{"title":"Response to initial treatment with glucocorticoids in TAFRO syndrome and implications for secondary treatment.","authors":"Ryutaro Tominaga, Kento Umino, Seina Honda, Daizo Yokoyama, Atsuto Noguchi, Shuka Furuki, Shunsuke Koyama, Rui Murahashi, Hirotomo Nakashima, Kazuki Hyodo, Shin-Ichiro Kawaguchi, Yumiko Toda, Daisuke Minakata, Masahiro Ashizawa, Chihiro Yamamoto, Kaoru Hatano, Kazuya Sato, Ken Ohmine, Shin-Ichiro Fujiwara, Yoshinobu Kanda","doi":"10.1007/s12185-025-03933-1","DOIUrl":"https://doi.org/10.1007/s12185-025-03933-1","url":null,"abstract":"<p><p>The study aimed to investigate the therapeutic effect of various initial treatments incorporating glucocorticoid (GC) in TAFRO syndrome (thrombocytopenia, anasarca, fever, reticulin fibrosis, and organomegaly). Cases of TAFRO syndrome up to November 2023 were retrospectively collected. Overall survival (OS) and resistance to GC therapy were assessed, with resistance analyzed based on the time to the next treatment or death (TTNTD). The study included 95 patients, including 5 diagnosed at our hospital. OS did not differ significantly between patients who received GC monotherapy and those who had a second-line therapy added within 2 weeks (100-day OS rate: 86.6% vs. 77.7%; p = 0.338). Moreover, 100-day OS did not differ between patients who received GC pulse therapy within 2 weeks and those who did not (77.5% vs. 93.1%, p = 0.129). In multivariate analyses, pretreatment severity score ≥ 8 (hazard ratio [HR], 2.99; 95% confidence interval [CI] 1.05-8.50) and platelets ≥ 6.9 × 10^4/µL (HR, 2.26; 95% CI 1.01-5.02) were significantly associated with shorter TTNTD. Additional second-line or GC pulse therapy provided no advantage in the hyperacute phase. Higher severity scores and platelet values may predict resistance to GC therapy.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143065525","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":"Long-term safety and efficacy of fostamatinib in Japanese patients with primary immune thrombocytopenia.","authors":"Masataka Kuwana, Yoshiaki Tomiyama","doi":"10.1007/s12185-025-03924-2","DOIUrl":"https://doi.org/10.1007/s12185-025-03924-2","url":null,"abstract":"<p><p>Fostamatinib had superior efficacy to a placebo and acceptable safety profiles for at least 1 year in a phase 3 study of Japanese patients with primary immune thrombocytopenia. Here, we report the 3-year safety and efficacy of fostamatinib in that study. Data from 33 patients who received at least one dose of fostamatinib were analyzed. A platelet response > 50,000/µL (at two consecutive visits at least 28 days apart while receiving fostamatinib) was achieved in 16 patients (48%). The median total duration of a platelet response > 50,000/µL was 589 (range: 106-1003) days. Gastrointestinal disorders, such as diarrhea, hypertension, and hepatic enzyme elevation, were the most common fostamatinib-related adverse events. Most events occurred within 12 weeks of treatment. No thromboembolisms, treatment-related infections, or moderate or severe treatment-related bleeding events were observed. In summary, this extension study of a clinical trial found a sustained platelet response without new safety signals during 3-year treatment with fostamatinib.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143052423","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}
Lin-Sen Feng, Hui-Yuan Li, Ai Tang, Meng-Li Xu, San-Bin Wang
{"title":"Venetoclax and azacitidine in combination with homoharringtonine, cytarabine, and aclarubicin for salvage therapy of relapsed/refractory T cell acute lymphoblastic leukemia.","authors":"Lin-Sen Feng, Hui-Yuan Li, Ai Tang, Meng-Li Xu, San-Bin Wang","doi":"10.1007/s12185-025-03915-3","DOIUrl":"https://doi.org/10.1007/s12185-025-03915-3","url":null,"abstract":"<p><strong>Background: </strong>The treatment of relapsed/refractory T cell acute lymphoblastic leukemia (R/R T-ALL) is a significant challenge in hematologic oncology, and no standard salvage treatment plan exists. Both Chinese and international clinical guidelines recommend combination chemotherapy including venetoclax.