Dilated cardiomyopathy in children: Clinical course and prognosis

Summary The clinical profile of 19 patients with dilated cardiomyopathy ages 2–18 years (mean 13.4±4 years) was reviewed to detect any factors that might be predictive for their survival. Follow-up ranged from 5 to 105 months (mean 39±33 months). Routine treatment consisted of digitalis and diuretics: 14 patients received antiarrhythmics, 6 received vasodilators, and 12 were managed with immunosuppression. There were 12 survivors and 7 nonsurvivors: The 1-year mortality was 21.2% and the 2-year mortality 35.8%. All deaths were within first 2 years. Of the 12 patients who survived 2 years, a significant improvement was noticed in 9. In 3 patients tachycardia-induced cardiomyopathy was diagnosed, and abolition of supraventricular tachycardia was followed by improvement and regression of cardiomegaly. Endomyocardial boopsy was performed in 16 patients. Four with a histologic diagnosis of active myocarditis survived, and in 3 of them a considerable improvement was noticed. Of the 12 patients with nonspecific histologic findings, 6 died (p<0.05). There were no significant differences between survivors and nonsurvivors for any of the following parameters: incidence of severe heart failure (NYHA class III–IV) and severe ventricular arrhythmias (Lown class III–V), relative heart volume, echocardiographic left ventricular diastolic diameter and shortening fraction, and the hemodynamic parameters of cardiac index, left ventricular ejection fraction, left ventricular end-diastolic pressure, and left ventricular end-diastolic volume index.     

儿童肾移植术后的免疫抑制治疗

目的 探讨长期存活的儿童肾移植受者不同免疫抑制方案的有效性与安全性.方法 回顾性分析34例存活5年以上的儿童受者免疫抑制剂使用情况,按所使用的免疫抑制剂不同分为5组:A组:环孢素A+强的松;B组:环孢素A+硫唑嘌呤+强的松;C组:环孢素A+霉酚酸酯+强的松;D组:他克莫司+硫唑嘌呤+强的松;E组:他克莫司+霉酚酸酯+强的松.结果 术后按Kaplan-Meier法得出1、3、5年人/肾存活率分别为100%/97%、91%/87.8%、84.4%/80.9%.术后1、3、5年时肾存活的33例、30例和28例中,服用环孢素A者为16/33(48.5%)、18/30(60%)、15/28(53.6%),服用他克莫司者为17/33(51.5%)、12/30(40%)、13/28(46.4%);服用硫唑嘌呤者为7/33(21.2%)、7/30(23.3%)、9/28(32.1%),服用霉酚酸酯者为26/33(78.8%)、21/30(70%)、17/28(60.7%).移植1年后环孢素A血浓度谷值100～150 ng/ml,他克莫司血浓度1.5～3 ng/ml.各组不同时期的环孢素A和他克莫司剂量和浓度均无明显差异.霉酚酸酯剂量维持在10 mg·kg-1·d-1,强的松5～10 mg/d.服药不依从者占30%.移植肾丢失5例,原因分别为排异反应1例,移植肾带功能死亡4例(肺部感染和药物性肝功能损害各2例).并发症包括高血压(35.7%)、高血脂(28.6%)、感染(17.9%)、牙龈增生(14.3%)、多毛(10.7%)、糖尿病(3.6%).结论 环孢素A/他克莫司、霉酚酸酯、强的松三联免疫抑制治疗是儿童肾移植受者主要的抗排异方案,须定期监测血药浓度,个体化调整剂量.     

