Health-related quality of life and its determinant factors in Thai children with cancer: parents vs. children perspectives.

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/ .

Improving comprehension, recall and attention using multimedia-informed assent among pediatric oncology patients: A comparative randomized controlled trial.

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.

Treatment outcomes among high-risk neuroblastoma patients receiving non-immunotherapy regimen: Multicenter study on behalf of the Thai Pediatric Oncology Group.

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.

Impact of treatment refusal and abandonment on survival outcomes in pediatric osteosarcoma in Southeast Asia: A multicenter study.

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.

The efficacy and safety of midazolam with fentanyl versus midazolam with ketamine for bedside invasive procedural sedation in pediatric oncology patients: A randomized, double-blinded, crossover trial.

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.

Single daily dosing versus divided dosing intravenous ondansetron to prevent chemotherapy-induced nausea and vomiting among children: A comparative randomized double-blind controlled trial.

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.

Unusual Case of Concurrent Retroperitoneal Congenital Infantile Fibrosarcoma and Cellular Type Congenital Mesoblastic Nephroma.

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.

Clinical outcomes and prognostic factors to predict treatment response in high risk neuroblastoma patients receiving topotecan and cyclophosphamide containing induction regimen: a prospective multicenter study.

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/m2/day) and cyclophosphamide (400 mg/m2/day) for 5 days followed by cisplatin (50 mg/m2/day) for 4 days combined with etoposide (200 mg/m2/day) for 3 days on the third and fifth cycles and cyclophosphamide (2100 mg/m2/day) for 2 days combined with doxorubicin (25 mg/m2/day) and vincristine (0.67 mg/m2/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.

Molecular Characterization of Hb H and AEBart's Diseases in Thai Children: Phramongkutklao Hospital Experiences.

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.

The clinical effect of fentanyl in comparison with ketamine in analgesic effect for oncology procedures in children: a randomized, double-blinded, crossover trial.

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.

Isolated Ocular Relapse in Childhood Acute Lymphoblastic Leukemia during Second Interim Maintenance Phase of Chemotherapy: Case Report.

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.

Knowledge of attention deficit hyperactivity disorder and its associated factors among teachers in 3 large primary schools in Phra Nakorn Sri Ayutthaya Province, Thailand.

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.

Pediatric acute leukemia: the effect of prognostic factors on clinical outcomes at Phramongkutklao Hospital, Bangkok, Thailand.

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.

The factors of ketamine that affect sedation in children with oncology procedures: parent satisfaction perspective.

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.

Nationwide Study of Factors Impacting Survival Outcome and Consequences in Children with Reactivation/Refractory Langerhans Cell Histiocytosis.

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.

Pediatric hemophagocytic lymphohistiocytosis in a tropical country: Results of a multicenter study in Thailand.

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/mm3 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/mm3 was the only independent prognostic factor to define survival outcome.

Practices and challenges for hemophilia management under resource constraints in Thailand.

Hemophilia is an inherited bleeding disorder caused by deficiency of a specific coagulation factor. Factor VIII deficiency is responsible for hemophilia A while factor IX deficiency is responsible for hemophilia B. As per the 2020 annual global survey by the World Federation of Hemophilia, only 1828 Thai hemophiliacs have been registered to the national healthcare system. The reason for the low number is the underdiagnosis which is a major concern in the real-world practice among Asian countries. In Thailand, most hemophiliacs are diagnosed by general practitioners, pediatricians or internists at rural hospitals and are referred to hemophilia specialists at the Hemophilia Treatment Centers (HTCs). Despite the challenges pertaining to infrastructure and cost of treatment, Thailand has progressed substantially in providing the required hemophilia care, as evidenced by an evolution in acquiring and sharing knowledge as well as collaborative efforts among multiple stakeholders over the past three decades. In this letter-to-the-editor, the authors have summarized the practices for and challenges faced with hemophilia management in Thailand.

National strategic advocacy to manage patients with inherited bleeding disorders in low and lower-middle income countries.

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.

Outcome of Kasabach-Merritt phenomenon: the role of vincristine as monotherapy: report of a case.

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.

The Reliability of the Thai version of Health-Related Quality of Life Questionnaire: PedsQL 3.0 Cancer Module.

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.