Patients In Argentina Research Articles

Real world evaluation of etelcalcetide in the treatment of secondary hyperparathyroidism in hemodialysis patients in Argentina.

Secondary hyperparathyroidism (sHPT) is a common complication in patients with chronic kidney disease (CKD). Recently, etelcalcetide (EC), an intravenous calcimimetic, has been introduced as a treatment. We evaluated the efficacy of EC in treating sHPT. We conducted a multicenter, observational, retrospective study involving hemodialysis patients with sHPT, treated for at least 3 months with EC. We analyzed baseline and follow-up values of intact parathyroid hormone (iPTH), calcium (Ca), phosphate (P), and alkaline phosphatase (ALP). Age, sex, time on dialysis, dialysate calcium concentration, and use of active vitamin D and phosphate binders were also recorded. Patients were divided into those receiving EC as de novo or after at least 3 months of cinacalcet treatment, and according to sHPT severity: PTH <1000 and >1000 pg/mL. The study included 196 patients. Mean age was 52 ± 15 years; 52.3% were male. Median time on hemodialysis was 46.5 (20-72) months. Significant reductions were observed in baseline iPTH (1053 pg/mL), Ca (8.7 mg/dL), and P (5.7 mg/dL) over 2 years (p < 0.0001), while ALP levels remained stable. iPTH reduction >30% was achieved in 37.5%, 64%, 66.7%, and 62.5% of patients at 3, 6, 12, and 24 months, respectively. EC was administered as initial treatment in 53% of patients, while 47% were switched from cinacalcet. Significantly iPTH reduction was observed in both groups. Greater reductions were noted in patients with initial PTH >1000 pg/mL (p = 0.009). Two patients discontinued due to severe hypocalcemia. EC effectively lowered iPTH and P levels, with a sustained effect over 2 years.

Insights into Acinetobacter baumannii AMA205's Unprecedented Antibiotic Resistance.

The rise of antibiotic-resistant bacteria in clinical settings has become a significant global concern. Among these bacteria, Acinetobacter baumannii stands out due to its remarkable ability to acquire resistance genes and persist in hospital environments, leading to some of the most challenging infections. Horizontal gene transfer (HGT) plays a crucial role in the evolution of this pathogen. The A. baumannii AMA205 strain, belonging to sequence type ST79, was isolated from a COVID-19 patient in Argentina in 2021. This strain's antimicrobial resistance profile is notable as it harbors multiple resistance genes, some of which had not been previously described in this species. The AmpC family β-lactamase blaCMY-6, commonly found in Enterobacterales, had never been detected in A. baumannii before. Furthermore, this is the first ST79 strain known to carry the carbapenemase blaNDM-1 gene. Other acquired resistance genes include the carbapenemase blaOXA-23, further complicating treatment. Susceptibility testing revealed high resistance to most antibiotic families, including cefiderocol, with significant contributions from blaCMY-6 and blaNDM-1 genes to the cephalosporin and carbapenem resistance profiles. The A. baumannii AMA205 genome also contains genetic traits coding for 111 potential virulence factors, such as the iron-uptake system and biofilm-associated proteins. This study underscores A. baumannii's ability to acquire multiple resistance genes and highlights the need for alternative therapies and effective antimicrobial stewardship to control the spread of these highly resistant strains.

P144 PHENOTYPIC CHARACTERISATION OF HEART FAILURE WITH PRESERVED EJECTION FRACTION ACCORDING TO BODY MASS INDEX: SUBANALYSIS OF THE ARGENTINE ARFEY-PRESER REGISTRY

Background: Nutritional status, characterised by body mass index (BMI), may influence therapeutic decisions in heart failure with preserved ejection fraction (HFpEF). The objectives of this study were to identify clinical and echocardiographic characteristics associated with nutritional status and to analyse therapeutic strategies in HFpEF patients in Argentina. Materials and Methods: This prospective, multicentre observational registry enrolled outpatients or those discharged from hospitals, with prior informed consent. The study spanned from December 2022 to February 2024. Inclusion criteria were: 1) presence of current signs and symptoms, LVEF ≥50%, 2) structural heart disease with LVH, LAE, or average E/e’ ≥15 on Doppler, 3) elevated natriuretic peptides: sinus rhythm: NTproBNP &gt;125 pg/ml or BNP &gt;35 pg/ml; Atrial fibrillation/flutter: NTproBNP &gt;375 pg/ml or BNP &gt;105 pg/ml; and 5) commitment to 6-month and 1-year follow-ups. Exclusion criteria comprised: 1) acute coronary syndrome, 2) coronary artery bypass grafting, or valve replacement within the preceding 3 months, 3) confirmed infiltrative or hypertrophic cardiomyopathy, severe valvular disease, 4) severe illness with &lt;1-year prognosis, and chronic renal failure on permanent dialysis. Results: A total of 449 patients were enrolled, with 440 having available BMI data for this analysis. Patient enrolment was consistent across centres and nationwide. BMI analysis revealed 0.7% undernutrition, 16.6% normal weight, 32% overweight, and 50.7% obesity. In Figure1 showed the analysis of clinical, laboratory, echocardiographic, and HF severity variables according to BMI. Significant differences were found in treatment, with lower use of RAS inhibitors and beta-blockers in non-normal weight patients compared to those with overweight and obesity. Conclusions: For a similar HFpEF severity profile, the phenotype according to BMI suggests different clinical characteristics. Underweight/normal weight patients were older, with more LAE and pulmonary hypertension, and fewer risk factors. Obesity was present in half of the cases, characterised by younger patients with a high burden of cardiovascular risk factors. This impacts the use of certain heart failure medications.

