Real-life Observational Study Research Articles

Isavuconazole as prophylaxis and therapy for invasive fungal diseases: a real-life observational study.

Isavuconazole is a relatively new antifungal agent indicated for the management of various invasive fungal diseases (IFDs), including invasive aspergillosis. Information on real-world experience with isavuconazole is scarce. This retrospective observational study aimed to describe the usage of isavuconazole in clinical practice with an in-depth evaluation of individual isavuconazole exposure. Patients treated with isavuconazole were evaluated based on retrospective data, including therapeutic drug monitoring (TDM) data and efficacy and safety data. Additionally, we calculated the individual isavuconazole exposure described by the average AUC24 over the first 7 days of treatment by means of non-linear mixed-effects modelling and compared this with the currently desired lower target AUC of 60 mg·h/L. Ninety-nine patients treated with isavuconazole were evaluated. In our real-life cohort, isavuconazole was often deployed off-label in patients with non-classical host factors and infections with non-Aspergillus and non-Mucorales species. Isavuconazole was most often chosen for its safety profile, even after prior triazole treatment with manifestations of toxicity. TDM and subsequent dosage adjustments were frequently performed. The individual average AUC24 over 7 days was above 60 mg·h/L in 29 out of 77 (37.7%) patients. This overview provides practical insights that can aid clinicians in the management of their patients with IFD. Our study shows that isavuconazole was used in a diverse patient population and was well tolerated overall. Individual isavuconazole exposure reflected by the average AUC24 over the first 7 days of treatment was generally low and variable. Dosage adjustments following TDM were frequently performed. Our experience shows that isavuconazole is a feasible alternative after prior azole treatment.

Characterization of omalizumab updosing patterns and predictive factors in chronic spontaneous urticaria: A prospective multicentric observational study.

Limited information is available on the use of omalizumab (OMA) updosing since its introduction as a second-line therapy in chronic spontaneous urticaria (CSU) in 2014. Practical guidelines from health authorities are lacking, and the specific characteristics of patients requiring higher doses remain unknown. Our objectives were to characterize the patterns of OMA updosing (defined as changes in dose and/or injection intervals), to identify the predictive factors associated with updosing, and to improve CSU management. We conducted a prospective, multicentric, real-life observational study, including patients diagnosed with CSU and starting OMA. The data were collected at 0, 3, 6, and 9 months. The primary endpoint was the frequency of OMA updosing at 3 months. The secondary endpoints included an analysis of updosed patients' profile, and an assessment of OMA efficacy and safety. We included 153 patients. Twenty percent of patients were updosed at 3 months, and 27% in total during the 9-month follow-up. Practitioners mainly chose to increase the frequency of injections (66%). At baseline, the updosed patients were more likely to have more severe CSU (UCT < 4, p < 0.030), a lower lymphocyte count (<2000/mm3, p = 0.037), and low IgE levels (<70 UI/mL, p = 0.024). The side effects of OMA were not more frequent after updosing. One in five patient underwent updosing within just 3 months. OMA updosing is frequent in particular in cases of severe disease and low IgE blood levels.

Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis with Mildly Decreased and Normal Lung Function: A Real-Life Observational Study.

Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis with Mildly Decreased and Normal Lung Function: A Real-Life Observational Study.

Single-centre real-life observational study on mortality and outcomes: decompressive craniectomy and brain death in traumatic brain injury, haemorrhage, and other cerebral diseases

