The relationship between liver stiffness by two-dimensional shear wave elastography and iron overload status in transfusion-dependent patients.

Increased liver stiffness (LS) can be result of increased liver iron concentration (LIC) which may not yet be reflected in the liver fibrotic status. The objective of our study was to examine relationship between hemochromatosis, LS, and serum ferritin level in transfusion-dependent patients. We recruited all 70 transfusion-dependent patients, whose median age was 15, referred for evaluating LIC status by magnetic resonance imaging (MRI) followed by two-dimensional ultrasonography shear wave elastography (2D-SWE). Thalassemia beta affected the majority of the patients. The optimal cut point for prediction of severe hemochromatosis using median SWE (kPa) and SWV (m/s) was ≥ 7.0 kPa and ≥ 1.54 m/s, respectively, with sensitivity of 0.76 (95% confidence interval [CI] 0.55, 0.91) and, specificity of 0.69 (95%CI 0.53, 0.82). When combing the optimal cut point of SWE (kPa) at ≥ 7.0 and serum ferritin ≥ 4123 ng/mL, the sensitivity increased to 0.84 (95%CI 0.64, 0.95) with specificity of 0.67 (95%CI 0.50, 0.80), positive predictive value (PPV) of 0.60 (95%CI 0.42, 0.76), and negative predictive value (NPV) of 0.88 (95%CI 0.71, 0.96). Simultaneous tests of 2D-SWE and serum ferritin for prediction of severe hemochromatosis showed the highest sensitivity of 84% (95%CI 0.64-0.95), as compared to 2D-SWE alone at 76% (95%CI 0.55, 0.91) or serum ferritin alone at 44% (95%CI 0.24-0.65). We recommend measuring both 2D-SWE and serum ferritin in short interval follow up patients. Adding 2D-SWE to management guideline will help in deciding for aggressive adjustment of iron chelating medication and increased awareness of patients having severe hemochromatosis.

Survival outcomes of myeloid leukemia associated with Down syndrome and de novo acute myeloid leukemia in children: Experience from a single tertiary center in Thailand.

Few studies have reported the survival outcomes of myeloid leukemia associated with Down syndrome (DS) in resource-limited countries. This study aimed to compare characteristics and survival outcomes of children with acute myeloid leukemia (AML) between those with and without DS in Thailand. The medical records of AML patients aged 0-15 years treated in a major tertiary center in Southern Thailand between October 1978 and December 2019 were reviewed retrospectively. The overall (OS) and event-free survivals (EFS) rates were calculated using the Kaplan-Meier method. A total of 362 AML patients were included, of which 41 (11.3%) had DS. The mean age at diagnosis of the DS patients was 2.5 ± 1.9 years and most of them (90.2%) were under the age of five. The DS patients had lower initial white blood cell counts and peripheral blasts compared to the non-DS patients. The AML-M7 subtype was more common in the DS than in the non-DS patients (80.5% vs. 9.1%, p < 0.01, respectively). The 5-year OS and EFS rates of the DS patients were lower compared to the non-DS patients (12.9% vs. 20.5%, p = 0.05 and 13.7% vs. 18.4%, p = 0.03, respectively). DS patients had a significantly higher rate of early and treatment-related deaths compared to non-DS patients (30.3% vs. 13.5%, p < 0.01 and 39.4% vs. 19.5%, p = 0.02, respectively). Over the study period, there were a decrease in early death rate and an increase in survival rates of DS patients, which suggests that chemotherapy regimens and supportive care have improved over time.

Prevalence and risk factors of disseminated intravascular coagulation in childhood acute lymphoblastic leukemia.

