Search Results (3,806)

Background: Left atrial appendage closure (LAAC) is an established alternative to oral anticoagulation for stroke prevention in patients with nonvalvular atrial fibrillation who are at high risk of thromboembolic events or bleeding complications. Methods: In this single-center retrospective study, we analyzed 70 consecutive patients who underwent successful LAAC with the Watchman™ device between 2012 and 2024. Acute procedural outcomes, long-term thromboembolic and bleeding events, transfusion requirements and mortality were evaluated. Mean follow-up duration was 1210 days. Results: Procedural success was achieved in 98.6% of cases with a low periprocedural complication rate. Ischemic stroke/transient ischemic attack occurred in 2.8% of patients; no hemorrhagic strokes or stroke-related deaths were observed. LAAC resulted in a significant reduction in both the number (144 vs. 56 events; 2.36 vs. 1.55 events per patient, p < 0.05) and severity of bleeding events. Nonetheless, 42.9% of patients required bleeding-related hospitalization after implantation, predominantly within the first 6 months during dual antiplatelet therapy. Overall mortality was 40% with a 12% yearly mortality rate; heart failure and infections were leading causes of death. Pre- and postprocedural transfusion requirements were independently associated with a six-fold increase in mortality risk (HR = 5.97). Conventional risk scores (CHA₂DS₂-VASc, HAS-BLED) failed to predict transfusion needs; atrial enlargement, right ventricular dysfunction, smoking and alcohol consumption were associated with higher risk. Conclusions: LAAC is a safe and effective alternative to long-term anticoagulation, significantly reducing bleeding burden without increasing thromboembolic mortality. Persistent postprocedural bleeding remains a major determinant of long-term prognosis, underscoring the need for individualized, multidisciplinary post-implant management. Full article

Background: Iron deficiency remains a major nutritional challenge, partly due to the limited stability and bioavailability of conventional iron formulations in foods and during digestion. In this study, iron–protein microparticles (IP-MPs) based on bovine serum albumin (IA-MPs) and hemp protein (IH-MPs) were developed via coprecipitation and evaluated as food-compatible iron delivery systems. Methods: Iron–protein microparticles (IP-MPs) were fabricated by a coprecipitation technique. The stability of IP-MPs was investigated in a three-phase digestion model. The uptake of IP-MPs by Caco-2 cells as well as the Ferritin concentration in Caco-2 cells were investigated. Results: Particle morphology and size distribution were strongly dependent on the protein matrix, with hemp protein microparticles exhibiting greater size uniformity and higher stability under simulated gastric conditions. In a standardized in vitro gastrointestinal digestion model, both IP-MP formulations preserved iron predominantly in the bioactive Fe(II) state and remained sufficiently intact to reach the intestinal phase. Biocompatibility and iron uptake were assessed using Caco-2 cell monolayers. Neither formulation induced cytotoxic effects, while iron delivered via IP-MPs showed enhanced cellular uptake compared to a commercial iron supplement and ferrous sulfate. The amount of Fe(II) detected in the basolateral compartment of IH-MP and IA-MP samples (1.4 µg and 1.3 µg, respectively) was higher than that observed for Floradix^® samples (approximately 0.7 µg) and corresponded to about 25% of the total iron applied. Functional iron bioavailability, assessed by ferritin formation, was significantly higher for IP-MPs, with hemp protein microparticles yielding the strongest ferritin response. Conclusions: These results demonstrate that iron–protein microparticles, particularly those based on hemp protein, effectively improve iron stability during digestion and enhance cellular iron bioavailability, highlighting their potential for application in iron fortification and functional food systems. Full article

Chronic lung allograft dysfunction (CLAD) remains the principal limitation to long-term survival after lung transplantation (LT). Early molecular alterations within the graft may precede clinically overt functional decline, but their biological significance remains incompletely defined. In this single-center exploratory pilot study, 16 bilateral lung transplant recipients underwent bronchoalveolar lavage (BAL) sampling at 7 days, 15 days, and 3 months post-transplantation. BAL-derived microRNA (miRNA) profiles were analyzed longitudinally and correlated with long-term clinical outcomes, including CLAD development and phenotypic classification into bronchiolitis obliterans syndrome (BOS) or restrictive allograft syndrome (RAS), over extended follow-up (mean 98 months). Distinct early miRNA signatures were detectable within the first weeks after transplantation and were associated with divergent long-term clinical trajectories. Specific miRNAs, namely let-7e-5p and miR-30d-3p, were associated with subsequent CLAD, whereas differential expression patterns distinguished trajectories toward BOS or RAS. Enrichment analyses highlighted networks related to innate immune activation, hypoxia, tissue remodeling, and PI3K–mTOR signaling. Notably, the occurrence of acute rejection did not differ significantly between patients who developed CLAD and those who remained stable. These findings, although preliminary, suggest that early BAL-derived miRNA profiles may reflect biologically distinct graft states associated with long-term CLAD phenotypes. Full article

