Therapeutic Advances in Hematology最新文献

{"title":"Adding venetoclax or hypomethylating agents to induction chemotherapy as first-line treatment for adults with acute myeloid leukemia: a retrospective case-cohort study","authors":"Fangfei Xu, Kuangguo Zhou, Duanhao Gong, Wei Huang","doi":"10.1177/20406207241275850","DOIUrl":"https://doi.org/10.1177/20406207241275850","url":null,"abstract":"Background:The response rate of traditional first-line induction chemotherapy (IC) for newly diagnosed acute myeloid leukemia needs to be improved, but it is not clear whether adding venetoclax or hypomethylating agents (HMAs) to IC will improve the response rate.Objective:To determine whether venetoclax or HMAs could increase the response rate of IC in patients with newly diagnosed acute myeloid leukemia (AML).Design:A retrospective, propensity score matching analysis.Methods:Newly diagnosed AML patients at Tongji Hospital between 2021 and 2023 were included in this study. By matching cases and controls based on age, gender, baseline bone marrow blast cell proportion, type of AML, and the National Comprehensive Cancer Network (NCCN) risk stratification group, we compared the response rate (CR, CR/CRi, ORR, and MRD negative) and hematological adverse events in newly diagnosed AML treated with IC plus venetoclax or HMAs versus IC alone after one cycle of IC.Results:The addition of venetoclax could improve CR/CRi of IC (83.8% for IC plus venetoclax vs 66.1% for IC alone, p = 0.029). The addition of venetoclax to IA regimen did not improve CR/CRi of IA regimen (76.9% for IA plus venetoclax vs 76.2% for IA alone, p = 0.986). The addition of HMAs could not only improves CR/CRi of IC (85.3%% for IC plus HMAs vs 65.4% for IC alone, p = 0.002) but also improves CR/CRi of IA regimen (91.3% for IA plus HMAs vs 70.0% for IA alone, p = 0.034). The addition of HMAs could improve CR/CRi of patients with adverse mutations (FLT3, IDH1/2, K/NRAS) after IC. The addition of venetoclax and HMAs both extended the duration of agranulocytosis and thrombocytopenia.Conclusion:Adding HMAs might improve CR/CRi of IC including IA. Adding venetoclax might not improve CR/CRi of IA. A well-designed prospective randomized controlled study is now warranted.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Is less more? Intravenous immunoglobulin for pediatric immune thrombocytopenia","authors":"Eyal Elron, Joanne Yacobovich, Orly Efros, Osama Tanous, Sarina Levy-Mendelovich, Esti Shamba, Orna Steinberg-Shemer, Tracie Goldberg, Shai Izraeli, Oded Gilad","doi":"10.1177/20406207241279202","DOIUrl":"https://doi.org/10.1177/20406207241279202","url":null,"abstract":"Objectives:Treatment of pediatric immune thrombocytopenia (ITP) is guided by the risk of bleeding. Intravenous immunoglobulin (IVIg) is one of the first-line therapy options for new-onset pediatric ITP. However, the exact optimal dose of IVIg has not been determined.Methods:This retrospective cohort study included all hospitalized children with newly diagnosed ITP receiving IVIg as first-line therapy during 2010–2020. We compared the safety and efficacy of two common IVIg dose regimens, 1 and 2 g/kg. Outcomes were short and long-term treatment responses and adverse events to the different doses.Results:A total of 168 children were included in our cohort. Eighty-two children were treated with 1 g/kg of IVIg and 86 with 2 g/kg. There was no difference in sustained response (platelet count &gt; 20 × 10<jats:sup>9</jats:sup>, &gt; 14 days) between the groups (74.3% vs 76.7%, respectively, p = 0.72) and maximal platelet counts following treatment ( p = 0.44). No difference was found regarding the percentage of chronic ITP between the two groups (24.4% in the 1 g/kg group as compared to 17.4% in the 2 g/kg group; p = 0.34). Logistic regression analysis demonstrated there was no effect of the IVIg dose on treatment failure and development of chronic ITP. As anticipated, 47.7% of adverse events were in the 2 g/kg group and 32.9% in the 1 g/kg group, with borderline statistical significance ( p = 0.06).Conclusion:The initial treatment of newly diagnosed pediatric ITP using a 1 g/kg IVIg regimen may give comparable results to the double dose of 2 g/kg in attaining a prolonged safe hemostatic threshold, without impacting the incidence of chronic disease.