Successful allogeneic peripheral blood stem cell transplantation in Wiskott-Aldrich Syndrome Patients: first report in Thailand.

Wiskott-Aldrich syndrome (WAS), an X-linked recessive disorder, is characterized by primary progressive T cell immunodeficiency, impaired antipolysaccharide antibody production, eczema, and thrombocytopenia. Stem cell transplantation is the only curative therapy. To evaluate the use of allogeneic peripheral stem cell transplantation (PBSCT) in this group of patients, we performed allogeneic PBSCT in two WAS patients (3 and 12 years old). The conditioning regimen consisted of busulfan 4 mg/kg/day for 4 days, and cyclophosphamide 50 mg/kg/day for 4 days. Graft-versus-host disease prophylaxis was consistent with cyclosporin A and methotrexate. Peripheral blood stem cells were collected from their brother donors (6 and 16 years old) by continuous flow leukapheresis after mobilization with granulocyte-colony-stimulating factor at a dose of 7.5 microg/kg/day for 5 days. Both recipients achieved neutrophils engraftment on days 11 and 12. The first patient achieved platelets engraftment on day 30. The second patient did not have platelet count below 20.0 x 10(9)/l during PBSCT procedure. Both did not develop acute or chronic graft-versus-host disease. At present, they are healthy after PBSCT. The follow up time after transplantation is 1,170 days and 269 days, respectively. Allogeneic PBSCT is economically feasible for WAS. The cost of PBSCT in Thailand is 20 to 30% less than bone marrow and cord blood stem cell transplantation. The cost of the transplant procedure for each patient in Thailand is US $ 12,000. This study is the first report of a successful stem cell transplantation in WAS patients in Thailand.

Thromboembolic complications in Thai pediatric patients.

Sixty patients with thromboembolic complications from 1987 to 1997 at the Department of Pediatrics, Ramathibodi Hospital were retrospectively studied. Twenty patients were infants and 40 patients were children and adolescents with a mean age of 18 days and 8 years, respectively. The sites of thromboembolic complications were in the central nervous system, 27.5 per cent; skin as purpura fulminans or necrotic lesions, 24.5 per cent; gangrene of the toe, finger or colon, 19 per cent; deep vein thrombosis, 16 per cent; and other sites such as heart and lungs, 13 per cent. Most of them had triggering conditions (80%) and underlying diseases (76.7%) causing thromboembolism. The low levels of either antithrombin III, protein C or protein S were found in 42 per cent (15/36). The management included administration of standard or low molecular weight heparin if not contraindicated, replacement of fresh frozen plasma 10 ml/kg twice a day and treatment of underlying and triggering conditions. The fatality rate was 15 per cent (9/60). Subsequent episodes of thromboembolism occurred in 6 patients including: pulmonary emboli in one patient with protein C deficiency who refused warfarin administration, deep vein thrombosis in 2 patients with unidentified etiology, and necrotic skin lesions in 3 patients with vasculitis who did not respond to treatment. In conclusion, a comprehensive investigation and specific treatment for patients with thromboembolic complications are emphasized in order to prevent recurring episodes.

Neonatal lupus erythematosus in Thailand.

Systemic lupus erythematosus (SLE) is not uncommon in Thai children and adults but neonatal lupus erythematosus has never been reported in Thailand. A case of NLE born of a mother without prior history of SLE was reported. He was initially misdiagnosed as congenital self-healing histiocytosis because of seborrheic dermatitis-like lesions, petechiae, hepatosplenomegaly and thrombocytopenia. Features supporting the diagnosis of NLE in this case were presented.

Childhood trauma, country report (Thailand).

In Thailand, each year approximately 15,000 people die from accidents, a figure exceeded only by cardiovascular diseases. Motor vehicle accidents (MVA) is the principal cause of death and injuries in children of all ages except preschool group. In 1988 there were 554,452 cases of injuries out of which 10,031 died from MVA. In Bangkok alone MVA accounts for an average of 300 childhood and teenage death and 1,900 cases of injuries each year. Falls, burns and poisonings are relatively high in children less than 4 years old while MVA and injuries from sharp and blunt objects of both accidental and non-accidental cases increase with age and become the two leading causes of injury in older children. The sex ratio (F:M) in preschool ages are 1:1.4 and 1:2 in preteen. Poisonings, though less common, are considered to be very important because of their severity. Drugs, hydrocarbon, insecticides, lead and corrosive substances are leading agents. As for injuries caused by animals, 150 cases of rabies were reported each year while around 5,000 cases of snake bites were found in 1987 and 20 per cent of the victims were children. The study from Ramathibodi Hospital revealed that the majority of accidents (65-72%) occur at home and 20 per cent in the street in children younger than 12 years. Peak incidence were found between 5-9 pm. During weekend and holidays the incidence is higher. Ninety-five per cent of the accidents reported were mild cases, 15 per cent moderate, 3 per cent severe and less than 1 per cent caused death.