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1.
Pediatr Transplant ; 28(3): e14758, 2024 May.
Artigo em Inglês | MEDLINE | ID: mdl-38659218

RESUMO

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.


Assuntos
COVID-19 , Transplante de Células-Tronco Hematopoéticas , Humanos , COVID-19/epidemiologia , COVID-19/terapia , COVID-19/mortalidade , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Criança , Masculino , Feminino , Estudos Retrospectivos , Adolescente , Turquia/epidemiologia , Pré-Escolar , Fatores de Risco , SARS-CoV-2 , Lactente , Transplante Homólogo , Índice de Gravidade de Doença
2.
Pediatr Nephrol ; 39(7): 2199-2207, 2024 Jul.
Artigo em Inglês | MEDLINE | ID: mdl-38324191

RESUMO

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.


Assuntos
Injúria Renal Aguda , Transplante de Células-Tronco Hematopoéticas , Humanos , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Injúria Renal Aguda/epidemiologia , Injúria Renal Aguda/etiologia , Injúria Renal Aguda/terapia , Injúria Renal Aguda/mortalidade , Masculino , Feminino , Criança , Fatores de Risco , Estudos Retrospectivos , Estudos de Casos e Controles , Pré-Escolar , Adolescente , Incidência , Prognóstico , Terapia de Substituição Renal/estatística & dados numéricos , Terapia de Substituição Renal/efeitos adversos , Lactente
3.
Pediatr Transplant ; 28(1): e14696, 2024 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-38317343

RESUMO

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.


Assuntos
Infecções por Adenoviridae , Transplante de Células-Tronco Hematopoéticas , Criança , Humanos , Estudos Retrospectivos , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Transplante de Células-Tronco Hematopoéticas/métodos , Infecções por Adenoviridae/complicações , Infecções por Adenoviridae/diagnóstico , Adenoviridae , Viremia/diagnóstico , Viremia/etiologia
4.
J Thromb Thrombolysis ; 57(3): 466-472, 2024 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-38085469

RESUMO

Revascularization treatments (IV thrombolysis, mechanical thrombectomy) related to ischemic stroke have developed in recent years. With devices such as NIRS, non-invasive monitoring of treatment efficacy is provided. In this study, we aimed to use near-infrared spectroscopy (NIRS) as an objective monitoring method to see the effect of intravenous (IV) thrombolysis or mechanical thrombectomy treatments applied for cerebral oxygenation in patients with acute ischemic stroke. This study was carried out as a prospective study involving patients admitted to the emergency department in the years 2021-2022. NIRS measured regional oxygen saturation (rSO2) of both hemispheres of the brain before IV thrombolysis treatment, during the treatment at 0. min, 15. min, 30. min, 45. min, 60. min, after the treatment, and before and after the mechanical thrombectomy procedure. The significance level of the change in rSO2 values measured by NIRS was examined. 80 patients were included in the study. IV thrombolysis was applied to 58 patients, mechanical thrombectomy was applied to 5 of them, and both treatments were applied to 17 of them. In patients receiving IV thrombolysis, a significant difference was found in the affected hemisphere between the NIRS values measured at 0.min-15.min, 0.min-30.min, 0.min-45.min, 0.min-60.min, 0.min-post-treatment, 15.min-60.min (p < 0.001). In the patients included in the study, there was a strong and significant negative correlation between the deltaNIHSS value and the deltaNIRS values in the affected hemisphere (r=- 0.307, p = 0.013). There was a significant increase in the NIRS measurement values during and after the IV thrombolysis treatment in the affected hemisphere in the group with clinical improvement (p < 0.001). It is thought that IV thrombolysis or mechanical thrombectomy treatment applied to patients admitted to the emergency department with acute ischemic stroke can be followed objectively by NIRS.


