{"title":"Quality of life of children and adolescents with hemophilia receiving low-dose prophylactic treatment.","authors":"Zahra Badiei, Atefeh Mokhtari, Hamid Farhangi, Maryam Heidarian, Elham Bakhtiari","doi":"10.1007/s12185-025-03969-3","DOIUrl":"https://doi.org/10.1007/s12185-025-03969-3","url":null,"abstract":"<p><strong>Objective: </strong>Compared to episodic treatment, low-dose prophylaxis (LDP) leads to better quality of life (QOL) by decreasing spontaneous bleeding in severe hemophilia A and B. LDP has been used to treat children under 16 years of age in Iran since 2017. The present study assesses the QOL of these patients.</p><p><strong>Methods: </strong>Patients aged 8-16 years with severe hemophilia A and B receiving escalating LDP were included. Patients and parents completed the Hemophilia Quality of Life Questionnaire for Children (Haemo-QOL).</p><p><strong>Results: </strong>Twenty-five children and 15 adolescents were included. The total score for children and parents was 39.7 ± 14.5 and 39.1 ± 15, respectively (p = 0.88). The total score for adolescents and parents was 36.5 ± 15 and 41 ± 15.3, respectively (p = 0.42). The domains of friends and treatment in children and domains of friends and family in parents of children had the highest and lowest scores, respectively. The domains of sport/school and coping in adolescents and the domains of physical health and coping in parents of adolescents had the highest and lowest scores, respectively. The overall QOL of adolescents was better than that of children.</p><p><strong>Conclusion: </strong>LDP resulted in a marked improvement in most domains of QOL. LDP can be appropriate for societies with limited access to factor concentrate.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143779968","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Discovery of a second, distinct development pattern of leukemic conversion from paroxysmal nocturnal hemoglobinuria.","authors":"Junji Tokushige, Kazuki Taoka, Masako Nishikawa, Masahiro Jona, Kumi Nakazaki, Yutaka Yatomi, Mineo Kurokawa","doi":"10.1007/s12185-025-03923-3","DOIUrl":"10.1007/s12185-025-03923-3","url":null,"abstract":"<p><p>The incidence of leukemic conversion during the clinical course of paroxysmal nocturnal hemoglobinuria (PNH) has been reported to be 0.6-2.9%. Such an association is logically linked to the progression of PNH to acute leukemia, especially the M6 subtype of acute myeloid leukemia (AML-M6). In many of these cases (11/26, 42%), leukemic conversion from PNH is associated with development of AML-M6. A literature review including our cases showed that this leukemic conversion from PNH has two distinct development patterns. In type 1, leukemic clones were derived from non-PNH clones in most cases, and the PNH phenotype of erythrocytes disappeared with progression. In one of our cases, however, the patient was diagnosed with concomitant PNH and AML-M6, and leukemic cells were observed alongside CD55-negative and CD59-negative PNH clones. In Type 2 cases such as this one, conversion of PNH is characterized by the coexistence of leukemic cells with PNH clones. Flow cytometry revealed that CD34-positive blast cells were deficient in CD55 and CD59. In Type 2, PNH clones do progress into malignancies, albeit rarely, demonstrating a distinct second development pattern of leukemic conversion from PNH.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"543-546"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11923016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Prognostic value of Glasgow prognostic score in hematological malignancies: a systematic review and meta-analysis.","authors":"Lijun Jiang, Wenting Jin","doi":"10.1007/s12185-025-03935-z","DOIUrl":"10.1007/s12185-025-03935-z","url":null,"abstract":"<p><strong>Objective: </strong>The Glasgow prognostic score (GPS) is used to predict the prognosis of several cancers. This first systematic review and meta-analysis evaluated the role of GPS in predicting overall survival (OS) and progression-free survival (PFS) in patients with hematological malignancies.</p><p><strong>Methods: </strong>Embase, PubMed, CENTRAL, Scopus, and Google Scholar were screened for studies evaluating the prognostic role of GPS in hematological malignancies.