{"title":"Proportion and characteristics of bone marrow dysplasia in advanced HIV infection.","authors":"Dimmy Prasetya, Chrisan Bimo Prayuda, Syifa Nur Maulida, Khairiyah Darojat, Rudi Wisaksana, Basti Andriyoko","doi":"10.1080/16078454.2025.2609373","DOIUrl":"10.1080/16078454.2025.2609373","url":null,"abstract":"<p><strong>Purpose: </strong>This study aims to determine the proportion of bone marrow dysplasia in advance of HIV infection.</p><p><strong>Patients and methods: </strong>A cross-sectional study was conducted on advanced HIV patients, stage III and IV, with cytopenia at Hasan Sadikin General Hospital, Bandung, from January 2021 to November 2023. Patient characteristics and laboratory data were obtained from patients diagnosed with HIV stages III and IV who presented with cytopenia and underwent bone marrow examinations, as documented in the medical records. The differences between the two groups were analyzed using the Mann-Whitney <i>U</i> test or the Independent T-test for numerical data and Chi-square or Fisher's exact for categorical data.</p><p><strong>Results: </strong>A total of 42 subjects were enrolled, consisting of 8 (19%) HIV stage III and 34 (81%) stage IV patients. Patients with HIV stage IV exhibited a higher incidence of pancytopenia, with 15 cases (44.1%). The bone marrow cellularity in HIV stage IV was predominantly hypocellular, accounting for 22 cases (64.7%). Dyserythropoiesis was the most common type of bone marrow dysplasia observed in HIV stage IV, present in 19 cases (55.9%). The prevalence of Bone Marrow Dysplasia in HIV stage IV was higher than in HIV stage III, with 23 (64.7%) and 2 (37.5%), respectively (<i>p</i> <i>=</i> 0.2348).</p><p><strong>Conclusion: </strong>Cytopenias related to HIV are common and tend to worsen as the disease advances. This emphasizes the importance of investigating the potential for bone marrow dysplasia and conducting cytogenetic diagnostic testing for myelodysplastic syndromes (MDS), as a significant non-AIDS-defining hematological malignancy in individuals with advanced HIV.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2609373"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146105329","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":"Secondary poor graft function after autologous stem cell transplantation in multiple myeloma: a case-based expert review and successful rescue with secondary autologous stem cell infusion.","authors":"Jiawen You, Yuanyuan Zhang, Yuan Meng, Xiaojun Liu, Xiaoning Song, Jinqiao Zhang, Meirong Zang, Jing Suo, Jiapei Lu, Yuxin Dai, Jianbo Meng","doi":"10.1080/16078454.2026.2633462","DOIUrl":"10.1080/16078454.2026.2633462","url":null,"abstract":"<p><strong>Introduction: </strong>Secondary poor graft function (PGF) after autologous hematopoietic stem cell transplantation (Auto-HSCT) for multiple myeloma (MM) is rare, often delayed in recognition, and lacks standardized salvage algorithms.</p><p><strong>Areas covered: </strong>Using a case-based expert-review format, we summarize diagnostic hallmarks, exclusion work-up, mechanistic drivers, and practical management, with emphasis on autologous stem-cell boost as a rescue option.</p><p><strong>Case summary: </strong>A 59-year-old woman with IgG κ MM achieved timely neutrophil and platelet engraftment after Auto-HSCT, then developed recurrent transfusion-dependent pancytopenia approximately two months later. Relapse, occult infection/viral reactivation, immune cytopenia, nutritional deficiency, and drug-related myelosuppression were systematically excluded, supporting secondary PGF. Growth factors and thrombopoietin-receptor agonists produced only transient benefit. A second infusion of cryopreserved autologous peripheral blood stem cells (PBSCs) (3.621 × 10<sup>6</sup> CD34<sup>+</sup>/kg) without re-conditioning led to rapid platelet recovery within 7 days and durable trilineage hematopoiesis.</p><p><strong>Expert opinion: </strong>Secondary PGF after Auto-HSCT appears multifactorial, involving quantitatively adequate but qualitatively fragile grafts, inflammatory/microenvironmental injury, and therapy-related megakaryocytic vulnerability (including heavy lenalidomide exposure). When backup cells are available and reversible causes are excluded, early unpreconditioned autologous PBSC boost is a safe, feasible, and likely under-utilized salvage strategy. Strategic PBSC banking and early recognition may prevent life-threatening PGF.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2633462"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147270765","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}