P153 NATIONAL SURVEY OF ARGENTINE DOCTORS ON HYPERTENSION

Background and Objective: Hypertension (HTN) is the leading cardiovascular risk factor, yet its control remains low. Identifying factors influencing diagnosis, potential clinical inertia, and the measuring devices used is crucial. Objective: This study aims to understand factors in medical practice affecting HTN knowledge and control, and to assess the availability and use of blood pressure (BP) measurement devices. Methods: This observational, cross-sectional study is based on a 25-question self-administered survey by the Argentine Society of Hypertension (SAHA) and the Argentine Society of Medicine (SAM), targeting doctors treating HTN patients in Argentina. This report focuses on BP measurement. Results: A total of 1,042 responses were received, with women comprising 45.8% (n:471). Responses came from 430 cardiologists (41.7%), 380 internists (36.8%), 68 HTN specialists (6.6%), 54 primary care physicians (5.2%), and 100 from other specialties (9.7%). An own validated automatic arm BP monitor was used by 44.5%, a manual aneroid by 36.5%, an institution's validated automatic arm monitor by 10.9%, and a fixed wall aneroid by 8.2%. BP was measured in all patients by 89% of respondents, sometimes by 8.5%, and only if the patient was hypertensive by 1.7%. Ambulatory blood pressure monitoring (ABPM) was always requested before treatment by 25.2%, sporadically by 34%, never by 4.1%, and only for BP between 130/85 mmHg and 159/99 mmHg by 36.7%. The 24-hour ABPM values for initiating pharmacological treatment were &gt;140/90 mmHg (40.9%), &gt;135/85 mmHg (39%), and &gt;130/80 mmHg (20.1%). Conclusions: Discrepancies exist in HTN patient care. Only 20% of doctors start pharmacological treatment at 24-hour ABPM values above 130/80 mmHg. Enhancing education and information could improve HTN control strategies.

Clinical efficacy of a next-generation sequencing (NGS) and immunohistochemistry (IHC) panel in patients with advanced cancer in Argentina.

e15154 Background: The analysis of somatic genomic alterations, evaluation of DNA mismatch repair (MMR) proteins, and examination of PD-L1 expression in tumor biopsies are crucial for prognosis and informed therapeutic decisions. However, their clinical implementation in Argentina faces challenges due to economic costs and lack of evidence. To address this, we conducted a retrospective study to assess the impact of this molecular panel testing on treatment decisions and clinical management of cancer patients in Argentina. Additionally, we evaluated its ability to detect clinically relevant alterations and analyzed the frequency and co-occurrence of mutations in our cohort. Methods: Tumor tissue samples from 266 patients with primary tumors, representing 26 different anatomical sites (53% lung cancer), underwent molecular testing using the Optimus panel developed by Biomakers. The assay integrates somatic sequencing of 52 genes through NGS with IHC assessment of MMR and PD-L1 proteins. A retrospective evaluation of the clinical effectiveness of the test was conducted on 133 patients. Genomic and clinical data were analyzed using R with the pairwise Fisher’s exact test applied for statistical analysis. Results: This panel facilitates molecular characterization, identifying clinically relevant genetic alterations in 65% of patients and detecting deficiency in MMR proteins in 3%. Findings align with global clinical trial availability for identified alterations in 97% of cases. KRAS is the most frequently mutated gene (38%), followed by EGFR (19%), PIK3CA (12%), BRAF (9%), CDK4, and CTNNB1 (5% each), correlating with lung cancer overrepresentation. Simultaneous alterations in EGFR, PIK3CA, CTNNB1, and KIT genes were observed. Pan-tumoral interaction analysis revealed mutual exclusivity between KRAS mutations and those in EGFR, BRAF, CDK4, and CTNNB1. CTNNB1 mutations co-occurred with EGFR mutations, akin to KIT and PDGFRA (p&lt;0.05). The retrospective analysis indicated that the Optimus panel contributed to clinical management of patients in 56% of cases, with 85% detecting clinically relevant variants, aiding treatment definition in 74% of these cases. Conclusions: The Biomakers-developed Optimus panel empowers the molecular characterization of genomic and proteomic biomarkers across tumor types. Its significance in clinical practice lies in contribution to vital decision-making processes, particularly in treatment selection.

