Linear growth in homozygous beta-thalassemia and beta-thalassemia/hemoglobin E patients under different treatment regimens.

The effects on linear growth and development among thalassemic patients under different treatment regimens were compared. Twelve homozygous beta-thalassemia (homozygous beta-thal) and 36 beta-thalassemia/Hb E (beta-thal/Hb E) were studied longitudinally between 1977 and 1998. Eighteen cases (10 homozygous beta-thal and 8 beta-thal/Hb E) received hypertransfusion with iron chelation by desferrioxamine. Another 30 cases (2 homozygous beta-thal and 28 beta-thal/Hb E) were given a low transfusion (depending on their clinical requirement). Their heights were measured serially and are presented as a standard deviation score (SDS). There was no significant difference in initial basic hematological data and ferritin levels between either group. However, the hypertransfused group, seemed to be clinically more severely affected than the other group as evidenced by early age at initial transfusion, the early onset of anemia and diagnosis and also their large acquired iron load after a period of transfusion. The average height SDS of the hypertransfused patients was within the 50th percentile +/- 1 SD during the first decade of life in both sexes and both genotypes. Whereas, in patients who were transfused infrequently, the SDS was always below the -1 SD and decreased gradually. In severe beta-thal/Hb E cases, their growth SDS showed no difference from those with homozygous beta-thal. Normal linear growth in those with homozygous beta thal and severe beta-thal/Hb E was only seen in the group that underwent hypertransfusion and this regimen contributed to normal growth during the first ten years of life. However, adequate iron chelation and hormonal treatment in these patients were also required in order to achieve normal adult height.

Penicilliosis-associated hemophagocytic syndrome in a human immunodeficiency virus-infected child: the first case report in children.

Infection-associated hemophagocytic syndrome (IAHS) has been found in many systemic infectious conditions with a high mortality rate. Disseminated Penicillium marneffei infection is a common opportunistic condition among HIV-infected patients in many regions in Southeast Asia. We report the first case of IAHS caused by penicilliosis in an HIV-infected child who presented with cytopenias and recovered promptly after antifungal and intravenous immunoglobulin therapy.

Successful prophylaxis of intracranial hemorrhage in infants with severe congenital factor VII deficiency.

During the period 1984-1992, 2 severe cases (1 male, 1 female) of congenital F VII deficiency with intracranial hemorrhage (ICH) were referred to the Department of Pediatrics, Siriraj Hospital Bangkok, Thailand at the ages of 1 and 3 months old. They both responded very well to fresh frozen plasma (FFP) transfusion therapy. Subsequently, both had repeated episodes of ICH (repeated ICH) 5 and 6 times, despite the 10-14 days of replacement therapy for each episode and eventually died at the ages of 11 and 13 months. Since September 1996, another 2 severe cases (2 females) of congenital F VII deficiency who had ICH within their first month of life were referred to us. In order to prevent repeated ICH, we started a prophylactic regime after the second episode of ICH, by giving FFP 10 ml/kg twice a week. The average duration of follow up was 21 months (at 8 and 34 months). All of them (aged 14, and 38 months old) are doing well at this time and free from repeated ICH. From this observation, if there is FFP available, this regime is an effective way to prevent repeated ICH in infants with severe congenital Factor VII deficiency.

Gaucher's disease;thirty-two years experience at Siriraj Hospital.

Gaucher's disease, a lysosomal disorder, is not a common disease in Thailand. During the period 1966-1998 we saw 20 patients with Gaucher's disease at the Department of Pediatrics. Siriraj Hospital. The patients came from different regions of the country but mostly from the central part of Thailand. There were 8 males and 12 females from 13 families of Thai, Thai-Chinese, Thai-Laos and Chinese-Chinese in origin. A history of consanguinity was present in 2 families. The age of onset was 2 months-4 years and the age when they were diagnosed was 4 months-15 years. The most common clinical features included splenomegaly, hepatomegaly, growth retardation, pallor, bleeding disorders and neurological abnormalities. The diagnosis was made by the clinical manifestations, hematologic complications and demonstration of Gaucher cells in the bone marrow and/or other tissues. In one family, the diagnosis was confirmed by evaluation of glucocerebrosidase activities in skin fibroblasts. The management of these patients was symptomatic ie packed red cell and platelet transfusion, splenectomy and other supportive measures. Most patients died of bleeding or infection at an early age.

Clostridium difficile infection in febrile neutropenic malignancy children.

C.difficile infections were studies in 30 febrile neutropenic malignancy children with diagnoses of ALL (15), AML (9), non-Hodgkin lymphoma (3) and others (3). All children have been on chemotherapy during induction phase (15), maintenance phase (9), consolidation phase (3) and pulse therapy phase (3). They were treated with ceftazidime and amikacin intravenously for at least 7 days. Stool specimens were collected before and 2 and 4 weeks after starting antibiotics. C.difficile toxin A in stools were detected by ELISA method which showed positive results in 11 cases (36.7%). There were 5, 5, and 2 positive specimens at before and 2, and 4 weeks respectively. All 5 cases of the first group had history of the last receiving antibiotic longer than 3 months. All of positive C.difficile toxin cases had no diarrhea or abdominal pain. Chemotherapy and cephalosporin were the important factors for colonization of C.difficile in malignancy children.

Itraconazole for treatment of oral candidosis in pediatric cancer patients.

Oral candidosis commonly occurs in malignancy children undergoing antineoplastic chemotherapy. Inadequate response to antifungal treatment leads to a risk of disseminated infection. The aim of this study is to evaluate the efficacy and side effects of itraconazole on treatment of oral candidosis. Fourteen children with malignancy undergoing chemotherapy received itraconazole 100-200 mg/day for 10 days to treat oral candidosis. The severity of disease was defined as mild and moderate depending on the number of lesions and symptoms. Oropharyngeal lesions and symptoms were recorded initially and daily. Blood chemistries were done on day 0, day 10 and day 16. The overall response rate was 87.5 per cent. The mild group (4 cases) had a response rate of 100 per cent which had lesions and symptoms resolved on day 2 and day 1.5 +/- 0.7 respectively. The moderate group (10 cases) had 8 responders (80%) whose lesions and symptoms resolved on day 6 +/- 2.5 and day 4.1 +/- 2.3 respectively. Side effects and abnormal blood chemistry values. were not seen.

Pyruvate kinase deficiency in an alpha-thalassemia family: first case report in Thailand.

In Thailand, the most common cause of chronic hemolytic anemia is thalassemia hemoglobinopathy. We report here a 10-year-old girl with pyruvate kinase (PK) deficiency who was initially diagnosed to have Hb H disease, like her sister. The patient had a history of neonatal jaundice which required blood exchange transfusion twice and phototherapy. She became anemic and regular blood transfusion was required since the age of 2 1/2 months. She was very anemic compared to her sister and was transfusion dependent. Besides, she never had red cell inclusion bodies, thus re-evaluation was performed. The diagnosis of red cell pyruvate kinase deficiency and the exclusion of Hb H disease was achieved after cessation of blood transfusion for 3 months. The family study also confirmed the diagnosis. The patient is now on high transfusion and iron chelation. She is doing well with mild splenomegaly.