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1.
Br J Haematol ; 205(1): 236-242, 2024 Jul.
Article in English | MEDLINE | ID: mdl-38811201

ABSTRACT

Pyruvate kinase (PK) is a key enzyme of anaerobic glycolysis. The genetic heterogeneity of PK deficiency (PKD) is high, and over 400 unique variants have been identified. Twenty-nine patients who had been diagnosed as PKD genetically in seven distinct paediatric haematology departments were evaluated. Fifteen of 23 patients (65.2%) had low PK levels. The PK:hexokinase ratio had 100% sensitivity for PKD diagnosis, superior to PK enzyme assay. Two novel intronic variants (c.695-1G>A and c.694+43C>T) have been described. PKD should be suspected in patients with chronic non-spherocytic haemolytic anaemia, even if enzyme levels are falsely normal. Total PKLR gene sequencing is necessary for the characterization of patients with PKD and for genetic counselling.


Subject(s)
Anemia, Hemolytic, Congenital Nonspherocytic , Introns , Pyruvate Kinase , Pyruvate Metabolism, Inborn Errors , Humans , Pyruvate Kinase/deficiency , Pyruvate Kinase/genetics , Male , Female , Pyruvate Metabolism, Inborn Errors/genetics , Child , Child, Preschool , Anemia, Hemolytic, Congenital Nonspherocytic/genetics , Turkey , Infant , Adolescent , Mutation
2.
Pediatr Transplant ; 28(3): e14751, 2024 May.
Article in English | MEDLINE | ID: mdl-38659194

ABSTRACT

BACKGROUND: The published experience concerning autologous peripheral blood stem cell collection in children is very limited. METHODS: The data of pediatric patients who underwent autologous stem cell mobilization and apheresis between January 2011 and April 2020 were analyzed retrospectively. RESULTS: We studied retrospectively 64 mobilization and apheresis procedures in 48 pediatric patients (34 males, 14 females), mean age of 7.31 ± 5.38 (range, 1.5-19.7) years, the underlying disease was mostly neuroblastoma (NBL). The body weight of 21 patients (43.75%) was 15 kg or less. The targeted autologous peripheral stem cell apheresis (APSCA) was successfully achieved in 98% of patients. Neuroblastoma patients were younger than the rest of the patients and underwent apheresis after receiving fewer chemotherapy cycles than others and all of them mobilized within the first session successfully. Plerixafor was added to mobilization in nine heavily pretreated patients (18.7%), median two doses (range, 1-4 doses). 11 patients (22.9%) underwent radiotherapy (RT) before mobilization with doses of median 24 Gy (range, 10.8-54.0 Gy). Patients with RT were older at the time of apheresis and had received more chemotherapy courses than patients without RT. As a result, patients with a history of RT had significantly lower peripheral CD34+ cells and CD34+ yields than those without RT. In 17 patients (35.4%), 22 different complications were noted. The most common complications were catheter-related infections (n:10, 20.8%), followed by catheter-related thrombosis in eight patients (16.7%). CONCLUSIONS: Patients who had far less therapy before apheresis were more likely to mobilize successfully. Our study provides a detailed practice approach including complications during APSCA aiming to increase the success rates of apheresis in transplantation centers.


Subject(s)
Blood Component Removal , Hematopoietic Stem Cell Mobilization , Neoplasms , Peripheral Blood Stem Cell Transplantation , Transplantation, Autologous , Humans , Female , Male , Hematopoietic Stem Cell Mobilization/methods , Child , Retrospective Studies , Child, Preschool , Adolescent , Infant , Blood Component Removal/methods , Peripheral Blood Stem Cell Transplantation/methods , Neoplasms/therapy , Young Adult , Peripheral Blood Stem Cells
3.
Pediatr Transplant ; 28(1): e14696, 2024 Feb.
Article in English | MEDLINE | ID: mdl-38317343

