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BACKGROUND: Pediatric acute kidney injury (AKI) is a global health concern with an associated mortality risk disproportionately pronounced in resource-limited settings. There is a pertinent need to understand the epidemiology of pediatric AKI in vulnerable populations. Here, we proposed a prospective study to investigate the epidemiology and associated risk factors of "severe dialysis dependent AKI" in children among South Asian nations which would be the first and largest of its kind. METHODS: The ASPIRE study (part of PCRRT-ICONIC Foundation initiative) is a multi-center, prospective observational study conducted in South Asian countries. All children and adolescents ≤ 18 years of age who required dialysis for AKI in any of the collaborating medical centers were enrolled. Data collection was performed until one of the following endpoints was observed: (1) discharge, (2) death, and (3) discharge against medical advice. RESULTS: From 2019 to 2022, a total of 308 children with severe AKI were enrolled. The mean age was 6.17 years (63% males). Secondary AKI was more prevalent than primary AKI (67.2%), which predominantly occurred due to infections, dehydration, and nephrotoxins. Common causes of primary AKI were glomerulonephritis, hemolytic uremic syndrome, lupus nephritis, and obstructive uropathy. Shock, need for ventilation, and coagulopathy were commonly seen in children with severe AKI who needed dialysis. The foremost kidney replacement therapy used was peritoneal dialysis (60.7%). The mortality rate was 32.1%. CONCLUSIONS: Common causes of AKI in children in South Asia are preventable. Mortality is high among these children suffering from "severe dialysis dependent AKI." Targeted interventions to prevent and identify AKI early and initiate supportive care in less-resourced nations are needed.
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BACKGROUND: Primary hyperoxalurias (PHs) constitute rare disorders resulting in abnormal glyoxalate metabolism. PH-associated phenotypes range from progressive nephrocalcinosis and/or recurrent urolithiasis to early kidney failure. METHODS: A retrospective study was conducted for patients with confirmed PH diagnoses from three tertiary centers in Saudi Arabia. Detailed clinical molecular diagnosis was performed for 25 affected individuals. Whole exome sequencing (WES)-based molecular diagnosis was performed for all affected individuals. RESULTS: The male:female ratio was 52% male (n = 13) and 48% female (n = 12), and consanguinity was present in 88%. Nephrolithiasis and/or nephrocalcinosis were present in all patients. Kidney stones were present in 72%, nephrocalcinosis in 60%, hematuria in 32%, proteinuria in 16%, abdominal pain in 36%, developmental delay in 8%, and chronic kidney disease stage 5 (CKD stage 5) was observed in 28% of the patients. The most common PH disorder was type I caused by variants in the AGXT gene, accounting for 56%. The GRHPR gene variants were identified in 4 patients, 16% of the total cases. Seven patients did not reveal any associated variants. Missense variants were the most commonly observed variants (48%), followed by frame-shift duplication variants (28%). CONCLUSIONS: Characterization of the genetic and clinical aspects of PH in this unique population provides direction for improved patient management and further research. A higher resolution version of the Graphical abstract is available as Supplementary information.
