International Journal of Hematology最新文献

Detection of Talaromyces marneffei in a HIV-infected patient from peripheral blood observation through Sysmex XN WNF/WNR channels. 通过 Sysmex XN WNF/WNR 通道从外周血观察结果中检测出一名艾滋病毒感染者体内的马拉氏菌。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-14 DOI: 10.1007/s12185-024-03855-4

Qiang Meng, Jin Deng, Yang Fu

引用次数: 0

Improved survival among elderly patients with aggressive adult T-cell leukemia/lymphoma: Impact of mogamulizumab-containing chemotherapy. 提高侵袭性成人T细胞白血病/淋巴瘤老年患者的生存率：含莫干单抗化疗的影响。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-10 DOI: 10.1007/s12185-024-03857-2

Miki Hashimoto, Takeharu Kato, Kenichi Yokota, Hikaru Sakamoto, Makiko Horai, Masataka Taguchi, Hidehiro Itonaga, Shinya Sato, Maki Baba, Koji Ando, Yoshitaka Imaizumi, Yasushi Miyazaki

{"title":"Improved survival among elderly patients with aggressive adult T-cell leukemia/lymphoma: Impact of mogamulizumab-containing chemotherapy.","authors":"Miki Hashimoto, Takeharu Kato, Kenichi Yokota, Hikaru Sakamoto, Makiko Horai, Masataka Taguchi, Hidehiro Itonaga, Shinya Sato, Maki Baba, Koji Ando, Yoshitaka Imaizumi, Yasushi Miyazaki","doi":"10.1007/s12185-024-03857-2","DOIUrl":"10.1007/s12185-024-03857-2","url":null,"abstract":"Due to the poor prognosis of adult T-cell leukemia/lymphoma (ATL), new treatments are urgently needed, especially for elderly patients with aggressive ATL. The anti-CCR4 antibody drug mogamulizumab (MOG) has been approved for the treatment of untreated ATL. To analyze the impact of MOG on elderly patients, we conducted a retrospective analysis of patients aged 70 years and older with aggressive ATL diagnosed at our institution between 2015 and 2021. Among 32 patients, including those who received best supportive care, the median survival time (MST) and 2-year overall survival (OS) rate were 14.6 months (range, 0.0-83.7), and 34.7% [95% confidence interval (CI), 18.2-51.9], respectively, which were better than outcomes in our previous study. The MST and 2-year OS for patients treated with MOG-containing chemotherapy were 18.1 months (range, 4.0-83.7) and 45.0% (95%CI, 23.1-64.7), respectively, demonstrating clear improvement. Adverse events observed with MOG-containing treatment, such as myelosuppression and skin rash, were similar to those reported previously. Univariate analysis identified comorbidity as a predictor of poor outcomes, but not intensity of MOG-containing treatment, suggesting a different mechanism of action than that of classical chemotherapy. Our study suggests that MOG-containing treatments are an option for elderly patients with ATL.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"694-704"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142400227","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}

引用次数: 0

Long-term safety profile of sutimlimab in adult Japanese patients with cold agglutinin disease. 日本成年冷凝集素病患者使用 sutimlimab 的长期安全性概况。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-14 DOI: 10.1007/s12185-024-03842-9

Yoshitaka Miyakawa, Eriko Sato, Yoshiaki Ogawa, Jun-Ichi Nishimura, Masashi Nishimi, Osamu Kawaguchi, Sayaka Tahara, Masaki Yamaguchi

