Received: 15 March 2017
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Revised: 22 May 2017
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Accepted: 23 May 2017
DOI: 10.1002/ajh.24801
A JH
RESEARCH ARTICLE
Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pramod K. Mistry1
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Julie L. Batista2 | Hans C. Andersson3 | Manisha Balwani4 |
Thomas Andrew Burrow5 | Joel Charrow6 | Paige Kaplan7 | Aneal Khan8 | Priya S. Kishnani9 | Edwin H. Kolodny10 | Barry Rosenbloom11 | C. Ronald Scott12 | Neal Weinreb13 1
Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut; 2Sanofi Genzyme, Cambridge, Massachusetts; 3Hayward Genetics Center, Tulane University Medical School, New Orleans, Louisiana; 4Icahn School of Medicine at Mount Sinai, New York City, New York; 5College of Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas; 6Northwestern University Feinberg School of Medicine, Chicago, Illinois; 7Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania; 8The University of Calgary, Calgary, Alberta, Canada; 9 Division of Medical Genetics, Duke University School of Medicine, Durham, North Carolina; 10New York University School of Medicine, New York, New York; 11 Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, California; 12 University of Washington, Seattle, Washington; 13Departments of Human Genetics and Medicine (Hematology), University of Miami Miller School of Medicine, Miami, Florida
Abstract This study tests the hypothesis that the prevalence of severe clinical manifestations in Gaucher disease type 1 (GD1) patients at the time of treatment initiation has changed since alglucerase/ imiglucerase enzyme replacement therapy (ERT) was approved in the United States (US) in 1991. US alglucerase/imiglucerase-treated GD1 patients from the International Collaborative Gaucher Group Gaucher Registry clinicaltrials.gov NCT00358943 were stratified by age at ERT initiation (
|
Revised: 22 May 2017
|
Accepted: 23 May 2017
DOI: 10.1002/ajh.24801
A JH
RESEARCH ARTICLE
Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry Pramod K. Mistry1
|
Julie L. Batista2 | Hans C. Andersson3 | Manisha Balwani4 |
Thomas Andrew Burrow5 | Joel Charrow6 | Paige Kaplan7 | Aneal Khan8 | Priya S. Kishnani9 | Edwin H. Kolodny10 | Barry Rosenbloom11 | C. Ronald Scott12 | Neal Weinreb13 1
Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut; 2Sanofi Genzyme, Cambridge, Massachusetts; 3Hayward Genetics Center, Tulane University Medical School, New Orleans, Louisiana; 4Icahn School of Medicine at Mount Sinai, New York City, New York; 5College of Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas; 6Northwestern University Feinberg School of Medicine, Chicago, Illinois; 7Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania; 8The University of Calgary, Calgary, Alberta, Canada; 9 Division of Medical Genetics, Duke University School of Medicine, Durham, North Carolina; 10New York University School of Medicine, New York, New York; 11 Cedars-Sinai/Tower Hematology Oncology, Beverly Hills, California; 12 University of Washington, Seattle, Washington; 13Departments of Human Genetics and Medicine (Hematology), University of Miami Miller School of Medicine, Miami, Florida
Abstract This study tests the hypothesis that the prevalence of severe clinical manifestations in Gaucher disease type 1 (GD1) patients at the time of treatment initiation has changed since alglucerase/ imiglucerase enzyme replacement therapy (ERT) was approved in the United States (US) in 1991. US alglucerase/imiglucerase-treated GD1 patients from the International Collaborative Gaucher Group Gaucher Registry clinicaltrials.gov NCT00358943 were stratified by age at ERT initiation (
