Acta Haematologica[JOURNAL]
Sun
200 papers
BEAC Induction Regimen and Prior Thoracic Radiotherapy Increases the Risk of ASCT-Associated Pneumonitis.
Kaprio E, Jokimäki A, Kuitunen H … +4 more , Kontiainen J, Selander T, Turpeenniemi-Hujanen T, Kuittinen O
Acta Haematol · 2026 Mar · PMID 41802094 · Full text
INTRODUCTION: High-dose chemotherapy (HDT) followed by autologous stem cell transplant (ASCT) has been standard of care in the treatment of relapsed and refractory lymphoma. Some severe toxicities are associated with thi...
INTRODUCTION: High-dose chemotherapy (HDT) followed by autologous stem cell transplant (ASCT) has been standard of care in the treatment of relapsed and refractory lymphoma. Some severe toxicities are associated with this treatment modality. Pulmonary toxicity is one of these significant adverse effects and a potential cause of treatment-related mortality. METHODS: In this retrospective study, we report the incidence, risk factors, and outcome of treatment-induced pneumonitis in 286 lymphoma patients receiving HDT followed by ASCT. RESULTS: The cumulative incidence of treatment-induced pneumonitis was 4.6% and occurred in 11/286 patients. Three months incidence rate was 2.6%. Most of the patients diagnosed with treatment-induced pneumonitis had received BEAC as HDT regimen. The risk of treatment-induced pneumonitis was higher if HDT-ASCT was given in later treatment lines. Also, a prior thoracic radiotherapy as part of first-line treatment was associated with higher risk for pneumonitis after HDT-ASCT. In 2 patients, the pneumonitis did not respond to high-dose steroid treatment. CONCLUSION: This study provides new information about the incidence of pneumonitis associated with HDT-ASCT and its risk factors, which might be useful to take into consideration during and after treatment with HDT-ASCT.
Impact of COVID-19 Pandemic on Progression-Free Survival Estimates in Oncology: The Role of Non-Informative Censoring.
Lesan V, Munteanu C
Acta Haematol · 2026 Mar · PMID 41774606 · Publisher ↗
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Effectiveness of Integrative Medicine in the Management of Lymphoma Survivors: An Exploratory Preference-Based Controlled Trial.
Levy Yurkovski I, Attias S, Schiff E … +8 more , Cohen-Naznin O, Anahory E, Gross-Geva Y, Oved A, Bleiweiss N, Caspi-Biran S, Awad S, Tadmor T
Acta Haematol · 2026 Mar · PMID 41770661 · Full text
INTRODUCTION: Lymphoma has recovery rates above 60%, but many survivors experience impaired quality-of-life (QoL) requiring survivorship care. This study evaluated the role of an integrative oncology (IO) clinic in manag...
INTRODUCTION: Lymphoma has recovery rates above 60%, but many survivors experience impaired quality-of-life (QoL) requiring survivorship care. This study evaluated the role of an integrative oncology (IO) clinic in managing lymphoma survivors. METHODS: In this exploratory preference-based controlled trial, adults in remission after lymphoma treatment were allocated to two groups: those attending the IO survivorship clinic (intervention) and those declining (control). The intervention included complementary medicine, spiritual, and social support, delivered weekly for up to 6 months in addition to standard follow-up. The primary outcome was QoL improvement (EQ-5D-5L index). Secondary outcomes included symptom relief (MYCAW), cognitive function, and perceived control. RESULTS: Twenty-nine patients were enrolled: 15 in the intervention and 14 in the control group. Over the first 3 months, a significant time × group interaction in EQ-5D-5L scores favored the intervention (p = 0.005), reflecting superior QoL trajectory. MYCAW concerns also improved significantly in the intervention group across 6 months (p = 0.005 and p = 0.03). At 3 months, FACT Cog-Oth scores were significantly higher in the intervention arm (p = 0.01), indicating better "other" cognitive functions (e.g., memory, clarity, confusion). To note, adherence to IO mainly decreased after 3 months. CONCLUSION: In this exploratory preference-based study, an IO survivorship clinic for lymphoma survivors was associated with improvements in QoL, especially for adherent patients. Given the preference-based design, these findings should be interpreted cautiously and viewed as hypothesis-generating rather than confirmatory. Further studies are warranted to evaluate long-term benefits and sustainability of this approach.
First Comprehensive Characterization of Lip Lymphoma: A Population-Based Study.
Loap P, Kirova Y
Acta Haematol · 2026 Feb · PMID 41701662 · Full text
INTRODUCTION: Lip lymphoma is an exceptionally rare extranodal lymphoma with limited data to guide clinical management. We conducted the first large-scale population-based analysis to characterize its epidemiology, treat...
