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Chalinee Monsereenusorn,1 Thitiyaporn Sricharoen,2 Piya Rujkijyanont,1 Detchvijitr Suwanpakdee,3 Apichat Photia,1 Nawachai Lertvivatpong,1 Chanchai Traivaree1 1Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and Phramongkutklao College of Medicine, Bangkok, Thailand; 2Buayai Hospital, Nakhon Ratchasima, Thailand; 3Division of Infectious Disease, Department of Pediatrics, Phramongkutklao Hospital and Phramongkutklao College of Medicine, Bangkok, ThailandCorrespondence: Chalinee MonsereenusornDivision of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and Phramongkutklao College of Medicine, 315 Ratchawithi Road, Ratchathewi, Bangkok, 10400, ThailandEmail chalinee_monsereenusorn@pedpmk.orgBackground: The most common complication among pediatric oncology patients is febrile neutropenia (FN). Invasive fungal disease (IFD) is suspected when fever persists > 4– 7 days after empirical antibiotics. Its clinical characteristics and predictive factors associated with IFD among pediatric oncology patients with FN were thus explored.Methods: Pediatric oncology patients with FN between January 1, 2012 and December 31, 2016 were enrolled in this study. Clinical characteristics, including laboratory investigations, treatment modalities, and final outcomes of IFD were retrospectively reviewed and analyzed.Results: In all, 73 patients with 180 episodes of confirmed diagnosis of FN were studied. Median age at diagnosis was 6.2 years, with equal sex distribution. The most common diagnosis was acute lymphoblastic leukemia (n=91, 51%), followed by acute myeloid leukemia (n=47, 26%), Burkitt’s lymphoma (n=7, 4%) and neuroblastoma (n=7, 4%). Median absolute neutrophil count at FN diagnosis was 0 (0– 806) cells/mm3. IFD was diagnosed for 25 (14%) episodes. Mortality rates for FN and IFD were 4% and 20%, respectively. Respiratory compromise, oxygen requirement, hypotension, prolonged hospitalization, duration of fever and neutropenia, bacteremia, bacteriuria, funguria, abnormal liver-function results, and prolonged broad-spectrum antibiotic administration were factors associated with IFD (P< 0.05). Prolonged duration between initiation of fever and antifungal administration for nearly 10 days was an independent factor in prediction of IFD occurrence (P= 0.014).Conclusion: Respiratory compromise, oxygen requirement, hypotension, prolonged hospitalization, duration of fever and neutropenia, bacteremia, bacteriuria, funguria, abnormal liver-function results and prolonged broad-spectrum antibiotic administration were factors associated with IFD. Duration between initiation of fever and antifungal administration of nearly 10 days were considered a risk factors of IFD among patients with FN.IRB Reference Number: IRBRTA 825/2560.Keywords: febrile neutropenia, invasive fungal disease, pediatric oncology, risk factors, mortality

Piya Rujkijyanont,1 Chanchai Traivaree,1 Kantang Satayasoontorn,2 Apichat Photia,1 Nawachai Lertvivatpong,1 Chalinee Monsereenusorn1 1Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and Phramongkutklao College of Medicine, Bangkok, Thailand; 2Department of Pathology, Phramongkutklao Hospital and Phramongkutklao College of Medicine, Bangkok, ThailandCorrespondence: Chalinee MonsereenusornDivision of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and Phramongkutklao College of Medicine, 315 Ratchawithi Road Ratchathewi, Bangkok, 10400, ThailandEmail chalinee_monsereenusorn@pedpmk.orgBackground: The most common type of vascular tumors reported among children is hemangioma. The determinant factors to predict clinical outcomes among those patients were not well studied.Objective: The study aimed to explore clinical characteristics and treatment approaches as well as associated prognostic factors of vascular tumors specifically in a pediatric population.Methods: Pediatric patients with a confirmed diagnosis of vascular tumors between January 1, 2005 and December 31, 2017 were enrolled in this study. Clinical data includes initial clinical manifestations with associated complications, and diagnostic studies were used. To establish a diagnosis, the treatment modalities provided and final outcomes were retrospectively reviewed and analyzed.Results: In all, 50 patients with a confirmed diagnosis of vascular tumors were enrolled. The median age at diagnosis was 11.5 years with equal gender distribution. The most common type of vascular tumor was hemangioma (n=41, 82%), followed by pyogenic granuloma (n=4, 8%), kapasiform hemangioendothelioma with Kasabach-Merritt phenomenon (n=2, 4%), infantile hepatic hemangioma (n=2, 4%), and juvenile nasal angiofibroma (n=1, 2%). The median age at diagnosis among patients with cutaneous vascular tumors (12.4 years) was significantly older than the age of those with visceral vascular tumors (1.3 years) witha P-value of 0.009. The mean size among patients with visceral tumors (7.46± 4.84 cm) was significantly greater than the size among patients with cutaneous tumors (3.21± 3.7 cm) with a P-value of 0.023. The size of the tumor was the only independent risk factor associated with clinical outcomes.Conclusion: Age at diagnosis in cutaneous vascular tumors was significantly older than in visceral vascular tumors. Clinical outcomes are favorable among most patients and the size of the tumors is an independent risk factor associated with outcomes.Keywords: pediatric, vascular tumor, prognostic factor, size, outcome

Apichat Photia,1 Chanchai Traivaree,1 Chalinee Monsereenusorn,1 Piyarat Simthamnimit,2 Piya Rujkijyanont1 1Division of Hematology-Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand; 2Department of Pediatrics, Fort Prachaksilapakhom Hospital, Udonthani, ThailandCorrespondence: Piya RujkijyanontDivision of Hematology-Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, 315 Ratchawithi Road, Ratchathewi, Bangkok 10400, ThailandTel/Fax +66 2 644 4130Email piya_rujk@yahoo.comPurpose: Red blood cell transfusion is a key element of treatment among patients with transfusion-dependent thalassemia (TDT). Volume overload and HCC syndrome (hypertension, convulsion, and intracranial hemorrhage) are fatal complications related to transfusion. Furosemide has been widely used to prevent hypertension secondary to volume overload with unclear supportive evidence. This study aimed to evaluate the efficacy of furosemide to prevent volume overload among children and young adults diagnosed with TDT.Methods: Patients diagnosed with TDT were enrolled and randomized to receive either furosemide pretransfusion or no furosemide pretransfusion. After 3 weeks to 4 months of wash-out periods, those patients underwent the alternate regimens as per crossover design of the study. Clinical and laboratory parameters including blood pressure and NT-proBNP levels were measured before and after each transfusion. The difference of those parameters between two randomized groups and their potential associated factors were analyzed.Results: In all, 30 patients undergoing 60 red blood cell transfusions were enrolled in the study. All were randomized and crossover was designed as receiving and not receiving furosemide pretransfusion. No transfusion reactions, symptoms of volume overload and HCC syndrome were observed. No statistically significant correlation was found between pretransfusion furosemide and the difference between pre- and posttransfusion systolic blood pressure (2 mmHg systolic blood pressure difference in pretransfusion furosemide and 1.5 mmHg in no pretransfusion furosemide; p-value = 0.721), as well as between pretransfusion furosemide and the difference between pre- and posttransfusion NT-proBNP levels (− 3.8 pg/mL NT-proBNP level difference in pretransfusion furosemide and − 2.4 pg/mL in no pretransfusion furosemide; p-value = 0.490). No significant correlation was also observed even in selected patients with high NT-proBNP levels (p-value = 0.262). Associated factors affecting the difference between pre- and posttransfusion NT-proBNP levels were analyzed, and none of those were affected concerning the difference in the levels.Conclusion: Furosemide has been included in standard transfusion guidelines in many institutions. Our study provided important evidence of the unnecessary use of the drug in preventing volume overload particularly in pediatric and young adult patients with TDT.Thai Clinical Trials Registry (Tctr) Number: TCTR20180209001. Registered 6 February 2018, https://www.clinicaltrials.in.th/.Keywords: NT-proBNP, furosemide, transfusion, thalassemia, volume overload

