International Registry Of Patients Research Articles

Impact of Hemoglobin Levels on Composite Cardiac Arrest or Stroke Outcome in Patients With Respiratory Failure Due to COVID-19.

Anemia has been associated with an increased risk of both cardiac arrest and stroke, frequent complications of COVID-19. The effect of hemoglobin level at ICU admission on a composite outcome of cardiac arrest or stroke in an international cohort of COVID-19 patients was investigated. Retrospective analysis of prospectively collected database. A registry of COVID-19 patients admitted to ICUs at over 370 international sites was reviewed for patients diagnosed with cardiac arrest or stroke up to 30 days after ICU admission. Anemia was defined as: normal (hemoglobin ≥ 12.0 g/dL for women, ≥ 13.5 g/dL for men), mild (hemoglobin 10.0-11.9 g/dL for women, 10.0-13.4 g/dL for men), moderate (hemoglobin ≥ 8.0 and < 10.0 g/dL for women and men), and severe (hemoglobin < 8.0 g/dL for women and men). Patients older than 18 years with acute COVID-19 infection in the ICU. None. Of 6926 patients (median age = 59 yr, male = 65%), 760 patients (11.0%) experienced stroke (2.0%) and/or cardiac arrest (9.4%). Cardiac arrest or stroke was more common in patients with low hemoglobin, occurring in 12.8% of patients with normal hemoglobin, 13.3% of patients with mild anemia, and 16.7% of patients with moderate/severe anemia. Time to stroke or cardiac arrest by anemia status was analyzed using Cox proportional hazards regression with death as a competing risk. Covariates selected through clinical knowledge were age, sex, comorbidities (diabetes, hypertension, obesity, and cardiac or neurologic conditions), pandemic era, country income, mechanical ventilation, and extracorporeal membrane oxygenation. Moderate/severe anemia was associated with a higher risk of cardiac arrest or stroke (hazard ratio, 1.32; 95% CI, 1.05-1.67). In an international registry of ICU patients with COVID-19, moderate/severe anemia was associated with increased hazard of cardiac arrest or stroke.

500. PRIMARY ESOPHAGEAL LYMPHOMA: UNDERSTANDING PROGNOSTIC FACTORS AND OUTCOMES

Abstract Background Primary Esophageal Lymphoma (PEL) represents a rare subtype of gastrointestinal lymphomas, posing unique diagnostic and therapeutic challenges. Given its rarity, comprehensive insights into its prognostic factors and outcomes remain limited. This study examines the epidemiology, treatment modalities, and outcomes of PEL, aiming to identify significant prognostic factors that influence patient survival. Methods A systematic review of the literature was conducted across PubMed/Medline and Scopus databases, focusing on cases of adult PEL. Data extraction included demographic information, treatment modality, and patient outcomes. Survival rates were calculated, and prognostic factors were analyzed through unifactorial analysis. Results Out of 56 studies, 69 cases of PEL were analyzed, with a male-to-female ratio of 2.8:1 and mean age of 55.5 years. Patients were predominantly treated with chemotherapy (52.1%) while 20% underwent surgical treatment. The mean survival reported was 49.3 months with one-, three-, and five-year survival rates of 84.5%, 72.3%, and 28.9%, respectively, exhibiting a less favorable prognosis compared to common lymphoma types. Survival analysis revealed that local lymphadenopathy had a significant impact on prognosis (p=0.008), while gender, age, treatment approach, and type of lymphoma were not significant determinants of survival. Immunocompromised status showed a marginally significant effect on survival (p= 0.053). Discussion The study highlights the less favorable prognosis of PEL compared to more common lymphomas, emphasizing the importance of recognizing patients with unfavorable prognostic factors such as local lymphadenopathy and immunocompromised status. The findings advocate for the establishment of an international patient registry to better understand and manage PEL, aiming to standardize diagnostic criteria and treatment strategies.

Emerging Indications for Hyperbaric Oxygen Treatment: Registry Cohort Study.

