Clinical hematology international最新文献

{"title":"Secondary failure of platelet recovery after allogeneic hematopoietic stem cell transplantation in pediatric acquired aplastic anemia: Incidence, risk factors, and clinical outcomes.","authors":"Yue Shang, Yuanyuan Ren, Lipeng Liu, Xin Wang, Chaoqian Jiang, Xia Chen, Yang Wan, Aoli Zhang, Xiaoyan Zhang, Fang Liu, Li Zhang, Yumei Chen, Yao Zou, Xiaojuan Chen, Wenyu Yang, Xiaofan Zhu, Ye Guo","doi":"10.46989/001c.171012","DOIUrl":"https://doi.org/10.46989/001c.171012","url":null,"abstract":"<p><p>Secondary failure of platelet recovery (SFPR) poses a significant risk for life-threatening complications following hematopoietic stem cell transplantation (HSCT). However, the incidence, clinical outcomes, and risk factors associated with SFPR in the context of allogeneic HSCT (allo-HSCT) for pediatric patients with acquired aplastic anemia (AA) remain unclear. We conducted a retrospective review of 86 consecutive acquired AA children who underwent allo-HSCT at our center from December 2017 to November 2023. The 1-year cumulative incidence of SFPR was found to be 11.6% (95% confidence interval [CI]: 6.2%-21.1%). The median time to SFPR was 2 months (range: 1-5 months) post-transplantation. To clarify the risk factors for SFPR, 9 SFPR cases and 69 non-SFPR cases were further analyzed. Multivariable analyses revealed that age, pre-transplantation body mass index (BMI) and a history of grade Ⅲ-Ⅳ acute graft-versus-host disease (aGVHD) were the most significant predictors for SFPR post-transplantation (p < 0.05). Additionally, we found that the 2-year overall survival rate was significantly lower in children with SFPR compared to those without (74.1% vs. 96.7%; p < 0.005). Ultimately, 4 patients achieved full platelet count recovery, with 2 patients becoming transfusion-independent. However, 1 patient still requires periodic platelet transfusions, and 2 patients unfortunately died.Among the 6 patients who received thrombopoietin receptor agonist therapy, an overall response rate of 66.6% was observed, suggesting the potential efficacy of this treatment approach in managing SFPR. This study underscores the need for vigilant monitoring of platelet recovery in pediatric HSCT recipients and highlights the need for further research into the etiology of SFPR to improve clinical outcomes.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"67-76"},"PeriodicalIF":0.0,"publicationDate":"2026-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13632644/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149120169","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":"Elranatamab in relapsed/refractory multiple myeloma patients on hemodialysis: A real-world case series.","authors":"Hikmettullah Batgi, Fatoş Dilan Köseoğlu, Denis Çetin, Semih Başcı, Sinem Namdaroğlu, Nur Akad Soyer","doi":"10.46989/001c.171328","DOIUrl":"https://doi.org/10.46989/001c.171328","url":null,"abstract":"<p><strong>Background: </strong>Renal impairment is a frequent and adverse prognostic factor in multiple myeloma (MM), with dialysis-dependent patients largely excluded from pivotal trials of B-cell maturation antigen (BCMA)-directed therapies. Elranatamab, a BCMA×CD3 bispecific antibody, has demonstrated significant efficacy in relapsed/refractory multiple myeloma (RRMM); however, data in end-stage renal disease are extremely limited. We report real-world outcomes of four dialysis-dependent RRMM patients treated with elranatamab.</p><p><strong>Results: </strong>Patients with a median age of 67 years (range 61-80) had received a median of six prior lines of therapy (range 4-7). All were refractory to proteasome inhibitors, immunomodulatory drugs, and anti-CD38 therapy, and none had prior BCMA-directed treatment. Baseline glomerular filtration rate ranged from 5 to 20 mL/min, and all patients were dialysis-dependent. At data cutoff, all four patients were evaluable for response. Two patients achieved complete response (CR), one achieved very good partial response, and one achieved partial response, resulting in an overall response rate of 100%. Treatment was ongoing in all four patients. Cytokine release syndrome (CRS) occurred in one patient (grade 1) and was self-limited. No immune effector cell-associated neurotoxicity syndrome (ICANS) events were observed. One patient developed severe pneumonia requiring intensive care unit admission; treatment was temporarily interrupted and successfully resumed without step-up dosing, maintaining CR. Hematologic toxicity was limited, with grade-4 lymphopenia in one patient. No dialysis-related complications or unexpected toxicities were observed. All patients received infection prophylaxis after hemodialysis, including trimethoprim-sulfamethoxazole, valacyclovir, and levofloxacin, with intravenous immunoglobulin administered.