Search Results (800)

Background: Children with inflammatory bowel disease (IBD) are at heightened risk for vaccine-preventable infections because of underlying immune dysregulation and long-term immunosuppressive therapy. Despite published guidelines affirming vaccine safety, real-world coverage remains suboptimal. It is a pilot, single-country survey designed to explore baseline knowledge and practices regarding vaccination in paediatric IBD within a specific local healthcare context. Objective: The objective of this study is to evaluate the knowledge, attitudes, and practices of paediatric gastroenterologists (PGs) regarding the immunisation of children with IBD. Methods: We conducted an exploratory pilot, cross-sectional survey of paediatric gastroenterologists in Russia, focusing on immunisation knowledge and practical barriers in routine care. A cross-sectional, anonymous online survey was distributed to PGs nationwide between January 2022 and April 2022. The online questionnaire explored demographic characteristics, awareness of international recommendations, perceptions of vaccine safety at various disease and treatment stages, and routine vaccination practices. Responses were analysed with non-parametric statistics (α = 0.05). In a parallel prospective cohort, the vaccination certificates of 98 paediatric IBD patients (January 2022–April 2023) were audited to quantify real-world coverage. Results: Fifty-one PGs completed the survey. Forty-one per cent agreed that vaccines do not provoke IBD flares, while 17.6% considered live vaccines acceptable during immunosuppressive remission. Nearly one-third (32%) did not personally oversee immunisation, and 18% occasionally discouraged vaccination during therapy. Only 35.3% deemed baseline serology essential before starting immunosuppression; 46.5% supported antibody checks immediately prior to vaccination. The certificate audit revealed a full schedule completion rate of 66.3% for measles–mumps–rubella and 74.2% for hepatitis B, contrasting with parental reports of 82.3% complete coverage. Conclusions: Knowledge gaps, limited guideline awareness, and parental concerns contribute to suboptimal vaccination of paediatric IBD patients. Targeted educational initiatives, clearer shared-care pathways, and routine certificate audits are needed to close the coverage gap and reduce infection-related morbidity. Findings are hypothesis-generating and reflect local practice; as a pilot study, results should be interpreted with caution and may not generalise beyond similar settings. Full article

The therapeutic landscape of adults with B-cell precursor acute lymphoblastic leukemia (BCP-ALL) is undergoing a paradigm shift, driven by the development of immunotherapy-based “chemo-free” and “chemo-light’ regimens. These strategies aim to achieve high efficacy with reduced toxicity, particularly in older adults who may not tolerate intensive chemotherapy. In Philadelphia chromosome-positive (Ph+) BCP-ALL, the incorporation of ABL tyrosine kinase inhibitors (TKIs) with blinatumomab (CD3/CD19 bispecific T-cell engager) has shown remarkable efficacy, with some studies reporting molecular response rates in the range of 90–100% and long-term survival exceeding 80% without the need for intensive chemotherapy or allogeneic hematopoietic cell transplantation (allo-HCT). In Philadelphia-negative (Ph−) BCP- ALL, an immunotherapy-based combination of blinatumomab and inotuzumab ozogamicin (anti-CD22 antibody-drug conjugate) has demonstrated high rates of complete remission and measurable residual disease (MRD) negativity, with manageable toxicity. While chimeric antigen receptor (CAR) T-cell therapy remains a transformative option for relapsed/refractory B-ALL, its integration into frontline treatment is still under investigation. Ongoing trials are evaluating the optimal sequencing and combinations of these agents and their potential to obviate the need for chemotherapy and/or allo-HCT in selected patients. As evidence continues to accumulate, chemo-free and chemo-light regimens, incorporating minimal chemotherapy with targeted agents to balance efficacy and reduced toxicity, are poised to redefine the standard of care for adults BCP-ALL, offering the possibility of durable remissions with reduced treatment-related morbidity. Full article