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Hiperoxalúria Primária , Nefrocalcinose , Nefrolitíase , Masculino , Humanos , Feminino , Nefrocalcinose/epidemiologia , Nefrocalcinose/genética , Nefrocalcinose/diagnóstico , Hiperoxalúria Primária/complicações , Hiperoxalúria Primária/diagnóstico , Hiperoxalúria Primária/epidemiologia , Estudos Retrospectivos , Arábia Saudita/epidemiologia , Nefrolitíase/genéticaRESUMO
BACKGROUND: Acute kidney injury (AKI) is a complication of coronavirus disease 2019 (COVID-19). The reported incidence of AKI, however, varies among studies. We aimed to evaluate the incidence of AKI and its association with mortality and morbidity in children infected with severe acute respiratory distress syndrome coronavirus 2 (SARS-CoV-2) who required hospital admission. METHODS: This was a multicenter retrospective cohort study from three tertiary centers, which included children with confirmed COVID-19. All children were evaluated for AKI using the Kidney Disease Improving Global Outcomes (KDIGO) definition and staging. RESULTS: Of 89 children included, 19 (21 %) developed AKI (52.6 % stage I). A high renal angina index score was correlated with severity of AKI. Also, multisystem inflammatory syndrome in children (MIS-C) was increased in children with AKI compared to those with normal kidney function (15 % vs. 1.5 %). Patients with AKI had significantly more pediatric intensive care admissions (PICU) (32 % vs. 2.8 %, p < 0.001) and mortality (42 % vs. 0 %, p < 0.001). However, AKI was not associated with prolonged hospitalization (58 % vs. 40 %, p = 0.163) or development of MIS-C (10.5 % vs. 1.4 %, p = 0.051). No patient in the AKI group required renal replacement therapy. Residual renal impairment at discharge occurred in 9 % of patients. This was significantly influenced by the presence of comorbidities, hypotension, hypoxia, heart failure, acute respiratory distress, hypernatremia, abnormal liver profile, high C-reactive protein, and positive blood culture. CONCLUSIONS: AKI occurred in one-fifth of children with SARS-CoV-2 infection requiring hospital admission, with one-third of those requiring PICU. AKI was associated with increased morbidity and mortality, and residual renal impairment at time of discharge.
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Injúria Renal Aguda/epidemiologia , Injúria Renal Aguda/virologia , COVID-19/complicações , Injúria Renal Aguda/diagnóstico , Injúria Renal Aguda/mortalidade , Criança , Pré-Escolar , Creatinina/sangue , Cuidados Críticos , Feminino , Taxa de Filtração Glomerular , Humanos , Incidência , Tempo de Internação , Masculino , Prevalência , Fatores de Risco , SARS-CoV-2 , Síndrome de Resposta Inflamatória Sistêmica/complicaçõesRESUMO
BACKGROUND: Approximately 50% of children with steroid-sensitive nephrotic syndrome (SSNS) will suffer from frequent relapses or steroid dependency, prompting the use of so-called steroid-sparing drugs. In this pilot study, we compare the efficacy and safety of rituximab to oral cyclophosphamide as first-line steroid-sparing medications. METHODS: A prospective open-label non-randomized study of children with frequent relapsing or steroid-dependant SSNS. Exclusion criteria were steroid-resistant disease, prescription of immunosuppressive agents other than prednisolone or levamisole, evidence of impaired kidney function, leucopenia, or active infection. The recruited children were allocated either to the oral cyclophosphamide (3 mg/kg/day for 8 weeks) or intravenous rituximab treatment (two doses of 375 mg/m2/dose, 2 weeks apart) and were monitored for relapses and side effects for 12 months. RESULTS: Forty-six subjects were included from two centers; 27 received cyclophosphamide and 19 received rituximab. One-year relapse-free survival was reached in 17 (58.6%) patients treated with cyclophosphamide compared to 16 (84.2%) with rituximab (adjusted HR 0.36; 95% CI 0.09-1.45; p = 0.151). The mean interval to relapse was 6.9 months in the cyclophosphamide group (N = 10) and 6.3 months in the rituximab group (N = 3). Both treatments were associated with a significant (p < 0.001) reduction in prescribed dose of oral alternate-day steroid from 1.02 to 0.36 mg/kg (cyclophosphamide) and 0.86 to 0.08 mg/kg (rituximab). Importantly, a significantly (p = 0.003) higher percentage of patients achieved complete withdrawal of steroid within 3 months of commencing study treatment in the rituximab (73.7%) versus cyclophosphamide (29.6%) group. Transient leucopenia was the most frequent adverse effect observed in the cyclophosphamide group (18.5%) and one patient (3.4%) had acute hepatotoxicity besides severe leucopenia and neutropenia in the 7th week of treatment with complete recovery with the withdrawal of cyclophosphamide and maintenance of remission. A minor infusion-related reaction in the form of a generalized macular skin rash was observed in one patient (5%) in the rituximab group. CONCLUSIONS: Rituximab is non-inferior to cyclophosphamide and safe as a first-line steroid-sparing agent in children with SSNS. A larger multicenter study is required to assess superiority over cyclophosphamide. Graphical abstract.
