Therapeutic Advances in Hematology最新文献

{"title":"Anemia is associated with long-term exposure to PM_2.5 and its components: a large population-based study in Southwest China.","authors":"Congyuan He,&nbsp;Linshen Xie,&nbsp;Lingxi Gu,&nbsp;Hongyu Yan,&nbsp;Shiyu Feng,&nbsp;Chunmei Zeng,&nbsp;Wangjiu Danzhen,&nbsp;Xuehui Zhang,&nbsp;Mingming Han,&nbsp;Zhifeng Li,&nbsp;Zhuoma Duoji,&nbsp;Bing Guo,&nbsp;Juying Zhang,&nbsp;Feng Hong,&nbsp;Xing Zhao","doi":"10.1177/20406207231189922","DOIUrl":"https://doi.org/10.1177/20406207231189922","url":null,"abstract":"<p><strong>Background: </strong>Anemia is linked to PM_2.5 (particulate matter with aerodynamic diameters of ⩽2.5 μm) exposure, which can increase the risk of various negative health outcomes. It remains unclear which PM_2.5 components are associated with anemia and the respective contribution of each component to this association.</p><p><strong>Objective: </strong>This study aimed at investigating the association between PM_2.5 and anemia in the general population and to identify the most critical PM_2.5 toxic components in this association.</p><p><strong>Design: </strong>Cross-sectional study.</p><p><strong>Methods: </strong>Our study involved a large cohort of 73,511 individuals aged 30-79 from China's multi-ethnic population. We employed satellite observations and the chemical transport model (GEOS-Chem)to estimate the long-term exposure to PM_2.5 and its components. Anemia was defined, according to WHO guidelines, as Hb levels below 130 g/L for men and below 120 g/L for women. Through logistic regression, we investigated the association between PM_2.5 components and anemia. By utilizing weighted quantile sum (WQS) analysis, we identified key components and gained insights into their combined impact on anemia. Overall, our study sheds light on the relationship between PM_2.5 exposure, its constituents, and the risk of anemia in a large cohort.</p><p><strong>Results: </strong>PM_2.5 and three components, nitrate (NIT), organic matter (OM), and soil particles (SOIL), were associated with anemia. Per-standard deviation increase in the 3-year average concentrations of PM_2.5 [odds ratio (OR): 1.14, 95% confidence interval (CI): 1.01, 1.28], NIT (1.20, 1.06, 1.35), OM (1.17, 1.04, 1.32), and SOIL (1.22, 1.11, 1.33) were associated with higher odds of anemia. In WQS regression analysis, the WQS index was associated with anemia (OR: 1.29, 95% CI: 1.13, 1.47). SOIL has the highest weight among all PM_2.5 components.</p><p><strong>Conclusions: </strong>Long-term exposure to PM_2.5 and its constituents is associated with anemia. Moreover, SOIL might be the most critical component of the relationship between PM_2.5 and anemia. Our research increases the evidence of the association between PM_2.5 and anemia in the general population, and targeted emission control measures should be taken into consideration to mitigate the adverse effects of PM_2.5-related anemia.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/91/fe/10.1177_20406207231189922.PMC10467225.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10305351","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treating accelerated and blast phase myeloproliferative neoplasms: progress and challenges.","authors":"Helen O Ajufo,&nbsp;Julian A Waksal,&nbsp;John O Mascarenhas,&nbsp;Raajit K Rampal","doi":"10.1177/20406207231177282","DOIUrl":"https://doi.org/10.1177/20406207231177282","url":null,"abstract":"<p><p>Myeloproliferative neoplasms (MPNs) are a group of clonal hematologic malignancies that include polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). MPNs are characterized by activating mutations in the JAK/STAT pathway and an increased risk of transformation to an aggressive form of acute leukemia, termed MPN-blast phase (MPN-BP). MPN-BP is characterized by the presence of ⩾20% blasts in the blood or bone marrow and is almost always preceded by an accelerated phase (MPN-AP) defined as ⩾10-19% blasts in the blood or bone marrow. These advanced forms of disease are associated with poor prognosis with a median overall survival (mOS) of 3-5 months in MPN-BP and 13 months in MPN-AP. MPN-AP/BP has a unique molecular landscape characterized by increased intratumoral complexity. Standard therapies used in <i>de novo</i> acute myeloid leukemia (AML) have not demonstrated improvement in OS. Allogeneic hematopoietic stem cell transplant (HSCT) remains the only curative therapy but is associated with significant morbidity and mortality and infrequently utilized in clinical practice. Therefore, an urgent unmet need persists for effective therapies in this advanced phase patient population. Here, we review the current management and future directions of therapy in MPN-AP/BP.