Dravet Syndrome: An Electroclinical, Genetic, Treatment, and Outcome Study of 35 Patients in Argentina

AbstractWe analyzed the electroclinical features, molecular findings, treatment, disease course, and outcomes of patients with Dravet syndrome (DS) with positive genetic markers seen at a public hospital in Argentina. A retrospective study was conducted assessing the clinical records of 44 patients who met the diagnostic criteria for DS according to the 2022 classification of epilepsy of the International League Against Epilepsy seen at our center between March 2018 and June 2023. Of 44 patients, 35 (18 males and 17 females), in whom genetic studies yielded positive results, were included. Median age was 9 years (range 4 to 16 years), and the median time of follow-up was 10 years (range 3 to 14 years). The mean age at onset was 7 months. The first seizure was associated with febrile illness in all patients, and in 11 (31.4%), seizures were immediately preceded by either infectious disease or vaccination. Heterozygous pathogenic/likely pathogenic SCN1A variants were detected in 32 of the original 44 patients (73%), of which 47% were novel. Variants in other genes related to DS (HCN1, STXB1, and SCN1B) were identified in three patients. Cognitive delay and motor impairment were found to be more severe in patients that had multiple and drug-resistant seizures and in those who had the complete phenotype with myoclonic seizures. Novel SCN1A gene variants were identified in nearly half of the patients. The prognosis for cognitive development is unfavorable. Seizures are not well controlled with antiseizure medications and early treatment with ketogenic dietary therapy as well as cannabidiol should be considered.

Abstract PO3-08-06: Challenges in Implementing Genetic Counseling and Genetic Testing for Breast Cancer: Insights from the First 1000 Families Assessed in a Public University Hospital in Argentina

Abstract Introduction: Integrating genetic testing into breast cancer patients care is crucial for personalized treatment recommendations, risk reduction strategies, and family members evaluation. However, Argentina faces several concerns in implementing these services. Objective: This study aims to explore the challenges encountered in implementing genetic testing for breast cancer, drawing insights from the assessment of the first 1000 patients at a public university hospital in Argentina. Methods: We conducted a retrospective analysis of patients who underwent genetic counseling in our institution from January 2015 to June 2023. We also analyzed patients who underwent surgery from June 2022 to May 2023. Results: 1032 patients were evaluated in our unit. 836 patients (81%) met the National Comprehensive Cancer Network (NCCN) criteria for genetic testing. Genetic testing was requested for 575 patients (68.7%) Only 44% of patients were able to access the test, with 60% receiving only the BRCA1/2 test (see Table 1 and 2 for details). In the analysis of breast cancer surgeries (n=246), 62% (n=154) met the NCCN criteria for genetic testing. However, only 56% of these patients underwent genetic counseling prior to their surgery and only 15 (9.86%) received their results before primary treatment. Discussion Unfortunately, only 9.8% of patients have the results of genetic testing prior to their primary treatment, indicating a critical need for timely referrals and mainstream genetic testing. To counteract, our university hospital's breast cancer unit has taken proactive measures since January 2015. Genetic counseling has been established as a standard of care for all patients, and a streamlined workflow has been implemented to optimize referrals. Trained professionals, including a genetic counselor, psycho-oncologist, and geneticist, have been integrated into core teams. Additionally, healthcare professional continued education has played a pivotal role in improving referrals. Additionally, we have introduced an easy workflow to optimize referrals to genetic counseling. Completion rate of requested genetic tests is concerning, with 56% of tests not being completed, and 60% of the completed tests limited to BRCA1/2. Improving access to genetic testing is imperative. Currently, comprehensive genetic testing is inaccessible to patients without medical coverage, and even those with coverage face difficulties in testing access. Moreover, patient education plays a vital role in promoting the importance of genetic testing and its impact on patient care and their families. In collaboration with the Argentinean Breast Pathology Society, we have developed two informative videos that reinforce knowledge provided by primary care physicians and breast surgeons. Conclusion, Our study is the first in Argentina to analyze the numerous challenges faced in conducting genetic testing for breast cancer in a public hospital. By addressing these challenges collectively, we can facilitate the effective integration of genetic counseling and testing into routine practice for breast cancer patients in Argentina. Table 1: Breast Cancer Genetic Counseling Unit Patients (2015-2023) Table 2: Breast Cancer Genetic Counseling Unit Patients studies (2015-2023) Citation Format: Dolores Mansilla, Diana Bequelman, Jorgelina Cavallero, Hernan Ursino, Berman Gaston, Ipiña Martin, Eduardo Armanasco, Andrea Aguilar, Valeria Caceres, Eugenia Azar. Challenges in Implementing Genetic Counseling and Genetic Testing for Breast Cancer: Insights from the First 1000 Families Assessed in a Public University Hospital in Argentina [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO3-08-06.

Treatment of Alzheimer's Disease in the New Era of Monoclonal Antibodies Against Cerebral Amyloid-β: Pharmacological Prescription and Knowledge in Argentina.

