Sources of error in measurement of minimal residual disease in childhood acute lymphoblastic leukemia.

Introduction: The level of minimal residual disease (MRD) in marrow predicts outcome and guides treatment in childhood acute lymphoblastic leukemia (ALL) but accurate prediction depends on accurate measurement.

Methods: Forty-one children with ALL were studied at the end of induction. Two samples were obtained from each iliac spine and each sample was assayed twice.

Elective administration in infants of low-dose recombinant activated factor VII (rFVIIa) in cardiopulmonary bypass surgery for congenital heart disease does not shorten time to chest closure or reduce blood loss and need for transfusions: a randomized, double-blind, parallel group, placebo-controlled study of rFVIIa and standard haemostatic replacement therapy versus standard haemostatic replacement therapy.

We investigated the effectiveness of prophylactic administration of recombinant activated factor VII (rFVIIa) for cardiopulmonary bypass surgery in children under 1 year old with congenital heart disease (CHD) in a double-blinded, placebo-controlled study. The rFVIIa dose was 40 microg/kg and all patients also received standard haemostatic replacement therapy. The primary endpoint was the time to chest closure from neutralization of heparin with protamine sulphate as this could be most objectively and accurately measured during surgery.

Factor V Leiden mutation: a contributory factor for cerebral palsy?

Fifty-seven children with cerebral palsy (CP) and imaging evidence of vascular thrombosis (study group) and 167 children with CP and other imaging finds (control group)were selected. Sixty-one per cent of the study group were male and 53 (93%) had spastic hemiplegia compared with the control group, of whom 55% were male and 54 (32%) had a diagnosis of spastic hemiplegia. Mean age was 5 years 11 months (SD 5y 1mo) for the study group and 7 years 7 months (SD 4y 7mo) for the control group.

Spontaneous liver hemorrhage during laparotomy for necrotizing enterocolitis: a potential role for recombinant factor VIIa.

Necrotizing enterocolitis remains a serious condition in very low birth weight infants, particularly in those infants who require surgery. Perioperative hemorrhage is a potentially fatal complication in this population. We describe our experience in 4 premature infants with necrotizing enterocolitis who received recombinant factor VIIa to manage life-threatening intraoperative hemorrhage.

Intended management of children with acute idiopathic thrombocytopenic purpura: a national survey.

Objective: In Australia acute idiopathic thrombocytopenic purpura (ITP) is mainly treated by paediatricians (either general paediatricians or paediatric haematologists/oncologists). A survey was conducted to gauge the current practice of treating children with acute ITP in Australia.

Methods: All practising Australian paediatricians registered by the Royal Australasian College of Physicians were surveyed regarding their intended management of children with acute ITP.

Impairments of attention following treatment with cranial irradiation and chemotherapy in children.

Neurobehavioral impairments are frequently reported following treatment for childhood cancers, with cranial irradiation (CRT). This study investigated attention and information processing skills, predicting that these skills would be impaired due to the vulnerability of cerebral white matter in early childhood. Three treatment groups were studied: (i) CRT+chemotherapy (n = 35); (ii) chemotherapy alone (n = 19); (iii) healthy children (n = 35).

Reduced bone density among children with severe hemophilia.

Objective: Children with severe hemophilia are at risk for reduced bone mineral density (BMD) because of reduced weight-bearing exercise and hepatitis C infection. Reduced bone density in childhood is a risk factor for osteoporosis in later life.

Study Design: We performed a cross-sectional survey of bone density among 19 children with severe hemophilia, at the Royal Children's Hospital.

Cost-utility analysis of recombinant factor VIIa (NovoSeven) in six children with long-standing inhibitors to factor VIII or IX.

The high cost of treating patients with inhibitors in an environment of restricted budgets warrants consideration of cost-effectiveness. We determined the clinical response, effect on quality of life and the cost-effectiveness of treatment with rFVIIa in six boys with long-standing inhibitors to factors VIII or IX, compared with other treatment regimes previously used in these patients. The study used a longitudinal before-and-after design and was conducted in three phases.

