Int J Hematol
· 2026 Jan · PMID 41225257
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Wiskott-Aldrich syndrome (WAS) is a rare X-linked recessive disorder characterized by microthrombocytopenia, eczema, recurrent infections, and immune dysregulation, affecting approximately 1 in 100,000 live births. While...Wiskott-Aldrich syndrome (WAS) is a rare X-linked recessive disorder characterized by microthrombocytopenia, eczema, recurrent infections, and immune dysregulation, affecting approximately 1 in 100,000 live births. While the disorder was historically fatal in early childhood, advances in hematopoietic stem cell transplantation (HSCT), gene therapy, and supportive care have improved survival, though morbidity remains high. Mutations in the WAS gene disrupt the WAS protein (WASp), which is essential for actin cytoskeleton dynamics, thereby impairing immune cell function. Over 466 mutations correlate with disease severity, from severe classic WAS to milder X-linked thrombocytopenia (XLT). Supportive therapies manage symptoms, while HSCT offers a cure for severe cases. Gene therapy has emerged as a promising alternative, with early gamma-retroviral trials showing efficacy but also a risk of leukemogenesis. Third-generation lentiviral vectors with self-inactivating LTRs (long terminal repeats) demonstrate improved safety and immune restoration in clinical trials. This review evaluates the evolution of gene therapy for WAS, from early trials to emerging genome editing approaches, assessing their efficacy, safety, and potential to transform clinical outcomes.
Saika W, Yamazaki H, Tanaka S
… +13 more, Lin M, Oshima S, Gao Z, Nomura M, Zang W, Koike Y, Xiao M, Ito H, Matsumoto N, Yamasaki T, Nishimura K, Murata M, Inoue D
Int J Hematol
· 2026 Mar · PMID 41219678
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SF3B1 mutations are among the most common splicing factor mutations in myeloid malignancies, yet the mechanisms linking aberrant splicing to metabolic phenotypes remain incompletely understood. We previously demonstrated...SF3B1 mutations are among the most common splicing factor mutations in myeloid malignancies, yet the mechanisms linking aberrant splicing to metabolic phenotypes remain incompletely understood. We previously demonstrated that SF3B1 mutations cause nonsense-mediated decay of BRD9, a core component of the non-canonical BAF chromatin remodeling complex. Here, we investigated how the SF3B1-BRD9 pathway contributes to metabolic reprogramming in hematopoietic cells. Using BRD9-depleted murine models and analyses of SF3B1-mutated samples, we found that BRD9 depletion markedly upregulates ALOX5, which plays a key role in lipid peroxidation, particularly by oxidizing polyunsaturated fatty acids. BRD9 and ALOX5 expressions are negatively correlated, and the presence of SF3B1 mutations is associated with ALOX5 upregulation in AML datasets. Notably, transcriptomic analysis demonstrated preferential upregulation of ALOX5 in mature myeloid lineages rather than stem/progenitor fractions. Mechanistically, integrated RNA-seq/ChIP-seq and Hi-C analyses revealed that BRD9 loss enhances CTCF occupancy at the ALOX5 locus boundary, enabling aberrant chromatin loop formation that drives transcriptional activation. These events increase lipid peroxidation and ferroptosis susceptibility in hematopoietic cells as evidenced by enhanced BODIPY-C11 oxidation and erastin sensitivity. Our findings reveal a spliceosome-to-chromatin-to-metabolism pathway in which SF3B1 mutations promote ferroptosis through BRD9-mediated chromatin dysregulation, highlighting the previously unrecognized metabolic rewiring in myeloid malignancies.