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/71/a8/10.1177_20406207231177282.PMC10410182.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10513536","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Considerations for shared decision management in previously untreated patients with hemophilia A or B.","authors":"Jan Astermark,&nbsp;Jan Blatný,&nbsp;Christoph Königs,&nbsp;Cédric Hermans,&nbsp;Victor Jiménez-Yuste,&nbsp;Daniel P Hart","doi":"10.1177/20406207231165857","DOIUrl":"https://doi.org/10.1177/20406207231165857","url":null,"abstract":"<p><p>Recent advances in therapeutics are now providing a wide range of options for adults and children living with hemophilia. Although therapeutic choices are also increasing for the youngest individuals with severe disease, challenges remain about early management decisions, as supporting data are currently limited. Parents and healthcare professionals are tasked with helping children achieve an inclusive quality of life and maintain good joint health into adulthood. Primary prophylaxis is the gold standard to optimize outcomes and is recommended to start before 2 years of age. A range of topics need to be discussed with parents to aid their understanding of the decisions they can make and how these will affect the management of their child/children. For those with a family history of hemophilia, prenatal considerations include the possibility of genetic counseling, prenatal investigations, and planning for delivery, together with monitoring of the mother and neonate, as well as diagnosis of the newborn and treatment of any birth-associated bleeding. Subsequent considerations, which are also applicable to families where infant bleeding has resulted in a new diagnosis of sporadic hemophilia, involve explaining bleed recognition and treatment options, practical aspects of initiating/continuing prophylaxis, dealing with bleeds, and ongoing aspects of treatment, including possible inhibitor development. Over time, optimizing treatment efficacy, in which individualizing therapy around activities can play a role, and long-term considerations, including retaining joint health and tolerance maintenance, become increasingly important. The evolving treatment landscape is creating a need for continually updated guidance. Multidisciplinary teams and peers from patient organizations can help provide relevant information. Easily accessible, multidisciplinary comprehensive care remains a foundation to care. Equipping parents early with the knowledge to facilitate truly informed decision-making will help achieve the best possible longer-term health equity and quality of life for the child and family living with hemophilia.</p><p><strong>Plain language summary: </strong><b>Points to be taken into account to help families make decisions to best care for children born with hemophilia</b> Medical advances are providing a range of treatment options for adults and children with hemophilia. There is, however, relatively limited information about managing newborns with the condition. Doctors and nurses can help parents to understand the choices for infants born with hemophilia. We describe the various points doctors and nurses should ideally discuss with families to enable informed decision-making. We focus on infants who require early treatment to prevent spontaneous or traumatic bleeding (prophylaxis), which is recommended to start before 2 years of age. Families with a history of hemophilia may benefit from discussions before pregnancy, including how an affected c","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10126613/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9365173","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efgartigimod alfa for the treatment of primary immune thrombocytopenia.","authors":"Catherine Broome","doi":"10.1177/20406207231172831","DOIUrl":"https://doi.org/10.1177/20406207231172831","url":null,"abstract":"<p><p>Primary immune thrombocytopenia (ITP) is an acquired autoimmune disorder characterized by isolated thrombocytopenia. Most patients with ITP have antiplatelet antibodies of the immunoglobulin G (IgG) subtype which through interaction with platelet and megakaryocyte glycoproteins result in increased platelet destruction and inhibition of platelet production. There are a variety of therapeutic options available for the treatment of ITP