Evidence map›Paper›PMID 30885997›Full record

ArticleBlood advances2019

Stem cell transplantation for osteopetrosis in patients beyond the age of 5 years.

Polina Stepensky, Sigal Grisariu, Batia Avni, Irina Zaidman, Bella Shadur, Orly Elpeleg, Mehtap Sirin, Manfred Hoenig, Catharina Schuetz, Ingrid Furlan and 5 more

Open access · goldAbstract read
In one paragraph

Article in Blood advances, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.

0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed
2.6field-weighted citation impact, top 10% 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

20 citing papers in PubMed, 41 citations in OpenAlex.

  1. Review
  2. Correction of osteopetrosis in the neonateFrontiers in endocrinology · 2024
    Article
  3. Article
  4. Metabolic bone disorders and the promise of marine osteoactive compounds.Cellular and molecular life sciences : CMLS · 2023
    Review
  5. Article
  6. Origin of Osteoclasts: Osteoclast Precursor Cells.Journal of bone metabolism · 2023
    Article
  7. Review
  8. Bone marrow transplantation as a therapy for autosomal dominant osteopetrosis type 2 in mice.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2022
    Article
  9. Review
  10. Article
  11. Article
  12. Article
  13. Review
  14. Article
  15. Review
  16. SNX10 gene mutation in infantile malignant osteopetrosis: A case report and literature review.Zhong nan da xue xue bao. Yi xue ban = Journal of Central South University. Medical sciences · 2021
    Review
  17. Article
  18. Article
  19. Article
  20. Article
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

15 authors at 4 institutions in 4 countries.

Polina StepenskyDepartment of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel.
Sigal GrisariuDepartment of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel.
Batia AvniDepartment of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel.
Irina ZaidmanDepartment of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel.
Bella ShadurDepartment of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel.
Orly ElpelegMonique and Jacques Roboh Department of Genetic Research, Hadassah-Hebrew University Medical Center, Ein Kerem, Jerusalem, Israel; and.
Mehtap SirinDepartment of Pediatrics and Adolescent Medicine.
Manfred HoenigDepartment of Pediatrics and Adolescent Medicine.
Catharina SchuetzDepartment of Pediatrics and Adolescent Medicine.
Ingrid FurlanDepartment of Pediatrics and Adolescent Medicine.
Meinrad BeerDepartment of Radiology, and.
Stephanie von HarsdorfDepartment of Internal Medicine III, University Medical Center Ulm, Ulm, Germany.
Donald BunjesDepartment of Internal Medicine III, University Medical Center Ulm, Ulm, Germany.
Klaus-Michael DebatinDepartment of Pediatrics and Adolescent Medicine.
Ansgar S SchulzDepartment of Pediatrics and Adolescent Medicine.ORCID 0000-0002-7798-7996
Office of Adolescent Health · USHadassah Medical Center · ILUniversität Ulm · DEGarvan Institute of Medical Research · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Osteopetrosis (OP) is a rare disease caused by defective osteoclast differentiation or function. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment available in the infantile "malignant" form of OP. Improved clinical and genetic diagnosis of OP has seen the emergence of a cohort of patients with less severe and heterogeneous clinical presentations. This intermediate form of OP does not call for urgent intervention, but patients accumulate debilitating skeletal complications over years and decades, which are severe enough to require curative treatment and may also require intermittent transfusion of blood products. Here we present data from 7 patients with intermediate OP caused by mutations in

Indexed as

AdolescentAdultChildFollow-Up StudiesHematopoietic Stem Cell TransplantationHumansMutationOsteopetrosisQuality of LifeTissue DonorsTransplantation ConditioningYoung Adult

Identifiers

PMID30885997
PMCPMC6436016
OpenAlexW2922457648

What OpenQuestion holds

Textmetadata
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.