Shen Y, Zhang P, He A
… +8 more, Wang J, Liu J, Zhao W, Gu L, Wang J, Lei B, Li X, Yang Y
Int J Hematol
· 2026 Feb · PMID 41219677
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Azacitidine (AZA) monotherapy demonstrates efficacy for post-transplant maintenance in patients with high-risk myeloid malignancies. However, no study has directly compared combination regimens. In this retrospective coh...Azacitidine (AZA) monotherapy demonstrates efficacy for post-transplant maintenance in patients with high-risk myeloid malignancies. However, no study has directly compared combination regimens. In this retrospective cohort study, 59 patients (AML = 56, MDS = 3) received AZA-based maintenance post-allogeneic hematopoietic stem cell transplantation (allo-HSCT), as monotherapy (n = 33), AZA plus interferon-α (IFN-α) (n = 15), or AZA plus targeted agents (n = 11). At the median 31-month follow-up, the overall relapse rate was 10.2% (6/59), with comparable rates across groups (AZA: 9.1%, AZA + IFN-α: 13.3%, AZA + targeted: 9.1%) (P = 0.850). Three 3-year relapse-free survival (89.4%; 95%CI 84.7-94.1%) and overall survival (84.4%; 95%CI 78.9-89.9%) rates did not differ significantly between monotherapy and combination regimens (RFS P = 0.975; OS P = 0.770). Overall adverse event rates showed no statistical difference (P > 0.05), although febrile reactions were more common with IFN-α combination regimens (P < 0.001). These findings demonstrate that AZA monotherapy has non-inferior efficacy compared with combination regimens and has a favorable toxicity profile, establishing it as a viable backbone for maintenance therapy in MRD-negative patients. Biomarker-driven combinations warrant prospective validation.
Yabe M, Furukawa S, Takeyama M
… +5 more, Sasai K, Shimonishi N, Nakajima Y, Ogiwara K, Nogami K
Int J Hematol
· 2026 Mar · PMID 41217634
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BACKGROUND: von Willebrand disease (VWD) is a hereditary bleeding disorder caused by quantitative or qualitative deficiencies in von Willebrand factor (VWF). Our previous studies demonstrated that emicizumab, a bispecifi...BACKGROUND: von Willebrand disease (VWD) is a hereditary bleeding disorder caused by quantitative or qualitative deficiencies in von Willebrand factor (VWF). Our previous studies demonstrated that emicizumab, a bispecific monoclonal antibody that mimics activated factor VIII (FVIII) cofactor activity, enhances thrombus formation under high-shear conditions in whole blood from patients with VWD (PwVWD). However, its effect on plasma coagulation potential remains unclear. AIM: To evaluate the coagulation-enhancing effect of emicizumab in plasma samples from PwVWD using global coagulation assays. METHODS: Plasma samples from PwVWD (type 1, n = 4; type 2, n = 9; type 3, n = 3) were spiked with emicizumab (50 or 100 µg/mL), recombinant VWF (rVWF; 2.4 U/mL), or plasma-derived FVIII/VWF concentrate (pd-FVIII/VWF; 1/2.4 U/mL). Coagulation potential was assessed using clot waveform analysis (Ad|min1|) and thrombin generation assay (Peak-Th). RESULTS: Emicizumab dose-dependently increased Ad|min1| in all type 1 VWD samples and improved Peak-Th in two cases. In type 2 VWD, Ad|min1| increased in all samples, while Peak-Th improved in six cases. In type 3 VWD, emicizumab increased both parameters in all cases. CONCLUSION: Emicizumab enhanced ex vivo coagulation potential in plasma samples from PwVWD across all subtypes, supporting its potential as a therapeutic option.
Aso Y, Nishikawa T, Mori Y
… +16 more, Kodama Y, Iwanaga M, Yoshida M, Haruyama T, Sakata M, Katayama O, Okuhiro K, Honda S, Nagamatsu K, Moroga Y, Takano K, Kawano R, Nakano S, Yahiro T, Nishizono A, Ogata M
Int J Hematol
· 2026 Mar · PMID 41214386
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Hematopoietic stem cell transplantation (HSCT) recipients are highly susceptible to viral infections, many of which are missed by conventional diagnostic tests owing to limited coverage and slow turnaround times. This si...Hematopoietic stem cell transplantation (HSCT) recipients are highly susceptible to viral infections, many of which are missed by conventional diagnostic tests owing to limited coverage and slow turnaround times. This single-center, prospective observational study evaluated a rapid multiplex PCR (mPCR) assay to detect 13 DNA viruses for diagnosis and management of post-HSCT viral infections. Between December 2020 and March 2025, 63 HSCT recipients with suspected viral infections underwent mPCR testing of blood (n = 51) or cerebrospinal fluid (CSF) (n = 12) in parallel with standard diagnostics. The mPCR results were reported within 24 h and integrated into clinical decision-making. The etiology was identified in 44 of 51 (86%) blood samples (30 infections) and 9 of 12 (75%) CSF samples (5 infections). mPCR provided the sole etiological diagnosis in 19 of 63 (30%) episodes: 15 of 51 (29%) blood and 4 of 12 (33%) CSF samples. mPCR enabled targeted treatment in 15 of 63 (24%) episodes: 11 of 51 (22%) blood and 4 of 12 (33%) CSF samples. Unexpected infections, such as varicella-zoster virus meningitis and human herpesvirus 7 encephalitis, were identified only by mPCR. Rapid mPCR provided timely, actionable diagnosis and directly influenced the clinical management of post-HSCT patients.