Background and Objectives: Inflammatory bowel diseases (IBDs) are chronic conditions of the digestive tract, often requiring life-long treatments in order to achieve and maintain remission. However, treatment adherence among patients with IBD can frequently be suboptimal, which can compromise disease control and long-term outcomes. The aim of this study was to analyze the adherence rate and to identify factors that significantly influence treatment adherence in patients with IBD. Materials and Methods: The study employed a cross-sectional design and was conducted at the Fundeni Clinical Institute, a tertiary medical center in Bucharest, Romania. The treatment adherence was assessed using the Medication Adherence Report Scale-5 (MARS-5), with patients scoring greater than 23 considered adherent. Anxiety, depression and perceived stress were assessed using the Depression, Anxiety and Stress Scale-21 (DASS-21). Perceived social support was measured with the Multidimensional Scale of Perceived Social Support (MSPSS), and coping strategies were assessed using the Brief Coping Orientation to Problems Experienced Inventory (Brief COPE Inventory). Results: A total of 188 patients were included in the final analysis. Of these, 99 patients (52.7%) were male and 109 (58.0%) had a diagnosis of Crohn’s disease. The majority of patients (81.9%) were receiving treatment with advanced therapies, including biologics or small molecules. Forty patients were receiving their therapy through more than one route of administration. Optimal adherence was noted in 160 patients (85.1%). Patients treated with advanced therapies (biologics and small molecules) had significantly higher odds of optimal adherence (OR 10.52, 95% CI: 4.3–25.74, p < 0.001), with a rate of adherence of 92.2%. Significantly lower odds of adherence were found for the oral (OR 0.35, 95% CI: 0.14–0.83, p = 0.01) and rectal (OR 0.09, 95% CI: 0.03–0.29, p < 0.001) routes of administration, while the intravenous administration had higher odds of adherence (OR 4.85, 95% CI: 1.02–22.9, p = 0.04) compared to the subcutaneous route. Other factors associated with an improved adherence were being retired (OR 3.5, 95% CI: 1.13–10.8, p = 0.029) and using positive reframing (p = 0.04), planning (p = 0.01) and venting (p = 0.02) as coping strategies; active smoking (OR 0.26, 95% CI: 0.11–0.6, p = 0.002), active disease (OR 0.36, 95% CI: 0.16–0.81, p = 0.014) and behavioral disengagement (p = 0.04) were associated with impaired treatment adherence. No significant differences in adherence were observed between disease phenotypes. Conclusions: The route of administration, smoking status and psychosocial factors, such as perceived stress of social support and coping strategies, may play an important role in influencing treatment adherence in patients with IBD. While the disease phenotype was not associated with differences in adherence, patients with active disease had significantly lower odds of optimal adherence. Full article

Accurate assessment of histological remission is a critical goal in the management of ulcerative colitis (UC); however, routine evaluation is hindered by the invasiveness of endoscopy and biopsy. Non-invasive alternatives like intestinal ultrasound (IUS) and faecal calprotectin (FC) show promise for monitoring mucosal inflammation, though their ability to predict histological healing remains underexplored. This systematic review and meta-analysis aimed to evaluate the diagnostic accuracy of IUS, FC, and their combined use for detecting histologic remission in patients with UC. A comprehensive literature search identified two eligible studies comprising 72 patients. Pooled estimates for IUS demonstrated high sensitivity (0.84, 95% CI: 0.35–0.98) but variable specificity (0.78, 95% CI: 0.08–0.99), while FC alone exhibited high sensitivity (0.85, 95% CI: 0.72–0.92) with moderate specificity (0.60, 95% CI: 0.38–0.79). Notably, only one study assessed the combined diagnostic approach, reporting superior performance with sensitivity and specificity of 0.88 and 0.80, respectively. The certainty of the evidence was rated as moderate. These exploratory findings suggest that a multimodal, non-invasive approach combining IUS and FC may improve diagnostic accuracy in detecting histological remission in UC, potentially reducing reliance on invasive procedures. However, given the limited number of studies included and the high degree of heterogeneity, these results should be interpreted with caution. Further large-scale, methodologically robust studies are needed to validate these preliminary findings and establish standardized diagnostic protocols. Full article