Chanchai Traivaree,1 Napakjira Likasitthananon,2 Chalinee Monsereenusorn,1 Piya Rujkijyanont1 1Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand; 2Department of Pediatrics, Lat Krabang Hospital, Bangkok, Thailand Background: High-dose methotrexate (HD-MTX) is widely used as a standard chemotherapeutic agent in pediatric cancers. Most research studies have confirmed the therapeutic efficacy of HD-MTX; however, strategies to prevent side effects vary among institutions, especially in developing countries, with limited monitoring of plasma methotrexate level.Objective: To evaluate the effect of intravenous hydration during HD-MTX administration on plasma methotrexate clearance in pediatric oncology patients.Materials and methods: This study retrospectively reviewed 165 courses of HD-MTX administered to children with acute lymphoblastic leukemia (ALL), non-Hodgkin’s lymphoma (NHL), or osteosarcoma. Demographic data of patients were collected. Adverse complications related to HD-MTX and 72-hour plasma methotrexate level were analyzed between patients receiving intravenous hydration ≥3,000 mL/m2/day and those receiving hydration

Chanchai Traivaree,1 Chalinee Monsereenusorn,1 Piya Rujkijyanont,1 Warakorn Prasertsin,2 Boonchai Boonyawat3 1Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand; 2Department of Pediatrics, Queen Savang Vadhana Memorial Hospital, Chonburi, Thailand; 3Division of Genetics, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand Introduction: Beta-thalassemia is a group of inherited hemolytic anemias and one of the most common genetic disorders in Thailand. The clinical spectrum of beta-thalassemia disease ranges from mild to severe clinical symptoms including mild beta-thalassemia intermedia (TI) and severe beta-thalassemia major (TM). Objective: This study aimed to determine the correlation between beta-globin gene (HBB) mutations and their phenotypic manifestations by evaluating patients’ clinical characteristics, transfusion requirements, growth and hematologic parameters, and hemoglobin typing among pediatric patients treated at Phramongkutklao Hospital. Materials and methods: Seventy beta-thalassemia patients, including 63 with beta-thalassemia/hemoglobin E (HbE) and 7 with either homozygous or compound heterozygous beta-thalassemia, were enrolled in this study. Their clinical presentation, growth parameters and laboratory findings were reviewed and analyzed. The mean follow-up time was 10.52±5.62 years. Mutation analysis in each individual was performed using multiplex amplification refractory mutation system (M-ARMS), direct DNA sequencing of beta-globin gene and gap PCR for 3.4 kb deletion detection. Results: All 7 homozygous and compound heterozygous beta-thalassemia patients were classified in TM. Among 63 patients with beta-thalassemia/HbE, 58 were classified in TM and 4 were classified in TI. Mean age at diagnosis was 0.8±0.49 years for homozygous or compound heterozygous beta-thalassemia and 3.43±3.5 years for beta-thalassemia/HbE. The most common HBB mutation was HBB:c.126_129delCTTT [codon 41/42 (-TCTT)] found in 34 alleles (48.6%). The height for age was also lower in homozygous beta-thalassemia patients (

Chanchai Traivaree,1,* Boonchai Boonyawat,2,* Chalinee Monsereenusorn,1 Piya Rujkijyanont,1 Apichat Photia1 1Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand; 2Division of Genetics, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand *These authors contributed equally to this work Background: α-Thalassemia, one of the major thalassemia types in Thailand, is caused by either deletion or non-deletional mutation of one or both α-globin genes. Inactivation of three α-globin genes causes hemoglobin H (Hb H) disease, and the combination of Hb H disease with heterozygous hemoglobin E (Hb E) results in AE Bart’s disease. Objective: This study aimed to characterize the clinical and hematological manifestations of 76 pediatric patients with Hb H and AE Bart’s diseases treated at Phramongkutklao Hospital, a tertiary care center for thalassemia patients in central Thailand.Patients and methods: Seventy-six unrelated pediatric patients, 58 patients with Hb H disease and 18 patients with AE Bart’s disease, were enrolled in this study. Their clinical presentations, transfusion requirement, laboratory findings, and mutation analysis were retrospectively reviewed and analyzed.Results: A total of 76 pediatric patients with Hb H and AE Bart’s diseases who mainly lived in central Thailand were included in this study. The clinical severities of patients with non-deletional mutations were more severe than those with deletional mutations. Eighty-six percent of patients with non-deletional AE Bart’s disease required more blood transfusion compared to 12.5% of patients with deletional AE Bart’s disease. Non-deletional AE Bart’s disease also had a history of urgent blood transfusion with the average of 6±0.9 times compared to 1±0.3 times in patients with deletional Hb H disease. The difference was statistically significant.Conclusion: This study revealed the differences in clinical spectrum between patients with Hb H disease and those with AE Bart’s disease in central Thailand. The differentiation of α-thalassemia is essential for appropriate management of patients. The molecular diagnosis is useful for diagnostic confirmation and genotype–phenotype correlation. Keywords: genotype, phenotype, Hb H disease, AE Bart’s disease, Thai children

Chanchai Traivaree,1 Chalinee Monsereenusorn,1 Arunotai Meekaewkunchorn,2 Premsak Laoyookhong,3 Saranya Suwansingh,4 Boonchai Boonyawat5 1Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, 2Division of Hematology/Oncology, Department of Pediatrics, 3Division of Neonatology, Department of Pediatrics, Queen Sirikit National Institute of Child Health, Bangkok, 4Division of Hematology/Oncology, Department of Pediatrics, Chiangrai Prachanukroh Hospital, Chiang Rai, 5Division of Genetics, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand Abstract: Congenital factor VII (FVII) deficiency is a rare inherited coagulopathy. The clinical manifestations and clinical findings vary widely, ranging from asymptomatic to life-threatening bleeding, including intracranial hemorrhage (ICH), with prolonged prothrombin time, normal partial thromboplastin time and normal platelet counts, which are confirmed by the low level of FVII assay. Treatment consists of fresh frozen plasma (FFP), prothrombin complex concentrates (PCCs), and recombinant activated FVII to treat bleeding and prophylactic therapy. Here, we report four patients with FVII levels T was the most common (50%) mutation identified in our study, followed by the K376X nonsense mutation (37.5%). In our study, we found that genetic information affected the severity of congenital FVII deficiency with ICH. Keywords: mutation analysis, factor VII deficiency, Thai children

