Annals of Hematology最新文献

{"title":"Successful use of ruxolitinib in steroid-refractory chronic GvHD affecting central nervous system: a case report.","authors":"Ewa Karakulska-Prystupiuk, Beata Zakrzewska-Pniewska, Agnieszka Tomaszewska, Marta Hałaburda-Rola, Wiesław Wiktor Jędrzejczak, Grzegorz Władysław Basak","doi":"10.1007/s00277-026-07064-3","DOIUrl":"https://doi.org/10.1007/s00277-026-07064-3","url":null,"abstract":"<p><p>Chronic graft-versus-host disease (cGvHD) is a major complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT). Beyond its classical manifestations, cGvHD may involve additional organs, including the central nervous system (CNS), where involvement remains poorly characterized, and therapeutic options are undefined, particularly in steroid-refractory cases. We report the case of a 50-year-old woman who underwent allo-HSCT for myelodysplastic syndrome and subsequently developed classical acute followed by chronic GvHD-overlap syndrome. At 2 years and 9 months post-transplant, she presented with progressive spastic paraparesis, and brain MRI revealed multifocal hyperintense white-matter lesions. A diagnosis of possible CNS-cGvHD was established. Due to poor response and intolerance to prolonged corticosteroid therapy, ruxolitinib was initiated at 10 mg/day and subsequently increased to 20 mg/day, with concomitant tapering of prednisone and immunoglobulin supplementation. Over 12 months, the patient demonstrated both clinical and radiological improvement, including significant functional recovery and marked regression of CNS lesions. This case suggests that ruxolitinib may represent a potential therapeutic option for atypical CNS involvement in cGvHD. Although clinical improvement likely reflected combined treatments, the neurological and radiological response suggests meaningful contribution from JAK inhibition. Further studies are needed to better define the role of ruxolitinib in this rare manifestation of cGvHD.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855668","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":"Romiplostim in the management of severe aplastic anemia: a comprehensive clinical review.","authors":"Mostafa F Mohammed Saleh, Abdulrahman Nasiri, Ahmed Kotb, Haroon Alfadil, Hazza Alzahrani, Mahmoud Aljurf, Ali Alahmari","doi":"10.1007/s00277-026-07057-2","DOIUrl":"https://doi.org/10.1007/s00277-026-07057-2","url":null,"abstract":"<p><p>Severe aplastic anemia (SAA) is a rare bone marrow failure disorder with high morbidity and mortality, particularly in patients ineligible for hematopoietic stem cell transplantation. While immunosuppressive therapy (IST) remains standard first-line treatment, a significant proportion of patients fail to respond or relapse. Thrombopoietin receptor agonists (TPO-RAs) have emerged as promising therapeutic options to enhance hematopoiesis. Among them, romiplostim has shown increasing efficacy across multiple treatment settings. To comprehensively review the clinical efficacy, safety, and optimal use of romiplostim in the treatment of SAA, based on data from clinical trials, real-world studies, and pediatric case series. A systematic literature search was conducted in PubMed, Web of Science, and Scopus through March 2026 using keywords related to \"romiplostim\" and \"severe aplastic anemia.\" Studies included prospective trials, retrospective cohorts, and mechanistic studies reporting hematologic response, transfusion independence, and adverse events in SAA patients treated with romiplostim. Romiplostim demonstrated consistent efficacy across pediatric and adult populations in 19 studies involving more than 390 patients with severe aplastic anemia. Hematologic response rates ranged from 41% to 95%, with trilineage recovery observed in up to 55% of patients. High-dose regimens (10-20 µg/kg/week) and early initiation were associated with superior outcomes, particularly in eltrombopag-refractory cases. Long-term use was well tolerated, with low rates of clonal evolution or serious toxicity. Romiplostim is an effective and safe therapeutic option in SAA, particularly in patients who are refractory to or intolerant of standard therapies. Its integration into frontline and salvage regimens offers the potential for durable trilineage responses and transfusion independence. Further studies are needed to define its role in combination strategies and optimal dosing and duration of therapy.