Assuntos
Isquemia Encefálica , AVC Isquêmico , Trombólise Mecânica , Acidente Vascular Cerebral , Humanos , Acidente Vascular Cerebral/tratamento farmacológico , Acidente Vascular Cerebral/induzido quimicamente , Isquemia Encefálica/diagnóstico , Isquemia Encefálica/tratamento farmacológico , AVC Isquêmico/etiologia , Espectroscopia de Luz Próxima ao Infravermelho , Estudos Prospectivos , Seguimentos , Trombectomia/métodos , Terapia Trombolítica/métodos , Resultado do Tratamento , Serviço Hospitalar de Emergência , Fibrinolíticos
5.
Allergy Asthma Proc ; 44(5): e22-e28, 2023 09 01.
Artigo em Inglês | MEDLINE | ID: mdl-37641226

RESUMO

Background: Anti-thymocyte globulin (ATG) has been successfully used for decades to prevent graft versus host disease before hematopoietic stem cell transplantation (HSCT) as a part of conditioning regimen. However, sometimes hypersensitivity reactions may limit its use. Objective: To evaluate hypersensitivity reactions experienced during rabbit-ATG infusion among children and present successful desensitization protocol. Methods: The medical records of pediatric patients who were given rabbit-ATG treatment at our tertiary center hospital HSCT unit between 2019 and 2022 were reviewed retrospectively. Diagnosis of the patients, age at the time of HSCT, gender, presence of hypersensitivity reaction to rabbit-ATG, and management were evaluated. Characteristics of the reaction and presence of hypersensitivity reaction to other drugs were also noted. If performed, desensitization protocols were evaluated retrospectively. Results: We evaluated 81 patients; 66.6% of them (n = 54) were boys. The mean age of the patients was 8.78 ± 5.48 years. Hypersensitivity to rabbit-ATG was seen in six patients (7.4%). Four of them (4.9%) had anaphylaxis; two (2.4%) had urticaria. Intradermal test performed to every patient before the first dose of ATG infusion was detected a positive result in 1 patient (1.2%) . None of these seven patients had allergic reactions to other drugs before. Successful ATG desensitization was performed in five patients by using a 12-16 step protocol due to patients' reaction severity. Conclusion: This study aimed to evaluate hypersensitivity reactions with rabbit-ATG in children. A successful desensitization protocol with rabbit-ATG is presented. Desensitization must be performed with an experienced team very carefully in the absence of alternative drug.


Assuntos
Anafilaxia , Urticária , Humanos , Soro Antilinfocitário/efeitos adversos , Estudos Retrospectivos , Anafilaxia/diagnóstico , Anafilaxia/etiologia , Anafilaxia/prevenção & controle , Testes Intradérmicos
6.
Biology (Basel) ; 12(7)2023 Jul 22.
Artigo em Inglês | MEDLINE | ID: mdl-37508466

RESUMO

The revolutionary CRISPR/Cas9 genome-editing technology has emerged as a powerful tool for plant improvement, offering unprecedented precision and efficiency in making targeted gene modifications. This powerful and practical approach to genome editing offers tremendous opportunities for crop improvement, surpassing the capabilities of conventional breeding techniques. This article provides an overview of recent advancements and challenges associated with the application of CRISPR/Cas9 in plant improvement. The potential of CRISPR/Cas9 in terms of developing crops with enhanced resistance to biotic and abiotic stresses is highlighted, with examples of genes edited to confer disease resistance, drought tolerance, salt tolerance, and cold tolerance. Here, we also discuss the importance of off-target effects and the efforts made to mitigate them, including the use of shorter single-guide RNAs and dual Cas9 nickases. Furthermore, alternative delivery methods, such as protein- and RNA-based approaches, are explored, and they could potentially avoid the integration of foreign DNA into the plant genome, thus alleviating concerns related to genetically modified organisms (GMOs). We emphasize the significance of CRISPR/Cas9 in accelerating crop breeding processes, reducing editing time and costs, and enabling the introduction of desired traits at the nucleotide level. As the field of genome editing continues to evolve, it is anticipated that CRISPR/Cas9 will remain a prominent tool for crop improvement, disease resistance, and adaptation to challenging environmental conditions.

