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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

Overview of attention for article published in New England Journal of Medicine, October 2017
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  • In the top 5% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (99th percentile)
  • High Attention Score compared to outputs of the same age and source (97th percentile)

Mentioned by

news
134 news outlets
blogs
11 blogs
policy
1 policy source
twitter
225 X users
patent
2 patents
facebook
19 Facebook pages
wikipedia
1 Wikipedia page
googleplus
1 Google+ user

Citations

dimensions_citation
422 Dimensions

Readers on

mendeley
499 Mendeley
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Title
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Published in
New England Journal of Medicine, October 2017
DOI 10.1056/nejmoa1700554
Pubmed ID
Authors

Florian Eichler, Christine Duncan, Patricia L Musolino, Paul J Orchard, Satiro De Oliveira, Adrian J Thrasher, Myriam Armant, Colleen Dansereau, Troy C Lund, Weston P Miller, Gerald V Raymond, Raman Sankar, Ami J Shah, Caroline Sevin, H Bobby Gaspar, Paul Gissen, Hernan Amartino, Drago Bratkovic, Nicholas J C Smith, Asif M Paker, Esther Shamir, Tara O'Meara, David Davidson, Patrick Aubourg, David A Williams

Abstract

Background In X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by demyelination and neurodegeneration. Disease progression, which leads to loss of neurologic function and death, can be halted only with allogeneic hematopoietic stem-cell transplantation. Methods We enrolled boys with cerebral adrenoleukodystrophy in a single-group, open-label, phase 2-3 safety and efficacy study. Patients were required to have early-stage disease and gadolinium enhancement on magnetic resonance imaging (MRI) at screening. The investigational therapy involved infusion of autologous CD34+ cells transduced with the elivaldogene tavalentivec (Lenti-D) lentiviral vector. In this interim analysis, patients were assessed for the occurrence of graft-versus-host disease, death, and major functional disabilities, as well as changes in neurologic function and in the extent of lesions on MRI. The primary end point was being alive and having no major functional disability at 24 months after infusion. Results A total of 17 boys received Lenti-D gene therapy. At the time of the interim analysis, the median follow-up was 29.4 months (range, 21.6 to 42.0). All the patients had gene-marked cells after engraftment, with no evidence of preferential integration near known oncogenes or clonal outgrowth. Measurable ALD protein was observed in all the patients. No treatment-related death or graft-versus-host disease had been reported; 15 of the 17 patients (88%) were alive and free of major functional disability, with minimal clinical symptoms. One patient, who had had rapid neurologic deterioration, had died from disease progression. Another patient, who had had evidence of disease progression on MRI, had withdrawn from the study to undergo allogeneic stem-cell transplantation and later died from transplantation-related complications. Conclusions Early results of this study suggest that Lenti-D gene therapy may be a safe and effective alternative to allogeneic stem-cell transplantation in boys with early-stage cerebral adrenoleukodystrophy. Additional follow-up is needed to fully assess the duration of response and long-term safety. (Funded by Bluebird Bio and others; STARBEAM ClinicalTrials.gov number, NCT01896102 ; ClinicalTrialsRegister.eu number, 2011-001953-10 .).

X Demographics

X Demographics

The data shown below were collected from the profiles of 225 X users who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 499 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 499 100%

Demographic breakdown

Readers by professional status Count As %
Researcher 84 17%
Student > Ph. D. Student 68 14%
Student > Bachelor 63 13%
Other 32 6%
Student > Master 31 6%
Other 94 19%
Unknown 127 25%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 113 23%
Medicine and Dentistry 100 20%
Agricultural and Biological Sciences 32 6%
Neuroscience 30 6%
Immunology and Microbiology 17 3%
Other 57 11%
Unknown 150 30%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 1267. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 11 July 2023.
All research outputs
#10,658
of 25,498,750 outputs
Outputs from New England Journal of Medicine
#502
of 32,538 outputs
Outputs of similar age
#155
of 331,491 outputs
Outputs of similar age from New England Journal of Medicine
#9
of 280 outputs
Altmetric has tracked 25,498,750 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 99th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 32,538 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 122.4. This one has done particularly well, scoring higher than 98% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 331,491 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 99% of its contemporaries.
We're also able to compare this research output to 280 others from the same source and published within six weeks on either side of this one. This one has done particularly well, scoring higher than 97% of its contemporaries.