FARMACIA HOSPITALARIA最新文献

Objectives: To assess changes in quality of life in patients with rare disease hereditary transthyretin amyloidosis after initiating vutrisiran. Secondary objectives included evaluating effectiveness and safety.

Methods: Prospective, observational study, conducted in a real-world clinical setting between November 2023 and May 2024. Quality of life was assessed using Norfolk QOL-DN questionnaire at baseline and six months after treatment initiation. Statistical analysis was performed using SPSS Statistics®, and the Wilcoxon signed-rank test was applied to compare outcomes.

Results: Twenty-five patients were included. The median Norfolk QOL-DN score changed from 54 [40.5; 77.5] to 48 [32.0; 83.0] after six months (p = 0.935). NIS scores decreased from 41 [14.0; 70] to 22 [6.0; 66.5] (p = 0.177). Serum TTR levels were maintained or reduced to undetectable levels in all patients. NT-proBNP decreased from 464.0 [102.0; 827.0] to 345.0 pg/mL [146.0; 995.0] (p = 0.518). No treatment-related adverse events were reported.

Conclusion: Although statistical significance was not reached, vutrisiran maintained or improved quality of life and clinical status in a real-world population that was more pretreated and had greater disease burden than the clinical trial. Combined with its favorable safety profile, subcutaneous administration, and extended dosing interval-preferred by patients-vutrisiran represents a promising therapeutic option for ATTRv amyloidosis. Long-term real-world studies are warranted to confirm these findings.

Objective: The effectiveness of alemtuzumab in patients with relapsing-remitting multiple sclerosis (RRMS) have been demonstrated in clinical trials. The primary endpoint was to describe the annual effectiveness of alemtuzumab over a 4-year period, according to the different parameters of the NEDA-3 concept (no evidence of disease activity) in clinical practice.

Methods: A retrospective, observational multicentric open study of patients with RRMS treated with alemtuzumab between 2015 and 2024. Effectiveness was assessed according to different parameters of the NEDA-3 concept (absence of relapses, stability in disability status according to the EDSS scale, and absence of new lesions and/or contrast enhancement in brain magnetic resonance imaging) with annual follow-up for up to four years.

Results: A cohort of 32 patients (71.9% women, mean age 40.3 ± 11 years) were included. The proportion of patients achieving NEDA-3 was 51.7% (15/29) in the first year, 56.2% (12/26) in the second year, 47.4% (9/19) in the third year, and 47.1% (8/17) in the fourth year. At the end of the study, 82% of patients remained relapse-free, 59% had stable disability according to the EDSS scale, and over 47% were free of radiological activity.

Conclusions: Alemtuzumab has proven to be effective, over 4 years, in clinical practice in patients with RRMS according to the different parameters of the NEDA-3 concept.

Introduction: The standard first-line treatment for metastatic small-cell lung cancer (SCLC) is platinum-based chemotherapy in combination with etoposide. The aim of this study was to evaluate the real-world effectiveness and safety of atezolizumab plus chemotherapy in SCLC and to explore factors associated with survival.

Methods: A retrospective, observational, multicenter study was conducted in patients diagnosed with SCLC who received first-line treatment with atezolizumab in combination with platinum-etoposide chemotherapy between November 2021 and March 2025. Treatment effectiveness was evaluated by assessing the objective response rate (ORR), treatment duration, progression-free survival (PFS) and overall survival (OS). Univariate and multivariate Cox models were used to explore associations between clinical variables and survival outcomes.

Results: A total of 76 patients with SCLC were included. Median age was 65 years, and 17.1% had an Eastern Cooperative Oncology Group performance status (ECOG PS) ≥2. The median duration of the treatment was 4.43 months (Interquartile range: 0.4-6.2). The ORR was 81.67% (95% CI: 70.08-89.44%). The median PFS was 7.2 months (95% CI: 6.5-8.6), and the median OS was 9.4 months (95% CI: 7.0-13.06). Patients with ECOG ≥2, hepatic metastases, and age ≥ 65 years were associated with a poor survival prognosis. Sex and the presence of brain metastases were not significantly associated with survival. Grade ≥ 3 treatment-related adverse events were observed in 44.7% patients, with no treatment-related deaths.

Conclusions: In this real-world cohort, atezolizumab plus platinum-etoposide achieved high response rates and acceptable survival with a manageable safety profile in extensive-stage SCLC. Baseline ECOG performance status, hepatic metastases, and older age were associated with worse survival, underscoring the prognostic value of clinical factors. These observational data complement, but do not validate, the results of IMpower133, as no randomized control group or formal trial emulation methods were used.

Background: The Pharmaceutical Care in Infectious Diseases Group (AFinf) is composed of a coordinating group of 11 pharmacists, 1 fellow, 1 resident pharmacist intern, 4 senior consultants and 221 associate members (July 2025). The aim is to understand and describe the profile of the members as a basis for defining future strategic lines to improve their participation.

Methods: An online survey was distributed to all AFinf group members, including questions about their professional activity, expectations and potential contributions to the group.

Results: The 41.7% of associated professionals participated in the survey. The median length of experience in the area of infectious diseases was 5 years (RIC 2-10). Antimicrobial stewardship programmes represented the main area of expertise (73.9%; 95% CI: 63.2-84.5), followed by pharmacokinetic monitoring (16.9%; 95% CI: 7.8-26.0). The priorities of the adherents were to receive training and education (52.3%; 95% CI: 40.2-64.5%) as well as the need to share doubts and experiences (27.7%; 95% CI: 16.8-38.6). Among the respondents, 50.0% (95% CI: 39.0-61.0) consider that they could contribute ideas and experiences, while 31.3% (95% CI: 21.1-41.4) research/collaborate on projects and 18.7% (95% CI: 10.2-27.3) participate in teaching and other group activities.

