Multicenter Registry-based Study Research Articles

Prospective Multicenter Registry-Based Study on Thyroid Storm: The Guidelines for the Management from Japan are Useful.

The mortality rate in thyroid storm (TS) has been reported to be higher than 10%. We aimed to evaluate the effectiveness of the 2016 guidelines for the management of TS proposed by the Japan Thyroid Association and Japan Endocrine Society. Prospective registry-based study through a secure web platform. Prospective multicenter registry. Patients with new-onset TS were registered in the Research Electronic Data Capture (REDCap). On day 30 after admission, clinical information and prognosis of each patient were added to the platform. On day 180, the prognosis was described. This study included 110 patients with TS. The median of Acute Physiology and Chronic Health Evaluation (APACHE) II score was 13, higher than the score in the previous nationwide epidemiological study, 10 (p = 0.001). Nonetheless, the mortality rate at day 30 was 5.5%, approximately half compared with 10.7% in the previous nationwide survey. Lower body mass index, shock and lower left ventricular ejection fraction were positively associated with poor prognosis at day 30, while the lack of fever ≥ 38℃ was related to the outcome. The mortality rate in patients with an APACHE II score ≥12 for whom the guidelines were not followed was significantly higher than the rate in patients for whom the guidelines were followed (50% vs. 4.7%) (p = 0.01). Prognosis seemed better than in the previous nationwide survey, even though disease severity was higher. The mortality rate was lower when the guidelines were followed. Thus, the guidelines are useful for managing TS.

A Multi-Center Retrospective Analysis of Outcomes Post Allogeneic Stem Cell Transplantation in AML Patients with TET2 Mutations: A Study on Behalf of the Global Committee and the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

Background Mutations in TET2 present in about 15% of adult AML are reported in some limited series as an adverse prognostic factor for overall survival. However the impact of allogeneic hematopoietic cell transplantation (allo-HCT) for the treatment of these patients remains unclear. We addressed this issue in a EBMT global multi-center registry-based study. Patients and Methods 644 adult AML patients with TET2 mutations receiving first non ex-vivo depleted allo-HCT from 2013-2022 in 127 centers were analyzed. All patients achieved first complete remission (CR1) before allo-HCT. Results The median age of the 644 AML patients was 59.4 (range, 18.1-86.3) years. 556 patients (86.3%) had de novo, and 88 (13.7%) had secondary AML, respectively. 367 (57.2%) patients were male. The median interval from diagnosis to allo-HCT was 4.9 (IQR, 3.8-6.2) months. Patients were categorized into favorable -(N=23, 3.9%), intermediate-(N=475, 79.8%) and adverse (N=97, 16.3%) risk categories according to cytogenetic characteristics. Conditioning regimen was myeloablative (MAC) in 46.7% of patients. 149 patients (23.1%) received a matched-sibling donor, 176 (27.3%) a haploidentical donor (Haplo), 268 (41.6%) a 10/10 unrelated donor (UD) and 51 (7.9%) a 9/10 UD allo-HCT, respectively. For the entire cohort, with a median follow-up of 1.9 years, the 30-day cumulative incidence of engraftment was 97.6%. The 100-day cumulative incidence of grade II-IV aGVHD was 22.9% and the 2-year cumulative incidence of extensive cGVHD was 13.7%. In addition, the 2-year relapse incidence and NRM were 23.2% and 12.8%, respectively. Finally, the 2-year OS, LFS and GRFS were 69.8%, 63.9% and 49.8%, respectively. In multivariable analyses, a donor type 9/10 UD was associated with a higher RI (HR=2.33, 95% CI 1.21-4.48; p=0.01), a lower LFS (HR=2.20, 95% CI 1.31-3.70; p<0.01) and OS (HR=2.29, 95% CI 1.33-3.96; p<0.01) as compared to MSD as reference. Compared to MSD, 10/10 UD and Haplo had similar outcomes. The adverse-cytogenetic group was associated with higher RI (HR=1.85, 95% CI 1.19-2.86; p<0.01), and higher incidence of grade II-IV acute GVHD (HR=1.74, 95% CI 1.14-2.64; p=0.01) as compared to other groups combined as reference. Compared to de novo AML (reference), secondary AML was associated with lower LFS (HR=1.49, 95% CI 1.03-2.15; p<0.05) and OS (HR=1.68, 95% CI 1.15-2.45; p<0.01). When age increased, patients faced higher RI (HR for 10y increment=1.24, 95% CI 1.01-1.53; p<0.05) and extensive chronic GVHD (HR=1.34, 95% CI 1.02-1.77; p<0.05), which translated into higher NRM (HR=1.66, 95% CI 1.23-2.24; p<0.01), lower LFS (HR=1.36, 95% CI 1.14-1.62; p<0.01) and OS (HR=1.60, 95% CI 1.31-1.96; p<0.01). Later year of transplantation was associated with lower incidence of II-IV acute GVHD (HR for 2y increment=0.76, 95% CI 0.63-0.92; p<0.01), chronic GVHD (HR=0.67, 95% CI 0.54-0.83; p<0.01) and extensive chronic GVHD (HR=0.72, 95% CI 0.52-0.99; p<0.05). Female to male, patient CMV status and interval between diagnosis and HCT did not affect significantly the transplant outcomes in multivariable analysis. Conclusion No significant difference was observed in outcomes of AML patients with TET2 mutations following MSD, HAPLO and 10/10 UD allo-HCTs, while 9/10 UD was associated with higher relapse incidence and lower survival outcomes. Higher patient age, secondary AML and adverse cytogenetics predicted worse transplant outcomes. GVHD prevention improved with time for these patients.