儿童肾移植临床免疫抑制用药探讨

目的　探讨儿童肾移植免疫抑制治疗特点。方法　回顾性分析 31例儿童肾移植患者(男 18例,女 13例)的临床资料。根据起始免疫抑制方案不同,分为 3组:A组(环孢素A+硫唑嘌呤+泼尼松)7例;B组(环孢素A+霉酚酸酯 +泼尼松)17例;C组 (他克莫司 +霉酚酸酯 +泼尼松)7例。统计 3组免疫抑制药物调整及维持剂量、移植肾功能变化和术后并发症。结果　1年人 /肾存活率为100% /96.8%,3年人 /肾生存率为94.4% /88.9%。三组各观察点肌酐实测值差异无显著性意义 (P<0.05)。他克莫司组药物副作用小且能保持良好的肾功能。泼尼松调整幅度大、维持量小,与其他组差异有显著性意义(P<0.01)。结论　肾移植是儿童终末期肾病的有效治疗措施。遵循儿童个体差异的免疫抑制治疗是移植后预防排斥的重点。他克莫司、霉酚酸酯、泼尼松三联抗排斥治疗是儿童肾移植理想的免疫抑制治疗方案。     

Evaluation of reproductive functions in male adolescents following renal transplantation

Abstract:  The aim of this study was to analyze the semen variables and hormone profiles in kidney transplanted male adolescents. Eight post-pubertal male patients who underwent successful renal tx during the peripubertal period and who had ESRD during childhood were enrolled in the study. Patients who underwent tx before 14 yr old (group I) and patients who underwent tx after 14 yr old (group II) were evaluated separately. Semen was collected and analyzed. Serum levels of LH, FSH, and testosterone were measured and found to be normal in all patients except one. The mean age at the diagnosis of CKD was six yr and 13 yr in groups I and II, respectively. The mean age at the time of tx was 12 yr in the first and 17.8 yr in the second group. The patients in group I had received prednisone, cyclosporine A and azathioprine with a longer duration of time compared with patients in group II. Sperm counts (15.5 ± 15.7 vs. 82.3 ± 64.2 millions/mL) and sperm motilities (37.8 ± 30.9 vs. 57.8 ± 22.1%) were lower in group I than group II. Only one patient in group II had normal sperm parameters and azospermia was observed in one patient from group I. We conclude that the earlier onset and the longer duration of uremia, the more impairment of reproductive function. Also, it seems that duration of exposure to corticosteroids or cyclosporine combined with azathioprine contribute to sperm dysfunction in peripubertal transplanted boys.     

Late nonresponsiveness to steroids in children with the nephrotic syndrome.

Among 195 nephrotic children, ten developed resistance to prednisone therapy after responding to this drug on one or more occasions (late nonresponders). All were found to have "minimal lesions" on renal biopsy. Nine of these patients went into remission: one responded to further prednisone therapy, one went into remission while receiving azathioprine, and the remaining seven children responded to cyclophosphamide. Five of these seven patients subsequently relapsed; three of them have continued to respond to prednisone. The other two eventually became steroid resistant a second time, but in both instances a second course of cyclophosphamide again induced a remission. These nine patients have been followed for periods ranging from 6 months to 9.5 years (median = 53 months); all are doing well and have normal renal function. The tenth patient died from sepsis four months after the onset of steroid resistance.     

麦考酚酸酯治疗幼年特发性关节炎全身型的疗效分析

目的：评价麦考酚酸酯（mycophenolate mofetil, MMF）治疗幼年特发性关节炎全身型（system onset juvenile idiopathic arthritis, SoJIA）的临床疗效。方法：35例确诊为SoJIA并初次接受治疗的患儿随机分为3组,即传统治疗方案组（对照组）,15例;MMF 1 组：传统治疗方案治疗2周后,病情无缓解,加用MMF治疗,7例;MMF 2 组：采用非甾体抗炎药+泼尼松+MMF治疗方案,13例。观察3组患儿治疗后2、4、12周的症状、体征、实验室指标及不良反应,并随访3~6个月。结果：治疗前MMF2组病程显著长于对照组,差异有统计学意义（P<0.05）。治疗2周后,与对照组相比,MMF1组和MMF2组的泼尼松用量明显减少,ESR明显减低（P<0.05）;MMF1组患儿体温高于另两组（P<0.05）。治疗4周时,与对照组比较,MMF1组泼尼松用量减少（P<0.05）,ESR降低（P<0.05）;MMF2组泼尼松用量减少,体温下降并恢复至正常,WBC值降低,ESR降低,铁蛋白降低,差异均具有统计学意义（P<0.05）;MMF1和MMF2组相比,MMF2组体温更低（P<0.05）。MMF治疗组均未出现MMF用药的副作用。结论：MMF联合治疗能更好地控制SoJIA患儿病情,更快缓解其临床症状,减少联合应用的糖皮质激素药物的剂量,并有良好的安全性。     

