Acta Haematologica[JOURNAL]
Sun
200 papers
Shorter Duration of Blinatumomab Administration to 14 Days Has Same Efficacy and Safety Profile in Treatment of Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia: A Retrospective Single-Center Study.
Kong J, Miao W, Lu J … +4 more , Liu Y, Kong X, Qiu H, Song B
Acta Haematol · 2025 · PMID 39467525 · Publisher ↗
INTRODUCTION: Treatment of patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (r/r BCP-ALL) remains a significant clinical challenge. Many new strategies are changing the treatment landscape...
INTRODUCTION: Treatment of patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia (r/r BCP-ALL) remains a significant clinical challenge. Many new strategies are changing the treatment landscape of r/r BCP-ALL in recent years. Blinatumomab has improved outcomes in r/r BCP-ALL, though high treatment costs and extended hospital stays are significant concerns. We considered that shortening the duration of blinatumomab administration during induction therapy might solve these problems. METHODS: We retrospectively analyzed 19 patients with r/r BCP-ALL treated with different durations of blinatumomab, where 10 patients received blinatumomab for 14 days (Bli 14D group) and 9 received it for a longer duration (LT group, 21-28 days). RESULTS: The overall response rate (ORR) was 63.2% (12/19) of patients in total, and the ORRs in 14D and LT groups were almost the same (60% and 66.6%, respectively). The median overall survival was not reached in either groups. The median event-free survival time was 4.1 months in LT group and not reached in D14 group. The most common adverse events were consistent with previous reports, including cytokine release syndrome, neurologic toxicity, and hematological toxicity. CONCLUSION: A 14-day blinatumomab administration may be a promising and well-tolerated regimen in r/r BCP-ALL, offering the same ORR and survival rates.
Efficacy of Haploidentical Allogeneic Hematopoietic Cell Transplantation following Two Courses of Venetoclax and Azacytidine Therapy in Patients over 55 Years Old with Acute Myelogenous Leukemia.
Cao J, Zhuang X, Luo D … +6 more , Pei R, Lu Y, Chen D, Li S, Du X, Liu X
Acta Haematol · 2025 · PMID 39462494 · Publisher ↗
INTRODUCTION: The combination of venetoclax (VEN) and azacytidine (AZA) has demonstrated potential in achieving rapid and effective remissions in elderly patients with acute myeloid leukemia (AML). Allogeneic hematopoiet...
INTRODUCTION: The combination of venetoclax (VEN) and azacytidine (AZA) has demonstrated potential in achieving rapid and effective remissions in elderly patients with acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation is a promising potential cure for high-risk AML, as VEN-based therapies have a worse prognosis in elderly patients. This study aimed to assess the efficacy of sequential haploidentical HSCT following two courses of VEN and AZA therapy in patients with AML aged 55 years and older. METHODS: We conducted a retrospective study on AML patients aged 55-70 years who received intensive chemotherapy or two courses of VEN/AZA therapy, followed by haploidentical allo-HSCT (haplo-HSCT) based on disease risk degree, measurable residual disease status, and patient's preference. RESULTS: Between January 2019 and December 2023, 141 newly diagnosed AML patients received initial treatment with intensive chemotherapy or VEN/AZA therapy. Among them, 64 patients received haplo-HSCT, while 77 did not. The 1-year overall survival (OS) and relapse-free survival (RFS) of patients who received haplo-HSCT were significantly higher than those who did not receive haplo-HSCT (p < 0.05). Among patients who received transplantation, there was no significant difference in 1-year OS and RFS between the VEN/AZA and intensive chemotherapy groups: 76.3% versus 69.3% (p = 0.367) for OS, and 74.5% versus 69.7% (p = 0.473) for RFS. High-risk ELN stratification and the presence of ≥4 gene mutations were associated with lower OS and RFS in both univariate and multivariate analyses. CONCLUSIONS: AML patients over 55 years of age who received haplo-HSCT after two courses of VEN/AZA therapy had outcomes similar to those who received haplo-HSCT after intensive chemotherapy, suggesting that two courses of VEN/AZA therapy as a bridge to haplo-HSCT are feasible for patients over 55 years old.
Real-World Use of Ruxolitinib in Patients with Myelofibrosis and Anemia or Thrombocytopenia at Diagnosis.
Yu J, Bland E, Schuler T … +2 more , Cordaro T, Braunstein E
Acta Haematol · 2025 · PMID 39433041 · Full text
INTRODUCTION: Ruxolitinib is approved for treatment of myelofibrosis. We evaluated ruxolitinib in patients with anemia (hemoglobin <10 g/dL) or thrombocytopenia (platelet count ≤100 × 109/L) at diagnosis. METHODS: This w...