BackgroundDecompressive hemicraniectomy (DHC) is used after severe brain damages with elevated, refractory intracranial pressure (ICP). In a non age-restricted population, mortality rates and long-term outcomes following DHC are still unclear. This study’s objectives were to examine both, as well as to identify predictors of unfavourable outcomes.MethodsWe undertook a retrospective observational analysis of patients aged 18 years and older who underwent DHC at the University Hospital of Bonn between 2018 and 2020, due to traumatic brain injury (TBI), haemorrhage, tumours or infections. Patient outcomes were assessed by conducting telephone interviews, utilising questionnaires for modified Rankin Scale (mRS) and extended Glasgow Outcome scale (GOSE). We evaluated the health-related quality of life using the EuroQol (EQ-5D-5L) scale.ResultsA total of 144 patients with a median age of 58.5 years (range: 18 to 85 years) were evaluated. The mortality rate was 67%, with patients passing away at a median of 6.0 days (IQR [1.9–37.6]) after DHC. Favourable outcomes, as assessed by the mRS and GOSE were observed in 10.4% and 6.3% of patients, respectively. Cox regression analysis revealed a 2.0% increase in the mortality risk for every year of age (HR = 1.017; 95% CI [1.01–1.03]; p = 0.004). Uni- and bilateral fixed pupils were associated with a 1.72 (95% CI [1.03–2.87]; p = 0.037) and 3.97 (95% CI [2.44–6.46]; p < 0.001) times higher mortality risk, respectively. ROC-analysis demonstrated that age and pupillary reactivity predicted 6-month mortality with an AUC of 0.77 (95% CI [0.69–0.84]). The only parameter significantly associated with a better quality of life was younger age.ConclusionsFollowing DHC, mortality remains substantial, and favourable outcomes occur rarely. Particularly in elderly patients and in the presence of clinical signs of herniation, mortality rates are notably elevated. Hence, the indication for DHC should be set critically.

Outpatient parenteral antibiotic therapy (OPAT) through elastomeric continuous infusion pumps in a real-life observational study: Characteristics, safety, and efficacy analysis

IntroductionThis study aimed to present real-life data on the use, efficacy, and safety of administering antibiotic therapy through portable elastomeric pumps (pEP) in the outpatient setting. MethodsThis retrospective observational cohort study was conducted from January 2020 to May 2023 in a large academic hospital in Rome, Italy. All patients receiving antibiotic therapy via pEP were included up to a follow-up period of 90 days after the end of antibiotic therapy.The primary outcome was the treatment response. Secondary endpoints were adverse events attributable to the drug administered, the vascular catheter, or the infection itself. ResultsOf the 490 patients referred to our outpatient parenteral antibiotic therapy (OPAT) unit, 94 (19.2%) received antibiotic therapy via pEP and were included in the final analysis. The most frequently treated infections were those involving bone and prosthetics, including spondylodiscitis (n=27; 28.8%). Most infections were due to Pseudomonas aeruginosa (n=55; 48.3%). Cefepime (n=32; 34.0%), piperacillin/tazobactam (n=29; 30.9%), ceftolozane/tazobactam (n=7; 7.5%), and oxacillin (n=7; 7.5%) were the most frequently administered antibiotics. The infection cure rate reached 88.3% (n=83). 12 patients (12.8%) reported adverse events, of which half (6.4%) were drug-related and half (6.4%) were line-related. ConclusionsOPAT through portable elastomeric infusion pumps proved to be safe and effective. It also contributed to the reduction of healthcare costs, fully respecting the principles of personalized medicine. This strategy has emerged as a promising tool for antibiotic stewardship and infection control.

From Emergency Department to clinic center: management of patients with Heart Failure with Reduced Ejection Fraction

Aims: primary outcomes - cardiovascular and all-cause mortality evaluation within 1 year in elderly patients enrolled with Angiotensin Receptor Inhibitor (ARNI) and/or Sodium Glucose Co-Transporter Type 2 Inhibitors (SGLT2-i) in an Internal Medicine Department; recurrent hospitalization assessment for acute Heart Failure with Reduced Ejection Fraction (HFrEF); length of hospitalization analysis compared to not-enrolled patients. Secondary endpoints - to identify any parameters predicting the length of hospitalization; safety assessment through the incidence of complications and treatment interruption. Materials and Methods: prospective observational real-life cohort study that analyzes the recruitment during hospitalization and follow-up 3-6-12 months after discharge. Several clinical parameters were recorded for each patient of any ethnicity, considering a diagnosis of acute HFrEF with NT-proBNP and chest x-ray. Results: the enrolled group (34 patients) showed a hospitalization period lower than the not-enrolled one. The rehospitalizations percentage was inferior to Randomized Clinical Trials (RCTs) for ARNI. Cardiovascular-cause mortality, symptomatic hypotension, and Urinary Tract Infections (UTI) were lower than RCTs data. Age, eGFR, NT-proBNP, Cumulative Illness Rating Scale (CIRS), and severe comorbidities, except for EF, predicted the lengthening of hospitalization. Hypoglycemia wasn’t recorded. Conclusions: ARNI/SGLT2-i are effective and safe in elderly patients. Comorbidities and bio-humoral features influence HFrEF and quality of life. The future aim is to confirm the results obtained so far.