Alternate day corticosteroid therapy and growth in renal transplant children

Corticosteroid therapy is probably the main factor inducing stunted growth in pediatric renal transplant recipients. Results of a randomized study in 35 children who received their kidney between 1981 and 1984 are reported. All patients had normal renal function and were taking azathioprine and prednisone to ensure immunosuppression. Eighteen months after transplantation, patients with normal renal biopsy results were randomized to receive further daily corticosteroid therapy (group A) for an additional year or to alternate-day corticosteroid therapy (group B). Chronologic age, bone age, renal function, and previous growth retardation were strictly comparable, in the two groups. During the first year, only prepubertal children in group B exhibited catch-up growth. In children undergoing puberty, annual statural gain was greater in group B (5.6 cm versus 3.2 cm in group A: p less than 0.001). Group A children were switched to alternate-day corticosteroid therapy one year after initiation of the study and exhibited improved growth after this change. No patient had renal function deterioration under alternate-day corticosteroid therapy, throughout the study period. Alternate-day prednisone should be offered to pediatric renal transplant recipients with satisfactory renal function as a mean for protecting growth potential.     

Efficacy of cyclosporin A in children with type 2 autoimmune hepatitis.

The conventional treatment of autoimmune hepatitis (AIH) with prednisone and azathioprine induces remission in most cases but is often associated with poorly tolerated side effects. We carried out a retrospective study to evaluate the efficacy of and the tolerance to cyclosporin treatment in 15 children and adolescents with type 2 AIH. Eight children received cyclosporin as primary immunosuppression because of risk factors for poor tolerance of steroids. Five other patients with relapsing AIH refused to resume treatment with steroids and were treated with cyclosporin. In both groups alanine aminotransferase activity returned to normal within 6 months. Side effects were minimal and well tolerated. No relapse occurred in 10 patients after 1 to 6 years. Cyclosporin was withdrawn in 3 patients after 1, 2, and 3 years and replaced by low doses of prednisone in combination with azathioprine. In 2 other children with acute liver failure, which progressed despite treatment with steroids and azathioprine, the addition of cyclosporin was followed by normalization of prothrombin time.     

Cyclosporine 0.05% ophthalmic emulsion for the treatment of radiation‐associated dry eye in children

Dry eye disease is a well‐known late complication of radiation therapy and is often difficult to treat. We evaluated the usefulness of cyclosporine 0.05% ophthalmic emulsion for the treatment of radiation‐associated dry eye in children. Eleven children received cyclosporine 0.05% emulsion twice daily after failure of conventional therapy. After 6 months, dry eye manifestations improved in three children (27.3%). The remaining eight children showed no improvement with cyclosporine 0.05% ophthalmic emulsion. These results suggest that twice‐daily cyclosporine 0.05% ophthalmic emulsion has limited use in children with refractory radiation‐associated chronic dry eye. Pediatr Blood Cancer 2013; 60: E35–E37. © 2013 Wiley Periodicals, Inc.     

