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Background: Treatment choices for patients with advanced-stage mycosis fungoides (MF) or Sézary syndrome (SS) who have failed first-line systemic therapies can be challenging, as several options are available. However, most evidence is based on observational and early phase studies due to the rarity of the disease. Mogamulizumab has recently been approved for the treatment of adult patients with MF or SS who have received at least one prior systemic therapy; it has a good tolerability profile prompting its use in combination with other agents. This article aims at describing the role of the concomitant use of bexarotene with mogamulizumab in this setting., Cases Presentation: To add information in the field, we describe our experience with four patients with MF/SS who failed first- and second-line treatments and started the combination mogamulizumab in addition to bexarotene. The combination of bexarotene with mogamulizumab in patients with advanced MF/SS after the failure of bexarotene alone obtained a response in all the four patients observed. The response was maintained longer than expected., Conclusions: The combination is promising and deserves further study.

We present the case of an HIV/HCV-coinfected patient with HIV-related pulmonary hypertension (HRPH) who experienced a good clinical and functional response to bosentan, with a subsequent switch to oral sildenafil due to increased transaminase levels. Bosentan resulted less handy in this case, probably due to both side effects and co-morbidities.

From November 1985 through December 1996, 128 patients aged 1 day to 18 years (mean age, 4.7 yrs; median, 3 years) were listed for heart transplant. Forty-seven (36.1%) died after a mean wait of 3.1 months, and 62 underwent transplant after a mean wait of 4 months. Two patients underwent retransplantation. The 1-, 5-, and 11-year actuarial survival rate for the patients who underwent heart transplantation is 68%, 62%, and 42%, respectively. The follow-up ranges from O to 132 months, with a mean follow-up of 39 months. At present, 36 patients, including the 2 who received a retransplant, are alive. Most of them have normal growth, development, and neurologic outcome.

From November 1985 to 31 July 1997, 65 pediatric patients underwent heart transplantation at Bambino Gesù Hospital in Rome. Two of them underwent retransplantation, both 6 years after the first transplant. The 67 transplant patients had a mean age of 59 months; 11 were under 1 year of age. Their indications for transplantation were cardiomyopathies (38), lymphocytic myocarditis (8), and congenital heart diseases (19). Two patients of the first group successfully received a combined heart and kidney transplant. The 1-, 5-, and 11-year actuarial survival rates for the 65 patients who underwent heart transplantation were 68%, 62%, and 42%, respectively. In the 1st postoperative year in patients who had had cardiomyopathy, a total of 50 episodes of acute rejection (AR), with one death, occurred (mean 1.7 AR/patient per year +/- 1.5) and, in patients who had had congenital heart diseases, 19 ARs (one death) occurred with a mean of 1.58 AR/patient per year +/- 1.4. The incidence of AR was significantly higher in patients who had had myocarditis with a total of 26 episodes (mean 3.7 AR/patient per year +/- 2) and one death. Rehabilitation of heart transplanted children and infants was complete (NYHA class 1) in 52% of patients of this series. We conclude that heart transplantation may give a good intermediate and long-term survival in selected patients; the extension of indications to desperately ill patients, or patients with systemic diseases or complex congenital heart diseases may bring less encouraging results, but should not be definitely excluded. Scarcity of donors remains the main limit, being still the first cause of death for patients on our waiting list. Our limited experience seems to suggest that, as described in adults, the cellular amplification of the immune response might affect the post-heart transplant follow up of pediatric patients with myocarditis resulting in a poor outcome for this population.

A 7-year-old boy had undergone heart transplantation (HT) at 1 year of age. The immunosuppressive regimen consisted of cyclosporine and azathioprine (Cy+Aza). During the follow-up there were 7 episodes of moderate rejection: 4 of them occurred during the first 3 months. He had cytomegalovirus (CMV) seroconversion 4 years after HT. After 5 years since he underwent primary HT, cardiac catheterization and selective coronary angiography, performed on a yearly basis, showed triple vessel occlusive disease. The treadmill test was positive. During the following year, the patient's clinical condition deteriorated: in May '92 he underwent retransplantation. Cross-match was negative and there were no common HLA-DR antigens between the first and the second donor heart. Only 1 rejection episode occurred during the first 18 months of follow-up. Despite the shortage of donor hearts we feel that retransplantation is justified as an elective procedure in pediatric patients with cardiac allograft vasculopathy (CAV).

Objective: To investigate the overall occurrence of congenital heart disease in 103 pedigrees with a proband affected with atrioventricular canal., Design: Family study of patient series., Setting: Department of Pediatric Cardiology, Bambino Gesu' Hospital, Rome, Italy., Participants: One hundred three consecutive patients with atrioventricular canal and normal karyotype and their first- and second-degree relatives., Interventions: None., Measurements/main Results: In 12 pedigrees (11.7%), one or more relatives had concordant or discordant congenital heart disease. Congenital heart disease occurred in four (1.9%) of the 206 parents of probands, in four (3.6%) of the 111 siblings, and in five (0.8%) of the 644 uncles or aunts. None of the grandparents had congenital heart disease. Atrioventricular canal affected several family members in five pedigrees., Conclusions: Familial aggregation of atrioventricular canal in several pedigrees requires careful collection of family histories, accurate cardiologic evaluation of the first- and second-degree relatives of the probands, and fetal echocardiography in all pregnant women in at-risk families.