Alzheimer's disease (AD) presents a significant global health challenge. Understanding the current and upcoming treatment landscape is crucial for effectively managing patients. The aim of this study was to assess the pattern of prescription and knowledge about new therapies by physicians who treat AD patients in Argentina. A cross- sectional and analytic study was conducted. A survey was elaborated about pharmacological treatment in AD. Statistical analysis of answers of specialists in cognitive disorders (SCD), non-specialists in cognitive disorders (NSCD), recommended treatment, non-recommended treatment (NRT), and off-label treatment was performed. 155 physicians answered the survey. A 19.35%prescribed at least one NRT for dementia. 78.06%prescribed at least an off-label treatment or an NRT for mild cognitive impairment (MCI). 31%would prescribe monoclonal antibodies (MABs) against cerebral amyloid-β (Aβ) to AD patients, and 42.6%responded that they were not aware of any adverse effect of these. Quetiapine was the most frequent treatment for psychotic symptoms (88.4%) and escitalopram (32.3%) for apathy. A 70%of potential prescribers of MABs (n = 100) would request biomarkers of cerebral Aβ in the initial assessment. There were significant differences between the responses of SCD and NSCD regarding the prescription of MABs (52.17%versus 23.08, respectively) and knowledge about adverse events (76.09%versus 38.46%, respectively). A considerable percentage of physicians indicated NRT and off-label medication in MCI and dementia. In Argentina, there are many physicians who would indicate a MABs for AD, but many are not completely aware of its safety profile.

Seventy years of pheochromocytomas and paragangliomas in Argentina. The FRENAR database

Pheochromocytomas and paragangliomas (PPGL) are neuroendocrine tumors characterized by the excessive production of catecholamines. This study aims to describe the clinical characteristics of PPGL cases in Argentina over recent decades. A multicenter retrospective cross-sectional analysis was carried out using a database comprising both pediatric and adult patients with confirmed PPGL diagnoses based on pathological reports.A cohort of 486 patients with PPGL was recruited. Women represent 58.4% of the patients, with a mean age of 38.3 years old at the time of diagnosis and 15.2% of the patients were under the age of 18. Hypertension, as well as classic signs and symptoms, were present in 80.9% of the patients. The adrenal incidentaloma, as a mode of presentation, increased in the last two decades rising from 3.9% (1953–2000) to 21.8% (2001–2022), p<0.001.Most tumors were located within the adrenal glands, accounting 83.0% of the cases, with bilateral occurrences noted in 20.0%. The median tumor size was 4.8cm. Local recurrence and metastases were observed in 10.9% and 12.2%. Out of 412 patients, 87.0% exhibited urinary excretion elevation of catecholamines and/or their metabolites. Furthermore, 148 patients, representing 30.4% of the study population, displayed a distinct genetic profile indicative of hereditary syndromes. The distribution of hereditary syndromes revealed that MEN2, VHL, and PGL4 constituted the most prevalent syndromes.This population-based study, spanning seven decades, offers valuable insights into the demographic and clinical characteristics of PPGL patients in Argentina.

Patients' and Caregivers' Experiences Navigating the Burden of Atopic Dermatitis in Argentina.

Background and Objectives: Little is known about patients' and caregivers' experiences with atopic dermatitis (AD) in Argentina, so a survey was administered to learn more. Materials and Methods: A 53-item anonymous survey was administered in Spanish to adult AD patients (n = 334) and caregivers (n = 339) of pediatric AD patients in Argentina (total n = 673). Demographics, healthcare provider information, financial burden, disease severity, disease burden, level of disease-specific education, and experience with shared physician/patient decision making were collected. Linear and logistic regression models were used for statistical comparisons. Results: Survey respondents were overwhelmingly female (90.8%), as was the overall patient population (72.8%). Patients were seen mostly by healthcare specialists (66.8% dermatologists, 13.5% pediatricians, 7.7% allergists, and 7.2% general practitioners). Only 2.8% of respondents reported no symptoms, while 33.3%, 52.4%, and 11.5% reported mild, moderate, and severe AD disease, respectively. Anxiety/depression and pain/discomfort were the most impactful on respondents' quality of life. Caregivers of children with moderate to severe AD and adult patients with severe AD reported a significant financial burden, including using savings or not purchasing food or other essentials to afford medical care. Few people reported receiving disease-specific education or having their own treatment priorities taken into consideration. For adult patients, receiving disease education and being asked about treatment priorities were associated with higher treatment satisfaction and AD control. Discussion: Mental health, pain/discomfort, and financial worries are the most important burdens for adult AD patients and caregivers of children with AD in Argentina. We recommend prioritizing disease-specific education and shared decision making to improve AD care in Argentina.

Identifying Who Benefits the Most from a Community Health Worker-Led Multicomponent Intervention for Hypertension.