Effective therapy for severe Henoch-Schonlein purpura nephritis with prednisone and azathioprine: a clinical and histopathologic study

OBJECTIVES: To validate a scoring system to assess the severity of renal lesions and to correlate histology with clinical findings. We also examined the efficacy of treatment with prednisone (1 to 2 mg/kg/d) and azathioprine (1 to 2 mg/kg/d) for severe Henoch-Schonlein purpura (HSP) nephritis. METHODS: Twenty patients were evaluated retrospectively. All underwent biopsy before treatment, and 13 underwent biopsy after therapy. We developed a scale based on glomerular, tubulointerstitial (TI), and vascular changes and assigned all specimens acuity, chronicity, and TI scores. The outcomes of 17 patients were compared with those of a historical control group. RESULTS: Chronicity score at initial biopsy increased with increasing delay between onset of renal involvement and first biopsy (rho = 0.55, P =.016) but did not progress after treatment was initiated. Both acuity (rho = 0.57,P =. 016) and TI (rho = 0.69, P =.003) scores correlated with clinical severity at first biopsy. The TI score correlated negatively with serum albumin (rho = -.60, P <.01). Significantly more patients in the study group than in the control group had a favorable outcome (15 [88%] of 17 vs 32 [54%] of 59, P =.011). CONCLUSIONS: Our scale reflects disease activity and highlights the importance of TI changes in severe HSP nephritis. Outcome comparisons indicate that early treatment with prednisone and azathioprine prevents progression of chronic changes and improves outcome.     

Sequential analysis of the lipid profile of children post-renal transplantation

Persistent hyperlipidemia and hypercholesterolemia post-transplantation are risk factors for accelerated atherosclerosis. To evaluate the natural history of lipid abnormalities in children post-transplantation, this study utilized a cohort of 29 patients who were all treated with the same three- drug maintenance immunosuppression (cyclosporine, azathioprine, and prednisone) and whose dosing regimen was rigidly controlled. Fasting blood samples were taken monthly to determine lipid profiles measuring total cholesterol (CHOL), triglycerides (TG), high-density lipoprotein cholesterol (HDL), very low density lipoprotein cholesterol (VLDL), and levels of lipoprotein (a) (LP(a)). A mean value was determined for each of five time periods: 0-3 months, 3-6 months, 6-9 months, 9-12 months and 12-15 months post-transplant. A single specimen of fasting lipid profile was drawn from 21 non-immunosuppressed children attending an ambulatory pediatric clinic and used as control. Despite significant reductions in the cyclosporine and prednisone doses post-transplantation, significant reduction in any of the lipid parameters was only noted after the first year. Reductions in the HDL fraction and in the TG level were noted during the 12-15 month period, however the values obtained in the patient population were significantly elevated for CHOL, TG, LDL and VLDL compared to controls. This study, using a fixed protocol, suggests that the lipid profile should be measured at one year post-transplant in all transplant patients, and if subsequent follow-up continues to exhibit abnormally elevated levels of CHOL and LDL, interventional therapy should be considered.     

Oral tacrolimus treatment of severe colitis in children

OBJECTIVE: To evaluate the efficacy of oral tacrolimus as an induction agent in steroid-refractory severe colitis. STUDY DESIGN: Open-label, multicenter trial of oral tacrolimus in patients with severe colitis. Patients not responding to conventional therapy received tacrolimus, 0.1 mg/kg/dose given twice a day, and the dosage was adjusted to achieve blood levels between 10 and 15 ng/mL. Response was defined as improvement in a number of clinical parameters (including abdominal pain, diarrhea, rectal bleeding, and cessation of transfusions). Patients who responded by 14 days continued to receive tacrolimus, and 6-mercaptopurine or azathioprine was added as a steroid-sparing agent 4 to 6 weeks after the tacrolimus was instituted. RESULTS: Fourteen patients were enrolled in the study. One patient elected to withdraw after 48 hours. Of the 13 remaining, 9 (69%) responded and were discharged. Tacrolimus was continued for 2 to 3 months in the responders, except for 1 patient who was given tacrolimus for 11 months. After 1 year of follow-up, only 5 (38%) patients were receiving maintenance therapy; the other 4 responders had undergone colectomy. CONCLUSION: Although tacrolimus is effective induction therapy for severe ulcerative or Crohn's colitis, fewer than 50% of patients treated will successfully achieve a long-term remission.     