INTRODUCTION: Ruxolitinib is approved for treatment of myelofibrosis. We evaluated ruxolitinib in patients with anemia (hemoglobin <10 g/dL) or thrombocytopenia (platelet count ≤100 × 109/L) at diagnosis. METHODS: This was a retrospective, secondary analysis of a Cardinal Health Oncology Provider Extended Network medical chart review of adults with myelofibrosis diagnosed between 2012 and 2016 who received first-line ruxolitinib. RESULTS: 176 patients received first-line ruxolitinib and were included in this analysis. At diagnosis, 120 patients had hemoglobin concentrations <10 g/dL and 59 had a platelet count ≤100 × 109/L. Most patients (95%) with thrombocytopenia also had anemia. Median time of observation after diagnosis was 21.4 months. Among patients with anemia or thrombocytopenia, ruxolitinib dose at end of study was ≥10 mg twice daily (bid) in 88.3% and 83.1%, respectively. Ruxolitinib treatment was ongoing in 76.1% of patients overall and was rarely discontinued for anemia or thrombocytopenia (n = 2 total, 1.1%). Per the treating physician, 79.7% of patients had improved symptoms and 62.7% improved spleen size. CONCLUSION: Most patients with myelofibrosis and anemia or thrombocytopenia at diagnosis tolerated and maintained a ruxolitinib dose ≥10 mg bid for nearly 2 years, resulting in clinical benefit. This real-world evidence supports observations from prospective clinical trials of ruxolitinib in myelofibrosis.
Sudden Blast Crisis in a Chronic Myeloid Leukemia Patient in Treatment-Free Remission: A Case Report and Literature Review.
Liang Q, Liu Z, Wu Y … +2 more , Zhu H, Yang Y
Acta Haematol · 2025 · PMID 39433036 · Publisher ↗
INTRODUCTION: Treatment-free remission (TFR) has emerged as a new goal in the treatment of chronic myeloid leukemia (CML). TFR is considered a safe intervention because patients who experienced molecular relapse usually...
INTRODUCTION: Treatment-free remission (TFR) has emerged as a new goal in the treatment of chronic myeloid leukemia (CML). TFR is considered a safe intervention because patients who experienced molecular relapse usually responded well to tyrosine kinase inhibitors resumption and regained molecular response quite efficiently. Nevertheless, there have been reports of occurrence of blast crisis during TFR. CASE PRESENTATION: We report a case of sudden lymphoid blast crisis in a CML patient who had been in TFR for 21 months without any prior molecular loss. Whole-exon sequencing identified a frameshift mutation of SETD2. In addition, we reviewed the current literature on cases of blast crisis in TFR. Only eleven cases of blast crisis have been reported among thousands of patients who discontinued tyrosine kinase inhibitor (TKI) therapy, including our patient. Of these cases, nine presented with lymphoid blast crisis. Additional gene mutations are frequently observed. CONCLUSION: This case, along with others, emphasizes the necessity of implementing a long-term monitoring strategy following TKI discontinuation due to the potential for late onset of blast crisis. Systematic genetic studies in patients failing TFR should be properly carried out to further understand the mechanism and, eventually, to predict or prevent such adverse event in patients in TFR.
Erratum.
Acta Haematol · 2024 · PMID 39389047 · Publisher ↗
Abstract loading — click title to view on PubMed.
Belantamab Mafodotin in Relapsed/Refractory AL Amyloidosis: Real-World Multi-Center Experience and Review of the Literature.
Lebel E, Vainstein V, Milani P … +7 more , Palladini G, Shragai T, Lavi N, Magen H, Assayag M, Avivi I, Gatt ME
Acta Haematol · 2025 · PMID 39357511 · Full text
INTRODUCTION: Treatment for relapsed/refractory AL amyloidosis (AL) is an unmet need. The safety and efficacy of belantamab mafodotin (BLM) in multiple myeloma are known, whereas in AL data are limited. METHODS: We repor...
INTRODUCTION: Treatment for relapsed/refractory AL amyloidosis (AL) is an unmet need. The safety and efficacy of belantamab mafodotin (BLM) in multiple myeloma are known, whereas in AL data are limited. METHODS: We report a multi-center cohort of AL patients receiving BLM, and review all previous data on BLM therapy in AL. RESULTS: Twelve patients with a median of 3 (range 2-9) prior lines of therapy were included. The overall hematological response rate (ORR) was 75% (9/12), including 5 complete responses. Six of the 10 evaluable patients had organ responses. The median event-free survivals/overall survivals were 22.3 and 28.8 months, respectively. Grade 3 toxicities were mostly infections and keratopathy, occurring in 7/12 (58%). Hematological toxicities were rare. No grade 4/5 toxicities occurred. The review of the previous series reveals BLM provides an ORR of 60-83% with similar rates of corneal toxicity. CONCLUSION: BLM, being an off-the-shelf therapy, with acceptable toxicity even in frail patients, may be a valuable option in AL, with a high ORR, and a signal for durable responses and high-quality organ responses.
Intake of Proton Pump Inhibitors Is Associated with a Shorter Time to First Treatment in Early-Stage Chronic Lymphocytic Leukemia.
