Impact of HbE mutation on the clinical severity of HbH disease: A multicentre study from Thailand.

Haemoglobin (Hb) H disease and HbH disease with co-inherited HbE mutation are the most prevalent forms of α-thalassaemia in Southeast Asia. Data were limited when comparing clinical phenotypes between these two patient groups. We conducted a Thai multicentre study and enrolled 588 patients [median (IQR) age 13.

Multicenter Study of Diagnostic Tool for Patients with Hemophilia: From Bedside to Comprehensive Investigations.

Background: Hemophilia cannot be diagnosed in most laboratories of economically less-developed countries leading to high mortality and morbidity rates.

Aim: A diagnostic tool was established ranging from bleeding assessment and a simple bedside test of mixing venous clotting time (VCT) to comprehensive DNA analysis for patients with hemophilia.

Methods: Patients with known (n=80) and suspected hemophilia (n=14) were included.

Molecular characteristics of hereditary red blood cell membrane disorders in Thailand: a multi-center registry.

Red blood cell (RBC) membrane disorders represent a significant category of hereditary hemolytic anemia; however, information from Southeast Asia is limited. We established a national registry aiming to characterize RBC membrane disorders and their molecular features in Thailand. A total of 100 patients (99 kindreds) diagnosed with RBC membrane disorders between 2011 and 2020 from seven university hospitals were enrolled.

Justification of Universal Iron Supplementation for Infants 6-12 months in Regions with a High Prevalence of Thalassemia.

Introduction: Many clinicians hesitate to adopt a universal infant iron supplementation program due to the risk of increased iron absorption for those with thalassemia. We aimed to determine thalassemia prevalence in 6- to 12-month-old infants, along with the iron status of those with and without thalassemia.

Methods: We performed a cross-sectional descriptive study of infants attending the Well Baby Clinic at Thammasat University Hospital for routine checkups.

Thalassemia-related complications in pediatric, adolescent, and young adult patients with transfusion-dependent thalassemia: A multicenter study in Thailand.

Introduction: Management of transfusion-dependent thalassemia (TDT) can be challenging due to numerous potential disease-related complications and comorbidities in particular age groups. The objective of this study was to report thalassemia-related complications and risk factors in pediatric, adolescent, and young adult patients with TDT.

Methods: A multicenter web-based registry was conducted in patients with TDT aged 25 years and younger from eight university hospitals covering all parts of Thailand.

Red blood cell alloimmunization and other transfusion-related complications in patients with transfusion-dependent thalassemia: A multi-center study in Thailand.

Background: Thalassemia is a common genetic disease in Southeast Asia. Red blood cell (RBC) transfusion is an essential treatment for severe forms of thalassemia. We performed a study to demonstrate RBC alloimmunization and other transfusion-related complications in patients with transfusion-dependent thalassemia (TDT).

Pediatric primary central nervous system tumors registry in Thailand under National Health Security Office schemes.

Background: Few epidemiological studies of pediatric central nervous system (CNS) tumors have been performed using data from Southeast Asian national registries. Therefore, we aimed to examine data on CNS tumors from the first national childhood CNS tumor registry in Thailand.

Methods: Newly diagnosed children with benign and malignant primary CNS tumors from 20 nationwide hospitals were included.

Health-Related Quality of Life in Thai Children with Thalassemia as Evaluated by PedsQL and EQ-5D-Y: A Single-Center Experience.

Background: Thalassemia remains a challenging chronic disease in Thailand, but national prenatal screening, along with better treatment and management, may have improved health-related quality of life HRQoL for pediatric patients. We aimed to measure the HRQoL of transfusion-dependent TDT and non-transfusion dependent NTDT of these pediatric patients at our institute.

Methods: We included all patients 2 - 18 years old, with TDT and NTDT, using the Pediatric Quality of Life Inventory 4.

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.

Hematopoietic Stem Cell Transplantation for Severe Thalassemia Patients from Haploidentical Donors Using a Novel Conditioning Regimen.

