RESUMEN
Experimental economics is, nowadays, a well-established approach to investigate agents' behavior under economic incentives. In the last decade, a fast-growing number of studies have focused on the application of experimental methodology to health policy issues. The results of that stream of literature have been intriguing and strongly policy oriented. However, those findings are scattered between different health-related topics, making it difficult to grasp the overall state-of-the-art. Hence, to make the main contributions understandable at a glance, we conduct a systematic literature review of laboratory experiments on the supply of health services. Of the 1248 articles retrieved from 2011, 56 articles published in peer-review journals have met our inclusion criteria. Thus, we have described the experimental designs of each of the selected papers and we have classified them according to their main area of interest.
RESUMEN
Our experiment assesses the level of coordination on clinical best practice among physicians and investigates whether the release of guidelines helps in supporting coordination. Based on three clinical vignettes using current national guidelines, physicians evaluate the appropriateness of each of the proposed courses of action. Afterwards, physicians are allowed to ask which action corresponds to national guidelines and change their ratings, if desired. On average, slightly more than half of the sample coordinated on appropriateness evaluations. Empirical analysis indicates that several organizational and individual variables influence the level of coordination. Additionally, the release of national guidelines improved both the level of conformity and coordination. Our findings suggest changes in implementation practices to increase the impact of these shared protocols in the health field.
Asunto(s)
Guías de Práctica Clínica como Asunto , Humanos , Médicos , Masculino , Femenino , Adulto , Adhesión a Directriz , Persona de Mediana EdadRESUMEN
BACKGROUND: Necrotizing colitis (NC) represents a severe, although rare, complication in patients with anorexia nervosa. OBJECTIVE: We report the only case of NC with good prognosis in a severely malnourished AN patient. METHOD: Available patient records, imaging, and biochemical data were evaluated. DISCUSSION: This case represents an unusual gastrointestinal complication and underlines the important clinical role that Parenteral Nutrition (PN) has for some selected very malnourished AN patients. The severe starvation may compromise the normal function of gastrointestinal tract and it can also lead to an incomplete tolerance of enteral refeeding thus making PN required to reach the nutritional rehabilitation.
Asunto(s)
Anorexia Nerviosa/complicaciones , Colitis/etiología , Adolescente , Colitis/patología , Colitis/terapia , Femenino , Humanos , Desnutrición/complicaciones , Desnutrición/etiología , Necrosis , Nutrición Parenteral , PronósticoRESUMEN
Patients on long-term parenteral nutrition (PN) are at significantly increased risk for the development of metabolic bone disease (MBD); this condition is characterized by incomplete mineralization of osteoid with consequent disturbances ranging from osteopenia to severe bone disease with fractures. The aim of the study was: (1) to evaluate the prevalence of MBD, (2) to identify the PN- or intestinal failure (IF)-related factors and (3) to assess annual changes of bone mineral status. Since September 2005 all patients affected by IF and treated with PN started a BMD evaluation program using dual-energy X-ray absorptiometry (DXA). Twenty-four IF patients were included [15 with short bowel syndrome (SBS), 5 with severe protracted diarrhea and 4 with chronic intestinal pseudostruction]. The bone mineral density (BMD) Z-score was significantly lower in patients than in the control group. In our series SBS patients showed a BMD Z-score significantly higher in comparison with the medical causes of IF. No significant correlations were found between bone mineral status and PN duration and nutrient intake. Nine IF patients were submitted to a second DXA evaluation after 1 year from the baseline. All bone mineral variables were significantly increased at the second DXA evaluation. The high prevalence of MBD in IF patients undergoing long-term treatment with PN requires that these patients undergo careful and periodic monitoring of their bone mineral status; patients with congenital gut dysfunctions, such as epithelium defects and motility anomalies, are at major risk of developing this complication, probably due to the association with extra-intestinal causes of bone loss.