Sex differences in 82Rubidium PET-derived myocardial flow reserve and implications for long-term cardiac outcomes

Abstract Introduction Coronary small vessel disease (CMD), defined by reduced myocardial flow reserve (MFR) in the absence of obstructive coronary artery disease, holds prognostic information on major cardiac events (MACE). We aimed to investigate if the prognostic value of MFR is independent of sex. Method A multicenter registry-based study of 7169 consecutive patients referred to clinically 82Rubidium PET myocardial perfusion imaging. We excluded patients with abnormal perfusion (defects >5% at rest or stress), previous revascularization, cardiomyopathy, or a diagnosis of heart failure. Global MFR≤2 was considered reduced. Patients were followed in registries for major adverse cardiac events (MACE) comprising all-cause mortality and admission for myocardial infarction, stroke, heart failure or unstable angina pectoris. Results A total of 2591 patients were included, 51% women, median age 69 [IQR 60-75] years. Women were older and men had a higher burden of cardiovascular risk factors. Overall, 31% had MFR≤2 (34% women vs 28% men, p<0.001). Mean MFR was lower in women compared with men (2.30 (SD 0.66) vs 2.44 (0.70), respectively) with higher myocardial blood flow both at rest and during stress among women (both p<0.001, Figure 1). In a linear regression model, sex remained associated with MFR after adjustments for age (p<0.001). At 3-year follow-up, MACE was less frequent in women than men (7.0% vs 9.4%, p=0.03). MFR≤2 was associated with MACE with an age-adjusted hazard ratio (HR) of 1.83 (95% CI: 1.20-2.77, p<0.01) in women and 1.75 (1.21-2.54, p<0.01) in men (p for interaction 0.82). The association remained significant after adjustments for left ventricular ejection fraction and charlson comorbidity index with a HR of 1.58 (1.07-2.31, p<0.05) in men and a HR of 1.68 (1.09-2.58, p<0.05) in women (p for interaction 0.69). On a continuous scale, MFR per every 0.1 unit decrease remained significantly associated to MACE (Figure 2). Conclusions Among patients suspected of obstructive CAD, CMD is more prevalent among women than men. The increased hazard of adverse outcomes with CMD did not differ by sex. This study provides new insight into the phenotype and prognosis of patients with CMD.Figure 1:Baseline distribution of MFRFigure 2:HR for MACE

269 Association Between Initial pH Levels and Neurological Outcomes in Out-of-Hospital Cardiac Arrest Patients with Shockable Rhythm: A Multi-Center Registry-Based Study

269 Association Between Initial pH Levels and Neurological Outcomes in Out-of-Hospital Cardiac Arrest Patients with Shockable Rhythm: A Multi-Center Registry-Based Study

Defibrillation testing during S-ICD implantation: How relevant? Results from a multicenter study