Izutsu K, Watanabe M, Toubai T
… +10 more, Tsukamoto T, Maruyama D, Kumode T, Fukuhara N, Ogawa N, Satomi N, Nishimura Y, Honda H, Chyla B, Takizawa J
Int J Hematol
· 2026 Feb · PMID 41212498
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This phase 2 study (NCT05105841) evaluated the safety and efficacy of a fixed-duration 12-cycle regimen of venetoclax plus obinutuzumab in Japanese patients with previously untreated chronic lymphocytic leukemia (CLL) or...This phase 2 study (NCT05105841) evaluated the safety and efficacy of a fixed-duration 12-cycle regimen of venetoclax plus obinutuzumab in Japanese patients with previously untreated chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The primary efficacy endpoint was the complete remission (CR)/complete remission with incomplete marrow recovery (CRi) rate, assessed by an independent review committee (IRC) according to the 2008 International Workshop on CLL criteria. Ten patients (6 male, 4 female; 9 CLL, 1 SLL) with a median age of 69.5 years (range 52-76) received venetoclax for a median duration of 11.3 months (range 9.2-12.4). The IRC-assessed CR/CRi rate based on the best overall response was 90.0% (95% confidence interval 55.5%, 99.7%). All patients experienced at least one treatment-emergent adverse event (TEAE), and three patients (30.0%) experienced at least one serious TEAE. The most common TEAEs included infusion-related reactions (60.0%), decreased neutrophil count (50.0%), and nausea (40.0%). Nine patients (90.0%) experienced TEAEs related to venetoclax, while all ten patients (100.0%) had TEAEs related to obinutuzumab. One patient (10.0%) developed COVID-19 pneumonia, necessitating the discontinuation of venetoclax. These findings demonstrate the high efficacy and manageable safety profile of venetoclax plus obinutuzumab in this patient population.
Katayama S, Moriya K, Imamura T
… +13 more, Tamefusa K, Sakaguchi K, Ishihara T, Nishi M, Usami I, Hama A, Hasegawa D, Sato A, Suenobu S, Moriya-Saito A, Horibe K, Hara J, Japan Association of Childhood Leukemia Study Group (JACLS)
Int J Hematol
· 2026 Feb · PMID 41212497
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Iriyama N, Nakayama H, Nakagawa M
… +23 more, Takahata A, Sato E, Takano M, Okada K, Toyama K, Hayashi T, Abe T, Tanaka K, Senpuku Y, Osada Y, Watanabe J, Yamamoto K, Sekiguchi Y, Oshikawa G, Kimura Y, Tokuhira M, Suzuki K, Kumagai T, Ikezoe T, Toyota S, Miura K, Nakazato T, Mori T
Int J Hematol
· 2026 Mar · PMID 41206814
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AIM AND PURPOSE: This study aimed to investigate the relationship between myeloma cell maturity, clinical profiles, and treatment outcomes in patients with multiple myeloma (MM). PATIENTS AND METHODS: The J-CHARGE-MM dat...AIM AND PURPOSE: This study aimed to investigate the relationship between myeloma cell maturity, clinical profiles, and treatment outcomes in patients with multiple myeloma (MM). PATIENTS AND METHODS: The J-CHARGE-MM database includes data from 1200 MM patients diagnosed between January 2011 and January 2023. Immunophenotyping by flow cytometry using antibodies against CD38, CD49e, and MPC-1 was used to evaluate myeloma cell maturity. RESULTS: Out of 969 patients evaluable for myeloma cell maturity, 115 were classified as mature type, 626 as intermediate type, and 228 as immature type. Patients with the immature type were more likely to have the IgG type and 1q21 gain/amplification. Treatment response and outcome analysis showed that patients with the immature type had lower response rates and worse time to next treatment (TTNT) with bortezomib-based therapy compared to those with the mature or intermediate type. Lenalidomide-based therapy partially mitigated the adverse impact of the immature type. However, daratumumab combination therapy showed higher response rates and better TTNT, which was almost equivalent between the mature/intermediate and immature types. CONCLUSION: Stratifying patients with MM based on myeloma cell maturity provides meaningful information for choosing the best treatment strategy and improving patient outcomes.
