Inferior Clinical Outcomes of Pediatric Rhabdomyosarcoma in Thailand: A 16-Year Experience in a Single Tertiary Institution.

INTRODUCTION: There is limited data available on the treatment outcomes of pediatric rhabdomyosarcoma (RMS) in Asian populations. Therefore, we aimed to review the baseline characteristics, clinical outcomes, and prognostic factors in children with RMS from Thailand. METHODS: The data of children under 15 years of age diagnosed with RMS between 2003 and 2019 from a large tertiary hospital in Southern Thailand were retrospectively reviewed. Descriptive statistics were utilized to describe the clinical characteristics. The Kaplan-Meier method was utilized to estimate survival. Cox proportional hazards regression analysis was utilized to determine prognostic factors that affect survival. RESULTS: A total of 42 children RMS were included in this study. The median age at diagnosis was 6.4 years (IQR, 2.4-10.2). Among these patients, 11 (26%) were older than 10 years, and 13 (31%) presented with metastatic disease at diagnosis. The 5-year overall survival (OS) rate was 39% for all children. Age greater than 10 years (hazard ratio (HR): 3.3, 95% CI: 1.2-9.2) and metastatic disease at diagnosis (hazard ratio (HR): 2.8, 95% CI: 1.1-7.5) were independently associated with poorer survival. The 3-year OS for children with metastatic disease (stage IV) was 15% (95% CI: 4.3-55). CONCLUSION: The percentage of metastatic disease in our cohort was higher than that in previous reports and may have contributed to a poorer outcome. Age greater than 10 years and metastatic disease at diagnosis were noted as adverse prognostic factors.

Cleft lip and/or palate: 10 years experience at a pediatric cleft center in Southern Thailand.

OBJECTIVE: To study the clinical characteristics, demographic data, and associated congenital anomalies of pediatric patients with cleft lip and/or palate in Southern Thailand. DESIGN: Retrospective, hospital-based study. SETTING: Cleft Clinic Center, Songklanagarind Hospital PARTICIPANTS: Children with cleft lip and/or palate who were born or seen at Songklanagarind Hospital between January 1997 and December 2006. MAIN OUTCOME MEASURE: Clinical features including demographic data, types of cleft, associated anomalies, family history of clefts, and physical growth. RESULTS: A total of 153 children were seen during the studied period, of whom 36 (23.5%) had isolated cleft lip, 32 (20.9%) had isolated cleft palate, and 85 (55.6%) had combined cleft lip and palate. Twenty-seven children (17.7%) had a family history of clefts. Congenital malformations (syndromic cleft) were found in 20 children (13%), and chromosomal abnormalities were found in four of these (20%). There were no significant differences among the three groups (isolated cleft lip, isolated cleft palate, and combined cleft lip and palate) in maternal and paternal ages, gestational age, birth weight, family history of cleft, or associated malformations. The physical growth parameters of children with nonsyndromic cleft were the same as in the general population. Children with syndromic cleft were significantly lighter at birth and had grown up significantly shorter and lighter, with smaller head circumference. CONCLUSIONS: Chromosomal abnormalities are commonly found in children with syndromic cleft. Children with nonsyndromic cleft have normal growth; whereas, those with syndromic cleft have some degree of prenatal and postnatal growth restriction.

Prevalence of impaired glucose metabolism in beta-thalassemic children receiving hypertransfusions with a suboptimal dosage of iron-chelating therapy.

A cross-sectional study of impaired glucose metabolism was carried out in 48 beta-thalassemic patients receiving hypertransfusions. An oral glucose tolerance test (OGTT) was performed using the method and criteria of the American Diabetes Association (ADA). Diabetes mellitus was diagnosed in two patients, and impaired glucose tolerance was found in four patients, giving a prevalence of impaired glucose metabolism of 12.5% in our patient population. The significant clinical characteristics associated with the diagnosis of impaired glucose metabolism were wasting (-2.15/-0.86 SDS, p = 0.025), stunting (-2.69/-1.22 SDS, p = 0.03), higher ferritin levels (8679/4710 microg/L, p = 0.005), splenectomy (50/9.5%, p = 0.012), and lower area under curve (AUC) of insulin secretion after OGTT (40.0/77.7, p = 0.002). The significant decrease of AUC insulin in thalassemic patients with an impaired glucose tolerance test suggests that the pathogenesis may originate from pancreatic beta-cell damage rather than from insulin resistance. In conclusion, the prevalence of impaired glucose tolerance in our population of thalassemic patients receiving hypertransfusions with suboptimal iron chelating therapy was 12.5%. The clinical characteristics of thalassemic patients who developed impaired glucose tolerance were wasting, stunting, higher ferritin levels, splenectomy, and lower AUC insulin.

Thyroid function in beta-thalassemic children receiving hypertransfusions with suboptimal iron-chelating therapy.

A cross-sectional study of thyroid function, free thyroxine (FT) and thyrotropin (TSH) concentrations, was carried out in 51 transfusion-dependent beta-thalassemic patients receiving suboptimal iron-chelating therapy. Nine patients had normal FT4 levels with elevated TSH levels (5.9-15.6 mLU/L), consistent with the diagnosis of compensated primary hypothyroidism and giving a prevalence of abnormal thyroid function of 17.6%. All patients with abnormal thyroid function had negative thyroid antibodies. No particular risk factor for abnormal thyroid function could be identified. Of the nine patients with compensated primary hypothyroidism, one patient showed a further increase in TSH level after 1 year of follow-up. The results of the present study emphasize the importance of thyroid function monitoring in hypertransfused beta-thalassemic patients.

A low dose adrenocorticotropin test (1 microg ACTH) for the evaluation of adrenal function in children with beta-thalassemia receiving hypertransfusion with suboptimal iron-chelating therapy.

A cross-sectional study of adrenal function was carried out in 48 patients with beta-thalassemia who were receiving hypertransfusion with suboptimal desferoxamine. A low dose adrenocorticotropic hormone (1 microg ACTH) stimulation test was performed using the cut-off criteria of peak cortisol for adrenal sufficiency >18 microg/dl. Adrenal impairment was diagnosed in 22 patients, giving a prevalence of 45.8%. The peak cortisol concentrations in normal and impaired adrenal function groups were 26.22 +/- 2.84 and 14.03 +/- 3.12 microg/dl, respectively, and the mean basal morning cortisol was 8.93 +/- 2.97 and 6.52 +/- 2.45 microg/dl, respectively. There was no significant difference in any clinical characteristic between the patients with impaired adrenal function and those with normal adrenal function.