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Ciclofosfamida/administração & dosagem , Imunossupressores/administração & dosagem , Síndrome Nefrótica/tratamento farmacológico , Rituximab/administração & dosagem , Criança , Pré-Escolar , Feminino , Humanos , Masculino , Projetos Piloto , Estudos Prospectivos , Indução de Remissão/métodosRESUMO
Following publication of the original article [1].
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BACKGROUND: Acute kidney injury (AKI) in critically ill children is associated with increased mortality and morbidity. In this study we evaluated the effect of AKI severity on the incidence of short-term mortality and morbidity. METHODS: Multicenter prospective cohort study was conducted over two years period. We used the Kidney Disease Improving Global Outcomes (KDIGO) to diagnose and stage AKI. RESULTS: A total of 511 out of 1367 included children (37.4%; 95% CI: 34.8-40.0) were diagnosed with AKI. They were categorized into three KDIGO stages: stage I (mild) in 47.5% (95% CI: 43.2-52.0), stage II (moderate) in 32.8% (95% CI: 28.8-37.1) and stage III (severe) in 19.7% (95% CI: 16.4-23.5). Stage II and III AKI had higher risk of mortality and longer length of stay (LOS) in hospital. Children with stage III AKI were more likely to require mechanical ventilation, referral to pediatric nephrology and discharge with abnormal creatinine level (above 100 uml\L). Hypervolemia, hypocalcemia, anemia, and acidosis were found to be independent risk factors of mortality. CONCLUSION: The extent of severity of AKI is directly associated with increased mortality, LOS and short-term morbidity.
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Injúria Renal Aguda/sangue , Injúria Renal Aguda/diagnóstico , Estado Terminal , Unidades de Terapia Intensiva Pediátrica , Índice de Gravidade de Doença , Equilíbrio Hidroeletrolítico/fisiologia , Injúria Renal Aguda/epidemiologia , Pré-Escolar , Estudos de Coortes , Estado Terminal/epidemiologia , Feminino , Humanos , Unidades de Terapia Intensiva Pediátrica/tendências , Masculino , Estudos Prospectivos , Fatores de Tempo , Resultado do TratamentoRESUMO
Hypokalemic periodic paralysis (HypoPP) is a relatively rare but treatable disorder caused by mutations in the CACNA1S gene. HypoPP patients may experience paralytic episodes associated with hypokalemia and, infrequently, may develop late-onset proximal myopathy. The paralytic attacks are characterized by reversible flaccid paralysis and, in most cases, spare the respiratory muscles and heart. We report a case of CACNA1S periodic paralysis precipitated by vigorous exercise in a 14-year-old boy who presented with sudden-onset paralysis of both his upper and lower extremities. Laboratory evaluation revealed a markedly low serum potassium level. The patients symptoms resolved after correction of the potassium abnormality, and he was discharged with no neurological deficits. Although rare, HypoPP must be differentiated from other causes of weakness and paralysis so that proper treatment can be promptly initiated to ensure good outcomes.
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Canais de Cálcio Tipo L/genética , Paralisia Periódica Hipopotassêmica/diagnóstico , Adolescente , Eletrocardiografia , Humanos , Paralisia Periódica Hipopotassêmica/genética , Masculino , Mutação de Sentido IncorretoRESUMO
OBJECTIVE: To determine physicians` attitudes and stated practice in the management of patients with spinal muscular atrophy (SMA). We also aimed to explore their knowledge about consensus statement for standard of care in SMA and the role of new treatment modalities in changing the method of practice in the management of these cases. METHODS: This is a quantitative observational cross-sectional study, conducted from February to May 2017 among physicians who manage SMA patients in Kingdom of Saudi Arabia. The study cohort included pediatric neurologists, adult neurologists, and physicians of other sub-specialties who manage SMA patients. We used online and paper-based questionnaires. RESULTS: Half of the 169 participants were aware of the consensus guidelines for the care of SMA patients. With regard to the newly released Nursinersen treatment protocol for SMA-diagnosed patients, half of the participants were uncertain, and the other half were hesitant about its outcomes. Junior physicians tended to be significantly more inclined to reverse the do-not-resuscitate (DNR) status of an SMA-diagnosed child than more senior physicians. CONCLUSION: Our results indicate the existence of wide differences in physician practice with children of SMA disease. Our data demonstrate a need for increased awareness of consensus guidelines and further awareness about the physician`s role in the variability of care for children with SMA.
