Ueki T, Fuji S, Kato M
… +15 more, Suzuki R, Ogata M, Maruyama Y, Sakaida E, Shirane S, Toyosaki M, Hiramoto N, Nagafuji K, Uchida N, Uehara Y, Yano S, Kimura T, Atsuta Y, Fukuda T, Transplant Complications Working Group of the Japan Society for Hematopoietic Cell Transplantation
Graft failure after allogeneic hematopoietic stem cell transplantation is a life-threatening complication requiring retransplantation. Cord blood is frequently used in this setting, although the optimal conditioning regi...Graft failure after allogeneic hematopoietic stem cell transplantation is a life-threatening complication requiring retransplantation. Cord blood is frequently used in this setting, although the optimal conditioning regimen remains unclear. Recently, a modified short-course fludarabine-based conditioning regimen has been used in clinical practice for salvage cord blood retransplantation in Japan.Using Japanese registry data, we analyzed 465 patients with hematologic malignancies who developed graft failure and underwent cord blood retransplantation within 2 months after allogeneic hematopoietic stem cell transplantation between 2008 and 2016. Of these, 84 patients received a modified short-course fludarabine-based conditioning regimen consisting of fludarabine 30 to 90 mg/m over 1 to 3 days, cyclophosphamide 2 g/m, and low-dose total body irradiation (2 Gy).One-year overall survival and progression-free survival were higher with the modified short-course regimen than with other regimens (45.1% vs 29.9%, p = 0.028; 44.0% vs 26.8%, p = 0.008, respectively). Engraftment at day 56 did not differ significantly between groups (64.3% vs 57.4%, p = 0.178).The modified short-course regimen was associated with improved overall and progression-free survival and may represent a feasible option for salvage cord blood retransplantation after graft failure.
Single-system, single-site (SS-s) Langerhans cell histiocytosis (LCH) is usually managed with observation or local therapy; however, a subset of patients requires systemic chemotherapy, although the optimal intensity for...Single-system, single-site (SS-s) Langerhans cell histiocytosis (LCH) is usually managed with observation or local therapy; however, a subset of patients requires systemic chemotherapy, although the optimal intensity for this group remains unclear. In this study, we retrospectively reviewed patients with newly diagnosed or recurrent SS-s LCH who were treated at our institution. They received 6 weeks of induction chemotherapy with cytarabine, vincristine, and prednisolone, followed by 1 year of oral maintenance therapy with 6-mercaptopurine and methotrexate. Of these patients, 15 were evaluable (13 newly diagnosed and 2 recurrent). At the end of induction, all patients achieved a good or partial response and were administered maintenance therapy, after which they achieved a good response. With a median follow-up of 5.6 years, all patients were alive without subsequent relapse, death, or central nervous system (CNS) complications. No grade 3 or 4 nonhematologic toxicities were observed, except transient increases in liver enzymes during maintenance therapy. No adverse events occurred that required hospitalization. These results suggest that cytarabine-based induction followed by oral maintenance may be an effective and tolerable option for patients with SS-s LCH requiring systemic therapy.
Kashimura M, Fujioka Y, Guo YM
… +3 more, Takahashi T, Moriya K, Takahashi N
Int J Hematol
· 2026 Jun · PMID 42377752
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Achieving treatment-free remission (TFR) in patients with chronic myeloid leukemia (CML) requires several years of tyrosine kinase inhibitor (TKI) therapy and a sustained deep molecular response (DMR). We report an excep...Achieving treatment-free remission (TFR) in patients with chronic myeloid leukemia (CML) requires several years of tyrosine kinase inhibitor (TKI) therapy and a sustained deep molecular response (DMR). We report an exceptional case of a 54-year-old woman who achieved long-term TFR despite discontinuing dasatinib after only 9 months due to grade 3 interstitial pneumonitis. At the time of discontinuation, the duration of DMR was 128 days. Following cessation, BCR::ABL1 transcript levels transiently rebounded to 0.0729% on the International Scale but subsequently declined spontaneously without resumption of TKI therapy. The patient has remained in TFR for more than 7 years. Immunological analysis revealed clonal expansion of a unique CD56CD57CD8 T-cell population possessing natural killer (NK)-like properties, as confirmed by T-cell receptor gene rearrangement analysis. This specific clone persisted after discontinuation, suggesting a role in the sustained immune surveillance of residual leukemia stem cells. These findings suggest that the quality of immune reconstitution, specifically the induction of innate-like effector T-cell clones, may be a more critical determinant of TFR success than the absolute duration of therapy.
