Hematology Reports最新文献

{"title":"Navigating the New Era in Myelodysplastic Neoplasms: A Review of Prognostic Implications of the IPSS-M Score and 2022 WHO Classification.","authors":"Mihai-Emilian Lapadat, Oana Stanca, Nicoleta Mariana Berbec, Cristina Negotei, Andrei Colita","doi":"10.3390/hematolrep17060058","DOIUrl":"10.3390/hematolrep17060058","url":null,"abstract":"<p><p>Myelodysplastic neoplasms represent a diverse group of clonal hematopoietic stem cell disorders characterized by ineffective hematopoiesis, peripheral cytopenias, and an inherent risk of progression to acute myeloid leukemia. Accurate risk assessment and patient stratification are critical to optimizing therapeutic approaches and clinical outcomes. In 2022, significant advancements reshaped both the classification and prognostic stratification of MDSs. The revised WHO Classification introduced crucial genetically defined subtypes, particularly those involving biallelic <i>TP53</i> inactivation and <i>SF3B1</i> mutations, shifting the emphasis from traditional morphology-based criteria to molecular ones. Simultaneously, morphological subtypes such as hypoplastic and hyperfibrotic MDSs were established as distinct entities with unique prognostic implications. At the same time, the introduction of the International Molecular Prognostic Scoring System (IPSS-M) provided a more precise prognostic stratification by integrating comprehensive molecular data alongside traditional clinical and cytogenetic parameters. Several validation studies have confirmed IPSS-M's superior discriminative power compared to previous models, notably IPSS-R, improving predictions regarding overall survival and leukemia transformation. Nevertheless, practical considerations regarding the widespread application of IPSS-M have emerged, including concerns over economic feasibility and accessibility of advanced molecular testing methods, such as extensive Next-Generation Sequencing panels. This review synthesizes the recent literature and critical studies validating these classification and prognostic updates, discussing their clinical impact, practical considerations, and implications for targeted therapeutic strategies. By focusing on molecular pathogenesis, the latest classification systems and prognostic models promise significant advances in patient-specific management, setting the stage for future innovations in treatment and improved patient outcomes.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 6","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12641622/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145587209","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Role of Daratumumab, Lenalidomide, and Dexamethasone in Transplantation-Eligible Patients with Multiple Myeloma After the Failure of Bortezomib-Based Induction Therapy.","authors":"Shun Ito, Takashi Hamada, Masaru Nakagawa, Takashi Ichinohe, Hironao Nukariya, Toshihide Endo, Kazuya Kurihara, Yuichi Takeuchi, Shimon Otake, Hiromichi Takahashi, Hideki Nakamura, Katsuhiro Miura","doi":"10.3390/hematolrep17060057","DOIUrl":"10.3390/hematolrep17060057","url":null,"abstract":"<p><p><b>Background/Objectives:</b> The role of daratumumab, lenalidomide, and dexamethasone (DRd) in autologous stem cell transplantation (ASCT)-eligible patients with multiple myeloma (MM) after first-line bortezomib, cyclophosphamide, and dexamethasone (VCd) treatment is not yet established. <b>Methods:</b> We retrospectively evaluated ASCT-eligible patients with MM who received second-line therapy with DRd after initial induction therapy with VCd between 2017 and 2023 (salvage group). For comparison, patients who successfully underwent per-protocol treatment with VCd induction, followed by ASCT during the same period, were selected (control group). <b>Results:</b> Eight patients with a median age of 61 years (range, 36-68 years) were included in the salvage group. After a median of 5 DRd cycles, the best response was partial response (PR) in two patients (25%) and a very good partial response (VGPR) in six (75%). All patients underwent ASCT, resulting in PR in one (13%), VGPR in four (50%), and stringent complete response in three (38%). Measurable residual disease (MRD) assessed using multicolor flow cytometry was negative in four patients (50%). The controls included thirteen patients with a median age of 60 years (range, 44-64 years). While most patients in both groups received various post-ASCT therapies, the post-ASCT 2-year time to the next treatment rate was slightly better in the salvage group than in the control group (88% vs. 49%, <i>p</i> = 0.089). However, hypogammaglobulinemia was more common in the salvage group (75% vs. 15%, <i>p</i> = 0.018). <b>Conclusions:</b> This small case series suggests that DRd is promising for ASCT-eligible patients with MM after VCd failure.