Background: The bidirectional cavo-pulmonary anastomosis is a useful surgical procedure for patients with complex congenital heart disease characterized by univentricular physiology. This operation significantly increases effective pulmonary blood flow and rarely causes distortion of the main pulmonary arterial branches. The aim of this study was to evaluate the clinical and pathophysiologic consequences of bidirectional cavo-pulmonary anastomosis as well as the effectiveness of this surgical approach as a definitive palliation., Methods: Clinical, hemodynamic and functional data of 12 patients who had undergone a bidirectional cavo-pulmonary anastomosis were reviewed. Each patient underwent clinical examination, chest x-ray, ambulatory EKG, bidimensional color-Doppler echocardiography, spirometric analysis, lung perfusion scintigraphy, cardiac catheterization and angiography, and stress test. Stress test data were compared to those obtained by a normal population of age-matched children., Results: After a mean follow-up interval of 28 +/- 20 months all patients showed a good clinical condition. Improved oxygenation, exercise tolerance and growth velocity were noted in all patients. Only 34% of patients showed abnormalities of chest x-ray, 29% manifested significant arrhythmias during ambulatory EKG and 20% showed abnormal ventricular function echocardiographically. Spirometry was normal in all patients, while lung perfusion scintigraphy was constantly abnormal with right/left and upper/lower perfusion mismatch. At cardiac catheterization a reduction of end diastolic volume (from 106.9 +/- 50.6 to 67.3 +/- 41.6 ml, p < 0.05) and end systolic volume (from 58.0 +/- 27.9 to 32.5 +/- 33.5 ml, p < 0.05) was noted. The functional evaluation of these patients was highly abnormal due to an impaired response to effort as evidenced by work time (p < 0.0001 vs normal) and stress-induced cardiovascular modifications., Conclusions: On the basis of these findings, we suggest that: 1) bidirectional cavo-pulmonary anastomosis is useful as an intermediate step towards a Fontan procedure, as it improves the hemodynamic performance of the systemic ventricle; 2) conversely, bidirectional cavo-pulmonary anastomosis should not be considered a form of definitive palliation for complex congenital heart disease because in these patients the response to exercise remains unsatisfactory.

Background: Total cavopulmonary connection has been proposed as a rational alternative to atriopulmonary connection for complex Fontan operations. In addition to fluid dynamic advantages proposed by de Leval, total right heart bypass may address an emerging important issue after repair of single ventricle: late atrial arrhythmias. The purpose of this study is to document the postoperative hemodynamic findings in 22 consecutive patients who received a total extracardiac right heart bypass with an inferior vena cava-to-pulmonary artery extracardiac Dacron conduit with a modified Glenn anastomosis (superior vena cava-to-pulmonary artery anastomosis)., Methods and Results: Twenty-eight patients with complex congenital heart disease underwent this surgical procedure. One patient died (early mortality, 3.5%). Mean follow-up was 13.9 months. Postoperative cardiac catheterization and echo Doppler studies were performed in 22 of the 27 survivors. In 18 of 22 patients, hemodynamic data were satisfactory; a preferential direction of caval flows to both lungs was observed. Echo Doppler assessments show that forward cavopulmonary flow appears as a predominant early diastolic event, in contrast to what occurs in atriopulmonary connections. This hemodynamic model emphasizes the possible role of the diastolic ventricular performance (as a "suction pump") in Fontan circulation. Early postoperative atrial arrhythmias were observed in two of the survivors., Conclusions: The technical advantages and the hemodynamic benefits of this form of right heart bypass are encouraging. Although the use of artificial material in this procedure is extensive, none of the survivors showed thromboembolic complications or peel formations with narrowing and/or obstruction. Further investigations during a longer follow-up are needed to confirm the early and intermediate results, especially the reduction of late atrial arrhythmias.

The bidirectional cavopulmonary anastomosis is a surgical procedure suitable for patients with cyanotic congenital heart disease and univentricular physiology. This operation is able to increase the effective pulmonary blood flow without any additional load on the cardiac work and without any further distortion on the pulmonary artery branches. The cavopulmonary anastomosis can represent the first stage for patients destined for Fontan repair or a definitive palliative operation in high risk Fontan candidates. In order to test the hypothesis of a definitive palliation by cavopulmonary anastomosis in this kind of patients, we evaluated the hemodynamic data before and after this surgical approach and compared these data with their clinical and functional outcome. We evaluated 74 patients submitted to bidirectional cavopulmonary anastomosis by either hemodynamic or functional evaluation. End-diastolic and end-systolic ventricular volumes were significantly reduced by bidirectional cavopulmonary anastomosis (p less than 0.0005). Despite these data and a normal ambulatory ECG, spirometry and echocardiographic analysis, the stress test showed discouraging results. In fact, mean work time and peak heart rate were significantly different from normal values showing an impaired functional capacity of these children. In conclusion we think that bidirectional cavopulmonary anastomosis can not be considered an adequate definitive palliation but it represents a very good stage to preserve the pulmonary arteries and to prepare the systemic ventricle towards the Fontan repair.