Kawahigashi T, Shibusawa M, Miyashita A
… +2 more, Mori H, Kataoka Y
Int J Hematol
· 2026 Jun · PMID 42323469
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Bleeding is a major complication in patients with hematologic malignancies, who frequently develop severe thrombocytopenia. The effectiveness of prophylactic tranexamic acid (TXA) in reducing clinically bleeding remains...Bleeding is a major complication in patients with hematologic malignancies, who frequently develop severe thrombocytopenia. The effectiveness of prophylactic tranexamic acid (TXA) in reducing clinically bleeding remains uncertain. We conducted a systematic review and meta-analysis of randomized controlled trials to evaluate whether prophylactic TXA reduces WHO grade ≥ 2 bleeding and transfusion requirements. Studies enrolling adults receiving chemotherapy, immunotherapy, or hematopoietic stem cell transplantation, or those expected to develop severe thrombocytopenia were eligible; studies including patients with prior thromboembolism, therapeutic anticoagulation, or platelet transfusion refractoriness were excluded. MEDLINE, EMBASE, and Cochrane CENTRAL were searched through April 2025. Two reviewers independently extracted data, assessed risk of bias with the Cochrane RoB 2 tool, and synthesized results using random-effects meta-analysis. Six trials involving 1086 participants were included. TXA showed little to no reduction in WHO grade ≥ 2 bleeding compared with control (OR 0.84, 95% CI 0.64-1.12; moderate certainty). For other outcomes, TXA had minimal effects on red blood cell and platelet transfusion requirements (low certainty), and thromboembolic events were uncommon, with no clear signal of increased risk (low certainty). Prophylactic TXA likely offers limited clinical benefit for patients with hematologic malignancies but appears generally safe.
Int J Hematol
· 2026 Jun · PMID 42315760
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Although novel drugs have recently been introduced in the clinic, the survival rates of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are not yet satisfactory; thus, developing novel strategies with di...Although novel drugs have recently been introduced in the clinic, the survival rates of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are not yet satisfactory; thus, developing novel strategies with different modes of action is urgently needed. Immunotherapy against AML/MDS is less feasible than that for B-cell malignancies because commonly and uniformly expressed cell-surface targets are lacking, and HLA constraints further limit patient eligibility. Careful patient selection based on high and homogeneous target antigen expression in AML/MDS cells may serve as a biomarker. In this review, we first describe T-cell receptor (TCR) T-cell therapy development processes, then review recent clinical trials and future perspectives.
Kanda Y, Takenaka K, Tanabe T
… +4 more, Omoto K, Kamimura K, Kudou K, Kaneko M
Int J Hematol
· 2026 Jun · PMID 42283791
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The efficacy and safety of maribavir, an oral benzimidazole nucleoside that inhibits viral kinase UL97, were evaluated in hematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT) recipients with cytomega...The efficacy and safety of maribavir, an oral benzimidazole nucleoside that inhibits viral kinase UL97, were evaluated in hematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT) recipients with cytomegalovirus (CMV) infection. This phase III, multicenter, open-label, single-arm, interventional study (ClinicalTrials.gov: NCT05137717; Japan Registry of Clinical Trials: jRCT2021210056) investigated oral maribavir 400 mg twice daily for 8 weeks. Japanese HSCT/SOT recipients aged ≥ 16 years with symptomatic or asymptomatic CMV infection (including those resistant/refractory to ganciclovir, valganciclovir, or foscarnet) and without central nervous system CMV tissue-invasive disease or CMV retinitis were enrolled. Primary endpoints were confirmed CMV viremia clearance at week 8 for efficacy and treatment-emergent adverse events (TEAEs) for safety. Among 61 patients enrolled across 22 sites, 41 patients (median age 56.0 years; 36/41 [87.8%] were HSCT recipients) received ≥ 1 maribavir dose. At week 8, twenty-eight patients (28/41, 68.3%) achieved confirmed CMV viremia clearance. Overall, 36 patients (36/41, 87.8%) had ≥ 1 TEAE, including nausea (10/41, 24.2%), anemia (7/41, 17.1%), pyrexia (6/41, 14.6%), and headache (6/41, 14.6%). Two deaths were deemed unrelated to maribavir. In conclusion, maribavir was effective in achieving CMV viremia clearance, with an acceptable safety profile and tolerability, in Japanese HSCT or SOT recipients with CMV infection.