Asunto(s)
Densidad Ósea/fisiología , Enfermedades Óseas Metabólicas/epidemiología , Enfermedades Gastrointestinales/terapia , Nutrición Parenteral en el Domicilio/efectos adversos , Absorciometría de Fotón , Adolescente , Algoritmos , Estatura , Peso Corporal , Enfermedades Óseas Metabólicas/diagnóstico por imagen , Enfermedades Óseas Metabólicas/prevención & control , Niño , Preescolar , Enfermedad Crónica , Dieta , Femenino , Enfermedades Gastrointestinales/congénito , Enfermedades Gastrointestinales/fisiopatología , Humanos , Estudios Longitudinales , Vértebras Lumbares/química , Masculino , Prevalencia , Factores de Riesgo , Síndrome del Intestino Corto/fisiopatología , Síndrome del Intestino Corto/terapiaRESUMEN
OBJECTIVES: The study aimed to assess, in pediatric patients presenting to the emergency department (ED), the incidence of visit to the ED for functional constipation (FC), symptoms, signs of presentation, and management from ED physicians. DESIGN: This is a retrospective study of hospital records for a period of 1 year at the ED of "Bambino Gesù" Children's Hospital, Rome, Italy. Children younger than 15 years discharged from ED with a diagnosis of FC in the 1-year period were included. INTERVENTIONS: We analyzed medical records of 202 patients (<15 years) with FC diagnosis at discharge. Main outcome measures included incidence, demographic characteristics, clinical presentations of FC patients, and ED physicians' interventions. RESULTS: Two hundred two FC cases were studied in a 12-month study period. Compared with the total number of ED consultations, the incidence of FC was 0.4%. The number of patients 4 years or younger was much higher than patients older than 4 years (P < .0001). Bowel frequency of 3 bowel movements or less per day, acute abdominal pain, and stool retention were found to be significantly more frequent than the other presenting symptoms (P < .0001). The number of patients beginning a therapy after ED discharge was significantly higher compared with that already treated before ED visit (P < .0001). Discharged patients were referred to community pediatricians significantly more frequently than to pediatric gastroenterologists (P = .003). CONCLUSIONS: Emergency department physicians have an important role in the diagnosis and management of FC despite its relatively low incidence. Indeed, ED intervention in many cases leads not only to recognition this disease but also to an approach for therapeutic strategy, avoiding complications of chronic constipation.
Asunto(s)
Estreñimiento , Adolescente , Distribución por Edad , Niño , Preescolar , Estreñimiento/diagnóstico , Estreñimiento/epidemiología , Estreñimiento/terapia , Continuidad de la Atención al Paciente , Servicio de Urgencia en Hospital/estadística & datos numéricos , Femenino , Humanos , Incidencia , Lactante , Recién Nacido , Masculino , Anamnesis , Examen Físico , Estudios Retrospectivos , Ciudad de Roma/epidemiologíaRESUMEN
BACKGROUND AND AIM: Parenteral nutrition (PN) is the primary treatment for intestinal failure, which is considered irreversible in patients who remain partially or fully dependent on PN. Causes of irreversible intestinal failure are short bowel syndrome (SBS), motility disorders (MD), and severe protracted diarrhea (SPD). The aim of this study was to report the clinical outcome in these patients in relation to the underlying disease. PATIENTS AND METHODS: From January 1, 1989 to December 31, 2006, 218 intestinal failure patients were observed in our center, but only 96 (48 SBS, 39 SPD, and 9 MD) were included because they required at least 50% of their total calories as PN for not less than 3 months. In these patients, survival and complication rates were evaluated. RESULTS: The survival rate was significantly higher in SBS patients than in the other groups (P < 0.01). SBS patients showed a higher rate of major complications, although only intestinal failure-associated liver disease was significantly higher (P < 0.001). In our series, MD was the main cause of irreversible intestinal failure. CONCLUSIONS: The potential for bowel adaptation is higher in surgical than in medical causes of intestinal failure and does not seem to be influenced by complications of intestinal failure. SBS, although worsened by the major number of complications, was not the main category contributing to intestinal failure.
