RESUMO
BACKGROUND: Chronic granulomatous disease (CGD) is a primary immunodeficiency with increased susceptibility to several bacteria, fungi, and mycobacteria, caused by defective or null superoxide production by the NADPH oxidase enzymatic complex. Accepted treatment consists mainly of antimicrobial prophylaxis. The role of human recombinant subcutaneous interferon-gamma (IFNγ) is less clear since the available evidence on its efficacy derives mainly from a single clinical trial that has been challenged. OBJECTIVE: We aimed to assess the efficacy and safety of IFNγ as an added treatment for CGD when compared to antimicrobial prophylaxis alone. METHODS: A literature search was conducted using MeSH terms "Chronic granulomatous disease" AND ("interferon gamma" OR "interferon-gamma"), as well as antibiotics, placebo, no therapy, clinical trial, and trial, on MEDLINE, EMBASE, LILACS, WHOs, CENTRAL, KOREAMED, The Cochrane Library, clinicaltrials.gov, and abstracts from meetings, from 1976 to July 2022. We included clinical trials (CT) and prospective follow-up studies and registered the number of serious infections (requiring hospitalization and IV antibiotics) and deaths, adverse events, and autoimmune complications, in patients treated for CGD with antimicrobial prophylaxis plus IFN-γ, versus antimicrobial prophylaxis alone. We assessed the quality of the studies using risk of bias and STROBE. We performed a meta-analysis by calculating both Peto's odds ratio (OR) and risk reduction (RR) through the Mantel-Haenszel method with a fixed-effect model, using Review Manager 5.4, and we reported the number needed to treat (NNT). RESULTS: We identified 54 matches from databases and 4 from other sources. We excluded 12 duplicates, 7 titles, and 9 abstracts for relevance, after which we had 30 eligible studies. Twenty-four were then excluded after reading the full text. Six papers were included: one randomized CT and 5 follow-up studies. In total, 324 patients with Chronic granulomatous disease were followed for 319 months under treatment with antibiotic prophylaxis plus interferon-gamma or placebo (or antibiotic prophylaxis alone), reported between the years 1991 and 2016. Three of the studies included a control group, allowing for the aggregate analysis of efficacy (prevention of serious infections). The aggregate OR was 0.49, with a 95% confidence interval of 0.19 to 1.23. The risk ratio for serious infection was 0.56 (95%CI 0.35-0.90) under IFN-γ. The meta-analysis thus favors interferon-gamma for a risk reduction of serious infection. DISCUSSION: The results from this meta-analysis support the use of IFN-γ in the treatment of patients with CGD. However, we found insufficient clinical evidence and believe more clinical trials are needed to better assess the efficacy and long-term safety of IFN-γ.
Assuntos
Antibacterianos , Doença Granulomatosa Crônica , Humanos , Estudos Prospectivos , Antibacterianos/uso terapêutico , Doença Granulomatosa Crônica/tratamento farmacológico , AntibioticoprofilaxiaRESUMO
Las trampas extracelulares de los neutrófilos son estructuras fundamentalmente compuestas de cromatina y proteínas granulares, que una vez liberadas constituyen un mecanismo de defensa que tiene la capacidad de atrapar y destruir microorganismos patógenos. El proceso que libera estas estructuras es conocido como NETosis y en el caso que provoque muerte celular, esta es diferente a la apoptosis y a la necrosis. Si bien no se conocen todos los eventos moleculares involucrados en la formación de las NETs, se sabe que dependiendo del estímulo, las especies reactivas del oxígeno son esenciales para que ocurra la descondensación de la cromatina y se lleve a cabo el proceso de NETosis(AU)