Wakayama T, Imoto N, Ukai S
… +2 more, Ito R, Kurahashi S
Int J Hematol
· 2026 Jun · PMID 42268480
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BACKGROUND: Wilms tumor gene 1 (WT1) is frequently identified in hematologic malignancies; however, its utility for measurable residual disease (MRD) assessment remains controversial. We investigated the prognostic impac...BACKGROUND: Wilms tumor gene 1 (WT1) is frequently identified in hematologic malignancies; however, its utility for measurable residual disease (MRD) assessment remains controversial. We investigated the prognostic impact of WT1 dynamics in peripheral blood (PB) before and after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). METHODS: Of 85 patients with AML or MDS who underwent allo-HSCT at our institution between January 2016 and March 2025, 51 who had WT1 in PB assessed before and approximately 100 days after transplantation were analyzed retrospectively. Patients were stratified by WT1 status before and/or after allo-HSCT. WT1 expression < 50 copies/µg RNA was defined as MRD-negative. RESULTS: In univariate analysis, 2-year progression-free survival (PFS), overall survival (OS), and cumulative incidence of relapse (CIR) differed significantly between Group A (MRD-/MRD-), Group B (MRD+/MRD-), and Group C (MRD+/MRD+) (PFS: 93.8% vs 67.0% vs 31.2%; OS: 93.8% vs 85.4% vs 48.6%; CIR: 0% vs 28.2% vs 68.7%). In multivariate analysis, conversion from WT1 positivity to negativity after allo-HSCT was associated with a favorable prognosis. No patients who were WT1-negative before allo-HSCT relapsed in this cohort. CONCLUSION: WT1 status before and after allo-HSCT is a relevant prognostic marker.
Izutsu K, Fujimoto A, Rai S
… +17 more, Maruyama D, Ohmachi K, Yokohama A, Tsukasaki K, Uchida T, Kuroda J, Choi I, Hidaka M, Ando J, Sakamoto T, Kato H, Minami H, Yoshida S, Yamamoto M, Gillings M, Onogi H, Takeuchi K
Int J Hematol
· 2026 Jun · PMID 42262647
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T follicular helper (TFH)-derived peripheral T-cell lymphomas (PTCLs) harbor frequent mutations in epigenetic regulators and are sensitive to epigenetic therapies. The histone deacetylase inhibitor tucidinostat demonstra...T follicular helper (TFH)-derived peripheral T-cell lymphomas (PTCLs) harbor frequent mutations in epigenetic regulators and are sensitive to epigenetic therapies. The histone deacetylase inhibitor tucidinostat demonstrated efficacy in relapsed/refractory PTCL, especially angioimmunoblastic T-cell lymphoma (AITL), in a phase IIb trial; however, the lack of TFH phenotyping has prevented assessment of the predictive value of this phenotype across a broader PTCL spectrum. Therefore, we retrospectively analyzed patients originally diagnosed with AITL or PTCL not otherwise specified (NOS) based on 2008 World Health Organization criteria who participated in the aforementioned trial. TFH phenotype was defined as the expression of ≥ 2 TFH markers. Among the 23 evaluable patients, the TFH group (n = 17) included six with AITL and 11 with PTCL-NOS exhibiting TFH features, and the non-TFH group included six patients with PTCL-NOS. The objective response rate was numerically higher in the TFH group (12/17, 70.6%) than in the non-TFH group (2/6, 33.3%; P = 0.162). Three patients with the TFH phenotype maintained a progression-free survival exceeding 3 years. These findings highlight the potential long-term benefit of tucidinostat in patients with TFH-derived PTCL, supporting the distinct susceptibility of this subtype to epigenetic modulation.