BACKGROUND: Few studies have examined disseminated intravascular coagulation (DIC) in childhood acute lymphoblastic leukemia (ALL). Our aims were to evaluate the prevalence, risk factors and outcomes of DIC at ALL presentation and during induction chemotherapy. METHODS: The medical records of ALL patients aged <15 years were retrospectively reviewed. Logistic regression analysis was used to identify risk factors. The Kaplan-Meier method was used to depict survival. RESULTS: Of the 312 patients, 48 (15.4%) and 76 (24.4%) had DIC at presentation and during induction chemotherapy, respectively. Risk factors for DIC at presentation (OR and 95% CI) were antibiotics prior to admission 2.34 (1.17-4.89), white blood cell count ≥100 × 109/L 2.39 (1.04-5.72), platelets <100 × 109/L 5.44 (1.84-23.4) and high National Cancer Institute (NCI) risk 2.68 (1.08-6.62). Risk factors for DIC during induction chemotherapy were antibiotics prior to admission 1.86 (1.07-3.27), high peripheral blasts 1.01 (1.00-1.02) and transaminitis 2.02 (1.18-3.48). Five-year overall survival of patients who had DIC was significantly lower than those who did not (45.0% vs. 74.1%, p <0.001). CONCLUSION: Antibiotics prior to admission, hyperleukocytosis, thrombocytopenia and high NCI risk were risk factors of DIC at presentation. Antibiotics prior to admission, high peripheral blasts and transaminitis were risk factors of DIC during induction chemotherapy. IMPACT: There are only two studies, both published before 2000, evaluating risk factors of DIC in pediatric ALL patients without reporting outcomes. DIC was associated with lower remission and survival rates in pediatric ALL patients. We identified the risk factors of DIC at presentation as antibiotics prior to admission, hyperleukocytosis, thrombocytopenia and high NCI risk. The risk factors of DIC during induction chemotherapy were antibiotics prior to admission, high peripheral blasts and aspartate transaminitis. Pediatric ALL patients who have the aforementioned risk factors should be closely monitored for DIC secondary to infection, and early treatment with appropriate antimicrobial agents is recommended.

Transient abnormal myelopoiesis in Down syndrome: Experience of long term follow up from a single tertiary center in Thailand.

Transient abnormal myelopoiesis (TAM) is a unique disease occurring in Down syndrome (DS) infants from which most patients have spontaneous remission. This study aimed to evaluate the incidence and outcomes of TAM in a tertiary center in Thailand. We reviewed the records of 997 DS patients diagnosed between June 1993 and October 2019. From the 997 DS patients, 32 had been diagnosed with TAM. The incidence of TAM was 3.2% and an overall survival rate of 87.5%. A total of 2/28 who survived (7.1%) subsequently developed AML-DS at the ages of 2.1 and 4.5 years, respectively. The risk factors related with death included maternal multiparity, sepsis, skin bleeding, subcutaneous nodules, high WBC count, low hemoglobin, and elevated AST level.Abbreviations.

Treatment outcomes in childhood acute lymphoblastic leukemia: 40-year experience from a single tertiary center in Thailand.

Studies on the long-term treatment outcomes of childhood acute lymphoblastic leukemia (ALL) in resource-limited countries are scarce. The purpose of this study was to assess the evolution of survival outcomes of pediatric ALL in a tertiary care center in Thailand over a 40-year period. We retrospectively reviewed the medical records of pediatric patients who were diagnosed with ALL and treated at our center between June 1979 and December 2019. We classified the patients into 4 study periods depending on the therapy protocol used to treat the patients (period 1: 1979-1986, period 2: 1987-2005, period 3: 2006-2013, and period 4: 2014-2019). The Kaplan-Meier method was used to determine overall and event-free survival (EFS) for each group. The log-rank test was used to identify statistical differences. Over the study period, 726 patients with ALL were identified, 428 boys (59%) and 298 girls (41%), with a median age at diagnosis of 4.7 years (range: 0.2-15 years). The study periods 1, 2, 3, and 4 had 5-year EFS rates of 27.6%, 41.6%, 55.9%, and 66.4%, and 5-year overall survival (OS) rates of 32.8%, 47.8%, 61.5%, and 69.3%, respectively. From periods 1 to 4, both the EFS and OS rates increased significantly (p <. 0001). Age, study period, and white blood cell (WBC) count were all significant prognostic indicators for survival outcomes. The OS of patients with ALL treated in our center improved significantly over time from 32.8% in period 1 to 69.3% in period 4.

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.

Acute kidney injury and childhood acute myeloid leukemia.

BACKGROUND: Acute kidney injury (AKI) appears to be particularly common in children with acute myeloid leukemia (AML), although the epidemiology data on this patient population is sparse. The objective of this study was to assess the prevalence and factors associated with AKI in childhood AML during chemotherapy treatment. METHODS: The medical records of 112 children aged under 15 years diagnosed with AML who received chemotherapy in a major tertiary-care referral center in southern Thailand were reviewed. Logistic regression was used to identify factors associated with AKI. RESULTS: Fifty-six (50%) children had AKI events. The median time from AML diagnosis to the first AKI was 29.5 days (interquartile range: 11.0-92.8) and the median follow-up time was 10.9 months (interquartile range: 3.6-31.1). Age at diagnosis ≥ 10 years (OR 2.75, 95% CI 1.09-6.93), glomerular filtration rate < 90 mL/min/1.73 m2 at AML diagnosis (OR 7.58, 95% CI 1.89-30.5), and septic shock (OR 22.0, 95% CI 4.63-104.3) were independently associated with AKI. CONCLUSIONS: Childhood AML has a high rate of kidney injury with 50% having AKI. Age ≥ 10 years at diagnosis, impaired kidney function before treatment, and septic shock were strongly associated with AKI. A higher resolution version of the Graphical abstract is available as Supplementary information.