Neutrophil extracellular traps (NETs) are structures mainly composed of chromatin and granule proteins that once released constitute a defense mechanism due to their ability to trap and destroy pathogen microorganisms. The process by which these structures are released is known as NETosis and in case this may lead to cell death is different to apoptosis and necrosis. Although all the molecular events involved in the formation of NETs are poorly understood, it is known that depending on the stimulus, reactive oxygen species (ROS) are essential to the chromatin decondensation and subsequent NETs formation(AU)
Assuntos
Humanos , Armadilhas Extracelulares , NADP/fisiologia , Neutrófilos/imunologia , Doença Granulomatosa Crônica/tratamento farmacológico , Doença Granulomatosa Crônica/genéticaAssuntos
Doença Granulomatosa Crônica/tratamento farmacológico , Hipoglicemiantes/administração & dosagem , Macrófagos/efeitos dos fármacos , Mitocôndrias/metabolismo , Fagocitose , Tiazolidinedionas/administração & dosagem , Adolescente , Adulto , Animais , Células Cultivadas , Criança , Modelos Animais de Doenças , Feminino , Doença Granulomatosa Crônica/imunologia , Humanos , Mediadores da Inflamação/metabolismo , Macrófagos/imunologia , Masculino , Camundongos , Pessoa de Meia-Idade , PPAR gama/metabolismo , Fagocitose/efeitos dos fármacos , Pioglitazona , Espécies Reativas de Oxigênio/metabolismo , Adulto JovemRESUMO
La enfermedad granulomatosa crónica (EGC) es una inmunodeficiencia primaria de la fagocitosis. Se presenta un paciente de 13 años de edad que a partir el mes de nacido presentó infecciones recurrentes: diarreas, neumonías, tuberculosis pulmonar, gingivo-estomatitis, celulitis, adenitis, abscesos cutáneos y hepáticos recidivantes. Al examen físico presentó una disminución severa del peso y la talla para la edad, palidez cutáneo-mucosa, periodontitis crónica, hiperlaxitud, aumento del hemiabdomen derecho y adenopatías generalizadas. Los estudios inmunológicos mostraron concentraciones normales de las inmunoglobulinas (Ig) séricas IgM: 0,98 g/L (0,69 - 2,69 g/L), IgA: 2,76 g/L (1,58 - 3,94 g/L) e IgE: 11,70 UI/ml (hasta 50 UI/mL), respectivamente, y ligeramente aumentadas de IgG: 17,2 g/L (7,81 - 15,30 g/L), C3 y C4 normales: 1,28 g/L (0,9 - 1,7 g/L) y 0,30 g/L (0,2 - 0,4 g/L), respectivamente. Las subpoblaciones linfocitarias T CD3, CD4 y CD8 positivas estuvieron normales: 62 por ciento (52 - 78 por ciento), 45 por ciento (25 - 48 por ciento) y 15 por ciento (9 - 35 por ciento), y los linfocitos B CD19 positivos estuvieron normales: 24 por ciento (8 - 24 por ciento). El índice opsonofagocítico mostró valores normales en los tiempos 15 y 60 min: 35 por ciento (22,99 - 53,95 por ciento) y 12,50 por ciento (6,63 - 28,4 3 por ciento). La prueba de reducción de nitroazul de tetrazolium espontánea y con agente inductor (Cándida albicans) fue negativa. Se concluyó como una EGC ligada al cromosoma X. El tratamiento incluyó el drenaje de los abscesos hepáticos recidivantes, uso de antimicrobianos y antimicóticos potentes e interferón gamma, con lo que disminuyó la frecuencia e intensidad de las infecciones. El diagnóstico y el tratamiento precoces de la EGC disminuyen la morbilidad y mortalidad de estos enfermos...
