Children with chronic organ failure possibly ending in organ transplantation: a survey in an Italian region of 5,000,000 inhabitants

Aim: The Italian Piedmont region sponsored in 2005 a population-based registry to assess the epidemiology of childhood chronic organ failure involving kidneys, liver, heart or lungs.
Methods: Patients in chronic organ failure who were younger than 18 years were selected, and entered the registry when accomplishing the standard failure criteria for each organ. The cases were reported by the general paediatricians of the region and integrated with the data gathered by the Children University Hospital, a tertiary care centre.
Results: In Piedmont (647 727 inhabitants < 18 years), a total of 146 children (217 cases per million of paediatric population) were found to be affected by chronic organ failure (mean age 10 years; range 0–17). The organ failure involved kidneys in 68 subjects (48%), liver in 24 (17%), heart in 21 (15%) and lungs in 28 (20%), and was severe in 32 subjects (6 on transplantation waiting list). The most represented disease leading to chronic renal failure was renal hypodysplasia (79%). Chronic liver failure was mostly caused by biliary atresia (30%), autoimmune hepatitis (25%) and Wilson's disease (21%). Dilated cardiomyopathy (62%) and surgically treated congenital cardiopathy were the two leading causes of chronic heart failure. The most represented disease leading to chronic lung failure was cystic fibrosis (89%).
Conclusion: This is the first report of the literature focusing on the epidemiology of chronic organ failure in children encompassing a region of 4 000 000 inhabitants. This clinical condition is rare, but medically and socially very demanding not only in childhood but the life along, as most of these patients will need solid organ transplantation decades later.     

DIALYSIS AND RENAL TRANSPLANTATION OF CHILDREN IN EUROPE, 1974

ABSTRACT. About a third of all children with end-stage renal failure in Europe were accepted for treatment by regular dialysis or transplantation during 1974. The number of specialised paediatric dialysis centres increased. The number of renal transplants also increased, but fewer living donor grafts and retransplants were performed in 1974. The best survival was again observed on home dialysis. Patient survival after a cadaver graft improved. Children more than ten years old appeared to have a better survival on dialysis and a somewhat better graft function than younger patients. Rehabilitation was similar on home dialysis and after transplantation, but full school activity in ordinary school was reported in only 40% of all children on hospital dialysis. Children on haemodialysis tended to dialyse more frequently in 1974, than before, but for fewer hours per week. Hepatitis was still a problem and only a small reduction in the incidence was noted during 1974. Children on dialysis required more blood transfusions than adults. Body growth on haemodialysis was retarded to a similar degree on boys and in girls; 70 % of the haemodialysed and 63 % of the transplanted children had a growth velocity below the third percentile. Growth rate was noted to fall after the first year on dialysis.     

DIALYSIS AND RENAL TRANSPLANTATION OF CHILDREN IN EUROPE, 1974

Abstract. About a third of all children with end-stage renal failure in Europe were accepted for treatment by regular dialysis or transplantation during 1974. The number of specialised paediatric dialysis centres increased. The number of renal transplants also increased, but fewer living donor grafts and retransplants were performed in 1974. The best survival was again observed on home dialysis. Patient survival after a cadaver graft improved. Children more than ten years old appeared to have a better survival on dialysis and a somewhat better graft function than younger patients. Rehabilitation was similar on home dialysis and after transplantation, but full school activity in ordinary school was reported in only 40 % of all children on hospital dialysis. Children on haemodialysis tended to dialyse more frequently in 1974, than before, but for fewer hours per week. Hepatitis was still a problem and only a small reduction in the incidence was noted during 1974. Children on dialysis required more blood transfusions than adults. Body growth on haemodialysis was retarded to a similar degree on boys and in girls; 70% of the haemodialysed and 63% of the transplanted children had a growth velocity below the third percentile. Growth rate was noted to fall after the first year on dialysis.     

Current status of liver transplantation

Liver transplantation is accepted therapy for acute or chronic liver failure. Survival after LT has improved significantly in developed countries and this has increased the awareness of this treatment modality in the developing world. Successful LT in both children and adults have now been reported from India. Chronic liver failure secondary to cholestatic liver disease in the most frequent indication for LT, with biliary with atresia as the single commonest cause. Innovative techniques such as reduced size, splint, and living donor liver transplantation are being applied more often to decrease long waiting times and reduce associated morbidity and mortality. Early postoperative complications include primary graft failure, venous thrombosis, rejection, biliary complications and infections. Late complication includes CMV or EBV infections, side effects of immunosuppression, post transplantation lymphoproliferative disease and late biliary strictures. Most children achieve good quality of life. There are still many lessons to learn and there are future challenges such as the ever increasing problems of donor scarcity and the search for potent but less toxic immunosuppressive agents.     

