Validity and reliability test of the Indonesian version of the PedHALshort questionnaire.

INTRODUCTION: The PedHAL questionnaire is employed in measuring the activities of haemophilia children. The PedHALshort was developed in 2022 to determine the most relevant item for the assessment. Haemo-QoL questionnaire assesses the quality of life of haemophilia children. AIM: Determine the validity and reliability of the PedHALshort Indonesia language version compared to the Haemo-QoL in the Indonesian population. METHODS: A cross-sectional study was conducted in Jakarta, Indonesia. The subjects were children (4-16 years old) with moderate or severe haemophilia A or B with repeated joint bleeding. The daily activity was assessed using the PedHALshorts and Haemo-QoL. The validity and reliability were tested. The validity was determined with Pearson correlation test, construct validity was assessed using convergent and discriminant validity. The Spearman correlation was used to determine which domains of Haemo-QoL correlated with PedHALshort. The reliability was analyzed using test-retest reliability. Cronbach's α was used to determined internal consistency. RESULTS: Fifty children were included in the study (78% haemophilia A, 70% severe group). All domains were valid (r > .284). The convergent validity test showed PedHALshort had a moderate negative correlation with physical health and sports school domain of Haemo-QoL (r = -.479, p < .001). The discriminant validity test showed the PedHALshort demonstrated a moderate negative correlation with physical health (r = -.585), view (r = -.590), sport school domain (r = -.430) in severe haemophilia subjects. The internal consistency of the PedHALshort questionnaire was high (Cronbach's α of .85 (.74-.92)). CONCLUSION: The PedHALshort is valid and reliable, hence, it can be used to measure the functional physical activity of children with haemophilia.

Comparison of Yearly Cost Related to Complications Between Deferasirox and Deferiprone Monotherapy in Thalassemia.

BACKGROUND: Hemoglobin disorders such as thalassemia major have created an economic burden on the health care system. Iron chelation therapy (ICT) is the most expensive cost component in patients with thalassemia. ICT was administered to reduce the toxic effects of iron overload. This study aims to compare the costs of iron chelators as monotherapy in patients with thalassemia major in Indonesia, specifically in Cipto Faculty of Medicine, Universit. METHODS: This is a retrospective analytical observational study. Data were collected from the thalassemia registry from 2016 to 2019. Patients' age, gender, type of thalassemia, and type of iron chelation were recorded. Complications and total annual costs were evaluated. All thalassemia patients aged ≥2 years who were only receiving monotherapy ICT and had no history of therapy switching were eligible. We excluded subjects who moved out to other facilities or lost to follow-up. RESULTS: From a total of 256 subjects, 249 subjects were included. The median age is 28 years old. Both sexes were represented equally. As many as 96.8% of subjects have thalassemia beta. Deferiprone was the most common iron chelator used (86.7%). Complications were observed in the subjects based on 4-year data collection; most of them were cardiomyopathy, diabetes mellitus, delayed puberty, and malnutrition ( P =0.422; P =0.867; P =0.004; and P =0.125, respectively). Deferiprone had a lower mean annual cost of USD 3581 than deferasirox, which had a cost of USD 6004. CONCLUSIONS: Cardiomyopathy, diabetes mellitus, delayed puberty, and malnutrition were the most common complications found in the study. This study showed that deferiprone should be taken as consideration as a drug of choice to treat iron overload in thalassemia provided by Indonesian national health insurance which is less costly despite the probability of complications found after the treatment was given. Further investigations are required to evaluate contributing factors of complications in thalassemia.

Novel retinal findings in ß-thalassemia major: older age and higher ferritin level as the risk factors.

PURPOSE: To investigate retinal changes in ß-thalassemia major patients and identify their association with systemic risk factors. METHODS: In this prospective study, 120 ß-thalassemia major patients received complete ophthalmic examinations (best-corrected visual acuity, contrast sensitivity, color vision, and indirect ophthalmoscopy) and retinal imaging using color fundus photography and fundus autofluorescence imaging. Patients were grouped according to the presence of thalassemia-related retinal changes. The association between systemic risk factors (age, type and duration of iron chelator use, history of splenectomy, hemoglobin level, and ferritin level) and thalassemia-related retinal changes was investigated using logistic regression analysis. RESULTS: Thalassemia-related retinal changes were identified in 36.7% of patients. Several distinct retinal changes were observed, including retinal refractile bodies in 10% of patients and retinal hemorrhage in 5.8% of patients. Fundus autofluorescence imaging showed abnormal patterns in 36.3% of patients with thalassemia-related retinal changes and 18.4% of patients without thalassemia-related retinal changes. Age (odds ratio [OR] = 1.10, 95% confidence interval [CI] 1.03-1.18) and ferritin level (OR 1.16, 95% CI 1.01-1.33) were associated with thalassemia-related retinal changes. CONCLUSION: Novel retinal changes were observed in ß-thalassemia major patients. This study identified older age and higher ferritin level as risk factors for thalassemia-related retinal changes.

