Acta Haematologica[JOURNAL]
Sun
200 papers
Failure of Treatment-Free Remission after a Prolonged Deep Molecular Response in Patients with Chronic Myeloid Leukemia.
Nasnas PE, Jabbour EJ, Sasaki K … +4 more , Issa GC, Masarova L, Short NJ, Haddad FG
Acta Haematol · 2025 · PMID 38574468 · Publisher ↗
INTRODUCTION: Treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with chronic myeloid leukemia in chronic phase (CML-CP). Although uncommon, some patients may stil...
INTRODUCTION: Treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with chronic myeloid leukemia in chronic phase (CML-CP). Although uncommon, some patients may still experience molecular or hematologic relapse after treatment discontinuation, even after a prolonged duration of remission. CASE PRESENTATION: In this case series, we report the outcome of 3 patients with CML-CP who were treated with tyrosine kinase inhibitors and achieved a deep molecular response for ≥8 years, but eventually experienced disease relapse after treatment discontinuation. We discuss the importance of regular monitoring after treatment discontinuation as well as future strategies to increase the chances of TFR in patients with CML-CP. CONCLUSION: Despite the high chances of TFR after TKI discontinuation in patients with CML-CP who remain in sustained and prolonged DMR, relapses can still occur. Regular monitoring with RT-PCR is recommended to detect any recurrence of the disease.
Consensus-Based Expert Recommendations on the Management of Hemophilia A in the Gulf Region.
Abdulla Alzahrani H, Warsi A, Mullah-Ali A … +6 more , Alotaibi G, Abu-Riash M, Alshahrani M, Siddiqui M, Owaidah T, Hashmi S
Acta Haematol · 2025 · PMID 38565097 · Full text
BACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region....
BACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript was to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region. SUMMARY: An expert panel was assembled comprising ten clinical hematologists from Kuwait, Oman, Saudi Arabia, and the UAE. The Delphi methodology was used to obtain a consensus on statements relating to several aspects of hemophilia A. A consensus was reached for all statements by means of an online, anonymized voting system. The consensus statements pertain to screening and diagnosis, treatment approaches, and requirements for the implementation of gene therapy. KEY MESSAGES: There are significant challenges that hinder the optimal management of hemophilia A in the Gulf region. The consensus statements presented provide specific recommendations to improve diagnostic and treatment approaches, promote multidisciplinary care, and optimize regional data generation and reporting. These statements also delineate the requirements for the establishment of gene therapy centers for hemophilia A in the region. BACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript was to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region. SUMMARY: An expert panel was assembled comprising ten clinical hematologists from Kuwait, Oman, Saudi Arabia, and the UAE. The Delphi methodology was used to obtain a consensus on statements relating to several aspects of hemophilia A. A consensus was reached for all statements by means of an online, anonymized voting system. The consensus statements pertain to screening and diagnosis, treatment approaches, and requirements for the implementation of gene therapy. KEY MESSAGES: There are significant challenges that hinder the optimal management of hemophilia A in the Gulf region. The consensus statements presented provide specific recommendations to improve diagnostic and treatment approaches, promote multidisciplinary care, and optimize regional data generation and reporting. These statements also delineate the requirements for the establishment of gene therapy centers for hemophilia A in the region.
Utility of Echocardiogram in Neutropenic Patients with Gram-Positive Bacteremia: A Retrospective Study.
Sternbach N, Ben-Zvi H, Wolach O … +4 more , Yeshurun M, Raanani P, Yahav D, Shargian L
Acta Haematol · 2024 · PMID 38527433 · Publisher ↗
INTRODUCTION: Hemato-oncology patients are vulnerable to bloodstream infections due to immunocompromised state and use of intravascular catheters. Data regarding risk of infective endocarditis (IE) among those with gram-...
INTRODUCTION: Hemato-oncology patients are vulnerable to bloodstream infections due to immunocompromised state and use of intravascular catheters. Data regarding risk of infective endocarditis (IE) among those with gram-positive bacteremia are limited. We aimed to evaluate the incidence of IE among neutropenic hemato-oncology patients and explore the yield of echocardiogram in this population. METHODS: We conducted a single retrospective study of all hospitalized hemato-oncology neutropenic patients with gram-positive blood cultures between 2007 and 2021. Data regarding patients' characteristics, blood cultures, and echocardiogram were collected. RESULTS: The study included 241 patients, with 283 isolates. Coagulase-negative Staphylococcus (CONS) was the most commonly isolate found, followed by Streptococcus viridans. Transthoracic echocardiography (TTE) was performed in 45% of patients overall, of which 5.8% had additional transesophageal echocardiogram (TEE). Only a single case of IE was identified in a 47-year-old multiple myeloma patient with neutropenic fever, S. viridans bacteremia, and stroke caused by septic emboli. TTE and TEE failed to demonstrate valvular pathology consistent with IE. CONCLUSION: In our experience, the yield of echocardiogram in hemato-oncological neutropenic patients with bacteremia is extremely low, owing to reduced probability of IE in this population, and thus could be avoided in most cases.
