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1.
N Engl J Med ; 386(24): 2295-2302, 2022 06 16.
Artigo em Inglês | MEDLINE | ID: mdl-35704481

RESUMO

Lifelong immunosuppression is required for allograft survival after kidney transplantation but may not ultimately prevent allograft loss resulting from chronic rejection. We developed an approach that attempts to abrogate immune rejection and the need for post-transplantation immunosuppression in three patients with Schimke immuno-osseous dysplasia who had both T-cell immunodeficiency and renal failure. Each patient received sequential transplants of αß T-cell-depleted and CD19 B-cell-depleted haploidentical hematopoietic stem cells and a kidney from the same donor. Full donor hematopoietic chimerism and functional ex vivo T-cell tolerance was achieved, and the patients continued to have normal renal function without immunosuppression at 22 to 34 months after kidney transplantation. (Funded by the Kruzn for a Kure Foundation.).


Assuntos
Transplante de Células-Tronco Hematopoéticas , Síndromes de Imunodeficiência , Transplante de Rim , Síndrome Nefrótica , Osteocondrodisplasias , Doenças da Imunodeficiência Primária , Arteriosclerose/genética , Arteriosclerose/terapia , Rejeição de Enxerto/prevenção & controle , Humanos , Síndromes de Imunodeficiência/terapia , Rim/fisiologia , Transplante de Rim/efeitos adversos , Síndrome Nefrótica/genética , Síndrome Nefrótica/terapia , Osteocondrodisplasias/genética , Osteocondrodisplasias/terapia , Doenças da Imunodeficiência Primária/genética , Doenças da Imunodeficiência Primária/terapia , Embolia Pulmonar/genética , Embolia Pulmonar/terapia , Condicionamento Pré-Transplante/métodos
2.
Biol Blood Marrow Transplant ; 23(9): 1437-1446, 2017 Sep.
Artigo em Inglês | MEDLINE | ID: mdl-28495643

RESUMO

Although T cell immune reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been well studied, long-term B cell immune reconstitution remains less characterized. We evaluated humoral immune reconstitution among 71 pediatric allo-HSCT recipients. Although tetanus toxoid antibody levels were normal at 1 year after allo-HSCT, antipolysaccharide carbohydrate antibodies remained persistently low for up to 5 years. While naive B cell counts normalized by 6 months, IgM memory B cell deficiency persisted for up to 2 years (P = .01); switched memory B cell deficiency normalized by 1 year after allo-HSCT. CD4+ T cell immune reconstitution correlated with that of switched memory B cells as early as 6 months after allo-HSCT (r = .55, P = .002) but did not correlate with IgM memory B cells at any time point after allo-HSCT. Taken together, this suggests that allo-HSCT recipients have impaired antibody immune reconstitution, mainly due to IgM memory B cell maturation block, compared with more prompt T cell-dependent switched memory cell immune reconstitution. We further explored other factors that might affect humoral immune reconstitution. The use of total body irradiation was associated with lower naive B cells counts at 6 months after HSCT (P = .04) and lower IgM (P = .008) and switched (P = .003) memory B cells up to 2 years. Allo-HSCT recipients with extensive chronic graft-versus-host disease had lower IgM memory B cell counts (P = .03) up to 2 years after allo-HSCT. The use of cord blood was associated with better naive (P = .01), IgM (P = .0005), and switched memory (P = .006) B cells immune reconstitution. These findings may inform future prophylaxis and treatment strategies regarding risk of overwhelming infection, graft-versus-host disease, and post-allogeneic HSCT revaccination.


Assuntos
Transplante de Células-Tronco de Sangue do Cordão Umbilical , Neoplasias Hematológicas/imunologia , Transplante de Células-Tronco Hematopoéticas , Hemoglobinopatias/imunologia , Reconstituição Imune/efeitos da radiação , Imunidade Inata/efeitos da radiação , Síndromes de Imunodeficiência/imunologia , Linfócitos B/efeitos dos fármacos , Linfócitos B/imunologia , Linfócitos B/patologia , Linfócitos B/efeitos da radiação , Linfócitos T CD4-Positivos/efeitos dos fármacos , Linfócitos T CD4-Positivos/efeitos da radiação , Criança , Pré-Escolar , Feminino , Seguimentos , Doença Enxerto-Hospedeiro/prevenção & controle , Neoplasias Hematológicas/patologia , Neoplasias Hematológicas/terapia , Hemoglobinopatias/patologia , Hemoglobinopatias/terapia , Humanos , Imunidade Inata/efeitos dos fármacos , Imunoglobulina M/sangue , Imunoglobulina M/deficiência , Síndromes de Imunodeficiência/patologia , Síndromes de Imunodeficiência/terapia , Memória Imunológica/efeitos dos fármacos , Memória Imunológica/efeitos da radiação , Lactente , Cinética , Masculino , Agonistas Mieloablativos/uso terapêutico , Estudos Retrospectivos , Condicionamento Pré-Transplante/métodos , Transplante Homólogo , Irradiação Corporal Total
3.
Biol Blood Marrow Transplant ; 23(2): 211-234, 2017 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-27713092

