Late Left Ventricular Outflow Tract Obstruction Following the Rastelli Operation: Expectations Out to 20 Years.

BACKGROUND: Consensus is lacking regarding the optimal operation for transposition, ventricular septal defect, and pulmonary stenosis. METHODS: Between 1968 and 2012, a total of 76 patients underwent the Rastelli procedure, with 52 mid- or long-term survivors. A bracketing analysis was used to estimate the likelihood of late left ventricular outflow tract obstruction (LVOTO). RESULTS: Early mortality decreased over the period of study, with no hospital mortality since 2000. Among one year survivors, 10- and 20-year survival was 90% and 72%, respectively. Freedom from reoperation for LVOTO was 87% at 20 years, with no increase in risk among patients having the procedure before 5 years of age. Available late echocardiographic or catheterization data indicated mild or no LVOTO at a median of 14.3 years in a subset of 38 patients. Estimated freedom from major LVOTO at 20 years is bracketed between the estimate of 87% freedom from reoperation for LVOTO at 20 years and the 78% freedom from reoperation for LVOTO or cardiac death by 20 years. CONCLUSION: The Rastelli operation can be performed in the current era with an early mortality that approaches 0% and with 20-year survival that exceeds 70%. The late risk of important LVOTO appears to range from about 13% to 22% at 20 years, with no increase in risk among patients operated upon before the age of 5 years.

Health-Related Quality of Life in Adult Survivors After the Fontan Operation.

The primary objective of this study was to ascertain the long-term health-related quality of life (HRQOL) of adult patients who underwent a childhood Fontan operation for palliation of univentricular cardiac anomalies. The secondary objective was to compare the long-term HRQOL of Fontan survivors to that of pediatric heart transplant recipients. This cross-sectional study examined adult survivors (>19 years) who underwent a Fontan operation during childhood (Fontan group) or a pediatric heart transplant (HT group) between 1988 and 2011 (23-year span). HRQOL was assessed using the short form 36 survey. The study group consisted of 49 Fontan group patients and 13 HT group patients who responded to the survey. HRQOL scores of the Fontan group were similar to those of an age-controlled healthy US population in social and mental functioning, energy or vitality, and overall mental component score (P ≥ 0.2). However, Fontan scores in physical functioning, bodily pain, general health, and overall by physical component were significantly lower than those of the age-controlled US population (P < 0.05). No differences were identified between Fontan and HT patients. This favorable life-satisfaction period (average 18 years) should be considered when informing patients and families of expectations with the Fontan pathway vs certain higher-risk procedures.

Maintenance steroid use at 30 days post-transplant and outcomes of pediatric heart transplantation: A propensity matched analysis of the Pediatric Heart Transplant Study database.

BACKGROUND: Maintenance steroid (MS) use in pediatric heart transplantation is variable. The purpose of this study was to evaluate the impact of MS use on graft outcomes. METHODS: All patients <18 years old in the Pediatric Heart Transplant Study database at the time of first heart transplant between 1993 and 2011 who survived ≥30 days post-transplant and were from centers with a protocolized approach to MS use were included (N = 2,178). Patients were grouped by MS use at 30 days post-transplant as MS+ or MS- (no MS use). Propensity score analysis was used to generate matched groups of MS+ and MS- patients based on pre-transplant and peri-transplant factors. Kaplan-Meier survival analysis was used to compare freedom from graft loss, graft loss secondary to rejection, rejection, rejection with severe hemodynamic compromise (RSHC), malignancy, and infection between groups. RESULTS: Of patients, 1,393 (64%) were MS+ and 785 (36%) were MS-. There were 315 MS- patients who had propensity matched MS+ controls. Kaplan-Meier estimates showed no difference in graft loss (p = 0.9) or graft loss secondary to rejection (p = 0.09). At 1 year post-transplant, there was no difference in freedom from rejection (p = 0.15) or malignancy (p = 0.07), but there was lower freedom from RSHC and infection in the MS- group (p = 0.05 and p = 0.02, respectively). CONCLUSIONS: MS use at 30 days post-transplant was not associated with enhanced graft survival after pediatric heart transplant. MS- patients had a higher incidence of RSHC and infection. These risks should be taken into consideration when determining MS use for pediatric recipients of heart transplants.

The modern Fontan operation shows no increase in mortality out to 20 years: a new paradigm.