Hemodynamic failure as an indication to urgent liver transplantation in infants with giant hepatic hemangiomas or vascular malformations--report of four cases

Abstract:  The aim of this study was to present acute hemodynamic failure as a rare indication for liver transplantation in neonates and infants with liver hemangiomatosis. We report four patients aged one to six months with giant liver hemangiomas, with huge arterio-venous shunting within these malformations. In three, many skin hemangiomas were found. All children developed right ventricular failure. In two, a trial of pharmacological reduction was attempted with corticosteroids and cyclophosphamide. In one patient, the arterio-venous fistulas were embolized without any improvement in hemodynamic status. Two children underwent rescue hepatic artery surgical ligation, which did not prevent heart and then multiorgan failure including liver failure. After unsuccessful conventional therapy, all infants were considered for urgent liver transplantation; in three cases, it was performed with a living-related donor, and in one case with a deceased donor. All patients are alive and well with the follow-up between nine and 37 months after transplantation. Liver transplantation should be considered as a rescue treatment in children with hepatic vascular malformations leading to hemodynamic insufficiency when conventional therapy is unsuccessful and multiorgan failure develops.     

Combination of cyclophosphamide, vincristine, and prednisone, followed by maintenance chemotherapy, with and without radiotherapy, in the management of patients with non-Hodgkin's lymphomas.

Sixty patients with non-Hodgkin's lymphomas were treated with a cyclophosphamide, vincristine, and prednisone (CVP) induction regimen, either alone (stage IV) or in combination with radiotherapy (stages I, II, III). The response rates for lymphocytic and histiocytic lymphomas were 82 and 86%. The complete remission (CR) rates were 66 and 71% with a median duration of 13 and 5.5 months respectively. Nodular types responded better than diffuse ones in both lymphocytic (CR rate 85% vs 45%; median duration 24+ months vs 2.5 months) and histiocytic lymphoma (CR rate 100% vs 0%). In lymphocytic lymphomas, survival in the responder group was 90% at 24 months vs only 20% in the nonresponder group (median survival 14.2 months). In the group with nodular lymphocytic lymphoma responding to therapy, there was a 100% survival rate at 24 months. The median survival for patients treated with chemotherapy alone (stage IV) and not responding to therapy, was 22 months vs 14.5 months in the whole nonresponder group (stages I, II, III, IV), suggesting a detremental effect of rediotherapy in the nonresponder group. In histiocytic lymphomas, the median survivals in the responder and nonresponder groups were 19 months and 3 months respectively. These results corroborate the excellent efficacy of the CVP regimen. They also indicate that, after CVP induction, 2 major prognostic factors are the histologic type and the nature of the response to therapy.     

Combination of cyclophosphamide,vincristine, and prednisone,followed by maintenance chemotherapy,with and without radiotherapy,in the management of patients with non-Hodgkin's lymphomas

Sixty patients with non-Hodgkin's lymphomas were treated with a cyclophosphamide, vincristine, and prednisone (CVP) induction regimen, either alone (stage IV) or in combination with radiotherapy (stages I, II, III). The response rates for lymphocytic and histocytic lymphomas were 82 and 86%. The complete remission (CR) rates were 66 and 71% with a median duration of 13 and 5.5 months respectively. Nodular types responded better than diffuse ones in both lymphocytic (CR rate 85% vs 45%; median duration 24+ months vs 2.5 months) and histiocytic lymphoma (CR rate 100% vs 0%). In lymphocytic lymphomas, survival in the responder group was 90% at 24 months vs only 20% in the nonresponder group (median survival 14.5 months). In the group with nodular lymphocytic lymphoma responding to therapy, there was a 100% survival rate at 24 months. The median survival for patients treated with chemotherapy alone (stage IV) and not responding to therapy, was 22 months vs 14.5 months in the whole nonresponder group (stages I, II, III, IV), suggesting a detrimental effect of radiotherapy in the nonresponder group. In histiocytic lymphomas, the median survivals in the responder and nonresponder groups were 19 months and 3 months respectively. These results corroborate the excellent efficacy of the CVP regimen. They also indicate that, after CVP induction, 2 major prognostic factors are the histologic type and the nature of the response to therapy.     