Background and objective: Thiopurines (azathioprine (AZA) and 6-mercaptopurine (6-MP)), used to maintain remission in inflammatory bowel diseases (Crohn’s disease (CD), ulcerative colitis (CU)) and autoimmune hepatitis (AIH), are responsible for a number of adverse effects. One is leukopenia, mainly due to neutropenia and less known lymphopenia. This study aimed to assess the incidence rate of lymphopenia in pediatric patients with CD, CU, and AIH treated with azathioprine (AZA) and to evaluate the impact of lymphopenia on the occurrence of opportunistic infections and its relationship with disease activity, treatment, and nutritional status. Materials and methods: A retrospective analysis was carried out in ninety-eight (98) paediatric patients, suffering from CD, CU, or AIH and treated with AZA, in order to assay blood cell count and thiopurine metabolite levels, assess the mean AZA dose, measure the anthropometric parameters, evaluate disease activity vs. the treatment administered, and to find out concomitant infections. Results: Lymphopenia was diagnosed in twenty-two (22) children and evaluated as severe in two (2) cases, which were associated with treatment discontinuation. The percentage of patients with lymphopenia in the CD group (34.5%) was significantly higher vs. the CU (3.7%) and AIH (7.7%) groups. The prevalence rates of the patients with low and moderate-to-high disease activity were 13.9% and 46.1%, respectively. The patients with lymphopenia demonstrated higher prevalence rates of mild respiratory tract and skin infections (identified in 32%). No cases of opportunistic infections were reported. Conclusions: Lymphopenia affected approximately one-quarter of the patients observed, the condition being transient in most cases and not demanding any therapy modifications. In no case was it associated with the occurrence of any opportunistic infections. It was significantly more common in the patients with Crohn’s disease and the subgroup with a more intense course of the disease, obviously suggesting a need for more frequent follow-up of the patients in those subgroups. The AZA therapy did not seem to be associated with lymphopenia occurrence in any significant way. Full article

Background: The therapeutic response in Graves’ Disease (GD) remains largely unpredictable. Patients often experience persistent symptoms that are poorly correlated with thyroid hormone levels, an undefined treatment duration, and the need for long-term or definitive therapies. Based on the nuclear antagonistic properties of L-carnitine (LCT) on thyroid hormone action and the immunomodulatory role of selenium (Se), we aimed to assess the impact of adding a combined LCT and Se supplement to standard methimazole (MMI) therapy on the biochemical profile and quality of life (QoL) of patients with overt GD. Methods: This multicenter prospective randomized trial enrolled 60 consecutive patients with newly diagnosed overt GD. Participants were randomized to receive either standard treatment with MMI alone (Control Group) or MMI plus the combined LCT/Se supplement (Intervention Group). TSH, fT3, fT4, and TSH–receptor antibodies (TRAb) levels were evaluated every two months for up to 24 months or until spontaneous remission or definitive therapy. At each visit, patients completed a symptom questionnaire addressing the frequency of typical thyrotoxic symptoms. Results: No significant differences were observed between groups in the trend or time-to-normalization of TSH, fT3, and fT4 levels. However, the Intervention Group reached TRAb negativity significantly earlier (HR = 2.35 (1.14–4.81), p = 0.016), with a synergistic interaction with MMI therapy. MMI requirements were consistently lower in the Intervention Group, both in average dosage (p = 0.013) and cumulative dose (p = 0.020). The rate of spontaneous remission was significantly higher (OR = 11.22 (3.35–46.11), p < 0.001). Overall symptom burden did not differ significantly between groups; however, the supplement exerted an independent effect in reducing the severity of tremor, irritability, mood lability, heat intolerance, and exertional dyspnea. Conclusions: Our findings suggest the clinical benefits of adding combined LCT and Se supplementation to MMI in the treatment of overt GD, including shorter disease duration, lower cumulative MMI exposure and earlier TRAb normality, that could positively influence TRAb-related prognostic outcomes. Full article