新生儿机械通气时撤机失败原因分析

目的分析新生儿机械通气时呼吸机撤机失败原因。方法对自1999年4月至2006年2月我院儿内科新生儿重症监护病房（NICU）,应用人工呼吸机235例中出现撤机失败的29例进行临床分析。结臬应用机械通气治疗新生儿呼吸衰竭时,撤机失败率早产儿组较足月儿组和过期产儿组高（P〈0．01）;多脏器功能损害组较非多脏器功能损害组高（P〈0．01）;机械通气时间3d以上组较机械通气0—3d组高（P〈0．05）;无静脉营养支持组较静脉营养支持组高（P〈0．05）。结论及时治疗原发病、合理使用呼吸机、加强机械通气管理、及时适时撤机、正确诊治呼吸机相关性肺炎、加强营养支持,是减少呼吸机撤机失败的关键。     

��ͯ��˥��76���ٴ���Ԥ�����

目的总结76例儿童肝衰竭临床资料,探讨影响儿童肝衰竭预后的相关因素。方法选择重庆医科大学附属儿童医院2001年6月至2010年11月收治的76例病历资料,用EXCEL及SPSS18.0软件分析,单因素分析用累积比数logistic回归模型分析,多因素分析用logistic回归模型分析。结果 (1)儿童肝衰竭治疗好转、治愈率低(17.1%)。(2)本组病例病因不明32例(42.1%),病因明确44例(57.9%),胆道畸形、巨细胞病毒、乙型肝炎病毒及EB病毒感染占72.7%。(3)使用非生物型人工肝治疗12例(15.8%),治疗显效2例,有效1例,治疗无效9例。(4)单因素分析提示,年龄、凝血酶原时间活动度(PTA)、白蛋白、肝性脑病、住院时间、并发症的种数对预后的影响有统计学意义(P均<0.05);多因素分析提示,肝性脑病、住院时间是影响患儿预后的独立危险因素(P均<0.05)。结论儿童肝衰竭病情进展快、病死率高,病因不明确占多数,肝性脑病、住院时间是影响患儿预后的独立危险因素。分析各种因素对儿童肝衰竭预后的影响可为疾病的诊断、治疗提供帮助。     

Predictors of Outcome of Myocarditis

Heart failure from myocarditis may be transient or may progress to unremitting severe cardiac failure. This study was performed to determine the outcomes and prognostic features of pediatric patients with myocarditis. Patients with the diagnosis of myocarditis between 1990 and 2001 were identified through the coding system of Yale–New Haven Hospital. A total of 28 patients were included, with ages ranging from 1 day to 20 years. Before discharge, 11 patients developed unremitting severe cardiac failure. Of the remaining 17 patients, at the time of discharge 10 had normal systolic function and 7 had decreased systolic function. Unremitting cardiac failure developed in 9 of 14 patients (64%) with an ejection fraction <30% and in only 2 of 14 (14%) of those with an ejection fraction 30% on admission (p < 0.01). Furthermore, shortening fraction <15%, left ventricular dilatation, and moderate to severe mitral regurgitation on admission as well as arrhythmia were significantly associated with development of unremitting severe cardiac failure. In this series of patients with myocarditis, by the time of discharge 39% of the patients had developed unremitting severe cardiac failure, 25% had depressed systolic function, and 36% had normal systolic function. Predictive factors at admission for poor outcome were ejection fraction <30%, shortening fraction <15%, left ventricular dilatation, and moderate to severe mitral regurgitation.Data from this study was presented as a poster at the 2002 annual meeting of the Society for Pediatric Research and Pediatric Academic Societies.     

Recent advances in the treatment of congestive heart failure

Treatment of heart failure should include correction of the underlying cause. These causes include large left to right shunts, obstructive lesions, arrhythmias, primary myocardial disease etc. The main pharmacological therapy includes inotropic agents, vasodilators and diuretics. Inotropic agents increase myocardial contractility and include digoxin, intravenous dopamine, dobutamine and isoproterenol. Vasodilators improve cardiac pump performance by decreasing the vascular resistance and/or increasing the venous capacitance. Commonly used vasodilator agents include angiotensin converting enzyme inhibitors (captopril, enalapril etc.), hydralazine, prazosin hydrochloride etc. Diuretics inhibit salt and water reabsorption promoting their excretion. Furosemide, thiazide diuretics, aldactone, are commonly used diuretics. Electrolyte and acid-base imbalance can occur on chronic diuretic therapy. Cardiac transplantation is considered for patients where all medical management has failed.     