Asunto(s)
Adaptación Fisiológica/fisiología , Enfermedades Intestinales/epidemiología , Enfermedades Intestinales/terapia , Nutrición Parenteral/métodos , Adolescente , Causas de Muerte , Niño , Preescolar , Diarrea/epidemiología , Diarrea/mortalidad , Diarrea/terapia , Femenino , Motilidad Gastrointestinal/fisiología , Humanos , Lactante , Enfermedades Intestinales/mortalidad , Masculino , Nutrición Parenteral/efectos adversos , Prevalencia , Pronóstico , Síndrome del Intestino Corto/epidemiología , Síndrome del Intestino Corto/mortalidad , Síndrome del Intestino Corto/terapia , Análisis de Supervivencia , Factores de TiempoRESUMEN
BACKGROUND & AIMS: To assess prevalence of bone mineral density (BMD) reduction and relationship between bone mineral status and anthropometric assessment, nutritional intake and physical activity in adolescents with early anorexia nervosa (AN). METHODS: Fifty-seven consecutive AN patients and 57 healthy controls underwent anthropometric status, bone density, body composition and physical activity evaluations. In AN patients clinical features and nutritional intake were also assessed. RESULTS: Thirty-five patients with AN (62%) and 44 healthy subjects (77%) (pNS) showed normal BMD. Mean value of BMD Z-score was -0.6+/-0.9 in AN patients and -0.2+/-1.4 in controls (pNS). Weight at diagnosis and lean mass resulted the main predictor of bone loss but also height, best weight before diagnosis and BMI resulted correlated with bone mineral status in AN patients. Additionally, AN patients maintained good levels of protein intake and sport activity CONCLUSIONS: Early diagnosis may prevent bone loss in AN patients. Protein intake and moderate physical activity seem to be useful to maintain an adequate bone mineral status.
Asunto(s)
Anorexia Nerviosa/fisiopatología , Composición Corporal/fisiología , Densidad Ósea/fisiología , Proteínas en la Dieta/administración & dosificación , Ejercicio Físico/fisiología , Absorciometría de Fotón , Adolescente , Antropometría , Índice de Masa Corporal , Densidad Ósea/efectos de los fármacos , Huesos/metabolismo , Estudios de Casos y Controles , Femenino , Humanos , Estado NutricionalRESUMEN
BACKGROUND: Inflammatory bowel disease (IBD) has been associated with several polymorphisms in genes likely involved in innate immune responses and integrity of epithelial mucosal barrier. A major role in adult Crohn's disease (CD) has been defined for 3 polymorphisms in the CARD15 gene, whereas variants in the SLC22A4, SLC22A5, and DLG5 genes could have a minor contribution to IBD susceptibility. METHODS: We analyzed a panel of 6 polymorphisms within these genes in 227 Italian early-onset IBD patients (134 CD, 93 ulcerative colitis [UC]; age at diagnosis Asunto(s)
Enfermedades Inflamatorias del Intestino/genética
, Péptidos y Proteínas de Señalización Intracelular/genética
, Proteínas de la Membrana/genética
, Proteínas de Transporte de Catión Orgánico/genética
, Polimorfismo Genético
, Proteínas Supresoras de Tumor/genética
, Adolescente
, Edad de Inicio
, Niño
, Colitis Ulcerosa/genética
, Enfermedad de Crohn/genética
, Genotipo
, Humanos
, Italia
, Proteína Adaptadora de Señalización NOD2
, Simportadores
RESUMEN