Kohso A, Iwamoto S, Niwa K
… +9 more, Nannya Y, Toki T, Hanaki R, Toyoda H, Deguchi T, Ogawa S, Terui K, Ito E, Hirayama M
Int J Hematol
· 2026 Feb · PMID 41201769
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Transient abnormal myelopoiesis (TAM) occurs in approximately 10% of neonates with Down syndrome (DS), and about 20% of these cases progress to myeloid leukemia associated with DS (ML-DS) within the first four years of l...Transient abnormal myelopoiesis (TAM) occurs in approximately 10% of neonates with Down syndrome (DS), and about 20% of these cases progress to myeloid leukemia associated with DS (ML-DS) within the first four years of life. TAM usually arises from a single clone harboring an identical GATA1 mutation, but approximately 8% of cases carry heterogeneous clones with distinct GATA1 mutations. To date, no reliable method has been established to determine which clone progresses to ML-DS. Here, we report a unique TAM case with multiple distinct GATA1 mutations in which a minor clone at the time of diagnosis evolved into overt ML-DS by 59 days of age. It is particularly noteworthy that changes in blast surface marker expression detected by flow cytometry (FCM) following a single course of low-dose cytarabine coincided with the emergence of the leukemogenic clone. This case illustrates that the use of FCM may serve as a valuable tool for the early detection of leukemogenic clonal expansion in TAM patients harboring multiple GATA1 mutations.
Imanaka R, Numata H, Katsuoka Y
… +10 more, Uoshima N, Hara S, Ando J, Ota S, Yoshimoto G, Matsuoka A, Morita T, Tsutsui A, Kosugi-Kanaya M, Goto T
Int J Hematol
· 2026 Feb · PMID 41201768
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Venetoclax (VEN) with azacitidine (AZA) is the standard treatment for patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. However, real-world evidence on dosing, scheduling, and outc...Venetoclax (VEN) with azacitidine (AZA) is the standard treatment for patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. However, real-world evidence on dosing, scheduling, and outcomes is lacking, particularly for patients with prior myelodysplastic syndrome (MDS) or AZA treatment, who have been excluded from clinical trials. This was a multicenter retrospective study of VEN + AZA in 120 patients newly diagnosed with AML between June 2021 and September 2022. The cohort had a median age of 77 years, 52% had secondary AML, 74% had previously been diagnosed with MDS, and 39% had previously received AZA. During cycle 1, half of the patients received 400 mg of VEN for a median of 27 days, with a median holding period of 12 days. With a median follow-up of 13.6 months, the rate of complete remission (CR) or CR with incomplete blood count recovery was 56.7% in VEN + AZA-treated patients in the overall cohort and 56.5% in patients with prior MDS. Median overall survival was 14.8 months for the overall cohort and 15.4 months for those with prior MDS. The real-world outcomes were comparable to those of clinical trials.
Smoldering multiple myeloma (SMM) represents a heterogeneous precursor stage between monoclonal gammopathy of undetermined significance and symptomatic multiple myeloma (MM). Advances in diagnostic criteria, particularly...Smoldering multiple myeloma (SMM) represents a heterogeneous precursor stage between monoclonal gammopathy of undetermined significance and symptomatic multiple myeloma (MM). Advances in diagnostic criteria, particularly the adoption of the SLiM-CRAB framework, have improved the distinction between SMM and MM and reduced the risk of misclassification. Contemporary risk stratification models, such as the Mayo 20/2/20 criteria and International Myeloma Working Group refinements, integrate both static and dynamic biomarkers to provide more accurate predictions of progression. Genomic and immunological studies have further revealed key drivers of disease evolution, patterns of clonal evolution, and immune dysregulation, which may guide therapeutic decisions. Clinical trials have demonstrated that early intervention with lenalidomide or daratumumab can delay progression. In contrast, intensive multi-agent regimens, such as those used in the GEM-CESAR and ASCENT trials, have achieved high rates of minimal residual disease negativity and suggest curative potential. Nevertheless, the long-term survival benefits of these strategies remain uncertain, and the risks of toxicity in asymptomatic patients require careful consideration. Ongoing studies, including CAR-PRISM and ImmunoPRISM, are evaluating cellular and antibody-based therapies for high-risk SMM. Collectively, these advances highlight the need for refined, individualized approaches that balance the benefits and risks in the management of SMM.