RESUMO

Chronic graft-versus-host disease (GVHD) is the leading cause of late, nonrelapse mortality and disability in allogeneic hematopoietic cell transplantation recipients and a major obstacle to improving outcomes. The biology of chronic GVHD remains enigmatic, but understanding the underpinnings of the immunologic mechanisms responsible for the initiation and progression of disease is fundamental to developing effective prevention and treatment strategies. The goals of this task force review are as follows: This document is intended as a review of our understanding of chronic GVHD biology and therapies resulting from preclinical studies, and as a platform for developing innovative clinical strategies to prevent and treat chronic GVHD.


Assuntos
Ensaios Clínicos como Assunto/normas , Doença Enxerto-Hospedeiro , Aloenxertos , Animais , Biomarcadores , Doença Crônica , Citocinas/metabolismo , Endotélio Vascular/patologia , Fibrose , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/imunologia , Doença Enxerto-Hospedeiro/patologia , Doença Enxerto-Hospedeiro/terapia , Humanos , Inflamação , Interferon gama/fisiologia , Camundongos , Modelos Animais , Modelos Imunológicos , Especificidade de Órgãos , Subpopulações de Linfócitos T/imunologia , Terminologia como Assunto , Imunologia de Transplantes , Cicatrização
4.
Biol Blood Marrow Transplant ; 21(5): 780-92, 2015 May.
Artigo em Inglês | MEDLINE | ID: mdl-25644957

RESUMO

Biology-based markers to confirm or aid in the diagnosis or prognosis of chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic cell transplantation or monitor its progression are critically needed to facilitate evaluation of new therapies. Biomarkers have been defined as any characteristic that is objectively measured and evaluated as an indicator of a normal biological or pathogenic process, or of a pharmacologic response to a therapeutic intervention. Applications of biomarkers in chronic GVHD clinical trials or patient management include the following: (1) diagnosis and assessment of chronic GVHD disease activity, including distinguishing irreversible damage from continued disease activity; (2) prognostic risk to develop chronic GVHD; and (3) prediction of response to therapy. Sample collection for chronic GVHD biomarkers studies should be well documented following established quality control guidelines for sample acquisition, processing, preservation, and testing, at intervals that are both calendar and event driven. The consistent therapeutic treatment of subjects and standardized documentation needed to support biomarker studies are most likely to be provided in prospective clinical trials. To date, no chronic GVHD biomarkers have been qualified for use in clinical applications. Since our previous chronic GVHD Biomarkers Working Group report in 2005, an increasing number of chronic GVHD candidate biomarkers are available for further investigation. This paper provides a 4-part framework for biomarker investigations: identification, verification, qualification, and application with terminology based on Food and Drug Administration and European Medicines Agency guidelines.


Assuntos
Biomarcadores/metabolismo , Ensaios Clínicos como Assunto , Doença Enxerto-Hospedeiro/diagnóstico , Doença Enxerto-Hospedeiro/metabolismo , Doença Enxerto-Hospedeiro/terapia , Humanos , Prognóstico , Terminologia como Assunto , Estados Unidos , United States Food and Drug Administration
5.
Blood ; 122(5): 623-4, 2013 Aug 01.
Artigo em Inglês | MEDLINE | ID: mdl-23908441

RESUMO

In this issue of Blood, Dertschnig and colleagues1 demonstrate in mice that acute graft-versus-host disease (GHVD) results in a marked reduction of autoimmune receptor­expressing medullary thymic epithelial cells (Aire1 mTEC) and a decrease in the diversity of Aire-dependent tissue-restricted peripheral selfantigens (TRAs) required for effective negative thymic selection. Both of these abnormalities are reversed by the peritransplant administration of the epithelial protectant drug, fibroblast growth factor 7(Fgf7).


