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A Study Using Real-world Data to Examine Outcomes in Children and Adults Who Develop a Blood Clotting Disorder Called Thrombotic Microangiopathy (TMA) After Undergoing a Hematopoietic Stem Cell Transplant (HSCT).
Sponsor: Alexion Pharmaceuticals, Inc.
Summary
The purpose of this study was to assess OS outcomes in pediatric (≥ 28 days of age at the time of HSCT-TMA diagnosis) and adult participants diagnosed with HSCT-TMA(referred to as TMA from hereon) within 52 weeks after HSCT and who were either complement inhibitor treatment naïve or had been treated with eculizumab.
Official title: A Secondary Real-world Data Study of Pediatric and Adult Participants With Thrombotic Microangiopathy (TMA) After Hematopoietic Stem Cell Transplant (HSCT)
Key Details
Gender
All
Age Range
4 Weeks - Any
Study Type
OBSERVATIONAL
Enrollment
307
Start Date
2025-01-22
Completion Date
2026-03-31
Last Updated
2026-07-28
Healthy Volunteers
No
Locations (43)
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Boston, Massachusetts, United States
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Akron, Ohio, United States
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Salt Lake City, Utah, United States
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Brussels, Brussels Capital, Belgium
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Liège, Belgium
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Fortaleza, Ceará, Brazil
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Brasília, Federal District, Brazil
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Curitiba, Paraná, Brazil
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Recife, Pernambuco, Brazil
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Porto Alegre, Rio Grande do Sul, Brazil
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Jaú, São Paulo, Brazil
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Rio de Janeiro, Brazil
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São Paulo, Brazil
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São Paulo, Brazil
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São Paulo, Brazil
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São Paulo, Brazil
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Pessac, Gironde, France
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Toulouse, Haute Garonne, France
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Grenoble, Isere, France
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Paris, France
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Thessaloniki, Central Macedonia, Greece
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Pátrai, West Greece, Greece
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Rome, Lazio, Italy
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Ancona, Italy
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Brescia, Italy
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Florence, Italy
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Genova, Italy
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Naples, Italy
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Pavia, Italy
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Roma, Italy
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Torino, Italy
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Fukushima, Fukushima, Japan
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Tsukuba, Ibaraki, Japan
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Saitama-shi, Saitama, Japan
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Wakayama, Wakayama, Japan
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Seoul, South Korea
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Seoul, South Korea
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Seoul, South Korea
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Barcelona, Spain
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Barcelona, Spain
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London, Greater London, United Kingdom
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Newcastle, Tyne & Wear, United Kingdom
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Birmingham, West Midlands, United Kingdom