Background: Foot pain often persists in patients with rheumatoid arthritis (RA), even during clinical remission. However, its causes are not fully understood. Identifying factors specifically associated with metatarsal pain, rather than generalized foot pain, may improve targeted management strategies. Objectives: The aim of this study was to compare the clinical, biomechanical, and radiological characteristics of RA patients in remission with isolated metatarsal pain versus those with pain in other foot regions, and to identify independent predictors of metatarsal pain. Methods: This cross-sectional study included 118 RA patients in remission, classified into two groups: isolated metatarsal pain (n = 61) and pain in other foot regions (n = 57). Clinical variables (demographics, disease duration, treatment, comorbidities), biomechanical measures (ankle, first metatarsophalangeal and subtalar joint mobility, hallux valgus severity, foot type), radiographic findings (erosions, subluxations), and ultrasound-detected synovitis in the 2nd–5th metatarsophalangeal (MTP) joints were recorded. Independent predictors were identified using binary logistic regression. Results: Patients with metatarsal pain had higher rates of severe hallux valgus, MTP synovitis, and dislocations ≥ 50%. Independent predictors were hallux valgus (OR = 5.428, 95% CI: 1.528–19.287, p = 0.009), MTP synovitis (OR = 2.093, 95% CI: 1.337–3.275, p = 0.001), and MTP dislocations (OR = 2.092, 95% CI: 1.275–3.432, p = 0.003). Conclusions: Persistent metatarsal pain in RA remission is associated with a distinct structural and biomechanical profile. Comparing foot pain by location may help identify clinically relevant patterns and support more individualized assessment and treatment strategies. Due to the cross-sectional design, causality cannot be established. Full article

Background/Objectives: After first-line treatment, elderly patients with acute myeloid leukemia (AML) often become unfit to continue intensive chemotherapy despite having achieved a response. This trial aimed to determine the efficacy of maintenance treatment with azacitidine in AML patients who are ineligible to continue intensive treatment after remission. Methods: A single-arm, multicenter, phase II clinical trial (EudraCT: 2010-020432-18) including patients with AML with complete (CR) or partial remission (PR) after one or two cycles of intensive induction chemotherapy and ineligible to continue intensive treatment was conducted. Efficacy was measured as the response rate, progression-free survival (PFS), and overall survival (OS), and was assessed in the intention-to-treat (ITT) population (all patients). Quality of life was also assessed. Results: Thirty-two patients were included, with a mean age of 73.3 years (SD 3.8) (53.1% male); sixteen patients (50.0%) reached the sixth treatment cycle. The best response was CR in 11 patients (68.8%) and PR in 2 patients (12.5%) at cycle six, and CR in 15 patients (46.9%) and PR in 5 patients (15.6%) overall. The median PFS was 6.7 months (95% CI 3.1–8.7), and OS was 11.5 months (95% CI 6.6–15.9). The daily function (p = 0.0296), cognitive function (p = 0.0412), and social function (p = 0.0275) scales of the EORTC QLQ-C30 questionnaire significantly improved. Thirty-one patients (96.9%) experienced adverse events; six (1.9%) were serious. Conclusions: Azacitidine is a safe and well-tolerated maintenance treatment option for AML patients unfit for intensive therapy following a response to induction, although the single-arm phase II design precludes direct causal inference. Patients achieved promising results regarding PFS and remission rates, with improved quality of life. Full article

