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Purpose: Pediatric Cardiac Quality of Life Inventory (PCQLI) is a disease-specific pediatric cardiac health-related quality of life (HRQOL) instrument that is reliable, valid, and generalizable. We aim to demonstrate PCQLI responsiveness in children undergoing arrhythmia ablation, heart transplantation, and valve surgery before and after cardiac intervention., Methods: Pediatric cardiac patients 8-18 years of age from 11 centers undergoing arrhythmia ablation, heart transplantation, or valve surgery were enrolled. Patient and parent-proxy PCQLI Total, Disease Impact and Psychosocial Impact subscale scores were assessed pre- and 3-12 months follow-up. Patient clinical status was assessed by a clinician post-procedure and dichotomized into markedly improved/improved and no change/worse/much worse. Paired t-tests examined change over time., Results: We included 195 patient/parent-proxies: 12.6 ± 3.0 years of age; median follow-up time 6.7 (IQR = 5.3-8.2) months; procedural groups - 79 (41%) ablation, 28 (14%) heart transplantation, 88 (45%) valve surgery; clinical status - 164 (84%) markedly improved/improved, 31 (16%) no change/worse/much worse. PCQLI patient and parent-proxies Total scores increased (p ≤ 0.013) in each intervention group. All PCQLI scores were higher (p < 0.001) in the markedly improved/improved group and there were no clinically significant differences in the PCQLI scores in the no difference/worse/much worse group., Conclusion: The PCQLI is responsive in the pediatric cardiac population. Patients with improved clinical status and their parent-proxies reported increased HRQOL after the procedure. Patients with no improvement in clinical status and their parent-proxies reported no change in HRQOL. PCQLI may be used as a patient-reported outcome measure for longitudinal follow-up and interventional trials to assess HRQOL impact from patient and parent-proxy perspectives., (© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.)

Data on the clinical importance of newly detected donor-specific anti-HLA antibodies (ndDSAs) after pediatric heart transplantation are lacking despite mounting evidence of the detrimental effect of de novo DSAs in solid organ transplantation. We prospectively tested 237 pediatric heart transplant recipients for ndDSAs in the first year posttransplantation to determine their incidence, pattern, and clinical impact. One-third of patients developed ndDSAs; when present, these were mostly detected within the first 6 weeks after transplantation, suggesting that memory responses may predominate over true de novo DSA production in this population. In the absence of preexisting DSAs, patients with ndDSAs had significantly more acute cellular rejection but not antibody-mediated rejection, and there was no impact on graft and patient survival in the first year posttransplantation. Risk factors for ndDSAs included common sensitizing events. Given the early detection of the antibody response, memory responses may be more important in the first year after pediatric heart transplantation and patients with a history of a sensitizing event may be at risk even with a negative pretransplantation antibody screen. The impact on late graft and patient outcomes of first-year ndDSAs is being assessed in an extended cohort of patients., (© 2018 The American Society of Transplantation and the American Society of Transplant Surgeons.)

Background: Post-Fontan protein-losing enteropathy (PLE) is associated with significant morbidity and mortality. Although heart transplantation (HTx) can be curative, PLE may increase the risk of morbidity before and after HTx. This study analyzed the influence of PLE influence on waiting list and post-HTx outcomes in a pediatric cohort., Methods: Fontan patients listed for HTx and enrolled in the Pediatric Heart Transplant Study from 1999 to 2012 were stratified by a diagnosis of PLE, and the association of PLE with waiting list and post-HTx mortality, rejection, and infection was analyzed., Results: Compared with non-PLE Fontan patients (n = 260), PLE patients listed for HTx (n = 96) were older (11.9 years vs 7.6 years; p = 0.003), had a larger body surface area (1.1 m(2) vs 0.9 m(2); p = 0.0001), had lower serum bilirubin (0.5 vs 0.9 mg/dl; p = 0.01), lower B-type natriuretic peptide (59 vs 227 pg/ml; p = 0.006), and were less likely to be on a ventilator (3% vs 13%; p = 0.006). PLE patients had lower waiting list mortality than non-PLE Fontan patients (p < 0.0001). There were no intergroup differences for post-HTx survival or times to the first infection or rejection. PLE was not independently associated with increased post-HTx mortality at any time point., Conclusions: In this multicenter cohort, the diagnosis of PLE alone was not associated with increased waiting list mortality or post-HTx morbidity or mortality. Given the limitations of our data, this analysis suggests that PLE patients in the pediatric age group have outcomes similar to their non-PLE counterparts. Additional multicenter studies of PLE patients with targeted collection of PLE-specific information will be necessary to fully delineate the risks conferred by PLE for HTx., (Copyright © 2015 International Society for Heart and Lung Transplantation. Published by Elsevier Inc. All rights reserved.)

