to AE. 18 months PFS and OS were 60.5% and 77%, respectively. Eight (61%) patients had AEs of any grade, and 3 (23%) of grade 3-4; 2 stopped ZAN due to congestive heart failure and extreme fatigue.</p><p><strong>Conclusion: </strong>The results of this real-world high-risk population are consistent with prospective studies highlighting the efficacy and safety of ZAN.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":1.7,"publicationDate":"2024-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142779024","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":"Haploidentical allogeneic hematopoietic cell transplantation following two courses of venetoclax and azacytidine therapy in patients over 55 years old with acute myelogenous leukemia: Comment.","authors":"Hinpetch Daungsupawong, Viroj Wiwanitkit","doi":"10.1159/000542843","DOIUrl":"https://doi.org/10.1159/000542843","url":null,"abstract":"","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-3"},"PeriodicalIF":1.7,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142765464","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":"Coronary Artery Disease and Microvascular Ischemia in Patients with Cardiac Amyloidosis.","authors":"Osnat Itzhaki Ben Zadok, Iuliana Vaxman, Sara Hoss, Yeela Talmor-Barkan, Tali Steinmetz, Pia Raanani, Ran Kornowski, Ashraf Hamdan","doi":"10.1159/000542510","DOIUrl":"https://doi.org/10.1159/000542510","url":null,"abstract":"<p><strong>Introduction: </strong>The prevalence of pre-existing obstructive coronary artery disease (CAD) and the occurrence of anginal chest pain as a presenting symptom in patients with light chain (AL) and transthyretin (ATTR) cardiac amyloidosis (CA) are undetermined.</p><p><strong>Methods: </strong>A single-center analysis of clinical, laboratory, imaging and angiographic characteristics of CA cohort.</p><p><strong>Results: </strong>Included were 98 CA patients (43 AL, 47 wtATTR, 8 mutant ATTR). Eighteen patients (18%) had pre-existing obstructive CAD at the time of CA diagnosis. These patients were older and had worse left ventricular ejection fraction, yet revealed similar cardiac biomarkers' levels. The 3-year survival rate was comparable between patients with versus without pre-existing CAD (p=0.974). Anginal chest pain was a presenting symptom of newly-diagnosed CA in 24% (n=19) of patients with no pre-existing CAD, 53% (n=10) of which had AL-CA. Two patients had an acute myocardial infarction. The prevalence of diabetes mellitus, dyslipidemia, hypertension and smoking was similar among CA patients presenting with versus without chest pain. Of the newly-diagnosed CA patients with no pre-existing CAD who underwent symptoms evaluation (n=37), 99mTc-Sestamibi myocardial perfusion scintigraphy demonstrated stress-induced perfusion defects in 45% (9/20) and normal study in 45% (9/20) of patients. Coronary evaluation revealed non-obstructive coronary artery lesions or normal coronaries in 75% of patients (18/24).</p><p><strong>Conclusion: </strong>CA patients may initially present with anginal chest pain and myocardial perfusion defects which may reflect coronary microvascular ischemia. CA should be considered in the differential diagnosis of patients presenting with chest pain, non-obstructive CAD and elevated cardiac biomarkers.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":1.7,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142714996","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":"Historical Perspective of Allogeneic Hematopoietic Stem Cell Transplantation for Multiple Myeloma.","authors":"Muhammad Faisal Aslam, Asfand Yar Cheema, Daniyal Shahid, Bibi Maryam, Debduti Mukhopadhyay, Mishaal Munir, Ali Najam, Hossam M Ali, Qaiser Bashir, Faiz Anwer","doi":"10.1159/000542704","DOIUrl":"https://doi.org/10.1159/000542704","url":null,"abstract":"<p><strong>Background: </strong>Advances in novel therapies have improved outcomes for multiple myeloma (MM) patients and the use of allo-SCT has decreased. Current guidelines no longer support allo-SCT as consolidation therapy for newly diagnosed MM, even in high-risk cases.</p><p><strong>Summary: </strong>Allo-SCT is now typically considered only within clinical trials for young, high-risk patients with relapsed or refractory MM (RRMM). It has not proven favorable despite its historical use. CAR T-cell therapy and bispecific antibodies have shown promise in treating triple- and penta-exposed/refractory MM, yet relapse remains common with poor survival rates. The efficacy of allo-SCT following BCMA-directed therapy and other new T-cell-directed therapies is unclear. Allo-SCT might be a viable option for eligible patients who relapse after these therapies, or where such options are unavailable. Advancements in reduced-intensity conditioning regimens have led to lower toxicity and transplant-related (TR) morbidity, lower graft versus host disease (GVHD) and TR mortality. Expanded use of alternative donors, like haploidentical donors, has yielded comparable outcomes. Better post-transplant GVHD regimens and maintenance strategies to prevent relapse have been developed.</p><p><strong>Key messages: </strong>This review analyzes available literature to better understand the safety, efficacy, and current role of allo-SCT in managing MM. Newer regimens are needed as routine use of allo-SCT cannot be recommended.