Uncontrolled hypertension is a major public health challenge in low- and middle-income countries. The Hypertension Control Program in Argentina (HCPIA) showed that a community health worker-led multicomponent intervention was effective for blood pressure (BP) reduction in resource-limited settings, but whether the intervention was equally effective across participant subgroups is unknown. To identify participants who benefit the most from the HCPIA BP control intervention. This secondary analysis used data from HCPIA, a successful 18-month cluster-randomized trial in 18 health centers with 1,432 low-income hypertensive patients in Argentina. Fifteen baseline characteristics were used to define subgroups. The proportion of controlled BP (<140/90 mmHg) was estimated using generalized linear mixed models with arm-by-subgroup interaction terms. The distribution of trial BP response among intervention patient subgroups was assessed. Participants were 53.0% female, a mean age of 56 years, and 17.4% controlled BP at baseline. After the intervention, 72.9% of intervention and 52.2% of control participants had controlled BP. The intervention was more effective in physically inactive patients (OR = 2.76, 95% CI: 1.82 and 4.21; p for interaction = 0.04), moderately active patients (OR = 3.08, 95% CI: 1.90 and 4.99; p for interaction = 0.03), and those with uncontrolled BP at baseline (OR = 2.77, 95% CI: 2.15 and 3.57; p for interaction = 0.05). Among intervention participants, 20.2% had no BP response (BP change < -4 mmHg), 41.3% had a moderate BP response (BP change: -4 mmHg to -24 mmHg), and 38.5% had a high BP response (BP change > -24 mmHg). Women (p=0.01), those who were physically inactive (p=0.03), and those not taking antihypertensive medications at baseline (p=0.001) had the greatest BP response. The effect of the intervention was consistent across many subgroups with some key groups showing a particularly strong intervention effect. These findings could be useful for planning future hypertension control programs in low- and middle-income countries.

Clinical characterization of long-term multiple sclerosis (COLuMbus) patients in Argentina: A cross-sectional non-interventional study

BackgroundMost Multiple Sclerosis (MS) clinical trials fail to assess the long-term effects of disease-modifying therapies (DMT) or disability. MethodsCOLuMbus was a single-visit, cross-sectional study in Argentina in adult patients with ≥10 years of MS since first diagnosis. The primary endpoint was to determine patient disability using the Expanded Disability Status Scale (EDSS). The secondary endpoints were to evaluate the distribution of diagnoses between relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS), patient demographics, disease history, and the risk of disability progression. The relationship between baseline characteristics and the current disability state and the risk of disability progression was assessed. ResultsOut of the 210 patients included, 76.7 % had a diagnosis of RRMS and 23.3 % had been diagnosed with SPMS, with a mean disease duration of 17.9 years and 20.5 years, respectively. The mean delay in the initial MS diagnosis was 2.6 years for the RRMS subgroup and 2.8 years for the SPMS subgroups. At the time of cut-off (28May2020), 90.1 % (RRMS) and 75.5 % (SPMS) of patients were receiving a DMT, with a mean of 1.5 and 2.0 prior DMTs, respectively. The median EDSS scores were 2.5 (RRMS) and 6.5 (SPMS). In the RRMS and SPMS subgroups, 23 % and 95.9 % of patients were at high risk of disability, respectively; the time since first diagnosis showed a significant correlation with the degree of disability. ConclusionsThis is the first local real-world study in patients with long-term MS that highlights the importance of recognizing early disease progression to treat the disease on time and delay disability.

Experience with Blinatumomab in Pediatric Patients in Argentina. Multi-Center Study

Introduction. Very high-risk and relapsed pediatric acute lymphoblastic leukemia (ALL) require high-dose chemotherapy and hematopoietic stem cell transplant (HSCT) as consolidation of treatment. Patients with positive minimal residual disease (MRD) previous to HSCT have lower survival rates. Immunotherapy is thus useful for management of MRD previous to HSCT with less chemotherapy-associated morbidity. Blinatumomab (Blina) is a bi specific monoclonal antibody against CD19 and CD3ε. Objectives. To report the experience with Blina in children with B-ALL in Argentina and assess its toxicity, effectiveness and overall survival (OS). Patients and methods. Retrospective analytical study. The main Argentinian pediatric hospitals participated. Patients younger than 17 years old with B-ALL in 1 st, 2 nd or 3 rd CR who had received 1 or 2 cycles of Blina were included. Those with negative MRD and those who didn´t complete a cycle were excluded. Toxicity was evaluated according to CTC 4.0 and effectiveness according to pre and post-infusion MRD. OS was calculated using Kaplan Meier with GraphPad Prism V5.0. Results. Patients. Thirty patients received Blina and 6 were excluded: 3 due to negative MRD, 2 didn´t complete 1 cycle and 1 for both reasons.Eligible: 24 patients. The median age was 5.5 (range 0.8-17) years. Patients under 1 year: 3. Distribution by sex F/M= 1/1.8. Patients with High Risk ALL represented 42% (n=10) and relapsed ALL 58% (n=14), 10 in 1 st relapse and 4 in 2 nd relapse. Sixteen patients received 1 cycle and 8 received 2. Median MRD previous to Blina was 0.71 (range 0.023-49)%. Thirteen patients received it in CR (MRD &amp;lt;1%): 7 in 1 st CR, 4 in 2 nd CR, and 2 in 3 rd CR. MRD was 1-5% in 6 patients and &amp;gt;5% in 5. Toxicity. During the 1 st and 2 nd weeks, 25% (n=6) and 16% (n=4) presented GII-III neurological toxicity respectively. Blina infusion was temporarily withdrawn for 3 hours to 12 days in 6 patients but all completed the cycle. During the 1 st week, 41.5% (n=10) presented fever. During the entire 1 st cycle 25% (n=6) presented GIII-IV hematological toxicity, 2 patients presented GIV and 1 GIII infectious toxicity. Regarding the 8 patients who received a 2 nd cycle, 1 patient presented febrile neutropenia and 2 presented GIII infectious toxicity without evidence of neurological toxicity. Systemic inflammatory response syndrome was not evidenced. There were no fatal events related to Blina administration. Efficacy. After the 1 st cycle of Blina 58% became MRD negative (n=14), 17% remained in CR (n=4) and 25% didn´t show any response. All patients who received a 2 nd cycle were in CR; however, 5 of them became MRD negative and 3 relapsed later. Ten of the 13 patients who received Blina in CR with MRD+ became negative and 3 relapsed. Of the 5 with MRD 1-5%, 4 became negative, 1 achieved CR with MRD+ and 1 progressed. Of the 5 patients with MRD&amp;gt;5%, 2 achieved CR but none became MRD negative. Fifty percent of the patients received HSCT (n=12): 6 haploidentical, 2 related allogeneic and 4 unrelated. With a median follow-up of 3.5 (1.7-56.9) months, OS at 36 months was 20.3%. Nine patients are alive and in CR (37.5%), 6 post-HSCT and 3 post-Blina still receiving treatment. Five died in CR (4 due to HSCT complications) and 10 died due to disease relapse/progression (3 post HSCT and 7 post-Blina). Conclusions. Blina infusion was safe with the previously described toxicities. The highest response rate was evidenced in patients in CR or with MRD 1-5% pre-Blina. The low survival rate in this cohort may be due to the high rate of relapse/progression and toxicity associated with HSCT, although HSCT is essential to consolidate Blina´s response. Blina therapy is effective in patients with very high risk or relapsed ALL with +MRD. The strategy should focus on the precise indication of patients with MRD+ as a bridge to HSCT.