Predictive factors for adverse outcome of advanced-stage childhood lymphoblastic lymphoma: a single tertiary center retrospective study in Thailand.

Childhood lymphoblastic lymphoma (LL) is a highly aggressive neoplasm which has achieved favorable survival outcomes in many developed countries. However, few studies have reported treatment outcomes of childhood LL in resource-limited counties, nor has a prognostic scoring system been developed. The objectives of this study were to evaluate survival outcomes and identify prognostic factors associated with inferior outcomes of childhood LL in a referral center in March 1985 and April 2017 were retrospectively reviewed. Seventy-five advanced-stage LL patients were included, 47 (62.7%) of whom had stage IV at initial diagnosis. The 5-year DFS and OS rates were 44.6% and 44.7%, respectively. There were 3 significant prognostic factors associated with worse outcomes: presence of B symptoms, low albumin level < 3.5 g/dL and serum LDH level > 500 IU/L. From these three factors, we assigned a score of 1 for each and total scores of 0, 1, 2, and 3 could predict 5-year OS rates of 92.3%, 50.9%, 24.7% and 0%, respectively (p < 0.05). The survival of children in this study was lower than in other studies of advanced-stage childhood LL. We identified 3 adverse prognostic factors and developed a prognostic model for clinical use in advanced-stage childhood LL.

Childhood-onset systemic lupus erythematosus and immune thrombocytopenia: Prevalence and risk factors.

BACKGROUND: There are few studies examining the prevalence and clinical risk factors for subsequent systemic lupus erythematosus (SLE) development after long-term follow-up in childhood immune thrombocytopenia (ITP). The aims of this study were to evaluate the prevalence and risk factors for subsequent SLE development in childhood ITP. METHODS: The medical records of childhood ITP patients aged under 15 years in a major tertiary care center in Southern Thailand were retrospectively reviewed. The Kaplan-Meier method was used to estimate the cumulative probability of subsequent SLE development after ITP. Logistic regression analysis was used to identify independent risk factors for SLE development. RESULTS: A total of 473 childhood ITP cases were included in the study. During a mean follow-up time of 6.1 ± 6.7 years, the prevalence of subsequent SLE development was 2.96%. Older age at ITP diagnosis (odds ratio [OR]: 1.24, 95% CI: 1.07-1.45) and chronic ITP (OR: 24.67, 95% CI: 3.14-100.0) were independent risk factors. The cumulative probabilities of subsequently developing SLE at 5 and 10 years after diagnosis of ITP were 3.8% (95% CI: 1.4-6.2) and 6.5% (95% CI: 2.9-9.8), respectively. CONCLUSION: Older age at ITP diagnosis and chronic ITP were risk factors for subsequent SLE developed in childhood ITP.

Successful Treatment of Gaucher Disease With Matched Sibling Hematopoietic Stem Cell Transplantation: A Case Report and Literature Review.

BACKGROUND: Gaucher disease (GD) is the most common lysosomal storage disease and requires long-term enzyme replacement therapy (ERT), which is costly and inconvenient for resource-limited countries such as Thailand. The authors present the case of a 1-year-old boy who was diagnosed with GD type 1 with a homozygous mutation at c.1448 T>C (L444P). He was treated with ERT and matched sibling hematopoietic stem cell transplantation (HSCT) was performed 6 months after the ERT was initiated. At a 3-year follow-up after the HSCT, he had full engraftment and the Lyso-GL1 levels were also at an acceptable level, which indicated disease remission. In conclusion, the authors suggest HSCT for long-term remission of GD in children.

Outcome and Prognostic Factors of Childhood Hodgkin Disease: Experience From a Single Tertiary Center in Thailand.