Hyperbaric oxygen (HBO2) treatment is used across a range of medical specialties for a variety of applications, particularly where hypoxia and inflammation are important contributors. Because of its hypoxia-relieving and anti-inflammatory effects HBO2 may be useful for new indications not currently approved by the Undersea and Hyperbaric Medical Society. Identifying these new applications for HBO2 is difficult because individual centers may only treat a few cases and not track the outcomes consistently. The web-based International Multicenter Registry for Hyperbaric Oxygen Therapy captures prospective outcome data for patients treated with HBO2 therapy. These data can then be used to identify new potential applications for HBO2, which has relevance for a range of medical specialties. Although hyperbaric medicine has established indications, new ones continue to emerge. One objective of this registry study was to identify cases where HBO2 has been used for conditions falling outside of current Undersea and Hyperbaric Medical Society-approved indications and present outcome data for them. This descriptive study used data from a web-based, multicenter, international registry of patients treated with HBO2. Participating centers agree to collect data on all patients treated using standard outcome measures, and individual centers send deidentified data to the central registry. HBO2 treatment programs in the United States, the United Kingdom, and Australia participate. Demographic, outcome, complication, and treatment data, including pre- and posttreatment quality of life questionnaires (EQ-5D-5L) were collected for individuals referred for HBO2 treatment. Out of 9726 patient entries, 378 (3.89%) individuals were treated for 45 emerging indications. Post-COVID-19 condition (PCC; also known as postacute sequelae of COVID-19; 149/378, 39.4%), ulcerative colitis (47/378, 12.4%), and Crohn disease (40/378, 10.6%) accounted for 62.4% (n=236) of the total cases. Calciphylaxis (20/378, 5.3%), frostbite (18/378, 4.8%), and peripheral vascular disease-related wounds (12/378, 3.2%) accounted for a further 13.2% (n=50). Patients with PCC reported significant improvement on the Neurobehavioral Symptom Inventory (NSI score: pretreatment=30.6; posttreatment=14.4; P<.001). Patients with Crohn disease reported significantly improved quality of life (EQ-5D score: pretreatment=53.8; posttreatment=68.8), and 5 (13%) reported closing a fistula. Patients with ulcerative colitis and complete pre- and post-HBO2 data reported improved quality of life and lower scores on a bowel questionnaire examining frequency, blood, pain, and urgency. A subset of patients with calciphylaxis and arterial ulcers also reported improvement. HBO2 is being used for a wide range of possible applications across various medical specialties for its hypoxia-relieving and anti-inflammatory effects. Results show statistically significant improvements in patient-reported outcomes for inflammatory bowel disease and PCC. HBO2 is also being used for frostbite, pyoderma gangrenosum, pterygium, hypospadias repair, and facial filler procedures. Other indications show evidence for improvement, and the case series for all indications is growing in the registry. RR2-10.2196/18857.

Overlapping hearing and communication profiles for the deletion and the RAI1 variant form of Smith-Magenis Syndrome (SMS)

Smith-Magenis syndrome (SMS), a rare, genetically linked complex developmental disorder is associated with hearing loss and delays in speech-language development. Approximately 70 % of those with SMS have a genetic deletion within chromosome 17p11.2 and 10 % have a single gene mutation in the same area, known as the RAI1 variant form of SMS. Previous studies presented preliminary comparisons of the phenotype of those with deletion versus the mutation form of SMS, but none focused on hearing, speech-language, and communication profiles. The current study examined patient registry data and compared two groups of individuals with SMS to determine if genetic differences influence the communication phenotype and to determine the importance of the RAI1 gene in hearing, speech, and language abilities in children. Speech-language and hearing data from the international SMS Patient Registry for 33 subjects with SMS were analyzed: 23 with a genetic deletion (SMS (del)) and 10 with an RAI1 mutation (SMS (RAI1)). Hearing status, otopathology findings, early speech-language milestones, mode of communication, intelligibility, vocal quality, language abilities, and literacy skills were analyzed. There were small differences between the groups for hearing status, otopathological findings, mode of communication, voice quality, intelligibility, speech-language abilities, and literacy. Overlap in the speech-language phenotype between groups confirms previous hypotheses that suggest haploinsufficiency of the RAI1 gene is responsible for the SMS phenotype and that the RAI1 gene is critical for speech-language development. Future studies should include direct testing of receptive and expressive language abilities, including analyses of language samples, with larger groups of individuals to replicate and extend the current findings.

Impact of postoperative necrotizing enterocolitis after neonatal cardiac surgery on neurodevelopmental outcome at 1 year of age.

