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Arch. argent. pediatr ; 122(1): e202310061, feb. 2024. tab, ilus
Article in English, Spanish | BINACIS, LILACS | ID: biblio-1525854


El síndrome de Wiskott-Aldrich es un error innato de la inmunidad de herencia ligada al cromosoma X, producido por variantes en el gen que codifica la proteína del síndrome de Wiskott-Aldrich (WASp). Reportamos el caso clínico de un paciente de 18 meses con diagnóstico de Wiskott-Aldrich que no presentaba donante antígeno leucocitario humano (HLA) idéntico y recibió un trasplante de células progenitoras hematopoyéticas (TCPH) con donante familiar haploidéntico. La profilaxis para enfermedad de injerto contra huésped incluyó ciclofosfamida (PT-Cy). El quimerismo del día +30 fue 100 % del donante y la evaluación postrasplante de la expresión de la proteína WAS fue normal. Actualmente, a 32 meses del trasplante, presenta reconstitución hematológica e inmunológica y quimerismo completo sin evidencia de enfermedad injerto contra huésped. El TCPH haploidéntico con PT-Cy se mostró factible y seguro en este caso de síndrome de WiskottAldrich en el que no se disponía de un donante HLA idéntico.

Wiskott-Aldrich syndrome (WAS) is an X-linked genetic disorder caused by mutations in the gene that encodes the Wiskott-Aldrich syndrome protein (WASp). Here, we report the clinical case of an 18-month-old boy diagnosed with Wiskott-Aldrich syndrome, who did not have an HLA-matched related or unrelated donor and was treated successfully with a hematopoietic stem cell transplant (HSCT) from a haploidentical family donor. Graft-versus-host disease (GvHD) prophylaxis included post-transplant cyclophosphamide (PT-Cy). At day +30, the peripheral blood-nucleated cell chimerism was 100% and the WAS protein had a normal expression. Currently, at month 32 post-transplant, the patient has hematological and immune reconstitution and complete donor chimerism without evidence of GvHD. HSCT with PT-Cy was a feasible and safe option for this patient with WAS, in which an HLA matched donor was not available.

Humans , Male , Infant , Wiskott-Aldrich Syndrome/diagnosis , Wiskott-Aldrich Syndrome/genetics , Wiskott-Aldrich Syndrome/therapy , Hematopoietic Stem Cell Transplantation/adverse effects , Graft vs Host Disease/etiology , Bone Marrow Transplantation/adverse effects , Cyclophosphamide
Goiânia; SES-GO; out. 2023. 1-19 p. graf, tab, quad.(Estatística geral de doação e transplantes de orgãos - Goiás).
Monography in Portuguese | LILACS, CONASS, ColecionaSUS, SES-GO | ID: biblio-1515946


Estatística geral de doação e transplantes de orgãos - Goiás que tem como objetivo transcrever em números os resultados de todo o trabalho executado pela Gerência de Transplantes em Goiás

General statistics on organ donation and transplants - Goiás which aims to transcribe into numbers the results of all the work carried out by the Transplant Management in Goiás

Transplants/statistics & numerical data , Bone Marrow Transplantation/statistics & numerical data , Corneal Transplantation/statistics & numerical data , Kidney Transplantation/statistics & numerical data , Liver Transplantation/statistics & numerical data
Goiânia; SES-GO; ago. 2023. 1-19 p. graf, tab.(Estatística geral de doação de orgãos e transplantes de orgãos - Goiás).
Monography in Portuguese | LILACS, CONASS, ColecionaSUS, SES-GO | ID: biblio-1444028


Estatística geral de doação de orgãos e transplantes de orgãos - Goiás tem como objetivo transcrever em números os resultados de todo o trabalho executado pela Gerência de Transplantes em Goiás

General statistics of organ donation and organ transplants - Goiás aims to transcribe in numbers the results of all the work carried out by the Transplant Management in Goiás

Humans , Male , Female , Transplants/statistics & numerical data , Tissue Donors , Tissue and Organ Procurement/statistics & numerical data , Brain Death , Bone Marrow Transplantation/statistics & numerical data , Corneal Transplantation/statistics & numerical data
Oncología (Guayaquil) ; 33(1)4 de Abril 2023.
Article in Spanish | LILACS | ID: biblio-1427630


El trasplante de progenitores hematopoyéticos se ha convertido en una opción curativa y de sobrevida libre de enfermedad que las alcanzadas con otras modalidades terapéuticas en ciertas patologías congénitas o tumorales. A inicios del año 2006 se diseñó el proyecto para la creación de la Unidad de Trasplante de progenitores hematopoyéticos del Hospital de SOLCA ­Guayaquil. En Junio de 2006, la Unidad de Trasplante de Médula Ósea (UTMO) inicia los primeros trasplantes, uno autólogo y otro alogénico, y a partir de entonces se han realizado 375 trasplantes de progenitores hematopoyéticos, de los cuales 166 han sido de tipo alogénicos, 147 con progenitores hematopoyéticos obtenidos desde la sangre periférica o médula ósea propiamente dicha y 19 con células obtenidas desde la sangre de cordón umbilical, así como 209 trasplantes de tipo autólogo. Actualmentese ha diseñado un proyecto de ampliación que contempla una infraestructura con 20 habitaciones para hospitalización y un área para manipulación celular más amplia con equipamiento complementario, lo cual permitirá incrementar la cartera de servicios, a saber: la opción del trasplante alogénico de tipo haploidéntico y ciertos procedimientos de inmunoterapia adoptiva con células T

Hematopoietic stem cell transplantation has become a curative and disease-free survival option than those achieved with other therapeutic modalities in specific congenital or tumor pathologies. At the beginning of 2006, the project for the creation of the Hematopoietic Stem Cell Transplant Unit of the Hospital de SOLCA ­ Guayaquil was designed. In June 2006, the Bone Marrow Transplant Unit (UTMO) began the first transplants, one autologous and the other allogeneic. Since then, 375 hematopoietic progenitor transplants have been performed, of which 166 have been allogeneic, 147 with hematopoietic progenitors obtained from peripheral blood or bone marrow itself, and 19 with cells obtained from umbilical cord blood, as well as 209 autologous transplants. An expansion project has been designed that includes an infrastructure with 20 rooms for hospitalization and a larger area for cell manipulation with complementary equipment, which will make it possible to increase the portfolio of services, namely: the option of haploidentical allogeneic transplantation and specific adoptive T-cell immunotherapy procedures.