Int J Hematol
· 2026 Jun · PMID 42260206
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Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are closely related bone marrow (BM) failure syndromes linked by shared immunological mechanisms. The frequent emergence of PNH-type blood cells during A...Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are closely related bone marrow (BM) failure syndromes linked by shared immunological mechanisms. The frequent emergence of PNH-type blood cells during AA, together with the co-occurrence of the disorders and their overlapping therapeutic responses, supports the concept that they represent a spectrum of immune-mediated hematopoietic injury rather than distinct disease entities.AA is a prototypical immune-mediated BM failure characterized by cytotoxic T-cell-driven suppression of hematopoietic stem and progenitor cells. Within this immune-hostile environment, glycosylphosphatidylinositol (GPI)-anchor-deficient PNH clones commonly emerge and persist, a phenomenon best explained by immune selection rather than intrinsic proliferative advantage. Although most PNH clones detected in AA remain small and clinically silent, their presence reflects ongoing or past immune pressure within the BM microenvironment.Advances in high-sensitivity flow cytometry have revealed that minor PNH populations are frequent in AA, indicating that immune-driven clonal selection occurs at an early stage of hematopoiesis. Longitudinal studies further demonstrate that clonal dynamics parallel changes in immune activity, supporting their relevance for risk-adapted monitoring.In this review, we highlight immune-mediated BM failure as the common soil from which PNH clones arise and discuss implications for diagnosis, monitoring, and phenotype-driven management.
Matsuda M, Ieko M, Komiyama Y
… +15 more, Masutani R, Inoue M, Kouno H, Sasaki H, Shimomura D, Ikebe A, Naito S, Yuki S, Kanouchi K, Kouno A, Sakuma T, Sato M, Shizuka R, Hashiguchi T, Working group for standardization of sample handling procedure in coagulation tests, Japanese Society for Laboratory Hematology
Int J Hematol
· 2026 Jun · PMID 42249206
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Preanalytical variables strongly influence coagulation test results; however, their combined effects remain insufficiently evaluated. This study examined whole-blood and plasma stability under conditions mimicking curren...Preanalytical variables strongly influence coagulation test results; however, their combined effects remain insufficiently evaluated. This study examined whole-blood and plasma stability under conditions mimicking current sample storage and transport practices, focusing on three variables: time, temperature, and mechanical agitation. Coagulation tests were performed using four manufacturers' systems, and sample stability was assessed using percentage changes with a 10% criterion and statistical analysis. Among all conditions tested, frozen plasma was consistently the most stable across assays. Whole blood stored at room temperature showed relatively smaller changes, whereas refrigerated whole blood exhibited the largest variation, with factor VIII activity and von Willebrand factor antigen levels decreasing to ≤ 30% even in samples obtained from healthy participants. In some refrigerated samples, clotting times shortened, leading to false-negative lupus anticoagulant results. Mechanical agitation had only marginal effects compared with time and temperature. Sample stability differed substantially between whole blood and plasma and across test types, and the alteration of sample quality accelerated depending on time and temperature conditions, leading to variable testing results. This study underscores the importance of immediate frozen plasma preparation after blood collection to prevent misinterpretation in clinical practice, particularly when prolonged storage is unavoidable, as in outsourced testing.
Int J Hematol
· 2026 Jun · PMID 42247117
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Nucleophosmin 1 (NPM1) gene aberrations are among the most common genetic alterations in acute myeloid leukemia (AML). NPM1 mutations (NPM1c), detected in 30-40% of adult AML cases, are well-characterized and associated...Nucleophosmin 1 (NPM1) gene aberrations are among the most common genetic alterations in acute myeloid leukemia (AML). NPM1 mutations (NPM1c), detected in 30-40% of adult AML cases, are well-characterized and associated with distinct clinical and molecular features. In contrast, NPM1 rearrangements (NPM1-r), involving rare fusion partners such as MLF1, CCDC28A, HAUS1, and RARA, are much less common and have historically been poorly understood. However, recent studies have begun to shed light on the molecular mechanisms and clinical characteristics of NPM1-r AML, revealing overlapping features with NPM1c-AML. Both types share key leukemogenic mechanisms, including cytoplasmic mislocalization of NPM1, interaction with XPO1, and sustained HOX gene expression, which drive leukemic transformation. NPM1-r AML may exhibit unique clinical characteristics, including a higher prevalence in younger patients and potentially a poorer prognosis, although further validation in larger cohorts is needed. Therapeutic strategies targeting XPO1 and Menin, which have shown promise in NPM1c-AML, may also hold potential for NPM1-r AML. Continued research is essential to further elucidate the biology of this rare AML subtype and to establish optimized treatment strategies.