Background: Solar urticaria is a rare and disabling photodermatosis. Due to its low prevalence, most available data regarding treatment are derived from observational studies and case series, and a systematic evaluation of treatment efficacy is lacking. This systematic review and meta-analysis aims to assess therapeutic outcomes across treatment modalities in order to guide clinical care. Methods: We conducted a systematic literature search across PubMed, ScienceDirect, the Cochrane Library, and ClinicalTrials.gov. Studies reporting treatment outcomes in patients with solar urticaria were included. Pooled response rates were calculated for each treatment modality. Results: Out of 508 studies initially identified, 38 met the inclusion criteria. Antihistamines were evaluated in 21 studies (376 patients), with a pooled response rate (partial or complete) of 83.0% (95% CI, 70.4–91.1%) and a complete response rate of 7.7% (95% CI, 1.7–28.3%). Phototherapy was assessed in 11 studies (145 patients), showing a similar overall response (89.8%; 95% CI, 77.9–95.3%) but a higher complete response rate (39.8%; 95% CI, 18.3–66.1%). Omalizumab, evaluated in nine studies (76 patients), demonstrated the highest efficacy, with 93.2% (95% CI, 73.8–98.5%) achieving response and 68.4% (95% CI, 48.5–83.2%) complete remission. Limited data on IVIG, cyclosporine, and plasmapheresis suggested partial efficacy in selected refractory cases. Conclusions: This meta-analysis may support clinical decision-making by clinicians. A stepwise approach is suggested: high-dose H₁ antihistamines as first-line therapy, phototherapy as an alternative option in patients with access to treatment centers, and omalizumab for those with insufficient response. In refractory cases, additional options might be considered. Full article

Background/Objectives: Chronic hepatitis C virus (HCV) infection is associated with a wide spectrum of extrahepatic manifestations, involving the immune, dermatologic, endocrine, vascular, and neuropsychiatric systems. Among these, mixed cryoglobulinemic vasculitis (CryoVas) remains one of the most clinically relevant complications. This work aims to provide a structured overview of HCV-related extrahepatic conditions and to analyze the clinical and virological outcomes of direct-acting antivirals (DAAs) in CryoVas patients. Methods: We first categorized and reviewed extrahepatic manifestations of HCV across five major domains: immune, inflammatory/metabolic/vascular, dermatological, thyroid, and neuropsychiatric. Subsequently, we conducted a comparative analysis of five clinical studies evaluating the impact of DAA therapy in patients with CryoVas. Data on demographics, clinical symptoms, treatment regimens, sustained virological response, and clinical response were extracted and summarized. Results: HCV was found to be associated with numerous extrahepatic conditions, including mixed cryoglobulinemia, non-Hodgkin lymphoma, autoimmune thyroiditis, insulin resistance, and neurocognitive symptoms. In the CryoVas subgroup analysis, virological response rates were uniformly high (88.9–100%), but clinical remission varied significantly. Complete response ranged from 39% to 90%, highlighting a discrepancy between viral eradication and extrahepatic symptom resolution. These findings underscore the need for individualized follow-up and further investigation into persistent immunological dysfunction post-sustained virological response (SVR). However, clinical outcomes were more variable: complete response (CR) varied between 39% and 90%, partial response (PR) ranged from 4% to 42%, and no response (NR) was reported in 0% to 40% of cases. Although significant improvement in key manifestations such as purpura, arthralgia, and neuropathy was frequently observed, a subset of patients continued to exhibit residual or refractory symptoms despite achieving SVR. Conclusions: HCV infection exerts multisystemic effects that extend beyond liver pathology. While DAAs offer near-universal virological clearance, the heterogeneous clinical response in CryoVas underscores the need for closer monitoring of extrahepatic outcomes. Future research should assess whether combining DAAs with immunomodulatory strategies can improve symptom control and long-term outcomes in patients with severe or refractory CryoVas. Full article