including corticosteroids, IVIgG, TPO-RA, rituximab, fostamatinib, and splenectomy. Long-term remissions with any of these therapies can vary widely and patients may require additional therapy. The neonatal Fc receptor (FcRn) plays a pivotal role in IgG and albumin physiology through recycling pathways. Efgartigimod is a human IgG1-derived fragment that has been modified by ABDEG technology to increase its affinity for FcRn at both physiologic and acidic pH. The binding of efgartigimod to FcRn blocks the interaction of IgG with FcRn facilitating increased lysosomal degradation of IgG and decreasing total IgG levels. Based on the mechanism of action and the known pathophysiology of ITP as well as the efficacy of other therapies such as intravenous immunoglobulin (IVIG), the use of efgartigimod in patients with ITP is attractive. This article will briefly discuss the pathophysiology of ITP, current treatments, and the data available on efgartigimod in ITP.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/0d/49/10.1177_20406207231172831.PMC10176552.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9475887","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment de-escalation in Philadelphia chromosome-positive B-cell acute lymphoblastic leukemia: the emerging role of chemotherapy-free regimens.","authors":"Fadi G Haddad,&nbsp;Jacki Sawyers,&nbsp;Nicholas J Short","doi":"10.1177/20406207231151294","DOIUrl":"https://doi.org/10.1177/20406207231151294","url":null,"abstract":"<p><p>The management of Philadelphia chromosome-positive (Ph-positive) acute lymphoblastic leukemia (ALL) has witnessed major progress over the past two decades. Initially, the incorporation of the first-generation BCR::ABL1 tyrosine kinase inhibitor (TKI) imatinib into intensive chemotherapy regimens improved outcomes compared with chemotherapy alone. The combinations of chemotherapy with second- or third-generation TKIs further improved outcomes, with higher rates of complete molecular remission (CMR) and superior survival. The combination of ponatinib plus chemotherapy resulted in durable remissions and prolonged long-term survival, even in patients who did not receive allogeneic stem cell transplantation (SCT). The promising results seen with later-generation TKIs have caused many to re-evaluate the role of allogeneic SCT for patients who achieve CMR with potent TKI regimens. Recently, the chemotherapy-free combinations of blinatumomab plus TKIs were shown to be safe and effective in newly diagnosed Ph-positive ALL, sparing patients the toxicities associated with intensive chemotherapy. In particular, encouraging early results have been seen with the combination of blinatumomab plus ponatinib, suggesting that this regimen may represent a chemotherapy-free and SCT-sparing strategy for patients with Ph-positive ALL. Herein, we discuss the current evidence for frontline therapies of Ph-positive ALL, the treatment de-escalation strategies over time, and the role of allogeneic SCT in view of the emergence of newer chemotherapy-free regimens using potent TKIs.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9900664/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10681624","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"REFLECT: prospective multicenter non-interventional study evaluating the effectiveness and safety of Sandoz rituximab (SDZ-RTX; Rixathon^®) in combination with CHOP for the treatment of patients with previously untreated CD20-positive diffuse large B-cell lymphoma.","authors":"Manfred Welslau,&nbsp;Boris Kubuschok,&nbsp;Julian Topaly,&nbsp;Burkhard Otremba,&nbsp;Thomas Wolff,&nbsp;Galyna Bryn","doi":"10.1177/20406207231183765","DOIUrl":"https://doi.org/10.1177/20406207231183765","url":null,"abstract":"<p><strong>Background: </strong>REFLECT is the first prospective study of Sandoz biosimilar rituximab (SDZ-RTX) in patients with diffuse large B-cell lymphoma (DLBCL).</p><p><strong>Objective: </strong>To evaluate the 2-year effectiveness and safety of SDZ-RTX as first-line treatment for DLBCL.</p><p><strong>Design: </strong>Real-world, multicenter, open-label, single-arm, non-interventional, post-approval study of SDZ-RTX in combination with cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) in patients with treatment-naïve CD20‑positive DLBCL.</p><p><strong>Methods: </strong>Treatment-naïve, CD20-positive adult patients (⩾18 years) with DLBCL eligible for therapy with R-CHOP were treated with SDZ-RTX-CHOP every 2 or 3 weeks for 6-8 cycles. The effectiveness of SDZ-RTX was measured by the complete response (CR) rate at the end of R-CHOP treatment, as assessed by the treating physician. Progression-free survival (PFS) was assessed at 24 months.