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1.
Curitiba; s.n; 20220602. 150 p. ilus, graf, tab.
Thesis in Portuguese | LILACS, BDENF | ID: biblio-1381154

ABSTRACT

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.


Subject(s)
Humans , Male , Female , Adult , Middle Aged , Aged , Quality of Life , Health , Bone Marrow Transplantation , Hematopoietic Stem Cell Transplantation , Habits , Hematologic Diseases
2.
Hematol., Transfus. Cell Ther. (Impr.) ; 44(2): 163-168, Apr.-June 2022. tab, graf
Article in English | LILACS | ID: biblio-1385054

ABSTRACT

Abstract Introduction Hemorrhagic cystitis (HC) is a common complication of haploidentical hematopoietic stem cell transplantation (haplo-HSCT), characterized by irritative symptoms of the urinary tract and a higher morbidity and mortality rate. The worldwide incidence is reported between 10% and 70%. The use of alkylating agents and BK viral infection are the most frequent etiologies. The aim of this study was to report the HC incidence in an outpatient haplo-HCST program with a reduced intensity-conditioning (RIC) regimen, cataloguing risk factors, complications and final outcomes. Methods The medical database of patients who received a haplo-HSCT between January 2012 and November 2017 was retrospectively analyzed. Demographic variables, general characteristics and HC incidence were included. Results One hundred and eleven patients were included, 30 (27%) of whom developed HC, most of them (70%) being grade II, with a 30-day (7-149) median time of post-transplant HC onset. The BK virus was detected in 71% of the urine samples analyzed. All HC patients responded to treatment, except two (6.6%), who died due to HC complications. Conclusions There was no difference in the HC incidence or severity, compared to that reported when performing haplo-HSCT in hospitalized patients, although the donor-recipient sex mismatch did relate to a higher HC incidence.


Subject(s)
Humans , Male , Female , Child, Preschool , Child , Adolescent , Adult , Middle Aged , Young Adult , BK Virus , Hematopoietic Stem Cell Transplantation , Cystitis , Transplantation, Haploidentical , Incidence , Cyclophosphamide
3.
Article in English | WPRIM | ID: wpr-929069

ABSTRACT

Antibody-mediated rejection (AMR) is one of the major causes of graft loss after transplantation. Recently, the regulation of B cell differentiation and the prevention of donor-specific antibody (DSA) production have gained increased attention in transplant research. Herein, we established a secondary allogeneic in vivo skin transplant model to study the effects of romidepsin (FK228) on DSA. The survival of grafted skins was monitored daily. The serum levels of DSA and the number of relevant immunocytes in the recipient spleens were evaluated by flow cytometry. Then, we isolated and purified B cells from B6 mouse spleens in vitro by magnetic bead sorting. The B cells were cultured with interleukin-4 (IL-4) and anti-clusters of differentiation 40 (CD40) antibody with or without FK228 treatment. The immunoglobulin G1 (IgG1) and IgM levels in the supernatant were evaluated by enzyme-linked immunosorbent assay (ELISA). Quantitative reverse transcription-polymerase chain reaction (RT-qPCR) and western blotting were conducted to determine the corresponding levels of messenger RNA (mRNA) and protein expression in cultured cells and the recipient spleens. The results showed that FK228 significantly improved the survival of allogeneic skin grafts. Moreover, FK228 inhibited DSA production in the serum along with the suppression of histone deacetylase 1 (HADC1) and HDAC2 and the upregulation of the acetylation of histones H2A and H3. It also inhibited the differentiation of B cells to plasma cells, decreased the transcription of positive regulatory domain-containing 1 (Prdm1) and X-box-binding protein 1 (Xbp1), and decreased the expression of phosphorylated inositol-requiring enzyme 1 α (p-IRE1α), XBP1, and B lymphocyte-induced maturation protein-1 (Blimp-1). In conclusion, FK228 could decrease the production of antibodies by B cells via inhibition of the IRE1α-XBP1 signaling pathway. Thus, FK228 is considered as a promising therapeutic agent for the clinical treatment of AMR.


Subject(s)
Animals , Depsipeptides , Endoribonucleases , Hematopoietic Stem Cell Transplantation , Histone Deacetylase Inhibitors/pharmacology , Mice , Protein Serine-Threonine Kinases , Skin Transplantation
4.
Acta Paul. Enferm. (Online) ; 35: eAPE03502, 2022. tab, graf
Article in Portuguese | LILACS, BDENF | ID: biblio-1364238

ABSTRACT

Resumo Objetivo Construir e validar uma tecnologia informativa para famílias de crianças/adolescentes com câncer submetidos ao transplante de células-tronco hematopoiéticas. Métodos Estudo metodológico fundamentado no Modelo do Cuidado Centrado no Paciente e Família e User-Centered Design, realizado em quatro etapas: avaliação das necessidades de informações; construção teórica e desenvolvimento do aplicativo; validação de conteúdo e semântica pelo comitê de juízes e população-alvo; avaliação da usabilidade por experts em informática. Os participantes do estudo foram profissionais e famílias de crianças com câncer. Para a validação de conteúdo e semântica, estabeleceu-se percentual de concordância entre juízes de 80% e Índice de Validade de Conteúdo de 0,8. Para a avaliação da usabilidade foram utilizadas as heurísticas de Nielsen. Os dados foram analisados por meio de estatística descritiva. Resultados Após identificação das necessidades de informações da família, na literatura e no estudo de campo, e construção do aplicativo, este foi validado por especialistas, obtendo percentual de concordância de 87% e índice de validade de conteúdo de 0,87; e pela população-alvo, de 98% e 0,98, respectivamente. Quanto a usabilidade, atingiu o grau de severidade simples. O aplicativo móvel TMO-App apresenta 268 telas e 95 ilustrações, contemplando as necessidades de informações da família antes, durante e após o transplante. Conclusão Os métodos utilizados para o desenvolvimento e validação mostraram-se satisfatórios para atingir os objetivos propostos. O aplicativo construído é confiável, de fácil uso, útil, completo e adequado. O estudo avança na proposição de nova estratégia informativa para promover o empoderamento da família em situação de doença crônica.