Humans , Male , Female , Adult , Middle Aged , Young Adult , Bone Marrow Transplantation , Bone Marrow Neoplasms , Cord Blood Stem Cell Transplantation , Peripheral Blood Stem Cell Transplantation
ABCS health sci ; 48: e023304, 14 fev. 2023. ilus, tab
Article in English | LILACS | ID: biblio-1518577


INTRODUCTION: Bone marrow transplants primarily depend on people who previously registered to be donors. From then on, the search for compatibility between donor and recipient begins. OBJECTIVE: To describe the historical landmarks and the legal apparatus of bone marrow donor banks in Brazil based on an integrative review. METHODS: LILACS database and PubMed and SciELO journals were used. The term bone marrow transplantation was the descriptor. Eligibility criteria were: articles with the theme of Bone Marrow Transplantation (BMT) and studies carried out on the national territory. RESULTS: A total of 88,855 articles were identified, among which 185 met the eligibility criteria. After they were thoroughly read, 14 articles were selected. The studies pointed out fragments that dealt with important historical landmarks for the establishment of bone marrow transplantation as a conventional treatment for oncohematological diseases. CONCLUSION: The use of BMT has a history of more than thirty years in Brazil. However, none of the articles identified specifically addresses the historical content of bone marrow transplantation.

Tissue Donors , Bone Marrow Transplantation/history , Bone Marrow Transplantation/legislation & jurisprudence , Stem Cell Transplantation , Brazil
Rev. Bras. Cancerol. (Online) ; 69(1)jan.-mar. 2023.
Article in English | LILACS, SES-SP | ID: biblio-1512127


Introduction: Hematologic malignancies, including lymphomas and leukemias, may be treated with autologous or allogeneic bone marrow transplantation. However, these approaches can increase the risk of infection, sepsis, graft-versus-host disease, and nephrotoxicity, possibly resulting in acute kidney injury (AKI). Objective: To evaluate AKI in patients with lymphomas or leukemia submitted to bone marrow transplantation (BMT). Method: Retrospective, observational cohort study of cases from a database of 256 patients (53.9% males) hospitalized for BMT between 2012 and 2014 at a cancer hospital in São Paulo, Brazil. Of these, 79 were selected randomly for analysis. Demographic data, length of hospitalization, and associated morbidities were recorded. AKI was identified according to Kidney Diseases Improving Global Outcomes (KDIGO) criteria. Results: The most frequent diagnoses for the 79 cases were non-Hodgkin's lymphoma (30.4%), acute myeloid leukemia (26.6%), and Hodgkin's lymphoma (24.1%). The probability of 100 days-survival after BMT was 81%, and three years after BMT was 61%. In-hospital mortality was significantly higher among patients who presented AKI during hospitalization (p<0.001). However, there was no difference in overall life expectancy (p=0.770). Conclusion: A significant prevalence of AKI was found in patients with leukemia or lymphoma while they were hospitalized for BMT, resulting in significantly increased rates of in-hospital mortality. The presence of AKI during hospitalization was not associated with a subsequent reduction in life expectancy.

Introdução: As neoplasias hematológicas, incluindo linfomas e leucemias, podem ser tratadas com transplante autólogo ou halogênico de medula óssea. No entanto, essas abordagens podem aumentar o risco de infecção, sepse, doença do enxerto contra o hospedeiro e nefrotoxicidade, possivelmente resultando em lesão renal aguda (LRA). Objetivo: Avaliar LRA em pacientes com linfomas ou leucemia submetidos a transplante de medula óssea (TMO). Método: Estudo de coorte observacional retrospectivo de casos de um banco de dados de 256 pacientes (53,9% do sexo masculino) internados por TMO entre 2012 e 2014 em um hospital oncológico de São Paulo, Brasil. Destes, 79 prontuários foram selecionados aleatoriamente para análise. Dados demográficos, tempo de internação e morbidades associadas foram registrados. A LRA foi identificada de acordo com os critérios de Kidney Diseases Improving Global Outcomes (KDIGO). Resultados: Os diagnósticos mais frequentes da amostra de 79 casos foram linfoma não Hodgkin (30,4%), leucemia mieloide aguda (26,6%) e linfoma de Hodgkin (24,1%). A probabilidade de sobrevivência em 100 dias após o TMO foi de 81% e, em três anos após o TMO, foi de 61%. A mortalidade intra-hospitalar foi significativamente maior entre os pacientes que apresentaram LRA durante a internação (p<0,001). No entanto, não houve diferença na expectativa de vida geral (p=0,770). Conclusão: Neste estudo, observou-se prevalência significativa de LRA em pacientes com leucemia ou linfoma durante a internação por TMO, resultando em aumento significativo das taxas de mortalidade intra-hospitalar. A presença de LRA durante a hospitalização não se associou a uma subsequente redução da expectativa de vida

Introducción: Las neoplasias malignas hematológicas, incluidos los linfomas y las leucemias, pueden tratarse con trasplante autólogo o alogénico de médula ósea. Sin embargo, estos enfoques pueden aumentar el riesgo de infección, sepsis, enfermedad de injerto contra huésped y nefrotoxicidad, lo que posiblemente provoque lesión renal aguda (IRA). Objetivo: Evaluar el FRA en pacientes con linfomas o leucemias sometidos a trasplante de médula ósea (TMO). Método: Se realizó un estudio de cohorte observacional retrospectivo de casos de una base de datos de 256 pacientes (53,9% hombres) hospitalizados por TMO entre 2012 y 2014 en un hospital de cáncer en São Paulo, Brasil. De estos, 79 registros fueron seleccionados aleatoriamente para su análisis. Se registraron los datos demográficos, la duración de la hospitalización y las morbilidades asociadas. La IRA se identificó según los criterios de Kidney Diseases Improving Global Outcomes (KDIGO). Resultados: Los diagnósticos más frecuentes en la muestra de 79 casos fueron linfoma no Hodgkin (30,4%), leucemia mieloide aguda (26,6%) y linfoma de Hodgkin (24,1%). La probabilidad de supervivencia 100 días después del BMT fue del 81% y tres años después del BMT fue del 61%. La mortalidad hospitalaria fue significativamente mayor entre los pacientes que presentaron FRA durante la hospitalización (p<0,001). Sin embargo, no hubo diferencia en la esperanza de vida global (p=0,770). Conclusión: En este estudio, se observó una prevalencia significativa de LRA en pacientes con leucemia o linfoma mientras estaban hospitalizados por TMO, lo que resultó en un aumento significativo de las tasas de mortalidad hospitalaria. La presencia de FRA durante la hospitalización no se asoció con una reducción posterior de la esperanza de vida.