Understanding failure to thrive

Failure to thrive is encountered in general practice and across all disciplines of paediatrics. It is most commonly diagnosed in children less than 2 years of age, but can also affect those older. There is no consensus regarding a definition, but is understood to reflect sub-optimal growth. The causes are often multi-factorial with a wide spectrum of aetiologies, including a combination of both organic and non-organic factors. Most cases are due to poor nutrition, but neglect as the sole cause or contributing factor should always be remembered. Common age-related aetiologies are described and we aim to provide a practical approach to diagnosis. The importance of taking a comprehensive history, elucidating relevant physical signs and proceeding to a logical method of relevant investigation is described. Treatment is dictated by the underlying pathology, but general therapeutic measures and a multidisciplinary team approach are vital in treatment. The prognosis will vary according to aetiology, and is good in those with unintentional poor calorie intake who recover without any long term physical or developmental problems.     

Haemorrhagic shock and encephalopathy

Two infants are described with a fulminant disorder characterised by profound circulatory collaps and shock, generalised convulsions and unremitting coma, bleeding due to severe DIC, fever, diarrhoea, metabolic acidosis and renal and hepatic failure. Both infants died shortly after onset of the symptoms. Autopsy mainly revealed haemorrhages in different organs, anoxaemic lesions in the brain and a normal structure of liver and pancreas. No causative agent could be demonstrated.We believe that both patients suffered from haemorrhagic shock and encephalopathy, a mostly fatal disorder which has recently been described. Although the clinical and biochemical features are very distinctive, this syndrome is probably heterogeneous and its differentiation from some other dis-orders may be difficult. Its pathogenesis is unknown but there are some indications that intravascular activity of trypsin may play a role. During a study of the two families we obtained abnormal results of immunologic tests in most members: the interpretation of this finding remains conjectural.Haemorhagic shock and encephalopathy may occur more frequently than the restricted literature on this subject suggests. Future studies will have to deal with the question of identity and pathogenesis.Abbreviation HSE haemorrhagic shock and encephalopathy     

Haemorrhagic shock-encephalopathy syndrome: plasmapheresis as a therapeutic approach

We present the case of a 4.5-week-old boy with acute encephalopathy, shock, intestinal bleeding and disseminated intravascular coagulation. The clinical course and typical laboratory parameters were compatible with a diagnosis of haemorrhagic shock-encephalopathy syndrome (HSE). Immediate shock treatment, repeated haemodialysis and plasmapheresis did not prevent a fatal outcome 4 days after the onset of clinical symptoms.Abbreviations HSE haemorrhagic shock-encephalopathy syndrome - HUS haemolytic uraemic syndrome     

小儿血液透析267例次临床分析

报道我院自１９７８年１０月￣１９９４年１１月为３７例肾功能衰竭患儿进行２６７例次血液透析,占同期我院血液透析中心总数１９４８４例次的１．３％,结果：痊愈１４例,好转１６例,死亡７例,主要并发症为失衡综合征（１９．１％）,低血压（１８．０％）和心力衰竭及肺水肿（１．９％）,通过血液透析,降低了小儿明功能衰竭的死亡率。根据临床实践及有关实验研究,提出有关小儿血液透析必须遵循的特点及做法。     

Evaluation of diagnostic criteria of acute renal failure in premature infants

A prospective study was performed to investigate the validity of renal failure index (RFI) or fractional excretion of sodium (FENa) in preterm infants. The subjects were 128 newborn infants, 72 with oliguria and 56 without renal dysfunction (control). Oliguric infants were divided into two categories: acute renal failure (ARF) and prerenal failure (PRF), according to creatinine clearance (Ccr). Furthermore, all subjects were divided into four groups according to gestation, that is, 38 infants with gestational age of 25–28 weeks (group 1), 28 with 29–30 weeks (group 2), 38 with 31–36 weeks (group 3) and 24 of > 37 weeks (group 4). As a result, differentiation between ARF and PRF was valid when the RFI or FENa was used in infants of > 29 weeks gestation (groups 2, 3 and 4). Although infants of > 31 weeks gestation (groups 3 and 4) who present with an RFI > 3 or an FENa > 3% may be diagnosed as having ARF, infants in group 2 with an RFI of > 8 or an FENa of > 6% may be diagnosed as having ARF. For the infants in group 1, the application of RFI or FENa for diagnosis of ARF may be limited because of some overlap among the groups.     