Tadmor T, Tadmor T, Melamed G … +4 more , Alapi H, Gazit S, Patalon T, Rokach L
Acta Haematol · 2025 · PMID 39348818 · Publisher ↗
INTRODUCTION: Proton pump inhibitors (PPIs) are one of the most widely used drugs worldwide [Gut Liver. 2017;11(1):27-37]. The use of PPI has become a common practice and is overprescribed for all patients with cancer in...
INTRODUCTION: Proton pump inhibitors (PPIs) are one of the most widely used drugs worldwide [Gut Liver. 2017;11(1):27-37]. The use of PPI has become a common practice and is overprescribed for all patients with cancer including patients with hematological malignancies. In the current study, we aimed to explore retrospectively the effect of PPI, on time to first treatment (TTFT) in a large cohort of patients with chronic lymphocytic leukemia (CLL) who were under watch-and-wait approach. METHODS: The cohort is based on anonymized data obtained from electronic medical records of Maccabi Healthcare Services (MHS) members, who is the second-largest healthcare organization in Israel, with 2.5 million insured patients, and received a diagnosis of CLL during this period. RESULTS: Our cohort included 3,474 patients with CLL who are treatment-naïve, and the median follow-up was 1,745 days (602-3,700). A total of 1,061 patients (30.5%) received a PPI agent, for a minimum of 3 months during the watch-and-wait period. The intake of PPI was found to be associated with a shorter TTFT: among PPI users, the 10-year treatment-free ratio is 79.2%, while among non-PPI users it is 90.6%. CONCLUSION: Routine use of PPI in CLL patients may negatively impact their clinical course. Biology of this primary observation requires further investigation.
Bosutinib Treatment of Chronic Myeloid Leukemia in Lombardy.
Iurlo A, Bucelli C, Intermesoli T … +16 more , Elena C, D'Adda M, Agostani E, Fiamenghi C, Maffioli M, Orofino N, Lunghi F, Gardellini A, Carraro MC, Inzoli A, Gigli F, Palazzolo R, Bertolli V, Cattaneo D, Pungolino EM, Gambacorti-Passerini C
Acta Haematol · 2025 · PMID 39293408 · Publisher ↗
INTRODUCTION: Up to 30% of CML patients will require a therapeutic change during follow-up due to intolerance and/or resistance to first-line tyrosine kinase inhibitor (TKI) approach. In this context, bosutinib (BOS) has...
INTRODUCTION: Up to 30% of CML patients will require a therapeutic change during follow-up due to intolerance and/or resistance to first-line tyrosine kinase inhibitor (TKI) approach. In this context, bosutinib (BOS) has not only demonstrated its efficacy, but also presents a favorable safety profile, without comorbid conditions representing an absolute contraindication to its use. METHODS: To gain further into BOS treatment in real life, we conducted a retrospective analysis on the outcome of CML patients receiving BOS in 18 hematological centers, all belonging to the "REL" (Lombard Hematology Network). RESULTS: Of 546 regularly followed CML cases, a total of 132 patients were reported as being treated with BOS, most frequently (62.9%) in second line. Interestingly, most patients (63.6%) switched to BOS due to intolerance to the previous TKI, while resistance to the last treatment was reported in the remaining 36.4% of patients. Despite a permanent discontinuation rate of 18.9%, over 80% of patients achieved at least a major molecular response and seven cases were able to attempt treatment-free remission. CONCLUSION: Although in this survey BOS represented the preferred option especially in patients intolerant rather than resistant to previous TKIs, we confirmed that BOS represents a safe and effective therapeutic option beyond the first line in the real-life setting.
Role of Autologous Transplant in Newly Diagnosed Multiple Myeloma Patients Treated with Novel Triplets: A Systematic Review and Meta-Analysis.
Amitai I, Gurion R, Raanani P … +5 more , Vaxman I, Yeshurun M, Magen H, Gafter-Gvili A, Shargian L
Acta Haematol · 2025 · PMID 39284295 · Publisher ↗
INTRODUCTION: High-dose therapy with melphalan followed by autologous stem cell transplant in the upfront setting (upfront ASCT) has significantly improved clinical outcomes of myeloma patients and become the standard of...