The Ratio of Serum Urea Nitrogen to Albumin Is a Better Predictor of Overall Survival in Multiple Myeloma Patients than Urea Nitrogen Alone.
Shao J, Zhang E, Chen H … +2 more , Cai Z, Dong M
Acta Haematol · 2025 · PMID 38527425 · Full text
INTRODUCTION: Multiple myeloma (MM) is a malignant proliferative disease of plasma cells. Abnormally cloned plasma cells secrete large amounts of monoclonal immunoglobulins in the bone marrow of MM patients. Serum urea n...
INTRODUCTION: Multiple myeloma (MM) is a malignant proliferative disease of plasma cells. Abnormally cloned plasma cells secrete large amounts of monoclonal immunoglobulins in the bone marrow of MM patients. Serum urea nitrogen (sUN) is a byproduct of protein metabolism, and its effect on MM patients' prognoses remains unknown. Therefore, we analyzed MM patients' clinical data to explore the role of sUN and sUN/serum albumin (sUAR) in the baseline tumor load and MM prognosis of MM patients. METHODS: We downloaded the clinical data of 762 MM patients from the MMRF database. After excluding those without baseline sUN, 452 patients were finally included in the study. Smoothed curve fitting, threshold analysis, Tamhane's T2 test, multivariate-adjusted Cox regression analysis, Kaplan-Meier (K-M) curves, and receiver operating characteristic (ROC) analysis were applied in the study. RESULTS: There were 452 newly diagnosed MM patients included in this study. In most patient groups, sUN and sUAR were positively linked with β2-microglobulin (β2-MG) and lactic dehydrogenase (LDH) according to smoothing curve fitting and threshold analysis. The higher the ISS stage, the greater the values of sUN and sUAR. Furthermore, smoothed curve fitting and threshold analysis showed that sUN was correlated with overall survival (OS), although sUAR had a stronger correlation with OS and could be applied to a broader group. The results of a multivariate-adjusted Cox regression analysis demonstrated that sUN and sUAR were independent prognostic factors for OS. The K-M curve confirmed the correlation between higher sUN and sUAR levels and worse OS. β2-MG and LDH are generally recognized prognostic factors of OS. ROC analysis revealed that sUN might boost β2-MG and LDH's predictive value and sUAR had a higher predictive value. CONCLUSION: This retrospective study based on the MMRF database showed that high sUN and sUAR levels were positively associated with β2-MG, LDH, and ISS staging, and sUAR exhibited a stronger correlation with OS than sUN alone. INTRODUCTION: Multiple myeloma (MM) is a malignant proliferative disease of plasma cells. Abnormally cloned plasma cells secrete large amounts of monoclonal immunoglobulins in the bone marrow of MM patients. Serum urea nitrogen (sUN) is a byproduct of protein metabolism, and its effect on MM patients' prognoses remains unknown. Therefore, we analyzed MM patients' clinical data to explore the role of sUN and sUN/serum albumin (sUAR) in the baseline tumor load and MM prognosis of MM patients. METHODS: We downloaded the clinical data of 762 MM patients from the MMRF database. After excluding those without baseline sUN, 452 patients were finally included in the study. Smoothed curve fitting, threshold analysis, Tamhane's T2 test, multivariate-adjusted Cox regression analysis, Kaplan-Meier (K-M) curves, and receiver operating characteristic (ROC) analysis were applied in the study. RESULTS: There were 452 newly diagnosed MM patients included in this study. In most patient groups, sUN and sUAR were positively linked with β2-microglobulin (β2-MG) and lactic dehydrogenase (LDH) according to smoothing curve fitting and threshold analysis. The higher the ISS stage, the greater the values of sUN and sUAR. Furthermore, smoothed curve fitting and threshold analysis showed that sUN was correlated with overall survival (OS), although sUAR had a stronger correlation with OS and could be applied to a broader group. The results of a multivariate-adjusted Cox regression analysis demonstrated that sUN and sUAR were independent prognostic factors for OS. The K-M curve confirmed the correlation between higher sUN and sUAR levels and worse OS. β2-MG and LDH are generally recognized prognostic factors of OS. ROC analysis revealed that sUN might boost β2-MG and LDH's predictive value and sUAR had a higher predictive value. CONCLUSION: This retrospective study based on the MMRF database showed that high sUN and sUAR levels were positively associated with β2-MG, LDH, and ISS staging, and sUAR exhibited a stronger correlation with OS than sUN alone.
Long Non-Coding RNA RP11-252C15.1 Is a Potential Biomarker of Prognosis and Hallmark for Leukemogenesis in Children with B-Cell Precursor Acute Lymphoblastic Leukemia.
