ABSTRACT
This meta-analysis evaluated the impact of prophylactic post-chemotherapy granulocyte colony-stimulating factor (G-CSF) in patients with acute myeloid leukemia (AML). Overall, the relapse rate, overall survival, event-free survival, and mortality rate were similar in G-CSF (+) compared to G-CSF (-) patients. However, the relative risk (RR) of relapse was higher in children and in secondary AML patients who were treated with G-CSF compared to the G-CSF (-) group [RR, 95% confidence interval: 1.26, 1.04-1.52, and 1.12 (1.02-1.24)]. Treatment with post-chemotherapy G-CSF should be prescribed with caution in pediatric patients with AML and secondary AML as possibly increasing the relapse risk.
Subject(s)
Granulocyte Colony-Stimulating Factor , Leukemia, Myeloid, Acute , Adolescent , Child , Humans , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Antineoplastic Combined Chemotherapy Protocols/adverse effects , Granulocyte Colony-Stimulating Factor/therapeutic use , Granulocyte Colony-Stimulating Factor/administration & dosage , Leukemia, Myeloid, Acute/drug therapy , Leukemia, Myeloid, Acute/mortality , Neoplasm Recurrence, Local/prevention & control , RecurrenceABSTRACT
OBJECTIVE: To determine the prevalence of iron overload in children with acute lymphoblastic leukemia (ALL) after treatment cessation and establish a cutoff value for serum ferritin level as an indicator of iron overload. BACKGROUND: Early detection and monitoring of iron overload in patients with leukemia is crucial. METHODS: In this prospective cohort study, 66 pediatric patients with ALL who were treated at a tertiary referral center affiliated with Shiraz University of Medical Sciences in Shiraz, Southern Iran, were investigated from July 2020 to December 2022. Serum ferritin levels were measured 6 months after treatment completion. T2* magnetic resonance imaging of the liver and heart was done for all patients. The receiver operating characteristic curve was used to illustrate the area under the receiver operating characteristic curve to assess the diagnostic value of serum ferritin level and total transfusion volume. RESULTS: A total of 24 patients (36.4%) had iron overload in the heart or liver based on T2 magnetic resonance imaging findings. Serum ferritin level was a highly accurate diagnostic marker for iron overload in pediatric patients with ALL, with a sensitivity of 95.8%, and specificity of 85.7% for a cutoff value of 238.5 ng/mL. Also, blood transfusion was a good predictor of iron overload a sensitivity of 75% and specificity of 81% for a cutoff value of 28.3 mL/kg. CONCLUSION: We identified specific cutoff values for serum ferritin and blood transfusion volume to predict iron overload with high sensitivity and specificity. These markers offer a cost-effective and accessible approach for periodic screening of iron deposition, particularly in resource-constrained settings.
Subject(s)
Iron Overload , Leukemia , Humans , Child , Ferritins , Prospective Studies , Iron Overload/diagnosis , Iron Overload/etiology , Heart , Magnetic Resonance Imaging/methods , Liver/pathology , Leukemia/pathologyABSTRACT
BACKGROUND: The SARS-CoV-2 infection has been associated with potentially endothelial damage and coagulation cascade activation that cause thrombosis. There is limited information on thrombosis and anticoagulant therapy in children with coronavirus disease 2019 (COVID-19). AIMS: This study evaluates the outcome of thromboprophylaxis in children younger than 18-year old with COVID-19 infection. METHODS: A retrospective study was conducted on 184 hospitalized pediatric patients with confirmed COVID-19 infection. A designed questionnaire was made to collect all demographic, clinical, and laboratory data. According to World Health Organization, the patients were classified as asymptomatic/mild, moderate, severe, and critically ill. RESULTS: The mean age of the patients was 7.04±5.9 (1 wk to younger than 18 y). Overall, 33 patients received anticoagulant therapy. All patients who passed away (n=19) belonged to the critical group. One patient (1.28%) was complicated with deep vein thrombosis despite taking thromboprophylaxis, and 1 (1.28%) with pulmonary thromboembolism while the patient did not take an anticoagulant. CONCLUSIONS: Our data showed a lower rate of thrombosis (1.4%) than adult patients with COVID-19. It may underline the role of anticoagulants in moderate to severe/critically ill children with COVID-19 infection. Expert opinion and personal experience are necessary, while we have a significant knowledge gap in understanding COVID-19-associated coagulopathy and thrombotic risk in children.