Background: Relapsed/refractory acute myeloid leukemia (R/R AML) remains a therapeutic challenge due to disease heterogeneity, resistance mechanisms, and poor tolerability to intensive regimens. Venetoclax (VEN), a BCL-2 inhibitor, has shown promise in combination with hypomethylating agents (HMAs), but data on response timing in the R/R setting are limited. The aim of this study was to assess the efficacy, safety, and kinetics of response to HMA-VEN therapy in a real-world cohort of R/R AML patients, with particular focus on early versus late responders. Methods: This prospective single-center study included 33 adult patients with R/R AML treated with VEN plus either azacitidine (AZA) or decitabine (DEC) from 2018 to 2021. The primary endpoint was the composite complete remission (cCR) rate and the rate of early and late response, respectively, occurring within two cycles of therapy or later; secondary endpoints included overall survival (OS), relapse-free survival (RFS), time to relapse (TTR), and safety. Results: The cCR was 58%, with complete remission (CR) or CR with incomplete recovery (CRi) achieved in 52% of patients. Median OS was 9 months. No significant differences in OS or TTR were observed between early (≤2 cycles) and late (>2 cycles) responders. Eight responders (42%) underwent allogeneic hematopoietic stem cell transplantation (HSCT), with comparable transplant rates in both groups of responders. Toxicity was manageable. Grade 3–4 neutropenia occurred in all patients, and febrile neutropenia occurred in 44% of patients. An Eastern Cooperative Oncology Group (ECOG) score >2 was associated with inferior response and shorter treatment duration. Conclusions: HMA-VEN therapy is effective and safe in R/R AML, including for patients with delayed responses. The absence of a prognostic disadvantage for late responders supports flexible treatment schedules and suggests that the continuation of therapy may be beneficial even without early blast clearance. Tailored approaches based on performance status and comorbidities are warranted, and future studies should incorporate minimal residual disease (MRD)-based monitoring to refine response assessment. Full article

Background/Objectives: Acute severe ulcerative colitis (ASUC) is often managed by tacrolimus induction therapy followed by maintenance therapy. We compared the effectiveness of ustekinumab versus vedolizumab as maintenance therapies after tacrolimus induced improvement in patients with ASUC. Methods: This single-center retrospective cohort study included patients with ASUC who received tacrolimus induction therapy followed by ustekinumab or vedolizumab between January 2018 and November 2024. The primary outcome was clinical remission at week 16. Secondary and exploratory outcomes included clinical remission at week 8, biologic persistence, and relapse risk. An inverse probability of treatment weighting (IPTW) analysis was performed using the following covariates: male sex, prior biologics or JAK inhibitors, partial Mayo score, CRP, and albumin. Results: Among 235 tacrolimus-treated patients, 29 received ustekinumab and 22 received vedolizumab. After IPTW adjustment, the clinical remission rates were significantly higher in the ustekinumab group at both week 8 (82.1% vs. 51.8%, p = 0.02) and week 16 (85.4% vs. 36.8%, p = 0.02). Biologic persistence was significantly higher in the ustekinumab group (p = 0.004), and ustekinumab significantly reduced the hazard of relapse in multivariable analyses (HR 0.42 [95% CI: 0.20–0.88], p = 0.02). Conclusions: Ustekinumab showed greater effectiveness than vedolizumab in terms of achieving remission at 16 weeks after tacrolimus induction therapy in patients with ASUC. Full article

Background and Aim: Long-term treatment with biologic therapy alongside immunomAfodulators in patients with inflammatory bowel disease (IBD) can be associated with severe side effects. The objective of this study was to determine whether discontinuing anti-TNF treatment after two years in patients who have achieved mucosal healing is associated with lower relapse rates. Materials and Methods: A total of 67 patients with IBD from a single tertiary IBD Center who had achieved mucosal healing were enrolled in this retrospective study. In this single-center retrospective study (January 2014–December 2022), we screened 67 IBD patients in deep remission (endoscopic mucosal healing after ≥2 years of anti-TNF therapy). After excluding three patients without histologic data, 64 patients (25 ulcerative colitis, 39 Crohn’s disease) were analyzed. Mayo endoscopic sub-score and SES-CD were used to evaluate endoscopic activity after two years of anti-TNF therapy. Histological activity was assessed using the GHAS (for CD) and Nancy index (for UC). Results: A total of 67 patients were screened, of whom 3 were excluded due to a lack of biopsies. Of the 64 included patients, 39.06% (25/64) had UC and 60.9% (39/64) had CD, with a mean disease duration of 11.6 ± 8.0 years. All patients were in endoscopic remission at the time of therapy de-escalation, and 60.9% (39/64) also achieved histological remission (“deep remission”). In the follow-up of 38.6 months (IQR 30–48) after biologic therapy was stopped, 57.8% (37/64) relapsed with a median time to relapse of 13.5 months (IQR 8–24) off anti-TNF—a total of 34 patients required a restarting of biologic therapy. Using Spearman’s correlation, a moderate connection was observed between histological activity at withdrawal and subsequent relapse (rho = 0.467, p < 0.001). The probability of relapsing within 4 years after anti-TNF cessation was significantly higher (OR 2.72) in patients with histologically active disease at the time of de-escalation. Conclusions: Achieving ‘deep remission’ (clinical, endoscopic, and histological healing) may be a suitable parameter for making decisions on when to de-escalate therapy; however, given that over half of patients in endoscopic remission relapse after discontinuation, any de-escalation should be approached with caution and individualized patient assessment. Full article