Background and Objectives: Laparoscopic procedures have become a routine approach in colorectal surgery. We aimed to evaluate intraoperative, postoperative and pathological outcomes of laparoscopic right hemicolectomy in comparison with open right hemicolectomy. Materials and Methods: We reviewed our database for colorectal surgery and collected data regarding right hemicolectomies performed over a period of 10 years regarding patient characteristics, operative outcomes and postoperative outcomes. We compared laparoscopic with open right hemicolectomies. All the anastomoses in the laparoscopic group were performed intracorporeally. Results: We included 384 cases, 74 (19.3%) laparoscopic and 310 (80.7%) open right hemicolectomies. Baseline characteristics were comparable between the two groups. Conversion rate was low (2.7%). A drain was placed more often in the open colectomies (p < 0.001). Laparoscopic colectomies lasted longer by 25 min on average in the entire cohort (p = 0.002) and by 30 min in cancer-only cases without concomitant procedures (p < 0.001). Laparoscopic procedures yielded more lymph nodes (p = 0.007), as well as longer distal resection margins (p < 0.001) and total specimen (p < 0.001). There was no difference between the two approaches concerning intraoperative complications (p = 0.36) or need for transfusion (p = 0.708). There was also no difference regarding overall (p = 0.361) or major complications (p = 1), as well as anastomotic leak (p = 0.475), surgical site infections (p = 0.275) or readmission rates (p = 1). Hospitalisation duration was shorter by 3 days after laparoscopic surgery in the entire cohort (p < 0.001), as well as when cancer-only cases without concomitant procedures were considered (p < 0.001). Conclusions: Laparoscopic right hemicolectomy with intracorporeal anastomosis provides perioperative safety and pathology outcomes comparable to open surgery, while significantly reducing hospital stay. Full article

Background/Objectives: Patients with severe hemophilia A on prophylaxis with emicizumab exhibit a mild/moderate bleeding phenotype that requires the use of either recombinant FVIII (rFVIII) or bypassing agents (BPAs) in patients with inhibitors, in the case of breakthrough bleeding or surgery. Since factor IX (FIX) limits the formation of the FIXa–emicizumab–FX complex, exogenously added FIX might enhance complex formation and thrombin generation. This study aimed to compare the procoagulant effects of various FIX concentrates with recombinant activated FVII (rFVIIa), activated prothrombin complex concentrate (aPCC), and rFVIII in SHA patients with and without inhibitors under emicizumab prophylaxis. Methods: Hemostatic changes were monitored using two optimized global coagulation assays: rotational thromboelastometry and calibrated automated thrombin generation. Tubes containing corn trypsin inhibitor (CTI) were used during blood collection to prevent activation. Low concentrations of tissue factor (TF) were used to trigger coagulation in both assays. Results: Ex vivo addition of recombinant FIX concentrates significantly increased the procoagulant activity of emicizumab, achieving levels comparable to therapeutic doses of rFVIIa or rFVIII, and the proportion of active FIXa within the concentrates is a major contributor to their procoagulant function. We assessed the influence of FVIII inhibitors on the hemostatic efficacy of rFIX concentrates and BPAs, finding that rFIX-induced thrombin generation increased in the presence of inhibitors, and no significant differences were observed with BPAs. Conclusions: These findings suggest that FIX concentrates could be an effective alternative to BPAs for emicizumab-treated patients, particularly those with inhibitors. Further studies are needed to confirm their in vivo efficacy and to evaluate thrombotic risk. Full article