Int J Hematol
· 2026 Jun · PMID 42230475
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BCR::ABL1-negative myeloproliferative neoplasms (Ph-negative MPNs) are clonal hematologic malignancies characterized by aberrant myeloid proliferation driven by canonical driver mutations in JAK2, MPL, or CALR. The ident...BCR::ABL1-negative myeloproliferative neoplasms (Ph-negative MPNs) are clonal hematologic malignancies characterized by aberrant myeloid proliferation driven by canonical driver mutations in JAK2, MPL, or CALR. The identification of these driver lesions has transformed diagnosis and therapeutic development; however, disease phenotype, clinical behavior, and treatment response are further shaped by cooperating mutations, clonal architecture, and the order of mutation acquisition. In parallel with these genetic insights, transcriptional and cellular readouts reflecting disease-state activity are increasingly recognized as critical complements to genotype-based classification. In this context, platelet-derived CREB3L1 mRNA expression has emerged as a lineage-associated biomarker that distinguishes neoplastic from reactive blood cell increases and reflects tumor-intrinsic proliferative states. Recent therapeutic advances increasingly aim to move beyond pathway-level cytoreduction toward disease modification and clonal control, including interferon-based strategies, mutation-selective approaches targeting oncogenic JAK2 or mutant CALR, and allele-directed therapeutic concepts currently under clinical and preclinical development. This review synthesizes recent advances in the genetic architecture, clonal evolution, and therapeutic targeting of Ph-negative MPNs, with particular emphasis on integrating mutational profiling and functional biomarkers to refine diagnosis, guide disease-modifying therapies, and enable mechanism-aligned molecular monitoring.
Int J Hematol
· 2026 May · PMID 42209950
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Efanesoctocog alfa is an extended half-life recombinant factor VIII (FVIII) designed for prophylaxis in hemophilia A (HA). However, global coagulation potential after repeated short-interval dosing remains unclear. We as...Efanesoctocog alfa is an extended half-life recombinant factor VIII (FVIII) designed for prophylaxis in hemophilia A (HA). However, global coagulation potential after repeated short-interval dosing remains unclear. We assessed coagulation potential following repeated short-interval administration of efanesoctocog alfa. Efanesoctocog alfa and rurioctocog alfa (1, 2, and 3 IU/mL) were added to FVIII-deficient plasma samples, and coagulation potential was assessed using thrombin generation assays. Efanesoctocog alfa and rurioctocog alfa were intravenously administered at 100 IU/kg to HA mice once every 24 h for three consecutive days. Coagulation function was assessed by rotational thromboelastometry. Activated partial thromboplastin time (aPTT), FVIII activity by chromogenic assay (FVIII:C), thrombin-antithrombin complex (TAT), and D-dimer were measured 5 min after each dose. Thrombin generation potential in FVIII-deficient plasma spiked with efanesoctocog alfa was comparable to that in plasma spiked with rurioctocog alfa. In HA mice, rotational thromboelastometry parameters, aPTT, TAT, and D-dimer were similar with efanesoctocog alfa and rurioctocog alfa, whereas FVIII:C by chromogenic assay was higher with efanesoctocog alfa than with rurioctocog alfa. In conclusion, global coagulation potential after short-interval administration of efanesoctocog alfa was similar to that after rurioctocog alfa under the experimental conditions.
Recent advances in multiple myeloma (MM) therapy, including quadruplet regimens, chimeric antigen receptor T cell therapies, and bispecific antibodies, have dramatically improved outcomes, necessitating disease assessmen...Recent advances in multiple myeloma (MM) therapy, including quadruplet regimens, chimeric antigen receptor T cell therapies, and bispecific antibodies, have dramatically improved outcomes, necessitating disease assessment tools that are more sensitive than the conventional serological criteria. Consequently, minimal residual disease (MRD) negativity has emerged as a robust surrogate endpoint for survival. This review summarizes the current methodologies for MRD detection, primarily next-generation sequencing (NGS) and next-generation flow (NGF), and evaluates the prognostic significance of MRD negativity across the disease spectrum. We herein discuss key data from major clinical trials in both newly diagnosed and relapsed/refractory settings, highlighting the high rates of MRD negativity achieved with novel therapeutic classes. A central focus is the evolving paradigm of MRD-adapted therapy, which utilizes dynamic MRD assessment to guide treatment decisions by allowing escalation in high-risk patients or de-escalation to reduce toxicity in deep responders. In addition, we address the optimal timing for MRD testing and the challenges of integrating these assessments into routine clinical practice. In conclusion, this review emphasizes the growing utility of MRD as not only a prognostic biomarker but also a critical tool to drive personalized treatment strategies and further enhance long-term outcomes in MM patients.