</p><p><strong>Results: </strong>A total of 169 patients [52.1% female, median (range) age 70 (24-94) years] with DLBCL were included in the full analysis set. At baseline, 19.5% and 24.3% of patients had Ann Arbor disease stage III or IV, respectively, and most patients (80.5%) had Eastern Cooperative Oncology Group Performance Status of 0 or 1. A total of 100 (59.2%) patients completed the 24-month observation period. In total, 110 [65.1%; 95% confidence interval (CI): 57.4-72.3] patients achieved CR as best response and 50 (29.6%; 95% CI: 22.8-37.1) patients achieved partial response. Overall best response rate was 94.7% (95% CI: 90.1-97.5). One-year PFS was 84.9% (95% CI: 78.2-89.6), while 2-year PFS was 78.5% (95% CI: 70.9-84.4); median PFS was not reached within the observational period. A total of 143 (84.6%) patients experienced ⩾1 adverse event, 53 (31.4%) of which were suspected to be related to study drug.</p><p><strong>Conclusion: </strong>This real-world, 2-year study reconfirms that first-line treatment of CD20-positive DLBCL with R-CHOP using SDZ-RTX is effective and well tolerated.</p><p><strong>Registration: </strong>N/A.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b0/5a/10.1177_20406207231183765.PMC10363888.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9930028","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of damoctocog alfa pegol prophylaxis in patients ⩾40 years with severe haemophilia A and comorbidities: <i>post hoc</i> analysis from the PROTECT VIII study.","authors":"Mark T Reding,&nbsp;Ingrid Pabinger,&nbsp;Pål Andrè Holme,&nbsp;Monika Maas Enriquez,&nbsp;Maria Elisa Mancuso,&nbsp;Shadan Lalezari,&nbsp;Wolfgang Miesbach,&nbsp;Giovanni Di Minno,&nbsp;Robert Klamroth,&nbsp;Cedric Hermans","doi":"10.1177/20406207231166779","DOIUrl":"https://doi.org/10.1177/20406207231166779","url":null,"abstract":"<p><strong>Background: </strong>Advances in treatment have enabled patients with haemophilia A to live longer and therefore may be subjected to comorbidities associated with ageing, in addition to disease-associated morbidities. There have been few reports to date on efficacy and safety of treatment specifically in patients with severe haemophilia A and comorbidities.</p><p><strong>Objective: </strong>To explore the efficacy and safety of damoctocog alfa pegol prophylaxis in patients with severe haemophilia A aged ⩾40 years with comorbidities of interest.</p><p><strong>Design: </strong>A <i>post hoc</i> analysis of data from the phase 2/3 PROTECT VIII study and its extension.</p><p><strong>Methods: </strong>Bleeding and safety outcomes were analysed in a subgroup of patients aged ⩾40 years with ⩾1 comorbidity receiving damoctocog alfa pegol (BAY 94-9027; Jivi^®) prophylaxis.</p><p><strong>Results: </strong>Thirty-four patients with severe haemophilia A were included in this analysis, with a mean age of 49.4 years at time of enrolment. The most prevalent comorbidities were hepatitis C (<i>n</i> = 33; chronic, <i>n</i> = 23), hepatitis B (<i>n</i> = 8) and hypertension (<i>n</i> = 11). Four patients had human immunodeficiency virus. All received damoctocog alfa pegol prophylaxis for the entire study [median (range) time in study = 3.9 (1.0-6.9) years]. During the main study and extension, median total annualised bleeding rates (ABRs) (Q1; Q3) were 2.1 (0.0; 5.8) and 2.2 (0.6; 6.0), respectively; median joint ABRs were 1.9 (0.0; 4.4) and 1.6 (0.0; 4.0), respectively. Mean adherence with prophylaxis schedule was greater than 95% throughout the study. No deaths or thrombotic events were reported.</p><p><strong>Conclusion: </strong>Efficacy, safety and adherence of damoctocog alfa pegol were confirmed in patients aged ⩾40 years with haemophilia A and one or more comorbidities, with data for up to 7 years supporting its use as a long-term treatment option in this group.</p><p><strong>Plain language summary: </strong>Advances in treatment mean that people with haemophilia A are now living longer and, as a result, may have additional medical conditions that occur with ageing. We aimed to investigate the efficacy and safety of the long-acting replacement factor VIII damoctocog alfa pegol in people with severe haemophilia A who had additional medical conditions. To do this, we investigated the recorded information about patients aged 40 years of age or older who had been treated with damoctocog alfa pegol in a previously completed clinical trial. We found that the treatment was well-tolerated; no deaths or thrombotic events (undesirable clotting events) were reported. Treatment was efficacious in reducing bleeding in this group of patients. The findings support the use of damoctocog alfa pegol as a long-term treatment for older patients with haemophilia A and coexisting conditions.