Resumen Objetivo Elaborar y validar una tecnología informativa para familias de niños/adolescentes con cáncer sometidos al trasplante de células madre hematopoyéticas. Métodos Estudio metodológico fundamentado en el Modelo del Cuidado Centrado en el Paciente y la Familia y User-Centered Design, realizado en cuatro etapas: evaluación de las necesidades de información, construcción teórica y desarrollo de la aplicación, validación semántica y del contenido por el comité de jueces y el público destinatario, evaluación de usabilidad por especialistas en informática. Los participantes del estudio fueron profesionales y familias de niños con cáncer. Para la validación semántica y de contenido, se estableció un porcentaje de concordancia entre los jueces del 80 % y un Índice de Validez de Contenido de 0,8. Para la evaluación de usabilidad se utilizaron las heurísticas de Nielsen. Los datos fueron analizados mediante estadística descriptiva. Resultados Después de identificar las necesidades de información de la familia, en la literatura y en el estudio de campo, y de elaborar la aplicación, esta fue validada por especialistas y obtuvo un porcentaje de concordancia del 87 % y un Índice de Validez de Contenido de 0,87. A su vez, los resultados del público destinatario fueron 98 % y 0,98, respectivamente. Respecto a la usabilidad, alcanzó un nivel de severidad simple. La aplicación móvil TMO-App presenta 268 pantallas y 95 ilustraciones que contemplan las necesidades de información de la familia antes, durante y después del trasplante. Conclusión Los métodos utilizados para el desarrollo y validación demostraron ser satisfactorios para alcanzar los objetivos propuestos. La aplicación elaborada es confiable, de fácil uso, útil, completa y adecuada. El estudio avanza en la propuesta de una nueva estrategia informativa para promover el empoderamiento de familias en situación de enfermedad crónica.


Abstract Objective To develop and validate an informative technology for families of children/adolescents with cancer undergoing hematopoietic stem cell transplantation. Methods A methodological study grounded on the Patient-and Family-Centered Care Model and User-Centered Design, conducted in four stages: assessment of information needs; theoretical construction and development of the mobile application; content and semantic validation by a committee of experts and target population; usability evaluation by computer experts. The participants of the study were professionals and families of children with cancer. For content and semantic validation, an inter-rater agreement percentage of 80% and a Content Validity Index of 0.8 were established. For usability evaluation, Nielsen's heuristics were used. The data were analyzed using descriptive statistics. Results After the information about the family's needs was obtained from the published literature and field study, the application was developed and validated by experts. Agreement of 87%, and a content validity index of 0.87, were achieved with the expert group; 98% and 0.98, respectively, with the target population. With regard to usability, the level of simple severity was reached. The BMT-App mobile application contains 268 screens and 95 illustrations, addressing the family's information needs before, during, and after transplantation. Conclusion The methods used for development and validation were satisfactory to achieve the intended objectives. The application constructed is reliable, easy to use, useful, complete, and adequate. The study advances the proposition of a new informative strategy to promote empowerment of the family confronting chronic disease.


Subject(s)
Humans , Male , Female , Child , Adolescent , Adult , Middle Aged , Hematopoietic Stem Cell Transplantation , Mobile Applications , Neoplasms , Family/psychology , Epidemiology, Descriptive , Patient-Centered Care , Evaluation Studies as Topic
5.
Rev. latinoam. enferm. (Online) ; 30: e3569, 2022. tab, graf
Article in Portuguese | LILACS, BDENF | ID: biblio-1376959

ABSTRACT

Resumo Objetivo: analisar os fatores associados ao insucesso do Transplante de Células-Tronco Hematopoiéticas (TCTH) em pacientes submetidos ao retransplante de Células-Tronco Hematopoiéticas (RCTH). Método: estudo quantitativo do tipo caso-controle para avaliar pacientes submetidos ao RCTH. Para tanto, utilizou-se amostra pareada de dois controles para cada caso (2:1). O grupo caso foi constituído pelos prontuários de saúde com todos os pacientes que foram submetidos ao RCTH (28) e o grupo controle (56) incluiu pacientes que receberam apenas um transplante. Três variáveis nortearam o pareamento: sexo, diagnóstico e tipo de transplante. Resultados: vinte e quatro (85,71%) pacientes do grupo caso receberam retransplante devido a recidiva da doença e quatro (14.29%) devido a falha do enxerto. Uma diferença estatística foi encontrada na análise entre os pacientes que não usaram o ácido ursodesoxicólico, analgésicos opioides ou imunossupressores. A necessidade de um RCTH entre aqueles que usaram estes medicamentos de forma inapropriada foi 16,12, 12,79 e 4,5 vezes maior, respectivamente, do que entre os que as usaram corretamente. Conclusão: houve uma diferença relacionada ao motivo que levou ao retransplante e os indivíduos analisados. A conclusão é que a razão preditiva para retransplante nesta amostra foi a recidiva da doença.


Abstract Objective: to analyze the factors associated with the failure of Hematopoietic Stem Cell Transplantation (HSCT) in patients undergoing Hematopoietic Stem Cell Retransplantation (HSCR). Method: this study implemented a quantitative approach and was a case-control type which addressed patients undergoing HSCR. To do so, a paired sample of two controls was used for each case (2:1). The case group consisted of the medical records of all patients who underwent HSCR (28) and the control group (56) of those who underwent only one transplant. Three variables guided the pairing: gender, diagnosis and type of transplant. Results: a total of 24 (85.71%) patients in the case group were re-transplanted due to disease relapse and four (14.29%) due to graft failure. There was a statistical difference in the analysis between patients who did not use ursodeoxycholic acid, opioid analgesics and immunosuppressants. The need for HSCR among those who used these medications inappropriately was 16.12, 12.79 and 4.5 times more likely, respectively, than those who used them correctly. Conclusion: there was a difference regarding the reasons which led to the retransplantation and the analyzed subjects, and this study concluded that the predictive reason for retransplantation in the studied sample was disease relapse.


Resumen Objetivo: analizar los factores asociados con el fracaso del Trasplante de Células Madre Hematopoyéticas (TCMH) en pacientes sometidos al Retrasplante de Células Madre Hematopoyéticas (RCMH). Método: estudio cuantitativo de tipo caso-control que abordó pacientes sometidos al RCMH. Para esto, se utilizó una muestra pareada de dos controles para cada caso (2:1). El grupo caso estuvo formado por los registros médicos de todos los pacientes que fueron sometidos al RCMH (28) y el grupo control (56) por los que fueron sometidos a un solo trasplante. Tres variables guiaron el emparejamiento: género, diagnóstico y tipo de trasplante. Resultados: un total de 24 (85.71%) pacientes en el grupo caso fueron retransplantados debido a la recaída de la enfermedad y 4 (14.29%) por el fracaso del injerto. Hubo una diferencia estadística en el análisis entre los pacientes que no usaron ácido ursodesoxicólico, analgésicos opioides e inmunosupresores. La necesidad de RCMH entre los que usaron estos medicamentos de manera inapropiada se encontraba 16,12 - 12,79 y 4,5 veces más probable, respectivamente, que aquellos que los usaron correctamente. Conclusión: hubo diferencia en cuanto a las razones que llevaron al retrasplante de los sujetos analizados. Este estudio concluyó que la razón predictiva del retrasplante, en la muestra estudiada, fue la recidiva de la enfermedad.