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Gerenciamento Clínico , Fidelidade a Diretrizes , Conhecimentos, Atitudes e Prática em Saúde , Atrofia Muscular Espinal/terapia , Médicos/psicologia , Adulto , Protocolos Clínicos , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Neurologistas/psicologia , Guias de Prática Clínica como AssuntoRESUMO
OBJECTIVE: Interleukin 13 (IL-13) plays a critical pro-inflammatory role in asthma. Several single nucleotide polymorphisms (SNPs) are associated with asthma susceptibility in specific populations; however, further replicative studies in other ethnic groups are mandatory. METHODS: The association between IL-13 SNPs rs762534, rs20541, rs1295686, and rs1800925 (risk alleles A, A, T, and A, respectively) and asthma predisposition in a Saudi Arabian cohort was examined via a case-control cross-sectional study. RESULTS: The frequencies of alleles between asthmatics and control populations were significantly different for rs20541 and rs1295686 SNPs (p < 0.001), whereas the frequencies of genotypes between asthmatics and controls were significantly different only for rs20541. The association of the risk (minor) alleles with asthma was examined using the dominant genetic model. Individuals with at least one copy of the risk alleles A (for rs20541) and T (for rs1295686) had significantly greater odds of being asthmatic (OR = 2.13, 95% CI = 1.39-3.26, p < 0.0001; OR = 1.69, 95% CI = 1.12-2.54, p = 0.008) relative to their most common homozygous genotypes. On the other hand, the minor A alleles for rs762534 and rs1800925 were not significantly associated with asthma risk. Regarding haplotype association analysis, individuals with at least one copy of the minor "risk" allele for both rs20541 and rs1295686 (CATG and CATA, respectively) had greater odds of being asthmatic relative to CGCG haplotype; however, this trend was not statistically significant (p > 0.3). CONCLUSIONS: IL-13 minor T and A alleles for rs1295686 and rs20541, respectively, were associated with significantly higher risk of asthma in the Saudi Arabian population.
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Asma/genética , Interleucina-13/genética , Adolescente , Adulto , Fatores Etários , Idoso , Alelos , Asma/epidemiologia , Criança , Pré-Escolar , Estudos Transversais , Feminino , Genótipo , Humanos , Hipersensibilidade Imediata/epidemiologia , Imunoglobulina E/sangue , Masculino , Pessoa de Meia-Idade , Polimorfismo de Nucleotídeo Único , Arábia Saudita/epidemiologia , Fatores Sexuais , Fatores Socioeconômicos , Adulto JovemRESUMO
BACKGROUND: Acute kidney injury (AKI) is a common problem encountered in critically ill children with an increasing incidence and evolving epidemiology. AKI carries a serious morbidity and mortality in patients requiring admission to a pediatric intensive care unit (PICU). METHODS: We undertook a prospective cohort study of PICU admissions at three tertiary care hospitals in the Kingdom of Saudi Arabia over 2 years. The Kidney Disease Improving Global Outcomes (KDIGO) definition was used to diagnose AKI. RESULTS: A total of 1367 pediatrics PICU admissions were included in the study. AKI affected 511 children (37.4%), with 243 children (17.8%) classified as stage I (mild), 168 patients (12.3%) stage II (moderate), and 100 children (7.3%) were classified as stage III (severe). After adjustment for age, sex, and underlying diagnosis, in-hospital mortality was six times more likely among patients with AKI as compared to patients with normal renal function (adjusted OR: 6.5, 95% CI: 4.2-10). AKI was also a risk factor for hypertension (adjusted OR: 4.1, 95% CI: 2.8-5.9) and prolonged stay in the PICU and hospital, as it increased the average number of admission days by 10 (95% CI: 8.6-11) days in the PICU and 12 (95% CI: 10-14) days in the hospital. CONCLUSIONS: One-third of PICU admissions were complicated with AKI. AKI was associated with increased hospital mortality and the length of stay in both PICU and hospital.