HematologyPub Date : 2026-12-01Epub Date: 2026-02-01DOI: 10.1080/16078454.2026.2620148
Omar Ahmed Alshaikhi, Elaf Yahia Faraj Alnasser
{"title":"GNE-related thrombocytopenia (Thrombocytopenia-12) in a 3-month-old from a Middle Eastern background infant: a case report.","authors":"Omar Ahmed Alshaikhi, Elaf Yahia Faraj Alnasser","doi":"10.1080/16078454.2026.2620148","DOIUrl":"10.1080/16078454.2026.2620148","url":null,"abstract":"<p><strong>Background: </strong>Congenital thrombocytopenia represents a diagnostically challenging group of disorders due to overlapping clinical presentations among various etiologies.</p><p><strong>Case presentation: </strong>A 3-month-old infant presented with severe thrombocytopenia (platelet count 6,000/μL), neutropenia, and bone marrow findings of megakaryocytic hypoplasia, initially suggestive of congenital amegakaryocytic thrombocytopenia (CAMT). Comprehensive genetic testing identified a homozygous pathogenic variant in the GNE gene (NM_001128227.2:c.1768G > A, p.Gly590Arg), establishing the definitive diagnosis of GNE-related thrombocytopenia (Thrombocytopenia-12, THC12).</p><p><strong>Clinical significance: </strong>This case highlights three critical aspects: first, the essential role of genetic testing in differentiating congenital thrombocytopenias; second, the distinct management implications of THC12 compared to CAMT; and third, the need for long-term monitoring given the potential for late-onset myopathy despite initial isolated hematologic manifestations.</p><p><strong>Management: </strong>Therapeutic interventions included intravenous immunoglobulin, platelet transfusions, and thrombopoietin receptor agonists, with concurrent evaluation for potential hematopoietic stem cell transplantation.</p><p><strong>Conclusion: </strong>This report underscores THC12 as an important diagnostic consideration in infants with congenital thrombocytopenia and emphasizes the necessity of genetic confirmation to guide appropriate clinical management and family counseling.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2620148"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146099903","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":"Whole transcriptome sequencing reveals differences in the pathogenesis of myelodysplastic syndromes and acute myeloid leukemia.","authors":"Miao Zhou, Tongyu Li, Yun Li, Ying Chen, Wanchuan Zhuang, Guifang Ouyang","doi":"10.1080/16078454.2026.2638615","DOIUrl":"10.1080/16078454.2026.2638615","url":null,"abstract":"<p><strong>Objectives: </strong>Myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) are closely related hematologic malignancies. However, the transcriptomic distinctions between them are not fully elucidated. This study aimed to identify transcriptional differences between MDS and AML using whole transcriptome sequencing to provide a theoretical basis for improved diagnosis and treatment.</p><p><strong>Methods: </strong>Whole transcriptome sequencing was conducted to profile the expression of mRNAs and non-coding RNAs in MDS and AML samples. Subsequent bioinformatic analyzes were performed to identify differentially expressed genes and their potential functional roles.</p><p><strong>Results: </strong>Comprehensive sequencing revealed 1926 differentially expressed mRNAs, 80 miRNAs, 1553 lncRNAs, and 1635 circRNAs between MDS and AML. Functional enrichment analysis indicated that these differentially expressed genes are predominantly involved in key pathways such as ferroptosis, immune response, and cell cycle regulation. Further investigation highlighted a significant association of differentially expressed lncRNAs and circRNAs with the ferroptosis pathway. Key molecules implicated in ferroptosis included AQP3, ALOX5, ADAM23, STK11, AIFM2, hsa-miR-4433b-3p, hsa-miR-1307-3p, hsa-miR-23a-5p, and hsa-miR-3916.</p><p><strong>Discussion: </strong>The distinct transcriptomic profiles uncovered in this study reveal critical molecular differences in the pathogenesis of MDS and AML. The strong enrichment of ferroptosis-related pathways, mediated by specific coding and non-coding RNAs, suggests a pivotal role for this biological process in disease development and progression from MDS to AML.</p><p><strong>Conclusion: </strong>Our study identified crucial genes and non-coding RNAs linked to ferroptosis, implicating their potential roles in MDS and AML pathogenesis and offering valuable candidate targets for future research.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2638615"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147325695","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}