Long-Term Follow-up of the AST Trial of Treatment-Free Remission in Chronic Myeloid Leukemia Patients

Background: Treatment-free remission (TFR) is a new goal for chronic myeloid leukemia (CML) patients who achieved a long deep molecular response (DMR) with better quality of life, reduced adverse events and high economic impact. The primary objective of the AST trial was to estimate molecular relapse rate at 24 month in CP-CML patients who discontinued TKI. Secondary objective to explore predictive markers for sustained TFR through clinical and immunological analysis ( innate inmune response). This update provides longer follow-up data and includes a new cohort of patients. Patients and Methods: AST-Argentina Stop Trial (NCT05926128) is a multicenter prospective study that included patients from 15 centers. Recruited patients ≥ 18 years, treated with TKI for at least 4 years, sustained MR 4.0 for ≥ 2 years and confirmed typical BCR-ABL1 b3a2 and/or b2a2 transcripts. Molecular studies were centralized in 2 internationally standardized laboratories and were performed monthly during the first 6 months, every 2 months until the first year, and every 3 months during the second year. TKI was restarted due to loss of major molecular response (MMR). Expression of multiple NK cell immunophenotypic markers(CD3-CD56+) was analyzed by flow cytometry at discontinuation time and 3 months later. The nonparametric Mann-Whitney test was used to compare the differences between relapsed and responding patients. Molecular relapse-free survival was estimated using the Kaplan-Meier method and optimal cut-off points were obtained using ROC curves. Comparisons between survival curves were made using the Log rank test method. The differences were considered statistically significant with p-values less than 0.05. Multivariate analysis was performed using the COX regression model. Results: A total of 81 patients divided into 2 cohorts were evaluated: 46 patients recruited between February 2018 and July 2020 (1st cohort) and 35 patients recruited between July 2022 and April 2023. Between these two periods, recruitment was stopped due to the COVID19 pandemic. This analysis focuses on the 1st cohort that has completed 24 months in the study. The median age was 57.5 years (24-86), median duration of treatment before discontinuation was 10.5 years (4.2-20). TKI treatment prior to discontinuation was as follows: Imatinib in 34 (73%) patients, Dasatinib 8 (17%), and Nilotinib 4 (9%). According to Sokal risk score: 22 (48%) were low risk, 14 (30%) intermediate, and 10 (22%) high risk. Seventeen patients (37%) lost MMR, leading to a 63% molecular relapse-free survival rate with a median follow-up of 47 months (41-52). All patients who lost MMR recovered it after restarting the same TKI, with a median time to response of 2.8 months (1-7). With a prolonged follow-up, 2 late relapses were observed (18 and 42 months) (Fig 1). For a univariate analysis, the continuous variables were dichotomized seeking to maximize the difference between the different cohorts. The differences were significant in the variables time in DMR (cut-off point: 51 months, p=0.0166)(HR=0.97 (0.94 - 0.99); p=0.013). Two of the NK cell markers showed significant differences between groups at month 3 after starting treatment, PD1 (Mann-Whitney p=0.035; Log-Rank test p=0.033) and NK-p44 (Mann-Whitney p=0.015; Log -Rank test p=0.020). Multivariate analysis using the Cox model that included variables as gender, age at diagnosis, time in DMR, time in TKI prior to discontinuation, Sokal score, and type of inhibitor, the clinical predictor of time in DMR was the only predictor, significantly associated with a higher rate of TFR, (HR = 0.97 (0.95 - 0.99); p=0.020). No patient experienced disease progression to advanced stages or death for any reason. Conclusion:This is the first multicenter study of TKI discontinuation in CML patients in Argentina showing that TKI can be safely discontinued in those who achieve and maintain a DMR before discontinuation. Molecular relapse-free survival rate was slightly higher than that reported in the international literature, and longer follow-up confirmed the sustained response. DMR before discontinuation showed to be a robust predictor of TFR. Although the sample size was increased, longer follow-up is needed to validate these findings in the second cohort. Potential advantages of TFR will not only impact in patients' quality of life but also show considerable reduction in the use of economic resources.

