Fetal rhabdomyomatous nephroblastoma: a tumour of good prognosis but resistant to chemotherapy.

Fetal rhabdomyomatous nephroblastoma (FRN) is a rare variant of Wilms' tumour. The tumour chiefly consists of fetal striated muscle with particularly distinct striations and central nuclei. To determine the effect of (preoperative) chemotherapy in the treatment of this subtype of nephroblastoma, a retrospective analysis was performed. By 1 November 1991, SIOP 9 had registered 852 patients (pts) from 55 centres. We report on 13 children diagnosed with FRN between 1988 and 1992 with a median age of 2 years and 1 month (range 1 month-8 years 6 months). There were 7 boys and 6 girls. 9 patients were classified as stage I, 2 as stage II, 1 as stage III and 1 as stage V. 12 patients received preoperative chemotheraphy with actinomycin-D and vincristine for 2 weeks (1 pt), 4 weeks (5 pts) and 8 weeks (6 pts) respectively. The average tumour volume at registration (determined by ultrasonography) in 12/13 patients was 965 cm3 (range 17.3-2520 cm3). 3/7 of the FRN patients showed no tumour regression after 4 weeks preoperative CT and 4/8 after 8 weeks preoperative chemotheraphy (compared with only 28 and 34%, after 4 and 8 weeks CT, of all trial patients). Of 13 patients, 10% are alive and free of disease with a mean follow up of 4 years. This variant of Wilms' tumour is a poor responder to preoperative chemotherapy and is associated with a generally favourable outcome in most of all unilateral cases when treated by surgery.

Kidney transplantation in patients with Wilms' tumor.

We report our experience of renal transplantation in three patients treated for Wilms' tumor (with lung metastasis in two of them), and review 26 previously reported cases in order to define the current indications of transplantation in this setting. Our patients, aged 5-12 years, were transplanted 13-95 months after completion of Wilms' tumor treatment. All three are alive and tumor-free, two with a functioning graft 20 and 97 months after transplantation. Two findings emerge from the review of the literature. First, posttransplant mortality is influenced by the delay between completion of tumor treatment and transplantation. Mortality reaches 79% when that delay is less than one year but falls to 27% when that delay exceeds one year. Second, the prognostic value of pretransplant metastasis depends on its location. All four patients with pretransplant abdominal metastasis died with active metastatic disease. By contrast, of three patients treated before transplantation for metastasis confined to the lung, two are alive and tumor free. We conclude that renal transplantation should be offered to patients successfully treated for Wilms' tumor for at least one year, even if the disease has been complicated by pulmonary metastasis. Several long-term survivors attest that the disease can be cured even under maintenance immunosuppression.

Bone marrow transplantation in sickle cell disease: the Belgian experience.

Twenty eight patients underwent bone marrow transplantation (BMT) for sickle cell anemia in Belgium. The patients were originating from Central Africa, were young and symptomatic. Engraftment occurred in all patients and was sustained in 25. In 3 patients, a bone marrow rejection was observed. One of these patients underwent a second BMT and had an uneventful recovery. One patient died 3 months after BMT of graft-versus-host disease (GVHD). Twenty seven patients are alive and 25 are free of vaso-occlusive related manifestations with a follow-up ranging from 4 to 78 months. So far, eight patients went back to Africa and continue to do well.

Severe idiopathic congenital aplastic anemia: role of hematopoietic growth factors.

A baby boy with constitutional aplastic anemia refractory to treatment with hematopoietic growth factors underwent BMT from an unrelated HLA-compatible donor. The BM failed to engraft but the combination of a conditioning regimen, immunosuppression and hematopoietic growth factors resulted in a recovery of the patient's own BM and a good outcome.

Bone marrow transplantation from an unrelated donor for Fanconi's anaemia: two unusual complications.