Ohashi KE, Fukuhara S, Maeshima AM
… +10 more, Shibata M, Makino H, Hattori D, Sasaki H, Makita S, Iwaki N, Munakata W, Fukuda T, Ando M, Izutsu K
Int J Hematol
· 2026 Feb · PMID 41171506
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Synchronous central nervous system (CNS) and systemic diffuse large B-cell lymphoma (synDLBCL) is a rare, aggressive entity with poor prognosis and limited data guiding optimal management. We retrospectively analyzed 25...Synchronous central nervous system (CNS) and systemic diffuse large B-cell lymphoma (synDLBCL) is a rare, aggressive entity with poor prognosis and limited data guiding optimal management. We retrospectively analyzed 25 patients with synDLBCL treated at our institution between 2012 and 2021. Among the 20 patients undergoing curative-intent treatment, most received less-intensive CNS-directed therapies, including R-CHOP with intrathecal chemotherapy or high-dose methotrexate. The 3-year progression-free survival rate was 33%, with CNS progression as the predominant site of treatment failure. Genetic profiling in 12 patients revealed a high prevalence (83%) of the MCD subtype, characterized by frequent MYD88 and CD79B mutations, irrespective of immunohistochemical cell-of-origin classification. These findings align with the genetic landscape of primary CNS lymphoma, underscoring the limitations of less-intensive CNS-directed therapies in achieving durable CNS control in synDLBCL. Our findings highlight the need for CNS-penetrant, intensified frontline strategies and support the investigation of Bruton's tyrosine kinase inhibitor-containing regimens in this population. Multicenter collaborative studies with standardized diagnostic and treatment approaches are essential to improve outcomes in synDLBCL.
Choi H, Yamauchi T, Mori Y
… +14 more, Matsuo C, Nishihara H, Irifune H, Nakao F, Ishihara D, Imanaga H, Sasaki K, Sakoda T, Jinnouchi F, Miyawaki K, Shima T, Kikushige Y, Akashi K, Kato K
Int J Hematol
· 2026 Jan · PMID 41171505
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Immune checkpoint inhibitors (CPIs) targeting PD-1, such as nivolumab and pembrolizumab, have become integral to the treatment of relapsed or refractory classical Hodgkin lymphoma (cHL), frequently serving as a bridge to...Immune checkpoint inhibitors (CPIs) targeting PD-1, such as nivolumab and pembrolizumab, have become integral to the treatment of relapsed or refractory classical Hodgkin lymphoma (cHL), frequently serving as a bridge to autologous stem cell transplantation (ASCT). However, recent reports suggest that CPI exposure prior to ASCT may increase the risk of engraftment syndrome (ES), an inflammatory complication that emerges during neutrophil recovery. We present three cases of cHL treated by ASCT was performed following anti PD-1-based salvage therapy. Two of the three patients developed ES, with one case requiring high-dose corticosteroid pulse therapy due to severe respiratory symptoms. We examined lymphocyte counts and the interval between the final CPI dose and transplantation as potential contributing factors. While lymphocyte counts did not correlate with ES severity, the patient with the highest ES severity had the shortest CPI-to-transplant interval. These findings suggest that recent CPI exposure may contribute to ES, possibly via prolonged immune activation. As CPIs become more widely used in pre-ASCT regimens, clinicians should remain alert to the potential for delayed inflammatory toxicities and consider the timing of transplantation when planning post-CPI management strategies.
Perumal HA, Nagarajan S, Ullattil PK
… +3 more, Unnikrishnan MK, Nair AR, Dutta D
Int J Hematol
· 2026 Feb · PMID 41166053
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BACKGROUND: Extramedullary plasmacytoma is a rare tumor, that primarily occurs in the head and neck region. We report a case series on head and neck extramedullary plasmacytoma treated with radiation therapy to provide i...BACKGROUND: Extramedullary plasmacytoma is a rare tumor, that primarily occurs in the head and neck region. We report a case series on head and neck extramedullary plasmacytoma treated with radiation therapy to provide insights regarding clinical outcomes. MATERIALS AND METHODS: This was a retrospective study on radiation therapy outcomes in patients with head and neck extramedullary plasmacytoma treated between January 2010 and December 2024. The treatment course was evaluated, in terms of local tumor control rates and treatment-related toxicities. RESULTS: A total of 200 plasmacytoma patients were screened, and 6 patients with extramedullary disease were included in the study. The most common site of occurrence was the larynx. Radiation therapy was administered at a dose of 45-50 Gy in 20-25 fractions. At a mean follow-up of 8 years, overall survival and disease-free survival were 8 and 7 years, respectively. A local control rate of 83% was achieved through radiation therapy alone. One of six patients progressed to multiple myeloma at 3 months; and all patients were alive as of the last follow-up. CONCLUSION: In head and neck extramedullary plasmacytoma, radiation offers excellent local control with acceptable toxicities while also preserving organ function.