Conclusion: This study has allowed us to better understand the professional profile of pharmacists who are members of the AFinf group and to analyze their concerns and possible contributions.

Objective: The objective is to describe the real-world effectiveness and safety of atezolizumab in combination with carboplatin and etoposide as first-line treatment for patients with extensive-stage small-cell lung cancer.

Methods: We conducted a retrospective observational study of 48 patients with extensive-stage small-cell lung cancer treated with atezolizumab, carboplatin, and etoposide between March 2022 and May 2024 at a tertiary care center. The primary endpoints were progression-free survival and overall survival, analyzed using the Kaplan-Meier method. Safety outcomes and clinical predictors of survival were also assessed using software SPSS v26.

Results: After a median follow-up of 7.6 months (range: 1.5-28.4), median progression-free survival was 5.8 months and median overall survival was 7.1 months. Baseline ECOG performance status (ECOG 1 and 2) and the presence of brain metastases were associated with reduced overall survival. Patients who received ≤2 cycles of maintenance atezolizumab showed inferior survival outcomes. Grade 3-4 treatment-related adverse events occurred in 60.4% (n = 29) of patients, with hematologic toxicity being the most frequent.

Conclusions: In this real-world cohort, atezolizumab in combination with carboplatin and etoposide demonstrated feasibility and a manageable safety profile in first-line treatment of extensive-stage small-cell lung cancer. Longer follow-up and larger sample sizes are warranted to confirm these findings and further define prognostic markers in clinical practice.

The standardization of hospital pharmacy practice remains a challenge in Latin America due to the heterogeneity of healthcare systems. The Basel Statements, developed by the International Pharmaceutical Federation, provide guidelines aimed at improving patient safety and medication management; however, their implementation across the region is inconsistent.

Objective: To evaluate the application of the Basel Statements Self-Assessment Questionnaire in hospitals across Latin America and identify priority areas for improvement.

Method: A multicenter study was conducted in eight hospitals from eight countries. A 33-item questionnaire based on the Basel Statements was electronically distributed to clinical and hospital pharmacists. A descriptive analysis was performed.

Results: Only 25% of hospitals reported effective integration of pharmacists into clinical decision-making. Fewer than one-third had adequate medication traceability systems. Additionally, only 37.5% reported having regulations for the use of dietary supplements. Lack of standardization in medication storage and administration was also observed, increasing the risk of medication errors.

Conclusions: Implementation of the Basel Statements in Latin America is heterogeneous and requires targeted interventions. Strengthening pharmacists' involvement in multidisciplinary teams, improving traceability systems, and developing stronger regulations to support the safe use of medications are essential steps to enhance patient safety in the region.

Objective: To evaluate treatment persistence and dosing interval extension with faricimab in neovascular age-related macular degeneration (nAMD) in real-world practice.

Methods: Retrospective observational study conducted in a tertiary hospital (March 2024-March 2025). Patients receiving faricimab (treatment-naïve or pre-treated with anti-VEGF therapy), with ≥1 post-loading dose, were included. Dosing intervals were analyzed at baseline, 6 and 12 months, Adherence was assessed with the medication possession ratio (MPR), with >80% considered adherent. Persistence was defined as the time from treatment initiation to discontinuation or end of follow-up. Persistence was estimated using Kaplan-Meier survival analysis.

Results: We included 129 patients (148 eyes), mean age 74.5 ± 8.85 years; 55% were female. A total of 39 patients (30.2%) were treatment-naïve and 90 (69.8%) were pretreated. At 12 months, 48.8% of naïve and 55.5% of pretreated patients achieved 8-12 weeks intervals. Mean persistence was 12.2 months (SD 0.2; 95% CI: 11.8-12.6). The median was not reached by the end of the study. Persistence rate was 93% at 6 and 12 months. Only one patient discontinued due to inefficacy. No serious adverse events or endophthalmitis occurred.

Conclusions: Faricimab showed excellent persistence and extended dosing intervals in real-world practice. This is the first study specifically evaluating faricimab real-world persistence in nAMD.

Objective: To assess pharmacobezoar formation in a case of intoxication following the massive ingestion of extended-release divalproex sodium tablets, using an in vitro model conducted in the pharmacy laboratory.

Methods: An in vitro model was created to simulate gastric conditions and evaluate pharmacobezoar formation. Simulated gastric fluid was prepared according to the European Pharmacopeia procedure. The conglomerate's size and pH were measured at 30 min, 1 h, 2 h, 4 h, 8 h, 12 h, and 24 h intervals. Serum valproic acid concentrations were determined at all mentioned time points and then compared with the patient's serum concentrations.

Results: The in vitro model revealed the formation of a solid pharmacobezoar (5 x 6 cm) after 2 h (pH = 1.5), which remained stable for 24 h. The peak serum concentration of valproic acid occurred approximately 22 h post-ingestion, reaching 656 μg/mL. The in vitro assay indicated a similar peak concentration around 24 h after the tablets were immersed in the simulated gastric fluid.

Conclusions: The experiment conducted supports the hypothesis of pharmacobezoar formation following excessive ingestion of extended-release divalproex sodium tablets. Understanding which drug formulations can potentially cause pharmacobezoars formation is crucial for toxicological management.