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251402","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Systematic literature review of the indirect costs and humanistic burden of β-thalassemia","authors":"Yesim Aydinok, Sneha Purushotham, Aylin Yucel, Mrudula Glassberg, Sohan Deshpande, Barbara Potrata, Myrto Trapali, Farrukh Shah","doi":"10.1177/20406207241270872","DOIUrl":"https://doi.org/10.1177/20406207241270872","url":null,"abstract":"Background:β-Thalassemia is an inherited blood disorder requiring lifetime management of anemia and its complications.Objective:This study aimed to determine the indirect costs and humanistic burden of β-thalassemia.Design:A systematic literature review was conducted.Data sources and methods:Searches were conducted in Embase, MEDLINE, MEDLINE In-Process, and EconLit (November 1, 2010, to November 25, 2020). Studies reporting indirect costs and health-related quality of life (HRQoL) for patients with β-thalassemia were eligible.Results:Seventy-five publications were included. Mean annual days lost due to transfusion-related absenteeism ranged from 15.6 to 35 days. Patients spent a mean of 592 min (standard deviation (SD): 349) daily on disease management on transfusion days and 91 min (SD: 221) daily on non-transfusion days. Patients with non-transfusion-dependent β-thalassemia (NTDT) showed worse HRQoL versus those with transfusion-dependent β-thalassemia (TDT) on the 36-item Short Form Health Survey (75.8 vs 66.5; p = 0.021). Caregivers of patients with TDT had more severe stress compared with patients (20.17 vs 18.95; p = 0.006), as measured by the standardized Cohen Perceived Stress Questionnaire.Conclusion:TDT is associated with substantial indirect costs and caregiver burden, and NTDT is associated with worse HRQoL. There is an unmet need for novel treatments in both TDT and NTDT that minimize patient and caregiver burden.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251403","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phase I/II study of the clinical activity and safety of GSK3326595 in patients with myeloid neoplasms","authors":"Justin Watts, Mark D. Minden, Kimo Bachiashvili, Andrew M. Brunner, Sameem Abedin, Timothy Crossman, Magdalena Zajac, Veronica Moroz, Jacqueline L. Egger, Aarti Tarkar, Brandon E. Kremer, Olena Barbash, Gautam Borthakur","doi":"10.1177/20406207241275376","DOIUrl":"https://doi.org/10.1177/20406207241275376","url":null,"abstract":"Background:GSK3326595 is a potent, selective, reversible protein arginine methyltransferase 5 (PRMT5) inhibitor under investigation for treatment of myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML). In preclinical models of AML, PRMT5 inhibition decreased proliferation and increased cell death, supporting additional clinical research in myeloid neoplasms.Objectives:To determine the clinical activity, safety, tolerability, dosing, additional measures of clinical activity, pharmacokinetics, and pharmacodynamics of GSK3326595.Design:In part 1 of this open-label, multicenter, multipart, phase I/II study, adults with relapsed/refractory myeloid neoplasms (e.g., MDS, CMML, and AML) received monotherapy with 400 or 300 mg oral GSK3326595 once daily. Study termination occurred prior to part 2 enrollment.Methods:Clinical activity was determined by the clinical benefit rate (CBR; proportion of patients achieving complete remission (CR), complete marrow remission (mCR), partial remission, stable disease (SD) &gt;8 weeks, or hematologic improvement). Adverse events (AEs) were assessed by incidence and severity. Exploratory examination of spliceosome mutations was performed to determine the relationship between genomic profiles and clinical response to GSK3326595.Results:Thirty patients with a median age of 73.5 years (range, 47–90) were enrolled; 13 (43%) and 17 (57%) received 400 and 300 mg of GSK3326595, respectively. Five (17%) patients met CBR criteria: 4 (13%) with SD &gt;8 weeks and 1 (3%) achieving mCR. Of five patients with clinical benefit: three had SRSF2 mutation, one U2AF1, and one was splicing factor wild-type. Frequent GSK3326595-related AEs were decreased platelet count (27%), dysgeusia (23%), fatigue (20%), and nausea (20%). GSK3326595 had rapid absorption, with a T<jats:sub>max</jats:sub> of approximately 2 h and a terminal half-life of 4–6 h.Conclusion:GSK3326595 monotherapy had limited clinical activity in heavily pretreated patients despite robust target engagement. The safety profile was broadly consistent with other published PRMT5 inhibitor studies.Trial registration:ClinicalTrials.gov: NCT03614728.