INTRODUCTION: Lip lymphoma is an exceptionally rare extranodal lymphoma with limited data to guide clinical management. We conducted the first large-scale population-based analysis to characterize its epidemiology, treatment patterns, and outcomes. METHODS: We identified all lip lymphoma cases (2000-2022) from the SEER database (17 registries). Patients were categorized as localized primary lip lymphoma (stage I) or lymphoma involving the lip area (stages II-IV). RESULTS: Among 82 patients (median age 62 years, 54.9% female), age-adjusted incidence was 0.0459 per million per year. MALT lymphoma predominated (46.3%). With the median follow-up of 74.5 months, 21 deaths (25.6%) occurred, only 5 (6.1%) lymphoma related. Ten-year overall and cancer-specific survival rates were 72.7% and 92.6%, respectively. In multivariate analysis, age (HR 1.115/year, p < 0.001) and non-low-grade histology (HR 3.251, p = 0.043) independently predicted survival. All lymphoma-related deaths occurred in patients with aggressive or unknown histologies, none in confirmed low-grade B-cell lymphomas. No treatment strategy (surgery, radiotherapy, or systemic treatment) showed a significant superiority in terms of survival. CONCLUSION: Lip lymphoma demonstrates excellent prognosis, particularly for low-grade B-cell histologies. Age and histological grade represent principal prognostic factors guiding treatment strategies.
Prognostic Role of Circular RNAs in Mantle Cell Lymphoma: A Competing Endogenous RNA Network Analysis.
Huang CX, Ma XD, Zhuang W … +1 more , Zou Y
Acta Haematol · 2026 Jan · PMID 41615902 · Publisher ↗
INTRODUCTION: This study evaluated the prognostic role of circular RNAs (circRNAs) in individuals diagnosed with mantle cell lymphoma (MCL), using the framework of the competing endogenous RNA (ceRNA) network model. METH...
INTRODUCTION: This study evaluated the prognostic role of circular RNAs (circRNAs) in individuals diagnosed with mantle cell lymphoma (MCL), using the framework of the competing endogenous RNA (ceRNA) network model. METHODS: Differentially expressed circRNAs were identified from the GSE159808 dataset, and differentially expressed messenger RNAs (mRNAs) were extracted from GSE32018. Corresponding microRNAs (miRNAs) were retrieved to construct a ceRNA regulatory network relevant to MCL. Functional enrichment analysis and survival analysis were performed on 35 mRNAs incorporated in the ceRNA network to identify transcripts associated with survival outcomes in MCL. A prognostically relevant ceRNA subnetwork was then constructed based on these results. RESULTS: Thirteen circRNAs (9 upregulated, 4 downregulated) and 457 differentially expressed mRNAs were identified. The initial ceRNA network included 11 circRNAs, 40 miRNAs, and 35 mRNAs. Gene Ontology analysis indicated enrichment in pathways related to dentin-containing tooth development, the plasma membrane signaling receptor complex, and growth factor receptor binding. Kyoto Encyclopedia of Genes and Genomes (KEGG) analysis demonstrated significant involvement in the B-cell receptor signaling pathway, Fc gamma (Fcγ) receptor-mediated phagocytosis, and the Wnt signaling pathway. Survival analysis identified six mRNAs significantly associated with overall survival in individuals with MCL. These results were used to derive a refined prognostic ceRNA network consisting of 6 mRNAs, 7 miRNAs, and 6 circRNAs. CONCLUSION: CircRNAs may regulate MCL prognosis by modulating miRNA-mRNA interactions within the ceRNA network. These findings suggest a regulatory mechanism by which circRNAs contribute to molecular pathways influencing disease progression and survival in MCL.
The Gastro-Intestinal Microbiota in Haematology.
Moreno-Mirón JM, Ruiz-Argüelles GJ, Gallardo-Pérez MM … +3 more , Moreno-Mirón A, Rivera-Aguilar AP, Gale RP
Acta Haematol · 2026 Jan · PMID 41610050 · Full text
BACKGROUND: The gastro-intestinal microbiota is a key regulator of systemic immunity and inflammatory tone and it contributes to normal haematopoiesis through microbial metabolites, barrier integrity, and host-microbe im...