Boonchai Boonyawat,1 Chalinee Monsereenusorn,2 Chanchai Traivaree2 1Division of Genetics, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand; 2Division of Hematology/Oncology, Department of Pediatrics, Phramongkutklao Hospital and College of Medicine, Bangkok, Thailand Background: Beta-thalassemia is one of the most common genetic disorders in Thailand. Clinical phenotype ranges from silent carrier to clinically manifested conditions including severe beta-thalassemia major and mild beta-thalassemia intermedia. Objective: This study aimed to characterize the spectrum of beta-globin gene mutations in pediatric patients who were followed-up in Phramongkutklao Hospital. Patients and methods: Eighty unrelated beta-thalassemia patients were enrolled in this study including 57 with beta-thalassemia/hemoglobin E, eight with homozygous beta-thalassemia, and 15 with heterozygous beta-thalassemia. Mutation analysis was performed by multiplex amplification refractory mutation system (M-ARMS), direct DNA sequencing of beta-globin gene, and gap polymerase chain reaction for 3.4 kb deletion detection, respectively. Results: A total of 13 different beta-thalassemia mutations were identified among 88 alleles. The most common mutation was codon 41/42 (-TCTT) (37.5%), followed by codon 17 (A>T) (26.1%), IVS-I-5 (G>C) (8%), IVS-II-654 (C>T) (6.8%), IVS-I-1 (G>T) (4.5%), and codon 71/72 (+A) (2.3%), and all these six common mutations (85.2%) were detected by M-ARMS. Six uncommon mutations (10.2%) were identified by DNA sequencing including 4.5% for codon 35 (C>A) and 1.1% initiation codon mutation (ATG>AGG), codon 15 (G>A), codon 19 (A>G), codon 27/28 (+C), and codon 123/124/125 (-ACCCCACC), respectively. The 3.4 kb deletion was detected at 4.5%. The most common genotype of beta-thalassemia major patients was codon 41/42 (-TCTT)/codon 26 (G>A) or betaE accounting for 40%. Conclusion: All of the beta-thalassemia alleles have been characterized by a combination of techniques including M-ARMS, DNA sequencing, and gap polymerase chain reaction for 3.4 kb deletion detection. Thirteen mutations account for 100% of the beta-thalassemia genes among the pediatric patients in our study. Keywords: mutation analysis, beta-globin gene, Thai children

Objectives: This study aimed to enhance the learning experience among medical students by empowering them to co-create learning tools and classroom activities., Methods: A cross-sectional study was conducted with 10 participants from Year 2 of the new curriculum volunteering to participate in this study. Five were selected based on their diversities and empowered to design learning tools and class activities. Student satisfaction was presented as mean scores. A comparison of self-confidence scores in subjects learned before and after the class was analyzed using the paired t-test. Comparisons of multiple-choice question (MCQ) scores before and after the class between Years 2 (n = 96) and Year 3 of the previous curriculum attending inclass teaching (n = 98) were analyzed using the independent sample t-test., Results: A high level of satisfaction (M=87.5, SD=15.7%) and significant improvements in student self-confidence in subjects learned between before (M=46.4, SD=20.8%) and after (M=82.7, SD=16.9%) the class were noted (t (223) = -23.73, p<.001). Additionally, Year 2 students achieved significantly higher MCQ scores after the class (M=85.6, SD=19.0%) compared with the scores from Year 3 (M=77.3, SD=23.6%) (t (190) = 3.32, p<.001)., Conclusions: Empowering medical students to co-create learning tools and class activities could positively enhance their learning experience. The result of this study addressed the importance of student empowerment with well-designed student-centered learning strategies based on their learning environment. Additional qualitative research is required to better understanding the "why" and "how" behind the findings of this study.

Background: Health-related quality of life (HRQOL) is a vital assessment to demonstrate the achievement in pediatric cancer care parallels that of medical treatment. The Pediatric Quality of Life Inventory (PedsQL) 3.0 Cancer Module in the Thai Version has become a standard tool to access the HRQOL among Thai children with cancer and their families. This study aimed to explore the HRQOL and factors related to HRQOL among pediatric oncology patients using the PedsQL 3.0 Cancer Module., Methods: In 2018-2019, a single-institution, cross-sectional study was conducted among children with cancer and their families in Bangkok, Thailand. A paired-sample t-test was performed to evaluate the differences between the HRQOL scores of the child and parents' reports. Linear regression was used to evaluate factors associated with HRQOL and which particular domains of the PedsQL 3.0 Cancer Module were influenced., Results: Eighty-five children with cancer and their families were enrolled. The patients' mean age was 10.82 ± 5.48 years. The most common cancer types included acute lymphoblastic leukemia (n = 32, 67%), central nervous system tumors (n = 13, 15%), osteosarcoma (n = 10, 27%) and neuroblastoma (n = 9, 24%), in rank. The mean HRQOL scores in child and parent reports were 74.37 ± 15.7 and 70.42 ± 17.15, respectively (p = 0.034). Factors negatively correlated to HRQOL in parent reports were the number of outpatient visits (p = 0.019) and hospital admissions (p = 0.002). The number of hospitalizations was the only independent factor that affected HRQOL (p = 0.044). The number of outpatient visits and/or hospital admissions was influenced by pain and hurt, nausea, procedural anxiety and communication domains (p < 0.05). Only the number of hospitalizations was an independent factor influencing the procedural anxiety domain in HRQOL (p = 0.005)., Conclusion: HRQOL among Thai children with cancer was desirable from both children's and parent's perspectives. Differences between child and parent HRQOL scores were observed. The number of outpatient visits and hospital admissions affected HRQOL, particularly in the procedural anxiety aspect., Trial Registration: Thai Clinical Trials Registry (TCTR) Number: TCTR20200904001 (04/09/2020),  https://www.thaiclinicaltrials.org/ ., (© 2024. The Author(s).)

Background: Disease reactivation/refractory remains a major challenge in managing Langerhans cell histiocytosis (LCH). Outcomes and late sequelae should be explored., Methods: A multi-institutional retrospective study was conducted to describe clinical characteristics, predictive factors, outcomes and late sequelae of pediatric reactivation/refractory LCH in Thailand., Results: In all, 47 patients were studied, 25 (53.2%) patients had disease reactivation and 22 (46.8%) patients had refractory LCH. The median reactivation and refractory time were 1.59 and 0.33 years from diagnosis, respectively (p <0.001). The most common site of reactivation/refractory was the bone (n = 26, 55%), and 20 (42.6%) patients developed late sequelae. The 5-year overall survival (OS) was 76.1%. Patients with reactivation and refractory LCH performed similarly in 5-year OS (88% vs. 63%, p = 0.055). Prognostic factors associated with mortality were liver, spleen, hematopoietic system and lung reactivation (p <0.05). Lung reactivation was the only independent risk factor associated with the survival outcome (p = 0.002)., Conclusions: The outcomes of pediatric patients between reactivation and refractory LCH in Thailand were similarly desirable and mortality was minimal although late sequelae may evolve. Pulmonary reactivation/refractory was an independent risk factor associated with survival.

Introduction: Inherited bleeding disorders (IBDs) including hemophilia, von Willebrand disease, platelet disorders, mucocutaneous bleeding disorders and coagulation factor deficiencies are rarely found and under-recognized in low and lower-middle-income countries. Some patients succumbed to serious bleeding without diagnosis and treatment during childhood., Area Covered: Diagnosis, management, and prevention should be integrated into the existing health care system. Although some countries have not implemented appropriate health care infrastructure, an initiative plan should be set up by cooperation of experienced experts and health care providers. Identification of patients with IBDs should be started in the antenatal setting to search for females at risk of carrier state. The investigations include bleeding assessment, mixing venous clotting time, coagulogram, coagulation factor assay and mutation detection. Genotypic analysis is helpful for confirming the definite diagnosis, carrier detection as well as prenatal diagnosis for females at risk of bearing an offspring with severe bleeding manifestations. Management involves replacement therapy ranging from blood component to virus-inactivated factor concentrate. Appropriate research is an essential backbone for improving patients' care., Expert Opinion: Effective national strategic advocacy to manage patients with IBDs requires intensive collaboration among policy makers, health care providers, patients, and family members.

