Predictors of severe adverse outcomes in febrile neutropenia of pediatric oncology patients at a single institute in Thailand.

Febrile neutropenia in pediatric oncology patients may lead to severe infection, with adverse events including septic shock or death. The aim of the study was to investigate the prevalence of severe adverse outcomes and to determine the associated risk factors. This is a retrospective cohort study of pediatric oncology patients with febrile neutropenia from October 2013 to September 2017 at Thammasat University Hospital, Thailand. Clinical assessment and time-to-event of severe outcomes were analyzed. There were 95 febrile neutropenic episodes; severe adverse outcomes were documented in 11 (11.5%), with no infection-associated mortalities. Those with severe outcomes were older, received prophylactic granulocyte-colony stimulating factor (G-CSF), and had documented infection, lower initial ANC, and central venous catheter insertion. The proportional hazard regression model revealed age ≥ 10 years (hazard ratio [HR], 5.96; p = 0.005), prophylactic G-CSF (HR, 4.52; p = 0.028), and microbiologically documented infections (HR, 12.53; p = 0.017) independently predicted severe adverse outcomes. Although severe adverse outcomes occurred in only 11.5% of our febrile neutropenic episodes, we identified a few risk factors that may help predict those at highest risk.

Two Novel GATA1 Mutations in Transient Abnormal Myelopoiesis of Thai Neonates with Down Syndrome.

Children with Down syndrome (DS) are 150 times more likely to develop acute myeloid leukemia (ML-DS), compared with those without. One risk factor is transient abnormal myelopoiesis (TAM). Somatic truncating GATA1 mutations are found in most TAM patients and are markers for future ML-DS. We identified two novel frameshift mutations in our seven newborns with DS and TAM: a heterozygous mutation of 17 nucleotide duplication (c.154_170 dup) and a heterozygous 9-nucleotide deletion combined with a 2-nucleotide insertion (c.150_158delins CT). Both mutations introduced a truncated GATA1 protein. Thus, neonates with DS and TAM require frequent ML-DS monitoring.

Modified desensitization protocols for a pediatric patient with anaphylactic reaction to deferoxamine.

Thalassemia major is an inherited form of chronic hemolytic anemia that results in iron overload due to regular blood transfusions. Deferoxamine is used as chelating agentfor treatment ofpatients with chronic iron overload worldwide. Anaphylactic reaction to deferoxamine is rare, and the mechanism ofdeferoxamine-induced anaphylaxis is not well understood. Only afewpediatric cases ofsuccessful desensitization for deferoxamine hypersensitivity have been described, and a different protocol has been used in each report. We report a case ofanaphylaxis to deferoxamine in a thirteen-years-old Thai boy with Hemoglobin E/ß-thalassemia disease who underwent successful desensitization. He had been receiving blood transfusions since the age often months. At age eleven, the patient began treatment with deferoxamine. Treatment was interrupted after the occurrence ofanaphylaxis, with urticaria, wheezing and gastrointestinal symptoms. A skin prick test was positive, indicating a type 1 hypersensitivity reaction. Deferoxamine desensitization was attempted with various differentprotocols. Finally, the patient could tolerate deferoxamine therapy at the dose previously administered. We proposed this modified subcutaneous desensitization protocolforpediatric cases that develop allergic reactions to deferoxamine.

Biopsychosocial predictors of health-related quality of life in children with thalassemia in Thammasat University Hospital.

OBJECTIVES: To determine health-related quality of life (HRQOL) in children with thalassemia in order to explore physical and psychosocial factors affecting on their QOL. MATERIAL AND METHOD: A cross-sectional study was conducted at Thammasat University Hospital, Pathum Thani. Sociodemographic factors and clinical characteristics were obtained from seventy-five of transfusion-dependent and non-transfused thalassemia patients. The PedsQL 4.0 Generic Core Scales (Thai version) were administered to determine the patients and their parents' perspectives. RESULTS: The mean (SD) of total HRQOL score was 78.50 (2.05) for children who were self-reporting and it was 73.41 (2.22) for parent proxy-report, that were comparable with population norms. The stepwise multiple regression analysis indicated that total HRQOL score of child self-report was negatively predicted by lower family income, early age onset of anemia before 2 years and under covered by Universal Health Coverage Scheme. The negative predictors of total HRQOL score of parent proxy-report were regular transfusion every 1-2 months, while self medical payment was positively predictive. CONCLUSION: The HRQOL in children with thalassemia was not only determined by disease severity and treatment but also by family financial impacts for caring of children. Health care interventions should be implemented to support in various domains of life.