Acta Haematologica最新文献

{"title":"Inferior Survival in Therapy-Related Chronic Myelomonocytic Leukemia: A Population-Based Analysis.","authors":"Arjun Syal, Douglas Tremblay","doi":"10.1159/aha/abkag010","DOIUrl":"https://doi.org/10.1159/aha/abkag010","url":null,"abstract":"<p><strong>Background: </strong>The prognostic impact of therapy-related chronic myelomonocytic leukemia (t-CMML) compared with de novo CMML (dnCMML) remains uncertain, in part due to the absence of therapy-related designations in cancer registries.</p><p><strong>Methods: </strong>Using the Surveillance, Epidemiology, and End Results (SEER) database, we compared outcomes between de novo CMML (dnCMML) and therapy-related CMML (t-CMML), operationally defined by antecedent chemotherapy and/or radiotherapy and diagnosis as a subsequent primary malignancy. Survival was evaluated by antecedent treatment exposure and within major antecedent malignancy groups. Additional operational definitions of t-CMML were evaluated to assess the robustness of findings.</p><p><strong>Results: </strong>Among 8,432 patients diagnosed with CMML between 2000 and 2022 after exclusion of antecedent myeloid malignancies, 594 (7.0%) met criteria for the primary definition of t-CMML. Median overall survival (OS) for the entire CMML cohort was 18 months, with shorter median OS observed for t-CMML compared with de novo CMML (14 vs 19 months). On multivariable Cox regression adjusted for age, sex, race, and era of diagnosis, t-CMML was associated with an increased risk of death (hazard ratio 1.20, 95% CI 1.09-1.32). Compared with dnCMML, inferior OS was observed among patients with recorded chemotherapy exposure but not among those treated with radiotherapy alone. Direct comparisons between chemotherapy-exposed and radiotherapy-only patients showed no significant difference in OS in the pooled adjusted analysis or within individual antecedent malignancy groups. Increasing definitional stringency was associated with stepwise worsening OS.</p><p><strong>Conclusion: </strong>Registry-defined t-CMML was associated with inferior survival compared with de novo disease. Although chemotherapy-exposed t-CMML was associated with poorer outcomes relative to dnCMML, OS did not differ significantly between chemotherapy-exposed and radiotherapy-only patients in direct pooled or malignancy-specific comparisons. Studies integrating detailed treatment histories with molecular and cytogenetic data are needed to clarify the biology and prognostic significance of t-CMML.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1"},"PeriodicalIF":2.8,"publicationDate":"2026-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148918093","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":"Complete Remission to Imatinib in a Myeloid Neoplasm with Basophilia and <i>PRKG2::PDGFRA</i> Gene Fusion: A Case Report.","authors":"Kirsten A Maddox, Shefali Tushar Patel, Shuko Harada, Diana Morlote, Fady M Mikhail, Pankit Vachhani","doi":"10.1159/aha/abkag009","DOIUrl":"10.1159/aha/abkag009","url":null,"abstract":"<p><strong>Introduction: </strong>The three most common myeloid/lymphoid neoplasms (MLNs) with eosinophilia and tyrosine kinase gene fusions have rearrangements of the genes coding the alpha and beta subgroups of platelet-derived growth factor receptors (PDGFRA, PDGFRB) and fibroblast growth factor receptor one (FGFR1). The most common partner gene of PDGFRA rearrangement is FIP1L1. However, a few notable exceptions to this have been reported.</p><p><strong>Case presentation: </strong>In our report, we present the case of a patient with high symptom burden and peripheral blood basophilia who initially went undiagnosed. Then, an unusual PRKG2::PDGFRA translocation with next-generation sequencing panel was detected. Treatment with imatinib led to complete remission. We discuss the clinical presentation, review the literature, and comment on its pathogenesis.</p><p><strong>Conclusion: </strong>In patients with myeloproliferative features, the absence of peripheral blood eosinophilia does not rule out MLN with tyrosine kinase gene fusions. Next-generation sequencing panels that detect RNA fusions may help uncover cryptic or rare fusions. MLN with PDGFRA rearrangement responds well to imatinib.