Acta Haematologica[JOURNAL]
Sun
200 papers
Dapagliflozin in Patients with homozygous β-Thalassemia and Albuminuria.
Tsiotsios K, Vamvakas SS, Labropoulou V … +10 more , Lazaris V, Davoulou P, Kalavrizioti D, Bratsiakou A, Paraskevas T, Kourakli A, Goumenos D, Papachristou E, Symeonidis A, Papasotiriou M
Acta Haematol · 2026 Jun · PMID 42360912 · Publisher ↗
Introduction Patients with homozygous β-thalassemia show continuously improved survival, which has contributed to the development of kidney complications, predominantly expressed as increased albuminuria. In particular,...
Introduction Patients with homozygous β-thalassemia show continuously improved survival, which has contributed to the development of kidney complications, predominantly expressed as increased albuminuria. In particular, patients with transfusion-dependent thalassemia (TDT), frequently develop chronic kidney complications related to chronic anemia, iron overload, and exposure to iron chelation therapy. Although sodium-glucose co-transporter 2 inhibitors (SGLT-2is) have demonstrated renoprotective effects in patients with chronic kidney disease (CKD) from other causes, their efficacy in patients with homozygous β-thalassemia and established CKD remains unknown. Methods In this prospective, open-label study we investigated the impact of dapagliflozin (10 mg/day), in patients with homozygous β-thalassemia and an albumin to creatinine ratio (ACR)>30 mg/g. Study end-points included the effect on ACR and kidney function at 6 months after treatment initiation. Furthermore, we examined the effect on systolic (SBP) and diastolic blood pressure (DBP), and on urine monocyte chemoattractant protein-1 (MCP-1) and matrix metalloproteinase-9 (MMP-9) levels. Results Twelve patients with ACR>30 mg/g received dapagliflozin and 18 patients with normal ACR were followed up for 6 months. Twenty three healthy adult individuals age- and sex-matched to the β-thalassemia group were used as normal controls. Patients with ACR>30 mg/g showed a significant reduction of ACR and SBP at 6 months (p=0.034, p=0.025 respectively) while kidney function remained stable. Patients with homozygous β-thalassemia showed increased baseline urine MCP-1 levels, while MMP-9 showed no differences versus normal controls. Those, who received dapagliflozin exhibited a significant decrease in urine MCP-1 (p=0.022) while MMP-9 remained stable. There were no significant adverse events. Conclusion Dapagliflozin at a standard dose of 10 mg daily is safe and effectively reduces albuminuria and urinary MCP-1 in patients with homozygous β-thalassemia and established CKD, suggesting potential kidney anti-inflammatory effects. Further studies with larger cohorts and longer follow-up are needed to determine its impact on CKD progression and long-term outcomes.
Efficacy of second-generation FLT3 inhibitors in FLT3-mutated AML: A meta-analysis of randomized controlled trials.
Yu Q, Cao J, Chen Y … +3 more , Ma X, Hu K, Qiu Y
Acta Haematol · 2026 Jun · PMID 42319886 · Publisher ↗
BACKGROUND: Second-generation FLT3 inhibitors have demonstrated clinical efficacy in FLT3-mutated acute myeloid leukemia (AML), although results across randomized trials remain inconsistent. This meta-analysis aimed to e...
BACKGROUND: Second-generation FLT3 inhibitors have demonstrated clinical efficacy in FLT3-mutated acute myeloid leukemia (AML), although results across randomized trials remain inconsistent. This meta-analysis aimed to evaluate the efficacy of second-generation FLT3 inhibitors in patients with FLT3-mutated AML. METHODS: We systematically searched databases including PubMed, Web of Science, and the Cochrane Library (from inception to August 2025) to identify randomized controlled trials (RCTs) evaluating second-generation FLT3 inhibitors for treating FLT3-mutated AML. The primary endpoints were overall survival (OS) and progression-free survival (PFS). A random-effects model was used to calculate pooled hazard ratios (HRs) and their 95% confidence intervals (95% CIs). The study protocol was registered in PROSPERO (CRD420251132477). RESULTS: This meta-analysis included eight RCTs involving 2,231 patients. The second-generation FLT3 inhibitors evaluated were gilteritinib and quizartinib, and control treatments included placebo or active non-FLT3-inhibitor regimens. Patients treated with second-generation FLT3 inhibitors exhibited significant improvements in both OS (HR: 0.72, 95% CI: 0.62-0.84; P<0.001) and PFS (HR: 0.73, 95% CI: 0.63-0.86; P<0.001). Subgroup analysis revealed that gilteritinib showed improvements in both OS (HR: 0.71, P=0.001) and PFS (HR: 0.71, P<0.001), and quizartinib significantly improved OS (HR: 0.72, P=0.013) with no significant difference in PFS (HR: 0.75, P=0.142). Among patients with newly diagnosed AML, second-generation FLT3 inhibitors did not significantly improve PFS (HR: 0.73, P=0.127) but significantly improved OS (HR: 0.80, P=0.037). Among patients with relapsed/refractory AML, significant benefits were observed in both OS (HR: 0.67, P<0.001) and PFS (HR: 0.74, P=0.04). Benefits were observed across both placebo-controlled and active-comparator trials. CONCLUSION: Second-generation FLT3 inhibitors significantly improve survival outcomes in FLT3-mutated AML, particularly for gilteritinib and in relapsed/refractory disease. Further studies are needed to clarify mutation subtype-specific and dose-specific effects.