TRUE REFRACTORY HYPERTENSION IN 2023- LESSONS LEARNED FROM A NATIONAL REGISTRY OF 1.82 MILLION ADULTS

Objective: Refractory hypertension (refHTN) is defined as blood pressure that remains uncontrolled on maximal therapy, which is defined as the use of 5 or more antihypertensive agents of different drug classes. We aimed to define the prevalence of apparent and true refHTN in a real life national registry of an Health Maintenance Organization (HMO). Design and method: In this real-life observational study we utilized the Maccabi Healthcare Services (MHS) database during 2023. Data includes pharmacy prescriptions and purchases. Apparent refHTN was defined as patients included in the Maccabi “Hypertension Registry” during 2023 and taking 5 different antihypertensive medications of different classes, of which four were from pre-specified groups (diuretics, CCB, RAAS inhibitors and MRAs). True refHTN (unlike apparent refHTN) was defined as being fully adherent to the antihypertensive medication regimen with at least 6 prescriptions and purchases (refills) during 2023 as well as blood pressure which was not optimally controlled during the year (less than 80% of blood pressure measurements equal or below the well-defined target of 130/80mmHg). Results: Out of 1,821,685 adult members of Maccabi HMO during 2023, 361,721 patients were defined as hypertensive patients (20%) and included in the registry. 0.5% (1750) of the hypertensive population were at some stage during 2023 on at least 5 anti-hypertensive medications (i.e., apparent refHTN). Of those only 264 patients (15%) were adherent to their medications during the whole year. Mean age was 71.3 years. Of the well adherent group, 205 patients (77%) were not optimally controlled and thus regarded as true refHTN. Therefore in total, true refHTN was evident in 0.056% of hypertensive population in Maccabi HMO. Conclusions: In an “optimal world” which lacks physician inertia, four medication regimen is prescribed (Diuretics, CCB, RAAS inhibitors and MRAs) and there is willingness to prescribe a fifth and a sixth medication, if needed (alpha blockers, beta blockers, central alpha-2 agonists or direct vasodilators). In the unlikely case of full adherence to all medications, true refHTN is extremely rare (0.056% of hypertensive patients). This suggests that most hypertensive patients can be well controlled using the current anti hypertensive armamentarium.

Belimumab corticosteroid-sparing treatment in systemic lupus erythematosus: a real-life observational study (BESST study).

Data about the impact of Belimumab on corticosteroid sparing in real life are scarce. To assess the corticosteroid-sparing effect among patients with systemic lupus erythematosus (SLE) treated with Belimumab in real-life settings. Multicentric observational retrospective study including patients with SLE and having received Belimumab for at least 6months between 2011 and 2020, in eight French hospitals. "Low dose" referred to patients receiving up to 7.5mg of prednisone a day and "Very low dose" to those receiving strictly ≤ 5mg prednisone a day The primary endpoint was the reduction of daily prednisone dose after six months of Belimumab. The secondary endpoint was a change in the proportion of patients with low or very low dose of prednisone as well as those without prednisone during the Belimumab course. Censoring occurred for patients who stopped Belimumab. Bivariate analyses were performed using the Wilcoxon signed-rank test. The threshold for statistical significance was set at p < 0.05. Thirty patients were included. All were female with a median age of 38years. A significant reduction in prednisone dose was observed at month 6 (10 [7-20] vs 6.75 [2-9] mg, p < 0.0001), continued until month 12 (10 [7-20] mg vs 5 [0-7.12] mg, p < 0.001) and was sustained until month 24. The proportion of patients with very low dose of prednisone and those without prednisone progressively increased during the Belimumab course. Introducing Belimumab in patients with SLE, in real-life conditions, is associated with early and sustained corticosteroid-sparing effect.

Post-Transplant Cyclophosphamide versus Anti-Thymocyte Globulin in Patients with Hematological Malignancies Treated with Allogeneic Hematopoietic Stem Cell Transplantation from Haploidentical and Matched Unrelated Donors: A Real-Life Experience.