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 6","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12641952/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145587269","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Gut Microbiome Role in Multiple Myeloma: Emerging Insights and Therapeutic Opportunities.","authors":"Mina Y George, Nada K Gamal, Daniel E Mansour, Ademola C Famurewa, Debalina Bose, Peter A Messiha, Claudio Cerchione","doi":"10.3390/hematolrep17060056","DOIUrl":"10.3390/hematolrep17060056","url":null,"abstract":"<p><p>Multiple myeloma is a hematological cancer depicted by the proliferation of plasma cells within the bone marrow, causing immune dysfunction and other abnormalities. The gut microbiome, the microbial community in the gastrointestinal tract, was found to modulate systemic immunity, inflammation, and metabolism. Although the interplay between gut microbiome and multiple myeloma has been found in recent research, there is a gap in knowledge linking the effect of the microbiome on the pathogenesis and treatment of multiple myeloma. The imbalance in the gut microbiome, dysbiosis, may influence multiple myeloma pathogenesis through immune modulation and inflammation. Certain microbial species have been associated with multiple myeloma progression, complications, and therapeutic responses to treatment. Moreover, microbiome-derived metabolites, short-chain fatty acids, can influence the immune circuits associated with multiple myeloma progression. Understanding the bidirectional relationship between multiple myeloma and gut microbiota may provide insights into enhanced treatment and the development of new microbiome-based interventions. The current review provides a comprehensive highlight of current evidence linking the gut microbiome with multiple myeloma, demonstrating its significant roles in the development, progression, and treatment of multiple myeloma. Additionally, it focuses on the therapeutic potential of modulating the gut microbiome as a novel adjunct strategy in multiple myeloma management.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 6","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12641986/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145587241","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Outcomes for Primary Central Nervous System Lymphoma from a Single Institution.","authors":"Sruthi Dontu, Jacob Boccucci, Michael Chahin, Amany Keruakous, Anand Jillella, Jorge Cortes, Vamsi Kota, Locke Bryan, Ayushi Chauhan","doi":"10.3390/hematolrep17060055","DOIUrl":"10.3390/hematolrep17060055","url":null,"abstract":"<p><p><b>Background</b>: Primary central nervous lymphoma (PCNSL) is a rare, aggressive, non-Hodgkin's lymphoma. Outcomes are poor with standard induction of high-dose methotrexate (HD-MTX)-based regimens and consolidation. We present retrospective data from the Georgia Cancer Center. <b>Methods</b>: A single retrospective chart review was conducted on all PCNSL patients from 2013 to 2023 to assess for various factors influencing care. <b>Results</b>: Of a total of 38 PCNSL patients, 6 died and 2 were lost to follow-up prior to therapy initiation, leading to a total of 30 patients for analysis. The median age was 62.3 (21-82 years). One patient had HIV/AIDS. Two patients were on immunosuppression for either kidney transplant or multiple sclerosis (MS). The HIV and MS cases were Epstein-Barr Virus (EBV)-positive. Completion of ≥six cycles of induction was predictive of response. <b>Conclusions</b>: PCNSL remains an area of high unmet need. Recent studies have shown that HD-MTX-based therapy and autologous stem cell transplantation afterwards leads to improved outcomes regardless of age; however, non-relapse mortality is important to consider. Our data from a primarily elderly and sub-rural cohort reiterate the efficacy of combination chemoimmunotherapy and impact of induction cycle number on response, regardless of age. A multidisciplinary approach and targeted agent maintenance should be considered to improve outcomes in the elderly.