Subject(s)
COVID-19 , Thrombosis , Venous Thromboembolism , Humans , Child , Adolescent , COVID-19/complications , Anticoagulants/therapeutic use , Retrospective Studies , Critical Illness , SARS-CoV-2 , Venous Thromboembolism/etiology , Thrombosis/etiology , Thrombosis/prevention & control , Thrombosis/drug therapyABSTRACT
BACKGROUND: Understanding the determinants of long-term overall survival (OS) of thalassemia patients (TPs) is the mainstay of care. METHODS: As a retrospective survey, we assessed the data of 769 TPs who had regular follow-up and blood transfusion for at least 30 years from 1990 - 2019. We utilized semi-parametric proportional hazards mixture cure-rate regression to discover the factors with a significant effect on short- and long-term OS separately. RESULTS: The 25- and 30-year OS for the TPs were calculated to be 98.7% and 90.4%, respectively. Each five-year age escalation was associated with a 30% decrease in the probability of being short-term survivors (HR = 1.06, p = 0.047). Parental family relationship influenced both cured (OR = 3.00, p = 0.017) and uncured (HR = 0.50, p = 0.046) TPs. Moreover, the type of iron chelation drug, liver iron concentration, and normal EF results had a significant effect on long-term OS. Aging, parental consanguinity, liver and cardiac siderosis, higher ferritin levels, and low hemoglobin level were associated with poorer prognosis in TPs. CONCLUSIONS: However, deferoxamine followed by multiple drugs as iron chelation, severe liver siderosis, and abnormal EF declined the probability of long-term OS among TPs. This can be considered by health policy decision-makers to enforce the screening program more strictly.
Subject(s)
Iron Overload , Siderosis , Thalassemia , beta-Thalassemia , Humans , Iron , Iron Chelating Agents , Prognosis , Retrospective Studies , Siderosis/complications , Thalassemia/therapy , beta-Thalassemia/complications , beta-Thalassemia/therapyABSTRACT
Oxidative stress is a major mechanism contributing to the progression of ß-thalassemia. To assess the effect of vitamin E and N-acetyl cysteine (NAC) as antioxidant agents on total oxidative stress (TOS) status and total antioxidant capacity (TAC) in patients with transfusion-dependent ß-thalassemia (TDT). In this open-label randomized controlled trial, from May to August 2019, 78 eligible patients with TDT over the age of 18 were enrolled. All patients were registered at the Thalassemia Clinic of Shiraz University of Medical Sciences in Southern Iran. Patients were randomly allocated to the NAC group (10 mg/kg/day, orally), vitamin E group (10 U/kg/day, orally), and control group. The duration of the study was 3 months. The mean age of the participants was 28.5 ± 5.1 (range: 18-41) years. At the end of the study, TOS significantly decreased only in the vitamin E group (mean difference (MD), 95% confidence interval (CI): 0.27 (0.03-0.50), P = 0.026). TAC significantly decreased in both supplemented groups at the 3rd month of treatment (NAC group: MD (95% CI): 0.11 (0.04-0.18), P = 0.002 and vitamin E group: 0.09 (0.01-0.16), P = 0.022 respectively). Hemoglobin did not significantly change at the end of the study in each group (P > 0.05). Mild transient adverse events occurred in 4 patients of the NAC group and 5 patients of the vitamin E group with no need to discontinue the treatment. Vitamin E can be a safe and effective supplement in improving oxidative stress in patients with TDT. Moreover, it seems that a longer duration of using antioxidant supplements needs to make clinical hematologic improvement in TDT patients.
