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There remains high morbidity and mortality with mechanical circulatory support (MCS) in failing bidirectional Glenn (BDG) physiology. We performed a retrospective analysis of children with BDG physiology supported with MCS before and after 2018. Fourteen patients met inclusion criteria (median age 1.5 years, weight 9 kg). Prior to 2018 (n = 7), with variable anticoagulation and strategies including pulsatile VAD, continuous flow VAD, and extracorporeal membrane oxygenation (ECMO), 3 (43%) of patients were transplanted with a total of 536 patient-days of support (median 59 days). Major hemocompatability-related adverse event (MHRAE) rate was 63 per 100 patient-months. After 2018 (n = 7), using a staged support strategy (ECMO to pulsatile VAD) and bivalirudin anticoagulation, 5 (71%) patients were transplanted with a total of 1260 patient-days of support (median 188 days) and MHRAE rate of 24 per 100 patient-months. Despite challenging physiology, we have observed improved survival and reduced MHRAE despite longer support duration.
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BACKGROUND: As more pediatric patients become candidates for heart transplantation (HT), understanding pathological predictors of outcome and the accuracy of the pretransplantation evaluation are important to optimize utilization of scarce donor organs and improve outcomes. The authors aimed to investigate explanted heart specimens to identify pathologic predictors that may affect cardiac allograft survival after HT. METHODS: Explanted pediatric hearts obtained over an 11-year period were analyzed to understand the patient demographics, indications for transplant, and the clinical-pathological factors. RESULTS: In this study, 149 explanted hearts, 46% congenital heart defects (CHD), were studied. CHD patients were younger and mean pulmonary artery pressure and resistance were significantly lower than in cardiomyopathy patients. Twenty-one died or underwent retransplantation (14.1%). Survival was significantly higher in the cardiomyopathy group at all follow-up intervals. There were more deaths and the 1-, 5- and 7-year survival was lower in patients ≤10 years of age at HT. Early rejection was significantly higher in CHD patients exposed to homograft tissue, but not late rejection. Mortality/retransplantation rate was significantly higher and allograft survival lower in CHD hearts with excessive fibrosis of one or both ventricles. Anatomic diagnosis at pathologic examination differed from the clinical diagnosis in eight cases. CONCLUSIONS: Survival was better for the cardiomyopathy group and patients >10 years at HT. Prior homograft use was associated with a higher prevalence of early rejection. Ventricular fibrosis (of explant) was a strong predictor of outcome in the CHD group. We presented several pathologic findings in explanted pediatric hearts.
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Rechazo de Injerto , Supervivencia de Injerto , Cardiopatías Congénitas , Trasplante de Corazón , Humanos , Niño , Masculino , Femenino , Preescolar , Lactante , Adolescente , Cardiopatías Congénitas/cirugía , Cardiopatías Congénitas/patología , Rechazo de Injerto/patología , Rechazo de Injerto/epidemiología , Estudios Retrospectivos , Resultado del Tratamiento , Estudios de Seguimiento , Cardiomiopatías/cirugía , Cardiomiopatías/patología , Reoperación , Recién Nacido , Análisis de SupervivenciaRESUMEN
BACKGROUND: Pediatric heart transplant (HT) candidates experience high waitlist mortality due to a limited donor pool that is constrained in part by anti-HLA sensitization. We evaluated the impact of CDC and Flow donor-specific crossmatch (XM) results on pediatric HT outcomes. METHODS: All pediatric HTs between 1999 and 2019 in the OPTN database were included. Donor-specific XM results were sub-categorized based on CDC and Flow results. Primary outcomes were treated rejection in the first year and time to death or allograft loss. Propensity scores were utilized to adjust for differences in baseline characteristics. RESULTS: A total of 4,695 pediatric HT patients with T-cell XM data were included. After propensity score adjustment, a positive T-cell CDC-XM was associated with 2 times higher odds of treated rejection (OR 2.29 (1.56, 3.37)) and shorter time to death/allograft loss (HR 1.50 (1.19, 1.88)) compared to a negative Flow-XM. HT recipients who were Flow-XM positive with negative/unknown CDC-XM did not have higher odds of rejection or shorter time to death/allograft loss. An isolated positive B-cell XM was also not associated with worse outcomes. Over the study period XM testing shifted from CDC- to Flow-based assays. CONCLUSIONS: A positive donor-specific T-cell CDC-XM was associated with rejection and death/allograft loss following pediatric HT. This association was not observed with a positive T-cell Flow-XM or B-cell XM result alone. The shift away from performing the CDC-XM may result in loss of important prognostic information unless the clinical relevance of quantitative Flow-XM results on heart transplant outcomes is systematically studied.