BACKGROUND: The gastro-intestinal microbiota is a key regulator of systemic immunity and inflammatory tone and it contributes to normal haematopoiesis through microbial metabolites, barrier integrity, and host-microbe immune signalling. Disruption of this has been increasingly linked to the development, clinical course, and treatment-related complications of haematological disorders, including clonal haematopoiesis of indeterminate potential (CHIP), leukaemias, and plasma cell neoplasms (PCNs). SUMMARY: This review synthesises current evidence on how gut microbiota composition and function intersect with haematopoietic regulation and haematological disease biology. We summarise proposed mechanisms - including microbe-derived metabolites (e.g., short-chain fatty acids), pattern-recognition receptor signalling, intestinal permeability, and cytokine-mediated inflammation - that may influence haematopoietic stem and progenitor cell behaviour and immune cell differentiation. We then discuss disease-specific associations of dysbiosis with CHIP, leukaemias, and PCN, as well as the impact of common haematology interventions (antibiotics, chemotherapy, immunomodulatory therapies, and transplantation) on microbial ecology and downstream clinical outcomes. Finally, we highlight methodological challenges and outline priorities for longitudinal, mechanistic, and multi-omics studies to enable microbiota-informed risk stratification and therapeutic modulation. KEY MESSAGES: (1) The gut microbiota influences haematopoiesis via immune signalling, microbial metabolites, and maintenance of mucosal barrier function. (2) Dysbiosis is associated with CHIP, leukaemias, and PCN, and may contribute through chronic inflammation and altered immune homeostasis. (3) Haematological therapies frequently reshape the microbiota; these changes may affect infection risk, treatment tolerance, and outcomes. (4) Current evidence is largely associative; rigorously designed longitudinal and interventional studies are needed to establish causality and guide clinical translation.
Prelims.
Acta Haematol · 2026 · PMID 41587135 · Publisher ↗
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International Prognostic Scoring System, Revised International Prognostic Scoring System, and Molecular International Prognostic Scoring System Had No Identical Prognostic Power Depending on Treatment in Myelodysplastic Syndromes.
Abichou G, Loschi M, Sammut R … +8 more , Sedaki B, Benachour S, Fileni C, Ferrero-Vacher C, De Pooter N, Mouanes-Abelin J, Dadone-Montaudie B, Cluzeau T
Acta Haematol · 2026 Jan · PMID 41575889 · Publisher ↗
INTRODUCTION: The Revised International Prognostic Scoring System (IPSS-R) has long been the standard prognostic tool in the management of myelodysplastic syndromes (MDS). Recently, a new clinical-molecular prognostic mo...
INTRODUCTION: The Revised International Prognostic Scoring System (IPSS-R) has long been the standard prognostic tool in the management of myelodysplastic syndromes (MDS). Recently, a new clinical-molecular prognostic model, the IPSS-M, was introduced. This model integrates MDS-related gene mutations and offers improved accuracy and precision in predicting patient outcomes and survival. In this study, we compare the prognostic value and predictive capacity of these prognostic scores across different treatment groups. METHODS: IPSS, IPSS-R, and IPSS-M scores were calculated at diagnosis for all eligible MDS patients in this cohort. Sankey diagrams were generated to visually compare the risk stratification across the three scoring systems. RESULTS: A total of 394 patients were included in this retrospective analysis. During the course of the disease, 59.1% required treatment, with 38.1% (150 patients) receiving erythropoiesis-stimulating agents (ESA) and 19.0% (75 patients) treated with hypomethylating agents (HMA). The IPSS-M was calculated for 281 patients and reclassified 41.9% of cases: 20.3% (n = 57) were upstaged and 21.7% (n = 61) were downstaged. All three scoring systems demonstrated significant stratification of overall survival (OS) in the global cohort (p < 0.001 for IPSS, IPSS-R, and IPSS-M). Notably, IPSS failed to predict OS in patients treated with azacitidine, and none of the scores successfully predicted OS in patients treated with ESA. CONCLUSION: The IPSS-M effectively reclassified 41.9% of patients, upstaging 20.2% and downstaging 21.7%. However, our analysis found that none of the prognostic scores effectively predicted outcomes for ESA-treated patients. Further studies are needed to assess whether the predictive value of these scoring systems is influenced by the specific treatment modalities used.
Chronic Graft-versus-Host Disease: A Review of Current Treatments beyond Second-Line and Emerging Therapies.
Saadeh A, Jibrin D, Haddadin M
Acta Haematol · 2026 Jan · PMID 41525301 · Publisher ↗
BACKGROUND: Chronic graft-versus-host disease (cGVHD) remains a major cause of late morbidity and non-relapse mortality following allogeneic hematopoietic cell transplantation. Early identification and intervention are c...