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Injúria Renal Aguda/epidemiologia , Criança , Pré-Escolar , Estudos de Coortes , Feminino , Mortalidade Hospitalar , Humanos , Lactente , Unidades de Terapia Intensiva Pediátrica/estatística & dados numéricos , Tempo de Internação/estatística & dados numéricos , Masculino , Estudos Prospectivos , Fatores de RiscoRESUMO
OBJECTIVE: To assess the knowledge and attitudes of physicians in different specialties who are involved in the care of children with FS. METHODS: We assessed knowledge and attitudes in the management of Febrile seizure (FS) among physicians working in different specialties in the Kingdom of Saudi Arabia using a questionnaire-based cross-sectional study conducted from September-December 2016. RESULTS: Of the 300 physicians who responded to the questionnaire, 178 (59.3%) were males, 119 (39.7%) were consultants, 92 (30.7%) were specialists, and 89 (29.7%) were residents. The majority were general pediatric consultants. Our study showed that the consultants were more aware of the definition of simple FS in comparison to other groups of physicians, and the difference was statistically significant. However, there was no difference between pediatric neurologists and general pediatricians. There was a statistically significant difference among various specialties in the perceived need to perform routine lumbar puncture, neuroimaging, and serum electrolyte determination in the evaluation of children with FS. On the other hand, there was no difference in the perceived need to perform an electroencephalogram among physicians in different specialties. CONCLUSION: The study highlighted the wide variation in knowledge and attitudes of physicians in different specialties with different levels of experience toward the management of FS. The use of clinical practice guidelines will help minimize this diversity.
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Gerenciamento Clínico , Conhecimentos, Atitudes e Prática em Saúde , Médicos/normas , Convulsões Febris/terapia , Adulto , Feminino , Humanos , Masculino , Guias de Prática Clínica como Assunto , Arábia Saudita , Convulsões Febris/diagnósticoRESUMO
This practice guideline was developed by the chronic kidney disease (CKD) Task Force, which was composed of clinical and methodological experts. The Saudi Arabian Ministry of Health and its health holding company commissioned this guideline project to support the realization of Vision 2030's health-care transformation pillar. The synthesis of these guidelines was guided by the Grading of Recommendations Assessment, Development, and Evaluation (GRADE)- ADOLOPMENT methodology. The final guidelines addressed 12 clinical questions on the management of blood pressure in patients with CKD through a set of recommen-dations and performance measures. The recom-mendations included antihypertensive agents in children; renin- angiotensin system inhibition (RASi) versus non-RASi in adults; intensive versus standard blood pressure targets; early versus late assessment for kidney replacement therapy (KRT); late versus early preparation strategies for KRT; CKD symptoms during assessment for KRT or conservative manage-ment; initiation of KRT in patients with deteriorating CKD; choice of KRT modality or conservative management in certain CKD patient groups; changing or discontinuing KRT modalities; the frequency of reviews for KRT or conservative management; and information, education, and support. These conditional recommendations were based on a low to very low certainty of evidence, which highlights the need for high-quality randomized trials com-paring different antihypertensive agents in patients with CKD.