Assuntos
Antígenos/genética , Células Epiteliais/imunologia , Doença Enxerto-Hospedeiro/genética , Doença Enxerto-Hospedeiro/imunologia , Timo/metabolismo , Animais , Feminino
6.
J Allergy Clin Immunol ; 133(2): 335-47, 2014 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-24139498

RESUMO

The Primary Immune Deficiency Treatment Consortium (PIDTC) is a network of 33 centers in North America that study the treatment of rare and severe primary immunodeficiency diseases. Current protocols address the natural history of patients treated for severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome, and chronic granulomatous disease through retrospective, prospective, and cross-sectional studies. The PIDTC additionally seeks to encourage training of junior investigators, establish partnerships with European and other International colleagues, work with patient advocacy groups to promote community awareness, and conduct pilot demonstration projects. Future goals include the conduct of prospective treatment studies to determine optimal therapies for primary immunodeficiency diseases. To date, the PIDTC has funded 2 pilot projects: newborn screening for SCID in Navajo Native Americans and B-cell reconstitution in patients with SCID after hematopoietic stem cell transplantation. Ten junior investigators have received grant awards. The PIDTC Annual Scientific Workshop has brought together consortium members, outside speakers, patient advocacy groups, and young investigators and trainees to report progress of the protocols and discuss common interests and goals, including new scientific developments and future directions of clinical research. Here we report the progress of the PIDTC to date, highlights of the first 2 PIDTC workshops, and consideration of future consortium objectives.


Assuntos
Síndromes de Imunodeficiência , Transplante de Células-Tronco Hematopoéticas , Humanos , Síndromes de Imunodeficiência/diagnóstico , Síndromes de Imunodeficiência/terapia , Recém-Nascido , Triagem Neonatal , Projetos Piloto , Sociedades Científicas
7.
Biol Blood Marrow Transplant ; 20(10): 1508-15, 2014 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-24979733

RESUMO

To determine the role of regulatory T lymphocytes (Tregs) in the pathogenesis of human chronic graft-versus-host disease (GVHD) and its clinical resolution, we evaluated long-term recipients of pediatric allogeneic hematopoietic stem cell transplantation (HSCT). Seventy-one recipients were evaluated, 30 of whom had a history of chronic GVHD, including 16 with active chronic GVHD and 14 with resolved chronic GVHD. There were no significant clinical differences and no differences in the frequency of Tregs (CD4(+), CD127(-), CD25(+)) between the recipients with active chronic GVHD and those with resolved chronic GVHD. Using the Miyara/Sakaguchi classification scheme to identify functional Tregs, a decreased frequency of functional resting Tregs (rTregs) was identified in recipients with active chronic GVHD (P = .009 compared with normal donors; P = .001 compared with HSCT recipients without history of chronic GVHD; P = .005 compared with recipients with resolved chronic GVHD). The frequency and number of recent thymic emigrants in rTregs were normal in recipients with resolved chronic GVHD, but persistently decreased in recipients with active chronic GVHD. These results support the hypothesis that the reestablishment of normal numbers of functional rTregs is required for the clinical resolution of chronic GVHD.


Assuntos
Doença Enxerto-Hospedeiro/terapia , Doenças Hematológicas/terapia , Transplante de Células-Tronco Hematopoéticas , Linfócitos T Reguladores/imunologia , Condicionamento Pré-Transplante , Adolescente , Antígenos CD/genética , Antígenos CD/imunologia , Contagem de Linfócito CD4 , Proliferação de Células , Criança , Pré-Escolar , Doença Crônica , Estudos Transversais , Feminino , Expressão Gênica , Doença Enxerto-Hospedeiro/genética , Doença Enxerto-Hospedeiro/imunologia , Doença Enxerto-Hospedeiro/patologia , Doenças Hematológicas/genética , Doenças Hematológicas/imunologia , Doenças Hematológicas/patologia , Humanos , Lactente , Masculino , Agonistas Mieloablativos/uso terapêutico , Linfócitos T Reguladores/efeitos dos fármacos , Linfócitos T Reguladores/patologia , Transplante Homólogo
8.
Blood ; 120(18): 3635-46, 2012 Nov 01.
Artigo em Inglês | MEDLINE | ID: mdl-22968453