HematologyPub Date : 2026-12-01Epub Date: 2026-03-02DOI: 10.1080/16078454.2026.2634435
Seraj Brugi, Eliane Young
{"title":"Measuring the neuropsychological consequences of thrombotic thrombocytopenic purpura: a rapid literature review.","authors":"Seraj Brugi, Eliane Young","doi":"10.1080/16078454.2026.2634435","DOIUrl":"10.1080/16078454.2026.2634435","url":null,"abstract":"<p><strong>Objectives: </strong>Thrombotic thrombocytopenic purpura (TTP) is a hematological disorder associated with potentially severe long-term health outcomes, including neuropsychological consequences. Guidelines recommend cognitive assessment for people with TTP, and an understanding of how the condition affects various cognitive domains can support decision-making for clinicians when selecting assessment measures.</p><p><strong>Methods: </strong>A rapid literature review was conducted using MEDLINE Ultimate, CINAHL Ultimate and PsycINFO to appraise the existing evidence base regarding the impact of TTP on cognition, the measures used to assess cognitive impairment, and the correlates of cognitive impairment in this population group.</p><p><strong>Results: </strong>Fourteen articles were eligible for inclusion and the results were summarized using a narrative synthesis. TTP is associated with impairment across all assessed cognitive domains. Cognitive impairment is associated with abnormal cerebral findings but not ADAMTS13 levels or severity and frequency of acute episodes. The sensitivity of brief screening measures in detecting TTP-related cognitive change is unclear.</p><p><strong>Conclusion: </strong>Comprehensive cognitive assessment following TTP, including an exploration of possible confounding factors such as poor mental health, is recommended. Caution when relying on brief screening measures is advised.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2634435"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147325754","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}
HematologyPub Date : 2026-12-01Epub Date: 2026-03-02DOI: 10.1080/16078454.2026.2631219
Jing Chen, Fanjun Cheng, Jun Fang
{"title":"Neutrophil extracellular trap-related genes in PTCL: identification, prognosis and drug interaction prediction via bioinformatics-machine learning.","authors":"Jing Chen, Fanjun Cheng, Jun Fang","doi":"10.1080/16078454.2026.2631219","DOIUrl":"10.1080/16078454.2026.2631219","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to identify neutrophil extracellular trap-related genes (NET-RGs), explore their prognostic significance, and predict drug interactions in peripheral T-cell lymphoma (PTCL).</p><p><strong>Methods: </strong>Differentially expressed NET-RGs (DE-NRGs) between PTCL and normal tissues were screened. Functional enrichment analysis was conducted. Bioinformatics and machine learning were used to identify hub genes and assess their diagnostic value. Univariate and multivariate analyses were used to evaluate prognostic roles. Correlation and immune infiltration analyses were performed to explore relationships with the tumor microenvironment (TME). Clinical data were collected from PTCL patients who received potential agents (lenalidomide) as first-line treatment.</p><p><strong>Results: </strong>A total of 31 DE-NRGs were identified (18 upregulated and 13 downregulated), enriched in inflammatory response, extracellular matrix organization, and infection involvement. Four hub genes (AKT2, MAPK14, IRF1, and TNF) were identified as effective PTCL diagnostic markers. Higher AKT2/MAPK14 expression correlated with poorer overall survival (OS), while elevated TNF expression associated with better OS; AKT2 and TNF independently predicted OS. These genes were implicated in modulating TME remodeling. Potential therapeutic agents (e.g. capivasertib, lenalidomide) were predicted, and lenalidomide may represent a feasible initial treatment option for PTCL, with an objective response rate (ORR) of 40.0% and a maximum survival duration exceeding 50 months.</p><p><strong>Conclusion: </strong>NET-RGs play crucial roles in diagnosis, prognosis, and TME regulation, and lenalidomide, a putative TNF-targeting agent, may represent a feasible initial treatment option in PTCL.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2631219"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147344134","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}
HematologyPub Date : 2026-12-01Epub Date: 2026-02-03DOI: 10.1080/16078454.2026.2619261
Yuxiu Xu, Xiaoli Sun, Jing Yang, Dongsheng Ma, Jiangwei Wan, Tingting Lei, Tao Wang
