Purpose: Idiopathic nephrotic syndrome (INS) is a common glomerular disease observed in children. Depending on their response to steroids, patients can be classified either as having ...steroid-sensitive nephrotic syndrome (SSNS) or steroid-resistant nephrotic syndrome (SRNS). Whereas SSNS patients respond favorably to steroid treatment and achieve a complete remission with normal renal functions, SRNS patients do not, and are at high risk of developing end-stage renal diseases (ESRD). The aim of this study was to analyze the long-term outcomes of patients with INS.
Materials and Methods: In our medical center, records of children diagnosed with INS were evaluated. Demographic information, laboratory results, response to treatment, and clinical progression were analyzed.
Result: Ninety-one children (64% male) with a mean age of 11.1±4.1 years (3.5-18) were included in the patient cohort, with a mean age of diagnosis of 5.2±3.8 years (1-16.2) and a mean follow-up period of 5.7±2.8 years (2-12). Sixty-eight (75%) patients had SSNS, and 23 (25%) patients had SRNS. Among the SSNS patients, 18 (31%) were steroid-dependent, 12 (20%) were frequently relapsing, and 29 (49%) were infrequently relapsing. Renal biopsy was performed on 29 (32%) patients, 59% had focal segmental glomerulosclerosis. The complete remission rate was 94% for all patients, with 100% for SSNS and 74% for SRNS. ESRD was developed for 9% of patients with SRNS.
Conclusion: The response to steroid treatment serves as a valuable prognostic indicator for INS as it plays a pivotal role in mitigating the risk of progression toward end-stage renal failure.
Amaç: İdiyopatik nefrotik sendrom (İNS) çocuklarda yaygın bir glomeruler hastalıktır. Steroid tedavisinin yanıtına göre steroide hassas nefrotik sendrom (SHNS) veya steroid dirençli nefrotik sendrom (SDNS) olarak sınıflandırılır. SHNS hastaları steroid tedavisinde normal böbrek fonksiyonları ile tam remisyon elde ederken, SDNS hastaları bunun aksine olumlu yanıt vermez ve son dönem böbrek hastalığı gelişme riski yüksektir. Bu çalışmanın amacı İNS’li çocukların uzun dönem sonuçlarını değerlendirmektir.
Gereç ve Yöntem: Merkezimizde İNS tanısı alan çocukların tıbbi kayıtları geriye dönük olarak değerlendirildi. Demografik bilgileri, laboratuvar bulguları, tedavi yanıtları ve klinik seyri analiz edildi.
Bulgular: Çalışmaya ortalama yaşı 11.1±4.1 (3.5-18) olan 91 çocuk (%64, erkek) dahil edildi. Ortalama tanı yaşı 5.2±3.8 yıl (1-16.2) ve ortalama izlem süreleri 5.7±2.8 yıl (2-12) idi. Hastaların 68 (%75)’i steroide hassas, 23 (%25)’ü steroide dirençli idi. Steroide hassas olanların 18 (%31)’i steroide bağımlı, 12 (%20)’si sık tekrarlayan, 29 (%49)’u seyrek tekrarlayan idi. Böbrek biyopsi 29 (%32) hastaya yapıldı, %59’unun histopatolojik bulgusu fokal segmental glomeruloskleroz idi. Tüm hastaların %94’ünde tam remisyon sağlandı, SHNS hastalarının tamamı, SDNS hastaların %74’ü tam remisyonda idi. SDNS hastalarının %9’unda son dönem böbrek yetmezliği gelişti.
Sonuç: Steroid cevabı İNS’da son dönem böbrek yetmezliğine ilerlemeyi azalttığından iyi prognoz göstergesidir.