Leukemia , Bone Marrow Transplantation , Acute Kidney Injury , Lymphoma
Chinese Journal of Hematology ; (12): 635-641, 2023.
Article in Chinese | WPRIM | ID: wpr-1012205


Objective: To observe the effect of platelets on hematopoietic stem cell (HSCs) implantation in mice with radiation-induced bone marrow injury and bone marrow transplantation models. Methods: ①Male C57BL/6 mice were divided into a single irradiation group and a radiation infusion group after receiving (60)Co semimyeloablative irradiation for 18-10 weeks. The irradiation infusion group received 1×10(8) platelets expressing GFP fluorescent protein. ② The allogeneic bone marrow transplantation model was established. The experimental groups included the simple transplantation group (BMT) and the transplantation infusion group (BMT+PLT). The BMT group was infused through the tail vein only 5 × 10(6) bone marrow cells, the BMT+PLT group needs to be infused with bone marrow cells at the same time 1× 10(8) platelets. ③ Test indicators included peripheral blood cell and bone marrow cell counts, flow cytometry to detect the proportion of hematopoietic stem cell (HSC) and hematopoietic progenitor cells, bone marrow cell proliferation and apoptosis, and pathological observation of vascular niche damage and repair. Results: ①On the 3rd, 7th, 14(th), and 21st days after irradiation, the bone marrow cell count of the infusion group was higher than that in the single irradiation group (P<0.05), and the peripheral blood cell count was also higher. A statistically significant difference was found between the white blood cell count on the 21st day and the platelet count on the 7th day (P<0.05). In the observation cycle, the percentage of bone marrow cell proliferation in the infusion group was higher, while the percentage of apoptosis was lower. ② The results of bone tissue immunofluorescence after irradiation showed that the continuity of hematopoietic niche with red fluorescence was better in the irradiation infusion group. ③The chimerism percentage in the BMT+PLT group was always higher than that in the BMT group after transplantation.④ The BMT+PLT group had higher bone marrow cell count and percentage of bone marrow cell proliferation on the 7th and 28th day after transplantation than that in the BMT group, and the percentage of bone marrow cell apoptosis on the 14th day was lower than that in the BMT group (P<0.05). After the 14th day, the percentage of stem progenitor cells in the bone marrow cells of mice was higher than that in the BMT group (P<0.05). ⑤The immunohistochemical results of bone marrow tissue showed that the continuity of vascular endothelium in the BMT+PLT group was better than that in the BMT group. Conclusion: Platelet transfusion can alleviate the injury of vascular niche, promotes HSC homing, and is beneficial to hematopoietic reconstruction.

Mice , Animals , Bone Marrow Transplantation , Bone Marrow , Mice, Inbred C57BL , Hematopoietic Stem Cells , Bone Marrow Diseases , Hematopoietic Stem Cell Transplantation , Mice, Inbred BALB C
Journal of Experimental Hematology ; (6): 233-240, 2023.
Article in Chinese | WPRIM | ID: wpr-971130


OBJECTIVE@#To establish an intestinal organoid model that mimic acute graft versus host disease (aGVHD) caused intestinal injuries by using aGVHD murine model serum and organoid culture system, and explore the changes of aGVHD intestine in vitro by advantage of organoid technology.@*METHODS@#20-22 g female C57BL/6 mice and 20-22 g female BALB/c mice were used as donors and recipients for bone marrow transplantation, respectively. Within 4-6 h after receiving a lethal dose (8.0 Gy) of γ ray total body irradiation, a total of 0.25 ml of murine derived bone marrow cells (1×107/mice, n=20) and spleen nucleated cells (5×106/mice, n=20) was infused to establish a mouse model of aGVHD (n=20). The aGVHD mice were anesthetized at the 7th day after transplantation, and the veinal blood was harvested by removing the eyeballs, and the serum was collected by centrifugation. The small intestinal crypts of healthy C57BL/6 mice were harvested and cultivated in 3D culture system that maintaining the growth and proliferation of intestinal stem cells in vitro. In our experiment, 5%, 10%, 20% proportions of aGVHD serum were respectively added into the organoid culture system for 3 days. The formation of small intestinal organoids were observed under an inverted microscope and the morphological characteristics of intestinal organoids in each groups were analyzed. For further evaluation, the aGVHD intestinal organoids were harvested and their pathological changes were observed. Combined with HE staining, intestinal organ morphology evaluation was performed. Combined with Alcian Blue staining, the secretion function of aGVHD intestinal organoids was observed. The distribution and changes of Lgr5+ and Clu+ intestinal stem cells in intestinal organoids were analyzed under the conditions of 5%, 10% and 20% serum concentrations by immunohistochemical stainings.@*RESULTS@#The results of HE staining showed that the integrity of intestinal organoids in the 5% concentration serum group was better than that in the 10% and 20% groups. The 5% concentration serum group showed the highest number of organoids, the highest germination rate and the lowest pathological score among experimental groups, while the 20% group exhibited severe morphological destruction and almost no germination was observed, and the pathological score was the highest among all groups(t=3.668, 4.334,5.309,P<0.05). The results of Alican blue staining showed that the secretion function of intestinal organoids in serum culture of aGVHD in the 20% group was weaker than that of the 5% group and 10% of the organoids, and there was almost no goblet cells, and mucus was stainned in the 20% aGVHD serum group. The immunohistochemical results showed that the number of Lgr5+ cells of intestinal organoids in the 5% group was more than that of the intestinal organoids in the 10% aGVHD serum group and 20% aGVHD serum group. Almost no Clu+ cells were observed in the 5% group. The Lgr5+ cells in the 20% group were seriously injuried and can not be observed. The proportion of Clu+ cells in the 20% group significantly increased.@*CONCLUSION@#The concentration of aGVHD serum in the culture system can affect the number and secretion function of intestinal organoids as well as the number of intestinal stem cells in organoids. The higher the serum concentration, the greater the risk of organoid injury, which reveal the characteristics of the formation and functional change of aGVHD intestinal organoids, and provide a novel tool for the study of intestinal injury in aGVHD.

