Evidence map›Paper›PMID 24797180›Full record

Trial reportBone marrow transplantation2014

Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease.

M Bhatia, Z Jin, C Baker, M B Geyer, K Radhakrishnan, E Morris, P Satwani, D George, J Garvin, G Del Toro and 8 more

Abstract readClinical Trial
PubMed Publisher
In one paragraph

Trial report in Bone marrow transplantation, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
45citing papers in PubMed, 1 pooled it
6.6field-weighted citation impact, top 2% 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

45 citing papers in PubMed, 1 synthesis or guideline pooled it, 102 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Trial
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  5. Health-related quality of life after allogeneic hematopoietic stem cell transplantation for sickle cell disease.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2015
    Trial
  6. Review
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  9. Article
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  11. Article
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  14. The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2024
    Review
  15. Article
  16. Article
  17. Article
  18. 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

18 authors at 6 institutions in 1 country.

M BhatiaDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
Z Jin1] Department of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA [2] Department of Biostatistics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
C BakerDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
M B GeyerDepartment of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
K RadhakrishnanDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
E MorrisDepartment of Pediatrics, New York Medical College, Valhalla, NY, USA.
P SatwaniDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
D GeorgeDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
J GarvinDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
G Del ToroDepartment of Pediatrics, Wyckoff Heights Medical Center, New York, NY, USA.
W ZuckermanDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
M T LeeDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
M LicursiDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
R HawksDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
E SmilowDepartment of Pediatrics, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
L A Baxter-LoweDepartment of Surgery, University of California San Francisco, San Francisco, CA, USA.
J SchwartzDepartment of Pathology, Columbia University, NewYork-Presbyterian Morgan Stanley Children's Hospital, New York, NY, USA.
M S Cairo1] Department of Pediatrics, New York Medical College, Valhalla, NY, USA [2] Department of Medicine, New York Medical College, Valhalla, NY, USA [3] Department of Pathology, New York Medical College, Valhalla, NY, USA [4] Department of Microbiology and Immunology, New York Medical College, Valhalla, NY, USA [5] Department of Cell Biology and Anatomy, New York Medical College, Valhalla, NY, USA.
Columbia University · USMorgan Stanley Children's Hospital · USNew York Medical College · USHarvard University · USUniversity of California, San Francisco · USWyckoff Heights Medical Center · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BU and CY (BU/CY; 200 mg/kg) before HLA-matched sibling allo-SCT in children with sickle cell disease (SCD) is associated with ~85% EFS but is limited by the acute and late effects of BU/CY myeloablative conditioning. Alternatives include reduced toxicity but more immunosuppressive conditioning. We investigated in a prospective single institutional study, the safety and efficacy of a reduced-toxicity conditioning (RTC) regimen of BU 12.8-16 mg/kg, fludarabine 180 mg/m(2), alemtuzumab 54 mg/m(2) (BFA) before HLA-matched sibling donor transplantation in pediatric recipients with symptomatic SCD. Eighteen patients, median age 8.9 years (2.3-20.2), M/F 15/3, 15 sibling BM and 3 sibling cord blood (CB) were transplanted. Mean whole blood and erythroid donor chimerism was 91% and 88%, at days +100 and +365, respectively. Probability of grade II-IV acute GVHD was 17%. Two-year EFS and OS were both 100%. Neurological, pulmonary and cardiovascular function were stable or improved at 2 years. BFA RTC and HLA-matched sibling BM and CB allo-SCT in pediatric recipients result in excellent EFS, long-term donor chimerism, low incidence of GVHD and stable/improved organ function.

Indexed as

AdolescentAdultAlemtuzumabAnemia, Sickle CellAntibodies, Monoclonal, HumanizedAntineoplastic Combined Chemotherapy ProtocolsBusulfanChildChild, PreschoolFemaleHematopoietic Stem Cell TransplantationHumansMalePilot ProjectsProspective StudiesSiblingsAlemtuzumabAntibodies, Monoclonal, HumanizedBusulfanfludarabineVidarabine

Identifiers

PMID24797180
OpenAlexW1996057204

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.