Subject(s)
Acetylcysteine/administration & dosage , Oxidative Stress/drug effects , Vitamin E/administration & dosage , beta-Thalassemia/drug therapy , Acetylcysteine/pharmacology , Adolescent , Adult , Antioxidants/administration & dosage , Antioxidants/analysis , Antioxidants/metabolism , Blood Transfusion , Dietary Supplements , Female , Humans , Iran , Male , Oxidants/blood , Oxidation-Reduction/drug effects , Time Factors , Vitamin E/pharmacology , Young Adult , beta-Thalassemia/blood , beta-Thalassemia/therapyABSTRACT
BACKGROUND: Hemophagocytic lymphohistiocytosis (HLH) is a syndrome of excessive inflammation. We aimed to describe the clinical and laboratory findings of HLH patients secondary to Visceral leishmaniasis (VL) and their treatment outcome during a 4-year follow-up period compared to primary HLH. METHOD: Forty children with primary HLH confirmed by genetic study and 20 children with HLH secondary to VL confirmed by a blood or bone marrow polymerase chain reaction from 2014 to 2018 in Shiraz, Fars province, Southern Iran, were enrolled. RESULTS: The median age at diagnosis was 11.5 months (range 1-170), and 56.7% were male. Fever and splenomegaly were the most frequent clinical presentations. 93.3% of the subjects had an HScore > 169, which had a good correlation with HLH-2004 criteria (r = 0.371, P = 0.004). Patients with primary HLH experienced more thrombocytopenia (P = 0.012) and higher alanine transaminase (P = 0.016), while patients with VL-associated HLH had higher ferritin (P = 0.034) and erythrocyte sedimentation rate (P = 0.011). Central nervous system (CNS) involvement occurred in 38.3% of patients. The mortality rate was higher in patients with CNS disease (61% vs. 35%, P = 0.051). The 3-yr overall survival rate was 35.9%. (24% in primary HLH and 100% in VL-associated HLH, P < 0.001). In Cox regression analysis, platelet count < 100,000/ µ l (hazard ratio 4.472, 95% confidence interval 1.324-15.107, P = 0.016) correlated with increased mortality in patients with primary HLH. CONCLUSION: VL is a potential source of secondary HLH in regions with high endemicity. Treatment of the underlying disease in VL-associated HLH is sufficient in most cases, with no need to start etoposide-based chemotherapy.
Subject(s)
Leishmaniasis, Visceral/complications , Lymphohistiocytosis, Hemophagocytic/diagnosis , Lymphohistiocytosis, Hemophagocytic/parasitology , Adolescent , Alanine Transaminase/blood , Blood Sedimentation , Central Nervous System Diseases/complications , Child , Child, Preschool , Female , Ferritins/blood , Fever , Follow-Up Studies , Humans , Infant , Infant, Newborn , Iran , Lymphohistiocytosis, Hemophagocytic/mortality , Lymphohistiocytosis, Hemophagocytic/therapy , Male , Polymerase Chain Reaction , Splenomegaly/diagnosis , Survival Rate , Thrombocytopenia/complications , Treatment OutcomeABSTRACT
BACKGROUND: Statins are a potential treatment for venous thromboembolism (VTE) prophylaxis complementary to conventional anticoagulants without associated bleeding complications. This study aimed to compare pro-thrombotic activities of different classes of lipid-lowering drugs in an active comparator design and determine whether there is a relation between statin versus fibrate/niacin use and pro-coagulant factor outcomes. METHODS: This is a cross-sectional analysis of participants from the Netherlands Epidemiology of Obesity study using any class of lipid-lowering drugs, including any types of statins, niacin, and fibrates. We performed linear regression analyses to determine fibrinogen, factor (F) VIII, FIX, and FXI activity in statins versus fibrate/niacin users and adjusted for age, sex, tobacco smoking, body mass index (BMI), hypertension, diabetes, and prevalent cardiovascular disease. RESULTS: Among 1043 participants, the mean age was 58.4 ± 5.2 years, 61% were men, and the mean BMI was 31.3 ± 4.5 kg/m2. Clinical characteristics were balanced between statin and fibrate/niacin users. Statin users had lower mean FXI (18.3 IU/dL, 95% confidence interval (CI) 9.4 to 27.3) levels compared to fibrate/niacin users. The level of FVIII (15.8 IU/dL, 95% CI - 0.003 to 31.6), and FIX (11.3 IU/dL, 95% CI - 0.4 to 23.2) were lower in statin users than fibrate/niacin users with marginal statistical significance. CONCLUSION: Current statin use was associated with lower plasma levels of FXI than fibrate/niacin use. The effects on coagulation factors may, in part, explain the benefit of statin therapy rendered in primary and secondary prevention of VTE.