Background: Post-transplant cyclophosphamide (PTCY) is widely used as graft versus host disease (GvHD) prophylaxis in allogeneic hematopoietic stem cell transplant (HSCT) recipients, with reported clinical benefits in patients who underwent transplant from a matched unrelated donor (MUD). However, real-life data on clinical efficacy and safety of PTCY in haploidentical and MUD transplantations are still poor. Methods: In our real-life retrospective observational study, we included a total of 40 consecutive adult patients who underwent haploidentical or MUD HSCT for various hematological malignancies and who received PTCY (n = 24) or ATG (n = 16) as GvHD prophylaxis at Hematology Units from hospitals of Salerno and Avellino, Italy, and clinical outcomes were compared. Results: We showed protective effects of PTCY against disease relapse with the relapse rate after transplantation of 16% versus 50% in the ATG arm (p = 0.02). All-cause mortality was lower (36% vs. 75%; p = 0.02) and the 2-year overall survival was slightly superior in patients administered PTCY (61% vs. 42%; p = 0.26). Conclusions: We support the use of PTCY, even in a real-life setting; however, the optimization of this protocol should be further investigated to better balance relapse prevention and GvHD prophylaxis.

Efficacy and tolerability of tirbanibulin 1% ointment in the treatment of cancerization field: a real-life Italian multicenter observational study of 250 patients.

Tirbanibulin 1% ointment is approved for the field treatment of Olsen grade I actinic keratoses (AKs) of the face and scalp. We performed a multicenter retrospective study involving 15 dermatologic units in Italy to investigate the efficacy and tolerability of tirbanibulin in a real-life setting. 250 patients were enrolled. Tirbanibulin, 1% ointment, was applied daily for five consecutive days. The efficacy of treatment was measured with modifications of the Actinic Keratosis Area and Severity Index (AKASI). A satisfactory response was defined by complete (100% reduction in the number of lesions) or partial clearance (75-99%) of treated AKs. Overall, the AKASI score was significantly reduced in the studied population (mean, from 4.1 ± 2.7 to 1.4 ± 1.5; P < 0.001). A satisfactory response was observed in 222 (88.8%) cases. The proportion of satisfactory responses was higher when follow-up was performed after 8 weeks (34/35, 97.1%). The reduction in AKASI was significant in patients with Olsen grade II or III lesions (from 5.3 ± 2.8 to 1.6 ± 1.6; P < 0.001). A satisfactory response was observed in 91/104 (87.5%) cases. AKASI reduction was also significant in patients with trunk or limb AKs (from 7.0 ± 1.3 to 2.0 ± 1.6; P = 0.018) since a satisfactory response was observed in 7/8 (87.5%) cases. Tirbanibulin was well tolerated; all adverse events (AEs) included transient local reactions at the site of treatment. Overall, 231 patients had at least one AE. Only 7 (2.8%) grade 4 AEs were recorded. Our retrospective study confirmed that tirbanibulin 1% ointment is effective and well tolerated in a real-life setting and is also promising for Olsen grade II and grade III AKs and AKs localized on difficult-to-treat areas.

Iron Supplementation May Improve the Patient's Level of Satisfaction in Not-Low-Ferritin Telogen Effluvium: A Real-Life Observational Study.

Telogen effluvium (TE) is the most common cause of alopecia in women. Treatment should address the etiological factors and may include adjuvant therapies. In practice, physicians may employ modified approaches and utilize various combinations of topical and oral molecules. In this real-life observational study, the aim was to evaluate the response of TE to iron supplementation. The population consisted of all patients who sought consultation for TE at our dermatology department between March 2021 and February 2022. Eligible participants were women, aged between 18 and 65, having a clinical diagnosis of TE, and intended for treatment with iron supplementation. Exclusion criteria comprised current pregnancy, chronic or active inflammatory disease, newly discovered dysthyroidism, concurrent use of hair supplements, topical minoxidil, or any other medications. The response was assessed based on the patient's level of satisfaction, a significant indicator, given the substantial psychological impact of TE on women's daily lives. The analysis included 200 women. The average age was 32.9 ± 11.4 years. A recent history of COVID-19 or treated dysthyroidism was present in 18.5% and 8% of patients, respectively, but did not impact their response. Significantly, patients with baseline ferritin ≥50 ng/ml were mostly "very satisfied", those with baseline ferritin <50 ng/ml were mostly "not satisfied", and those with unknown levels were mostly "partially satisfied" with iron supplementation. A high dose of elemental iron and a prolonged duration of treatment significantly improved the patients' level of satisfaction. Iron supplementation can improve the patient's level of satisfaction in TE even if serum ferritin is not low.