We analyzed the impact of postoperative necrotizing enterocolitis (NEC) after cardiac surgery in neonatal age on neurodevelopmental (ND) outcome at 1 year of age. Using data from the Swiss Neurodevelopmental Outcome Registry for Children with Congenital Heart Disease (ORCHID), we analyzed perioperative variables including postoperative NEC (Bell's stage ≥2) and 1-year ND outcome (Bayley III). The included patients (n = 101) had congenital heart disease (CHD), categorized as follows: 77 underwent biventricular repair for CHD with two functional chambers, 22 underwent staged palliation until the Fontan procedure for CHD with single ventricle physiology (n = 22), or 4 underwent single ventricle palliation or biventricular repair for borderline CHD (n = 4). Neonatal cardiopulmonary bypass (CBP) surgery was performed at a median age (IQR) of 8 (6) days. NEC occurred in 16 patients. Intensive care unit (ICU) length of stay (LOS) and the total duration of the hospitalization were longer in children with NEC than those in others (14 with vs. 8 days without NEC, p < 0.05; 49 with vs. 32 days without NEC, p < 0.05). The Bayley III scores of the analyzed patients determined at an age of 11.5 ± 1.5 months showed cognitive (CCS) (102.2 ± 15.0) and language scores (LCS) (93.8 ± 13.1) in the normal range and motor composite scores (MCS) (88.7 ± 15.9) in the low-normal range. After adjusting for socioeconomic status and CHD type, patients with NEC had lower CCS scores [β = -11.2 (SE 5.6), p = 0.049]. Using a cumulative risk score including NEC, we found a higher risk score to be associated with both lower CCS [β = -2.8 (SE 1.3), p = 0.030] and lower MCS [β = -3.20 (SE 1.3), p = 0.016]. Postoperative NEC is associated with longer ICU and hospital LOS and contributes together with other complications to impaired ND outcome at 1 year of age. In the future, national and international patient registries may provide the opportunity to analyze large cohorts and better identify the impact of modifiable perioperative risk factors on ND outcome. ClinicalTrials.gov identifier: NCT05996211.

An international multicentre experience with the third-generation laser balloon system: safety, efficiency, and efficacy

Abstract Background Pulmonary vein isolation (PVI) with the use of an endoscopic laser balloon (ELB) is an alternative option for catheter ablation of atrial fibrillation (AF). The third-generation system has several improvements, including the novel RAPID mode (RM), potentially allowing a one-shot, continuous, circumferential laser lesion for PVI. Objective In an international, multicentric cohort, we assessed the third-generation ELB’s safety, efficacy, and long-term effectiveness. We also focused on the learning curve (LC) and the use of RM. Methods Between June 2019 and December 2022, we prospectively obtained data on all the patients who consecutively underwent ablation of AF using the third-generation ELB in eight Italian and one German hospital. Each operator attempted to use the RM to the maximum extent in every procedure. Follow-up (FU) visits were scheduled at 3, 6, 12 and 24 months and included a Holter-ECG of 24 hours or, if the patient was a carrier, a device interrogation (implantable loop recorder or two-chamber/three-chamber pacemaker/defibrillator). For FU analysis, we included patients with data available after the blanking period (the first 90 days after the procedure). We analysed the LC, comparing the outcomes of the first fifteen patients of each centre with the remaining patients. In addition, we investigated the impact of an extensive use of the RM (at least 90% of all PV circumferences) on the procedural and FU outcomes. Results we enrolled 427 patients. Of these, 328 had a FU &amp;gt;90 days. The mean total procedural and fluoroscopic times were 105 (66-150) and 17 (10-28) minutes, respectively. Serious adverse procedural events occurred in 11 patients (2.6 %) as follows: 3 periprocedural transient ischaemic attacks, 4 pericardial tamponades, 1 persistent phrenic nerve palsy and 3 severe vascular complications. The mean FU period was of 365 days (194-533). AF recurrence-free rate was 76.7% (95% CI, 70.8%-81.5%) at 12 months. Median RM use was 94% (81-100) of the PVs circumference. RM could be used for at least 90% of all PV circumferences in 188 (55.8%) patients, and the operators could perform an RM-only procedure in 82 (24.3%) patients. Patients treated after the LC or with extensive use of RM showed significantly reduced procedural times and rates of AF recurrence. In a multivariate analysis, the use of the RM was not an independent predictor of a lower recurrence rate at FU, demonstrating only a tendency toward significance (86.0% [77.3-91.6] vs 71.3% [61.3-79.2], HR 0.52, CI 0.26-1.07, p 0.075). Conclusion In this prospective, multicentre, international registry of patients treated with the third-generation ELB, the system proved efficient, safe, and effective. Completing the LC and using the RM reduced procedural time and arrhythmia recurrence. However, using RM was not an independent predictor of a lower recurrence rate.

Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.

Background: Gaucher disease (GD) is a rare, autosomal, recessive condition characterized by hepatosplenomegaly, thrombocytopenia, anemia, and bone abnormalities, often requiring life-long treatment. Velaglucerase alfa has improved hematologic and visceral parameters in clinical trials; however, limited long-term efficacy and safety data are available. Methods: The Gaucher Outcome Survey (GOS), a structured and validated international registry for patients with confirmed GD, provides an opportunity to evaluate long-term data from patients receiving velaglucerase alfa. Results: This analysis included 376 treatment-naïve children and adults with GD enrolled in GOS, including 20 with type 3 GD, who initiated velaglucerase alfa through participation in clinical trials or as part of their clinical management and continued treatment for a mean (range) time of 6.6 (0.003-18.6) years. Initial improvements in hematologic and visceral parameters and the biomarkers glucosylsphingosine (lyso-GL1) and chitotriosidase were observed after one year of treatment and were maintained throughout the follow-up period. Of 129 (34.3%) patients who developed adverse events during the follow-up period, events were considered related to treatment in 33 (8.8%). None led to treatment discontinuation. There were 21 deaths overall, none of which were considered related to treatment. Conclusions: This analysis of data from the GOS registry supports the safety and efficacy of velaglucerase alfa in patients with GD.