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855707","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":"Gilteritinib response in acute myeloid leukemia harboring a rare FLT3 juxtamembrane domain mutation with subsequent clonal evolution.","authors":"Fumihiko Nakao, Takahiro Shima, Ken Takigawa, Yuichiro Semba, Fumiaki Jinnouchi, Takuji Yamauchi, Jun Odawara, Yoshikane Kikushige, Yasuo Mori, Koji Kato, Takahiro Maeda, Koichi Akashi","doi":"10.1007/s00277-026-07061-6","DOIUrl":"https://doi.org/10.1007/s00277-026-07061-6","url":null,"abstract":"<p><p>Mutations in the FMS-like tyrosine kinase 3 (FLT3) gene are among the most clinically relevant molecular abnormalities in acute myeloid leukemia (AML); however, the therapeutic significance of rare non-canonical FLT3 mutations involving the juxtamembrane domain (JMD) remains poorly defined. We report a 34-year-old woman with acute monocytic leukemia harboring a rare FLT3-JMD missense mutation (V579A) who experienced early relapse following standard induction and consolidation chemotherapy. Targeted next-generation sequencing revealed the presence of FLT3 V579A along with a concurrent truncating ARID1A mutation. Salvage therapy with the type I FLT3 inhibitor gilteritinib induced rapid hematologic remission within three weeks, allowing successful bridging to haploidentical allogeneic hematopoietic stem cell transplantation. Three months after transplantation, the patient relapsed, and genomic analysis demonstrated loss of the FLT3-mutated clone with the emergence and expansion of TP53-mutated independent clones, indicating clonal evolution and an apparent shift away from FLT3-dependent disease biology. This case suggests that AML harboring rare FLT3-JMD point mutations may exhibit transient dependence on FLT3 signaling and may respond to FLT3 inhibition despite the absence of canonical FLT3 alterations. These findings highlight the potential value of extended molecular profiling and longitudinal genomic assessment to identify potential therapeutic targets and mechanisms of resistance in relapsed AML.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855616","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":"Safety and efficacy of chidamide for maintenance therapy after allogeneic hematopoietic stem cell transplantation in patients with T-ALL/T-LBL.","authors":"Kuangfei Wang, Xiaojing Li, Xiaofan Li, Xiaohong Yuan, Xianling Chen, Xiaohui Lai, Nainong Li, Ping Chen","doi":"10.1007/s00277-026-07016-x","DOIUrl":"https://doi.org/10.1007/s00277-026-07016-x","url":null,"abstract":"<p><p>Post-transplant relapse remains a primary obstacle to long-term survival in patients with T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/T-LBL), underscoring the need for effective maintenance therapies. Although chidamide, a selective histone deacetylase inhibitor, has shown activity in T-cell malignancies, its role as post-transplant maintenance has been scarcely studied. To evaluate the efficacy and safety of chidamide as maintenance therapy in T-ALL/T-LBL patients after allogeneic hematopoietic stem cell transplantation (allo-HSCT). We retrospectively analyzed 75 T-ALL/T-LBL patients who underwent allo-HSCT (December 2019-January 2024). Thirty-eight patients received chidamide-based maintenance, and 37 served as controls (no maintenance or non-chidamide regimens). The primary endpoints were overall survival (OS) and progression-free survival (PFS); safety endpoints included graft-versus-host disease (GVHD) incidence and treatment-related adverse events. Chidamide maintenance was associated with significantly improved OS and PFS compared with the non-chidamide group (OS: p = 0.001; PFS: p = 0.006). Multivariate Cox regression identified chidamide maintenance (OS: p = 0.003; PFS: p = 0.006) and complete remission status before transplantation (OS: p = 0.013; PFS: p = 0.012) as independent favorable prognostic factors. Chidamide did not increase acute GVHD; chronic GVHD was numerically higher (26.32% vs. 13.51%) but mostly mild to moderate and manageable. Adverse events were primarily hematologic toxicity (bone marrow suppression) and gastrointestinal, generally manageable and rarely required treatment discontinuation. Chidamide maintenance after allo-HSCT significantly improves survival outcomes in T-ALL/T-LBL patients with an acceptable safety profile, while pre-transplant remission status further refines prognosis. Prospective studies are warranted to confirm these findings and optimize maintenance strategies in this setting.