{"title":"Apoptosis-related genes influence prognosis and immune characteristics of diffuse large B-cell lymphoma.","authors":"Yuxiu Xu, Xiaoli Sun, Jing Yang, Dongsheng Ma, Jiangwei Wan, Tingting Lei, Tao Wang","doi":"10.1080/16078454.2026.2619261","DOIUrl":"10.1080/16078454.2026.2619261","url":null,"abstract":"<p><strong>Objectives: </strong>Dysregulation of apoptosis-related genes (ARGs) may contribute to tumorigenesis and impact patient prognosis, but their specific influence on prognosis and immune characteristics in diffuse large B-cell lymphoma (DLBCL) remains unclear.</p><p><strong>Methods: </strong>Gene expression profiles were collected from GEO datasets GSE10846 (training set; <i>n</i> = 414) and GSE181063 (validation set; <i>n</i> = 1310), totaling 1724 DLBCL samples. Univariate Cox and LASSO Cox regression analyses were performed to identified key ARGs, which were used to construct a risk score model. Patients were stratified into high/low-risk groups using the risk score. Functional enrichment analyses were conducted to explore biological functions and pathways. Immune cell infiltration was assessed using the CIBERSORT algorithm.</p><p><strong>Results: </strong>39 ARGs were significantly associated with overall survival in DLBCL patients. The risk score model effectively stratified patients into high/low-risk groups with distinct survival outcomes. Consensus clustering revealed distinct molecular subtypes with varying prognoses and biological characteristics. Enrichment analyses indicated that the prognostic genes are involved in critical pathways related to apoptosis and immune responses. High-risk patients exhibited higher immune scores and a distinct tumor immune microenvironment.</p><p><strong>Conclusion: </strong>Apoptosis-related genes are valuable prognostic biomarkers in DLBCL and are associated with distinct immune characteristics. The risk model may aid in personalized risk assessment and inform treatment strategies. These findings provide insights into potential therapeutic targets by modulating apoptosis and immune responses in DLBCL.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2619261"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146112951","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":"Adverse karyotype amplifies risk in secondary acute myeloid leukemia (AML) and AML with myelodysplasia-related changes.","authors":"Toshihiro Matsukawa, Shota Yoshida, Masahiro Onozawa, Fumiaki Fujii, Jun Nagai, Tomoki Takahashi, Shuichi Ota, Junichi Hashiguchi, Akio Mori, Takuto Miyagishima, Makoto Ibata, Yasutaka Kakinoki, Tatsuo Oyake, Satoshi Yamamoto, Toshiaki Hayashi, Kentaro Wakasa, Tomoyuki Saga, Tetsuya Igarashi, Satoshi Iyama, Yutaka Tsutsumi, Katsuya Fujimoto, Masaaki Adachi, Masahiro Yoshida, Daigo Hashimoto, Takanori Teshima","doi":"10.1080/16078454.2026.2628479","DOIUrl":"10.1080/16078454.2026.2628479","url":null,"abstract":"<p><strong>Background: </strong>Secondary acute myeloid leukemia (sAML) and AML with myelodysplasia-related changes (AML-MRC) are associated with poor prognosis, but the impact relative to de novo AML remains controversial. We investigated clinical and genetic features in a multicenter Japanese cohort before CPX-351 approval.</p><p><strong>Methods: </strong>We retrospectively analyzed 294 patients with newly diagnosed AML registered in the Hokkaido Leukemia Net between 2022 and 2023. Propensity score matching was used to adjust baseline variables. Genetic profiles were assessed in 160 matched patients.</p><p><strong>Results: </strong>In the matched cohort, sAML/AML-MRC did not show inferior overall survival compared with non-sAML/non-AML-MRC (<i>P</i> = 0.90). Adverse karyotypes were the predominant determinant among sAML/AML-MRC. Among sAML/AML-MRC patients, adverse karyotypes were associated with poorer survival than those without adverse karyotypes (<i>P</i> = 0.0075). In contrast, non-sAML/non-AML-MRC groups did not affect survival regardless of adverse karyotypes (<i>P</i> = 0.51). Among 65 patients with ELN 2017 adverse-risk, those with TP53 mutations had markedly shorter survival than those with TP53 wild-type (<i>P</i> = 0.018).</p><p><strong>Conclusions: </strong>sAML/AML-MRC with an adverse karyotype had a dismal outcome. These findings provide a benchmark for risk stratification in the pre- CPX-351 era.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2628479"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146157286","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}
HematologyPub Date : 2026-12-01Epub Date: 2026-01-06DOI: 10.1080/16078454.2025.2611526