Efficacy and safety of tildrakizumab in elderly patients: real-world multicenter study (ESTER – study)

Purpose of the article: Interleukin-23 inhibitors, such as tildrakizumab, have emerged as safe and effective options for the management of psoriasis. Yet their efficacy in elderly patients (aged 65 years or more), particularly in those with difficult-to-treat areas involvement, remains insufficiently explored. We conducted this real-life retrospective multicentric observational study to assess the effectiveness of tildrakizumab in elderly patients with moderate-to-severe psoriasis, with involvement of difficult-to-treat areas.Materials and methods: We enrolled forty-nine patients aged 65 years old or more (mean age 73.1 ± 6.0), all treated with tildrakizumab for at least 28 weeks. The effectiveness of tildrakizumab was assessed by Static Physician’s Global Assessment of Genitalia (sPGA-G), fingernail-PGA (f-PGA), palmoplantar PGA (pp-PGA), scalp-specific PGA (sc-PGA), and Psoriasis Area and Severity Index (PASI) scores.Results: Significant improvements in PASI scores were observed within 28 weeks of treatment, with 77.5%, 60%, and 45.2% of patients achieving PASI75, PASI90, and PASI100, respectively. The mean PASI decreased significantly from baseline (13.6 ± 9.9) to 1.3 ± 1.7 at week 28. More than 90% of patients had clear sPGA-G and pp-PGA scores and over 70% had clear f-PGA and sc-PGA scores after 28 weeks.Conclusions: Our findings suggest that tildrakizumab could be a valuable option for the treatment of elderly patients, including those with difficult-to-treat areas involvement.

Effectiveness of ixekizumab over 24 months in different clinical scenarios in psoriatic arthritis: results from the Gruppo Italiano Studio Early Arthritis multicentric prospective registry.

We aimed to evaluate ixekizumab (IXE) effectiveness, drug survival and clinical response predictors in moderate-severe psoriatic arthritis (PsA) patients in different clinical scenarios. This was a multicentre real-life observational study based on Gruppo Italiano Studio Early Arthritis (GISEA) registry of IXE treatment in PsA patients (January 2019-June 2023). Data were collected at baseline and every six months. 223 PsA outpatients were included. Statistically significant improvement was observed after 6 (T6), 12 (T12) and 24 (T24) months of therapy for tender and swollen joint count (TJC and SJC), Visual Analogue Scale (VAS)-pain and Disease Activity in PSoriatic Arthritis (DAPSA) score. DAPSA remission was reached at T12 in 22% and at T24 in 18.5% of patients. At baseline, higher fibromyalgia and combination therapy with conventional synthetic disease-modifying anti-rheumatic drugs (csDMARDs) in females with respect to males and higher Psoriasis Area Severity Index (PASI) in males than in females were observed. Therapeutic effectiveness showed in males higher DAPSA and VAS-pain reduction, higher percentage of males in DAPSA remission/low disease activity (LDA) at T6, and higher ∆PASI at T6 and T12 than in female patients. At multivariate analysis, male sex was predictive for treatment response at T6 [p=0.02, odds ratio (OR) 2.49 (95% confidence interval 1.11-5.54)], while it lost significance at T12. IXE effectiveness was highlighted after 6 months at both joint and skin levels and lasted up to 24 months in different clinical scenarios, making IXE effective in the complexity of managing PsA in a real-life setting.

Injection site reactions after dupilumab or tralokinumab for atopic dermatitis

Background: Injection site reaction (ISR) is a local phenomenon defined as a constellation of symptoms, including swelling, erythema, pruritus, and pain around the site of injection. Objective: ISR is reported as a frequent adverse event after subcutaneous injection (SCI) of several biologics. Methods: We performed an observational real-life study to compare dupilumab and tralokinumab as regards ISR, analysing frequency, duration and intensity of symptoms related to SCI. From January 2023 to June 2023, we enrolled adult patients affected by moderate to severe AD and being on dupilumab or tralokinumab treatment. A 12 items questionnaire was administered to all enrolled patients. Results and conclusions: Three hundred and ninety-two patients were included. ISR was a frequent occurrence in both the treatment groups, with tralokinumab causing ISR more frequently than dupilumab. However, the reactions were generally mild and no patient stopped therapy.