Mice , Female , Animals , Mice, Inbred C57BL , Bone Marrow Transplantation , Graft vs Host Disease , Stem Cells , Organoids
Hematol., Transfus. Cell Ther. (Impr.) ; 44(4): 549-554, Oct.-dec. 2022. tab, ilus
Article in English | LILACS | ID: biblio-1421524


ABSTRACT Objective: The objective of this study was to evaluate the oral health condition and dental treatments performed in patients in pre-allogeneic HCT. Method: The records of patients treated during 2018 at a Brazilian HCT service were reviewed. The following oral health data were obtained: 1. Decayed, missing and filled teeth / correlated index for primary dentition (DMFT/dmft); 2. Quality of oral hygiene and 3. Dental pathologies: 3.1 Periodontal infectious focus, 3.2 Endodontic infectious focus and 3.3 Carie incidence. All dental procedures performed were surveyed. Results: Thirty-three patients were included, with a mean age of 28.42 (±16.37), 20 male (60%) and 13 female. The average DMFT/dmft found in this study was 10.24 (± 8.37), similar to the index found in the population in southeastern Brazil. The younger study population presented a DMFT/dmft considered high, when compared to the general population. A total of 27.2% of the patients had active caries lesions, 33.3%, foci of periodontal infection, 15.1%, endodontic infectious focus and 40%, poor oral hygiene. Almost half of the patients (48.4%) had to undergo dental intervention, 24.2% needing periodontal scaling, 21.2%, fillings and 12.1%, tooth extractions. Conclusion: We conclude that the studied population had an important incidence of dental pathologies and infectious conditions that could complicate throughout HCT, especially in younger patients, therefore presenting a high demand for dental treatment in the pre-HCT. Studies that assess the impact of dental conditioning on the outcomes of HCT with an emphasis on dental infectious complications, days of hospitalization and survival are necessary."

Humans , Male , Female , Child, Preschool , Child , Adolescent , Adult , Middle Aged , Young Adult , Oral Health , Hematopoietic Stem Cell Transplantation , Transplantation, Homologous , Bone Marrow Transplantation , Focal Infection
Rev. bras. ortop ; 57(5): 891-895, Sept.-Oct. 2022. graf
Article in English | LILACS | ID: biblio-1407710


Abstract Freiberg disease is a rare condition whose diagnosis requires a high clinical suspicion. Avascular necrosis of the metatarsal head progresses with articular collapse, leading to forefoot pain and limitation of the daily activities. Several surgical techniques have been described to address the disease, and since it is usually diagnosed in later-stages, most of them include joint-destructive procedures. The use of arthroscopy on the small joint of the foot has arisen in the last few years, but its application in Freiberg disease is still scant. Joint-preserving procedures have been advocated for cases of early-stage disease, aiming to relieve symptoms while preventing the progression of the disease. In the present report, we describe a successful treatment of a 12-year-old patient with early-stage Freiberg disease using core decompression and bone marrow graft through a minimally-invasive approach assisted by arthroscopy.

Resumo A doença de Freiberg é rara, e seu diagnóstico requer alto grau de suspeita clínica. A necrose avascular da cabeça do metatarso progride com colapso articular, e causa dor no antepé e limitação da atividade. Há diversas técnicas cirúrgicas para o tratamento da doença; como o diagnóstico geralmente é feito em estágios posteriores, a maioria dessas técnicas inclui procedimentos de destruição articular. A artroscopia tem sido utilizada nas pequenas articulações do pé nos últimos anos, mas sua aplicação na doença de Freiberg ainda é escassa. Procedimentos de preservação articular têm sido preconizados na doença em estágio inicial, com o objetivo de aliviar os sintomas e impedir a progressão. Neste relato, descrevemos o tratamento bem-sucedido de uma paciente de 12 anos de idade com doença de Freiberg em estágio inicial por meio de descompressão central e enxerto de medula óssea, por meio de abordagem minimamente invasiva assistida por artroscopia.

Humans , Female , Child , Arthroscopy , Metatarsal Bones , Bone Marrow Transplantation , Foot
Biomédica (Bogotá) ; 42(3): 531-540, jul.-set. 2022. tab, graf
Article in Spanish | LILACS | ID: biblio-1403604


Introducción. El trasplante de células madre hematopoyéticas es la opción curativa para algunas enfermedades y está aumentando el tiempo de supervivencia de los pacientes. La calidad de vida relacionada con la salud en estos pacientes no se evalúa de manera sistemática. Objetivos. Describir la calidad de vida relacionada con la salud y las complicaciones en niños con trasplante de células madre hematopoyéticas. Materiales y métodos. Es un estudio transversal en pacientes pediátricos sobrevivientes al trasplante. Se midió la calidad de vida relacionada con la salud, utilizando el cuestionario KIDSCREEN-27 en pacientes entre 8 y 14 años y la SF-12™ (Short Form-12) en pacientes mayores de 14 años. El análisis estadístico se realizó en el software Stata 12. Utilizamos el modelo de Rasch, trasladando estimación de parámetros a valores t para obtener el resultado de los cuestionarios. Resultados. En total, 42 pacientes respondieron alguno de los cuestionarios. Los eventos adversos más frecuentes fueron "enfermedad crónica de injerto Vs. contra huésped" y "complicaciones endocrinas". De acuerdo con la normalidad de datos del KIDSCREEN-27, los puntajes de las dimensiones "ambiente escolar" y "soporte social y pares" fueron inferiores al percentil 50. En el cuestionario SF-12™, el grupo que utilizaba inmunosupresores tuvo un menor puntaje en el componente físico. Conclusiones. En general, los resultados del KIDSCREEN-27 sugieren un cierto déficit de calidad de vida en pacientes entre 8 y 14 años. Los cuestionarios mostraron confiabilidad en la muestra.