Abstract Funding Acknowledgements Type of funding sources: None. Introduction Defibrillation testing (DFT) is still recommended in patients with subcutaneous implantable cardioverter-defibrillator (S-ICD) to ensure proper detection of ventricular fibrillation and confirm effective defibrillation. Given the significant risk of complications, recent studies, are reconsidering the interest of the DFT in S-ICD. The purpose of this study was to assess the clinical "real-life" outcomes of DFT over a mid-term follow-up period after third-generation S-ICD implantation. Methods and Results 368 patients (pts) (47.9 ± 15.5 years old; male 72.8%) implanted with Generation 3 S-ICD system were included in this retrospective multicenter registry-based study. Mean left ventricular ejection fraction (LVEF) was 40.8% (primary prevention 56.1%). For 99.2% pts, S-ICD implantation was performed using the two-incision technique and intermuscular positioning of the generator was performed for all pts. 81% pts underwent ICD testing during implantation. Reasons for not performing DFT were: very low LVEF (3.5%), intracardiac thrombus (2.4%) and VF induction failure. In 2.2% (8 pts) DFT was unsuccessful, causes were: induction of ventricular tachycardias with rates falling below the shock zone for 3 pts while shocks were ineffective for 5 pts. One patient required revision for contralateral lead implantation, while sensing vector reprogramming and shock polarity modification were efficient for the others. The mean impedance was 68.4 (±14.7) Ω and time to shock therapy ranged from 10 to 33 s, with a mean of 16.7 (±4) s. A total of 36 appropriate shocks (10.1%) have been observed after a FU period of 11.02±8.8 months (1.8 shocks per patient, several shocks for 12pts, failure of the first shock for 5 patients). 17 patients (4.6%) underwent S-ICD system extraction. The reasons were pocket site complication (7 pts), pacing indication for cardiac resynchronization therapy or bradycardia (3 pts), and S-ICD lead dysfunction (4 pts). Extraction after heart transplant was needed in 3 pts. Among the 4 pts explanted for system dysfunction, only one patient had a DFT during implantation. After a mean follow-up of 19±12.1 months, the mortality rate was 3.8% (no S-ICD-related deaths were reported). There was no significant difference between the 2 groups of pts with and without DFT testing (p=0.629). Conclusion The DFT remains for the moment highly recommended during S-ICD implantations. Results from randomized studies are still needed to confirm the relevance of DFT during S-ICD implantation.

Stroke-Specific Predictors of Major Bleeding in Anticoagulated Patients With Stroke and Atrial Fibrillation: A Nationwide Multicenter Registry-Based Study.

The congestive heart failure, hypertension, age, diabetes, previous stroke/transient ischemic attack (CHA2DS2-VASc) and hypertension, abnormal renal/liver function, stroke, bleeding history or predisposition, labile international normalized ratio, elderly, drugs/alcohol (HAS-BLED) scores have been validated in estimating the risks of ischemic stroke and major bleeding, respectively, in patients with atrial fibrillation (AF). This study investigated stroke-specific predictors of major bleeding in patients with stroke and AF who were taking oral anticoagulants (OACs). Subjects were selected from patients enrolled in the Korean ATrial fibrillaTion EvaluatioN regisTry in Ischemic strOke patieNts (K-ATTENTION) nationwide multicenter registry between 2013 and 2015. Patients were excluded if they were not taking OACs, had no brain imaging data, or had intracranial bleeding directly related to the index stroke. Major bleeding was defined according to International Society of Thrombosis and Haemostasis criteria. Cox regression analyses were performed to assess the associations between clinical variables and major bleeding and Kaplan-Meier estimates were performed to analyze event-free survival. Of a total of 3,213 patients, 1,414 subjects (mean age of 72.6 years, 52.5% males) were enrolled in this study. Major bleeding was reported in 34 patients during the median follow-up period of 1.73 years. Multivariable analysis demonstrated that initial National Institutes of Health Stroke Scale scores (hazard ratio [HR] 1.07, p=0.006), hypertension (HR 3.18, p=0.030), persistent AF type (HR 2.51, p=0.016), and initial hemoglobin level (HR 0.74, p=0.001) were independently associated with major bleeding risk. Except for hypertension, these associations remained significant after adjusting for the HAS-BLED score. Intracranial atherosclerosis presented a trend of association without statistical significance (HR 2.21, p=0.050). This study found that major bleeding risk was independently associated with stroke-specific factors in anticoagulated patients with stroke and AF. This has the clinical implication that baseline characteristics of patients with stroke and AF should be considered in secondary prevention, which would bring the net clinical benefit of balancing recurrent stroke prevention with minimal bleeding complications.