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 6","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12641803/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145587254","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Vitamin K Antagonists (VKAs) and Novel Oral Anticoagulants (NOACs) Safety Comparison Based on Data from EudraVigilance Database.","authors":"Pier Paolo Olimpieri, Fanny Erika Palumbo, Gaetano Giuffrida, Edoardo Milanetti, Cecilia Gozzo, Elisa Lucia Scebba, Giovanni Luca Romano, Giovanni Enrico Lombardo, Andrea Duminuco, Calogero Vetro, Davide Giuseppe Castiglione, Giuseppe Alberto Palumbo, Salvatore Scarso, Filippo Drago, Lucia Gozzo","doi":"10.3390/hematolrep17050054","DOIUrl":"10.3390/hematolrep17050054","url":null,"abstract":"<p><p><b>Background</b>: Clinical trials comparing novel oral anticoagulants (NOACs) with warfarin reported a lower mortality rate and a reduced incidence of bleeding with NOACs. However, these studies do not allow for final conclusions about safety. Moreover, direct comparisons among NOACs are not available. <b>Objectives</b>: The aim of this study was to analyze data from EudraVigilance in order to compare OAC safety profiles. <b>Methods</b>: We searched for all suspected adverse drug reactions (ADRs) from OACs collected in the EudraVigilance up to March 2019. We calculated the reporting odds ratios (RORs) in order to assess the risk of reporting specific ADRs among drugs. Moreover, OAC safety profiles were investigated through correspondence analysis and visualized in contribution biplots. <b>Results</b>: A total of 244,149 individual case safety reports (ICSRs; 431,354 ADRs) related to OACs were retrieved from EudraVigilance. About 80% of ICSRs refer to NOACs, especially rivaroxaban. Gastrointestinal (Gastr) and central nervous system (Nerv) disorders were the most represented categories. More than 90% of ADRs were serious and almost 9% fatal, with the highest ROR reported for dabigatran. Both fatal and non-fatal ADRs reported for Vitamin K Antagonists (VKAs) differed from those reported for NOACs. Among the latter, dabigatran and rivaroxaban showed similar profiles, while apixaban differed from all other OACs, even in the case of fatal ADRs. <b>Conclusions</b>: As expected, data collected from EudraVigilance showed differences among drugs, probably related to their specific characteristics and/or the peculiar utilization in clinical practice. Further investigations are needed to better compare the safety profile of OACs.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 5","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12563034/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145388931","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Assessment of Pharmacokinetics, Clinical Outcomes, and Costs After Switching from Standard Half-Life to Extended Half-Life FVIII in Well-Controlled Hemophilia A Patients.","authors":"Maria Choví-Trull, Juan Eduardo Megías-Vericat, Santiago Bonanad-Boix, Saturnino Haya-Guaita, Ana Rosa Cid-Haro, Marta Aguilar-Rodriguez, Tomás Palanques-Pastor, Javier Garcia-Pellicer, Jose Luis Poveda-Andrés","doi":"10.3390/hematolrep17050053","DOIUrl":"10.3390/hematolrep17050053","url":null,"abstract":"<p><p><b>Objective:</b> This study aimed to analyze pharmacokinetic and clinical parameters (bleeding rates and joint health) before and after switching from standard half-life (SHL) factor VIII (FVIII) to extended half-life (EHL) PEGylated turoctocog alfa pegol in patients with severe/moderate hemophilia A (HA) on prophylaxis, one year prior to and following the switch in a real-world setting. <b>Methods:</b> A single-center, comparative, observational, sequential, retrospective, multidisciplinary study was designed. The population pharmacokinetic parameters were estimated using the WAPPS-Hemo^® platform. The annualized bleeding rate (including total and joint bleeds), joint health (Hemophilia Joint Health Score), FVIII consumption, administration frequency, and treatment costs were analyzed. <b>Results:</b> Eight patients with severe (n = 7) or moderate (n = 1) HA on prophylaxis were included after switching to turoctocog alfa pegol. With this regimen, the median FVIII half-life was 16.8 (15.2-19.1) hours, the area under the curve (AUC) was 18,182 (12,879-21,214) IU·h/dL, and the incremental recovery was 2.2 IU/dL per (1.6-2.4) IU/kg. The patients required a median of 2.0 infusions per week (2.0-2.0), corresponding to a weekly consumption of 57.8 (54.2-61.1) IU/kg. Clinically, the prophylactic regimen was associated with fewer infusions per week, stable joint health, and a reduction in overall treatment costs. <b>Conclusions:</b> Prophylaxis with turoctocog alfa pegol provided the expected pharmacokinetic profile of an EHL-FVIII concentrate, enabled a lower infusion frequency, and was linked to a decreased treatment burden and cost while maintaining joint health.