An eight-year-old boy with Fanconi's anaemia received a bone marrow transplantation from an unrelated donor. Sustained engraftment was achieved, but he developed major complications including acute grade III graft-versus-host disease, autoimmune haemolytic anaemia with positive Coombs' test and free antibodies against Rhesus group, and a virus-associated haemophagocytic syndrome; this latter complication was successfully treated with acyclovir. Sixteen months after transplantation he has full haematopoietic reconstitution, but with persisting mild autoimmune haemolysis.

Haematopoietic stem cell transplantation for sickle cell anaemia: the first 50 patients transplanted in Belgium.

Fifty patients affected by sickle cell anaemia underwent transplantation of HLA-identical haematopoietic stem cells (bone marrow, 48; cord blood, 2). Two groups of patients were considered for transplantation. Group 1 included 36 permanent residents of a European country who, retrospectively, met the inclusion criteria accepted at a consensus conference held in Seattle in 1990, wherein children were selected because they already had evidence of a morbid course. Group 2 included 14 patients who were transplanted earlier, had not received more than three blood transfusions and were transplanted because they had decided to return to their country of origin. Kaplan-Meier estimates of overall survival, event-free survival and disease-free survival at 11 years of the whole grafted population are 93, 82 and 85%, respectively. In group 1, overall survival, EFS and DFS were 88, 76 and 80% and in group 2, 100, 93 and 93%, respectively. Clinical manifestations of the disease, as well as disease associated haemolytic anaemia, disappeared in all successfully treated patients. Recovery of spleen function was present in seven out of 10 evaluated patients. Adverse events (death, absence of engraftment, mixed chimerism and relapse) occurred more frequently in group 1 than in group 2 (25% vs 7%, P< 0.001). Acute graft-versus-host disease (GVHD) was present in 20 patients (grade I or II, 19; grade III, 1), chronic GVHD in 10 (limited, 7; extensive, 3). One patient developed an acute myeloid leukaemia. Gonadal dysfunction was present in all patients (six boys and eight girls) transplanted close to or after puberty, although transient in one adolescent girl.

"Spontaneous" erythroid progenitor cells in the circulation and monosomy 7 in juvenile chronic myelogenous leukemia.

Cytogenetic examination of bone marrow cells from two patients with juvenile chronic myelogenous leukemia (JCML) showed a monosomy 7. Peripheral blood from these two children was found to contain large numbers of cells capable of erythroid colony formation in tissue culture without added erythropoietin. We conclude that erythroid progenitor cells are part of the malignant clone in JCML.

High dose melphalan in children with advanced malignant disease. A pharmacokinetic study.

Nine children with poor-prognosis malignancies--seven with advanced neuroblastoma and two with metastatic Ewing's sarcoma--were given high doses of melphalan (HDM), 150 mg/m2 (3 patients) and 180 mg/m2 (6 patients), as a 'late intensification' agent combined with noncryopreserved autologous bone marrow transplants. Melphalan levels in the plasma decreased biphasically, with mean half-lives of 6.6 min and 3.0 h. At the time of marrow reinfusion (12-21 h after HDM) the melphalan plasma level was generally below 0.1 microgram/ml. The renal contribution to melphalan clearance was low, a mean of 5.8% of the injected dose being found in patients' urine over the 12 h following HDM administration. No significant difference was seen in pharmacokinetic parameters between patients undergoing and not undergoing forced diuresis.

Alkylating activity in serum, urine, and CSF following high-dose ifosfamide in children.