RESUMO

We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34(+) cells. Four subjects were treated without pretransplantation cytoreduction and remained on ADA enzyme-replacement therapy (ERT) throughout the procedure. Only transient (months), low-level (< 0.01%) gene marking was observed in PBMCs of 2 older subjects (15 and 20 years of age), whereas some gene marking of PBMC has persisted for the past 9 years in 2 younger subjects (4 and 6 years). Six additional subjects were treated using the same gene transfer protocol, but after withdrawal of ERT and administration of low-dose busulfan (65-90 mg/m(2)). Three of these remain well, off ERT (5, 4, and 3 years postprocedure), with gene marking in PBMC of 1%-10%, and ADA enzyme expression in PBMC near or in the normal range. Two subjects were restarted on ERT because of poor gene marking and immune recovery, and one had a subsequent allogeneic hematopoietic stem cell transplantation. These studies directly demonstrate the importance of providing nonmyeloablative pretransplantation conditioning to achieve therapeutic benefits with gene therapy for ADA-deficient severe combined immunodeficiency.


Assuntos
Agamaglobulinemia/terapia , Transplante de Medula Óssea/métodos , Terapia Genética/métodos , Vetores Genéticos , Transplante de Células-Tronco Hematopoéticas/métodos , Imunodeficiência Combinada Severa/terapia , Adenosina Desaminase/deficiência , Adolescente , Antígenos CD34/metabolismo , Criança , Pré-Escolar , Feminino , Humanos , Lactente , Masculino , Retroviridae/genética , Transdução Genética , Condicionamento Pré-Transplante , Adulto Jovem
9.
Clin Immunol ; 149(1): 146-55, 2013 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-23994768

RESUMO

Multi-center evaluations of pediatric patients with juvenile systemic sclerosis (jSSc) have suggested that the pathogenesis of jSSc may differ from that of systemic sclerosis (SSc) in adult patients. Therefore, we undertook to identify abnormalities in the T lymphocytes of jSSc patients and to determine if they differed from the abnormalities reported in the T lymphocytes of adult SSc patients. We identified decreases in the frequency of resting regulatory T lymphocytes and an increased frequency of CD45RA expressing effector memory (EMRA) CD4 T lymphocytes, which were characterized by an increased frequency of CCR7 protein expressing cells. Neither the increases in the EMRA subpopulation nor the increased CCR7 protein expression have been reported in adult SSc patients. The decrease in resting regulatory T lymphocytes in jSSc patients may permit the expansion of the disease initiating CD4 T lymphocytes present in the CCR7 expressing EMRA CD4 T lymphocyte subpopulation.


Assuntos
Linfócitos T CD4-Positivos/imunologia , Escleroderma Sistêmico/imunologia , Subpopulações de Linfócitos T/imunologia , Adolescente , Adulto , Criança , Feminino , Perfilação da Expressão Gênica , Humanos , Masculino , Análise de Sequência com Séries de Oligonucleotídeos , Receptores CCR7/genética , Escleroderma Sistêmico/genética , Adulto Jovem
10.
J Clin Immunol ; 31(4): 615-22, 2011 Aug.
Artigo em Inglês | MEDLINE | ID: mdl-21541793

RESUMO

Autologous hematopoietic stem cell transplantation (HSCT) has been used for the treatment of both adult and pediatric autoimmune diseases. However, HSCT has significant side effects (neutropenia, thrombocytopenia, infertility, cardiotoxicity) and costs (HSC collection/harvesting, blood product support). In an attempt to avoid the toxicities and costs associated with HSCT, we investigated whether immune ablation similar to that achieved following myeloablative HSCT could be achieved by the intensive administration of an anti-CD52 antibody (Campath-1H antibody). The first patient treated with the treatment regime, who had refractory juvenile polymyositis, achieved immune ablation (the elimination of pre-therapy antigen-specific T lymphocyte immunity) and has had stable clinical improvement for more than 6 years.


Assuntos
Anticorpos Monoclonais/uso terapêutico , Antígenos CD/imunologia , Antígenos de Neoplasias/imunologia , Glicoproteínas/imunologia , Polimiosite/terapia , Adolescente , Alemtuzumab , Anticorpos Monoclonais/administração & dosagem , Anticorpos Monoclonais/imunologia , Anticorpos Monoclonais Humanizados/administração & dosagem , Anticorpos Monoclonais Humanizados/farmacologia , Anticorpos Monoclonais Humanizados/uso terapêutico , Anticorpos Antineoplásicos/administração & dosagem , Anticorpos Antineoplásicos/farmacologia , Anticorpos Antineoplásicos/uso terapêutico , Doenças Autoimunes/imunologia , Doenças Autoimunes/terapia , Antígeno CD52 , Feminino , Humanos , Polimiosite/imunologia
12.
J Clin Immunol ; 29(2): 231-7, 2009 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-18807155