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-21"},"PeriodicalIF":1.7,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142714998","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":"RESULTS OF LONG-TERM THERAPY WITH A BIOSIMILAR OF ECULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA.","authors":"Alexander D Kulagin, Vadim V Ptushkin, Elena A Lukina, Igor L Davydkin, Alexander V Korobkin, Tatiana S Konstantinova, Elena Yu Komartseva, Natalia V Minaeva, Tatiana A Mitina, Olesya U Klimova, Evgeniya G Arshanskaya, Vitalii D Latyshev, Oksana A Markova, Eugene V Zuev","doi":"10.1159/000542294","DOIUrl":"https://doi.org/10.1159/000542294","url":null,"abstract":"<p><strong>Introduction: </strong>The study aimed to assess the safety, immunogenicity, and efficacy of long-term therapy with biosimilar of eculizumab (Elizaria®) in PNH patients.</p><p><strong>Methods: </strong>The study included 30 patients with PNH who had completed previous clinical trials. Of these, 25 patients continued receiving the biosimilar product, and 5 patients switched from the originator product Soliris. The maximum duration of follow-up was 104 weeks, during which the investigational product was administered 52 times at a standard dose.</p><p><strong>Results: </strong>Throughout the study, the levels of LDH, hemoglobin, reticulocytes, and PNH clone remained stable compared to baseline, regardless of the previous therapy (p > 0.05). There were no significant differences in the number of patients with chronic kidney disease at different visits, as well as in the number of patients who received donor red blood cell and platelet transfusions during the study (p > 0.05). There were two cases of adverse reactions reported in two patients (6.6%): elevated aspartate aminotransferase (3.3%) and alopecia (3.3%). Immunogenicity analysis showed no significant differences in the frequency of anti-drug antibody detection compared to baseline (p > 0.05).</p><p><strong>Conclusion: </strong>The study findings confirm the long-term efficacy and safety of biosimilar in patients with PNH.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":1.7,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142715000","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":"Shorter Duration of Blinatumomab Administration to 14 Days Has Same Efficacy and Safety Profile in Treatment of Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia: A Retrospective Single-Center Study.","authors":"Jinyu Kong, Wenjing Miao, Jialing Lu, Yin Liu, Xin Kong, Huiying Qiu, Baoquan Song","doi":"10.1159/000542060","DOIUrl":"10.1159/000542060","url":null,"abstract":"<p><strong>Introduction: </strong>Treatment of patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (r/r BCP-ALL) remains a significant clinical challenge. Many new strategies are changing the treatment landscape of r/r BCP-ALL in recent years. Blinatumomab has improved outcomes in r/r BCP-ALL, though high treatment costs and extended hospital stays are significant concerns. We considered that shortening the duration of blinatumomab administration during induction therapy might solve these problems.</p><p><strong>Methods: </strong>We retrospectively analyzed 19 patients with r/r BCP-ALL treated with different durations of blinatumomab, where 10 patients received blinatumomab for 14 days (Bli 14D group) and 9 received it for a longer duration (LT group, 21-28 days).</p><p><strong>Results: </strong>The overall response rate (ORR) was 63.2% (12/19) of patients in total, and the ORRs in 14D and LT groups were almost the same (60% and 66.6%, respectively). The median overall survival was not reached in either groups. The median event-free survival time was 4.1 months in LT group and not reached in D14 group. The most common adverse events were consistent with previous reports, including cytokine release syndrome, neurologic toxicity, and hematological toxicity.</p><p><strong>Conclusion: </strong>A 14-day blinatumomab administration may be a promising and well-tolerated regimen in r/r BCP-ALL, offering the same ORR and survival rates.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-6"},"PeriodicalIF":1.7,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142520636","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":"Thiamine-Responsive Megaloblastic Anemia Syndrome Mimicking Myelodysplastic Neoplasm.","authors":"Christina Klötzer, Franziska Schnabel, Anne-Sophie Kubasch, Madlen Jentzsch, Georg-Nikolaus Franke, Jens Uhlig, Helene Faust, Robin-Tobias Jauss, Henry Oppermann, Denny Popp, Klaus H Metzeler, Johannes R Lemke, Vladan Vučinić, Uwe Platzbecker","doi":"10.1159/000542286","DOIUrl":"10.1159/000542286","url":null,"abstract":"<p><strong>Introduction: </strong>Thiamine-responsive megaloblastic anemia syndrome (TRMA) is a rare autosomal recessive disease with a homozygous or compound-heterozygous mutation in the SLC19A2 gene characterized by megaloblastic anemia, diabetes mellitus (DM), and sensorineural hearing loss with onset in childhood. Folic acid and vitamin B12 in serum are normal with dysplastic erythropoiesis in the bone marrow often mimicking myelodysplastic neoplasms (MDS) as a potential differential diagnosis. Thiamine substitution leads to normalization of anemia, without effects on hearing loss or DM.