Chronic granulomatous disease (CGD) is a primary immunodeficiency with a defect of the phagocytosis process. A 13 year-old adolescent with recurrent life-threatening episodes since one month of birth is presented. The main clinical manifestations included diarrhea, stomatitis, cellulitis, lymphadenitis, pneumonia, granuloma formation, pulmonary tuberculosis, pulmonary and hepatic abscesses. Physical examination showed poor growth, hepatomegaly, adenopathies, hyperextension of extremities and chronic gingivitis. Immunological studies showed normal concentrations of immunoglobulins (Ig): IgM: 0,98 g/L (0,69 - 2,69 g/L), IgA: 2,76 g/L (1,58 - 3,94 g/L) and IgE: 11,70 UI/mL ( < 50 UI/ml), C3 and C4 (1,28 g/L (0,9 - 1,7 g/L) and 0,30 g/L (0,2 - 0,4 g/L), respectively, and hypergammaglobulinemia of 17,2 g/L (7,81 - 15,30 g/L). Lymphocytes count T CD3, CD4 and CD8 positive were normal: 62 percent (52 - 78 percent), 45 percent (25 - 48 percent) y 15 percent (9 - 35 percent) and B lymphocytes count was also normal: 24 percent (8 - 24 percent). Opsonophagocytic index was normal at time 15 and 60 minutes: 35 percent (22,99 - 53,95 percent) and 12,50 percent (6,63 - 28,43 percent), respectively. Diagnosis was confirmed with negative nitroblue tetrazolium test . Treatment with antibiotics, fungistats, as well as gamma interferon contributed to a favorable response, presenting a lower amount of infectious episodes as well as a recovery of weight and height. Early diagnosis and treatment of CGD has improved prognosis and reduced patients´ morbidity and mortality...
Assuntos
Humanos , Masculino , Criança , Diagnóstico Precoce , Doença Granulomatosa Crônica/diagnóstico , Doença Granulomatosa Crônica/tratamento farmacológicoRESUMO
OBJETIVO: Relatar caso ilustrativo de doença granulomatosa crônica cujo diagnóstico ocorreu durante o aparecimento do primeiro episódio infeccioso, colaborando com a iniciativa do Brazilian Group for Immunodeficiency para a sensibilização do pediatra geral em relação ao diagnóstico precoce das imunodeficiências primárias, o que está associado a melhor qualidade de vida e maior sobrevida desses indivíduos. DESCRIÇÃO DE CASO: Paciente do sexo masculino, 39 dias de vida, admitido em pronto-socorro pediátrico por febre alta há cinco dias e irritabilidade. No dia seguinte, observou-se abscesso cervical, isolando-se Staphylococcus aureus comunitário. Durante a internação, ocorreram outros abscessos superficiais e em cadeias ganglionares profundas, além de resposta lenta aos antimicrobianos. Solicitou-se investigação para imunodeficiências, que confirmou a hipótese de doença granulomatosa crônica por quantificação dos ânions superóxido e teste de redução do nitrobluetetrazolio. Paciente foi encaminhado a serviço especializado, no qual identificou-se doador de medula óssea compatível, realizando-se o transplante seis meses após o diagnóstico. Quatro meses após o transplante, ocorreu normalização do burst oxidativo, indicando sucesso. COMENTÁRIOS: O paciente mostrou apresentação típica da doença, o que permitiu seu diagnóstico por pediatras gerais já na primeira infecção, tendo como consequência o acompanhamento por especialistas em imunodeficiências primárias, a introdução da profilaxia antimicrobiana e a procura bem sucedida de doador de medula HLA-compatível.
OBJECTIVE: To report a case of chronic granulomatous disease diagnosed during the first infectious episode in order to collaborate with the Brazilian Group for Immunodeficiency, in sensitizing the general pediatrician that the early diagnosis of primary immunodeficiency results in better quality of life and longer life expectancy for the patients. CASE DESCRIPTION: Male patient, 39 days, admitted to the pediatric emergency ward with fever for the last five days and irritability. On the following day, a cervical abscess was noted and a community Staphylococcus aureus was isolated. During hospital stay, other abscesses were observed in the skin and in the deep ganglia chains, with a slow response to antibiotics. Investigation of immunodeficiency was requested and chronic granulomatous disease was confirmed by quantification of superoxide anions and nitrobluetetrazolium tests. The patient was transferred to a specialized clinic for bone marrow transplantation, performed six months after diagnosis. Four months afterwards, the normalization of oxidative burst was noted, indecating the success of the transplantation. COMMENTS: The patient showed a typical presentation of the disease, which allowed its diagnosis by general pediatricians on the first infection, allowing the follow-up by experts in primary immunodeficiencies, the introduction of antimicrobial chemoprophylaxis, and the successful search for an HLA-compatible bone marrow donor.