Invasive candidiasis (IC) remains a significant cause of morbidity and mortality among critically ill, hematologic, and neonatal patients worldwide. Rapid and accurate diagnosis is essential to guide timely antifungal therapy and improve outcomes. Among available diagnostic tools, 1,3-β-D-glucan (BDG), a polysaccharide component of the fungal cell wall, has emerged as a key biomarker. BDG assays allow for early detection of probable IC, often preceding positive blood cultures, and offer prognostic information based on serial measurements. Species-specific differences in Candida cell wall composition influence BDG release and diagnostic sensitivity. Candida albicans generally correlates with high BDG levels, whereas Nakaseomyces glabrata, Candida parapsilosis, and Candida auris exhibit variable or lower glucan exposure, limiting assay sensitivity. BDG performance is affected by patient-specific factors, such as prior surgery, transfusions, or coexisting bacterial infections, which may lead to false-positive results. Molecular techniques, including PCR-based assays, provide complementary diagnostic accuracy and species identification, and their combination with BDG testing enhances sensitivity up to 90%. Serial BDG monitoring supports risk stratification and treatment response assessment, with persistent elevations predicting worse outcomes. In neonatal and pediatric populations, optimal cut-off values remain under investigation, highlighting the need for integration with clinical and microbiological data. Overall, BDG represents a valuable adjunct in a multimodal diagnostic workflow, providing both diagnostic and prognostic insights in invasive candidiasis management. Full article

Background: Children with β-thalassemia major (β-TM) survive longer due to advances in transfusion and chelation therapy; however, cardiovascular complications have emerged as a leading cause of long-term morbidity. Chronic hemolysis, oxidative stress, and iron overload may promote early endothelial dysfunction and premature vascular aging, yet their impact on myocardial deformation in pediatric patients remains incompletely characterized. Objectives: To evaluate subclinical myocardial dysfunction and arterial stiffness in children with β-TM and to investigate hemolysis-related changes in asymmetric dimethylarginine (ADMA) and L-arginine as biomarkers of endothelial dysfunction in relation to cardiovascular involvement. Methods: Twenty-four children with β-TM and 20 age-matched healthy controls were included. Cardiac structure and myocardial deformation were assessed by conventional echocardiography, tissue Doppler imaging, and speckle-tracking strain analysis. Arterial stiffness was evaluated using oscillometric pulse wave analysis and bilateral carotid intima–media thickness (CIMT). Serum ADMA and L-arginine levels were measured, and hemoglobin, reticulocyte count, and ferritin levels were recorded. Results: Children with β-thalassemia major demonstrated significantly increased arterial stiffness compared with controls, including higher PWV (4.61 ± 0.37 vs. 4.38 ± 0.31), AIx@75 (augmentation index at 75 bpm) (28.5 ± 8.34 vs. 22.8 ± 6.51), left CIMT [0.45 (0.39–0.51) vs. 0.41 (0.38–0.46)], and right CIMT [0.43 (0.39–0.54) vs. 0.40 (0.34–0.46)]. In addition, patients exhibited reduced global longitudinal strain (−19.3 ± 2.91 vs. −21.84 ± 1.91), prolonged isovolumetric relaxation time [53 (37–71) vs. 45 (37–55)], and elevated E/Em (8.44 ± 2.19 vs. 6.92 ± 1.10). ADMA levels were significantly higher in patients (0.54 ± 0.19 vs. 0.39 ± 0.22) and were positively associated with reticulocyte counts and inversely correlated with hemoglobin levels. In addition, both ADMA and ferritin levels were positively correlated with arterial stiffness indices and left ventricular filling pressures. Conclusions: Children with β-thalassemia major exhibit features suggestive of early cardiovascular aging, including impaired myocardial deformation, diastolic involvement, and increased arterial stiffness. The observed association between ADMA levels and markers of hemolysis, vascular stiffness, and myocardial deformation highlights the potential involvement of endothelial dysfunction in premature myocardial–vascular remodeling. These findings suggest that ADMA may serve as a promising biomarker for early cardiovascular risk in pediatric β-thalassemia major; however, further longitudinal and multi-center studies are needed to confirm its clinical utility for risk stratification. Full article