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/50/cd/10.1177_20406207231166779.PMC10126693.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9363934","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of venetoclax-based regimens for the treatment of relapsed/refractory multiple myeloma: a systemic review and meta-analysis.","authors":"Linchun Xu,&nbsp;Shaoze Lin,&nbsp;Xueyang Xing,&nbsp;Yongzhong Su","doi":"10.1177/20406207231155028","DOIUrl":"https://doi.org/10.1177/20406207231155028","url":null,"abstract":"<p><strong>Background: </strong>Patients with relapsed/refractory multiple myeloma (RRMM) usually have dismal prognostic outcomes. Venetoclax, a selective inhibitor of antiapoptotic protein B-cell lymphoma-2 (BCL-2), demonstrates antimyeloma activity in plasma cells with t(11;14) or high BCL-2 expression.</p><p><strong>Objectives: </strong>This meta-analysis aimed to investigate the efficacy and safety of venetoclax-based therapy in RRMM.</p><p><strong>Design: </strong>This is a meta-analysis study.</p><p><strong>Data sources and methods: </strong>PubMed, Embase, and Cochrane were searched for studies published up to 20 December 2021. Overall response rate (ORR), rate of very good partial response or better (≧VGPR), and complete response (CR) rate were pooled with the random-effects model. Safety was evaluated by the incidences of grade ≧3 adverse events. Subgroup analysis and meta-regression were performed to identify the causes of heterogeneities. All the analyses were conducted by STATA 15.0 software.</p><p><strong>Results: </strong>A total of 14 studies with 713 patients were included for analysis. The pooled ORR, rate of ≧VGPR, and CR for all patients were 59% [95% confidence interval (CI) = 45-71%], 38% (95% CI = 26-51%), and 17% (95% CI = 10-26%), respectively. The median progression-free survival (PFS) ranged from 2.0 months to not reached (NR), and the median overall survival (OS) ranged from 12.0 months to NR. Meta-regression showed that patients treated with more drugs combined or less heavily pretreated had higher response rates. Patients with t(11;14) had superior ORR [relative risk (RR) = 1.47, 95% CI = 1.05-2.07], ≧VGPR (RR = 1.71, 95% CI = 1.12-2.60), CR (RR = 1.86, 95% CI = 1.34-2.57), PFS [hazard ratio (HR) = 0.47, 95% CI = 0.30-0.65], and OS (HR = 0.30, 95% CI = 0.08-0.52) compared with patients without t(11;14). Most grade ≧3 adverse events were hematologic, gastrointestinal, and infectious related and were manageable.</p><p><strong>Conclusion: </strong>Venetoclax-based therapy is an effective and safe option for RRMM patients, especially those with t(11;14).</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/13/8f/10.1177_20406207231155028.PMC9989383.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9438227","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Avatrombopag treatment response in patients with immune thrombocytopenia: the REAL-AVA 1.0 study.","authors":"Abiola Oladapo,&nbsp;Scott Kolodny,&nbsp;Michael Vredenburg,&nbsp;Elyse Swallow,&nbsp;Debbie Goldschmidt,&nbsp;Kirthana Sarathy,&nbsp;Priscilla Lopez,&nbsp;Hillary Maitland,&nbsp;John Yee","doi":"10.1177/20406207231179856","DOIUrl":"https://doi.org/10.1177/20406207231179856","url":null,"abstract":"<p><strong>Background: </strong>Thrombopoietin-receptor agonists (TPO-RAs) are used to treat immune thrombocytopenia (ITP), a disorder characterized by prolonged low platelet counts (PCs) that pose a risk of serious bleeding episodes. Avatrombopag (AVA) is the most recently approved TPO-RA for the treatment of chronic ITP. A high proportion of patients responded to AVA in clinical trials, and treatment was well-tolerated; however, limited real-world effectiveness data have been reported to date.</p><p><strong>Objectives: </strong>To describe demographic and clinical characteristics, treatment patterns, and outcomes following the initiation of AVA in patients with ITP in the United States.</p><p><strong>Design: </strong>This is a retrospective study using administrative claims data from the Komodo Healthcare Map (1 February 2017 to 28 February 2022) linked with PC laboratory data.