BACKGROUND: Chronic graft-versus-host disease (cGVHD) remains a major cause of late morbidity and non-relapse mortality following allogeneic hematopoietic cell transplantation. Early identification and intervention are critical to improving outcomes. Corticosteroids continue to serve as the first-line therapy; however, treatment-refractory cases are common and associated with poor outcomes. Ruxolitinib has become the standard second-line agent, yet beyond ruxolitinib, treatment selection varies widely due to a lack of comparative data and standardized sequencing strategies. A comprehensive review of key therapeutic trials in chronic GVHD was conducted, with a focus on second-line treatments and beyond. Emerging and investigational therapies were also included through analysis of recent literature and ongoing studies. SUMMARY: There is currently no established sequencing of agents beyond second-line therapies. The approval of belumosudil, axatilimab, and ibrutinib has expanded therapeutic options, with each agent offering a unique mechanism of action and demonstrating promising organ-specific response rates. KEY MESSAGES: The therapeutic landscape for chronic GVHD is evolving, with several newer agents beginning to demonstrate utility in third-line settings. However, the selection of third-line agents remains largely dependent on clinical judgment, prior treatment history, and the specific organs involved. There is a continuing and critical need for well-designed, comparative trials to establish optimal treatment strategies.
Fluoroquinolone Prophylaxis for Febrile Neutropenia in Autologous Hematopoietic Cell Transplantation: Outcomes and Risk Factors.
Pinto FBR, Meucci FAMO, Cortez AC … +5 more , Cordeiro AC, Bovolenta VDA, Nascimento MM, Schmidt-Filho J, Batista MV
Acta Haematol · 2026 Jan · PMID 41505366 · Publisher ↗
INTRODUCTION: Febrile neutropenia (FN) is a frequent complication following autologous hematopoietic cell transplantation (auto-HCT). Prophylactic use of fluoroquinolones (FQs) has been shown to reduce FN incidence; howe...
INTRODUCTION: Febrile neutropenia (FN) is a frequent complication following autologous hematopoietic cell transplantation (auto-HCT). Prophylactic use of fluoroquinolones (FQs) has been shown to reduce FN incidence; however, its routine use remains controversial due to increasing concerns regarding antimicrobial resistance and uncertain impact on clinically relevant outcomes. METHODS: We conducted a retrospective cohort study including adult patients who underwent auto-HCT at A.C.Camargo Cancer Center between 2016 and 2021. Patients were divided into two groups according to institutional prophylaxis policy: those who received levofloxacin prophylaxis during neutropenia between 2016 and 2018 (Px group, n = 201) and those who did not receive FQ prophylaxis between 2018 and 2021 (NPx group, n = 169). The primary outcome was the incidence of FN. Secondary outcomes included microbiologically documented infections, antimicrobial resistance patterns, and 30- and 100-day all-cause mortality. RESULTS: FN occurred more frequently in the NPx group compared with the Px group (92% vs. 81%, p = 0.002). There were no significant differences between groups in the rate of microbiologically documented infections (23% vs. 14%, p = 0.09), 30-day mortality (3% vs. 1%, p = 0.25), or 100-day mortality (3% vs. 2%, p = 0.48). Antimicrobial resistance profiles differed significantly: the Px group had a lower proportion of pan-susceptible isolates (17% vs. 61%) and higher rates of extended-spectrum β-lactamase-producing organisms (27% vs. 6%) and methicillin-resistant bacteria (36% vs. 10%) compared with the NPx group (all p < 0.001). CONCLUSION: Although FQ prophylaxis was associated with a lower incidence of FN after auto-HCT, it did not translate into reductions in short-term mortality and was associated with significantly higher rates of antimicrobial resistance. Based on these findings, our institution does not recommend routine FQ prophylaxis for patients undergoing auto-HCT.
Improving Cure Rates of B-Cell Acute Lymphoblastic Leukemia with Chimeric Antigen Receptor T Cells.
Valtis YK, Park JH
Acta Haematol · 2025 Dec · PMID 41474667 · Publisher ↗
BACKGROUND: Chimeric antigen receptor T (CAR T) cell therapy was first approved for the treatment of children and young adults with relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL). Since then, two additional...
BACKGROUND: Chimeric antigen receptor T (CAR T) cell therapy was first approved for the treatment of children and young adults with relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL). Since then, two additional CAR T products have been approved for adult R/R B-ALL. SUMMARY: Through clinical trials and real-world evidence, we have learned that CAR T therapies generally lead to high upfront response rates with usually deep remissions, negative for measurable residual disease (MRD). Despite that, many patients eventually relapse, prompting a need for better prognostication and techniques to prolong remissions and maximize cures. Here, we focus our review on previously known and recently discovered prognostic factors for sustained remissions and potential avenues for improved cure rates with CAR T therapy for ALL. Lastly, we comment on the importance of removing barriers to accessing CAR T cells for patients with ALL. KEY MESSAGES: Relevant prognostic factors vary across products and clinical settings. To improve cure rates, investigators are designing new CAR T products, using CAR T cells in earlier treatment settings, and focusing on addressing access barriers.