Acute management of cerebral venous thrombosis: Indications, technique, and outcome of endovascular treatment in two high-volume centers.

After several uncontrolled studies and one randomized clinical trial, there is still uncertainty regarding the role of endovascular treatment (EVT) in cerebral venous thrombosis (CVT). This study aims to describe and assess different acute management strategies in the treatment of CVT. We performed a retrospective analysis of an international two-center registry of CVT patients admitted since 2019. Good outcome was defined as a return to baseline modified Rankin scale at three months. We described and compared EVT versus no-EVT patients. We included 61 patients. Only one did not receive systemic anticoagulation. EVT was performed in 13/61 (20%) of the cases, with a median time from diagnosis to puncture of 4.5 h (1.25-28.5). EVT patients had a higher median baseline NIHSS [6 (IQR 2-17) vs 0 (0-2.7), p = 0.002)] and a higher incidence of intracerebral hemorrhage (53.8% vs 20.3%, p = 0.03). Recanalization was achieved in 10/13 (77%) patients. Thrombectomy was performed in every case with angioplasty in 7 out of 12 patients and stenting in 3 cases. No postprocedural complication was reported. An improvement of the median NIHSS from baseline to discharge [6 (2-17) vs 1(0-3.75); p < 0.001] was observed in EVT group. A total of 31/60 patients (50.8%) had good outcomes. Adjusting to NIHSS and ICH, EVT had a non-significant increase in the odds of a good outcome [aOR 1.42 (95%CI 0.73-2.8, p = 0.307)]. EVT in combination with anticoagulation was safe in acute treatment of CVT as suggested by NIHSS improvement. Selected patients may benefit from this treatment.

Patient-reported impact of symptoms in adrenoleukodystrophy (PRISM-ALD)

BackgroundAdrenoleukodystrophy (ALD) is a multifaceted, X-linked, neurodegenerative disorder that comprises several clinical phenotypes. ALD affects patients through a variety of physical, emotional, social, and other disease-specific factors that collectively contribute to disease burden. To facilitate clinical care and research, it is important to identify which symptoms are most common and relevant to individuals with any subtype of ALD.MethodsWe conducted semi-structured qualitative interviews and an international cross-sectional study to determine the most prevalent and important symptoms of ALD. Our study included adult participants with a diagnosis of ALD who were recruited from national and international patient registries. Responses were categorized by age, sex, disease phenotype, functional status, and other demographic and clinical features.ResultsSeventeen individuals with ALD participated in qualitative interviews, providing 1709 direct quotes regarding their symptomatic burden. One hundred and nine individuals participated in the cross-sectional survey study, which inquired about 182 unique symptoms representing 24 distinct symptomatic themes. The symptomatic themes with the highest prevalence in the overall ALD sample cohort were problems with balance (90.9%), limitations with mobility or walking (87.3%), fatigue (86.4%), and leg weakness (86.4%). The symptomatic themes with the highest impact scores (on a 0–4 scale with 4 being the most severe) were trouble getting around (2.35), leg weakness (2.25), and problems with balance (2.21). A higher prevalence of symptomatic themes was associated with functional disability, employment disruption, and speech impairment.ConclusionsThere are many patient-relevant symptoms and themes that contribute to disease burden in individuals with ALD. These symptoms, identified by those having ALD, present key targets for further research and therapeutic development.

Outcome predictors of post-COVID conditions in the European Academy of Neurology COVID-19 registry.

Several neurological manifestations are part of the post-COVID condition. We aimed to: (1) evaluate the 6-month outcome in the cohort of patients with neurological manifestations during the COVID-19 acute phase and surviving the infection, and find outcome predictors; (2) define the prevalence and type of neurological symptoms persistent at six months after the infection. Data source was an international registry of patients with COVID-19 infection and neurological symptoms, signs or diagnoses established by the European Academy of Neurology. Functional status at six-month follow-up was measured with the modified Rankin scale (mRS), and defined as: "stable/improved" if the mRS at six months was equal as or lower than the baseline score; "worse" if it was higher than the baseline score. By October 30, 2022, 1,003 lab-confirmed COVID-19 patients were followed up for a median of 6.5months. Compared to their pre-morbid status, 522 patients (52%) were stable/improved, whereas 465 (46%) were worse (functional status missing for 16). Age, hospitalization, several pre-COVID-19 comorbidities, and COVID-19 general complications were predictors of a worse status. Amongst neurological manifestations, stroke carried the highest risk for worse outcome (OR 5.96), followed by hyperactive delirium (2.8), and peripheral neuropathies (2.37). On the other hand, hyposmia/hypogeusia (0.38), headache (0.40), myalgia (0.45), and COVID-19 vaccination (0.52) were predictors of a favourable prognosis. Persisting neurological symptoms or signs were reported by 316/1003 patients (31.5%), the commonest being fatigue (n = 133), and impaired memory or concentration (n = 103). Our study identified significant long-term prognostic predictors in patients with COVID-19 and neurological manifestations.