</p><p><strong>Conclusion: </strong>In this real-world case series of dialysis-dependent RRMM patients, elranatamab demonstrated high response rates, low incidence of CRS, absence of neurotoxicity, and manageable infectious complications. These findings support the feasibility and clinical activity of elranatamab in patients undergoing hemodialysis. Larger prospective studies are warranted to confirm these encouraging results.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"60-66"},"PeriodicalIF":0.0,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13625312/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149120164","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":"Transplant outcomes after intensive chemotherapy or venetoclax plus azacitidine in acute myeloid leukemia: a multicenter retrospective study in Japan.","authors":"Hiroyuki Muranushi, Junya Kanda, Tadakazu Kondo, Yasuyuki Arai, Ikue Okamura-Shiki, Takashi Ikeda, Hiroki Amagase, Takehiro Okuda, Daishi Nakagawa, Takeshi Maeda, Yasunori Ueda, Kazunori Imada, Kazuhiro Yago, Tomoharu Takeoka, Mutsumi Okada, Yasuko Miyahara, MItsumasa Watanabe, Nobuyoshi Arima, Toshiyuki Kitano, Masakatsu Hishizawa, Katsuhiro Io, Satoko Oka, Kosuke Asagoe, Takashi Akasaka, Kouhei Yamashita, Akifumi Takaori-Kondo","doi":"10.46989/001c.170281","DOIUrl":"10.46989/001c.170281","url":null,"abstract":"<p><p>The present retrospective multicenter study in Japan evaluated the role of venetoclax plus azacitidine (V/A) before first allogeneic hematopoietic stem cell transplantation (SCT) in 144 transplant-eligible acute myeloid leukemia (AML). Thirty-seven patients received V/A prior to SCT, mainly as salvage therapy for refractory and adverse-risk AML. Patients were classified into four groups according to remission status at SCT and V/A use. The 1-year overall survival (OS), cumulative incidence of relapse (CIR) and non-relapse mortality (NRM) of those who achieved complete remission (CR) with intensive chemotherapy (IC) were 93%, 5% and 4%, respectively. The 1-year OS, CIR and NRM of those who were refractory/intolerant to IC and achieved CR with V/A were 93%, 7% and 7%, respectively. The 1-year OS, CIR and NRM of those who were refractory to IC and proceeded directly to SCT were 52%, 54% and 13%, respectively. The 1-year OS, CIR and NRM of those who were refractory/intolerant to IC and refractory to V/A were 41%, 39% and 29%, respectively. Overall, use of V/A prior to SCT salvaged some AML patients who were refractory/intolerant to IC, and the transplant outcomes of these patients were favorable. Further studies are needed to optimize treatment for AML prior to SCT.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"48-59"},"PeriodicalIF":0.0,"publicationDate":"2026-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13602127/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149042497","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":"Healthcare professionals' knowledge and attitudes toward the microbiome and fecal microbiotherapy or microbiota transfer in hematology and oncology: an international survey.","authors":"Marion Larue, Giorgia Battipaglia, Hendrik Poeck, Riccardo Masetti, Christoph Stein-Thoeringer, Jaime Sanz, Daniela Weber, Annalisa Ruggeri, Zinaida Peric, Raphael Duarte, Mohamad Mohty, Florent Malard","doi":"10.46989/001c.166241","DOIUrl":"10.46989/001c.166241","url":null,"abstract":"<p><p>The gut microbiome has emerged as a critical factor influencing cancer progression, treatment response, and complications in hematology and oncology. Fecal microbiota transfer (FMT) is being explored as a novel biotherapeutic strategy, yet the knowledge and attitudes of healthcare professionals towards its clinical use remain poorly defined. We conducted an international survey of 152 healthcare professionals, including hematologists, oncologists, transplant specialists, and others. Most respondents were affiliated with academic centers (66.0%) and practiced primarily in hematology (48.0%) or transplantation (34.0%). Awareness of the microbiome's clinical relevance was widespread, but perceptions of microbiome profiling and FMT remained cautious. One-third of participants were in favor of routine microbiome profiling, while half considered it promising but still investigational. Direct clinical experience with FMT was limited: only 20.4% had prescribed it and 14.5% had referred patients. Nonetheless, over half indicated they would consider adopting FMT in the near future. The main reported barriers to broader use included limited regulatory approval (63.8%), logistical or supply challenges (57.9%), and physician skepticism or lack of knowledge (51.3%). Despite these challenges, more than 70% of respondents expressed strong interest in further education, standardized guidelines, and training opportunities to support safe and effective implementation. This survey highlights a strong enthusiasm regarding microbiome-based interventions in cancer and transplantation care. While current clinical use remains limited, healthcare professionals recognize the therapeutic potential of FMT and call for structured education, multidisciplinary collaboration, and clear regulatory frameworks to enable its integration into routine practice.