[Brachytherapy in the treatment of vesicoprostatic rhabdomyosarcomas in children]. / Curiethérapie dans le traitement des rhabdomyosarcomes vésicoprostatiques de l'enfant.

Brachytherapy has been widely used at the Institut Gustave-Roussy since 1972 in pediatric oncology. In genitourinary rhabdomyosarcoma, because of its ballistic and physical characteristics, it represents the optimal treatment whenever irradiation is required and brachytherapy feasible. Between 1976 and 1998, 23 children with bladder or prostate rhabdomyosarcoma were treated with a protocol including brachytherapy, with five of them treated with a salvage brachytherapy. All but one brachytherapy was performed during the surgery. Among the 18 brachytherapies performed as a first-line treatment, eight presented a tumoral evolution: five presented a local evolution, one a local and nodal evolution and two a nodal evolution. Brachytherapy allowed a conservative treatment among ten out of 11 children alive with no evidence of disease. Among the five patients with salvage brachytherapy, two presented a second recurrence. Sequelae were minimal, consisting of one grade I rectitis and one asymptomatic vesical and ureteral reflux. These results are consistent with the published data using more radical treatment. Brachytherapy can represent an alternative to radical surgery, when indications are clearly defined in bladder or prostate rhabdomyosarcoma. This type of treatment can be performed only integrated with other treatments, more particularly with surgery. This approach requires a close cooperation between the different specialists: pediatricians, surgeons and brachytherapists.

Long-term outcome of liver transplantation in patients with glycogen storage disease type Ia.

Liver transplantation may be indicated in patients with GSD type Ia when dietary treatment fails or when hepatic adenomas develop, because they carry a risk of liver cancer or severe intratumoral haemorrhage. Published reports on the results of liver transplantation in patients with GSD Ia include 10 patients and provide little information on long-term outcome. In particular, it is not known whether liver transplantation prevents renal failure due to focal segmental glomerulosclerosis. We report here on 3 patients with GSD Ia in whom liver transplantation was performed at 15, 17 and 23 years of age because of multiple hepatic adenomas in all 3 patients with a fear of malignant transformation, and of poor metabolic balance and severe growth retardation in the youngest one. Renal function was normal in all patients. During the 6-8 years following transplantation, the quality of life has initially greatly improved, with none of the previous dietary restraints and a spectacular increase in height. However, long-term complications included chronic hepatitis C in one patient, gouty attacks in another and focal segmental glomerulosclerosis with progressive renal insufficiency in the third. These results: (1) confirm that liver transplantation restores a normal metabolic balance in patients with GSD Ia, allows catch-up growth and improves the quality of life; (2) suggest that liver transplantation may be considered in teenagers with unresectable multiple adenomas because of a lack of clear-cut criteria to detect malignant transformation early; and (3) suggest that liver transplantation does not prevent focal segmental glomerulosclerosis associated with GSD Ia.

Peliosis hepatis with initial presentation as acute hepatic failure and intraperitoneal hemorrhage in children.

Peliosis hepatis, a condition characterized by the presence of blood-filled lacunar spaces in the liver, usually has a chronic presentation pattern and is mainly reported in adult patients in association with chronic wasting disorders and after administration of various drugs. The present report concerns two previously healthy young children in whom peliosis hepatis initially presented as acute hepatic failure and who had Escherichia coli pyelonephritis. Both patients had active intraperitoneal hemorrhage from the peliotic liver lesions, and liver ultrasonography showed multiple hypoechoic areas of different sizes, which in this context should suggest the diagnosis. One child died from hypovolemic shock and the other recovered. This study indicates that acute peliosis hepatis can be a serious life-threatening disease in children.

Paediatric liver transplantation with a split graft: experience at Bicêtre.

UNLABELLED: This study evaluates the results of paediatric liver transplantation (PLT) with split liver grafts at Bicêtre hospital. PATIENTS AND METHODS: Between January 1, 1988 and December 31, 1995, 205 PLT were performed in 180 children. One auxiliary PLT was excluded from the study. The graft was a whole (WLG), reduced (RLG) and split liver graft (SLG) in 76, 112 and 16 cases respectively. The SLG consisted of segments II + III in 14 cases, and II + III + IV in 2 cases. Results of PLT with SLT, RLT and WLG were retrospectively compared. Minimal follow-up was 12 months. RESULTS: In elective PLT, actual 1 year patient (graft) survival were 93.3% (84.4%) with WLG (n = 64), 84.1% (76.4%) with RLG (n = 72), 81.8% (81.8%) with SLG (n = 11). In urgent LT, actual patient (graft) survival were 100% (83.3%) with WLG (n = 6), 58.6% (52.5%) with RLG (n = 40), 25% (20%) with SLG (n = 5). Specific complications of the splitting technique were: 2 Budd-Chiari syndromes in 2 early patients, without any new case after modification of the technique of left hepatic vein to inferior vena cava anastomosis; 4 bile leaks (25%) from the left hepatic duct to Roux-en-Y loop anastomosis. DISCUSSION: In our experience, the results of PLT with SLG were satisfactory in the elective situation, but disappointing in urgent cases. In the current context of liver graft shortage, appropriate use of this sophisticated and demanding technique depends on the experience of the team, recipient's condition, and logistic considerations.