Pan L, Zheng Y, Zheng H
Acta Haematol · 2024 · PMID 38527424 · Publisher ↗
INTRODUCTION: Improved understanding of the prognostic biomarkers associated with childhood acute lymphoblastic leukemia (ALL) is needed for accurate risk group stratification. This study aimed to identify potential long...
INTRODUCTION: Improved understanding of the prognostic biomarkers associated with childhood acute lymphoblastic leukemia (ALL) is needed for accurate risk group stratification. This study aimed to identify potential long non-coding RNA (lncRNA) markers and evaluate their prognostic value in children with ALL. METHODS: We selected 50 children with newly diagnosed ALL and 20 age-matched patients with idiopathic immune thrombocytopenia (controls). RNA sequencing was performed to identify differentially expressed lncRNAs between the ALL and control groups. Correlation analysis was performed to determine the relationships between candidate lncRNAs, clinical features, and the risk of leukemogenesis. RESULTS: A total of 1,019 differentially expressed lncRNAs were identified between the ALL and control groups. Reverse transcriptase (RT-qPCR) revealed that lncRNA RP11-252C15.1 and lncRNA RP11-701P16.2 were significantly upregulated in patients with ALL. Furthermore, correlation analysis showed that lncRNA RP11-252C15.1 and lncRNA RP11-701P16.2 represent potential predictors of leukemogenesis; however, only lncRNA RP11-252C15.1 was associated with clinical features and outcome in children with B-cell precursor ALL (BCP-ALL). In vitro experiments confirmed that lncRNA RP11-252C15.1 was significantly overexpressed in BCP-ALL cell lines and promoted proliferation and repressed apoptosis in MHH-CALL-3 cells. CONCLUSION: lncRNA RP11-252C15.1 is a potential oncogene in BCP-ALL pathogenesis and a prognostic biomarker in children with BCP-ALL.
The IRE1α Endonuclease Plays a Dual Role in Regulating the XBP1/miRNA-34a Axis and PD-1 Expression within Natural Killer Cells in Hodgkin Lymphoma.
Bednarska K, Thillaiyampalam G, Mujaj S … +10 more , Nourse J, Gunawardana J, Sabdia MB, Law SC, Pilaar A, Cui Q, de Long LM, Vari F, Gandhi MK, Cristino AS
Acta Haematol · 2024 · PMID 38479365 · Publisher ↗
INTRODUCTION: Hodgkin lymphoma (HL) is deficient in major histocompatibility complex class I, rendering it susceptible to antitumoral immunity by natural killer (NK) cells. Despite the functional impairment of PD-1+ NK c...
INTRODUCTION: Hodgkin lymphoma (HL) is deficient in major histocompatibility complex class I, rendering it susceptible to antitumoral immunity by natural killer (NK) cells. Despite the functional impairment of PD-1+ NK cells in HL, the underlying mechanisms of NK cell dysfunction remain unclear. METHODS: This study involved 14 HL patients and SNK10/KHYG-1 cell lines to assess NK cell activation against cancer cells. Activation was measured through transcript (PCR) and protein expression (flow cytometry). Regulatory mechanisms associated with IRE1α activation were validated through knockdown and luciferase reporter assays. RESULTS: Our findings reveal a novel role for IRE1α-endonuclease in fine-tuning NK cell effector functions by orchestrating the XBP1s/microRNA-34a-5p/PD-1 axis. When NK cells encounter cancer cells, IRE1α endonuclease activates the decay of microRNA-34a-5p, resulting in increased expression of XBP1s and PD-1. IRE1α-endonuclease activation enhances NK cell functions while promoting PD-1 expression. In turn, PD-1 is directly regulated by microRNA-34a-5p, which binds to the 3'UTR of PD-1 transcript to repress PD-1 protein on the NK cell surface. Importantly, IRE1α-pathway activation is impaired in NK cells from HL patients. CONCLUSION: The IRE1α endonuclease emerges as a key player, simultaneously regulating the XBP1s/microRNA-34a-5p/PD-1 axis in NK cells, a process disrupted in HL. Targeting the IRE1α-pathway holds promise as a therapeutic strategy to optimize NK cell functions in Hodgkin lymphoma treatments.
Efficacy of Preexposure Prophylaxis with Monoclonal Antibody Tixagevimab-Cilgavimab against Emerging SARS-CoV-2 Resistant Variants in Patients with Chronic Lymphocytic Leukemia.
Benjamini O, Tadmor T, Avigdor A … +10 more , Gershon R, Kliker L, Fares F, Atari N, Laevsky I, Abdelkader B, Hod T, Golan-Shany O, Mandelboim M, Rahav G
Acta Haematol · 2024 · PMID 38471491 · Full text
INTRODUCTION: Preexposure prophylaxis with monoclonal antibodies (mAbs) was developed in addition to COVID-19 vaccine for immunocompromised and those with insufficient immune response, among them patients with CLL. Omicr...