{"title":"Long-term safety profile of sutimlimab in adult Japanese patients with cold agglutinin disease.","authors":"Yoshitaka Miyakawa, Eriko Sato, Yoshiaki Ogawa, Jun-Ichi Nishimura, Masashi Nishimi, Osamu Kawaguchi, Sayaka Tahara, Masaki Yamaguchi","doi":"10.1007/s12185-024-03842-9","DOIUrl":"10.1007/s12185-024-03842-9","url":null,"abstract":"Sutimlimab, a complement inhibitor, has recently been approved in Japan for treating cold agglutinin disease (CAD). We report the safety and efficacy of sutimlimab in Japanese patients with CAD who completed a global phase 3 clinical trial (CARDINAL/CADENZA: 26-week treatment with 1-2 years of open-label extension [OLE] periods) and subsequently participated in the Japanese OLE study. Patients with a recent history of blood transfusion (CARDINAL, n = 3) and those without (CADENZA, n = 4) were analyzed (71.4% female; median [range] baseline age: 70 [46-83] years). For CARDINAL/CADENZA, the treatment duration (median [range]) was 140.9 (104.9-157.3) weeks, and the cessation period was 70 (61-133) weeks. For the Japanese OLE study, the treatment duration was 47.1 (15.1-49.1) weeks. Three (42.9%) patients experienced treatment-related and treatment-emergent adverse events (TEAEs): injection site erythema, cystitis bacterial, viral infection, and blood pressure increased during CARDINAL/CADENZA. One (14.3%) patient experienced one treatment-related TEAE (urinary tract infection) during the Japanese OLE study. One patient died of renal failure, considered unrelated to sutimlimab, that was exacerbated by hepatorenal syndrome due to liver cirrhosis and bacterial peritonitis, in addition to CKD. Hemoglobin and bilirubin levels improved during treatment but deteriorated after withdrawal and recovered on retreatment. Sutimlimab was well tolerated over a median of 3.8 years, with no new safety concerns identified during retreatment.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"656-664"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11588873/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142464640","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}

引用次数: 0

Clinical characteristics in adolescents and young adults with polycythemia vera and essential thrombocythemia in Japan. 日本多发性红细胞症和原发性血小板增多症青少年患者的临床特征。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-28 DOI: 10.1007/s12185-024-03862-5

Yuka Sugimoto, Keiki Nagaharu, Eiko Ohya, Kohshi Ohishi, Isao Tawara, Tomoki Ito, Akihiko Gotoh, Mika Nakamae, Fumihiko Kimura, Michiaki Koike, Keita Kirito, Hideho Wada, Kensuke Usuki, Takayuki Tanaka, Takehiko Mori, Satoshi Wakita, Toshiki I Saito, Akiko M Saito, Kazuya Shimoda, Toshiro Kurokawa, Akihiro Tomita, Yoko Edahiro, Yoshinori Hashimoto, Hitoshi Kiyoi, Koichi Akashi, Itaru Matsumura, Katsuto Takenaka, Norio Komatsu

{"title":"Clinical characteristics in adolescents and young adults with polycythemia vera and essential thrombocythemia in Japan.","authors":"Yuka Sugimoto, Keiki Nagaharu, Eiko Ohya, Kohshi Ohishi, Isao Tawara, Tomoki Ito, Akihiko Gotoh, Mika Nakamae, Fumihiko Kimura, Michiaki Koike, Keita Kirito, Hideho Wada, Kensuke Usuki, Takayuki Tanaka, Takehiko Mori, Satoshi Wakita, Toshiki I Saito, Akiko M Saito, Kazuya Shimoda, Toshiro Kurokawa, Akihiro Tomita, Yoko Edahiro, Yoshinori Hashimoto, Hitoshi Kiyoi, Koichi Akashi, Itaru Matsumura, Katsuto Takenaka, Norio Komatsu","doi":"10.1007/s12185-024-03862-5","DOIUrl":"10.1007/s12185-024-03862-5","url":null,"abstract":"We report the first large-scale retrospective cohort study on adolescent and young adult (AYA) polycythemia vera (PV) and essential thrombocythemia (ET) in Japan, a subgroup analysis using Japanese multicenter registry data (JSH-MPN-R18). This study included patients with PV (n = 31) or ET (n = 141) aged 20 to 39 years at the initial visit. Hemorrhage-free survival (HFS) was better in AYA ET than in non-AYA ET (5-year HFS: 100% vs. 88.6%, p < 0.01), which might be attributed to differences in antithrombotic treatment rates between AYA and non-AYA patients. Although thrombosis-free survival did not differ statistically, the percentage of venous thrombotic events (TEs) among total TEs was higher in AYA compared to non-AYA PV and ET in Japan (26.0% vs. 6.0%, p < 0.01), but much lower than figures reported in European or US cohorts. Cytoreductive therapy (CRT) was administered to 25.8% of AYA patients with PV and 43.3% of AYA patients with ET, and the reason was usually unrelated to high risk of thrombosis. These results could be used to develop a more appropriate strategy for managing PV and ET in the Japanese AYA population.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"684-693"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142521857","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}