Limited prognostic value of ELN classification and relevance of molecular ontogeny in acute myeloid leukemia post myeloproliferative neoplasms: a retrospective multicenter study.
Chiche E, Fileni C, Bonnet S … +16 more , Bertoli S, Kuykendall AT, Gastaud L, Loschi M, Venton G, De Mas V, Delabesse E, Marceau-Renaut A, Preudhomme C, Courtier F, Murati A, Dadone-Montaudie B, Sallman D, Récher C, Vey N, Cluzeau T
Acta Haematol · 2026 Jun · PMID 42296061 · Publisher ↗
Acute myeloid leukemia (AML) post myeloproliferative neoplasm (MPN) have very poor prognosis and are often excluded from most clinical trials. We retrospectively collected data from 166 patients, including 156 with avail...
Acute myeloid leukemia (AML) post myeloproliferative neoplasm (MPN) have very poor prognosis and are often excluded from most clinical trials. We retrospectively collected data from 166 patients, including 156 with available treatment data and 83 with NGS data treated in France and US. 2022 ELN risk categories were favorable, intermediate and adverse in 3 (2%), 17 (13%) and 110 (85%), respectively. ORR was 57%, 20% and 25% in patients treated by intensive chemotherapy (IC), hypomethylating agents (HMA) and BSC (including low intensity treatments as hydroxyurea and low-dose cytarabine), respectively. 31 patients (22%) underwent allogeneic stem cell transplantation (ASCT). Median overall survival (OS) was 7.2 months without significant difference between IC and HMA (9,5 and 8,6 months, respectively). OS was significantly improved in patients allografted (6.7 vs 1.3 months, respectively, p<0.001). Even though 2017 and 2022 ELN risk categories were not prognostic for OS, we observed a prognostic impact on OS of Lindsley's classifier (p=0.014). In multivariate analysis, JAK2 mutation was associated with worse OS (p=0.037), whereas SRSF2 showed a trend toward adverse prognosis (p=0.057). Among functional groups, only spliceosome mutations predicted poor prognosis (p=0.015). In conclusion, we confirmed poor prognosis of AML post MPN. Classical prognostic classification was not validated in our cohort. We observed poor outcome using IC or HMA encouraging us to propose new clinical trials in this specific subgroup. Only ASCT was able to improve prognosis.
Real-World Data on First Relapse in Multiple Myeloma: Treatment Patterns and Outcomes in Finland Using Automated Data Collection.
Vikkula J, Remes K, Laatikainen O … +3 more , Utriainen M, Tikka S, Lievonen J
Acta Haematol · 2026 Jun · PMID 42284247 · Publisher ↗
OBJECTIVES: This study aimed to characterize multiple myeloma (MM) patients at the first relapse and examine treatment outcomes. METHODS: This registry-based retrospective study examined relapsed multiple myeloma patient...
OBJECTIVES: This study aimed to characterize multiple myeloma (MM) patients at the first relapse and examine treatment outcomes. METHODS: This registry-based retrospective study examined relapsed multiple myeloma patients in two Finnish Wellbeing Services Counties (Helsinki and Uusimaa, and Southwest Finland) from 2013 to 2022. Relapse was identified per current guidelines using serum paraprotein, plasma immunoglobulins, and free light chain data. Data were collected from hospital data lakes, Social Insurance Institution and Digital and Population Services Agency. Patients were stratified by age (≤70 vs >70), index year, and time to first relapse (≤2 vs >2 years post-diagnosis). RESULTS: Of 1,221 patients treated for MM between 2012-2022, 286 met the study inclusion criteria. Median overall survival from the relapse was higher in the younger patients (32.4 months vs 20.0 months). In the first half of follow-up, lenalidomide was the most common pharmacotherapy in both age groups. After 2018, CD38-based treatments and pomalidomide increased significantly for younger patients, remaining limited in the elderly. CONCLUSIONS: While new therapies have been adopted for younger MM patients, the treatment of older patients at first relapse has had little change. Given their poorer outcomes, there is a need to improve care for older MM patients.
Comparative efficacy of donor lymphocyte infusions in augmenting graft-versus-leukemia effect after allogeneic hematopoietic stem cell transplantation for patients with myeloid malignancies.