Assuntos
Humanos , Masculino , Lactente , Abscesso , Doença Granulomatosa Crônica/diagnóstico , Doença Granulomatosa Crônica/tratamento farmacológico , Síndromes de Imunodeficiência/diagnóstico , Transplante de Medula ÓsseaRESUMO
Chronic granulomatous disease (CGD) is a primary immunodeficiency, a phagocyte defect that appears in 1:200,000 live births and is produced by mutations in the genes that codify for the enzyme nicotinamide adenine dinucleotide phosphate oxidase (NADPH oxidase). The inheritance form is X linked (> 60%) or autosomic recesive (30-40%). The NADPH oxidase is responsible for the production of reactive oxygen species (ROS) in the activated phagocyte ("respiratory burst"). When present, mutations on the NAPDH oxidase genes do not allow the ROS production, making the neutrophils of these patients incapable to destroy pathogens. These patients are especially susceptible to infections by staphylococcus, fungi and some gram-negative bacteria. The main clinical manifestations include recurrent life-threatening episodes of lymphadenitis, abscess, pneumonias, osteomyelitis, granuloma formation and sepsis. The diagnosis is suggested by a history of recurrent infections, familiar cases, fail to grow and confirmed with an altered test of ROS production and the specific mutation. Allogenic stem cells transplant is the curative treatment. The early diagnosis and the treatment with prophylactic antibiotics and interferon-gamma have modified favorably the morbidity and mortality of these patients.
Assuntos
Doença Granulomatosa Crônica , Doença Granulomatosa Crônica/diagnóstico , Doença Granulomatosa Crônica/tratamento farmacológico , HumanosRESUMO
Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by early onset of recurrent and severe infections. The molecular defects causing CGD are heterogeneous and lead to absence, low expression, or malfunctioning of one of the phagocyte NADPH oxidase components. It is known that mutations leading to CGD reside within the genes encoding four essential components of the oxidase designated as gp91-phox (phagocyte oxidase), p22-phox, p47-phox and p67-phox. gp91- together with p22-phox form the membrane cytochrome b(558) and play an essential role in the transfer of electrons following assembly of the active oxidase with the cytoplasmic p47- and p67-phox components. In hematopoietic cells, CYBB expression (the gene encoding gp91-phox) is limited to the granulocyte and monocyte/macrophage lineages during the process of terminal differentiation. CYBB is responsive to a number of inflammatory cytokines, especially interferon-gamma (IFN-gamma) and tumor necrosis factor alpha (TNF-alpha). Cytokines have been also studied for activation of phagocytes respiratory burst. IFN-gamma stimulates superoxide release and is a prophylactic agent for CGD. It has been shown in vitro and in vivo to correct at least in part alterations of the oxidative metabolism, and to improve their microbicidal function. It has demonstrated clinical benefit in the majority of patients with CGD, reducing the relative risk of severe infections in 70%. In this study, we review mechanisms showing that IFN-gamma improves the splicing efficiency of CYBB gene transcripts in a particular group of CGD patients. The present article is an informative review of recent patents related to the use of interferon gamma therapy in chronic granulomatous disease.
Assuntos
Doença Granulomatosa Crônica/tratamento farmacológico , Interferon gama/uso terapêutico , HumanosRESUMO
Chronic granulomatous disease (CGD) is an infrequent inherited disorder characterized by recurrent infections and abnormal granuloma formation. Patients with CGD have an exuberant inflammatory response and an increased risk of developing autoimmunity. We present the case of a 1-year-old boy with CGD who developed several of the characteristic clinical features of Kawasaki Disease. His illness responded to intravenous immunoglobulin, aspirin, and corticosteroids.