The International Association for the Study of Lung Cancer Pleural Mesothelioma Staging Project: Expanded Database to Inform Revisions in the Ninth Edition of the TNM Classification of Pleural Mesothelioma

The International Association for the Study of Lung Cancer collaborated with the International Mesothelioma Interest Group to propose the first TNM stage classification system for diffuse pleural mesothelioma in 1995, accepted by the Union for International Cancer Control and the American Joint Committee on Cancer for the sixth and seventh edition stage classification manuals. The International Association for the Study of Lung Cancer Staging and Prognostic Factors Committee Mesothelioma Domain developed and analyzed an international registry of patients with pleural mesothelioma and updated TNM descriptors for the eighth edition of the stage classification system. To inform revisions for the forthcoming ninth edition of the TNM stage classification system, data submission was solicited for patients diagnosed between 2013 and 2022 with expanded data elements on the basis of the first project’s exploratory analyses, including pleural thickness measurements, updated surgical nomenclature, and molecular markers. The resulting database consisted of a total of 3598 analyzable cases from Europe, Australia, Asia, North America, and South America, with a median age of 71 years (range: 18–99 y), 2775 (77.1%) of whom were men. With only 1310 patients (36.4%) undergoing curative-intent operations, this iteration of the database includes far more patients treated nonsurgically compared with prior. Four separate manuscripts on T, N, M, and stage groupings submitted to this journal will summarize analyses of these data and will serve collectively as the primary source of the proposed changes to the upcoming ninth edition of the pleural mesothelioma stage classification system.

Prognostic performance of the IABP-SHOCK II Risk Score among cardiogenic shock subtypes in the critical care cardiology trials network registry

BackgroundRisk stratification has potential to guide triage and decision-making in cardiogenic shock (CS). We assessed the prognostic performance of the IABP-SHOCK II score, derived in Europe for acute myocardial infarct-related CS (AMI-CS), in a contemporary North American cohort, including different CS phenotypes. MethodsThe Critical Care Cardiology Trials Network (CCCTN) coordinated by the TIMI Study Group is a multicenter network of cardiac intensive care units (CICU). Participating centers annually contribute ≥2 months of consecutive medical CICU admissions. The IABP-SHOCK II risk score includes age >73 years, prior stroke, admission glucose >191 mg/dl, creatinine >1.5 mg/dl, lactate >5 mmol/l, and post-PCI TIMI flow grade <3. We assessed the risk score across various CS etiologies. ResultsOf 17,852 medical CICU admissions 5,340 patients across 35 sites were admitted with CS. In patients with AMI-CS (n=912), the IABP-SHOCK II score predicted a >3-fold gradient in in-hospital mortality (low risk = 26.5%, intermediate risk =52.2%, high risk = 77.5%, p<0.0001; c-statistic=0.67; Hosmer-Lemeshow p=0.79). The score showed a similar gradient of in-hospital mortality in patients with non-AMI-related CS (n=2,517, p<0.0001) and mixed shock (n=923, p<0.001), as well as in left ventricular (<0.0001), right ventricular (p = 0.0163) or biventricular (<0.0001) CS. The correlation between the IABP-SHOCK II score and SOFA was moderate (r2=0.17) and the IABP-SHOCK II score revealed a significant risk gradient within each SCAI Stage. ConclusionsIn an unselected international multicenter registry of patients admitted with CS, the IABP- SHOCK II score only moderately predicted in-hospital mortality in a broad population of CS regardless of etiology or irrespective of right, left, or bi-ventricular involvement.

Respiratory impairments in patients suffering from Fabry disease - A cross-sectional study.