Abdelgamid M, Hashi NJ, Hefazi M … +18 more , Matin A, Gauto Mariotti E, Hogan WJ, Litzow MR, Alkhateeb HB, Shah MV, Durani U, Kenderian SS, Foran JM, Murthy HS, Kharfan-Dabaja MA, Roy V, Ayala E, Patnaik MM, Dingli D, Diguardo MA, Jacob EK, Mangaonkar AA
Acta Haematol · 2026 Jun · PMID 42258587 · Publisher ↗
BACKGROUND: The efficacy of donor lymphocyte infusions (DLI) among various myeloid malignancies (particularly, genetic subtypes) and the optimal timing of DLI initiation remains unclear. METHODS: This was a retrospective...
BACKGROUND: The efficacy of donor lymphocyte infusions (DLI) among various myeloid malignancies (particularly, genetic subtypes) and the optimal timing of DLI initiation remains unclear. METHODS: This was a retrospective study of 62 patients with myeloid malignancies treated with an alloHSCT and DLI from years 2001-2022. DLI indication was therapeutic 55 (89%), pre-emptive 6 (10%) and prophylactic 1 (2%) with complete remission (CR/CRi) in twenty patients with common diagnoses being 9 (45%) acute myeloid leukemia, 6 (30%) myelodysplastic syndromes, and 3 (15%) chronic myelomonocytic leukemia among others. RESULTS: Among patients who received therapeutic DLI (n=55), the best response was CR/CRi in 16 (29%) patients. At a median follow-up from the date of DLI of 75 (95% CI 35-121) months, there were 49 (79%) deaths with a median OS of 6 (95% CI 4-14) months, higher in patients in CR/CRi versus no CR/CRi (median 51 versus 4 months, P=0.008). Presence of cytogenetic abnormalities such as complex karyotype, deletions in chromosome 5, 7 and 17p, and mutations in TP53, KRAS, NRAS, RUNX1 or JAK2 were associated with adverse outcomes. CONCLUSION: DLI is an effective treatment strategy for post-transplant relapse of all myeloid malignancies, with specific genetic subtypes showing poorer outcomes and survival.
COVID-19 infection in patients with follicular lymphoma and related indolent B-cell lymphomas receiving anti-CD20 monoclonal antibody maintenance therapy.
Kuniyoshi S, Tanaka K, Akuzawa Y … +7 more , Homma S, Okura M, Okamura T, Kiguchi T, Sugimori H, Hashimoto K, Tamura H
Acta Haematol · 2026 Jun · PMID 42258574 · Publisher ↗
INTRODUCTION: Coronavirus disease 2019 (COVID-19) poses significant risks to immunocompromised individuals. Patients receiving maintenance therapy with anti-CD20 monoclonal antibodies for follicular lymphoma (FL) may be...
INTRODUCTION: Coronavirus disease 2019 (COVID-19) poses significant risks to immunocompromised individuals. Patients receiving maintenance therapy with anti-CD20 monoclonal antibodies for follicular lymphoma (FL) may be especially vulnerable because of prolonged B-cell depletion. However, data from Japan, particularly from the later stages of the pandemic, remain limited. This study aimed to investigate the incidence, severity, duration, mortality, and treatment discontinuation associated with COVID-19 in this population. METHODS: We retrospectively analyzed all consecutive 72 patients with FL or related indolent B-cell lymphomas, including transformed FL, who received anti-CD20 maintenance therapy (rituximab or obinutuzumab) at our institute between January 2020 and September 2023. All patients had an indolent lymphoma background. Data on clinical features, laboratory values, disease severity at initial diagnosis according to Ministry of Health, Labour and Welfare guidelines, hospitalization, outcomes, duration of PCR positivity (>21 days defined as prolonged), and maintenance therapy continuation were reviewed. Fisher's exact test was used for analysis. RESULTS: Among the 72 patients (median age: 72 years), 16 (22.2%) developed COVID-19. Of these, 5 had received rituximab and 11 obinutuzumab. The median lymphocyte count at infection was 498/μL; no patient had serum IgG <500 mg/dL. Eight infected patients developed oxygen-requiring disease at initial assessment (50.0% of infected patients; 11.1% overall). Prolonged infection occurred in 9 patients (56.3% of infected patients; 12.5% overall). All four deaths were COVID-19-related and occurred in obinutuzumab recipients (25.0% of infected patients; 5.6% overall). Lymphocyte count <200/μL was associated with mortality (p = 0.027), although this finding should be interpreted cautiously because of the small number of events. Maintenance therapy was discontinued in 5 of 8 patients infected during therapy. CONCLUSION: COVID-19 infection during anti-CD20 maintenance therapy led to severe illness and prolonged viral shedding in some cases. Severe lymphopenia may be a potential risk marker for mortality, but this finding should be interpreted cautiously because of the exploratory nature of the analysis.
Shared Care Offers an Effective Framework for Managing Tagraxofusp Treatment in Blastic Plasmacytoid Dendritic Cell Neoplasm.
Ambinder A, LeBlanc TW, Levy MY … +5 more , Patel AB, McCloskey J, Duffield E, Katsetos J, Mannis G
Acta Haematol · 2026 Jun · PMID 42241367 · Publisher ↗
Tagraxofusp, a first-in-class CD123-targeted therapy, is the only approved treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN). Shared-care models enable coordination and care delivery between specialized...