Patients with severe thalassemia commonly have a survival that is significantly shorter than that of the general population. Allogeneic hematopoietic stem cell transplantation (allo-SCT) is the only established treatment that is potentially curative, but it is limited by the availability of donors and the medical condition of the patient. To expand the donor pool to include haploidentical related donors, we introduced a program consisting of a pharmacologic pretransplant immune suppression phase (PTIS) and 2 courses of dexamethasone and fludarabine, followed by pretransplant conditioning with fludarabine-i.

Two Novel 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 mutations are found in most TAM patients and are markers for future ML-DS.

Infant feeding practices in relation to iron status and other possible nutritional deficiencies in Pathumthani, Thailand.

Background And Objectives: Iron deficiency (ID) is the most common micronutrient deficiency worldwide and usually leads to impaired neurodevelopment. Appropriate introduction of complementary foods is mandatory for all infants to prevent iron insufficiency. We aimed to demonstrate feeding behaviors in relation to infant iron status and also identify potential concomitant nutrient inadequacies.

Long-Term Outcomes of Modified St Jude Children's Research Hospital Total Therapy XIIIB and XV Protocols for Thai Children With Acute Lymphoblastic Leukemia.

Background: We studied long-term outcomes and prognostic features of Thai children with acute lymphoblastic leukemia treated with modified St Jude Children's Research Hospital (SJCRH) protocols.

Patients And Methods: Pediatric patients newly diagnosed with acute lymphoblastic leukemia were included. From 1997 to 2003, the first group received modified Total Therapy XIIIB (previous protocol).

A molecular study on the role of alpha-hemoglobin-stabilizing protein in hemoglobin H disease.

The clinical course of hemoglobin H (HbH) disease is remarkably variable. It is not completely clear how genetic and environmental factors interplay to modify clinical severity in affected individuals. Previous studies suggested that altered structure or function of alpha-hemoglobin-stabilizing protein (AHSP) could modify the clinical phenotypes of thalassemias.

Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors.

Thalassemia-free survival after allogeneic stem cell transplantation (SCT) is about 80-90% with either matched-related or -unrelated donors. We explored the use of a mismatched-related ('haplo- ') donor. All patients received two courses of pretransplant immunosuppressive therapy (PTIS) with fludarabine (Flu) and dexamethasone (Dxm).

Curative Stem Cell Transplantation for Severe Hb H Disease Manifesting From Early Infancy: Phenotypic and Genotypic Analyses.

Most people with Hb H disease live normal lives; however, a minority of cases requires lifelong regular transfusions. An atypical form of nondeletional Hb H disease was reported in a Thai boy, characterized by severe persistent hemolytic anemia since the age of 2 months. Molecular diagnosis revealed the apparent compound heterozygosity for the Southeast Asian (- -(SEA)) and α2 polyadenylation (polyA) signal (AATAAA>AATA- -) deletions.

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.

Central diabetes insipidus: an unusual complication in a child with juvenile myelomonocytic leukemia and monosomy 7.

Central diabetes insipidus (DI) is well-documented as a presenting feature of myelodysplastic syndrome and acute myeloid leukemia in adults. However, DI is unusual in pediatric patients with myeloid malignancies. We report here this rare complication in a child with neurofibromatosis type 1 who developed juvenile myelomonocytic leukemia and monosomy 7.

Comparative outcomes of Thai children with acute lymphoblastic leukemia treated with two consecutive protocols: 11-year experience.

The aim of this study was to determine outcomes and prognostic features of children with acute lymphoblastic leukemia (ALL) treated with two modified St Jude Children's Research Hospital protocols at Ramathibodi Hospital (1997-2008). Event-free survival rates of the "previous protocol" were 88.3 ± 3.

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.

Impaired glucose tolerance and insulin resistance in survivors of childhood acute lymphoblastic leukemia: prevalence and risk factors.

Aim/purpose: Survivors of acute lymphoblastic leukemia (ALL) are at increased risks of impaired glucose metabolism, insulin resistance, and metabolic syndrome. The aim of our study was to determine the prevalence of alterations in glucose metabolism and the predisposing factors of these disturbances in survivors of childhood ALL.

Patients And Methods: In 131 ALL survivors, an oral glucose tolerance test was conducted to determine beta-cell function/insulin sensitivity.