Journal of hematology最新文献

{"title":"Prevalence of Viral Infections and Serious Complications in Pediatric Hematopoietic Stem Cell Transplant Patients: A Ten-Year Single-Institution Retrospective Study.","authors":"Calvin E Lau, David J DiTullio, Holly Wilhalme, LaVette Bowles, Theodore B Moore, Satiro N De Oliveira","doi":"10.14740/jh1376","DOIUrl":"10.14740/jh1376","url":null,"abstract":"<p><strong>Background: </strong>Pediatric hematopoietic stem cell transplant (pHSCT) patients are at risk for many life-threatening post-transplant complications, notably relapse, graft-versus-host disease (GvHD), and infection.</p><p><strong>Methods: </strong>This retrospective study reviewed 10 years of pHSCT at a single institution, assessing for risk factors for post-transplantation viral infections (herpes simplex virus (HSV), varicella-zoster virus (VZV), Epstein-Barr virus (EBV), cytomegalovirus (CMV), human herpes virus 6 (HHV6), adenovirus (ADNV), and human polyoma virus 1 (BK virus)), and characterizing adverse infectious outcomes.</p><p><strong>Results: </strong>Overall, 139 patients received 151 transplants. With respect to graft source: 73 (48.3%) were bone marrow, 67 (44.4%) umbilical cord blood (UCB), and 11 (7.2%) peripheral blood stem cells (PBSCs). Forty-one deaths occurred, for an overall mortality rate of 29.5%. The overall incidence of post-transplant viral infections was 47.7% (n = 72). Incidence of post-transplant infection varied by virus type: 3.97% HSV, 0.67% VZV, 3.97% EBV, 24.5% CMV, 14.5% HHV6, 12.6% ADNV, and 12.6% BK virus. Viral encephalitis, though relatively uncommon, was primarily caused by HHV6 and more common in UCB transplants. Overall, cell source and donor source were identified with statistically significant correlation to both risk of infection and mortality.</p><p><strong>Conclusions: </strong>Post-transplant viral infection remains as a serious adverse event in pediatric patients, and thus prospective studies should be performed to implement early intervention and more aggressive treatment in select high-risk patients. More studies specifically addressing infection risks in cord blood transplants and risk factors for post-transplant viral encephalitis are warranted.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 1","pages":"1-13"},"PeriodicalIF":1.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11809597/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399274","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":"Increased Transferrin Concentrations Are Not Associated With Thrombosis in People Living at High Altitude.","authors":"Ricardo Amaru, Josef Prchal, Tomas Ganz, Xu Zhang, Daniela Paton, Mireya Carrasco, Emma Mancilla, Victor R Gordeuk","doi":"10.14740/jh1388","DOIUrl":"10.14740/jh1388","url":null,"abstract":"<p><strong>Background: </strong>Bolivian Andean Aymara highlanders, living at 4,000 m for 14,000 years, have evolved genetic adaptations to hypoxia. These include <i>EGLN1</i> encoding prolyl hydroxylase 2 (PHD2), a regulator of transferrin transcription. Transferrin level increases in hypoxia and iron deficiency. Contrasting reports indicate that elevated transferrin is associated with experimentally induced thrombosis in mice undergoing short-stay at high altitude, but with decreased thrombosis in a congenital disorder of hypoxia-sensing.</p><p><strong>Methods: </strong>A retrospective study was conducted in people living at high altitude (3,650 - 4,150 m). We analyzed serum transferrin concentration and thrombosis history in Aymara patients with high-altitude erythrocytosis (n = 149, median age 55 years, female gender 30%, iron deficiency 23%) or high-altitude anemia (n = 137, median age 43 years, female gender 86%, iron deficiency 57%).</p><p><strong>Results: </strong>The median transferrin concentration was 339 mg/dL in erythrocytosis patients versus 310 mg/dL in anemia patients (P = 0.037); it was 367 mg/dL in iron deficient versus 312 mg/dL in iron replete patients (P < 0.001). Thrombosis history was present in 13% of erythrocytosis and 8% of anemia patients (P = 0.25) and was present in 16% of iron deficient and 7% of iron replete patients (P = 0.017). After adjustment for erythrocytosis and iron deficiency in multivariate regression analysis, the mean (95% confidence interval) transferrin concentration was 277 (237 - 316) mg/dL in 30 patients with thrombosis history versus 324 (306 - 341) mg/dL in 256 patients without thrombosis history (P = 0.018). Similar trends occurred for the subgroups of arterial thrombosis history (P = 0.044) and venous thrombosis history (P = 0.22).