The pharmacokinetics of alkylating activity were studied in 17 children treated i.v. with ifosfamide (IF) at 3 g/m2 as a 1-h infusion for 2 consecutive days every 3 weeks, with mesna as a uroprotector. Two patients were treated for a newly diagnosed rhabdomyosarcoma according to the current SIOP (International Society of Pediatric Oncology) protocol. The other 15 patients were treated in a phase II study and presented with one of the following malignancies in relapse: neuroblastoma (7), osteosarcoma (3), soft tissue sarcoma (2), Wilms' tumor (1), non-Hodgkin's lymphoma (1), and acute lymphoblastic leukemia (1). Plasma alkylating activity levels determined by using 4(4'-nitro-benzyl)-pyridine showed considerable inter-individual and intercyclic variations and decreased biphasically, with mean alpha and beta half-lives of 60 min and 6-7 h, respectively. Probably as a result of liver mixed-function oxidase induction, on the 2nd day of treatment the terminal half-lives were shorter, the plasma exposures were lower, and the mean plasma clearances were higher. Renal excretion was almost complete after 24 h, accounting for a mean of 19% of the injected dose. The CSF alkylating activity levels, obtained in four children, were always lower than the plasma levels and ranged from 8 to 51 micrograms/ml, with a mean CSF/plasma ratio of 0.53 +/- 0.23 during the first 12 h. We conclude that IF alkylating activity was biphasically cleared from the plasma, with significant interindividual and intercyclic variability, that the renal contribution to the clearance was low, and that high levels of CSF alkylating activity could possibly contribute to the CNS toxic side effects observed in pediatric patients treated with high-dose IF/mesna.

Health care workers: vectors of influenza virus? Low vaccination rate among hospital health care workers.

The influenza vaccination rate among health care workers is not very high and needs to be improved. The authorities of our university hospital decided to encourage vaccination among health care workers and to include it in the hospital quality of care program. A survey about knowledge of influenza disease and its vaccination was undertaken, and 37.2% of the workers participated in the survey. Among them, 31.8% were vaccinated. The vaccination rate was higher when workers were older, men, physicians, technicians, or laboratory workers, vaccinated in the previous flu season and had fewer contacts with patients. The variables with the highest impact on the vaccination rate were vaccination the previous year, belief that vaccination is highly effective, and affirmation that prevention is important. Our study shows that an active vaccination campaign is also necessary for health workers to put them in the stream of usually vaccinated people.

[Eruptive fever of rare cause: familial hemophagocytic lymphohistiocytosis]. / Une fièvre éruptive de cause rare: la lymphohistiocytose hémophagocytaire familiale.

BACKGROUND: Clinical onset of familial lymphohistiocytosis is non-specific so that the diagnosis of this rare and severe disease is difficult. CASE REPORT: An 8 week-old girl was admitted suffering from fever and rash. She had hepatosplenomegaly. She developed pancytopenia (Hb: 6.6 g/100 ml; WBC: 4500/mm3; platelets: 25,000/mm3) impaired liver function tests (prothrombin: 15%, blood bilirubin: 40 mg/l; SGOT: 160 mU/ml) and hypofibrinogenemia (0.3 g/l) within a few days. Bone marrow examination showed diffuse histiocytic infiltration and erythrophagocytosis, suggesting a syndrome of inappropriate macrophage activation. The age of the patient, parental consanguinity and absence of specific infection led to diagnosis of familial erythrophagocytic lymphohistiocytosis. The patient died 18 days after clinical onset. CONCLUSION: The diagnosis of this unusual syndrome in infants is strongly supported by parental consanguinity as seen in our case or a positive family history. In this condition, erythrophagocytosis is often a marked feature.

[Combined hereditary deficiency in factors VII and X revealed by a prolonged partial thromboplastin time]. / Déficit héréditaire combiné en facteurs VII et X révélé par un allongement du temps de Quick.

BACKGROUND: Congenital factors VII and X deficiency is rare. Association of both deficiencies is exceptional. CASE REPORT: A 3 year-old boy, born to consanguinous Moroccan parents, had a prolonged partial thromboplastin time discovered fortuitously. This finding led to the diagnosis of combined factors VII and X deficiency. His siblings had the same deficiencies. CONCLUSION: Profound deficiencies in factors VII and X are inherited following an autosomal-recessive mode. These deficiencies may be asymptomatic, only discovered by prolonged partial thromboplastin time. They may also be revealed by intracranial bleeding and other severe hemorrhages. Treatment consists of administration of factor VII or PPSB.