Subject(s)
Humans , Male , Female , Child, Preschool , Child , Adolescent , Adult , Middle Aged , Recurrence , Reoperation , Hematopoietic Stem Cells , Retrospective Studies , Hematopoietic Stem Cell Transplantation
6.
Rev. Bras. Cancerol. (Online) ; 68(1)jan./fev./mar. 2022.
Article in Portuguese | LILACS | ID: biblio-1370283

ABSTRACT

Introdução: Pacientes com câncer hematológico, candidatos a transplante de células-tronco hematopoiéticas (TCTH), requerem um autocuidado extenso, com tratamentos complexos e comportamentos de saúde adequados que são considerados fator-chave. Frente a essas complexidades, surge a importância do letramento em saúde. Objetivo: Descrever o nível de letramento e avaliar as inter-relações existentes entre letramento em saúde, estado nutricional, condições sociodemográficas e clínicas nos pacientes com câncer hematológicos e candidatos a TCTH em um hospital universitário terciário de Fortaleza-CE. Método: Estudo de caráter transversal realizado com 69 pacientes candidatos a TCHT, atendidos nesse hospital, que responderam à versão validada e traduzida do Newest Vital Sign (NVS-Br). Os dados clínicos e sociodemográficos foram coletados por meio da análise dos prontuários. O estado nutricional foi avaliado pelo índice de massa corporal (IMC), circunferência da cintura (CC) e CC associada à relação cintura-quadril (RCQ). Resultados: O letramento inadequado foi encontrado em 78,3% dos pacientes. Os fatores foram associados à idade (p=0,024), à baixa renda (p=0,005) e a menos anos de estudo (p=0,047). Conclusão: O estudo aponta para a necessidade de se melhorar o grau de letramento em saúde dos pacientes, a fim de obter melhores resultados no tratamento, principalmente naqueles que apresentam idade mais avançada, baixa renda e menor escolaridade


Introduction: Patients with hematologic cancer candidates for hematopoietic stem cell transplantation (HSCT) require extensive selfcare, with complex treatments and appropriate health behaviors that are considered a key factor. The importance of health literacy emerges because of these complexities. Objective: To describe the level of literacy and assess the interrelationships between health literacy, nutritional status, clinical and sociodemographic conditions in patients with hematologic cancer and candidates for HSCT in a tertiary university hospital in Fortaleza-CE. Method: Cross-sectional study conducted with 69 HSCT candidates, treated at a university hospital, who responded to the validated and translated version of the Newest Vital Sign (NVS-Br). Clinical and sociodemographic data were collected through the analysis of medical records. Nutritional status was assessed using body mass index (BMI), waist circumference (WC) and WC associated with hip circumference (WH). Results: Inadequate literacy was found in 78.3% of patients. Factors were associated with age (p=0.024), low income (p=0.005) and less years of education (p=0.047). Conclusion: The results indicated the necessity to improve the level of health literacy of patients for better treatment results, especially in older adults with low income and poor education level


Introducción: Los pacientes con cáncer hematológico candidatos a trasplante de células madre hematopoyéticas (TCMH) requieren un autocuidado extenso, con tratamientos complejos y conductas de salud adecuadas que se consideran un factor clave. Frente a estas complejidades, surge la importancia de la alfabetización en salud. Objetivo: Describir el nivel de alfabetización y evaluar las interrelaciones entre alfabetización en salud, estado nutricional, datos clínicos y condiciones sociodemográficas en pacientes con cáncer hematológico candidatos a TCMH en un hospital universitario terciario de Fortaleza-CE. Método: Estudio transversal realizado con 69 candidatos a TCMH, atendidos en este hospital, que respondieron a la versión validada y traducida del Newest Vital Sign (NVSBr). Los datos clínicos y datos sociodemográficos se recolectaron mediante el análisis de historias clínicas. El estado nutricional se evaluó mediante el indice de masa corporal (IMC), la circunferencia de la cintura (CC) y la CC asociada con la circunferencia de la cadera (CCR). Resultados: Se encontró alfabetización inadecuada en el 78,3% de los pacientes. Los factores se asociaron con la edad (p=0,024), bajos ingresos (p=0,005) y menos años de educación (p=0,047). Conclusión: Este estudio apunta a la necesidad de mejorar el nivel de alfabetización en salud de los pacientes con el fin de obtener mejores resultados de tratamiento, especialmente en aquellos que son mayores, tienen bajos ingresos y tienen menos educación


Subject(s)
Humans , Male , Female , Leukemia , Nutritional Status , Hematopoietic Stem Cell Transplantation , Health Literacy , Hematology
7.
Chinese Journal of Pediatrics ; (12): 323-328, 2022.
Article in Chinese | WPRIM | ID: wpr-935695

ABSTRACT

Objective: To investigate the characteristics, risk factors and outcomes of thalassemia major (TM) children with pericardial effusion (PE) after allo-geneic hematopoietic stem cell transplantation (allo-HSCT). Methods: Clinical data of 446 TM children received allo-HSCT at Shenzhen Children's Hospital between January 2012 and December 2020 were analyzed retrospectively. Patients were divided into PE and non-PE group according to the occurrence of PE. Chi-square tests were used to investigate the risk factors that were associated with the development of PE. Kaplan-Meier method was used for survival analysis of the 2 groups. Results: Twenty-five out of 446 patients (5.6%) developed PE at a time of 75.0 (66.5, 112.5) days after allo-HSCT. Among these patients, 22 cases (88.0%) had PE within 6 months after allo-HSCT and 19 patients (76.0%) had PE within 100 days after allo-HSCT. The diagnoses of PE were confirmed using echocardiography. Pericardial tamponade was observed in only 1 patient, who later undergone emergency pericardiocentesis. The rest of patients received conservative managements alone. PE disappeared in all patients after treatment. Risk factors that were associated with the development of PE after allo-HSCT included the gender of patients, the type of transplantation, the number of mononuclear cells (MNC) infuse, pulmonary infection after HSCT and transplantation associated thrombotic microangiopathy (TA-TMA) (χ²=3.99, 10.20, 14.18, 36.24, 15.03, all P<0.05). In 239 patients that received haploidentical HSCT, the development of PE was associated with the gender of patients, pulmonary infection after HSCT and TA-TMA (χ²=4.48, 20.89, 12.70, all P<0.05). The overall survival rates of PE and non-PE groups were 96.0% (24/25) and 98.6% (415/421). The development of PE was not associated with the overall survival of TM children after allo-HSCT (χ²=1.73, P=0.188). Conclusions: PE mainly develop within 100 days after allo-HSCT in pediatric TM recipients. Haploidentical grafts, female gender, pulmonary infection after HSCT and TA-TMA are the main risk factors associated with PE development after transplant. However, the presence of PE don't have a significant impact on the outcomes of pediatric TM patients after allo-HSCT.