ABSTRACT

BACKGROUND: We aimed to evaluate our pediatric HSCT recipients routinely monitored for adenoviremia and to determine the adequacy of this monitoring in predicting adenoviral disease (AD). METHODS: A retrospective cohort of patients who underwent allogeneic HSCT between January 2021 and August 2022, and routinely monitored for adenoviremia by real-time PCR was included in our survey. Demographic and clinical data of the patients were recorded. Incidence rates, risk factors, and mortality rates related to adenoviremia, and AD were analyzed. RESULTS: Among 104 HSCTs performed in 94 patients adenovirus (AdV) was revealed in 27 (26%) episodes and adenoviremia in 18 (17.3%) HSCT episodes. AD without adenoviremia developed in nine episodes (8.6%). Disseminated disease was significantly more frequently detected in episodes with adenoviremia (p = .008). GVHD was independent risk factor for AdV detection (OR: 8.6, 95% CI: 2.03-33.7, p = .001). Viremia developed within a shorter time interval after HSCT in isolated episodes of adenoviremia compared to those with concomitant AD (p = .006). Initial and peak viral loads were significantly higher in adenoviremia with AD (p < .001). Mortality was higher in the AdV-detected episodes (p < .001) than in the AdV-undetected episodes. AdV-related mortality was found to be 22.2%. Adenoviremia increased the risk of mortality (OR: 1.2, 95% CI: 0.22-1.33, p = .01). CONCLUSIONS: Adenoviremia monitoring is an important process in the detection of AD. Since some patients may develop AD without accompanying by adenoviremia, monitoring for AdV in blood samples should be supported with other monitoring methods in order to evaluate the probable involvement of different organs or systems.


Subject(s)
Adenoviridae Infections , Hematopoietic Stem Cell Transplantation , Child , Humans , Retrospective Studies , Hematopoietic Stem Cell Transplantation/adverse effects , Hematopoietic Stem Cell Transplantation/methods , Adenoviridae Infections/complications , Adenoviridae Infections/diagnosis , Adenoviridae , Viremia/diagnosis , Viremia/etiology
4.
Pediatr Transplant ; 28(3): e14758, 2024 May.
Article in English | MEDLINE | ID: mdl-38659218

ABSTRACT

BACKGROUND: Data on the risk factors and outcomes for pediatric patients with SARS-CoV-2 infection (COVID-19) following hematopoietic stem cell transplantation (HSCT) are limited. OBJECTIVES: The study aimed to analyze the clinical signs, risk factors, and outcomes for ICU admission and mortality in a large pediatric cohort who underwent allogeneic HSCT prior to COVID-19 infection. METHOD: In this nationwide study, we retrospectively reviewed the data of 184 pediatric HSCT recipients who had COVID-19 between March 2020 and August 2022. RESULTS: The median time from HSCT to COVID-19 infection was 209.0 days (IQR, 111.7-340.8; range, 0-3845 days). The most common clinical manifestation was fever (58.7%). While most patients (78.8%) had asymptomatic/mild disease, the disease severity was moderate in 9.2% and severe and critical in 4.4% and 7.6%, respectively. The overall mortality was 10.9% (n: 20). Deaths were attributable to COVID-19 in nine (4.9%) patients. Multivariate analysis revealed that lower respiratory tract disease (LRTD) (OR, 23.20, p: .001) and lymphopenia at diagnosis (OR, 5.21, p: .006) were risk factors for ICU admission and that HSCT from a mismatched donor (OR, 54.04, p: .028), multisystem inflammatory syndrome in children (MIS-C) (OR, 31.07, p: .003), and LRTD (OR, 10.11, p: .035) were associated with a higher risk for COVID-19-related mortality. CONCLUSION: While COVID-19 is mostly asymptomatic or mild in pediatric transplant recipients, it can cause ICU admission in those with LRTD or lymphopenia at diagnosis and may be more fatal in those who are transplanted from a mismatched donor and those who develop MIS-C or LRTD.