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Anti-Hipertensivos , Pressão Sanguínea , Insuficiência Renal Crônica , Terapia de Substituição Renal , Humanos , Insuficiência Renal Crônica/terapia , Insuficiência Renal Crônica/fisiopatologia , Insuficiência Renal Crônica/diagnóstico , Arábia Saudita , Anti-Hipertensivos/uso terapêutico , Criança , Terapia de Substituição Renal/normas , Adulto , Pressão Sanguínea/efeitos dos fármacos , Hipertensão/tratamento farmacológico , Hipertensão/fisiopatologia , Hipertensão/diagnóstico , ConsensoRESUMO
OBJECTIVES: To study childhood nephrolithiasis and nephrocalcinosis caused by metabolic disorders, distal renal tubular acidosis (dRTA), and familial hypomagnesemia, hypercalciuria, and nephrocalcinosis (FHHNC). METHODS: We retrospectively evaluated 86 children presented over 10 years (2011-2021), with nephrolithiasis (89%) and nephrocalcinosis (11%) caused by metabolic disorders (62%), FHHNC (21%), and dRTA (17%). RESULTS: The mean age at discovery was 72.7 months. The underlying metabolic etiologies included hyperoxaluria (38%), cystinuria (32%), hypercalciuria (24%), and hyperuricosuria (6%). Genetic testing was carried out for 23 patients. Hyperoxaluria was typically treated medically (75%). However, the majority progressed to end-stage kidney disease (ESKD). Most children with cystinuria, hypercalciuria, and hyperuricosuria required medical and surgical intervention. Patients with FHHNC typically presented with nephrocalcinosis. Genetic testing revealed Claudin-16 mutations in 7 children. Patients often progressed to stage II-IV chronic kidney disease (61%) and ESKD (6%). Patients with dRTA typically presented with nephrocalcinosis (80%), as well as poor weight gain and failure to thrive (86%), and medical treatment included sodium bicarbonate and potassium replacement. Despite nephrocalcinosis progression, most patients had normal renal function (53%), although the remaining 47% progressed to chronic kidney disease (none reached ESKD). CONCLUSION: Childhood nephrolithiasis is mainly related to metabolic disorders and is associated with poor renal outcomes. Nephrocalcinosis and nephrolithiasis have poor outcomes when associated with FHHNC, while nephrocalcinosis associated with dRTA has relatively good renal outcomes.
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Nefrocalcinose , Nefrolitíase , Criança , Testes Genéticos , Humanos , Hipercalciúria/complicações , Hipercalciúria/epidemiologia , Hipercalciúria/genética , Nefrocalcinose/complicações , Nefrocalcinose/epidemiologia , Nefrolitíase/complicações , Nefrolitíase/epidemiologia , Estudos RetrospectivosRESUMO
IMPORTANCE: Hypermanganesemia with dystonia type 2 is a rare autosomal recessive neurodegenerative disorder characterized by the loss of previously acquired milestones, dystonia, parkinsonian features, a high serum manganese level, and characteristic neuroimaging findings such as bilateral and symmetrically increased T1 and decreased T2/fluid-attenuated inversion recovery signal intensity in the basal ganglia. This condition is secondary to a mutation in the SLC39A14 gene. OBJECTIVE: To present a series of three cases of hypermanganesemia with dystonia type 2, which was genetically confirmed secondary to a mutation in the SLC39A14 gene, and to describe the treatment and clinical course in these cases. DESIGN: A retrospective case series. SETTING: University, Tertiary hospital. PARTICIPANTS: Three unrelated pediatric patients with hypermanganesemia with dystonia type 2, genetically confirmed to be secondary to a mutation in the SLC39A14 gene. EXPOSURES: Chelation therapy using calcium disodium edetate. MAIN OUTCOME(S) AND MEASURE(S): The response to chelation therapy based on clinical improvements in motor and cognition developments. RESULTS: All three patients were started on chelation therapy using calcium disodium edetate, and two of them showed an improvement in their clinical course. The chelation therapy could alter the course of the disease and prevent deterioration in the clinical setting. CONCLUSIONS AND RELEVANCE: Early diagnosis and intervention with chelating agents, such as calcium disodium edetate, will help change the outcome in patients with hypermanganesemia with dystonia type 2. This finding highlights the importance of early diagnosis and treatment in improving the outcomes of patients with treatable neurodegenerative disorders.
