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Background: Hemoglobin H disease ( HbH ) , a hemoglobinopathy resulting from abnormal alpha globin genes, is classified into two categories: deletional HbH ( DHbH ) and non-deletional HbH ( NDHbH ) . The alpha-mutation genotypes exhibit a range of clinical anemias, which differentially impact patient growth., Objectives: This retrospective study assessed the growth of HbH patients at Siriraj Hospital, Mahidol University., Methods: Patients diagnosed with HbH between January 2005 and April 2021 were analyzed using growth standard scores of the Thai Society for Pediatric Endocrinology (2022 version) and BMI-for-age Z scores of the World Health Organization. Growth failure was defined as a patient's height for age exceeding two standard deviations below the mean., Results: Of the 145 HbH patients, 75 ( 51.7 %) had NDHbH, with -- SEA / α CS α being the most common genotype ( 70 patients; 93.3 %) . The mean baseline hemoglobin level was significantly lower in NDHbH patients than in DHbH patients (8.16 ± 0.93 g / dL vs. 9.51 ± 0.68 g / dL; P < 0.001 ) . Splenomegaly and growth failure prevalences were higher in NDHbH patients (37.3 % vs. 0%, with P < 0.001, and 22.7% vs. 8.6%, with P = 0.020, respectively). Multivariable analysis revealed splenomegaly > 3 cm was associated with growth failure (OR = 4.28; 95% CI, 1.19-15.39; P = 0.026)., Conclusions: NDHbH patients exhibited lower hemoglobin levels and more pronounced splenomegaly than DHbH patients. Growth failure can occur in both HbH types but appears more prevalent in NDHbH. Close monitoring of growth velocity is essential, and early treatment interventions may be required to prevent growth failure., Competing Interests: Competing interests: The authors declare no conflict of Interest.

Background: 6-Mercaptopurine (6-MP), a thiopurine agent, is a essential medication for treating pediatric acute lymphoblastic leukemia (ALL). However, its side effects of neutropenia and hepatotoxicity might interrupt treatment, resulting in poor outcomes. Inosine triphosphate pyrophosphatase ( ITPA ), an enzyme in the thiopurine pathway, may prevent the accumulation of toxic thiopurine metabolites. Studies on ITPA and thiopurine-associated toxicities are scarce., Methods: This study retrospectively investigated 1- to 15-year-old children with ALL who received 6-MP during the maintenance phase of treatment between 2000 and 2020. Toxicity during the first year of maintenance therapy and the mean dose of 6-MP were analyzed., Results: The 209 patients had a median age of 4.8 (0.3-14.8) years. Of these, 124 patients (59.3%) had wild-type ITPA , 73 patients (34.9%) had heterozygous ITPA 94C>A (het ITPA ), and 12 patients (5.7%) had homozygous ITPA 94C>A (hom ITPA ), with an allele frequency of 0.23. The incidence of neutropenia among ITPA polymorphisms did not significantly differ ( P = 0.813). In patients harboring hom ITPA , transaminitis was more frequent than other polymorphisms but without a significant difference ( P = 0.063). The mean dose of 6-MP for patients with hom ITPA was significantly lower than that for patients with het ITPA or wild-type ITPA ( P = 0.016)., Conclusions: Hom ITPA had a higher incidence of transaminitis and required a significantly larger dose reduction of 6-MP than wild-type ITPA . Further study is warranted to elucidate the effects of ITPA polymorphisms on toxicity in patients with ALL treated with 6-MP., Competing Interests: Competing interests: The authors declare no conflict of Interest.

Background: Several disseminated intravascular coagulation (DIC) scoring systems are used for prognosticating the clinical outcomes of patients with DIC. However, research on children is scarce. Therefore, this study compared the clinical outcomes of overt and non-overt DIC using the International Society on Thrombosis and Hemostasis (ISTH) DIC scoring system., Methods: This retrospective study reviewed data on children aged one month to 15 years diagnosed with DIC between 2003 and 2014., Results: Of 244 patients, 179 (73.4%) had overt DIC, and 65 (26.6%) had non-overt DIC. The most common causes were infection (84.8%), tissue injury (7%), and malignancies (2.9%). The 28-day case fatality rate was significantly higher for overt than non-overt DIC (76% vs. 15.6%; P < 0.001). DIC scores were significantly associated with mortality (R2 = 0.89). Each clinical parameter (platelet count, prothrombin time, and fibrin degradation products) was associated with mortality ( P = 0.01). On multivariable analysis, the factors associated with death were platelet counts ≤ 50 000 cells/mm3 (OR, 2.42; 95% CI, 1.08-5.42; P = 0.031); overt DIC score (OR, 7.62; 95% CI, 2.94-19.75; P < 0.001); renal dysfunction (OR, 2.92; 95% CI, 1.34-6.37; P = 0.007); shock (OR, 39.62; 95% CI, 4.99-314.84; P = 0.001); and acute respiratory distress syndrome (OR, 25.90; 95% CI, 3.12-214.80; P = 0.003)., Conclusions: The 28-day case-fatality rate was significantly higher for patients with overt than non-overt DIC and concordant with ISTH scores. ISTH DIC scores can be used as a clinical predictor for DIC in children., Competing Interests: Competing interests: The authors declare no conflict of Interest.

Background: Patients with transfusion-dependent thalassemia ( TDT ) risk iron overload and require iron chelation therapy. Second-line therapy is warranted for patients demonstrating poor chelation responses., Patients and Methods: We retrospectively studied the serum-ferritin (SF), and liver-iron-concentration (LIC) outcomes of patients with TDT treated with twice-daily dosing of deferasirox (TDD-DFX) > 24 months, after failing to respond to once-daily deferasirox (OD-DFX)., Results: We enrolled 22 OD-DFX nonresponders (14 males and eight females; median age, 9.2 [3-15.5] years). The median blood transfusion was 216 (206-277) ml/kg/year. The median TDD-DFX treatment period was 30 (24-35) months. Before initiating TDD-DFX, the median SF level was 2,486 (1,562-8,183) ng/ml, while the median LIC was 6.6 (3.2-19) mg/g dry wt. There were 18 TDD-DFX responders (81.8%) and 4 TDD-DFX nonresponders. The median SF-level change was -724 (-4,916 to 1,490) ng/mL. The median LIC change was -2.14 (-13.7 to 6.8) mg/g dry wt. The 1-year and 2-year SF levels and LICs were statistically significant (SF, P = 0.006/0.005; and LIC, 0.006/0.005, respectively). There were no treatment interruptions secondary to adverse events. In the follow-up of the TDD-DFX responder group, 11 of the 18 had a reduced dose, whereas the remaining seven continued with the same dose., Conclusions: TDD-DFX appears to be an alternative treatment approach for patients refractory to OD-DFX, with a favorable long-term safety profile. Further studies with larger groups and pharmacogenetic analyses of OD-DFX responders are warranted to determine the efficacy and safety profile of TDD-DFX., Competing Interests: Competing interests: The authors declare no conflict of Interest.