Tagraxofusp, a first-in-class CD123-targeted therapy, is the only approved treatment for blastic plasmacytoid dendritic cell neoplasm (BPDCN). Shared-care models enable coordination and care delivery between specialized cancer treatment centers and local institutions, allowing eligible patients to receive tagraxofusp. This review provides guidance on best practices for effective shared-care implementation for tagraxofusp in patients with BPDCN, including appropriate patient selection, adequate local institutional readiness, proactive collaboration and communication between institutions, integrated co-management between institutions, and requisite staff, patient, and caregiver education. Key Messages: Shared care is particularly suitable for tagraxofusp treatment, as adverse events can be grade 3 or greater, but typically only occur in cycle 1, if they are to occur at all. Based on clinical experience, this article discusses the application of a shared-care model for tagraxofusp treatment in BPDCN.
Clinical effect analysis of frozen-thawed-deglycerolized red blood cells.
Wang Y, Rao Q, Ma Y … +2 more , Zhang Y, Li X
Acta Haematol · 2026 May · PMID 42154638 · Publisher ↗
Objective： To analyze the clinical infusion effect of frozen-thawed-deglycerolized red blood cells (FTD-RBCs) and provide solutions for situations such as blood supply shortages. Methods： In this retrospective study, 2...
Objective： To analyze the clinical infusion effect of frozen-thawed-deglycerolized red blood cells (FTD-RBCs) and provide solutions for situations such as blood supply shortages. Methods： In this retrospective study, 280 patients receiving FTD-RBCs and 280 patients receiving suspended red blood cells (SRBCs) in our hospital from December 2019 to June 2022 were included. We collected the course records and laboratory test results, including basic information, total amount of transfusion, as well as RBC count, hemoglobin concentration (HGB) and hematocrit（HCT）24 h before and after the transfusion, and whether there were adverse blood transfusion reactions. Clinical efficacy analysis of FTD-RBCs was conducted by Mann-Whitney U test and Kruskal-Wallis H test. Results: In the 280 patients of our study, RBC count, HGB and HCT increased significantly after transfusion of FTD-RBCs (P＜0.05). For transfusion of one FTD-RBC unit, the median of differences in RBC count, HGB and HCT before and after transfusion were 0.310*1012/L, 9.735 g/L and 2.800%, respectively. The increments of the three indicators in the FTD-RBC group were all lower than those in the SRBC group, but the differences were not statistically significant (P > 0.05). None of the patients in this study had adverse transfusion reactions after transfusion of FTD-RBCs. In addition, we found that the efficacy of FTD-RBCs transfusion was associated with sex, age, body weight, disease type and blood type of the patient (P＜0.05). Conclusion: The infusion of FTD-RBCs for clinical patients is safe and effective, and it is an effective blood supply supplement in situations of blood shortage such as public health events. Key words：Frozen-thawed-deglycerolized RBCs, RBC count, HGB, HCT.
Treatment Decision-making for Individuals with Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL) in the United States.
Coombs CC, LeBlanc TW, Winfree KB … +8 more , Muehlenbein CE, Desai U, Kirson NY, Holub A, Qu A, Sears E, Koffman B, Thompson MC
Acta Haematol · 2026 May · PMID 42096360 · Publisher ↗
BACKGROUND: As available treatment options for relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) increase, treatment decisions become increasingly complex. This cross-sectional study evaluated patient and phys...
BACKGROUND: As available treatment options for relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) increase, treatment decisions become increasingly complex. This cross-sectional study evaluated patient and physician decision-making when selecting treatment for R/R CLL in the United States. METHODS: After an initial pilot, 100 individuals with R/R CLL and 100 physicians who treat R/R CLL completed web-based surveys on factors that influenced treatment selection and levels of involvement during treatment selection. Categorical measures were summarized using numbers/ proportions and continuous measures were summarized using means/standard deviations. RESULTS: Individuals with R/R CLL and physicians prioritized treatment efficacy (individuals: N=47 of 78, 60.3%; physicians: N=79 of 100, 79.0%) and potential side effects (individuals: N=43 of 78, 55.1%; physicians: N=50 of 79, 63.3%) when selecting treatment. Differences in the relative importance of specific factors were observed. Financial and cost factors were important for individuals while medical history was important for physicians. Both cohorts reported a shared decision-making process. CONCLUSIONS: Building upon previous findings in the front-line CLL setting, this study confirms that individuals with R/R CLL and physicians commonly consider efficacy and safety of different treatment options when making their decision. Relative importance of specific factors within these and other categories differs across the samples.
Diagnostic Pathways and Procedural Risk in Primary Central Nervous System Lymphoma: A Nationwide Analysis.