引用次数: 0

Assay variables and early clinical evaluation of low-angle light scattering for platelet function analysis. 用于血小板功能分析的低角度光散射的检测变量和早期临床评估。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-22 DOI: 10.1007/s12185-024-03859-0

Galina S Svidelskaya, Vera P Sorkina, Anastasia A Ignatova, Evgeniya A Ponomarenko, Aleksandr V Poletaev, Elena A Seregina, Valentin A Manuvera, Pavel A Zharkov, Igor V Mindukshev, Stepan Gambaryan, Mikhail A Panteleev

{"title":"Assay variables and early clinical evaluation of low-angle light scattering for platelet function analysis.","authors":"Galina S Svidelskaya, Vera P Sorkina, Anastasia A Ignatova, Evgeniya A Ponomarenko, Aleksandr V Poletaev, Elena A Seregina, Valentin A Manuvera, Pavel A Zharkov, Igor V Mindukshev, Stepan Gambaryan, Mikhail A Panteleev","doi":"10.1007/s12185-024-03859-0","DOIUrl":"10.1007/s12185-024-03859-0","url":null,"abstract":"Introduction: The recently developed platelet aggregation technique based on low-angle light scattering (LaSca) in diluted platelet-rich plasma (PRP) requires only a small sample volume and provides information about platelet aggregation and shape change. This study aimed to investigate the influence of preanalytical and analytical variables and to validate the method in a real-life pediatric hematology hospital setting.Methods: Platelet aggregation was induced by ADP in diluted PRP in the presence of 2 mM calcium at 23 °C. The study included healthy adults (n = 30), healthy children (n = 20), and pediatric patients with suspected or diagnosed platelet function abnormalities (n = 25).Results: The assay parameters were stable for at least 3 h after isolation of PRP and were sensitive to plasma dilution in the range of 2-8%. The initial aggregation velocity was significantly reduced in pediatric patients compared with healthy children (p < 0.05). ADP-induced light transmission amplitude was moderately correlated with LaSca amplitude of aggregation in healthy children (p = 0.52, p < 0.05) but not in pediatric patients.Conclusions: We standardized the protocol for platelet aggregation assessment by LaSca and characterized the influence of preanalytical and analytical variables on it.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"717-724"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142464629","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}

引用次数: 0

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-08 DOI: 10.1007/s12185-024-03858-1

Radu Chiriac

引用次数: 0

Adult-onset severe paroxysmal cold hemoglobinuria after COVID-19 successfully treated with sutimlimab. 使用 sutimlimab 成功治疗 COVID-19 后成人发作的严重阵发性冷性血红蛋白尿。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-09-20 DOI: 10.1007/s12185-024-03851-8

Keijiro Sato, Yusuke Nakamura, Ryosuke Hara, Ryuhei Kayama, Shunya Sunohara, Sayaka Okajima, Shintaro Kazama, Wataru Komaba, Ryuto Ishikawa, Masahiko Sumi, Hikaru Kobayashi

{"title":"Adult-onset severe paroxysmal cold hemoglobinuria after COVID-19 successfully treated with sutimlimab.","authors":"Keijiro Sato, Yusuke Nakamura, Ryosuke Hara, Ryuhei Kayama, Shunya Sunohara, Sayaka Okajima, Shintaro Kazama, Wataru Komaba, Ryuto Ishikawa, Masahiko Sumi, Hikaru Kobayashi","doi":"10.1007/s12185-024-03851-8","DOIUrl":"10.1007/s12185-024-03851-8","url":null,"abstract":"Paroxysmal cold hemoglobinuria (PCH) is a form of cold autoimmune hemolytic anemia characterized by the presence of the Donath-Landsteiner antibody, which triggers complement-mediated intravascular hemolysis when the body temperature changes from cold to warm. PCH occurs primarily in children as a rare, self-limiting disease following viral infections. In contrast, adult-onset PCH is very rare and associated with a diverse range of underlying conditions, which complicates its management and treatment. We describe a case of adult-onset PCH following COVID-19, effectively managed with a single dose of sutimlimab, a selective classical complement pathway inhibitor. This intervention was performed during a life-threatening hemolytic crisis, at a time requiring swift decision-making when specific tests to differentiate from other hemolytic anemias were not readily available. This case illustrates the potential of using a single dose of sutimlimab to manage life-threatening hemolytic crises in PCH, highlighting the significance of inhibiting the classical complement pathway.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"735-742"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142286348","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}