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251404","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Targeting B-cell maturation antigen for treatment and monitoring of relapsed/refractory multiple myeloma patients: a comprehensive review","authors":"David Yashar, Bernard Regidor, Marissa-Skye Goldwater, Sean Bujarski, Ashley Del Dosso, James R. Berenson","doi":"10.1177/20406207241275797","DOIUrl":"https://doi.org/10.1177/20406207241275797","url":null,"abstract":"Despite major therapeutic advancements in recent years, multiple myeloma (MM) remains an incurable disease with nearly all patients experiencing relapsed and refractory disease over the course of treatment. Extending the duration and durability of clinical responses will necessitate the development of therapeutics with novel targets that are capable of robustly and specifically eliminating myeloma cells. B-cell maturation antigen (BCMA) is a membrane-bound protein expressed predominantly on malignant plasma cells and has recently been the target of several novel therapeutics to treat MM patients. This review will focus on recently approved and currently in development agents that target this protein, including bispecific antibodies, antibody-drug conjugates, and chimeric antigen receptor T-cell therapies. In addition, this protein also serves as a novel serum biomarker to predict outcomes and monitor disease status for MM patients; the studies demonstrating this use of BCMA will be discussed in detail.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142251405","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vedolizumab for second-line treatment of steroid-refractory gastrointestinal late acute graft-versus-host disease.","authors":"Yingling Zu, Ruirui Gui, Zhen Li, Juan Wang, Pei Li, Ying Liu, Xiaofeng Dong, Jian Zhou","doi":"10.1177/20406207241276982","DOIUrl":"https://doi.org/10.1177/20406207241276982","url":null,"abstract":"<p><strong>Background: </strong>Late acute graft-versus-host disease (aGVHD) is a complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with little data regarding treatment and outcomes. There is no standard treatment for gastrointestinal (GI) late aGVHD, especially for steroid-refractory (SR) GI late aGVHD. Vedolizumab, a monoclonal antibody inhibiting the migration of both naive and activated lymphocytes into the GI endothelium, has been verified to be effective for SR GI aGVHD.</p><p><strong>Methods: </strong>We retrospectively analyzed the clinical efficacy and safety of vedolizumab as the second line for SR GI late aGVHD in seven patients after allo-HSCT.</p><p><strong>Results: </strong>Four patients received two doses of vedolizumab infusion, while three patients received only one dose of vedolizumab infusion. The complete response and partial response rates were 57.1% (4/7) and 42.9% (3/7), respectively. No patient progressed to chronic GVHD during the period of follow-up. There was no severe adverse event related to vedolizumab.</p><p><strong>Conclusion: </strong>Our data suggest that vedolizumab is expected to ameliorate SR GI late aGVHD. Further data on the treatment timing, efficacy, and safety of vedolizumab are warranted in prospective clinical trials.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11380122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154997","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 use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS.","authors":"Wolfgang Miesbach, Nicola Curry, Paul Knöbl, Charles Percy, Rita Santoro, Alvin H Schmaier, Karolin Trautmann-Grill, Kayode Badejo, Jie Chen, Masoud Nouri, Pooja Oberai, Robert Klamroth","doi":"10.1177/20406207241260332","DOIUrl":"10.1177/20406207241260332","url":null,"abstract":"<p><strong>Background: </strong>Recombinant porcine factor VIII (rpFVIII; susoctocog alfa) is indicated for the treatment of bleeding events (BEs) in adults with acquired hemophilia A (AHA).