</p><p><strong>Conclusions: </strong>In individuals with extreme environmental hypoxia, we found no evidence that increased transferrin is associated with increased thrombosis history. Rather, we observed a trend to decreased thrombosis history with increased transferrin levels.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"14 1","pages":"20-25"},"PeriodicalIF":1.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11809596/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399308","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":"Retrospective Study of CD20 Expression Loss in Relapsed or Refractory B-Cell Non-Hodgkin Lymphoma.","authors":"Joseph P Marshalek, Xin Qing, Marcin Dragan, Sarah Tomassetti","doi":"10.14740/jh1341","DOIUrl":"10.14740/jh1341","url":null,"abstract":"<p><strong>Background: </strong>CD20-targeted therapies are widely used in the management of B-cell lymphomas. Re-treatment with CD20-directed agents is common; however, previous research has demonstrated loss of CD20 expression at relapse in a subset of patients.</p><p><strong>Methods: </strong>In this single-center retrospective cohort of 243 patients, CD20 analysis was performed by immunohistochemistry (IHC) and/or flow cytometry at diagnosis and at relapse if a biopsy was performed.</p><p><strong>Results: </strong>Of 109 patients with relapsed or refractory B-cell lymphoma, 59 patients with CD20-positive lymphoma at diagnosis underwent a biopsy at relapse for a total of 76 biopsies across all relapses. The rate of partial or complete CD20 expression loss was 11.9% (four patients with partial loss, three patients with complete loss). There were four cases of CD20 loss at first relapse (three IHC, one flow cytometry), two at second relapse (one IHC, one IHC and flow cytometry), and one at fifth relapse (IHC and flow cytometry). CD20 antigen escape was observed in marginal zone lymphoma, follicular lymphoma, and diffuse large B-cell lymphoma (DLBCL). All patients with CD20 expression loss previously received rituximab. Among patients with CD20 antigen escape, 85.7% had stage IV disease, and median overall survival after CD20 loss was 4 months. In the group of five patients with indolent lymphoma and CD20 expression loss, three patients (60%) had concurrent transformation to high-grade lymphoma.</p><p><strong>Conclusions: </strong>This study, which reinforces the importance of repeating a biopsy at relapse before implementing CD20-directed therapy, is particularly relevant given the widespread use of rituximab along with the emerging significance of CD20-targeted bispecific antibodies in the management of B-cell lymphomas.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 6","pages":"268-277"},"PeriodicalIF":1.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650554/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854333","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":"Sequential Use of Prednisolone and Cyclosporine Is Effective in the Management of Immunotherapy-Related Hemolytic Anemia.","authors":"Cherian Verghese, Niravkumar Brahmbhatt, Ghulam Ghous, Kapil Meleveedu, Nancy Vander Velde, Mark Hunter, Hari Parameshwaran","doi":"10.14740/jh1344","DOIUrl":"10.14740/jh1344","url":null,"abstract":"<p><p>Immune checkpoint inhibitors (CPIs) can cause immune-related organ dysfunctions, including nephritis, pneumonitis, thyroiditis, hepatitis, colitis and more rarely hematological toxicities like immune-related autoimmune hemolytic anemia (irAIHA). Very few cases of irAIHA associated with immunotherapy have been reported, and treatment protocols remain unclear. This is partly because not all irAIHA cases are Coomb's test positive. Causes of anemia in cancer patients undergoing treatment with chemotherapy with or without immunotherapeutic agents can also be multiple. This makes it difficult to initially diagnose irAIHA, especially when CPIs are used concurrently with chemotherapy. Once alternate causes have been ruled out, a treatment plan for irAIHA is initiated based on grade of the anemia. Grade 1-2 irAIHA cases are managed with supportive interventions. However, cessation of therapy is recommended for life-threatening (grade 4) toxicity, severe (grade 3) toxicity that is recurring, or moderate (grade 2) toxicity that does not resolve with appropriate treatment for 3 months. Management of irAIHA usually involves methylprednisolone for 2 - 4 weeks with a slow taper after hemoglobin has normalized. But some cases do not respond to steroids alone and require cessation of immunotherapy or selecting alternate immunosuppressive agents. We report a protocol for treatment of grade 4 irAIHA secondary to programmed death protein 1 (PD-1) blocker pembrolizumab.