Comparison of Deferiprone to Deferasirox and Deferoxamine to Cardiac and Hepatic T2* MRI in Thalassemia Patients: Evidence-based Case Report.

BACKGROUND: there are currently three iron chelator readily available for patients Indonesia; deferiprone/DFP (branded as Ferriprox), deferasirox/DFX (branded as Exjade) and deferoxamine/DFO (branded as Desferal). This study aims to determine which iron chelator is the most efficient in reducing cardiac and hepatic iron overload (measured by means of T2* MRI). METHODS: journal search with determined MeSH term was done in PubMed and Scopus. Studies that looked upon thalassemia major patient in all ages with usage of monotherapy iron chelation and its effect on myocardial T2* MRI and/or liver T2* MRI was included. Appraisal of studies was done using Oxford's CEBM appraisal tools and Joanna Brigs Institute critical appraisal tools. RESULTS: total of 11 studies with grand total of 611 samples were included. Mean T2* MRI value or (when available) mean changes in T2* MRI value after usage of specific iron chelator was gained from all the studies included. Comparison study and individual studies shows better control and increase of myocardial T2* MRI in those with DFP, and of liver T2* in those with good adherence to DFO chelation. CONCLUSION: DFP is superior in controlling or reducing myocardial iron load (as proven by mT2* MRI) and DFO had better capabilities in controlling or reducing hepatic iron load (as proven by liver T2* MRI). Studies with longer observation and larger samples is needed to see a significant changes of T2* MRI in DFX.

Seroconversion among children with HBsAg-positive mothers in Indonesia and factors affecting the anti-HBs titers.

Background and aim: Around 2% of newborns are at risk of hepatitis B virus (HBV) infection from their mothers. To prevent this, infants born to HBsAg-positive mothers are given hepatitis B immune globulin (HBIG) and hepatitis B (HB) vaccine as immunoprophylaxis. This study aims to investigate the efficacy of immunoprophylaxis in infants born to HBsAg-positive mothers and the contributing factors. Methods: The study was conducted on a group of 87 children, ranging from nine months to under 36 months, born to HBsAg-positive mothers and received immunoprophylaxis within 24 h after birth followed by a national immunization schedule at the Community Health Center (CHC) in three administrative cities of DKI Jakarta. We measured the levels of HBsAg and anti-HBs, and utilized ordinal logistic regression models to identify factors that influence the anti-HBs titers after vaccination. Results: Out of 87 children, only one child had positive HBsAg results. The data showed that 88.5% of the children had seroprotection with anti-HBs levels ≥10 mIU/mL. Additionally, 48.3% of the children had a high protective response with anti-HBs levels ≥100 mIU/mL, while 11.5% had a non-protective response. Children under one year of age, with a family history of HBV carriers, and who received five doses of the HB vaccine exhibited higher levels of anti-HBs titer category with adjusted OR 3.9 (95%CI: 1.3-11.6), 5.3 (95%CI: 1.1-27.4), and 8.3 (95%CI: 2-34.8), respectively. Conclusion: The administration of HBIG and HB vaccine successfully prevented vertical transmission, resulting in a high seroprotection rate.

PATTERN DYSTROPHY-LIKE CHANGES ASSOCIATED WITH A VERY HIGH SERUM FERRITIN LEVEL IN ß-THALASSEMIA MAJOR.

PURPOSE: To report a rare case of pattern dystrophy-like retinal changes in a young ß-thalassemia major patient with a very high serum ferritin level. METHODS: Observational case report of a ß-thalassemia major patient with bilateral pattern dystrophy-like retinal changes. RESULTS: An 18-year-old man experienced blurring of vision in both eyes. Patient had a very high serum ferritin level and had a history of using various iron-chelating drugs. Funduscopy revealed bilateral widespread retinal pigment epithelium changes with macular involvement, which resembled pattern dystrophy-like changes. Fundus autofluorescence imaging showed extensive area of decreased autofluorescence signal interspersed with increased autofluorescence signals, whereas optical coherence tomography demonstrated macular retinal pigment epithelium thinning and interdigitation zone disruption corresponding to retinal pigment epithelium damage and atrophy. CONCLUSION: Pattern dystrophy-like changes should be considered as a possible manifestation of retinal changes in patients with ß-thalassemia major. Iron overload, which is indicated by a very high serum ferritin level, might have a role in the formation of this rare retinal manifestation in ß-thalassemia major.