Ischemic necrosis of bile ducts complicating Schönlein-Henoch purpura.

Gastrointestinal complications of Schönlein-Henoch purpura are frequent and sometimes severe. However, there seem to be no reports of liver involvement. A child is described in whom Schönlein-Henoch purpura was complicated by bile duct lesions, resulting in biliary cirrhosis and requiring liver transplantation. At surgical removal, the liver had lesions of bile ducts and of adjacent small blood vessels in the hilum, very similar to those complicating hepatic artery thrombosis after liver transplantation. These findings suggest that Schönlein-Henoch purpura can be complicated by vasculitis of the peribiliary vessels resulting in ischemic necrosis of the bile ducts. Schönlein-Henoch purpura can be added to the list of causes of ischemic cholangiopathies.

[Bulky superinfected tinea capitis of the scalp. Treatment by surgical resection and reconstruction by cutaneous expansion]. / Volumineuse teigne surinfectée du cuir chevelu. Traitement par résection chirurgicale et reconstruction par expansion cutanée.

BACKGROUND: Tinea capitis are usually cured by medical treatment. We present an unusual case which required a surgical treatment. CASE REPORT: A bulky infected kerion ignored for several weeks extended to a large part of the scalp of a 4-year-old girl. Antibiotics and antifungal agents were ineffective so that a total resection was necessary. The loss of substance was covered by a cutaneous graft; the large alopecia was secondarily repaired by a cutaneous expansion. Evolution has been followed for the past 10 years. CONCLUSION: It is exceptional that surgical treatment should be required for this condition.

Portal vein complications after liver transplantation for biliary atresia.

The objective of this report is to review portal complications (PC) after pediatric liver transplantation (LT) for biliary atresia (BA) in the Bicêtre surgical series. From January 1, 1988, to February 28, 1995, 96 children with BA underwent 115 LTs Portal anastomosis was done on either the recipient portal vein (n = 85) or superior mesenteric vein (n = 11). No antiaggregative agents were administered postoperatively. Median follow-up was 50 months (range, 12 to 97). Nineteen PC (16.5%) occurred in 17 recipients: 16 portal thrombosis (PT) and 3 portal stenosis (PS). Fifteen instances of early PT occurred between days 0 and 17 (median, day 2). Emergency thrombectomy was performed in 9 cases (successful in 5). Three children underwent a secondary portosystemic shunt (successful in 2). Three PS were cured by either surgery or balloon dilatation. Four children died, 3 are alive with portal hypertension (PHT), and 10 are alive without PHT. Three-year patient actuarial survival is 82.4% in PC cases and 82% in others (NS). Significant risk factors of PC are young age and weight at the time of LT, small portal vein, and emergency LT. Analysis of our own results and review of the literature suggest that prevention of PC depends primarily on appropriate surgical technique. Reduction of postoperative hypercoagulability may also play an important role: a meta-analysis of 1,257 published pediatric LT show an overall risk of PT of 2.2% in teams using aspirin with or without dipyridamole compared with 7.8% when no antiaggregative agents are given (P = .0001).

Ureteroureteral anastomosis in the treatment of reflux associated with ureteral duplication.

PURPOSE: Reflux is the most common pathological condition associated with ureteral duplication. Based on 18 cases of reflux associated with ureteral duplication that were managed by ipsilateral ureteroureteral anastomosis, we analyzed this method of treatment and its advantages. MATERIALS AND METHODS: Between 1978 and 1991, 19 ipsilateral ureteroureterostomies were performed in 18 children. No complications were noted perioperatively or postoperatively. Average 38-month followup was uneventful. RESULTS: End-to-side ipsilateral ureteroureterostomy behind the bladder is simple and safe with respect to bladder integrity, and so it decreases morbidity and hospitalization. CONCLUSIONS: Indications for this operation are well-defined and they must be recognized.

Determinants of life span after Kasai operation at the era of liver transplantation.