Background: Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition caused by genetic mutation or various triggers disturbing the immune system., Methods: A multicenter retrospective study of pediatric patients with HLH receiving a diagnosis between January 2005 and December 2019 from three pediatric oncology centers was conducted to explore the clinical characteristics and determine prognostic factors associated with outcomes among Thai children., Results: In all, 78 patients with HLH with a median age at diagnosis of 3.17 (range, .08-17.83) years were enrolled. The male to female ratio was 1.2:1. The most common type of HLH was infection-associated hemophagocytic syndrome (IAHS) (n = 59, 75%) of which Epstein-Barr virus was the most common pathogen. Thrombocytopenia, hyperbilirubinemia, and treatment response at weeks 2 and 8 after initiating treatment were associated with mortality. Platelet count <50,000 cells/mm 3 was the only independent prognostic factor to define survival outcome (p-value .035). Two-year overall survival rate was 71.3% (95% confidence interval, 59.2%-80.3%). Survival rates between IAHS, malignant associated HLH, macrophage activation syndrome, and unspecific HLH did not significantly differ (p-value .571)., Conclusion: IAHS was the most common cause among pediatric HLH in Thailand. The outcomes of Thai children with HLH were comparable to those of developed countries. Platelet count <50,000 cells/mm 3 was the only independent prognostic factor to define survival outcome., (© 2022 John Wiley & Sons Australia, Ltd.)

Children with cancer often require sedation before undergoing invasive procedures. Fentanyl, ketamine, and midazolam are effective drugs widely used for procedural sedation. This study aimed to investigate the efficacy and safety of midazolam-fentanyl (M-F) compared with midazolam-ketamine (M-K) for bedside procedural sedation among pediatric oncology patients. A randomized, double-blinded, crossover trial was conducted among children with cancer requiring procedural sedation for invasive procedures. Patients were randomly assigned either intravenous M-F or M-K and subsequently received the alternate regimens following the crossover design of the study. The efficacy and safety of the sedations including sedation time intervals, nausea score, vomiting episodes, pain score, adverse effects, and parent's satisfaction were evaluated. In all, 58 patients with 116 procedural sedations were enrolled. M-K provided a shorter induction time (0:58 vs. 1:23 min) ( p  = 0.005), but longer sedation (9:02 vs. 5:50 min) ( p  = 0.019) and emergence time (4:26 vs. 0:56 min) ( p  = 0.011) compared with M-F. Sedation routes affected the sedation time intervals. Patients had higher rates of vomiting (0, range 0-8 vs. 0, range 0-2) ( p  = 0.033) but experienced less pain (0 vs. 2) ( p  = 0.008) in the M-K group. Overall satisfaction and other adverse effects were comparable among both sedation regimens. Combined sedative drugs are recommended to improve the effectiveness of bedside procedural sedation. M-K provided shorter induction, but longer sedation and emergence time compared with M-F. These findings correlated with sedative routes. Patients receiving M-K experienced a higher rate of vomiting, but less painfulness compared with M-F. Overall satisfaction and tolerable side effects were comparable among both sedative regimens.

Background: Neuroblastoma is the most common extracranial malignant solid tumor during childhood. Despite intensified treatment, patients with high-risk neuroblastoma (HR-NBL) still carry a dismal prognosis. The Thai Pediatric Oncology Group (ThaiPOG) proposed the use of a multimodality treatment to improve outcomes of HR-NBL in non-immunotherapy settings., Methods: Patients with HR-NBL undergoing ThaiPOG protocols (ThaiPOG-NB-13HR or -18HR) between 2013 and 2019 were retrospectively reviewed. Patient demographic data, treatment modalities, outcomes, and prognostic factors were evaluated and analyzed., Results: A total of 183 patients with HR-NBL undergoing a topotecan containing induction regimen were enrolled in this study. During the consolidation phase (n = 169), 116 patients (68.6%) received conventional chemotherapy, while 53 patients (31.4%) underwent hematopoietic stem cell transplantation (HSCT). The 5-year overall survival (OS) and event-free survival (EFS) were 41.2% and 22.8%, respectively. Patients who underwent HSCT had more superior 5-year EFS (36%) than those who received chemotherapy (17.1%) (p = .041), although they both performed similarly in 5-year OS (48.7% vs. 39.8%, p = .17). The variation of survival outcomes was observed depending on the number of treatment modalities. HSCT combined with metaiodobenzylguanidine (MIBG) treatment and maintenance with 13-cis-retinoic acid (cis-RA) demonstrated a desirable 5-year OS and EFS of 65.6% and 58.3%, respectively. Poorly or undifferentiated tumor histology and cis-RA administration were independent factors associated with relapse and survival outcomes, respectively (p < .05)., Conclusion: A combination of HSCT and cis-RA successfully improved the outcomes of patients with HR-NBL in immunotherapy inaccessible settings., (© 2022 Wiley Periodicals LLC.)

Background: Assent should be obtained in all children involved in research in keeping with their level of maturity. Traditional assent forms contain too much information and are difficult to read. The study aimed to identify an effective tool to enhance children's comprehension during the assent process and focused on those with cancer who are likely more engaged in research involving greater than minimal risk., Methods: In all, 116 children with cancer were randomized to receive either a paper-based assent document or a multimedia-based assent document. Open-ended and multiple-choice questions were used to assess comprehension and recall. Time spent on the documents and children's behavior during the assent process was recorded to determine their attention and satisfaction., Results: Children randomized to a multimedia-based assent document achieved significant higher comprehension and recall assessment scores (p-values <.001). The high score achievement significantly correlated with the child's age with adjusted odds ratio (OR) of 1.90 (p-value <.001; 95% confidence interval [CI]: 1.35-2.66) for comprehension assessment and 1.59 (p-value .001; 95% CI: 1.20-2.12) for recall assessment. Children randomized to a multimedia-based assent document had significant longer time spent on the document (p-value .001) with less numbers of inattention (p-value <.001) and expressed more signs of enjoyment during the assent process (p-values <.001)., Conclusion: Multimedia-based assent document successfully enhanced comprehension, recall, and attention with more satisfaction compared with a traditional paper-based document among children with cancer. This approach may be considered as an alternative format for children engaging in research involving greater than minimal risk., (© 2022 Wiley Periodicals LLC.)