Post-Transplant Lymphoproliferative Disorder following Heart Transplantation Presenting as a Retroperitoneal Plasmacytoma: A Case Report and Review of the Literature.
Vilian Y, Sherban A, Goldberg E … +8 more , Wax T, Cohen E, Hezkelo N, Sheena L, Vaxman I, Raanani P, Krause I, Goldberg I
Acta Haematol · 2025 Dec · PMID 41442435 · Full text
INTRODUCTION: Plasma cell neoplasms (PCNs) are a rare type of post-transplant lymphoproliferative disease (PTLD). Extramedullary plasmacytoma is a rare type of PCN, and is infrequently located in the retroperitoneum. Acc...
INTRODUCTION: Plasma cell neoplasms (PCNs) are a rare type of post-transplant lymphoproliferative disease (PTLD). Extramedullary plasmacytoma is a rare type of PCN, and is infrequently located in the retroperitoneum. According to the current literature, heart transplant recipients are considered at intermediate risk for developing PTLD. CASE PRESENTATION: We report a case of a 70-year-old patient with a history of heart transplantation, who was admitted to the hospital with back pain and swelling of the left lower limb. Imaging revealed a large retroperitoneal mass involving the left kidney, descending colon, left iliopsoas muscle, bladder, and prostate. Histopathological analysis confirmed the diagnosis of extramedullary plasmacytoma. Immunosuppressive therapy was reduced and chemotherapy was initiated; however, the patient passed away after two cycles of treatment. CONCLUSION: To the best of our knowledge, only once has retroperitoneal plasmacytoma in a heart transplant recipient been previously reported. Our case should raise suspicion for recognizing and considering this rare diagnosis in similar clinical scenarios.
A Case Series of SGLT2i Improving Hemoglobin in Adults with Myelodysplastic Syndrome.
Olar P, Lee S, Fralick M
Acta Haematol · 2025 Dec · PMID 41442419 · Publisher ↗
INTRODUCTION: The objective of our case series was to assess the change in hemoglobin for adults with myelodysplastic syndrome (MDS) started on a sodium glucose cotransporter 2 inhibitor (SGLT2i) for their diabetes, hear...
INTRODUCTION: The objective of our case series was to assess the change in hemoglobin for adults with myelodysplastic syndrome (MDS) started on a sodium glucose cotransporter 2 inhibitor (SGLT2i) for their diabetes, heart failure, or chronic kidney disease. CASE PRESENTATION: Four patients with bone marrow biopsy confirmed low- or intermediate-risk MDS were referred for consideration of SGLT2i between July 2024 and December 2024. The patients were followed prospectively and had a repeat complete blood count at least 3 months after starting empagliflozin at 10 mg daily. The patients provided written consent for their results to be published. Median age was 78 years (range 76-82 years), 3 of the 4 patients were men, and the median baseline hemoglobin was 11.6 g/dL (IQR 9.3-12.8). Over a median duration of 4 months of taking empagliflozin, the median repeat hemoglobin was 12.8 g/dL (IQR 10.3-13.7). CONCLUSIONS: In our case series of 4 patients with MDS started on empagliflozin, we observed an increase in hemoglobin for all 4 patients. Larger studies are needed to assess whether the increase in hemoglobin is sufficiently robust and sustained to reduce a person's need for future red blood cell transfusion or other treatments for MDS.
Hans-Based Cell of Origin Is Not of Prognostic Significance in Diffuse Large B-Cell Lymphoma Patients: A Retrospective Single-Center Study Conducted in 326 RCHOP-Treated Patients.
Symeonidou M, Intzes SG, Zagoridis K … +14 more , Karatisidis L, Mprotsis T, Kanelis G, Vrachiolias G, Stamatiou I, Bezirgiannidou Z, Malkots B, Papoutselis M, Roumpakis C, Skendros P, Tsikouras P, Liapis K, Kotsianidis I, Spanoudakis E
Acta Haematol · 2025 Dec · PMID 41411222 · Publisher ↗
INTRODUCTION: Diffuse large B-cell lymphoma (DLBCL) is a highly aggressive lymphoma with dismal outcome after disease progression. Individual risk assessment can stratify patients into different treatment strategies. MET...