ABSTRACT
Severe coronavirus disease 2019 (COVID-19) accompanies hypercytokinemia, similar to secondary hemophagocytic lymphohistiocytosis (sHLH). We aimed to find if HScore could predict disease severity in COVID-19. HScore was calculated in hospitalized children and adult patients with a proven diagnosis of COVID-19. The need for intensive care unit (ICU), hospital length of stay (LOS), and in-hospital mortality were recorded. The median HScore was 43.0 (IQR 0.0-63.0), which was higher in those who needed ICU care (59.7, 95% CI 46.4-72.7) compared to those admitted to non-ICU medical wards (38.8, 95% CI 32.2-45.4; P = 0.003). It was also significantly higher in patients who died of COVID-19 (105.1, 95% CI 53.7-156.5) than individuals who survived (41.5, 95% CI 35.8-47.1; P = 0.005). Multivariable logistic regression analysis revealed that higher HScore was associated with a higher risk of ICU admission (adjusted OR = 4.93, 95% CI 1.5-16.17, P = 0.008). The risk of death increased by 20% for every ten units increase in HScore (adjusted OR 1.02, 95% CI 1.00-1.04, P = 0.009). Time to discharge was statistically longer in high HScore levels than low levels (HR = 0.41, 95% CI 0.24-0.69). HScore is much lower in patients with severe COVID-19 than sHLH. Higher HScore is associated with more ICU admission, more extended hospitalization, and a higher mortality rate. A modified HScore with a new cut-off seems more practical in predicting disease severity in patients with severe COVID-19.
Subject(s)
COVID-19/diagnosis , Severity of Illness Index , Adolescent , Adult , Aged , Aged, 80 and over , COVID-19/mortality , COVID-19/pathology , COVID-19/therapy , COVID-19 Testing , Child , Child, Preschool , Critical Care/statistics & numerical data , Cytokine Release Syndrome/diagnosis , Cytokine Release Syndrome/virology , Female , Hospital Mortality , Hospitalization , Humans , Infant , Iran/epidemiology , Length of Stay/statistics & numerical data , Logistic Models , Male , Middle Aged , Predictive Value of Tests , Prognosis , Prospective Studies , Survival Analysis , Young AdultABSTRACT
BACKGROUND: Hemophagocytic lymphohistiocytosis (HLH) is a life-threatening syndrome that predominantly affects infants from birth to 18 months of age, characterized by fever and multiorgan failure. Liver injury has been rarely reported as a presenting sign in the neonatal period. This study reports a case with HLH in the neonatal period who presented with acute liver failure. CASE PRESENTATION: Herein, a 3-day-old female newborn was admitted with cytopenia, increased liver enzymes, hypofibrinogenemia, and markedly elevated serum ferritin. Hemophagocytosis of bone marrow biopsy confirmed the diagnosis of HLH. The newborn was treated with HLH-2004 protocol, but she finally died from multiorgan failure. CONCLUSION: Growing awareness of HLH as a cause of liver failure in the neonatal period can be associated with early treatment and reduces mortality in this group of patients.