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1"},"PeriodicalIF":2.8,"publicationDate":"2026-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148896249","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":"Positive Anti-Neutrophil Antibodies Test in Autoimmune Neutropenia Experience from Single-Center Cohort.","authors":"Yehonatan Sherf, Tzvika Porges, Ellie Ilan, Lilach Bonstein, Itai Levi","doi":"10.1159/aha/abkag008","DOIUrl":"10.1159/aha/abkag008","url":null,"abstract":"<p><strong>Introduction: </strong>Autoimmune neutropenia (AIN) is characterized by presence of anti-neutrophil antibodies and exclusion of other diagnosis. In contrast to pediatrics, data in adults are limited. We aimed to assess the basic characteristics of the disease, outcomes, and clinical impact in that group.</p><p><strong>Methods: </strong>We performed a retrospective cohort study of 180 adults with isolated neutropenia tested for anti-neutrophil antibodies at our center (2017-2023). Patients were classified as antibody positive or negative. A second comparison was conducted with a matched healthy control group for comparison. Clinical outcomes like infections, hospitalizations, antibiotic treatments, and mortality were analyzed.</p><p><strong>Results: </strong>Of 180 patients, 134 (74.4%) were antibody positive. No significant differences were found between antibody-positive and -negative groups in baseline characteristics and clinical outcomes. Compared with the healthy control group (3,600 patients), antibody-positive patients had higher rates of hospitalizations (29% vs. 15%, p < 0.001) and ER visits (50% vs. 32%, p = 0.002) but similar infection and death rates (0.7% vs. 2.4%, p = 0.4).</p><p><strong>Conclusion: </strong>Our findings suggest that adult AIN follows a benign course, similar to that of matched cohort. We also did not find any major difference between antibody-positive and -negative patients, raising questions about the utility of anti-neutrophil antibody testing in adults.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1"},"PeriodicalIF":2.8,"publicationDate":"2026-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148823908","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":"Predictive Value of Plasma sPECAM-1, t-PAIC, and sCLEC-2 for Early Severe Bleeding in Patients with Acute Promyelocytic Leukemia.","authors":"Lijun Hao, Xiang Li, Teng Xu, Yanjie Cui, Qin Huang, Yuanyuan Yu, Changmin Wang","doi":"10.1159/aha/abkag003","DOIUrl":"10.1159/aha/abkag003","url":null,"abstract":"<p><strong>Introduction: </strong>This study evaluated the predictive value of plasma sPECAM-1, t-PAIC, and sCLEC-2 (individually and in combination) for early severe bleeding events in patients with acute promyelocytic leukemia (APL).</p><p><strong>Methods: </strong>A retrospective review of 319 APL patients was conducted. Patients were divided into occurrence and nonoccurrence groups based on severe bleeding events. Plasma markers were measured. Logistic regression, ROC, calibration, decision curve analyses, and internal validation were performed.</p><p><strong>Results: </strong>Plasma sPECAM-1, sCLEC-2, and t-PAIC were significantly elevated in the occurrence group. Elevated levels of these markers, along with reduced levels of FIB, were independently associated with early severe bleeding events in patients with APL. AUCs for sPECAM-1, sCLEC-2, and t-PAIC were 0.759, 0.855, and 0.820, respectively; the three-marker combination achieved an AUC of 0.929, outperforming any single marker and the traditional model (DIC score, FIB, D-D, and WBC) (AUC = 0.804). Adding the new biomarkers to the traditional model further increased the AUC to 0.959 (p < 0.001). Internal validation yielded a corrected AUC of 0.916, with good calibration (p = 0.069). The combined model provided net clinical benefit across threshold probabilities of 4-98%.</p><p><strong>Conclusion: </strong>Plasma sPECAM-1, t-PAIC, and sCLEC-2 are elevated in APL patients with early severe bleeding. Their combined detection demonstrates high predictive efficacy for early severe bleeding in APL. However, the generalizability of this model requires further validation through large-scale, multicenter, prospective studies.