INTRODUCTION: Diffuse large B-cell lymphoma (DLBCL) is a highly aggressive lymphoma with dismal outcome after disease progression. Individual risk assessment can stratify patients into different treatment strategies. METHODS: We retrospectively collected data from 326 DLBCL patients treated in a single center with RCHOP from 01/2000 to 06/2023. Immunohistochemistry by Han's algorithm was used to classify patients according to cell of origin (COO). The Kaplan-Meier estimator of survival and Cox regression analysis were used. RESULTS: Time to progression (TTP) and overall survival (OS) were not different according to COO. Univariate analysis reveals International Prognostic Index (IPI), b2-microglobulin ≥3.5 mg/L, bulky disease, and abnormal LDH, but not Han's defined COO, as the strongest predictors for progression. IPI score in multivariate analysis was the only prognostic factor for OS and together with high b2-microglobulin levels were independently prognostic factors for TTP. Accordingly, b2-microglobulin ≥3.5 mg/L was an independent prognostic factor for progression both in GC (hazard ratio [HR] 0.249 [(95% CI: 0.087-0.649), p = 0.004] and non-GC DLBCL patients (HR 0.380 [95% CI: 0.177-0.813], p = 0.011). CONCLUSION: COO according to Hans index is not a significant prognostic factor for DLBCL patients, but b2-microglobulin ≥3.5 mg/L and IPI 2-5 in both GC and non-GC patients can predict individually a higher risk for progression.
Anexelekto (Axl)/Mer Inhibitor Tamnorzatinib in Patients with Relapsed/Refractory Acute Myeloid Leukaemia: Results from a Phase I (Monotherapy) and Phase II (Combination with Venetoclax) Clinical Study.
Kasner MT, Courtenay-Luck N, DiNardo C … +10 more , Post SM, Baratam P, Magrath GN, Nakamura T, Fujii A, Prados S, Honda N, Mcbride M, Edwards-Holmes P, Stuart R
Acta Haematol · 2025 Dec · PMID 41385448 · Full text
INTRODUCTION: Relapsed/refractory (R/R) acute myeloid leukaemia (AML) is a life-threatening haematological malignancy without effective treatments. Anexelekto (Axl) and Mer receptor tyrosine kinases have emerged as impor...
INTRODUCTION: Relapsed/refractory (R/R) acute myeloid leukaemia (AML) is a life-threatening haematological malignancy without effective treatments. Anexelekto (Axl) and Mer receptor tyrosine kinases have emerged as important therapeutic targets in AML for their crucial role in survival of AML cells. Tamnorzatinib (ONO-7475) is a potent and highly selective inhibitor of Axl/Mer. We report first-in-human study of tamnorzatinib (NCT03176277) in patients with R/R AML. METHODS: Tamnorzatinib was administered as monotherapy (n = 20) to determine an appropriate biological dose of tamnorzatinib and then in combination (n = 22) with venetoclax to evaluate safety and clinical efficacy. RESULTS: Tamnorzatinib was safe and well tolerated as monotherapy (3, 6, and 10 mg) and in combination (6 mg) with venetoclax. No dose-limiting toxicities were observed at any dose level. Near-maximal Axl/Mer inhibition was observed following 6 mg tamnorzatinib alone and in combination therapy. No complete remission (CR) with partial haematologic recovery was observed with combination therapy. However, decreased transfusion dependency was observed; in the venetoclax-resistant subgroup (n = 14), 1 (7.1%) patient achieved CR with incomplete haematologic recovery and 1 (7.1%) patient achieved morphologic leukaemia-free state. CONCLUSION: Tamnorzatinib alone and in combination with venetoclax was safe and well tolerated but failed to induce robust clinical efficacy in R/R AML.
Patients with Prior Cerebral Venous Sinus Thrombosis Presenting to the Emergency Department with Neurological Symptoms: The Yield of Neuroimaging.
Grishin E, Budnik I, Efros O … +4 more , Cohen O, Kenet G, Levy-Mendelovich S, Barg AA
Acta Haematol · 2025 Dec · PMID 41379744 · Full text
INTRODUCTION: Cerebral venous sinus thrombosis (CVST) is a rare but potentially life-threatening condition. Despite advances in diagnostic imaging and therapeutic strategies, data regarding risk factors for CVST progress...