Subject(s)
Antineoplastic Combined Chemotherapy Protocols/adverse effects , Liver Failure/etiology , Lymphohistiocytosis, Hemophagocytic/pathology , Multiple Organ Failure/etiology , Fatal Outcome , Female , Humans , Infant, Newborn , Lymphohistiocytosis, Hemophagocytic/complications , Lymphohistiocytosis, Hemophagocytic/drug therapy , Multiple Organ Failure/pathologyABSTRACT
BACKGROUND: Ocular involvement may occur via several mechanisms in patients with transfusion-dependent ß-thalassemia (TDT) mainly chronic anemia, iron overload and iron chelator toxicity. We aimed to evaluate the frequency of abnormal ocular findings and their relationship with hematologic parameters in TDT patients. METHODS: In this cross-sectional study from January 2018 to January 2019, a total of 79 patients with TDT over the age of 18 who were on iron-chelation therapy (ICT) were consecutively investigated. All patients were registered at the Thalassemia Comprehensive Center affiliated with Shiraz University of Medical Sciences, Shiraz, Southern Iran. Complete ophthalmic examination was performed by an expert ophthalmologist. Clinical and hematologic parameters were collected from the patients´ medical records. RESULTS: The mean age ± standard deviation (SD) of the patients was 28.4 ± 5.6 years (range: 18-43). Twenty-four patients (30.4%) were male and 29 (36.7%) were splenectomized. The mean ± SD of the best-corrected visual acuity (VA) was 0.960 ± 0.086 decimal, (range: 0.6-1), 0.016 ± 0.046 logMar, (range: 0-0.2). The frequency of patients with VA > 0.1 logMar was 3 (3.8%). The mean intraocular pressure (IOP) was 14.88 ± 3.34 (6-25) mmHg. Fundus abnormalities were observed in 8 patients (10.1%), consisting of increased cup-disk ratio (3.8%), vessel tortuosity (2.5%), retinal pigment epithelium degeneration (2.5%), myelinated nerve fiber layer (1.3%), and internal limiting membrane wrinkling (1.3%). No significant association was observed between fundus abnormalities, VA, or IOP with hematologic parameters (P > 0.05). TDT patients with diabetes mellitus had significantly higher IOP (P = 0.010) but similar frequency of fundus abnormalities with non-diabetic patients (P > 0.05). CONCLUSIONS: The frequency of ocular abnormalities in our patients was lower than the previous reports. The frequency of fundus abnormalities were similar in diabetic and non-diabetic thalassemia patients indicating close monitoring and proper management of the disease and comorbidities in these patients.
Subject(s)
Iron Overload , Thalassemia , beta-Thalassemia , Adult , Cross-Sectional Studies , Female , Humans , Iran/epidemiology , Male , Middle Aged , beta-Thalassemia/complications , beta-Thalassemia/epidemiologyABSTRACT
INTRODUCTION: Neuroblastoma commonly required multimodal therapy containing surgery, chemotherapy, radiotherapy, and immunotherapy. CASE REPORT: In our case, who had refractory metastatic neuroblastoma, we use histone deacetylase inhibitor (panobinostat) in combination with chemotherapy agents and iodine-131-meta-iodobenzylguanidine (MIBG) therapy. MANAGEMENT AND OUTCOME: This approach leads to successfully treat the patient. MIBG scan and bone marrow examination after therapy revealed no evidence of tumor. Now, she underwent autologous transplantation six months ago and free of tumor. CONCLUSION: Panobinostat can cause apoptosis induction in refractory metastatic neuroblastoma in combination with MIBG therapy and chemotherapy.
Subject(s)
3-Iodobenzylguanidine/administration & dosage , Antineoplastic Combined Chemotherapy Protocols/administration & dosage , Brain Neoplasms/drug therapy , Neuroblastoma/drug therapy , Panobinostat/administration & dosage , Brain Neoplasms/diagnostic imaging , Child , Combined Modality Therapy/methods , Female , Histone Deacetylase Inhibitors/administration & dosage , Humans , Iodine Radioisotopes/administration & dosage , Neoplasms, Second Primary/diagnostic imaging , Neoplasms, Second Primary/therapy , Neuroblastoma/diagnostic imaging , Radiopharmaceuticals/administration & dosage , Stem Cell Transplantation/methods , Transplantation, Autologous/methods , Treatment OutcomeABSTRACT
BACKGROUND: Fanconi anemia (FA) is a heterogeneous genetic disorder characterized by congenital anomalies, early-onset bone marrow failure, and a high predisposition to cancers. Up to know, different genes involved in the DNA repair pathway, mainly FANCA genes, have been identified to be affected in patients with FA. CASE PRESENTATION: Here, we report clinical, laboratory and genetic findings in a 3.5-year-old Iranian female patient, a product of a consanguineous marriage, who was suspicious of FA, observed with short stature, microcephaly, skin hyperpigmentation, anemia, thrombocytopenia and hypo cellular bone marrow. Therefore, Next Generation Sequencing was performed to identify the genetic cause of the disease in this patient. Results revealed a novel, private, homozygous frameshift mutation in the FANCF gene (NM_022725: c. 534delG, p. G178 fs) which was confirmed by Sanger sequencing in the proband. CONCLUSION: Such studies may help uncover the exact pathomechanisms of this disorder and establish the genotype-phenotype correlations by identification of more mutations in this gene. It is the first report of a mutation in the FANCF gene in Iranian patients with Fanconi anemia. This new mutation correlates with a hematological problem (pancytopenia), short stature, and microcephaly and skin hyperpigmentation. Until now, no evidence of malignancy was detected.