Induction of immune tolerance and suppression of anaphylaxis in a child with haemophilia B by simple plasmapheresis and antigen exposure.

Anaphylaxis to factor IX (FIX) in patients with haemophilia B is a rare and life-threatening complication that has been reported to occur in association with the development of inhibitors to FIX. Management of these patients is difficult. This report presents an 18-month-old boy with a frame-shift mutation of the FIX gene and FIX coagulant level of <1% who developed anaphylactoid reactions to low and high purity plasma-derived FIX concentration infusions and an inhibitor measuring 1.

Cognitive and academic outcome following cranial irradiation and chemotherapy in children: a longitudinal study.

Cranial irradiation therapy (CRT) and chemotherapy are associated with neurobehavioural deficits. Many studies have investigated late effects of these treatments, but few have evaluated changes in abilities over time. This study employed a longitudinal design to map abilities following these treatments.

High incidence of treatment failure with vincristine, etoposide, epirubicin, and prednisolone chemotherapy with successful salvage in childhood Hodgkin disease.

Background And Procedure: In an attempt to further reduce the long-term toxicity of chemotherapy for childhood Hodgkin disease (HD), the Australian and New Zealand Children's Cancer Study Group between 1990 and 1996 enrolled 53 children with biopsy-proven and imaging-staged HD into a chemotherapy-only treatment regimen using 5-6 courses of vincristine, etoposide, epirubicin, and prednisolone (VEEP).

Results: There were 23 events in these children with 3 progressive disease (PD), 8 partial remissions (PR), and 12 relapses. In the stage I patients, there were 8 events (35%).

Cross-reactivity to porcine factor VIII of factor VIII inhibitors in patients with haemophilia in Australia and New Zealand.

Background: Inhibitory antibodies which neutralise factor VIII develop in 10-20% of individuals with inherited haemophilia A and rarely as autoantibodies in normal individuals to cause acquired haemophilia. The antibodies are directed against human factor VIII but cross-react to varying degrees with porcine factor VIII. Porcine factor VIII can be used for treatment in individuals with low cross-reactivity.

Effect of the Philadelphia chromosome on minimal residual disease in acute lymphoblastic leukemia.

The Philadelphia translocation is associated with a poor prognosis in adults and children with acute lymphoblastic leukemia, even though the majority of patients achieve remission. To test the hypothesis that the translocation leads to drug resistance in vivo, we studied 61 children and 20 adults with acute lymphoblastic leukemia and used the level of minimal residual disease at the end of induction as the measure of drug resistance in vivo. In children the presence of the translocation was associated with a significant increase in residual disease, indicating higher drug resistance in vivo; five of seven Philadelphia-positive children but only five of 54 Philadelphia-negative children had a minimal residual disease level >10(-3), a level which is associated with a high risk of relapse in childhood acute lymphoblastic leukemia of standard risk.

Neurobehavioural sequelae following cranial irradiation and chemotherapy in children: an analysis of risk factors.

Neurobehavioural deficits are commonly reported following treatment for childhood cancers. This study examined the impact of cranial irradiation (CRT) and chemotherapy in children, aiming to identify factors detrimental to long-term outcome. The study compared survivors of acute lymphoblastic leukemia (ALL), treated with CRT and chemotherapy (CRT group: n = 100), survivors of cancers treated with chemotherapy only (n = 50) and healthy controls (n = 100) for intelligence, academic achievement, information processing, learning, and executive function.

Coexistent T-cell lymphoblastic lymphoma and an atypical myeloproliferative disorder associated with t(8;13)(p21;q14).

This report describes a neoplasm exhibiting both lymphoid and myeloid differentiation associated with an acquired balanced translocation between chromosomes 8 and 13 occurring in a 10-year-old boy. Serial lymph node biopsies revealed the presence of both lymphoblastic lymphoma and an atypical myeloproliferative disorder within the same node. Immunophenotyping was consistent with the presence of an immature T-cell population within the nodal biopsy specimens.