Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases

被引：0

作者：

Miguel Sampayo-Cordero

Bernat Miguel-Huguet

Almudena Pardo-Mateos

Andrea Malfettone

José Pérez-García

Antonio Llombart-Cussac

Javier Cortés

Marc Moltó-Abad

Cecilia Muñoz-Delgado

Marta Pérez-Quintana

Jordi Pérez-López

机构：

[1] Medica Scientia Innovation Research (MedSIR),Colorectal Unit, Department of Surgery

[2] Hospital de Bellvitge,Unit of Rare Diseases

[3] Albiotech consultores y redacción científica S.L.,Department of Internal Medicine

[4] IOB,Department of Internal Medicine

[5] Institute of Oncology,undefined

[6] QuironSalud Group,undefined

[7] FISABIO - Hospital Arnau de Vilanova,undefined

[8] Hospital Vall d’Hebron,undefined

[9] Hospital Gregorio Marañón,undefined

[10] Hospital San Juan de Dios,undefined

来源：

Orphanet Journal of Rare Diseases | / 14卷

关键词：

Systematic review; Meta-analysis; Case reports; Clinical studies; Mucopolysaccharidosis type II; Enzyme replacement therapy; Evidence-based medicine;

D O I：

暂无

中图分类号：

学科分类号：

摘要：

引用

共 4 条

[1] Agreement between results of meta-analyses from case reports and clinical studies, regarding efficacy and safety of idursulfase therapy in patients with mucopolysaccharidosis type II (MPS-II). A new tool for evidence-based medicine in rare diseases
Sampayo-Cordero, Miguel
Miguel-Huguet, Bernat
Pardo-Mateos, Almudena
Malfettone, Andrea
Perez-Garcia, Jose
Llombart-Cussac, Antonio
Cortes, Javier
Molto-Abad, Marc
Munoz-Delgado, Cecilia
Perez-Quintana, Marta
Perez-Lopez, Jordi
ORPHANET JOURNAL OF RARE DISEASES, 2019, 14 (01)
[2] Agreement between the results of meta-analyses from case reports and from clinical studies regarding the efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age: An example of case reports meta-analyses as an useful tool for evidence-based medicine in rare diseases
Sampayo-Cordero, Miguel
Miguel-Huguet, Bernat
Pardo-Mateos, Almudena
Molto-Abad, Marc
Munoz-Delgado, Cecilia
Perez-Lopez, Jordi
MOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : 69 - 75
[3] The Value of Case Reports in Systematic Reviews from Rare Diseases. The Example of Enzyme Replacement Therapy (ERT) in Patients with Mucopolysaccharidosis Type II (MPS-II)
Sampayo-Cordero, Miguel
Miguel-Huguet, Bernat
Malfettone, Andrea
Perez-Garcia, Jose Manuel
Llombart-Cussac, Antonio
Cortes, Javier
Pardo, Almudena
Perez-Lopez, Jordi
INTERNATIONAL JOURNAL OF ENVIRONMENTAL RESEARCH AND PUBLIC HEALTH, 2020, 17 (18) : 1 - 16
[4] The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: "The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis"
Sampayo-Cordero, Miguel
Miguel-Huguet, Bernat
Malfettone, Andrea
Perez-Garcia, Jose Manuel
Llombart-Cussac, Antonio
Cortes, Javier
Pardo, Almudena
Perez-Lopez, Jordi
FRONTIERS IN MOLECULAR BIOSCIENCES, 2021, 8

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