Trasplante de células madre hematopoyéticas para la enfermedad de Gaucher
References
Referencias de los estudios excluidos de esta revisión
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Almon E. Novel enzyme replacement therapy for Gaucher disease; on‐going phase 3 clinical trial with recombinant human glucocerebrosidase expressed in plant cells [abstract]. Proceedings of the 8th European Working Group on Gaucher Disease (EWGGD); 2008 June 4‐7th; Budapest, Hungary2008:18.
Baker KS. Study of pulmonary complications in Pediatric Patients With Storage Disorders undergoing Allogeneic Hematopoietic stem cell transplantation. www.clinicaltrials.gov.
Barranger J. Phase I study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene into peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher disease. www.clinicaltrials.gov.
de Fost M, Aerts JM, Groener JE, Maas M, Akkerman EM, Wiersma MG, et al. Low frequency maintenance therapy with imiglucerase in adult type 1 Gaucher disease: a prospective randomised controlled trial. Haematologica 2007;92(2):215‐21.
Elstein D, Abrahamov A, Hadas‐Halpern I, Meyer A, Zimran A. Low‐dose low frequency imiglucerase as a starting regimen of enzyme replacement therapy for patients with type 1 Gaucher disease. QJM:monthly journal of the Association of Physicians 1998;91(7):483‐8.
Elstein D, Heitner R, Dweck A, Attias D, Atarescu G, Zimran A. OGT918 as substrate reduction therapy in type 1 gaucher disease [abstract]. 6th Meeting of the European Hematology Association. Frankfurt , Germany, 2001:Abst 212.
Grabowski GA, Barton NW, Pastores G, Dambrosia JM, Banerjee TK, McKee MA, et al. Enzyme therapy in gaucher diseasetype1: comparative efficacy of mannose terminated glucocerebrosidase from natural and recombinant sources. Annals of Internal Medicine 1995;122(1):33‐9.
Mehta A. A multicentre, randomised, dose frequency study of the safety and efficacy of cerezyme infusions every 4 weeks versus every 2 weeks in the maintenance therapy of patients with type 1 gaucher disease [abstract]. Proceedings of the 8th European Working Group on Gaucher Disease (EWGGD); 2008 June 4‐7th; Budapest, Hungary2008:11.
Orchard P. Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation. www.clinicaltrials.gov.
Schiffmann R, Fitzgibbon EJ, Harris C, DeVile C, Davies EH, Abel L, et al. Randomized, controlled trial of miglustat in Gaucher's disease type 3. Annals of Neurology2008; Vol. 64, issue 5:514‐22.
Weinreb N on behalf of AT2101 Study Group. A phase 2 clinical trials of the pharmacological chaperone AT2101 for the treatment of Gaucher disease [abstract]. Journal of Inherited Metabolic Disease 2008;108:Abstract 427‐P.
Weinreb NJ. A phase 2 clinical trial of the pharmacological chaperone AT2101 for the treatment of gauchers disease [abstract]. Proceedings of the 8th European Working Group on Gaucher Disease (EWGGD); 2008 June 4‐7; Budapest, Hungary. 2008:14.
Wenstrup RJ, Bailey L, Grabowski GA, Moskovitz J, Oestrich AE, Wu W, et al. Gaucher disease: alendronate disodium improves bone mineral density in adults receiving enzyme therapy. Blood 2004;104(5):1253‐7.
Referencias adicionales
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August CS, Palmieri M, Nowell P, Elkins WL, D'Angio G, Glew RH, et al. Bone marrow transplantation (BMT) in Gaucher's disease. Paediatric Research 1984;18:236A.
Beutler E, Grabowski GA. Gaucher Disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D, Childs B, Kinzler KW, Vogelstein B editor(s). The Metabolic and Molecular Bases of Inherited Disease. 8th Edition. Vol. III, New York: McGraw‐Hill, 2001:3635‐68.
Beutler E. The treatment of Gaucher disease in countries with limited health care resources. Indian Journal of Human Genetics 2005;11(3):121‐7.
Beutler E. Gaucher disease: multiple lessons from a single gene disorder. Acta Paediatrica 2006;Suppl 451:103‐9.
Chan KW, Wong LTK, Applegarth D, Davidson AGF. Bone marrow transplantation in Gaucher's disease: effect of mixed chimeric state. Bone Marrow Transplantation 1994;14(2):327‐30.
Erikson A, Johansson K, Monsson JE, Svennerholm L. Enzyme replacement therapy of infantile Gaucher disease. Neuropediatrics 1993;24(4):237‐8.