Subject(s)
COVID-19 , Hematopoietic Stem Cell Transplantation , Humans , COVID-19/epidemiology , COVID-19/therapy , COVID-19/mortality , Hematopoietic Stem Cell Transplantation/adverse effects , Child , Male , Female , Retrospective Studies , Adolescent , Turkey/epidemiology , Child, Preschool , Risk Factors , SARS-CoV-2 , Infant , Transplantation, Homologous , Severity of Illness Index
5.
Pediatr Nephrol ; 39(7): 2199-2207, 2024 Jul.
Article in English | MEDLINE | ID: mdl-38324191

ABSTRACT

BACKGROUND: Acute kidney injury (AKI) is a common complication of hematopoietic stem cell transplantation (HSCT) with increased mortality and morbidity. Understanding the risk factors for AKI is essential. This study aimed to identify AKI incidence, risk factors, and prognosis in pediatric patients post-HSCT. METHODS: We conducted a retrospective case-control study of 278 patients who were divided into two groups: those with AKI and those without AKI (non-AKI). The groups were compared based on the characteristics and clinical symptoms of patients, as well as post-HSCT complications and the use of nephrotoxic drugs. Logistic regression analysis was employed to identify the risk factors for AKI. RESULTS: A total of 16.9% of patients had AKI, with 8.5% requiring kidney replacement therapy. Older age (OR 1.129, 95% CI 1.061-1.200, p < 0.001), sinusoidal obstruction syndrome (OR 2.562, 95% CI 1.216-5.398, p = 0.011), hemorrhagic cystitis (OR 2.703, 95% CI 1.178-6.199, p = 0.016), and nephrotoxic drugs, including calcineurin inhibitors, amikacin, and vancomycin (OR 17.250, 95% CI 2.329-127.742, p < 0.001), were identified as significant independent risk factors for AKI following HSCT. Mortality rate and mortality due to AKI were higher in stage 3 patients than those in stage 1 and 2 AKI (p = 0.019, p = 0.007, respectively). Chronic kidney disease developed in 1 patient (0.4%), who was in stage 1 AKI (2.1%). CONCLUSIONS: AKI poses a serious threat to children post-HSCT, leading to alarming rates of mortality and morbidity. To enhance outcomes and mitigate these risks, it is vital to identify AKI risk factors, adopt early preventive strategies, and closely monitor this patient group.


Subject(s)
Acute Kidney Injury , Hematopoietic Stem Cell Transplantation , Humans , Hematopoietic Stem Cell Transplantation/adverse effects , Acute Kidney Injury/epidemiology , Acute Kidney Injury/etiology , Acute Kidney Injury/therapy , Acute Kidney Injury/mortality , Male , Female , Child , Risk Factors , Retrospective Studies , Case-Control Studies , Child, Preschool , Adolescent , Incidence , Prognosis , Renal Replacement Therapy/statistics & numerical data , Renal Replacement Therapy/adverse effects , Infant
6.
Eur J Pediatr ; 183(5): 2155-2162, 2024 May.
Article in English | MEDLINE | ID: mdl-38367066

ABSTRACT

The purpose of this study was to evaluate the association between interleukin-33 (IL-33) and its receptor Soluble Suppression of Tumorigenicity-2 (sST2) levels and bacterial infections during febrile neutropenia (FN) in pediatric patients with acute lymphoblastic leukemia (ALL). In this prospective, case-control study, participants were divided into 3 groups: ALL patients with FN (Group A), ALL patients without neutropenia and fever (Group B), and healthy children without infection and chronic disease (Group C). There were 30 cases in each group. Blood samples for IL-33 and sST2 have been drawn from patients in Group A before the initiation of treatment and on days 1 and 5 of treatment, and from patients in Groups B and C at initiation. At admission, mean IL-33 level (39.02 ± 26.40 ng/L) in Group B and mean sST2 level (185.3 ± 371.49 ng/ml) in Group A were significantly higher than the other groups (p = 0.038, p < 0.001, respectively). No difference was observed in the mean IL-33 and sST2 levels in the 5-day follow-up of patients in Group A (p = 0.82, p = 0.86, respectively). IL-33 and sST2 levels were not associated with fever duration, neutropenia duration or length of hospitalization. While C-reactive protein (CRP) was significantly higher in patients with positive blood culture (p = 0.021), IL-33 (p = 0.49) and sST2 (p = 0.21) levels were not associated with culture positivity.  Conclusion: IL-33 and sST2 levels were not found valuable as diagnostic and prognostic markers to predict bacterial sepsis in patients with FN. What is Known: • Neutropenic patients are at high risk of serious bacterial and viral infections, but the admission symptom is often only fever. • Febrile neutropenia has a high mortality rate if not treated effectively. What is New: • Febrile neutropenia is not only caused by bacterial infections. Therefore, new biomarkers should be identified to prevent overuse of antibiotics. • Specific biomarkers are needed to diagnose bacterial sepsis in the early phase of febrile neutropenia.