Hemotherapy in small ruminants is indicated for several acute and chronic conditions; however, its clinical use is often limited by the difficulty in identifying suitable donors, particularly regarding blood volume availability and hematologic compatibility. Xenotransfusion in small ruminants with bovine blood may represent a practical alternative in emergency situations involving severe anemia when homologous donors are unavailable. This study evaluated the clinical, hematologic, biochemical, and blood gas responses of sheep subjected to acute blood loss followed by bovine whole blood xenotransfusion. Six healthy adult castrated male sheep (mean body weight 44.3 ± 7.2 kg) underwent removal of 40% of their estimated total blood volume. Parameters were assessed before hemorrhage induction (T0) and at times T30, T6h, T12h, T24h, T48h, T72h, T96h, T5d, T6d, T7d, T8d and T16d after transfusion. Acute blood loss significantly reduced packed cell volume and erythrocyte count at T0 (p < 0.05). After xenotransfusion, packed cell volume increased at T30min, T6h, and T12h and remained stable until T72h (p < 0.05), with progressive erythrocyte recovery and sustained macrocytosis. Total leukocyte count remained unchanged, whereas platelets increased at T7D (p < 0.05). Total protein decreased at T0 and subsequently increased. Transient elevations in urea, creatinine, glucose, pO₂, and SO₂ were observed (p < 0.05), without acid–base imbalance. Clinical parameters progressively stabilized, and no severe transfusion reactions occurred. Bovine whole blood xenotransfusion may represent a promising therapeutic alternative for sheep subjected to acute blood loss under the experimental conditions evaluated in this study. The procedure was associated with improvements in clinical, hematological, and biochemical parameters, and no severe transfusion reactions were observed during the monitoring period. These findings support the potential clinical applicability of this approach as an emergency intervention in situations where homologous donors are not readily available. Full article

Background/Objectives: Damage Control Resuscitation (DCR) is a critical strategy in the management of severe trauma, focusing on the optimisation of the patient’s physiological condition. This study reviews current DCR strategies, emphasizing the mitigation of the “diamond of death”—hypothermia, acidosis, coagulopathy, and hypocalcemia—while addressing complex disturbances like respiratory distress syndrome (ARDS) and (acute kidney injury) AKI in high-ISS (Injury Severity Score) patients. Methods: A systematic review of 59 contemporary sources was conducted, encompassing clinical trials (e.g., CRASH-2), military-to-civilian protocol translations, and guidelines from the C and European Resuscitation Council. The analysis focused on pre-hospital interventions, in-hospital transfusion protocols, and the impact of transport logistics on survival. Results: Evidence highlights that aggressive crystalloid resuscitation (over 5 L) significantly increases mortality, favoring balanced blood component therapy (1:1:1 ratio) or Whole Blood guided by viscoelastic testing like rotational thromboelastometry (ROTEM) or thromboelastography (TEG). Pre-hospital success is driven by rapid hemorrhage control via tourniquets, early administration of Tranexamic Acid (TXA), no aggressive crystalloids, permissive hypotension, proactive calcium supplementation is recommended in early care. Furthermore, the integration of Helicopter Emergency Medical Services (HEMS) is independently associated with improved survival in multi-organ trauma by reducing time to definitive care and facilitating “en-route” damage control. Conclusions: The evolution of rescue strategies focused on mitigating the effects of the diamond of death, combined with the implementation of permissive hypotension and optimized HEMS logistics, constitutes the foundation of a modern model aimed at minimizing mortality in multi-organ trauma. Full article

Background: Coronary button reimplantation is a key determinant of operative safety in the modified Bentall procedure (MBP), and technical modifications aimed at improving anastomotic stability and hemostasis continue to evolve. This study investigated the early outcomes of a posterior Teflon-felt-reinforced coronary button technique in comparison with the conventional approach. Methods: Between January 2021 and May 2025, a total of 57 patients who underwent an elective modified Bentall procedure were included and divided into two groups: the conventional coronary button group (CCB, n = 30) and the posterior Teflon-felt-reinforced coronary button group (RCB, n = 27). Operative variables and early postoperative outcomes (including bleeding, re-exploration, and 30-day mortality) were compared between the two groups. Results: The CCB group included 9 women and 21 men with a mean age of 59.5 ± 9.6 years, whereas the RCB group consisted of 5 women and 22 men with a mean age of 57.3 ± 8.9 years. The mean maximum aortic root diameter was 49.6 ± 5.3 mm, and the mean ascending aortic diameter was 50.8 ± 4.9 mm. Aortic cross-clamp (ACC) and cardiopulmonary bypass (CPB) times were similar between the groups (p = 0.330 and p = 0.214, respectively). After excluding patients who underwent planned coronary artery bypass grafting (CABG; n = 8), the incidence of unplanned CABG was higher in the CCB group than in the RCB group [6 (24.0%) vs. 2 (8.3%); p = 0.136]. Postoperative 24-h chest tube drainage tended to be lower (p = 0.060), and re-exploration for bleeding occurred less frequently (11.1% vs. 30.0%, p = 0.076), with no coronary button-related bleeding after reinforcement. The RCB group required significantly fewer transfused blood products, including red blood cells, fresh frozen plasma, and platelets (all p < 0.01). Intensive care unit stay was shorter in the reinforced group (p < 0.01), with a trend toward reduced hospital stay (p = 0.085). Early mortality was comparable (p = 0.356). Conclusions: Posterior Teflon-felt-reinforced coronary button anastomosis may improve early hemostatic stability and provide additional mechanical support during coronary reimplantation in the modified Bentall procedure; confirmation in larger cohorts is required. Full article

