Evidence map›Paper›PMID 26260077›Full record

ArticleMolecular genetics and metabolism

Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.

Patricia I Dickson, Ilkka Kaitila, Paul Harmatz, Anton Mlikotic, Agnes H Chen, Alla Victoroff, Merry B Passage, Jacqueline Madden, Steven Q Le, David E Naylor and 1 more

Open access · greenAbstract readClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed, 1 pooled it
3.2field-weighted citation impact, top 8% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.

  1. Pooled it
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  5. Review
  6. Article
  7. Mucopolysaccharidosis Type I.Diagnostics (Basel, Switzerland) · 2020
    Review
  8. Article
  9. Review
  10. Review
  11. Article
  12. Review
  13. Review
  14. Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 5 institutions in 2 countries.

Patricia I DicksonDepartment of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States. Electronic address: pdickson@ucla.edu.
Ilkka KaitilaMedical Genetics, University of Helsinki, Helsinki University Hospital, Helsinki, Finland.
Paul HarmatzUCSF Benioff Children's Hospital Oakland, Oakland, CA, United States.
Anton MlikoticDepartment of Radiology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States.
Agnes H ChenDepartment of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States; Department of Neurology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States.
Alla VictoroffDepartment of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States.
Merry B PassageDepartment of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States.
Jacqueline MaddenUCSF Benioff Children's Hospital Oakland, Oakland, CA, United States.
Steven Q LeDepartment of Pediatrics, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States.
David E NaylorDepartment of Neurology, Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, CA, United States.
Mucopolysaccharidosis I Intrathecal Research Collaborative
The Lundquist Institute · USHarbor–UCLA Medical Center · USUCSF Benioff Children's Hospital · USUCLA Medical Center · USUniversity of Helsinki · FI

Funding

Engaging University of California Stakeholders for Biorespository ResearchUL1TR000004 · NCATS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI GRANDIS, JENNIFER RUBIN · 2012 to 2015
$78.0M
ZONA-FREE HAMSTER OVA ASSAY AS AN INDICATOR OF MALE FERTILITYM01RR000425 · NCRR · LUNDQUIST INSTITUTE FOR BIOMEDICAL INNOVATION AT HARBOR-UCLA MEDICAL CENTER · PI ANDERSON, GAIL V · 1985 to 2011
$74.2M
UCLA Clinical and Translational Science InstituteUL1TR000124 · NCATS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI DUBINETT, STEVEN M. · 2012 to 2015
$57.0M
Pilot/Phase I Study of Intrathecal Laronidase for Spinal Cord Compression in MucoR01FD003450 · FDA · LUNDQUIST INSTITUTE FOR BIOMEDICAL INNOVATION AT HARBOR-UCLA MEDICAL CENTER · PI DICKSON, PATRICIA I · 2008 to 2009
$454k
FDA HHS FD003450FDA HHS R01 FD003450NCATS NIH HHS UL1 TR000004NCATS NIH HHS UL1 TR000124NCATS NIH HHS UL1TR000124NCRR NIH HHS M01 RR000425NCRR NIH HHS UL1 RR0241315
6 · The paper itself

Abstract

Enzyme replacement therapy with laronidase (recombinant human alpha-l-iduronidase) is successfully used to treat patients with mucopolysaccharidosis type I (MPS I). However, the intravenously-administered enzyme is not expected to treat or prevent neurological deterioration. As MPS I patients suffer from spinal cord compression due in part to thickened spinal meninges, we undertook a phase I clinical trial of lumbar intrathecal laronidase in MPS I subjects age 8 years and older with symptomatic (primarily cervical) spinal cord compression. The study faced significant challenges, including a heterogeneous patient population, difficulty recruiting subjects despite an international collaborative effort, and an inability to include a placebo-controlled design due to ethical concerns. Nine serious adverse events occurred in the subjects. All subjects reported improvement in symptomatology and showed improved neurological examinations, but objective outcome measures did not demonstrate change. Despite limitations, we demonstrated the safety of this approach to treating neurological disease due to MPS I.

Indexed as

AdolescentAdultCervix UteriChildConstriction, PathologicFemaleHumansIduronidaseMaleMucopolysaccharidosis IRecombinant ProteinsSpinal CanalYoung AdultIduronidaseRecombinant ProteinsAlpha-l-iduronidaseEnzyme replacement therapyHurlerIntrathecalLysosomal storage diseaseScheie

Identifiers

PMID26260077
PMCPMC4572891
OpenAlexW1684788719

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.