Evidence map›Paper›PMID 22829629›Full record

Trial reportBlood2012

Transplanted bone marrow mononuclear cells and MSCs impart clinical benefit to children with osteogenesis imperfecta through different mechanisms.

Satoru Otsuru, Patricia L Gordon, Kengo Shimono, Reena Jethva, Roberta Marino, Charlotte L Phillips, Ted J Hofmann, Elena Veronesi, Massimo Dominici, Masahiro Iwamoto and 1 more

Registry-linked trialOpen access · bronzeAbstract readClinical Trial
In one paragraph

Trial report in Blood, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00187018 (Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta), which is not on this map. Cited by 76 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
76citing papers in PubMed, 3 pooled it
12.6field-weighted citation impact, top 1% 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.

NCT00187018 nacompletednot on this map

Marrow Mesenchymal Cell Therapy for Osteogenesis Imperfecta: A Pilot Study

TypeinterventionalSponsorSt. Jude Children's Research HospitalRan2004 to 2007Enrolled9ConditionsOsteogenesis ImperfectaArmsBone marrow transplant
3 · Its place in the literature

Who cites it

76 citing papers in PubMed, 3 syntheses or guidelines pooled it, 143 citations in OpenAlex.

  1. Pooled it
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  19. Curative Cell and Gene Therapy for Osteogenesis Imperfecta.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research · 2022
    Review
  20. Review

16 more citing papers are in PubMed but not listed here.

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.

Satoru OtsuruDivision of Oncology/Blood and Marrow Transplantation, The Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA 19104, USA.
Patricia L Gordon
Kengo Shimono
Reena Jethva
Roberta Marino
Charlotte L Phillips
Ted J Hofmann
Elena Veronesi
Massimo Dominici
Masahiro Iwamoto
Edwin M Horwitz
Children's Hospital of Philadelphia · USSt. Jude Children's Research Hospital · USUniversity of Modena and Reggio Emilia · ITUniversity of Missouri · USUniversity of Pennsylvania · US

Funding

Viral Vector Technology (VVTSR)P30CA021765 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI CHARLES ROBERTS · 1985 to 2026
$166.9M
Biology/Transplantation of Multipotent Marrow Stem CellsR01HL077643 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI HORWITZ, EDWIN M · 2005 to 2009
$1.9M
NCI NIH HHS P30 CA021765NCI NIH HHS P30 CA21765NHLBI NIH HHS R01 HL077643
6 · The paper itself

Abstract

Transplantation of whole bone marrow (BMT) as well as ex vivo-expanded mesenchymal stromal cells (MSCs) leads to striking clinical benefits in children with osteogenesis imperfecta (OI); however, the underlying mechanism of these cell therapies has not been elucidated. Here, we show that non-(plastic)-adherent bone marrow cells (NABMCs) are more potent osteoprogenitors than MSCs in mice. Translating these findings to the clinic, a T cell-depleted marrow mononuclear cell boost (> 99.99% NABMC) given to children with OI who had previously undergone BMT resulted in marked growth acceleration in a subset of patients, unambiguously indicating the therapeutic potential of bone marrow cells for these patients. Then, in a murine model of OI, we demonstrated that as the donor NABMCs differentiate to osteoblasts, they contribute normal collagen to the bone matrix. In contrast, MSCs do not substantially engraft in bone, but secrete a soluble mediator that indirectly stimulates growth, data which provide the underlying mechanism of our prior clinical trial of MSC therapy for children with OI. Collectively, our data indicate that both NABMCs and MSCs constitute effective cell therapy for OI, but exert their clinical impact by different, complementary mechanisms. The study is registered at www.clinicaltrials.gov as NCT00187018.

Indexed as

AnimalsBody HeightBody WeightBone Marrow TransplantationBone MatrixCells, CulturedChildCollagenFemaleFlow CytometryGene ExpressionHumansLeukocytes, MononuclearLumbar VertebraeMaleMesenchymal Stem CellsCollagen

Identifiers

PMID22829629
PMCPMC3433095
OpenAlexW1978035979

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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.