Qureshi K, Arakali S, Anzueto J … +8 more , Huddleston D, Hobbs J, Saenz S, Thakkar R, Jayaram RH, Goulart C, Alok K, Gupta M
Acta Haematol · 2026 May · PMID 42081433 · Publisher ↗
INTRODUCTION: Primary central nervous system lymphoma (PCNSL) is an aggressive malignancy in which timely diagnosis is critical. While neurosurgical biopsy remains the gold standard, cerebrospinal fluid (CSF)-based diagn...
INTRODUCTION: Primary central nervous system lymphoma (PCNSL) is an aggressive malignancy in which timely diagnosis is critical. While neurosurgical biopsy remains the gold standard, cerebrospinal fluid (CSF)-based diagnostics are frequently deployed. Owing to the rarity of this condition, little is known about clinical characteristics, test utilization, social determinants of health, and complication rates in the context of securing a PCNSL diagnosis. We evaluated approaches for diagnosing PCNSL, post-procedural complications, and clinical and socioeconomic associations with adverse events. METHODS: We queried the PearlDiver Mariner claims database to identify adults with newly diagnosed PCNSL between 2011 and 2023. CPT and ICD codes were used to identify lumbar puncture (LP) and/or neurosurgical biopsy performed before diagnosis. Patients were stratified by diagnostic pathway: LP-only, Biopsy-only, or both LP and biopsy. We used logistic regression to identify predictors of adverse events. Results Among 2,549 total patients, 1957 (76.8%), 398 (15.6%), and 194 (7.6%) comprised the LP-only, Biopsy-only, or both LP and biopsy pathways, respectively. LP-only patients were younger, more frequently male, Medicaid-insured, and economically disadvantaged (P< 0.001 for each). Rates of post-procedural thromboembolic complications were 5.4%, 10.1%, and 6.7% for LP-only, Biopsy-only, and both LP and biopsy, respectively (P<0.002). Biopsy-only was associated with increased odds of procedural complications (OR 3.21, 95% CI 1.79-5.69, P<0.001), thromboembolic events (OR 1.85, 95% CI 1.39-2.45, P<0.001), and medical complications (OR 1.87, 95% CI 1.49-2.33, P<0.001) compared to LP alone. CONCLUSIONS: The choice of diagnostic modalities utilized to secure a diagnosis of PCNSL may be influenced by patient-level clinical and socioeconomic factors. PCNSL patients may represent a uniquely high-risk subgroup for neurosurgical biopsy, emphasizing the importance of advancing CSF-based diagnostics.
Prognostic Factors and Survival Outcomes in Ovarian Lymphoma: A Population-Based Analysis.
Loap P, Kirova Y
Acta Haematol · 2026 Apr · PMID 42060552 · Full text
INTRODUCTION: Primary ovarian lymphoma represents a rare clinical entity accounting for 0.5% of non-Hodgkin lymphomas and 1.5% of ovarian neoplasms. Current literature is limited to small case series, with poorly defined...
INTRODUCTION: Primary ovarian lymphoma represents a rare clinical entity accounting for 0.5% of non-Hodgkin lymphomas and 1.5% of ovarian neoplasms. Current literature is limited to small case series, with poorly defined prognostic factors and optimal treatment strategies. METHODS: This retrospective cohort study utilized the SEER database (2000-2020) to identify patients with ovarian lymphoma. Patients were categorized as localized primary ovarian lymphoma (L-POL, stage I) or lymphoma involving the ovary (LIO, stages II-IV, or unknown). Cox proportional hazards regression identified independent prognostic factors for overall survival (OS) and cancer-specific survival (CSS). RESULTS: A total of 221 patients were identified, representing the largest ovarian lymphoma cohort ever reported. The cohort included 75 L-POL (33.9%) and 146 LIO (66.1%) patients. Median age was 49 years. Diffuse large B-cell lymphoma was the most common histology (62.0%). L-POL patients demonstrated higher proportions of Black patients (13.3% vs. 4.1%, p = 0.022), follicular lymphoma (22.7% vs. 12.3%), and unilateral disease (89.3% vs. 61.6%). At median follow-up of 80 months, 5-year and 10-year CSS rates were 77.7% and 75.3% overall, and 87.4% and 85.5% for L-POL patients. In multivariate analysis, age (HR 1.035, p < 0.001), L-POL (grade I) classification (HR 0.435, p = 0.023), and high-grade histology (HR 4.898, p = 0.009) were independent prognostic factors for CSS. Surgery, radiotherapy, and chemotherapy showed no survival benefit in multivariate analysis. Surgery was performed in 86.9% of patients, with 9.9% undergoing inappropriate debulking procedures. CONCLUSIONS: L-POL represents a distinct clinical presentation with excellent long-term prognosis. Age, L-POL classification, and histological grade are independent prognostic factors. The high rate of inappropriate surgical procedures, ranging from hysterectomy and debulking to pelvic exenteration, underscores the critical need for improved preoperative recognition. Optimal management should emphasize limited surgical intervention for diagnosis and fertility preservation, followed by systemic chemotherapy tailored to lymphoma subtype and stage.
<italic>Acta Haematologica</italic> in the Era of Artificial Intelligence.