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Rechazo de Injerto , Supervivencia de Injerto , Trasplante de Corazón , Humanos , Niño , Masculino , Femenino , Rechazo de Injerto/inmunología , Rechazo de Injerto/epidemiología , Preescolar , Estudios Retrospectivos , Prueba de Histocompatibilidad , Adolescente , Lactante , Donantes de TejidosRESUMEN
BACKGROUND: Short-term outcomes using steroid avoidance immune suppression are encouraging in pediatric heart transplant (HT) recipients at low risk of antibody-mediated rejection. We assessed medium-term outcomes in pediatric HT recipients initiated on a steroid avoidance protocol at our institution using surveillance biopsies. METHODS: All primary HT recipients during 2006-2020 who did not have a donor-specific antibody were eligible for immune suppression consisting of 5-d Thymoglobulin/steroid induction followed by a tacrolimus-based, steroid-free regimen. We assessed freedom from graft failure (death or retransplant), acute rejection, posttransplant lymphoproliferative disease, and cardiac allograft vasculopathy. RESULTS: Overall, 150 of 181 primary HT recipients were eligible for steroid avoidance regimen. Their median age was 8.7 y, 41% had congenital heart disease, 23% were sensitized, and 35% were on a mechanical support. The median follow-up was 6.1 y. Eleven patients (8%) were on maintenance steroids at discharge and 13% at 1 y. Graft survival was 94% at 1 y and 87% at 5 y. Freedom from rejection was 73% at 1 y and 64% at 5 y. Freedom from posttransplant lymphoproliferative disease was 96% at 1 y and 95% at 5 y. Freedom from moderate cardiac allograft vasculopathy was 94% at 5 y. Eight patients developed diabetes. Estimated glomerular filtration rate was <60 mL/min/1.73 m 2 in 5% of the cohort at 5 y. CONCLUSIONS: Pediatric HT recipients at low risk of antibody-mediated rejection have excellent medium-term survival and relatively low incidence of posttransplant morbidities when managed using a steroid avoidance immune suppression protocol.
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Trasplante de Corazón , Inmunosupresores , Humanos , Niño , Inmunosupresores/efectos adversos , Terapia de Inmunosupresión/métodos , Esteroides , Tacrolimus/efectos adversos , Anticuerpos , Trasplante de Corazón/efectos adversos , Rechazo de Injerto , Supervivencia de InjertoAsunto(s)
Enfermedad Coronaria , Cardiopatías , Trasplante de Corazón , Adulto , Humanos , Niño , Iluminación , Enfermedad Coronaria/diagnóstico , AloinjertosRESUMEN
Patients who have undergone hematopoietic stem cell transplantation (HSCT) in childhood have a higher risk of diastolic heart failure (HF). The rate of progression of diastolic dysfunction in aging pediatric patients is unknown and is more difficult to assess in young patients secondary to changes in diastolic indices as they grow. HSCT recipients at our center were previously found to have decline in diastolic function indices at 1 year after HSCT. This study provides follow-up of this cohort, using age-normalized z-scores to assess whether the decline in diastolic function noted at 1-year post-HSCT persists, worsens, or improves over time. Patients age <21 years who underwent HSCT at Boston Children's Hospital/Dana-Farber Cancer Center between 2005 and 2008 with ≥3 surveillance echocardiograms, including 1 performed pre-HSCT, were included. Diastolic measures included mitral inflow (E/A ratio) and Doppler tissue imaging of left ventricular lateral wall (LV lateral e'), LV septal wall (septal e') and right ventricular free wall (RV e'). Systolic function was measured by LV ejection fraction (LVEF). Normalization by age was done using z-scores, and >±2 SD was defined as abnormal in linear modeling of diastolic dysfunction and systolic dysfunction over time. In a subset of patients with adequate post-HSCT images of the entire left atrium (LA), LA volume and LA strain analyses also were performed. The study cohort comprised 61 patients (41% female; median age at HSCT, 10.7 years; median follow-up, 7.4 years). Diastolic index z-scores declined by -.045/year for LV lateral e', -.06/year for LV septal e', and -.14/year for RV e' (P < .01). The E/A ratio z-score increased by .034/year (P = .028). Linear modeling demonstrated that LV lateral e' and LV septal e' would become abnormal at 25 and 20 years post-HSCT, respectively, whereas RV e' would become abnormal sooner, at 12.6 years. LVEF z-score declined by -.04/year (P < .01) and was estimated to become abnormal at 40 years post-HSCT. Exposure to total body irradiation (TBI) was associated with worsening diastolic indices, lower LVEF (P ≤ .002), and decreased LA reservoir strain (42.0% versus 45.0%; P = .016) and conduit strain (-31.5% versus -35.1%; P = .029), although there was significant overlap between TBI and anthracycline exposure. Treatment with anthracyclines even at low doses (median, 150 mg/m2) was associated with declining LVEF but not with changes in diastolic indices. Long-term survivors of childhood HSCT exhibit declines in both LV and RV diastolic function indices. These results inform the rate of progression of LV and RV diastolic dysfunction indices over time in long-term survivors of pediatric HSCT. A significant association was observed between TBI and diastolic dysfunction and a decline in LVEF. Treatment with anthracyclines even at low doses was associated with a mild decline in LVEF. Our results can inform a lifespan perspective on disease management in this population, encourage clinicians and patients to be vigilant in following guideline-directed surveillance echocardiography, and inform anticipatory responses by clinicians as patients transition from pediatric care to adult care.
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Atrios Cardíacos , Trasplante de Células Madre Hematopoyéticas , Adulto , Humanos , Niño , Femenino , Adulto Joven , Masculino , Estudios de Seguimiento , Sobrevivientes , Trasplante de Células Madre Hematopoyéticas/efectos adversos , AntraciclinasRESUMEN
STUDY OBJECTIVE: The immunosuppressant tacrolimus is a first-line agent to prevent graft rejection following pediatric heart transplant; however, it suffers from extensive inter-patient variability and a narrow therapeutic window. Personalized tacrolimus dosing may improve transplant outcomes by more efficiently achieving and maintaining therapeutic tacrolimus concentrations. We sought to externally validate a previously published population pharmacokinetic (PK) model that was constructed with data from a single site. DATA SOURCE: Data were collected from Seattle, Texas, and Boston Children's Hospitals, and assessed using standard population PK modeling techniques in NONMEMv7.2. MAIN RESULTS: While the model was not successfully validated for use with external data, further covariate searching identified weight (p < 0.0001 on both volume and elimination rate) as a model-significant covariate. This refined model acceptably predicted future tacrolimus concentrations when guided by as few as three concentrations (median prediction error = 7%; median absolute prediction error = 27%). CONCLUSION: These findings support the potential clinical utility of a population PK model to provide personalized tacrolimus dosing guidance.
