Replacement Therapy In Children Research Articles

P190 Features of dosing of enzyme replacement therapy in children with cystic fibrosis in the Russian Federation: cross-sectional study

P190 Features of dosing of enzyme replacement therapy in children with cystic fibrosis in the Russian Federation: cross-sectional study

Progress in the application of regional citrate anticoagulation in continuous renal replacement therapy in children

局部枸橼酸抗凝是儿童连续性肾脏替代治疗的首选抗凝方式，但国内普及率仍较低。本文回顾国内外枸橼酸抗凝相关文献，针对局部枸橼酸抗凝的应用要点包括适应证及禁忌证、血流量及枸橼酸的输入速度、治疗剂量和液体成分、补钙量、代谢并发症的防治等及相关进展进行综述。.

Impact of nephrology care trajectories pre-CKD stage 5 on initiation of kidney replacement therapy in children.

To improve pre-emptive kidney transplantation (PKT) in children and limit starting dialysis in an emergency, we aimed to describe nephrology care trajectories pre-CKD stage 5. We included all children in France who, between 2010 and 2016, started kidney replacement therapy (KRT): standard dialysis (reference group) and emergency dialysis or PKT. We identified four pre-CKD stage 5 nephrology care trajectories before KRT that were extracted from the national exhaustive medical-administrative database and used logistic regression to explore associations between patient characteristics, care trajectories, and KRT initiation. Six hundred forty-three pediatric patients started KRT in France; 406 started dialysis and 30.5% emergency dialysis. The "optimal" care trajectory encompassed 179 patients, 82.7% with at least 18months nephrology follow-up. Conversely, the "no care" trajectory encompassed 118 patients with no nephrology follow-up before KRT. The "severe" trajectory encompassed 128 patients; 93% hospitalized more than once a year and 18% in an intensive care unit. Finally, the "irregular" trajectory encompassed 127 patients, 77% and 46% with irregular laboratory monitoring and CKD drug delivery, respectively. With the "optimal" trajectory as the reference, probability of emergency dialysis was higher with the "irregular" and "no care" trajectories (odds ratio 3.02 [95% confidence interval 1.18-7.66] and 26.5 [10.8-64.8], respectively), and PKT was reduced with the "severe" trajectory (0.43 [0.23-0.82]). We identified a group of patients with irregular follow-up who may benefit the most from interventions aiming at improving adherence to treatment and earlier diagnosis of their CKD to improve access to PKT. A higher resolution version of the Graphical abstract is available as Supplementary information.

Evaluation of Clinical Features and Renal Replacement Therapy in Children with Typical Hemolytic Uremic Syndrome

Objective: A subset of typical Hemolytic-uremic syndrome (HUS) patients requires initiation of renal replacement therapy (RRT) despite aggressive medical intervention. This study aims to evaluate the clinical and laboratory findings of patients diagnosed with typical HUS and efficacy and safety of peritoneal dialysis (PD) and hemodialysis (HD) were compared in managing these cases. Material and Methods: Patients having a diagnosis of typical HUS were retrospectively determined, and assessment the effects of RRT modalities on renal survival. Results: The present study included 49 children with typical HUS, and the mean age was 2.99 ± 1.88 years. Neurological involvement was detected in 15 (30.6%) patients. The neutrophil/lymphocyte ratio (NLR) was significantly higher in the neurological involvement group [odds ratio (OR) 15.42, 95% CI (3.39-70.1), p = 0.005]. In total, 43 (87.7%) patients received RRT and the median time from the first recorded symptom to RRT was six days (range 3-11). While throughout RRT, 10.2% of patients required mechanical ventilation. In this study, 26 (53.1%) patients were managed with PD, and 17 (34.6%) patients were managed with HD. During follow-up, 10.2% of all patients progressed to chronic kidney disease (CKD), proteinuria was detected in 6.1% and hypertension in 4.08%. The risk of CKD was significantly lower in the 3-month period in PD patients than in HD patients (odds ratio (OR) 7.69, 95% CI (0.77-76.07), p = 0.04). Conclusion: We concluded that PD might be as effective and safer as HD concerning kidney recovery. Clinical features and inflammatory markers can predict neurological involvement in typical HUS cases.