Subject(s)
Child , Female , Hematopoietic Stem Cell Transplantation/adverse effects , Humans , Pericardial Effusion/etiology , Retrospective Studies , Risk Factors , Thrombotic Microangiopathies/complications , beta-Thalassemia/therapy
8.
Chinese Journal of Hematology ; (12): 287-292, 2022.
Article in Chinese | WPRIM | ID: wpr-929637

ABSTRACT

Objective: To evaluate the efficacy and toxicity profiles of idarubicin, cytarabine, and cyclophosphamide (IAC) in relapse/refractory acute myeloid leukemia (AML) . Methods: This study was a prospective, randomized controlled clinical trial with the registration number NCT02937662. The patients were randomly divided into two groups. The experimental group was treated with an IAC regimen, and the regimen of the control group was selected by doctors according to medication experience. After salvage chemotherapy, allogeneic hematopoietic stem cell transplantation (allo-HSCT) was conducted as far as possible according to the situation of the patients. We aimed to observe the efficacy, safety, and toxicity of the IAC regimen in relapse/refractory AML and to explore which is the better regimen. Results: Forty-two patients were enrolled in the clinical trial, with a median age of 36 years (IAC group, 22 cases and control groups, 20 cases) . ①The objective response rate was 71.4% in the IAC group and 40.0% in the control group (P=0.062) ; the complete remission (CR) rate was 66.7% in the IAC group and 40.0% in the control group (P=0.121) . The median follow-up time of surviving patients was 10.5 (range:1.7-32.8) months; the median overall survival (OS) was 14.1 (range: 0.6-49.1) months in the IAC group and 9.9 (range: 2.0-53.8) months in the control group (P=0.305) . The 1-year OS was 54.5% (95%CI 33.7%-75.3%) in the IAC group and 48.2% (95%CI 25.9%-70.5%) in the control group (P=0.305) , with no significant difference between these two regimens. ②The main hematologic adverse events (AEs) were anemia, thrombocytopenia, and neutropenia. The incidence of grade 3-4 hematologic AEs in the two groups was 100% (22/22) in the IAC group and 95% (19/20) in the control group. The median time of neutropenia after chemotherapy in the IAC group and control group was 20 (IQR: 8-30) and 14 (IQR: 5-50) days, respectively (P=0.023) . ③The CR rate of the early relapse (relapse within 12 months) group was 46.7% and that of the late relapse (relapse after 12 months) group was 72.7% (P=0.17) . The median OS time of early recurrence was 9.9 (range:1.7-53.8) months, and that of late recurrence patients was 19.3 (range: 0.6-40.8) months (P=0.420) , with no significant differences between the two groups. The 1-year OS rates were 45.3% (95%CI 27.2%-63.3%) and 66.7% (95%CI 40.0%-93.4%) , respectively (P=0.420) . Survival analysis showed that the 1-year OS rates of the hematopoietic stem cell transplantation group and non-hematopoietic stem cell transplantation group were 87.5% (95%CI 71.2%-100%) and 6.3% (95%CI 5.7%-18.3%) , respectively. The OS rate of the hematopoietic stem cell transplantation group was significantly higher than that of the non-hematopoietic stem cell transplantation group (P<0.001) . Conclusion: The IAC regimen is a well-tolerated and effective regimen in relapsed/refractory AML; this regimen had similar efficacy and safety with the regimen selected according to the doctor's experience for treating relapsed/refractory AML. For relapsed/refractory patients with AML, allogeneic hematopoietic stem cell transplantation should be attempted as soon as possible to achieve long-term survival.


Subject(s)
Adult , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Cyclophosphamide/therapeutic use , Cytarabine/therapeutic use , Hematopoietic Stem Cell Transplantation , Humans , Idarubicin/therapeutic use , Leukemia, Myeloid, Acute/drug therapy , Neutropenia , Prospective Studies , Recurrence , Retrospective Studies
9.
Chinese Journal of Hematology ; (12): 408-413, 2022.
Article in Chinese | WPRIM | ID: wpr-929628

ABSTRACT

Objective: To investigate the influence of the number of high-risk cytogenetic abnormalities (HRCA) on the clinical characteristics and prognosis of patients with newly diagnosed multiple myeloma (MM) . Methods: A total of 360 patients with newly diagnosed MM admitted to Jiangsu Province Hospital between November 2013 and September 2020 were included in this study. Cytoplasmic light chain immunofluorescence with fluorescence in situ hybridization (cIg-FISH) was used to detect HRCA. Cytogenetic abnormalities were combined with clinical characteristics and outcomes for further analysis. Results: Among the 360 patients, 120 patients (33.3%) presented with no HRCAs, and 175 (48.6%) , 61 (16.9%) , and four (1.1%) patients had one, two, and three HRCA (s) , respectively. Patients were divided into three groups, including the no-HRCA group, one-HRCA group, and ≥two-HRCA group, according to the number of HRCAs. There were significant differences in the R-ISS stage, hemoglobin level, albumin level, and the proportion of bone marrow plasma cells among the three groups (P<0.05) . The COX proportional-hazards model identified extramedullary disease (P=0.018) , HRCA ≥ 2 (P=0.001) , and absence of autologous hematopoietic stem cell transplantation (P<0.001) as independent risk factors for progression free survival (PFS) and identified lactate dehydrogenase (LDH) level ≥ 220 U/L (P<0.001) , HRCA ≥2 (P=0.001) , and absence of autologous hematopoietic stem cell transplantation (P=0.005) as independent risk factors for overall survival (OS) . The median PFS was 28 months, 22 months, and 14 months (P=0.005) for the three cohorts, and their OS was not reached,60 months, and 30 months (P=0.001) , respectively. Conclusions: HRCA ≥ 2 is an independent risk factor for decreased survival in patients with newly diagnosed MM. More HRCAs result in heavier tumor burden, as well as a higher risk of disease progression and death.


