The incidence of cardiac morbimortality in acute myeloid leukemia (AML) is not well known. We aim to estimate the cumulative incidence (CI) of cardiac events in AML patients and to identify risk factors for their occurrence. Among 571 newly diagnosed AML patients, 26 (4.6%) developed fatal cardiac events, and among 525 treated patients, 19 (3.6%) experienced fatal cardiac events (CI: 2% at 6 months; 6.7% at 9 years). Prior heart disease was associated with the development of fatal cardiac events (hazard ratio (HR) = 6.9). The CI of non-fatal cardiac events was 43.7% at 6 months and 56.9% at 9 years. Age ≥ 65 (HR = 2.2), relevant cardiac antecedents (HR = 1.4), and non-intensive chemotherapy (HR = 1.8) were associated with non-fatal cardiac events. The 9-year CI of grade 1–2 QTcF prolongation was 11.2%, grade 3 was 2.7%, and no patient had grade 4–5 events. The 9-year CI of grade 1–2 cardiac failure was 1.3%, grade 3–4 was 15%, and grade 5 was 2.1%; of grade 1–2, arrhythmia was 1.9%, grade 3–4 was 9.1%, and grade 5 was 1%. Among 285 intensive therapy patients, median overall survival decreased in those experiencing grade 3–4 cardiac events (p < 0.001). We observed a high incidence of cardiac toxicity associated with significant mortality in AML.

Background: Information regarding the impact on healthcare systems of secondary acute myeloid leukemia (sAML) is scarce. Methods: A retrospective review of medical charts identified patients aged 60–75 years with sAML between 2010 and 2019. Patient information was collected from diagnosis to death or last follow-up. Outpatient resource use, reimbursement, frequency and duration of hospitalization, and transfusion burden were assessed. Forty-six patients with a median age of 64 years were included. Anthracycline plus cytarabine regimens were the most common induction treatment (39 patients, 85%). The ratio of the total days hospitalized between the total follow-up was 29%, with a sum of 204 hospitalizations (average four/patient; average duration 21 days). The total average reimbursement was EUR 90,008 per patient, with the majority (EUR 77,827) related to hospital admissions (EUR 17,403/hospitalization). Most hospitalizations (163, mean 22 days) occurred in the period before the first allogeneic hematopoietic stem cell transplant (alloHSCT), costing EUR 59,698 per patient and EUR 15,857 per hospitalization. The period after alloHSCT (in only 10 patients) had 41 hospitalizations (mean 21 days), and a mean reimbursement cost of EUR 99,542 per patient and EUR 24,278 per hospitalization. In conclusion, there is a high consumption of economic and healthcare resources in elderly patients with sAML receiving active treatments in Spain.

Background: In recent years, numerous recurrently mutated genes have been identified in acute myeloid leukemia (AML), some of which, such as FLT3 and IDH1/2, serve as therapeutic targets, offering new treatment options. Rapid mutational analysis is crucial for timely and optimal therapy selection. This study aims to develop and validate a rapid, cost-effective, and sensitive screening method for detecting IDH1, IDH2, and FLT3-TKD2 mutations using polymerase chain reaction (PCR) and high-resolution melting curve analysis (HRM). Methods: A PCR-HRM assay was developed to simultaneously detect mutations in IDH1, IDH2, and FLT3-TKD2. The method was applied to a cohort of 1363 AML patients, and its performance, including turnaround time, was evaluated through comparison with next-generation sequencing (NGS) results. Results: The PCR-HRM method demonstrated a positive percent agreement of 98%, 98%, and 92% for IDH1, IDH2, and FLT3-TKD2, respectively, and a negative percent agreement of 100% for all three genes compared to NGS. No false positives were observed, and false negatives were detected in less than 1% of cases, mostly in FLT3-TKD2, all occurring below the established limit of detection. The turnaround time and cost of PCR-HRM were significantly lower than those of NGS. Conclusions: This method offers a highly sensitive, specific, and time-efficient approach for the simultaneous detection of IDH1, IDH2, and FLT3-TKD2 mutations in AML patients. Its rapid turnaround time and cost-effectiveness make it a valuable tool for routine clinical screening, facilitating timely and targeted treatment decisions.