INTRODUCTION: Preexposure prophylaxis with monoclonal antibodies (mAbs) was developed in addition to COVID-19 vaccine for immunocompromised and those with insufficient immune response, among them patients with CLL. Omicron variant and its sublineages evolved mutations that escape mAbs neutralizing effect, yet the extent of which was not studied. METHODS: We evaluated anti-spike titers and neutralization activity of COVID-19 wild-type (WT), Delta, Omicron, BA.2, BA.4, and BA.5 before and after tixagevimab-cilgavimab (TGM/CGM) dose of 150/150 mg or 300/300 mg in patients with CLL. RESULTS: 70 patients were tested 2 weeks before and 4 weeks after receiving TGM/CGM mAbs. After TGM/CGM, anti-spike ab level increased 170-folds from 13.6 binding antibody unit (BAU)/mL (IQR, 0.4-288) to 2,328 BAU/mL (IQR, 1,681-3,500). Neutralization activity increased in all variants and was 176-folds higher in WT and 55-folds higher in Delta compared to 10-folds higher in Omicron and its sublineages (BA.2 ×11, BA.4 ×4, BA.5 ×18). Over follow-up period of 3 months, 20 patients (29%) with CLL acquired COVID-19 infection, all recovered uneventfully. In a multivariate analysis, anti-spike antibody titer was found a significant predictor for post-TGM/CGM COVID-19 infection. CONCLUSION: Efficacy of preexposure prophylaxis with TGM/CGM in patients with CLL is significantly reduced in era of Omicron and its sublineages BA.2, BA.4, and BA.5.
[Momelotinib bei Myelofibrose und Anämie].
Acta Haematol · 2024 · PMID 38467116 · Publisher ↗
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The Effect of Mind-Body Intervention on Lymphocyte Doubling Time and Treatment-Free Survival in Treatment-Naïve Chronic Lymphocytic Leukemia Patients.
Shapira S, Hirschberger N, Ofran Y … +5 more , Mizrahi B, Mandel E, Benjamini O, Rabinowicz N, Zoref-Lorenz A
Acta Haematol · 2024 · PMID 38461815 · Publisher ↗
INTRODUCTION: Mind-body intervention (MBI) serves as a supportive aid in oncology. We hypothesized that MBI could impact the progression of chronic lymphocytic leukemia (CLL) in the "watch and wait" (w&w) phase. METH...
INTRODUCTION: Mind-body intervention (MBI) serves as a supportive aid in oncology. We hypothesized that MBI could impact the progression of chronic lymphocytic leukemia (CLL) in the "watch and wait" (w&w) phase. METHODS: We conducted a non-randomized, prospective controlled study between the years 2020 and 2022 on 76 treatment-naïve CLL patients in the w&w phase. Thirty-seven patients were included in the intervention arm and received MBI, while 39 patients were included in the control group. The primary and secondary endpoints were prolongation of lymphocyte doubling time (LDT) and treatment-free survival (TFS). LDT was compared at 0, 180, 360, and 540 days using paired t tests. TFS was compared between the intervention and control groups using the log-rank test. Cox proportional hazards models were used to calculate the hazard ratio (HR) and 95% confidence interval (CI) for TFS in the intervention group compared to the control, stratified by the study covariates. RESULTS: MBI prolonged LDT at all time points, including at day 360 (median of 2.47 years; CI 1.05-3.9; p = 0.001). TFS at 18 months was longer in the intervention group compared to the control group (HR 0.23; CI 0.06-0.79, p = 0.01). CONCLUSIONS: MBI was associated with prolonged LDT and TFS in patients with CLL in the w&w phase. These results provide a basis for a larger randomized controlled trial.
Peeling Back the Layers: Recurrent Talquetamab Skin Toxicity after Supportive Stem Cell Boost in Multiple Myeloma.
Heini AD, Bacher VU, Akhoundova D … +2 more , Seipel K, Pabst T
Acta Haematol · 2024 · PMID 38447541 · Full text
INTRODUCTION: Bispecific antibodies have meaningfully expanded the therapeutic armamentarium in multiple myeloma. Talquetamab is a CD3+ T-cell-redirecting antibody targeting GPRC5D, which is expressed on multiple myeloma...
INTRODUCTION: Bispecific antibodies have meaningfully expanded the therapeutic armamentarium in multiple myeloma. Talquetamab is a CD3+ T-cell-redirecting antibody targeting GPRC5D, which is expressed on multiple myeloma plasma cells as well as in keratinized tissues. Due to the expression pattern, toxicity of talquetamab involves skin toxicity. CASE PRESENTATION: Here we report the case of a patient who was treated with talquetamab after relapse after CAR-T therapy. The patient developed a severe recurrence of talquetamab-mediated skin toxicity after the administration of a supportive hematopoietic stem cell boost to treat persistent late cytopenias after CAR-T therapy. CONCLUSION: This case underscores the complex dynamics between novel immunotherapies like talquetamab and stem cell-based interventions in the context of MM treatment, shedding light on the need for personalized approaches to maximize the benefits of these therapies while minimizing their associated adverse effects.