Summary Introduction The clinical tests currently in use for obstructive nephropathy (such as renal ultrasonography, differential radionuclide renal scans and urinary creatinine concentration data) ...are not efficient predictors of the subsequent clinical course. Novel and simple biomarkers are required which, if proven, could be clinically beneficial in determining if a patient is eligible for surgery or reno-protective therapy. More recently, the interest of clinicians has focused on the potential of urinary neutrophil gelatinase-associated lipocalin (uNGAL), urinary kidney injury molecule-1 (uKIM-1) and urinary liver-type fatty acid-binding proteins (uL-FABP) as biomarkers for renal function in children with hydronephrosis (HN). Objective The purpose of this study was to investigate possible clinical applications of uNGAL, uKIM-1 and uL-FABP as beneficial non-invasive biomarkers to determine whether or not surgical intervention is required in children with HN. Study design Renal ultrasonography and radionuclide renal scans were used as diagnostic tools to detect HN. Patients were divided into two groups based on the antero-posterior diameter of their renal pelvis and the presence of dysfunction. Group 1 included 26 children with severe HN (with dysfunction), and group 2 consisted of 36 children with mild HN (without dysfunction). Urine samples were collected from 62 children with HN and 20 healthy children. Results Hydronephrosis was more common in males than in females, with a male to female ratio of 9:1 in the study sample. The incidence of left kidney involvement (32 patients) was slightly higher than right kidney involvement (28 patients). Compared with controls and group 2, the ratio of uNGAL to creatinine was significantly higher in group 1 ( p < 0.05). The biomarker uNGAL/Cr exhibited fairly good diagnostic accuracy, with an area under the curve of 0.68 95% confidence interval 0.6–0.7 and an optimal cut-off value of 0.16 ng/mg Cr (sensitivity 58%, specificity 75%) ( p < 0.05). There was a positive correlation between the uNGAL/Cr ratio and the uKIM-1/Cr ratio ( r = 0.582, p < 0.05) and uL-FABP/Cr ratio ( r = 0675, p < 0.05) in group 1. Discussion The results clearly demonstrated that children with hydronephrosis and dysfunction had significantly increased uNGAL, and uNGAL/Cr concentrations. However, uKIM-1, uKIM-1/Cr, uL-FABP and uL-FABP/Cr concentrations were not significantly different when compared with controls. These results support the use of uNGAL concentrations as an early marker for renal dysfunction in HN. Conclusions The study clearly demonstrated that pediatric patients with hydronephrosis and dysfunction had significantly higher uNGAL to creatinine concentrations as compared with controls. Table Urine concentration of KIM-1/Cr, NGAL/Cr and L-FABP/Cr in patients and controls. Parameters Group 1 (HN with obstruction) Group 2 (HN without obstruction) Control group NGAL/Cr (ng/mg Cr) 1.8 (0 – 16) 0.9 (0 – 21) 0.5 (0 – 14) KIM-1/Cr (ng/mg Cr) 169 (39 – 2809) 215 (37 – 1351) 159 (6 – 525) L-FABP/Cr (ng/mg Cr) 1.2 (0 – 15) 1.5 (0 – 11) 0.8 (0 – 7) Cr: creatinine; HN: hydronephrosis, KIM-1: kidney injury molecule-1; L-FABP: liver fatty-acid-binding protein; NGAL: neutrophil gelatinase-associated lipocalin. a p <0.05 between group 1 and group 2; and between group 1 and controls.
aHUS is caused by the over‐activation and dysregulation of the alternative complement pathway. Data regarding outcomes of pediatric aHUS patients after kidney transplantation are still very scarce. ...Accordingly, the aim of this study was to describe the clinical findings and outcomes of pediatric aHUS patients after renal transplantation. This is a retrospective, multicenter study including 12 patients from the national registry system. Among the 12 patients, eight had received prophylactic eculizumab and none of those patients (except one) had experienced aHUS recurrence during a median follow‐up period of 58.5 (min‐max, 4‐94) months. Although eculizumab had been started on the day before transplantation in one of them, aHUS recurrence occurred during the transplantation procedure. Eculizumab had been stopped in only one patient who had no complement gene mutation after 35 months of therapy, and recurrence had not been observed during the 19 months of follow‐up. In three patients, maintenance doses had been spaced out without any recurrence. One additional patient with anti‐CFH antibody received only two doses of eculizumab for transplantation and had been followed for 46 months without aHUS recurrence. The remaining three patients had not received anti‐C5 therapy and none of those patients experienced aHUS recurrence during a median follow‐up period of 21 (min‐max, 9‐42) months. Prophylactic eculizumab is a safe and effective treatment for the prevention of aHUS recurrence. Eculizumab interval prolongation, discontinuation, and transplantation without eculizumab prophylaxis can be tried in selected patients with close follow‐up.