The aim of this work is to determine the influence of age, extrahepatic biliary lesions pattern (EHBP) and association to polysplenia syndrome (PS) on 10 years outcome of 164 patients with biliary atresia (BA) treated from 1984 to 1992 by initial Kasai operation (KO) and secondary liver transplantation (LT) when necessary. Actuarial crude survival without or after LT (CS), actuarial survival with native liver (NLS) and jaundice-free actuarial survival with native liver (JFS) were calculated from 1 to 10 years versus age (under/over 45 days), EHBP (favorable/ unfavorable) and PS (no/yes). Overall 10-year CS is 70%, overall 10-year NLS and JFS are 14%. In univariate analysis, age at KO under 46 days, favorable EHBP (BA with patent gallbladder, and/or cystic dilatation of extrahepatic bile duct, or BA restricted to choledocus), and absence of PS are significant determinants of a better outcome regarding CS, NLS and JFS. EHBP is more discriminant than age. Influence of PS in this series is redundant with that of EHBP since 11/11 patients with PS had unfavorable EHBP.

Urgent liver transplantation for biliary atresia: the experience in Bicêtre.

According to French rules for cadaver organ sharing, children with biliary atresia (BA) complicated with acute necrosis (ALN) can be registered on the waiting list for liver transplantation (LT) in a special intermediate grade urgent code. Over a 7 years period, 100 children have been submitted to elective LT for BA and 15 to urgent LT. Urgent procedures accounted for 25% of LT for BA in patients aged 0-2 years and 67% (8/12) in patients under 1 year of age. Children actuarial survival at 1, 12 and 48 months was respectively 66%, 60% and 60% versus 92%, 86% and 85%, deaths occurring earlier in the urgent group. Graft actuarial survival at 1, 12 and 48 months were 60%, 53% and 53% versus 85%, 77% and 76% (p < 0.05), respectively. Outcome of children and grafts after LT is not significantly different in BA cases and in other urgent indications, excluding retransplantations. In a LT program based on cadaver organ donation, allocation of in an urgent registration code to children with BA and ALN offers them more than 50% chance to escape death and does not result in wasting of grafts.

Spontaneous perforation of the biliary tract in infancy: a series of 11 cases.

Eleven patients presenting with spontaneous perforation of the biliary tract were treated at Bicêtre Hospital between 1971 and 1993. Three groups were individualised, each with a different pattern of local presentation: generalised biliary peritonitis (n = 2), localised biliary peritonitis (n = 4), secondary biliary stenosis (n = 5). In each case, cholestatic jaundice developed after a postnatal symptom-free interval. Ten patients were operated on. Perforation was located in the cystic duct (n = 2), at the junction of the cystic and hepatic ducts (n = 4), in the common hepatic duct (n = 1) or common bile duct (n = 1). The site of perforation was no longer identifiable in two cases with stenosis. A cholecystectomy was performed in the 2 cases with cystic duct perforation; in the cases of lesions of the main duct, either simple external biliary drainage (n = 3) or biliary reconstruction (n = 5) was carried out. Postoperative complications included bile leak (n = 2), ascending cholangitis (n = 1), portal vein thrombosis (n = 2). Five patients were submitted to further surgery including biliary revision (n = 3), porto-systemic shunt (n = 1), and other procedures (n = 2). One infant died from postoperative sepsis; 2 were lost to follow-up, one of which probably did not survive; 4 are alive and well. Late sequelae are present in 4 children: portal hypertension (n = 1), mild residual bile duct dilatation without cholestasis (n = 1), and mild to moderate liver fibrosis (n = 2). Prompt diagnosis and appropriate treatment should improve the prognosis of this rare condition.

Conventional treatment of biliary atresia: long-term results.

From 1968 to 1983, 271 patients were treated for biliary atresia by a group of surgeons from the same pediatric surgical unit, in Paris, using procedures adapted to the local anatomy and all derived from the Kasai technique. Eighty children have survived more than 10 years since the surgery, without the need for liver transplantation during the 10-year period. However, three children died subsequently from complications of the liver disease. Thirteen others later underwent liver transplantation, which accounted for three additional deaths. Thus, of the 64 patients left for study, 38 had a good result with respect to serum bilirubin level, but 18 of them still have symptoms of portal hypertension. Among another group of 14 patients with serum bilirubin levels between 18 and 36 mumol/L, 11 are leading a near-normal life. The mean follow-up period for this study is 14 years; the oldest patient is aged 24 years. One patient, already the mother of a normal son, is awaiting her second baby; she was treated by portocholecystostomy at 2 months of age. As a rule, liver transplantation should not be considered an alternative to the Kasai operation as initial treatment of biliary atresia. It may be the only form of treatment for survivors without jaundice, if survival becomes compromised by complications owing to portal hypertension or pulmonary shunts.

