Between January 1, 1989, and December 31, 1994, we have treated 122 primary heart recipients with FK 506 (group I) and 121 with cyclosporine (group II). Fifty patients in the cyclosporine (CyA) group received no lympholytic induction (CyA alone) and 71 others received lympholytic induction with either rabbit antithymocyte globulin or OKT3 (CyA+LI). The mean follow-up was longer in the FK 506 group than in the CyA groups (3.2 +/- 1.3 vs 2.3 +/- 1.8 years; p< 0.01). Patient survival did not differ on the basis of the type of immunosuppression used. At 3 months after transplantation, the freedom from rejection in the FK 506 group was higher than that of the CyA-alone group (47% vs 22%, p < 0.01) but similar to that of the CyA+LI group (47% vs 53%). The linearized rejection rate (episodes/100 patient-days) of the FK 506 group (0.09 episodes) was lower (p < 0.05) than that of the CyA-alone group (0.26) and the CyA+LI group (0.13). The requirement for pulsed steroids to treat rejection was less in common in the FK 506 group than in either CyA group. Eighteen patients in the CyA group had refractory rejections; all resolved with FK 506 rescue. Two patients in the FK 506 group had refractory rejection that resolved with total lymphoid irradiation (n=1) and methotrexate therapy (n=1). Patients receiving FK 506 had a lower risk of hypertension and required a lower dose of steroids. Although the mean serum creatinine concentration at 1 year was higher in the FK 506 group, this difference disappeared after 2 years. No patients required discontinuation of FK 506 because of its side effects. Our intermediate-term results indicate that FK 506 compares favorably with CyA as a primary immunosuppressant in heart transplantation.
D uring the last few years, pediatric cardiologists have witnessed a dramatic change in the utilization of the cardiac catheterization laboratory.1-21 Improved noninvasive diagnostic techniques have narrowed indications for diagnostic cardiac catheterizations while the laboratory is now increasingly being used for therapeutic procedures. Recently, numerous catheter techniques, increased numbers of persons and centers using these techniques, and the increased number of lesion types thought to be amenable to catheter therapy have caused concern about the appropriateness of some applications of pediatric therapeutic cardiac catheterization.Compared with diagnostic cardiac catheterization, therapeutic catheter procedures require more time and resources, are costlier and riskier, and demand more technical training and expertise. High levels of skill and expertise are required of the operator who performs the various therapeutic catheterization techniques. These procedures should only be performed in institutions with appropriate facilities, personnel, and programs.22 These considerations, combined with the rapid increase in the number of laboratories and cardiologists performing therapeutic catheterization procedures, cause concerns about hospital and physician credentialing, hospital and physician peer review, and human subjects investigational review. These concerns have prompted this report on the current status of pediatric therapeutic cardiac catheterization and its important new techniques as well as the development of guidelines for specific credentialing and review.
The most common variety of ventricular septal defect, a perimembranous defect, is frequently associated with a so-called aneurysm of the membranous septum. Previous studies have suggested that ventricular septal defects associated with an aneurysm of the membranous septum tend to spontaneously decrease in size or close more than defects without such an aneurysm. To better define the natural history of this entity, clinical and catheterization data from 87 patients with ventricular septal defect and aneurysm of the membranous septum were reviewed. The initial evaluation was made at a median age of 0.3 years (range 0.1 to 11), with the final evaluation at a median age of 10 years (range 1.5 to 20) and a median duration of follow-up of 8.6 years (range 1.2 to 18.8). Approximately 75% of the ventricular septal defects had a small or no left to right shunt at last evaluation. Overall, 48 patients (55%) had no significant change in the size of the defect, and 39 (45%) showed improvement during the period of observation. Only four patients (5%) had spontaneous closure of the defect. Of the 49 patients who presented with a large left to right shunt, with or without congestive heart failure, 23 (47%) had persistence of a shunt large enough to warrant surgery. When spontaneous improvement occurred, it did so by 6 years of age in all but one patient. Therefore, a continued tendency for a ventricular septal defect associated with an aneurysm of the membranous septum to spontaneously decrease in size or close after this age may be less likely than previously suggested.(ABSTRACT TRUNCATED AT 250 WORDS)
PTLD is a major complication after transplantation. Treatment options for PTLD are not standardized, usually sequential, starting with reduction in immunosuppression. Recently, we have used a dual combination of rituximab and reduced dose chemotherapy (R/C) directly after failed RI. We retrospectively identified 30 pediatric PTLD cases across four organ systems at our center from 1995 to 2008. We assessed recent outcomes of PTLD in children, comparing the responses to different regimens. Two-yr failure-free survival was best in renal and heart recipients (80-88%), followed by liver (57%) and lung (0%). Of note, two patients were Epstein-Barr peripheral blood viral load low positive but tumor EBER negative. Three patients had no detectable viral load but were EBER positive. The R/C regimen (n = 8) had the highest CR rate (100%), low recurrence (12%) and lowest mortality (12%). Interferon (n = 4) had 75% CR, 33% recurrence and 25% mortality. Rituximab/prednisone (n = 5) had 80% CR, 50% recurrence and 20% mortality. Other chemotherapy (n = 7, including all 4 T-cell PTLDs) had 57% CR, 0% recurrence and 14% mortality. Direct dual R/C combination therapy after failed RI is effective and offers another treatment option for B-cell PTLD.
Patients with restrictive cardiomyopathy (RC) have impaired diastolic function, but intact systolic function until later stages of the disease, ultimately leading to heart failure. Primary RC is often sporadic, but also may be inherited in an autosomal dominant fashion, particularly the idiopathic forms. Recently there has been great interest in inherited cardiomyopathy associated with myocyte desmin deposition ('desminopathies'). In some such families, desmin or alpha-B crystallin gene mutation is the underlying cause, and the desmin accumulation affects skeletal muscle as well, usually causing skeletal myopathy. We describe a large family with apparent autosomal dominant inheritance of desmin-associated RC spanning four generations, with the age of onset and severity/rate of progression being highly variable. This family is relatively unique in that there is no symptom-based evidence of skeletal muscle involvement, and the known desminopathy and cardiomyopathy genes/loci have been ruled out. These data support literature suggesting that desmin deposition may be associated with different underlying gene defects, and that a novel desminopathy gene is responsible for the condition in this family.
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