Assessing the health-related quality of life (HRQOL) is highly recommended as a standard of care for children with cancer in conjunction with medical treatment. The Pediatric Quality of Life Inventory (PedsQL) Cancer Module is a standard tool designed to assess the HRQOL among pediatric oncology patients. This study aimed to evaluate the reliability and correlation of the PedsQL 3.0 Cancer Module in Thai version between child and parent reports. A cross-sectional study was conducted on 85 Thai children with cancer and their families. Excellent internal consistency of the PedsQL 3.0 Cancer Module of the Thai version was addressed among child and parent reports (0.92 and 0.94, respectively). Overall positive correlations were also found between child and parent reports ( r = 0.61, P < .001). However, the statistically significant differences of HRQOL scores between child and parent reports were determined on procedural anxiety (70.05 ± 26.67 vs 60.03 ± 25.6, P = .003), treatment anxiety (88.15 ± 17.37 vs 76.82 ± 26.7, P = .001), worry (66.67 ± 25.59 vs 55.34 ± 30.37, P = .003) and the total score (74.37 ± 15.7 vs 70.42 ± 17.15, P = .034). This study demonstrated desirable internal reliability with positive correlations between child and parent reports of the PedsQL 3.0 Cancer Module in Thai version, although possible differences between child and parent HRQOL scores should be considered., Competing Interests: Declaration of Conflicting Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2022.)

Background: Treatment refusal and abandonment (TxRA) are major barriers to improving outcomes among children with sarcomas of the extremities as curative treatment options bearing on amputation or disfiguring surgery, particularly in countries with limited resources. A multi-institutional retrospective study was conducted to determine the predictive factors for TxRA among patients with osteosarcoma associated with survival outcomes across Southeast Asia (SEA)., Methods: Pediatric patients with osteosarcoma treated between January 1998 and December 2017 in four SEA pediatric oncology centers from three countries were studied. Nelson-Aalen estimates, Kaplan-Meier method, and Cox's proportion hazard model were applied to address the cumulative incidence, survival outcomes, and to identify prognostic factors associated with TxRA., Results: From a total of 208 patients with osteosarcoma enrolled; 18 (8.7%) patients refused and 41 (19.7%) patients abandoned treatment. Income classification of countries, age at diagnosis, tumor size, disease extent, chemotherapy protocols, and types of surgery were associated with TxRA. Tumor size more than 15 cm was an independent risk factor associated with TxRA. The 5-year overall and relapse-free survivals were 49.4% and 50.4%, respectively. However, these rates declined further to 37.9% and 35.8%, respectively, when TxRA were considered as events. Tumor size larger than 15 cm and metastatic disease were independent risk factors associated with TxRA-sensitive outcomes., Conclusion: The prevalence of TxRA was high in SEA, particularly in lower middle-income countries. Factors associated with TxRA related to tumor burden. Treatment outcomes could be substantially improved by lowering the refusal and abandonment rates., (© 2022 Wiley Periodicals LLC.)

Background: Pediatric osteosarcoma outcomes among developed and developing countries have not been previously compared. Countries in Southeast Asia (SEA) have a wide variety of socioeconomic statuses. A multi-institutional retrospective study was conducted to determine the prognostic factors and outcomes for pediatric osteosarcoma in SEA., Methods: Pediatric patients with osteosarcoma treated between 1998 and 2017 in 4 SEA pediatric oncology centers were studied. Countries were classified using the World Bank Atlas method. Kaplan-Meier method and Cox's Proportion Hazard Model were applied to estimate survival outcomes and identify prognostic factors., Results: In all, 149 patients with osteosarcoma with a mean age of 12.48±3.66 years were enrolled. The localized to metastatic disease ratio was 1.5:1. The 5-year overall survival (OS) and event-free survival (EFS) were 53.8% and 42%, respectively. Prognostic factors associated with outcomes were country, stage of disease, MTX-containing regimens, and surgery type (p-value <0.05). In patients with localized disease, EFS was superior with limb-salvage surgery (62%) than amputation or rotationplasty (40%) (p-value 0.009). MTX-containing chemotherapies provided higher OS (45.3%) and EFS (37.9%) than non-MTX regimens (12.3% and 10.7%, respectively) among metastatic patients (p-value 0.004 and 0.005, respectively). Metastatic disease was an independent prognostic factor for death but not relapse outcome.  Conclusion: The disease outcomes in SEA were acceptable compared to developed countries. The stage of disease was the only independent prognostic factor. MTX-containing regimens and limb-salvage surgery should be considered where possible.

Background: Langerhans cell histiocytosis (LCH) is a rare disease characterized by the various systems involved and clinical manifestations with a wide range of symptoms., Objectives: To describe clinical characteristics, imaging, treatment, and outcomes of pediatric LCH at Phramongkutklao Hospital, Bangkok, Thailand., Methods: We conducted a 20-year retrospective review of the medical records of patients diagnosed with LCH from birth to 21 years old from January 1, 1997, to December 31, 2016., Results: In all, 14 patients with median age of 2.5 years were studied. Six (43%) patients had single-system (SS) LCH. Five patients (63%) with multisystem (MS) LCH (n = 8. 57%) had risk-organ involvement (RO+). All patients had plain X-ray imaging of their skull with 11 (79%) showing abnormal findings. Tc-99m bone imaging and fluorodeoxyglucose F18 (FDG) positron emission tomography (PET)-computed tomography (CT) demonstrated abnormal findings in 8 (89%) and 4 (29%) patients, respectively. The 5-year event-free survival (EFS) for patients with RO+ MS-LCH was less than that for those without risk-organ involvement (RO-) MS-LCH and SS-LCH (20% vs. 100%, P = 0.005). Hematological dysfunction, hypoalbuminemia, and conjugated hyperbilirubinemia may be worse prognostic factors for RO+ MS-LCH., Conclusion: FDG-PET-CT might have a greater accuracy to detect LCH disease than conventional plain X-ray and Tc-99m bone imaging. RO+ MS-LCH has been encountered with relapse and poor outcomes. Hematopoietic involvement, hypoalbuminemia, and conjugated hyperbilirubinemia may be worse prognostic factors for RO+ MS-LCH., (© 2021 Ponrachet Kitticharoenjit et al., published by Sciendo.)

Hepatitis B is a major global health concern and can be prevented in the era of vaccination. Impaired immunological memory to primary immunization is a common chemotherapy-related complication among cancer survivors. The study aimed to determine protective immunity against hepatitis B virus (HBV) and anamnestic response to booster vaccination. In all, 107 pediatric cancer survivors previously immunized with primary hepatitis B vaccination were enrolled. A hepatitis B booster dose was administered to those with suboptimal seroprotection (anti-HBs < 10 mIU/mL) and 2 additional doses were subsequently administered at 1 and 6 months to those whose anti-HBs remained low. Clinical and serologic parameters were analyzed. Sero-protective rate against HBV (anti-HBs ≥ 10 mIU/mL) among survivors was 20.6% with geometric mean titer (GMT) of 95.7 ± 265.6 mIU/mL. Anamnestic response was 61% after a booster vaccine among those with suboptimal seroprotection and 100% after 2 additional booster doses among those whose anti-HBs remained low. GMTs among those survivors after the First and third booster vaccines were 320.0 ± 412.4 mIU/mL and 826.5 ± 343.8 mIU/mL, respectively. Age at diagnosis was a significant independent risk factor for adequate seroprotection (adjusted OR = 0.84, 95%CI: 0.71-0.99) with a P -value of .034. No associated risk factors to predict optimal anamnestic response to booster vaccination were identified. Loss of immunological memory to primary hepatitis B immunization is an inevitable complication among most pediatric cancer survivors; therefore, assessing adequate seroprotection is essentially required. For those with limited accessibility to serologic tests, completion of full 3-booster-dose series is alternative and highly recommended., Competing Interests: Declaration of Conflicting Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2021.)