Subject(s)
Chromosome Aberrations , Hematopoietic Stem Cell Transplantation , Humans , In Situ Hybridization, Fluorescence , Multiple Myeloma/genetics , Prognosis , Retrospective Studies , Transplantation, Autologous
10.
Chinese Journal of Hematology ; (12): 400-407, 2022.
Article in Chinese | WPRIM | ID: wpr-929627

ABSTRACT

Objective: To explore the differences in the biological effects of different expansion systems on natural killer (NK) cells, as well as the safety and preliminary clinical efficacy in the treatment of patients with recurrence after allogeneic hematopoietic stem cell transplantation (allo-HSCT) . Methods: Peripheral blood cells from healthy donors were stimulated with either CD3 combined with CD52 or K562 feeder cells loaded with IL-21/4-1BB to induce NK cell expansion. Changes in the NK cell phenotype, cytokine secretion, and cytotoxicity before and after expansion were detected. We also evaluated the safety and clinical efficacy of two different expansion strategies for patients received NK infusion. Results: Compared with the CD3/CD52 monoclonal antibody amplification system, the feeder cell expansion group had a higher purity of NK cells and higher expression ratios of NK cell surface activation receptors such as DNAM-1 and NKp30, while inhibitory receptor CTLA-4 expression was low and NKG2D/CD25/CD69/ Trail/PD-1/TIM-3/TIGIT had no statistically significant differences between the groups. Further functional results showed that the expression level of KI67 in NK cells after expansion in the two groups increased significantly, especially in the feeder cell expansion group. Simultaneously, the perforin and granzyme B levels of NK cells in the feeder cell expansion group were significantly higher than in the CD3/CD52 expansion group. A retrospective analysis of eight patients who received monoclonal antibody-expanded NK cell reinfusion and nine patients with trophoblast cell-expanded NK cell reinfusion was done. The disease characteristics of the two groups were comparable, NK cell reinfusion was safe, and there were no obvious adverse reactions. Clinical prognostic results showed that in the CD3/CD52 monoclonal antibody amplification group, the MRD conversion rate was 50% (2/4) , and the feeder cell expansion group was 50% (3/6) . After 5 years of follow-up from allo-HSCT, three patients in the monoclonal antibody expansion group had long-term survival without leukemia, and the remaining five patients had died; two patients died in the feeder cell expansion group, and the other six patients had long-term survival. Six cases had GVHD before NK cell reinfusion, and GVHD did not aggravate or even relieved after NK cell reinfusion. Conclusions: Preliminary results show that the biological characteristics of NK cells with diverse expansion strategies are significantly different, which may affect the clinical prognosis of patients with recurrence or persistent minimal residual disease after HSCT. The two groups of patients treated with NK cells from different expansion strategies had no obvious adverse reactions after NK cell infusion, but efficacy still needs to be further confirmed.


Subject(s)
Antibodies, Monoclonal/pharmacology , Graft vs Host Disease/metabolism , Hematopoietic Stem Cell Transplantation , Humans , Killer Cells, Natural , Retrospective Studies , Treatment Outcome
11.
Chinese Journal of Hematology ; (12): 247-254, 2022.
Article in Chinese | WPRIM | ID: wpr-929565

ABSTRACT

Objective: This study aimed to explore the prognostic value of the revised international prognostic scoring system (IPSS-R) and the WHO prognostic scoring system (WPSS) in patients with myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Methods: The clinical data of 184 patients with MDS who received allo-HSCT from July 2016 to June 2019 were retrospectively analyzed. IPSS-R and WPSS were performed at diagnosis and before transplantation. The prognostic values of IPSS-R and WPSS and potential risk factors were explored. Results: With a median follow-up of 21.9 (0.5-47.5) months, the two-year overall survival (OS) and progression-free survival (PFS) rates were (75.1±3.4)% and (71.6±3.6)% , respectively. The two-year cumulative relapse rate and nonrelapse mortality rate were (11.9±0.1)% and (16.5±0.1)% , respectively. There were no significant differences in OS and PFS between the IPSS-R ≤3.5 and >3.5 groups at diagnosis (P=0.409; P=0.724). No significant differences in OS and PFS between the WPSS ≤2 and >2 groups (P=0.426; P=0.726) were observed as well. When the patients were reevaluated before transplantation, the OS and PFS of the IPSS-R ≤3.5 group were significantly better than >3.5 group [OS: (88.6±4.1)% vs (65.8±5.3)% , P=0.003; PFS: (87.6±4.2)% vs (60.5±5.8)% , P=0.002]. However, there were no significant differences in OS and PFS among the WPSS ≤2 and >2 groups (P=0.584; P=0.565). In addition, the OS and PFS of the improved group based on IPSS-R were significantly better than those of the unimproved group before transplantation [OS: (83.8±4.6)% vs (69.3±5.8)% , P=0.027; PFS: (82.8±4.4)% vs. (64.0±7.2)% , P=0.006]. Multivariate analysis indicated that a pretransplant IPSS-R of >3.5 (P=0.021, HR=2.510, 95% CI 1.151-5.476) and TP53 mutation (P=0.047, HR=2.460, 95% CI 1.014-5.971) were independent risk factors for OS, whereas a pretransplant IPSS-R of >3.5 (P=0.017, HR=2.457, 95% CI 1.175-5.141) and pretransplant cytogenetic poor and very poor (P=0.008, HR=2.765, 95% CI 1.305-5.856) were independent risk factors for PFS. Conclusion: A pretransplantation evaluation of IPSS-R could help determine the prognosis of patients with MDS undergoing allo-HSCT. In addition, patients with improved IPSS-R scores before undergoing allo-HSCT had a better prognosis.


Subject(s)
Hematopoietic Stem Cell Transplantation , Humans , Myelodysplastic Syndromes/therapy , Prognosis , Retrospective Studies , Risk Factors
12.
Chinese Journal of Hematology ; (12): 221-228, 2022.
Article in Chinese | WPRIM | ID: wpr-929561

