Clinical course and outcome of children with single ventricle physiology at King Chulalongkorn Memorial Hospital.

BACKGROUND: Children with single ventricle physiology comprise 10% of all children with congenital heart disease (CHD) and one-third of children with cyanotic CHD seen at King Chulalongkorn Memorial Hospital. The prognosis of these children is generally thought to be poor but no study of the outcome has previously been done in this hospital and in Thailand. OBJECTIVES: To study the clinical course and outcome of children with single ventricle physiology at King Chulalongkorn Memorial Hospital during the current era. MATERIAL AND METHOD: One hundred and seventeen patients with single ventricle physiology were initially seen at this hospital during the year 1999-2001. Retrospective chart reviews were carried out in 90 children with available medical records. The status of the patients was determined in 2003 at clinic visits, by phone calls and mail. RESULTS: The main diagnoses were tricuspid atresia (TA, n = 10), pulmonary atresia with intact ventricular septum (PA/IVS, n = 16), single ventricle associated with cardiac malposition or heterotaxy syndrome (malposition, n = 35), hypoplastic left heart syndrome (HLHS, n = 11) and other complex single ventricle (others, n = 18). Most children did not have other major anomalies. Survival of patients with TA and PA/IVS was approximately 92 and 87% at 1 and 4 years, respectively. For patients in the other three groups, 1 and 4 year survival was 69 and 42%, respectively. Patients with HLHS had the worse survival, partly because of decisions not to pursue further treatments by the parents. Among HLHS patients who underwent Norwood procedure, the 1 and 4 year survival were 83% and 42%, respectively. Approximately 40 and 90% of all patients underwent heart surgery at 1 and 4 years follow-up, respectively. CONCLUSION: Despite limited resource, the short-term outcome of Thai children with single morphologic left ventricle (TA and PA/IVS) is reasonably good. Timely evaluation and treatment of these children toward Fontan procedure should be ensured. After proper discussion with the parents, palliative care is still acceptable for patients with single morphologic right ventricle or other complex single ventricle due to poor survival in the current era. As access to health care improves, re-evaluation of these outcomes is necessary to find the best strategy for the management of these patients.

A cost-benefit analysis of intravenous immunoglobulin treatment in children with Kawasaki disease.

To determine the long-term cost-benefit of intravenous immunoglobulin (IVIG) treatment in Children with Kawasaki Disease (KD), a model was made to compare the total cost for management of these children with and without the use of IVIG. Long-term (10-21 years) follow-up of 594 KD patients treated in the pre-IVIG era reported by Kato, et al. was used to calculate cost using previous cost studies from Chulalongkorn Hospital. Reduction of CAA from 25 per cent to 4 per cent with IVIG treatment was assumed based on previous published data. Total cost was slightly lower for the non-IVIG treatment group compared to the IVIG treatment group (33,451,129 baht vs 35,001,195 baht) for the duration of follow-up in Kato's model. Cost per effectiveness analysis showed more effectiveness in the IVIG treatment group (359,576 baht vs 383,614 baht). Net cost analysis similarly demonstrated lower costs in the IVIG treatment group (25,365,215 baht vs 33,451,129 baht). Incremental cost-effectiveness analysis demonstrated supplementary costs of 13,663 baht for one case in the reduction of coronary involvement and 387,517 baht for one life saved in the IVIG-treated group. Estimation of total costs for follow-up and treatment for healthy life (until 60 years old) was more expensive in the non-IVIG treatment than the IVIG treated group (75,482,803 baht vs 29,883,833 baht). The authors conclude that treatment of all KD cases in Thailand with IVIG is likely to result in lower cost and better outcome when compared to no treatment with the IVIG policy.

Clinical, pathological, and electron microscopic findings in two Thai children with Pompe disease.

The authors report on a Thai boy who first presented at age 7 months and an unrelated Thai girl in her neonatal period with hypotonia, cardiomegaly and hepatomegaly. Their chest roentgenograms showed markedly enlarged hearts, EKGs showed abnormally shortened PR intervals with gigantic QRS complexes, and electron microscopic studies of their skin samples showed glycogen accumulations surrounded by membranes. The boy died at age 22 months and the girl at age 9 months due mainly to cardiorespiratory failure. Autopsy of the girl showed marked accumulation of glycogen in the liver, heart and numerous additional tissues including her brain. The clinical, pathological, and electron microscopic findings of these two children are consistent with the diagnosis of Pompe disease. Pompe disease is an autosomal recessive disorder of glycogen metabolism resulting from deficiencies in activity of the lysosomal acid alpha-glucosidase. Definite diagnosis of the disease can be made from a biochemical test or a mutation analysis. To the authors' knowledge, no service laboratories in Thailand offer the tests. Because Thai children have occasionally been reported to be affected by Pompe disease, an attempt to establish a definite diagnostic test for Pompe disease in Thailand should be encouraged. With a definite diagnosis, the proper genetic counseling and prenatal diagnosis could be offered to the families.