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 6","pages":"285-289"},"PeriodicalIF":1.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650556/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854394","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":"Optimizing Falling Drop Hemoglobin Method by Comparing Capillary Versus Venous Blood and Determining the Stability of the Copper Sulfate Solution.","authors":"Amogh Chariyamane, Tim R Randolph","doi":"10.14740/jh1337","DOIUrl":"10.14740/jh1337","url":null,"abstract":"<p><strong>Background: </strong>Anemia is a global health issue that affects over 1 billion people and contributes to maternal mortality and birth defects. Low-resource, tropical areas face a dual challenge: high prevalence of anemia and inability to access affordable testing methods. The falling drop hemoglobin method has been developed by our lab to quantify hemoglobin concentration and assess anemia by timing the descent of venous blood in a column of copper sulfate solution, without using electricity or batteries. This research aimed to optimize the falling drop hemoglobin method by evaluating the use of capillary blood to reduce within sample variance and assessing copper sulfate stability to determine shelf life in expected working conditions.</p><p><strong>Methods: </strong>The falling drop hemoglobin method was performed on both venous and capillary blood samples collected directly from the fingertip by dispensing 44 µL of blood in a copper sulfate column. A microhematocrit was performed on the venous blood sample and converted mathematically to a hemoglobin level to serve as the standard. Copper sulfate stability was assessed for 32 weeks among three solutions: solution prepared fresh on day of testing, solution incubated at room temperature, and solution incubated at 37.7 °C.</p><p><strong>Results: </strong>Capillary blood yielded higher average descent times and higher standard deviations than venous blood. Collecting precisely 44 µL of capillary blood proved challenging and impractical. In copper sulfate stability testing, freshly prepared solution yielded the highest average descent time. A one-way analysis of variance (ANOVA) test and Tukey's honestly significant difference (HSD) post hoc testing revealed no significant difference between mean descent times of freshly prepared and 37.7 °C solutions (P = 0.26) and between room temperature and 37.7 °C solutions (P = 0.64), but a significant difference between freshly prepared and room temperature solutions (P = 0.04).</p><p><strong>Conclusions: </strong>This study found that capillary blood did not present a more accurate alternative to venous blood in the falling drop hemoglobin test, and copper sulfate did not degrade over 32 weeks at 37.7 °C. This lends support for the current use of venous blood in the test, and for use of copper sulfate solution in tropical climates, where the test is most necessary, with a shelf life of at least 32 weeks.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 6","pages":"261-267"},"PeriodicalIF":1.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650553/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854294","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":"Spontaneous Regression of Plasmablastic Lymphoma Associated With Methotrexate After Withdrawal.","authors":"Takayuki Goto, Kiichi Hatano, Nobuhiro Kanemura, Hiroki Makita, Hideko Goto","doi":"10.14740/jh1361","DOIUrl":"10.14740/jh1361","url":null,"abstract":"<p><p>Plasmablastic lymphoma (PBL) is a malignant lymphoma with poor prognosis that occurs in immunocompromised and elderly patients. We describe the case of a 75-year-old woman with PBL as a methotrexate-associated lymphoproliferative disorder (MTX-LPD). She presented with multiple oral ulcers and mass-like shadows in the lung fields. Biopsy of the oral ulcer revealed medium to large irregular round monotypic B cells positive for cluster of differentiation (CD)138, CD79a, immunoglobulin λ, and Epstein-Barr virus-encoded small ribonucleic acid <i>in situ</i> hybridization, and PBL was diagnosed. The patient showed negative results for human immunodeficiency virus and had a history of taking MTX for rheumatoid arthritis, suggesting MTX-LPD. Following discontinuation of MTX, the oral ulcers resolved 1 month later without recurrence, and lung lesions decreased in size over time. Because MTX-LPD can take the form of PBL and may resolve with MTX withdrawal alone, therapeutic interventions should be carefully considered. While PBL is typically highly aggressive and requires prompt treatment, MTX-LPD cases can sometimes resolve without further treatment, depending on the clinical course. However, in cases where the disease shows progression or when spontaneous regression does not occur, additional therapeutic interventions may be necessary to manage the disease effectively.