Of 353 children who underwent surgical repair of a congenital heart defect, including closure of a ventricular septal defect (VSD), 12 patients (four with tetralogy of Fallot, five with a VSD, and three with a double-outlet right ventricle) developed subaortic stenosis, which was diagnosed one to six years after the surgical procedure. Five patients required surgical treatment of the subaortic stenosis, and one required percutaneous balloon angioplasty. Postsurgical subaortic stenosis appears to be an uncommon progressive acquired disease.

The use of oral propafenone was assessed in 57 children. Mean age was 4.8 +/- 5.2 years (range 1 day to 17 years). Twenty-three patients were less than 1 year of age (40%) and 10 were less than 1 month of age (18%). Arrhythmias included supraventricular tachycardia in 32 patients (57%), atrial flutter in six (10%), and ventricular tachycardia in 19 (33%). Cardiac diagnoses included: an otherwise normal heart in 30 (53%); unoperated congenital heart defect in 11 (19%); cardiomyopathy in nine (16%); and an operated congenital heart defect in seven (12%). Mean dose of propafenone was 13.1 mg/kg/day (range 8 to 15). Success (assessed by relief of symptoms and serial 24-hour ECGs) was achieved in 16 of 32 patients with supraventricular tachycardia (50%), three of six with atrial flutter (50%), and 8 of 19 with ventricular tachycardia (42%). Drug efficacy in patients with and without organic heart disease was 30% (8 of 27) and 63% (19 of 30), respectively (p less than 0.05). Mean duration of therapy in patients in whom the drug was effective was 22.9 +/- 13.2 months (range 6 to 60). Growth was unimpaired in all patients. A neonate with nonsustained monomorphic ventricular tachycardia had sustained ventricular tachycardia during the second day of propafenone therapy at a dose of 13 mg/kg/day. Sustained ventricular tachycardia disappeared when the drug was discontinued. Another patient had an asymptomatic and transient increase in the glutamic pyruvic transaminase level. No adverse hemodynamic effects were observed. In conclusion, propafenone is an effective antiarrhythmic drug in children with normal hearts; it is less effective in patients with heart disease.(ABSTRACT TRUNCATED AT 250 WORDS)

The patterns of pulmonary circulation were assessed by angiography in a series of 24 patients with right atrial isomerism (asplenia syndrome) and pulmonary atresia. Mean age at catheterization was 21.5 days (range 1 day-11 months). The pulmonary arteries were confluent in 22 cases (91.7%). Eighteen patients (75%) had ductus-dependent pulmonary circulation in presence of confluent pulmonary arteries, similar to cases of pulmonary atresia combined with complex congenital heart disease. The ductus appeared in continuity with the aortic arch, shaping an acute angle with the descending aorta, as in cases of pulmonary atresia with ventricular septal defect. A bilateral ductus was present in 5 cases (20.8%), 3 with confluent and 2 with nonconfluent pulmonary arteries. One patient presented multiple systemic collaterals to the lungs. All the other patients had normal arborization of the pulmonary arteries. Seven patients presented an obstructed total anomalous pulmonary venous connection: 4 in inferior vena cava and 3 in superior vena cava. These anatomical observations provide useful information for the surgical palliation of these anomalies and, probably, warrant pre-operative angiographic evaluation in all the patients with right atrial isomerism.

The muscular ventricular septal defect associated with the atrioventricular canal is a malformation which has not yet been extensively studied. Between June 1982 and December 1989, 151 patients with atrioventricular canal underwent echocardiography and angiocardiography in our Department. Of these 95 (62.9%) had a complete form and 56 (37.1%) a partial. Among the 151 patients, 81 (53.6%) presented Down syndrome. We found 5 muscular ventricular septal defects in 4 patients; in 3 cases there was a single defect and in one case two defects. These defects were midmuscular in all patients and one patient also presented an apical defect. All 4 patients with muscular ventricular septal defect presented a complete atrioventricular canal and aortic coarctation; 3 out of 4 patients had a hypoplastic left ventricle with absence of Down syndrome. The muscular ventricular septal defect is a malformation which is rarely associated with atrioventricular canal (4/151 = 2.6%). In our experience, it was always associated with a complete form with aortic coarctation and was very rare in Down syndrome patients (1/81 = 1.2%). These findings may represent a peculiar association of anomalies which may be caused by fetal hemodynamic mechanisms.