Evaluation of endocrine functions before and after enzyme replacement therapy in children with mucopolysaccharidosis

There is a scarcity of data concerning endocrine function and the effects of enzyme replacement therapy (ERT) on growth and other endocrine functions in patients with mucopolysaccharidosis (MPS). This study retrospectively evaluated height increase, bone mineral density (BMD), and other endocrine functions in MPS patients who received ERT for at least two years in our clinic. The clinical findings, hormonal analysis (TSH, fT4, ACTH, cortisol, FSH, LH, testosterone, estradiol, prolactin, GH, IGF-1, IGFBP-3), and BMD measurements of 10 MPS patients, aged 0-18 years, before and after ERT were recorded and compared retrospectively. Four patients were MPS Type-2, five were MPS Type-4, and one was MPS Type-6. Nine of the patients were male, and one was female. The mean treatment onset age was 7.6 years. The mean ± standard deviation of the patients' height SDS was -4.61±3.01 before treatment and -5.65±2.42 after treatment. While the mean BMD Z-score of the patients was -2.33±1.77 before treatment, it was -0.94±1.52 after treatment. Apart from growth retardation (one of the most prominent disease features), no severe dysfunction was detected in other endocrine organs in our patients with MPS. Severe short stature was evident at the time of diagnosis in MPS patients, and their height SDS worsened, despite ERT. However, osteoporotic BMD Z-scores at diagnosis improved after ERT. No serious dysfunction was seen in other endocrine organs.

Evaluation of renal replacement therapy in children and adolescents in the state of Amazonas, Brazil

Objective:To describe the characteristics of stage-5 chronic kidney disease (CKD) children and adolescents undergoing renal replacement therapy (RRT) in Amazonas, Brazil, estimating the frequencies of current and new cases, describing the presence of anemia and bone metabolism disorders.Methods:Thirty-five patients aged 7 to 19 years-old on hemodialysis (HD) or peritoneal dialysis (PD) were studied between June 2018 and April 2019. The frequencies of current and new cases were estimated based on the 0 to 19 years-old population of Amazonas, in the same period. Data were collected about the underlying cause and diagnosis of CKD, dialysis, and biochemical analysis.Results:The frequencies of current and new cases were 24 and 15 patients per million people of compatible age (pmpca), respectively. The causes of CKD were nephrotic syndrome (22.8%), nephritic syndrome (14.3%), and neurogenic bladder (14.3%); in 48.6%, the cause was unknown/not investigated. Ten patients underwent renal biopsy, seven with segmental and focal glomerulosclerosis. The majority (80%) were on HD, with an average kt/V of 1.4, and in 51.4% the vascular access was the double lumen catheter. Hypocalcemia was found in 82.8% of patients, hyperphosphatemia in 57.2%, vitamin D insufficiency or deficiency in 60%, and altered parathyroid hormone values in 48.6%. Hemoglobin was low in 80%, with absolute/functional iron deficiency in 28.6%.Conclusions:In children and adolescents of Amazonas, Brazil, we found 24 pmpca with stage-5 CKU currently in RRT and 16.3 pmpca per year of new cases requiring RRT. Most patients were adolescents on HD, half without a causal diagnosis of CKD, with a high frequency of anemia and bone metabolism disorder.

Allergic Reactions at Enzyme Replacement Therapy in Children with Mucopolysaccharidosis Type II

Mucopolysaccharidosis type II (MPS II; Hunter syndrome) is rare hereditary disease caused by changes in the IDS gene and associated deficiency of lysosomal enzyme iduronate-2-sulfatase (I2S). The main treatment scheme for children with MPS II is enzyme replacement therapy (ERT) with recombinant human I2S. The major issue of ERT is development of allergic (sometimes up to severe anaphylaxis) reactions to recombinant enzymes. The article covers features of infusion-related reactions to ERT, it describes pathogenesis, diagnostic criteria management algorithm of anaphylaxis. Whereas, there is the need of further studies on allergic infusion-related reactions to ERT in children.

Outcome of immunoglobulin replacement therapy in children with rhinosinusitis.

Outcome of immunoglobulin replacement therapy in children with rhinosinusitis.