7.
Turk Kardiyol Dern Ars ; 51(5): 328-332, 2023 07.
Artigo em Inglês | MEDLINE | ID: mdl-37450454

RESUMO

OBJECTIVE: Beta thalassemia major is an inherited hemoglobin disorder resulting in chronic hemolytic anemia. Cardiac involvement is the main cause of death in patients. Speckle-tracking echocardiography is a feasible method for the evaluation of cardiac function via an assessment of the longitudinal deformation of the myocardium through the cardiac cycle. The aim of our study is to evaluate the association between vitamin D deficiency and deformation of the left ventricular myocardium measured by speckle-tracking echocardiography in children with thalassemia major. METHODS: In this prospective study, 33 thalassemic patients with vitamin D deficiency were enrolled. Cardiac magnetic resonance T2* value, conventional echocardiography, and speckle tracking, and also left ventricular longitudinal and circumferential strain values were measured. Myocardial functions of the patients with vitamin D deficiency or insufficiency were evaluated by speckle-tracking echocardiography before and after vitamin D replacement. RESULTS: The mean age of the patients was 15.4 ± 3.09 years. Vitamin D level was deficient in 30 (90%) and insufficient in 3 (10%) of them. Speckle-tracking analysis showed a significantly decreased absolute value of the left ventricular global longitudinal strain before vitamin D replacement. A significant improvement in the global longitudinal strain was detected after vitamin D replacement (P < 0.05). A statistically significant increase was observed in parameters showing left ventricular systolic and diastolic functions after vitamin D replacement. CONCLUSION: Vitamin D deficiency is frequently observed and causes decreased contractility in thalassemic patients. In our study, we observed that our patients' cardiac functions had improved after vitamin D replacement therapy.


Assuntos
Disfunção Ventricular Esquerda , Deficiência de Vitamina D , Talassemia beta , Humanos , Criança , Adolescente , Talassemia beta/complicações , Talassemia beta/patologia , Vitamina D , Estudos Prospectivos , Ecocardiografia/métodos , Miocárdio/patologia , Função Ventricular Esquerda
8.
Ulus Travma Acil Cerrahi Derg ; 29(5): 553-559, 2023 May.
Artigo em Inglês | MEDLINE | ID: mdl-37145041

RESUMO

BACKGROUND: Thoracic and abdominal computed tomography scans are widely used modalities for trauma patients in emergency department (ED). However, alternative diagnostic and follow-up tools are also needed, due to limitations such as high cost and exces-sive radiation exposure. This study aimed to investigate the utility of repeated extended focused abdominal sonography for trauma (rE-FAST) performed by the emergency physician in patients with stable blunt thoracoabdominal trauma. METHODS: This was a prospective, single-center diagnostic accuracy study. Patients with blunt thoracoabdominal trauma admitted to the ED were included in the study. The E-FAST was performed on the patients included in the study at the 0th h, the 3rd h, and the 6th h during their follow-up. Then, the diagnostic accuracy metrics of E-FAST and rE-FAST were calculated. RESULTS: The sensitivity and specificity of E-FAST in determining thoracoabdominal pathologies were found to be 75% and 98.7%, respectively. The sensitivity and specificity for specific pathologies were 66.7% and 100% for pneumothorax, 66.7% and 98.8% for hemothorax, and 66.7% and 100% for hemoperitoneum, respectively. The sensitivity and specificity of rE-FAST in determining thoracal and/or abdominal hemorrhage in stable patients were found to be 100% and 98.7%, respectively. CONCLUSION: E-FAST successfully rules in thoracoabdominal pathologies in patients with blunt trauma, with its high specificity. However, only a rE-FAST might be sensitive enough to exclude traumatic pathologies in these stable patients.


Assuntos
Traumatismos Abdominais , Ferimentos não Penetrantes , Humanos , Estudos Prospectivos , Ultrassonografia/métodos , Traumatismos Abdominais/cirurgia , Hemoperitônio , Ferimentos não Penetrantes/diagnóstico por imagem , Sensibilidade e Especificidade
9.
Indian J Hematol Blood Transfus ; 38(4): 691-697, 2022 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-36258726

RESUMO

During CD34 + stem cell count to determine the number of stem cells in the allografts from pediatric donors, we noticed a considerable amount of early hematogones (eHGs) within the stem cell gate in flow cytometry. Since the number of hematogones causes a decrease in the total number of stem cells counted within the graft, we planned a retrospective study to analyze the effect of eHGs on transplant outcomes. We also wanted to show how allografts containing high amounts of early HGs affect transplant outcomes. Quantification of CD34 numbers and the number of eHGs were determined by flow cytometry. Patients were divided into 2 groups according to the number of CD 34+ cells calculated after subtracting the number of hematogones within the allograft. Those who received < 2 × 106/kg CD34+ cells and ≥ 2 × 106/kg were defined as group 1 and 2, respectively. Twenty-six patients and their 26 donors were included in the study. The median age of patients was 6.5 years and 5.4 years in Group 1 and 2, respectively. The median donor age was 9 years in Group 1 and 7 years in Group 2. The ages and genders were similar in the two groups (p > 0.05). The number of nucleated cells given to both groups was not different. The number of early hematogones given to both groups was similar (p = 0.93). The mean times to myeloid and platelet engraftments were also similar in the two groups. In this study, we provided trilineage engraftment to all patients in two groups. We could not find a considerable effect of these eHGs in myeloid and platelet engraftments. However, the number of patients included in our study is low, therefore we suggest a study including a large number of donors in order to confirm our findings.

