Evidence map›Paper›PMID 29767845›Full record

Trial reportBritish journal of haematology2018

Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome.

Nancy A Kernan, Stephan Grupp, Angela R Smith, Sally Arai, Brandon Triplett, Joseph H Antin, Leslie Lehmann, Tsiporah Shore, Vincent T Ho, Nancy Bunin and 6 more

2 registry-linked trialsOpen access · hybridAbstract readClinical Trial, Phase IIIMulticenter Study
In one paragraph

Trial report in British journal of haematology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 80 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
80citing papers in PubMed, 1 pooled it
9.1field-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.

NCT00628498 phase3completednot on this map

Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study

TypeinterventionalSponsorJazz PharmaceuticalsRan2007 to 2016Enrolled1,206ConditionsHepatic Veno-Occlusive DiseaseArmsDefibrotide
NCT03963999 phase4withdrawnnot on this mapstarted 2020, after this paper: background citation

Validating Ultrasound Biomarkers for Hepatic Sinusoidal Obstruction Syndrome in Pediatric Hematopoietic Cell Transplant Patients

TypeinterventionalSponsorChildren's Mercy Hospital Kansas CityRan2020 to 2025Enrolled0ConditionsSinusoidal Obstruction Syndrome, Veno Occlusive Disease, Hepatic, Bone Marrow Transplant Complications, Stem Cell Transplant ComplicationsArmsUltrasound Elastography, Contrast Enhanced Ultrasound (CEUS)
3 · Its place in the literature

Who cites it

80 citing papers in PubMed, 1 synthesis or guideline pooled it, 121 citations in OpenAlex.

  1. A Meta-Analysis Evaluating the Incidence of Bleeding Events With Intravenous Defibrotide Treatment Outside the Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome Setting.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis
    Pooled it
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20 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

16 authors at 9 institutions in 2 countries.

Nancy A KernanMemorial Sloan-Kettering Cancer Center, New York, NY, USA.
Stephan GruppThe Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Angela R SmithUniversity of Minnesota, Minneapolis, MN, USA.
Sally AraiStanford University Medical Center, Stanford, CA, USA.
Brandon TriplettSt Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-8220-9980
Joseph H AntinDana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
Leslie LehmannDana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
Tsiporah ShoreWeill Cornell Medical College, New York, NY, USA.
Vincent T HoDana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
Nancy BuninThe Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Massimo IacobelliTechitra Srl, Milan, Italy.
Wei LiangJazz Pharmaceuticals, Palo Alto, CA, USA.
Robin HumeJazz Pharmaceuticals, Palo Alto, CA, USA.
William TappeJazz Pharmaceuticals, Palo Alto, CA, USA.
Robert SoifferDana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
Paul RichardsonDana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.ORCID 0000-0002-7426-8865
Harvard University · USJazz Pharmaceuticals (United States) · USChildren's Hospital of Philadelphia · USCornell University · USMemorial Sloan Kettering Cancer Center · USStanford Medicine · USSt. Jude Children's Research Hospital · USTechint (Italy) · ITUniversity of Minnesota · US

Funding

X-RAY CRYSTALLOGRAPHYP30CA008748 · NCI · SLOAN-KETTERING INSTITUTE FOR CANCER RES · PI Nicholas D Socci · 1985 to 2026
$347.4M
NCI NIH HHS P30 CA008748
6 · The paper itself

Abstract

Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic stem cell transplant (HSCT) conditioning and chemotherapy. Defibrotide is approved for treatment of hepatic VOD/SOS with pulmonary or renal dysfunction [i.e., multi-organ dysfunction (MOD)] after HSCT in the United States and severe VOD/SOS after HSCT in patients aged older than 1 month in the European Union. Defibrotide was available as an investigational drug by an expanded-access treatment programme (T-IND; NCT00628498). In the completed T-IND, the Kaplan-Meier estimated Day +100 survival for 1000 patients with documented defibrotide treatment after HSCT was 58·9% [95% confidence interval (CI), 55·7-61·9%]. Day +100 survival was also analysed by age and MOD status, and post hoc analyses were performed to determine Day +100 survival by transplant type, timing of VOD/SOS onset (≤21 or >21 days) and timing of defibrotide treatment initiation after VOD/SOS diagnosis. Day +100 survival in paediatric patients was 67·9% (95% CI, 63·8-71·6%) and 47·1% (95% CI, 42·3-51·8%) in adults. All patient subgroups without MOD had higher Day +100 survival than those with MOD; earlier defibrotide initiation was also associated with higher Day +100 survival. The safety profile of defibrotide in the completed T-IND study was similar to previous reports.

Indexed as

Hematopoietic Stem Cell TransplantationHepatic Veno-Occlusive DiseaseMultiple Organ FailureAdolescentAdultAgedAge FactorsAllograftsChildChild, PreschoolDisease-Free SurvivalFemaleHumansInfantInfant, NewbornMaledefibrotidePolydeoxyribonucleotidesdefibrotidehaematopoietic stem cell transplantmulti-organ dysfunctionsinusoidal obstruction syndromeveno-occlusive disease

Identifiers

PMID29767845
PMCPMC6032999
OpenAlexW2803440649

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.