The Risk of Drug Interactions in Older Primary Care Patients after Hospital Discharge: The Role of Drug Reconciliation.

Drug-drug interactions (DDIs) represent an important clinical problem, particularly in older patients, due to polytherapy, comorbidity, and physiological changes in pharmacodynamic and pharmacokinetic pathways. In this study, we investigated the association between drugs prescribed after discharge from the hospital or clinic and the risk of DDIs with drugs used daily by each patient. We performed an observational, retrospective, multicenter study on the medical records of outpatients referred to general practitioners. DDIs were measured using the drug interaction probability scale. Potential drug interactions were evaluated by clinical pharmacologists (physicians) and neurologists. Collected data were analyzed using the Statistical Package for the Social Sciences. During the study, we evaluated 1772 medical records. We recorded the development of DDIs in 10.3% of patients; 11.6% of these patients required hospitalization. Logistic regression showed an association among DDIs, sex, and the number of drugs used (p = 0.023). This observational real-life study shows that the risk of DDIs is common in older patients. Physicians must pay more attention after hospital discharge, evaluating the treatment to reduce the risk of DDIs.

Sofosbuvir/Velpatasvir/Voxilaprevir for Hepatitis C Virus Retreatment in Difficult-to-treat Patients: A Real-life Observational Study from India

Background and AimHepatitis C (HCV) treatment fail to achieve sustained virological response at 12 weeks (SVR12) in 5-10% and require retreatment with second-line drugs. We report our experience of sofosbuvir/velpatasvir/voxilaprevir use for HCV retreatment in a small cohort of difficult-to-treat Indian patients. MethodsWe reviewed our HCV databases to identify the patients who had failed to achieve SVR12 after treatment with sofosbuvir in combination with either daclatasvir or ledipasvir or velpatasvir with/without ribavirin on one or more occasions. Participants were excluded if they had (i) decompensated cirrhosis or (ii) HIV co-infection or (iii) chronic kidney disease or (iv) prior organ transplantation. All the participants were treated with sofosbuvir/velpatasvir/voxilaprevir plus ribavirin for 12 weeks. Treatment outcome was categorized as successful or failure if HCV RNA was undetectable or detectable at SVR12, respectively. ResultsFifteen patients (male 67%; genotype-3 80%) were included in analysis. Ten (67%) had cirrhosis. Five, eight, and two participants had previously failed to one, two, and three courses of pegylated-interferon free, sofosbuvir containing DAA regimens respectively. Fourteen participants had failed to at least one course of sofosbuvir/velpatasvir combination. Fourteen patients achieved SVR12 and one patient was lost to follow-up. Treatment was successful in 100% and 93.3% on per-protocol (PP) and intention to treat (ITT) analysis, respectively. ConclusionSofosbuvir/velpatasvir/voxilaprevir combination is an effective second line therapy in India for difficult to treat HCV patients.

Living on Oral Anticoagulants: Duke Anticoagulation Satisfaction Scale Results.

Direct oral anticoagulants (DOACs) are widely used in patients with atrial fibrillation and venous thromboembolism. The lack of the need for laboratory monitoring and a better safety than vitamin K antagonists (VKAs) has probably changed the quality of life of patients on these oral anticoagulants. This was a real-life prospective observational cohort study. The aim was to evaluate if a long-term treatment with DOACs could offer a better quality of life than VKAs. Moreover, age, gender, education level, time in therapeutic range for VKAs, taking medication once or twice a day for DOACs, the total daily number of medications and thrombotic and bleeding complications were considered as variables probably associated with the quality of life of these patients. Between January and December 2021, the Duke Anticoagulation Satisfaction Scale (DASS) 25-items was administered as an interview to patients on either VKAs or DOACs therapy. During the follow-up period, all of the patients were closely monitored to evaluate possible bleeding and thrombotic events. The analysis included 300 outpatients treated with VKAs and 254 treated with DOACs. In general, the quality of life was better in patients taking DOACs (DASS total score: DOACs = 44.7, 42.9-46.5 vs. VKAs = 51, 49.2-52.8, p < 0.0001) as the daily-life limitations, hassles and burdens and the psychological impact were less important than in patients on VKAs therapy. VKAs negatively influence the daily-life of the patients in terms of both less satisfaction and time-consuming tasks. DOACs confer a better quality of life even if some concerns emerge from not knowing how their therapy is working.