Subject(s)
Biomarkers , Febrile Neutropenia , Interleukin-1 Receptor-Like 1 Protein , Interleukin-33 , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Humans , Interleukin-33/blood , Female , Male , Interleukin-1 Receptor-Like 1 Protein/blood , Child , Prospective Studies , Case-Control Studies , Child, Preschool , Febrile Neutropenia/blood , Febrile Neutropenia/etiology , Febrile Neutropenia/diagnosis , Biomarkers/blood , Precursor Cell Lymphoblastic Leukemia-Lymphoma/blood , Precursor Cell Lymphoblastic Leukemia-Lymphoma/complications , Adolescent , Infant , Bacterial Infections/blood , Bacterial Infections/diagnosis
7.
Indian Pediatr ; 61(7): 621-626, 2024 Jul 15.
Article in English | MEDLINE | ID: mdl-38655891

ABSTRACT

OBJECTIVE: This study aimed to assess the efficacy of different oral iron preparations prescribed for prevention of iron deficiency anemia in healthy infants. METHODS: This retrospective study enrolled infants aged between 6 and 12 months who were initiated on iron prophylaxis at four months of age. Enrolled children consistently used specific iron preparations (ferrous, ferric or liposomal iron) and had their complete blood counts and serum ferritin levels assessed within the 6-12 month timeframe. Blood values and iron prophylaxis type (ferrous (Fe+2), ferric (Fe+3), liposomal iron) were recorded. Chi-square test was used to compare the hemoglobin and ferritin levels levels between groups. Univariate and multivariate regression analyses assessed the risk of anemia. RESULTS: The study included 371 children (ferrous sulphate - 60, iron hydroxide-polymaltose complex - 137 and liposomal ferric pyrophosphate - 174) with a mean (SD) age 9.1 (1.3) mo. Iron deficiency in different groups were: liposomal iron (46.0%), ferric iron (44.5%), and ferrous iron (5.0%). Mean (SD) serum ferritin levels (µg/L) were higher in the ferrous group [30.1 (10.8)] compared to infants receiving ferric [17.6 (14.50)] and liposomal iron [15.4 (12.1)] (P < 0.001). Mean (SD) hemoglobin levels (g/dL) were significantly higher in the ferrous group [12.4 (0.8)] compared to ferric [11.9 (1.1)] and liposomal iron group [12.0 (1.1)]; P =0.008. Multiple regression analysis showed that ferrous group was associated with a lower risk of iron deficiency [OR (95% CI) 0.04 (0.01-0.15), P < 0.001]. CONCLUSION: Ferrous iron demonstrated superior efficacy compared to ferric and liposomal iron. Further studies are needed to establish alternative iron preprations in children.


Subject(s)
Anemia, Iron-Deficiency , Ferric Compounds , Ferrous Compounds , Liposomes , Humans , Infant , Anemia, Iron-Deficiency/prevention & control , Anemia, Iron-Deficiency/blood , Retrospective Studies , Male , Female , Ferrous Compounds/administration & dosage , Ferrous Compounds/therapeutic use , Ferric Compounds/administration & dosage , Ferric Compounds/therapeutic use , Ferritins/blood , Iron/administration & dosage , Iron/blood , Iron/therapeutic use , Hemoglobins/analysis , Hemoglobins/drug effects
8.
Indian J Hematol Blood Transfus ; 40(1): 108-115, 2024 Jan.
Article in English | MEDLINE | ID: mdl-38312178