10.
Pediatr Transplant ; 26(8): e14397, 2022 12.
Artigo em Inglês | MEDLINE | ID: mdl-36207767

RESUMO

BACKGROUND: Refugee or asylum seekers (RAS) children are at increased risk of physical, developmental, and behavioral health issues. The aim of this study was to evaluate clinical and psychosocial outcomes of hematopoietic stem cell transplantation (HSCT) in RAS children and compare health-related quality of life (HRQOL) to those of Turkish peers. METHODS: This retrospective study included patients who underwent HSCT aged 0-18 years and completed 100-day post-transplant. The PedsQL 4.0 Generic Core Scale was used in children over 5 years old to compare HRQOL. RESULTS: A total of 166 RAS patients (M/F: 106 /60) underwent 174 HSCTs (six patients had two, and one had three HSCT) compared to 66 Turkish patients. The mean age of the patients in the RAS group was 7.8 ± 4.9 years and similar to controls. A total of 124 patients (75%) were from Syria, and 49 (25%) were from other countries in the Middle East and Africa. The cause of migration was war in 121 (74%) RAS patients. Complications of HSCT were no different between the groups. However, the rate of neutropenic sepsis was significantly higher in the RAS group (p = 0.004). The total scores of HRQOL were not different between RAS and controls. In the RAS group, ratings of social functioning were lower in patients with consanguinity or non-malignant disease or who had match-related donors. DISCUSSION: Identifying areas of difficulty in subscales of HRQOL may help physicians to classify patients who need additional supportive care. Regular monitoring and supporting physical needs may result in better functional outcomes after HSCT.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Refugiados , Humanos , Criança , Pré-Escolar , Qualidade de Vida/psicologia , Turquia , Estudos Retrospectivos , Transplante de Células-Tronco Hematopoéticas/psicologia
11.
Front Plant Sci ; 13: 951097, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-36061762

RESUMO

Plant diseases cause significant decreases in yield and quality of crops and consequently pose a very substantial threat to food security. In the continuous search for environmentally friendly crop protection, exploitation of RNA interferance machinery is showing promising results. It is well established that small RNAs (sRNAs) including microRNA (miRNA) and small interfering RNA (siRNA) are involved in the regulation of gene expression via both transcriptional and post-transcriptional RNA silencing. sRNAs from host plants can enter into pathogen cells during invasion and silence pathogen genes. This process has been exploited through Host-Induced Gene Silencing (HIGS), in which plant transgenes that produce sRNAs are engineered to silence pest and pathogen genes. Similarly, exogenously applied sRNAs can enter pest and pathogen cells, either directly or via the hosts, and silence target genes. This process has been exploited in Spray-Induced Gene Silencing (SIGS). Here, we focus on the role of sRNAs and review how they have recently been used against various plant pathogens through HIGS or SIGS-based methods and discuss advantages and drawbacks of these approaches.

