RESUMO
This review focuses on the therapeutic features of umbilical cord blood (UCB) cells as a source for allogeneic hematopoietic stem cell transplantation (aHSCT) in adult and child populations to treat malignant and nonmalignant hematologic diseases, genetic disorders, or pathologies of the immune system, when standard treatment (e.g., chemotherapy) is not effective or clinically contraindicated. In this article, we summarize the immunological properties and the advantages and disadvantages of using UCB stem cells and discuss a variety of treatment outcomes using different sources of stem cells from different donors both in adults and pediatric population. We also highlight the critical properties (total nucleated cell dose depending on HLA compatibility) of UCB cells that reach better survival rates, reveal the advantages of double versus single cord blood unit transplantation, and present recommendations from the most recent studies. Moreover, we summarize the mechanism of action and potential benefit of mesenchymal umbilical cord cells and indicate the most common posttransplantation complications.
Assuntos
Doença Enxerto-Hospedeiro , Doenças Hematológicas , Transplante de Células-Tronco Hematopoéticas , Células-Tronco Mesenquimais , Adulto , Criança , Humanos , Doenças Hematológicas/terapia , Células-Tronco Hematopoéticas , Resultado do Tratamento , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Sangue FetalRESUMO
Background and objectives: Acute lymphoblastic leukaemia (ALL) is associated with a cytokine imbalance and oxidative stress, which can be aggravated by malnutrition. Malnutrition, defined by the World Health Organisation (WHO) as obesity or undernutrition, can affect treatment complications and outcomes. Therefore, we aimed to analyse the change in the body mass index (BMI) z-score during induction, as well as evaluate the impact of childhood malnutrition on fevers at an ALL presentation and early response to therapy. Methods: An observational cohort study of 50 consecutive children with ALL, diagnosed in 2019-2022, was performed. Patients were divided into age groups of 0-5, 6-11, and 12-17 years. BMI-for-age z-scores were used to define undernutrition and overnutrition according to WHO growth standards. Results: The number of patients with an abnormal BMI increased from 3 (6%) at diagnosis to 10 (20%) at the end of induction (from 2 (4%) to 6 (12%) in overweight/obese, and from 1 (2%) to 4 (8%) in underweight patients). At the end of induction, all overweight/obese patients were 0-5 years old. On the other hand, a statistically significant decrease in the mean BMI z-score among patients aged 12-17 was observed (p = 0.005). The mean BMI z-score differed statistically significantly among children aged 0-5 presenting with and without fever (p = 0.001). The minimal residual disease (MRD) level at the end of induction was not related to BMI at diagnosis. Conclusions: Despite the use of steroids, adolescents are prone to losing weight during an ALL induction, in contrast to preschool children, who tend to gain weight under the same treatment. BMI at diagnosis was related to a fever of ≥38 °C (at ALL presentation) in the 0-5 age group. The results emphasise the importance of careful nutritional status monitoring, with younger and older children as important target groups for weight gain and weight loss interventions, respectively.
Assuntos
Desnutrição , Leucemia-Linfoma Linfoblástico de Células Precursoras , Pré-Escolar , Adolescente , Humanos , Criança , Recém-Nascido , Lactente , Estado Nutricional , Sobrepeso/complicações , Neoplasia Residual/complicações , Obesidade/complicações , Índice de Massa Corporal , Desnutrição/complicações , Leucemia-Linfoma Linfoblástico de Células Precursoras/complicações , Febre/complicaçõesRESUMO
The reported treatment outcomes of children treated for cancer in Eastern European countries are inferior to those in Northern/Western Europe. We hypothesized that recent survival rates could be comparable to the current standards and performed a population-based analysis of treatment outcome of childhood acute myeloid leukemia (AML) in Lithuania, a small Eastern European country. Children < 18 years old who were treated for AML from 2000 to 2013 were included (n = 54). Estimates of 5-year event-free (EFS5y) and overall survival (OS5y) rates were analyzed. Comparing periods 2000-2006 (n = 32) and 2007-2013 (n = 22), the EFS5y improved from 31 to 63% (p = 0.04), and the OS5y improved from 31 to 72% (p = 0.02) because of reductions in toxicity-related mortality (42 vs. 15%, p = 0.08) and relapse (43 vs. 25%, p = 0.08). The most significant improvement was demonstrated in high-risk patients (OS5y improved from 26 to 75%, p = 0.02) who benefited from hematopoietic stem cell transplantation: the post-transplant EFS5y increased from 13 to 86% (p = 0.01). CONCLUSIONS: The current survival rate of Lithuanian children treated for AML was comparable to the expected rate in other parts of Europe. What is Known: ⢠In the last three decades, significant improvement has been achieved in treating childhood cancer, with an overall survival (OS) rate of > 80% in high-income countries. The difference in survival rates between Northern/Western and Eastern European countries as well as between high- and middle-/low-income countries is as much as 20%. Recently, the 5-year event-free survival rate of acute myeloid leukemia (AML) has reached > 60% in high-income countries. The survival rates for myeloproliferative diseases were the lowest in Eastern European countries. ⢠The reported inferior survival rates were calculated based on outcome data of patients treated until 2007. The recent survival rates in Eastern European countries are unknown. What is New: ⢠Being a small Eastern European country, Lithuania has experienced good economic growth during the last decade. We hypothesized that economic growth and gain of experience could result in better survival rates of children treated for cancer in our country in recent years. ⢠A population-based analysis of treatment outcome of childhood AML treated in Lithuania in the recent years was performed for the first time. The survival rates of childhood AML in Lithuania are comparable to those of other high-income countries. Current survival rates of children treated for cancer in Eastern European countries could be comparable to the best current standards contributing to better European survival rates of childhood cancer in general.