Subject(s)
Fanconi Anemia Complementation Group F Protein/genetics , Fanconi Anemia/genetics , Genetic Association Studies , Genetic Predisposition to Disease/genetics , Sequence Deletion , Base Sequence , Child, Preschool , Consanguinity , Fanconi Anemia/physiopathology , Fanconi Anemia Complementation Group F Protein/metabolism , Female , Genotype , High-Throughput Nucleotide Sequencing , Homozygote , Humans , Iran , Pancytopenia/genetics , Pedigree , Sequence Analysis, ProteinABSTRACT
Recently, resveratrol showed induction of γ-globin mRNA synthesis in human erythroid precursors and reducing oxidative stress in red cells of thalassemia patients in many in vitro studies. We aimed to investigate the efficacy and safety of resveratrol, for the first time, in non-transfusion-dependent beta-thalassemia intermedia (B-TI) in Southern Iran. In this double-blind randomized clinical trial, 54 patients with B-TI were investigated during 6 months between October 2016 and March 2017. Patients were randomly allocated into three groups by simple randomization method. Group 1 (hydroxyurea (HU) and placebo, 18 patients), group 2 (resveratrol/piperine and placebo, 16 patients), and group 3(HU and resveratrol/piperine, 20 patients). Primary end point was considered as change in hemoglobin (Hb) levels and need for blood transfusion. Drug safety was considered as a secondary end point. Mean age of the patients was 28.2 ± 5.6 (18-42) years. Response rate was not significantly different among the three groups (P > 0.05). Higher percentages of adverse events were detected in groups 2 (31.3%) and 3 (25%) compared to group 1 (5.6%). However, the difference was not statistically significant (P > 0.05). All reported adverse events were gastrointestinal symptoms. Resveratrol showed a similar efficacy with HU in the small population of non-transfusion B-TI patients during a 6-month follow-up. Complications, mostly gastrointestinal, were observed more frequently in resveratrol groups compared to the HU group. Although it was not statistically significant, more attention should be given to safety and efficacy of resveratrol as an oral HbF-augmenting agent.
Subject(s)
Fetal Hemoglobin/drug effects , Stilbenes/administration & dosage , Stilbenes/adverse effects , beta-Thalassemia/drug therapy , Administration, Oral , Adolescent , Adult , Alkaloids/administration & dosage , Alkaloids/adverse effects , Benzodioxoles/administration & dosage , Benzodioxoles/adverse effects , Double-Blind Method , Female , Fetal Hemoglobin/metabolism , Humans , Iran , Male , Piperidines/administration & dosage , Piperidines/adverse effects , Polyunsaturated Alkamides/administration & dosage , Polyunsaturated Alkamides/adverse effects , Resveratrol , Treatment Outcome , Up-Regulation/drug effects , Young Adult , beta-Thalassemia/bloodABSTRACT
The aim of this study was to promote emotional well-being, hope, life satisfaction, and meaning to mothers of children with cancer utilizing a positive psychology intervention. The study population included 50 mothers who had children with cancer attending an outpatient oncology clinic. The participants (experimental and control groups) completed Ryff's Psychological Well-being Scale (PWBS), Satisfaction with Life Scale (SWLS), Snyder's Hope Scale (HS), and Meaning in Life Questionnaire (MLQ) before and after the intervention and also one month after the end of intervention. Variables of the study significantly increased in the experimental group compared to the control group.