Venous Thromboembolism Prophylaxis in Inflammatory Bowel Disease Inpatients: Systematic Review and Meta-Analysis.
McNeil R, Fredman D, Eldar O … +2 more , Gafter-Gvili A, Avni T
Acta Haematol · 2024 · PMID 38432204 · Full text
INTRODUCTION: Inflammatory bowel disease (IBD) patients are three times more likely to develop venous thromboembolism (VTE), and guidelines recommend prophylaxis during all hospitalizations. In this systematic review, we...
INTRODUCTION: Inflammatory bowel disease (IBD) patients are three times more likely to develop venous thromboembolism (VTE), and guidelines recommend prophylaxis during all hospitalizations. In this systematic review, we sought to assess for the benefits and risks of VTE prophylaxis in hospitalized IBD patients. METHODS: We performed a systematic review and meta-analysis. We searched MEDLINE and others up to 2/2022, for studies on IBD inpatients treated with prophylactic anticoagulation during hospitalization, compared to no prophylaxis. Primary efficacy and safety outcomes were any VTE and major bleeding, respectively. Results were pooled using random-effects models, calculating odds ratios (OR), and 95% confidence intervals (CI). The ROBINS-I tool was used to assess bias. RESULTS: We extracted data from 18 observational studies and 2 randomized-trial subgroups. The studies were highly variable regarding the included populations, interventions, and outcome definitions. Meta-analysis of all studies showed a nonsignificant effect of prophylaxis on VTEs (OR: 0.97 [95% CI: 0.49-1.95]). An analysis of eight lower-risk-of-bias studies showed a significant reduction in VTEs (OR: 0.27 [95% CI: 0.13-0.55], number needed to treat (NNT) 34.8 [95% CI: 26.8-49.8]). A significant protective effect persisted in several subgroups. Major bleeding was reported in three studies and showed a significant increase with prophylaxis (OR: 2.02 [95% CI: 1.11-3.67], number needed to harm (NNH) 113.6 [95% CI: 40.7-very-large-number]). CONCLUSION: In studies with lower-risk-of-bias, a significant reduction in VTEs was shown in patients treated with VTE prophylaxis (NNT = 35), which should be carefully considered against an increased major-bleeding risk (NNH = 114). However, current data are limited and randomized trials dedicated to IBD inpatients would aid in understating whether universal prophylaxis should be recommended.
Impacts of Posttransplant Cyclophosphamide Dose on Graft-versus-Leukemia Effects via HLA-B Leader in HLA-Haploidentical Peripheral Blood Stem Cell Transplantation.
Moriguchi M, Ido K, Okamura H … +11 more , Nakamae M, Sakatoku K, Makuuchi Y, Kuno M, Takakuwa T, Hirose A, Nishimoto M, Nakashima Y, Koh H, Hino M, Nakamae H
Acta Haematol · 2024 · PMID 38432202 · Publisher ↗
INTRODUCTION: The graft-versus-leukemia effect of HLA-B leader dimorphism, i.e., methionine (M) or threonine (T) at position -21 of the leader sequence, has been observed in HLA-haploidentical hematopoietic cell transpla...
INTRODUCTION: The graft-versus-leukemia effect of HLA-B leader dimorphism, i.e., methionine (M) or threonine (T) at position -21 of the leader sequence, has been observed in HLA-haploidentical hematopoietic cell transplantation with posttransplant cyclophosphamide (PTCy-haplo). However, the biological mechanism has been unclear, and the contributions of HLA-B leader genotype to risk reduction of relapse might be dependent on posttransplant cyclophosphamide (PTCy) doses. METHODS: To investigate whether the effect of HLA-B leader dimorphism was modified by the PTCy dose, we retrospectively analyzed 99 patients who received PTCy-haplo. RESULTS: In the low-dose PTCy group, the patient M+ HLA-B leader genotype did not significantly affect the cumulative incidence of relapse (CIR) but negatively impacted the overall survival (OS) compared to the M- genotype. In contrast, in the high-dose PTCy group, patients with the M+ genotype had a decreased CIR, but no significant difference in the OS was observed between patients with the M+ and M- genotypes. Regardless of PTCy doses, the patient M+ genotype had detrimental effects on nonrelapse mortality. CONCLUSION: Our findings suggest that the effect of the patient HLA-B leader genotype is modified by the PTCy dose, providing immunological insight into the PTCy dosage and supporting further studies to investigate the underlying mechanisms.