Min Song, Xiaoman Sun, Pengyu Wang, Ning Liu, Menghao Wei, Jie Shi
{"title":"Serum gamma-glutamyl transpeptidase/albumin ratio in patients with acute myeloid leukemia: a new perspective on prognostic assessment.","authors":"Min Song, Xiaoman Sun, Pengyu Wang, Ning Liu, Menghao Wei, Jie Shi","doi":"10.1080/16078454.2025.2611526","DOIUrl":"https://doi.org/10.1080/16078454.2025.2611526","url":null,"abstract":"<p><strong>Objectives: </strong>Evaluate the gamma-glutamyl transpeptidase-to-albumin ratio (GAR) as a prognostic biomarker in acute myeloid leukemia (AML).</p><p><strong>Methods: </strong>We retrospectively analyzed the data of 162 patients with AML. Receiver operating characteristic (ROC) curve analysis determined the optimal cutoff value for GAR. Multivariate analysis was performed to assess the independent prognostic role of GAR for overall survival (OS) and event-free survival (EFS). Subgroup analysis was conducted to explore the impact of GAR in specific patient groups.</p><p><strong>Results: </strong>ROC curve analysis showed an optimal cutoff value of 0.84 for GAR. Multivariate analysis revealed that GAR was an independent prognostic factor for OS and EFS. Subgroup analysis demonstrated that a high GAR was associated with shorter OS and EFS in patients with intermediate-risk stratification, those aged ≥60 years, or those that have not undergone hematopoietic stem cell transplantation.</p><p><strong>Discussion: </strong>GAR reflects nutrition, inflammation, and oxidative stress. This study shows its potential to supplement existing AML risk stratification. Future prospective studies are needed to validate its clinical utility.</p><p><strong>Conclusion: </strong>We propose GAR as a prognostic biomarker in AML cases, providing a new perspective for prognostic assessment.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2611526"},"PeriodicalIF":1.6,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145911346","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}
HematologyPub Date : 2026-12-01Epub Date: 2026-03-17DOI: 10.1080/16078454.2026.2642515
Dan Wang, Jia Liu, Aoxue Tan, Jinqiong Li, Xiaoli Chen, Yimei Feng, Xi Zhang
{"title":"Efficacy and safety of TPO-RA combined with sirolimus in the treatment of relapsed immune thrombocytopenia.","authors":"Dan Wang, Jia Liu, Aoxue Tan, Jinqiong Li, Xiaoli Chen, Yimei Feng, Xi Zhang","doi":"10.1080/16078454.2026.2642515","DOIUrl":"10.1080/16078454.2026.2642515","url":null,"abstract":"<p><strong>Background: </strong>Relapsed immune thrombocytopenia (ITP) poses challenges in treatment. Thrombopoietin receptor agonists (TPO-RAs) are recommended as second-line therapy, but the efficacy of monotherapy is limited to about 60%. Our preliminary studies suggest sirolimus is effective for relapsed ITP. This study aimed to evaluate the efficacy and safety of TPO-RA combined with sirolimus in treating relapsed ITP.</p><p><strong>Methods: </strong>A retrospective analysis was conducted on 50 patients with relapsed ITP, all of whom received TPO-RAs (hetrombopag and eltrombopag) combined with sirolimus therapy. The primary endpoints were the overall response rate (ORR) at 4, 8, and 12 weeks of treatment. Secondary endpoints included safety of this regimen and subgroup analysis.</p><p><strong>Results: </strong>ORR at 4, 8, and 12 weeks was 61%, 76%, and 82%, respectively, with a median time to response of 7 days. Mean platelet counts at 2-, 4-, 8-, and 12-weeks post-treatment were 71 ± 12.7 × 10<sup>9</sup>/L, 86 ± 10.9 × 10<sup>9</sup>/L, 128 ± 19.7 × 10<sup>9</sup>/L and 131 ± 17.2 × 10<sup>9</sup>/L, respectively. Whether it is hetrombopag or eltrombopag, when combined with sirolimus, there is no statistically significant difference in efficacy between the two subgroups. TPORAs combined with sirolimus as second-line treatment and as third-line or beyond treatment showed no difference in efficacy. Among ANA-positive patients, the ORR shows no significant difference compared to the ANA-negative group (<i>p</i> > 0.05). Patients with ITP for less than one year are more likely to benefit from this treatment regimen (<i>p</i> = 0.003).</p><p><strong>Conclusions: </strong>The combination of TPO-RAs and sirolimus significantly improved platelet counts and sustained response rates. The regimen demonstrated a manageable safety profile, offering a novel therapeutic option for relapsed ITP.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"31 1","pages":"2642515"},"PeriodicalIF":2.0,"publicationDate":"2026-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473507","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}