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 6","pages":"290-294"},"PeriodicalIF":1.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650557/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854395","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":"Methionine Synthase Reductase A66G Variant in Pediatric Acute Lymphoblastic Leukemia Patients.","authors":"Elrashed B Yasin, Haitham M H Qutob, Raed Alserihi","doi":"10.14740/jh1360","DOIUrl":"10.14740/jh1360","url":null,"abstract":"<p><strong>Background: </strong>Methionine synthase reductase, which is encoded by the methionine synthase reductase (<i>MTRR</i>) gene, plays a crucial role in the methylation reactions and the production of DNA and its epigenetic processes. There was a correlation between the <i>MTRR</i> (A66G) polymorphism and the likelihood of developing acute lymphoblastic leukemia (ALL). This study was carried out to investigate the correlation among pediatric ALL cases.</p><p><strong>Methods: </strong>Within the participant population of this case-control study, there were 86 individuals who had been diagnosed with ALL, and there were also 150 healthy persons who acted as the control group. To determine the <i>MTRR</i> (A66G) polymorphism, DNA was first extracted and then observed through the use of real-time polymerase chain reaction.</p><p><strong>Results: </strong>The results of the flow cytometry analysis showed that the prevalence of B-cell ALL (B-ALL) was much higher than that of T-cell ALL (T-ALL), which accounted for only 20 cases (23.3%). Upon comparing the hematological parameters of ALL subtypes in patients with T-ALL, it was discovered that there was a statistically significant higher mean total white blood count (P < 0.0005) and mean blast percentage (P = 0.050). Upon examination, it was discovered that both of these figures were much higher than the average. In accordance with the results of the molecular analysis, the occurrence of the <i>MTRR</i> homozygous GG genotype was found to be considerably lower in the patients' group (4.65%) than in the control group (20.67%). However, the <i>MTRR</i> homozygous AA and heterozygous AG were nearly similar in the two groups. The risk of acute lymphoblastic leukemia and <i>MTRR</i> genotypes, on the other hand, exhibited a correlation that was not statistically significant (P = 0.082).</p><p><strong>Conclusions: </strong>The study's findings showed that among pediatric ALL patients, the <i>MTRR</i> A66G polymorphism was not linked to an increased risk of ALL.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 6","pages":"278-284"},"PeriodicalIF":1.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650555/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854292","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":"A Case of Fulminant Cerebral Edema Leading to Death After Chimeric Antigen Receptor T-Cell Therapy.","authors":"Katherine Hickmann, Rachel DiLeo, Kathleen Faringer, Chelsea Peterson, Cyrus Khan, Yazan Samhouri","doi":"10.14740/jh1367","DOIUrl":"10.14740/jh1367","url":null,"abstract":"<p><p>Chimeric antigen receptor (CAR) T-cell therapy has transformed treatment of refractory B-cell malignancies; however, treatment puts patients at risk for side effects secondary to the amplified immune response it induces. Fulminant cerebral edema (FCE) is one of the rarest, yet most devastating side effects following CAR T-cell therapy. Due to this rarity, FCE has not been well characterized and the risk factors associated with its development are not fully understood. Here, we present a case of a 42-year-old male who passed away from FCE following CAR T-cell infusion with the primary goal to better understand which patients are at higher risk of developing FCE before and after infusion.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 6","pages":"295-299"},"PeriodicalIF":1.