Atypical hemolytic uremic syndrome (aHUS) occurs due to defective regulation of the alternative complement pathway (ACP) on vascular endothelial cells. Plasma based therapy (PT) was the mainstay of ...the treatment for aHUS for many years until the introduction of therapies targeting blockage of the complement system. The aim of this study was to evaluate patients with aHUS who had been treated with plasma based therapies alone.
The outcomes of seven genetically confirmed aHUS patients (2 girls, 5 males) were evaluated by means of clinical presentation, response to plasma therapy, course of the disease during the follow-up period and last status.
The median age of the patients at admission was 6.7 years (IQR 0.7-7.8). Three patients received plasma exchange therapy and the other four patients were treated with plasma infusions. One patient was lost to follow-up after one year; the median duration of follow-up for other patients was 3.7 years (IQR 2.7-6.5). During the follow up, two patients from our historical records when complement blocking therapies had not been in clinical use yet in Turkey, underwent kidney transplantation. One transplant patient experienced an acute rejection episode without graft loss. The remaining five patients had a glomerular filtration rate of more than 90 ml/min./1.73 m < sup > 2 < /sup > at the last visit.
Although we had a relatively small patient population, our findings indicate that PT might still be considered in selected patients particularly in countries where complement blocking therapies are difficult to reach due to their unavailability or costs that are not covered by the health care systems.
Background
During erythropoietin-stimulating agent (ESA) treatment, hemoglobin (Hb) levels usually fluctuate; this phenomenon is known as “Hb cycling (HC).” In this study, we aimed to evaluate the ...predictors of HC and its impact on left ventricular hypertrophy (LVH) as a patient-important outcome parameter in pediatric dialysis patients.
Methods
Records of patients followed up in nine pediatric nephrology centers between 2008 and 2013 were reviewed. More than 1 g/dL decrease or increase in Hb level was considered as HC. Patients were divided into two groups according to 12-month Hb trajectory as rare cycling (RC) (≤ 3) and frequent cycling (FC) (> 3 fluctuation) as well as three groups based on T-A-Hb levels: < 10, 10–11, and > 11 g/dL.
Results
Two hundred forty-five dialysis (160 peritoneal dialysis (PD) and 85 hemodialysis (HD)) patients aged 12.3 ± 5.1 (range 0.5–21) years were enrolled in this study. Fifty-two percent of the patients had RC, 45% had FC, and only 3% had no cycling. There were no differences between HC groups with respect to age, dialysis modality, having anemia, hospitalization rate, residual urine volume, and mortality. Although left ventricular mass index (LVMI) tended to be higher in RC than FC group (65 ± 37 vs 52 ± 23 g/m
2.7
,
p
= 0.056), prevalence of LVH was not different between the groups (
p
= 0.920). In regression analysis, FC was not a risk factor for LVH, but low T-A Hb level (< 10 g/dL) was a significant risk for LVH (OR = 0.414, 95% CI 0.177–0.966,
p
= 0.04). The target Hb levels were more often achieved in PD patients, and the number of deaths was significantly lower in non-anemic patients (Hb level > 11 g/dL).
Conclusion
Hb cycling is common among dialysis patients. Severity of anemia rather than its cycling has more significant impact on the prevalence of LVH and on inflammatory state.
Mortality Factors in Crush Syndrome Onan, Engin; Torun, Dilek; Kozanoğlu, Rüya ...