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"41-47"},"PeriodicalIF":0.0,"publicationDate":"2026-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13619122/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149077124","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":"Delphi expert consensus on phlebotomy limitations and treatment challenges in <i>HFE</i>-related hemochromatosis.","authors":"Jeremy Shearman, Domenico Girelli, Dominik J Schaer, Sant-Rayn Pasricha, Kris V Kowdley, John K Olynyk, Gregory J Kato, Sonya Abraham, Soraya Benchikh El Fegoun, Charlotte Pollet, Uta Merle","doi":"10.46989/001c.166769","DOIUrl":"10.46989/001c.166769","url":null,"abstract":"<p><p><i>HFE</i>-related hemochromatosis (HC) is caused by hepcidin dysregulation and is characterized by excessive iron absorption and accumulation in the liver, heart, and endocrine glands, which leads to complications including arthropathy, cirrhosis, and hepatocellular carcinoma. Standard of care (SoC) typically involves phlebotomy, with iron chelators used less frequently. However, these therapies may cause side effects and negatively impact quality of life, making them troublesome for some patients. Using the Delphi methodology, a survey was developed to identify unmet needs and clinical challenges within the current therapeutic landscape of HC, and to establish consensus statements for its management. Consensus was defined as ≥75% agreement. Thirty-two HC specialists from Europe, Australia and the USA responded to the survey. After three rounds of survey refinement, final consensus statements were compiled. The Delphi process identified key unmet needs in HC, including lack of alternatives to phlebotomy, persistent symptoms, and burden related to the use of current SoC. The consensus process helped establish definitions for high phlebotomy treatment burden, intolerance, and suboptimal response to phlebotomy. The process also identified the subgroup of patients overly burdened by phlebotomy. Patient-reported outcomes were considered key to assessing phlebotomy's impact, although they are rarely measured in clinical practice. The Delphi study highlighted the limitations of phlebotomy, with respondents identifying a high unmet need for patients who cannot be managed with or do not tolerate this approach. The study suggested exploring alternative therapy options for patients who experience high treatment burden or intolerance to phlebotomy.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"28-40"},"PeriodicalIF":0.0,"publicationDate":"2026-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13524205/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148852133","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":"Iron deficiency without anemia: An underrecognized and undertreated clinical condition with special focus on colorectal cancer.","authors":"Naga P Raja, Nagapavani Kandagari","doi":"10.46989/001c.165952","DOIUrl":"10.46989/001c.165952","url":null,"abstract":"<p><p>Iron deficiency is the most common micronutrient deficiency worldwide, affecting approximately 2 billion individuals. Iron deficiency without anemia (IDNA), the stage in which iron stores are depleted but hemoglobin remains within the reference range, is estimated to affect approximately 1-2 billion individuals, although the precise prevalence varies by population, ferritin threshold, and inflammatory status. IDNA remains underrecognized because diagnostic evaluation often focuses on hemoglobin concentrations rather than iron stores. A growing body of evidence demonstrates that IDNA is associated with clinically significant fatigue, impaired cognitive function, anxiety, reduced exercise capacity, and diminished quality of life. Randomized controlled trials and meta-analyses of iron repletion in iron-deficient non-anemic cohorts demonstrate clinically meaningful reductions in fatigue. Emerging observational data have also identified an association between iron deficiency and colorectal neoplasia in selected higher-risk populations such as older adults, men, and postmenopausal women. However, the magnitude of this risk and the role of routine endoscopic evaluation in patients with IDNA remain incompletely defined. This narrative review examines the epidemiology, pathophysiology, origins of under-recognition, clinical manifestations, diagnostic approach, treatment, association with colorectal cancer, and future directions for IDNA in adults.