Background: Surveillance endomyocardial biopsy (EMB) with right heart catheterization (RHC) is the standard of care for the assessment of post cardiac transplantation rejection. This procedure has traditionally relied upon fluoroscopy, which exposes both patient and staff to the risks of ionizing radiation. These risks may be of particular concern in the transplant patient who must undergo many such procedures lifelong. We present data on a new "ALARA - As Low As Reasonably Achievable" protocol to reduce radiation exposure during the performance of RHC with EMB., Methods: All cardiac transplantation patients < 21 years of age who underwent RHC with EMB at The Children's Hospital at Montefiore from 6/11-12/11 were included. EMB was performed after all right heart pressures including wedge pressure and thermodilution cardiac output were measured. A novel ALARA protocol consisting of multiple features including ultra-low frame rates (2-3 fps), low fluoro dose/frame (10-18 nGy/frame), use of the "air-gap" technique for patients < 20 kg, and multiple other techniques aimed at minimizing use of fluoroscopy were employed in all cases. Demographics, procedural data and patient radiation exposure levels were collected and analyzed., Results: 18 patients underwent 45 surveillance RHC with EMB in the study period and were the subject of this analysis. The mean age was 5.9 ± 6.1 years, weight was 20.4 kg ± 16.6 kg, and BSA was 0.75 ± 45 m(2) . PA fluoroscopy was used exclusively in 45/45. Vascular access was RFV (21/45; 47%), RIJV (17/45; 38%), LFV (4/45; 9%) and LIJV (3/45; 7%). The median number of EMB specimens obtained was 5 (range, 4-7). The median fluoroscopy time was 3.7 min (range, 1.2-9). The median air Kerma product (K) was 1.4 mGy (range, 0.4-14), and dose area product (DAP) was 15.8 uGym(2) (range, 3.5-144.5). The K and DAP are substantially lower than any prior published data for RHC/EMB in this patient group. There were no procedural complications., Conclusions: The use of a novel ALARA protocol for RHC and EMB in pediatric cardiac transplantation patients markedly reduced radiation exposure to levels far below any previously reported values without negatively affecting the safety or efficacy of these procedures., (Copyright © 2013 Wiley Periodicals, Inc.)

Limited options exist for mechanical circulatory support as a bridge to transplantation in pediatric patients with advanced heart failure. This is particularly true when it comes to intracorporeal technologies. We describe the first reported experience with the use of the HVAD in the US as a successful bridge to transplantation in a 13-yr-old patient., (© 2012 John Wiley & Sons A/S.)

This report describes the use of a donor heart with ventricular pre-excitation for pediatric orthotopic heart transplantation and the successful surgical cryoablation of the donor heart prior to transplantation. The issues related to the preoperative evaluation and surgical management of the donor heart with Wolff-Parkinson White syndrome are discussed., (© 2011 The Authors Journal compilation © 2011 The American Society of Transplantation and the American Society of Transplant Surgeons.)