12.
Pediatr Transplant ; 26(5): e14266, 2022 08.
Artigo em Inglês | MEDLINE | ID: mdl-35343635

RESUMO

BACKGROUND: Therapeutic drug monitoring (TDM) of intravenous busulfan (Bu) has been recommended for safe engraftment and decreased toxicity in children undergoing hematopoietic stem cell transplantation (HSCT). This study aims to compare HSCT-related outcomes, such as acute or chronic graft-versus-host disease (GvHD), sinusoidal obstructive syndrome (SOS), event-free survival (EFS), and overall survival (OS) in children with and without TDM for busulfan. METHODS: This retrospective study conducted between February 2012 and February 2021 at our Bone Marrow Transplantation Unit included 172 patients (34% girls) with a median age of 4.70 years (IQR 2.41-10.01). Group A consisted of 46 patients whose Bu doses were adjusted according to actual body weight, and group B consisted of 126 patients whose Bu dose adjustments made according to TDM. RESULTS: Totally, 32 patients (19%) developed moderate or severe SOS. The incidence of SOS was significantly higher in the group without TDM (29% vs. 15%, p = .041). A multivariable analysis showed that the presence of acute GvHD and one alkylating drug-containing conditioning regimen compared with two or three were associated with SOS (p = .03 and p = .002, respectively). In patients with TDM, cumulative Bu dose and area under curve also were not associated with SOS. Other HSCT-related outcomes such as acute or chronic GvHD, relapse and graft rejection rates, OS and EFS rates did not differ between the groups. CONCLUSIONS: TDM and making dose adjustments with Bayesian forecasting over four days of Bu therapy optimizes exposure and reduces the risk of SOS in children undergoing HSCT.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Teorema de Bayes , Bussulfano/uso terapêutico , Criança , Pré-Escolar , Monitoramento de Medicamentos , Feminino , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Masculino , Estudos Retrospectivos , Condicionamento Pré-Transplante/efeitos adversos
13.
Bone Marrow Transplant ; 57(5): 760-767, 2022 05.
Artigo em Inglês | MEDLINE | ID: mdl-35210564

RESUMO

We report the national data on the outcomes of hematopoietic stem cell transplantation (HSCT) for thalassemia major (TM) patients in Turkey on behalf of the Turkish Pediatric Stem Cell Transplantation Group. We retrospectively enrolled 1469 patients with TM who underwent their first HSCT between 1988 and 2020 in 25 pediatric centers in Turkey. The median follow-up duration and transplant ages were 62 months and 7 years, respectively; 113 patients had chronic graft versus host disease (cGVHD) and the cGVHD rate was 8.3% in surviving patients. Upon the last visit, 30 patients still had cGvHD (2.2%). The 5-year overall survival (OS), thalassemia-free survival (TFS) and thalassemia-GVHD-free survival (TGFS) rates were 92.3%, 82.1%, and 80.8%, respectively. cGVHD incidence was significantly lower in the mixed chimerism (MC) group compared to the complete chimerism (CC) group (p < 0.001). In survival analysis, OS, TFS, and TGFS rates were significantly higher for transplants after 2010. TFS and TGFS rates were better for patients under 7 years and at centers that had performed over 100 thalassemia transplants. Transplants from matched unrelated donors had significantly higher TFS rates. We recommend HSCT before 7 years old in thalassemia patients who have a matched donor for improved outcomes.


Assuntos
Doença Enxerto-Hospedeiro , Transplante de Células-Tronco Hematopoéticas , Talassemia , Talassemia beta , Criança , Doença Enxerto-Hospedeiro/etiologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Humanos , Estudos Retrospectivos , Talassemia/complicações , Talassemia/terapia , Condicionamento Pré-Transplante/efeitos adversos , Turquia/epidemiologia , Talassemia beta/complicações , Talassemia beta/terapia
14.
J Pediatr Hematol Oncol ; 44(3): e653-e657, 2022 Apr 01.
Artigo em Inglês | MEDLINE | ID: mdl-34486550

RESUMO

INTRODUCTION: Invasive fungal infections (IFIs) are significant causes of morbidity and mortality in leukemia patients. This study investigated antifungal treatment and prophylaxis features according to leukemia risk groups and treatment phases in pediatric acute lymphoblastic leukemia (ALL) patients who received Berlin-Frankfurt-Munster-based protocols. MATERIALS AND METHODS: We retrospectively examined ALL patients' data between the ages of 1 and 18 and treated them with Berlin-Frankfurt-Munster-ALL protocols between June 2013 and December 2016. RESULTS: A total of 446 febrile neutropenic attacks in 85 children were evaluated. Seventy-two patients received antifungals in 151 infection attacks, while 13 patients did not receive any antifungal treatment during chemotherapy. Empirical, preemptive, or proven treatments were given to 74.8%, 21.2%, and 4% of patients, respectively. The frequency of antifungal therapy increased linearly and significantly from the standard-risk group to the intermediate-risk (IR) group, high-risk (HR) group, and relapsed group. IR patients needed more antifungal therapy while receiving induction, whereas HR patients needed more throughout the induction and HR consolidation blocks than other phases. During induction, IR patients received antifungal therapy similar to HR patients' treatment in the induction and HR consolidation blocks. CONCLUSIONS: Antifungal therapy requirements increased as the severity and intensity of chemotherapy increased for all leukemia risk groups. The requirement of antifungal therapy for IR patients receiving induction was similar to that of HR patients; further studies are needed to evaluate the potential advantages of using primary antifungal prophylaxis in IR patients.