The addition of midostaurin (MIDO) to intensive chemotherapy (IC) improves survival in younger adults with FLT3 ‐mutated acute myeloid leukemia (AML); however, real‐world data in elderly patients (≥ 60 years) are limited. This large, retrospective, multicenter study from three European registries (PETHEMA, FILO, DATAML) evaluated MIDO+IC ( n = 194) versus IC alone ( n = 371) in 565 patients with FLT3 ‐mutated AML aged ≥ 60 years (median age 67.5 years; 35.6% ≥ 70 years). MIDO+IC was associated with lower day‐60 early death (8.2% vs. 21.4%, p < 0.0001) and higher composite complete remission (CRc) rates (78.9% vs. 63.1%, p < 0.0001). After a median follow‐up of 46.0 months, median overall survival (OS) was 24.2 months for MIDO+IC versus 8.7 months for IC ( p < 0.0001), with 5‐year OS rates of 40.6% vs. 12.9%, respectively. Event‐free survival (EFS; median 13.5 vs. 4.6 months; 5‐year EFS: 36.0% vs. 10.1%) and relapse‐free survival (RFS; median 20.2 vs. 8.0 months; 5‐year RFS: 45.4% vs. 15.7%) were also significantly improved (both p < 0.0001). The 5‐year cumulative incidence of relapse was lower with MIDO+IC (47.8% vs. 67.1%, p < 0.001). In multivariate analyses, midostaurin was an independent favorable prognostic factor for CRc (aOR 1.97 [95% CI: 1.29–2.98]), OS (aHR 0.46 [95% CI: 0.36–0.58]), EFS (aHR 0.49 [95% CI: 0.39–0.60]), and RFS (aHR 0.47 [CI: 0.36–0.62]) (all p ≤ 0.002). These benefits were confirmed by propensity score matching. This large real‐world study demonstrates that combining midostaurin with IC significantly improves remission rates and survival outcomes in elderly patients with FLT3 ‐mutated AML, supporting its consideration in this population.

Background/Objectives: The incidence of cardiac morbimortality in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) is unknown. Methods: We analyze the characteristics, incidence, risk factors, and outcomes of cardiac events in AML patients treated for second-line (2L) or third-line (3L) episodes. Results: Among 327 2L AML patients (median age 62 years old), 135 experienced cardiac events, with an incidence of 38.6% non-fatal and 1.3% fatal events at 6 months. The grade 1–2 incidence was 16.8%, and the grade 3–4 incidence was 23.5% at 6 months. Overall, 207 cardiac events occurred in the 2L cohort, the most frequent being hypertension (n = 45), bradycardia (n = 39), QTc prolongation (n = 35), heart failure (n = 33), syncope/presyncope (n = 22), arrhythmia (n = 18), and myocardial ischemia (n = 8). Median OS in the 2L cohort was 9.4 months, 21.4 months in patients with grade 1–2, 8.8 months in patients without a cardiac event, 7.6 months in grade 3–4 patients, and 2.1 months with in 5 patients (p = 0.0035). The multivariate analysis showed prior cardiologic antecedents (p = 0.013), intensive 2L chemotherapy (p = 0.01), and inclusion in a 2L clinical trial (p < 0.001) as independent risk factors for non-fatal cardiac events. Among 189 patients of the 3L cohort, the incidence of non-fatal and fatal cardiac events was 49.2% and 0% at 6 months, respectively. Non-fatal cardiac events were more frequent in patients with prior cardiac antecedents (p = 0.004). Conclusions: In summary, cardiotoxicity is a frequent and challenging complication in R/R AML patients. We identified the risk factors that could be relevant to implementing risk-adapted management guidelines, aiming to reduce morbi-mortality in this difficult-to-treat setting.

Background/Objectives: Information on the economic impact and healthcare resource utilization (HCRU) associated with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) is lacking. Methods: A retrospective review of medical records identified patients diagnosed with BPDCN between 2009 and 2023. Data on outpatient resource use, reimbursement, frequency and duration of hospitalizations, and transfusion burden were collected from diagnosis to death or last follow-up. Results: A total of 38 patients with a median age of 66 years were included. Conventional intensive chemotherapy (CHT) regimens were initially administered to 58% of patients, while targeted therapies were used in 37% of cases. The ratio of total days hospitalized to the overall follow-up period was 17%, with a total of 182 hospitalizations (average of 5 per patient; mean duration 20 days). The mean total reimbursement was EUR 109,104 per patient, primarily attributed to hospital admissions (EUR 89,158; EUR 18,118 per hospitalization). Hospitalizations before or without any allogeneic hematopoietic stem cell transplant (alloHSCT) accounted for 70% of total admissions, with an average overall cost of EUR 50,285 per patient (EUR 12,427 per hospitalization). AlloHSCT-period hospitalizations occurred in 18 patients, with a mean total reimbursement of EUR 122,497 per patient and EUR 30,464 per hospitalization. Conclusions: The active treatment of BPDCN imposes a high economic burden and extensive HCRU. Comparative pharmacoeconomic studies evaluating the cost-effectiveness of new therapies for BPDCN are needed to identify patient subgroups that may benefit most from these treatments.