</p><p><strong>Methods: </strong>Patients with ⩾1 diagnosis of ITP, ⩾1 paid prescription for AVA (index date), and ⩾1 month of pharmacy coverage after AVA initiation were selected. Baseline characteristics and follow-up steroid, immunosuppressant, and rescue medication use were described. The percentage of patients achieving clinically meaningful PC thresholds (⩾30 × 10⁹/l) were assessed among patients with ⩾1 PC following AVA initiation and prior to AVA discontinuation/switch (effectiveness subgroup).</p><p><strong>Results: </strong>A total of 205 patients met eligibility criteria and 49% reported TPO-RA use in the prior 6 months. Approximately 70% and 93% of patients did not require use of steroid or immunoglobulin rescue medication during follow-up, respectively. Among patients with concomitant steroid (<i>n</i> = 75) or immunosuppressant (<i>n</i> = 7) use at AVA initiation, 35% and 57% discontinued those treatments, respectively. Of the 21 patients in the effectiveness subgroup, 81% achieved clinically meaningful PC thresholds.</p><p><strong>Conclusion: </strong>A high proportion of evaluable patients with ITP in this real-world study achieved clinically meaningful PCs, without requiring rescue medication during AVA treatment, with many able to discontinue baseline concomitant steroid or immunosuppressant utilization. Despite limited availability of PC data, these results are consistent with results from the AVA pivotal clinical trials.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/03/09/10.1177_20406207231179856.PMC10350755.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10648384","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study.","authors":"Carmen Escuriola Ettingshausen,&nbsp;Cedric Hermans,&nbsp;Pål A Holme,&nbsp;Ana R Cid,&nbsp;Kate Khair,&nbsp;Johannes Oldenburg,&nbsp;Claude Négrier,&nbsp;Jaco Botha,&nbsp;Aurelia Lelli,&nbsp;Jerzy Windyga","doi":"10.1177/20406207231184323","DOIUrl":"https://doi.org/10.1177/20406207231184323","url":null,"abstract":"<p><strong>Background: </strong>The bypassing agent, activated prothrombin complex concentrate [aPCC, FEIBA (factor VIII inhibitor bypass activity); Baxalta US Inc, a Takeda company, Lexington, MA, USA], is indicated for the treatment of bleeding episodes, perioperative management, and routine prophylaxis in patients with hemophilia A or B with inhibitors. In certain countries, aPCC is also indicated for the treatment of bleeding episodes and perioperative management in patients with acquired hemophilia A.</p><p><strong>Objectives: </strong>To describe long-term, real-world effectiveness, safety, and quality-of-life outcomes for patients with congenital hemophilia A or B and high-responding inhibitors receiving aPCC treatment in routine clinical practice.</p><p><strong>Design: </strong>FEIBA Global Outcome (FEIBA GO; EUPAS6691) was a prospective, observational study.</p><p><strong>Methods: </strong>Investigators determined the treatment regimen and clinical monitoring frequency. The planned patient observation period was 4 years. Data are from the safety analysis set (patients who received ⩾1 aPCC infusion).</p><p><strong>Results: </strong>Overall, 50 patients received either aPCC prophylaxis (<i>n</i> = 37) or on-demand therapy (<i>n</i> = 13) at screening [hemophilia A, <i>n</i> = 49; hemophilia B, <i>n</i> = 1; median (range) age, 16.5 [2-71] years). Mean ± standard deviation overall annualized bleeding rate and annualized joint bleeding rate for patients receiving prophylaxis were 6.82 ± 11.52 and 3.77 ± 5.71, respectively, and for patients receiving on-demand therapy were 10.94 ± 11.27 and 6.94 ± 7.39, respectively. Overall, 177 and 31 adverse events (AEs) were reported in 28 of 40 and 10 of 13 patients receiving prophylaxis or on-demand therapy, respectively. Two serious AEs were considered possibly related to aPCC: acute myocardial infarction due to coronary artery embolism in one patient receiving prophylaxis. No thrombotic microangiopathy was reported. No AEs resulted in death.</p><p><strong>Conclusion: </strong>This study demonstrated the long-term, real-world effectiveness and consistent safety profile of aPCC as on-demand therapy and prophylactic treatment in patients with hemophilia and high-responding inhibitors.</p><p><strong>Trial registry: </strong>FEIBA Global Outcome Study; EUPAS6691 https://www.encepp.eu/encepp/viewResource.htm?id=32774.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/83/ca/10.1177_20406207231184323.PMC10387704.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10301373","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}