Validation of the Spanish ASQ translation: Screening pediatric patients for suicide-risk in Argentina

BackgroundThe high frequency of suicide risk in adolescents necessitates the development and validation of specific tools for systematic screening. To date, there are translated, but not validated suicide risk screening tools in Spanish. ObjectiveTo validate the Spanish version of the Ask Suicide-Screening Questions (ASQ) for suicide risk screening in pediatric patients in Argentina. MethodUsing a cross-sectional multicenter design, a convenience sample of pediatric patients aged 10 to 18 years old were recruited from outpatient/inpatient medical settings and private psychiatric clinics. The Spanish version of the Suicidal Ideation Questionnaire (SIQ) assessment tool was used as a standard criterion to validate the ASQ. ResultsA total of 301/380 pediatric patients were screened for suicide risk. Twentyeight percent of the entire sample (83/301) of youth screened positive on the ASQ, and 21% (62/301) screened positive on the SIQ/SIQ-JR and were considered “at risk” for suicide. Compared with the SIQ, the Spanish ASQ yielded a sensitivity of 96.8% (95% Confidence Interval [CI]: 88.8–99.6%), specificity of 90.4% (95% CI: 85.9–93.8%), positive predictive value of 72.3% (95 CI: 61.4–81.6%), and negative predictive value of 99.1% (95% CI: 96.7–99.9%). The positive Likelihood Ratio (LR) was 10.1 (95% CI: 6.1–14.0), and the negative LR was 0.03 (95% CI: −0.01–0.09). Kappa was 0.77 (95% CI: 0.69–0.86), and the Area Under the Curve was 0.94 (95% CI: 0.91–0.97). ConclusionThe Spanish language ASQ demonstrated strong psychometric properties, providing initial evidence that it is a valid tool for identifying Spanish-speaking youth at risk for suicide.

First identification of Strongyloides stercoralis infection in a pet dog in Argentina, using integrated diagnostic approaches

BackgroundStrongyloides stercoralis is a soil-transmitted intestinal nematode with a complex life cycle that primarily affects humans, non-human primates, dogs, and occasionally cats. This study presents, to the best of our knowledge, the first case of S. stercoralis infection and its genotyping in a domestic dog from Argentina.MethodsThe patient was a female wired-haired Teckel dog exhibiting recurrent coughing. Coproparasitological analysis using the Baermann technique revealed the presence of rhabditiform larvae morphologically compatible with S. stercoralis. To confirm this finding, molecular diagnosis (18S ribosomal RNA) and analysis of the cox1 gene were performed.ResultsWe identified a haplotype (HP20) that has previously only been related to S. stercoralis infection in dogs, but was found in the present study to be highly related to the haplotype (HP16) of a zoonotic variant and divergent from those previously described from human patients in Argentina. Furthermore, unlike in human cases following treatment with ivermectin, the dog was negative after moxidectin treatment according to polymerase chain reaction of the sampled faeces.ConclusionsThis case report shows the importance of further investigation into potential transmission events and prevalences of S. stercoralis in dogs and humans in South America. The results reported here should also encourage future work that examines different scenarios of infection with S. stercoralis in dogs and humans with the aim of integrating clinical management, diagnosis, treatment and follow-up strategies in the quest for new approaches for the treatment of this disease in animals and humans. The findings support the adoption of a One Health approach, which recognizes the interconnectedness between animal and human health, in addressing parasitic infections such as strongyloidiasis.Graphical

PB0080 The Effect of COVID-19 Pandemic in Haemophilia Patients in Argentina

PB0080 The Effect of COVID-19 Pandemic in Haemophilia Patients in Argentina

Frequency of new asymptomatic MRI lesions during attacks and follow-up of patients with NMOSD in a real-world setting.