引用次数: 0

Ruxolitinib for steroid-refractory chronic graft-versus-host disease: Japanese subgroup analysis of REACH3 study. Ruxolitinib治疗类固醇难治性慢性移植物抗宿主病：REACH3研究的日本亚组分析。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-03 DOI: 10.1007/s12185-024-03850-9

Souichi Shiratori, Kentaro Fukushima, Yasushi Onishi, Noriko Doki, Tatsunori Goto, Masaya Okada, Hirohisa Nakamae, Yoshinobu Maeda, Koji Kato, Takayuki Ishikawa, Tadakazu Kondo, Masako Toyosaki, Takashi Ikeda, Naoyuki Uchida, Akio Maki, Fumika Shimada, Takeshi Tajima, Tommaso Stefanelli, Takanori Teshima

{"title":"Ruxolitinib for steroid-refractory chronic graft-versus-host disease: Japanese subgroup analysis of REACH3 study.","authors":"Souichi Shiratori, Kentaro Fukushima, Yasushi Onishi, Noriko Doki, Tatsunori Goto, Masaya Okada, Hirohisa Nakamae, Yoshinobu Maeda, Koji Kato, Takayuki Ishikawa, Tadakazu Kondo, Masako Toyosaki, Takashi Ikeda, Naoyuki Uchida, Akio Maki, Fumika Shimada, Takeshi Tajima, Tommaso Stefanelli, Takanori Teshima","doi":"10.1007/s12185-024-03850-9","DOIUrl":"10.1007/s12185-024-03850-9","url":null,"abstract":"Ruxolitinib, a Janus kinase (JAK1-JAK2) inhibitor, has demonstrated safety and efficacy in patients with graft-versus-host disease (GvHD). This phase 3 randomized trial (REACH3) evaluated the efficacy and the safety of ruxolitinib 10 mg twice daily compared with investigator-selected best available therapy (BAT) in a subgroup of Japanese patients (n = 37) with steroid-refractory or dependent (SR/D) chronic GvHD. At data cut-off, treatment was ongoing in 17 patients and discontinued in 20. The overall response rate (complete or partial) at week 24 was greater with ruxolitinib than BAT (50% vs. 20%; odds ratio, 4.13 [95% CI, 0.90-18.9]). The best overall response rate (complete or partial response at any time point up to week 24) was higher with ruxolitinib than BAT (68.2% vs. 46.7%; odds ratio, 2.69 [95% CI, 0.66-10.9]). Ruxolitinib led to longer median failure-free survival than BAT (18.6 months vs. 3.7 months; hazard ratio, 0.34; [95% CI, 0.14-0.85]). The most common grade ≥ 3 adverse events up to week 24 were anemia (ruxolitinib: 22.7%; BAT: 6.7%) and pneumonia (22.7% and 20.0%, respectively). Ruxolitinib showed a higher response rate and improvement in failure-free survival in Japanese patients with SR/D chronic GvHD, with a safety profile consistent with the overall study population.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"705-716"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11588829/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142365174","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}

引用次数: 0

Long-term safety and efficacy of ropeginterferon alfa-2b in Japanese patients with polycythemia vera. 罗京干扰素 alfa-2b 对日本多发性红细胞症患者的长期安全性和有效性。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-10-03 DOI: 10.1007/s12185-024-03846-5

Keita Kirito, Yuka Sugimoto, Akihiko Gotoh, Katsuto Takenaka, Michiko Ichii, Tadaaki Inano, Shuichi Shirane, Masafumi Ito, Oleh Zagrijtschuk, Albert Qin, Hiroaki Kawase, Toshiaki Sato, Norio Komatsu, Kazuya Shimoda