A novel mutation in the putative DNA helicase XH2 is responsible for male-to-female sex reversal associated with an atypical form of the ATR-X syndrome.

We describe a pedigree presenting X-linked severe mental retardation associated with multiple congenital abnormalities and 46,XY gonadal dysgenesis, leading in one family member to female gender assignment. Female carriers are unaffected. The dysmorphic features are similar to those described in the alpha-thalassemia and mental retardation (ATR-X) syndrome, although there is no clinical evidence of alpha-thalassemia in this family. In addition, the family had other clinical features not previously observed in the ATR-X syndrome, including partial optic-nerve atrophy and partial ocular albinism. Mutations in a putative DNA helicase, termed XH2, have been reported to give rise to the ATR-X syndrome. We screened the XH2 gene for mutations in affected members of the family and identified a 4-bp deletion at an intron/exon boundary that removes an invariant 3' splice-acceptor site. The mutation cosegregates with the syndrome. The genomic deletion causes missplicing of the pre-mRNA, which results in the loss of 8 bp of coding sequence, thereby generating a frameshift and a downstream premature stop codon. Our finding increases the range of clinical features associated with mutations in the XH2 gene.

Prenatal ultrasonographic detection of abdominal and pelvic tumors: impact on management.

An abdominal or pelvic tumor in very seldom detected in a prenatal ultrasound examination. The most commonly detected tumors are, in order of decreasing frequency, sacrococcygeal teratomas, neuroblastomas and kidney tumors. According to the present state of the art, diagnosis of a sacrococcygeal teratoma only leads to specific monitoring of the pregnancy, because an early delivery or delivery by cesarean section will be necessary in some cases to improve the prognosis of child and/or mother. For other types of tumors investigations and actions should be delayed until after birth.

[Liver transplantation in children]. / La transplantation hépatique chez l'enfant.

The need for paediatric liver transplantation, which in most paediatric series is the remedy for biliary atresia after Kasai's operation has failed, is not sufficiently covered by organ retrieval at the present time. In most cases, survival after liver transplantation in children is approximately 80%. Mortality is still high due to intra-operative complications in most cases. Morbidity is related to vascular complications as well as to different types of infections which occur in nearly all the children. After the initial period, growth in most children returns to normal with normal physical and intellectual development. There remain a number of questions concerning the future of liver transplantation in children. What are the limits for proper indications and contraindications? What is the risk of life-long immunosuppressive treatment? Are there alternative modalities of treatment other orthotopic liver transplantation? It must be admitted that before progress has provided answers to these questions and a better understanding of the aetiology of many congenital or metabolic diseases in children as well as a better means of treatment or prevention, that the need for liver grafts in children will continue to rise as a consequence of its own success. One point must be emphasized, since we are concerned with very young children, care should always be given by specially trained health care providers.

Liver retransplantation in children.

Between January 1988 and December 1993, 132 children received 154 orthotopic liver transplantation (OLT) at Bicêtre Hospital (France). Among them, 18 children underwent two and 2 children underwent three transplants (retransplant rate; 15.2%). Retransplantations were classified into three groups according to the interval between both OLTs: 1 (< 7 days, n = 8), 2 (8 days to 6 months, n = 10) and 3 (> 6 months, n = 4). Each group was compared with a control group whose grafts survived more than 1 week. There were no significant differences regarding age (35 +/- 49, 23 +/- 13, and 33 +/- 15 versus 49 +/- 41 months), cold ischemic time (566 +/- 105, 470 +/- 144, and 476 +/- 58 versus 455 +/- 110 minutes) and reduced size graft ratio (62.5%, 60%, and 75%, versus 58.3%). The causes of graft failure included primary nonfunctioning liver (PNF, n = 8) all in group 1 cases, biliary complications associated with hepatic artery thrombosis (HAT, n = 10; group 2, 6; group 3, 4), chronic rejection (n = 3; group 2, 1; group 3, 2) and three others (uncontrollable acute rejection, fulminant hepatitis, and secondary dysfunction, all in the group 2). The authors were unable to detect the cause of PNF. The overall survival rate after retransplantation was 62.5%, 60.0% and 75%, respectively. To reduce the rate of retransplants and improve the prognosis after OLT, further investigation into the cause of PNF, refinements in the anastomotic technique of the hepatic artery, and also early treatment of HAT are some of the goals to be achieved.