Assuntos
Doença Granulomatosa Crônica/epidemiologia , Fatores Imunológicos/uso terapêutico , Síndrome de Linfonodos Mucocutâneos/epidemiologia , Comorbidade , Quimioterapia Combinada , Glucocorticoides/uso terapêutico , Doença Granulomatosa Crônica/tratamento farmacológico , Humanos , Imunoglobulinas Intravenosas/uso terapêutico , Lactente , Masculino , Síndrome de Linfonodos Mucocutâneos/tratamento farmacológico , Síndrome de Linfonodos Mucocutâneos/etiologia , Prednisolona/uso terapêuticoRESUMO
The diagnostic and treatment of verrucous lesions of the larynx involves a high level of suspicion by the physician attending the patient. The causes may go from unspecific laryngitis to neoplasia and granulomatous diseases. This kind of lesion is uncommon and the presentation aspects may vary broadly. The lesions in larynx are significant source of morbidity. The onset of symptoms is insidious and the diagnosis is usually delayed. Symptoms include dysphonia, dyspnea, dysphagia and odynophagia. Proper treatment depends upon tissue biopsy, identification of the causative organism, and the appropriate pharmacotherapy. As there are few papers presenting the clinical features of infectious granulomatous laryngitis (IGL) as leishmaniasis, tuberculosis and paracoccidiodomycosis affecting the larynx, we considered important to show the experience of a big Brazilian Laryngology Service in dealing with this potential worldwide problem. We present a retrospective chart review showing our institution's experience with IGL focusing in the diagnostic, treatment and prognosis aspects. Twenty-four patients were identified. Mycobacterium tuberculosis and Paracoccidiodis brasiliensis accounted for ten cases each, and Leishmania braziliensis the remaining four. Hoarseness was the most common symptom of infection. Up to one-third of patients with laryngeal involvement lacked laryngeal symptoms. The average delay from onset of symptoms to diagnosis was 7 months. All patients underwent direct laryngoscopy and biopsies. Caseating granulomas was the key histopathologic finding. Identification of the causative organism was uncommon. No evidence of concomitant malignancy was seen on biopsy. Despite treatment, almost 40% of patients had permanent sequelae of infection, including hoarseness, dyspnea, and dysphagia. Mycobacterium tuberculosis, P. brasiliensis, and L. braziliensis accounted for all cases of IGL. Patients may have laryngeal infection but lack laryngeal symptoms. Prompt diagnosis relies upon a high index of suspicion, especially when evaluating patients from endemic areas. Given the degree of tissue destruction, which accompanies infection, timely intervention may be important in the prevention of late sequelae. Despite appropriate therapy, a significant number of patients may have permanent sequelae of infection.
Assuntos
Doença Granulomatosa Crônica/diagnóstico , Laringite/diagnóstico , Adolescente , Adulto , Antibacterianos/uso terapêutico , Bactérias/isolamento & purificação , Biópsia , Criança , Diagnóstico Diferencial , Feminino , Seguimentos , Doença Granulomatosa Crônica/tratamento farmacológico , Doença Granulomatosa Crônica/microbiologia , Humanos , Laringite/tratamento farmacológico , Laringite/microbiologia , Laringoscopia , Masculino , Pessoa de Meia-Idade , Prognóstico , Estudos RetrospectivosRESUMO
BACKGROUND: Chronic granulomatous disease (CGD) is a primary phagocyte immunodeficiency. It is often accompanied by an exuberant and aberrant inflammatory response, with granulomata and obstruction of the gastrointestinal and genitourinary tracts and inflammatory bowel disease. Although corticosteroids are successful in managing the obstructive and inflammatory disorders of CGD, they are not ordinarily used for the management of infection because of the possibility of further compromising the patient's immune system. OBJECTIVES: To discuss the pros and cons of the use of corticosteroids for the treatment of infections in CGD. METHODS: We describe 2 patients with CGD and refractory infections who were successfully treated with systemic corticosteroids in addition to antimicrobial agents. We also review the medical literature in which corticosteroids have been used for CGD infection. RESULTS: Our cases add to 3 other reports in which antibiotics and corticosteroids were used successfully in patients with CGD. However, in the presence of a potential pathogen, notably, aspergilla, corticosteroids may mask or favor dissemination of the fungus, especially in adults. CONCLUSIONS: Corticosteroids may play an important adjunctive role in CGD refractory infections.
