Evidence map›Paper›PMID 35309862›Full record

ArticleJBMR plus2022

Open-Label Pilot Study of Interferon Gamma-1b in Patients With Non-Infantile Osteopetrosis.

Andrew Nguyen, Weston P Miller, Ashish Gupta, Troy C Lund, Daniel Schiferl, Lok Sze Kelvin Lam, Zorayr Arzumanyan, Paul J Orchard, Lynda E Polgreen

Open access · goldAbstract read
In one paragraph

Article in JBMR plus, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed
0.8field-weighted citation impact, top 32% 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

9 citing papers in PubMed, 11 citations in OpenAlex.

  1. 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

9 authors at 4 institutions in 2 countries.

Andrew NguyenLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance CA USA.ORCID https://orcid.org/0000-0001-9178-2500
Weston P MillerAudentes Therapeutics, An Astellas Company San Francisco, (formerly at University of Minnesota) San Francisco CA USA.
Ashish GuptaUniversity of Minnesota Minneapolis MN USA.
Troy C LundUniversity of Minnesota Minneapolis MN USA.
Daniel SchiferlBone Diagnostics Spring Branch TX USA.
Lok Sze Kelvin LamLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance CA USA.
Zorayr ArzumanyanLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance CA USA.
Paul J OrchardUniversity of Minnesota Minneapolis MN USA.
Lynda E PolgreenLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center Torrance CA USA.
UCLA Medical Center · USUniversity of Minnesota · USAstellas Pharma (Japan) · JPEKF Diagnostics (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The only treatment currently available for patients with severe infantile osteopetrosis is hematopoietic cell transplantation (HCT). HCT-related toxicity and mortality risks typically preclude its use in non-infantile patients, and other therapies are needed for these patients who have significant disease-related morbidity. Interferon gamma-1b is currently approved by the U.S. Food and Drug Administration (FDA) for treatment of severe infantile osteopetrosis (autosomal recessive osteopetrosis [ARO]). However, little is known about the effects of interferon gamma-1b in non-infantile osteopetrosis. Thus, this pilot study aimed at testing the safety and tolerability of interferon gamma-1b in patients with non-infantile osteopetrosis and assessing the clinical effects. We performed a 12-month, open-label, multi-center pilot study involving patients >1 year-old diagnosed radiographically with osteopetrosis. Patients were initiated on interferon gamma-1b subcutaneously 15 μg/m

Indexed as

ANALYSIS/QUANTITATION OF BONEBONE QCT/μCTDISEASES AND DISORDERS OF/RELATED TO BONEOSTEOPETROSISTHERAPEUTICS

Identifiers

PMID35309862
PMCPMC8914146
OpenAlexW4206831401

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.