Background: Chemotherapy-induced nausea and vomiting (CINV) is a common complication in cancer treatment. Ondansetron is an effective antiemetic drug widely used to prevent CINV; however, the effective administrative dosing strategies among pediatrics remain unclear. The study aimed to investigate clinical effectiveness of single daily dosing versus divided dosing ondansetron., Methods: In all, 194 children undergoing chemotherapy were randomized to receive either single daily dosing (0.3 mg/kg/dose) or divided dosing (0.15 mg/kg/dose every 8 hours) intravenous ondansetron for 24 hours. Clinical parameters including number of emesis episodes, nausea scores, appetite levels, parent's satisfaction, and adverse effects within 24 hours were analyzed., Results: No significant differences were found between the two dosing strategies concerning number of emesis episodes and parent's satisfaction. However, nonleukemic hematologic malignancies and concurrent administration of intrathecal methotrexate-hydrocortisone-cytarabine (IT-MHA) were associated with increased risk of acute-phase vomiting. Interestingly, none of the patients aged under 7 years, receiving divided dosing ondansetron, presented nausea symptoms compared with those receiving single daily dosing (p-value .034). No significant differences regarding headache were observed between the two dosing strategies and none of the patients experienced QTc prolongation., Conclusion: Ondansetron administered as divided dosing should be considered among children aged under 7 years to prevent chemotherapy-induced nausea and among patients receiving low emetogenic chemotherapy to maintain their appetite. Both administrative dosing strategies were well tolerated with no significant adverse effects., (© 2021 Wiley Periodicals LLC.)

Early recognition and management are the key elements to prevent febrile neutropenia associated mortality. The prospective observational study aimed to investigate prognostic accuracy of serum lactate to predict septic shock within 48 hours among hemodynamically stable children with febrile neutropenia. In all, 99 pediatric oncology patients who developed febrile neutropenia were enrolled in the study. Clinical information during 48 hours and serum lactate at the time of enrollment were analyzed. Among 99 participating patients, 10 developed septic shock and 4 of those expired. No significant difference was found of patients' baseline characteristics and basic laboratory parameters between patients with and without septic shock. Serum lactate was significantly elevated among patients developing septic shock ( P -value < .001) and those who expired ( P -value .002). Receiver operating characteristic (ROC) curve was created to identify the best cutoff value for initial serum lactate associated with the development of septic shock within 48 hours. Baseline serum lactate more than 2.5 mmol/L showed the largest area under the ROC curve to predict the septic shock development within 48 hours (ROC area, 0.90; 95% confidence interval [CI], 0.81-0.98), with sensitivity, specificity, negative predictive value, and accuracy of 80.0%, 92.1%, 97.6%, and 90.9%, respectively. Serum lactate level determined early at the time of febrile neutropenia was an effective surrogate marker for developing septic shock within 48 hours among hemodynamically stable, pediatric oncology patients. The level more than 2.5 mmol/L was the best threshold to start preemptive aggressive hemodynamic monitoring and prompt treatment to ensure adequate tissue perfusion., Competing Interests: Declaration of Conflicting Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article., (© The Author(s) 2021.)

Background: Glucose-6-phosphate dehydrogenase (G6PD) deficiency is the most common X-linked inherited erythroenzymopathy in Thailand. The clinical and hematological manifestations of G6PD deficiency are variable., Objective: This study aimed to characterize the genotype-phenotype correlation of G6PD mutations in Thai pediatric patients who were followed-up in Phramongkutklao Hospital, a tertiary center in central Thailand. Material and Method . A total of 102 children including 73 males (71.6%) and 29 females (28.4%) were included in our study. Mutation analysis was performed by direct DNA sequencing of all coding exons of the G6PD gene. Ninety-one patients (89.2%) were presented with neonatal hyperbilirubinemia and 11 patients (10.8%) were presented with acute hemolytic anemia beyond the neonatal period., Results: Molecular analysis of the G6PD gene in 102 G6PD-deficient Thai children identified 12 different mutations. G6PD Viangchan (871G > A) and G6PD Canton (1376G > T) were the first (46.2%) and the second (15.4%) most common identified mutations among both male and female G6PD-deficient individuals, respectively. All affected males were hemizygous for G6PD mutations and had an average G6PD level of 16.7 ± 11.5 (3-76) IU/ml.RBC. Majority of female patients (27 in 29, 93.1%) were heterozygous for G6PD mutations and had an average G6PD level of 133.6 ± 43.4 (9-195) IU/ml.RBC. Two female patients (6.9%) were either homozygous or compound heterozygous for the mutations and had G6PD level in the affected male range (35 and 10 IU/ml.RBC). Only 1 in 27 heterozygous females (3.7%) had G6PD level in the affected male range (9 IU/ml.RBC) which is possibly explained by nonrandom X-chromosome inactivation. The correlation of genotypes, G6PD levels, and clinical phenotypes was not demonstrated in our study in which all of the included G6PD-deficient patients were presented with neonatal hyperbilirubinemia and acute hemolytic anemia, since the genotype-phenotype correlation is normally demonstrated in chronic nonspherocytic hemolytic anemia (CNSHA) G6PD-deficient individuals., Conclusion: This study characterizes the molecular heterogeneity of G6PD variants causing G6PD deficiency in Thai children. Our study demonstrated the efficiency of direct DNA sequencing which can identify 12 missense mutations in Thai children., Competing Interests: The authors declare that they have no conflicts of interest., (Copyright © 2021 Boonchai Boonyawat et al.)

Background: Although congenital infantile fibrosarcoma (cIFS) is a rare soft tissue sarcoma among children, it constitutes one of the most common soft tissue sarcomas during the first year of life. Congenital mesoblastic nephroma (CMN) is the most common benign renal tumor usually developing during the first 3 months of life. cIFS and cellular type CMN (cCMN) share not only similar histopathologic features but identical molecular genetic abnormality including the ETV6/NTRK3 fusion gene. Here, we report an unusual case of cIFS occurring with cCMN., Case Presentation: An 18-month-old girl presented with a 1-month history of abdominal distension and a few days' history of a palpable abdominal mass. A large heterogenous mass sized 9.0×11.2×11.6 cm on the right side of the abdomen and an isolated heterogenous lesion sized 4×4.5 cm within the right kidney were noted from the imaging study. Pathologic findings were consistent with cIFS and cCMN of the right kidney. In addition, both pathologic specimens contained the ETV6/NTRK3 fusion gene., Conclusion: Although cIFS and cCMN share similar histopathologic features and molecular genetic abnormality, simultaneous occurrence of these 2 types of tumor is exceedingly rare. To our knowledge, this is the first unusual case report of concurrent cIFS and cCMN.