INTRODUCTION: High-dose therapy with melphalan followed by autologous stem cell transplant in the upfront setting (upfront ASCT) has significantly improved clinical outcomes of myeloma patients and become the standard of care for the past 30 years. However, with the advent of modern induction therapy, the role of upfront ASCT approach has been called into question. Several prospective studies have examined whether continuing with triplet therapy as consolidation with optional ASCT at relapse (triplet-alone) could result in comparable outcomes. METHODS: This was a systematic review and meta-analysis of randomized controlled trials comparing upfront ASCT versus triplet-alone approach among myeloma patients treated with triplet therapy, which included two novel agents and a corticosteroid, as induction. Cochrane Library, PubMed and conference proceedings were searched. Primary outcome was overall survival (OS). Secondary outcomes included progression-free survival (PFS), safety, and second primary malignancies (SPM). Subgroup analysis was conducted for high-risk cytogenetics. RESULTS: Our search yielded three trials, conducted between 2010-2018, including 1,737 patients. Two trials evaluated bortezomib plus lenalidomide (VRd) induction and the third study tested carfilzomib plus lenalidomide (KRd) induction. Maintenance was given in all trials to both arms. There was no difference in OS between the arms; the pooled OS in all patients and those with high-risk cytogenetics was hazard ratio (HR) 1.03 (95% CI, 0.85-1.26; I2 = 0%; 1,737 patients, 3 trials) and 0.85 (95% CI, 0.59-1.23; I2 = 0%; 222 patients, 2 trials), respectively. The pooled PFS for upfront ASCT versus triplet-alone was significantly improved in all the patients and in the high-risk cytogenetics subgroup, HR 0.67 (95% CI 0.59-0.76; I2 = 0%; 1,737 patients, 3 trials) and HR 0.59 (95% CI: 0.44-0.7; I2 = 0%; 306 patients, 3 trials), respectively. The risk of any grade 3-4 adverse events was higher in the upfront ASCT arm versus triplet-alone approach (relative risk = 1.17 [95% CI, 1.12-1.23; 1,737 patients]). The risk of secondary malignancies was reported in all three trials and was comparable between both arms. Two trials reported on secondary myeloid neoplasms, which were significantly higher among upfront ASCT arm versus triplet-alone approach, OR 9.7 (1.8-52.25, I2 = 0%, 1,422 patients). CONCLUSION: Although upfront ASCT approach, in the era of triplet therapy, resulted in a significantly longer PFS among all patients, this did not translate into a survival benefit, regardless of cytogenetic risk. Upfront ASCT was associated with an increased rate of secondary myeloid neoplasms. In the current plethora of innovative therapies, the role of upfront ASCT is debatable.
Beneficial Effect of Integrated Nutritional Interventions in Patients with Hematological Diseases Undergoing Hematopoietic Stem Cell Transplant.
Geng M, Sun Z
Acta Haematol · 2025 · PMID 39265560 · Publisher ↗
INTRODUCTION: The nutritional status of patients undergoing hematopoietic stem cell transplantation (HSCT) is critically important. This study aimed to assess the impact of comprehensive nutritional interventions on the...
INTRODUCTION: The nutritional status of patients undergoing hematopoietic stem cell transplantation (HSCT) is critically important. This study aimed to assess the impact of comprehensive nutritional interventions on the well-being of individuals with hematological diseases who underwent HSCT. METHODS: A total of 175 patients with hematological diseases who underwent HSCT were included, with 94 in the control group and 81 in the research group. Patients in the control group received standard nursing care, while those in the research group underwent integrated nutritional interventions. Nutritional status was evaluated using the mini nutritional assessment (MNA) and subjective global assessment (SGA), along with body measurements and serum levels of albumin, prealbumin, and hemoglobin. RESULTS: There were no significant differences in the proportion of malnourished patients evaluated by MNA or SGA between the control and research groups at admission. However, at discharge and 3 months of post-discharge, fewer patients in the research group were malnourished compared to the control group, as assessed by both MNA and SGA. At admission, there were no significant differences in albumin, prealbumin, hemoglobin levels, weight, calf circumference (CC), triceps skinfold thickness (TSF), or subscapular skinfold thickness (SSF) between groups. However, at discharge and 3 months of post-discharge, the levels of these indicators significantly decreased compared to those upon admission. Levels of albumin, prealbumin, and hemoglobin, as well as weight, CC, TSF, and SSF, were significantly higher in the research group than in the control group at both discharge and 3 months of post-discharge. CONCLUSION: HSCT led to a decline in nutritional status among patients with hematological diseases, but integrated nutritional interventions effectively improved their nutritional status.
Outcomes of Fedratinib in Routine Treatment of Ruxolitinib-Resistant or -Refractory Patients with Primary and Post-Polycythemia Vera or Essential Thrombocythemia Myelofibrosis: A Nationwide Retrospective Study.
Duek A, Tzinman A, Maziuk K … +13 more , Levy A, Ellis M, Stemer G, Shacham Abulafia A, Cohen A, Lavi N, Ronson A, Braester A, Shapira S, Canaani J, Volchek Y, Leiba R, Leiba M
Acta Haematol · 2025 · PMID 39265539 · Publisher ↗
INTRODUCTION: In recent years, fedratinib, a selective JAK2 inhibitor, has emerged as a potential therapeutic option for patients who have failed or are intolerant to ruxolitinib. Despite the promising results observed i...