Épidémiologie de la maladie rénale chronique chez l’enfant

Major advances have been made in the management of children with chronic kidney disease over the past 30 years. However, existing epidemiology data are primarily from kidney replacement therapy registries, and information available at earlier stages of chronic kidney disease is limited. The incidence and prevalence of chronic kidney disease stages 2 to 5 remain poorly understood. However, rare population-based studies suggest that the prevalence of all-stage chronic kidney disease may be as high as 1% of the pediatric population. Congenital disorders including congenital abnormalities of the kidney and urinary tract and hereditary nephropathies account for one-half to two-thirds of pediatric chronic kidney disease cases in middle and high-income countries, whereas acquired nephropathies seem to predominate in low-income countries. The progression of chronic kidney disease is slower in children with congenital disorders than in those with acquired nephropathy, particularly glomerular disease, resulting in a lower proportion of congenital abnormalities of the kidney and urinary tract as a cause of end-stage kidney disease compared to less advanced stages of chronic kidney disease. The incidence of kidney replacement therapy in the pediatric population ranged by country from 1 to 14 per million children of the same age in 2018 (approximately 8 per million children in France) in patients younger than 20 years. The prevalence of kidney replacement therapy in children under 20 years of age in 2018 ranged from 15-30 per million children in some Eastern European and Latin American countries to 100 per million children in Finland and the United States (56 per million children in France). Most children with end-stage kidney disease initiate kidney replacement therapy with dialysis (more frequently hemodialysis than peritoneal dialysis). In about 20% of cases, the initial kidney replacement therapy modality is a pre-emptive kidney transplantation. In high-income countries, 60-80% of prevalent children with end-stage kidney disease live with a functioning transplant (75% in France). While the survival of children with chronic kidney disease has continuously improved over time, mortality remains about 30 times higher than in the general pediatric population.

The role of nutrition and pancreatic enzyme replacement therapy in children with cystic fibrosis

Background Cystic fibrosis (CF) is an inherited genetic disorder with high mortality and morbidity. CF is strongly correlated with malnutrition due to higher energy losses, pancreatic insufficiency, chronic inflammation, higher resting energy expenditure, and feeding problems. Malnutrition in CF patients associated with worse survival. Thus, appropriate and prompt nutritional intervention should be addressed to reduced malnutrition in CF patients.
 Methods The literature search was performed on 9 August 2021 in four major databases such as MEDLINE, EBSCOhost, Cochrane Reviews, and Web of Sciences to find the role of nutrition and pancreatic enzyme replacement therapy in pediatrics population with cystic fibrosis.
 Recent findings In recent decades, early nutritional management and pancreatic enzyme replacement therapy (PERT) have been shown to improve CF patient’s outcomes. Nutrition should be given in higher calories compared to healthy individuals with close and regular nutritional status monitoring. High protein and fat diets are essential for CF patient’s overall survival. Adequate level of micronutrients should be ensured to avoid morbidity caused by micronutrients deficiency. Regular pancreatic insufficiency screening should be done annually in order to start PERT early. Further research focusing on body composition, growth chart, protein intake, and PERT are needed to further improve the management of CF patient.
 Conclusion Nutritional intervention and PERT play an important role in prolonging CF patient survival. Both treatments should be initiated early with nutritional status close monitoring and tailored to each individual. Collaboration with parents and children is critical to warrant that CF patients followed the dietary advice.

Tailored prophylaxis in children with severe hemophilia: A four-year Iranian study

BackgroundProphylaxis the current standard care for patients with severe hemophilia should be planned to optimize the replacement therapy and minimize bleeding. We report our single-center experience of tailored prophylaxis in children affected by hemophilia A (HA) and hemophilia B (HB). MethodsThis study was conducted on 55 patients, under 15 years, with HA (PWHA, n: 46) and HB (PWHB, n: 9) between 2015 and 2019. According to the phenotype, three prophylaxis regimens: 25−50 unit/kg once, twice, or three-times a week for PWHA, and two: 30−50 unit/kg once or twice a week for PWHB were administered. Following the occurrence of > 3 joint bleeding, or > 4 soft tissue bleeding, or one spontaneous major bleeding in the last 3 months, the prophylaxis regimen is changed. Annualized bleeding rate (ABR), annualized joint bleeding rate (AJBR), target joints development, inhibitor development, and hemophilia joint health score (HJHS) also were assessed. ResultsA mean ± SD of 2520 ± 1045 IU/kg/yr coagulation factor (F) VIII was used to reduce ABR and AJBR from 1.02 ± 1.11 and 0.8 ± 1.3 (in the first year of the study) to 0.27 ± 0.44 (P < 0.001) and 0.19 ± 0.38 (P = 0.004) (at the end of the study) in PWHA, respectively. Furthermore, in PWHB, in the first year of the study, with using 2168 ± 1216 IU/kg coagulation FIX, ABR and AJBR were 0.19 ± 0.39 and 0.06 ± 0.1. At the end of the study, ABR and AJBR were 0.02 ± 0.05 (p = 0.156) and 0.01 ± 0.03 (p = 0.361), respectively. During the study period, the mean number of the target joints and mean HJHS were 0.25 ± 0.57 and 7.6 ± 2.1 for PWHA and 0 and 6.3 ± 1.8 for PWHB, respectively.Finally, 5 PWHA (11 %) did not need dose-escalation in their prophylaxis regimen, whereas 31 (67 %) and 10 (21 %) PWHA needed two and three infusions a week, respectively. In PWHB, 7 (78 %) and 2 (22 %) were adjusted to receive a once and twice weekly regimen, respectively. ConclusionOur results suggest that tailored prophylaxis is an effective strategy to reduce the rate of bleeding and optimize the replacement therapy in children with hemophilia.