Background: Accurate identification of patients at high risk of perioperative blood transfusion is essential for optimizing patient blood management (PBM) strategies in oncological surgery. However, the performance of standard PBM eligibility criteria in real-world oncological settings remains incompletely characterized. Material and Methods: We conducted a retrospective, single-center analysis of 4228 consecutive patients undergoing elective oncological surgery of any complexity or liver transplantation over a 9-month period to assess transfusion need and estimate access to preoperative patient blood management (PBM) strategies to improve anemia management. Transfusion events were assessed within 24 h after surgery (PS24) and during the perioperative period (PO; 48 h before to 72 h after surgery). Two PBM eligibility strategies were applied to the same patient cohort and compared: (A) an observational approach, based on predefined PBM indicators (transfusion rate and transfusion index by surgical complexity), and (B) a multivariable modeling approach based on pre- and intraoperative anesthesiology assessment to estimate individual transfusion risk. Predictive performance of both strategies was evaluated using accuracy, Cramér’s V, area under the receiver-operating characteristic curve (AUC-ROC), and Brier score. Results: Overall, 7.7% of patients received transfusion within PS24 and 9.2% during PO. According to the observational approach, 23.8% of patients were classified as PBM-eligible, accounting for 89.2% of PS24 transfusions and 87.1% of PO transfusions. In the multivariable modeling approach, independent predictors of transfusion included surgical type (e.g., sarcoma surgery: OR 22.8 for PS24; OR 6.3 for PO; vs. senology surgery OR 1 for PS24; OR 1 for PO, respectively), anemia severity (moderate anemia: OR 64.3 and OR 107.9, respectively and mild anemia OR 3.38 and OR 3.65, respectively), high surgical complexity, operative time >3 h (>3 h: OR 8.83 and OR 8.65, respectively vs. <3 h OR 1 and OR 1, respectively), and ICU admission risk. The observational approach demonstrated stronger alignment with actual transfusion events (Cramér’s V = 0.44–0.47) and higher overall accuracy (90.8–92.3%); in contrast, a multivariable modeling approach showed superior discrimination (AUC = 0.94–0.95) and lower Brier scores, indicating better individual risk prediction. Conclusions: In a large real-world cohort of oncological surgical patients, standard PBM eligibility criteria effectively identified the majority of patients requiring perioperative transfusion. While multivariable modeling provided greater predictive precision, the observational PBM approach demonstrated strong clinical alignment and practical applicability. Integrating both strategies may support more effective transfusion risk stratification and PBM planning in oncological surgery. Full article

Background: MSCs possess strong immunoregulatory properties and play a central role in maintaining immune homeostasis by limiting inflammatory responses. Their function is highly plastic and influenced by environmental cues, including viral signals. How SARS-CoV-2-derived antigens affect MSC immunoregulation remains incompletely understood. This study aimed to investigate the impact of SARS-CoV-2 peptides on MSC-mediated immune modulation of T-cells. Methods: MSCs were stimulated directly with SARS-CoV-2 spike protein S peptides or cocultured with SARS-CoV-2 peptide-activated T-cells. TLR4 surface expression and receptor downstream signaling were assessed to evaluate pathway activation. MSC immunoregulatory function was analyzed by measuring suppression of TNF-α and IFN-γ expression and induction of CD4⁺FOXP3⁺ regulatory T-cells. TLR4 inhibition and lipopolysaccharide (LPS) stimulation were used to examine pathway specificity and interaction. Results: SARS-CoV-2 peptides activated TLR4-associated signaling in MSCs, increasing TLR4 expression and NF-κB phosphorylation. Peptide-treated MSCs showed impaired suppression of pro-inflammatory cytokines and reduced induction of regulatory T-cells. TLR4 inhibition prevented these effects. LPS induced similar effects, while combining LPS and peptide stimulation partially restored physiological T-cell cytokine suppression. Conclusions: SARS-CoV-2 peptides modulate MSC immunoregulatory function on T-cells via TLR4-dependent mechanisms. Full article