INTRODUCTION: In recent years, fedratinib, a selective JAK2 inhibitor, has emerged as a potential therapeutic option for patients who have failed or are intolerant to ruxolitinib. Despite the promising results observed in clinical studies, real-world evidence from the USA and Europe suggests that the efficacy of fedratinib may be less conclusive. We report the characteristics, treatment patterns, and clinical outcomes of patients with myelofibrosis (MF) treated with fedratinib following ruxolitinib failure in Israel's clinical practice. METHODS: This retrospective patient chart review included adults with a physician-reported diagnosis of MF, who initiated fedratinib after discontinuing ruxolitinib. Descriptive analyses characterized patient characteristics, clinical outcomes, and treatment patterns from MF diagnosis through ruxolitinib and fedratinib treatment. RESULTS: We extracted data for 16 eligible patients. Approximately 62.5% of the patients were female, and the median age was 77 (range: 63-85) years. The median duration of ruxolitinib therapy was 17 months (range: 3-84) months. Before the initiation of fedratinib, the median spleen size by palpation was 15.5 cm below the costal margin (range: 4-22 cm). After 3 months the median spleen size was 13 cm below the costal margin (range: 2-21 cm). Only 2 patients showed minimal improvement after 6 months, while 3 patients progressed, and 2 patients showed no change in the spleen size. The spleen response did not improve after 12 months of treatment. At this point, the median spleen size was 19 cm below the costal margin (range: 2-30 cm). Regarding the MF-related symptoms, 43.75% (n = 7) of patients reported some improvement, 37.5% (n = 6) showed no changes, whereas 18.75% (n = 3) of the population complained of worsening. Gastrointestinal toxicity was the most frequent adverse effect of the drug, while 31% of patients died. CONCLUSION: Our observations showed that in MF patients who have failed to ruxolitinib, the therapeutic value from fedratinib may be modest, especially when exposure time to ruxolitinib was more than 12 months. Early switching from ruxolitinib to fedratinib may yield a better result.
Real-World Impact of Routine Addition of Antithymocyte Globulin to Standard GVHD Prophylaxis in Myeloablative Unrelated Donor Transplants: Important Gains in Graft-versus-Host Disease Prevention though No Difference in Overall Survival.
Bai N, Limvorapitak W, Henderson R … +17 more , Abou Mourad Y, Chung S, Forrest D, Hay K, Kuchenbauer F, Nantel S, Narayanan S, Nevill T, Power M, Rodrigo J, Roy C, Sanford D, Song K, Stubbins R, Sutherland H, Toze C, White J
Acta Haematol · 2025 · PMID 39197435 · Publisher ↗
INTRODUCTION: Antithymocyte globulin (ATG) has been demonstrated to reduce the incidence of graft-versus-host disease (GVHD); however, it remains controversial whether these gains are offset by an increase in relapse. ME...
INTRODUCTION: Antithymocyte globulin (ATG) has been demonstrated to reduce the incidence of graft-versus-host disease (GVHD); however, it remains controversial whether these gains are offset by an increase in relapse. METHODS: We conducted a retrospective historical control study consisting of patients (n = 210) who underwent myeloablative allogeneic hematopoietic stem-cell transplantation (HSCT) from 2014 to 2020. RESULTS: The incidence of acute GVHD was lower in the ATG group (51.4%) than the non-ATG group (control) (70.0%, p = 0.010). The incidence of chronic GVHD was also lower in the ATG group at 1-year (36.4% vs. 62.9%, p < 0.001) and 2-year (40.0% vs. 65.7%, p < 0.001) post-HSCT. The mortality due to GVHD was higher in the control (18.5%) than the ATG group (4.3%; p = 0.024). The severe GVHD-relapse-free survival was higher in the ATG group (36.4%) than the control (12.9%; p < 0.001). Nevertheless, the 2-year overall survival was similar. CONCLUSION: Our results confirm the effectiveness of ATG in prevention of GVHD in the real-world setting and enhanced GVHD-free survival. An important result is the equalization of overall survival between the ATG and control groups at 1- and 2-year post-HSCT and implies that earlier GVHD-associated mortality may be offset by later relapse mortality producing similar overall survival over time.
Prognostic Value of the Pretransplant Fibrosis-4 Index on Non-Relapse and Overall Mortality following Unrelated Single-Unit Cord Blood Transplantation in Adults.
Konuma T, Monna-Oiwa M, Kato S … +3 more , Isobe M, Takahashi S, Nannya Y
Acta Haematol · 2025 · PMID 39197423 · Full text
INTRODUCTION: The fibrosis-4 (FIB-4) index is a noninvasive marker of liver fibrosis. The FIB-4 index predicts poor outcomes in patients with hepatic and non-hepatic diseases. However, the association of the FIB-4 index...