We aimed to assess the frequency of new asymptomatic lesions on brain and spinal imaging (magnetic resonance imaging (MRI)) and their association with subsequent relapses in a large cohort of neuromyelitis optica spectrum disorder (NMOSD) patients in Argentina. We retrospectively reviewed 675 MRI (225 performed during an attack and 450 during the relapse-free period (performed at least 3 months from the last attack)) of NMOSD patients who had at least 2 years of clinical and MRI follow-up since disease onset. Kaplan-Meier (KM) curves were used for depicting time from remission MRI to subsequent relapse. We included 135 NMOSD patients (64.4% were aquaporin-4-immunoglobulin G (AQP4-IgG)-positive). We found that 26 (19.26%) and 66 (48.88%) of patients experienced at least one new asymptomatic MRI lesion during both the relapse-free period and attacks, respectively. The most frequent asymptomatic MRI lesions were optic nerves followed by short-segment myelitis during the relapse-free period and attacks. KM curves did not show differences in the time taken to develop a new relapse. Our findings showed that new asymptomatic lesions are relatively frequent. However, the presence of new asymptomatic MRI lesions during the relapse-free period and at relapses was not associated with a shorter time to developing subsequent relapses.

Overall survival in treated ovarian cancer patients in Argentina: OCEANIA study.

e17595 Background: OCEANIA is a retrospective, real-world database study that evaluated treatment patterns and outcomes in a cohort of patients diagnosed with ovarian cancer (OC) in private healthcare settings in Latin America. The present analysis focused on evaluating up to five-year overall survival (OS) in a cohort of treated patients with OC in Argentina. Methods: Patients with an OC code diagnosed from 2010 to 2019 and with a cancer-related treatment claim (antineoplastics, surgery, and/or radiotherapy) were selected from a private healthcare provider’s database (Hospital Italiano de Buenos Aires) in Argentina. Patients were followed from diagnosis until the end of observation period or death. Frequency of OC patients by FIGO (International Federation of Gynecology and Obstetrics) stage was described. Cumulative treatment type was described through the whole follow-up period. One- to five-year survival Kaplan–Meier curves were built; patients who were lost to follow-up were censored. Results: 741 patients with OC were identified in the database with a mean (standard deviation) follow-up time of 31.7 (30.6) months; 539 (72.7%) were treated during this period. Among this population, 76.3% had information about FIGO staging: 73 (17.8%) were stage 1; 36 (8.8%) stage 2; 259 (63.0%) stage 3; and 43 (10.5%) stage 4. Cumulative distribution across treatment types revealed 355 (65.9%) patients received antineoplastics (321 [90.4%] received platinum-based treatment), 496 (92.0%) surgery and 18 (3.3%) radiotherapy. During a five-year observation window, 111 patients died (more than one half within two years of diagnosis). The adjusted cumulative OS for all treated patients in years 1–5 was 93.2%, 87.2%, 80.9%, 74.1%, and 69.0%, respectively (Table). Conclusions: OS is a key outcome in assessing the impact of current standards of care. Based on the results, although most (69.0%) of the patients survived after 5 years of follow-up, further improvement in OS presents a challenge. However, recent drug releases for OC management in Latin America may change this scenario. [Table: see text]

Toxicity and Outcomes of Ibrutinib in Chronic Lymphatic Leukemia-Real-World Results from the Study of 215 Patients in Argentina

Different pivotal works allowed the approval of ibrutinib as a CLL treatment, both in the first line and in relapsed/refractory patients, the adverse effects differ from conventional chemotherapy, the discontinuation rate was 10%, but in different real-life studies, showed a higher number of complications and medication suspension rates of 40 to 50%. To know the reality of the use of ibrutinib in CLL in our environment, we designed this work with the objectives of evaluating the safety profile of the drug with the description of the adverse effects, their incidence, and their severity. The clinical efficacy will also be studied with the determination of the response achieved, calculation of overall survival (OS) and progression-free survival. Also as an additional objective, the discontinuation rate and its causes will be obtained. A total of 215 patients in 26 centers throughout the country were retrospectively analyzed. 189 patients (88%) had a global response, and the majority 58% achieved a partial response. The 5-year overall survival was 60%, with no difference between patients with different numbers of previous lines. The progression-free survival of the entire group was 5.06 years. 44.7% of the population, presented at least 1 adverse effect. The most frequent ones were: bleeding, thrombocytopenia, pneumonia and diarrhea. Others presented: anemia, neutropenia, infections, AF, HTA, arthralgia, rash, opportunistic infections. Most adverse events were mild to moderate in grade and generally occurred within the first 6 months. The main cause of treatment suspension was the appearance of adverse effects, 37 patients had to suspend it, 27 due to adverse effects (72.97%). In our work, we found that ibrutinib, as a single agent, has outstanding activity in CLL, with a significant percentage of overall responses, even in patients with several lines of prior treatment. Most of the adverse effects were of a mild to moderate degree. The cardiovascular effects of TKI, HBP, and AF, are in percentages similar to those reported in other studies. If we analyze the percentage of treatment discontinuation, which is 17%, mostly due to adverse effects, this finding is similar to pivotal studies. In conclusion, our real-life study confirms the important activity of ibrutinib in patients with CLL, in the first line and relapses, highlighting the low percentage of treatment discontinuation in our environment. We believe our work reflects the real life and daily care of patients with CLL, under treatment with ibrutinib in our environment.