Introduction: Hematopoietic stem cell transplantation is the curative option for some diseases and is increasing patient survival. The health-related quality of life in these patients is not systematically evaluated. Objectives: The present study sought to describe the health-related quality of life and complications in children who underwent hematopoietic stem cell transplantation. Materials and methods: A cross-sectional study was conducted on pediatric transplanted survivors. Health-related quality of life was measured using the KIDSCREEN-27 scale and Short Form-12 (SF-12) in patients between 8 and 14 years of age and those over 14 years, respectively. Statistical analysis was performed using STATA 12 software. We used the Rasch model person parameter estimates translated into T-values to score the questionnaire. Results: A total of 42 children answered the questionnaires. The most frequent adverse events were chronic graft Vs. host disease and endocrine complications. According to European norm data in the KIDSCREEN-12 scale, scores for the school dimension and social and peer support were below the 50th. percentile. The group administered immunosuppressants had lower scores on the physical component of the SF-12™ scale. Conclusions: In general, the KIDSCREEN-27 does appear to suggest some quality-of-life deficit in younger children. The scales showed reliability in this population.

Quality of Life , Bone Marrow Transplantation , Child , Mental Health
Rev. ecuat. pediatr ; 23(2): 146-153, 15 de agosto 2022.
Article in Spanish | LILACS | ID: biblio-1397276


Introducción: El trasplante de células progenitoras hematopoyéticas (TCPH) es el trata-miento para la leucemia aguda en niños, el tipo de cáncer más común en edad pediátrica. El objetivo del presente estudio fue determinar la supervivencia global y libre de enferme-dad en un grupo de pacientes sometidos a TCPH y explorar los factores de riesgo pacientes pediátricos con leucemia aguda. Metodología: El presente estudio observacional incluye a pacientes pediátricos diagnosticados de leucemia mieloide aguda (LMA) o linfoide (LLA), sometidos a TCPH, de 2011 a 2018 presentados en el Hospital Infantil Federico Gómez. Se construyen curvas de Kaplan Meier para la supervivencia global, por subgrupos según tipo de leucemia y estado libre de enfermedad así como un estudio multivariable para medir factores de riesgo. Resultados: Se incluyeron 53 pacientes en el análisis. 5 pacientes (11%) tuvieron falla primaria del injerto. La supervivencia global fue del 28% a los 24 meses. Fallecieron 30 pacientes (67%). La mediana de supervivencia global fue de 11 meses. Para LMA fue de 8.9 meses y para LLA de 12.4 meses. Uno de los factores de riesgo constituyó la edad >10 años al momento del trasplante OR 5.2 (1.07-25.12), P=0.04 y el número de recaídas previas al trasplante OR 4.3 (1.2-15.07) P=0.025. Conclusión: Los pacientes que sobrevivieron un año libre de la enfermedad tenían un mejor pronóstico en general. En estudios relacionados a TCPH no se ha reportado que exista un rango de edad de los receptores de trasplante que esté relacionado a mayor mortalidad, por lo cual es un dato significativo como un factor de riesgo independiente.

Introduction: Hematopoietic stem cell transplantation (HSCT) is the treatment for acute leukemia in children, the most common type of cancer in children. The objective of the present study was to de-termine the overall and disease-free survival in a group of patients undergoing HSCT and to explore the risk factors for pediatric patients with acute leukemia. Methodology: This observational study includes all pediatric patients diagnosed with acute myeloid leukemia (AML) or lymphoid leukemia (ALL), undergoing HSCT from March 2011 to March 2018, presented at the Federico Gómez Children's Hospital. Kaplan Meier curves are constructed for overall survival by subgroups according to the type of leukemia and disease-free status, as well as a multivaria-ble study to measure risk factors. Results: 53 patients were included in the análisis. 5 patients (11%) had primary graft failure. Overall survival was 28% at 24 months. Thirty patients (67%) died. The median overall survival was 11 months. For AML, it was 8.9 months, and for ALL, it was 12.4 months. One of the risk factors was age >10 years at the time of transplant OR 5.2 (1.07-25.12) P=0.04 and the number of relapses prior to transplant OR 4.3 (1.2-15.07) P=0.025. Conclusión: Patients who survived one year free of the disease had a better prognosis. In studies relat-ed to HSCT, it has not been reported that there is an age range of transplant recipients that is related to higher mortality, which is why it is a significant and independent risk factor.

Humans , Child, Preschool , Child , Child , Bone Marrow Transplantation , Bone Marrow , Leukemia, Myeloid, Acute , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Survivorship
São Paulo; s.n; 20220720. 112 p.
Thesis in Portuguese | LILACS, BBO | ID: biblio-1379712


As alterações do paladar durante o transplante de células tronco hematopoiéticas (TCTH) são frequentemente descritas em períodos após a finalização do transplante, porém pouco se conhece sobre quais sabores e que tipo de alterações de percepção são vivenciadas pelos pacientes durante o transplante. O objetivo deste trabalho foi caracterizar as alterações do paladar e de despapilação presentes no período de neutropenia do TCTH, bem como verificar se há associação entre essas alterações e variáveis clínicas relacionadas ao transplante e à toxicidade na mucosa oral. Também se avaliou o impacto das alterações do paladar e da mucosa oral na qualidade de vida dos pacientes. Foram selecionados 52 pacientes de TCTH autólogo e alogênico, que realizaram testes de acuidade do paladar antes do condicionamento e durante a neutropenia. Os pacientes foram ainda submetidos a oroscopia para avaliação de mucosite oral e despapilação no dorso da língua. Foi também realizado inquérito quanto a xerostomia e a alterações no paladar. Foi realizado teste de acuidade na percepção dos sabores doce, amargo, azedo e salgado, ofertados em soluções contendo concentrações baixas e altas de substâncias estimuladoras desses sabores. A qualidade de vida foi avaliada pelo questionário EORTC QLQ-C30 e QLQ-H&N35. Detectou-se que os 46,1% dos pacientes exibiram alterações do paladar antes do condicionamento do TCTH, mas que essa frequência aumentou para 90,5% durante a neutropenia (teste do c2, p=0.042). O tipo de alteração mais comum foi a hipogeusia dos sabores de concentração mais forte, principalmente do amargo. Alta frequência de pacientes (72,0%) foi detectada exibindo despapilação no período de neutropenia, porém essa despapilação não foi associada às alterações de percepção de cada tipo de sabor. Não houve associação entre alterações do paladar e tipo de transplante, tipo de condicionamento e variáveis relacionadas à toxicidade na cavidade oral e no trato gastrointestinal. Houve associação significativa entre despapilação e duração da mucosite oral >=8 dias (OR= 5,62, IC95% = 0,98-60,30, p=0.039). As alterações salivares e do paladar reduziram significativamente na qualidade de vida durante a neutropenia em comparação ao período que antecedeu o condicionamento. Concluiu-se que as alterações do paladar já estão presentes antes do TCTH, mas há aumento da frequência dessas alterações, principalmente de hipogeusia. A despapilação na língua ocorreu após o condicionamento, e foi associada a maior tempo de duração da mucosite oral. O impacto das alterações salivares e do paladar na qualidade de vida do paciente durante a neutropenia é alto e devem ser minimizados mediante a adoção de estratégias mais abrangentes, que incluam manutenção da integridade da mucosa oral.