Assuntos
Corticosteroides/uso terapêutico , Anti-Infecciosos/uso terapêutico , Broncopneumonia/tratamento farmacológico , Doença Granulomatosa Crônica/tratamento farmacológico , Abscesso Hepático/tratamento farmacológico , Broncopneumonia/etiologia , Broncopneumonia/microbiologia , Pré-Escolar , Quimioterapia Combinada , Feminino , Doença Granulomatosa Crônica/complicações , Humanos , Hidrocortisona/uso terapêutico , Abscesso Hepático/etiologia , Masculino , Prednisona/uso terapêuticoRESUMO
La enfermedad granulomatosa crónica (EGC) es una inmunodeficiencia primaria causada por mutaciones en los genes que codifican para cualquiera de las 4 subunidades que conforman a la enzima adenina dinucleótido fosfato oxidasa (NADPH oxidasa), encargada de regular la producción de especies oxidantes microbicidas que constituyen la primera vía de defensa del organismo contra los microorganismos infecciosos. Esta es una deficiencia muy heterogénea clasificada en EGC ligada al cromosoma X (subunidad gp91-phox) y autosómica recesiva, donde puede estar afectada cualquiera de las siguientes subunidades de la enzima: p22-phox, p47-phox y p67-phox. Dentro de los hallazgos clínicos más frecuentes se encuentran entre otros la linfadenopatía, hepatoesplenomegalia, neumonía. Para el tratamiento de la enfermedad se utilizan antibióticos de amplio espectro y el interferón gamma para el tratamiento de infecciones severas en la EGC ligada al cromosoma X. Actualmente se están realizando estudios para utilizar la terapia génica somática como posible cura de la enfermedad
Assuntos
Humanos , Doença Granulomatosa Crônica/genética , Doença Granulomatosa Crônica/tratamento farmacológico , Doenças Genéticas Ligadas ao Cromossomo X , Doença Granulomatosa Crônica/diagnósticoRESUMO
Eosinophilic cystitis is an uncommon disease in children, and its association with chronic granulomatous disease (CGD) has been previously reported in only five patients. In all those patients the disease showed either a self-limited benign course or a rapid response to corticosteroid treatment. The authors describe a child with X-linked CGD who developed eosinophilic cystitis with a recurrent course and difficult therapeutic management. The authors also discuss the pathogenesis of granuloma formation in CGD and review the literature for current therapies for these complications.
Assuntos
Cistite/etiologia , Eosinofilia/etiologia , Doença Granulomatosa Crônica/complicações , Ciclosporina/uso terapêutico , Doença Granulomatosa Crônica/tratamento farmacológico , Humanos , Imunossupressores/uso terapêutico , Lactente , Masculino , Dados de Sequência Molecular , Prednisona/uso terapêutico , Recidiva , Resultado do Tratamento , Ultrassonografia , Bexiga Urinária/diagnóstico por imagemRESUMO
The aim of this study was to investigate the effect of recombinant human interferon-gamma (rHuIFN-gamma) therapy on the release of nitric oxide (NO) by neutrophils (NEU) and mononuclear cells (MON) from patients with chronic granulomatous disease (CGD). Five patients with this rare disease received rHuIFN-gamma (50 micrograms/m2 of body surface, given by subcutaneous injection three times a week) for 6 months. Clinical and laboratory evaluations were performed before and after 1 and 6 months of rHuIFN-gamma therapy. Nitric oxide release by NEU and MON was assessed by the ability of these cells to inhibit thrombin-induced washed platelet aggregation. The nitrite (NO2-) and nitrate (NO3-) levels in the supernatant of cultured NEU and MON, as well as in plasma and urine (24 h diuresis), were quantified by high-performance liquid chromatography (HPLC). Conventional immunologic tests for assessing phagocyte and lymphocyte functions and humoral immunity were also performed. Therapy with rHuIFN-gamma for 6 months did not enhance NO synthesis by NEU or MON from the patients with CGD. The urinary but not plasma levels of NO2- and NO3- were elevated after rHuIFN-gamma therapy. Phagocyte and lymphocyte functions as well as humoral immunity were not affected by rHuIFN-gamma therapy. Although few patients were available for the study, we conclude that therapy with rHuIFN-gamma for 6 months did not enhance the synthesis of NO by NEU and MON in CGD patients. Whether the increased excretion of NO2- and NO3- in the urine of CGD patients after rHUIFN-gamma therapy reflects an induction of NO-synthase in cells other than leukocytes remains to be investigated.