Primary Nodal Epstein-Barr Virus-Positive T-Cell/NK-Cell Lymphoma: Real-World Experience.
Choi DH, Yoon SE, Cho J … +2 more , Kim SJ, Kim WS
Acta Haematol · 2024 · PMID 38432198 · Publisher ↗
INTRODUCTION: Primary nodal Epstein-Barr virus-positive T-cell/NK-cell lymphoma (PTCL-EBV) is a disease entity newly recognized in the World Health Organization's classification of hematolymphoid tumors, 5th edition (WHO...
INTRODUCTION: Primary nodal Epstein-Barr virus-positive T-cell/NK-cell lymphoma (PTCL-EBV) is a disease entity newly recognized in the World Health Organization's classification of hematolymphoid tumors, 5th edition (WHO-HAEMS5) and the International Consensus Classification of Mature Lymphoid Neoplasms (ICC). Previously, it was classified as a subtype within peripheral T-cell lymphoma, not otherwise specified, and was known to have a poor prognosis. However, the clinical features and treatment outcomes are not well known. METHODS: This retrospective observational study was conducted on patients diagnosed with PTCL-EBV at Samsung Medical Center through a pathology review from 2000 to 2020. We analyzed the clinical data from 14 patients, immunohistochemistry, and survival outcomes including overall survival (OS) and progression-free survival (PFS) for each treatment regimen. PFS was defined as the time from the start of chemotherapy to the confirmation of disease progression on imaging, and hematopoietic stem-cell transplantation (HSCT) was considered a consolidation treatment. OS was defined as the time from diagnosis to the time of death. RESULTS: 25% (1 out of 4) were beta-F1 positive, and 67% (4 out of 6) were T-cell receptor gamma (TCRγ) positive. T-cell intracellular antigen (TIA-1) and granzyme B exhibited positive results in all cases (3 out of 3), whereas the NK-cell marker CD56 was positive in only 11% of patients (1 out of 9). CD3 was observed in all of the patients (11 out of 11). The CD4 was 43% positive (3 out of 7). The CD8 was investigated in 8 patients, with 37.5% positive (3 out of 8). Hepatosplenomegaly was observed in 55% of patients (6 out of 11), and 70% (7 out of 10) of patients displayed B symptoms at the time of diagnosis. Patients who received CHOP (cyclophosphamide, doxorubicin, vincristine, prednisolone) or CVP (cyclophosphamide, vincristine, prednisolone) treatment had a median PFS of 2.2 months (95% CI: 1.9-2.5 months), and patients who received other treatments had a median PFS of 5.1 months (NA). The objective response rate (ORR) for ICE/dexa (ifosfamide, carboplatin, etoposide, dexamethasone) as the first- or second-line treatment was 100% (3 out of 3). But ORR of CHOP or CVP as the first-line treatment was 33.3% (3 out of 9). The median OS for the group that received HSCT (3 out of 11) after achieving a response was 34.6 months (95% CI: 0-74.6 months), and the median OS for the group that did not receive HSCT (8 out of 11) was 5.0 months (95% CI: 2.1-7.9 months) (p = 0.04). CONCLUSIONS: In conclusion, in the context of PTCL-EBV, despite a limited sample size, the ICE/Dexa regimen shows potential benefits in terms of ORR and PFS. Furthermore, the application of HSCT following the attainment of a complete response may prove advantageous.
Impact of COVID-19 on Patients with Beta-Thalassemia Major: An Observational Study.
Vacca N, Locci C, Serra F … +5 more , Chicconi E, Puci MV, Sotgiu G, Amadu AM, Antonucci R
Acta Haematol · 2024 · PMID 38417413 · Publisher ↗
INTRODUCTION: The prevalence of COVID-19 is slightly lower, and its mortality is higher in beta-thalassemia patients than in the general population. We evaluated the impact of COVID-19 in terms of incidence, clinical cou...
INTRODUCTION: The prevalence of COVID-19 is slightly lower, and its mortality is higher in beta-thalassemia patients than in the general population. We evaluated the impact of COVID-19 in terms of incidence, clinical course, management, and specific antibody response to vaccination, in a cohort of patients with beta-thalassemia major. METHODS: We retrospectively enrolled all transfusion-dependent beta-thalassemia major patients attending the Thalassemia Day Care Center of the University Hospital of Sassari, Italy, from March 1, 2020, to May 31, 2021. For each patient, demographic, clinical, laboratory, instrumental, and therapy data were collected. Patients aged ≥16 years received two doses of mRNA COVID-19 vaccine. Anti-SARS-CoV-2 serum antibodies were tested before and after the first vaccine dose. RESULTS: A total of 68 patients (median age: 36.5 years; IQR: 13-42 years) were included. Nasopharyngeal swab (NPS) for SARS-CoV-2 detection by RT-PCR was positive in 5 (7.35%) of 68 patients (4 symptomatic). No COVID-19-related complications, hospitalizations, or deaths were observed. The transfusion regimen and iron chelation therapy were not significantly changed. Prior to COVID-19 vaccination, anti-SARS-CoV-2 antibodies were tested in 61 patients, 51 negative and 10 positive; five of the latter were also positive for SARS-CoV-2 on NPS. The 46 vaccinated subjects had an antibody response, with higher levels in subjects previously infected with SARS-CoV-2. CONCLUSION: Our findings suggest that patients with beta-thalassemia major are not at a higher risk of contracting SARS-CoV-2 infection and developing a severe form of COVID-19 despite being considered more vulnerable than the general population.