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1"},"PeriodicalIF":2.8,"publicationDate":"2026-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148760506","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":"Isolated Myeloid Sarcoma of the Thoracic Spine as Initial Presentation of Acute Promyelocytic Leukemia: A Case Report and Literature Review.","authors":"Xiaoyong Chen, Jialing Lu, Junjie Hou, Jihao Zhou, Yang Wang, Xin Zhao, Chun Feng, Guoqiang Li, Lina Hu, Jizhang Bao, Beibei Qu, Yuanfei Mao, Peng Ke, Ying Xu","doi":"10.1159/000553021","DOIUrl":"https://doi.org/10.1159/000553021","url":null,"abstract":"<p><strong>Introduction: </strong>Acute promyelocytic leukemia (APL), a subtype of acute myeloid leukemia, typically presents with severe bleeding. However, the occurrence of APL with isolated myeloid sarcoma (MS) as the sole initial presentation is extremely rare, lacking typical clinical and laboratory features, which complicates diagnosis and management.</p><p><strong>Case presentation: </strong>A 45-year-old male was admitted for 2-month lower back pain. Spinal MRI showed T10 vertebral signal changes; histopathology confirmed the vertebral mass was myeloid origin, with PML::RARα fusion detected via PCR. Initial blood counts, coagulation tests, and bone marrow (BM) examinations were normal. However, 21 days postoperatively, the patient's platelet count and fibrinogen level declined. A subsequent BM examination revealed abundant abnormal promyelocytes, and FISH confirmed the presence of the PML::RARα fusion gene, diagnosing APL with MS and central nervous system (CNS) involvement. He achieved complete remission with all-trans retinoic acid-based chemotherapy.</p><p><strong>Conclusion: </strong>This is a rare case of isolated thoracic vertebra promyelocytic sarcoma as the initial symptom, which later progressed to APL with MS and CNS involvement. Additionally, we performed a comprehensive literature review of 13 other cases of APL with spinal MS at the time of diagnosis. In aggregate, APL presenting with spinal MS as the sole initial manifestation is extremely rare.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":2.8,"publicationDate":"2026-08-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148668056","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":"Eltrombopag-Induced Serum Pigmentation in Severe Aplastic Anemia: A Case Report.","authors":"Eva Desbrousses, Tifenn Charbonnel, Flavie Arbion, Hugo Alarcan, Nicolas Vallet","doi":"10.1159/000553383","DOIUrl":"10.1159/000553383","url":null,"abstract":"<p><strong>Introduction: </strong>Severe aplastic anemia (SAA) is a bone marrow failure disorder characterized by profound pancytopenia associated with a high risk of infection and bleeding.</p><p><strong>Case presentation: </strong>We report a case of a 19-year-old patient with a history of acute hepatitis who was admitted to the hospital with acquired SAA attested by 2% cellularity at bone marrow trephine biopsy. In the absence of matched sibling donor, immunosuppressive therapy with horse anti-thymocyte globulin, cyclosporine, and eltrombopag was initiated. Following centrifugation, serum obtained from blood samples addressed to the laboratory displayed a rare manifestation of brown color. Intravascular hemolysis was excluded based on normal haptoglobin and lactate dehydrogenase ranges, although bilirubin was mildly elevated. The brown staining disappeared after therapy discontinuation. Eltrombopag is a chromogenic molecule which absorbs visible light associated with serum coloration.</p><p><strong>Conclusion: </strong>This rare case highlights drug-induced serum pigmentation as a surprising manifestation in patients receiving eltrombopag and its normalization following drug withdrawal.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-5"},"PeriodicalIF":2.8,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13585414/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148597416","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Inverted U-Shaped Relationship between Pre-Discontinuation Tyrosine Kinase Inhibitor Dose Intensity and Treatment-Free Remission Outcome in Chronic Myeloid Leukemia.","authors":"Dae-Ho Choi, Jun Ho Jang, Chul Won Jung","doi":"10.1159/aha/abkag001","DOIUrl":"10.1159/aha/abkag001","url":null,"abstract":"<p><strong>Introduction: </strong>Treatment-free remission (TFR) is an established goal for patients with chronic myeloid leukemia in chronic phase (CML-CP) who achieve sustained deep molecular response (DMR) on tyrosine kinase inhibitor (TKI) therapy. However, the relationship between pre-discontinuation dose intensity (DI) and TFR outcome remains poorly characterized.