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11650552/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142854289","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":"Successful Treatment of Idiopathic Multicentric Castleman Disease With Rash as the Initial Symptom Using a Rituximab-Based Regimen.","authors":"Li Zhu, Yi Liu, Fang Yu, Xue Jiao Yin, Qiu Mei Yao, Hai Tao Meng, Liang Shun You, Hong Yan Tong","doi":"10.14740/jh1313","DOIUrl":"10.14740/jh1313","url":null,"abstract":"<p><p>Idiopathic multicentric Castleman disease (iMCD) is a rare lymphoproliferative disorder characterized by enlarged lymph nodes and systemic inflammation, often involving multiple organ dysfunction. However, cutaneous involvement in iMCD is rare and heterogeneous, and studies on the treatment of iMCD with skin involvement are scarce. Here, we present a rare case of iMCD with prominent facial skin involvement, which showed significant improvement with rituximab-based regimen treatment.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 5","pages":"245-249"},"PeriodicalIF":1.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11526581/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142566996","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":"Siltuximab in Idiopathic Multicentric Castleman Disease: Real-World Experience.","authors":"Ciprian Jitaru, Argyris Symeonidis, Sorina Badelita, Eirini Katodritou, Andrei Colita, Anastasia Mpanti, Anamaria Bancos, Bogdan Tigu, Petra Rotariu, Laura Urian, Ioana Rus, Delia Dima, Anca Bojan, Marc Damian, Vasiliki Labropoulou, Mihai Stefan Muresan, Despina Fotiou, Bogdan Fetica, Bobe Petrushev, Angela Dascalescu, Dimitra Dalampira, Sanda Buruiana, Catalin Constantinescu, Mihnea Zdrenghea, Meletios A Dimopoulos, Ciprian Tomuleasa, Evangelos Terpos","doi":"10.14740/jh1343","DOIUrl":"10.14740/jh1343","url":null,"abstract":"<p><strong>Background: </strong>Castleman disease (CD) is a very rare, non-malignant lymphoproliferative disorder that can be classified as unicentric or multicentric (MCD). MCD is associated with systemic symptoms, including organ dysfunction due to cytokine dysregulation, primarily interleukin-6 (IL-6). The anti-IL-6 monoclonal antibody siltuximab is recommended as a frontline treatment for idiopathic MCD (iMCD), but real-world data on its use in routine clinical practice are limited. This study aimed to assess disease response and survival outcomes in patients with iMCD treated with siltuximab therapy in real-world settings in Greece and Romania.</p><p><strong>Methods: </strong>This retrospective cohort study included adult patients with iMCD treated with siltuximab in clinical practice across Greece and Romania between January 2017 and December 2022. The primary endpoint was overall response rate and secondary endpoints included survival and safety outcomes. Response assessments were performed according to the Castleman Disease Collaborative Network guidelines. Patients were followed until death, loss to follow-up or study conclusion (October 2023).</p><p><strong>Results: </strong>Forty-eight patients with iMCD were included in the study. Mean age at baseline was 65 years, with significant age differences between patients from Greece (74 years) and Romania (54 years). The majority of patients were male (68.8%) and received one prior line of therapy (75%). Patients included in the study received a median of nine cycles of siltuximab. Response data were available for 38 patients. The overall response to siltuximab was 71.1%, with 55.3% of patients achieving a complete response, and 15.8% a partial response. The estimated overall survival rate at 3 years was 74% and the median survival was 123 months. The most common adverse events (> 5%) included elevated liver enzymes, anxiety, allergic reactions and nausea/diarrhea. Serious adverse events were experienced by 16.7% of the patients.</p><p><strong>Conclusions: </strong>Our results suggest that siltuximab-based therapy is effective in treating iMCD in real-world settings in Greece and Romania. To our knowledge, this study represents the largest real-world analysis of siltuximab in European patients with iMCD so far.</p>","PeriodicalId":15964,"journal":{"name":"Journal of hematology","volume":"13 5","pages":"207-215"},"PeriodicalIF":1.3,"publicationDate":"2024-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11526577/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142566974","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}