</p><p><strong>Methods: </strong>Efficacy and safety of venetoclax, azacitidine, homoharringtonine, cytarabine, and aclarubicin (VA-HAA) combination therapy were retrospectively analyzed in 3 patients with R/R T-ALL at the Department of Hematology, 920th Hospital of the Joint Logistics Support Force, Chinese People's Liberation Army.</p><p><strong>Results: </strong>The chemotherapy resulted in CR/CRi with negative flow MRD in all 3 patients. Quantitative negative conversion was achieved in 2 patients with fusion genes, and the frequency of monoclonal TCR gene rearrangements was significantly reduced in 1 patient. All patients received stem cell rescue after the chemotherapy. Hematologic toxicity may be manageable, with a median of 24 days for complete recovery of neutrophils (ANC) and 36 days for partial recovery of platelets. There were no major bleeding events or chemotherapy-related deaths.</p><p><strong>Conclusion: </strong>VA-HAA may be an effective and safe salvage treatment for R/R T-ALL, and prospective clinical trials are needed to verify its specific clinical efficacy.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143023352","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":"https://doi.org/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":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-21","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":"Post-transplant TKIs for Ph+ ALL: practices to date and clinical significance.","authors":"Satoshi Nishiwaki, Seitaro Terakura, Takanobu Morishita, Tatsunori Goto, Yuichiro Inagaki, Kotaro Miyao, Nobuaki Fukushima, Daiki Hirano, Naoyuki Tange, Shingo Kurahashi, Yachiyo Kuwatsuka, Masanobu Kasai, Hiroatsu Iida, Kazutaka Ozeki, Masashi Sawa, Tetsuya Nishida, Hitoshi Kiyoi","doi":"10.1007/s12185-025-03917-1","DOIUrl":"https://doi.org/10.1007/s12185-025-03917-1","url":null,"abstract":"<p><p>Post-transplant tyrosine kinase inhibitors (TKIs) show promise in preventing relapse after allogeneic hematopoietic cell transplantation (allo-HCT) for Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ALL). However, their real-world use and efficacy remain unclear. A comprehensive study across seven centers included Ph+ALL patients who underwent allo-HCT between 2002 and 2022. Post-transplant TKIs were administered in 28% of patients (49 of 173 transplanted in complete remission): 7% as prophylaxis during complete molecular remission (CMR), and 21% in response to measurable residual disease (MRD) positivity. Median first post-transplant TKI duration was 13.7 months for the prophylactic group and 4.0 months for the MRD-triggered group. Prophylactic TKIs appear particularly beneficial for patients not in CMR at allo-HCT, showing a trend towards higher 5-year relapse-free survival (RFS) compared to those not receiving prophylactic TKIs (100% vs. 73%; P = 0.11). Significant RFS differences were observed between the prophylactic, non-TKI, and MRD-triggered groups. However, patients with white blood cell counts <15000/µl at diagnosis and no additional chromosomal abnormalities-an MRD-triggered high efficacy cluster-demonstrated comparable 5-year RFS regardless of TKI strategy (100% vs. 85% vs. 80%; P = 0.87). This cluster highlights the potential effectiveness of MRD-triggered TKI administration in select low-risk patients, suggesting tailored TKI strategies based on risk factors.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143005061","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":"Clinical practice guidelines for management of disseminated intravascular coagulation in Japan 2024. Part 3: solid cancers and vascular abnormalities.","authors":"Yoshinobu Seki, Kohji Okamoto, Takayuki Ikezoe, Kazuma Yamakawa, Seiji Madoiwa, Toshimasa Uchiyama, Hidesaku Asakura, Shinya Yamada, Shin Koga, Hiroyasu Ishikura, Takashi Ito, Toshiaki Iba, Mitsuhiro Uchiba, Kaoru Kawasaki, Noriaki Kawano, Satoshi Gando, Shigeki Kushimoto, Yuichiro Sakamoto, Toshihisa Tamura, Kenji Nishio, Mineji Hayakawa, Takeshi Matsumoto, Toshihiko Mayumi, Hideo Wada","doi":"10.1007/s12185-024-03912-y","DOIUrl":"https://doi.org/10.1007/s12185-024-03912-y","url":null,"abstract":"<p><p>This study discusses disseminated intravascular coagulation (DIC) associated with solid cancers and various vascular abnormalities, both of which generally exhibit chronic DIC