BACKGROUND: Helicobacter pylori infection is likely acquired in childhood. Helicobacter pylori is recognized as a cause of gastritis and peptic ulcer. OBJECTIVE: To investigate some noninvasive tests, particularly H pylori fecal antigen, for the diagnosis of H pylori infection in comparison with the gold-standard invasive test, esophagogastroduodenoscopy with biopsy. METHODS: We studied 250 patients (102 male; age range, 3-18 years) who underwent esophagogastroduodenoscopy with biopsy (histologic examination and rapid urease test) for a suspicious upper gastrointestinal disease; in all of them, fecal H pylori antigen, serum H pylori immunoglobulin G, and cytotoxin-associated gene product A immunoglobulin G were measured. Sensitivity and specificity of noninvasive tests were compared with those of the gold-standard esophagogastroduodenoscopy with biopsy. RESULTS: Ninety-three patients (37%) had positive histopathologic (Giemsa staining) and rapid urease test results. The H pylori fecal antigen revealed a sensitivity of 97%, a specificity of 98%, a positive predictive value of 97%, and a negative predictive value of 98%; serum H pylori immunoglobulin G had a sensitivity of 86%, a specificity of 80%, a positive predictive value of 72%, and a negative predictive value of 90%; and serum cytotoxin-associated gene product A immunoglobulin G had a sensitivity of 83%, a specificity of 80%, a positive predictive value of 71%, and a negative predictive value of 89%. CONCLUSIONS: Our study demonstrates that among noninvasive and easily applicable tests, particularly in small children, H pylori fecal test is simple, suitable, and has high accuracy for the screening of H pylori-positive patients.
Asunto(s)
Infecciones por Helicobacter/diagnóstico , Helicobacter pylori/aislamiento & purificación , Adolescente , Antígenos Bacterianos/análisis , Proteínas Bacterianas/sangre , Biopsia , Niño , Preescolar , Endoscopía del Sistema Digestivo , Heces/microbiología , Femenino , Gastritis/patología , Infecciones por Helicobacter/inmunología , Humanos , Hiperemia/patología , Técnicas para Inmunoenzimas , Inmunoglobulina G/análisis , Masculino , Valor Predictivo de las Pruebas , Estudios Prospectivos , Antro Pilórico/patología , Saliva/inmunología , Sensibilidad y Especificidad , UreasaRESUMEN
OBJECTIVES: The aim of this study was to investigate how the differences across the regional reimbursement mechanisms and in particular the use of the DRGs impact on the level in the high technology equipment diffusion. METHODS: Based on hospital sector data at a regional level we build up indicators to measure the regional diffusion of high technological medical equipment in the period 1997-2007. These indicators are regressed on regional healthcare characteristics to investigate the relationship between the different reimbursement systems offered by Italian regions and the level of high technological medical equipment. RESULTS: Our results suggest that the per-case payment system is generally associated with a lower level of regional technology endowment per million of inhabitants, especially for the complex and expensive medical equipment. CONCLUSIONS: Our findings cast some doubts that an effective regulation of reimbursement mechanisms cannot limit the excessive diffusion of medical equipment that is a relevant driver of the increase in expenditure.