Suita N, Kanemaru A, Komori N
… +3 more, Nitta K, Fujita H, Kawabata H
Int J Hematol
· 2026 May · PMID 42204127
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We reviewed 70 patients with prolonged activated partial thromboplastin time (aPTT) who underwent cross‑mixing tests (CMT) at our hospital. Median age was 70 years, median aPTT was 65.8 s, and 41 (58.5%) underwent CMT fo...We reviewed 70 patients with prolonged activated partial thromboplastin time (aPTT) who underwent cross‑mixing tests (CMT) at our hospital. Median age was 70 years, median aPTT was 65.8 s, and 41 (58.5%) underwent CMT for preoperative screening. Visual CMT showed 13 deficiency‑pattern, 4 inhibitor‑pattern, and 53 lupus anticoagulant (LAC)‑pattern cases. Direct oral anticoagulants (DOACs) explained one deficiency‑pattern and two LAC‑pattern cases. All inhibitor‑pattern patients presented with bleeding and were diagnosed with acquired hemophilia A (AHA). Based on additional tests, including dilute Russell's viper venom time, LAC was considered the cause of aPTT prolongation in 59 cases (84.3%). No congenital hemophilia was identified. In CMT, an increase in aPTT after incubation in 50% mixed plasma clearly differentiated AHA (> 30 s) from LAC (< 25 s). All preoperative cases were ultimately attributed to LAC or DOACs; 37 patients proceeded to surgery without abnormal bleeding. These findings suggest that most preoperative aPTT prolongations seen in community practice are due to LAC, whereas AHA is rare. CMT provides same‑day results and is useful for rapidly excluding AHA in preoperative evaluation.
Int J Hematol
· 2026 May · PMID 42201452
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Immunosuppressants can be used to treat chronic graft-versus-host disease (cGVHD) following allogeneic hematopoietic cell transplantation. However, real-world retrospective studies on practical regimens and health outcom...Immunosuppressants can be used to treat chronic graft-versus-host disease (cGVHD) following allogeneic hematopoietic cell transplantation. However, real-world retrospective studies on practical regimens and health outcomes remain limited. To identify challenges associated with systemic treatment of cGVHD, we analyzed anonymized Japanese medical insurance claims data. Among 1090 patients with cGVHD, 81% received systemic treatment with immunosuppressive drugs. The most common initial regimen was a combination of tacrolimus and steroids. Second-line treatment was provided in 44% of patients including the addition of steroids, calcineurin inhibitors, or mycophenolate mofetil, or switching to another drug class. Third-line treatment was provided in 23% and steroid monotherapy was the most commonly used. Immunosuppressive treatment was temporarily discontinued in 79% of patients but was re-initiated in approximately a half of them. Young recipients (< 15 years) and umbilical cord blood recipients had lower rates of treatment re-initiation and conferred lower mortality. Various complications were observed after transplantation but most of them gradually resolved, with the exception of chronic bone, eye, and respiratory system disorders. The immunocompromised state of these patients was also reflected in the frequent use of anti-infective agents. The adverse effects associated with long-term steroid use pose a clinical challenge, highlighting the need for alternative treatments.
Hashimoto Y, Hosoda S, Omura H
… +1 more, Tanaka T
Int J Hematol
· 2026 May · PMID 42160001
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PURPOSE AND METHODS: The availability of high-concentration 20% subcutaneous immunoglobulin (SCIG 20%) and hyaluronidase-facilitated subcutaneous immunoglobulin 10% (fSCIG 10%) has recently expanded therapeutic options f...PURPOSE AND METHODS: The availability of high-concentration 20% subcutaneous immunoglobulin (SCIG 20%) and hyaluronidase-facilitated subcutaneous immunoglobulin 10% (fSCIG 10%) has recently expanded therapeutic options for immunoglobulin replacement therapy in patients with secondary immunodeficiency (SID). The present study aimed to confirm the effectiveness and tolerability of each SCIG formulation in eight patients with SID switched from SCIG 20% to fSCIG 10% and to inform SCIG selection in clinical practice. RESULTS: The number of infection events varied among patients and was generally low during each SCIG administration. At the individual patient level, no significant difference was observed in infection rates between SCIG 20% and fSCIG 10%. Local adverse events occurred more frequently with fSCIG 10% than with SCIG 20%. Local adverse events with fSCIG 10% persisted as treatment continued but remained mild. CONCLUSION: These results suggest that dosing interval preferences, adverse event tolerability, and shared decision-making are important considerations in SCIG selection.