In childhood, Hodgkin disease (HD) has an excellent outcome in developed countries. There are few studies on outcomes of HD from resource-limited countries. This study aimed to assess clinical outcomes and factors associated with survival rates of childhood HD in a tertiary care center in Thailand. We retrospectively reviewed the medical records of pediatric HD patients between March 1985 and August 2017. Seventy-two children diagnosed with HD were identified. Pretreatment clinical and laboratory factors were assessed by Cox regression analysis to predict event-free survival (EFS) and overall survival (OS). The overall 5-year EFS and OS rate was 70.7% and 75.5%, respectively. Multivariate analysis identified 3 factors predicting inferior EFS: high-risk group (stages III-B, IV-B), splenomegaly, and platelet count >400,000/µL. The prognostic markers were assigned a score of 1 for each factor. For a total score of 0, the 5-year EFS and OS rates were 95% and 86%; scores 2 to 3, 33% and 54%, respectively. In conclusion, our study identified 3 factors predicting inferior EFS. These adverse prognostic factors can be used in clinical practice for predicting outcomes in pediatric HD.

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

BACKGROUND: Few epidemiological studies of pediatric central nervous system (CNS) tumors have been performed using data from Southeast Asian national registries. Therefore, we aimed to examine data on CNS tumors from the first national childhood CNS tumor registry in Thailand. METHODS: Newly diagnosed children with benign and malignant primary CNS tumors from 20 nationwide hospitals were included. Two eras in the Thai registry were studied to compare national protocol effectiveness, including 2003-2005 (before establishment of a pediatric CNS tumor protocol) and 2011-2012 (post-establishment). RESULTS: The first study period had 300 patients with an incidence of 7.5/1,000,000 person-years and the second had 168 patients with an incidence of 13.24/1,000,000 person-years. The three most common tumors were gliomas, medulloblastoma/primitive neuroectodermal tumor (PNET), and germ cell tumors. The most common tumor site was the cerebellum, followed by the brainstem and pineal region. Five- and 10-year overall survival (OS) rates were 46.62% (95% confidence interval [CI] 40.85-52.18) and 41.78% (95% CI 36.11-47.34), respectively, for the first period. The second period had a 5-year OS of 64.75% (95% CI 56.70-71.68). OS rates for gliomas, germ cell tumors, medulloblastoma/PNET, and ependymomas were better in the second period than in the first period. CONCLUSIONS: The incidence of primary childhood CNS tumors in our study is lower compared with other reports. Improvement of OS in the second study period might be because of establishment of the Thai Pediatric Oncology Group, and national protocols for childhood CNS tumors.

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.

Childhood cancer incidence and survival in Thailand: A comprehensive population-based registry analysis, 1990-2011.

BACKGROUND: Southeast Asia is undergoing a transition from infectious to chronic diseases, including a dramatic increase in adult cancers. Childhood cancer research in Thailand has focused predominantly on leukemias and lymphomas or only examined children for a short period of time. This comprehensive multisite study examined childhood cancer incidence and survival rates in Thailand across all International Classification of Childhood Cancer (ICCC) groups over a 20-year period. METHODS: Cancer cases diagnosed in children ages 0-19 years (n = 3574) from 1990 to 2011 were extracted from five provincial population-based Thai registries, covering approximately 10% of the population. Descriptive statistics of the quality of the registries were evaluated. Age-standardized incidence rates (ASRs) were calculated using the Segi world standard population, and relative survival was computed using the Kaplan-Meier method. Changes in incidence and survival were analyzed using Joinpoint Regression and reported as annual percent changes (APC). RESULTS: The ASR of all childhood cancers during the study period was 98.5 per million person-years with 91.0 per million person-years in 1990-2000 and 106.2 per million person-years in 2001-2011. Incidence of all childhood cancers increased significantly (APC = 1.2%, P < 0.01). The top three cancer groups were leukemias, brain tumors, and lymphomas. The 5-year survival for all childhood cancers significantly improved from 39.4% in 1990-2000 to 47.2% in 2001-2011 (P < 0.01). CONCLUSIONS: Both childhood cancer incidence and survival rates have increased, suggesting improvement in the health care system as more cases are identified and treated. Analyzing childhood cancer trends in low- and middle-income countries can improve understanding of cancer etiology and pediatric health care disparities.

Survival Outcome of Alternative Medicine Treatment for Newly Diagnosed Acute Leukemia in Children.