Whole-Genome Sequencing of SARS-CoV-2 Infection in a Cluster of Immunocompromised Children in Indonesia.

Background: Thus far, Indonesia has recorded over 4,000,000 confirmed COVID-19 cases and 144,000 fatalities; 12.8% of cases have been in children under 18 years. Whole-genome viral sequencing (WGS) of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has been demonstrated to help differentiate hospital-acquired infection from community-acquired coronavirus disease 2019 (COVID-19) infection. Our study highlighted the use of WGS to investigate the origin of infection among pediatric oncology patients in Jakarta. The aim of our study was to evaluate clinical and laboratory characteristics and also the efficacy of using WGS to confirm hospital-acquired COVID-19 infection in a cluster of immunocompromised children within a single ward of a tertiary hospital in metropolitan Jakarta based on quasispecies, viral load, and admission dates. Method: Real-time reverse-transcription polymerase chain reaction (RT-PCR) from nasopharyngeal (NP) swabs was used to diagnose the patients and also guardians and healthcare workers (HCWs) in the ward, followed by WGS of RT-PCR positive cases to establish their phylogenetic relationships. Result: Using WGS, we showed that SARS-CoV-2 transmission in a cluster of children with underlying malignancy was characterized by high similarity of whole virus genome, which suggests nosocomial transmission.

The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload.

Limited data are available on the use of deferiprone in children younger than 10 years of age. This study evaluated the safety and efficacy of a new liquid formulation of deferiprone for the treatment of transfusional iron overload in children 1-10 years old. One hundred children (91 thalassemia major, 8 Hb E-ß thalassemia, and 1 sickle cell disease) were enrolled for a 6-month treatment with deferiprone (50 to 100 mg/kg/d). The safety profile was similar to or better than that reported in earlier studies with deferiprone tablets in older children and adults. No unexpected adverse reactions were observed. Gastrointestinal intolerance (GI) was observed in 11% and an increased serum ALT in 12% of the children. Both events were transient. Mild neutropenia, observed in 6% of patients, did not progress to agranulocytosis and resolved despite continuous deferiprone treatment. Two patients experienced agranulocytosis that resolved without complications upon discontinuation of therapy. Deferiprone use was associated with a significant decline in mean serum ferritin level from 2532±1463 µg/L at baseline to 2176±1144 µg/L (P<0.0005). The results of this study show a favorable benefit/risk ratio of deferiprone oral solution for the treatment of young children with transfusional iron overload.

Pneumococcal vaccination for splenectomized patients with thalassemia major in Indonesia.

INTRODUCTION: Streptococcus pneumoniae is a capsulated bacterium that can cause severe infection in patients with thalassemia major, particularly those who have undergone splenectomy. The absence of the spleen as well as zinc deficiency in splenectomized patients with thalassemia major increases the possibility of developing invasive pneumococcal infection. The aims of this study are to evaluate pneumococcal IgG levels following PCV and PPV immunizations and the effect of zinc supplementation on qualitative specific immune responses in splenectomized patients with thalassemia. METHODS: Splenectomized patients with thalassemia major were administered a PCV pneumococcal vaccine (Prevenar 13®) at the start of the trial, after which they were randomly assigned to 2 groups (zinc and placebo group). After 8weeks, the patients received a PPV pneumococcal vaccine (Pneumovax®). Zinc syrup was provided to the zinc group at a dose of 1.5mg/kg/day (maximum of 50mg/day). Pneumococcal IgG examinations were conducted at the start of the trial and after 12weeks. RESULTS: In the group without PPV, the median initial pneumococcal IgG value was 315 (ranging from 65 to 1419) mU/mL for the zinc group and 338.5 (ranging from 82 to 1648) mU/mL for the placebo group. The median final pneumococcal IgG value was 1812.5 (ranging from 834 to 2444) mU/mL for the zinc group and 2857.5 (ranging from 834 to 2624) for the placebo group. The increase in the pneumococcal IgG value between the two groups was comparable (p=0.642). In the group with previous PPV, the median initial pneumococcal IgG value was 1333 (ranging from 793 to 2031) mU/mL for the zinc group and 880 (ranging from 74 to 1686) mU/mL for the placebo group. The median final pneumococcal IgG value was 1487 (ranging from 635 to 1757) mU/mL for the zinc group and 1012 (ranging from 292 to 1732) mU/mL for the placebo group. The increase in the pneumococcal IgG value between the two groups was comparable (p=0.528). CONCLUSION: There is no difference in the increase in pneumococcal IgG level in splenectomized patients with thalassemia major prior to and after receiving PPV. There were no differences observed in the development of pneumococcal IgG following zinc supplementation.