</p><p><strong>Results: </strong>Twelve studies were eligible. Meta-analysis showed that patients with GPS of ≥1 and ≥2 had worse OS than those with GPS of 0. We noted that both GPS scores of ≥1 and ≥2 were associated with significantly poor PFS in patients with hematological malignancies. Results remained robust on sensitivity analysis.</p><p><strong>Conclusion: </strong>GPS can be used as a predictor of OS and PFS in patients with hematological malignancies. High GPS scores can lead to a twofold higher risk of poor OS and PFS.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"450-461"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143079787","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Rapid tapering of cyclosporine after allogeneic transplantation for high-risk hematological malignancies.","authors":"Ayumi Gomyo, Shinichi Kako, Masakatsu Kawamura, Shunto Kawamura, Junko Takeshita, Nozomu Yoshino, Yukiko Misaki, Kazuki Yoshimura, Shinpei Matsumi, Yu Akahoshi, Masaharu Tamaki, Machiko Kusuda, Kazuaki Kameda, Hidenori Wada, Koji Kawamura, Miki Sato, Kiriko Terasako-Saito, Shun-Ichi Kimura, Hideki Nakasone, Yoshinobu Kanda","doi":"10.1007/s12185-024-03913-x","DOIUrl":"10.1007/s12185-024-03913-x","url":null,"abstract":"<p><p>Rapid tapering of cyclosporine (CsA) in the early phase after allogeneic transplantation may induce a potent graft-versus-leukemia/lymphoma (GVL) effect. We retrospectively reviewed the outcomes of patients with high-risk hematological malignancies who underwent their first transplantation at our institution. The blood CsA concentration was maintained at around 300 ng/ml. Our planned schedule for tapering CsA in patients without graft-versus-host disease (GVHD) or with limited GVHD was to reduce the dose by 10% per week starting from day 30 for related HSCT or from day 50 for unrelated HSCT. In total, we began tapering CsA in 36, and classified them into 2 an \"On-schedule group\" or \"Delayed group\" based on the timing of starting tapering. The cumulative incidences of grade II-IV acute GVHD overall were 33.8% and 39.4% (P = 0.746) in the On-schedule and Delayed groups. The On-schedule group showed no significant difference in non-relapse mortality, but showed a trend toward a higher relapse rate, resulting in significantly worse overall survival (55.6% vs 72.2% at 1y, P = 0.025) and worse disease-free survival (38.9% vs 66.7% at 1y, P = 0.059). These findings suggest that early CsA tapering after HSCT in high-risk patients was not effective.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"504-510"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143255490","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The medium-fluorescence reticulocyte ratio is an independent predictor of G6PD deficiency neonates.","authors":"Mingliang Rao, Wenna Luo, Baojing Wu","doi":"10.1007/s12185-025-03916-2","DOIUrl":"10.1007/s12185-025-03916-2","url":null,"abstract":"<p><strong>Objective: </strong>G6PD deficiency is a potentially life-threatening condition in neonates presenting with hyperbilirubinemia. This study aims to identify clinical and laboratory predictors of G6PD deficiency in neonates presenting with hyperbilirubinemia.</p><p><strong>Methods: </strong>This was a retrospective study of 227 term neonates admitted to Heyuan People's Hospital from January 2019 to October 2023. Hematological parameters and bilirubin were compared between those with G6PD deficiency and those with normal G6PD.</p><p><strong>Results: </strong>Term neonates with G6PD deficiency had higher levels of total bilirubin, indirect bilirubin, mean corpuscular volume, mean corpuscular hemoglobin, immature reticulocyte fraction, high-fluorescence reticulocyte ratio, medium-fluorescence reticulocyte ratio, and content of reticulocytes than those with normal G6PD, but lower levels of red blood cells, hemoglobin, hematocrit, and low-fluorescence reticulocyte ratio. Medium-fluorescence ratios (OR = 1.291, P = 0.028) independently predicted G6PD deficiency in neonates. The optimal cut-off value for medium-fluorescence ratios was > 18.55%. The area under the curve for diagnosing G6PD deficiency was 0.924 (95% confidence interval: 0.886-0.962, P < 0.0001), with a sensitivity of 82.6% and specificity of 86.2%.</p><p><strong>Conclusion: </strong>MFR emerged as a potentially valuable predictor for G6PD deficiency in neonates.