</p><p><strong>Objectives: </strong>To assess the safety, utilization, and effectiveness of rpFVIII in clinical practice.</p><p><strong>Design: </strong>EU post-authorization safety study (PASS) (NCT03199794) was a multicenter, noninterventional, post-authorization safety study conducted in adults with AHA.</p><p><strong>Methods: </strong>Data were collected retrospectively or prospectively for up to 180 days after the last rpFVIII dose. The primary objective was safety, as assessed by adverse events (AEs), serious AEs (SAEs), and AEs of special interest (AESIs) (e.g. immunogenicity, hypersensitivity reactions, thromboembolic events). Secondary endpoints included immunogenicity, rpFVIII hemostatic effectiveness, and rpFVIII utilization.</p><p><strong>Results: </strong>Fifty patients were enrolled; 31 completed the study. The median (range) follow-up for patients who completed or discontinued the study was 178 (26-371) days. The median (range) first dose of rpFVIII was 54.0 (11-200) U/kg. Thirty patients reported 46 SAEs; 5 SAEs were considered probably related to rpFVIII, of which 1 was lack of rpFVIII efficacy, and 4 were AESIs: drug resistance due to FVIII inhibition (one patient), antibody test positive for anti-pFVIII inhibitors (one patient), and <i>de novo</i> anti-pFVIII inhibitors (two patients). No hypersensitivity reactions or thromboembolic events were reported. Of the 50 initial BEs, 37 resolved [in a median (interquartile range) of 8.0 (4.0-16.0) days].</p><p><strong>Conclusion: </strong>Results from this real-world study support the use of rpFVIII for AHA, aligning with findings from the clinical trial of rpFVIII (NCT01178294) in the treatment of BEs in adults with AHA.</p><p><strong>Trial registration: </strong>EUPAS16055; NCT03199794.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11369870/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142126734","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":"Comparable efficacy and safety of generic and branded imatinib for patients with chronic myeloid leukemia in China.","authors":"Fang Cheng, Di Wu, Zheng Cui, Qiang Li, Weiming Li, Yu Zhang","doi":"10.1177/20406207241270806","DOIUrl":"10.1177/20406207241270806","url":null,"abstract":"<p><strong>Background: </strong>Generics imatinib became an alternative treatment option for chronic myeloid leukemia (CML) patients in China. However, clinicians and patients alike harbor concerns regarding the long-term safety of generic imatinib.</p><p><strong>Objectives: </strong>Patients with chronic phase CML receiving frontline imatinib treatment.</p><p><strong>Design: </strong>A retrospective study was used to evaluate the blood concentration, effectiveness, and safety of generic in 170 CML patients.</p><p><strong>Methods: </strong>Imatinib plasma concentrations were detected by high-performance liquid chromatography-tandem mass spectrometry.</p><p><strong>Results: </strong>Among the 170 patients, 73 (42.9%) patients treated with branded imatinib as first-line therapy, while 22 (12.9%) switched to generic imatinib during treatment due to economic considerations. No significant differences in trough concentrations between branded and generic imatinib (1549.9 ± 648.8 ng/mL vs 1479.0 ± 507.0 ng/mL; <i>p</i> = 0.95). During the 2-year follow-up, there were no significant differences in molecular response rates (major molecular response (MMR): 33.3% vs 37.0%; deep molecular response: 56.9% vs 42.9%, <i>p</i> = 0.17) between the branded and generic imatinib. Both groups showed similar rates of switching to second-generation tyrosine kinase inhibitor (11.8% vs 15.1%, <i>p</i> = 0.56). Furthermore, there were no significant differences in event-free survival or failure-free survival between branded and generic imatinib. Twenty-two (12.9%) switched to generic imatinib during treatment, 68.2% maintained their level of response, 27.3% improved, and only one patient (4.5%) lost MMR. There were no significant differences in the incidence of various adverse events.