Objective: This study was designed to evaluate the applicability and effectiveness of the enhanced informed consent form (ICF) methodology, proposed by the Strategic Initiative for Developing Capacity in Ethical Review (SIDCER), in paediatric research requiring parental consent. The objective of this study was to compare the parental understanding of information between the parents who read the SIDCER ICF and those who read the conventional ICF., Design: A prospective, randomized, controlled design., Setting: Paediatric Outpatients Department, Phramongkutklao Hospital, Thailand., Participants: 210 parents of children with thalassemia (age=35.6 ± 13.1 years)., Interventions: The parents were randomly assigned to read either the SIDCER ICF (n=105) or the conventional ICF (n=105) of a paediatric drug trial., Primary and Secondary Outcome Measures: Parental understanding of trial information was determined using 24 scenario-based questions. The primary endpoint was the proportion of parents who obtained the understanding score of more than 80%, and the secondary endpoint was the total score., Results: Forty-five parents (42.9%) in the SIDCER ICF group and 29 parents (27.6%) in the conventional ICF group achieved the primary endpoint (relative risk=1.552, 95% CI 1.061 to 2.270, p=0.021). The total score of the parents in the SIDCER ICF group was significantly higher than the conventional ICF group (18.07±3.71 vs 15.98±4.56, p=0.001)., Conclusions: The SIDCER ICF was found to be superior to the conventional ICF in improving parental understanding of trial information., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Background: Neuroblastoma is the most common extra-cranial solid tumor among children. Despite intensive treatment, patients with advanced disease mostly experience dismal outcomes. Here, we proposed the use of topotecan and cyclophosphamide containing induction regimen as an upfront therapy to high risk neuroblastoma patients., Methods: Patients with high risk neuroblastoma undergoing ThaiPOG high risk neuroblastoma protocol from 2016 to 2017 were studied. All patients received 6 cycles of induction regimen consisting of 2 cycles topotecan (1.2 mg/m 2 /day) and cyclophosphamide (400 mg/m 2 /day) for 5 days followed by cisplatin (50 mg/m 2 /day) for 4 days combined with etoposide (200 mg/m 2 /day) for 3 days on the third and fifth cycles and cyclophosphamide (2100 mg/m 2 /day) for 2 days combined with doxorubicin (25 mg/m 2 /day) and vincristine (0.67 mg/m 2 /day) for 3 days on the fourth and sixth cycles. Treatment response after the 5th cycle before surgery and treatment-related toxicities after each topotecan containing induction cycle were evaluated. Relevant prognostic factors were analyzed to measure the treatment response among those patients., Results: In all, 107 high risk neuroblastoma patients were enrolled in the study. After the 5th cycle of induction regimen, the patients achieved complete response (N = 2), very good partial response (N = 40), partial response (N = 46) and mixed response (N = 19). None of the patients experienced stable disease or disease progression. The most significant prognostic factor was type of healthcare system. The most common adverse effect was febrile neutropenia followed by mucositis, diarrhea and elevated renal function., Conclusion: The topotecan and cyclophosphamide containing induction regimen effectively provides favorable treatment response. The regimen is well tolerated with minimal toxicity among patients with high risk neuroblastoma in Thailand.

Background: Thalassemia is a common congenital hemolytic disorder. In severe cases, regular blood transfusion is essentially required. The role of premedications to prevent transfusion reactions is varied among institutions with no standard guideline., Objective: To prospectively compare the risk of transfusion reactions in thalassemia patients premedicated with acetaminophen and chlorpheniramine maleate (CPM) versus placebo prior to blood transfusion., Material and Method: A randomized, double-blinded, placebo-controlled transfusion reaction study of 147 eligible patients was analyzed. All administered red blood cell (RBC) products were leukoreduced blood products. Patients were monitored and followed for the development of transfusion reactions for 24 hours after RBC transfusion., Results: A total of 73 patients randomized to receive active drugs consisting of acetaminophen and CPM were compared to 74 patients receiving placebo. The overall incidences of febrile reaction and urticarial rash were 6.9% and 22% in the patients randomized to receive active drugs comparing with 9.5% and 35.2% in the patients receiving placebo with no significant differences between two groups. However, delayed development of urticarial rash at 4-24 hours after RBC transfusion was significantly higher in female and patients receiving placebo., Conclusion: Administration of premedications in thalassemia patients receiving RBC transfusion without a history of transfusion reactions does not decrease the overall risk of transfusion reactions. However, the use of CPM might be beneficial to prevent delayed urticarial rash in those patients especially in females (Thai Clinical Trial Registry (TCTR) study ID: 20140526001).

Objective: This study aimed to test the applicability and effectiveness of the enhanced informed consent form (ICF) methodology, proposed by the Strategic Initiative for Developing Capacity in Ethical Review (SIDCER), in neonatal research requiring maternal consent., Design: A single-centre open-label randomised controlled study., Setting: Antenatal care clinics at Phramongkutklao Hospital, Thailand., Patients: 234 pregnant women who were at risk of preterm labour were enrolled; 232 individuals completed the study., Interventions: The participants were randomly assigned to read either the SIDCER ICF or the conventional ICF., Main Outcome Measures: The participants' understanding of essential trial-related information was assessed using 25 closed-ended questions. The primary endpoint was the proportion of the participants who obtained the satisfactory level of understanding at 80% (score of ≥20/25)., Results: 72.5% (87/120) of the participants in the SIDCER ICF group and 59.8% (67/112) of the conventional ICF group achieved the primary endpoint (relative risk (RR)=1.212, 95% CI 1.005 to 1.462, p=0.041). The superiority of the SIDCER ICF over the conventional ICF was significant, particularly among the participants whose education was at the high school level or below (63.5% vs 44.1%, RR=1.441, 95% CI 1.022 to 2.030, p=0.031)., Conclusions: The SIDCER ICF methodology is applicable to neonatal research requiring maternal consent. The SIDCER ICF significantly improved the understanding of pregnant women, particularly among those with lower levels of education. The present study confirms the value of the SIDCER ICF methodology in research involving individuals with a limited academic background., Competing Interests: Competing interests: None declared, (© Article author(s) (or their employer(s) unless otherwise stated in the text of the article) 2018. All rights reserved. No commercial use is permitted unless otherwise expressly granted.)

Background: Alpha-thalassemia is a common genetic disorder in Thailand and is caused by either deletion or non-deletional mutation of one or both α-globin genes. Inactivation of three α-globin genes causes Hb H disease and interaction of Hb H disease with heterozygous Hb E results in AEBart’s disease., Objective: The present study aimed to characterize the genotype of α-globin gene in 81 pediatric patients with Hb H and AEBart’s diseases in Phramongkutklao Hospital, a tertiary care center for thalassemic patients in central Thailand., Material and Method: Eighty one unrelated pediatric patients including 60 patients with Hb H disease and 21 patients with AEBart’s disease were enrolled in our study. Mutation analysis was performed by multiplex gap-PCR, multiplex-ARMS and direct DNA sequencing of both HBA1 and HBA2 genes, respectively., Results: A total of 81 pediatric patients with Hb H and AEBart’s diseases who mainly lived in central Thailand were included in the present study. Eight different α-thalassemia mutations interacting to produce seven genotypes of α-globin gene in both Hb H and AEBart’s diseases were identified. The number of patients in the non-deletional form was higher than in the deletional form for both Hb H (51.6% VS 48.4%) and AEBart’s diseases (52.4% VS 47.6%). The SEA deletion (--SEA) was the most common (98.8%) α-thalassemia 1 mutation. While 3.7-kb deletion (-α3.7) was the most common (90%) α-thalassemia 2 deletion, Hb CS was the most common (90%) non-deletional a-thalassemia 2. Uncommon non-deletional α-thalassemia 2 mutation identified in our study were Hb QS, Hb PS and initiation codon mutation, respectively., Conclusion: All of the α-thalassemia mutation in our pediatric patients with Hb H and AEBart’s diseases have been characterized by the combination of molecular techniques including multiplex gap-PCR, multiplex-ARMS and DNA sequencing of HBA1 and HBA2 genes.