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"526-532"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143005063","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Zanubrutinib in Japanese treatment-naive and relapsed/refractory patients with Waldenström macroglobulinemia and CLL/SLL.","authors":"Koji Izutsu, Takayuki Ishikawa, Kazuyuki Shimada, Kohmei Kubo, Takeshi Kondo, Katsuya Fujimoto, Tomoaki Fujisaki, Shingo Kurahashi, Koji Nagafuji, Rika Sakai, Tatsuro Jo, Tomonori Nakazato, Kazutaka Sunami, Senji Kasahara, Aileen Cohen, Motohisa Takai, Jinhua Zhong, Masahiro Takeuchi","doi":"10.1007/s12185-025-03925-1","DOIUrl":"10.1007/s12185-025-03925-1","url":null,"abstract":"<p><p>Zanubrutinib is a selective second-generation Bruton tyrosine kinase inhibitor approved in various B-cell malignancies globally. The phase 1/2 BGB-3111-111 study evaluated the efficacy and safety of zanubrutinib 160 mg twice daily orally in Japanese patients with treatment-naive or relapsed/refractory mature B-cell malignancies. Here, efficacy results from Part 2 in chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL; n = 19) and Waldenström macroglobulinemia (WM; n = 19), and safety results from Parts 1 (N = 6) and 2 (N = 49) are presented, with the first dose between 30 January, 2020, and 31 October, 2022. As of 10 May, 2023, investigator-assessed overall response rates were 100% (19/19) and 94.7% (18/19) in CLL/SLL and WM, respectively, with median follow-up of 27.9 and 26.8 months; 24-month progression-free survival rates were 71.4% and 100% in treatment-naive and relapsed/refractory CLL/SLL and 83.9% and 100% in treatment-naive and relapsed/refractory WM, respectively. In patients with B-cell malignancies, any-grade treatment-emergent adverse events (TEAEs) occurred in 53 (96.4%) and serious TEAEs in 18 (32.7%). Common TEAEs were platelet count decreased (18.2%), pyrexia (18.2%), COVID-19 (14.5%), and neutrophil count decreased (12.7%). With median follow-up > 2 years, zanubrutinib demonstrated durable efficacy in Japanese patients with CLL/SLL or WM and a favorable safety profile consistent with global phase 3 studies.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"483-493"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11922998/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143440878","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Isatuximab plus pomalidomide and dexamethasone in frail individuals with relapsed/refractory multiple myeloma in Japan.","authors":"Nami Tagami, Michihiro Uchiyama, Kenshi Suzuki, Heigoroh Shirai, Takeshi Seto, Shinsuke Iida","doi":"10.1007/s12185-024-03904-y","DOIUrl":"10.1007/s12185-024-03904-y","url":null,"abstract":"<p><p>This post-marketing surveillance (PMS) assessed the safety and effectiveness of isatuximab plus pomalidomide and dexamethasone (Isa-Pd) for relapsed or refractory multiple myeloma (RRMM) in frail individuals during real-world use in Japan. Data from all individuals with RRMM treated with Isa-Pd in Japan between October 2020 and October 2021 were collected, with follow-up continued up to 12 months after starting Isa-Pd or until discontinuation. In the overall PMS population, 40 participants were classified as frail (33.3%) and 29 as fit/intermediate (24.2%), and 51 had no frailty score (42.5%). Incidence of adverse drug reactions in each group was 77.5%, 65.5%, and 37.3%. In frail versus fit/intermediate participants, bone-marrow suppression occurred in 72.5% versus 44.8%, infectious diseases in 17.5% versus 10.3%, and infusion-related reactions in 7.5% versus 3.5%. Heart failure occurred in one participant with no frailty score. The rates of overall response and very good partial response or better were higher (p = 0.101) in fit/intermediate participants (56.0% and 36.0%) than in frail participants (38.5% and 18.0%). Rates of treatment discontinuation due to disease progression were similar between groups. These findings support the safety and effectiveness of Isa-Pd for frail individuals with RRMM in real-life settings in Japan.