Superior cavopulmonary anastomosis timing and outcomes in infants with single ventricle.

OBJECTIVES: We sought to identify factors associated with the timing and surgical outcomes of the superior cavopulmonary anastomosis. METHODS: The Pediatric Heart Network's Infant Single Ventricle trial database identified participants who underwent superior cavopulmonary anastomosis. Factors potentially associated with age at superior cavopulmonary anastomosis, length of stay and death by 14 months of age were evaluated. Factors included subject demographics, cardiac anatomy, measures from neonatal hospitalization and pre-superior cavopulmonary anastomosis visit, adverse events, echocardiographic variables, intraoperative variables, superior cavopulmonary anastomosis type, and number of concurrent cardiac surgical procedures. Age at superior cavopulmonary anastomosis was analyzed using Cox proportional hazards regression. Natural log length of stay was analyzed by multiple linear regression. RESULTS: Superior cavopulmonary anastomosis was performed in 193 subjects at 5.2 months of age (interquartile range, 4.2, 6.2) and weight of 5.9 kg (interquartile range, 5.3, 6.6). The median length of stay was 7 days (interquartile range, 6, 10). There were 3 deaths and 1 transplant during the superior cavopulmonary anastomosis hospitalization, and 3 deaths and 3 transplants between discharge and 14 months of age. Age at superior cavopulmonary anastomosis was associated with center and interstage adverse events. A longer length of stay was associated with younger age and greater case complexity. Superior cavopulmonary anastomosis type, valve regurgitation, ventricular ejection fraction, and ventricular end-diastolic pressure were not independently associated with age at superior cavopulmonary anastomosis or the length of stay. CONCLUSIONS: Greater case complexity and more frequent interstage adverse events are associated with an earlier age at superior cavopulmonary anastomosis. Significant variation in age at superior cavopulmonary anastomosis among centers, independent of subject factors, highlights a lack of consensus regarding the optimal timing. Factors associated with length of stay could offer insights for improving presuperior cavopulmonary anastomosis care and surgical outcome.

Interstage mortality after the Norwood procedure: Results of the multicenter Single Ventricle Reconstruction trial.

OBJECTIVE: For infants with single ventricle malformations undergoing staged repair, interstage mortality is reported at 2% to 20%. The Single Ventricle Reconstruction trial randomized subjects with a single morphologic right ventricle undergoing a Norwood procedure to a modified Blalock-Taussig shunt (MBTS) or a right ventricle-to-pulmonary artery shunt (RVPAS). The aim of this analysis was to explore the associations of interstage mortality and shunt type, and demographic, anatomic, and perioperative factors. METHODS: Participants in the Single Ventricle Reconstruction trial who survived to discharge after the Norwood procedure were included (n = 426). Interstage mortality was defined as death postdischarge after the Norwood procedure and before the stage II procedure. Univariate analysis and multivariable logistic regression were performed adjusting for site. RESULTS: Overall interstage mortality was 50 of 426 (12%)-13 of 225 (6%) for RVPAS and 37 of 201 (18%) for MBTS (odds ratio [OR] for MBTS, 3.4; P < .001). When moderate to severe postoperative atrioventricular valve regurgitation (AVVR) was present, interstage mortality was similar between shunt types. Interstage mortality was independently associated with gestational age less than 37 weeks (OR, 3.9; P = .008), Hispanic ethnicity (OR, 2.6; P = .04), aortic atresia/mitral atresia (OR, 2.3; P = .03), greater number of post-Norwood complications (OR, 1.2; P = .006), census block poverty level (P = .003), and MBTS in subjects with no or mild postoperative AVVR (OR, 9.7; P < .001). CONCLUSIONS: Interstage mortality remains high at 12% and is increased with the MBTS compared with the RVPAS if postoperative AVVR is absent or mild. Preterm delivery, anatomic, and socioeconomic factors are also important. Avoiding preterm delivery when possible and close surveillance after Norwood hospitalization for infants with identified risk factors may reduce interstage mortality.

Minimal clinically important differences in the vaginal insertion and successful intercourse items of the sexual encounter profile.