Taste Buds , Bone Marrow Transplantation , Dysgeusia
Notas enferm. (Córdoba) ; 22(39): 23-32, junio 2022.
Article in Spanish | LILACS, BDENF, BINACIS, UNISALUD | ID: biblio-1380255


El Trasplante de Médula ósea es actualmente, una alternativa en patologías oncológicas, que busca curación y sobrevida del paciente, los cuidados de enfermería en todas las etapas del tratamiento están encaminados a proporcionar atención oportuna y eficaz, con la finalidad de prevenir, tratar y superar complicaciones propias del proceso. La educación de los pacientes y cuidadores favorece a que el paciente aprenda a autocuidarse, a disminuir su ansiedad, a realizar cambios en su comportamiento y estilo de vida y a prevenir la no adherencia al tratamiento. El objetivo fue diseñar un programa educativo sobre los cuidados que el paciente y su familia debe conocer al ingreso y estadía en el servicio de trasplante de Médula Osea. Diseño Metodológico: la búsqueda se efectuó en bases de datos: Pubmed y Google Académico y Scielo. Se analizaron 13 artículos para el desarrollo del trabajo. Resultados: Educar a los pacientes y sus familias sobre el proceso del trasplante de Medula Osea es un gran desafío que necesita de actualización permanente del personal de enfermería. Brindar conocimientos sobre medidas preventivas y pautas que ayuden a sobrellevar este proceso, permitirá tener al paciente como un miembro activo en sus cuidados, disminuyendo su ansiedad y si es necesario realizando modificaciones en su estilo de vida[AU]

Bone Marrow Transplantation is currently an alternative in oncological pathologies, which seeks healing and patient survival, here nursing care at all stages of treatment is aimed at providing timely and effective care, in order to prevent, treat and overcome complications of the process. The education of patients and caregivers favors the patient learning to care for himself, to reduce his anxiety, to make changes in his behavior and lifestyle and to prevent non-adherence to treatment. The objective was to design an educational program on the care that the patient and his family should know during their admission and stay in the Bone Marrow Transplant Service. Methodological design: the search was carried out in the database: Pubmed and Google Scholar and Scielo. 13 articles were analyzed for the development of the work. Results: Educating patients and their families about the bone marrow transplant process is a great challenge that requires permanent updating of the nursing staff. Providing knowledge about preventive measures and guidelines to help cope with this process will allow us to have the patient as an active member in their care, reducing their anxiety and, if necessary, making changes to their lifestyle[AU]

O Transplante de Medula Óssea é atualmente uma alternativa nas patologias oncológicas, que busca a cura e a sobrevivência do paciente, aqui a assistência de enfermagem em todas as etapas do tratamento visa proporcionar uma assistência oportuna e eficaz, | 24a fim de prevenir, tratar e superar complicações do processo. A educação de pacientes e cuidadores favorece que o paciente aprenda a cuidar de si mesmo, a diminuir sua ansiedade, a realizar mudanças em seu comportamento e estilo de vida e a prevenir a não adesão ao tratamento. O objetivo foi elaborar um programa educativo sobre os cuidados que o paciente e sua família devem conhecer durante sua admissão e permanência no serviço de transplante de medula óssea. Desenho metodológico: a busca foi realizada nas bases de dados: Pubmed e Google Acadêmico e Scielo. 13 artigos foram analisados para o desenvolvimento do trabalho. Resultados: Educar os pacientes e seus familiares sobre o processo de transplante de medula óssea é um grande desafio que exige atualização permanente da equipe de enfermagem. Fornecer conhecimento sobre medidas preventivas e orientações para auxiliar no enfrentamento desse processo nos permitirá ter o paciente como um membro ativo em seu cuidado, reduzindo sua ansiedade e, se necessário, realizando mudanças em seu estilo de vida[AU]

Humans , Anxiety , Bone Marrow , Patient Education as Topic , Bone Marrow Transplantation , Life Style , Nursing Care
Curitiba; s.n; 20220602. 150 p. ilus, graf, tab.
Thesis in Portuguese | LILACS, BDENF | ID: biblio-1381154


Resumo: O objetivo desta tese foi avaliar as alterações da qualidade de vida relacionada à saúde dos pacientes adultos com câncer hematológico, submetidos ao transplante de células-tronco hematopoéticas, nos primeiros cinco anos após o procedimento. Trata-se de um estudo quantitativo, longitudinal, observacional e analítico, realizado em hospital público do sul do Brasil, referência na América Latina para esta modalidade de tratamento. Foram incluídos 55 participantes com idade igual ou superior a 18 anos, que se submeteram a esta terapia. A coleta de dados ocorreu de setembro de 2013 a janeiro de 2021, com avaliações em dez etapas: pré-transplante (antes de iniciar o condicionamento), pancitopenia, pré-alta hospitalar, após 100 dias, após 180 dias, Follow up 1 (após 360 dias), e anualmente até completar cinco anos da realização do procedimento. Foram aplicados um instrumento para coleta de dados sociodemográficos e clínicos e os questionários de Qualidade de vida relacionada à saúde Quality of life Questionnaire Core 30 e Functional Assessment of Cancer Therapy - Bone Marrow Transplant, ambos traduzidos, adaptados e validados para o português - Brasil. Em relação ao diagnóstico, as leucemias estão presentes em 65% dos casos; quanto à modalidade de tratamento, o transplante de células-tronco alogênico foi realizado em 71% dos pacientes. No que diz respeito aos óbitos, a causa de maior incidência foi por recidiva da doença (44%), e o maior número ocorreu no primeiro ano (37%). A qualidade de vida global (56,6/100) e geral (90,7/148) apresentou os menores escores na etapa de pancitopenia, com melhores índices no quinto ano, (80,4/100) e (116,1/148), respectivamente. A análise com o modelo linear generalizado misto evidenciou alterações significativas nos escores dos domínios de qualidade de vida relacionada à saúde entre as etapas ao longo do tempo. Foi comprovada a hipótese de que os pacientes com melhores escores nos domínios de qualidade de vida relacionada à saúde observados no início do tratamento têm maior sobrevida. Os resultados do estudo inferem as correlações entre os domínios mensurados e encontram, assim, sustentação no modelo conceitual teórico utilizado. As contribuições consistem em reafirmar a dimensionalidade do constructo qualidade de vida relacionada à saúde, além de agregar conhecimento acerca das alterações autopercebidas pelos pacientes durante o tratamento.