Assuntos
Infecções Fúngicas Invasivas , Leucemia Mieloide Aguda , Leucemia-Linfoma Linfoblástico de Células Precursoras , Adolescente , Antifúngicos/uso terapêutico , Criança , Pré-Escolar , Humanos , Lactente , Infecções Fúngicas Invasivas/tratamento farmacológico , Infecções Fúngicas Invasivas/etiologia , Infecções Fúngicas Invasivas/prevenção & controle , Leucemia Mieloide Aguda/tratamento farmacológico , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Estudos Retrospectivos , Fatores de Risco
16.
Anatol J Cardiol ; 26(1): 57-62, 2022 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-34927587

RESUMO

OBJECTIVE: Anthracyclines are widely used in the treatment of acute lymphoblastic leukemia (ALL). However, cardiotoxicity is the most critical side effect that requires dose limitation. It is thought to occur at first exposure, but the clinical presentation may occur years later. In this study, we aimed to determine the time of initial damage and cardiotoxicity that develops in children with ALL. METHODS: In this prospective study, 13 patients with newly diagnosed intermediate-risk precursor B cell ALL treated with the ALL-IC BFM 2009 protocol were included. Conventional echocardiography, tissue Doppler imaging (TDI), and speckle-tracking echocardiography (STE) were performed in all the patients before chemotherapy, after completing the induction phase, and at the end of the reinduction phase. RESULTS: The mean age of the patients was 7.8±4.6 (3.1-16.3) years. Myocardial velocity during systole (Sm) determined by TDI at the interventricular septum significantly decreased during the induction phase. Despite a decrease in STE parameters, a statistically significant reduction was determined in the global longitudinal strain rate at both left and right ventricles at the end of the induction. Nevertheless, a statistically significant increase was observed among the conventional echocardiographic findings in the left ventricular end-diastolic diameter at the end of the reinduction. CONCLUSION: During the treatment of ALL, subclinical anthracycline-associated cardiotoxicity develops in the early stages of treatment. The findings detected by TDI and STE could be missed by conventional echocardiography. We recommend evaluating patients with these newly developed techniques to detect subclinical cardiotoxicity at an early stage and starting appropriate therapy on time.


Assuntos
Leucemia-Linfoma Linfoblástico de Células Precursoras , Disfunção Ventricular Esquerda , Antraciclinas/efeitos adversos , Cardiotoxicidade/diagnóstico por imagem , Criança , Pré-Escolar , Ecocardiografia , Humanos , Leucemia-Linfoma Linfoblástico de Células Precursoras/induzido quimicamente , Leucemia-Linfoma Linfoblástico de Células Precursoras/tratamento farmacológico , Estudos Prospectivos , Disfunção Ventricular Esquerda/induzido quimicamente
17.
Pediatr Transplant ; 25(7): e14136, 2021 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-34505744

RESUMO

BACKGROUND: Pneumatosis cystoides intestinalis (PCI) is a disorder in which widespread air sacs are present in mucosa, submucosa, subserosa, and intraabdominal area of the intestinal wall. It has a heterogeneous clinical presentation as a rare complication of intestinal graft-versus-host disease (GVHD). Computed tomography is the preferred imaging method for the diagnosis. Since the air sacs could be ruptured spontaneously, the presence of free air in the peritoneal cavity does not confirm intestinal perforation. The conservative treatment approach is sufficient in cases that do not require urgent surgical intervention, such as perforation or obstruction. CASE: Here, we present a 2.5-year-old patient diagnosed with primary hemophagocytic lymphohistiocytosis (pHLH), who underwent allogeneic hematopoietic stem cell transplantation from a matched unrelated donor (MUD) and developed PCI secondary to intestinal GVHD 14th months after HSCT. CONCLUSIONS: Pneumatosis cystoides intestinalis, which is a rare complication, should be kept in mind, especially in patients with intestinal GVHD and receiving intensive immunosuppressive, octreotide, and steroid treatment after HSCT.