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BACKGROUND: Atypical hemolytic uremic syndrome (aHUS) is a rare, life-threatening thrombotic microangiopathy (TMA), which has been treated successfully with eculizumab. The optimal duration of eculizumab in treating patients with aHUS remains poorly defined. METHODS: We conducted a multicenter retrospective study in the Arabian Gulf region for children of less than 18 years of age who were diagnosed with aHUS and who discontinued eculizumab between June 2013 and June 2021 to assess the rate and risk factors of aHUS recurrence. RESULTS: We analyzed 28 patients with a clinical diagnosis of aHUS who had discontinued eculizumab. The most common reason for the discontinuation of eculizumab was renal and hematological remission (71.4%), followed by negative genetic testing (28.6%). During a median follow-up period of 24 months after discontinuation, 8 patients (28.5%) experienced HUS relapse. The risk factors of recurrence were positive genetic mutations (p = 0.020). On the other hand, there was no significant relationship between the relapse and age of presentation, the need for acute dialysis, the duration of eculizumab therapy before discontinuation, or the timing of eculizumab after the presentation. Regarding the renal outcomes after discontinuation, 23 patients were in remission with normal renal function, while 4 patients had chronic kidney disease (CKD) (three of them had pre-existing chronic kidney disease (CKD) before discontinuation, and one case developed a new CKD after discontinuation) and one patient underwent transplantation. CONCLUSIONS: The discontinuation of eculizumab in patients with aHUS is not without risk; it can result in HUS recurrence. Eculizumab discontinuation can be performed with close monitoring of the patients. It is essential to assess risk the factors for relapse before eculizumab discontinuation, in particular in children with a positive complement variant and any degree of residual CKD, as HUS relapse may lead to additional loss of kidney function. Resuming eculizumab promptly after relapse is effective in most patients.
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OBJECTIVES: To present our experience of treating steroid-dependent nephrotic syndrome (SDNS) in children with repeated doses of rituximab (RTX) with a relatively long follow-up, and to discuss the role of the histopathology type and previous immune-suppressor (IS) drugs on the outcome of these patients. METHODS: The patients included in this prospective study were children with SDNS who were in remission on a high-dose steroid or with additional IS drugs. All patients underwent renal biopsy before RTX treatment. Intravenous RTX was administered monthly at 375 mg/m2 for 4 doses. Response to treatment was defined as maintaining remission with no steroid-sparing agents or prednisone for one year. RESULTS: Seventeen (14 males) patients were enrolled. Approximately 76% had minimal change disease (MCD) and 3 (18%) patients had immunoglobulin M (IgM) nephropathy. Approximately 85% of MCD and 33% of IgM nephropathy showed complete response to RTX. CONCLUSION: Compared to other IS used to treat SDNS, RTX showed a significant decrease in relapse rate with fewer side effects. The dose and interval should be modified according to the patient's characteristics, such as medical history, pathology type, and previous IS agents.
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Síndrome Nefrótica , Criança , Humanos , Imunoglobulina M , Imunossupressores/uso terapêutico , Masculino , Síndrome Nefrótica/tratamento farmacológico , Estudos Prospectivos , Proteinúria , Recidiva , Rituximab/uso terapêutico , Resultado do TratamentoRESUMO
Background: Renal stones (nephrolithiasis and urolithiasis) and nephrocalcinosis are uncommon in children; however, their incidences in pediatric populations have been increasing. Patients and Methods: This multicenter retrospective study compared the clinical presentation, etiology, and outcomes of childhood nephrolithiasis or urolithiasis with those of nephrocalcinosis. Results: The study included 144 children: 93 with renal stones and 51 with nephrocalcinosis. The mean age at presentation was 72 months and 54 months for children with renal stones and nephrocalcinosis, respectively. A history of consanguinity was found in 65% and 76% of the cases of renal stones and nephrocalcinosis, respectively. Congenital anomalies of the kidneys and urinary tract (CAKUT) were present in 28 and 9.8% of the patients with renal stones and nephrocalcinosis, respectively. The most common symptoms of renal stones were flank pain (29%), hematuria (15%), and dysuria (11%). Urinary tract infection was the primary presentation in the nephrocalcinosis group (18%), followed by failure to thrive (16%), polyuria (12%), and dehydration (12%). The majority of renal stone cases were caused by metabolic disorders, including hyperoxaluria (18%), cystinuria (18%), hypercalciuria (12%), and hyperuricosuria (2%). In contrast, the most common underlying disorders in cases of nephrocalcinosis were familial hypomagnesemia, hypercalciuria, nephrocalcinosis (35%), distal renal tubular acidosis (23%), and Bartter syndrome (6%). Clinical outcomes were significantly better in children with nephrolithiasis/urolithiasis than in those with nephrocalcinosis, who showed radiological evidence of worsening/persistent calcinosis and progressed more frequently to chronic kidney disease (stage II-IV) and end-stage kidney disease. Conclusion: The average age at presentation for children with renal stones was greater than that for those presenting with nephrocalcinosis. More than 25% of the children with renal stones were found to have CAKUT. Nephrocalcinosis was associated with worse clinical outcomes related to kidney function and disease resolution than nephrolithiasis.