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Trasplante de Corazón , Trasplante de Riñón , Niño , Humanos , Tacrolimus , Modelos Biológicos , InmunosupresoresRESUMEN
BACKGROUND: Fecal microbiota transplant (FMT) is an effective treatment for recurrent Clostridioides difficile infection (CDI). Safety concerns around FMT are increased in immunocompromised populations, such as solid organ transplant (SOT) recipients. Outcomes among adult SOT recipients suggest FMT is efficacious and safe; however, pediatric SOT data are lacking. METHODS: We describe the efficacy and safety of FMT among pediatric SOT recipients in a single-center retrospective study from March 2016 to December 2019. Successful FMT was defined as no recurrence of CDI within 2 mo of FMT. We identified 6 SOT recipients ages 4-18 y who received FMT a median of 5.3 y post-SOT. RESULTS: Success after a single FMT was 83.3%. One liver recipient did not achieve cure after 3 FMTs and remains on low-dose vancomycin. One serious adverse event (SAE) occurred; cecal perforation and bacterial peritonitis occurred following colonoscopic FMT coordinated with intestinal biopsy in a kidney transplant recipient. He achieved full recovery and CDI cure. There were no other SAEs. There were no adverse events related to immunosuppression or transplantation status including: bacteremia, cytomegalovirus activation or reactivation, allograft rejection, or allograft loss. CONCLUSIONS: In this limited series, efficacy of FMT in pediatric SOT is comparable to efficacy in the general pediatric recurrent CDI population. There may be an increased risk of procedure-related SAE in SOT patients and larger cohort studies are needed.
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Clostridioides difficile , Infecciones por Clostridium , Trasplante de Órganos , Masculino , Adulto , Humanos , Niño , Trasplante de Microbiota Fecal/efectos adversos , Estudios Retrospectivos , Resultado del Tratamiento , Infecciones por Clostridium/epidemiología , Receptores de Trasplantes , Trasplante de Órganos/efectos adversosRESUMEN
BACKGROUND: Currently there are no immunosuppression regimens FDA-approved to prevent rejection in pediatric heart transplantation (HT). In recent years, everolimus (EVL) has emerged as a potential alternative to standard tacrolimus (TAC) as the primary immunosuppressant to prevent rejection that may also reduce the risk of cardiac allograft vasculopathy (CAV), chronic kidney disease (CKD) and cytomegalovirus (CMV) infection. However, the 2 regimens have never been compared head-to-head in a randomized trial. The study design and rationale are reviewed in light of the challenges inherent in rare disease research. METHODS: The TEAMMATE trial (IND 127980) is the first multicenter randomized clinical trial (RCT) in pediatric HT. The primary purpose is to evaluate the safety and efficacy of EVL and low-dose TAC (LD-TAC) compared to standard-dose TAC and mycophenolate mofetil (MMF). Children aged <21 years at HT were randomized (1:1 ratio) at 6 months post-HT to either regimen, and followed for 30 months. Children with recurrent rejection, multi-organ transplant recipients, and those with an estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2 were excluded. The primary efficacy hypothesis is that, compared to TAC/MMF, EVL/LD-TAC is more effective in preventing 3 MATEs: acute cellular rejection (ACR), CKD and CAV. The primary safety hypothesis is that EVL/LD-TAC does not have a higher cumulative burden of 6 MATEs (antibody mediated rejection [AMR], infection, and post-transplant lymphoproliferative disorder [PTLD] in addition to the 3 above). The primary endpoint is the MATE score, a composite, ordinal surrogate endpoint reflecting the frequency and severity of MATEs that is validated against graft loss. The study had a target sample size of 210 patients across 25 sites and is powered to demonstrate superior efficacy of EVL/LD-TAC. Trial enrollment is complete and participant follow-up will be completed in 2023. CONCLUSION: The TEAMMATE trial is the first multicenter RCT in pediatric HT. It is anticipated that the study will provide important information about the safety and efficacy of everolimus vs tacrolimus-based regimens and will provide valuable lessons into the design and conduct of future trials in pediatric HT.