[Congenital dilatation of the common bile duct in children. Study of a series of 52 cases]. / Dilatation congénitale de la voie biliaire principale chez l'enfant. Etude d'une série de 52 cas.

OBJECTIVE: The aim of this study was to evaluate results at long-term follow-up of patients suffering from congenital dilatation of the bile duct according to the type of operative procedure. METHOD: A retrospective study of 52 patients who were operated on during a 25-year period was carried out. Of 52 patients, 43 (83%) were female and 9 (17%) were male, with a ratio of 4.8/1. In six cases, the diagnosis was made antenatally by ultrasonography. Transhepatic cholangiography showed an extrahepatic dilatation in 25 cases (48%) and a combined extra and intrahepatic dilatation in 27 cases (52%). A common bilio-pancreatic channel was found in 25 patients. Mean age at the time of operation was 4.2 years. Operative procedures included: cyst excision and hepaticojejunostomy in 47 cases, cystojejunostomy or cystoduodenostomy in 4 cases and cystostomy in one case. There were two postoperative deaths. RESULTS: Long-term follow-up was established in 43 of 50 survivors (80%). One patient was reoperated because of portal hypertension. Cholangitis developed in 11 patients: 2/2 patients who had undergone in internal drainage and 9/41 patients who had undergone a cyst excision with hepaticojejunostomy. In patients with cyst excision, cholangitis developed twice as frequently in those with associated intrahepatic dilatation. Two of 11 children with recurrent cholangitis died following sepsis, and three others were reoperated because of intrahepatic bile duct stones, one of these 15 years later. CONCLUSION: A total excision of the extrahepatic biliary tract, followed by hepaticojejunostomy is considered as a treatment of choice. The site of the biliary anastomosis should be adapted to the type of dilatation of intrahepatic bile duct in the case of a combined extra and intrahepatic dilatation.

Lessons from the first 100 liver transplantations in children at Bicêtre Hospital.

The authors report their experience with 100 liver transplantations at Bicêtre Hospital. From 1988 to 1991, 85 children received a total of 100 liver grafts (mean age, 44.4 months; two thirds were under 3 years of age). Fifty-four percent of the grafts were reduced-size. Cyclosporine, steroids, and azathioprine were used for immunosuppression. The actuarial survival rate at 4 years is 86%. Retransplantation was performed in 14 children (16%). Forty-four patients (49%) had another operation. Biliary complications (17%), hepatic artery thrombosis (HAT) (14%), and hemoperitoneum (14%) were the most frequent surgical complications. Retransplantation was avoided in 50% of the patients who underwent urgent artery revision for thrombotic complications. It appeared that ABO-incompatible were better tolerated in children without ABO alloantibodies at the time of transplantation. The survival rates of ABO-identical, -compatible, and -incompatible liver grafts did not differ (61%, 50%, and 57% respectively). The results suggest that an aggressive policy of reintervention, including retransplantation, is necessary to achieve a satisfactory survival rate and quality of life. Children lacking ABO alloantibodies at the time of transplantation might tolerate ABO-incompatible liver grafts better.

Outcome of ABO-incompatible liver transplantation in children with no specific alloantibodies at the time of transplantation.

The shortage of suitable liver donors for children has motivated the use of ABO-incompatible (ABO-I) grafts for transplantation in urgent situations. However, survival after ABO-I liver grafts has been reported at about 30% as compared with 80% in cases of ABO-identical or -compatible liver grafts. This difference has been attributed to antibody-mediated, hyperacute or chronic liver rejection, due to preformed ABO antibodies (alloantibodies). In this study, we report our results with ABO-I livers in children without alloantibodies at the time of transplantation. From January 1988 to June 1993, 143 OLT were performed in 122 children. Eight children received 8 ABO-I liver grafts. Of these, 7 patients were included in the study. All 7 were alloantibody free before OLT. Five children were spontaneously alloantibody free, while in 2 children, the plasma alloantibodies were eliminated before and after transplantation using intravenous infusion of specific blood group antigens of the donor blood group (soluble antigens). Immunosuppression consisted of a triple-drug treatment combining CsA, AZA, and steroids. The follow-up period was between 10 and 48 months. One child died from a surgical complication. Six children survived, but 1 died 10 months later from intestinal obstruction. There were no graft losses and no episodes of hyperacute or chronic rejection. The graft and patient survival rate was 71%. There was a 28% incidence of rejection, but all were mild (requiring steroid boluses only). Our results suggest that the absence of ABO alloantibodies at the time of and after transplantation can protect ABO-I liver grafts against antibody-mediated rejection, whether hyperacute or chronic, and that soluble antigens are effective in eliminating alloantibodies in children.