INTRODUCTION: Despite adoption of the successful vaginal insertion (Q2) and intercourse (Q3) items of the sexual encounter profile (SEP) as end points in clinical trials, there are no objective data on what constitute minimal clinically important differences (MCIDs) in these items. AIM: The objective was to estimate the MCID for SEP Q2 and Q3. METHODS: Using data from 17 randomized, controlled trials of the phosphodiesterase type 5 inhibitor tadalafil, we estimated MCIDs for the SEP using anchor-based approaches. The 17 studies included 3,345 patients treated for 12 weeks. The anchor for the MCID is the minimal improvement measure calculated using change from baseline to 12 weeks on the following question: "Over the past 4 weeks, when you attempted sexual intercourse how often was it satisfactory for you?" MCIDs were developed using analysis of variance- and receiver operating characteristic (ROC)-based methods in a subset of studies (N = 11) by comparing patients with and without minimal improvement (N = 863). MCIDs were validated in the remaining six studies (N = 377). MAIN OUTCOME MEASURES: The main outcome measures of this study are SEP Q2 and Q3. RESULTS: Using the ROC-based approach, the MCID for SEP Q2 was 21.4%, with estimated sensitivity of 0.55 and specificity of 0.73; the MCID for SEP Q3 was 23.0%, with estimated sensitivity of 0.72 and specificity of 0.78. MCIDs for SEP Q2/Q3 varied significantly (P < 0.001) according to baseline erectile dysfunction (ED) severity. MCIDs distinguished between patients in the validation sample classified as no change or minimally improved in each ED etiology, ED duration, and age group, but less well across geographic regions. CONCLUSIONS: The contextualization of treatment-related changes into clinically relevant terms is essential to understanding treatment efficacy, interpreting results across studies, and for effective patient management. Overall, there was a better balance between sensitivity and specificity of the MCIDs using the ROC-based approach for the SEP intercourse success item than for the vaginal insertion item.

Minimal clinically important differences in the erectile function domain of the International Index of Erectile Function scale.

BACKGROUND: Despite widespread adoption of the six-item erectile function (EF) domain of the International Index of Erectile Function (IIEF) as a clinical trial end point, there are currently no objective data on what constitutes a minimal clinically important difference (MCID) in the EF domain. OBJECTIVE: Estimate the MCID for the IIEF EF domain. DESIGN, SETTING, AND PARTICIPANTS: Anchor-based MCIDs were estimated using data from 17 randomized, double-blind, placebo-controlled, parallel-group clinical trials of the phosphodiesterase type 5 inhibitor (PDE5-I) tadalafil for 3345 patients treated for 12 wk. MEASUREMENTS: The anchor for the MCID is the minimal improvement measure calculated using change from baseline to 12 wk on IIEF question 7: "Over the past 4 weeks, when you attempted sexual intercourse how often was it satisfactory for you?" MCIDs were developed using analysis of variance (ANOVA)- and receiver operating characteristic (ROC)-based methods in a subset of studies (n=11) by comparing patients with and without minimal improvement (n=863). MCIDs were validated in the remaining six studies (n=377). RESULTS AND LIMITATIONS: The ROC-based MCID for the EF domain was 4, with estimated sensitivity and specificity of 0.74 and 0.73, respectively. MCIDs varied significantly (p<0.0001) according to baseline ED severity (mild: 2; moderate: 5; severe: 7). MCIDs consistently distinguished between patients in the validation sample classified as no change or minimally improved overall and by geographic region, ED etiology, and age group. MCIDs did not differ by age group, geographic region, or ED etiology. Current analyses were based on 17 clinical trials of tadalafil. Results need to be replicated in studies using other PDE5-Is or in nonpharmacologic intervention studies. CONCLUSIONS: The contextualization of treatment-related changes in terms of clinically relevant improvement is essential to understanding treatment efficacy, to interpreting results across studies, and to managing patients effectively. This analysis provides, for the first time, anchor-based estimates of MCIDs in the EF domain score of the IIEF.