Effectiveness of Different Antimicrobial Strategies for Staphylococcal Prosthetic Joint Infection: Results From a Large Prospective Registry-Based Cohort Study

BackgroundTreatment of staphylococcal prosthetic joint infection (PJI) usually consists of surgical debridement and prolonged rifampicin combination therapy. Tailored antimicrobial treatment alternatives are needed due to frequent side effects and drug-drug interactions with rifampicin combination therapy. We aimed to assess the effectiveness of several alternative antibiotic strategies in patients with staphylococcal PJI.MethodsIn this prospective, multicenter registry-based study, all consecutive patients with a staphylococcal PJI, treated with debridement, antibiotics and implant retention (DAIR) or 1-stage revision surgery between January 1, 2015 and November 3, 2020, were included. Patients were treated with a long-term rifampicin combination strategy (in 2 centers) or a short-term rifampicin combination strategy (in 3 centers). Antimicrobial treatment strategies in these centers were defined before the start of the registry. Patients were stratified in different groups, depending on the used antimicrobial strategy. Cox proportional hazards models were used to compare outcome between the groups.ResultsTwo hundred patients were included and stratified in 1 long-term rifampicin group (traditional rifampicin combination therapy) or 1 of 3 short-term rifampicin groups (clindamycin or flucloxacillin or vancomycin monotherapy, including rifampicin for only 5 postoperative days). Adjusted hazard ratios (aHRs) for failure in patients treated with short-term rifampicin and either flucloxacillin or clindamycin were almost equal to patients treated with long-term rifampicin combination therapy (aHR = 1.21; 95% confidence interval, .34–4.40).ConclusionsA short-term rifampicin strategy with either clindamycin or flucloxacillin and only 5 days of rifampicin was found to be as effective as traditional long-term rifampicin combination therapy. A randomized controlled trial is needed to further address efficacy and safety of alternative treatment strategies for staphylococcal PJI.

Metastatic Death Based on Presenting Features and Treatment for Advanced Intraocular Retinoblastoma: A Multicenter Registry-Based Study

Purpose:To evaluate presenting features, tumor size, and treatment methods for risk of metastatic death due to advanced intraocular retinoblastoma (RB).Design:International, multicenter, registry-based retrospective case series.Participants:A total of 1841 patients with advanced RB.Methods:Advanced RB was defined by 8th edition American Joint Committee on Cancer (AJCC) categories cT2 and cT3 and new AJCC-Ophthalmic Oncology Task Force (OOTF) Size Groups (1: < 50% of globe volume, 2: > 50% but < 2/3, 3: > 2/3, and 4: diffuse infiltrating RB). Treatments were primary enucleation, systemic chemotherapy with secondary enucleation, and systemic chemotherapy with eye salvage.Main Outcome Measures:Metastatic death.Results:The 5-year Kaplan–Meier cumulative survival estimates by patient-level AJCC clinical subcategories were 98% for cT2a, 96% for cT2b, 88% for cT3a, 95% for cT3b, 92% for cT3c, 84% for cT3d, and 75% for cT3e RB. Survival estimates by treatment modality were 96% for primary enucleation, 89% for systemic chemotherapy and secondary enucleation, and 90% for systemic chemotherapy with eye salvage. Risk of metastatic mortality increased with increasing cT subcategory (P < 0.001). Cox proportional hazards regression analysis confirmed a higher risk of metastatic mortality in categories cT3c (glaucoma, hazard ratio [HR], 4.9; P = 0.011), cT3d (intraocular hemorrhage, HR, 14.0; P < 0.001), and cT3e (orbital cellulitis, HR, 19.6; P < 0.001) than in category cT2a and with systemic chemotherapy with secondary enucleation (HR, 3.3; P < 0.001) and eye salvage (HR, 4.9; P < 0.001) than with primary enucleation. The 5-year Kaplan–Meier cumulative survival estimates by AJCC-OOTF Size Groups 1 to 4 were 99%, 96%, 94%, and 83%, respectively. Mortality from metastatic RB increased with increasing Size Group (P < 0.001). Cox proportional hazards regression analysis revealed that patients with Size Group 3 (HR, 10.0; P = 0.002) and 4 (HR, 41.1; P < 0.001) had a greater risk of metastatic mortality than Size Group 1.Conclusions:The AJCC-RB cT2 and cT3 subcategories and size-based AJCC-OOTF Groups 3 (> 2/3 globe volume) and 4 (diffuse infiltrating RB) provided a robust stratification of clinical risk for metastatic death in advanced intraocular RB. Primary enucleation offered the highest survival rates for patients with advanced intraocular RB.