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Cardiopatías , Trasplante de Corazón , Trasplante de Riñón , Insuficiencia Renal Crónica , Humanos , Niño , Tacrolimus/uso terapéutico , Tacrolimus/farmacología , Everolimus/farmacología , Ácido Micofenólico/uso terapéutico , Ácido Micofenólico/farmacología , Trasplante de Riñón/efectos adversos , Inmunosupresores/uso terapéutico , Inmunosupresores/farmacología , Insuficiencia Renal Crónica/etiología , Cardiopatías/etiología , Quimioterapia Combinada , Supervivencia de InjertoRESUMEN
There is a growing population of pediatric and adult patients supported with the HeartMate 3 ventricular assist device (HM3 VAD) all of whom require anticoagulation. Apixaban is an anticoagulant requiring less testing than warfarin which has been shown to be effective in other indications. We report five pediatric and young adult patients managed on HM3 VAD with apixaban anticoagulation for 1589 days of VAD support between January 6, 2019 and January 7, 2022. The median age was 17 years and the weight was 69 kg. Four patients had congenital heart disease (2 single-ventricle Fontan circulation, and 2 biventricular circulations) and one had dilated cardiomyopathy. Apixaban was initiated at a median of 7 days postoperatively and doses were titrated based on peak apixaban-specific anti-Xa chromogenic analysis levels (goal 150-250 ng/ml). All patients received aspirin 81 mg daily. There was one major hemocompatibility-related event observed (outflow graft thrombus in the setting of medication nonadherence and chronic VAD infection); there was no major bleeding, death, or stroke. Three patients underwent heart transplantation and two remain on VAD support. In this limited series, apixaban paired with a level-based dosing regimen and low-dose aspirin provided safe and effective antithrombosis with only one hemocompatibility-related event related to medication non-adherence.
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Insuficiencia Cardíaca , Corazón Auxiliar , Adolescente , Niño , Humanos , Adulto Joven , Anticoagulantes/efectos adversos , Aspirina , Insuficiencia Cardíaca/cirugía , Estudios Retrospectivos , Resultado del TratamientoRESUMEN
BACKGROUND: In order to improve transparency within the patient selection process, a transplant listing advisory committee was formed within the Boston Children's Hospital Pediatric Transplant Center. Its mission is to promote equity in access to organ transplantation by ensuring that the institutional transplant selection criteria are fair, unbiased, and nondiscriminatory. The committee conducts comprehensive case and data review of individual characteristics and reviews in aggregate to identify potential systems bias. METHODS: Charts for 256 patients evaluated for transplant from 3/2016 to 3/2019 were reviewed. Among these, 64 (25%) patients were declined for transplant. Univariate logistic regression analysis was used to identify demographic variables and vulnerable status factors associated with being declined. Odds ratios (OR) are reported. RESULTS: Among all patients, median age was 8.5 years and 58% were male. Asian patients were more likely to be declined than White patients (OR = 5.3, Wald p = .007). Socioeconomic factors that affected likelihood of listing decline included concerns for caregivers' ability to manage and understand care requirements (OR = 3.8, p = .011), caregiver employment status (OR = 1.9, p = .042), and use of public assistance programs (OR = 2.2, p = .05). Patients with severe neurodevelopmental delay were more likely to be declined for listing (OR = 3.7, p = .019). CONCLUSION: This analysis identified areas of potential bias related to race, socioeconomic status, and neurodevelopmental delay where initiatives can be targeted. Advisory committees are an important aspect of evaluating equity in transplant center selection policy and practice.
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Trasplante de Órganos , Listas de Espera , Humanos , Masculino , Niño , Femenino , Factores Socioeconómicos , Clase Social , EmpleoRESUMEN
BACKGROUND: Cardiac allograft vasculopathy (CAV) is a leading cause of long-term morbidity and mortality in pediatric heart transplant (HTx) recipients. Exercise stress echocardiography (ESE) has been shown to be useful in the detection of angiographically confirmed coronary artery disease in children. However, the prognostic utility of ESE for prediction of cardiac events in HTx survivors is unknown. OBJECTIVES: We aim to assess whether an abnormal (positive) ESE is be associated with a higher risk of future cardiovascular (CV) outcomes in pediatric HTx recipients. METHODS: We conducted a retrospective review of CV outcomes in a cohort of 95 pediatric HTx recipients who underwent 188 ESEs over a 10-year period. A composite endpoint for CV events including myocardial infarction, hospitalization for nonrejection heart failure, coronary revascularization, need for repeat transplantation, and death was used. Based on the interpretation of the ESE results, each ESE study was classified for this study as either positive (abnormal) or negative (normal) for ischemia. Results of the coronary angiograms performed