Higgins JPT, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta‐analyses. BMJ 2003;327(7414):557‐60.
Higgins JPT, Altman DG (editors). Chapter 8: Assessing risk of bias in included studies. In: Higgins JPT, Green S (editors).. Cochrane Handbook for Systematic Reviews of Interventions Version 5.0.0 (updated February 2008). The Cochrane Collaboration, 2008. Available from www.cochrane‐handbook.org.
Hobbs JR, Jones KH, Shaw PJ, Lindsay I, Hancock M. Beneficial effect of pre‐transplant splenectomy on displacement bone marrow transplantation for Gaucher's syndrome. Lancet 1987;1(8542):1111‐5.
Jmoudiak M, Futerman AH. Gaucher disease: pathological mechanisms and modern management. British Journal of Haematology 2005;129(2):178‐88.
Kumar L. Haematopoietic stem cell transplantation: current status. National Medical Journal of India 2007;20(3):128‐37.
Mehta A. Epidemiology and natural history of Gaucher's disease. European Journal of Internal Medicine 2006;17:S2‐S5.
Morales LE. Gaucher's Disease: A Review. Annals of Pharmacotherapy 1996;30(4):381‐8.
Parmar MKB, Torri V, Stewart L. Extracting summary statistics to perform meta‐analyses of the published literature for survival end‐points. Statistics in Medicine 1998;17(24):2815‐34.
Peters C, Steward CG. Haematopoietic stem cell transplantation for Inherited metabolic diseases : an overview of outcomes and practice guidelines. Bone Marrow Transplantation 2003;31(4):229‐39.
Rappeport JM, Ginns EI. Bone‐marrow transplantation In severe Gaucher's disease. New England Journal of Medicine 1984;311(2):84‐8.
Ringden O, Groth CJ, Erikson A, Backman L, Granqvist S, Mansson JE, et al. Long‐term follow‐up of the first successful Bone marrow transplantation in Gaucher disease. Transplantation 1988;46(1):66‐70.
Ringden O, Groth CG, Erikson A, Granqvist S, Mansson JE, Sparrelid E. Ten years experience of bone marrow transplantation for gaucher disease. Transplantation 1995;59(6):864‐70.
Schmitz J, Poll LW, Dahl SV. Therapy of adult Gaucher disease. Haematologica 2007;92(2):148‐52.
Starer F, Sargent JD, Hobbs JR. Regression of the radiological changes of Gaucher's disease following bone marrow transplantation. British Journal of Radiology 1987;60(720):1189‐95.
Steward CG, Jarisch A. Haemopoietic stem cell transplantation for genetic disorders. Archives of Disease in Childhood 2005;90(12):1259‐63.
Svennerholm L, Erikson A, Groth CG, Ringden O, Mansson JE. Norbottnian type of Gaucher disease ‐ Clinical, Biochemical and Molecular Biology aspects: Successful treatment with Bone marrow transplantation. Developmental Neuroscience 1991;13(4‐5):345‐51.
Tsai P, Lipton JM, Sahdev I, Najfeld V, Rankin LR, Slyper AH, et al. Allogeneic Bone marrow transplantation in Severe Gaucher disease. Pediatric Research 1992;31(5):503‐7.
Young E, Chatterton C, Vellodi A, Winchester B. Plasma chitotriosidase activity in Gaucher disease patients who have been treated either by bone marrow transplantation or by enzyme replacement therapy with alglucerase. Journal of Inherited Metabolic Disease 1997;20(4):595‐602.
Zimran A, Elstein D, Abrahamov A. Enzyme replacement therapy in type 1 and type 3 Gaucher's disease. Lancet 1995;345(8947):451‐2.
Characteristics of studies
Characteristics of excluded studies [ordered by study ID]
| Study | Reason for exclusion |
| Intervention not relevant, enzyme replacement therapy trial. | |
| Not an RCT/Quasi/CCT. Outcomes not relevant | |
| Not an RCT/Quasi/CCT. Intervention not relevant. | |
| Intervention not relevant, enzyme replacement therapy trial. | |
| Intervention not relevant, enzyme replacement therapy trial. | |
| Intervention not relevant, enzyme replacement therapy trial. | |
| Intervention not relevant, enzyme replacement therapy trial. | |
| Intervention not relevant, enzyme replacement therapy trial. | |
| Not an RCT/quasi/CCT. | |
| Intervention not relevant, Miglustat trial. | |
| Intervention not relevant, AT2101(pharmacological chaperone) therapy trial. | |
| Intervention not relevant, enzyme replacement therapy trial. |
CCT: clinical controlled trial
RCT: randomised controlled trial