ABSTRACT

Prophylaxis is the gold standard for the management of hemophilia A patients. It has been shown that prophylaxis regulated with pharmacokinetic (PK) data reduces frequency of bleeding and cost of treatment. To determine the best prophylaxis regimen, PK dosing tools using the Bayesian method have been developed. We aimed to compare two PK dosing tools. Blood samples were drawn before, 4, 24, and 48 h after FVIII infusions from patients with severe hemophilia A and inhibitor negative. FVIII levels were measured by PTT-based one-stage assay method. PK parameters obtained using WAPPS and myPKFiT, which are web-accessible PK dosing tools using Bayesian algorithm, and daily prophylaxis dose estimated by the programs were compared. Forty-two hemophilia A patients [median age 13 years (IQR 8.9-16.4)] included in the study. There was no difference between the daily dose of FVIII given for prophylaxis and the dose recommended by the myPKFiT for the 1% trough level; whereas, a significant difference was found with the WAPPS. The half-lives of FVIII did not differ between the two dosing tools; however, significant differences were found in the estimated dose, clearances, and times to 1% trough level. There was no significant difference between PK data of patients who received Advate® and those who received non-Advate® factor concentrates. Choice of PK dosing tool can affect recommended FVIII dose. However, target trough levels should be individualized according to bleeding phenotype and daily activity of patient. Supplementary Information: The online version contains supplementary material available at 10.1007/s12288-023-01671-0.

9.
Pediatr Infect Dis J ; 43(7): e231-e234, 2024 Jul 01.
Article in English | MEDLINE | ID: mdl-38621150

ABSTRACT

BACKGROUND: Herbaspirillum species are nonfermenting, aerobic, helical or curved, Gram-negative bacteria belonging to the class Betaproteobacteria, order Burkholderiales. To date, only a few studies have reported on the epidemiology, clinical symptoms, antibiotic susceptibility profiles, treatment and outcomes of Herbaspirillum huttiense -related infections in pediatric patients. METHODS: The aim of this study was to present 3 years of H.huntiense data, antibiotic susceptibility profiles, systemic antibiotics and antibiotic lock therapy (ALT) options and clinical outcomes. RESULTS: Fourteen episodes of infection in 12 patients were included in this retrospective study. The patients had a male/female ratio of 1:1 and a median age of 160.5 months (range, 3-198 months). Catheter-related bloodstream infection (CRBSI) was detected in 11 patients. Only 1 patient developed catheter-related infective endocarditis. The patient's catheter was removed, and she was successfully treated with systemic antibiotics for 4 weeks. Systemic antibiotics were used in all infections related to H. huttiense . In septic, critically ill patients, the catheter was removed, and systemic antibiotics were started. Port catheters were removed in 5 patients. ALT was performed in clinically stable patients. ALT using amikacin was administered to 6 patients through the port catheter. Two patients had a 2nd attack. After the 2nd ALT treatment, 1 patient cured, and the catheter of the other patient was removed due to persistent microbial growth in cultures. Antimicrobial susceptibility testing of the reported isolates showed susceptibility to meropenem (90%), ceftazidime (87%) and piperacillin/tazobactam (65%), with 92% resistance to colistin. CONCLUSION: H. huttiense is an emerging pathogen in CRBSI. Piperacillin/tazobactam, ceftazidime and meropenem appear to be good therapeutic options for the treatment of H. huttiense infections. ALT and systemic antibiotics can be used in H. huttiense -CRBSI to sterilize and preserve the central venous catheter.


Subject(s)
Anti-Bacterial Agents , Catheter-Related Infections , Gram-Negative Bacterial Infections , Herbaspirillum , Microbial Sensitivity Tests , Humans , Female , Male , Child , Retrospective Studies , Anti-Bacterial Agents/therapeutic use , Anti-Bacterial Agents/pharmacology , Herbaspirillum/drug effects , Herbaspirillum/genetics , Catheter-Related Infections/microbiology , Catheter-Related Infections/drug therapy , Child, Preschool , Infant , Gram-Negative Bacterial Infections/drug therapy , Gram-Negative Bacterial Infections/microbiology , Gram-Negative Bacterial Infections/epidemiology , Adolescent
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