El trasplante de células hematopoyéticas pediátrico en la región de murcia. El inicio de una singladura
- Fita, Ana Maria
- Miguel Blanquer Director/a
- A. Sánchez Salinas Director/a
Universidad de defensa: Universidad de Murcia
Fecha de defensa: 29 de mayo de 2018
- Luis Madero López Presidente
- Ana María García Hernández Secretario/a
- Antonio Pérez Martínez Vocal
Tipo: Tesis
Resumen
Hematopoietic stem cell transplantation (SCT) it is a therapeutic procedure, which has allowed to improve long term survival in children affected by malignant and non malignant hemopathies, solid tumors, storage disorders, autoimmune diseases and congenital immunodeficiencies. They were studied SCT performed during the period 2007-2015, in pediatric patients at the Hospital Clínico Universitario Virgen de la Arrixaca (HCUVA). PRIMARY ENDPOINT To compare feasibility, safety and efficacy in pediatric SCT with other pediatric series. SECONDARY ENDPOINTS Study the graft composition, engraftment kinetics, early and late complications. Analyse the disease-free survival (DFS), relapse/progression, event-free survival (EFS) and overall survival (OS). PATIENTS AND METHODS Retrospective study of the 107 SCT performed in 91 pediatric patients at the HCUVA, by reviewing their medical records. The following variables were registered: patient and donor (age, sex, diagnosis, disease status prior SCT, CMV serology, blood type), SCT (number, donor type, HLA compatibility, source of stem cells), conditioning, graft-versus-host disease (GVHD) prophylaxis, graft composition (total nucleated cells, CD34+, CD3+, ?? CD3+, ?? CD3+, CD3+CD4+, CD3+CD8+, CD19+, CD56+, microbiology, cellular viability, CFU-GM, BFU-E), assessment of engraftment (leucocytes, neutrophils, platelets), transfusion requirements (red blood concentrates, platelet pools), engraftment rate and graft failure, post-aloSCT chimerism, immune reconstitution (CD3+, CD4+, CD8+, CD56+, CD19+, CD4+/CD8+ at day +30, +60, +90, +180, +270, +360), post SCT immunotherapy (donor-lymphocyte infusion), early post SCT complications (mucositis, infections, hemorrhagic cystitis, acute GVHD, hepatic veno-occlusive disease, hepatic and renal toxicity, engraftment syndrome, metabolic complications, infectious and non infectious pulmonary complications, neurologic complications), late post SCT complications (chronic GVHD, pulmonary, endocrine, other ). Statistical analysis was performed with the IBM© SPSS© Statistics 21.0 version (Chicago, Illinois, EE. UU.) software for the descriptive study (frecuency, percentage and for categorical variables; median and rank for continous variables). To study the association between a certain fact and a categorical variable, chi-square test or Fisher's exact test) were used. To compare media values of quantitative continous variables, T-Test or Mann-Whitney non-parametric test were performed. The comparison of more than two means was performed with analysis of variance (ANOVA) in the case of meeting the conditions of normality and homogeneity of variances (Levene test). Otherwise the nonparametric test H of Kruskal-Wallis was used (with Bonferroni correction in case of obtaining a result p <0.05). Probabilities of DFS, EFS and OS were estimated by the non-parametric test Kaplan-Meier and the Log-Rank test was used for univariate comparisons. R© (R 3.3.2, R Foundation for Statistical Computing, 2016) software and cmprsk package were used for the estimation of cumulative incidence ± standard error for acute and chronic GVHD, relapse and transplant related mortality. In each case competitive risks factors were taken into account. Statistical signification was considered if p<0.05 and Microsoft¿ Excel and Word 2011 14.6.6 version software were used to perform the rest of charts and the work edition. CONCLUSIONS The performance of the SCT procedure in pediatric patients in the HCUVA is feasible, safe and has shown similar efficacy to published studies, in terms of graft composition, engraftment kinetics, early and late complications, DFS, relapse/ progression, EFS, OS and TRM.