{"title":"Long-term safety and efficacy of ropeginterferon alfa-2b in Japanese patients with polycythemia vera.","authors":"Keita Kirito, Yuka Sugimoto, Akihiko Gotoh, Katsuto Takenaka, Michiko Ichii, Tadaaki Inano, Shuichi Shirane, Masafumi Ito, Oleh Zagrijtschuk, Albert Qin, Hiroaki Kawase, Toshiaki Sato, Norio Komatsu, Kazuya Shimoda","doi":"10.1007/s12185-024-03846-5","DOIUrl":"10.1007/s12185-024-03846-5","url":null,"abstract":"Ropeginterferon alfa-2b (ropegIFN), a new-generation interferon-based agent, has been approved in Japan for patients with polycythemia vera (PV) who are ineligible for or respond inadequately to conventional treatment. However, long-term outcomes with ropegIFN in Japanese patients have not been reported. This extension of a phase 2 study of ropegIFN in Japanese patients with PV aimed to determine its long-term safety/efficacy, and changes over time in JAK2 V617F allele burden. Here, we report data from the phase 2 study and subsequent extension over a period of 36 months. The primary endpoint was the complete hematologic response (CHR) maintenance rate without phlebotomy (hematocrit value < 45% without phlebotomy during the previous 12 weeks, platelet count ≤ 400 × 109/L, and white blood cell count ≤ 10 × 109/L). The CHR maintenance rates were 8/27 (29.6%), 18/27 (66.7%), and 22/27 (81.5%) at 12, 24, and 36 months, respectively. No thrombotic or hemorrhagic events occurred. The median allele burden change from baseline was - 74.8% at 36 months. All patients experienced adverse events; 25/27 (92.6%) experienced adverse drug reactions (ADRs), but no serious ADRs or deaths occurred. This interim analysis demonstrated the safety and efficacy of ropegIFN over 36 months in Japanese patients with PV.","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"675-683"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11588802/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142365173","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}

引用次数: 0

Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings. 罗米波司汀治疗慢性特发性血小板减少性紫癜的长期安全性和有效性。

IF 1.7 4区医学

International Journal of Hematology Pub Date : 2024-12-01 Epub Date: 2024-09-29 DOI: 10.1007/s12185-024-03847-4

Naoshi Obara, Shigeki Hatanaka, Yukie Tsuji, Koji Higashi

{"title":"Long-term safety and effectiveness of romiplostim for chronic idiopathic thrombocytopenic purpura in real-world settings.","authors":"Naoshi Obara, Shigeki Hatanaka, Yukie Tsuji, Koji Higashi","doi":"10.1007/s12185-024-03847-4","DOIUrl":"10.1007/s12185-024-03847-4","url":null,"abstract":"Idiopathic thrombocytopenic purpura (ITP), an autoimmune hematologic disorder characterized by severe platelet count reduction, can be treated with romiplostim. However, post-marketing safety and effectiveness data for romiplostim in Japan are scarce. This prospective, observational, post-marketing Specified Use-Results Survey evaluated the real-world safety and effectiveness of romiplostim for 2 years. All patients treated with romiplostim during the survey period were eligible. Of the 1622 patients in the safety analysis set, 94.08% (1526/1622) had chronic ITP. The mean single dose of romiplostim was stable after 12 weeks and remained < 6 μg/kg in approximately 70% of patients until 104 weeks. Within 2 years, 14.92% of patients discontinued romiplostim because of adverse events, while 6.47% discontinued because of suspected adverse drug reactions. In contrast, 14.00% of patients discontinued romiplostim because of symptom improvement. Before romiplostim initiation, platelet count was < 2.0 × 104/µL in 60.54% of patients, and the mean platelet count was 2.84 ± 5.76 × 104/µL. Platelet count was 9.19 ± 13.01 × 104/µL after 4 weeks, and remained between 10.34 ± 10.72 and 12.38 ± 12.63 × 104/µL from 8 to 104 weeks of treatment. No specific concerns were revealed regarding the safety and effectiveness of romiplostim in chronic ITP; the findings demonstrated a favorable risk-benefit balance for romiplostim in this population. Trial registration: UMIN000047864 ( www.umin.ac.jp/ctr ).","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"665-674"},"PeriodicalIF":1.7,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11588964/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142346187","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}

引用次数: 0