Ulusal travma ve acil cerrahi dergisi = Turkish journal of trauma & emergency surgery : TJTES,
03/2024, Letnik:
30, Številka:
3
Journal Article
Recenzirano
Odprti dostop
Crush Syndrome is a major cause of morbidity and mortality following large-scale catastrophic earthquakes. Since there are no randomized controlled studies on Crush Syndrome, knowledge on this ...subject is limited to expert experience. The primary objective is to analyze the epidemiological and demographic characteristics, clinical outcomes, and mortality factors of earthquake victims after the Pazarcik and Elbistan earthquakes on February 6, 2023.
This cross-sectional and observational retrospective study evaluated 610 earthquake victims who presented to our center between February 6 and April 30, 2023. Among these patients, 128 with Crush Syndrome were included in the study. Patient information was gathered from hospital records during their stay and from national registries upon referral. The primary outcome was to identify risk factors for mortality. Demographic and laboratory data were analyzed by acute kidney injury (AKI) stages; mortality-affecting factors were identified through regression analysis.
Of the 128 Crush Syndrome patients (100 adults, 28 children), 64 were female. The AKI rate was 32.8%. Among patients with AKI, the frequency of hemodialysis requirement was 69%, and the mortality rate was 14.2%. The overall mortality rate for patients with Crush Syndrome was 4.6%, compared to 3.9% (19/482) in earthquake victims without Crush Syndrome (p=0.705). Notably, low systolic blood pressure at admission was the only factor significantly affecting mortality in Crush Syndrome patients (Hazard Ratio HR: 1.088, p=0.021, 95% Confidence Interval CI).
Our study highlights low systolic blood pressure upon admission as a significant risk factor for increased mortality in Crush Syndrome patients. This finding may contribute to the literature by emphasizing the importance of monitoring blood pressure under rubble and administering more aggressive fluid therapy to patients with low systolic blood pressure.
Purpose: Urinary tract infection is one of the most common infections in childhood. Because of the long term sequelae, differentiation of pyelonephritis from cystitis is important. The aim of this ...study is to determine the value of biomarkers such as C-reactive protein and procalcitonin and whether preferred to predict pyelonephritis in children without 99mTcDimercaptosuccinic Acid scan. Material and Methods: Fifty children aged 3 months to 16 years with a first urinary tract infection were included in this retrospective observational study. The medians, sensitivity, specificity, and cut-off values of serum C - reactive protein and procalcitonin to predict pyelonephritis were determined. Results: Thirty-two (64%) patients were diagnosed with pyelonephritis and 18 (36%) were diagnosed with cystitis. The cut-off value for C - reactive protein was 34 mg/L to predict pyelonephritis, with 69% sensitivity and 61% specificity. The cut-off value for procalcitonin was 0.23 ng/mL to predict pyehlonephritis, with 69% sensitivity and 66% specificity. In combination, these biomarkers were 63% sensitive and 78% specific to predict pyelonephritis. Conclusion: Using a combination of procalcitonin and C-Reactive Protein is preferred to predict pyelonephritis in children, instead of the 99mTc-Dimercaptosuccinic Acid scan. Because of its disadvantages, the 99mTcDimercaptosuccinic Acid scan should be avoided in children.
