Evidence map›Paper›PMID 31701524›Full record

Trial reportClinical pharmacology and therapeutics2020

Proposed Therapeutic Range of Treosulfan in Reduced Toxicity Pediatric Allogeneic Hematopoietic Stem Cell Transplant Conditioning: Results From a Prospective Trial.

Robert Chiesa, Joseph F Standing, Robert Winter, Zohreh Nademi, Jan Chu, Danielle Pinner, Frank Kloprogge, Susan McLellen, Persis J Amrolia, Kanchan Rao and 15 more

Open access · hybridAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Clinical pharmacology and therapeutics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed, 1 pooled it
2.3field-weighted citation impact, top 11% 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

15 citing papers in PubMed, 1 synthesis or guideline pooled it, 31 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Review
  5. Article
  6. Review
  7. Article
  8. Article
  9. Article
  10. Review
  11. Review
  12. Review
  13. Treosulfan-based conditioning for inborn errors of immunity.Therapeutic advances in hematology · 2021
    Review
  14. Allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency.Hematology. American Society of Hematology. Education Program · 2020
    Review
  15. 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

25 authors at 6 institutions in 2 countries.

Robert ChiesaBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Joseph F StandingPharmacy Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Robert WinterChemical Pathology Department, Great Ormond Street Hospital for Children,, NHS Foundation Trust, London, UK.
Zohreh NademiBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Jan ChuBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Danielle PinnerBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Frank KloproggeInstitute for Global Health, University College London, London, UK.
Susan McLellenClinical Biochemistry, Integrated Laboratory Medicine Directorate, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Persis J AmroliaBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Kanchan RaoBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Giovanna LucchiniBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Juliana SilvaBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Oana CiocarlieBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Arina LazarevaBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Andrew R GenneryBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Bilyana DonchevaPharmacy Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Andrew J CantBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Sophie HambletonBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Terence FloodBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Elizabeth RogersonBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Kirsty DevineBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Helen PruntyChemical Pathology Department, Great Ormond Street Hospital for Children,, NHS Foundation Trust, London, UK.
Simon HealesChemical Pathology Department, Great Ormond Street Hospital for Children,, NHS Foundation Trust, London, UK.
Paul VeysBone Marrow Transplantation Department, Great Ormond Street Hospital for Children, NHS Foundation Trust, London, UK.
Mary SlatterBone Marrow Transplantation Department, Great North Children's Hospital, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Great Ormond Street Hospital for Children NHS Foundation Trust · GBNewcastle upon Tyne Hospitals NHS Foundation Trust · GBGreat North Children's Hospital · GBGreat Ormond Street Hospital · GBInstitute of Infection and Immunity · CAUniversity College London · GB

Funding

Medical Research Council MR/M008665/1Medical Research Council MR/P014534/1
6 · The paper itself

Abstract

Treosulfan is given off-label in pediatric allogeneic hematopoietic stem cell transplant. This study investigated treosulfan's pharmacokinetics (PKs), efficacy, and safety in a prospective trial. Pediatric patients (n = 87) receiving treosulfan-fludarabine conditioning were followed for at least 1 year posttransplant. PKs were described with a two-compartment model. During follow-up, 11 of 87 patients died and 12 of 87 patients had low engraftment (≤ 20% myeloid chimerism). For each increase in treosulfan area under the curve from zero to infinity (AUC

Indexed as

Hematopoietic Stem Cell TransplantationTransplantation ConditioningAdolescentBusulfanChildChild, PreschoolDose-Response Relationship, DrugDrug Administration ScheduleDrug MonitoringEnglandFemaleHumansInfantInfant, NewbornInfusions, IntravenousMaleBusulfanfludarabineMyeloablative AgoniststreosulfanVidarabine

Identifiers

PMID31701524
PMCPMC7484914
OpenAlexW2986767939

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.