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BACKGROUND: Lumbar puncture (LP) remains an essential diagnostic procedure for neurological and infectious diseases. However, it remains a source of anxiety to patients and families. This research aimed to assess the impact of a newly developed educational simplified video about LP, in the parents' native language, that is tailored to their social background and beliefs and to assess whether it can facilitate their consent for the procedure. METHODS: This prospective, interventional study was conducted at the outpatient pediatric clinics at a teaching hospital. The conventional arm used verbal explanation about LP. The second method utilized a standardized video, having the same information as the conventional arm, with streaming of graphic depictions. Parents' knowledge and perceived LP risks were measured before and after the intervention. RESULTS: Two hundred and one parents were enrolled, with no significant differences in the socio-demographic and baseline characteristics. Both verbal and video-based counseling were found to provide a statistically significant increase in knowledge scores, and a Wilcoxon signed-rank test showed that knowledge gains for both groups were statistically significant (Verbal Explanation: W = 2693, n = 83, P < .001 and Video: W = 5538, n = 117, P < .001). However, the conventional verbal counseling resulted in more consistent gain of knowledge (SD = 14.5) as compared to the video group (SD = 18.94). The video group reported higher perceived risk (Mean 8.2, SD 3.59) than the verbal explanation group (Mean 7.12, SD 2.51). The less educated parents perceived LP procedure to be of significantly higher risk after watching the video (P < .001). It was found that 73.6% of persons with perceived intention to refuse LP changed their opinion after either of the counseling interventions, with significant opinion difference pre- and post-counseling intervention (P value .002). CONCLUSIONS: Video education in parents' native language about LP is as effective as conventional verbal education for informed consent, with the additional advantage of reproducibility and more illustrations to give parents better insight. However, this video should be followed by direct interaction with parents to ensure their full understanding and address any further concerns.
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BACKGROUND: The COVID-19 global pandemic caused by severe acute respiratory syndrome coronavirus 2 infection, warranted attention for whether it has unique manifestations in children. Children tend to develop less severe disease with a small percentage present with clinical manifestations of paediatric multisystem inflammatory syndrome and have poor prognosis. We studied the characteristics of COVID-19 in children requiring hospitalisation in the Kingdom of Saudi Arabia and assessed the clinical presentation and the risk factors for mortality, morbidity, and paediatric intensive care (PICU) admission. METHODS: We conducted a retrospective analysis of COVID-19 patients under 15 years hospitalised at three tertiary academic hospitals between 1 March and 30 June 2020. RESULTS: Eighty-eight children were enrolled (>20% were infants). Seven (8%) were in critical condition and required PICU admission, and 4 (4.5%) died of which 3 met the full diagnostic criteria of multi-system inflammatory syndrome and had a high Paediatric Risk of Mortality (PRISM) score at the time of admission. The initial polymerase chain reaction (PCR) test result was positive for COVID-19 in most patients (97.7%), and the remaining two patients had positive result in the repeated confirmatory test. In a subset of patients (20 subjects), repeated PCR testing was performed until conversion to negative result, and the average duration for conversion was 8 (95% CI: 5.2-10.5) days Children requiring PICU admission presented with signs of respiratory distress, dehydration, and heart failure. Most had fever (71.4%) and tonsillitis; 61.4% were discharged within 7 days of hospitalisation. Risk factors for mortality included skin rash, hypotension, hypoxia, signs of heart failure, chest radiograph suggestive of acute respiratory distress syndrome, anaemia, leucocytosis, hypernatraemia, abnormal liver enzymes, and high troponin I, and risk factors for prolonged hospitalisation (>7 days) included the presence of comorbidities, leucopaenia, hyponatraemia, and elevated C-reactive protein. CONCLUSIONS: The majority of hospitalised children had a brief febrile illness and made a full recovery, but a minority had severe disease.