Association between active cooling and lower mortality among patients with heat stroke and heat exhaustion.

Body cooling is recommended for patients with heat stroke and heat exhaustion. However, differences in the outcomes of patients who do or do not receive active cooling therapy have not been determined. The best available evidence supporting active cooling is based on a case series without comparison groups; thus, the effectiveness of this method in improving patient prognoses cannot be appropriately quantified. Therefore, we compared the outcomes of heat stroke patients receiving active cooling with those of patients receiving rehydration-only therapy. This prospective observational multicenter registry-based study of heat stroke and heat exhaustion patients was conducted in Japan from 2010 to 2019. The patients were stratified into the “severe” group or the “mild-to-moderate” group, per clinical findings on admission. After conducting multivariate logistic regression analyses, we compared the prognoses between patients who received “active cooling + rehydration” and patients who received “rehydration only,” with in-hospital death as the endpoint. Sex, age, onset situation (i.e., exertional or non-exertional), core body temperature, liver damage, renal dysfunction, and disseminated intravascular coagulation were considered potential covariates. Among those who received active cooling and rehydration-only therapy, the in-hospital mortality rates were 21.5% and 35.5%, respectively, for severe patients (n = 231) and 3.9% and 5.7%, respectively, for mild-to-moderate patients (n = 578). Rehydration-only therapy was associated with a higher in-hospital mortality in patients with severe heat illness (adjusted odds ratio [aOR], 3.29; 95% confidence interval [CI], 1.21–8.90), whereas the cooling methods were not associated with lower in-hospital mortality in patients with mild-to-moderate heat illness (aOR, 2.22; 95% CI, 0.92–5.84). Active cooling was associated with lower in-hospital mortality only in the severe group. Our results indicated that active cooling should be recommended as an adjunct to rehydration-only therapy for patients with severe heat illness.

Pregnancy Outcome in Women With APECED (APS-1): A Multicenter Study on 43 Females With 83 Pregnancies.

ContextAutoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy (APECED; also known as autoimmune polyendocrine syndrome type 1) has a severe, unpredictable course. Autoimmunity and disease components may affect fertility and predispose to maternal and fetal complications, but pregnancy outcomes remain unknown.ObjectiveTo assess fetal and maternal outcomes and course of clinical APECED manifestations during pregnancy in women with APECED.Design and SettingA multicenter registry-based study including 5 national patient cohorts.Patients321 females with APECED.Main Outcome MeasureNumber of pregnancies, miscarriages, and deliveries.ResultsForty-three patients had altogether 83 pregnancies at median age of 27 years (range, 17–39). Sixty (72%) pregnancies led to a delivery, including 2 stillbirths (2.4%) and 5 (6.0%) preterm livebirths. Miscarriages, induced abortions, and ectopic pregnancies were observed in 14 (17%), 8 (10%), and 1 (1.2%) pregnancies, respectively. Ovum donation resulted in 5 (6.0%) pregnancies. High maternal age, premature ovarian insufficiency, primary adrenal insufficiency, or hypoparathyroidism did not associate with miscarriages. Women with livebirth had, on average, 4 APECED manifestations (range 0-10); 78% had hypoparathyroidism, and 36% had primary adrenal insufficiency. APECED manifestations remained mostly stable during pregnancy, but in 1 case, development of primary adrenal insufficiency led to adrenal crisis and stillbirth. Birth weights were normal in >80% and apart from 1 neonatal death of a preterm baby, no serious perinatal complications occurred.ConclusionsOutcome of pregnancy in women with APECED was generally favorable. However, APECED warrants careful maternal multidisciplinary follow-up from preconceptual care until puerperium.

Australia and New Zealand (ANZ) outcomes of high-dose chemotherapy and stem cell transplantation for relapsed or refractory germ cell tumors (GCT) from 1999 to 2019: Multicenter registry-based study with ABMTRR.