RESUMO

BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is the only cure for patients with severe combined immunodeficiency (SCID). The purpose of this study was to evaluate long-term neurodevelopment of patients with SCID following myeloablative chemotherapy and HSCT. MATERIALS AND METHODS: Sixteen pediatric patients diagnosed with SCID were tested using the Bayley Scales of Infant Development and the validated Vineland Adaptive Behavior Scales (VABS) pre- and 1-year post-HSCT. Three years post-HSCT, there were 11 patients available for testing and four patients available 5 years post-HSCT. Patients greater than 3 years of age were administered the Wechsler Preschool and Primary Scale of Intelligence. Both raw scores and scaled scores were analyzed. RESULTS: There was a significant decrease 1 year post-HSCT in the Bayley Mental Developmental Index (MDI) [92.5 (pre) vs. 70.81 (1 year post), p < 0.0001] and the VABS [99.73 (pre) vs. 79.87 (1 year post), p = <0.0001]. There was a significant decrease over time in the MDI [95.00 (pre) vs. 72.64 (1 year post) vs. 71.82 (3 years post), p < 0.0001], but no significant change between 1 and 3 years post-HSCT. There was no change in the Bayley Psychomotor Development Scale (PDI) [82.4 (pre) vs. 84.8 (1 year post), p = 0.68]. The PDI scores decreased over time [86.29 (pre) vs. 86 (1 year post) vs. 74.14 (3 years post), p = 0.045]. Although there was a decrease in scaled scores, there was not a loss of skills. Analysis of raw scores showed that there was an increase in the raw test scores, which indicated that these children acquired developmental skills, but at a slower rate than normal infants and toddlers. Younger children had a more significant decrease in adaptive scores compared with older children. CONCLUSIONS: These findings may reflect the effects of the isolation and prolonged hospitalization that characterizes the immediate post-transplant period. Patients miss out on social interactions and learning opportunities that normally occur at their respective stages of development. These restrictions keep patients from acquiring developmentally appropriate cognitive skills as well as gross and fine motor developmental milestones. Longitudinal follow-up will be important to quantify acquisition of skills.


Assuntos
Adaptação Psicológica , Cognição , Transplante de Células-Tronco Hematopoéticas , Desempenho Psicomotor , Imunodeficiência Combinada Severa/psicologia , Imunodeficiência Combinada Severa/cirurgia , Alemtuzumab , Anticorpos Monoclonais/uso terapêutico , Anticorpos Monoclonais Humanizados , Anticorpos Antineoplásicos/uso terapêutico , Antineoplásicos/uso terapêutico , Bussulfano/uso terapêutico , Desenvolvimento Infantil , Ciclofosfamida/uso terapêutico , Seguimentos , Humanos , Lactente , Agonistas Mieloablativos/uso terapêutico , Testes Neuropsicológicos , Imunodeficiência Combinada Severa/tratamento farmacológico , Resultado do Tratamento
13.
Pediatr Blood Cancer ; 52(1): 139-42, 2009 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-18819128

RESUMO

Hemophagocytic lymphohistiocytosis (HLH) is a rare autosomal recessive disorder of infancy and childhood that is invariably fatal if not treated. We report on the first patient to receive post-natal HSCT for HLH after receiving in utero chemotherapy for disease stabilization.


Assuntos
Linfo-Histiocitose Hemofagocítica/diagnóstico , Linfo-Histiocitose Hemofagocítica/terapia , Antineoplásicos/uso terapêutico , Transtornos Cognitivos , Deficiências do Desenvolvimento , Feminino , Terapias Fetais , Feto , Seguimentos , Transplante de Células-Tronco Hematopoéticas , Humanos , Recém-Nascido , Linfo-Histiocitose Hemofagocítica/fisiopatologia , Gravidez
14.
J Allergy Clin Immunol ; 122(6): 1087-96, 2008 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-18992926