ABSTRACT

Objective: To investigate whether haplotype hematopoietic stem cell transplantation (haplo-HSCT) is effective in the treatment of pre transplant minimal residual disease (Pre-MRD) positive acute B lymphoblastic leukemia (B-ALL) compared with HLA- matched sibling donor transplantation (MSDT) . Methods: A total of 998 patients with B-ALL in complete remission pre-HSCT who either received haplo-HSCT (n=788) or underwent MSDT (n=210) were retrospectively analyzed. The pre-transplantation leukemia burden was evaluated according to Pre-MRD determinedusing multiparameter flow cytometry (MFC) . Results: Of these patients, 997 (99.9% ) achieved sustained, full donor chimerism. The 100-day cumulative incidences of neutrophil engraftment, platelet engraftment, and grades Ⅱ-Ⅳ acute graft-versus-host disease (GVHD) were 99.9% (997/998) , 95.3% (951/998) , and 26.6% (95% CI 23.8% -29.4% ) , respectively. The 3-year cumulative incidence of total chronic GVHD was 49.1% (95% CI 45.7% -52.4% ) . The 3-year cumulative incidence of relapse (CIR) and non-relapse mortality (NRM) of the 998 cases were 17.3% (95% CI 15.0% -19.7% ) and 13.8% (95% CI 11.6% -16.0% ) , respectively. The 3-year probabilities of leukemia-free survival (LFS) and overall survival (OS) were 69.1% (95% CI 66.1% -72.1% ) and 73.0% (95% CI 70.2% -75.8% ) , respectively. In the total patient group, cases with positive Pre-MRD (n=282) experienced significantly higher CIR than that of subjects with negative Pre-MRD [n=716, 31.6% (95% CI 25.8% -37.5% ) vs 14.3% (95% CI 11.4% -17.2% ) , P<0.001]. For patients in the positive Pre-MRD subgroup, cases treated with haplo-HSCT (n=219) had a lower 3-year CIR than that of cases who underwent MSDT [n=63, 27.2% (95% CI 21.0% -33.4% ) vs 47.0% (95% CI 33.8% -60.2% ) , P=0.002]. The total 998 cases were classified as five subgroups, including cases with negative Pre-MRD group (n=716) , cases with Pre-MRD<0.01% group (n=46) , cases with Pre-MRD 0.01% -<0.1% group (n=117) , cases with Pre-MRD 0.1% -<1% group (n=87) , and cases with Pre-MRD≥1% group (n=32) . For subjects in the Pre-MRD<0.01% group, haplo-HSCT (n=40) had a lower CIR than that of MSDT [n=6, 10.0% (95% CI 0.4% -19.6% ) vs 32.3% (95% CI 0% -69.9% ) , P=0.017]. For patients in the Pre-MRD 0.01% -<0.1% group, haplo-HSCT (n=81) also had a lower 3-year CIR than that of MSDT [n=36, 20.4% (95% CI 10.4% -30.4% ) vs 47.0% (95% CI 29.2% -64.8% ) , P=0.004]. In the other three subgroups, the 3-year CIR was comparable between patients who underwent haplo-HSCT and those received MSDT. A subgroup analysis of patients with Pre-MRD<0.1% (n=163) was performed, the results showed that cases received haplo-HSCT (n=121) experienced lower 3-year CIR [16.0% (95% CI 9.4% -22.7% ) vs 40.5% (95% CI 25.2% -55.8% ) , P<0.001], better 3-year LFS [78.2% (95% CI 70.6% -85.8% ) vs 47.6% (95% CI 32.2% -63.0% ) , P<0.001] and OS [80.5% (95% CI 73.1% -87.9% ) vs 54.6% (95% CI 39.2% -70.0% ) , P<0.001] than those of MSDT (n=42) , but comparable in 3-year NRM [5.8% (95% CI 1.6% -10.0% ) vs 11.9% (95% CI 2.0% -21.8% ) , P=0.188]. Multivariate analysis showed that haplo-HSCT was associated with lower CIR (HR=0.248, 95% CI 0.131-0.472, P<0.001) , and superior LFS (HR=0.275, 95% CI 0.157-0.483, P<0.001) and OS (HR=0.286, 95% CI 0.159-0.513, P<0.001) . Conclusion: Haplo HSCT has a survival advantage over MSDT in the treatment of B-ALL patients with pre MRD<0.1% .


Subject(s)
B-Lymphocytes , Graft vs Host Disease , HLA Antigens/genetics , Haplotypes , Hematopoietic Stem Cell Transplantation/adverse effects , Humans , Leukemia, B-Cell/complications , Leukemia, Lymphocytic, Chronic, B-Cell/complications , Neoplasm, Residual , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Recurrence , Retrospective Studies , Siblings
13.
Chinese Journal of Hematology ; (12): 215-220, 2022.
Article in Chinese | WPRIM | ID: wpr-929560

ABSTRACT

Objective: This study aimed to determine the efficacy of dose-enhanced immunochemotherapy followed by autologous peripheral blood stem cell transplantation (ASCT) in young patients with newly diagnosed high-risk aggressive B-cell lymphoma. Methods: A retrospective study was conducted to examine the clinical and survival data of young patients with high-risk aggressive B-cell lymphoma who received dose-enhanced immunochemotherapy and ASCT as first-line treatment between January 2011 and December 2018 in Blood Diseases Hospital. Results: A total of 63 patients were included in the study. The median age range was 40 (14-63) years old. In terms of the induction therapy regimen, 52 cases received R-DA-EP (D) OCH, and the remaining 11 received R-HyperCVAD/R-MA. Sixteen (25.4% ) patients achieved partial response in the mid-term efficacy assessment, and ten of them were evaluated as complete response after transplantation. The median follow-up was 50 (8-112) months, and the 3-year progression-free survival (PFS) rate and overall survival (OS) rate were (83.9±4.7) % and (90.4±3.7) % , respectively. Univariate analysis demonstrated that age-adjusted international prognostic index ≥2 scores was a negative prognostic factor for OS (P=0.039) , and bone marrow involvement (BMI) was an adverse prognostic factor for OS (P<0.001) and PFS (P=0.001) . However, multivariate analysis confirmed that BMI was the only independent negative predictor of OS (P=0.016) and PFS (P=0.001) . Conclusions: The use of dose-enhanced immunochemotherapy in combination with ASCT as first-line therapy in the treatment of young, high-risk aggressive B-cell lymphoma results in good long-term outcomes, and BMI remains an adverse prognostic factor.


Subject(s)
Adult , Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Disease-Free Survival , Hematopoietic Stem Cell Transplantation , Humans , Lymphoma, B-Cell , Peripheral Blood Stem Cell Transplantation , Prognosis , Retrospective Studies , Stem Cell Transplantation , Transplantation, Autologous
14.
Chinese Journal of Hematology ; (12): 141-145, 2022.
Article in Chinese | WPRIM | ID: wpr-929546

ABSTRACT

Objective: To evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (auto-HSCT) in elderly patients (≥65 years old) with multiple myeloma (MM) . Methods: From June 1, 2006 to July 31, 2020, 22 MM patients (≥65 years old) who were diagnosed in the First Affiliated Hospital, Sun Yat-sen University and received novel drug induction followed by auto-HSCT were analyzed retrospectively. These patients were evaluated for important organ functions before transplantation, and the International Myeloma Working Group frail score was used in 2016 to screen out transplant-eligible patients. Results: The median (interquartile range, IQR) age at the time of transplantation of the 22 patients was 66.75 (IQR 4.50) years. A total of 20 patients received stem cell mobilization. The median number of mononuclear cells collected was 4.53×10(8)/kg, that of CD34(+) cells was 3.37×10(6)/kg, and the median number of apheresis procedures performed was 2. After stem cell transfusion, the median time of neutrophil implantation was 11 days, that of platelet implantation was 13 days, and the treatment-related mortality was 0 at 100 days after transplantation. The median follow-up was 48.7 months. The median time to progression time was not reached, and the median overall survival time was 111.8 months. Conclusion: Auto-HSCT is a safe and effective treatment for selected elderly patients of 65 years or older with MM.