INTRODUCTION: The fibrosis-4 (FIB-4) index is a noninvasive marker of liver fibrosis. The FIB-4 index predicts poor outcomes in patients with hepatic and non-hepatic diseases. However, the association of the FIB-4 index with mortality and liver-related clinical outcomes following cord blood transplantation (CBT) is unclear. METHODS: We retrospectively evaluated the impact of the pretransplant FIB-4 index on outcomes in 336 adults following single-unit unrelated CBT at our institution. RESULTS: In multivariate analyses, when the FIB-4 index <1.3 group was used as the reference, non-relapse mortality was significantly higher in the FIB-4 index 1.3-2.67 (hazard ratio [HR], 2.51; 95% confidence interval [CI], 1.19-5.30) and FIB-4 index >2.67 (HR, 2.34; 95% CI, 1.12-4.90) groups. Overall mortality was significantly higher in the FIB-4 index >2.67 group (HR, 1.66; 95% CI, 1.00-2.73), but with only marginal significance in the FIB-4 index 1.3-2.67 group (HR, 1.59; 95% CI, 0.96-2.64). Hematopoietic recovery, acute and chronic graft-versus-host disease of the liver, and veno-occlusive disease/sinusoidal obstruction syndrome were not associated with the pretransplant FIB-4 index. CONCLUSION: The pretransplant FIB-4 index is accurate and useful in predicting mortality in adult patients undergoing single-unit unrelated CBT.
Outcomes of Patients with Myeloid Malignancies and Cardiovascular Disease Undergoing Allogeneic Stem Cell Transplantation.
Sanchez-Petitto G, Goloubeva O, Childress J … +14 more , Iqbal T, Masur J, An M, Muhammad S, Lawson J, Li G, Barr B, Emadi A, Duong VH, Hardy NM, Rapoport AP, Baer MR, Niyongere S, Yared JA
Acta Haematol · 2025 · PMID 39191224 · Publisher ↗
INTRODUCTION/BACKGROUND: Reduced-intensity conditioning (RIC) and nonmyeloablative (NMA) regimens have enabled patients with cardiovascular disease (CVD) to undergo allogeneic stem cell transplantation (allo-HSCT). Howev...
INTRODUCTION/BACKGROUND: Reduced-intensity conditioning (RIC) and nonmyeloablative (NMA) regimens have enabled patients with cardiovascular disease (CVD) to undergo allogeneic stem cell transplantation (allo-HSCT). However, little is known about long-term outcomes, including cardiovascular (CV) complications. METHODS: We retrospectively studied 99 consecutive patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who underwent allo-HSCT between September 1, 2013, and November 30, 2020. Overall survival (OS), progression-free survival (PFS), nonrelapse mortality (NRM), cumulative incidence of relapse, and cumulative incidence of acute and chronic graft-versus-host disease (GvHD) were compared in patients with and without CV risk factors or disease. RESULTS: Preexisting CVD was present in 34 of 99 patients (34%). CVD patients more commonly had reduced-intensity conditioning (91% vs. 60%, p = 0.001) and unrelated donors (56% vs. 35%, p = 0.04). Early adverse cardiac events occurred more frequently in the CVD versus no-CVD group (38% vs. 14%), particularly arrhythmias (21% vs. 5%; p = 0.04). CVD patients tended to have poorer OS and PFS outcomes (HR = 1.98, [1.00, 3.92]; HR = 1.89, [0.96-3.72], respectively). OS rate at 1, 2, and 3 years for CVD versus no-CVD patients was 66% versus 72%, 55% versus 64%, and 46% versus 62%, respectively. Causes of death in the CVD and no-CVD groups were infections (53% vs. 28%), relapsed disease (32% vs. 52%), and CV events (10% vs. 3%). CONCLUSION: Based on these data, predictive models to identify patients with CVD with higher risk of post-allo-HSCT complications and mortality and strategies to mitigate these risks should be developed.
Disseminated Mucormycosis and T-Cell-Depleted Allogeneic Stem Cell Transplantation: An Unusual Case Study.
Dragoi OD, Shah M, Potter V … +6 more , Avenoso D, Krishnamurthy P, Abdolrasouli A, Schelenz S, Chandra J, Mehra V
Acta Haematol · 2025 · PMID 39168110 · Publisher ↗
INTRODUCTION: Invasive fungal infections are a primary cause of morbidity and mortality in patients with haematological malignancies. CASE PRESENTATION: We describe an unusual clinical and radiological presentation of in...
INTRODUCTION: Invasive fungal infections are a primary cause of morbidity and mortality in patients with haematological malignancies. CASE PRESENTATION: We describe an unusual clinical and radiological presentation of invasive mucormycosis (IM) in a 69-year-old patient with relapsed acute myeloid leukaemia. The patient was diagnosed with disseminated IM with involvement of the central nervous system in an atypical location, lung, spleen, muscle, bone, and heart, after having completed induction and bridging chemotherapy to allogeneic haematopoietic stem cell transplant (HSCT). Her clinical presentation was atypical with mild neurological symptoms slowly progressing over 2 months and without appropriate signs of systemic inflammation. Mucorales was eventually confirmed from bronchoalveolar lavage and subdural collection. CONCLUSION: This report highlights the difficult challenges of managing disseminated IM in an immunocompromised patient, where close multidisciplinary specialist care enabled successful treatment, followed by T-cell-depleted allogeneic HSCT for a high-risk haematological malignancy.
Congenital Hypodysfibrinogenemia due to γ326Cys→Tyr Mutation: Third Ever-Described Case Associated with Recurrent Venous Thrombosis and COVID Vaccine.