INTRODUCTION: Cerebral venous sinus thrombosis (CVST) is a rare but potentially life-threatening condition. Despite advances in diagnostic imaging and therapeutic strategies, data regarding risk factors for CVST progression among patients remain limited. METHODS: The retrospective cohort study aimed to evaluate the yield of neuroimaging and to assess risk factors for CVST progression among patients presenting to the emergency department (ED) after a prior CVST diagnosis. We collected data from the hospital's electronic medical records on patients diagnosed with CVST at our tertiary care center between January 2002 and April 2023. For patients who had subsequent ED visits related to their initial CVST diagnosis, data regarding demographics, clinical presentation, imaging outcomes, and alterations in therapeutic management were retrieved. RESULTS: Our initial cohort included 251 patients diagnosed with CVST. Of these, 107 (43%) patients returned to the ED with symptoms potentially related to CVST. Headache was the most common presenting symptom (59%), and imaging was performed in 71% of relevant ED visits. Thrombus progression was observed in only 6% of cases. No significant associations were found between demographic factors, clinical presentation, anticoagulation status, and neuroimaging findings. Among patients whose initial CVST diagnosis occurred more than 2 years prior to ED evaluation, only those with thrombophilia experienced thrombus progression. CONCLUSIONS: Thrombus progression is a rare finding among patients with a history of CVST presenting to the ED with neurological complaints. Pediatric patients showed low rates of thrombotic worsening, suggesting a more judicious use of neuroimaging in this population. No significant risk factor was found to predict the risk of CVST progression.
Hemophagocytic Syndromes in Adults: Real-World Data on Mortality from a Tertiary Reference Center.
Wojtovicova T, Aujesky D, Schefold JC … +16 more , Daskalakis M, Furrer H, Novak U, Pabst T, Maurer B, Möller B, Abegglen RC, Exadaktylos A, Berzigotti A, Banz Y, Bacher U, Zeerleder SS, Bonadies N, Tichelli A, Angelillo-Scherrer A, Rovó A
Acta Haematol · 2025 Dec · PMID 41329624 · Full text
INTRODUCTION: Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by excessive immune activation, cytokine storm, and aberrant macrophage function. Although HLH is well studied in...
INTRODUCTION: Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by excessive immune activation, cytokine storm, and aberrant macrophage function. Although HLH is well studied in children, data on adult HLH remain limited. Our primary goal was to examine in-hospital mortality and its associated risk factors in patients with HLH in a tertiary center. METHODS: This retrospective study at University Hospital Bern queried the hospital database using the i2b2 system to analyze adult HLH patients, assessing clinical, laboratory data, treatments, and outcomes according to the HLH-2004 criteria and Saint-Antoine score. RESULTS: From 845,846 patients seen in the hospital between 2014 and 2021, a cohort of 54 adult HLH patients was identified. The overall mortality rate was 40.7%. In univariate analysis, we found that deceased patients with HLH were significantly older than surviving patients (median age of 69.6 [range 22-83] vs. 52.5 [24-79] years old [p = 0.002]). Patients with HLH were significantly more likely to have cardiopulmonary and neurological complications, higher alkaline phosphatase levels, lower platelet counts, need platelet transfusions, and lower response rate to the HLH therapy. In multivariate analysis, age (HR 0.94; 95% CI 0.89-0.99; p = 0.024), cardiopulmonary (HR 7.045; 95% CI 1.28-38.66, p = 0.025), neurologic complications (HR 5.55; 95% CI 1.01-30.51; p = 0.04), and the requirement of platelet transfusions (HR 6.22; 95% CI 1.16-33.20; p = 0.032) were all independently associated with in-hospital mortality. CONCLUSIONS: This study identifies risk factors whose early presence can be used to stratify management strategies and improve prognosis in patients with HLH.
Minimal Clonal Plasma Cell Contamination of Peripheral Stem Cell Grafts Has an Adverse Prognostic Impact in Patients with Multiple Myeloma Undergoing Autologous Transplantation.
Liu Y, Shao D, Chang Y … +8 more , Wang Y, Dou X, Peng N, Wen L, Wang F, Huang X, Mo X, Lu J
Acta Haematol · 2025 Nov · PMID 41252321 · Publisher ↗
INTRODUCTION: The significance of autografts' contamination by clonal plasma cells on clinical outcome in newly diagnosed multiple myeloma (NDMM) remains controversial. METHODS: We retrospectively reviewed the clinical a...