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"18-27"},"PeriodicalIF":0.0,"publicationDate":"2026-08-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13449291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148690489","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":"Incidental perioperative detection of congenital methemoglobinemia: A prospective case series from a South Indian referral centre.","authors":"Pooja Pushparaj, Aboobacker Mohamed Rafi","doi":"10.46989/001c.164967","DOIUrl":"10.46989/001c.164967","url":null,"abstract":"<p><strong>Background: </strong>Congenital methemoglobinemia is a rare inherited disorder caused by impaired reduction of methemoglobin to functional hemoglobin, resulting in functional hypoxia despite normal arterial oxygen tension. Due to its often-mild clinical phenotype, diagnosis is frequently delayed or made incidentally.</p><p><strong>Methods: </strong>This prospective case series of surgical patients was conducted at a tertiary care center in South India over a one-year period. Patients incidentally diagnosed with methemoglobinemia during perioperative or acute medical evaluation were included. Diagnosis was established using arterial blood gas analysis with co-oximetry and confirmed biochemically by erythrocyte NADH-cytochrome b5 reductase activity.</p><p><strong>Results: </strong>Ten surgical patients were identified, all originating from a single geographic region. Methemoglobin levels ranged from 14.4% to 29.1%. Most patients were asymptomatic or mildly symptomatic, with diagnosis prompted by refractory hypoxemia and a characteristic saturation gap. Enzyme assays confirmed Type I congenital methemoglobinemia in all cases. Management was largely supportive, with methylene blue reserved for symptomatic individuals.</p><p><strong>Conclusions: </strong>This case series highlights incidental perioperative detection as a key diagnostic opportunity and suggests possible regional clustering of congenital methemoglobinemia. Increased clinical awareness and targeted screening may improve the diagnosis of this underrecognized condition.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"10-17"},"PeriodicalIF":0.0,"publicationDate":"2026-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13428680/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148665053","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":"Natural killer cells from patients relapsing on daratumumab therapy express an exhausted-associated phenotype.","authors":"Katrine Fladeland Iversen, Line Nederby, Thomas Lund, Trine Andreasen Leth, Torben Plesner","doi":"10.46989/001c.163405","DOIUrl":"10.46989/001c.163405","url":null,"abstract":"<p><p>The monoclonal antibody daratumumab can induce antibody-dependent cellular cytotoxicity (ADCC) via CD16 expressed by natural killer (NK) cells. Prior studies have shown that the number of NK cells decreased after initiation of daratumumab treatment. We used flow cytometry to evaluate the expression of immune checkpoint receptors on NK cells from patients with newly diagnosed multiple myeloma (NDMM) compared to myeloma patients progressing during treatment with daratumumab (DRMM). In accordance with prior studies, we found that the percentage of NK cells was significantly lower in the DRMM group compared to the NDMM group. In addition, the percentage of the cytotoxic CD56dim NK cell subset was lower in the DRMM group, and a lower portion of these NK cells expressed CD16, the receptor mediating ADCC. There was no difference in the expression of the inhibitory receptor PD-1, but the NK cells from DRMM patients expressed an exhausted-associated phenotype with a lower expression of the stimulatory receptor DNAM-1 and a higher expression of the inhibitory receptor TIGIT. Dysfunctional NK cells have been observed in other patients progressing on daratumumab. Our data show that patients progressing on DARA-containing regimens display NK cells with an inhibitory-skewed phenotype. Whether this reflects a driver, biomarker, or consequence of resistance requires further functional and longitudinal investigation.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 3","pages":"1-9"},"PeriodicalIF":0.0,"publicationDate":"2026-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13348759/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148427489","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":"Current landscape of academic and point of care CAR-T cell therapy in low-middle income countries.","authors":"Sofía I Quezada-Ramírez, José E Montelongo-Cepeda, Gabriel A González-López, Rosario Salazar-Riojas, Dalila M Alvarado-Navarro, Andrés Gómez-De León, David Gómez-Almaguer","doi":"10.46989/001c.163505","DOIUrl":"10.46989/001c.163505","url":null,"abstract":"<p><p>Chimeric antigen receptor T-cell (CAR-T) therapy has emerged as a transformative treatment for patients with relapsed or refractory hematologic malignancies, offering new possibilities beyond conventional transplantation. Since their approval in high-income countries, CAR-T products have demonstrated remarkable efficacy; however, their implementation in low- and