Raanani P
Acta Haematol · 2026 Apr · PMID 42048274 · Publisher ↗
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Persistent Splenomegaly Is Associated with Morbidity in Tanzanian Children with Sickle Cell Anemia: Secondary Analysis of the SPHERE Trial.
Ambrose EE, Tomlinson G, Ngoya PS … +5 more , Stuber SE, Latham TS, Ware RE, Makubi AN, Smart LR
Acta Haematol · 2026 Apr · PMID 42048272 · Full text
INTRODUCTION: African children with sickle cell anemia (SCA) often have splenomegaly with recurrent cytopenias that complicate management, rather than the splenic atrophy typically observed in SCA. METHODS: We analyzed t...
INTRODUCTION: African children with sickle cell anemia (SCA) often have splenomegaly with recurrent cytopenias that complicate management, rather than the splenic atrophy typically observed in SCA. METHODS: We analyzed the presence, persistence, and impact of splenomegaly in the Stroke Prevention with Hydroxyurea Enabled through Research and Education (SPHERE) trial (NCT03948867), an open-label trial of dose-escalated hydroxyurea to prevent stroke in Tanzanian children with SCA. Palpable splenomegaly was recorded at each visit. Annual ultrasound measurements were compared to age- and height-related references. Longitudinally, palpable splenomegaly was categorized as infrequent (present in <5% visits), intermittent (10-50% visits), or persistent (>50% visits). Adverse events (AEs) and hydroxyurea dose-limiting toxicities (DLTs) were compared using incidence rate ratios (IRRs) among the three categories. RESULTS: At enrollment, splenomegaly was identified in 48/196 (25%) by palpation and 78 (40%) by ultrasound. Across 221 patient-years of treatment in 53 children, splenomegaly was intermittent in 19% and persistent in 21%. At 12 months, children with persistent splenomegaly were compared to those with infrequent splenomegaly and had achieved similar hydroxyurea dose (23.1 vs. 27.6 mg/kg/day, p = 0.136), hemoglobin (9.0 vs. 9.1 g/dL, p = 0.877), and HbF (23.8 vs. 24.3%, p = 0.481), but experienced more vaso-occlusive AE (IRR = 3.8, p value 0.122), transfusions (IRR = 7.1, p value 0.014), and DLTs (IRR = 3.2, p value 0.073). CONCLUSION: Splenomegaly is common in Tanzanian children with SCA, often persistent, worsens clinical outcomes, and complicates hydroxyurea. Its cause remains unclear, and further investigation is urgently needed to clarify its etiology, improve its management, and ensure optimal hydroxyurea treatment.
Venetoclax-Based Regimens in Chronic Myelomonocytic Leukemia: A Systematic Review and Meta-Analysis.
Abdulgayoom M, Afana MS, Saleh LH … +5 more , Manthiri AA, Aweer NA, Bakheet M, Al-Mashdali AF, Mohamed SF
Acta Haematol · 2026 Apr · PMID 41945493 · Full text
INTRODUCTION: Chronic myelomonocytic leukemia (CMML) is a biologically heterogeneous myelodysplastic/myeloproliferative neoplasm with limited disease-modifying treatment options beyond hypomethylating agents (HMAs) and a...
INTRODUCTION: Chronic myelomonocytic leukemia (CMML) is a biologically heterogeneous myelodysplastic/myeloproliferative neoplasm with limited disease-modifying treatment options beyond hypomethylating agents (HMAs) and allogeneic hematopoietic stem cell transplantation. Venetoclax, a selective BCL-2 inhibitor, is increasingly used off-label in CMML, but its CMML-specific efficacy and safety remain incompletely defined. METHODS: We conducted a systematic review and meta-analysis in accordance with PRISMA 2020. Eligible studies included adult patients with CMML treated with venetoclax-based regimens and reporting extractable CMML-specific outcomes. PubMed, Embase, Cochrane CENTRAL, conference proceedings, and trial registries were searched through August 2025. Random-effects meta-analyses of proportions were performed for complete remission (CR), marrow complete remission (mCR), and overall response rate (ORR). RESULTS: Seventeen publications representing nine unique studies were included, comprising 145 venetoclax-treated CMML patients. Most regimens combined venetoclax with azacitidine, decitabine, or oral decitabine-cedazuridine, with heterogeneous dosing schedules and frequent CYP3A-guided dose modifications. Responses typically occurred early, within one to two treatment cycles, but durability was generally modest. The pooled CR rate was 19.1% (95% CI: 9.4-34.9; I2 = 55%), the pooled mCR rate was 36.4% (95% CI: 24.7-50.0; I2 = 21%), and the pooled ORR was 71.9% (95% CI: 56.5-83.4; I2 = 56%). Venetoclax-based therapy was associated with substantial myelosuppression, including frequent grade ≥3 neutropenia and thrombocytopenia, with clinically relevant infectious complications. Early mortality was low in studies reporting short-term outcomes. CONCLUSION: Venetoclax-based regimens demonstrate measurable but limited activity in CMML, with high overall response rates but low CR rates and modest durability. Prospective CMML-specific trials are needed to define optimal dosing, clarify comparative effectiveness, and identify patients most likely to benefit.