382 Background: High dose chemotherapy (HDCT) with autologous stem-cell transplant (ASCT) is effective in advanced germ cell tumours (GCT) that are refractory to, or progress after, first-line therapies. Five-year overall survival in North American and European series range from 48-60%1,2,3. This study aimed to assess ANZ outcomes for quality assurance. Methods: Retrospective multi-centre audit of all male patients with GCT who underwent HDCT and ASCT from 1999-2019. Patients were identified from the Australasian Bone Marrow Transplant Recipient Registry. Primary outcomes included overall and progression- free survival (OS, PFS). Results: 111 patients were identified at 13 centres, each treating a median (range) of 7 (1-27) patients. Median (range) age was 30 (14-68) years. 88 (79%) had testicular primary. 16% were pure seminoma. Median time from first diagnosis to first stem cell cycle was 11 months (range 3 months-38 years). Prior to ASCT, 35% had primary refractory disease and 65% had relapsed. IPFSG risk score was very low in 5%, low in 13%, intermediate in 36%, high in 25%, and very high in 21%. HDCT regimen was CE in 78% (as part of TI-CE regimen in 38%), Carbop-EC-T in 6%, ICE in 6%, CEC in 5% and other in 4%. 89% completed all planned HDCT and ASCT cycles. Five treatment related deaths occurred. Progressive disease on treatment occurred in 14%. At median follow-up time 4.4 years (95% CI: 2.9 to 6.0), 51% were disease-free, 13% alive with disease, 34% deceased. 3 patients displayed late progression over 2 years after ASCT. The estimated 1, 2 and 5-year PFS rates were 62%, 57% and 52% respectively and OS rates were 73%, 65% and 61%. Survival by IPFSG and IGCCG risk categories are displayed in the table below. Conclusions: This is the first registry-based audit of HDCT for metastatic GCT from ANZ, which has demonstrated our outcomes are comparable with best international practice.

Safety of Endoscopic Ultrasound-Guided Fine-Needle Aspiration for Pancreatic Solid Pseudopapillary Neoplasm Before Surgical Resection: A European Multicenter Registry-Based Study on 149 Patients.

The results of only a few endoscopic ultrasound-guided fine-needle aspiration (EUS-FNA) for pancreatic solid pseudopapillary neoplasm (SPN) have been published, and the safety of the procedure has never been investigated. Our study compared the recurrence rate in patients with and without preoperative EUS-FNA. This European multicenter registry-based study was conducted in 22 digestive units, and retrospectively included all patients who underwent complete resection of a pancreatic SPN from 2000 to 2018. Patients with and without initial EUS-FNA were compared, and postsurgery recurrence and the associated risk factors were evaluated. A complete resection of a pancreatic SPN was performed in 149 patients (133 women, 89%), with a mean age of 34 (standard deviation, 14) years. There were no significant differences between the with (78 patients) and without (71 patients) EUS-FNA groups, except for age and tumor size and location.Preoperative EUS-FNA allowed pancreatic SPN diagnosis in 63/78 cases (81%). After a mean follow-up of 43 (standard deviation, 36) months, recurrence was noted in 4 patients (2.7%). Preoperative EUS-FNA was not correlated with recurrence, but an older age (P = 0.005) was significant. Preoperative EUS-FNA does not affect pancreatic SPN recurrence. In this series, old age was significantly correlated with recurrence.

Prognostic Impact of Scoring Balloon Angioplasty After Rotational Atherectomy in Heavily Calcified Lesions Using Second-Generation Drug-Eluting Stents: A Multicenter Registry-Based Study