RESUMO

Allogeneic hematopoietic cell transplantation (HCT) has been used for 40 years to ameliorate or cure primary immune deficiency (PID) diseases, including severe combined immunodeficiency (SCID) and non-SCID PID. There is a critical need for evaluation of the North American experience of different HCT approaches for these diseases to identify best practices and plan future investigative clinical trials. Our survey of incidence and prevalence of PID in North American practice sites indicates that such studies are feasible. A conference of experts in HCT treatment of PID has recommended (1) a comprehensive cross-sectional and retrospective analysis of HCT survivors with SCID; (2) a prospective study of patients with SCID receiving HCT, with comparable baseline and follow-up testing across participating centers; (3) a pilot study of newborn screening for SCID to identify affected infants before compromise by infection; and (4) studies of the natural history of disease in patients who do or do not receive HCT for the non-SCID diseases of Wiskott-Aldrich syndrome and chronic granulomatous disease. To accomplish these goals, collaboration by a consortium of institutions in North America is proposed. Participation of immunologists and HCT physicians having interest in PID and experts in laboratory methods, clinical outcomes assessment, databases, and analysis will be required for the success of these studies.


Assuntos
Transplante de Células-Tronco Hematopoéticas , Síndromes de Imunodeficiência/terapia , Bases de Dados Factuais , Intervalo Livre de Doença , Educação , Seguimentos , Transplante de Células-Tronco Hematopoéticas/tendências , Humanos , Síndromes de Imunodeficiência/genética , Síndromes de Imunodeficiência/imunologia , Síndromes de Imunodeficiência/mortalidade , Recém-Nascido , Estudos Multicêntricos como Assunto , América do Norte , Projetos Piloto , Taxa de Sobrevida , Transplante Homólogo
17.
Hematol Oncol Clin North Am ; 25(1): 17-30, 2011 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-21236387

RESUMO

It is now more than 40 years since the first successful allogeneic hematopoietic stem cell transplantation (HSCT) for a child with severe combined immunodeficiency (SCID). In the succeeding years, HSCT for SCID patients have represented only a small portion of the total number of allogeneic HSCT performed. Nevertheless, the clinical and biologic importance of the patients transplanted for SCID has continued. SCID patients were the first to be successfully transplanted with nonsibling related bone marrow, unrelated bone marrow, T-cell depleted HSCT, and genetically corrected (gene transfer) autologous HSC. Many of the biologic insights now widely applied to allogeneic HSCT were first identified in the transplantation of SCID patients. This article reviews the clinical and biologic lessons that have been learned from HSCT for SCID patients, and how the information has impacted the general field of allogeneic HSCT.

18.
Immunol Allergy Clin North Am ; 30(1): 17-30, 2010 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-20113884

RESUMO

It is now more than 40 years since the first successful allogeneic hematopoietic stem cell transplantation (HSCT) for a child with severe combined immunodeficiency (SCID). In the succeeding years, HSCT for SCID patients have represented only a small portion of the total number of allogeneic HSCT performed. Nevertheless, the clinical and biologic importance of the patients transplanted for SCID has continued. SCID patients were the first to be successfully transplanted with nonsibling related bone marrow, unrelated bone marrow, T-cell depleted HSCT, and genetically corrected (gene transfer) autologous HSC. Many of the biologic insights now widely applied to allogeneic HSCT were first identified in the transplantation of SCID patients. This article reviews the clinical and biologic lessons that have been learned from HSCT for SCID patients, and how the information has impacted the general field of allogeneic HSCT.


Assuntos
Transplante de Células-Tronco Hematopoéticas/métodos , Imunodeficiência Combinada Severa/terapia , Células da Medula Óssea/imunologia , Células da Medula Óssea/patologia , Criança , Pré-Escolar , Seleção do Doador , Técnicas de Transferência de Genes , Doença Enxerto-Hospedeiro/etiologia , Doença Enxerto-Hospedeiro/imunologia , Transplante de Células-Tronco Hematopoéticas/efeitos adversos , Células-Tronco Hematopoéticas/imunologia , Células-Tronco Hematopoéticas/patologia , Humanos , Lactente , Depleção Linfocítica/métodos , Imunodeficiência Combinada Severa/imunologia , Linfócitos T/citologia , Linfócitos T/imunologia , Doadores de Tecidos , Transplante Autólogo , Transplante Homólogo
19.
Cell Stem Cell ; 7(1): 43-9, 2010 Jul 02.
Artigo em Inglês | MEDLINE | ID: mdl-20621049

RESUMO

A report by the International Society for Stem Cell Research (ISSCR)'s Task Force on Unproven Stem Cell Treatments outlines development of resources for patients, their families, and physicians seeking information on stem cell treatments.


Assuntos
Pesquisas com Embriões/ética , Pesquisas com Embriões/legislação & jurisprudência , Internet , Células-Tronco , Guias como Assunto , Humanos , Sociedades Médicas
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