near the time of ESE were also assessed and classified for this study as positive (abnormal) or negative (normal) for CAV according to standard HTx criteria for CAV. RESULTS: Fifty-one (27%) ESEs were positive for ischemia. There was a total of 35 CV events in 23 patients. A positive ESE was associated with increased risk of any CV event (hazard ratio = 3.55; 95% CI, 1.52, 8.28), as well as an increased risk of CV death (hazard ratio = 3.19; 95% CI, 1.23, 8.28). Freedom from composite CV outcome at 1, 2, and 3 years following a positive ESE was 89.9% (95% CI = 77.3%, 95.7%), 81.5% (95% CI = 65.9%, 90.5%), and 63.2% (95% CI = 41.9%, 78.5%), respectively. Freedom from composite CV outcome at 1, 2, and 3 years following a negative ESE was 99.3% (94.8, 99.9), 98.4% (93.6, 99.6), and 97.0% (90.6, 99.1), respectively. No patient died within 1 year of a negative ESE. CONCLUSIONS: In this largest study of ESE in pediatric HTx recipients, a positive or abnormal ESE is associated with increased future CV morbidity and mortality. Conversely, a negative ESE can help predict CV event-free survival. Even in the setting of a normal coronary angiogram, our pilot data show that an abnormal ESE may still be clinically important. Use of ESE in follow-up may improve risk stratification and management of pediatric HTx recipients.
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Enfermedad de la Arteria Coronaria , Cardiopatías , Trasplante de Corazón , Humanos , Niño , Ecocardiografía de Estrés/métodos , Pronóstico , Trasplante de Corazón/efectos adversos , Angiografía Coronaria , Enfermedad de la Arteria Coronaria/diagnóstico , Enfermedad de la Arteria Coronaria/etiología , Cardiopatías/etiologíaAsunto(s)
Varicela , Trasplante de Corazón , Varicela/prevención & control , Niño , Herpesvirus Humano 3 , Humanos , Receptores de Trasplantes , VacunaciónRESUMEN
We describe waiting times for pediatric heart transplant (HT) candidates after the 2016 revision to the US allocation policy. The OPTN database was queried for pediatric HT candidates listed between 7/2016 and 4/2019. Of the 1789 included candidates, 65% underwent HT, 14% died/deteriorated, 8% were removed for improvement, and 13% were still waiting at the end of follow-up. Most candidates were status 1A at HT (81%). Median wait times differ substantially by listing status, blood type, and recipient weight. The likelihood of HT was lower in candidates <25 kg and in those with blood type O; The <25 kg, blood type O subgroup experiences longer wait times and higher wait list mortality. For status 1A candidates, median wait times were 108 days (≤25 kg, blood type O), 80 days (≤25 kg, non-O), 47 days (>25 kg, O), and 24 days (>25 kg, non-O). We found that centers with more selective organ acceptance practices, based on a lower median Pediatric Heart Donor Assessment Tool (PH-DAT) score for completed transplants, experience longer status 1A wait times for their listed patients. These data can be used to counsel families and to select appropriate advanced heart failure therapies to support patients to transplant.
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Cardiopatías Congénitas , Trasplante de Corazón , Obtención de Tejidos y Órganos , Niño , Humanos , Políticas , Donantes de Tejidos , Estados Unidos , Listas de EsperaRESUMEN
BACKGROUND: Fontan physiology results in multiorgan dysfunction, most notably affecting the liver and kidney. We evaluated the utility of Model for End-Stage Liver Disease Excluding INR (MELD-XI) score, a score evaluating the function of both liver and kidney to identify Fontan patients at increased risk for morbidity and mortality post-heart transplant. METHODS: The Pediatric Heart Transplant Society database was queried to identify Fontan patients listed for heart transplant between January 2005 and December 2018. MELD-XI scores were calculated at listing and heart transplant. A multivariable analysis was conducted to identify risk factors for post-heart transplant mortality. Demographic, clinical characteristics, and survival differences were evaluated and compared between the high and low MELD-XI score cohorts. The impact of changing MELD-XI scores during the waitlist period on post-heart transplant outcomes was also evaluated. RESULTS: Of 565 Fontan patients who underwent transplantation, 524 (93%) had calculable MELD-XI scores at the time of heart transplant: 421 calculable at listing and 392 calculable at listing and at heart transplant. On multivariable analysis, only MELD-XI score (squared) (hazard ratio, 1.007), history of protein-losing enteropathy (hazard ratio, 2.1), and ventricular assist device use at transplant (hazard ratio, 3.4) were risk factors for early phase post-heart transplant mortality. Patients with high MELD-XI scores at heart transplant had inferior survival post-heart transplant (P = .02); those in the high MELD-XI score cohort at wait listing and heart transplant tend to have the worst post-heart transplant survival; however, this was not significant (P = .42). CONCLUSIONS: The MELD-XI, an easily calculated score, serves as a valuable aid in identifying pediatric Fontan patients at increased risk for post-heart transplant mortality.