Immunosuppressive Therapy of Chronic Myocarditis in Children: Three Cases and the Design of a Randomized Prospective Trial of Therapy

Three infants, each with a clinical picture of dilated cardiomyopathy, underwent endomyocardial biopsy. Immunohistologic analysis revealed chronic myocarditis. In one infant, a postviral etiology of chronic myocarditis could be assessed on the basis of molecular techniques. Therapy with azathioprine and prednisone resulted in the normalization of echocardiographic findings. Based on these observations, a randomized, multicenter treatment study of chronic myocarditis in children (TCMC) has been initiated.     

甲泼尼龙和环磷酰胺冲击治疗儿童重症系统性红斑狼疮的临床研究

目的　了解甲泼尼龙 (MP)和环磷酰胺 (CTX)冲击治疗儿童重症系统性红斑狼疮(SeverJOSLE)的临床效果和转归。方法　确诊的儿童系统性红斑狼疮 30例 ,初治 2 2例 ,其中狼疮性肾炎 (LN) 2 0例 ,9例纳入前病程 3～ 1 2个月。 30例患儿中 ,2 3例有肾损伤的临床表现 ,其中 ,WHO Ⅳ型 4例 ,WHO Ⅱ型 2例 ,WHO Ⅴ型 1例 ,WHO Ⅲ型 1例 ,7例肾损伤同时伴 1项或以上的神经系统损伤的临床表现 :舞蹈症、癫样发作、脑血管意外 (CAV)和器质性脑综合征 (OBS)等 ;有神经系统症状不伴肾损伤临床表现的 9例 ;3例狼疮危象 (lupuscrisis)。其余 7例不伴肾损伤临床表现 ,仅有轻微或一过性中枢神经系统损伤。根据治疗方法分为 3组 :A组 ,1 8例 ,临床或病理证实合并LN或精神神经性狼疮 (NPLE) ,采用MP +CTX冲击治疗 ;B组 ,7例 ,临床无LN和 (或 )NPLE ,MP冲击诱导后 ,小剂量泼尼松维持治疗 ,并以雷公藤多甙 (Twhf)接续治疗 ;C组 ,5例 ,临床以LN为主 ,糖皮质激素 (GS)治疗。观察各组的近期、远期疗效和远期临床转归。结果　平均随访时间为 (37 2± 2 4 8)个月。近期疗效 :A组与B组间SLEDAI 2K的差别主要是肾脏权重计分 ,9个月时 ,A组的变化最大 ,计分与B组相似 ,C组SLEDAI 2K计分在中度活动范围 ,主要是肾脏的权重结果。远期     

HRQOL implications of treatment with dexamethasone for children with acute lymphoblastic leukemia (ALL)

BACKGROUND: Dexamethasone is increasingly used as the steroid of choice in trials for standard risk children with acute lymphoblastic leukemia (ALL). Improvements in event-free survival (EFS) have been attributed to lower CNS relapse rates, However, there are concerns that dexamethasone may be more toxic than previous conventional therapy with prednisone. Such toxicity raises questions about the implications for child neuropsychological function and HRQOL. Patients participating in the UK ALL 99/01 trial were randomized to receive dexamethasone or prednisone as their steroid in induction and maintenance chemotherapy. We compared the HRQOL and behavior in children randomized to receive both these agents. PROCEDURE: Standardized questionnaires to assess parent and child HRQOL at 3-6 months after diagnosis (T1) and 1 year later (T2) completed by mothers in family homes. Forty-five mothers of a child with ALL (32 male, 13 female; average age at T1, 7 years 3 months; at T2, 8 years 3 months) completed HRQOL questionnaires. RESULTS: For the total group, child HRQOL scores improved and behavior problems decreased significantly from T1 to T2. Comparison of HRQOL scores between the 17 children randomized to dexamethasone and 28 children randomized to prednisone showed no significant differences. The rate of improvement in HRQOL from T1 to T2 did not differ between children randomized to dexamethasone or prednisone. CONCLUSIONS: Dexamethasone is increasingly used in the treatment of ALL and has been linked with improved survival rates. Long-term use of dexamethasone raises questions about neuropsychologic toxicity. Although HRQOL increased significantly over the year for all children, the extent of this increase did not differ by chemotherapy. These results should contribute to lessened concerns about use of dexamethasone in the treatment of ALL.     