Asunto(s)
Tecnología Biomédica/estadística & datos numéricos , Difusión de Innovaciones , Hospitales/estadística & datos numéricos , Mecanismo de Reembolso , Tecnología Biomédica/economía , Grupos Diagnósticos Relacionados/economía , Grupos Diagnósticos Relacionados/estadística & datos numéricos , Economía Hospitalaria , Humanos , Italia , Programas Nacionales de Salud/economía , Programas Nacionales de Salud/organización & administración , Programas Nacionales de Salud/estadística & datos numéricosRESUMEN
BACKGROUND: Thyroid autoimmunity (TA) is often associated with coeliac disease (CD). OBJECTIVE: To evaluate, in children and adolescents with CD on a gluten-free diet (GFD): (1) the prevalence of TA; (2) the impact of TA on growth and the need for L-thyroxine (L-T4) treatment, during a longitudinal survey. METHOD: Between January and December 2005, 545 patients with CD, prospectively followed up until December 2007, and 622 controls were screened for TA. Antithyroperoxidase and antithyroglobulin antibodies were assayed and, if positive, serum free tri-iodothyronine, free thyroxine and thyroid-stimulating hormone (TSH) assays and thyroid ultrasound were performed. L-T4 was started if TSH was >5.5 mU/ml at two successive measurements. RESULTS: There was no significant difference in TA prevalence between patients with CD on a GFD (10%) and controls (8.2%). Duration of GFD differed significantly in coeliac patients with TA in comparison with those without TA (7.9±0.9 and 10.2±0.3 years, p<0.001), but no significant difference was found for weight and height gain (1.8±1.0 vs 3.7±1.5 and 2.1±1.2 kg/year vs 4.0±1.1 cm/year, respectively). At the end of the follow-up an increase of 7% in the prevalence of patients with CD with TA requiring L-T4 was found. CONCLUSIONS: TA seems no more common in paediatric and adolescent patients with CD on a GFD than in controls; its clinical evolution does not seem to impact on growth. Therefore, a long-term regular screening programme for thyroid disease may not be necessary for all patients with CD on a GFD, but only for those who are suspected of having thyroid diseases.
Asunto(s)
Enfermedades Autoinmunes/etiología , Enfermedad Celíaca/complicaciones , Enfermedades de la Tiroides/etiología , Adolescente , Enfermedad Celíaca/dietoterapia , Niño , Preescolar , Dieta Sin Gluten , Femenino , Estudios de Seguimiento , Trastornos del Crecimiento/etiología , Humanos , Hipotiroidismo/tratamiento farmacológico , Hipotiroidismo/etiología , Lactante , Masculino , Estudios Prospectivos , Tiroxina/uso terapéutico , Adulto JovenRESUMEN
BACKGROUND: To date, a number of high-profile studies have yielded over 50 inflammatory bowel disease (IBD) disease genes/loci. The polymorphisms rs9858542 (BSN) and rs3197999 (MST1), on 3p21 locus, have been found associated with susceptibility to IBD. We aimed to replicate these associations in adult and early-onset cohorts of IBD Italian patients, by analyzing also potential gene-gene interactions with variants in NOD2/CARD15, IL23R, ATG16L1, and IRGM genes, and investigating genotype-phenotype correlation. METHODS: In all, 1808 patients with IBD, 855 with Crohn's disease (CD) and 953 with ulcerative colitis (UC), including 539 patients with their initial diagnosis <19 years of age, and 651 controls were analyzed for SNPs rs9858542 and rs3197999. RESULTS: BSN and MST1 were significantly associated with either CD (P(rs9858542) 2.5 x 10(-7); P(rs3197999) 3.9 x 10(-7)), and UC (P(rs9858542) = 3.1 x 10(-4); P(rs3197999) = 8 x 10(-4)). Prevalence of these variants was significantly increased in both adult and early-onset IBD patients. After stepwise logistic regression, the 2 variants were associated in adult UC with distal colitis (P(rs9858542) = 0.013, odds ratio [OR] = 2.04, 95% confidence interval [CI] = 1.16-3.59; P(rs3197999) = 0.018, OR 1.9, 95% CI 1.2-3.3), while the rs3197999 variant was inversely associated with occurrence of extraintestinal manifestations in adult CD(P = 0.017, OR 0.6, 95% CI 0.4-0.9). CONCLUSIONS: We confirmed the association of BSN and MST1 with IBD susceptibility, either in the adult or the early-onset cohorts. These variants appeared to influence either the distal location of the disease in the UC cohort and extraintestinal manifestations in CD patients.