INTRODUCTION: The significance of autografts' contamination by clonal plasma cells on clinical outcome in newly diagnosed multiple myeloma (NDMM) remains controversial. METHODS: We retrospectively reviewed the clinical and laboratory data of NDMM patients who underwent autologous stem cell transplantation (ASCT) and had received graft minimal residual disease (gMRD) examination by multi-color flow cytometry. RESULTS: From January 2011 to December 2022, 250 NDMM patients with complete cytogenetic information, gMRD information, and who received ASCT as consolidation were enrolled. Multi-flow cytometry can achieve a median detection sensitivity of 0.004%, and gMRD positivity was 12.4% at a median level of 0.0160% (interquartile range, 0.0049%, 0.05394%). Its presence was correlated with response to induction treatment, with percentages of 2.65%, 12.94%, 28.89%, and 57.14% of patients achieving complete response, very good partial response, partial response, and minimal response/stable disease, respectively. gMRD (+) patients had a higher risk of not achieving bone marrow MRD negativity post-ASCT. After a median follow-up of 33.5 months for the whole cohort, patients in the gMRD (+) group had significantly worse progression-free survival (PFS) than those in the gMRD (-) group did (34.8 vs. 65.0 months, p = 0.001). Multivariable analysis revealed that gMRD (-) was independently predictive of better PFS (hazard ratio 0.464, 95% confidence interval: 0.274-0.785, p = 0.004). We found the significance of gMRD on PFS was in high-risk subgroups and in patients who achieved ≤ partial response prior to ASCT. CONCLUSIONS: In conclusion, gMRD (+) was an independent risk factor for inferior progression-free survival, with the impact primarily affecting high-risk groups and patients who achieved ≤ partial response before ASCT.
Imatinib Treatment in Primary Acute Basophilic Leukemia with FIP1L1-PDGFRα Rearrangement: A Case Report.
Wu J, Li J, Yin J … +5 more , Huang D, Yi J, Lei Y, Guo Q, Zhang Z
Acta Haematol · 2025 Nov · PMID 41237070 · Publisher ↗
INTRODUCTION: Acute basophilic leukemia (ABL) is a rare form of acute leukemia, characterized by a high number of immature basophils in the blood. It is clinically associated with skin infiltration, organ enlargement, os...
INTRODUCTION: Acute basophilic leukemia (ABL) is a rare form of acute leukemia, characterized by a high number of immature basophils in the blood. It is clinically associated with skin infiltration, organ enlargement, osteolytic lesions, and symptoms of histamine excess, with rapid progression and poor prognosis. Due to its rarity and the lack of specialized diagnostic tests, there is no universally accepted diagnostic standard. CASE PRESENTATIONS: We report a rare case of ABL, which advances our understanding of the clinical manifestations and pathological mechanisms of the disease. The patient presented with symptoms indicative of hyperhistaminemia, and the diagnosis was confirmed through molecular testing, specifically detecting the FIP1L1::PDGFRA fusion gene. Detailed analysis of this case helped identify early symptoms and highlighted the relevance of hyperhistaminemia in ABL's clinical presentation. CONCLUSION: The patient successfully achieved a complete response to treatment and remained relapse-free during an 18-month follow-up. This case underscores the importance of accurate and timely diagnosis and individualized treatment, and it provides valuable insights for managing similar cases of ABL.
Diagnosis and Management of Philadelphia-Like Acute Lymphoblastic Leukemia in Adults.
Pourhassan H, Lee WY, Schwartz M … +2 more , Pullarkat V, Aldoss I
Acta Haematol · 2026 · PMID 41144606 · Publisher ↗
BACKGROUND: The entity of Philadelphia (Ph)-like acute lymphoblastic leukemia (ALL) accounts for approximately 20-30% of newly diagnosed adults with B-cell ALL cases in the USA. Compared to other B-cell subtypes, Ph-like...
BACKGROUND: The entity of Philadelphia (Ph)-like acute lymphoblastic leukemia (ALL) accounts for approximately 20-30% of newly diagnosed adults with B-cell ALL cases in the USA. Compared to other B-cell subtypes, Ph-like ALL is associated with overall poor prognosis and inferior outcomes with high measurable residual disease rates following induction therapy, increased risk of treatment failure and relapse, as well as short event-free and overall survival. SUMMARY: Here we aim to highlight Ph-like ALL genetic subtypes and methods of genomic profiling for diagnosis and disease prognostication and to summarize current management approaches for frontline treatment including multiagent chemotherapy, immunotherapy, tyrosine kinase and small molecule inhibitors, and the role of allogeneic stem cell transplantation. KEY MESSAGES: Despite the improvement in the treatment outcomes of adult patients with newly diagnosed B-cell ALL, patients with Ph-like ALL continue to do poorly with standard therapy. Thus, tailored therapeutic studies are indeed warranted to refine frontline treatment approaches and to improve outcomes for patients with Ph-like ALL.
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