</p><p><strong>Conclusion: </strong>Generic imatinib are equally effective and safe compared to branded molecules, both for newly diagnosed patients and those who switch from branded.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11325334/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141988974","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":"Successful treatment of hemophagocytic intravascular large B-cell lymphoma with CNS involvement with BTK inhibitor combined with rituximab and high-dose methotrexate.","authors":"Fangfei Shao, Wei Su, Xiujie Zhao, Jianping He, Xiaofen Wang, Feng Guo, Haowen Xiao","doi":"10.1177/20406207241270788","DOIUrl":"10.1177/20406207241270788","url":null,"abstract":"<p><p>This is a case of hemophagocytic intravascular large B-cell lymphoma (IVLBCL) with central nervous system (CNS) involvement. Although R-CHOP chemotherapy regimen has been shown significant improvement in survival rate. The prognosis and outcomes remain unsatisfactory, which is identified as outstanding challenges and need solutions. Gene and molecular profiling studies may provide new therapeutic strategies, especially the BCR/TLR/IL-1R/NF-κB signaling pathway in IVLBCL. Here, we treated the hemophagocytic IVLBCL CNS-involved patient with the Bruton tyrosine kinase inhibitor (BTKi) to block NF-κB pathway, and indicated that the second-generation BTKi zanubrutinib-based treatment was feasible and efficient.</p>","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-08-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11325336/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141988975","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":"Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes","authors":"Priyanka Raheja, Nana Kragh, Linda Bystrická, Daniel Eriksson, Khaoula Aroui, Marwa Mezghani, Sylvaine Barbier, Silvia Linari","doi":"10.1177/20406207241257917","DOIUrl":"https://doi.org/10.1177/20406207241257917","url":null,"abstract":"Background:Hemophilia-associated bleeding and resultant joint pain and mobility restrictions can predispose patients to poor health-related quality of life (HRQoL). Therefore, efficacy of a treatment needs to address more than just annualized bleed rates.Objectives:Describe the evolution of HRQoL, pain, and activity in patients with hemophilia A, treated with efmoroctocog alfa prophylaxis.Design:A post hoc analysis from Kids A-LONG (NCT01458106), A-LONG (NCT01181128), and long-term extension study ASPIRE (NCT01454739) assessed change in pain and activity-related patient-reported outcomes (PROs).Methods:Physical health, pain, and HRQoL were assessed by PROs for a cumulative treatment duration of up to ~6 years. The primary endpoint was change from baseline in EuroQoL (EQ)-5D and Haemophilia Quality of Life Questionnaire (Haem-A-QoL).Results:118 adult/adolescents and 71 pediatric patients were included. The proportion of adults and adolescents reporting no problem in the EQ-5D analysis of ‘ pain/discomfort’ significantly increased from A-LONG baseline (35.04%; 41/117) to ASPIRE month 30 (44.68%; 21/47; p = 0.024). Mean (standard deviation) Haem-A-QoL subdomain scores for ‘ feeling’ and ‘ physical health’ at A-LONG baseline improved by −3.24 (15.13; p = 0.018) and −3.85 (23.07; p = 0.047), respectively, at study end. Proportion of pediatric patients reporting no problem on the EQ-5D analysis of ‘ pain/discomfort’, significantly increased from A-LONG baseline (75.0%; 42/56) to ASPIRE baseline (95.56%; 43/45; p = 0.046). Satisfaction levels for pediatric patients were high at A-LONG baseline and maintained until study end.Conclusion:Long-term efmoroctocog alfa prophylaxis reduces pain and improves HRQoL in adult and adolescent patients with hemophilia A. In pediatric patients, it reduces perceived pain and maintains satisfaction levels.Trial registration:NCT01458106, NCT01181128, NCT01454739.","PeriodicalId":23048,"journal":{"name":"Therapeutic Advances in Hematology","volume":null,"pages":null},"PeriodicalIF":3.4,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141866676","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}