Erratum.
Acta Haematol · 2024 · PMID 38412838 · Full text
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Zanubrutinib plus Cytarabine in Patients with Refractory/Relapsed Primary Central Nervous System Lymphoma.
Lin Z, Ma J, Ma Y … +4 more , Li Q, Kang H, Zhang M, Chen B
Acta Haematol · 2024 · PMID 38408440 · Full text
INTRODUCTION: Primary central nervous system lymphoma (PCNSL) is a rare subtype of aggressive extranodal non-Hodgkin lymphoma. Currently, there is no standard of care for the treatment of refractory or relapsed PCNSL (r/...
INTRODUCTION: Primary central nervous system lymphoma (PCNSL) is a rare subtype of aggressive extranodal non-Hodgkin lymphoma. Currently, there is no standard of care for the treatment of refractory or relapsed PCNSL (r/r PCNSL). We conducted a prospective single-arm phase II study to evaluate zanubrutinib plus cytarabine for r/r PCNSL. METHODS: Using Simon's two-stage design, we analyzed 34 patients who received high-dose cytarabine (3.0 g/m2 once daily) for 2 days and zanubrutinib (160 mg twice daily) for 21 days each cycle for up to 6 cycles. The study was registered at <ext-link ext-link-type="uri" xlink:href="http://www.chictr.org.cn" xmlns:xlink="http://www.w3.org/1999/xlink">www.chictr.org.cn</ext-link> as #ChiCTR2000039229. RESULTS: The median follow-up was 19 months. The overall response rate was 64.7% (95% confidence interval [CI], 47.9-78.5%) with a complete remission or unconfirmed complete remission rate of 47.1% (16/34) and a partial remission rate of 17.6% (6/34). The median progression-free survival was 4.5 months (95% CI, 1.5-9.4), and the median OS was 18 months (95% CI, 9.5 to not estimable). The median duration of the response was 9 months (95% CI, 3.2 to not estimable). The most common treatment-emergent adverse events were thrombocytopenia (55.9%). No treatment-related death occurred. CONCLUSION: Zanubrutinib and cytarabine showed efficacy in r/r PCNSL with an acceptable safety profile.
Oncogenic MTOR Signaling Axis Compensates BTK Inhibition in a Chronic Lymphocytic Leukemia Patient with Richter Transformation: A Case Report and Review of the Literature.
Parigger T, Drothler S, Scherhäufl C … +13 more , Gassner FJ, Schubert M, Steiner M, Höpner JP, Hödlmoser A, Schultheis L, Bakar AA, Neureiter D, Pleyer L, Egle A, Greil R, Geisberger R, Zaborsky N
Acta Haematol · 2024 · PMID 38402867 · Full text
INTRODUCTION: Targeting the B-cell receptor pathway via ibrutinib, a specific inhibitor of Bruton's tyrosine kinase, has shown marked clinical efficacy in treatment of patients with chronic lymphocytic leukemia (CLL), th...
INTRODUCTION: Targeting the B-cell receptor pathway via ibrutinib, a specific inhibitor of Bruton's tyrosine kinase, has shown marked clinical efficacy in treatment of patients with chronic lymphocytic leukemia (CLL), thus becoming a preferred first line option independent of risk factors. However, acquired resistance to ibrutinib poses a major clinical problem and requires the development of novel treatment combinations to increase efficacy and counteract resistance development and clinical relapse rates. CASE PRESENTATION: In this study, we performed exome and transcriptome analyses of an ibrutinib resistant CLL patient in order to investigate genes and expression patterns associated with ibrutinib resistance. Here, we provide evidence that ibrutinib resistance can be attributed to aberrant mammalian target of rapamycin (MTOR) signaling. CONCLUSION: Thus, our study proposes that combined use of MTOR inhibitors with ibrutinib could be a possible option to overcome therapy resistance in ibrutinib treated patients.
Evaluating the Economic Burden of Acute Myeloid Leukemia in Canada.