[Antithrombin deficiency and thrombosis in a young child]. / Déficit en antithrombine et thromboses chez le jeune enfant.

BACKGROUND: Thromboses represent a rare event in children and may be due to a deficiency of antithrombin. CASE REPORT: A 10-year-old boy developed thrombosis due to a congenital quantitative deficiency in antithrombin, confirmed by molecular biology. His father was diagnosed with the same deficiency. The child was first treated with heparin and is now on antivitamin K. He is well 26 months after diagnosis. CONCLUSION: When a young patient presents with a thrombotic event, a congenital deficiency in one of the inhibitors of coagulation, one of which is antithrombin, should be looked for and the condition treated as soon as possible.

[Reversal of urinary diversion after radical surgery for rhabdomyosarcoma of the bladder]. / Dé-dérivation urinaire après chirurgie radicale pour rhabdomyosarcome de la vessie.

The authors report a case of reversal of urinary diversion in a 14 year old boy who had presented, three years previously with an embryonal rhabdomyosarcoma of the base of the bladder. The initial treatment had consisted of preoperative and postoperative chemotherapy combined with radical cystoprostatectomy with urinary diversion via a colonic conduit. The principle of the technique of reversal of urinary diversion are discussed in the light of this unusual case.

[The role of ultrasonography in abdominal pain in children in the emergency room]. / La place de l'échographie dans la douleur abdominale de l'enfant en salle d'urgences.

Our retrospective study aims at defining the influence of ultrasonography on the management of acute abdominal pain in a pediatric population. This technique appears to be essential in the diagnosis of intussusception and thus, should be recommended in all cases of acute abdominal pain in the population at risk for this disorder. On the other hand, ultrasonography only plays a minor role in the management of the other diseases we observed, especially in acute appendicitis.

A multicentre study of fluconazole versus oral polyenes in the prevention of fungal infection in children with hematological or oncological malignancies. Multicentre Study Group.

A randomized, comparative study was conducted in 502 patients in 23 centres world-wide to assess the efficacy and safety of fluconazole versus nystatin and amphotericin B for prevention of fungal infection in a severely immunocompromised pediatric population. Patients scheduled within 48 hours to undergo chemotherapy or radiotherapy for hematological or oncological malignancies were randomly allocated to receive 3 mg/kg oral fluconazole once daily, 50,000 U/kg oral nystatin four times daily or 25 mg/kg oral amphotericin B four times daily. Prophylaxis began with the initiation of chemotherapy or radiotherapy and continued throughout a patient's hospital stay or period of neutropenia as necessary. The mean duration of fluconazole prophylaxis was 27.8 days and of the oral polyenes 29.2 days. The outcome of prophylaxis with fluconazole was significantly superior to that with the polyenes (p = 0.01). Mycologically verified infections occurred in 5 patients (2.1%) given fluconazole and in 21 (8.4%) given polyenes (p = 0.002). Clinical evaluation at the end of prophylaxis showed that the clinical outcome was definitely or possibly successful in 87% in the fluconazole group and 82% in the polyenes group with no significant differences between the treatment groups. Mycological evaluation demonstrated reduction or control of colonization in 84% in the fluconazole group and 85% in the polyenes group, again with no significant between-group differences. Possibly drug-related side effects, mainly mild to moderate gastrointestinal disturbances, were reported in 38 patients given fluconazole, with eight subsequent withdrawals, and in 21 patients given oral polyenes, with three subsequent withdrawals. Laboratory test abnormalities occurred in 28 patients given fluconazole and 24 given polyenes.(ABSTRACT TRUNCATED AT 250 WORDS)

AIDS-resembling disease in a non-HIV-infected African born to an HIV-positive mother.

We report the case of a 10-month-old boy born to an HIV-positive mother who presented with symptoms compatible with AIDS. However, he became HIV-negative and his condition was probably due to combined cytomegalovirus and Epstein-Barr virus infections.