A Case Report of Tyrosine Kinase Inhibitor Interruption in a Patient with Multilineage Philadelphia-Positive Acute Lymphoblastic Leukemia.
Goulart H, Braish J, Short NJ … +4 more , Jain N, Sasaki K, Kantarjian H, Jabbour EJ
Acta Haematol · 2026 Mar · PMID 41915597 · Publisher ↗
INTRODUCTION: Measurable residual disease (MRD) assessment in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) commonly relies on reverse transcription polymerase chain reaction detection of BCR::A...
INTRODUCTION: Measurable residual disease (MRD) assessment in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) commonly relies on reverse transcription polymerase chain reaction detection of BCR::ABL1 transcripts. However, discordance between BCR::ABL1 PCR and next-generation sequencing (NGS)-based MRD assays targeting immunoglobulin/T-cell receptor (IG/TR) rearrangements may occur, particularly in cases of multilineage Ph+ ALL, complicating treatment decisions such as tyrosine kinase inhibitor (TKI) discontinuation. CASE PRESENTATION: We report a 54-year-old female with Ph+ ALL who achieved durable remission with sustained NGS MRD negativity by IG/TR despite persistent low-level BCR::ABL1 transcript positivity. Following self-discontinuation of ponatinib, she experienced a rapid rise in BCR::ABL1 transcripts and loss of cytogenetic remission in myeloid cells, while remaining morphologically in remission and NGS MRD-negative. Reintroduction of ponatinib resulted in a prompt transcript decline. CONCLUSION: This case highlights the clinical significance of multilineage Ph+ ALL and its distinct biological background, thereby underscoring caution with TKI discontinuation despite deep NGS MRD negativity and may support continued TKI therapy in this setting.
A Complex Case of Anti-e Mimicking Autoantibody Development Leading to Warm Autoimmune Hemolytic Anemia: Case Report.
Hale I, Kunes KL, Sabile J … +3 more , Dougherty J, Wong T, Heinrich M
Acta Haematol · 2026 Mar · PMID 41824628 · Publisher ↗
INTRODUCTION: Newly diagnosed autoimmune hemolytic anemia (AIHA) develops in 1-3 individuals per 100,000 people per year. Warm autoimmune hemolytic anemia (WAIHA) is the most common type of AIHA, accounting for approxima...
INTRODUCTION: Newly diagnosed autoimmune hemolytic anemia (AIHA) develops in 1-3 individuals per 100,000 people per year. Warm autoimmune hemolytic anemia (WAIHA) is the most common type of AIHA, accounting for approximately 70-80% of cases. This report outlines a complex presentation of DAT-negative, anti-e-mimicking autoantibody mediated WAIHA following a transient viral infection in an adult male patient with no prior transfusions. CASE PRESENTATION: The 55-year-old male patient presented with several weeks of flu-like symptoms followed by jaundice, dyspnea, and progressive fatigue. Initial laboratory workup revealed hemolytic anemia, and subsequent serologic testing identified a warm autoantibody with anti-C specificity, but was ultimately consistent with an anti-e mediated WAIHA. CONCLUSION: This report highlights a complex case of a patient with an autoantibody mimicking anti-e associated WAIHA and the challenge of identifying suitable transfusion options.
Reconsidering Asparaginase in Leukemia Therapy.
Frost M, Subbiah V, Cuglievan B … +1 more , Garcia MB
Acta Haematol · 2026 Mar · PMID 41824624 · Publisher ↗
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Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Pregnancy-Associated Aplastic Anemia.
Li X, Wu L, Li Y … +9 more , Mo W, Chen X, Zhou M, Zhou R, Xu S, Pan S, Wang C, Wang S, Zhang Y
Acta Haematol · 2026 Mar · PMID 41824622 · Publisher ↗
INTRODUCTION: The prognosis and management of pregnancy-associated aplastic anemia (PAA) following allogeneic hematopoietic stem cell transplantation (allo-HSCT) remain uncertain. This retrospective study was conducted t...
INTRODUCTION: The prognosis and management of pregnancy-associated aplastic anemia (PAA) following allogeneic hematopoietic stem cell transplantation (allo-HSCT) remain uncertain. This retrospective study was conducted to describe our clinical experience and to explore the efficacy and safety of allo-HSCT in patients with PAA. METHODS: A retrospective observational study was evaluated the safety and feasibility of allo-HSCT in 18 patients with PAA who were treated between January 2013 and November 2023. RESULTS: Median neutrophil engraftment occurred at 11.5 days (range: 6-15), and platelet engraftment occurred at 11.5 days (range: 7-28). The cumulative incidence (CuI) of both neutrophil and platelet engraftment was 100%. The CuI rates were 22.96% (95% CI, 13.68-27.32) for grade II acute graft-versus-host disease (aGVHD) and 12.61% (95% CI, 9.32-29.28) for chronic GVHD (cGVHD). No cases of grade III-IV aGVHD, extensive cGVHD, or relapse were observed. The CuI of transplantation-related mortality was 19.87% (95% CI, 13.15-30.73). Among the 15 surviving patients, the median follow-up was 47.0 months (range: 7.6-122.2 months). The overall survival and relapse/rejection-free rates were both 80.14% (95% CI, 69.27-86.86). CONCLUSION: These findings suggest that allo-HSCT is a viable and effective treatment option for PAA. The donor types included 8 matched sibling donors, 1 matched unrelated donor, 3 mismatched unrelated donors, and 6 haploidentical donors.