Kawai H, Fukushima H, Nannya Y
… +3 more, Onizuka M, Ogawa Y, Kawada H
Int J Hematol
· 2026 Jan · PMID 41148513
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De novo Ph-positive MDS with the micro BCR::ABL1 (e19a2/p230) transcript is rare. Here, we report a case of MDS with multilineage dysplasia in an 86-year-old woman. Reverse transcription polymerase chain reaction (RT-PCR...De novo Ph-positive MDS with the micro BCR::ABL1 (e19a2/p230) transcript is rare. Here, we report a case of MDS with multilineage dysplasia in an 86-year-old woman. Reverse transcription polymerase chain reaction (RT-PCR) showed the following karyotype: 46, XX, t(9;22)(q34.1;q11.2), i(17)(q10) [20], and p230 BCR::ABL1. Targeted NGS at diagnosis revealed mutations in CSF3R, BCOR, SRSF2, and ASXL1. Low-dose dasatinib (20 mg/day) reduced BCR::ABL1 levels (FISH negative, RT-PCR positive), but had no effect on anemia, dysplasia, or transfusion frequency. At six months, panel results showed loss of all mutations except ASXL1, as well as clearance of Ph-positive subclones. However, an ASXL1 founder clone persisted. Post-treatment chromosome analysis was not feasible because of poor cell growth. Serial genomics suggested that Ph positivity was a late/secondary event on a pre-existing ASXL1-mutant background. In Ph-positive de novo MDS, BCR::ABL1 transcript responses alone may not reflect disease control when an adverse founder clone persists. Integrating panel-based NGS with fusion transcript monitoring may improve therapeutic decision-making and prognostic assessment.
Nitta K, Takagi S, Tsuchihashi R
… +15 more, Kuno M, Watanabe O, Yamaguchi K, Kageyama K, Kaji D, Taya Y, Nishida A, Ishiwata K, Yamamoto H, Araoka H, Yamamoto G, Asano-Mori Y, Wake A, Taniguchi S, Uchida N
Int J Hematol
· 2025 Dec · PMID 41145908
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Pericardial effusion (PCE) is a serious complication after allogeneic hematopoietic cell transplantation, but its etiology is not fully understood, particularly the role of viral reactivation. We investigated the presenc...Pericardial effusion (PCE) is a serious complication after allogeneic hematopoietic cell transplantation, but its etiology is not fully understood, particularly the role of viral reactivation. We investigated the presence of DNA viruses in pericardial fluid from nine umbilical cord blood transplant recipients who underwent pericardiocentesis. Multiplex PCR detected DNA viruses in seven patients (78%), with Epstein-Barr virus being most common. The clinical context of viral detection appeared to differ by onset timing. In early-onset PCE (< 100 days), viral presence was often systemic and likely secondary to severe inflammation. In contrast, late-onset cases frequently occurred with chronic graft-versus-host disease and showed localized viral reactivation within the pericardium. These findings suggest DNA viruses are potential contributors to post-transplant PCE. Viral evaluation of pericardial fluid should be considered in these patients as it may influence therapeutic strategies.