Abstract: The objective of this thesis was to evaluate the changes in the health-related quality of life of adult patients with hematological cancer undergoing hematopoietic stem cell transplantation in the first five years after the procedure. This is a quantitative, longitudinal, observational and analytical study carried out in a public hospital in southern Brazil, a reference in Latin America for this treatment modality. We included 55 participants aged 18 years and over, who underwent this therapy. Data collection took place from September 2013 to January 2021, with evaluations in ten stages: pre-transplantation (before starting conditioning), pancytopenia, pre-hospital discharge, after 100 days, after 180 days, Follow up 1 ( after 360 days), and annually until completing five years of the procedure. An instrument for collecting sociodemographic and clinical data and the Health-related Quality of life Questionnaire Core 30 and Functional Assessment of Cancer Therapy - Bone Marrow Transplant questionnaires were applied, both translated, adapted and validated for Portuguese - Brazil. Regarding diagnosis, leukemias are present in 65% of cases; regarding the treatment modality, allogeneic stem cell transplantation was performed in 71% of the patients. With regard to deaths, the cause of highest incidence was disease recurrence (44%), and the highest number occurred in the first year (37%). The global (56.6/100) and general (90.7/148) quality of life had the lowest scores in the pancytopenia stage, with better rates in the fifth year (80.4/100) and (116.1/148), respectively. The analysis with the mixed generalized linear model showed significant changes in the scores of the health-related quality of life domains between the stages over time. The hypothesis was confirmed that patients with better scores in the domains of health-related quality of life observed at the beginning of treatment have greater survival. The study results infer the correlations between the measured domains and thus find support in the theoretical conceptual model used. The contributions consist of reaffirming the dimensionality of the health-related quality of life construct, in addition to adding knowledge about the self-perceived changes by patients during treatment.

Resumen: El objetivo de esta tesis fue evaluar los cambios en la calidad de vida relacionada con la salud de pacientes adultos con cáncer hematológico, sometidos a trasplante de células madre hematopoyéticas, en los primeros cinco años después del procedimiento. Se trata de un estudio cuantitativo, longitudinal, observacional y analítico realizado en un hospital público del sur de Brasil, referencia en América Latina para esta modalidad de tratamiento. Se incluyeron 55 participantes mayores de 18 años que se sometieron a esta terapia. La recolección de datos ocurrió de septiembre de 2013 a enero de 2021, con evaluaciones en diez etapas: pretrasplante (antes de iniciar el acondicionamiento), pancitopenia, alta prehospitalaria, después de 100 días, después de 180 días, Seguimiento 1 (después de 360 días), y anualmente hasta completar cinco años del procedimiento. Se aplicó un instrumento de recolección de datos sociodemográficos y clínicos y los cuestionarios Health-related Quality of life Questionnaire Core 30 y Functional Assessment of Cancer Therapy - Bone Marrow Transplant, ambos traducidos, adaptados y validados para portugués - Brasil. En cuanto al diagnóstico, las leucemias están presentes en el 65% de los casos; en cuanto a la modalidad de tratamiento, se realizó trasplante alogénico de células madre en el 71% de los pacientes. En cuanto a las defunciones, la causa de mayor incidencia fue la recidiva de la enfermedad (44%) y el mayor número se produjo en el primer año (37%). La calidad de vida global (56,6/100) y general (90,7/148) tuvieron las puntuaciones más bajas en la etapa de pancitopenia, con mejores tasas en el quinto año (80,4/100) y (116,1/148), respectivamente. El análisis con el modelo lineal generalizado mixto mostró cambios significativos en las puntuaciones de los dominios de calidad de vida relacionada con la salud entre las etapas a lo largo del tiempo. Se confirmó la hipótesis de que los pacientes con mejores puntajes en los dominios de calidad de vida relacionada con la salud observados al inicio del tratamiento tienen mayor sobrevida. Los resultados del estudio infieren las correlaciones entre los dominios medidos y así encuentran apoyo en el modelo teórico conceptual utilizado. Los aportes consisten en reafirmar la dimensionalidad del constructo calidad de vida relacionada con la salud, además de sumar conocimientos sobre los cambios autopercibidos por los pacientes durante el tratamiento.

Humans , Male , Female , Adult , Middle Aged , Aged , Quality of Life , Health , Bone Marrow Transplantation , Hematopoietic Stem Cell Transplantation , Habits , Hematologic Diseases
Revista Digital de Postgrado ; 11(1): 327, abr. 2022. ilus
Article in Spanish | LILACS, LIVECS | ID: biblio-1417141