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855678","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":"Impact of diagnosis-to-treatment interval on the outcome of patients with acute myeloid leukemia.","authors":"Samah Nassereddine, Yuan Feng, Jordan Selep, Firas El Chaer, Leah Wells, Ramey Elsarrag, Kimberly Doucette, Vanya Aggarwal, Lacey Williams, Imad Tabbara, Shanshan Liu, Guoquing Diao, Mary-Elizabeth M Percival, Catherine Lai","doi":"10.1007/s00277-026-07052-7","DOIUrl":"https://doi.org/10.1007/s00277-026-07052-7","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is considered an oncologic emergency, yet the optimal timing for treatment initiation remains uncertain. We conducted a multi-institutional retrospective study of 698 adults with newly diagnosed AML presenting to four academic centers across the United States. Diagnosis-to-treatment intervals (DTI) were categorized as < 5 days, 5-10 days, and > 10 days. Outcomes were analyzed using multivariable models adjusting for age, treatment intensity, ELN 2017 risk classification, and white blood cell count. Among younger patients, DTI was not associated with differences in survival outcomes. In contrast, older patients demonstrated improved survival with delayed treatment (DTI > 10 days), particularly those with lower white blood cell counts. No adverse effects from treatment delay were observed in younger cohorts. This retrospective study showed that prolonged DTI is associated with improved survival in older adults with newly diagnosed AML, challenging the traditional assumption that immediate therapy universally improves outcomes. These findings underscore the importance of individualized treatment timing in the era of precision oncology.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147863445","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":"Efficacy and safety of platelet-rich plasma injection for hemophilic chronic synovitis and arthropathy: a systematic review and meta-analysis.","authors":"Shaoyu Yin, Yijia Lu, Qi He, Hui Bi, Zeping Zhou","doi":"10.1007/s00277-026-07055-4","DOIUrl":"https://doi.org/10.1007/s00277-026-07055-4","url":null,"abstract":"<p><p>Platelet-rich plasma (PRP) has emerged as a potential therapeutic approach for hemophilia. Systematically summarize the evidence regarding the efficacy and safety of PRP therapy in hemophilia. PubMed, Embase, Web of Science, Cochrane Library, and ClinicalTrials.gov were searched. Risk of bias was assessed using Cochrane Risk of Bias (RoB 2) and the RoB for Non-Randomized Studies of interventions (ROBINS-I V2). Analyses were conducted separately for hemophilic synovitis and arthropathy given their distinct pathophysiology. A random-effects model was applied for meta-analysis. Evidence certainty was evaluated using the Grading of Recommendations Assessment, Development, and Evaluation (GRADE). Six studies were included: 1 randomized controlled trial (RCT) and 5 non-randomized trials. For chronic synovitis, pooled analyses demonstrated significant reductions in Visual Analogue Scale (mean difference: -3.36, - 4.42, and - 2.60 at 3, 6, and 12 months), Hemophilia Joint Health Score (mean difference: -3.00, - 4.72, and - 3.65 at 3, 6, and 12 months); however, all outcomes were rated as very low certainty. For arthropathy, the single high-quality RCT showed no clinically meaningful improvements in Western Ontario and McMaster Universities Osteoarthritis Index, Hemophilia Joint Health Score, Visual Analogue Scale, or magnetic resonance imaging compared with placebo over 24 months (high certainty). Adverse events were minimal, with injection site discomfort reported in 8.4% of patients (high certainty). The evidence for PRP in hemophilic chronic synovitis is of very low certainty, and the results should be interpreted with caution. High-certainty evidence does not support the routine use of PRP in hemophilic arthropathy.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855696","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":"Incidence of infections in patients treated with rituximab for autoimmune disorders of hematological Interest or non-Hodgkin lymphoma.","authors":"Salvatore Perrone, Riccardo Tomasello, Simona Raso, Ombretta Annibali, Mariantonietta Tafuri, Marta Mattana, Tania Soriano, David Fanciullo, Giovanni Manfredi Assanto, Giacinto Luca Pedone, Erminia Baldacci, Eugenio Santacroce, Uros Markovic, Gaetano Giuffrida, Manuela Giuseppa Ingrascì, Mario Biglietto, Claudia Cammarata, Matteo Totaro, Silvio Ligia, Bruno Fattizzo, Cristina Santoro, Sergio Siragusa, Mariasanta Napolitano","doi":"10.1007/s00277-026-07047-4","DOIUrl":"https://doi.org/10.1007/s00277-026-07047-4","url":null,"abstract":"<p><p>Rituximab is employed in the treatment of B-cell non-Hodgkin lymphomas (NHL) and autoimmune disorders of hematological interest (ADHI), such as autoimmune