middle-income countries (LMICs) remains limited due insufficient infrastructure, complex regulatory requirements and high costs. In some countries, like Mexico, these barriers are particularly pronounced, with limited access to clinical-grade vectors, early phase clinical research programs, and lack of government and private sector infrastructure and funding opportunities. Facing these challenges, there is a substantial patient population with B-cell acute lymphoblastic leukemia, multiple myeloma, or diffuse large B-cell lymphoma, who could benefit from CAR-T therapy. Advances in point-of-care manufacturing within academic settings -particularly the use of closed, semi-automated systems- have demonstrated feasibility in LMICs, by reducing costs and simplifying procedures, while maintaining product quality. These approaches may provide a viable alternative to commercial products, with the potential to lower the economic burden and increase accessibility. Regulatory innovation with the establishment of expert multidisciplinary oversight committees, will also be critical to ensure safe implementation. Ultimately, CAR-T therapy in LMICs is both a scientific and public health opportunity, and its integration into healthcare systems will depend on collaborative strategies that address financial, logistical, and policy barriers. By highlighting our experience, this review underscores the importance of developing locally adapted solutions to expand access to advanced cellular therapies in resource-constrained settings.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 2","pages":"63-72"},"PeriodicalIF":0.0,"publicationDate":"2026-06-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13321489/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148371439","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":"Comparison of patient, hospitalization and center characteristics from the EMMY centers, IFM centers, and all multiple myeloma centers in France.","authors":"Decaux Olivier, Ronan Garlantezec, Aurore Perrot, Margaret Macro, Bruno Royer, Karim Belhadj Merzoug, Laure Vincent, Thomas Chalopin, Laurent Frenzel, Arthur Bobin, Mohamad Mohty, Cyrille Touzeau, Patricia Zunic, Ronan LE Calloch, Florence Lachenal, Murielle Roussel, Valentine Richez-Olivier, Joel Fleury, Elena Loppinet, Isabelle Leduc, Mourad Tiab, Jacques Delaunay, Jean Gutnecht, Selim Corm, Fabienne Bazin, Pippa McKelvie-Sebileau, Sandrine Rollet, Heloise Mouton, Laurent Gibel, Chanaz Louni, Ariane Boumendile, Cyrille Hulin","doi":"10.46989/001c.161361","DOIUrl":"10.46989/001c.161361","url":null,"abstract":"<p><strong>Background: </strong>Multiple myeloma (MM) is an incurable hematological malignancy with increasing prevalence. While randomized controlled trials have demonstrated survival improvements, they underrepresent older and comorbid patients. Real-world data from observational cohorts are therefore essential.</p><p><strong>Objective: </strong>To assess the comparability of patient, hospitalization and center characteristics for patients treated in centers included in the French Epidemiology of Multiple MYeloma (EmmY) cohort with those treated in centers of the French-speaking Intergroupe Francophone du Myélome (IFM) network (including EmmY) and those treated in all centers treating MM in France between 2017-2023.</p><p><strong>Methods: </strong>We analyzed French national hospital discharge data (PMSI) to identify adults hospitalized with MM (ICD-10 C90) between August 2017 and December 2023. Patient demographics, comorbidities, hospitalization characteristics, and center profiles were compared between the three cohorts using standardized differences.</p><p><strong>Results: </strong>We identified 69,276 patients (including 50,243 IFM and 33,785 EmmY), 1,871,369 hospitalizations (including 1,484,288 IFM and 1,022,462 EmmY), and 323 centers (including 118 IFM and 70 EmmY). EmmY centers were larger and treated a higher mean proportion of MM patients (2.7%) per center than IFM (2.4%) and all MM centers (1.3%). No clinically meaningful differences were observed between EmmY and IFM centers regarding patient age, sex, comorbidities, treatment patterns, or hospitalization outcomes.</p><p><strong>Conclusions: </strong>The EmmY cohort is highly comparable to patients treated in specialized MM centers and those within the broader French MM population, supporting its validity as a robust real-world data source. It is well-suited for evaluating treatment pathways and outcomes in real-world MM populations, including patients underrepresented in randomized controlled trials.</p>","PeriodicalId":93942,"journal":{"name":"Clinical hematology international","volume":"8 2","pages":"50-58"},"PeriodicalIF":0.0,"publicationDate":"2026-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13218629/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148140219","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}