Subject(s)
Aged , Hematopoietic Stem Cell Mobilization/methods , Hematopoietic Stem Cell Transplantation/adverse effects , Humans , Multiple Myeloma/drug therapy , Retrospective Studies , Transplantation, Autologous/methods , Treatment Outcome
15.
Chinese Journal of Hematology ; (12): 120-127, 2022.
Article in Chinese | WPRIM | ID: wpr-929543

ABSTRACT

Objective: To evaluate the efficacy and prognosis of basiliximab in the treatment of steroid-refractory or steroid-dependent acute graft-versus-host disease (SR/SD-aGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT) . Methods: Clinical data of 87 patients with SR/SD-aGVHD in the skin, intestine, and liver after allo-HSCT at the Institute of Hematology & Blood Diseases Hospital Transplantation Center from January 2015 to December 2018 were retrospectively analyzed. The administration plan of basiliximab was as follows: 20 mg for adults and children weighing ≥35 kg and 10 mg for children weighing<35 kg. The drug was administered once on the 1st, 4th, and 8th days, respectively, and then once weekly. The efficacy was evaluated on the 7th, 14th, 21st, and 28th days after basiliximab treatment. Results: ①There were 51 males (58.6%) and 36 females (41.4%) , with a median (range) age of 34 (4-63) years. There were 54 cases of classic aGVHD, 33 of late aGVHD, 49 of steroid-refractory aGVHD, and 38 of steroid-dependent aGVHD. ②Thirty-five patients (40.2%) achieved complete remission (CR) , 23 (26.4%) achieved partial remission (PR) , and 29 had no remission (NR) . The total effective rate[overall response rate (ORR) ] was 66.7% (58/87) . ③The ORR of the classic and late aGVHD groups was 77.8% (42/54) and 48.5% (16/33) , respectively. ④The median (range) follow-up time was 154 (4-1813) days, the 6-month overall survival (OS) rate of the 87 patients was 44.8% (95% CI 39.5%-50.1%) and the 1-year OS was 39.4% (95%CI 34.2%-44.3%) . ⑤After treatment with basiliximab, the 6-month OS in the CR (35 cases) , PR (23 cases) , and NR (29 cases) groups was 80.0% (95%CI 73.2%-86.8%) , 39.1% (95%CI 28.9%-49.3%) , and 6.9% (95%CI 2.2%-11.6%) , respectively (χ(2)=34.679, P<0.001) , and the 1-year OS was 74.3% (95%CI 66.9%-81.7%) , 30.4% (95%CI 20.8%-40.0%) , and 3.4% (95%CI 0%-6.8%) , respectively (χ(2)=43.339, P<0.001) . The OS of the classic and late aGVHD groups was 57.4% (95%CI 50.7%-64.1%) and 24.2% (95%CI 16.7%-31.7%) , respectively (χ(2)=9.109, P=0.004) , and the 1-year OS was 51.9% (95%CI 45.1%-58.7%) and 18.2% (95%CI 11.5%-24.9%) , respectively (χ(2)=9.753, P=0.003) . ⑥Univariate and multivariate analyses showed that late aGVHD (OR=3.121, 95%CI 1.770-5.503, P<0.001) , Minnesota score high-risk group before medication (OR=3.591, 95%CI 1.931-6.679, P<0.001) , active infection before medication (OR=1.881, 95%CI 1.029-3.438, P=0.040) , and impairment of important organ function caused by non-GVHD (OR=3.100, 95%CI 1.570-6.121, P=0.001) were independent risk factors affecting the efficacy of basiliximab. Conclusion: Basiliximab has good efficacy and safety for SR/SD-aGVHD, but not in patients with late aGVHD, high-risk group of Minnesota score, and infection or impaired function of important organs.


Subject(s)
Acute Disease , Adult , Basiliximab/therapeutic use , Child , Female , Graft vs Host Disease/drug therapy , Hematopoietic Stem Cell Transplantation/adverse effects , Humans , Male , Middle Aged , Retrospective Studies , Steroids/therapeutic use
16.
Article in Chinese | WPRIM | ID: wpr-939715

ABSTRACT

Mesenchymal stem cells (MSC) have been widely used in tissue regeneration and treatment graft versus host disease (GVHD) and immune diseases due to their self-renewal, multi-differentiation and immunoregulatory potential. However, more and more scholars begin to put weight on the MSC -derived extracellular vesicles (MSC-EV) for its regulation of inflammation and immunity. MSC-EV can activate the relevant signal pathways and regulate the function and biological behaviors of cells via acting on target cells and mediating communication between cells. MSC-EV has important potential clinical applications for its powerful immunomodulatory and hematopoietic regulatory functions. It is considered as a potential therapeutic tool to treat autoimmune diseases and GVHD. This paper reviewed the immunomodulatory activity of MSC-EV as well as the research progress of MSC-EV in hematopoietic stem cell transplantation, and discussed its potential clinical applications in the future.


Subject(s)
Cell Differentiation , Extracellular Vesicles/transplantation , Graft vs Host Disease/metabolism , Hematopoietic Stem Cell Transplantation , Humans , Mesenchymal Stem Cell Transplantation , Mesenchymal Stem Cells
17.
Article in Chinese | WPRIM | ID: wpr-939713

ABSTRACT

OBJECTIVE@#To investigate the safety and efficacy of a new proteasome inhibitor Ixazomib followed by autologous hematopoietic stem cell transplantation (AHSCT) in the treatment of POEMS syndrome.@*METHODS@#The clinical manifestations, diagnosis and treatment process and follow-up results of 4 patients with POEMS syndrome who were treated with Ixazomib-based regimen combined with AHSCT in Wuhan No.1 Hospital from February 2018 to July 2020 were analyzed retrospectively. All patients were male, aged from 37-54 years old, with varying degrees of peripheral neuropathy, organ enlargement (liver, spleen or lymph nodes), circulatory overload (peripheral edema and/or pleural effusion), osteosclerosis, endocrine diseases (thyroid, gonads, etc.), skin changes (pigmentation, hemangioma, white nails, etc.), M protein, papilledema and other clinical manifestations and characteristics at the time of initial treatment. Two patients were pathologically diagnosed as hyaline vascular Castleman disease by lymph node biopsy. Three patients underwent lumbar puncture examinations and all showed elevated cerebrospinal fluid protein. All patients received at least 2 cycles of sequential AHSCT after induction chemotherapy based on ixazomib. The follow-up time was 10-28 months, and the median follow-up time was 16 months.@*RESULTS@#All cases survived. The complications were controllable during the treatment. Moreover, the clinical symptoms related to the disease were improved to a certain extent after the treatment. The levels of vascular endothelial growth factor (VEGF) showed a gradual decline.@*CONCLUSION@#Ixazomib combined with AHSCT is safe and effective in the treatment of POEMS syndrome.