Nanthan KR, Pedersen IS, Andersen DT … +1 more , Bor MV
Acta Haematol · 2024 · PMID 39140696 · Publisher ↗
INTRODUCTION: Congenital fibrinogen disorders are a heterogenous group of fibrinogen defects. CASE PRESENTATION: Here, we describe hypodysfibrinogenemia in a 33-year-old female patient with provoked recurrent deep vein t...
INTRODUCTION: Congenital fibrinogen disorders are a heterogenous group of fibrinogen defects. CASE PRESENTATION: Here, we describe hypodysfibrinogenemia in a 33-year-old female patient with provoked recurrent deep vein thrombosis (DVT) diagnosed based on decreased functional and antigenic fibrinogen levels with a decreased functional/antigenic fibrinogen ratio. Definitive diagnosis of congenital hypodysfibrinogenemia is done by genotyping using whole-exome sequencing, which identified the γ326Cys→Tyr mutation combined with single-nucleotide polymorphisms: rs2070011 and rs2070018 in FGA and rs1049636 in FGG. Fibrin structure assays showed reduced maximum polymerization rate. The mother of the proband shares the same γ326Cys→Tyr mutation and experienced a provoked DVT. CONCLUSION: Our case with DVT is the third ever-described occurrence of the mutation γ326Cys→Tyr that is associated with hypodysfibrinogenemia. The mechanism by which this mutation induces thrombosis remains unknown. Due to the high recurrence risk of thrombosis, the patient was treated with long-term reduced dose of rivaroxaban (10 mg daily) as secondary prophylaxis.
The Latin-American Experience in POEMS Syndrome: A Study of the GELAMM (Grupo de Estudio Latinoamericano de Mieloma Múltiple).
Gallardo-Pérez MM, Negrete-Rodríguez P, Gertz MA … +26 more , Peña C, Riva E, Gilli V, Rodríguez G, Samánez C, Ferreira J, Portiño S, Montaña J, León P, Gutiérrez Y, Del-Castanhel C, Seehaus C, Funes ME, Meneces-Bustillo R, Duarte P, Shanley C, Elvira G, Ochoa P, López-Vidal H, Martinez-Cordero H, Vasquez J, von-Glasenapp A, Donoso J, Viñuela JL, Ruiz-Delgado GJ, Ruiz-Argüelles GJ
Acta Haematol · 2025 · PMID 39128463 · Publisher ↗
INTRODUCTION: POEMS syndrome is a rare paraneoplastic syndrome caused by an underlying plasma cell disorder. The acronym refers to the following features: polyradiculoneuropathy, organomegaly, endocrinopathy, monoclonal...
INTRODUCTION: POEMS syndrome is a rare paraneoplastic syndrome caused by an underlying plasma cell disorder. The acronym refers to the following features: polyradiculoneuropathy, organomegaly, endocrinopathy, monoclonal paraproteinemia, and skin changes. METHODS: The study was conducted at 24 hematological centers across 8 Latin-American countries. The study included a total of 46 patients {median age was 52 years (interquartile range [IQR]: 42-61.5), 30 males and 16 females} fulfilling the POEMS syndrome criteria diagnosed over a period of 12 years (January 1, 2011, through July 31, 2023). Epidemiological and clinical data were collected in an ad hoc database sent to the members of GELAMM, as well as the Kolmogorov-Smirnov test and Kaplan-Meier estimates. RESULTS: All patients had polyneuropathy and monoclonal gammopathy; 89% had bone marrow plasma cell infiltration, 33% had sclerotic bone lesions. Only 10 patients underwent vascular endothelial growth factor (VEGF) testing in plasma samples. The paraproteinemia was IgG λ in 32% and IgA λ in 30%. 59% patients presented with cutaneous changes, mainly hyperpigmentation, 54% had organomegaly, and 74% endocrinopathy. The median interval from symptom onset to diagnosis was 7.7 months (IQR: 4.0-12.6). 69% of patients received a single line of treatment. The median follow-up period was 25 months (IQR: 9.37-52.0) and the 2-year overall survival rate was 100%. All patients who underwent transplantation (43%) are alive, with a median follow-up of 45.62 months (IQR: 15.46-70). CONCLUSION: This study investigates POEMS syndrome in Latin America and presents an initial overview of the disease in the region. VEGF usage is recommended for accurate diagnosis, but only 7 hematology centers in the region used it. Survival rate in Latin America is comparable with those observed internationally.
A Case of Recurrent Localized Pulmonary Nodular Light Chain Amyloidosis Treated with Daratumumab plus CyBorD.
Raddell T, Ashraf F, Zhao X … +2 more , Abramian O, Budak-Alpdogan T
Acta Haematol · 2025 · PMID 39074451 · Publisher ↗
INTRODUCTION: Nodular pulmonary amyloidosis (NPA) is a localized form of light chain (AL) amyloidosis often found incidentally and typically has an indolent and benign disease course treated with resection or local excis...