BACKGROUND: There is scarce information on the efficacy of alternative medicine (AM) alone as a treatment for newly diagnosed acute leukemia in children. We aimed to compare overall survival (OS) between children with newly diagnosed acute leukemia who received AM alone as the first-line treatment and those treated with conventional chemotherapy (CCT). METHODS: Two-to-one nearest-neighbor propensity score-matching using sex, initial white blood cell count, phenotype of leukemia, and period of diagnosis was performed on 184 patients who received CCT and 92 who received AM alone after being diagnosed with leukemia. A multivariable Cox proportional-hazards regression model was then applied to assess the effect of treatment on OS after adjusting for potential confounders. Hazard ratios (HR) and 95% confidence intervals (CI) are provided. RESULTS: After adjusting for initial white cell count and subtype of leukemia, children treated with AM alone had worse OS (HR 5.14, 95% CI 3.75-7.04) than those given CCT. The 5-year OS rate for newly diagnosed acute leukemia treated with AM medicine alone was 0%. CONCLUSION: AM without CCT is associated with poorer survival when compared with CCT.

Clinical Course and Outcome of Childhood Acquired Platelet Dysfunction with Eosinophilia.

Acquired platelet dysfunction with eosinophilia (APDE) is a syndrome which manifests as a transient state of platelet dysfunction in the presence of eosinophilia. The aim of this work was to study the clinical course and outcomes of children diagnosed with APDE. The hospital records of children with APDE were retrospectively reviewed and a total of 69 children were included. The mean (standard deviation) age at diagnosis was 6.9 (3.1) years. All of the patients presented with ecchymoses on the extremities, body, and face. None had serious bleeding symptoms. Platelet counts were within the normal range but all of the patients had abnormal platelet morphology by light microscopy. Parasitic infestation was found in 38% and most (87%) were treated with antiparasitic drugs. The median time from the onset of symptoms to remission was 2.6 months (95% CI 1.8-3.1). The overall complete remission rates at 3, 6, and 12 months were 61, 90, and 94%, respectively, with a median follow-up time after remission of 14.0 months (interquartile range 6.0-30.8). Neither univariate nor multivariate analysis indicated any statistically significant determinants for remission time. In our study, APDE was transient with spontaneous remission, no serious bleeding manifestations, and had a benign clinical course.

The relationship between myocardial and hepatic T2 and T2* at 1.5T and 3T MRI in normal and iron-overloaded patients.

Background Cardiac and liver iron assessment using magnetic resonance imaging (MRI) is non-invasive and used as a preclinical "endpoint" in asymptomatic patients and for serial iron measurements in iron-overloaded patients. Purpose To compare iron measurements between hepatic and myocardial T2* and T2 at 1.5T and 3T MRI in normal and iron-overloaded patients. Material and Methods The T2 and T2* values from the regions of interest (ROIs) at mid-left ventricle and mid-hepatic slices were evaluated by 1.5T and 3T MRI scans for healthy and iron-overloaded patients. Results For iron-overloaded patients, the myocardial T2 (1.5T) and myocardial T2 (3T) values were 60.3 ms (range = 56.2-64.8 ms) and 55 ms (range = 51.6-60.1 ms) (ρ = 0.3679) while the myocardial T2* (3T) 20.5 ms (range = 18.4-25.9 ms) was shorter than the myocardial T2* (1.5T) 35.9 ms (range = 31.4-39.5 ms) (ρ = 0.6454). The hepatic T2 at 1.5T and 3T were 19.1 ms (range = 14.8-27.9 ms) and 15.5 ms (14.6-20.4 ms) (ρ = 0.9444) and the hepatic T2* at 1.5T and 3T were 2.7 ms (range = 1.8-5.6 ms) and 1.8 ms (range = 1.1-2.9 ms) (ρ = 0.9826). The line of best fit exhibiting the linearity of the hepatic T2* (1.5T) and hepatic T2* (3T) had a slope of 2 and an intercept of -0.387 ms (R = 0.984). Conclusion Our study found myocardial T2 (1.5T) nearly equal to T2 (3T) with myocardial T2* (3T) 1.75 shorter than myocardial T2* (1.5T). The relationship of hepatic T2* (1.5T) and hepatic T2* (3T) was linear with T2* (1.5T) approximately double to T2* (3T) in iron-overloaded patients. This linear relationship between hepatic T2* (1.5T) and hepatic T2 (3T) could be an alternative method for estimating liver iron concentration (LIC) from 3T.

Clinical outcome of childhood chronic immune thrombocytopenia: A 38-year experience from a single tertiary center in Thailand.