Lachaine J, Beauchemin C, Dodat F … +6 more , Au Y, Evans WK, Leber B, Paulson K, Schuh A, Storring J
Acta Haematol · 2025 · PMID 38382483 · Publisher ↗
INTRODUCTION: Acute myeloid leukemia (AML) represents a significant burden for patients and their families, and to the healthcare system. This study estimated the total cost of illness associated with newly diagnosed AML...
INTRODUCTION: Acute myeloid leukemia (AML) represents a significant burden for patients and their families, and to the healthcare system. This study estimated the total cost of illness associated with newly diagnosed AML patients in Canada. METHODS: The economic burden of AML was estimated using an incidence-based model, analyzing different types of AML cases in Canada. Direct and indirect costs were calculated using scientific literature and Canadian clinical experts' inputs. Patients were categorized depending on their eligibility for intensive chemotherapy (fit and unfit patients) as well as according to age and cytogenetic markers. RESULTS: The total average cost of AML per patient is estimated to be CAD 178,073 with a cost of CAD 210,983 and CAD 145,163 for fit and unfit patients, respectively. The costs related to treatment represent half of the total average cost (52%), followed by hematopoietic stem cell transplant (23%), best supportive care (16%), productivity loss (6%), and wastage (4%). CONCLUSION: For patients with AML, the costs associated with fit patients are higher than unfit patients. Hospitalization and best supportive care costs are key cost drivers for the total costs of fit and unfit patients, respectively. This study highlights that AML is associated with a significant economic burden in Canada.
Clinical Outcomes, Survival, and Predictors in Lower-Risk Myelodysplastic Syndrome Patients Treated with Cyclosporine A.
Lu Y, Zhang L, Qu W … +3 more , Feng Z, Deng Y, Zhao L
Acta Haematol · 2024 · PMID 38368853 · Publisher ↗
INTRODUCTION: Therapeutic options to improve myelodysplastic syndrome (MDS)-related cytopenias in patients with lower-risk MDS are limited, and cyclosporin A (CSA) is an available option. METHODS: We retrospectively anal...
INTRODUCTION: Therapeutic options to improve myelodysplastic syndrome (MDS)-related cytopenias in patients with lower-risk MDS are limited, and cyclosporin A (CSA) is an available option. METHODS: We retrospectively analysed the clinical data of 153 consecutive patients with lower-risk MDS at our institution from July 1997 to October 2017. The propensity score matching method was used to balance the influence of confounding factors between patients with MDS treated with CSA and other conventional treatments (excluding CSA), and 50 pairs of cases were successfully identified for the final analysis. We assessed response rates, progression-free survival (PFS), overall survival (OS), and factors affecting response and survival. RESULTS: Haematological improvement (HI) was observed in 35 (70%) patients treated with CSA and in 25 (50%) patients treated with conventional therapies (p < 0.05). Treatment with CSA was a favourable prognostic factor for HI in lower-risk MDS patients in the entire population in univariate analysis (odds ratio (OR) 2.333, p < 0.05), but not in multivariate analysis. In the multivariate analysis, hypocellular marrow was the only independent prognostic factor for HI in the CSA group (OR 6.259, p < 0.05) and in the overall cohort (OR 3.102, p < 0.05). CSA treatment did not improve PFS or OS (p > 0.05). CONCLUSION: CSA is a safe treatment and can significantly improve cytopenias in a substantial proportion of patients with MDS, especially in individuals with hypocellular bone marrow. However, CSA is not associated with improved PFS or OS.
Acquired von Willebrand Syndrome Associated with a Smoldering Multiple Myeloma, Successfully Treated by Daratumumab, Lenalidomide, and Dexamethasone.
Iarossi M, Vekemans MM, Weynants N … +1 more , Hermans C
Acta Haematol · 2024 · PMID 38359808 · Publisher ↗
INTRODUCTION: Acquired von Willebrand syndrome (AvWS) is a rare entity with approximately 700 cases described in the literature. A number of etiologies are responsible for this condition, mainly lymphoproliferative, myel...
INTRODUCTION: Acquired von Willebrand syndrome (AvWS) is a rare entity with approximately 700 cases described in the literature. A number of etiologies are responsible for this condition, mainly lymphoproliferative, myeloproliferative syndromes and cardiac diseases. Management is aimed at preventing and treating bleeds, as well as treating the underlying pathology. In the case of a monoclonal gammopathy, there are limited evidence and high heterogeneity only based on old case reports, resulting in poor quality recommendations. It seems essential in 2023 to take into account and offer the new anti-myeloma treatments available. CASE PRESENTATION: We describe the case of a patient with an AvWS secondary to an IgG smoldering multiple myeloma, experiencing multiple bleeding, treated successfully with daratumumab, lenalidomide, and dexamethasone, after multiple treatment failure. CONCLUSION: Daratumumab, lenalidomide, and dexamethasone was demonstrated as a rapid and effective treatment for a patient with severe AvWS and multiple bleeding complications.
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