Sumitani R, Miki H, Nakamura S
… +14 more, Nakamura M, Hori T, Maeda Y, Oura M, Sogabe K, Yagi H, Takahashi M, Harada T, Fujii S, Okada N, Nishio S, Tomonari T, Abe M, Matsuoka KI
Int J Hematol
· 2026 Feb · PMID 41143947
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Liver involvement in systemic immunoglobulin light-chain (AL) amyloidosis is diagnosed by an increase in liver size and/or elevated serum alkaline phosphatase (ALP) levels. However, novel diagnostic approaches that refle...Liver involvement in systemic immunoglobulin light-chain (AL) amyloidosis is diagnosed by an increase in liver size and/or elevated serum alkaline phosphatase (ALP) levels. However, novel diagnostic approaches that reflect the organ-specific characteristics of liver involvement are needed. Shear wave elastography (SWE) is a noninvasive and repeatable technique for measuring tissue stiffness. In this study, we evaluated the clinical significance of SWE and serum biochemistry tests in the diagnosis of AL amyloidosis with liver involvement. Twenty-five patients with systemic AL amyloidosis were examined. Eight patients were diagnosed with liver involvement according to the current consensus criteria. In patients with and without liver involvement, hepatic shear wave velocity (SWV) was 1.93 ± 0.39 versus 1.36 ± 0.11 m/sec (p = 0.00099), and the gamma-glutamyltranspeptidase (γ-GTP) level was 165 ± 131 versus 36 ± 28 U/L (p = 0.00222). Receiver-operating characteristic curve analysis showed that SWV and γ-GTP levels had favorable diagnostic accuracy for liver involvement. Of note, two patients with liver involvement who achieved organ response showed a decrease in both SWV and γ-GTP levels. These results suggest that SWV and γ-GTP are useful for establishing accurate organ-specific diagnosis and response criteria for liver involvement in AL amyloidosis.
Chikagawa Y, Higashi D, Sakai S
… +15 more, Yoshino H, Nukii Y, Mura H, Nakagawa S, Yamada S, Yoroidaka T, Imi T, Zaimoku Y, Maruyama H, Hosokawa K, Aoki G, Yoshida A, Takamatsu H, Yamazaki H, Miyamoto T
Int J Hematol
· 2026 Jan · PMID 41143946
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A 53-year-old woman experienced relapse of diffuse large B cell lymphoma (DLBCL) 16 years after achieving a first complete metabolic response (CMR). Despite initially being refractory to salvage chemotherapy, she achieve...A 53-year-old woman experienced relapse of diffuse large B cell lymphoma (DLBCL) 16 years after achieving a first complete metabolic response (CMR). Despite initially being refractory to salvage chemotherapy, she achieved a second CMR and underwent umbilical cord blood transplantation (UCBT). On day 119 post-transplantation, she experienced a second relapse and received chimeric antigen receptor T (CAR T) cell therapy, achieving a third remission without cytokine release syndrome or graft-versus-host disease (GVHD). However, a third relapse occurred seven months after CAR T cell therapy. Epcoritamab treatment was initiated, resulting in CMR without severe complications. Subsequently, the patient underwent curative unrelated allogeneic peripheral blood hematopoietic stem cell transplantation (HSCT). She experienced no severe transplant-related complications, including serious GVHD or infections. She has remained in CMR for > 1 year after the second transplant. Epcoritamab appears to be an effective and safe treatment option for DLBCL relapse, even after both UCBT and CAR T cell therapy, suggesting that bridging therapy with epcoritamab followed by a second allogeneic HSCT may achieve long-term survival.
Yi JH, Park SS, Min CK
… +4 more, Lee JH, Eom HS, Lee JH, Kim K
Int J Hematol
· 2026 Feb · PMID 41134506
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Carfilzomib, lenalidomide, and dexamethasone (KRD) regimens are widely used for relapsed/refractory multiple myeloma (RRMM). However, their administration is limited to 18 cycles, and many patients relapse during subsequ...Carfilzomib, lenalidomide, and dexamethasone (KRD) regimens are widely used for relapsed/refractory multiple myeloma (RRMM). However, their administration is limited to 18 cycles, and many patients relapse during subsequent lenalidomide/dexamethasone (Rd) maintenance. As pomalidomide may overcome lenalidomide refractoriness, a phase II study was conducted to evaluate the efficacy and safety of carfilzomib, pomalidomide, and dexamethasone (KPD) in patients previously treated with KRD. Eligible patients received at least 12 cycles of KRD and six cycles of Rd, were aged 20-80 years, and had measurable disease. The KPD regimen consisted of 56 mg/m carfilzomib (days 1, 8, and 15), pomalidomide 4 mg (days 1-21), and weekly dexamethasone 40 mg, repeated every 28 days. The primary endpoint was the overall response rate (ORR). Between October 2021 and September 2023, 12 patients (median age 60 years, 67% male) were enrolled. The ORR was 75%, including three complete, four very good partial, and two partial responses. The median progression-free survival was 17.2 months. Grade ≥ 3 hematologic toxicities included neutropenia (75%), thrombocytopenia (50%), and anemia (33%). The study was terminated early because of slow accruals. KPD appears to be feasible for lenalidomide-refractory carfilzomib-sensitive RRMM.