The inherited X-linked disorder, Fabry disease, is caused by deficient lysosomal enzyme α-galactosidase A, with progressive accumulation of globotriaosylceramide in multiple organs including the upper and lower airways. To assess pulmonary function at the time of the first pulmonary function test (PFT) performed among the National Danish Fabry cohort and define the prevalence of affected lung function variables. A cross-sectional retrospective cohort study of 86 adult patients enrolled in one or both international patient registry databases for Fabry disease, Fabry Registry or FollowME with at least one PFT. The Mainz Severity Score Index (MSSI) was calculated to determine the disease severity. Lung function variables were examined by multivariate regression adjusted for important variables for developing airway illness. Seventeen patients (20%) showed obstructive airflow limitation and 7 (8%) a restrictive lung deficiency. Smoking status (p = .016) and MSSI (p < .001) were associated with increasing obstructive airway limitation. The prevalence of affected lung function among the National Danish Fabry cohort was 28%. Patients with classic gene variants frequently developed a decrease in lung function regardless of their smoking status, with significant relationship with disease severity.

Impact of high-profile transvalvular pumps in heasrt failure related cardiogenic shock

Abstract Background Heart failure-related cardiogenic shock (HF-CS) not related to acute MI is a leading cause of CS.(1) Congestion is a major predictor of mortality among patients with HF-CS, for whom in-hospital mortality remains between 25-40%.(2,3) Both low-profile (Impella CP) and high-profile (Impella 5.0/5.5) transvalvular pumps are used to manage these patients. In the last few years, use of Impella 5.0/5.5 pumps in these patients is increasing, although it is often used as an escalation strategy from other temporary mechanical circulatory support (tMCS) devices. Purpose To assess the impact of LV unloading using high profile transvalvular pump (Impella 5.0/5.5) compared to a low-profile pump (Impella CP) on outcomes in HF-CS. Methods The Cardiogenic Shock Working Group (CSWG) is a large, multi-center international prospective registry of CS patients.(1) For this analysis, we retrospectively reviewed patients with HF-CS who underwent Impella 5.0/5.5 therapy for HF-CS and compared them to patients who underwent an Impella CP. Patients who received an Impella device as part of their escalation strategy from another device (IABP) or concomitant or de-escalation strategy with ECMO were excluded. Results Of the 2,755 patients with HF-CS, 125 patients received an Impella 5.0/5.5 while 152 patients received an Impella CP alone; 13 patients were escalated from an Impella CP to 5.0/5.5 pump. Of these 292 patients who either received an Impella CP or 5.0/5.5 or a combination of the two, 228(78%) were male, 208(71%) were white with a mean LVEF at presentation of 19%. About a third (n=78, 27%) presented with de-novo HF-CS while 214(73%) were acute on chronic HF-CS. In-hospital mortality in the entire HF-CS cohort was 717(26%). Unadjusted mortality was higher in those who underwent Impella CP alone (n= 57; 38%) compared to patients with Impella 5.0/5.5 (n=27, 23%; p=0.01). This improvement in mortality was seen in acute on chronic HF-CS with Impella 5.5/5.0 vs CP (41% vs 26%, p=0.02), and as a trend in de-novo HF-CS (30% vs 14%, p=0.13). More patients on Impella 5.0/5.5 went on to receive heart replacement therapies (durable LVAD or heart transplantation) compared to Impella CP (56% vs 9%, p&amp;lt;0.001). Conclusion(s) Use of a high-profile transvalvular pump (Impella 5.0/5.5) was associated with improved mortality compared to a low-profile transvalvular pump (Impella CP) in HF-CS.

Endovascular treatment of cerebral sinus thrombosis due to vaccine-induced immune thrombotic thrombocytopenia.

There is little data on the role of endovascular treatment (EVT) of cerebral venous sinus thrombosis (CVST) due to vaccine-induced immune thrombotic thrombocytopenia (VITT). Here, we describe clinical characteristics and outcomes of CVST-VITT patients who were treated with EVT. We report data from an international registry of patients who developed CVST within 28 days of SARS-CoV-2 vaccination, reported between 29 March 2021 and 6 March 2023. VITT was defined according to the Pavord criteria. EVT was performed in 18/136 (13%) patients with CVST-VITT (92% aspiration and/or stent retrieval, 8% local thrombolysis). Most common indications were extensive thrombosis and clinical or radiological deterioration. Compared to non-EVT patients, those receiving EVT had a higher median thrombus load (4.5 vs 3). Following EVT, local blood flow was improved in 83% (10/12, 95% confidence interval [CI] 54-96). One (6%) asymptomatic sinus perforation occurred. Eight (44%) patients treated with EVT also underwent decompressive surgery. Mortality was 50% (9/18, 95% CI 29-71) and 88% (8/9, 95% CI 25-66) of surviving EVT patients achieved functional independence with a modified Rankin Scale score of 0-2 at follow-up. In multivariable analysis, EVT was not associated with increased mortality (adjusted odds ratio, 0.66, 95% CI 0.16-2.58). We describe the largest cohort of CVST-VITT patients receiving EVT. Half of the patients receiving EVT died during hospital admission, but most survivors achieved functional independence.