Subject(s)
Mental Health , Mothers/psychology , Neoplasms/psychology , Personal Satisfaction , Psychotherapy/methods , Adult , Child , Female , Hope , Humans , Mothers/statistics & numerical data , Neoplasms/therapy , Surveys and Questionnaires , Treatment Outcome , Young AdultABSTRACT
BACKGROUND: Human Leukocyte Antigen (HLA) has an important role in presenting self and non-self-antigens to T-cell receptors on T lymphocytes. For tissue transplantation and the possibility of graft rejection, these HLA antigens on the surface of the donor and recipient's cells should be checked. METHODS: In this study, a novel technique was used for HLA typing by cloning and sequencing. The most polymorphic exons of HLA-A, HLA-B, and HLA-DRB1 in five unrelated persons were cloned into the T-vector. Afterward, the sequencing data were analyzed using a new computer software named SAF (Second Allele Finder), which was designed for this study. RESULTS: Using this new technique, the HLA typing was performed in five unrelated persons, for four of them the HLA typing was done by the PCR-SSP method. Despite that the PCR-SSP method is typing in the first field, the results of this study were obtained in the third field, which is more valid and accurate. CONCLUSIONS: This study presents a new, accurate, and low cost technique for typing HLA loci in tissue transplantation especially in bone marrow transplantation.
Subject(s)
Alleles , Histocompatibility Testing/methods , Software , Female , Humans , Male , Polymerase Chain Reaction , Sequence Analysis, DNAABSTRACT
Because of insufficient erythropoiesis, peripheral hemolysis and increased gastrointestinal iron absorption, iron overload is still a matter of debate in ß-thalassemia intermedia (ß-TI) patients, which can be overcome using iron chelators. However, data on use of iron chelators in ß-TI patients is highly restricted. The aim of this study was to evaluate the efficacy of oral administration of deferasirox (Exjade(®) or DFX) by assessment of serum ferritin levels in ß-TI patients. In this quasi-experimental study, 50 ß-TI patients with serum ferritin levels >1000 ng/mL were selected and received oral DFX for 12 consecutive months. Iron overload was measured by checking serum ferritin levels every 2 months and the results were compared with the baseline level. The mean serum ferritin was decreased during 1 year of chelation therapy without any toxic effect. Although the difference between baseline ferritin and ferritin levels at the end of second month was not remarkable (p = 0.88), a significant reduction in serum ferritin was observed after 4 (p = 0.01), 6 (p = 0.001), 8 (p < 0.001), 10 (p < 0.001) and 12 months (p < 0.001) of chelation therapy compared to its baseline levels. There was no correlation between baseline ferritin levels and age (p = 0.574). In addition, no statistically significant difference was observed about change in serum ferritin levels after 6 and also 12 months of therapy between patients who had undergone splenectomy and those who did not (p = 0.796 and 0.859, respectively). Iron chelation therapy with DFX is safe and effective in reducing serum ferritin levels in ß-TI patients who suffer from side effects of iron overload.
Subject(s)
Benzoates/therapeutic use , Iron Chelating Agents/therapeutic use , Iron Overload/drug therapy , Iron Overload/etiology , Triazoles/therapeutic use , beta-Thalassemia/complications , Adolescent , Adult , Child , Deferasirox , Female , Ferritins/blood , Humans , Iron Overload/diagnosis , Iron Overload/metabolism , Male , Treatment Outcome , Young AdultABSTRACT
Purpose: Patients who receive frequent blood transfusions are at an elevated risk of developing hepatic fibrosis due to iron overload in the liver. In this study, we evaluated the effectiveness of transient elastography (TE) (FibroScan®) for assessing liver fibrosis in patients with pediatric cancer. Methods: We enrolled 106 consecutive cases of acute leukemia in individuals under 21 years of age. The participants were followed for 2 years. Based on their serum ferritin (SF) levels, the patients were divided into two groups: group 1 (SF≥300 ng/mL) and group 2 (SF<300 ng/mL). A liver FibroScan® was performed, and a p-value of less than 0.05 was considered statistically significant. Results: Among the various parameters in the liver function test (LFT), alkaline phosphatase was significantly higher in a subgroup of patients aged 5-8 years in group 2 compared to those in group 1. The indices of liver fibrosis determined by TE, including the FibroScan score, controlled attenuation parameter score, steatosis percentage, and meta-analysis of histological data in viral hepatitis score, as well as indirect serum markers of liver fibrosis such as the aminotransferase (AST)/alanine aminotransferase (ALT) ratio, Fibrosis 4 score, and AST to platelet ratio index, did not differ significantly between the two groups. The association between the TE results and LFT parameters was only significant for ALT. Conclusion: Transfusion-associated iron overload does not have a significant correlation with severe liver fibrosis. FibroScan® is not a sensitive tool for detecting early stages of fibrosis in survivors of pediatric leukemia.