We aimed to assess the impact of scoring balloon angioplasty (SBA) after rotational atherectomy (RA) on long-term clinical outcomes in patients who underwent percutaneous coronary intervention (PCI) using second-generation drug-eluting stents (DES). The long-term outcomes associated with SBA after RA in severely calcified lesions is unknown. Using the J2T ROTA registry data, we evaluated the clinical events of patients who underwent PCI using RA for heavily calcified lesions from January 2004 to December 2015. A total of 307 patients who underwent PCI with second-generation DES were analyzed and divided into the SBA (n = 96) and conventional balloon angioplasty (CBA) groups (n = 211). Eighty-two and 189 patients comprised the "SBA after small burr (SBA-SB)" and "CBA after small burr (CBA-SB)" subgroups, respectively, for the subgroup analysis. Study endpoints were incidence of 3-year major adverse cardiac events (MACE), target vessel revascularization (TVR), and target lesion revascularization (TLR). Kaplan-Meier analysis revealed that the incidence of 3-year cumulative MACE, TVR, and TLR were comparable between groups, and that the incidences of 3-year cumulative MACE, TVR, and TLR were significantly lower in the SBA-SB subgroup than in the CBA-SB subgroup (log-rank p = 0.008; log-rank p = 0.047; log-rank p = 0.045; respectively). Multivariate Cox regression model indicated that SBA after RA was an independent predictor of MACE (hazard ratio: 0.337; 95% confidence interval: 0.139 to 0.817; p = 0.016). Additional SBA following RA was associated with lower MACE incidence in patients undergoing RA with a small-sized burr.

Abstract TMP15: Thrombolysis in Stroke With Unknown Onset Based on Non-contrast Ct (TRUST CT). An International, Multicenter, Registry-based Study

Background: Thrombolysis in wake-up stroke (WUS) or stroke with unknown onset (SUO) has been recently proven to be safe and effective using magnetic resonance imaging (MRI). However, in most of the thrombolyzing hospitals worldwide MR imaging is not available. We hypothesize that pragmatic non-contrast CT (NCCT)-based WUS/SUO thrombolysis may be feasible and safe. Methods: TRUST-CT is an international multicenter registry-based study. WUS/SUO patients undergoing NCCT-based thrombolysis with NIHSS ≥ 4 and initial ASPECTS ≥ 7 are included and compared to propensity score matched non-thrombolyzed WUS/SUO controls. Primary endpoint is symptomatic intracranial hemorrhage (ICH); secondary endpoints include 24-hour NIHSS change of ≥ 4 and modified Rankin Score (mRS) at 90 days. Results: Until now 100 patients with NCCT-based WUS/SUO thrombolysis were included. As compared to 100 controls, the median admission NIHSS was 9 and 8, respectively; the median ASPECTS was 10 in both groups. 3 (3 %) patients and 0 controls suffered symptomatic ICH. A decrease of ≥ 4 NIHSS points was observed in 48 % of the thrombolyzed patients as compared to 25% in the control group (p = 0.001); 33% and 28% achieved mRS 0 to 1 at 90 days, respectively (p = 0.4). Conclusions: NCCT-based thrombolysis in WUS/SUO seems feasible, safe and eventually effective. Updated results will be presented and discussed in the context of current evidence.

Single- or double-unit UCBT following RIC in adults with AL: a report from Eurocord, the ALWP and the CTIWP of the EBMT

BackgroundThe feasibility of cord blood transplantation (CBT) in adults is limited by the relatively low number of hematopoietic stem/progenitor cells contained in one single CB unit. The infusion of two CB units from different partially HLA-matched donors (double CBT) is frequently performed in patients who lack a sufficiently rich single CB unit.MethodsWe compared CBT outcomes in patients given single or double CBT following reduced-intensity conditioning (RIC) in a retrospective multicenter registry-based study. Inclusion criteria included adult (≥18 years) patients, acute myeloid leukemia (AML) or acute lymphoblastic leukemia (ALL), complete remission (CR) at the time of transplantation, first single (with a cryopreserved TNC ≥ 2.5 × 107/kg) or double CBT between 2004 and 2014, and RIC conditioning.ResultsData from 534 patients with AML (n = 408) or ALL (n = 126) receiving a first single (n = 172) or double (n = 362) CBT were included in the analyses. In univariate analysis, in comparison to patients transplanted with a single CB, double CB recipients had a similar incidence of neutrophil engraftment but a suggestion for a higher incidence of grade II–IV acute GVHD (36 versus 28%, P = 0.08). In multivariate analyses, in comparison to single CBT recipients, double CBT patients had a comparable incidence of relapse (HR = 0.9, P = 0.5) and of nonrelapse mortality (HR = 0.8, P = 0.3), as well as comparable overall (HR = 0.8, P = 0.17), leukemia-free (HR = 0.8, P = 0.2) and GVHD-free, relapse-free (HR = 1.0, P = 0.3) survival.ConclusionsThese data failed to demonstrate better transplantation outcomes in adult patients receiving double CBT in comparison to those receiving single CBT with adequate TNC after RIC.