Assuntos
Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Linfo-Histiocitose Hemofagocítica/terapia , Pneumatose Cistoide Intestinal/etiologia , Pré-Escolar , Colonoscopia , Evolução Fatal , Doença Enxerto-Hospedeiro/complicações , Humanos , Masculino , Pneumatose Cistoide Intestinal/diagnóstico , Tomografia Computadorizada por Raios X
19.
Am J Hum Genet ; 108(7): 1301-1317, 2021 07 01.
Artigo em Inglês | MEDLINE | ID: mdl-34038740

RESUMO

Human C2orf69 is an evolutionarily conserved gene whose function is unknown. Here, we report eight unrelated families from which 20 children presented with a fatal syndrome consisting of severe autoinflammation and progredient leukoencephalopathy with recurrent seizures; 12 of these subjects, whose DNA was available, segregated homozygous loss-of-function C2orf69 variants. C2ORF69 bears homology to esterase enzymes, and orthologs can be found in most eukaryotic genomes, including that of unicellular phytoplankton. We found that endogenous C2ORF69 (1) is loosely bound to mitochondria, (2) affects mitochondrial membrane potential and oxidative respiration in cultured neurons, and (3) controls the levels of the glycogen branching enzyme 1 (GBE1) consistent with a glycogen-storage-associated mitochondriopathy. We show that CRISPR-Cas9-mediated inactivation of zebrafish C2orf69 results in lethality by 8 months of age due to spontaneous epileptic seizures, which is preceded by persistent brain inflammation. Collectively, our results delineate an autoinflammatory Mendelian disorder of C2orf69 deficiency that disrupts the development/homeostasis of the immune and central nervous systems.


Assuntos
Encefalite/genética , Doenças Mitocondriais/genética , Animais , Evolução Biológica , Sistemas CRISPR-Cas , Linhagem Celular , Encefalite/mortalidade , Feminino , Genes Recessivos , Glicogênio/metabolismo , Humanos , Inflamação/genética , Masculino , Proteínas de Membrana/genética , Doenças Mitocondriais/mortalidade , Linhagem , Convulsões/genética , Convulsões/mortalidade , Peixe-Zebra/genética
20.
Support Care Cancer ; 29(4): 2225-2230, 2021 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-32895740

RESUMO

OBJECTIVES: We aimed to evaluate the impact of pretreatment folate and vitamin B12 deficiencies on the frequency of complications and peripheral blood recovery, in children with acute lymphoblastic leukemia (ALL). METHODS: Pre-induction serum folate and vitamin B12 levels of 88 newly diagnosed ALL patients were evaluated retrospectively. Folate < 3 ng/mL and vitamin B12 < 200 pg/mL were accepted as deficiency. Median hemoglobin, absolute neutrophil count (ANC), and platelet counts, transfusion needs, and complications such as mucositis, febrile neutropenia (FN), bleeding at diagnosis, at 15th and 33rd day of induction, were assessed. Recovery of peripheral blood count, which was defined as an ANC > 1.0 × 109/L and platelet count > 100 × 109/L at 33rd day of chemotherapy were also evaluated. RESULTS: Folate or vitamin B12 deficiencies were observed in 21 (23%) and 40 (45%) children, respectively. Peripheral blood counts, complications rates, and transfusion needs were not statistically different between deficient and normal level groups during induction. The number of febrile days, though not statistically significant, was higher in the both deficient groups. Seventeen of 40 (42.5%) patients with vitamin B12-deficient and 12 of 21 (57.1%) folate-deficient patients experienced at least one episode of FN during induction. FN was more common in folate-deficient group, but that was not statistically significant. Complete peripheral blood recovery at 33rd day of induction was seen in 40% in the vitamin B12-deficient group and 28.6% in folate-deficient group. Peripheral blood recovery rate at day 33 was also similar in both deficient and normal level groups. CONCLUSION: Although pre-induction low serum levels of vitamin B12 and folate did not have statistically significant impact on disease-/treatment-related complications and peripheral blood recovery at induction, the frequency of FN and number of febrile day were higher in both deficiencies and folate-deficient patients, respectively.


Assuntos
Ácido Fólico/sangue , Quimioterapia de Indução/efeitos adversos , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Vitamina B 12/sangue , Criança , Estudos Transversais , Feminino , Humanos , Masculino , Leucemia-Linfoma Linfoblástico de Células Precursoras/sangue , Estudos Retrospectivos
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