</p><p><strong>Case presentation: </strong>We report about a 38-year-old male patient, presented with a 12-year history of anemia, insulin dependent DM, optic neuropathy, and a cataract since early childhood. The laboratory showed megaloblastic anemia. Other values were normal. The bone marrow smear showed dysplastic erythropoiesis with megaloblastic changes, and normal findings in cytogenetic and molecular genetic examinations. Next-generation sequencing-based diagnostics revealed a heterozygous missense variant in the SLC19A2 gene on the maternal allele and a 3.4 Mb inversion in the chromosomal region 1q24.2 with breaking points in FAM78B and SLC19A2 on the paternal allele. Treatment with oral thiamine 100 mg daily was initiated, and 12 weeks later hemoglobin levels and bone marrow morphology had normalized.</p><p><strong>Conclusion: </strong>Late-onset TRMA should be considered in adult patients with indicative comorbidities and a typical phenotype, which may mimic features of MDS.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-5"},"PeriodicalIF":1.7,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142520637","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":"Efficacy of Haploidentical Allogeneic Hematopoietic Cell Transplantation following Two Courses of Venetoclax and Azacytidine Therapy in Patients over 55 Years Old with Acute Myelogenous Leukemia.","authors":"Junjie Cao, Xianxu Zhuang, Danjie Luo, Renzhi Pei, Ying Lu, Dong Chen, Shuangyue Li, Xiaohong Du, Xuhui Liu","doi":"10.1159/000542034","DOIUrl":"10.1159/000542034","url":null,"abstract":"<p><strong>Introduction: </strong>The combination of venetoclax (VEN) and azacytidine (AZA) has demonstrated potential in achieving rapid and effective remissions in elderly patients with acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation is a promising potential cure for high-risk AML, as VEN-based therapies have a worse prognosis in elderly patients. This study aimed to assess the efficacy of sequential haploidentical HSCT following two courses of VEN and AZA therapy in patients with AML aged 55 years and older.</p><p><strong>Methods: </strong>We conducted a retrospective study on AML patients aged 55-70 years who received intensive chemotherapy or two courses of VEN/AZA therapy, followed by haploidentical allo-HSCT (haplo-HSCT) based on disease risk degree, measurable residual disease status, and patient's preference.</p><p><strong>Results: </strong>Between January 2019 and December 2023, 141 newly diagnosed AML patients received initial treatment with intensive chemotherapy or VEN/AZA therapy. Among them, 64 patients received haplo-HSCT, while 77 did not. The 1-year overall survival (OS) and relapse-free survival (RFS) of patients who received haplo-HSCT were significantly higher than those who did not receive haplo-HSCT (p < 0.05). Among patients who received transplantation, there was no significant difference in 1-year OS and RFS between the VEN/AZA and intensive chemotherapy groups: 76.3% versus 69.3% (p = 0.367) for OS, and 74.5% versus 69.7% (p = 0.473) for RFS. High-risk ELN stratification and the presence of ≥4 gene mutations were associated with lower OS and RFS in both univariate and multivariate analyses.</p><p><strong>Conclusions: </strong>AML patients over 55 years of age who received haplo-HSCT after two courses of VEN/AZA therapy had outcomes similar to those who received haplo-HSCT after intensive chemotherapy, suggesting that two courses of VEN/AZA therapy as a bridge to haplo-HSCT are feasible for patients over 55 years old.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":1.7,"publicationDate":"2024-10-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142492694","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 Use of Ruxolitinib in Patients with Myelofibrosis and Anemia or Thrombocytopenia at Diagnosis.","authors":"Jingbo Yu, Emily Bland, Tammy Schuler, Thomas Cordaro, Evan Braunstein","doi":"10.1159/000541549","DOIUrl":"10.1159/000541549","url":null,"abstract":"<p><strong>Introduction: </strong>Ruxolitinib is approved for treatment of myelofibrosis. We evaluated ruxolitinib in patients with anemia (hemoglobin <10 g/dL) or thrombocytopenia (platelet count ≤100 × 109/L) at diagnosis.</p><p><strong>Methods: </strong>This was a retrospective, secondary analysis of a Cardinal Health Oncology Provider Extended Network medical chart review of adults with myelofibrosis diagnosed between 2012 and 2016 who received first-line ruxolitinib.</p><p><strong>Results: </strong>176 patients received first-line ruxolitinib and were included in this analysis. At diagnosis, 120 patients had hemoglobin concentrations <10 g/dL and 59 had a platelet count ≤100 × 109/L. Most patients (95%) with thrombocytopenia also had anemia. Median time of observation after diagnosis was 21.4 months. Among patients with anemia or thrombocytopenia, ruxolitinib dose at end of study was ≥10 mg twice daily (bid) in 88.3% and 83.1%, respectively. Ruxolitinib treatment was ongoing in 76.1% of patients overall and was rarely discontinued for anemia or thrombocytopenia (n = 2 total, 1.1%). Per the treating physician, 79.7% of patients had improved symptoms and 62.7% improved spleen size.</p><p><strong>Conclusion: </strong>Most patients with myelofibrosis and anemia or thrombocytopenia at diagnosis tolerated and maintained a ruxolitinib dose ≥10 mg bid for nearly 2 years, resulting in clinical benefit. This real-world evidence supports observations from prospective clinical trials of ruxolitinib in myelofibrosis.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-11"},"PeriodicalIF":16.4,"publicationDate":"2024-10-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142455471","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}