</p><p><strong>Methods: </strong>We retrospectively analyzed 146 patients with CML-CP who attempted TFR at Samsung Medical Center between 2003 and 2025. The DI was defined as the time-weighted average ratio of actual to standard TKI dose during the 2-year period preceding discontinuation. The association between DI and TFR failure was modeled using Cox regression with restricted cubic splines (RCSs) in the DI subcohort (n = 126), using a DI of 1.0 as the reference.</p><p><strong>Results: </strong>In the full cohort, at median follow-up of 22.3 months, 50 (34.2%) patients experienced molecular relapse. TFR probability was 79.5% at 6 months and 61.9% at 48 months. In the DI subcohort, an unadjusted RCS model suggested a nonlinear, inverted U-shaped pattern, but the overall DI effect was not significant (likelihood ratio, p = 0.101; p for nonlinearity = 0.045). In exploratory post hoc groups, intermediate DI showed the highest TFR failure rate (49.0% vs. 25.0% and 28.6%; p = 0.035). The pattern did not reach significance after adjustment for TKI generation (p for nonlinearity = 0.074).</p><p><strong>Conclusion: </strong>These exploratory findings suggest that the biology underlying DMR maintenance, rather than the absolute pre-discontinuation dose, may influence TFR outcome. Given the modest sample size and post hoc design, this hypothesis-generating observation requires confirmation in independent cohorts.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1"},"PeriodicalIF":2.8,"publicationDate":"2026-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148576455","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":"Identifying Sexuality and Intimacy Needs in Patients with Multiple Myeloma: A Cross-Sectional Survey.","authors":"Ilana Levy Yurkovski, Sharon Peleg-Nesher, Gabriel Pazooky, Miri Zektser, Ekaterina Votinov, Ekaterina Frosinsky, Iuliana Vaxman, Varda Shoam, Tamar Tadmor, Natalia Kreiniz","doi":"10.1159/000553645","DOIUrl":"10.1159/000553645","url":null,"abstract":"<p><strong>Introduction: </strong>Sexual dysfunction and intimacy concerns are common in cancer but under-assessed in hematologic malignancies, including multiple myeloma (MM). MM-specific disease burden may compound age-related sexual dysfunction, yet structured data in this population are limited.</p><p><strong>Methods: </strong>We conducted an observational, cross-sectional, anonymous survey among adults with MM recruited from 3 Israeli hematology centers and the Israeli MM patient association. The Hebrew-language questionnaire assessed sexual dysfunction domains (before MM diagnosis vs. at survey completion), MM-related fears affecting intimacy (5-point Likert scale), reasons for sexual inactivity, and interest in supportive services. McNemar tests compared paired proportions; multivariable logistic regression explored associations with patient characteristics.</p><p><strong>Results: </strong>In this survey, 53 patients participated, with 62% aged >60 years; 79% partnered. Among respondents classifiable by sex (n = 34), most sexual dysfunction domains showed numerical increases from before diagnosis to survey completion. Two domains reached nominal statistical significance: low/no desire in women (21%-71%; p = 0.016) and erectile dysfunction in men (16%-53%; p = 0.016). The most common reasons for sexual inactivity were decreased desire, absence of a partner, and erectile dysfunction. Interest in supportive services was substantial: complementary medicine (27/53), hematologist consultation regarding sexual health (21/53), and sexual counseling (21/53) were most frequently endorsed. MM-related fears affecting intimacy were common, mostly of neuropathy (36%), weakness/fatigue (34%), and bone pain (32%).</p><p><strong>Conclusions: </strong>Sexual dysfunction and intimacy concerns are prevalent in MM and may worsen over the disease course. MM-specific fears are frequently endorsed and represent potentially addressable barriers to intimacy. Patients express clear interest in supportive services. Routine sexual health assessment and multidisciplinary supportive care should be integrated into MM clinical practice.