Lactoferrin versus iron hydroxide polymaltose complex for the treatment of iron deficiency anemia in children with cerebral palsy: a randomized controlled trial.

Iron deficiency anemia (IDA) is common among children with cerebral palsy (CP), and studies on the efficacy of lactoferrin (Lf) in the treatment of IDA are limited. This study aimed to compare the efficacy of Lf with that of iron hydroxide polymaltose complex (IPC) in the treatment of IDA in children with CP. This randomized controlled study, conducted at Alexandria University Children's Hospital, enrolled 70 children aged 1-10 years with CP and IDA; 35 children randomly received IPC, whereas the other 35 received Lf. Four children withdrew from the study; thus, only 66 children were analyzed (32 in the IPC group and 34 in the Lf group). At baseline, the hemoglobin level and other blood parameters were similar between the two intervention groups. After four weeks of treatment, both the IPC and Lf groups showed significant improvements in hemoglobin (Hb), serum ferritin (SF), serum iron, total iron-binding capacity, mean corpuscular volume, and mean corpuscular hemoglobin from baseline. Upon comparing the two treatment groups, adjusted mean Hb and SF changes in the Lf group were significantly higher than that of the IPC group (p =0.001and p= 0.033, respectively), and constipation was less likely to occur in the Lf group than the IPC group (p = 0.049 ).Conclusion: Lactoferrin is effective and superior to IPC as an oral iron replacement therapy in children with CP and IDA, as it has fewer side effects. What is Known: • Lactoferrin (LF) is a natural glycoprotein capable of treating iron deficiency anemia (IDA). • Studies on the efficacy of Lf in the treatment of IDA in children with cerebral palsy (CP) are limited. What is New? • This trial compared the efficacy of Lf and iron hydroxide polymaltose complex (IPC) as treatments of IDA in children with CP. • Lf is effective and even better than IPC as a treatment of IDA in children with CP, as it has fewer side effects.

Paediatric kidney transplantation in under-resourced regions-a panoramic view.

Kidney transplantation is the ideal choice of kidney replacement therapy in children as it offers a low risk of mortality and a better quality of life. A wide variance in the access to kidney replacement therapies exists across the world with only 21% of low- and low-middle income countries (LLMIC) undertaking kidney transplantation. Pediatric kidney transplantation rates in these under-resourced regions are reported to be as low as < 4 pmcp [per million child population]. A robust kidney failure care program forms the cornerstone of a transplant program. Even the smallest transplant program entails a multidisciplinary workforce and expertise besides ensuring family commitment towards long-term care and economic burden. In general, the short-term graft survival rates from under-resourced regions are comparable to most high-income countries (HIC) and the challenge lies in the long-term outcomes. This review focuses on specific issues relevant to kidney transplants in children in under-resourced regions by highlighting limitations in the capacity and health workforce, regulatory norms, medical issues, economic burden, factors beyond financial hardship and ethical considerations relevant to these regions. Finally, the perspective of strengthening transplant programs in these regions should factor in the bigger challenges that exist in achieving the health-related sustainable development goals by 2030.