Amaç: İdrar yolu enfeksiyonu, çocukluk çağının en sık görülen enfeksiyonlarından biridir. Piyelonefritin uzun dönemde görülen sekelleri nedeniyle sistitten ayırımı önemlidir. Bu çalışmanın amacı, çocuklarda 99mTc-Dimerkaptosüksinik Asid ile sintigrafik görüntüleme yapılmaksızın piyelonefrit ayırımını yapmakta prokalsitonin ve C-Reaktif Protein gibi belirteçlerin tanısal değerini saptamak, 99mTc-Dimerkaptosüksinik Asid ile sintigrafik görüntüleme yapılacak hastalar konusunda yol gösterici olup olmayacağını belirlemektir. Materyal ve Metod: Bu çalışmaya, 3 ay ile 16 yaş arasında, ilk kez idrar yolu enfeksiyonu geçiren 50 çocuk dahil edilmiştir. Retrospektif gözleme dayalı bir çalışmadır. Piyelonefrit tanısı için serum C-Reaktif Protein ve prokalsitonin düzeylerinin ortanca değeri, duyarlılık, özgüllük ve cut-off değerleri saptandı. Bulgular: Hastaların 32’si (%64) piyelonefrit tanısı alırken, 18 (%36) hasta sistit tanısı aldı. Piyelonefrit tanısı için CReaktif Protein cut-off değeri %69 duyarlılık ve %61 özgüllük ile 34 mg/L iken, prokalsitonin için bu değer %69 duyarlılık ve %66 özgüllük ile 0,23 ng/mL saptandı. Her iki belirteçin birlikte kullanılması ise %63 duyarlılık ve %78 özgüllük ile piyelonefrit tanısını destekledi. Sonuç: Çocuklarda piyelonefrit tanısını koymakta serum prokalsitonin ve C-Reaktif Proteinin birlikte değerlendirilmesi, 99mTc-Dimerkaptosüksinik Asid ile sintigrafik görüntüleme yapılacak hastalarda yol gösterici olabilir. Böylece çocuklar 99mTc-Dimerkaptosüksinik Asid ile sintigrafik görüntülemenin dezavantajlarından uzak tutulabilir.
Objective: It is unclear whether exon 2 mutations are variations or mutations that causes the disease. This study aimed to evaluate the clinical features and prognosis exon 2 mutations in Familial ...Mediterranean Fever. Methods: The clinical features, disease severity and prognosis of all patients with at least one exon 2 mutations were evaluated retrospectively. These data were compared separately for homozygous (Group 1), heterozygous (Group 2), compound heterozygous (Group 3), and complex alleles (Group 4), and the data were compared by grouping patients into those with and without exon 10 mutations. Results: There were a total of 119 patients with exon 2 mutations, including 11.7% in Group 1, 36.1% in Group 2, 21.8% in Group 3, and 30.2% in Group 4 were similar in terms of demographic data, clinical characteristics, and disease course. When compared patients with exon 10 mutations (+) to those with exon 10 mutations (-), the exon 10 mutations (+) group had a higher presence of chest pain (100%, p=0.02) and a significantly higher mean Pras severity score (6.66+ or -1.87, 6.01+ or -1.40; p=0.02). Additionally, a higher number of patients with exon 10 mutation (-) achieved remission with treatment (76 (67.9%), 36 (32.1%); p=0.03). Conclusion: Exon 2 mutations have a milder course and higher remission rates but they should be considered as Familial Mediterranean Fever disease because of their similar clinical presentation and response to colchicine treatment with exon 10 mutations. Early treatment and close follow-up should be performed. Keywords: Genetic variation; mutation; genetic disease, inborn; child
Background
ADCK4-related glomerulopathy is an important differential diagnosis in adolescents with steroid-resistant nephrotic syndrome (SRNS) and/or chronic kidney disease (CKD) of unknown origin. ...We screened adolescent patients to determine the frequency of
ADCK4
mutation and the efficacy of early CoQ10 administration.
Methods
A total of 146 index patients aged 10–18 years, with newly diagnosed non-nephrotic proteinuria, nephrotic syndrome, or chronic renal failure and end-stage kidney disease (ESKD) of unknown etiology were screened for
ADCK4
mutation.
Results
Twenty-eight individuals with bi-allelic mutation from 11 families were identified. Median age at diagnosis was 12.4 (interquartile range IQR 8.04–19.7) years. Upon first admission, all patients had albuminuria and 18 had CKD (6 ESKD). Eight were diagnosed either through the screening of family members following index case identification or during genetic investigation of proteinuria in an individual with a history of a transplanted sibling. Median age of these 8 patients was 21.5 (range 4.4–39) years. CoQ10 supplementation was administered following genetic diagnosis. Median estimated glomerular filtration rate (eGFR) just before CoQ10 administration was 140 (IQR 117–155) ml/min/1.73m
2
, proteinuria was 1,008 (IQR 281–1,567) mg/m
2
/day. After a median follow-up of 11.5 (range 4–21) months following CoQ10 administration, proteinuria was significantly decreased (median 363 IQR 175–561 mg/m
2
/day,
P
=0.025), whereas eGFR was preserved (median 137 IQR 113–158 ml/min/1.73m
2
,
P
=0.61).