The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data

BackgroundX-linked hypophosphatemia (XLH) is a rare, hereditary, progressive, renal phosphate-wasting disorder characterized by a pathological increase in FGF23 concentration and activity. Due to its rarity, diagnosis may be delayed, which can adversely affect outcomes. As a chronic disease resulting in progressive accumulation of musculoskeletal manifestations, it is important to understand the natural history of XLH over the patient’s lifetime and the impact of drug treatments and other interventions. This multicentre, international patient registry (International XLH Registry) was established to address the paucity of these data. Here we present the findings of the first interim analysis of the registry.ResultsThe International XLH Registry was initiated in August 2017 and includes participants of all ages diagnosed with XLH, regardless of their treatment and management. At the database lock for this first interim analysis (29 March 2021), 579 participants had entered the registry before 30 November 2020 and are included in the analysis (360 children [62.2%], 217 adults [37.5%] and 2 whose ages were not recorded [0.3%]; 64.2% were female). Family history data were available for 319/345 (92.5%) children and 145/187 (77.5%) adults; 62.1% had biological parents affected by XLH. Genetic testing data were available for 341 (94.7%) children and 203 (93.5%) adults; 370/546 (67.8%) had genetic test results; 331/370 (89.5%) had a confirmed PHEX mutation. A notably longer time to diagnosis was observed in adults ≥ 50 years of age (mean [median] duration 9.4 [2.0] years) versus all adults (3.7 [0.1] years) and children (1.0 [0.2] years). Participants presented with normal weight, shorter length or height and elevated body mass index (approximately − 2 and + 2 Z-scores, respectively) versus the general population. Clinical histories were collected for 349 participants (239 children and 110 adults). General data trends for prevalence of bone, dental, renal and joint conditions in all participants were aligned with expectations for a typical population of people with XLH.ConclusionThe data collected within the International XLH Registry, the largest XLH registry to date, provide substantial information to address the paucity of natural history data, starting with demographic, family history, genetic testing, diagnosis, auxology and baseline data on clinical presentation.

Antithrombotic management of patients with deep vein thrombosis and venous stents: an international registry

BackgroundIn patients with acute deep vein thrombosis (DVT) treated with catheter-based thrombolysis and venous stenting, poststenting anticoagulant management is uncertain. ObjectivesTo determine the type and duration of antithrombotic therapy used in patients who have received venous stents for treatment of acute lower extremity DVT. MethodsWe created an international registry of patients with leg DVT from 2005 to 2019 who received venous stents as part of their acute management. We collected data on baseline clinical characteristics and pre-venous and post-venous stent antithrombotic therapy. ResultsWe studied 173 patients with venous stents: 101 (58%) were aged ≤50 years, 105 (61%) were female, and 128 (74%) had risk factors for thrombotic disease. DVT was iliofemoral in 150 (87%) patients, and catheter-based treatment was given within 7 days of diagnosis in 92 (53%) patients. After venous stenting, 109 (63%) patients received anticoagulant-only therapy with a direct oral anticoagulant (29%), warfarin (22%), or low-molecular-weight heparin (10%), and 59 (34%) received anticoagulant-antiplatelet therapy. In patients taking anticoagulant-only therapy, 29% received indefinite treatment; in patients on anticoagulant-antiplatelet therapy, 19% received indefinite treatment. Factors associated with combined anticoagulant-antiplatelet therapy vs anticoagulant-only therapy were use of thrombolytic, thrombectomy, and aspiration interventions (odds ratio [OR], 5.11; 95% CI, 1.45-18.05); use of balloon angioplasty (OR, 2.62; 95% CI, 1.20-5.76); and immediate stent restenosis (OR, 7.2; 95% CI, 1.45-5.89). ConclusionAnticoagulant therapy without concomitant antiplatelet therapy appears to be the most common antithrombotic strategy in patients with DVT and venous stenting. More research is needed to determine outcomes of venous stenting in relation to antithrombotic therapy.

The ELEGANCE Registry: Working to Achieve Equity in Clinical Research Design

Summary Peripheral artery disease (PAD) disproportionately affects women and underrepresented, historically marginalized groups (UMGs); however, these patients are not well-represented in clinical research of PAD treatments. Most patients enrolled in trials are older white men. White women and underrepresented populations of any gender present differently and respond differently to therapies compared with white men. They must be represented in clinical studies of PAD in proportion to their numbers in the patient population to ensure generalizability of study findings and the development of optimal individualized care. The many obstacles to enrolling these populations in clinical studies include mistrust of the medical system, no proximity to clinics and providers who run clinical trials, limited understanding about the biomedical research process, language barriers, financial and transportation limitations, and inability to take time off from work or from caring for others in the household. Given the complexity of PAD and the need for clinical data from diverse patient populations that are not available from existing clinical trials, the authors created an international registry of patients treated with advanced therapies for PAD. The Drug-ELuting REGistry: ReAl-World Treatment of LesioNs in the Peripheral VasCulaturE (ELEGANCE) registry is a prospective international postmarket registry examining real-world use of drug-coated devices in patients with symptomatic PAD and includes several novel design elements aimed at enrolling at least 40% women and 40% underrepresented patients. As of December 1, 2022, over 800 patients were enrolled in the registry, ahead of target enrollment rates; of these, 44% are women and 47% are UMGs.