Assuntos
Leucemia Mieloide Aguda/mortalidade , Leucemia Mieloide Aguda/terapia , Adolescente , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapêutico , Criança , Pré-Escolar , Terapia Combinada , Feminino , Seguimentos , Transplante de Células-Tronco Hematopoéticas , Humanos , Lactente , Recém-Nascido , Lituânia/epidemiologia , Masculino , Melhoria de Qualidade , Estudos Retrospectivos , Análise de Sobrevida , Resultado do TratamentoRESUMO
BACKGROUND AND OBJECTIVE: Return visits (RVs) to a pediatric emergency department (ED) within a short period after discharge have an influence on overcrowding of the ED and reveal some weaknesses of the health care system. The aim of this study was to determine the rate of RVs and factors related to RVs to the pediatric ED in Lithuania. MATERIALS AND METHODS: A retrospective study in an urban, tertiary-level teaching hospital was carried out. Electronic medical records of all patients (n=44097) visiting the ED of this hospital between 1 January and 31 December 2013 were analyzed. Demographic and clinical characteristics of patients who return to the ED within 72h and those who had not visited the ED were compared. Factors associated with RVs were determined by multivariable logistic regression. RESULTS: Of the overall ED population, 33889 patients were discharged home after the initial assessment. A total of 1015 patients returned to the ED within 72h, giving a RV rate of 3.0%. Being a 0-7-year old, visiting the ED during weekdays, having a GP referral, receiving of laboratory tests and ultrasound on the initial visit were associated with greater likelihoods of returning to the ED. Patients who arrived to the ED from 8:01a.m. to 4:00p.m. and underwent radiological test were less likely to return to the ED within 72h. Diseases such as gastrointestinal disorders or respiratory tract/earth-nose-throat (ENT) diseases and symptoms such as fever or pain were significantly associated with returning to the ED. The initial diagnosis corresponded to the diagnosis made on the second visit for only 44.1% of the patients, and the highest rate of the congruity in diagnosis was for injuries/poisoning, surgical pathologies (77.2%) and respiratory tract diseases (76.9%). CONCLUSIONS: RVs accounted for only a small proportion of visits to the ED. RVs were more prevalent among younger patients and patients with a GP referral as well as performed more often after discharging from the ED in the evening and at night.
Assuntos
Serviço Hospitalar de Emergência/organização & administração , Hospitais Pediátricos/organização & administração , Readmissão do Paciente/estatística & dados numéricos , Adolescente , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Recém-Nascido , Lituânia , Masculino , Alta do Paciente/estatística & dados numéricos , Estudos Retrospectivos , Fatores de TempoRESUMO
BACKGROUND: Parenteral nutrition is commonly used to ensure nutrition support and prevent the harmful effects of malnutrition, which frequently occurs after allogeneic hematopoietic stem cell transplantation (aHSCT). Nevertheless, enteral nutrition supports the restoration of the gut barrier and microbiome as well as protects against infectious complications and acute graft-vs-host disease. Percutaneous endoscopic gastrostomy (PEG) may also be beneficial for gastric decompression and drug administration. METHODS: We performed a retrospective cohort study to evaluate the impact of PEG on treatment outcomes in 75 children who underwent aHSCT with (n = 34) or without (n = 41) PEG from 2005 to 2016. RESULTS: In 34 patients, PEG was used to ensure enteral nutrition support (n = 30), oral drug intake (n = 28), and abdominal decompression (n = 2). During the study period, we observed a beneficial association between PEG placement and transplant-related mortality as well as 5-year overall survival compared with the non-PEG group (12.9% vs 59.0%, P = 0.000; 85.3% vs 35.1%, P = 0.000, respectively). The beneficial impact of PEG was most prominent on 5-year overall survival in older children (12-17 years) with grafts from matched unrelated donors. CONCLUSIONS: PEG placement had a positive association with transplant outcomes in pediatric patients undergoing aHSCT. To confirm these results, larger prospective studies are needed.