Acute hepatic failure after open-heart surgery in children

Summary Acute hepatic failure (AHF) combined with acute renal failure (ARF) is a well-known complication of open-heart surgery in adults. The occurrence of this complication in two children after open-heart surgery for correction of congenital heart disease is reported. Hypotension occurred during the operation and was treated by catecholamine vasopressors. AHF set in during the postoperative course; it was manifested by impaired consciousness, hypoglycemia, hyperbilirubinemia, hyperammonemia, elevated liver enzymes and prolongation of the prothrombin time with failure of hemostasis. ARF also developed in both children. One of the patients survived the acute episode of hepatic failure. The importance of early diagnosis, routine close monitoring, and appropriate selection of vasopressors is emphasized.     

Acute heart failure and acute renal failure in Kawasaki disease

Acute renal failure and acute heart failure are rare in Kawasaki disease. We experienced two patients with Kawasaki disease who presented acute renal failure and acute heart failure. These two patients gave us an important insight into the understanding of water balance and fluid therapy in Kawasaki disease. One patient showed acute prerenal failure due to fluid exudation from the intravascular to the extravascular space, and subsequent acute heart failure. The other patient showed acute heart failure caused by fluid infusion for the treatment of dehydration. It is suggested that acute renal failure could be caused by a fluid shift from the intravascular to the extravascular space in Kawasaki disease. It is also demonstrated that the reserve of cardiac function could be decreased in patients with Kawasaki disease due to myocarditis even with normal echocardiography and chest X-rays.     

Statural Growth of Children with Chronic Renal Failure on Conservative Treatment

ABSTRACT. Statural growth was studied in 20 prepubertal children with chronic renal failure on conservative treatment followed-up 1.1 to 8.9 years (average 3.9). Five children reached end-stage renal failure during the follow-up period and underwent dialysis or transplantation. Most patients grew at a normal rate. During the observation period only 1 out of 20 children lost more than 0.5 height standard deviation score whereas 9 gained 0.5 to 3.1. A growth velocity above the 97th percentile for at least 1 year was observed in 6 patients. A normal growth rate and even catch-up growth is possible in children with chronic renal failure regardless of the degree of reduction of glomerular function.     

肝功能衰竭患者体外替代与支持治疗

肝功能衰竭患者病死率极高,虽然肝脏移植被认为是最有效的治疗方法,但由于供体有限和价格昂贵,使其应用受到限制.目前人工肝仍是肝功能衰竭患者除内科综合治疗外的惟一选择.本文就肝功能衰竭患者体外替代与支持治疗作简要综述.

Abstract：

The patients with liver failure have a high risk of death.Liver transplant is known as the most curative therapy for those patients,but the transplant operations are often restricted because of the donor deficiency and high medical cost.Now artificial liver is still the best choice to treat the liver failure.In the article,the research progress on the replacement and the support therapy in the patients with liver failure is reviewed.     

Perioperative renal failure in pediatric heart transplant recipients: outcome and risk factors

PRF is encountered in 10-13% of adult heart transplants. Only one study of a single center's experience with PRF has been reported in pediatric patients. This study examines the effect of PRF on pediatric heart transplant outcome using the UNOS database. A total of 3598 patients met inclusion criteria, of whom 254 (7%) had PRF. The PRF group comprised 31 recipients requiring PRE and 223 recipients requiring POST. Compared with No-PRF patients, PRE patients had similar survival rate and POST patients had decreased survival rate at 30 days, one, five, and 10-yr post-transplant (p < 0.001). PRF patients also had significantly lower graft survival at one, five, and 10 yr (p < 0.001). Risk factors for developing PRF included ECMO, ventilator, and inotropic support at listing and CHD as the listing diagnosis. PRF increased the duration of hospital stay and the incidence of chronic severe renal dysfunction. PRF that requires POST (whether or not it began pretransplant) has a significant negative impact on pediatric heart transplant outcome. Specific characteristics identify patients at particular high risk of developing PRF.     

Underlying undiagnosed inherited marrow failure syndromes among children with cancer

To study the prevalence of pediatric cancer patients who have underlying inherited bone marrow failure syndrome (IBMFS), we retrospectively reviewed the medical records of newly diagnosed pediatric cancer patients at The Hospital for Sick Children from June 2009 to May 2010, focusing on clinical, laboratory, and treatment‐related findings which may indicate underlying IBMFS. We found five (1.8%) patients out of 276 who had two or more findings suggestive of IBMFS. We conclude that a small fraction of patients with cancer have clinical features that indicate investigations to rule out underlying IBMFSs. A prospective study is needed to determine their prevalence.     

Hepatic hamartoma in a newborn

In a newborn infant with heart failure and hepatomegaly the radiological findings suggested hepatic haemangioendothelioma, but subsequently the tumour proved to be a hamartoma.