Chronic Myeloid Leukemia: Historical Perspective, Pathophysiology, and Treatment Advances.
Tungjitviboonkun S, Daran L, Unsuwan S
Acta Haematol · 2026 Mar · PMID 41824621 · Full text
BACKGROUND: Chronic myeloid leukemia (CML) was the first leukemia to be described and remains one of the most well-studied hematologic malignancies. Since its initial description in 1845, CML has evolved from a fatal hem...
BACKGROUND: Chronic myeloid leukemia (CML) was the first leukemia to be described and remains one of the most well-studied hematologic malignancies. Since its initial description in 1845, CML has evolved from a fatal hematologic disease into a manageable chronic condition. SUMMARY: This review traces the historical evolution of CML research, from its first clinical recognition in the mid-19th century to modern molecular diagnostics and targeted therapy. Key milestones include the discovery of the Philadelphia chromosome in 1960, identification of the BCR::ABL1 fusion gene in the 1980s, and the subsequent development of tyrosine kinase inhibitors (TKIs). The introduction of imatinib in the early 2000s revolutionized CML treatment, transforming a fatal disease into a chronic condition with near-normal life expectancy for most patients. Second- and third-generation TKIs have since been introduced to overcome drug resistance and target specific BCR::ABL1 mutations, such as T315I. Recently, research has focused on mechanisms of TKI resistance, novel signaling pathways, and strategies to achieve treatment-free remission (TFR). Emerging therapies such as vamotinib, KF1601, and combination regimens are being explored. Furthermore, new insights into non-kinase functions of BCR::ABL1 and the role of microRNAs in resistance open additional therapeutic avenues. KEY MESSAGES: CML remains the most successful example of how understanding molecular pathogenesis can directly translate into targeted therapy. Strategic shifts toward multi-generational TKIs and combination regimens are essential to address the challenges posed by clonal evolution and TKI resistance. Modern clinical goals have shifted to sustained TFR, aiming to transform CML from a chronic to a curable condition.
Differentiation Therapy in Acute Myeloid Leukemia: Advances in Phenotypic Screening and CRISPR-Based Functional Genomics.
Takahashi S
Acta Haematol · 2026 Mar · PMID 41824616 · Publisher ↗
BACKGROUND: Acute myeloid leukemia (AML) is a clinically and genetically heterogeneous malignancy characterized by a differentiation block in myeloid progenitors. Although advances in molecular targeted therapies have im...
BACKGROUND: Acute myeloid leukemia (AML) is a clinically and genetically heterogeneous malignancy characterized by a differentiation block in myeloid progenitors. Although advances in molecular targeted therapies have improved outcomes in selected subgroups, long-term prognosis remains poor for many patients. Differentiation therapy, exemplified by the success of all-trans retinoic acid in acute promyelocytic leukemia (APL), represents an alternative therapeutic paradigm that aims to overcome the differentiation blockade rather than directly inducing cytotoxicity. SUMMARY: This review summarizes recent advances in phenotypic screening and CRISPR-based functional genomics that have contributed to the discovery of novel differentiation-inducing strategies in AML. High-throughput phenotypic screening approaches using compound libraries, computational tools, and integrative transcriptomic analyses have identified several candidate differentiation inducers. For example, triciribine, an AKT inhibitor, has been identified as a differentiation-inducing compound in AML models. In parallel, CRISPR loss- and gain-of-function screens have uncovered multilayered regulatory networks governing AML differentiation, including transcriptional regulators (e.g., KAT6A), metabolic dependencies (e.g., NMNAT1, glucose transporter type 1), and post-transcriptional regulators (e.g., ZFP36L2, YTHDC1). Emerging computational approaches, such as the Lineage Maturation Index and single-cell data integration, further enhance target prioritization and improve the translational relevance of screening results. Despite these advances, differentiation therapy outside APL remains challenging due to partial maturation, context-dependent responses, and AML heterogeneity. KEY MESSAGES: Recent advances in phenotypic screening and CRISPR-based functional genomics have expanded our understanding of the molecular mechanisms governing AML differentiation and have revealed novel therapeutic vulnerabilities. Integration of these discovery platforms with computational and single-cell approaches may facilitate the development of differentiation-based strategies for a broader spectrum of AML patients.
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