Background/Objectives: The advent of tyrosine kinase inhibitors (TKIs) revolutionized the management of chronic myeloid leukemia (CML), achieving survival rates near those of the general population. Despite this success, prolonged therapy presents challenges, including physical, emotional, and financial burdens. Treatment-free remission (TFR), defined as sustained deep molecular response (DMR) after discontinuing TKIs, has emerged as a viable clinical goal. This study evaluates real-world data from the Canary Islands Registry of CML (RCLMC) to explore outcomes, predictors, and the feasibility of TFR. Methods: This retrospective observational study included 393 patients diagnosed with CML-CP between 2007 and 2023. Molecular response was monitored according to international guidelines. Survival probabilities were estimated using the Kaplan–Meier method. Logistic regression analysis was performed to identify predictors of molecular relapses after TKI discontinuation. Results: Of the 383 patients who received TKI treatment, 58.3% achieved molecular response grade 2 (MR2) (BCR-ABL1 ≤ 1%), 95.05% achieved MR2, and 50.5% reached MR4 within the first year. Of the 107 patients attempting TFR, 73.2% maintained remission at 36 months. Relapses occurred in 24 patients, all regaining molecular response upon reintroduction of TKIs. No cases of disease progression were observed. Conclusions: Our findings support the feasibility and safety of TFR in a real-world clinical setting for well-selected patients, with outcomes consistent with international studies. The study underscores the importance of molecular monitoring and patient-specific strategies to optimize outcomes. Full article

Background: CCAAT/enhancer-binding protein alpha–basic leucine zipper in-frame (CEBPA^bZIP-inf) mutations are associated with favorable outcomes in acute myeloid leukemia (AML). So far, there are limited data on integrating clinical and genomic features impacting the outcomes. Methods: Clinical and genomic data from consecutive patients with CEBPA^bZIP-inf were reviewed. A Cox proportional hazards regression was used to identify the variables associated with event-free survival (EFS), relapse-free survival (RFS) and survival. Results: 224 CEBPA^bZIP-inf patients were included in this study. In the 201 patients, except for the 19 receiving the transplant in the first complete remission with no events (the transplant cohort), multivariate analyses showed that IKZF1 mutations/deletions were significantly associated with poor EFS (p = 0.001) and RFS (p < 0.001); FLT3-ITD mutations, poor RFS (p = 0.048). In addition, increasing WBC count, lower hemoglobin concentration, non-intensive induction, and MRD positivity after first consolidation predicted poor outcomes. On the basis of the number of adverse prognostic covariates for RFS, the 201 patients were classified into low-, intermediate- or high-risk subgroups, and there were significant differences in the 3-year EFS, RFS and survival rates (all p < 0.001); however, except for survival in the low-risk group, these metrics were lower than those in the transplant cohort. Conclusions: We identified a potential high-risk population with adverse prognostic factors in CEBPA^bZIP-inf AML patients for which transplantation should be considered. Full article