ABSTRACT
Objective: Anthracycline administration in children is associated with cardiac dysfunction. Speckle-tracking echocardiography (STE) can detect subclinical cardiac damage that may go undetected by conventional two-dimensional (2D) echocardiography. This study aims to investigate medium-term anthracycline cardiotoxicity using STE and determine a safer administrable level of anthracyclines (ACs). Methods: This observational case-control study enrolled 37 healthy controls and 78 pediatric cancer survivors who received chemotherapy. The patients were divided into two groups: cardiotoxic received (CR) and cardiotoxic free (CF). Data on segmental longitudinal strain (LS), global LS (GLS), and 2D echocardiographic parameters were collected after a drug-free period of at least one year. Results: A total of 115 children with a mean age of 108 ± 55 months, of whom 66% were males, were included in the study. Both the groups of cancer survivors exhibited significantly reduced GLS compared to healthy controls (CR vs. controls, P = 0.001; CF vs. controls, P = 0.013), but no significant difference in left ventricular ejection fraction (LVEF) was observed (P = 0.75). Overall, cancer survivors treated with ACs demonstrated a significant reduction in strain in 10 left ventricular segments, particularly in the basal segments (P < 0.05). Among CR patients, those with impaired GLS (n = 43, GLS worse than -21.9) had significantly higher mean age and cumulative anthracycline dose compared to CR patients with normal GLS (age, P = 0.024; anthracycline dosage, P = 0.036). Using an anthracycline cutoff of 223 mg/m2 resulted in a higher detection rate (49% vs. 25%) and fewer missed cases (51% vs. 74%) compared to the 360 mg/m2 anthracycline cutoff. Conclusion: Childhood cancer survivors demonstrate significantly reduced GLS while preserving a normal LVEF, which does not differ significantly from reference values of healthy children. The reduction in strain appears to be associated with higher anthracycline doses and older age. Lowering the anthracycline threshold to 223 mg/m2 may improve the predictability of a decline in cardiac function using strain imaging at medium-term follow-up.
Subject(s)
Cerebrospinal Fluid Rhinorrhea/diagnosis , Chylous Ascites/diagnosis , Lymphatic Abnormalities/diagnosis , Pericardial Effusion/diagnosis , Cerebrospinal Fluid Rhinorrhea/complications , Chylous Ascites/complications , Diagnosis, Differential , Humans , Infant , Lymphatic Abnormalities/complications , Male , Pericardial Effusion/complications , PrognosisABSTRACT
Teratoma is a type of germ cell tumor layer that appears in the gonadal, sacrococcygeal, mediastinal, and retroperitoneal regions. Primary retroperitoneal teratoma is rare and asymptomatic but can present with symptoms due to a mass effect on neighboring organs. These tumors have to be considered in the differential diagnosis of a mass in the abdominal cavity of children to distinguish between Wilms' tumor, neuroblastoma, and other intra-abdominal lesions. We presented an infant boy with protrusion of the left upper quadrant of the abdomen and a palpable abdominal mass that had progressively enlarged. An abdominal computed tomography scan revealed a large retroperitoneal cystic, solid mass on the left side of the abdominal cavity, causing pressure on the left ureter. Also, hydronephrosis of the left kidney was seen with a decreased enhancement of the left kidney due to obstruction uropathy. The mass was suspicious on imaging for a retroperitoneal teratoma. The patient underwent laparotomy, and excision of the huge retroperitoneal mass was done. The final diagnosis was an immature teratoma grade 3, and the patient was discharged in good condition. Retroperitoneal teratomas are rare tumors in infants. These tumors would be an incident diagnosis or diagnosed by the mass effect of giant tumors on other organs. They must be considered in the differential diagnosis of intra-abdominal tumors in children. Hydronephrosis and obstructive uropathy can be rare consequences of the mass effects of these tumors.