patterns. Solid cancers are among the most significant underlying diseases that induce DIC. However, the severity, bleeding tendency, and progression of DIC vary considerably depending on the type and stage of the cancer, making generalization difficult. Moreover, during the process of creating these guidelines, it became apparent that despite solid cancers being a major underlying condition for DIC, there is a lack of high-quality research on DIC associated with solid cancers. Nevertheless, we developed recommendations for clinical questions (CQs) regarding the use of heparin and recombinant thrombomodulin. Additionally, statements concerning these five questions were provided. DIC associated with various vascular abnormalities, is characterized by hyperfibrinolytic activity and linked to underlying conditions such as aortic aneurysm, aortic dissection, vasculitis syndromes, and vascular malformations. These conditions must always be considered differential diagnoses when unexplained thrombocytopenia or bleeding tendencies are observed. Although no evidence was found to support the assignment of recommendation levels, three statements were made. However, traumatic vascular abnormalities have not been discussed in this context.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142948610","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":"A case of late-onset sinusoidal obstruction syndrome diagnosed histologically before meeting diagnostic criteria.","authors":"Takashi Kuroha, Tatsuo Furukawa, Seiichi Yoshikawa, Akane Kaihatsu, Hiroki Nemoto, Hiroyuki Usuda, Toshio Yano","doi":"10.1007/s12185-024-03909-7","DOIUrl":"https://doi.org/10.1007/s12185-024-03909-7","url":null,"abstract":"<p><p>Sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (VOD), is a life-threatening complication of hematopoietic stem cell transplantation. In severe cases, SOS/VOD progresses to multiple organ failure with a mortality rate higher than 80%. Early diagnosis and treatment based on severity assessment improve the prognosis of severe SOS/VOD, but conventional diagnostic criteria may be insufficient for an early diagnosis. We herein report a case of severe late-onset SOS/VOD that was histologically proven before clinical findings become evident. Although defibrotide therapy was started before the clinical diagnostic criteria were met, the disease progressed to multiple organ failure. This case suggests that some patients require earlier treatment before the appearance of clinical findings such as ascites and weight gain or hyperbilirubinemia, and supports the need to establish methods for predicting the onset and severity of SOS/VOD.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":""},"PeriodicalIF":1.7,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142931709","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":"Iron deficiency and phlebotomy in patients with polycythemia vera.","authors":"Yoko Edahiro, Norio Komatsu","doi":"10.1007/s12185-024-03868-z","DOIUrl":"10.1007/s12185-024-03868-z","url":null,"abstract":"<p><p>Polycythemia vera (PV) is a myeloproliferative neoplasm that is associated with an elevated risk of thrombosis. Treatment strategies are based on thrombosis risk classification. Phlebotomy is a commonly recommended treatment for all patients with PV, regardless of their risk classification, and reduces the incidence of thrombosis by lowering hematocrit levels. However, patients with PV frequently present with iron deficiency at diagnosis due to increased erythropoiesis, which repeated phlebotomy can exacerbate. This can produce symptoms that diminish quality of life, such as fatigue, lethargy, and impaired concentration. Recently, hepcidin mimetics have been developed to suppress iron utilization in erythropoiesis. Among them, rusfertide has been shown to control hematocrit levels without requiring phlebotomy. Further studies are needed to identify new treatment strategies for PV that also consider iron deficiency.</p>","PeriodicalId":13992,"journal":{"name":"International Journal of Hematology","volume":" ","pages":"39-44"},"PeriodicalIF":1.7,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142619950","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}