EP175 - One-year results of olipudase alfa enzyme replacement therapy in children with chronic visceral and neurovisceral acid sphingomyelinase deficiency

eP175 - One-year results of olipudase alfa enzyme replacement therapy in children with chronic visceral and neurovisceral acid sphingomyelinase deficiency

Comparison of Efficacy of Oral and Intravenous Iron Replacement Therapy in Children with Systolic Heart Failure and Iron Deficiency

Purpose Iron deficiency (ID) is associated with increased risk of ventricular assist device implantation, transplant, or death in children with systolic heart failure (HF). Oral iron replacement is standard therapy for ID. However retrospective studies in children with HF estimate its efficacy at ∼50%. There are no data comparing the efficacy of oral and intravenous (IV) iron therapy in children with systolic HF. Methods In July 2019, we instituted a screening and treatment protocol for ID in children with systolic HF. We performed a retrospective analysis of patients who were screened with this protocol between 07/2019 and 10/2020. ID was defined as the presence of ≥2 of the following 4 criteria on serum iron testing: iron 300 ng/mL, transferrin saturation Results Forty patients with systolic HF (21 male, median age 4.5 years, 28 cardiomyopathy and 12 congenital heart disease) met inclusion criteria. Nine received oral iron while 31 received IV iron. Post-therapy iron studies were available for 6/9 of the oral iron cohort and 28/31 of the IV iron cohort. The oral and IV iron cohorts were similar in terms of demographics, etiology or chronicity of HF, renal dysfunction, and mechanical ventilation. Pre-treatment serum BNP was higher in the IV iron cohort (median 1092 vs 55, p = 0.028) compared to the oral iron cohort. At 3 months, iron therapy was effective in 50% (3/6) of the oral iron cohort, compared to 86% (24/28) of those on IV iron (p = 0.086). By 6 months, ID was effectively treated in 50% (3/6) of patients on oral iron, compared to 89% (25/28) of patients on IV iron (p = 0.053). Demographics, etiology or chronicity of HF, BNP, renal dysfunction, and mechanical ventilation were not associated with efficacy of iron therapy at 3 or 6 months. Conclusion IV iron tended to be more effective than oral iron in treating ID in children with systolic HF, despite the IV iron cohort having higher BNP levels and being presumably sicker. This is the first pediatric data on the topic, although statistical significance was limited by small sample size. Larger studies are needed to guide effective therapy for ID in children with systolic HF.

Development of the "Hamburg Best Practice Guidelines for ICV-Enzyme Replacement therapy (ERT) in CLN2 Disease" Based on 6 Years Treatment Experience in 48 Patients.

Intracerebroventricular enzyme replacement therapy (ICV-ERT) for CLN2 disease represents the first approved treatment for neuronal ceroid lipofuscinosis (NCL) diseases. It is the first treatment where a recombinant lysosomal enzyme, cerliponase alfa, is administered into the lateral cerebral ventricles to reach the central nervous system, the organ affected in CLN2 disease. If untreated, CLN2 children show first symptoms such as epilepsy and language developmental delay at 2-4 years followed by rapid loss of motor and language function, vision loss, and early death. Treatment with cerliponase alfa has shown to slow the rapid neurologic decline. However, the mode of administration by 4 hour-long intracerebroventricular infusions every 14 days represents a potentially greater risk of infection compared to intravenous enzyme replacement therapies. The Hamburg NCL Specialty Clinic was the first site worldwide to perform intracerebroventricular enzyme replacement therapy in children with CLN2 disease. In order to ensure maximum patient safety, we analysed data from our center from more than 3000 intracerebroventricular enzyme replacement therapies in 48 patients over 6 years with regard to the occurrence of device-related adverse events and device infections. Since starting intracerebroventricular enzyme replacement therapy, we have also developed and continuously improved the “Hamburg Best Practice Guidelines for ICV–Enzyme Replacement Therapy (ERT) in CLN2 Disease.” Results from this study showed low rates for device-related adverse events and infections with 0.27% and 0.33%, respectively. Therefore, following our internal procedural guidelines has shown to improve standardization and patient safety of intracerebroventricular enzyme replacement therapy for CLN2 disease.

Renal replacement therapy in children and adolescents.

This Viewpoint article discusses a study recently published in Swiss Medical Weekly that describes the incidence of end stage renal disease, the primary diagnosis, modalities of renal replacement therapy and long-term outcomes in patients up to 20 years of age, as well as some aspects that differ from renal disease in adults.

Evaluation of non-infectious complications of peritoneal dialysis in children: a multicenter study.