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"476-482"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11923021/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142894290","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Steroid-resistant nephrotic syndrome due to renal-limited thrombotic microangiopathy and membranous nephropathy after allogeneic hematopoietic stem cell transplantation successfully treated with calcineurin inhibitors.","authors":"Shinri Okada, Masashi Nishikubo, Yoshimitsu Shimomura, Nobuhiro Hiramoto, Keisuke Osaki, Shigeo Hara, Tadakazu Kondo, Takayuki Ishikawa","doi":"10.1007/s12185-025-03930-4","DOIUrl":"10.1007/s12185-025-03930-4","url":null,"abstract":"<p><p>Transplantation-associated thrombotic microangiopathy (TMA) is a severe complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with high mortality. As calcineurin inhibitors (CNIs) reportedly contribute to TMA via drug-induced endothelial injury, treatment of TMA often involves CNI discontinuation or dose reduction. However, renal-limited TMA, defined as biopsy-proven renal TMA without the classical triad (hemolytic anemia, thrombocytopenia, and organ damage), has rarely been reported after allo-HSCT, and its optimal management remains unknown. Herein, we report three cases of renal-limited TMA after allo-HSCT that presented with nephrotic syndrome, in which renal biopsy showed TMA and concurrent membranous nephropathy. All patients were refractory to glucocorticoid monotherapy and the addition of CNIs led to complete remission of nephrotic syndrome. Renal-limited TMA after allo-HSCT may present as nephrotic syndrome with distinct pathophysiological features from renal-limited TMA in non-allo-HSCT recipients. Previous reports have suggested that renal-limited TMA after allo-HSCT is associated with renal graft-versus-host disease, and thus optimizing immunosuppressive therapy, including CNI treatment, may be useful. CNI treatment may be an option even in the presence of renal-limited TMA after allo-HSCT accompanied by concurrent membranous nephropathy.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"553-559"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143065526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Luana Conte, Giorgio De Nunzio, Roberto Lupo, Donato Cascio, Marco Cioce, Elsa Vitale, Chiara Ianne, Ivan Rubbi, Massimo Martino, Letizia Lombardini, Aurora Vassanelli, Simonetta Pupella, Simona Pollichieni, Nicoletta Sacchi, Fabio Ciceri, Stefano Botti
{"title":"Raising awareness may increase the likelihood of hematopoietic stem cell donation: a nationwide survey using artificial intelligence.","authors":"Luana Conte, Giorgio De Nunzio, Roberto Lupo, Donato Cascio, Marco Cioce, Elsa Vitale, Chiara Ianne, Ivan Rubbi, Massimo Martino, Letizia Lombardini, Aurora Vassanelli, Simonetta Pupella, Simona Pollichieni, Nicoletta Sacchi, Fabio Ciceri, Stefano Botti","doi":"10.1007/s12185-024-03894-x","DOIUrl":"10.1007/s12185-024-03894-x","url":null,"abstract":"<p><strong>Background: </strong>In Italy, the demand for allogeneic transplantation exceeds the number of compatible donors in the Italian Bone Marrow Donor Registry (IBMDR). This study aimed to explore the knowledge, beliefs, opinions, values, and feelings of the Italian population regarding stem cell donation.</p><p><strong>Methods: </strong>An online survey was shared via social media. Respondents were retrospectively identified as registered on the IBMDR (donor group) or never registered (non-donor group). Statistical analyses confirmed the relationship between knowledge level and willingness to donate. Six machine learning classifiers were trained using questionnaire responses to predict the probability of IBMDR registration.</p><p><strong>Results: </strong>A total of 1518 respondents participated. Characteristics identified in the non-donor group were a lower level of knowledge regarding donation needs (51.7% vs 24.4%, p < 0.001) and negative feelings such as fear (Z = - 2.2642, p = 0.02), confusion (Z = 4.4821, p < 0.001), and uncertainty (Z = 3.3425, p < 0.001). Higher knowledge predicted a greater likelihood of IBMDR enrollment. Machine learning analysis showed an AUC ranging from 0.65 to 0.81, depending on the classifier.</p><p><strong>Conclusions: </strong>The results underscore the need to improve strategies to raise awareness and knowledge of stem cell donation among the Italian population.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"511-525"},"PeriodicalIF":1.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142818095","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}