Int J Hematol
· 2026 May · PMID 42151711
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Post-marketing surveillance was conducted in Japan (2015-2024, UMIN000056049) to evaluate the long-term safety and effectiveness of darbepoetin alfa for anemia with myelodysplastic syndromes (MDS) for 5 years after treat...Post-marketing surveillance was conducted in Japan (2015-2024, UMIN000056049) to evaluate the long-term safety and effectiveness of darbepoetin alfa for anemia with myelodysplastic syndromes (MDS) for 5 years after treatment initiation. Among 1834 patients (median age: 79.0 years), the distribution of International Prognostic Scoring System (IPSS) risk categories was: Low, 39.3%; Intermediate-1, 45.1%; Intermediate-2, 7.7%; and High, 2.8%. The median (range) administration duration, dosing interval, and weekly dose were 330.0 (1-1863) days, 1.7 (0.2-41.5) weeks, and 240.0 (30.0-250.0) µg, respectively. Treatment discontinuation reasons included inadequate response (36.9%) and adverse events (AEs) (31.2%). AEs were reported in 75.1% and included pneumonia (16.6%) and transformation to acute myeloid leukemia (9.0%). Mean hemoglobin concentration (n = 1821) increased from 7.6 g/dL at baseline to > 9.0 g/dL after 52 weeks. Red blood cell transfusion volume decreased by ≥ 50% compared with baseline in 65.2% of patients. In transfusion-dependent patients (n = 909), the highest transfusion-independence rate was 40.9% at year 4-5. Five-year overall/leukemia-free survival rates (n = 1826/1814) by IPSS risk group were: Low, 49.5%/48.8%, Intermediate-1, 31.1%/30.2%, Intermediate-2, 11.9%/10.4%, and High, 0%/0%. Poor prognostic factors for MDS were IPSS risk category, age, chromosomal abnormalities, and platelet count. No new safety or effectiveness concerns were identified.Trial registration: University Hospital Medical Information Network; study ID: UMIN000056049.
Miyazawa Y, Kanaya S, Takei H
… +6 more, Kobayashi N, Osaki Y, Ogawa Y, Yokohama A, Saitoh T, Handa H
Int J Hematol
· 2026 May · PMID 42149416
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BACKGROUND: To evaluate the real-world effectiveness, overall survival (OS), durability of response, and tolerability of daratumumab-based therapy for systemic amyloid light-chain (AL) amyloidosis in Japan, where bortezo...BACKGROUND: To evaluate the real-world effectiveness, overall survival (OS), durability of response, and tolerability of daratumumab-based therapy for systemic amyloid light-chain (AL) amyloidosis in Japan, where bortezomib and daratumumab were only recently introduced, using 3-year follow-up data. METHODS: We retrospectively reviewed 43 patients diagnosed with AL amyloidosis. Fifteen patients received daratumumab-containing regimens (Dara group) and 28 received non-daratumumab therapy (non-Dara group). Clinical characteristics, hematologic response (HR), organ response (OR), OS, and treatment tolerability were compared. Four patients with Mayo 2012 stage IV disease underwent stepwise dose modification of daratumumab-based therapy. RESULTS: The Dara group showed significantly higher HR (93% vs. 35.7%) and OR (71.4% vs. 19.2%) and improved OS compared with the non-Dara group. At a median follow-up of 34 months, 11 of the 12 surviving patients in the Dara group maintained a complete HR. In Mayo stage IV cases, reduced-intensity induction with daratumumab and dexamethasone, followed by gradual intensification, achieved an OR in most patients evaluated. CONCLUSIONS: Daratumumab-based therapy resulted in high response rates, durable remission, and improved OS in patients with systemic AL amyloidosis. Daratumumab-based strategies may benefit patients across risk groups, including those with advanced cardiac involvement.