Asunto(s)
Cromosomas Humanos Par 3/genética , Colitis Ulcerosa/genética , Enfermedad de Crohn/genética , Predisposición Genética a la Enfermedad , Polimorfismo de Nucleótido Simple/genética , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Proteínas Relacionadas con la Autofagia , Proteínas Portadoras/genética , Niño , Preescolar , Colitis Ulcerosa/patología , Enfermedad de Crohn/patología , Femenino , Proteínas de Unión al GTP/genética , Estudios de Asociación Genética , Genotipo , Haplotipos/genética , Humanos , Lactante , Masculino , Persona de Mediana Edad , Proteína Adaptadora de Señalización NOD2/genética , Fenotipo , Receptores de Interleucina/genética , Adulto JovenRESUMEN
The inflammatory bowel diseases (IBD) Crohn's disease and ulcerative colitis are common causes of morbidity in children and young adults in the western world. Here we report the results of a genome-wide association study in early-onset IBD involving 3,426 affected individuals and 11,963 genetically matched controls recruited through international collaborations in Europe and North America, thereby extending the results from a previous study of 1,011 individuals with early-onset IBD. We have identified five new regions associated with early-onset IBD susceptibility, including 16p11 near the cytokine gene IL27 (rs8049439, P = 2.41 x 10(-9)), 22q12 (rs2412973, P = 1.55 x 10(-9)), 10q22 (rs1250550, P = 5.63 x 10(-9)), 2q37 (rs4676410, P = 3.64 x 10(-8)) and 19q13.11 (rs10500264, P = 4.26 x 10(-10)). Our scan also detected associations at 23 of 32 loci previously implicated in adult-onset Crohn's disease and at 8 of 17 loci implicated in adult-onset ulcerative colitis, highlighting the close pathogenetic relationship between early- and adult-onset IBD.
Asunto(s)
Variación Genética , Enfermedades Inflamatorias del Intestino/genética , Edad de Inicio , Mapeo Cromosómico , Colitis Ulcerosa/genética , Genoma Humano , Estudio de Asociación del Genoma Completo , Humanos , Enfermedades Inflamatorias del Intestino/epidemiologíaRESUMEN
OBJECTIVES: Our goal was to evaluate the possible correspondence between antitissue transglutaminase of immunoglobulin A class levels and stage of mucosal damage in patients affected by celiac disease. In addition, we assessed clinical use of antitissue transglutaminase values to predict biopsy results. METHODS: One thousand eight hundred eighty-six consecutive patients with symptoms suggestive of celiac disease and 305 healthy controls underwent determination of serum levels of immunoglobulin A and antitissue transglutaminase. An intestinal biopsy was performed in subjects with antitissue transglutaminase levels > or = 4 IU/mL and in subjects with negative antitissue transglutaminase levels but with clinical suspicion of celiac disease. Histologic grading of celiac disease was consistent with the Marsh classification. RESULTS: One hundred eighty-six subjects with positive antitissue transglutaminase levels and 91 patients with negative antitissue transglutaminase levels were submitted to biopsy. In all healthy subjects, antitissue transglutaminase results were negative. Histologic evaluations in patients with positive antitissue transglutaminase levels gave the following results: type 0 in 25 patients, type 1 in 3 patients, type 2 in 4 patients, type 3a in 22 patients, type 3b in 74 patients, and type 3c in 58 patients. None of the patients with negative antitissue transglutaminase levels showed histologic findings suggestive of celiac disease. The mean antitissue transglutaminase values in patients without mucosal atrophy were significantly lower than in patients with mucosal atrophy. Antitissue transglutaminase values > or = 20 IU/mL were found in only 1 patient without mucosal atrophy. CONCLUSIONS: Our study found a strong correspondence between antitissue transglutaminase levels and stage of mucosal injury; antitissue transglutaminase values > 20 IU/mL seemed to be strongly predictive of mucosal atrophy.