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 5","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12563216/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145388889","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Concomitant Acquired Hemophilia A and Acquired Von Willebrand Syndrome from Distinctive Autoantibodies: Case Report.","authors":"Richard Yu, Mackenzie Bowman, Arnaud Bonnefoy, Paula James, Chai W Phua","doi":"10.3390/hematolrep17050052","DOIUrl":"10.3390/hematolrep17050052","url":null,"abstract":"<p><p><b>Background and Clinical Significance:</b> Acquired hemophilia A (AHA) and acquired von Willebrand syndrome (AVWS) are rare bleeding disorders that do not often present concurrently. Here, we report a coexisting AHA and AVWS case due to underlying autoantibodies to factor VIII (FVIII) and von Willebrand factor (VWF). <b>Case Presentation:</b> A patient with gastrointestinal bleeding and prolonged aPTT was diagnosed with AHA and AVWS. The patient was started on immunosuppression with prednisone, cyclophosphamide, and intravenous immunoglobulin, alongside recombinant porcine FVIII replacement, susoctocog alfa. AVWS reduced the half-life of susoctocog alfa, requiring more frequent dosing and laboratory monitoring until AVWS resolved. The patient had two further relapses; the most recent was treated with Rituximab, following which remission has been maintained. <b>Conclusions:</b> Given the potential therapeutic implications, VWF testing should be considered as part of the diagnostic workup for AHA.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 5","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12564277/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145388910","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association of Cognitive Impairment with Reduced Health-Related Quality of Life and Depression Among Survivors of Thrombotic Thrombocytopenic Purpura.","authors":"Sruthi Selvakumar, Jia Yu, Jacob Meade, Shruti Chaturvedi","doi":"10.3390/hematolrep17050051","DOIUrl":"10.3390/hematolrep17050051","url":null,"abstract":"<p><p><b>Background</b>: Immune-mediated thrombotic thrombocytopenic purpura (iTTP) survivors exhibit increased rates of psychological comorbidities, cognitive impairment (CI), and reduced health-related quality of life (HRQoL). This cross-sectional study investigated the prevalence of CI and its association with reduced HRQoL and depression among iTTP survivors. <b>Methods</b><i>:</i> iTTP survivors completed the Beck Depression Inventory (BDI-II), the SF-36 for evaluation of HRQoL, and the NIH Toolbox Cognition Battery. SF-36 scores and fluid cognition and crystallized cognition composite scores from the cognition battery were compared to the reference population. We examined associations of cognitive impairment with depression and HRQoL. <b>Results</b>: We enrolled 47 patients with iTTP; 76.6% were female, the median age was 51 (IQR 39, 60), and the median number of episodes was 2 (1, 3.5). Compared to the reference, iTTP survivors had significantly lower mean scores in seven SF-36 domains (physical function, physical limitation, general, mental health, vitality, social functioning, and emotional limitation) as well as the mental component score (MCS) (<i>p</i> < 0.0001) and physical component scores (PCS) (<i>p</i> < 0.0001). A lower physical HRQoL score was observed in those with mild (49.3 vs. 37.7, <i>p</i> = 0.005) and major (49.3 vs. 38.4, <i>p</i> = 0.007) CI compared to no CI. The fluid cognition composite score correlated strongly with the SF-36 Physical Component Summary (r = 0.548, <i>p</i> = 0.0002) but not the Mental Component Summary (r = 0.113, <i>p</i> = 0.489). <b>Conclusions</b>: Cognitive impairment in iTTP survivors is associated with reduced physical HRQoL. Identifying and addressing cognitive deficits in iTTP may improve HRQoL. Given that 40% of participants had depressive symptoms, which were associated with reduced mental HRQoL, iTTP survivors may also benefit from routine mental health screening t.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 5","pages":""},"PeriodicalIF":1.9,"publicationDate":"2025-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12564109/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145388921","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Presentation, Diagnosis, and Management of Autosomal Dominant Common Variable Immunodeficiency Type XII with NFKB1 Mutation and Autoimmune Neutropenia Treated with Allogenic Stem Cell Transplantation.","authors":"Matthew Gold, Chandini Kannan, Ashley Schofield, Alane Rogers, Charles J Weeks, Sruthi Dontu, Joseph Suchomski, Nabil Ghani, Shawn Doss, Jacob Boccucci, Mei Zheng, Amany Keruakous","doi":"10.3390/hematolrep17050049","DOIUrl":"10.3390/hematolrep17050049","url":null,"abstract":"<p><p><b>Background and Clinical Significance:</b> Common Variable Immunodeficiency (CVID) is a prevalent manifestation of primary immunodeficiency disorder. The current mainstay of