Peritoneal dialysis (PD) is the most common kidney replacement therapy in children. Complications associated with PD affect treatment success and sustainability. The aim of this study was to investigate the frequency of PD-related non-infectious complications and the predisposing factors. Retrospective data from 11 centers in Turkey between 1998 and 2018 was collected. Non-infectious complications of peritoneal dialysis (NICPD), except metabolic ones, in pediatric patients with regular follow-up of at least 3months were evaluated. A total of 275 patients were included. The median age at onset of PD and median duration of PD were 9.1 (IQR, 2.5-13.2) and 7.6 (IQR, 2.8-11.9) years, respectively. A total of 159 (57.8%) patients encountered 302 NICPD within the observation period of 862 patient-years. The most common NIPCD was catheter dysfunction (n = 71, 23.5%). At least one catheter revision was performed in 77 patients (28.0%). Longer PD duration and presence of swan neck tunnel were associated with the development of NICPD (OR 1.191; 95% CI 1.079-1.315, p = 0.001 and OR 1.580; 95% CI 0.660-0.883, p = 0.048, respectively). Peritoneal dialysis was discontinued in 145 patients; 46 of whom (16.7%) switched to hemodialysis. The frequency of patients who were transferred to hemodialysis due to NICPD was 15.2%. Peritoneal dialysis-related non-infectious complications may lead to discontinuation of therapy. Presence of swan neck tunnel and long duration of PD increased the rate of NICPD. Careful monitoring of patients is necessary to ensure that PD treatment can be maintained safely.

Management of hypersensitivity reactions to enzyme replacement therapy in children with lysosomal storage diseases

BackgroundIntravenous recombinant enzyme replacement therapy (ERT) is currently available for 8 lysosomal diseases. Hypersensitivity reactions (HSRs) may be observed during this long-term treatment. ObjectiveTo evaluate the frequency and clinical treatment features of ERT HSRs and the management of desensitizations in children. MethodsMedical records were reviewed retrospectively for patients who received ERT. Those who had experienced HSRs to ERT were included in the study. The demographic characteristics of the patients, culprit enzyme, signs and symptoms, diagnostic tests, management of the reaction, and the protocol employed for the maintenance of ERT were recorded. ResultsDuring the study period, 54 patients received ERT in our institution. A total of 11 patients (20.4%) experienced HSR to ERT. All reactions were of immediate type. The most common symptoms were cutaneous manifestations. A total of 9 patients experienced urticaria, and 2 had anaphylaxis as initial reaction. Patients who had isolated cutaneous symptoms continued their treatments with antihistamines, corticosteroid premedication, slower infusion rate or both. Patients who had recurrent urticaria with these modalities or those who had anaphylaxis continued their ERT with desensitization (n = 8). A total of 3 patients required revisions in desensitization protocols because of recurrent anaphylaxis. ConclusionThe reactions that develop during this long-term treatment may be treated by premedication-prolonged infusion, but in some patients, desensitization protocols are necessary for the continuation of therapy. Revisions in desensitization protocols may be required.

Kidney transplantation and other methods of renal replacement therapy in children: 30 years of observations in one center.

Kidney transplantation (Tx) is regarded as the optimal treatment method for renal replacement therapy (RRT) for end-stage renal disease (ESRD) patients. Children qualified for Tx should receive the organ as soon as possible in order to improve their chances for healthy development. In our center, RRT for children with ESRD has been conducted for 36 years: hemodialysis (HD) since 1982, peritoneal dialysis (PD) since 1992 and the first transplant in 1987. To analyze the rates of different RRT methods in children with ESRD. Special attention was paid to Tx. We compared the rates of RRT methods over 3 subsequent decades (1987-1996, 1997-2006 and 2007-2017). In the period analyzed, 153 children aged from 2 weeks to 18 years were dialyzed. The mean age of the start of RRT was 9.4 years. In 80 children (52.2%), first method was HD, while in 73 patients (47.7%) it was PD. In 25 children, the type of dialysis was changed. Kidney transplantation was performed in 40%, 60.34% and 73% of patients dialyzed in the periods 1987-1996, 1997-2006 and 2007-2017, respectively. The average waiting time for a transplant in the abovementioned decades was 2.25 years, 2.65 years and 1.97 years, respectively. Three children underwent transplantation with a family donor; 1 boy received a transplanted kidney and liver. Two children underwent a preemptive transplant from a deceased donor. The percentage of children with ESRD treated with Tx continues to increase, but in our assessment, it still remains too low. Among the types of dialysis, PD was much more frequently used, which is consistent with pediatric recommendations. Small number of transplants from a living donor and preemptive transplants indicates the need to promote organ donation in Polish society.