Idiopathic restrictive cardiomyopathy (RCM) is a rare cardiomyopathy in children notable for severe diastolic dysfunction and progressive elevation of pulmonary vascular resistance (PVR). Traditionally, those with pulmonary vascular resistance indices (PVRI) >6 W.U. x m(2) have been precluded from heart transplantation (HTX). The clinical course of all patients transplanted for RCM between 1986 and 2006 were reviewed. Preoperative, intraoperative and postoperative variables were evaluated. A total of 23 patients underwent HTX for RCM, with a mean age of 8.8 +/- 5.6 years and a mean time from listing to HTX of 43 +/- 60 days. Preoperative and postoperative (114 +/- 40 days) PVRI were 5.9 +/- 4.4 and 2.9 +/- 1.5 W.U. x m(2), respectively. At time of most recent follow-up (mean = 5.7 +/- 4.6 years), the mean PVRI was 2.0 +/- 1.0 W.U. x m(2). Increasing preoperative mean pulmonary artery pressure (PA) pressure (p = 0.04) and PVRI > 6 W.U. x m(2) (chi(2)= 7.4, p < 0.01) were associated with the requirement of ECMO postoperatively. Neither PVRI nor mean PA pressure was associated with posttransplant mortality; 30-day and 1-year actuarial survivals were 96% and 86%, respectively. Five of the seven patients with preoperative PVRI > 6 W.U. x m(2) survived the first postoperative year. We report excellent survival for patients undergoing HTX for RCM despite the high proportion of high-risk patients.

Four years prior to transplantation, a 14-year-old boy with severe haemophilia A and a high-responding factor VIII (FVIII) inhibitor developed an anteroseptal myocardial infarct while receiving high doses of an activated prothrombin complex concentrate (PCC). Cardiac transplantation was required for survival because of the ensuing cardiomyopathy. At surgery, the patient's inhibitor titre was 1.8 Bethesda units (BU). High-dose bolus therapy, followed by a continuous infusion of FVIII provided excellent operative and initial postoperative haemostasis without additional blood-product support. Once anamnaesis developed on day 6 postoperatively, recombinant factor VIIa (rFVIIa) therapy was initiated. Haemostasis remained excellent, except for the transient increase in chest-tube bleeding that was noted on day 7. epsilon-Aminocaproic acid was added and haemostasis was re-established. On day 15, rFVIIa was replaced with alternate day infusions of prothrombin complex concentrates (PCCs). On day 21 following the transplant, the patient was discharged, remaining on daily FVIII immune tolerance and thrice-weekly PCC prophylaxis. He remains well 24 months after transplant with an inhibitor titre of 39 BU. This paper describes the second case of cardiac transplantation complicated by haemophilia and an inhibitor, and discusses preoperative planning and operative and postsurgical haemostasis management.

Pulmonary arteriovenous malformations (PAVMs) can occur following caval to pulmonary artery connection, Glenn and/or Fontan procedure, leading to severe cyanosis and exercise intolerance. It is unknown whether these abnormalities regress or persist following heart transplantation (HTx). Twenty patients with failed Fontan or Glenn procedures were screened for PAVMs prior to HTx by contrast echocardiography, selective pulmonary angiography, and pulmonary venous desaturation. Age at transplant, diagnosis, previous operations, time from Glenn to transplant, systemic oxygenation, hemoglobin level, and ventricular function were determined. The clinical course after HTx was characterized in three patients with significant PAVMs. Indications for HTx were exercise intolerance and severe cyanosis in one patient, and cyanosis and ventricular dysfunction in two. Pre-HTx, mean systemic saturation was 67%; mean pulmonary venous wedge saturation was 81%. Post-HTx, oxygen saturations were normal (> 96%) at 14, 40, and 180 days. Contrast echocardiography, performed 1 month to 3.3 yrs after HTx, showed no intrapulmonary shunting in two patients and minimal shunting in one. One patient suffered an embolic stroke from right-to-left shunting post-HTx. All patients are alive and well 35, 71, and 73 months post-HTx. In patients with single ventricle physiology, PAVMs are not an absolute contraindication to HTx. Heart-lung transplant may not be required for these patients.