Use of a first plant-based complete oral nutritional supplement for the management of disease related malnutrition: A real-life observational study in Switzerland

Use of a first plant-based complete oral nutritional supplement for the management of disease related malnutrition: A real-life observational study in Switzerland

Long-Term Eltrombopag in Children with Chronic Immune Thrombocytopenia: A Single-Center Extended Real-Life Observational Study in China

Background: We have previously confirmed the efficacy and safety of eltrombopag (ELT) in children with chronic immune thrombocytopenia (cITP). However, there is still a lack of results on long-term ELT therapy. Here, we report the long-term follow-up results ( between September 2018 and June 2023 ) on our previous study. Methods: This is a single-center real-life observational study that collected data from patients who previously enrolled in our children cITP study and had a follow-up period of more than 3 years by June 2023. Results: A total of 65 patients (28 males) were enrolled with a median age at ELT initiation of 6.34 (range 1.65, 14.13) years and a 47.07 (36.00, 57.00) months follow-up time with a 40.36 (10.53, 56.83) months of ELT therapy at the analysis time point. In total, 29.23% (19/65) (of) patients discontinued ELT because of a stable response, and 18.46% (12/65) patients switched to another platelet-enhancing therapy due to loss of response to therapy after 19.13 (14.53, 26.37) months. Among the 19 patients who discontinued ELT because of a stable response, 24.62% (16/65) children achieved 12 m of off ELT treatment(SRoT), the last recorded platelet count ranged from 56-166×10 9/L (median value 107×10 9/L), and 4.62% (3/65) patients relapsed at the 5th, 6th, and 9th months after discontinuation, respectively. Of the 12 patients who lost response to ELT after 19.13 (14.53, 26.37) months of therapy, four switched to avatrombopag, three to hetrombopag, two to Chinse traditional medicine, one had splenectomy and two received additional prednisolone acetate. Thirty-four patients maintained a sustained response while continuing on ELT treatment but with tapering doses. No significant adverse events were reported. In the first 12 months of ELT, there was a higher rate of PLT ≥ 100×10 9/L in SRoT patients than in tapering patients (62.50% [10/16] vs. 5.88% [2/34]) and a higher rate of PLT 70-100×10 9/L (31.25% [5/16] vs. 27.78% [10/36]) ( P=0.01). Conclusion: We report more than 3 years long-term clinical data on cITP children taking ELT. We show that 24.62% of the children who had a stable response to ELT achieved 12 m of SRoT. The treatment response of children in the first 12 months after the initiation of treatment may be a predictive factor for successful discontinuation. These data highlight no new safety concerns regarding the long-term use of ELT in children with cITP.

Aging Impact in Response to Different Classes of Biological Treatment in Psoriatic Patients: A Real-Life Observational Study

Over the last decade, the treatment landscape for moderate to severe psoriasis has undergone transformative changes with the advent of biotechnological drugs. Monoclonal antibodies targeting the IL-17 and IL-23 pathways have displayed remarkable clinical efficacy and safety, even among patients with complex comorbidities. These innovations have extended across various age groups within the psoriatic population. However, a scarcity of age-specific data remains regarding the efficacy and safety of these medications. Our study tries to bridge this gap by systematically presenting data obtained from the analysis of 1055 patients treated for psoriasis with anti-IL17 and anti-IL23 drugs during a 1-year period. The effectiveness and safety of anti-IL-17 and anti-IL23 drugs for moderate to severe psoriasis were assessed across four different age groups ranging from patients less than 26 years old to patients older than 65 years, divided in four year ranges. In the studied population, baseline PASI score was significantly higher in the age group of individuals over 65 years compared to those under 26 years old. Patients over 65 years also exhibited a slower rate of improvement in PASI-90 and PASI &lt; 3 at the 16-week mark compared to other age groups. However, no clinically significant differences in treatment response were found when comparing overall responses among different age groups. In age groups older than 26 years, anti-IL17 drugs seems faster in the achievement of PASI-100 when compared to anti-IL23 drugs. This trend became more pronounced with increasing age. The investigation provides insights into treatment responses and patient characteristics, highlighting the influence of age as a significant variable in patient management.