Clinical response to amino acid-based formula in neurologically impaired children with refractory esophagitis

OBJECTIVE: Chronic gastrointestinal symptoms and histologic changes of the esophagus unresponsive to standard treatments for gastroesophageal reflux disease (GERD) may be improved by the use of elemental formulas. The aim of our study was to evaluate the efficacy of a dietary trial in neurologically impaired children unresponsive to medical and surgical therapy for GERD. METHODS: Nine children (three boys and six girls; median age, 44 months; range, 13-180 months) affected by cerebral palsy associated with severe mental retardation and with long-standing history of GERD were fed the elemental formula, Neocate, for a minimum of 4 weeks. Before and after the dietary trial, each child underwent endoscopy with esophageal biopsy and a cellobiose/mannitol sugar permeability test. The diagnosis of GERD was based on the microscopic changes of the esophagus. RESULTS: Before the dietary trial, according to conventional histologic criteria, esophagitis was considered moderate in seven children and mild in two. Five of nine patients also had abnormal sugar permeability test results. During and after the dietary trial, seven of nine patients experienced resolution of their long-term symptom complaints. Furthermore, after the dietary trial, both endoscopic ( < 0.01) and histologic ( < 0.05) findings significantly improved. At 6-month follow-up, progressive reintroduction of individual dietary proteins, except for cow's milk protein, did not cause reappearance of the symptoms. CONCLUSIONS: In neurologically impaired children unresponsive to conventional antireflux treatments, a course of a highly restricted diet with an amino acid-based formula may bring an immediate and sustained, endoscopically and histologically proven improvement in long-standing gastrointestinal symptoms and esophagitis.     

Experience with heart transplantation in children

Between March 1981 and March 1986, 200 orthotopic heart transplantations were performed at the University of Pittsburgh. Fourteen of those procedures were carried out in children 2 to 16 years of age. Two children received combined liver and heart transplants; one because of familial hypercholesterolemia with associated ischemic heart disease, and the other because of dilated cardiomyopathy associated with intrahepatic biliary atresia. Eight patients had dilated cardiomyopathy, and two had myocarditis. Two had heart transplantations for congenital heart disease: one had multiple muscular ventricular septal defects repaired in infancy and had an associated cardiomyopathy, and the other developed a cardiomyopathic ventricle from a congenital right coronary artery to right atrial fistula. Chronic immune suppression consisted 0.2 to 0.5 mg/kg/d of prednisone and 5 to 50 mg/kg/d cyclosporine, with the addition of antithymocyte globulin for unresolved moderate or severe acute rejection. There were three early postoperative deaths: one from intracranial bleeding, one from Pseudomonas mediastinitis, and one from ischemic injury to transplanted organs. Early postoperative complications included reversible renal failure, hypertension, and seizures. Late problems were related to allograft rejection and side effects of cyclosporine and corticosteroids. Significant rejection episodes occurred in all patients surviving longer than 2 weeks, with seven requiring antithymocyte globulin. Two patients died 8 months following transplantation of severe acute and chronic rejection; another patient required retransplantation for ischemic cardiomyopathy resulting from chronic rejection but subsequently died of recurring rejection 3 months after the second transplantation.(ABSTRACT TRUNCATED AT 250 WORDS)