Asunto(s)
Enfermedad Celíaca/sangre , Enfermedad Celíaca/diagnóstico , Proteínas de Unión al GTP/inmunología , Inmunoglobulina A/sangre , Transglutaminasas/inmunología , Adolescente , Enfermedad Celíaca/enzimología , Niño , Preescolar , Femenino , Humanos , Lactante , Masculino , Proteína Glutamina Gamma Glutamiltransferasa 2RESUMEN
BACKGROUND & AIMS: The syndrome of immunodysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) is a rare disorder resulting in the expression of multiple autoimmune and allergic features. Early onset enteropathy and type 1 diabetes (T1D) are the most common clinical features. The IPEX syndrome is caused by mutations of the FOXP3 gene, which is essential for the development of regulatory T cells (Treg). We describe 2 unrelated patients with IPEX syndrome with a mild clinical phenotype and with novel FOXP3 mutations and the phenotypic and functional characterization of their Treg cells. METHODS: The FOXP3 gene was analyzed by sequencing amplimers from genomic DNA. Treg cells were characterized by evaluating the number of CD4+CD25+ T cells and their functional ability to suppress the proliferation of autologous CD4+CD25- effector T cells stimulated with anti-CD3 and anti-CD28 antibodies. RESULTS: A 7-year-old boy and a 24-year-old man presented with autoimmune enteropathy characterized by early onset persistent diarrhea not associated with T1D or other endocrinopathies. These 2 patients carry novel FOXP3 mutations that do not abrogate the function of the forkhead domain. They have normal numbers of CD4+CD25+ T lymphocytes, however, these show severely defective suppressive function in vitro. CONCLUSIONS: Our 2 patients show that IPEX patients may present with early onset enteropathy and long-term survival without T1D or other endocrinopathies. This milder phenotype may be associated with FOXP3 mutations that do not abrogate the function of the forkhead domain.
Asunto(s)
Enfermedades Genéticas Ligadas al Cromosoma X/genética , Predisposición Genética a la Enfermedad , Mutación , Poliendocrinopatías Autoinmunes/genética , Enteropatías Perdedoras de Proteínas/genética , Adulto , Biopsia con Aguja , Preescolar , Análisis Mutacional de ADN , Factores de Transcripción Forkhead/genética , Regulación de la Expresión Génica , Enfermedades Genéticas Ligadas al Cromosoma X/inmunología , Enfermedades Genéticas Ligadas al Cromosoma X/patología , Humanos , Inmunohistoquímica , Mucosa Intestinal/patología , Masculino , Fenotipo , Poliendocrinopatías Autoinmunes/inmunología , Poliendocrinopatías Autoinmunes/patología , Pronóstico , Enteropatías Perdedoras de Proteínas/inmunología , Enteropatías Perdedoras de Proteínas/patología , Enfermedades Raras , Índice de Severidad de la Enfermedad , SíndromeRESUMEN
OBJECTIVE: To evaluate the safety and efficacy of the administration of low doses of glucocorticoids in patients with cystic fibrosis (CF) by using autologous erythrocytes loaded with dexamethasone 21-phosphate. STUDY DESIGN: Nine consecutive CF patients (patients nos. 1-9) received autologous erythrocytes loaded with increasing amounts of dexamethasone 21-phosphate to obtain a slow delivery of dexamethasone in circulation. The appearance of possible adverse effects, the reproducibility of the procedure, and the dexamethasone pharmacokinetics were evaluated. Subsequently, patient no. 9 and eight additional patients (patient nos. 10-17) received dexamethasone 21-phosphate-loaded erythrocytes at 1-month intervals to evaluate the efficacy of continuous release in circulation of low doses of dexamethasone. RESULTS: Erythrocytes from CF patients can be processed to be loaded with increasing dexamethasone 21-P concentrations. Once reinfused in respective donors, a slow and prolonged delivery of dexamethasone in the blood stream was measured up to 28 days. Repeated administrations of drug-loaded erythrocytes at 4-week intervals for 15 months showed that very low doses of glucocorticoids provide significant improvement in FEV1 values and significant reduction of infective relapses due to Pseudomonas aeruginosa without adverse effects. CONCLUSIONS: The administration of very low doses of glucocorticoids using autologous erythrocytes is possible, with benefits for patients and without side effects. This method is likely to be extended to other chronic diseases.