OBJECTIVE: Dating back to the first published report of the Fontan circulation in 1971, multiple studies have examined the long-term results of this standard procedure for palliation of single-ventricle heart disease in children. Although the technique has evolved over the last 4 decades to include a polytetrafluorethylene (PTFE) conduit for a large percentage of patients, the long-term outcome has not yet been established. The aim of the current study was to investigate the possibility of a late increasing risk for death after 15 years among patients with a modern Fontan operation and to evaluate late morbidity. METHODS: Between January 1, 1988, and December 31, 2011, 207 patients underwent the Fontan procedure using an internal or external PTFE conduit plus a bidirectional cavopulmonary connection. Survival and late adverse events were analyzed. Risk factors for early and late mortality were examined using hazard function methodology. RESULTS: At 1, 10, and 20 years, survival for the entire cohort was 95%, 88%, and 76%, respectively, with no deaths in the last 6 years of the study. Hazard modeling showed a 1.3% risk of death per year 24 years after the Fontan procedure, with no late increasing hazard phase. Freedom from reoperations was greater than 90% at 20 years and freedom from thrombotic complications was 98% at 20 years (with greater than 80% of patients on aspirin alone). Survival curves were superimposable for 16- to 20-mm conduits, and the freedom from any reoperation including transplantation was greater than 90% after 20 years. Multivariable risk factor analysis identified only earlier date of operation as a predictor of early and late mortality. By era of surgery, the 10-year predicated survival is 89% for patients undergoing surgery in 2000 and 94% for patients in 2010. CONCLUSIONS: Early and late survival after a Fontan operation with a PTFE conduit is excellent, with no late phase of increasing death risk after 20 years. Late functional status is good, the need for late reoperation is rare, and thrombotic complications are uncommon on a standard medical regimen including aspirin as the only anticoagulation medication.

An international comparison of current strategies to prevent herpesvirus and fungal infections in hematopoietic cell transplant recipients.

Herpes virus (cytometalovirus [CMV], herpes simplex virus, varicella zoster virus) and invasive fungal infections continue to cause significant morbidity and mortality in allogeneic hematopoietic cell transplant (HCT) recipients despite the availability of effective therapies. In this study, we developed an Internet-based survey, which was distributed to all hematopoietic cell transplant centers participating in the Center for International Blood and Marrow Transplant Research (CIBMTR) program, to gather information on strategies utilized for the prevention of disease caused by herpes viruses and fungal infections between 1999 and 2003. The survey response rate was 72%, representing 175 programs from 32 countries. Generally, reported center strategies were in accord with the Center for Disease Control and Prevention guidelines published in 2000, with 81% of programs using low-dose acyclovir prophylaxis for herpes simplex virus seropositive patients, 99% of programs reporting use of a CMV prevention strategy during the first 100 days posttransplant for all patients at risk of CMV disease, and 90% of programs using antifungal prophylaxis. Seventy percent of programs reported routine use of a CMV prevention strategy in high-risk patients after day 100. The greatest departure from published guidelines was the use of acyclovir prophylaxis for varicella zoster virus seropositive recipients in 75% of programs. There were very few reported changes within centers in practices over the study time period. Significant regional variations were found with regard to surveillance procedures and treatment durations. There were no significant differences in treatment practices by center size and very few differences found between those centers that reported treating primarily pediatric patients versus primarily adult patients. In summary, our survey demonstrates overall agreement with published guidelines for the prevention of disease because of herpesviruses and fungal infections with significant regional differences found in duration of antiviral prophylaxis, duration of preemptive therapy, and duration and dosing of antifungal prophylaxis. Center size and age of primary patient population were not associated with many reported differences in strategies.

Allogeneic hematopoietic cell transplant for prolymphocytic leukemia.

The poor prognosis of patients with prolymphocytic leukemia (PLL) has led some clinicians to recommend allogeneic hematopoietic cell transplant (HCT). However, the data to support this approach is limited to case-reports and small case series. We reviewed the database of the Center for International Blood and Marrow Transplant Research (CIBMTR) to determine outcomes after allotransplant for patients with PLL. We identified 47 patients with a median age of 54 years (range: 30-75 years). With a median follow-up of 13 months, progression-free survival (PFS) was 33% (95% confidence interval [CI] 20%-47%) at 1 year. The most common cause of death was relapse or progression in 49%. The cumulative incidence of treatment-related mortality (TRM) at 1-year posttransplant was 28%. The small patient population prohibited prognostic factor analysis, but these data support consideration of allotransplant for PLL. Further study of a larger population of patients is needed to determine which patients are more likely to benefit.

Impact of prior imatinib mesylate on the outcome of hematopoietic cell transplantation for chronic myeloid leukemia.

Imatinib mesylate (IM, Gleevec) has largely supplanted allogeneic hematopoietic cell transplantation (HCT) as first line therapy for chronic myeloid leukemia (CML). Nevertheless, many people with CML eventually undergo HCT, raising the question of whether prior IM therapy impacts HCT success. Data from the Center for International Blood and Marrow Transplant Research on 409 subjects treated with IM before HCT (IM(+)) and 900 subjects who did not receive IM before HCT (IM(-)) were analyzed. Among patients in first chronic phase, IM therapy before HCT was associated with better survival but no statistically significant differences in treatment-related mortality, relapse, and leukemia-free survival. Better HLA-matched donors, use of bone marrow, and transplantation within one year of diagnosis were also associated with better survival. A matched-pairs analysis was performed and confirmed a higher survival rate among first chronic phase patients receiving IM. Among patients transplanted with advanced CML, use of IM before HCT was not associated with treatment-related mortality, relapse, leukemia-free survival, or survival. Acute graft-versus-host disease rates were similar between IM(+) and IM(-) groups regardless of leukemia phase. These results should be reassuring to patients receiving IM before HCT.