hemolytic anemia (AIHA), acquired hemophilia A (AHA), and immune thrombocytopenia (ITP). Rituximab induces profound B-cell depletion, reducing the titer of immunoglobulins and autoreactive antibodies, but secondary hypogammaglobulinemia increases risk of infections. We performed a retrospective, multicentric study across seven Italian centers to compare two cohorts of patients with either indolent NHL or ADHI treated with rituximab between March 2014 and March 2024. We evaluated the incidence and characteristics of infections and common antimicrobial prophylaxis adopted according to local clinical practice, in the absence of shared guidelines for ADHI. We included 285 patients (52% female; median age 55 years). The ADHI cohort comprised 187 patients (65%; 97 ITP, 78 AIHA, 12 AHA), while the control group included 98 i-NHL patients (34%). We documented 65 infectious events occurring in 65/285 patients (22.8%). Infection-related mortality occurred in 9 patients (4%). When stratified, the cumulative 180-day incidence of infections was 9.4% (IC95% 8.8%-10%) for NHL patients and 13.7% for ADHI (IC95% 12.2%-14.2%). Multivariate analysis identified prophylaxis (OR 2.62, p = 0.014) and post-treatment hypogammaglobulinemia (OR 2.73 p = 0.004) as risk factors. The risk of infection in patients with ADHI is high and comparable to that observed in NHL. Therefore, close monitoring of IgG levels is strongly advised, and mortality can be contained through rigorous antimicrobial stewardship.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147863503","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":"WT1 mRNA in peripheral blood enables early prognostic stratification in AML patients receiving venetoclax and azacitidine therapy.","authors":"Hiroyuki Sugiura, Noboru Asada, Takeru Asano, Yasushi Hiramatsu, Tomohiro Urata, Toshi Imai, Makoto Nakamura, Kazuhiko Yamamoto, Tatsunori Ishikawa, Masanori Makita, Takanori Yoshioka, Kazutaka Sunami, Kyosuke Saeki, Yuichiro Nawa, Maiko Kimura, Makoto Takeuchi, Masaya Abe, Shoji Asakura, Tomofumi Yano, Yoshinobu Maeda","doi":"10.1007/s00277-026-07046-5","DOIUrl":"https://doi.org/10.1007/s00277-026-07046-5","url":null,"abstract":"<p><p>Venetoclax and azacitidine (VEN/AZA), which target BCL-2 and DNA methylation, have demonstrated substantial efficacy, particularly in older or unfit patients with acute myeloid leukemia (AML). Wilms' Tumor-1 (WT1) mRNA, measurable in peripheral blood (PB), is an established biomarker for monitoring treatment response and predicting AML prognosis. We conducted a retrospective analysis of patients with AML treated with VEN/AZA within the Okayama Hematology Study Group (OHSG). Patients who showed a marked reduction in PB WT1 mRNA levels from baseline after the first or second treatment cycle had significantly improved overall survival (OS) and progression-free survival (PFS). Moreover, achieving PB WT1 mRNA negativity-regardless of whether this occurred early or later in the therapy།was consistently associated with superior OS and PFS. These findings suggest that PB WT1 mRNA is a sensitive and reliable biomarker for predicting treatment response and long-term outcomes in patients with AML receiving VEN/AZA.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855646","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":"MRD conversion during oral azacitidine maintenance correlates with outcomes in predominantly favorable-risk AML.","authors":"Noa Hurvitz, Irina Amitai, Ilana Levy, Ariella Tvito, Yishai Ofran, Tzvika Porges, Ofir Wolach, Adi Sherban, Noa Gross Even-Zohar, Vladimir Vainstein, Arnon Haran, Shlomzion Aumann, Robert Mikhelashvili, Avraham Frisch, Boaz Nachmias","doi":"10.1007/s00277-026-07054-5","DOIUrl":"https://doi.org/10.1007/s00277-026-07054-5","url":null,"abstract":"<p><strong>Background: </strong>Oral azacitidine improves survival in patients with AML in first complete remission who are not candidates for allogeneic transplantation. Most favorable-risk AML patients harbor molecular markers enabling sensitive measurable residual disease (MRD) monitoring. While MRD is a well-established prognostic factor in AML, the clinical significance of MRD dynamics during maintenance therapy remains incompletely defined.</p><p><strong>Methods: </strong>We conducted a multicenter, real-world retrospective study of 30 patients with AML, predominantly ELN favorable-risk, treated with oral azacitidine maintenance following intensive induction-consolidation. MRD was assessed longitudinally using standardized molecular and flow-based assays. Outcomes were analyzed according to MRD status at maintenance initiation and subsequent MRD conversion.