Conclusions
ADCK4
mutations are one of the most common causes of adolescent-onset albuminuria and/or CKD of unknown etiology in Turkey. CoQ10 supplementation appears efficacious at reducing proteinuria, and may thereby be renoprotective.
Purpose: Rituximab (RTX) has been offered as rescue therapy for patients with difficult-to-treat nephrotic syndrome (frequent relapsing, steroid-dependent and steroid resistant). We aimed to assess ...the efficacy and long-term outcomes of RTX treatment in children with difficult nephrotic syndrome and shared our experiences
Materials and Methods: Medical records of children with difficult nephrotic syndrome who were treated with RTX were retrospectively evaluated. The relapse-free survival rate at 12 month and monitoring of B-cell depletion were assessed.
Results: In the study included 20 children of which 8 had steroid-dependent (SDNS), 6 had frequent relapsing (FRNS), and 6 had steroid-resistant nephrotic syndrome (SRNS). The median number of relapses at 1 year before and after treatment in FRNS/SDNS patients receiving RTX treatment were compared. The median number of relapses decreased from 2 (1-4) to 0 (0-1) times/year. The mean duration of the follow-up period after RTX treatment was 23 (12-59) months, and 8 patients developed relapse. Repeated doses of RTX were administered to 5 patients who relapsed after RTX treatment. In these patients, CD19+B cells re-emerged during remission, while depletion of memory B-cells remained.
Conclusion: The RTX treatment prolonged the remission time in FRNS/SDNS patients, but it was ineffective in SRNS patients. It was determined that the RTX doses can be repeated to maintain remission in these patients, and the best memory B-cell counts can help in timing the repeat doses.
Amaç: Rituksimab (RTX), tedavisi zor nefrotik sendromlu (sık tekrarlayan, steroide bağımlı ve steroide dirençli) hastalar için kurtarma tedavisi olarak önerilmektedir. Amacımız zor nefrotik sendromlu çocuklarda RTX tedavisinin etkinliğini ve uzun dönem sonuçlarını değerlendirmek ve deneyimlerimizi paylaşmaktır.
Gereç ve Yöntem: RTX ile tedavi edilen zor nefrotik sendromlu çocukların tıbbi kayıtları geriye dönük olarak değerlendirildi. Oniki ayda nükssüz sağkalım oranı ve B hücre deplesyonun izlemi değerlendirildi.
Bulgular: Çalışmaya 8'i steroide bağımlı (SBNS), 6'sı sık tekrarlayan (STNS) ve 6'sı steroide dirençli nefrotik sendromlu (SDNS) 20 çocuk dahil edildi. RTX tedavisi alan STNS/SBNS hastalarının tedavi öncesi ve sonrası 1 yıllık ortalama nüks sayısı karşılaştırıldı. Ortalama nüks sayısı 2 (1-4)’den 0 (0-1) kez/yıla geriledi. RTX tedavisi sonrası ortalama takip süresi 23 ay (12-59) ve 8 hastada nüks gelişti. RTX tedavisi sonrası nüks eden 5 hastaya, tekrarlayan dozlarda RTX uygulandı. Bu hastalarda, CD19+B hücreleri remisyon sırasında yeniden ortaya çıkarken, hafıza B hücrelerinin deplesyonu devam etti.
Sonuç: RTX tedavisi, STNS/SBNS hastalarında remisyon süresini uzattı, ancak SDNS hastalarında etkisiz kaldı. Bu hastalarda remisyonun idamesi için RTX dozunun tekrarlanabileceği ve tekrarlama dozlarının zamanlamasında en iyi hafıza B hücre sayısının yardımcı olabileceği saptandı.
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