Assuntos
Doença Granulomatosa Crônica/tratamento farmacológico , Interferon gama/uso terapêutico , Leucócitos Mononucleares/efeitos dos fármacos , Neutrófilos/efeitos dos fármacos , Óxido Nítrico/sangue , Adolescente , Adulto , Bioensaio , Atividade Bactericida do Sangue/efeitos dos fármacos , Relação CD4-CD8 , Estudos de Casos e Controles , Quimiotaxia de Leucócito/efeitos dos fármacos , Criança , Pré-Escolar , Feminino , Doença Granulomatosa Crônica/sangue , Humanos , Leucócitos Mononucleares/metabolismo , Linfócitos/imunologia , Masculino , Neutrófilos/metabolismo , Nitratos/sangue , Nitratos/urina , Nitritos/sangue , Nitritos/urina , Fagocitose/efeitos dos fármacos , Proteínas RecombinantesRESUMO
Gastrointestinal manifestations of chronic granulomatous disease of childhood include granulomatous inflammatory bowel disease. Severe colitis and perirectal disease developed in a 12-year-old boy with chronic granulomatous disease while he was receiving interferon gamma therapy. The boy had a deficiency of the 22 kd light chain of the cytochrome b heterodimer. After conventional medical therapy proved to be ineffective, a rapid clinical response was obtained to cyclosporine.
Assuntos
Ciclosporina/uso terapêutico , Doença Granulomatosa Crônica/tratamento farmacológico , Aspergillus fumigatus/isolamento & purificação , Criança , Grupo dos Citocromos b/deficiência , Humanos , Interferon gama/uso terapêutico , Masculino , Pneumonia Bacteriana/tratamento farmacológico , Pneumonia Bacteriana/microbiologia , Resultado do TratamentoRESUMO
We report the outpatient management of three patients with X-linked chronic granulomatous disease, two of whom had episodes of gastric outlet obstruction and another, urinary bladder obstruction. These obstructive conditions were successfully treated with 2-week courses of orally administered corticosteroids with or without the addition of orally administered clindamycin. There were no infectious or other adverse reactions.
Assuntos
Obstrução da Saída Gástrica/tratamento farmacológico , Doença Granulomatosa Crônica/tratamento farmacológico , Prednisona/uso terapêutico , Obstrução do Colo da Bexiga Urinária/tratamento farmacológico , Administração Oral , Assistência Ambulatorial , Criança , Clindamicina/administração & dosagem , Clindamicina/uso terapêutico , Humanos , Masculino , Prednisona/administração & dosagem , RecidivaAssuntos
Agamaglobulinemia/tratamento farmacológico , Doença Granulomatosa Crônica/tratamento farmacológico , Sulfaleno/uso terapêutico , Sulfanilamidas/uso terapêutico , Trimetoprima/uso terapêutico , Infecções Bacterianas/prevenção & controle , Pré-Escolar , Combinação de Medicamentos , Humanos , Lactente , Estudos RetrospectivosRESUMO
In a retrospective study, the effect of long-term treatment with sulfamethoxazole-trimethoprim was evaluated in nine male patients with chronic granulomatous disease. During this treatment, a marked reduction was observed in the number of infectious episodes, the number of causative agents, and the number of surgical interventions. Furthermore, a significant reduction in days of hospitalization per year was found. The mean observation period was six years before and four years during treatment. Transient alopecia was observed in one patient during therapy. We conclude that prophylactic treatment with sulfamethoxazole-trimethoprim is beneficial in patients with chronic granulomatous disease.