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":2.8,"publicationDate":"2026-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13585415/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148547631","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comprehensive Analysis of Disulfidptosis-Related Genes in Multiple Myeloma: Implications for Prognosis and Immune Microenvironment.","authors":"Yuxiu Xu, Xiaoli Sun, Jing Yang, Dongsheng Ma, Jiangwei Wan, Tingting Lei, Tao Wang","doi":"10.1159/000553114","DOIUrl":"10.1159/000553114","url":null,"abstract":"<p><strong>Introduction: </strong>Multiple myeloma (MM) remains an incurable hematological malignancy with heterogeneous outcomes, necessitating the identification of novel prognostic markers for improved patient stratification and treatment.</p><p><strong>Methods: </strong>This study investigated the prognostic significance of disulfidptosis-related genes (DRGs) in MM, analyzing 1,272 samples from TCGA_MMRF and GSE4581 datasets. To explore the biological functions of key DRGs, siRNA was used to conduct in vitro experimental verification.</p><p><strong>Results: </strong>Through comprehensive bioinformatic analyses, we identified four genes (FLNA, GYS1, LRPPRC, and MYH10) significantly associated with MM prognosis. These DRGs linked the signature to disulfidptosis biology in MM. A novel four-gene signature effectively stratified patients into high-risk and low-risk groups with distinct survival outcomes, remained an independent prognostic factor, and varied significantly across disease stages. High-risk scores were associated with pathways involving mRNA processing, DNA replication, and cell-cycle progression, as well as immune shifts in neutrophil and plasma-cell fractions. Drug-sensitivity modeling showed distinct IC50 patterns between risk groups, with LRPPRC and GYS1 negatively correlated with sensitivity to selected agents. In vitro, LRPPRC knockdown in RPMI 8226 and U266 reduced viability and colony formation while increasing apoptosis, supporting the biological relevance of the signature.</p><p><strong>Conclusion: </strong>This novel DRG signature not only provides a valuable tool for patient stratification but also offers insights into the biological processes driving MM progression and potential therapeutic targets.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-16"},"PeriodicalIF":2.8,"publicationDate":"2026-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148468300","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":"Progression-Free Survival Effect Sizes in Randomized Controlled Trials of Targeted Cancer Therapies with Positive Outcomes.","authors":"Wei Zhang, Wen Yan, Mengyun Chen, Robert Peter Gale, Junren Chen","doi":"10.1159/000553384","DOIUrl":"10.1159/000553384","url":null,"abstract":"<p><strong>Introduction: </strong>Improvement in progression-free survival (PFS) is increasingly used by health authorities for cancer drug approvals.</p><p><strong>Method: </strong>We analyzed 106 randomized controlled trials (RCTs) with positive PFS outcomes published in JAMA Oncology, Lancet Oncology, Journal of Clinical Oncology, and Annals of Oncology during 2020-2023. RCTs were categorized as \"targeted\" (n = 83) or \"conventional\" (n = 23) by their interrogated treatment variables. Two measures of PFS effect sizes were analyzed: hazard ratio (HR) and PFS increment.</p><p><strong>Results: </strong>Among the RCTs of targeted therapies, the 25th-percentile value (Q1) of HR increased significantly (slope = 0.05 per year; p = 0.02) during the study period whilst Q1 of PFS increment decreased significantly (slope = -9.1 percentage points per year; p = 0.03). Similar trends were not observed among the RCTs of conventional therapies.</p><p><strong>Conclusions: </strong>Our analyses suggest a potential bias in accepting RCTs of targeted therapies despite decreased PFS effect sizes.</p>","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":2.8,"publicationDate":"2026-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13476059/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148395386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}