Objectives: Real-world data on surgical and multimodal management of upper tract urothelial carcinoma (UTUC) are limited. This study examined epidemiological trends, nephron-sparing surgery adoption, and the perioperative impact of lymphadenectomy (LND) and neoadjuvant chemotherapy (NAC). Methods: The German Nationwide Inpatient Data (GRAND) registry (2005–2023) identified UTUC patients undergoing radical nephroureterectomy (RNU), endoscopic laser destruction, or segmental ureteral resection (SUR) using OPS codes. Demographics, comorbidities, complications, and in-hospital mortality were extracted from ICD-10-GM data. Multivariable regression adjusted for baseline comorbidities assessed associations between treatment type, LND, NAC, and perioperative outcomes. Results: Among 53,427 UTUC patients, 77.3% underwent RNU, 13.8% endoscopic laser destruction, and 8.9% SUR. Endoscopic laser use rose from <10% (2005) to about 20% (2023). LND was performed in 13% of RNU cases, increasing from 1.1% to 19%. LND was associated with higher risks of transfusion (OR 1.47, 95% CI 1.37–1.57), acute kidney injury (OR 1.19, 95% CI 1.07–1.32), and ICU admission (OR 1.21, 95% CI 1.13–1.30), without affecting in-hospital mortality. NAC was given to 1.7% of patients, with a five-fold increase over time, and was associated with more transfusions (OR 1.28, 95% CI 1.07–1.52) and urinomas (OR 2.31, 95% CI 1.31–3.78), but not mortality. Conclusions: UTUC management is evolving, with growing use of endoscopic laser therapy and guideline-aligned lymphadenectomy during nephroureterectomy. Neoadjuvant chemotherapy remains underused despite acceptable perioperative safety, highlighting the need for increased awareness to optimize multimodal treatment. Full article

Background: An appropriate and effective management of haemophilia is currently based on a multidimensional evaluation of treatment adequacy. Current clinical practice however is still lacking standardised tools able to combine clinical, functional, and patient-reported outcomes. In this study a structured Monitoring Tool for haemophilia A and B was developed and validated through a Delphi-based expert consensus process. This study represents an expert consensus-based validation of a monitoring framework, rather than a clinical validation in patient cohorts. The tool is intended for use by haemophilia treaters during routine follow-up visits to support structured treatment reassessment. Score categories reflect the need for clinical re-evaluation or potential treatment optimisation, rather than disease severity. Methods: Italian haemophilia specialists were asked to participate to a panel over a two-round Delphi process. Experts rated the relevance of several predefined clinical domains—pharmacokinetics, bleeding episodes, joint health, adherence and quality of life (QoL)—and the individual items within each domain for patients on prophylactic or on-demand treatment. Consensus was defined by responses within an interquartile range (IQR) < 8. Section and item weights and Likert-based scoring values were used to reach a composite score between 0 and 100. Results: Consensus was achieved for all domains and items across haemophilia types and treatments, prophylaxis and on demand (Haemophilia A: 16 and 12 participants; Haemophilia B: 12 and 9, respectively). With reference to prophylaxis domains, bleeding episodes received the highest domain weight (31–32%), followed by joint health (27–29%) and adherence/QoL (21–23%) and pharmacokinetics (18–19%). For on-demand treatment, pharmacokinetics was excluded; bleeding episodes (38–40%) and joint health (35–37%) remained dominant. At the item level, dynamic joint health indicators (HJHS and HEAD-US changes) and longitudinal QoL changes consistently received the highest weights. The final scoring system categorised results as Excellent (0–25), Suboptimal (26–50), Poor (51–75), or Critical (76–100). Conclusions: The Delphi-validated Monitoring Tools provide a structured, weighted, and clinically relevant framework for assessing treatment adequacy in haemophilia A and B across prophylactic and on-demand settings. These tools allow multidimensional outcome assessment and may support a more consistent, personalised therapeutic decision-making. A prospective validation of the tool in clinical cohorts is warranted. Full article