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Trasplante de Corazón/mortalidad , Modelos Estadísticos , Adolescente , Niño , Preescolar , Femenino , Procedimiento de Fontan , Cardiopatías Congénitas/cirugía , Corazón Auxiliar , Humanos , Masculino , Enteropatías Perdedoras de Proteínas/mortalidad , Factores de RiesgoRESUMEN
Giant cell myocarditis (GCM) is a form of fulminant myocarditis that is rapidly progressive and frequently lethal even in children. Over the course of 20 years, a definitive histopathologic diagnosis of GCM has been made at our institution in only two pediatric patients, and in neither instance was the diagnosis of GCM rendered on initial cardiac biopsy. We present the two patients and highlight the similarities in their clinical presentation and their challenging and inconclusive- albeit histologically similar- initial cardiac biopsy findings.
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Trasplante de Corazón , Enfermedades del Sistema Inmune , Miocarditis , Biopsia , Niño , Células Gigantes/patología , Corazón , Humanos , Enfermedades del Sistema Inmune/patología , Miocarditis/diagnóstico , Miocarditis/patologíaAsunto(s)
Aneurisma Cardíaco/complicaciones , Ventrículos Cardíacos/diagnóstico por imagen , Ventrículos Cardíacos/fisiopatología , Miocarditis/complicaciones , Inmunodeficiencia Combinada Grave/complicaciones , Cateterismo Cardíaco/métodos , Cardiotónicos/uso terapéutico , Preescolar , Electrocardiografía/métodos , Células Gigantes , Aneurisma Cardíaco/terapia , Humanos , Imagen por Resonancia Magnética/métodos , Masculino , Milrinona/uso terapéutico , Miocarditis/tratamiento farmacológicoRESUMEN
AIM: To describe the clinical and hemodynamic characteristics of Fontan failure in children listed for heart transplant. METHODS: In a nested study of the Pediatric Heart Transplant Society, 16 centers contributed information on Fontan patients listed for heart transplant between 2005and 2013. Patients were classified into four mutually exclusive phenotypes: Fontan with abnormal lymphatics (FAL), Fontan with reduced systolic function (FRF), Fontan with preserved systolic function (FPF), and Fontan with "normal" hearts (FNH). Primary outcome was waitlist and post-transplant mortality. RESULTS: 177 children listed for transplant were followed over a median 13 (IQR 4-31) months, 84 (47%) were FAL, 57 (32%) FRF, 22 (12%) FNH, and 14 (8%) FPF. Hemodynamic characteristics differed between the 4 groups: Fontan pressure (FP) was most elevated with FPF (median 22, IQR 18-23, mmHg) and lowest with FAL (16, 14-20, mmHg); cardiac index (CI) was lowest with FRF (2.8, 2.3-3.4, L/min/m2). In the entire cohort, 66% had FP >15 mmHg, 21% had FP >20 mmHg, and 10% had CI <2.2 L/min/m2. FRF had the highest risk of waitlist mortality (21%) and FNH had the highest risk of post-transplant mortality (36%). CONCLUSIONS: Elevated Fontan pressure is more common than low cardiac output in pediatric failing Fontan patients listed for transplant. Subtle hemodynamic differences exist between the various phenotypes of pediatric Fontan failure. Waitlist and post-transplant mortality risks differ by phenotype.