</p><p><strong>Results: </strong>At initiation of oral azacitidine, 47% of patients were MRD-positive. MRD conversion to negativity occurred in 64% of these patients during maintenance. Relapse-free survival (RFS) was comparable between patients who were MRD-negative at baseline and those who converted to MRD negativity (24-months restricted median RFS 577 vs. 638 days), whereas patients with persistent MRD positivity experienced early relapse (24-months restricted median RFS 63 days). Despite inferior RFS, overall survival did not significantly differ between MRD-defined groups. Patients relapsing after oral azacitidine retained sensitivity to subsequent therapies, including venetoclax-azacitidine-based regimens, with high rates of molecular response.</p><p><strong>Conclusions: </strong>Oral azacitidine was associated with high rates of MRD conversion, conferring RFS comparable to patients who were MRD-negative at treatment initiation. Persistent MRD identified patients at high-risk for early relapse, while effective salvage therapies mitigated overall survival differences. These findings support prospective evaluation of MRD-guided maintenance strategies in favorable-risk AML but should be considered hypothesis-generating given the retrospective design and limited cohort size. The trial is on behalf of the Israel Acute Leukemia Group.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":" ","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855662","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":"The role of interleukin-1 beta as an early biomarker for renal dysfunction in Egyptian sickle cell disease patients","authors":"Nourhan Mohamed Nasr,&nbsp;Noha Khalifa Abd El Ghaffar,&nbsp;Shaimaa Mohamed El Asmar,&nbsp;Yasmin Mohamed Saber","doi":"10.1007/s00277-026-07000-5","DOIUrl":"10.1007/s00277-026-07000-5","url":null,"abstract":"<div><h3>Background</h3><p>Sickle cell nephropathy is a recognized complication increasingly seen in young individuals with sickle cell disease (SCD). The typical progression of glomerular disease is thought to involve hyperfiltration and albuminuria, eventually leading to a decline in glomerular filtration rates and ultimately end-stage renal disease (ESRD). This study sought to investigate the relationship between urinary interleukin-1 beta (IL-1β), a marker of inflammation, and existing biomarkers of renal damage in SCD patients. The goal was to identify IL-1β as a novel, early diagnostic biomarker for sickle nephropathy.</p><h3>Patients and methods</h3><p>This case-control study enrolled forty-six patients (both genders, aged 18–40 years) with sickle cell disease and forty-six healthy controls. All patients were subjected to the following: A complete medical history, physical examination, and laboratory investigation including a complete blood analysis, blood chemistry (which included kidney function tests and liver function, serum ferritin, urinalysis, hepatitis B surface antigen and hepatitis C antibody, estimated GFR, urinary IL-1 beta using the ELISA technique, and urinary albumin/creatinine ratio). These measurements were taken for both the case and control groups.</p><h3>Results</h3><p>The mean estimated glomerular filtration rate (eGFR) level showed a statistically significant increase in SCD patients. The median value of the albumin/creatinine ratio level was statistically different between cases and controls. The median value of urinary IL-1 beta level was statistically different between cases and controls. Assessment of correlations between urinary IL-1 beta and eGFR revealed a statistically significant negative correlation between them, while assessment of the correlation between urinary IL-1 beta and the urinary albumin/creatinine ratio revealed a statistically significant positive correlation between them in sickle cell patients.</p><h3>Conclusion</h3><p>In this study, significant renal hyperfiltration, a high frequency of albuminuria, and elevated urine IL-1β levels are all present in Egyptian adults with sickle cell disease. Urinary IL-1β, albuminuria, and eGFR are strongly correlated, highlighting the crucial role of inflammation in sickle cell nephropathy pathogenesis. A promising non-invasive biomarker for early renal injury and disease progression in sickle cell disease (SCD) may be urinary IL-1β.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 6","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-026-07000-5.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147828488","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}