Background: Advances in surgical and medical management have greatly improved long-term survival rates in patients with congenital heart disease (CHD). As these patients reach adulthood, myocardial dysfunction can occur, leading to cardiac transplantation., Methods and Results: We reviewed the pretransplantation and posttransplantation courses of 24 patients >18 years old (mean age, 26 years; range, 18 to 56 years) with CHD who received a transplant between January 1985 and September 1998. The relation between preoperative and perioperative risk factors for complications and death was assessed. Single ventricle was the pretransplantation diagnosis for 12 patients (50%), and d-transposition of the great vessels was the diagnosis for 4 patients (16%). Twenty-two patients had a mean of 2 previous operations. At cardiac transplantation, additional surgical procedures were required to correct extracardiac lesions in 18 patients (75%). Refractory heart failure was present in 22 patients, significant cyanosis was present in 7, and protein-losing enteropathy was present in 4. There were 5 early deaths due to bleeding (n=3) and infection (n=2). The Kaplan-Meier survival rate after cardiac transplantation was 79% at 1 year and 60% at 5 years. No anatomic or surgical risk factor was predictive of death. The outcome of patients with CHD who received a transplant was compared with that for patients without CHD (n=788). Mean bypass and ischemic times were significantly longer in patients with CHD than in patients without CHD. Survival rates after transplantation did not differ significantly between patients with and those without CHD (P=0.83)., Conclusions: Successful cardiac transplantation is obtainable in adults with complex CHD, with an outcome similar to that of patients without CHD. A detailed assessment of cardiac anatomy and careful surgical planning are essential to the pretransplantation and posttransplantation management of these patients.

This review details the indications for heart transplantation in children. Contraindications have evolved from absolute to relative. Controversial issues remain and this paper represents a consensus of more than a dozen centers that have programs that remain active performing pediatric heart transplants.

We have shown that sensitized and non-sensitized pediatric heart transplant (PHTx) recipients had similar 1 year patient and graft survival (CTOTC-04 study). In this follow-up study (CTOTC-09), we assess the impact of 'preformed' (at Tx) and first year newly detected donor specific antibody (ndDSA) on allograft function at 3 years. Consecutive children listed for Tx at 9 centers were prospectively enrolled. Management of sensitization, immunosuppression and rejection surveillance were standardized. The primary endpoint was PCWP at 3 years. Secondary endpoints included other hemodynamics, ejection fraction (EF), BNP, graft and patient survival and rejection. DSA was defined by single antigen Luminex testing with ≥1 antibody specific towards donor HLA antigens with MFI>1000. Of 407 enrolled, 370 achieved Tx (mean 8.7 years). Sensitization status at Tx: non-sensitized (n=169, 46%), sensitized/no DSA (n= 120, 32%), sensitized/DSA (n=76, 21%); 5 (1%) unknown. Subjects with and without early DSA (preformed and/or first year ndDSA) had similar PCWP at 3 years (table). There were no significant differences between the 2 groups for: other hemodynamics (PA, RA, RVEDP, LVEDP; all P > 0.08); cardiac index (P= 0.315); EF (P=0.682); and BNP (P=0.900). Subanalysis of the 3 sensitization groups at Tx stratified by presence/absence of first year ndDSA showed no significant impact of ndDSA on PCWP, EF or BNP (all P > 0.1). Graft and patient survival did not differ between groups, though freedom from all acute rejection types was inferior in subjects with DSA at Tx. Sensitization status at Tx and development of first year ndDSA are not associated with worse allograft function or inferior survival at 3 years. Ongoing follow-up, including assessment of CAD, is in progress. This information is critical for defining Tx strategies and the long-term impact of DSA on graft outcomes. [ABSTRACT FROM AUTHOR]