treatment is immunoglobulin replacement therapy; however, in patients with severe complications or refractory disease, hematopoietic stem cell transplant (HSCT) is indicated. Despite this, there has been little research regarding HSCT as a treatment for CVID, with few case reports demonstrating clinical benefit. <b>Case presentation:</b> We present a unique case of common variable immunodeficiency Type XII (CVID12) with rare NFKB mutation and its management. A 20-year-old female with autoimmune alopecia, eczema, and a congenital atrophic right kidney presented to the emergency department with a three-month history of intermittent fever, malaise, lymphadenopathy, mouth sores, diarrhea, and odynophagia, accompanied by a 5 lb. unintentional weight loss and night sweats. Previously, she received multiple steroid prescriptions for these symptoms, providing only temporary relief with each course. Lab findings revealed severe neutropenia and imaging demonstrated hepatosplenomegaly and lymphadenopathy. Flow cytometry revealed a slightly atypical CD8-positive T-cell population and bone marrow biopsy revealed variable cellular marrow with trilineage hematopoiesis. Genetic testing confirmed the diagnosis of Autosomal Dominant Common Variable Immunodeficiency Type XII with an NFKB1 mutation. Pre-transplant treatments included monthly IVIG, weekly rituximab, and daily filgrastim, all of which failed to improve her autoimmune neutropenia and hypogammaglobulinemia and failed to reduce her symptomatic burden. Given the patient's young age and refractory autoimmune neutropenia, it was decided to manage them definitively with hematopoietic stem cell transplantation (HSCT). She ultimately underwent allogenic stem cell transplantation (haploidentical, donor was the mother) with 3.96 × 10⁸/kg TNC without immediate post-transplant complications. <b>Conclusions:</b> This article demonstrates a rare case of NFKB1-positive CVID that was successfully treated with HSCT and highlights the importance of considering transplant therapy in younger patients with clinically significant, refractory autoimmune cytopenia.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 5","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12562562/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145388873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acquired Hypolipoproteinemia and Hemophagocytic Lymphohistiocytosis: A Case Series and Review.","authors":"Leo Reap, Ritwick S Mynam, Radhika Takiar, Vincent T Ma","doi":"10.3390/hematolrep17050050","DOIUrl":"10.3390/hematolrep17050050","url":null,"abstract":"<p><strong>Background: </strong>Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening hyperinflammatory syndrome characterized by uncontrolled macrophage activation. Secondary HLH is more common in adults and may be triggered by infection, malignancy, or autoimmune disease. Dyslipidemia, particularly hypolipoproteinemia, has been described but remains underexplored.</p><p><strong>Methods: </strong>We retrospectively reviewed 18 adult HLH cases diagnosed between 2012 and 2020 at two institutions where complete lipid profiles were obtained at or near diagnosis. HLH was defined according to HLH-2004 criteria.</p><p><strong>Results: </strong>Among 18 patients, 17 (94%) had secondary HLH, most commonly idiopathic (<i>n</i> = 5, 28%) or Epstein-Barr virus-associated (<i>n</i> = 3, 17%). Hypolipidemia was nearly universal: all (18/18) had HDL-C < 30 mg/dL, 15/18 (83%) had HDL-C < 20 mg/dL, and 12/18 (67%) had HDL-C < 10 mg/dL. LDL-C was <100 mg/dL in 12/18 (67%), with 6/18 (33%) undetectable. Triglycerides were variably elevated (median 279 mg/dL, range 96-1658 mg/dL). Three representative cases with profound hypolipoproteinemia demonstrated lipid normalization after HLH-directed therapy.</p><p><strong>Conclusions: </strong>Severe reductions in HDL-C and LDL-C appear to accompany HLH and may contribute to its pathophysiology by impairing antioxidant defenses, destabilizing membranes, and potentiating macrophage activation. This case series highlights a consistent association between hypolipoproteinemia and HLH, suggesting potential diagnostic value. However, the observational design and small cohort limit generalizability. Larger prospective studies are needed to clarify mechanisms and evaluate whether full lipid profiling should be incorporated into diagnostic algorithms.</p>","PeriodicalId":12829,"journal":{"name":"Hematology Reports","volume":"17 5","pages":""},"PeriodicalIF":1.2,"publicationDate":"2025-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12563620/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145388907","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}