More than 80% of acute lymphoblastic leukemia (ALL) in pediatric population is curable by using combinations of chemotherapy. However, 20% of the cases still suffer from disease relapse. The most common site of relapse is bone marrow. Relapse of childhood ALL involving the eyeball is rare. However, it occurs in 2.2% of relapsing children. The authors described a 10-year-old Thai boy with underlying ALL on therapy, presented with a one-month history of progressive visual loss of his right eye. The clinical and imaging studies strongly suggested the diagnosis of isolated ocular relapse. In this report, the authors presented the findings from successfully specific treatment consisting of systemic chemotherapy and radiation therapy on the affected eye. From other studies, the outcome was more favorable in cases of ocular relapse off therapy. In our study, one case of isolated ocular relapse ALL was reported.

Background: Children often require relief of pain and anxiety when undergoing painful procedures., Objective: To determine the differences by comparing fentanyl and ketamine used in cancer-diagnosed children undergoing painful procedures., Material and Method: A randomized, double-blinded, crossover trial was conducted with 55 children undergoing painful procedures (intrathecal chemotherapy and/or bone marrow aspiration/biopsy). Patients were randomly assigned in a double-blinded fashion to receive either intravenous fentanyl or ketamine at 1 mcg/kg/dose and 1 mg/kg/dose, respectively. The result in effectiveness of the drug was measured using three parameters, 1) satisfaction score ranging from 0 to 10, 2) perception of procedural pain using FLACC scale, Wong-Baker FACES Pain Rating Scale and Visual Analog Scale, and 3) the frequency of vomiting nausea score., Results: The satisfaction amongpatients receiving fentanyl was significantly greater than ketamine (p = 0.007). In addition, both painful and nausea/vomiting were significantly decreased in the patients receiving fentanyl (p = 0.002 and p < 0.001, respectively). No serious complications were observed, Conclusion: This study demonstrated that intravenous fentanyl generated a superior clinical effect in satisfaction, decreased pain and nausea/vomiting, and showed no significant side-effects over ketamine. Fentanyl may also be recommended as a reasonable option before undergoing oncology procedures in children with cancer.

Background: Leukemia is the most common malignancy in children. Multiple prognostic factors have been used in order to assist the clinician to decide appropriate risk-adjusted treatment for each patient; the current clinical outcomes of those patients have been significantly improved over the past decades., Objective: The purpose of this study was to examine survival outcome in children who were diagnosed with acute leukemia and treated in the Department of Pediatrics, Phramongkutklao Hospital during January 1, 2000 and July 31, 2013., Material and Method: The authors retrospectively reviewed the patients who were diagnosed with acute leukemia and treated at Phramongkutklao Hospital. Their clinical data were collected and analyzed based on clinicalfeatures inchluding age, initial WBC count at diagnosis, sex, immnunophenotype and cytogenetic abnormalities., Results: Total 152 patients with acute leukemia, 123 patients were diagnosed with acute lymnphoblastic leukemia (ALL) and 29 patients were diagnosed with acute myeloid leukemia (AML). The 5-year survival rates of ALL and AML patients were 72.63% and 30.30%, respectively. In addition, we found a correlation between the ALL patients' clinical outcomes and several prognostic factors including initial white blood cell count, CNS status at diagnosis and ploidy. However, there was no correlation between those factors and clinical outcomes in AML patients., Conclusion: Our treatment outcomes on patients with acute leukemia were similar to the reports from other countries. The several prognostic factors especially initial WBC at diagnosis can assist the clinician to select appropriate treatment option for each patient.

Background: The pain and its complication during sedation with ketamine remain a significant problem for children with hematologic malignancy., Objective: The purpose of the present study was to evaluate further the parental satisfaction for procedural sedation and analgesia during pediatric hematology/oncology procedures perfonned by pediatrician in the Department of Pediatrics, Phramongkutklao Hospital., Material and Method: The authors prospectively evaluated our experience using intravenous ketamine 1 mg/kg for oncology patients undergoing procedures at Department ofPediatrics, Phramongkutklao Hospital. The procedure was assessed by way ofaphysician-completed form and by evaluation of questionnaires given to parents to estimate levels of pain by using a 0 to 10 mm visual analog scale (VAS) at 2 hours after procedures and results in any adverse events with respect to age, gende, procedures performed, ketamine dosage and recovery time., Results: Total of46 children aged 6 months to 15 years with 46procedures were observed at pediatric unit post-procedure. The indicationsfor procedural sedation and analgesia included lumbar puncture and intrathecal chemotherapy (50%), bone marrow aspiration or biopsy (21.7%), and both plrocedures (28.3%). The median VAS scale during oncology procedures was 3, which were expressed by all the parents/guardians of the children treated. Adverse effects were observed in all children including nausea (30.4%), hypersalivation (26.1%), vomiting (21.7%), hallucinlation (4.2%). No child required admission to hospital and there were no serious complications., Conclusion: hntravenous ketamine 1 mg/kg is effective for invasive procedures in children with malignancy. The use of intravenous ketamine may produce psychedelic effects in children. These adverse effects may alter the child's comfort and parental satisfaction especially in the young children.

Background: Though attention deficit, hyperactivity disorder ADHD is a common problem in childhood. Thai teachers' knowledge regarding the disease has never been assessed., Objective: To identify knowledge of Thai teachers regardingADHD and its influencingfactors., Material and Method: Cross-sectional study was operated in three primary schools in Ayutthaya, Thailand. Standardized questionnaires comprised ofdemographic data, ADHD experiences and the Knowledge of Attention Deficit Disorder Scale, KADDS, were distributed to participating teachers. Results were reported using frequency, percent, mean, and standard deviation. Association between demographic and ADHD experiences and the KADDS score was identified by logistic regression analysis., Results: Lack ofknowledge of ADHD among teachers was apparent. Only 19.4% of them passed the total scale of KADDS. Teachers under 31-years-old were more likely to pass general information and signs, symptoms & diagnosis subscales and total scale. In addition, familiarity with ADHD patients was associated with passing scores of general information subscale and total scale., Conclusion: Despite public awareness of ADHD, Thai teachers lacked knowledge concerning the disease. Young teachers were more acquainted with ADHD. Direct experience with ADHD patient might help teachers develop their knowledge on ADHD.

Kasabach-Merrit phenomenon (KMP) is a rare disorder of pediatric hematological malignancies which is previously referred to Kasabach-Merrit syndrome (KMS). The disorder is characterized by prominent vascular mass resulting from abnormal proliferation of blood vessels, consumption coagulopathy and thrombocytopenia. The diagnosis is based upon three basic findings as above. The authors describe a 6-month-old girl with a huge ecchymotic mass at left buttock who is found to have thrombocytopenia and consumption coagulopathy. The clinical and imaging studies strongly suggested the diagnosis of KMP. Vincristine was administered after a trial of corticosteroids was failed to show clinical and laboratory improvement. After 2 weeks, the patient showed that the platelet count and fibrinogen level become to be normal without blood transfusion and gradually decreased in tumor size without any surgical procedure in 4 weeks and disappeared in 5 months without any complications. In the present report, the authors present the findings from successfully specific treatment with vincristine alone and supportive treatment for life-threatening hemorrhage with platelet concentrate and cryoprecipitate should be the best management in our situation.