BACKGROUND: There is limited information on long-term follow-up and prognostic factors for remission among children diagnosed with chronic immune thrombocytopenia (ITP). The aim of this study was to determine clinical outcomes and factors influencing remission in childhood chronic ITP. STUDY DESIGN: The hospital records of children aged 0-15 years diagnosed with chronic ITP were retrospectively reviewed. Kaplan-Meier curves were fit to estimate the median time to complete remission with 95% confidence intervals (CIs). Multivariate Cox proportional hazards regression models were used to identify independent factors for remission. RESULTS: A total of 113 patients were included in the analysis. The number of children achieving complete remission was 49 (46%) and the median time to remission was 7.1 years (95% CI: 4.8-11.0). The remission rates at 3, 5, 10, and 20 years were 25, 43, 60, and 75%, respectively. Factors influencing remission were platelets >60 × 109 /L at the onset of chronic ITP (hazard ratio [HR]: 7.24, 95% CI: 3.0-17.5) and treatment with intravenous immunoglobulin (HR: 0.37, 95% CI: 0.16-0.84). Age, gender, and clinical factors at the time of newly diagnosed ITP including bleeding manifestations, onset of symptoms, and history of preceding infection and vaccination were not predictive of remission. CONCLUSION: The spontaneous complete remission rates of chronic ITP were 43 and 60% at 5 and 10 years, respectively, and reached 75% at 20 years. A higher platelet level at diagnosis of chronic ITP and form of treatment were statistically significant indicators for achieving complete remission.

Predictive factors for resolution of childhood immune thrombocytopenia: Experience from a single tertiary center in Thailand.

BACKGROUND: Initial clinical factors that can reliably predict a successful within-1-year resolution of childhood immune thrombocytopenia (ITP) are still unclear. This study aimed to determine factors associated with within-12-month resolution of newly diagnosed childhood ITP. METHODS: The hospital records of 417 consecutive children aged less than 15 years with ITP were reviewed retrospectively and data related to the initial presentation were noted. Logistic regression analysis was used to determine which presenting features were associated with a favorable outcome within 12 months. RESULTS: Significant clinical and laboratory predictors for resolution of newly diagnosed childhood ITP within 12 months were abrupt onset less than 14 days, age less than 5 years, and platelet count at 4 weeks postdiagnosis of at least 100 × 109 l-1 . With these three significant predictors, the rate of within-1-year recovery was more than 97.2%, with a positive predictive value of 97.8% for newly diagnosed childhood ITP. CONCLUSION: Age less than 5 years, onset of bleeding less than 14 days, and follow-up platelet count at 4 weeks of at least 100 × 109 l-1 are significant predictive factors for disease resolution among children with newly diagnosed ITP.

Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5-year observational study (ENTRUST).

BACKGROUND: Children with red blood cell disorders may receive regular transfusions from an early age and consequently accumulate iron. Adequate iron chelation therapy can prevent organ damage and delayed growth/development. Deferasirox is indicated for treatment of pediatric patients with chronic iron overload due to transfusional hemosiderosis; however, fewer than 10% of patients in the registration studies were aged 2 to less than 6 years. PROCEDURE: Deferasirox, a once-daily oral iron chelator, was evaluated in young pediatric patients with transfusional hemosiderosis during the observational 5-year ENTRUST study. Patients aged 2 to less than 6 years at enrollment received deferasirox according to local prescribing information, with the primary objective of evaluating safety, specifically renal and hepatic function. Serum ferritin was observed as a surrogate efficacy parameter. RESULTS: In total, 267 patients (mean age 3.2 years) predominantly with ß-thalassemia (n = 176, 65.9%) were enrolled. Mean ± standard deviation deferasirox dose was 25.8 ± 6.5 mg/kg per day over a median of 59.9 months. A total of 145 patients (54.3%) completed 5 years' treatment. The proportion of patients with two or more consecutive postbaseline measurements (≥7 days apart) of serum creatinine higher than age-adjusted upper limit of normal (ULN) and alanine aminotransferase more than five times the ULN was 4.4% (95% confidence interval [CI]: 2.1-7.9) and 4.0% (95% CI: 1.8-7.4), respectively. Median serum ferritin decreased from 1,702 ng/ml at baseline to 1,127 ng/ml at 5 years. There were no new safety signals. CONCLUSIONS: Safety and efficacy of deferasirox in young pediatric patients in this long-term, observational study in everyday clinical practice were consistent with the known deferasirox profile.