La Aplasia Medular pura de Células Rojas es un trastorno que se caracteriza por anemia con ausencia casi completa de precursores de células rojas en la médula ósea, con contaje de leucocitos y plaquetas normales. La anemia de Diamond-Blackfan es un síndrome de insuficiencia de la médula ósea caracterizada por anemia, reticulocitopenia y disminución de precursores eritroides en la médula ósea. Se presenta el caso de un lactante menor masculino de 2 meses de edad, sin antecedentes familiares ni perinatales de importancia, cuya madre evidencia palidez cutáneo mucosa progresiva asociado a hiporexia; acude a centro de salud donde realizan paraclínicos que reportan hemoglobina en 1,7g/dL. Se realiza frotis de sangre periférica donde se muestra serie eritroide francamente afectada con contaje granulocítico y megacariocítico normales; se realiza biopsia y aspirado de médula ósea concluyéndose aplasia medular de serie roja y en vista de otros hallazgos clínicos, se plantea posible anemia de Diamond-lackfan. Se indica tratamiento con glucocorticoides, sin embargo por respuesta insuficiente, se inicia eritropoyetina aumentando dosis de forma progresiva, a pesar de la administración de la misma, amerita transfusiones sanguíneas de forma regular; se realizan estudios de compatibilidad con familiares de primer grado resultando positivos, actualmente es candidato a trasplante alogénico de médula ósea. Se concluye que a pesar de corresponder a un síndrome poco frecuente, debe sospecharse ante la presencia de anemia severa, sin pérdida sanguínea aguda y descarte previo de otras etiologías, además se plantea que el inicio oportuno del tratamiento es fundamental para la supervivencia de estos pacientes(AU)

Pure red cell aplasia medullary is a disorder characterized by anemia with almost complete absence of red cell precursors in the bone marrow, with leukocyte count and platelets. e Diamond-Blackfan anemia is a failure syndrome characterized by bone marrow anemia, reticulocytopenia and decreased erythroid precursors in the bone marrow. the case of an infant under 2 months of age presented no family or perinatal history major, whose mother progressive skin pallor evidence mucosa associated with hyporexia; go to health center where they perform paraclinical reporting hemoglobin 1.7 g /dL. peripheral blood smear where erythroid frankly affected with normal megakaryocytic granulocytic count shown is made; It biopsied and bone marrow aspirate concluding marrow red cell aplasia; possible anemia Diamond-Blackfan in light of other clinical findings arises. It stays with glucocorticoid treatment, however insufficient response, begins erythropoietin dose progressively increasing, despite it, warrants blood transfusions on a regular basis; compatibility studies performed with firstdegree resulting positive, currently a candidate for allogeneic bone marrow transplantation. It is concluded that despite being a rare syndrome should be suspected in severe anemia where there is acute blood loss, ruling out other etiologies; also timely initiation of treatment is critical to the survival of these patients(AU)

Humans , Male , Infant , Red-Cell Aplasia, Pure , Anemia, Diamond-Blackfan , Anemia , Bone Marrow , Hemoglobins , Bone Marrow Transplantation , Erythropoietin
Chinese Journal of Hematology ; (12): 272-278, 2022.
Article in Chinese | WPRIM | ID: wpr-929635


Objective: To establish an intramedullary transplantation model of primary megakaryocytes to evaluate the platelet-producing capacity of megakaryocytes and explore the underlying regulatory mechanisms. Methods: Donor megakaryocytes from GFP-transgenic mice bone marrow were enriched by magnetic beads. The platelet-producing model was established by intramedullary injection to recipient mice that underwent half-lethal dose irradiation 1 week in advance. Donor-derived megakaryocytes and platelets were detected by immunofluorescence staining and flow cytometry. Results: The proportion of megakaryocytes in the enriched sample for transplantation was 40 to 50 times higher than that in conventional bone marrow. After intramedullary transplantation, donor-derived megakaryocytes successfully implanted in the medullary cavity of the recipient and produce platelets, which showed similar expression of surface markers and morphology to recipient-derived platelets. Conclusion: We successfully established an in vivo platelet-producing model of primary megakaryocytes using magnetic-bead enrichment and intramedullary injection, which objectively reflects the platelet-producing capacity of megakaryocytes in the bone marrow.

Animals , Humans , Mice , Blood Platelets , Bone Marrow , Bone Marrow Cells , Bone Marrow Transplantation , Megakaryocytes/metabolism
Interface (Botucatu, Online) ; 26: e210455, 2022.
Article in Portuguese | LILACS | ID: biblio-1356352


Este artigo é parte inicial de uma pesquisa realizada em um centro de transplante de medula óssea, do Sistema Único de Saúde (SUS), cujos usuários são pessoas em situação de extrema vulnerabilidade. Sua proposta é analisar dimensões que atravessam a prática em saúde e não se reduzem a resultados imediatamente mensuráveis, podendo ser chamadas de dimensões intangíveis do cuidar. O objetivo é refletir sobre o encontro profissional-paciente, em sua relação com o adoecimento e a morte, sob o conceito psicanalítico de inconsciente. Para isso, foi realizado o relato de situações vividas no campo denominadas vinhetas clínicas; e, com base nas articulações teóricas elaboradas, consideram-se os vetores da biopolítica, ética e afeto como axiais à assistência. Conclui com a necessidade de propor perspectivas do cuidar que reconheçam a subjetividade concernida à prática assistencial. (AU)

This article is based on the initial stages of a study undertaken in a public bone marrow transplant center for extremely vulnerable people. We analyze dimensions that cut across health care practice not reduced to immediately measureable results, otherwise called intangible dimensions of care. We reflect on the intersection between health professionals and patients in relation to illness and death drawing on the psychoanalytic concept of the unconscious. To this end, we document situations experienced in the field called clinical vignettes. Drawing on the theoretical framework, we consider the vectors biopolitics, ethics and affection to be core aspects of care. We conclude that there is a need for approaches to care that recognize the subjectivity of health care practice. (AU)

Este artículo es parte inicial de una investigación realizada en un centro de trasplante de médula ósea del Sistema Único de Salud, cuyos usuarios son personas en situaciones de extremada vulnerabilidad. Su propuesta es analizar dimensiones que atraviesan la práctica en salud y no se reducen a resultados inmediatamente mensurables, pudiendo llamarse de dimensiones intangibles del cuidar. El objetivo es reflexionar sobre el encuentro profesional-paciente en su relación con la enfermedad y la muerte, bajo el concepto psicoanalítico de inconsciente. Para ello, se realizó el relato de situaciones vividas en el campo, denominadas viñetas clínicas; y a partir de las articulaciones teóricas elaboradas se consideran los vectores de la biopolítica, ética y afecto como axiales a la asistencia. Se concluye con la necesidad de proponer perspectivas del cuidar que reconozcan la subjetividad relativa a la práctica asistencial. (AU)

Humans , Patient Care Team , Physician-Patient Relations , Professional-Patient Relations , Ethics , Politics , Unified Health System , Bone Marrow Transplantation , Observational Study