Subject(s)
Adult , Boron Compounds , Glycine/analogs & derivatives , Hematopoietic Stem Cell Transplantation , Humans , Male , Middle Aged , POEMS Syndrome/therapy , Retrospective Studies , Transplantation, Autologous , Vascular Endothelial Growth Factor A
18.
Article in Chinese | WPRIM | ID: wpr-939709

ABSTRACT

AbstractObjective: To investigate the sample selection, result correction and clinical application value of multi nucleotide polymorphism chimerism detection method based on Next-generation sequencing.@*METHODS@#The chimerism samples from November 2018 to June 2020 were collected, and Pearson correlation coefficient (r) was used to analyze the consistency of bone marrow and peripheral blood results detected by MNPseq; according to the different information integrity before transplantation, the calibration model was constructed to analyze the correction value of the micro chimerism results in each model; the clinical results were retrospectively analyzed to verify the reliability and practicability of chimerism results correction and the clinical value of MNPseq method.@*RESULTS@#The results of bone marrow and peripheral blood chimerism detected by MNPseq method were consistent with each other and showed significant correlation (r=0.985, P<0.01). The three groups of calibration models were constructed according to different pre-transplant information. For the no donor and pre-transplant patients information group, the correction value was 1%; while for the group with pre-transplant patients and without donor information, 0.61% of the chimerism rate and 13 heterotopic points were used as the correction value; 0.26% of the chimerism rate and 21.57% of the heterotopic points were used as the correction value for the group with pre-transplantation patients and donor information. After correction, the number of the patients with incomplete chimerism decreased from 276 (74.19%) to 141 (37.91%) (P<0.01). Among 18 (18/141, 12.77%) patients with incomplete chimerism, the results of MNPseq in the patients were 25-39 days earlier than those in STR and flow MRD, and the result showed statistical significance.@*CONCLUSION@#MNPseq method can be used to monitor chimerism with peripheral blood instead of bone marrow samples, and the results can be corrected to detect the changes of graft status in vivo in a more timely manner.


Subject(s)
Chimerism , Hematopoietic Stem Cell Transplantation , Humans , Nucleotides , Reproducibility of Results , Retrospective Studies , Transplantation Chimera/genetics , Transplantation, Homologous
19.
Article in Chinese | WPRIM | ID: wpr-939681

ABSTRACT

OBJECTIVE@#To investigate the prognostic significance of dynamic detection of minimal residual disease (MRD) in patients with acute myeloid leukemia (AML) by 8-color flow cytometry.@*METHODS@#MRD of 282 AML patients who achieved remission after initial therapy was detected by 8-color flow cytometry. MRD threshold for predicting recurrence was determined by receiver operating characteristic (ROC) curve, and time from MRD-positive to clinical recurrence was analyzed. The differences in overall survival (OS) time and relapse-free survival (RFS) time of patients with different MRD-changes were compared, and the related factors of recurrence in patients with MRD-negative were analyzed by univariate and logistic regression analysis.@*RESULTS@#ROC curve determined that the MFC-MRD threshold for predicting the recurrence of AML was 0.105%, and the recurrence rate of MRD-positive patients was significantly higher than that of MRD-negative patients [52.45% (75/143 cases) vs 35.97% (50/139 cases), P=0.005]. The patients in MRD persistent positive group and negative to positive group recurred earlier than those in positive to negative group and negative-positive fluctuation group (P<0.005). Survival analysis showed that OS and RFS time of patients with MRD persistent positive were significantly shorter than those of patients with MRD persistent negative, positive to negative, and negative-positive fluctuation (P<0.005). There was no significant difference in OS and RFS between MRD negative to positive group and MRD persistent positive group (P>0.005), either between MRD persistent negative group and MRD positive to negative group (P>0.005). Among 139 MRD-negative patients, 50 recurred. Univariate and logistic regression analysis showed that the risk of recurrence increased with the increase of white blood cells level (95%CI: 1.000-1.013, P=0.045). The risk of recurrence in patients without hematopoietic stem cell transplantation (HSCT) was 9.694 times higher than that in patients who received HSCT (95%CI: 1.720-54.651, P=0.010), and in the high-risk group was 5.848 times higher than that in the low-risk group (95%CI: 1.418-24.121, P=0.015).@*CONCLUSION@#The prognosis of AML patients with different MRD changes is significantly different. No matter MRD-positive or MRD-negative at the initial remission, dynamic detection of MRD after treatment is more helpful to accurately guide treatment.


Subject(s)
Flow Cytometry , Hematopoietic Stem Cell Transplantation , Humans , Leukemia, Myeloid, Acute/drug therapy , Neoplasm, Residual/diagnosis , Prognosis , Recurrence , Transplantation, Homologous
20.
Article in Chinese | WPRIM | ID: wpr-939641

ABSTRACT

OBJECTIVES@#To evaluate the clinical effect of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in children with hyper-IgM syndrome (HIGM).@*METHODS@#A retrospective analysis was performed on the medical data of 17 children with HIGM who received allo-HSCT. The Kaplan Meier method was used for the survival analysis of the children with HIGM after allo-HSCT.@*RESULTS@#After allo-HSCT, 16 children were diagnosed with sepsis; 14 tested positive for virus within 100 days after allo-HSCT, among whom 11 were positive for Epstein-Barr virus, 7 were positive for cytomegalovirus, and 2 were positive for JC virus; 9 children were found to have invasive fungal disease. There were 6 children with acute graft-versus-host disease and 3 children with chronic graft-versus-host disease. The median follow-up time was about 2 years, and 3 children died in the early stage after allo-HSCT. The children had an overall survival (OS) rate of 82.35%, an event-free survival (EFS) rate of 70.59%, and a disease-free survival (DFS) rate of 76.47%. The univariate analysis showed that the children receiving HLA-matched allo-HSCT had a significantly higher EFS rate than those receiving HLA-mismatched allo-HSCT (P=0.019) and that the children receiving HLA-matched unrelated allo-HSCT had significantly higher OS, EFS, and DFS rates than those receiving HLA-mismatched unrelated allo-HSCT (P<0.05). Compared with the children with fungal infection after allo-HSCT, the children without fungal infection had significantly higher EFS rate (P=0.02) and DFS rate (P=0.04).@*CONCLUSIONS@#Allo-HSCT is an effective treatment method for children with HIGM. HLA-matched allo-HSCT and active prevention and treatment of fungal infection and opportunistic infection may help to improve the prognosis of such children.


Subject(s)
Child , Epstein-Barr Virus Infections , Graft vs Host Disease/prevention & control , Hematopoietic Stem Cell Transplantation/methods , Herpesvirus 4, Human , Humans , Hyper-IgM Immunodeficiency Syndrome , Retrospective Studies
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