INTRODUCTION: Nodular pulmonary amyloidosis (NPA) is a localized form of light chain (AL) amyloidosis often found incidentally and typically has an indolent and benign disease course treated with resection or local excision. We present a patient with recurrent localized AL amyloidosis who required further treatment. CASE PRESENTATION: A 63-year-old female with monoclonal gammopathy of undetermined significance (MGUS) was found to have pulmonary AL amyloid on wedge resection and later had recurrence. The patient did not have signs of clonal plasma cell proliferation or systemic AL amyloid. She was treated with daratumumab, cyclophosphamide, bortezomib, and dexamethasone. After initiation of treatment, the patient has had significant hematologic and radiographic response. CONCLUSION: The patient had NPA recurrence with organ dysfunction without systemic disease. Because the presentation of recurrent pulmonary AL amyloidosis is rare, there is no published evidence on treatment. However, the patient has had hematologic and radiographic improvement after initiating treatment with a systemic protocol.
Prognostic Factors for Chronic Thrombocytopenia in Systemic Lupus Erythematosus with Immune Thrombocytopenia.
Ahn SM, Choi EJ, Oh JS … +4 more , Kim YG, Lee CK, Yoo B, Hong S
Acta Haematol · 2025 · PMID 39074444 · Publisher ↗
INTRODUCTION: We aimed to identify the clinical characteristics and risk factors for chronic immune thrombocytopenia (ITP) in patients with systemic lupus erythematosus (SLE). METHODS: We retrospectively reviewed patient...
INTRODUCTION: We aimed to identify the clinical characteristics and risk factors for chronic immune thrombocytopenia (ITP) in patients with systemic lupus erythematosus (SLE). METHODS: We retrospectively reviewed patients diagnosed with SLE-associated ITP between January 2000 and December 2021. Patient characteristics were analyzed according to the progression of chronic thrombocytopenia. No response was defined as a platelet count <30 × 109/L or less than double the baseline count after treatment. Factors associated with chronic ITP were evaluated by logistic regression analysis. RESULTS: Among the 121 patients with SLE-associated ITP, 27 progressed to chronic ITP lasting more than 1 year after initial diagnosis. The median initial platelet count was significantly lower in patients with chronic thrombocytopenia than in those without the disease (16 vs. 51 × 109/L). Patients who did not achieve a response within 1 month of treatment exhibited a high probability of progressing to chronic ITP (55.6 vs. 22.3%, p < 0.001). Multivariable analysis revealed that severe thrombocytopenia at baseline (<20 × 109/L) (adjusted odds ratio [aOR] = 13.628, 95% confidence interval [CI] = 3.976-46.791) and no response within 1 month (aOR = 9.171, 95% CI = 2.776-30.298) were significantly associated with the risk of progression to chronic ITP in patients with SLE. Approximately one-quarter of the patients with SLE-associated ITP progressed to chronic ITP. CONCLUSION: Severe thrombocytopenia and failure to achieve a response within 1 month were risk factors for the development of chronic ITP in those patients.
Long-Term Follow-Up of Eltrombopag Treatment for Patients with Cyclosporin A Refractory/Relapsed Transfusion-Dependent Non-Severe Aplastic Anemia: A Report from a Single Center in China.
Hu Q, Yang Y, Yang C … +2 more , Chen M, Han B
Acta Haematol · 2025 · PMID 39019025 · Full text
INTRODUCTION: Aplastic anemia (AA) is characterized by bone marrow failure and cytopenia. Eltrombopag (ELT) is effective and safe for treating refractory/relapsed AA; however, reports on the long-term outcomes of transfu...
INTRODUCTION: Aplastic anemia (AA) is characterized by bone marrow failure and cytopenia. Eltrombopag (ELT) is effective and safe for treating refractory/relapsed AA; however, reports on the long-term outcomes of transfusion-dependent non-severe AA (TD-NSAA) are limited. METHODS: Patients with TD-NSAA refractory to immunosuppressive therapy (IST) or relapsed after IST, treated with ELT alone, and followed up for at least 12 months were retrospectively enrolled. The baseline characteristics of patients, efficacy and adverse effects of ELT, and relapse and clone evolution rates after ELT were documented. RESULTS: Of the 55 patients with TD-NSAA included, 24 (43.6%) were men. Median age at diagnosis was 46 (19-80) years. Twenty-four patients had relapsed TD-NSAA, and 31 patients had refractory TD-NSAA. During the median follow-up period of 28 (12-48) months, the overall and complete response rates at 3, 6, and 12 months of ELT treatment were 38.2, 60.0, and 52.7 and 9.1, 14.6, and 9.1%, respectively. After a median follow-up of 28 (12-48) months, 21.2% (7/33) of patients experienced relapse, with a median duration from ELT treatment to relapse of 14 (6-45) months. CONCLUSION: ELT was effective in patients with relapsed/refractory TD-NSAA, with tolerable adverse effects.
About
- Frequency
- Sun
- Papers found
- 200
- RSS feed
- Subscribe