Moraxella catarrhalis in People with Cystic Fibrosis

BACKGROUND: The gammaproteobacteria, Moraxella catarrhalis (MC) is commonly isolated from respiratory specimens from people with cystic fibrosis (PwCF), although its prevalence and clinical significance in cystic fibrosis (CF) remains largely unreported in international patient registries and the wider scientific literature. AIM AND OBJECTIVE: The aim of this study was to examine the time-to-first-isolation of MC and to correlate this with patient gender and CF transmembrane conductance regulator (CFTR) mutation type. MATERIALS AND METHODS: The microbiology of respiratory specimens from 100 adult (≥18 years) PwCF was examined (50 females; 50 males; mean age 24.6 years ± 6.25 (Standard deviation [SD]) from birth to present, equating to 2455 patient-years. Time-to-first-isolation was determined and correlated with (i) CFTR mutation type and (ii) PwCF gender. RESULTS: MC was isolated from 27 (27/100 [27%] adult CF patients [18 female/9 males]); (i) F508del/F508del homozygous (55.6%; 15/27) and (ii) F508del/other heterozygous (37.0%; 10/27) and (iii) Other (7.4%; 2/27). The median and mean time to the first isolation of MC was 60 months and 71.8 months (SD = 66.2 months; standard error of the mean = 12.7 months), respectively; shortest time was 1 month, the longest time was 254 months. The highest rate of MC isolation was from 2 years until 10 years and by the age of 18 years, approximately 96.3% of positive patients had recorded their first MC isolate. By 21.2 years, all positive patients had recorded their first MC isolation. There was no statistical significance in the time-to-first-isolation of MC in relation to the CFTR mutation group or with patient gender. CONCLUSIONS: MC is commonly isolated from respiratory specimens of PwCF. This work represents an advance in biomedical science as it identifies time points in the person with CF’s disease staging, which may allow mitigating infection prevention interventions to be established, as well as vigilance to help prevent MC acquisition.

Thrombotic and hemorrhagic complications of COVID-19 in adults hospitalized in high-income countries compared with those in adults hospitalized in low- and middle-income countries in an international registry

BackgroundCOVID-19 has been associated with a broad range of thromboembolic, ischemic, and hemorrhagic complications (coagulopathy complications). Most studies have focused on patients with severe disease from high-income countries (HICs). ObjectivesThe main aims were to compare the frequency of coagulopathy complications in developing countries (low- and middle-income countries [LMICs]) with those in HICs, delineate the frequency across a range of treatment levels, and determine associations with in-hospital mortality. MethodsAdult patients enrolled in an observational, multinational registry, the International Severe Acute Respiratory and Emerging Infections COVID-19 study, between January 1, 2020, and September 15, 2021, met inclusion criteria, including admission to a hospital for laboratory-confirmed, acute COVID-19 and data on complications and survival. The advanced-treatment cohort received care, such as admission to the intensive care unit, mechanical ventilation, or inotropes or vasopressors; the basic-treatment cohort did not receive any of these interventions. ResultsThe study population included 495,682 patients from 52 countries, with 63% from LMICs and 85% in the basic treatment cohort. The frequency of coagulopathy complications was higher in HICs (0.76%-3.4%) than in LMICs (0.09%-1.22%). Complications were more frequent in the advanced-treatment cohort than in the basic-treatment cohort. Coagulopathy complications were associated with increased in-hospital mortality (odds ratio, 1.58; 95% CI, 1.52-1.64). The increased mortality associated with these complications was higher in LMICs (58.5%) than in HICs (35.4%). After controlling for coagulopathy complications, treatment intensity, and multiple other factors, the mortality was higher among patients in LMICs than among patients in HICs (odds ratio, 1.45; 95% CI, 1.39-1.51). ConclusionIn a large, international registry of patients hospitalized for COVID-19, coagulopathy complications were more frequent in HICs than in LMICs (developing countries). Increased mortality associated with coagulopathy complications was of a greater magnitude among patients in LMICs. Additional research is needed regarding timely diagnosis of and intervention for coagulation derangements associated with COVID-19, particularly for limited-resource settings.